Here are 1-3 brief analogies for Sarepta Therapeutics (SRPT):

• Sarepta Therapeutics is like Vertex Pharmaceuticals for rare neuromuscular diseases, specializing in high-impact treatments for conditions like Duchenne muscular dystrophy.
• Sarepta Therapeutics is like the Biogen of gene and RNA therapies for debilitating neuromuscular disorders, focusing on advanced genetic medicines for specific patient populations.

• EXONDYS 51: A therapeutic designed to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 51 skipping.
• VYONDYS 53: A phosphorodiamidate morpholino oligomer (PMO) indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping.
• AMONDYS 45: A PMO antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping.
• ELEVIDYS: A one-time gene therapy approved for the treatment of ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the *DMD* gene.

Sarepta Therapeutics (SRPT) Major Customers

Sarepta Therapeutics primarily sells its specialized pharmaceutical products to other companies within the healthcare distribution chain, rather than directly to individuals. Their products, which are therapies for rare diseases like Duchenne muscular dystrophy, are distributed through a limited number of specialty distributors and specialty pharmacies.

According to Sarepta's latest financial filings (10-K), the major customer companies that accounted for a significant portion of their net product revenues are:

• AmerisourceBergen Drug Corporation (Symbol: ABC)
• Cardinal Health, Inc. (Symbol: CAH)
• McKesson Corporation (Symbol: MCK)

These three large pharmaceutical wholesalers collectively manage the distribution of Sarepta's products to pharmacies, hospitals, and other healthcare providers.

• Catalent (CTLT)
• Thermo Fisher Scientific (TMO)

Douglas S. Ingram, President & Chief Executive Officer

Douglas S. Ingram was appointed Sarepta's President, Chief Executive Officer, and a member of the Board in June 2017. He previously served as President and Chief Executive Officer of Chase Pharmaceuticals Corporation from December 2015 until November 2016, a clinical-stage biopharmaceutical company focused on neurodegenerative disorders that was acquired by Allergan. Prior to Chase Pharmaceuticals, Mr. Ingram was the President of Allergan, Inc. from July 2013 until its acquisition by Actavis in early 2015. He played an instrumental role in driving Allergan's growth from 1996 through early 2015. His career includes M&A history with the sale of Chase Pharmaceuticals to Allergan and Allergan's acquisition by Actavis.

Ryan H. Wong, Executive Vice President, Chief Financial Officer

Ryan H. Wong was appointed Sarepta's Executive Vice President and Chief Financial Officer in July 2025. Before this role, he was the company's Senior Vice President, Strategic Finance, Treasury and Investor Relations. Mr. Wong joined Sarepta in July 2021 as Vice President, Financial Strategy, Planning & Analysis. Prior to joining Sarepta, he worked at GW Pharmaceuticals plc, most recently as Vice President of Finance, and held various finance roles at Allergan.

Ian M. Estepan, President & Chief Operating Officer

Ian Estepan was appointed President and Chief Operating Officer in July 2025. He previously served as Sarepta's Executive Vice President and Chief Financial Officer from December 2020. Mr. Estepan joined Sarepta in January 2015 and was instrumental in securing $2.5 billion in capital to support the company's growth. Before Sarepta, he spent over 15 years managing a portfolio for Spectra Financial Group and began his career as a healthcare sector analyst at Salomon Smith Barney.

Louise R. Rodino-Klapac, Ph.D., President, R&D and Technical Operations

Louise R. Rodino-Klapac, Ph.D., was named President, R&D and Technical Operations in July 2025. She previously held the title of Executive Vice President and Chief Scientific Officer. Dr. Rodino-Klapac was the former head of the Laboratory for Gene Therapy Research at Nationwide Children's Hospital and co-founded Myonexus Therapeutics, serving as its Chief Scientific Officer before its acquisition by Sarepta in 2019.

Patrick E. Moss, Pharm.D., Executive Vice President, Chief Commercial Officer

Patrick E. Moss, Pharm.D., was named Executive Vice President and Chief Commercial Officer in July 2025. He joined Sarepta in 2015 as director, specialty distribution & payer access. Dr. Moss was instrumental in building the team that oversaw the launch of Sarepta's four therapies for Duchenne, including EXONDYS 51. His prior industry experience includes roles at Incyte Corporation, Vertex Pharmaceuticals, Inc., Amgen Inc., and Eli Lilly & Company.

