• A Sarepta Therapeutics focused on Duchenne muscular dystrophy, but using cell and exosome therapies instead of gene therapy.
• A Vertex Pharmaceuticals for Duchenne muscular dystrophy, but with cell and exosome therapies.
• A bluebird bio for Duchenne muscular dystrophy, but focused on cell and exosome therapies.

• CAP-1002: An investigational allogeneic cell therapy derived from cardiosphere-derived cells (CDCs) primarily being developed for Duchenne muscular dystrophy (DMD) associated cardiomyopathy.

Capricor Therapeutics (CAPR) is a clinical-stage biotechnology company focused on developing novel cell-based therapies for rare diseases, primarily Duchenne muscular dystrophy (DMD).

Given its stage of development, Capricor's primary business model involves the research, development, and partnering/licensing of its therapeutic candidates, rather than direct widespread commercial sales of a finished product to individuals or a broad range of companies.

Therefore, its major "customers" are typically other pharmaceutical companies that acquire commercialization rights or enter into development partnerships for specific regions.

Major Customer/Partner:

• Nippon Shinyaku Co., Ltd. (TYO: 4516)

Capricor Therapeutics has an exclusive commercialization and distribution agreement with Nippon Shinyaku Co., Ltd. for its lead investigational product, CAP-1002, in Japan for the treatment of Duchenne muscular dystrophy (DMD).

For other territories, such as the United States and Europe, CAP-1002 is currently in clinical development and not yet approved for commercial sale. Upon potential approval and commercialization in these regions, direct customers would likely include hospitals, specialized clinics, and potentially other pharmaceutical companies through further licensing or distribution agreements, rather than individual patients directly.

• Fujifilm Cellular Dynamics, Inc. (a subsidiary of FUJIFILM Holdings Corporation (FUJIY))

Linda Marbán, Ph.D. Chief Executive Officer and Director

Dr. Marbán is a co-founder of Capricor, Inc., a wholly-owned subsidiary of Capricor Therapeutics, Inc., having been with the company since 2005 and serving as its Chief Executive Officer since 2010. With over 20 years in the biotechnology field, she brings extensive experience in research, product development, and business development. From 2003 to 2009, Dr. Marbán held various senior roles at Excigen, Inc., a gene therapy biotechnology company, where she was responsible for operations and business development and oversaw the development of a biologic pacemaker. She also began her career in academic science at the Cleveland Clinic Foundation and Johns Hopkins University. As CEO, she negotiated the foundational license agreements for Capricor's intellectual property and secured approximately $27 million in non-dilutive grants and a loan award to fund R&D programs and clinical trials.

AJ Bergmann, M.B.A. Chief Financial Officer

Mr. Bergmann serves as the Chief Financial Officer for Capricor Therapeutics. His prior experience includes work at Gettleson, Witzer & O'Connor.

Karen Krasney, J.D. Executive Vice President and General Counsel

Ms. Krasney has served as Capricor's Executive Vice President, Secretary, and General Counsel since 2012. Her career spans over 40 years as general counsel for numerous corporations and private companies across various industries, with a focus on domestic and international corporate and business law and litigation. She has been involved in the medical technology arena since the mid-1990s, including serving as legal counsel for Biosensors International Group Ltd., a multinational medical device company, from 2002, and later as General Counsel in 2006.

Kristi A. H. Elliott, Ph.D. Chief Operating and Science Officer

Dr. Elliott joined Capricor in 2021 and currently leads product development across the company’s therapeutic pipeline, overseeing CMC operations, manufacturing, quality, and analytical and process development. With over 15 years of experience in biotechnology, she is recognized as an innovator in primary cell, stem cell, and exosome-based therapeutics. Before her current role, she was Capricor’s Vice President of Research and Development. Prior to joining Capricor, Dr. Elliott advised joint ventures between Harvest Capital Strategies and Intrexon Corp. and held several leadership roles at Intrexon, including Senior Director of Stem Cell and Exosome Development, where she led cross-functional teams and programs that included an FDA-approved autologous cell therapy.

Mark Awadalla Chief Development Officer

Mr. Awadalla serves as the Chief Development Officer for Capricor Therapeutics. His previous experience includes roles at Celularity, Mustang Bio, and Celgene.

Key Risks to Capricor Therapeutics (CAPR)

Capricor Therapeutics (CAPR) faces significant risks primarily centered around the regulatory approval of its lead therapeutic candidate, Deramiocel, coupled with substantial financial challenges and ongoing legal proceedings.

