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CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a $5.3 billion biotechnology company with strong financial health indicators and over 50% revenue growth in the past year, has received the green light from the U.S. Food and Drug Administration (FDA) to advance its clinical programs targeting various subtypes of limb-girdle muscular dystrophy (LGMD), as per a recent announcement from the biotechnology firm.
The FDA has confirmed that Sarepta can proceed with screening and dosing in its first-in-human clinical study, Study SRP-9005-101 (COMPASS), for LGMD type 2C/R5, also known as gamma-sarcoglycanopathy. This study will evaluate SRP-9005, an investigational gene therapy.
Additionally, the company has completed enrollment and dosing for Study SRP-9004-102 (DISCOVERY), a phase 1 proof-of-concept study for LGMD type 2D/R3, or alpha-sarcoglycanopathy. This study is assessing the safety and expression of the alpha-sarcoglycan protein following treatment with SRP-9004, another investigational gene therapy. According to InvestingPro data, Sarepta maintains a healthy current ratio of 4.2, indicating strong ability to fund its clinical programs.
For LGMD type 2E/R4, also known as beta-sarcoglycanopathy, Sarepta has wrapped up enrollment and dosing in the phase 3 clinical trial, Study SRP-9003-301 (EMERGENE). The primary endpoint of this global study is the biomarker expression of beta-sarcoglycan protein. A pre-Biologics License Application (BLA) meeting has taken place, and the program is eligible for the accelerated approval pathway. The company plans to submit a BLA in the second half of 2025.
Louise Rodino-Klapac, Ph.D., executive vice president and head of research and development at Sarepta, expressed optimism about the potential of gene therapy for treating rare genetic-based diseases and highlighted the progress across the company’s LGMD pipeline. The company’s financial performance supports this optimism, with InvestingPro analysis showing net income of $235 million in the last twelve months. Investors seeking deeper insights can access comprehensive analysis and 12+ additional ProTips through InvestingPro’s detailed research reports.
Limb-girdle muscular dystrophies are a group of genetic disorders causing progressive muscle weakness. With no disease-modifying treatments currently approved for LGMD, the advancement of Sarepta’s gene therapy programs represents a significant step toward addressing this unmet medical need. The company’s robust financial position, with analysts forecasting continued profit growth, positions it well to advance these critical programs.
This article is based on a press release statement from Sarepta Therapeutics.
In other recent news, Sarepta Therapeutics has been the focus of several analyst updates and regulatory developments concerning its gene therapy, ELEVIDYS, for Duchenne muscular dystrophy (DMD). Wells Fargo initiated coverage on Sarepta with an Overweight rating and a price target of $115, highlighting the commercial potential of ELEVIDYS despite recent adverse events. Guggenheim Securities adjusted its price target for Sarepta to $112, maintaining a Buy rating but citing concerns over market uptake and regulatory uncertainties following a patient death. Oppenheimer reduced its price target to $184, retaining an Outperform rating, and noted confidence in the drug’s benefit/risk profile despite the same adverse event.
H.C. Wainwright maintained a Neutral rating with a $75 target, addressing the European Medicines Agency’s temporary clinical hold on ELEVIDYS trials due to safety concerns. Barclays reiterated an Overweight rating with a $209 target, expecting only minor delays from the EU suspension and expressing confidence in the drug’s future prospects. These developments reflect ongoing evaluations of ELEVIDYS’s market potential and safety profile, amidst evolving regulatory landscapes and competitive pressures. Analysts have varied in their revenue projections, with Guggenheim estimating $1.739 billion in ELEVIDYS sales for 2025, aligning with broader market expectations. The company’s strategic decisions, including a share repurchase program, continue to be monitored in light of these recent events.
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