Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial and Operational Results

— Cash position of $388.7M; runway expected into 2H’23 —

CRANBURY, N.J. – Feb. 24, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial and operational results for the fourth quarter and year ended December 31, 2021.

“2021 was a very productive year marked by clinical data supporting potential durable cures in three devastating bone marrow-derived diseases, and for the first time in history by the demonstration of potential durable clinical benefit in a cardiac disease using a gene therapy approach,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “The stage is set for 2022 to be an important year for Rocket in which we anticipate progression of the Danon Disease program toward a registrational trial, as well as top-line data from our pivotal LAD-I and Fanconi Anemia clinical trials that move us toward registration. I am growing increasingly confident about our unique gene therapy approach at Rocket focused on addressing specific rare diseases using highly targeted, enabled technologies with a proven track record. Additionally, we anticipate in-house AAV cGMP manufacturing readiness in the second quarter of this year, as we rapidly scale our capabilities and work towards commercial readiness.”

Dr. Shah continued, “I am extremely proud of the positive momentum witnessed in 2021 and our steadfast focus on superior execution. Notably, we announced positive data from our Phase 1 trial in Danon Disease showing clinical, functional and biomarker improvements at one year or beyond, and we initiated treatment in the pediatric patient cohort at the low-dose, with pediatric data anticipated in the third quarter of 2022. In our pivotal Phase 2 trials in LAD-I and Fanconi Anemia, the favorable safety and efficacy observed are very encouraging as we approach top-line data readouts. We were also pleased with the progress of our PKD program demonstrating sustained normal-range hemoglobin through 12-months post-treatment in two adult patients in the Phase 1 trial. As we continue accelerating our current first wave of clinical programs, we are also poised to grow our Wave 2 pipeline based on our core R&D strategy from this year onward. We are excited by the momentum and look forward to continuing on our mission of seeking gene therapy cures for young patients and their families facing such devastating diseases.”

Key Pipeline and Operational Updates

Anticipated 2022 Milestones

RP-A501 for Danon Disease (AAV)

RP-L201 for Leukocyte Adhesion Deficiency-I (LVV)

RP-L102 for Fanconi Anemia (LVV)

RP-L301 for Pyruvate Kinase Deficiency (LVV)

Manufacturing Facility in Cranbury, New Jersey

Upcoming Investor Conferences

Fourth Quarter and Full Year 2021 Financial Results

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Cash position. Cash, cash equivalents and investments as of December 31, 2021, were $388.7 million.

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R&D expenses. Research and development expenses were $32.2 million and $125.5 million for the three and twelve months ended December 31, 2021, compared to $50.1 million and $105.4 million for the three and twelve months ended December 31, 2020. The increase in research and development expenses for the three and twelve months ended December 31, 2021, was primarily driven by an increase in manufacturing and development costs, compensation and benefits expense due to increased R&D headcount and an increase in non-cash stock compensation expense, offset by a decrease in non-cash new research agreements expenses.

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G&A expenses. General and administrative expenses were $12.2 million and $41.8 million for the three and twelve months ended December 31, 2021, compared to $9.1 million and $28.9 million for the three and twelve months ended December 31, 2020. The increase in general and administrative expenses for three and twelve months ended December 31, 2021, was primarily driven by an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount, an increase in office and administrative costs, and an increase in commercial preparation expenses, offset by a decrease in debt conversion expense.

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Net loss. Net loss was $44.2 million and $169.1 million or $0.69 and $2.67 per share (basic and diluted) for the three and twelve months ended December 31, 2021, compared to $60.9 million and $139.7 million or $1.08 and $2.52 per share (basic and diluted) for the three and twelve months ended December 31, 2020.

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Shares outstanding. 64,505,889 shares of common stock were outstanding as of December 31, 2021.

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Cash position. As of December 31, 2021, we had cash, cash equivalents and investments of $388.7 million. Rocket expects such resources will be sufficient to fund its operations into the second half of 2023, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our four clinical programs.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, Rocket’s plans for the advancement of its Danon Disease program following the lifting of the FDA’s clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, filed November 5, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

(amounts in thousands, except share and per share data)

		Three Months Ended December 31,								Twelve Months Ended December 31,
		2021				2020				2021				2020
Operating expenses:
Research and development		$	32,161			$	50,093			$	125,476			$	105,438
General and administrative			12,171				9,144				41,772				28,865
Total operating expenses			44,332				59,237				167,248				134,303
Loss from operations			(44,332	)			(59,237	)			(167,248	)			(134,303	)
Research and development incentives			500				-				1,000				-
Interest expense			(463	)			(1,640	)			(2,977	)			(6,967	)
Interest and other income net			849				235				3,068				2,150
Amortization of premium on investments - net			(801	)			(274	)			(2,912	)			(580	)
Net loss		$	(44,247	)		$	(60,916	)		$	(169,069	)		$	(139,700	)
Net loss per share attributable to common shareholders - basic and diluted		$	(0.69	)		$	(1.08	)		$	(2.67	)		$	(2.52	)
Weighted-average common shares outstanding - basic and diluted			64,470,930				56,284,599				63,235,417				55,380,740

		December 31, 2021				December 31, 2020
Cash, cash equivalents and investments			388,740				482,719
Total assets			497,020				590,824
Total liabilities			42,296				87,305
Total stockholders' equity			454,724				503,519

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Kevin Giordano

Director, Corporate Communications

Investors

Mayur Kasetty, M.D., M.B.A.

Director, Business Development & Operations

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