Rocket Pharmaceuticals Reports Second Quarter 2019 Financial Results and Operational Highlights

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 14th 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 17th 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.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's first two clinical programs using 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, and Leukocyte Adhesion Deficiency-I (LAD-I), a serve pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal. Rocket’s first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rocket’s pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO). 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 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 successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, 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 and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, 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, 2018. 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.

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