Sarepta Therapeutics (SRPT) faces several significant business risks, primarily stemming from regulatory and safety concerns surrounding its lead therapies, high product concentration, and ongoing financial challenges.

1. Regulatory and Safety Hurdles for Key Therapies: The most significant risk to Sarepta Therapeutics is the ongoing scrutiny and safety concerns related to its gene therapy, Elevidys, and other Duchenne muscular dystrophy (DMD) drugs. The company has experienced patient deaths linked to Elevidys, which led to a substantial drop in stock price, regulatory holds, and the suspension of guidance. The FDA has also requested a "black box" warning for Elevidys due to acute liver injury and acute liver failure risks, and there was a temporary pause in shipments for non-ambulatory patients and the ENVISION clinical study. The historical mixed feedback from the FDA on Sarepta's products highlights persistent regulatory uncertainty, which could delay approvals or limit the scope of product labels.
2. High Product Concentration and Reliance on DMD Portfolio: Sarepta's business is heavily concentrated on its Duchenne muscular dystrophy (DMD) portfolio, particularly Elevidys, which generated 43% of the company's total revenues in the past year. This overwhelming dependence on a single revenue source means that any setbacks or regulatory challenges with these therapies can have a disproportionately negative impact on the company's overall revenue prospects and financial stability.
3. Financial Challenges and Volatility: Sarepta continues to face significant financial hurdles, operating with unprofitability, negative operating cash flow, and negative net income margins. The capital-intensive nature of biotechnology research and development contributes to these challenges. The company also has substantial debt, including $1.15 billion in convertible senior notes maturing in September 2027, and a concerning debt-to-equity ratio. While Sarepta has undertaken a major restructuring, including workforce reductions and pipeline reprioritization, to achieve cost savings and address financial pressures, the company's stock price remains volatile and subject to significant fluctuations.

The potential approval and commercialization of Pfizer's Duchenne muscular dystrophy (DMD) gene therapy (PF-0693992) represents a clear emerging threat. If successful, Pfizer's therapy would directly compete with Sarepta's recently approved Elevidys, potentially fragmenting the market or offering a competing option with its own efficacy and safety profile.

Sarepta Therapeutics (SRPT) focuses on precision genetic medicine, with its main commercial products addressing Duchenne Muscular Dystrophy (DMD) and pipeline therapies targeting Limb-girdle Muscular Dystrophy (LGMD).

Duchenne Muscular Dystrophy (DMD)

Sarepta's commercial portfolio for DMD includes four FDA-approved therapies: Elevidys (a gene therapy), Exondys 51, Vyondys 53, and Amondys 45 (RNA-based therapies).

• The global Duchenne muscular dystrophy drug market was valued at approximately $2.5 billion in 2024 and is projected to reach between $5.9 billion and $19.5 billion by 2033-2034. Other estimates place the global market at $4.1 billion in 2024, growing to $19.46 billion by 2034, or $2.2 billion in 2023, expected to reach $7.4 billion by 2034.
• For the United States, the Duchenne muscular dystrophy drugs market size was $1.32 billion in 2024 and is projected to reach approximately $6.38 billion by 2034. North America held the largest share of the DMD drugs market in 2024, at 46%, with the U.S. contributing $1.3 billion to the North American market in 2023.

Limb-girdle Muscular Dystrophy (LGMD)

Sarepta has a gene therapy candidate, SRP-9003 (bidridistrogene xeboparvovec), in clinical trials for LGMD type 2E/R4.

• The global Limb-girdle muscular dystrophy market was valued at $1.36 billion in 2024 and is expected to grow to $1.96 billion by 2032. Another report indicates the global market reached $1.3 billion in 2023 and is anticipated to reach $2.4 billion by 2031.
• The 7 major Limb-girdle muscular dystrophy markets were valued at $612.1 million in 2024 and are projected to reach $865.0 million by 2035.
• North America dominated the global LGMD market with a 41.3% revenue share in 2024. The U.S. market for limb-girdle muscular dystrophy accounted for 82.4% of North America's revenue share in 2024.

