UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549

Form 10-Q

QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2021

OR

TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from           to

Commission file number: 001-36829

Rocket Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)

Delaware
 
04-3475813
(State or other jurisdiction of incorporation or organization)
 
(I.R.S. Employer Identification No.)

9 Cedarbrook Drive
Cranbury, NJ 08512
(Address of principal executive office) (Zip Code)

Registrant’s telephone number, including area code:
(646) 440-9100

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes  No

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes  No

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. See the definitions of “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer 
Accelerated filer
   
Non-accelerated filer
Smaller reporting company
Emerging growth company
 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes No

Securities registered pursuant to Section 12(b) of the Act:
Title of each class
Trading Symbol(s)
Name of each exchange on which registered
     
Common Stock, $0.01 par value
RCKT
Nasdaq Global Market

As of May 3, 2021, there were 63,396,954 shares of common stock, $0.01 par value per share, outstanding.




Table of Contents


Page
PART I - FINANCIAL INFORMATION
 
Item 1.
Financial Statements
4
 
Consolidated Balance Sheets at March 31, 2021 (unaudited) and December 31, 2020
4
     
 
Consolidated Statements of Operations for the three months ended March 31, 2021 and 2020 (unaudited)
5
     
 
Consolidated Statements of Comprehensive Loss for the three months ended March 31, 2021 and 2020 (unaudited)
6
     
 
Consolidated Statements of Stockholders’ Equity for the three months ended March 31, 2021 and 2020 (unaudited)
7
     
 
Consolidated Statements of Cash Flows for the three months ended March 31, 2021 and 2020 (unaudited)
8
     
 
Notes to Consolidated Financial Statements (unaudited)
9
Item 2.
Management’s Discussion and Analysis of Financial Condition and Results of Operations
19
Item 3.
Quantitative and Qualitative Disclosures About Market Risk
28
Item 4.
Controls and Procedures
28
PART II - OTHER INFORMATION
 
Item 1.
Legal Proceedings
28
Item 1A.
Risk Factors
29
Item 2.
Unregistered Sales of Equity Securities and Use of Proceeds
29
Item 3.
Defaults Upon Senior Securities
29
Item 4.
Mine Safety Disclosures
29
Item 5.
Other Information
29
Item 6.
Exhibits
30
Signatures
31

2

Table of Contents
Forward-looking statements

This Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 (Form 10-Q) contains forward-looking statements that involve risks and uncertainties, as well as assumptions that, if they never materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q are forward-looking statements. In some cases, you can identify forward-looking statements by words such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will,” “would,” or the negative of these words or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the U.S. Food and Drug Administration (“FDA”);
the timing of and our ability to submit regulatory filings with the FDA and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates;
our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting;
whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval or further development of any of our product candidates;
our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product candidates, within currently anticipated timeframes, or at all;
our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;
our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;
our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances, or joint ventures;
our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire;
unanticipated delays due to manufacturing difficulties, including the development of our direct manufacturing capabilities for our AAV programs, and any supply constraints or changes in the regulatory environment; our ability to successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws;
uncertainties associated with obtaining and enforcing patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims;
anticipated trends and challenges in our business and the markets in which we operate;
natural and manmade disasters, including pandemics such as COVID-19, and other force majeures, which could impact our operations, and those of our partners and other participants in the health care industry, and which could adversely impact our clinical studies, preclinical research activities, and drug supply;
our estimates regarding our capital requirements; and
our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities.

We caution you that the foregoing list may not contain all of the forward-looking statements made in this Quarterly Report on Form 10-Q.

Any forward-looking statements in this Quarterly Report on Form 10-Q reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. We have included important factors in the cautionary statements included in this Quarterly Report on Form 10-Q, particularly in the “Risk Factors” section incorporated by reference from our Annual Report for the year ended December 31, 2020, on Form 10-K, that could cause actual results or events to differ materially from the forward-looking statements that we make. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make or enter into.

You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results, performance or achievements may be materially different from what we expect. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.

This Quarterly Report on Form 10-Q also contains estimates, projections and other information concerning our industry, our business and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources.

3

Table of Contents

PART I — FINANCIAL INFORMATION

Item 1.
Financial Statements
Rocket Pharmaceuticals, Inc.
Consolidated Balance Sheets
($ in thousands, except shares and per share amounts)

   
March 31,
   
December 31,
 
 
2021
   
2020
 
Assets
           
Current assets:
           
Cash and cash equivalents
 
$
252,540
   
$
297,098
 
Investments
   
213,813
     
185,621
 
Prepaid expenses and other assets
   
5,469
     
4,626
 
Total current assets
   
471,822
     
487,345
 
Property and equipment, net
   
19,833
     
19,206
 
Goodwill
   
30,815
     
30,815
 
Restricted cash
   
1,461
     
1,568
 
Deposits
   
455
     
455
 
Operating lease right-of-use assets
   
1,787
     
914
 
Finance lease right-of-use asset
   
50,311
     
50,521
 
Total assets
 
$
576,484
   
$
590,824
 
Liabilities and stockholders' equity
               
Current liabilities:
               
Accounts payable and accrued expenses
 
$
33,168
   
$
25,472
 
Convertible notes, net of unamortized discount, current
   
4,986
     
4,875
 
Operating lease liabilities, current
   
683
     
626
 
Finance lease liability, current
   
1,655
     
1,644
 
Total current liabilities
   
40,492
     
32,617
 
Convertible notes, net of unamortized discount, non-current
   
35,544
     
35,066
 
Operating lease liabilities, non-current
   
1,299
     
498
 
Finance lease liabilities, non-current
   
19,028
     
18,988
 
Other liabilities
   
122
     
136
 
Total liabilities
   
96,485
     
87,305
 
Commitments and contingencies (Note 11)
   
     
 
                 
Stockholders' equity:
               
Preferred stock, $0.01 par value, authorized 5,000,000 shares:
               
Series A convertible preferred stock; 300,000 shares designated as Series A; 0 shares issued and outstanding
   
-
     
-
 
Series B convertible preferred stock; 300,000 shares designated as Series B; 0 shares issued and outstanding
   
-
     
-
 
Common stock, $0.01 par value, 120,000,000 shares authorized; 61,987,799 and 60,996,367 shares issued and outstanding at March 31, 2021 and December 31, 2020, respectively
   
619
     
610
 
Additional paid-in capital
   
842,477
     
825,794
 
Accumulated other comprehensive loss
   
(75
)
   
(42
)
Accumulated deficit
   
(363,022
)
   
(322,843
)
Total stockholders' equity
   
479,999
     
503,519
 
Total liabilities and stockholders' equity
 
$
576,484
   
$
590,824
 

The accompanying notes are an integral part of these consolidated financial statements.

4

Table of Contents

Rocket Pharmaceuticals, Inc.
Consolidated Statements of Operations
($ in thousands, except shares and per share amounts)
(unaudited)

 
Three Months Ended March 31,
 
   
2021
   
2020
 
 
           
Revenue
 
$
-
   
$
-
 
 
               
Operating expenses:
               
Research and development
   
28,542
     
16,957
 
General and administrative
   
10,680
     
7,163
 
Total operating expenses
   
39,222
     
24,120
 
Loss from operations
   
(39,222
)
   
(24,120
)
Research and development incentives
   
500
     
-
 
Interest expense
   
(1,729
)
   
(1,573
)
Interest and other income net
   
911
     
967
 
(Amortization of premium) accretion of discount on investments - net
   
(639
)
   
62
 
Net loss
 
$
(40,179
)
 
$
(24,664
)
Net loss per share attributable to common stockholders - basic and diluted
 
$
(0.65
)
 
$
(0.45
)
Weighted-average common shares outstanding - basic and diluted
   
61,574,405
     
54,883,120
 

The accompanying notes are an integral part of these consolidated financial statements.

5

Table of Contents

Rocket Pharmaceuticals, Inc.
Consolidated Statements of  Comprehensive Loss
(in thousands)
(unaudited)

 
Three Months Ended March 31,
 
   
2021
   
2020
 
 
           
Net loss
 
$
(40,179
)
 
$
(24,664
)
Other comprehensive loss
               
Net unrealized loss on investments
   
(33
)
   
(95
)
Total comprehensive loss
 
$
(40,212
)
 
$
(24,759
)

The accompanying notes are an integral part of these consolidated financial statements.

