Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Progress

Rhea-AI Summary

Rocket Pharmaceuticals (NASDAQ: RCKT) reported its Q4 and full-year 2024 financial results, highlighting significant progress in its gene therapy pipeline. The company's cash position stands at $372.3 million, providing runway into Q3 2026.

Key developments include ongoing dosing in the Phase 2 pivotal study of RP-A501 for Danon disease, with program updates expected in H1 2025. Long-term data from the Phase 1 study showed sustained efficacy up to five years. The company completed enrollment in the low-dose cohort of RP-A601 for PKP2-ACM, with initial data expected in H1 2025.

Financial results show R&D expenses decreased to $171.2 million from $186.3 million in 2023, while G&A expenses increased to $102.0 million from $73.3 million. Net loss was $258.7 million ($2.73 per share) compared to $245.6 million ($2.92 per share) in 2023. The company raised $182.5 million through a public offering in December 2024.

Positive

• Strong cash position of $372.3M with runway into Q3 2026
• Successful Phase 1 results for RP-A501 showing sustained efficacy up to 5 years
• R&D expenses decreased by $15.1M year-over-year
• Raised $182.5M through public offering
• Large market opportunity with PKP2-ACM affecting 50,000 people in US/Europe

Negative

• Net loss increased to $258.7M from $245.6M year-over-year
• G&A expenses increased by $28.7M (39%) to $102.0M
• FDA requested additional CMC information for KRESLADI approval
• BLA submission delayed to 2025 for KRESLADI

Insights

Biotechnology Financial Analyst

Rocket Pharmaceuticals reported its 2024 year-end results with $372.3 million in cash and investments, providing runway into Q3 2026. The company strengthened its financial position through a $182.5 million public offering in December 2024, which helps offset the $258.7 million net loss ($2.73 per share) for the year.

The company's strategic pivot toward cardiovascular gene therapies is yielding promising clinical validation. Long-term data from RP-A501 for Danon disease published in The New England Journal of Medicine demonstrated sustained therapeutic effects up to five years post-treatment. All evaluable patients showed cardiac LAMP2 expression with ≥10% reductions in left ventricular mass index and improvements in heart failure classification (NYHA Class II to Class I). These results represent potentially transformative outcomes for a fatal condition with no approved therapies.

Rocket has also made progress with its PKP2-ACM program (RP-A601), completing enrollment in the low-dose cohort with data expected in H1 2025. With approximately 50,000 patients affected in the US and Europe, this represents Rocket's largest market opportunity and could become a significant value driver if early data proves promising.

The 39% increase in G&A expenses (to $102.0 million) reflects aggressive commercial preparation, which appears premature given the company has no approved products yet. While the 8.1% decrease in R&D expenses (to $171.2 million) demonstrates improved operational efficiency, investors should monitor whether these cost-saving measures impact pipeline advancement.

Regulatory challenges persist, with KRESLADI (LAD-I) facing continued FDA scrutiny over CMC issues, pushing the complete BLA submission to 2025. Management's statement about "seeking to realize value in our full pipeline in a thoughtful manner" suggests potential partnerships or asset sales for non-cardiovascular programs to preserve capital and focus resources on their most promising assets.

Biotech R&D Expert

Rocket Pharmaceuticals' 2024 results showcase impressive clinical validation for their AAV-based cardiac gene therapy platform. The publication of RP-A501 Danon disease data in The New England Journal of Medicine represents a significant scientific milestone, as cardiac-targeted gene therapy has historically faced delivery challenges due to the heart's post-mitotic nature and physical barriers.

The long-term efficacy data for RP-A501 is particularly compelling from a mechanistic perspective. The sustained cardiac LAMP2 expression demonstrates successful transgene delivery and persistence - a critical hurdle for AAV therapies. The corresponding ≥10% reductions in left ventricular mass index and normalization of heart failure classification from NYHA Class II to Class I represent meaningful clinical translation of this molecular correction. The substantial improvements in cardiac biomarkers (troponin reduced by 84% and BNP by 57%) further validate the therapy's disease-modifying potential.

