Rocket Pharmaceuticals Reports Second Quarter 2019 Financial Results and Operational Highlights

– First Patient Dosed in RP-A501 Trial for Danon Disease –

– Multi-platform Gene Therapy Pipeline Advances with Four Rocket-sponsored Programs in the Clinic by Year End –

NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, reports financial results for the quarter ended June 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones.

“2019 is a transformative year for Rocket as we transition to a registrational-stage development company focused on the growth of our multi-platform gene therapy pipeline,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “This quarter, we made a lot of progress, and we now have three Rocket-sponsored gene therapy programs in the clinic, including our first AAV gene therapy program. The initiation of our Phase 1 clinical trial for Danon disease marked an important milestone for Rocket as we advanced the first AAV gene therapy for a monogenic heart failure syndrome into the clinic. With each clinical milestone, we are one step closer to rapidly providing potentially curative treatments to patients severely in need.”

Recent Pipeline Developments

• Two patients treated with RP-L102 “Process B” in the Phase 1 trial for Fanconi Anemia (FA) at Stanford University with initial data anticipated in six to nine months. Two patients were treated earlier this year at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, the lead U.S. clinical site. “Process B” incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing. The global Phase 2 study is anticipated to commence in the fourth quarter with alignment from the U.S. Food and Drug Administration (FDA). Ongoing follow-up data on patients from “Process A” and preliminary data on patients from “Process B” will be presented in the fourth quarter.
• Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
• Commencement of Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the registrational trial is underway with the first patient’s cells harvested in the second quarter. The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. Preliminary data will be presented in the fourth quarter.

Anticipated Milestones

• FA (RP-L102)
  • End-of-Phase 1 Meeting with FDA (3Q19)
  • Commencement of E.U. Phase 2 study (3Q19)
  • Commencement of U.S. Phase 2 study (4Q19)
  • Additional data for “Process A” (4Q19)
  • Initial Phase 1 data for “Process B” (4Q19)
• Danon Disease (RP-A501)
  • Phase 1 data (2020)
• LAD-I (RP-L201)
  • Initial Data from RP-L201 (4Q19)
• PKD (RP-L301)
  • IMPD clearance (3Q19)
  • Initiation of Phase 1 study (4Q19)
• IMO (RP-L401)
  • Initiation of clinical study (2020)

Upcoming Investor Conferences

• Leerink’s Spotlight Series on Rare & Genetic Diseases—August 7, 2019 in Boston, M.A.
• Citi’s 14^th Annual Biotech Conference—September 4, 2019 in Boston, M.A.
• Baird’s 2019 Global Healthcare Conference—September 5, 2019 in New York, N.Y.
• Morgan Stanley’s 17^th Annual Global Healthcare Conference—September 11, 2019 in New York, N.Y.
• Oppenheimer’s Fall Summit Focused on Specialty Pharma and Rare Disease Companies—September 23, 2019 in New York, N.Y.
• Ladenburg Thalmann’s 2019 Healthcare Conference—September 24, 2019 in New York, N.Y.

Second Quarter 2019 Financial Results

• Cash position. Cash, cash equivalents and investments as of June 30, 2019, were $257.8 million.
• Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
• R&D expenses. Research and development expenses were $14.0 million for the three months ended June 30, 2019, compared to $10.8 million for the three months ended June 30, 2018. The increase was primarily driven by an increase in clinical trial expenses of $2.1 million as the Phase 1 clinical trials commenced for Fanconi Anemia and Danon disease and an increase in license expense of $1.1 million.
• G&A expenses. General and administrative expenses were $4.4 million for the three months ended June 30, 2019, compared to $4.1 million for the three months ended June 30, 2018.
• Net loss. Net loss was $18.7 million or $0.38 per share (basic and diluted) for the three months ended June 30, 2019, compared to $15.8 million or $0.40 per share (basic and diluted) for the three months ended June 30, 2018.
• Shares outstanding. 50,332,435 shares of common stock were outstanding as of June 30, 2019.

Financial Guidance

• Cash position. As of June 30, 2019, we had cash, cash equivalents and investments of $257.8 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.