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Table of Contents

Rocket Pharmaceuticals, Inc.
Consolidated Statements of Stockholders’ Equity
For the Three Months Ended March 31, 2021 and 2020
(in thousands except share amounts)
(unaudited)

 
Common Stock
   
Treasury
   
Additional
Paid-In
   
Accumulated
Other
Comprehensive
   
Accumulated
   
Total
Stockholders'
 
   
Shares
   
Amount
   
Stock
   
Capital
   
Loss
   
Deficit
   
Equity
 
                                           
Balance at December 31, 2020
   
60,996,367
   
$
610
   
$
-
   
$
825,794
   
$
(42
)
 
$
(322,843
)
 
$
503,519
 
Issuance of common stock pursuant to exercise of stock options
   
991,432
     
9
     
-
     
8,783
     
-
     
-
     
8,792
 
Unrealized comprehensive loss on investments
   
-
     
-
     
-
     
-
     
(33
)
   
-
     
(33
)
Share-based compensation
   
-
     
-
     
-
     
7,900
     
-
     
-
     
7,900
 
Net loss
   
-
     
-
     
-
     
-
     
-
     
(40,179
)
   
(40,179
)
Balance at March 31, 2021
   
61,987,799
   
$
619
   
$
-
   
$
842,477
   
$
(75
)
 
$
(363,022
)
 
$
479,999
 

Balance at December 31, 2019
   
54,773,061
   
$
548
   
$
(53
)
 
$
489,925
   
$
20
   
$
(183,143
)
 
$
307,297
 
Issuance of common stock pursuant to exercise of stock options
   
386,974
     
3
     
-
     
(3
)
   
-
     
-
     
-
 
Share repurchase
   
(3,000
)
   
-
     
-
     
(72
)
   
-
     
-
     
(72
)
Sale of treasury stock
   
-
     
-
     
53
     
-
     
-
     
-
     
53
 
Issuance of treasury stock pursuant to exercise of stock options
   
-
     
-
     
(429
)
   
-
     
-
     
-
     
(429
)
Unrealized comprehensive loss on investments
   
-
     
-
     
-
     
-
     
(95
)
   
-
     
(95
)
Share-based compensation
   
-
     
-
     
-
     
3,961
     
-
     
-
     
3,961
 
Net loss
   
-
     
-
     
-
     
-
     
-
     
(24,664
)
   
(24,664
)
Balance at March 31, 2020
   
55,157,035
   
$
551
   
$
(429
)
 
$
493,811
   
$
(75
)
 
$
(207,807
)
 
$
286,051
 

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.
Consolidated Statements of  Cash Flows
(in thousands)
(unaudited)

 
Three Months Ended March 31,
 
   
2021
   
2020
 
             
Operating Activities:
           
Net loss
 
$
(40,179
)
 
$
(24,664
)
Adjustments to reconcile net loss to net cash used in operating activities:
               
Accretion of discount on convertible notes
   
589
     
844
 
Depreciation and amortization expense
   
674
     
118
 
Write down of property and equipment
   
-
     
63
 
Stock-based compensation
   
7,900
     
3,961
 
Accretion of discount (amortization of premium) on investments, net
   
620
     
(63
)
Changes in operating assets and liabilities:
               
Prepaid expenses and other assets
   
(842
)
   
(271
)
Accounts payable and accrued expenses
   
7,047
     
(1,932
)
Operating lease liabilities
   
(15
)
   
(452
)
Finance lease liability
   
(63
)
   
151
 
Other long term liabilities
   
(14
)
   
-
 
Net cash used in operating activities
   
(24,283
)
   
(22,245
)
Investing activities:
               
Purchases of investments
   
(103,815
)
   
(23,481
)
Proceeds from maturities of investments
   
74,969
     
56,805
 
Payments made to acquire right of use asset
   
-
     
(530
)
Purchases of property and equipment
   
(328
)
   
(5,207
)
Purchases of internal use software
   
-
     
(88
)
Net cash (used in) provided by investing activities
   
(29,174
)
   
27,499
 
Financing activities:
               
Issuance of common stock, pursuant to exercise of stock options
   
8,792
     
-
 
Common stock repurchase
   
-
     
(72
)
Proceeds from sale of treasury stock
   
-
     
53
 
Convertible notes refinancing costs to the lender
   
-
     
(98
)
Net cash provided by (used in) financing activities
   
8,792
     
(117
)
Net change in cash, cash equivalents and restricted cash
   
(44,665
)
   
5,137
 
Cash, cash equivalents and restricted cash at beginning of period
   
298,666
     
186,908
 
Cash, cash equivalents and restricted cash at end of period
 
$
254,001
   
$
192,045
 
                 
Supplemental disclosure of non-cash financing and investing activities:
               
Accrued purchases of property and equipment
 
$
337
   
$
2,796
 
Accrued purchases of internal use software
 
$
311
   
$
174
 
Retirement of treasury stock
 
$
-
   
$
72
 
Witholding tax payable on shares witheld in treasury stock
 
$
-
   
$
429
 
Unrealized loss on investments
 
$
(33
)
 
$
(95
)
Finance lease right of use asset and lease liability
 
$
-
   
$
20,179
 
Reclassification of construction in process to finance right of use asset
 
$
325
   
$
26,465
 
Supplemental cash flow information:
               
Cash paid for interest
 
$
1,347
   
$
1,495
 

The accompanying notes are an integral part of these consolidated financial statements.

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ROCKET PHARMACEUTICALS, INC.
Notes to Consolidated Financial Statements
($ in thousands, except share and per share data) (Unaudited)

1.
Nature of Business


Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. Rocket has four clinical-stage ex vivo lentiviral vector (“LVV”) programs. These include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I), a genetic disorder that causes the immune system to malfunction, Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia and Infantile Malignant Osteopetrosis (“IMO”), a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program are in registration-enabling studies in the United States (“U.S.”) and Europe (“EU”). In addition, in the U.S., Rocket has a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements. Additional discovery efforts on a gene therapy program for the less common FA subtypes C and G is ongoing.

2.
Risks and Liquidity


The Company has not generated any revenue and has incurred losses since inception. The Company’s operations are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, the absence of commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance-reporting capabilities.


The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies.


The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has experienced negative cash flows from operations and had an accumulated deficit of $363.0 million as of March 31, 2021. As of March 31, 2021, the Company has $466.4 million of cash, cash equivalents and investments. During the year ended December 31, 2020, the Company received net proceeds of $280.8 million from a public offering of 5,339,286 shares of common stock. Rocket expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2023.  In April 2021, the Company called for the redemption of the remaining $38.4 million principal balance of  its  6.25% 2022 Convertible Senior Notes due 2022 which were converted into common stock (See Notes 7 and 16).


In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies. 

3.
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies


Basis of Presentation


The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December 31, 2020 included in the Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 1, 2021 (“2020 Form 10-K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of March 31, 2021 and the results of its operations and its cash flows for the three months ended March 31, 2021. The financial data and other information disclosed in these consolidated notes related to the three months ended March 31, 2021 and 2020 are unaudited. The results for the three months ended March 31, 2021 are not necessarily indicative of results to be expected for the year ending December 31, 2021 and any other interim periods or any future year or period.

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Principles of Consolidation


The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with accounting principles generally accepted in the United States (“U.S. GAAP”). All intercompany accounts have been eliminated in consolidation.


Use of Estimates


The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these consolidated financial statements include but are not limited to goodwill impairment, the accrual of research and development (“R&D”) expenses, the valuation of equity transactions and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates.


Cash, Cash Equivalents and Restricted Cash


Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts.


Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s finance and operating leases (see Note 11 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s Corporate Credit Card. Cash, cash equivalents and restricted cash consist of the following:

 
 
 
March 31,
2021
   
December 31,
2020
 
 
           
Cash and cash equivalents
 
$
252,540
   
$
297,098
 
Restricted cash
   
1,461
     
1,568
 
 
 
$
254,001
   
$
298,666
 


Significant Accounting Policies


The significant accounting policies used in the preparation of these consolidated financial statements for the three months ended March 31, 2021 are consistent with those disclosed in Note 3 to the consolidated financial statements in the 2020 Form 10-K, except as noted below.


NYS Life Sciences Research and Development Tax Credit


New York State allows investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against their New York State Tax return for certain expenditures incurred in New York State, including applicable R&D related expenditures. The credit is recognized as research and development incentives when the eligibility and amount has been approved by New York State. During the three months ended March 31, 2021 and 2020, respectively, the Company recorded research and development incentive income of $0.5 million and $0, respectively.


Recent Accounting Pronouncements


In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements (“ASU 2016-13”). The new standard requires that expected credit losses relating to financial assets measured on an amortized cost basis and available-for-sale debt securities be recorded through an allowance for credit losses. It also limits the amount of credit losses to be recognized for available-for-sale debt securities to the amount by which carrying value exceeds fair value and also requires the reversal of previously recognized credit losses if fair value increases. The targeted transition relief standard allows filers an option to irrevocably elect the fair value option of ASC 825-10, Financial Instruments-Overall, applied on an instrument-by-instrument basis for eligible instruments. The new standard was effective beginning January 1, 2021. The adoption of ASU 2016-13, and related updates, did not have a material impact on the Company’s consolidated financial position and results of operations.


There were no other recent accounting pronouncements that impacted the Company for the three months ended March 31, 2021 or are expected to have a significant effect on the consolidated financial statements.


