RCKT Stock Up on FDA's RMAT Tag to Gene Therapy for Heart Failure

Rocket Pharmaceuticals RCKT announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational adeno-associated virus (AAV)-based gene therapy, RP-A601, for treating PKP2-arrhythmogenic cardiomyopathy (ACM).

PKP2-arrhythmogenic cardiomyopathy is a genetic heart condition where a PKP2 gene defect weakens heart muscle, causing abnormal rhythms and risk of sudden cardiac death.

The RMAT designation provides the candidate with increased opportunities to meet FDA officials, as well as arrange early meetings to discuss potential surrogate or intermediate endpoints. It opens up early interactions between the FDA and sponsors to facilitate accelerated approval and potential priority review of a product’s biologics license application (BLA).

Shares of Rocket Pharmaceuticals were up 16.8% yesterday following the announcement of the news. However, the stock has plunged 73.5% so far this year compared with the industry’s decline of 0.7%.

The FDA’s RMAT tag was based on positive safety and efficacy data from the phase I study, which evaluated RP-A601 for treating PKP2-ACM. Preliminary data from the ongoing phase I study demonstrated the gene therapy’s encouraging early safety and efficacy.

Also, treatment with RP-A601 was generally safe and well-tolerated.

RCKT Developing Another Gene-Therapy for Heart Diseases

Besides RP-A601, RCKT is developing another AAV-based gene therapy candidate, RP-A701, for treating BAG3-associated dilated cardiomyopathy (BAG3-DCM), a rare and inherited heart condition.

The FDA recently cleared the company’s investigational new drug application for RP-A701.

Rocket Pharmaceuticals is planning to conduct a phase I study evaluating RP-A701, an AAVrh.74-based gene therapy for the treatment of BAG3-DCM.

BAG3-associated dilated cardiomyopathy is a genetic heart disease that causes enlarged, weakened heart muscles, leading to heart failure.

RCKT's Recent Pipeline Setbacks

In May 2025, the company voluntarily paused dosing in the pivotal phase II study evaluating its gene therapy candidate, RP-A501, for treating patients with Danon disease, following a patient death. The FDA placed a clinical hold on the study.

Last June, the FDA issued a complete response letter to the BLA seeking approval for Kresladi to treat LAD-I, a rare genetic disorder.

This was the second time that the FDA had requested additional information from the company on Kresladi’s Chemistry Manufacturing and Controls, which is part of the BLA submission. In February 2024, the FDA extended the review period of the Kresladi BLA by an additional three months.

In light of these recent setbacks, the successful development of its other gene therapy candidates remains a key focus area for the company.

RCKT's Zacks Rank & Stocks to Consider

Rocket Pharmaceuticals currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the biotech sector are Arvinas ARVN, Keros Therapeutics KROS and Akero Therapeutics AKRO, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Arvinas’ 2025 loss per share have improved from $1.60 to $1.51. Loss per share estimates for 2026 have narrowed from $3.28 to $2.98 during the same period. ARVN stock has plunged 59.5% year to date.

Arvinas’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 82.09%.

In the past 60 days, estimates for Keros Therapeutics’ 2025 loss per share have improved from 79 cents to 29 cents. Loss per share estimates for 2026 have narrowed from $4.52 to $4.29 during the same period. KROS stock has plunged 59.5% year to date.

Keros Therapeutics’ earnings beat estimates in one of the trailing four quarters, while missing the same on the remaining three occasions, delivering an average surprise of 9,065.97%.

In the past 60 days, estimates for Akero Therapeutics’ 2025 loss per share have improved from $4.00 to $3.96. Loss per share estimates for 2026 have narrowed from $4.34 to $4.27 during the same period. Year to date, shares of AKRO have surged 95.3%.

Akero Therapeutics’ earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, the average surprise being 48.90%.

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This article originally published on Zacks Investment Research (zacks.com).

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