Mark White, MB.ChB, named Chief Medical Officer following more than 25 years at AstraZeneca; Jonathan Schwartz, M.D., appointed Chief Gene Therapy Officer<\/i><\/p>\n
Became founding consortium member of Rady Children\u2019s Institute for Genetic Medicine\u2019s BeginNGSTM<\/sup> newborn screening program to increase speed of diagnosis for genetic diseases<\/i><\/p>\n Cash, cash equivalents and investments of approximately $360M; expected operational runway into the first half of 2025<\/i><\/p>\n CRANBURY, N.J.–(BUSINESS WIRE<\/a>)–Rocket Pharmaceuticals, Inc.<\/a> (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending March 31, 2023, and updates from the Company\u2019s key pipeline developments, business operations and upcoming milestones.<\/p>\n \n\u201cI am very pleased with our first quarter progress, as we continued to advance our industry leading pipeline of now six programs across both AAV and LV platforms addressing life threatening cardiac and hematologic rare diseases, including two LV programs nearing fillings and four programs either demonstrating robust clinical results or strong preclinical proof of concept, \u201csaid Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. \u201cCorporate and program milestones remain on track, including the planned initiation of the Phase 2 pivotal trial in Danon Disease this second quarter and regulatory filings for LAD-I and Fanconi Anemia this second quarter and fourth quarter, respectively.\u201d<\/p>\n \nDr. Shah continued, \u201cIn addition, we look forward to presenting data across all four of our clinical programs at the upcoming ASGCT meeting this month, as well as our recently announced preclinical program for PKP2-ACM, one of the most prevalent and devastating forms of heart disease impacting nearly 50,000 adults and children in the U.S. and EU. Further, I am proud to announce that we became a Founding Member of BeginNGSTM<\/sup>, a national program that advocates for and facilitates newborn screening for approximately 400 genetic diseases. As we diligently work towards cures for rare diseases, encouraging early genetic screening is essential in providing rare disease patients and their families with an accurate and early diagnosis so they can identify and activate a disease management plan with their healthcare providers as soon as possible.\u201d<\/p>\n \n\u201cLastly, following a recent ATM sale of $17.2M in net proceeds and in-house manufacturing efficiencies, I am pleased to announce that we have extended our cash runway into the first half of 2025. We look forward to leveraging this strong cash position, which will take us through planned launches in 2024,” said Dr. Shah. \u201cTaken together, I am incredibly proud of the tremendous progress we have made in expeditiously developing one of the broadest and deepest pipeline of assets in gene therapy and building integrated capabilities spanning discovery through manufacturing and commercial to help address the unmet needs of patients facing these rare and devastating diseases. We look forward to continuing this progress into the next quarter and year.\u201d<\/p>\n Key Pipeline and Operational Updates<\/b><\/p>\n Details for oral presentations are as follows:<\/b><\/p>\n T<\/b>itle<\/b>: Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous (IV) Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy Title<\/b>: Lentiviral-Mediated Gene Therapy for Fanconi Anemia [Group A]: Results From Global RP-L102 Clinical Trials Title<\/b>: Global Phase 1 Study Results of Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency (PKD) Title<\/b>: Preclinical Efficacy of AAVrh.74-PKP2a (RP-A601): Gene Therapy for PKP2-associated Arrhythmogenic Cardiomyopathy Details for the poster presentation are as follows:<\/b><\/p>\n T<\/b>itle<\/b>: Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1\/2 Study First Quarter Financial Results<\/b><\/p>\n Financial Guidance<\/b><\/p>\n About Rocket Pharmaceuticals, Inc.<\/b><\/p>\n \nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company\u2019s 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 (LV) 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\u2019s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com<\/a>.<\/p>\n Rocket Cautionary Statement Regarding Forward-Looking Statements<\/b><\/p>\n \nVarious statements in this release concerning Rocket\u2019s future expectations, plans and prospects, including without limitation, Rocket\u2019s expectations regarding 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), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket\u2019s ongoing and planned clinical trials, the expected timing and outcome of Rocket\u2019s regulatory interactions and planned submissions, Rocket\u2019s plans for the advancement of its Danon Disease program, including its planned pivotal trial, 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 \u201caim,\u201d \u201canticipate,\u201d “believe,” \u201ccan,\u201d \u201ccontinue,\u201d \u201cdesign,\u201d \u201cestimate,\u201d “expect,” “intend,” \u201cmay,\u201d “plan,” \u201cpotential,\u201d “will give,” “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\u2019s 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\u2019s ongoing trials, 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, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket\u2019s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors\u2019 activities, including decisions as to the timing of competing product launches, pricing and discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket\u2019s Annual Report on Form 10-K for the year ended December 31, 2022, filed February 28, 2023 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.