Rocket Pharmaceuticals Reports Second Quarter 2022 Financial Results and Highlights Recent Progress

— Cash, cash equivalents and investments of $321.4M; expected operational runway into first half of 2024 —

CRANBURY, N.J. – August 8, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending June 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

“Rocket continues on an excellent trajectory following a strong and highly productive quarter that featured positive results across all four clinical programs, concrete steps taken toward manufacturing readiness and leadership, and filing preparedness for our first two gene therapies,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “Positive safety data from our Phase 1 study for Danon Disease demonstrated RP-A501 was well-tolerated in both pediatric patients. We now look forward to presenting early efficacy data from the pediatric cohort with three to six months of follow-up in late Q3. If early signals of efficacy and ongoing tolerability in the pediatric cohort are demonstrated along with evidence of longer-term safety and efficacy in the adult cohort, we expect to begin Phase 2 pivotal trial planning activities in Q4, including FDA alignment on study design and endpoints.”

Dr. Shah continued, “In parallel, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal trial in Danon Disease. To further strengthen our manufacturing and commercial capabilities, we appointed Mayo Pujols, one of the most seasoned cell and gene therapy technical operations and manufacturing leaders in the industry, as our Chief Technical Officer.”

“This quarter, we also shared positive top-line data from our pivotal Phase 2 trial for severe LAD-I showing that RP-L201 was well-tolerated and associated with 100% overall survival at one year,” said Dr. Shah. “We have initiated work towards regulatory filings planned for the first half of 2023. In addition, based on achievement of the primary endpoint in our pivotal Phase 2 study for Fanconi Anemia, we have initiated FDA dialogue around BLA planning activities."

“These steps deliver on our effort to best leverage our strong cash position to create value as we embark on transitioning from a clinical to a commercial-stage company. Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024,” concluded Dr. Shah. “Taken together, our positive data updates and steady progress this quarter continue to motivate us to push the boundaries of science and deliver on our mission to seek gene therapy cures for patients and families facing devastating, life-threatening diseases.”

Key Pipeline and Operational Updates

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Second Quarter Financial Results

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Cash position. Cash, cash equivalents and investments as of June 30, 2022, were $321.4 million.

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G&A expenses. General and administrative expenses were $12.9 million for the three months ended June 30, 2022, compared to $9.5 million for the three months ended June 30, 2021. The increase in general and administrative expenses was primarily driven by an increase in commercial preparation expenses, an increase in compensation and benefits expense due to increased G&A headcount, and an increase in legal expenses.

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Cash position. As of June 30, 2022, the Company had cash, cash equivalents and investments of $321.4 million. As of June 30, 2022, the Company sold 1.3 million shares of common stock for net proceeds of $17.3 million under its at-the-market facility. With the at-the-market facility proceeds, the Company expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2024, 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 as well as future pipeline 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, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program 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 Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q.  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.

Media

Kevin Giordano

Director, Corporate Communications

Investors

Jessie Yeung, M.B.A.

Vice President, Investor Relations and Corporate Finance

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