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4.
Fair Value of Financial Instruments
 

Items measured at fair value on a recurring basis are the Company’s investments. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy:

 
Fair Value Measurements as of
March 31, 2021 Using:
 
 
 
Level 1
   
Level 2
   
Level 3
   
Total
 
Assets:
                       
Cash and cash equivalents:
                       
Money market mutual funds
 
$
235,899
   
$
-
   
$
-
   
$
235,899
 
     
235,899
     
-
     
-
     
235,899
 
 
                               
Investments:
                               
United States Treasury securities
   
107,566
     
-
     
-
     
107,566
 
Corporate Bonds
   
-
     
100,247
     
-
     
100,247
 
Municipal Bonds
   
-
     
6,000
     
-
     
6,000
 
     
107,566
     
106,247
     
-
     
213,813
 
 
                               
 
 
$
343,465
   
$
106,247
   
$
-
   
$
449,712
 

 
Fair Value Measurements as of
December 31, 2020 Using:
 
 
 
Level 1
   
Level 2
   
Level 3
   
Total
 
Assets:
                       
Cash and cash equivalents:
                       
Money market mutual funds
 
$
193,312
   
$
-
   
$
-
   
$
193,312
 
United States Treasury securities
   
62,497
     
-
     
-
     
62,497
 
Corporate Bonds
   
-
     
501
     
-
     
501
 
Agency Bonds
   
-
     
8,015
     
-
     
8,015
 
 
   
255,809
     
8,516
     
-
     
264,325
 
 
                               
Investments:
                               
United States Treasury securities
   
112,328
     
-
     
-
     
112,328
 
Corporate Bonds
   
-
     
63,710
     
-
     
63,710
 
Municipal Bonds
   
-
     
6,000
     
-
     
6,000
 
Agency Bonds
   
-
     
3,583
     
-
     
3,583
 
     
112,328
     
73,293
     
-
     
185,621
 
 
                               
 
 
$
368,137
   
$
81,809
   
$
-
   
$
449,946
 


The Company classifies its money market mutual funds and U.S. Treasury securities as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices in active markets without any valuation adjustment. The Company classifies its Government, Corporate, Municipal and Agency Bonds as Level 2 assets as these assets are not traded in an active market and have been valued through a third-party pricing service based on quoted prices for similar assets.


The fair value of the 2021 Convertible Notes and the 2022 Convertible Notes as of March 31, 2021 was $8.1 million and $53.1 million, respectively (see Note 7).

5.
Property and Equipment, Net


The Company’s property and equipment consisted of the following:

 
 
 
March 31,
2021
   
December 31,
2020
 
Laboratory equipment
 
$
8,770
   
$
7,807
 
Machinery and equipment
   
9,973
     
9,933
 
Computer equipment
   
218
     
218
 
Furniture and fixtures
   
1,867
     
1,880
 
Leasehold improvements
   
29
     
29
 
Internal use software
   
1,696
     
1,385
 
 
   
22,553
     
21,252
 
Less: accumulated depreciation and amortization
   
(2,720
)
   
(2,046
)
 
 
$
19,833
   
$
19,206
 


During the three months ended March 31, 2021 and 2020, the Company recognized $0.7 million and $0.1 million of depreciation and amortization expense, respectively.

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6.
Accounts Payable and Accrued Expenses


At March 31, 2021 and December 31, 2020, the Company’s accounts payable and accrued expenses consisted of the following:

  
 
March 31,
   
December 31,
 
   
2021
   
2020
 
Research and development
 
$
22,675
   
$
14,962
 
Property and equipment
   
337
     
1,456
 
Right of use asset
   
325
     
-
 
Employee compensation
   
6,187
     
4,875
 
Accrued interest
   
449
     
1,122
 
Government grant payable
   
597
     
590
 
Professional fees
   
1,338
     
1,332
 
Internal use software
   
311
     
300
 
Other
   
949
     
835
 
 
 
$
33,168
   
$
25,472
 

7.
 Convertibles Notes


2021 Convertible Notes


On January 4, 2018, in connection with its reverse merger with Inotek Pharmaceuticals, Corporation (“Inotek”), the Company assumed the obligations of Inotek under its outstanding convertible notes, with an aggregate principal amount of $52.0 million, (the “2021 Convertible Notes”). The 2021 Convertible Notes were issued in 2016 and were scheduled to mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes are unsecured and accrue interest at a rate of 5.75% per annum and interest is payable semi-annually on February 1 and August 1 of each year. Each holder of the 2021 Convertible Notes (“Holder”) has the option until the close of business on the second business day immediately preceding the Maturity Date to convert all, or any portion, of the 2021 Convertible Notes held by it at a conversion rate of 31.1876 shares of the Company’s common stock per $1.00 principal amount of 2021 Convertible Notes (the “Conversion Rate”) which is $32.08 per share. The Conversion Rate is subject to adjustment from time to time upon the occurrence of certain events, including the issuance of stock dividends and payment of cash dividends.

 

The Company, at its option, may redeem for cash all or any portion of the 2021 Convertible Notes if the last reported sale price of a share of the Company’s common stock is equal to or greater than 200% of the Conversion Rate for the 2021 Convertible Notes then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending within the five trading days immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2021 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date.

 

The 2021 Convertible Notes are considered a hybrid financial instrument consisting of a fixed interest rate “host” and various embedded features that required evaluation as potential embedded derivatives under FASB ASC 815, Derivatives and Hedging (“ASC 815”). Based on the nature of the host instrument and the embedded features, management concluded that none of the conversion, put and redemption features required bifurcation and separate accounting from the host instrument. The Company determined that the Additional Interest was an embedded derivative that contains non-credit related events of default. As a result, the Additional Interest feature required bifurcation and separate accounting under ASC 815. Based on the amount of Additional Interest that would be owed and the likelihood of occurrence, the Company estimated the fair value of the Additional Interest feature to be insignificant upon issuance and as of March 31, 2021 and December 31, 2020. As of March 31, 2021, the stated interest rate was 5.75%, and the effective interest rate was 15.3%.


2022 Convertible Notes

 

On February 20, 2020 and June 5, 2020, the Company entered into separate, privately negotiated exchange agreements (the “Exchange Agreements”) with certain holders of the 2021 Convertible Notes. Pursuant to the Exchange Agreements, on February 20, 2020, the Company exchanged approximately $39.35 million aggregate principal amount of the 2021 Convertible Notes (representing approximately 76% of the aggregate outstanding principal amount of the 2021 Convertible Notes) for (a) approximately $39.35 million aggregate principal amount of 6.25% Convertible Senior Notes due August 2022 (the “2022 Convertible Notes”) (an exchange ratio equal to 1.00 2022 Convertible Note per exchanged 2021 Convertible Note) and (b) $0.1 million to pay the accrued and unpaid interest on the exchanged 2021 Convertible Notes from February 1, 2020, to February 20, 2020, the closing date of the February 20, 2020 exchange transactions. Additionally, the Company repurchased 3,000 shares of its common stock that have been retired for an aggregate amount of $71,670 from certain holders of the 2021 Convertible Notes participating in the exchange transactions in privately negotiated, private transactions.


Also pursuant to the Exchange Agreements, on June 12, 2020, the Company exchanged $7.5 million aggregate principal amount of the 2021 Convertible Notes for (a) $7.5 million aggregate principal amount of its newly issued 6.25% Convertible Senior Notes due 2022 (an exchange ratio equal to 1.00 2022 Convertible Notes per exchanged 2021 Convertible Notes) and (b) approximately $11,000 to pay the accrued and unpaid interest on the exchanged 2021 Convertible Notes from, and including, February 1, 2020, to, but excluding, the closing date of the exchange transaction, adjusted to take into account the unearned accrued interest on the 2022 Convertible Notes from, and including, February 20, 2020, to, but excluding, the closing date of the exchange transaction.


The 2022 Convertible Notes were issued in a private placement exempt from registration in reliance on Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”). The 2022 Convertible Notes issued in the exchange transaction were issued as additional notes pursuant to the Indenture, dated as of August 5, 2016, between the Company and Wilmington Trust, National Association, as trustee, as supplemented by the Second Supplemental Indenture, dated as of February 20, 2020, governing the 2022 Convertible Notes.

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The conversion rate for the 2022 Convertible Notes is initially 31.1876 shares of the Company’s common stock per 1.00 principal amount of 2022 Convertible Notes, which is equivalent to an initial conversion price of approximately $32.06 per share of common stock and is subject to adjustment under the terms of the 2022 Convertible Notes. The Company may redeem for cash all or any portion of the 2022 Convertible Notes, at its option, if the last reported sale price of its common stock is equal to or greater than 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending within the five trading days immediately preceding the date on which the Company provides written notice of redemption. The 2022 Convertible Notes Indenture contains customary terms and covenants and events of default. As of March 31, 2021, the stated interest rate was 6.25%, and the effective interest rate was 15.3%.

