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Rocket posts balance sheet with $275.9 million in cash, capitalized into 2022

Rocket Pharmaceuticals has reported a strong balance sheet with $275.9 million in cash, capitalized into 2022.

NEW YORK–(BUSINESS WIRE)–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

Anticipated Milestones

Upcoming Investor Conferences

Third Quarter Financial Results

Financial Guidance

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.

Selected Financial Information
Operating Results:
(amounts in thousands, except share and per share data)
Three Months Ended March 31,

2020

2019

Revenue

$

$

Operating expenses:
Research and development

16,957

15,137

General and administrative

7,163

3,808

Total operating expenses

24,120

18,945

Loss from operations

(24,120

)

(18,945

)

Research and development incentives

250

Interest expense

(1,573

)

(1,604

)

Interest and other income net

967

601

Accretion of discount on investments

62

247

Net loss

$

(24,664

)

$

(19,451

)

Net loss per share attributable to common shareholders – basic and diluted

$

(0.45

)

$

(0.43

)

Weighted-average common shares outstanding – basic and diluted

54,883,120

45,122,815

Selected Balance Sheet Information
(amounts in thousands)
March 31, December 31,

2020

2019

Cash, cash equivalents and investments

275,895

304,115

Total assets

372,751

372,121

Total liabilities

86,700

64,824

Total stockholders’ equity

286,051

307,297

Contacts

Claudine Prowse, Ph.D.

SVP, Strategy & Corporate Development

investors@rocketpharma.com

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