Sarepta Therapeutics (SRPT) is anticipated to drive future revenue growth over the next 2-3 years through several key initiatives:

1. Continued Growth of ELEVIDYS in Ambulatory Patients and Potential Re-expansion to Non-Ambulatory Patients: ELEVIDYS, Sarepta's gene therapy for Duchenne muscular dystrophy (DMD), is expected to be a significant revenue contributor. Despite a recent decision to suspend shipments to non-ambulatory patients and potential upcoming labeling discussions, Sarepta anticipates at least $500 million in annual revenue from ELEVIDYS infusions in the ambulant population for the full year 2025. The therapy received expanded U.S. approval in June 2024 to treat Duchenne muscular dystrophy patients aged four and above, with its accelerated approval for ambulatory patients converted to a full approval, significantly broadening its addressable market. Sarepta is also preparing for a prophylactic-sirolimus non-ambulatory study for ELEVIDYS, with initial data expected in the first half of 2026, which could potentially lead to re-expansion into this patient population.
2. Sustained Performance and Pursuit of Traditional Regulatory Approval for the PMO Franchise: Sarepta's phosphorodiamidate morpholino oligomer (PMO) therapies, including EXONDYS 51, VYONDYS 53, and AMONDYS 45, continue to be a core revenue generator. While the ESSENCE confirmatory study for AMONDYS 45 and VYONDYS 53 did not achieve statistical significance on its primary endpoint, the company reported positive and encouraging trends, particularly when excluding COVID-impacted data. Sarepta plans to engage with the FDA to discuss a path to traditional approval for these therapies based on these trends and significant multi-year real-world evidence. The company projects approximately $900 million in revenue from its PMO therapies in 2025.
3. Advancement and Potential Commercialization of New siRNA Therapies: Sarepta's pipeline includes several promising siRNA programs, acquired through a deal with Arrowhead Pharmaceuticals. Initial data from Phase 1/2 studies for SRP-1001 in facioscapulohumeral muscular dystrophy (FSHD) and SRP-1003 in myotonic dystrophy type 1 (DM1) are expected in early 2026. The successful development and potential launch of these new product candidates represent opportunities to expand into new therapeutic areas beyond Duchenne muscular dystrophy.
4. Regulatory Approval and Commercialization of SRP-9003 for Limb-Girdle Muscular Dystrophy Type 2E/R4: Sarepta is progressing with SRP-9003, a gene therapy candidate for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4). A regulatory filing with the FDA for this therapy is anticipated before the end of 2025. A successful approval and launch would mark Sarepta's entry into a new indication within rare muscular dystrophies, further diversifying its revenue streams.

Share Repurchases

• In November 2024, Sarepta Therapeutics' Board of Directors approved a share repurchase program authorizing up to $500.0 million of outstanding common stock over 18 months.
• During the three and six months ended June 30, 2025, the company repurchased 650,876 shares for a total cost of $25.3 million under this program.
• As of June 30, 2025, $475.0 million remained authorized for future share repurchases.

Share Issuance

• In February 2020, Sarepta issued and sold 2,522,227 shares of common stock to Roche Finance as part of a collaboration agreement.
• The company proposed to issue and sell an aggregate of 6,172,840 shares of common stock in an offering in October 2021.
• The weighted average number of shares of common stock outstanding for diluted earnings per share increased from 88,186,000 for the three months ended March 31, 2021, to 99,114,000 for the three months ended March 31, 2022, indicating an increase in shares.

Inbound Investments

• Sarepta recognized $175.5 million in collaboration revenue year-to-date and a $63.5 million milestone payment linked to ELEVIDYS approval in Japan, stemming from its collaboration with Roche.
• The collaboration arrangement with F. Hoffman-La Roche Ltd consistently generated significant collaboration and other revenues, amounting to $89.2 million and $89.5 million for the twelve months ended December 31, 2022, and 2021, respectively.

Outbound Investments

• In February 2025, Sarepta Therapeutics Investments, Inc. purchased 11,926,301 shares of Arrowhead common stock for $325.0 million in a private placement, related to an exclusive global licensing and collaboration agreement.
• In March 2021, Sarepta made an investment in StrideBio's Series B financing round.
• As of December 31, 2021, Sarepta held an equity investment of $2.5 million in Lysogene, owning 1,140,728 shares of its common stock.

Capital Expenditures

• Sarepta's capital expenditures are primarily focused on increasing manufacturing capabilities and expanding real estate facilities to support growth.
• Purchases of property and equipment, a key component of capital expenditures, totaled $61.6 million for the six months ended June 30, 2024, and $27.4 million for the six months ended June 30, 2023.
• The company's investments in plant, property, and equipment have shown a steady increase, reaching $361 million net as of Q1 2025, up from $181 million at Q3 2022.