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RCKT Stock Rises as FDA Lifts Clinical Hold on Danon Disease Study
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Key Takeaways
RCKT jumped 28.9% after the FDA lifted the clinical hold on its phase II Danon disease study.
The study will restart at a lower dose, with patients treated sequentially at four-week intervals.
RCKT also plans to use an immunomodulatory regimen similar to its phase I pediatric study.
Shares of Rocket Pharmaceuticals (RCKT - Free Report) surged 28.9% yesterday, after the FDA lifted the clinical hold on the pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.
The clinical hold had been in place since May, when one of the patients in the study experienced an acute systemic infection, an unexpected serious adverse event, which resulted in his untimely death. Following the turn of events, the company voluntarily paused further dosing in the study evaluating RP-A501 in Danon disease.
The FDA noted that RCKT has now satisfactorily addressed the issues outlined in the clinical hold, which resulted in the regulatory body lifting the hold within three months of placing it.
The FDA has now allowed the study to restart with a lower, recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg), given to three patients sequentially with a minimum four-week interval. This dosage aligns with lower ranges that showed efficacy across multiple biomarkers and clinical endpoints in the prior phase I study.
Additionally, Rocket Pharmaceuticals is looking to collaborate with investigators to implement an immunomodulatory regimen aligned with that used in the phase I pediatric cohort.
To date, six patients have been treated in the pivotal phase II study evaluating RP-A501 for treating Danon disease. More updates from the study are likely to be announced after the review of data from the next three patients.
RCKT's Price Performance
Shares of Rocket Pharmaceuticals have plunged 70.2% so far this year against the industry’s rise of 3%.
Image Source: Zacks Investment Research
RCKT's Focus on Gene Therapies for Rare Cardiovascular Conditions
As part of the recent portfolio reprioritization, the company is focusing on the development of its adeno-associated virus (AAV) gene therapies targeting rare cardiovascular indications.
RP-A501 is the most advanced AAV-based candidate in the company’s pipeline.
It can be inferred that the FDA’s swift approval to resume the Danon disease study with improved safety measures marks a significant regulatory milestone for Rocket Pharmaceuticals. It also highlights the potential of RP-A501 and the company’s broader gene therapy pipeline.
Another AAV-based gene therapy candidate is RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy. Rocket Pharmaceuticals also plans to start clinical studies on a new gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy.
In the past 60 days, estimates for Akero Therapeutics’ 2025 loss per share have narrowed from $3.93 to $3.90. Loss per share estimates for 2026 have narrowed from $4.27 to $4.21 during the same period. AKRO stock has surged 72.3% year to date.
Akero Therapeutics’ earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 49.24%.
In the past 60 days, estimates for Amarin’s 2025 loss per share have narrowed from $2.30 to $2.25. Loss per share estimates for 2026 have narrowed from $1.78 to $1.61 during the same period. AMRN stock has rallied 63.4% year to date.
Amarin’s earnings beat estimates in two of the trailing four quarters, met the same once and missed the same on the remaining occasion, the average surprise being 22.97%.
In the past 60 days, estimates for Aligos Therapeutics’ 2025 loss per share have narrowed from $9.71 to $8.24. Loss per share estimates for 2026 have narrowed from $10.45 to $8.74 during the same period. ALGS stock has plunged 77.8% year to date.
Aligos Therapeutics’ earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average negative surprise being 15.45%.
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RCKT Stock Rises as FDA Lifts Clinical Hold on Danon Disease Study
Key Takeaways
Shares of Rocket Pharmaceuticals (RCKT - Free Report) surged 28.9% yesterday, after the FDA lifted the clinical hold on the pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.
The clinical hold had been in place since May, when one of the patients in the study experienced an acute systemic infection, an unexpected serious adverse event, which resulted in his untimely death. Following the turn of events, the company voluntarily paused further dosing in the study evaluating RP-A501 in Danon disease.
The FDA noted that RCKT has now satisfactorily addressed the issues outlined in the clinical hold, which resulted in the regulatory body lifting the hold within three months of placing it.
The FDA has now allowed the study to restart with a lower, recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg), given to three patients sequentially with a minimum four-week interval. This dosage aligns with lower ranges that showed efficacy across multiple biomarkers and clinical endpoints in the prior phase I study.
Additionally, Rocket Pharmaceuticals is looking to collaborate with investigators to implement an immunomodulatory regimen aligned with that used in the phase I pediatric cohort.
To date, six patients have been treated in the pivotal phase II study evaluating RP-A501 for treating Danon disease. More updates from the study are likely to be announced after the review of data from the next three patients.
RCKT's Price Performance
Shares of Rocket Pharmaceuticals have plunged 70.2% so far this year against the industry’s rise of 3%.
Image Source: Zacks Investment Research
RCKT's Focus on Gene Therapies for Rare Cardiovascular Conditions
As part of the recent portfolio reprioritization, the company is focusing on the development of its adeno-associated virus (AAV) gene therapies targeting rare cardiovascular indications.
RP-A501 is the most advanced AAV-based candidate in the company’s pipeline.
It can be inferred that the FDA’s swift approval to resume the Danon disease study with improved safety measures marks a significant regulatory milestone for Rocket Pharmaceuticals. It also highlights the potential of RP-A501 and the company’s broader gene therapy pipeline.
Another AAV-based gene therapy candidate is RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy. Rocket Pharmaceuticals also plans to start clinical studies on a new gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy.
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 Akero Therapeutics (AKRO - Free Report) , Amarin (AMRN - Free Report) and Aligos Therapeutics (ALGS - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Akero Therapeutics’ 2025 loss per share have narrowed from $3.93 to $3.90. Loss per share estimates for 2026 have narrowed from $4.27 to $4.21 during the same period. AKRO stock has surged 72.3% year to date.
Akero Therapeutics’ earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 49.24%.
In the past 60 days, estimates for Amarin’s 2025 loss per share have narrowed from $2.30 to $2.25. Loss per share estimates for 2026 have narrowed from $1.78 to $1.61 during the same period. AMRN stock has rallied 63.4% year to date.
Amarin’s earnings beat estimates in two of the trailing four quarters, met the same once and missed the same on the remaining occasion, the average surprise being 22.97%.
In the past 60 days, estimates for Aligos Therapeutics’ 2025 loss per share have narrowed from $9.71 to $8.24. Loss per share estimates for 2026 have narrowed from $10.45 to $8.74 during the same period. ALGS stock has plunged 77.8% year to date.
Aligos Therapeutics’ earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average negative surprise being 15.45%.