Rocket Pharmaceuticals, Inc. today disclosed a corporate restructuring strategy to concentrate on its AAV cardiovascular platform and substantial regulatory focus on its lead gene therapy, KRESLADI™. The restructuring is designed to maximize near-term value, maximize capital efficiency, and position for long-term growth, the company explained.

The plan consists of a workforce reduction of approximately 30%, which is expected to reduce Rocket's 12-month cash burn by approximately 25%. Through such operational efficiency, Rocket is assured that the current cash balances will carry operations through to the second quarter of 2027.

The reorganization places highest priority on clinical programs in AAV cardiovascular platform, such as those for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM).

Rocket is also giving highest priority to filing and submitting regulatory responses to KRESLADI™ (formerly RP-L201), its severe leukocyte adhesion deficiency-I (LAD-I) therapy, following a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA).

“This redirection allows us to concentrate on our highest-potential cardiovascular programs while ensuring that we reach significant regulatory milestones," said Dr. Gaurav Shah, CEO of Rocket Pharmaceuticals.

"Our AAV cardiovascular platform has shown incredible promise to treat serious heart diseases of high unmet need, and we believe this prioritization places Rocket both on near-term clinical success and long-term sustainable value creation."

Focus on AAV Cardiovascular Platform

Rocket's pipeline for AAV gene therapy for cardiovascular diseases is aimed at monogenic heart diseases, or gene-defined heart failure. These include:

·         Danon Disease: An autosomal dominant hypertrophic cardiomyopathy that typically leads to premature heart failure.

·         PKP2-ACM: A potentially fatal arrhythmogenic cardiomyopathy that can cause sudden cardiac death.

·         BAG3-DCM: A dilated cardiomyopathy caused by mutations of the BAG3 gene, resulting in the enlargement and failure of the heart.

Collectively, these conditions affect over 100,000 patients in the U.S. and Europe. Rocket plans to administer single-infusion, curative therapies through the use of its AAV cardiovascular platform supporting precision-guided gene correction for each subtype of disease.

Regulatory Milestones for KRESLADI™ in LAD-I

Aside from the cardiovascular emphasis, Rocket is pursuing regulatory approval of KRESLADI™, a gene therapy for advanced LAD-I. This is a rare immune deficiency that causes repeated life-threatening infections in children.

Rocket is intending to submit completed responses to the FDA's CRL and may be eligible for a Priority Review Voucher (PRV) upon approval.

While proceeds from a theoretical PRV are not part of Rocket's existing financial estimates, a voucher would be substantial non-dilutive capital and commercial value.

Other Program Delays: An Indicator of Strategic Priorities

As part of this pipeline deferment, Rocket announced other programs with anticipated delays, including:

·         RP-L102 for Fanconi Anemia (FA): No longer expected approval by the FDA in 2026.

·         RP-L301 in Pyruvate Kinase Deficiency (PKD): Aside from that, development timelines have not been defined.

These realignments are indicative of a managed capital allocation strategy, focusing on investment in those most immediate areas of return and clinical advantage.

Financial Projections and Way Forward

The firm expects its lower operating expenses and frugal management of capital to extend its cash runway to Q2 2027. This does not consider any future proceeds from future PRVs or other future funding opportunities, and hence a conservative and prudent financial strategy.

Rocket will provide further details in its second-quarter 2025 earnings report this year, such as clinical timelines, regulatory submissions, and possible strategic collaborations.