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RCKT Aligns With FDA on Completing Phase II Study in Danon Disease
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Key Takeaways
RCKT will enroll nine more male patients to complete the 12-patient pivotal efficacy population.
The study will use a recalibrated RP-A501 dose and optimized immunomodulatory regimen.
Rocket Pharmaceuticals expects to finish dosing the remaining nine patients by mid-2027.
Rocket Pharmaceuticals (RCKT - Free Report) announced that it has reached alignment with the FDA on certain key elements of the pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.
The company has now reached alignment with the FDA on continued enrollment and dosing and key elements, including the recalibrated dose, pivotal patient population and 12-month co-primary endpoints, of the phase II study.
Shares of the company rose in pre-market trading on Wednesday following the announcement of the news.
More on RCKT’s Danon Disease Study
The FDA confirmed that the pivotal efficacy population will comprise 12 male patients treated with RP-A501 at the recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg), using commercial-grade product. Notably, the first three patients treated under the modified protocol will count toward the 12-patient pivotal population. The company will now enroll and treat nine more patients to complete the 12-patient pivotal population.
Rocket Pharmaceuticals expects to finish dosing these remaining nine patients by mid-2027.
Based on the available safety data, the FDA confirmed that enrollment and dosing can continue under the modified protocol at the recalibrated dose.
The pivotal phase II study will use the previously established 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index. Per the company, these endpoints are intended to support a potential accelerated approval pathway for RP-A501 in Danon disease.
The modified protocol includes the recalibrated dose of RP-A501 and an optimized immunomodulatory regimen comprising rituximab, sirolimus and corticosteroids, along with enhanced eligibility criteria, safety monitoring and risk-mitigation measures.
The company plans to share a comprehensive update on the Danon disease program at a virtual investor webinar next month.
Year to date, shares of Rocket Pharmaceuticals have lost 2.8% against the industry’s rise of 7.8%.
Image Source: Zacks Investment Research
RCKT's Gene Therapies in Rare Cardiovascular Conditions
RP-A501 is the most advanced AAV-based candidate in the company’s pipeline.
Last month, the company announced positive initial safety findings from the first three patients treated with RP-A501 under a modified protocol for a phase II study in Danon disease. The patients received a recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg) with a refined immunomodulatory regimen, and no cases of thrombotic microangiopathy, capillary leak syndrome or other significant safety concerns were observed.
Rocket Pharmaceuticals’ recent development activities highlight the potential of RP-A501 and the company’s broader gene therapy pipeline.
The company is also developing RP-A601 in an early-stage study for treating arrhythmogenic cardiomyopathy. The company also plans to start initial patient dosing in a phase I study evaluating another gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy in the second half of 2026.
Over the past 60 days, Amarin’s 2026 loss per share estimates have narrowed from 65 cents to 39 cents. During the same time, 2027 loss per share has narrowed from 51 cents to 50 cents. AMRN shares have lost 0.2% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed on the remaining occasion, delivering an average surprise of 62.27%.
Over the past 60 days, estimates for Anika Therapeutics’ 2026 bottom line have moved from a loss of 12 cents per share to earnings of $1.05 per share. Earnings estimates for 2027 have increased from 16 cents to 95 cents during the same time. ANIK’s shares have surged 117.6% year to date.
Anika Therapeutics’ earnings beat estimates in each of the trailing three quarters, with the average surprise being 950.00%.
Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.61 during the same time. RGEN’s shares have risen 6.7% year to date.
Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.
View the Zacks #1 Rank List
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RCKT Aligns With FDA on Completing Phase II Study in Danon Disease
Key Takeaways
Rocket Pharmaceuticals (RCKT - Free Report) announced that it has reached alignment with the FDA on certain key elements of the pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.
The company has now reached alignment with the FDA on continued enrollment and dosing and key elements, including the recalibrated dose, pivotal patient population and 12-month co-primary endpoints, of the phase II study.
Shares of the company rose in pre-market trading on Wednesday following the announcement of the news.
More on RCKT’s Danon Disease Study
The FDA confirmed that the pivotal efficacy population will comprise 12 male patients treated with RP-A501 at the recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg), using commercial-grade product. Notably, the first three patients treated under the modified protocol will count toward the 12-patient pivotal population. The company will now enroll and treat nine more patients to complete the 12-patient pivotal population.
Rocket Pharmaceuticals expects to finish dosing these remaining nine patients by mid-2027.
Based on the available safety data, the FDA confirmed that enrollment and dosing can continue under the modified protocol at the recalibrated dose.
The pivotal phase II study will use the previously established 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index. Per the company, these endpoints are intended to support a potential accelerated approval pathway for RP-A501 in Danon disease.
The modified protocol includes the recalibrated dose of RP-A501 and an optimized immunomodulatory regimen comprising rituximab, sirolimus and corticosteroids, along with enhanced eligibility criteria, safety monitoring and risk-mitigation measures.
The company plans to share a comprehensive update on the Danon disease program at a virtual investor webinar next month.
Year to date, shares of Rocket Pharmaceuticals have lost 2.8% against the industry’s rise of 7.8%.
Image Source: Zacks Investment Research
RCKT's Gene Therapies in Rare Cardiovascular Conditions
RP-A501 is the most advanced AAV-based candidate in the company’s pipeline.
Last month, the company announced positive initial safety findings from the first three patients treated with RP-A501 under a modified protocol for a phase II study in Danon disease. The patients received a recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg) with a refined immunomodulatory regimen, and no cases of thrombotic microangiopathy, capillary leak syndrome or other significant safety concerns were observed.
Rocket Pharmaceuticals’ recent development activities highlight the potential of RP-A501 and the company’s broader gene therapy pipeline.
The company is also developing RP-A601 in an early-stage study for treating arrhythmogenic cardiomyopathy. The company also plans to start initial patient dosing in a phase I study evaluating another gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy in the second half of 2026.
Rocket Pharmaceuticals, Inc. Price
Rocket Pharmaceuticals, Inc. price | Rocket Pharmaceuticals, Inc. Quote
RCKT’s Zacks Rank & Stocks to Consider
Rocket Pharmaceuticals currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amarin (AMRN - Free Report) , Anika Therapeutics (ANIK - Free Report) and Repligen (RGEN - Free Report) , each currently holding a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Over the past 60 days, Amarin’s 2026 loss per share estimates have narrowed from 65 cents to 39 cents. During the same time, 2027 loss per share has narrowed from 51 cents to 50 cents. AMRN shares have lost 0.2% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed on the remaining occasion, delivering an average surprise of 62.27%.
Over the past 60 days, estimates for Anika Therapeutics’ 2026 bottom line have moved from a loss of 12 cents per share to earnings of $1.05 per share. Earnings estimates for 2027 have increased from 16 cents to 95 cents during the same time. ANIK’s shares have surged 117.6% year to date.
Anika Therapeutics’ earnings beat estimates in each of the trailing three quarters, with the average surprise being 950.00%.
Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.61 during the same time. RGEN’s shares have risen 6.7% year to date.
Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.