  

In December 2020, $8.5 million principal amount, representing a carrying value of $7.6 million of the 2022 Convertible Notes was converted into 298,562 shares of the Company’s common stock.  On April 26, 2021, the Company called for the redemption of the remaining $38.4 million principal balance of the 2022 Convertible Notes (See Note 16) as the Company’s stock price traded above the Conversion Rate for at least 20 trading days during a 30-day consecutive trading period.


The table below summarizes the carrying value of the 2021 Convertible Notes as of March 31, 2021 and December 31, 2020:

 
March 31,
2021
   
December 31,
2020
 
Principal amount
 
$
5,150
   
$
5,150
 
Discount
   
(164
)
   
(275
)
Carrying value
 
$
4,986
   
$
4,875
 


Accretion of the 2021 Convertible Notes discount was $0.1 million and $0.6 million for the three months ended March 31, 2021 and 2020, respectively.


The table below summarizes the carrying value of the 2022 Convertible Notes as of March 31, 2021 and December 31, 2020:

 
March 31,
2021
   
December 31,
2020
 
Principal amount
 
$
38,350
   
$
38,350
 
Discount
   
(2,806
)
   
(3,284
)
Carrying value
 
$
35,544
   
$
35,066
 


Accretion of the 2022 Convertible Notes discount was $0.5 million and $0.2 million for the three months ended March 31, 2021 and 2020, respectively.

8.
Stock Based Compensation


Stock Option Valuation


The weighted average assumptions that the Company used in the Black-Scholes pricing model to determine the fair value of the stock options granted to employees, non-employees and directors were as follows:

 
Three Months Ended March 31,
 
 
 
2021
   
2020
 
 
     
Risk-free interest rate
   
0.62
%
   
1.48
%
Expected term (in years)
   
5.87
     
5.87
 
Expected volatility
   
69.41
%
   
77.75
%
Expected dividend yield
   
0.00
%
   
0.00
%
Exercise price
 
$
36.99
   
$
22.47
 
Fair value of common stock
 
$
36.99
   
$
22.47
 

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The following table summarizes stock option activity for the three months ended March 31, 2021 under the Second Amended and Restated 2014 Stock Option and Incentive Plan:

 
 
Number of
Shares
   
Weighted
Average
Exercise
Price
   
Weighted
Average
Contractual
Term (Years)
   
Aggregate
Intrinsic
Value
 
 
                       
Outstanding as of December 31, 2020
   
11,050,931
   
$
9.10
     
6.55
   
$
504,079
 
Granted
   
916,575
     
60.95
     
10.64
         
Exercised
   
(991,432
)
   
8.31
             
51,892
 
Cancelled
   
(83,244
)
   
31.82
                 
Outstanding as of March 31, 2021
   
10,892,830
   
$
13.42
     
6.57
   
$
349,406
 
 
                               
Options vested and exercisable as of March 31, 2021
   
7,856,137
   
$
5.70
     
5.55
   
$
300,452
 
Options unvested as of March 31, 2021
   
3,036,693
   
$
33.17
     
9.20
         


The weighted average grant-date fair value per share of stock options granted during the three months ended March 31, 2021 and 2020 was $36.99 and $15.03, respectively.


The total fair value of options vested during the three months ended March 31, 2021 and 2020 was $8.3 million and $17.3 million, respectively.


Stock-Based Compensation


Stock-based compensation expense recognized by award type was as follows:

 
Three Months Ended March 31,
 
 
 
2021
   
2020
 
 
     
Stock options
 
$
7,826
   
$
3,961
 
Restricted stock units
   
74
     
-
 
Total share based compensation expense
 
$
7,900
   
$
3,961
 



Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows:

 
Three Months Ended March 31,
 
 
 
2021
   
2020
 
 
     
Research and development
 
$
2,916
   
$
1,735
 
General and administrative
   
4,984
     
2,226
 
Total share based compensation expense
 
$
7,900
   
$
3,961
 


As of March 31, 2021, the Company had an aggregate of $55.7 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 3.01 years.


Restricted Stock Units (“RSU”)

 

The following table summarizes our RSU activity for the three months ended March 31, 2021:

 
Number of
Shares
   
Weighted
Average
Grant Date
Fair Value
 
             
Unvested as of December 31, 2020
   
20,000
   
$
25.06
 
Granted
   
3,500
     
62.32
 
Vested
   
-
     
-
 
Forfeited
   
-
     
-
 
Unvested as of March 31, 2021
   
23,500
     
30.61
 

9.
Stockholders’ Equity
 

The Company had warrants outstanding as of both March 31, 2021 and December 31, 2020, convertible into 7,051 and 603,386 shares of common stock at an exercise price of $24.82 and $57.11 per share, respectively which will expire on June 28, 2023 and December 21, 2030, respectively.

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10.
Net Loss Per Share


Basic and diluted net loss per share attributable to common stockholders was calculated as follows:

 
 
Three Months Ended March 31,
 
 
 
2021
   
2020
 
 
           
Numerator:
           
Net loss attributable to common stockholders
 
$
(40,179
)
 
$
(24,664
)
Denominator:
               
Weighted-average common shares outstanding - basic and diluted
   
61,574,405
     
54,883,120
 
Net loss per share attributable to common stockholders - basic and diluted
 
$
(0.65
)
 
$
(0.45
)


The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:

 
 
Three Months Ended March 31,
 
 
 
2021
   
2020
 
Shares issuable upon conversion of the 2021 Convertible Notes
   
160,536
     
394,327
 
Shares issuable upon conversion of the 2022 Convertible Notes
   
1,195,449
     
1,226,621
 
Warrants exercisable for common shares
   
610,437
     
14,102
 
Options to purchase common shares
   
10,892,830
     
10,538,890
 
 
   
12,859,252
     
12,173,940
 

11.
Commitments and Contingencies
 

The Company determines if an arrangement is a lease at inception. Operating and finance leases are presented in the Company’s consolidated balance sheet as right-of-use assets from leases, current lease liabilities and long-term lease liabilities. Certain of the Company’s lease agreements contain renewal options; however, the Company does not recognize right-of-use assets or lease liabilities for renewal periods unless it is determined that the Company is reasonably certain of renewing the lease at inception or when a triggering event occurs. As the Company’s leases do not provide an implicit rate, the Company estimated the incremental borrowing rate in calculating the present value of the lease payments using an estimate of the Company’s collateralized borrowing rate for debt with a similar term. The Company has utilized its incremental borrowing rate based on the long-term borrowing costs of comparable companies in the biotechnology industry. Since the Company elected to account for each lease component and its associated non-lease components as a single combined lease component, all contract consideration was allocated to the combined lease component. Some of the Company’s lease agreements contain rent escalation clauses (including index-based escalations). The Company recognizes the minimum rental expense on a straight-line basis based on the fixed components of a lease arrangement. The Company will amortize this expense over the term of the lease beginning with the lease commencement date. Variable lease components represent amounts that are not fixed in nature and are not tied to an index or rate and are recognized as incurred.


Finance Lease

 

The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research and development laboratories and 50,000 square feet dedicated to AAV Current Good Manufacturing Practice (cGMP) manufacturing facilities to support the Company’s pipeline. A smaller area within this facility was originally leased in August 2018, and the lease was amended in June 2019 to include the full building (such lease, as amended, the “NJ Lease Agreement”). The NJ Lease Agreement has a 15-year term from September 1, 2019, with an option to renew for two consecutive five-year renewal terms.

 

Estimated rent payments for the NJ Lease Agreement are $1.2 million per annum, payable in monthly installments, depending upon the nature of the leased space, and subject to annual base rent increases of 3%. The total commitment under the NJ Lease Agreement is estimated to be approximately $29.3 million over the 15-year term of the NJ Lease Agreement. The Company paid a cash security deposit of $0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected in deposits in the consolidated balance sheets as of March 31, 2021 and December 31, 2020.

 

The Company determined the lease commencement date was reached on March 15, 2020 when the construction of all landlord owned improvements had been substantially completed and when the Company began including its leasehold improvements on the balance sheet and move equipment into the space. Upon commencement of the NJ Lease Agreement in March 2020, the Company recognized total right-of-use assets of $47.7 million, with a corresponding lease liability of $20.2 million. The Company reclassified $26.5 million of construction costs in progress and $1.1 million of prepaid rent as part of the right of use asset upon the lease commencement date of March 15, 2020. During the three months ended March 31, 2021, the Company reclassified an additional $0.3 million bringing the aggregate reclassification to $32.4 million of construction costs in progress as part of the right of use asset.


Interest associated with the financing lease was $0.5 million and $0.2 million for the three months ended March 31, 2021 and 2020, respectively and is recorded as interest expense on the consolidated statements of operations.


