Rocket Pharmaceuticals Reports Third Quarter 2018 Financial Results and Operational Highlights

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceutical, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, reports financial results for the quarter ended September 30, 2018, and provides an update on the Company’s recent achievements, as well as upcoming milestones.

“We are excited about the ongoing and future developments at Rocket, driven by exceptional execution by both our team and partners. Key recent highlights include the presentation of our promising long-term clinical data from our ongoing Phase 1/2 clinical trial of first-generation RP-L102 in FA at ESGCT 2018 demonstrating durable engraftment, stabilization of previously-declining blood counts and progressive increases in corrected versus non-corrected peripheral blood leukocytes for all patients. In addition, we received clearance of our IND application in FA using our optimized ‘Process B’ of RP-L102 and we plan to start a U.S. clinical trial with no conditioning in early 2019,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket.

Shah continued, “We remain on track with our corporate goal of having potentially four gene therapy programs in the clinic in 2019, including our adeno-associated viral vector (AAV)-based gene therapy program, which we are excited to disclose this quarter. Our AAV candidate treats a monogenic, multi-organ disease that frequently causes mortality in the teenage years. It has a direct on-target mechanism of action, reversing a specific protein defect that is responsible for the full spectrum of the disease, as well a vector with high tropism for the affected tissues. This program will have the largest market opportunity of all of our programs. We look forward to sharing more details on this program ahead.”

Recent Pipeline and Corporate Updates

• FDA Clears IND Application for RP-L102 for FA. Today, the Company announced the clearance of its IND application for RP-L102 for FA utilizing “Process B” which incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell processing. The Company plans to initiate a clinical trial in early 2019 and will not utilize patient conditioning due to the select advantage that is present in FA. The planned clinical trial of “Process B” RP-L102 is expected to enroll approximately 12 FA patients at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, Hospital Niño Jesús/CIEMAT, and other leading centers in the U.S. and in the EU.
• Additional FA Data Presented at the 2018 Annual Congress of European Society of Gene and Cell Therapy (ESGCT). At ESGCT, the Company presented promising preclinical data from the Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO) LVV-based gene therapy programs, as well as updated clinical data from patients that have met the requisite 12-month follow-up time for long-term stem-cell engraftment analysis from the ongoing Phase 1/2 clinical trial of RP-L102 for FA under “Process A”, the first generation, non-optimized process. Patients continue to demonstrate durable engraftment as evidenced by progressively increasing vector copy number (VCN) in peripheral blood for up to 30 months. Patients also demonstrated improved resistance of bone marrow CD34+ cells in the presence mitomycin-C (MMC), and durable increases in peripheral T-lymphocytes chromosomal stability in the presence of diepoxybutane (DEB). MMC- and DEB-resistance are two key diagnostic measures of functional and phenotypic correction in FA. Trends for continued stabilization of previously-declining blood counts, and progressive increases of corrected versus non-corrected peripheral blood leukocytes, also continued.
• Rocket’s Chief Operating Officer and Head of Development, Kinnari Patel, Recognized as a “Rare Disease Champion.” At a special ceremony in September 2018, Kinnari Patel, Pharm.D., MBA, was honored in the PharmaVOICE 100 list of the most inspiring people in the life science industry.

Anticipated Milestones

• Preclinical data and disclosure of the AAV-based gene therapy program (4Q18)
• Investigational Medicinal Product Dossier (IMPD) filing in Spain for the LAD-I program (4Q18) and initiation of clinical trial (2019)
• IMPD filing in Spain for the PKD program (1H19)
• IND filing in the U.S. for the AAV-based program (2019)
• Initiation of FA clinical trial of “Process B” RP-L102 (Early 2019)
• Additional FA patient data (Next 12-18 months)

Upcoming Investor Conferences

• Evercore ISI HealthconX. Rocket is scheduled to present on Wednesday, November 28, at 1:15 p.m. Eastern Time
• Barclays Gene Editing & Gene Therapy Summit. Rocket is scheduled to present on Thursday. November 29, at 11:15 a.m. Eastern Time.

Third Quarter 2018 Financial Results

• Cash position. Cash, cash equivalents and investments as of September 30, 2018, were $159.5 million.
• Debt. Our cash position includes a $52.0 million fully convertible debenture which matures in August of 2021.
• R&D expenses. Research and development expenses were $13.1 million and $29.6 million for the three and nine months ended September 30, 2018, compared to $4.9 million and $10.0 million for the three and nine months ended September 30, 2017.
• G&A expenses. General and administrative expenses were $2.3 million and $15.0 million for the three and nine months ended September 30, 2018, compared to $1.8 million and $3.1 million for the three and nine months ended September 30, 2017.
• Net loss. Net loss was $16.1 million and $47.2 million or $(0.40) and $(1.22) per share (basic and diluted) for the three and nine months ended September 30, 2018, compared to $6.7 million and $12.9 million or $(0.99) and $(1.90) per share (basic and diluted) for the three and nine months ended September 30, 2017.
• Shares outstanding. Approximately 39.8 million shares of common stock were outstanding as of September 30, 2018.

Financial Guidance

• Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of September 30, 2018, will be sufficient to run its operations into 2020.

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 lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for an undisclosed rare pediatric disease. 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, including in collaboration with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO), 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, Rocket’s ability to commence a registrational study in FA within the projected time periods, 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, 2017. 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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