<\/p>\n \n\u00a0<\/p>\n<\/td>\n 2023<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2022<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n46,371<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n30,794<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n15,823<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n11,770<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n62,194<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n42,564<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(62,194<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(42,564<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(468<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(464<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n1,908<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n623<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n2,419<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(577<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(58,335<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(42,982<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(0.73<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.67<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n79,453,519<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n64,509,721<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n 2023<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2022<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n360,041<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n399,670<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n514,757<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n551,807<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n55,881<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n62,121<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n458,876<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n489,686<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \n\u00a0<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20230504005960\/en\/<\/a><\/span><\/p>\n Media Investors KEYWORDS:<\/b> New Jersey United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Health Genetics Research Pharmaceutical Science Biotechnology<\/p>\n\n
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Session<\/b>: Clinical Trials Spotlight Symposium
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Presenter<\/b>: Joseph Rossano, M.D., M.S., FAAP, FACC, Co-Director of the Cardiac Center and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia
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Session date and time<\/b>: Thursday, May 18, 2023, 8:00 a.m. \u2013 9:45 a.m. PT
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Location<\/b>: Concourse Hall 152 & 153
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Presentation number<\/b>: 9<\/p>\n
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Session<\/b>: Hematologic and Immunologic Diseases
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Presenter<\/b>: Agnieszka Czechowicz, M.D., Ph.D., Department of Pediatrics, Division of Hematology\/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine
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Session date and time<\/b>: Thursday, May 18, 2023, 3:45 p.m. \u2013 5:30 p.m. PT
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Location<\/b>: Room 501 ABC
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Presentation number<\/b>: 217<\/p>\n
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Session<\/b>: Hematologic and Immunologic Diseases
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Presenter<\/b>: Ami J. Shah, M.D., Clinical Professor of Pediatrics, Division of Hematology\/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine
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Session date and time<\/b>: Thursday, May 18, 2023, 3:45 p.m. \u2013 5:30 p.m. PT
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Location<\/b>: Room 501 ABC
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Presentation number<\/b>: 218<\/p>\n
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Session<\/b>: Late-Breaking Abstracts 1
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Presenter<\/b>: Christopher Herzog, Ph.D., Associate Vice President, AAV R&D, Rocket Pharma
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Session date and time<\/b>: Friday, May 19, 2023, 8:00 a.m. \u2013 9:45 a.m. PT
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Location<\/b>: Room 515AB
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Presentation number<\/b>: 2<\/p>\n
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Session<\/b>: Friday Poster Session
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Presenter<\/b>: Donald B. Kohn, M.D., Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy Program, University of California, Los Angeles
\n
Session date and time<\/b>: Friday, May 19, 2023, 12:00 p.m. PT
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Location<\/b>: Exhibit Hall\/West Hall A
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Presentation number<\/b>: 1547<\/p>\n\n
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\n \n \n \n \n <\/tr>\n \n \n \n Three Months Ended March 31,<\/b><\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n Operating expenses:<\/td>\n \n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n \n \n \n \n \n \n \n General and administrative<\/td>\n \n \n \n \n \n \n \n \n \n Total operating expenses<\/td>\n \n \n \n \n \n \n \n \n \n Loss from operations<\/td>\n \n \n \n \n \n \n \n \n \n Interest expense<\/td>\n \n \n \n \n \n \n \n \n \n Interest and other income, net<\/td>\n \n \n \n \n \n \n \n \n \n Accretion of discount and amortization of premium on investments, net<\/td>\n \n \n \n \n \n \n \n \n \n Net loss<\/td>\n \n \n \n \n \n \n \n \n \n Net loss per share – basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n Weighted-average common shares outstanding – basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n <\/tr>\n \n \n \n March 31, <\/b><\/td>\n \n December 31, <\/b><\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n Cash, cash equivalents, and investments<\/td>\n \n \n \n \n \n \n \n \n \n Total assets<\/td>\n \n \n \n \n \n \n \n \n \n Total liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Total stockholders’ equity<\/td>\n \n \n \n \n \n \n \n \n <\/p>\n
\n<\/b>
Kevin Giordano
\n
kgiordano@rocketpharma.com
\n<\/a><\/p>\n
\n<\/b>
Brooks Rahmer
\n
investors@rocketpharma.com<\/a><\/p>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20230504005960\/en\/1151092\/3\/Rocket_Pharma.jpg\")
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