Rocket's strategic pivot toward cardiovascular gene therapies aligns with areas of highest unmet need and competitive advantage. While Danon disease affects approximately 7,500 patients in the US and Europe, their PKP2-ACM program targets a significantly larger population of 50,000 patients. This strategic focus likely explains why Rocket appears to be deprioritizing their lentiviral programs, with KRESLADI (LAD-I) facing continued regulatory challenges.

The FDA's requests for additional CMC information for KRESLADI raise potential concerns about manufacturing consistency that investors should monitor. Gene therapy manufacturing remains a significant industry-wide challenge, particularly for companies transitioning from clinical to commercial production.

Rocket's approach to cardiac gene therapy differentiates from competitors by using direct intravenous delivery rather than more invasive approaches. Their demonstrated ability to achieve meaningful cardiac transduction with manageable immunosuppression regimens provides a potential platform advantage for their expanding cardiovascular portfolio, including the BAG3-DCM program advancing toward IND.

With multiple data readouts expected in 2025, including updates on the RP-A501 pivotal study and initial RP-A601 Phase 1 data, Rocket is positioned at a critical inflection point that could validate their cardiac gene therapy platform across multiple indications.

Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025

New England Journal of Medicine publication of Phase 1 study of RP-A501 to treat patients with Danon disease

Long-term data from the Phase 1 trial of RP-A501 for Danon disease presented at AHA on safety and meaningful efficacy

Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Initial data expected in the first half of 2025

Regulatory reviews ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia)

Cash, cash equivalents and investments of approximately $372.3M; expected operational runway into the third quarter of 2026

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the fourth quarter and year ended December 31, 2024.

“In 2024, we made strong progress in advancing our gene therapy pipeline, underscored by the New England Journal of Medicine publication of the Phase 1 study of RP-A501 for Danon disease and long-term data presented at AHA showing its safety and meaningful efficacy up to five years. Our momentum continues as we progress with the Phase 2 pivotal trial of RP-A501 and the Phase 1 trial of RP-A601 for PKP2-ACM, and we remain on track to submit the IND for BAG3-DCM in the first half of 2025,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Looking ahead to 2025, we will maintain our focus and resources on advancing our AAV cardiovascular programs while seeking to realize value in our full pipeline in a thoughtful manner, so we deliver the greatest value to our patients and shareholders.”

Recent Pipeline and Operational Updates

• Dosing in the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing.
  • Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.
  • Program update anticipated in the first half of 2025.
    
    
• Long-term data from the Phase 1 study of RP-A501 for Danon disease published in The New England Journal of Medicine and new data presented at the American Heart Association’s 2024 Late-Breaking Science sessions.
  • RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years).
  • Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months.
  • All evaluable patients had reductions in NYHA heart failure (from Class II to Class I; no longer displaying symptoms of heart failure), improvements in KCCQ (median 27-point increase), and substantial improvements in troponin (median reduction 84%) and BNP (median reduction 57%) observed 24-54 months after treatment.
    
    
• Progressed the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).
  • Completed enrollment of all patients in the low dose cohort, and initial data from the Phase 1 study is expected in the first half of 2025.
  • Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket’s pipeline of disclosed programs.
  • Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412.
    
    
• Progressed BAG3-associated dilated cardiomyopathy preclinical program.
  • Nonclinical, IND-enabling studies are ongoing.
  • Submission of the IND is anticipated in the first half of 2025.
    
    
• Progressed RP-L102 investigational gene therapy for Fanconi Anemia (FA).
  • Initiated rolling Biologics License Application (BLA) for RP-L102 and submission of the final module is anticipated in 2025.
  • Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is ongoing.
    
    
• U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADI^TM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).
  • Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I.
  • The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research.
  • Submission of complete BLA to resolve Complete Response Letter anticipated in 2025.
    
    
• Raised net proceeds of $182.5M through public offering.
  • On December 11, 2024, the Company completed a public offering of approximately 15.2 million shares of our common stock at a public offering price of $12.50 per share and pre-funded warrants to purchase 0.4 million shares of common stock at a price of $12.49 per warrant. The gross proceeds to Rocket from the public offering were approximately $194.7 million, net of $12.2 million of offering costs, commissions, legal and other expenses for net proceeds of $182.5 million.