Operating Leases

 

On June 7, 2018, the Company entered into a three-year lease agreement for office space in the Empire State Building in New York, NY (the “ESB Lease Agreement”). In connection with the ESB Lease Agreement, the Company established an irrevocable standby letter of credit (the “Empire LOC”) with a bank for $0.9 million which expired on June 30, 2020 and renewed automatically for a one-year period until its expiration date of July 31, 2021. On March 26, 2021, the Company entered in Amendment No. 1 to the ESB Lease Agreement (“ESB Lease Amendment”) that extended the term of the lease agreement to June 30, 2024, reduced the rent payments going forward, and reduced the Empire LOC down to $0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary. The Company has accounted for the ESB Lease Amendment as a modification to the ESB Lease Agreement. The Company remeasured lease liability and adjusted the operating lease right of use asset by $1.1 million.
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The Company has a certificate of deposit of $0.8 million and $0.9 million with a bank as collateral for the Empire LOC which is classified as part of restricted cash in the consolidated balance sheets as of March 31, 2021 and December 31, 2020.  This security deposit was reduced to $0.8 million in March 2021 in connection with the ESB Lease Amendment.

 

On January 4, 2018, in connection with the Reverse Merger, the Company assumed an operating lease for Inotek’s former headquarters in Lexington, Massachusetts, with a term ending in February 2023. In July 2018, the Company signed an agreement to sublease a portion of the Lexington, Massachusetts space and in September 2018, the Company signed an agreement to sublease the remaining portion of the Lexington, Massachusetts space. Rental income received under the sublease agreement totaled $0.1 million and $0.1 million for the three months ended March 31, 2021 and 2020, respectively. Rental income is netted against rent expense in the consolidated statement of operations.


Rent expense was $0.3 million for the three months ended March 31, 2021 and 2020.


The total restricted cash balance for the Company’s operating and finance leases at March 31, 2021 and December 31, 2020 was $1.0 million and $1.1 million, respectively.

Lease cost
 
March 31, 2021
 
Operating lease cost
 
$
196
 
Finance lease cost
       
Amortization of right of use assets
   
535
 
Interest on lease liablities
   
459
 
Total lease cost
 
$
1,190
 


The following table summarizes the maturity of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of March 31, 2021:

Maturity of operating lease liabilities
 
March 31, 2021
 
2021
   
544
 
2022
   
860
 
2023
   
488
 
2024
   
208
 
Total lease payments
 
$
2,100
 
Less: interest
   
(118
)
Total operating lease liabilities
 
$
1,982
 

Maturity of finance lease liability
 
March 31, 2021
 
2021
   
1,235
 
2022
   
1,689
 
2023
   
1,736
 
2024
   
1,791
 
Thereafter
   
48,768
 
Total lease payments
 
$
55,219
 
Less: interest
   
(34,536
)
Total finance lease liability
 
$
20,683
 

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Leases
 
March 31, 2021
 
 
     
Operating right-of-use assets
 
$
1,787
 
 
       
Operating current lease liabilities
   
683
 
Operating noncurrent lease liabilities
   
1,299
 
Total operating lease liabilities
 
$
1,982
 
 
       
Finance right-of-use assets
 
$
50,311
 
Finance current lease liability
   
1,655
 
Finance noncurrent lease liability
   
19,028
 
Total finance lease liability
 
$
20,683
 

Other information
     
Cash paid for amounts included in the measurement of lease liabilities:
     
Operating cash flows from operating leases
 
$
226
 
Cash flows from finance lease
 
$
408
 
Weighted-average remaining lease term - operating leases
 
2.7 years
 
Weighted-average remaining lease term - finance lease
 
23.4 years
 
Weighted-average discount rate - operating leases
   
4.90
%
Weighted-average discount rate - finance lease
   
8.96
%


Litigation


From time to time, the Company may be subject to other various legal proceedings and claims that arise in the ordinary course of its business activities. Although the results of litigation and claims cannot be predicted with certainty, the Company does not believe it is party to any other claim or litigation the outcome of which, if determined adversely to the Company, would individually or in the aggregate be reasonably expected to have a material adverse effect on its business. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other factors.


Indemnification Arrangements


Pursuant to its bylaws and as permitted under Delaware law, the Company has indemnification obligations to directors, officers, employees or agents of the Company or anyone serving in these capacities. The maximum potential amount of future payments the Company could be required to pay is unlimited. The Company has insurance that reduces its monetary exposure and would enable it to recover a portion of any future amounts paid. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.


Throughout the normal course of business, the Company has agreements with vendors that provide goods and services required by the Company to run its business. In some instances, vendor agreements include language that requires the Company to indemnify the vendor from certain damages caused by the Company’s use of the vendor’s goods and/or services. The Company has insurance that would allow it to recover a portion of any future amounts that could arise from these indemnifications. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.

12.
Agreements Related to Intellectual Property


The Company has various license and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property which is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols, or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in research and development activities has no alternative future uses.

13.
CIRM Grants


LAD-I CIRM Grant


On April 30, 2019, the California Institute for Regenerative Medicine (“CIRM”) awarded the Company up to $6.5 million under a CLIN2 grant award to support the clinical development of gene therapy for LAD-I. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase I/II patients enrolled at the U.S. clinical site, University of California, Los Angeles (“UCLA”) Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. In 2019, the Company received the first two grants from CIRM in the aggregate of $1.2 million, based on eligible costs incurred under the grant. The CIRM grant reimbursements are accrued as an offset against R&D expenses as reimbursable expenses are incurred. As of December 31, 2020, the Company met the next CIRM milestone and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $1.1 million. The Company received the $1.1 million milestone payment on January 4, 2021. As of March 31, 2021, the Company met the next CIRM milestone and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $1.0 million. The Company received the $1.0 million milestone payment on April 1, 2021.

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IMO CIRM Grant


On November 12, 2020, the CIRM awarded the Company up to $3.7 million under a CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of IMO. The Company received a $1.1 million pursuant to the grant on January 4, 2021 related to the CIRM IMO award and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $0.9 million as of December 31, 2020.  The Company recorded a reduction of research and development expense of $0.1 million for the three months ended March 31, 2021.

14.
Related Party Transactions


During April 2018, the Company entered into an agreement with a member of the Board of Directors for business development consulting services. Payments for the services under the agreement are $28 per quarter, and the Company may terminate the agreement with 14 days’ notice. The Company incurred expenses of $28 during the three months ended March 31, 2021 and 2020, relating to services provided under this agreement.


In June 2020, the Company entered into a consulting agreement with a member of the Board of Directors for pipeline development, new asset evaluation, and corporate strategy. In lieu of cash for services to be provided under the consulting agreement during its one-year term, the Company granted the board member options to purchase 9,784 options with a fair value of $0.1 million.


In October 2020, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services.  In exchange for the services provided under the agreement, the Company granted 10,000 restricted stock units which vest over a three-year period.


In December 2020, the Company entered into a consulting agreement with a related party. Pursuant to the consulting agreement, the related party provides certain business development and asset identification consulting services to the Company. The term of the consulting agreement is three years and may be terminated with 60 days’ notice by either party. In exchange for the business development services to be provided under the agreement, the Company issued a warrant exercisable for 603,386 shares of common stock. Pursuant to the consulting agreement, the related party is entitled to receive additional warrants exercisable for common stock upon identification of new assets for the Company to in-license. There were no warrants issued for the three months ended March 31, 2021.

15.
401(k) Savings Plan


The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected to the safe harbor match of 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three months ended March 31, 2021 and 2020 was $0.2 million and $0.1 million, respectively.

16.
Subsequent Events


On April 26, 2021, the Company completed a full redemption of its 2022 Convertible Notes prior to the redemption date. Holders of approximately $38.4 remaining million principal amount of the 2022 Convertible Notes converted such notes in accordance with the terms of the governing indenture into approximately 1.3 million shares of the Company’s common stock and cash in lieu of fractional shares. Following the redemption, approximately $5.2 million aggregate principal amount of the 2021 Convertible Notes remain outstanding.

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Item 2.
Management’s Discussion and Analysis of Financial Condition and Results of Operations

You should read the following discussion and analysis of our financial condition and results of operations together with the consolidated financial statements and related notes that are included elsewhere in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the fiscal year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission, or the SEC, on March 1, 2021 (“2020 Form 10-K”). This discussion contains forward-looking statements based upon current plans, expectations and beliefs that involve risks and uncertainties. Our actual results may differ materially from those anticipated in these forward-looking statements as a result of various factors, including, but not limited to, those discussed in the section entitled “Risk Factors” and elsewhere in this Quarterly Report on Form 10-Q. In preparing this MD&A, we presume that readers have access to and have read the MD&A in our 2020 Form 10-K, pursuant to Instruction 2 to paragraph of Item 303 of Regulation S-K. Unless stated otherwise, references in this Quarterly Report on Form 10-Q to “us,” “we,” “our,” or our “Company” and similar terms refer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. We have four clinical-stage ex vivo lentiviral vector (“LVV”). These include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I), a genetic disorder that causes the immune system to malfunction, Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia and Infantile Malignant Osteopetrosis (“IMO”), a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program are in registration-enabling studies in the United States (“U.S.”) and Europe (“EU”). In addition, in the U.S., we have a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. We have global commercialization and development rights to all of these product candidates under royalty-bearing license agreements. Additional discovery efforts on a gene therapy program for the less common FA subtypes C and G is ongoing.

