Sarepta Therapeutics (SRPT) shares soared 8.7% in the last trading session to close at $79.97. The move was backed by solid volume with far more shares changing hands than in a normal session. This compares to the stock's 30.2% loss over the past four weeks.This rise in share price can be attributed to the recovery in Sarepta’s share price after it tanked earlier this week on reports of a patient’s death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy.This biopha ...

Core Viewpoint - Sarepta Therapeutics' stock experienced a significant decline of over 20% following the report of a patient's death after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy (DMD) [1][4]. Company Overview - The patient who died was reported to have suffered from acute liver failure, a known risk associated with Elevidys. This incident marks the first death linked to the therapy, although the patient also had a recent CMV infection that may have contributed to the liver damage [2][3]. - Sarepta has communicated the event to health authorities and plans to update the prescribing information for Elevidys. The company asserts that the overall benefit-risk profile of the therapy remains positive, with over 800 patients treated [3]. Financial Impact - The decline in Sarepta's stock is attributed to concerns that this incident may deter doctors from prescribing Elevidys, potentially slowing its market adoption. Elevidys accounted for nearly 60% of the company's total revenues in Q4 2024, with projected net product revenues for the year between $2.9 billion and $3.1 billion, two-thirds of which are expected from Elevidys sales [4]. - Year-to-date, Sarepta's shares have decreased by nearly 40%, contrasting with a 6% growth in the industry [5]. Product Portfolio - Sarepta's portfolio includes four approved therapies for DMD, with Elevidys being the first one-shot gene therapy for the condition in the U.S. Since its launch in June 2023, Elevidys has shown significant sales potential, generating approximately $821 million in 2024 compared to $200 million in the previous year [7]. - The company has a partnership with Roche for the development of Elevidys, which has exclusive rights to market the therapy outside the U.S. [8]. - Other therapies in Sarepta's portfolio include Exondys 51, Vyondys 53, and Amondys 45, which collectively have the potential to address nearly one-third of all DMD patients in the U.S. [9].

Sarepta Therapeutics Inc (NASDAQ:SRPT) is down 23.2% to trade at $77.86 at last check, and earlier hit a 52-week low of $75.06. The security is pacing for its worst single-day percentage loss since October 2023 after the gene-editing company revealed a 16-year-old boy died from acute liver failure after receiving its gene therapy for a rare muscular dystrophy. Year to date, the equity is down 37.5%. Beam Therapeutics Inc (NASDAQ:BEAM) is down 6.8% to trade at $22.85 at last glance, brushing off a price-targ ...

About this content About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, ...

On Tuesday, Sarepta Therapeutics, Inc. SRPT stock is sliding. The company shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.The company reported a patient death following treatment with Elevidys, having suffered acute liver failure.Acute liver injury is a known possible side effect of Elevidys and other AAV-mediated gene therapies and is highlighted in the prescribing information.Also Read: Solid Biosciences Outpaces ...

I've been writing for Seeking Alpha since 2017 with a focus on healthcare and technology investments. With a background as a Registered Nurse, I analyze biotech stocks by evaluating clinical data, treatment guidelines, and market dynamics. My MBA provided a foundation of financial theory, allowing me to expand into technology investments, where I focus on identifying key valuation drivers and using DCF modeling for scenario-based forecasts. My thinking is influenced by books such as "Superforecasting" and " ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number : 001-14895 Sarepta Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 93-0797222 (State or other jurisdi ...

Sarepta Therapeutics, Inc. (SRPT) reported fourth-quarter 2024 earnings per share (EPS) of $1.50, which missed the Zacks Consensus Estimate of $1.87. However, the reported figure was significantly higher than the year-ago period’s 47 cents due to higher product sales.The loss included depreciation and amortization costs and stock-based compensation expenses. The adjusted EPS in the quarter was $1.90 compared with 82 cents in the year-ago period.Sarepta recorded total revenues of $658.4 million, up 66% year ...

Financial Data and Key Metrics Changes - For Q4 2024, the company reported total revenues of $658.4 million, an increase of $261.6 million compared to $396.8 million in Q4 2023 [64] - Net product revenue for Q4 2024 was $638 million, growing 75% year-over-year, and full-year net product revenue reached $1.8 billion, representing a 56% increase [14][64] - The company achieved a GAAP net income of $159 million for Q4 2024, compared to $45.7 million in Q4 2023, and a non-GAAP net income of $206 million, up from $86.6 million [66] Business Line Data and Key Metrics Changes - ELEVIDYS sales for Q4 2024 were $384.2 million, a 112% increase from the previous quarter, and total sales since its launch exceeded $1 billion [15][28] - The PMO franchise generated $254 million in Q4 2024, growing approximately 9% compared to Q4 2023, with individual revenues of $137.6 million for EXONDYS 51, $40.2 million for VYONDYS 53, and $76.2 million for AMONDYS 45 [31][32] Market Data and Key Metrics Changes - The company noted that ELEVIDYS has only treated about 5% of the on-label addressable patient population, indicating significant growth potential ahead [33][41] - The PMO franchise continues to perform well, growing organically without price increases, reflecting an increase in patients served in the Duchenne community [30] Company Strategy and Development Direction - The company aims to build on the success of Project Moonshot, focusing on expanding its gene therapy pipeline and achieving multiple potential blockbuster siRNA launches by the end of the decade [9][13] - The strategic collaboration with Arrowhead is expected to diversify the company's future, adding a broad platform of siRNA programs across various diseases [12][13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the 2025 net product revenue guidance of $2.9 billion to $3.1 billion, representing a 70% year-over-year growth [17][42] - The company anticipates continued quarter-over-quarter growth throughout 2025, driven by strong demand for ELEVIDYS and the PMO franchise [81] Other Important Information - The company plans to transition to suspension manufacturing to improve cost of goods sold (COGS) and is preparing for multiple upcoming clinical milestones in 2025 [20][21] - The company has approximately $1.5 billion in cash and investments as of December 31, 2024, and secured a $600 million revolving credit facility to support its strategic goals [71][72] Q&A Session Summary Question: What is the cadence of patient onboarding in Q1? - Management reiterated confidence in guidance for 2025, expecting growth quarter-over-quarter as the launch ramps up [80] Question: What is the prevalence of limb-girdle muscular dystrophy type 2E? - Management indicated that type 2E is an ultra-rare disease, with a 50-50 split between ambulatory and non-ambulatory patients [85][87] Question: Will the upcoming data for FSHD and DM1 be conclusive? - Management stated that the initial single ascending dose (SAD) data will provide important proof of biology, but further data will be needed for definitive conclusions [108][110] Question: What is the status of reimbursement for PMOs in ELEVIDYS treated patients? - Management noted early signs of reimbursement for both therapies, but emphasized that it is still early days to draw broad conclusions [115][117] Question: What is the timeframe for the $500 million share repurchase program? - The program has an 18-month timeframe, and management plans to be opportunistic in deploying capital as cash reserves are built back up [125][126]

Patients can't wait for the next breakthrough in medical research. So neither will we. Fourth Quarter and Full Year 2024 Financial Results Wednesday, February 26, 2025 MELANIE Living with limb-girdle muscular dystrophy ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 1 Forward Looking Statements In order to provide Sarepta's investors with an understanding of its current results and future prospects, forward-looking statements will be made during this conference call. Any statements made by Sarepta th ...