Fourth Quarter and Full Year 2024 Financial Results

• Cash position. Cash, cash equivalents and investments as of December 31, 2024, were $372.3 million.
• R&D expenses. Research and development expenses were $171.2 million for the twelve months ended December 31, 2024, compared to $186.3 million for the twelve months ended December 31, 2023. The decrease of $15.1 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $19.9 million. The decreases were partially offset by increases in the costs for professional fees and consultants of $4.0 million, non-cash stock compensation expense of $1.3 million and depreciation expense of $2.2 million.
• G&A expenses. General and administrative expenses were $102.0 million for the twelve months ended December 31, 2024, compared to $73.3 million for the twelve months ended December 31, 2023. The increase in G&A expenses was primarily driven by increases in commercial preparation related expenses which consisted of commercial strategy, medical affairs, market development and pricing analysis expenses of $17.6 million, legal expenses of $4.8 million, non-cash stock compensation expense of $3.2 million, and compensation and benefit expense of $2.1 million.
• Net loss. Net loss was $258.7 million or $2.73 per share (basic and diluted) for the twelve months ended December 31, 2024, compared to $245.6 million or $2.92 (basic and diluted) for the twelve months ended December 31, 2023.
• Shares outstanding. 106,453,818 shares of common stock were outstanding as of December 31, 2024.

Financial Guidance

• Cash position. As of December 31, 2024, Rocket had cash, cash equivalents and investments of $372.3 million. Rocket expects such resources will be sufficient to fund its operations into the third quarter of 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.

Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.

Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) 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 monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.

Rocket Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “could,” “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. These forward-looking statements include, but are not limited to, statements concerning Rocket’s 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’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, including the timing and outcome of the FDA’s review of the additional CMC information that Rocket will provide in response to the FDA’s request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket’s ability to transition to a commercial stage pharmaceutical company, and Rocket’s expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into the third quarter of 2026. 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 dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, 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, 2024, filed February 27, 2025 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.

		Three Months Ended December 31,								Twelve Months Ended December 31,
			2024				2023				2024				2023
Operating expenses:
Research and development		$	37,357			$	41,744			$	171,244			$	186,342
General and administrative			25,337				21,535				101,961				73,317
Total operating expenses			62,694				63,279				273,205				259,659
Loss from operations			(62,694	)			(63,279	)			(273,205	)			(259,659	)
Interest expense		$	(473	)			(470	)			(1,886	)			(1,875	)
Interest and other income, net			1,617				814				8,267				5,288
Accretion of discount on investments, net		$	1,223				3,275				8,078				10,651
Net loss		$	(60,327	)		$	(59,660	)		$	(258,746	)		$	(245,595	)
Net loss per share - basic and diluted		$	(0.62	)		$	(0.64	)		$	(2.73	)		$	(2.92	)
Weighted-average common shares outstanding - basic and diluted			97,530,032				93,336,541				94,807,773				84,009,004
		December 31, 2024				December 31, 2023
Cash, cash equivalents, and investments		$	372,336			$	407,495
Total assets			527,700				566,341
Total liabilities			64,466				73,767
Total stockholders' equity			463,234				492,574

 

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Investors

Meg Dodge

mdodge@rocketpharma.com

Media

Kevin Giordano

media@rocketpharma.com

Source: Rocket Pharmaceuticals, Inc.

FAQ

What were the key findings from RCKT's RP-A501 Phase 1 study for Danon disease?

The study showed sustained efficacy up to 5 years, with all evaluable patients showing cardiac LAMP2 expression, ≥10% reduction in LV mass index, improved heart failure symptoms, and substantial reductions in troponin (84%) and BNP (57%).

How much cash does Rocket Pharmaceuticals (RCKT) have and what is their runway?

RCKT has $372.3 million in cash, cash equivalents and investments as of December 31, 2024, with expected operational runway into Q3 2026.

What is the market size for RCKT's PKP2-ACM program?

PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket's pipeline.

What was Rocket Pharmaceuticals' (RCKT) net loss for full-year 2024?

RCKT reported a net loss of $258.7 million or $2.73 per share for the twelve months ended December 31, 2024.

How much did RCKT raise in their December 2024 public offering?

The company raised net proceeds of $182.5 million through a public offering of common stock and pre-funded warrants.