Recent Developments

On April 26, 2021, we completed a full redemption of our 2022 Convertible Notes prior to the redemption date. Holders of approximately $38.4 remaining million principal amount of the 2022 Convertible Notes converted such notes in accordance with the terms of the governing indenture into approximately 1.3 million shares of the Company’s common stock and cash in lieu of fractional shares.

On May 10, 2021 we announced that the RP-A501 Danon Disease program was placed on clinical hold by the FDA. No new drug-related safety events have been observed in the low- or high-dose adult cohorts of the Phase 1 trial. The FDA has requested that we pause patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management. All follow-up study activities will continue and no additional data are requested. We are continuing our dialogue with the agency to ensure safety measures are updated and harmonized adequately and anticipate additional patient treatment by 3Q2021.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid (“DNA”), which code for proteins that perform a broad range of physiologic functions in all living organisms. Although genes are passed on from generation to generation, genetic changes, also known as mutations, can occur in this process. These changes can result in the lack of production of proteins or the production of altered proteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence or segment of DNA is administered to a patient, most commonly for the purpose of treating a genetic disease that is caused by genetic mutations. Currently available therapies for many genetic diseases focus on administration of large proteins or enzymes and typically address only the symptoms of the disease. Gene therapy aims to address the disease-causing effects of absent or dysfunctional genes by delivering functional copies of the gene sequence directly into the patient’s cells, offering the potential for curing the genetic disease, rather than simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our gene therapy treatments. Viruses are particularly well suited as delivery vehicles because they are adept at penetrating cells and delivering genetic material inside a cell. In creating our viral delivery vehicles, the viral (pathogenic) genes are removed and are replaced with a functional form of the missing or mutant gene that is the cause of the patient’s genetic disease. The functional form of a missing or mutant gene is called a therapeutic gene, or the “transgene.” The process of inserting the transgene is called “transduction.” Once a virus is modified by replacement of the viral genes with a transgene, the modified virus is called a “viral vector.” The viral vector delivers the transgene into the targeted tissue or organ (such as the cells inside a patient’s bone marrow). We have two types of viral vectors in development, LVV and AAV. We believe that our LVV and AAV-based programs have the potential to offer a significant therapeutic benefit to patients that is durable (long-lasting).

The gene therapies can be delivered either (1) ex vivo (outside the body), in which case the patient’s cells are extracted and the vector is delivered to these cells in a controlled, safe laboratory setting, with the modified cells then being reinserted into the patient, or (2) in vivo (inside the body), in which case the vector is injected directly into the patient, either intravenously (“IV”) or directly into a specific tissue at a targeted site, with the aim of the vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greater regulatory acceptance of gene therapy have created a promising environment to advance gene therapy products as these products are being designed to restore cell function and improve clinical outcomes, which in many cases include prevention of death at an early age. The FDA approval of several gene therapies in recent years is supportive of a regulatory pathway forward for gene therapy products.

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Pipeline Overview

The chart below shows the current phases of development of Rocket’s programs and product candidates:


AAV Program:

Danon Disease:
Danon disease is a multi-organ lysosomal-associated disorder leading to early death due to heart failure. Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (“LAMP-2”), a mediator of autophagy. This mutation results in the accumulation of autophagic vacuoles, predominantly in cardiac and skeletal muscle. Male patients often require heart transplantation and typically die in their teens or twenties from progressive heart failure. Along with severe cardiomyopathy, other Danon disease symptoms can include skeletal muscle weakness, liver disease, and intellectual impairment. There are no specific therapies available for the treatment of Danon disease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease, which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and the EU, however new market research is being performed and the prevalence of patients may be updated in the future.

Danon disease is an autosomal dominant, rare inherited disorder characterized by progressive cardiomyopathy which is almost universally fatal in males even in settings where cardiac transplantation is available. Danon disease predominantly affects males early in life and is characterized by absence of LAMP2B expression in the heart and other tissues. Pre-clinical models of Danon disease have demonstrated that AAV-mediated transduction of the heart results in reconstitution of LAMP2B expression and improvement in cardiac function.

As of December 31, 2020, we have treated five patients in the RP-A501 Phase 1 clinical trial and completed the first cohort of the study evaluating an initial low-dose in male patients age 15 or greater. The preliminary data announced in December 2020 for the low dose cohort included safety and clinical activity results from the three patients treated with the low dose of 6.7×1013 genome copies (gc)/kilogram (kg) and early safety information from the two patients treated with the higher dose of 1.1×1014 gc/kg as of the cutoff date of November 2020.

In the three patients treated in the low dose cohort, RP-A501 showed manageable safety results. No unexpected and serious drug product-related adverse events or severe adverse events were observed. The most common adverse events were mild and were related to elevated transaminases post treatment. Elevation in transaminases was observed in all three low-dose patients and returned to baseline within the first one to two months post-treatment. There was also a transient and reversible decline in platelets observed in these three patients. These changes were largely responsive to corticosteroids and other immunosuppressive therapies. All patients were given oral steroids to prevent or minimize potential immune-related events.

At the higher dose administered (1.1×1014 gc/kg), additional immunosuppressive therapies were stipulated and administered to mitigate the immune response associated with RP-A501. One of the two treated patients, who received the higher absolute AAV9 dose and had some degree of pre-existing anti-AAV9 immunity, experienced a non-persistent, immune-related event that was classified as a drug product-related serious adverse event. This was believed to be likely due to complement activation, resulting in reversible thrombocytopenia and acute kidney injury requiring eculizumab and transient hemodialysis. This patient returned to baseline within three weeks and regained normal kidney function.

From the perspective of gene expression results, all three low dose patients demonstrated evidence of cardiac LAMP2B expression by Western blot and/or immunohistochemistry. In two of the three patients in the low dose cohort who had closely monitored compliance with the immunosuppressive regimen, high levels of cardiac LAMP2B expression were observed along with clinical biomarker improvements. In cardiac biopsies of the low dose patients, LAMP2B gene expression was observed in 67.8% of cells compared to normal as determined by immunohistochemistry at 9 months in one patient, and at 92.4% of cells compared to normal at month 12 in the other patient. In this latter patient, Western blot assessment showed 61% of normal LAMP2B protein expression at month 9. The 12-month Western blot data was still pending for all three patients as of the data cutoff.

The first patient in the low dose cohort was not as closely monitored for compliance with the immunosuppressive regimen as the other two patients. Although we did observe evidence of cardiac LAMP2B expression of approximately 15% of cells compared to normal as determined by immunohistochemistry at 12 months in this patient, we believe that such expression was likely limited by inconsistent compliance with the immunosuppressive regimen, as evidenced by transient increases in transaminase levels approximately one month after treatment and a lack of adverse events frequently associated with the immunosuppressive regimen. Additionally, in this patient, Western blot assessment showed 17.9% of normal LAMP2B protein expression at month 6.

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At least two of the three low dose patients demonstrated key clinical biomarker improvements consistent with improved cardiac function. Brain natriuretic peptide, a key marker of heart failure, improved in all three patients, including by greater than 50% in the two patients with closely monitored immunosuppressive regimen compliance. Additionally, creatine kinase myocardial band either improved or stabilized in these two patients. For the patient with the potential inconsistent immunosuppressive regimen compliance, the creatine kinase myocardial band was higher than at baseline at month 12. Notably, all three patients showed visible improvements in autophagic vacuoles, a hallmark of Danon disease pathology, as assessed by electron microscopy of cardiac tissue via endomyocardial biopsy. Additionally, two of the three low dose patients with closely monitored immunosuppressive regimen compliance demonstrated improvement in cardiac output as measured by invasive hemodynamics, including one patient who showed a 1.62-fold improvement in cardiac output at month 12, and one patient who showed a 1.35-fold improvement at month 9. For the patient with the apparent inconsistent immunosuppressive regimen compliance, the cardiac output was lower at month 12 than at baseline.

We expect to announce updated data from our open-label Phase 1 trial of RP-A501 in the fourth quarter of 2021.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from a mutation in a single FA gene. An estimated 60 to 70% of cases arise from mutations in the Fanconi-A (“FANCA”) gene, which is the focus of our program. FA results in bone marrow failure, developmental abnormalities, myeloid leukemia, and other malignancies, often during the early years and decades of life. Bone marrow aplasia, which is bone marrow that no longer produces any or very few red and white blood cells and platelets leading to infections and bleeding, is the most frequent cause of early morbidity and mortality in FA, with a median onset before 10 years of age. Leukemia is the next most common cause of mortality, ultimately occurring in about 20% of patients later in life. Solid organ malignancies, such as head and neck cancers, can also occur, although at lower rates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem cell transplant (“HSCT”), currently the most frequently utilized therapy for FA, have resulted in more frequent hematologic correction of the disorder, HSCT is associated with both acute and long-term risks, including transplant-related mortality, graft versus host disease (“GVHD”), a sometimes fatal side effect of allogeneic transplant characterized by painful ulcers in the GI tract, liver toxicity and skin rashes, as well as increased risk of subsequent cancers. Our gene therapy program in FA is designed to enable a minimally toxic hematologic correction using a patient’s own stem cells during the early years of life. We believe that the development of a broadly applicable autologous gene therapy can be transformative for these patients.

