Sarepta stock popped Wednesday after the biotech company reported promising results for its gene-silencing tech in two forms of muscular dystrophy.
The studies in facioscapulohumeral muscular dystrophy type 1 and myotonic dystrophy type 1 — or FSHD1 and DM1, respectively — are the first from Sarepta Therapeutics’ (SRPT) siRNA platform. These drugs essentially interrupt the process by which proteins are made.
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A single dose of the drugs, SRP-1001 in FSHD1 and SRP-1003 in DM1 “support reduction, or knockdown, of the target protein or mRNA,” Sarepta said in a news release. “In both studies, the majority of adverse events were mild to moderate and were not dose dependent.”
Sarepta noted both conditions are caused by too many mutant proteins or toxic messenger RNA, or mRNA. For that reason, RNA technology holds “significant promise.”
Sarepta stock jumped 35% to 23.77. Shares leapfrogged both their 50-day and 200-day moving averages.
Sarepta Stock: Rivaling Novartis
Analysts noted the data is still thin and early, making it tough to compare the results to Avidity Biosciences’ rival approach. Novartis (NVS) acquired Avidity for $12 billion in February.
RBC Capital Markets analyst Brian Abrahams says the results in FSHD1 are “looking intriguing at this stage, and suggest viability.” But the results from a multiple ascending dose study in the second half of the year will be key to de-risking the asset.
Notably, patients given SRP-1001 achieved a sevenfold higher concentration of siRNA in their muscles compared to the Avidity/Novartis drug. SRP-1001 also achieved 80% to 90% reductions in DUX4, the aberrant protein at the center of FSHD1. On its face, that appears better than the 53% reduction after treatment with Avidity/Novartis’ del-brax.
On the DM1 side, the 50% reduction in the problematic protein “will be interpreted favorably by many,” Abrahams said in a report. This looks comparable to del-brax.
But he kept his sector perform rating and 18 price target on Sarepta stock.
Leerink Partners analyst Joseph Schwartz says Sarepta’s results “are a bit of a mixed bag.”
“On one hand, it seems like they can achieve impressive muscle concentration, and the DUX-4 reduction looks good, while the DM1 data are very limited and safety remains an open debate,” he said in a client note.
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サレプタ、ノバルティスの19028374656574839201億ドルの筋ジストロフィー買収に挑む中、急騰
Sarepta stock popped Wednesday after the biotech company reported promising results for its gene-silencing tech in two forms of muscular dystrophy.
The studies in facioscapulohumeral muscular dystrophy type 1 and myotonic dystrophy type 1 — or FSHD1 and DM1, respectively — are the first from Sarepta Therapeutics’ (SRPT) siRNA platform. These drugs essentially interrupt the process by which proteins are made.
This video file cannot be played.(Error Code: 102630)
A single dose of the drugs, SRP-1001 in FSHD1 and SRP-1003 in DM1 “support reduction, or knockdown, of the target protein or mRNA,” Sarepta said in a news release. “In both studies, the majority of adverse events were mild to moderate and were not dose dependent.”
Sarepta noted both conditions are caused by too many mutant proteins or toxic messenger RNA, or mRNA. For that reason, RNA technology holds “significant promise.”
Sarepta stock jumped 35% to 23.77. Shares leapfrogged both their 50-day and 200-day moving averages.
Sarepta Stock: Rivaling Novartis
Analysts noted the data is still thin and early, making it tough to compare the results to Avidity Biosciences’ rival approach. Novartis (NVS) acquired Avidity for $12 billion in February.
RBC Capital Markets analyst Brian Abrahams says the results in FSHD1 are “looking intriguing at this stage, and suggest viability.” But the results from a multiple ascending dose study in the second half of the year will be key to de-risking the asset.
Notably, patients given SRP-1001 achieved a sevenfold higher concentration of siRNA in their muscles compared to the Avidity/Novartis drug. SRP-1001 also achieved 80% to 90% reductions in DUX4, the aberrant protein at the center of FSHD1. On its face, that appears better than the 53% reduction after treatment with Avidity/Novartis’ del-brax.
On the DM1 side, the 50% reduction in the problematic protein “will be interpreted favorably by many,” Abrahams said in a report. This looks comparable to del-brax.
But he kept his sector perform rating and 18 price target on Sarepta stock.
Leerink Partners analyst Joseph Schwartz says Sarepta’s results “are a bit of a mixed bag.”
“On one hand, it seems like they can achieve impressive muscle concentration, and the DUX-4 reduction looks good, while the DM1 data are very limited and safety remains an open debate,” he said in a client note.
IBD Newsletters
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Get these newsletters delivered to your inbox & more info about our products & services. Privacy Policy & Terms of Use
Thank You!
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Follow Allison Gatlin on X/Twitter at @AGatlin_IBD.
YOU MAY ALSO LIKE:
How Apogee, Up 20%, Plans To Take On Rival Regeneron’s Eczema Drug
Why Merck Might Have A Fight On Its Hands With Its $6.7 Billion Takeover Of Terns
Stocks To Buy And Watch: Top IPOs, Big And Small Caps, Growth Stocks
MarketSurge: Research, Charts, Data And Coaching All In One Place
Looking For The Next Apple Or Amazon? Start With These S&P 500-Beating Lists