Rocket Pharmaceuticals Reports First Quarter 2020 Financial Results and Highlights Recent Progress

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, today reports financial results for the quarter that ended March 31, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.

"During the first quarter, we continued to advance five programs across both the AAV and Lenti platforms," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "Notably this quarter, we completed patient treatment in the low dose cohort for RP-A501 in the Phase 1 Danon Disease trial with no dose-limiting toxicities, and are now pleased to report FDA and IDSMC clearance to advance to a higher dose cohort. This has been an important priority for us as Danon Disease affects so many young patients with devastating heart, muscle and CNS manifestations. We also continued to progress our clinical programs for FA, LAD-I, and PKD. Rocket is at an exciting stage of growth, with a rich pipeline of development-stage opportunities and the potential to transform the lives of patients afflicted with rare disease. In the coming months, we look forward to presenting updated FA ?Process A' and LAD-I ?Process B' data at ASGCT, treating our first patient in a higher dose cohort for RP-A501, and bringing our fifth program, IMO, to the clinic."

Dr. Shah continued, "With regard to the COVID-19 pandemic, we are well-positioned overall to continue our progress while we monitor and adjust to this challenging global crisis. We have experienced modest COVID-19-related pauses in patient enrollment and follow-up that are being managed on a patient-by-patient basis, along with some delays in data collection. Nonetheless, we remain on track for providing updates on our lentiviral pipeline. Assuming that COVID-19 associated delays have shorter-term impact, we remain committed to providing data for Danon by the end of the year. Lastly, we continue to actively monitor the pandemic and refine operations to support the complete safety and well-being of our patients, employees and community."

Key Pipelines and Operational Updates

• First cohort of patients completed in Phase 1 trial of RP-A501 for Danon Disease, with U.S. Food and Drug Administration (FDA) and Independent Data Safety Monitoring Committee (IDSMC) clearance to initiate a higher dose cohort. Rocket has treated the first three patients at the low dose of 6.7×10¹³ vector genomes (vg)/kilogram (kg). No investigational product (IP)-related dose-limiting safety concerns have been observed in any of the three patients treated. The low dose cohort demonstrates a favorable safety profile, and the Company has received clearance from the IDSMC and the FDA to move to a higher dose cohort. Patient treatment in this cohort is anticipated in the third quarter, with preliminary Danon data readout in the fourth quarter.
• The Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) clinical studies have continued to progress. Updated data from FANCOLEN-I (utilizing "Process A") will be presented at ASGCT, as will updates from the Phase 1 LAD-I study utilizing "Process B." Updates on FA "Process B" data will be presented in the fourth quarter.
• The Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) facility in Cranbury, New Jersey resumes construction after COVID-19 associated delays. Build-out of the New Jersey facility experienced some construction delays associated with the COVID-19 pandemic, but resumed on May 4, 2020. Occupancy in the new facility is still anticipated in the first half of 2020. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. As previously guided, the first cGMP clinical product release is expected in 2021.
• Recent publications for FA and Danon Disease provide greater insight into Rocket's approach for ongoing clinical trials. In February, the peer-reviewed journal Annals of Hematology published Rocket's comprehensive review of somatic mosaicism in Fanconi Anemia (FA), written in collaboration with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and other international research partners. The article, entitled, "Mosaicism in Fanconi Anemia: Concise review and evaluation of published cases with focus on clinical course of blood count normalization," highlights research describing mosaicism in FA and supports Rocket's approach of treating patients with RP-L102 without any pre-treatment conditioning measures. In March, Science Translational Medicine published positive preclinical data on Rocket's Danon program, demonstrating that vector-mediated transfer of LAMP2B to deficient mice improved heart function and survival. The article, "AAV9.LAMP2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease," underscores the promise of Rocket's AAV-based gene therapy candidate for Danon, RP-A501.
• Rocket recognizes Rare Disease Day with an event at New York City's Carnegie Hall. On February 29, 2020, Rocket hosted its second annual Rare Disease Day event highlighting the theme "I Am Rare, Hear Me Roar." Over 200 members of the rare disease and broader New York City biotech communities heard from patients and their families, learning more about the impact of rare disease and how to become partners in the fight to find new treatment options. The event culminated with the lighting of the Empire State Building in Rare Disease Day colors, honoring all those affected by rare disease.

Anticipated Milestones

• FA (RP-L102)
  • Additional "Process A" data update (2Q)
  • Preliminary "Process B" data (4Q)
• Danon Disease (RP-A501)
  • First patient treatment in higher dose (3Q)
  • Preliminary Phase 1 data (4Q)
• LAD-I (RP-L201)
  • Phase 1 data update on first patient (2Q)
  • Initiate Phase 2 study (4Q)
  • Phase 1 data update (4Q)
• PKD (RP-L301)
  • First patient treatment (3Q, previously 2Q)
  • Preliminary Phase 1 data (4Q)
• IMO (RP-L401)
  • Initiation of clinical study (4Q)

Upcoming Investor Conferences

• BofA Securities 2020 Virtual Health Care Conference, May 13, 2020
• Jefferies Virtual Healthcare Conference, June 2-4, 2020

Third Quarter Financial Results

• Cash position. Cash, cash equivalents and investments as of March 31, 2020, were $275.9 million.
• Debt. Our balance sheet includes $52.0 million of fully convertible notes.
• R&D expenses. Research and development expenses were $17.0 million for the three months ended March 31, 2020, compared to $15.1 million for the three months ended March 31, 2019. The increase was primarily driven by an increase in clinical trial expenses of $1.5 million.
• G&A expenses. General and administrative expenses were $7.2 million for the three months ended March 31, 2020, compared to $3.8 million for the three months ended March 31, 2019. The increase was primarily driven by fees incurred for the convertible notes exchange of $1.6 million, and an increase in compensation expense of $1.3 million due to increased headcount.
• Net loss. Net loss was $24.7 million or $0.45 per share (basic and diluted) for the three months ended March 31, 2020, compared to $19.5 million or $0.43 per share (basic and diluted) for the three months ended March 31, 2019.
• Shares outstanding. 55,126,474 shares of common stock were outstanding as of March 31, 2020.

Financial Guidance

• Cash position. As of March 31, 2020, we had cash, cash equivalents and investments of $275.9 million. Rocket expects such resources will be sufficient to fund its operations into 2022.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare 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. Rocket's pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. 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 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, 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 when clinical trial sites will resume normal business operations, 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, 2019, filed March 6, 2020 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.