In December 2020, we presented updated interim data from our FA at the 62nd American Society of Hematology (“ASH”) Annual Meeting.  The FA data presented at the ASH Annual Meeting were from seven of the nine patients treated (out of twelve patients enrolled) as of October 2020 in both the U.S. Phase 1 and global Phase 2 studies of RP-L102 for FA. Patients in these studies received a single intravenous infusion of “Process B” RP-L102 which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector. Preliminary data from these studies support “Process B” as a consistent and reproducible improvement over “Process A” which was used in earlier academic FA studies.

Seven patients had follow-up data of at least two-months and three of the seven patients had been followed for twelve-months or longer. As patients are treated with gene therapy product without the use of a conditioning regimen, the data indicated that RP-L102 was generally well-tolerated with no significant safety issues reported with infusion or post-treatment. One drug related serious adverse event of Grade 2 transient infusion-related reaction was observed. In five out of the seven patients for whom there was follow-up data, evidence of preliminary engraftment was observed, with bone marrow (“BM”) vector copy numbers (“VCNs”) from 0.16 to 0.22 (long-term follow-up only) and peripheral VCNs ranging from 0.01 (2-month follow-up) to 0.11 (long-term follow-up). Further, two of the three patients with greater than 12-months follow-up showed evidence of increasing engraftment, mitomycin-C (“MMC”) resistance and stable blood counts, which suggests a halt in the progression of bone marrow failure. The third patient with greater than 12-month follow-up contracted Influenza B nine months post-treatment resulting in progressive BM failure, for which, such patient received a successful bone marrow transplant at 18 months post-treatment.

We expect to report longer-term follow up on these patients in the second quarter of 2021.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion and migration, resulting from mutations in the ITGB2 gene encoding for the Beta-2 Integrin component, CD18. Deficiencies in CD18 result in an impaired ability for neutrophils (a subset of infection-fighting white blood cells) to leave blood vessels and enter tissues where these cells are needed to combat infections. As is the case with many rare diseases, accurate estimates of incidence are difficult to confirm; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. Severe LAD-I is notable for recurrent, life-threatening infections and substantial infant mortality in patients who do not receive an allogeneic HSCT. Mortality for severe LAD-I has been reported as 60 to 75% by age two in the absence of allogeneic HCST.

We currently have one ex-vivo program targeting LAD-I, RP-L201. RP-L201 is a clinical program that we in-licensed from CIEMAT. We have partnered with UCLA to lead U.S. clinical development efforts for the LAD-I program. UCLA and its Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is serving as the lead U.S. clinical research center for the registrational clinical trial for LAD-I, and HNJ is serving as the lead clinical site in Spain. GOSH in London is also a site for the LAD-I trial. This study has received a $6.5 million CLIN2 grant award from the California Institute for Regenerative Medicine (“CIRM”) to support the clinical development of gene therapy for LAD-I.

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The ongoing open-label, single-arm, Phase 1/2 registration enabling clinical trial of RP-L201 has treated four severe LAD-I patients to assess the safety and tolerability of RP-L201 to date. The first patient was treated at UCLA with RP-L201 in the third quarter of 2019. Enrollment is now complete in this study.

In April 2021, Rocket presented positive clinical updates from RP-L201 at the Clinical Immunology Society (CIS) Annual Meeting. The Phase 1/2 data presented in a poster at CIS 2021 are from four pediatric patients with severe LAD-I. RP-L201 was well tolerated with no safety issues reported with treatment or post-treatment. All four patients achieved hematopoietic reconstitution within 5-weeks and demonstrated CD18 expression substantially exceeding the 4-10% threshold associated with survival into adulthood. The first patient with 18-months follow up demonstrated durable CD18 expression of ~40%, peripheral blood vector copy number (VCN) levels of 1.2 at 12-months post-treatment and resolution of skin lesions with no new lesions. The second patient with 9-months of follow up demonstrated CD18 expression of ~28% and peripheral blood VCN levels of 0.75 at 6-months post-treatment with kinetics consistent with those of the first patient. The third and fourth patients demonstrated high CD18 expression of ~70% and ~51%, respectively at 3-months post treatment, and peripheral blood VCN kinetics consistent with those of the first two patients.

The LAD-I program received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and Priority Medicines (PRIME) designation from the European Medicines Agency (“EMA”), completing the full complement of all U.S. and EU accelerated regulatory designations for the program.

We expect to announce initial Phase 2 data for our LAD-I program in the third quarter of 2021.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting from mutations in the pyruvate kinase L/R (“PKLR”) gene encoding for a component of the red blood cell (“RBC”) glycolytic pathway. PKD is characterized by chronic non-spherocytic hemolytic anemia, a disorder in which RBCs do not assume a normal spherical shape and are broken down, leading to decreased ability to carry oxygen to cells, with anemia severity that can range from mild (asymptomatic) to severe forms that may result in childhood mortality or a requirement for frequent, lifelong RBC transfusions. The pediatric population is the most commonly and severely affected subgroup of patients with PKD, and PKD often results in splenomegaly (abnormal enlargement of the spleen), jaundice and chronic iron overload which is likely the result of both chronic hemolysis and the RBC transfusions used to treat the disease. The variability in anemia severity is believed to arise in part from the large number of diverse mutations that may affect the PKLR gene. Estimates of disease incidence have ranged between 3.2 and 51 cases per million in the white U.S. and EU population. Industry estimates suggest at least 2,500 cases in the U.S. and EU have already been diagnosed despite the lack of FDA-approved molecularly targeted therapies. Market research indicates the application of gene therapy to broader populations could increase the market opportunity from approximately 250 to 500 patients per year.

We currently have one ex-vivo LVV-based program targeting PKD, RP-L301. RP-L301 is a clinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 to initiate the global Phase 1 study cleared in October 2019. This program has been granted US and EMA orphan drug disease designation.

This global Phase 1 open-label, single-arm, clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial will be comprised of three cohorts to assess RP-L301 in young pediatric (age 8-11), older pediatric (age 12-17) and adult populations. The trial is designed to assess the safety, tolerability, and preliminary activity of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in pediatric patients. Stanford will serve as the lead site in the U.S. for adult and pediatric patients, HNJ will serve as the lead site in Europe for pediatrics, and Hospital Universitario Fundación Jiménez Díaz will serve as the lead site in Europe for adult patients. In July 2020, we treated the first patient in our clinical trial of RP-L301.

The data presented at the 2020 ASH Annual Meeting were from two adult PKD patients with significant anemia and transfusion requirements. Patient L301-006-1001 was treated with RP-L301. Preliminary data from this first patient supported initial tolerability of RP-L301, hemoglobin improvement to a normal range at 3-months post treatment and additional normalization of hemolysis markers. The patient was 31-years of age at the time of enrollment and had been followed for 3-months post treatment as of the data cutoff date of October 2020.

Patient L301-006-1001 received a cell dose of 3.9x106 cells/kilogram (“kg”) with a drug product mean VCN of 2.73. For this patient, hematopoietic reconstitution was observed in less than two weeks. Furthermore, the patient attained peripheral blood VCN of 2.21 at 1-month and 1.55 at 3-months and normalized hemoglobin (“Hgb”) and hemolysis markers at 3-months post-treatment. In particular, at baseline, the patient had Hb of approximately 7.4 grams (“g”)/deciliter (“dL”) to Hb of 14.3 g/dL at 3-months post treatment with RP-L301. In the two years prior to enrollment, the patient underwent approximately 14 transfusion episodes; subsequent to engraftment from RP-L301 treatment, the patient to date has not required any red blood cell transfusions. The patient also exhibited normalization of bilirubin, lactate dehydrogenase and erythropoietin levels at 3-months post treatment, each of which had been substantially elevated prior to study enrollment. The patient also had an increase in hepcidin and a decrease in reticulocytes at 3-months post treatment.

The data from Patient L301-006-1001 indicated that RP-L301 was generally well-tolerated and there were no serious safety issues or infusion-related complications observed 3-months post treatment. The patient experienced Grade 3 treatment-emergent adverse events of neutropenia, stomatitis, increased liver transaminase levels (AST and ALT) and a Grade 4 treatment-emergent adverse event of hypertriglyceridemia; the investigator did not consider these adverse events related to RP-L301.

Patient L301-006-1001 also experienced Grade 2 serious adverse events of chest pain, dyspnea, and nausea during the apheresis collection. The investigator considered these events related to apheresis, hyperleukocytosis and the mobilizing agents. They resolved with supportive care and without sequelae. Other events included Grade 2 bone pain and Grade 3 leukocytosis. The second cohort of this study will enroll older pediatric patients and is expected to be initiated in the first half of 2021.

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A second patient L301-006-1002, was 47 years old at the time of enrollment and had been recently treated with RP-L301, receiving a cell dose of 2.4x106 cells/kg with a mean drug product VCN of 2.08.

In March 2021, we announced updated positive preliminary clinical data from Phase 1 trial of RP-L301 for the treatment of PKD. The updated preliminary Phase 1 RP-L301 data are from two patients that showed sustained safety and tolerability 6- and 3-months after treatment, respectively. The two patients demonstrated durable normalization of hemoglobin levels from an average baseline of ~7.4 to 13.9 g/dL at 6-months post treatment in the first patient and from a baseline of ~7.0 g/dL to 13.8 g/dL at 3-months post treatment in the second patient. Respectively, the two patients demonstrated significant improvements in bilirubin 6- and 3-months after treatment, which had been substantially elevated prior to study enrollment.

The Phase 1 trial continues to enroll patients and we expect to report longer-term data in the fourth quarter of 2021, with a near-term update at the American Society for Cell and Gene Therapy Meeting in May 2021.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. During normal growth and development small areas of bone are constantly being broken down by special cells called osteoclasts, then made again by cells called osteoblasts. In IMO, the cells that break down bone (osteoclasts) do not work properly, which leads to the bones becoming thicker and not as healthy. Untreated IMO patients may suffer from a compression of the bone-marrow space, which results in bone marrow failure, anemia, and increased infection risk due to the lack of production of white blood cells. Untreated IMO patients may also suffer from a compression of cranial nerves, which transmit signals between vital organs and the brain, resulting in blindness, hearing loss and other neurologic deficits.

IMO represents the autosomal recessive, severe variants of a group of disorders characterized by increased bone density and bone mass secondary to impaired bone resorption. IMO typically presents in the first year of life and is associated with severe manifestations leading to death within the first decade of life in the absence of allogeneic HSCT, although HSCT results have been limited to-date and notable for frequent graft failure, GVHD and other severe complications.

Approximately 50% of IMO results from mutations in the TCIRG1 gene, resulting in cellular defects that prevent osteoclast bone resorption. As a result of this defect, bone growth is markedly abnormal. It is estimated that IMO occurs in 1 out of 250,000-300,000 within the general global population, although incidence is higher in specific geographic regions including Costa Rica, parts of the Middle East, the Chuvash Republic of Russia, and the Vasterbotten Province of Northern Sweden.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is a preclinical program that we in-licensed from Lund University, Sweden. This program has been granted ODD and Rare Pediatric Disease designation from the FDA. We have partnered with UCLA to lead U.S. clinical development efforts for the IMO program and UCLA will serve as the lead U.S. clinical site for IMO. The IND for RP-L401 to initiate a global Phase 1 study was cleared by the FDA in June 2020. The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status, and endocrine abnormalities.

In October 2020, we presented pre-clinical data from our LVV-based program targeting IMO, RP-L401, at the ESID 2020 Meeting. Preclinical data on IMO indicate that a modest level of engraftment can correct the disease phenotype in vivo, with increased long-term survival, tooth eruption, weight gain and normalized bone resorption. A comprehensive review of pre-clinical gene therapy investigations in TCIRG1-mediated osteopetrosis published in December 2020 supports acceleration into clinical development for RP-L401.

A clinical trial for RP-L401 was initiated in the fourth quarter of 2020 and recruitment is currently ongoing. On November 12, 2020, the CIRM awarded Rocket up to $3.7 million under a CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of IMO.

We expect to report initial Phase 1 clinical trial data for our IMO program in the third quarter of 2021.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated, rare pediatric diseases through the development and commercialization of potentially curative first-in-class gene therapies. To achieve these objectives, we intend to develop into a fully-integrated biotechnology company. In the near- and medium-term, we intend to develop our first-in-class product candidates, which are targeting devastating diseases with substantial unmet need, develop proprietary in-house analytics and manufacturing capabilities and continue to commence registration trials for our currently planned programs. In the medium and long-term, we expect to submit our first biologics license applications (“BLAs”) and establish our gene therapy platform and expand our pipeline to target additional indications that we believe to be potentially compatible with our gene therapy technologies. In addition, during that time, we believe that our currently planned programs will become eligible for priority review vouchers from the FDA that provide for expedited review. We have assembled a leadership and research team with expertise in cell and gene therapy, rare disease drug development and product approval.

We believe that our competitive advantage lies in our disease-based selection approach, a rigorous process with defined criteria to identify target diseases. We believe that this approach to asset development differentiates us as a gene therapy company and potentially provides us with a first-mover advantage.

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Financial Overview

Since our inception, we have devoted substantially all our resources to organizing and staffing, business planning, raising capital, acquiring, or discovering product candidates and securing related intellectual property rights, conducting discovery, research and development activities for the programs and planning for potential commercialization. We do not have any products approved for sale and have not generated revenue from product sales. From inception through March 31, 2021, we raised net cash proceeds of approximately $654.1 million from investors through both equity and convertible debt financing to fund operating activities. As of March 31, 2021, we had cash, cash equivalents and investments of $466.4 million.

Since inception, we have incurred significant operating losses. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of the current or future product candidates and programs. We had net losses of $40.2 million for the three months ended March 31, 2021 and $139.7 million for the year ended December 31, 2020. As of March 31, 2021, we had an accumulated deficit of $363.0 million. We expect to continue to incur significant expenses and higher operating losses for the foreseeable future as we advance our current product candidates from discovery through preclinical development and clinical trials and seek regulatory approval of our product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to product manufacturing, marketing, sales and distribution. Furthermore, we expect to incur additional costs as a public company as we transition out of emerging growth company status. Accordingly, we will need additional financing to support continuing operations and potential acquisitions of licensing or other rights for product candidates.

Until such a time as we can generate significant revenue from product sales, if ever, we will seek to fund our operations through public or private equity or debt financings or other sources, which may include collaborations with third parties and government programs or grants. Adequate additional financing may not be available to us on acceptable terms, or at all. We can make no assurances that we will be able to raise the cash needed to fund our operations and, if we fail to raise capital when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more product candidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain profitability. Even if we are able to generate product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including from product sales, and we do not expect to generate any revenue from the sale of products in the near future. If our development efforts for product candidates are successful and result in regulatory approval or license agreements with third parties, we may generate revenue in the future from product sales.

Operating Expenses

Research and Development Expenses

Our R&D program expenses consist primarily of external costs incurred for the development of our product candidates. These expenses include:
expenses incurred under agreements with research institutions that conduct R&D activities including, process development, preclinical, and clinical activities on our behalf;
costs related to process development, production of preclinical and clinical materials, including fees paid to contract manufacturers and manufacturing input costs for use in internal manufacturing processes;
consultants supporting process development and regulatory activities;
patent fees; and
costs related to in-licensing of rights to develop and commercialize our product candidate portfolio.

We recognize external development costs based on contractual payment schedules aligned with program activities, invoices for work incurred, and milestones which correspond with costs incurred by the third parties. Nonrefundable advance payments for goods or services to be received in the future for use in R&D activities are recorded as prepaid expenses.

Our direct R&D expenses are tracked on a program-by-program basis for product candidates and consist primarily of external costs, such as research collaborations and third-party manufacturing agreements associated with our preclinical research, process development, manufacturing, and clinical development activities. Our direct R&D expenses by program also include fees incurred under license agreements. Our personnel, non-program and unallocated program expenses include costs associated with activities performed by our internal R&D organization and generally benefit multiple programs. These costs are not separately allocated by product candidate and consist primarily of:
salaries and personnel-related costs, including benefits, travel and stock-based compensation, for our scientific personnel performing R&D activities;
facilities and other expenses, which include expenses for rent and maintenance of facilities, and depreciation expense; and
laboratory supplies and equipment used for internal R&D activities.

Our R&D activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development. As a result, we expect that R&D expenses will increase substantially over the next several years as we increase personnel costs, including stock-based compensation, support ongoing clinical studies, seek to achieve proof-of-concept in additional product candidates, advance preclinical programs to clinical programs, and prepare regulatory filings for product candidates.

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We cannot determine with certainty the duration and costs to complete current or future clinical studies of product candidates or if, when, or to what extent we will generate revenues from the commercialization and sale of any of its product candidates that obtain regulatory approval. We may never succeed in achieving regulatory approval for any of our product candidates. The duration, costs, and timing of clinical studies and development of product candidates will depend on a variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any clinical studies and other R&D activities that we undertake;
future clinical study results;
uncertainties in clinical study enrollment rates;
changing standards for regulatory approval; and
the timing and receipt of any regulatory approvals.

We expect R&D expenses to increase for the foreseeable future as we continue to invest in R&D activities related to developing product candidates, including investments in manufacturing, as our programs advance into later stages of development and as we conduct additional clinical trials. The process of conducting the necessary clinical research to obtain regulatory approval i