1. Regulatory Approval Uncertainty: The most prominent risk for Capricor Therapeutics is the uncertainty surrounding the regulatory approval of its cell therapy, Deramiocel, for Duchenne muscular dystrophy (DMD)-related cardiomyopathy. The U.S. Food and Drug Administration (FDA) previously issued a complete response letter (CRL) for Deramiocel's Biologics License Application (BLA), citing insufficient evidence of effectiveness and unresolved Chemistry, Manufacturing, and Controls (CMC) issues. Although Capricor has since announced positive topline results from its Phase 3 HOPE-3 study, the FDA's final stance on this data and its interpretation of efficacy remain critical given the agency's tightened standards and increased scrutiny of high-risk biotech ventures. Delays or a failure to obtain regulatory approval would significantly impede the company's ability to commercialize its primary product candidate.
2. Financial Viability and Commercialization: Capricor Therapeutics operates with zero reported revenue and has incurred significant net losses, primarily due to substantial research and development expenses. The company's financial stability and long-term viability are heavily dependent on the successful regulatory approval and subsequent commercialization of Deramiocel. Without an approved product generating revenue, the company continues to rely on its cash reserves, which, while providing a buffer, are not sufficient to withstand prolonged regulatory rejections or extensive litigation.
3. Legal and Litigation Risks: Capricor Therapeutics is currently facing a class-action lawsuit alleging that its CEO misled investors by presenting an overly optimistic outlook, which led to a substantial decline in stock value following the FDA's CRL for Deramiocel. Such legal battles can be as financially damaging as clinical trial failures. Furthermore, biotechnology and pharmaceutical companies are generally susceptible to securities class action litigation, especially due to stock price volatility associated with critical events like clinical trial outcomes and regulatory decisions.

The emergence and increasing adoption of gene therapies for Duchenne Muscular Dystrophy (DMD) represents a clear threat. Specifically, Sarepta Therapeutics' ELEVIDYS (delandistrogene moxeparvovec-ropr), a one-time gene therapy for DMD, received accelerated FDA approval in June 2023. This therapy, and others in development, aim to address the underlying genetic cause of DMD by delivering a micro-dystrophin gene. In contrast, Capricor's lead product, CAP-1002, is an allogeneic cardiosphere-derived cell therapy focused on improving cardiac and skeletal muscle function and reducing inflammation, requiring repeat dosing. The potential for gene therapies to become a primary or foundational treatment option for a significant portion of the DMD patient population could diminish the market opportunity and clinical positioning for adjunctive or symptom-modifying therapies like CAP-1002.

Duchenne Muscular Dystrophy (DMD)

• The global Duchenne Muscular Dystrophy drugs market was valued at approximately USD 4.1 billion in 2024 and is projected to reach USD 19.46 billion by 2034, growing at a Compound Annual Growth Rate (CAGR) of 16.85% from 2025 to 2034. Other estimates place the global DMD therapeutics market at US$ 2.19 billion in 2024, projected to surge to US$ 6.64 billion by 2033.
• In the United States, the Duchenne Muscular Dystrophy drugs market was valued at approximately USD 1.32 billion in 2024 and is projected to reach USD 6.38 billion by 2034, with a CAGR of 17.06% from 2025 to 2034. The U.S. DMD therapeutics market contributed approximately US$ 950 million in 2024, as part of North America's 43.5% market share. The North American Duchenne Muscular Dystrophy treatment market size is expected to grow from US$ 1.2 billion in 2024 to US$ 5.234 billion by 2033 at a 17.9% CAGR.
• Across the seven major markets (7MM, including U.S., EU4, UK, and Japan), the DMD market is projected to grow from $2.3 billion in 2023 to $5.2 billion in 2033. The U.S. accounted for 84.8% of the total market share in the 7MM in 2023.

Exosome Therapeutics

• The global exosome therapeutics market is estimated to be valued at approximately USD 299.4 million in 2025 and is expected to reach USD 1961.1 million by 2032. Another report values the global exosome therapeutics market size at USD 0.71 billion in 2025, projected to climb to USD 2.21 billion by 2030, with a CAGR of 25.5% during 2025-2030.
• The global exosome diagnostic and therapeutic market size was valued at $224.34 million in 2020 and is projected to reach $2.9 billion by 2030.
• North America leads the exosome market, accounting for 53% of the 2024 revenue. The U.S. accounted for 52.0% of the global exosomes market share.

Cardiomyopathy

• The global cardiomyopathy market size was valued at USD 512.95 million in 2024 and is expected to reach USD 684.38 million by 2032, at a CAGR of 3.67%. Other estimates show the global cardiomyopathies market growing from $3.07 billion in 2021 to $9.63 billion in 2031 (across 68 markets).
• North America dominates the cardiomyopathy market, holding 41.8% of the revenue share in 2024. The U.S. cardiomyopathy market captured the largest revenue share of 79% within North America in 2025.
• For the seven major markets (7MM), the cardiomyopathies market is expected to grow from $1.6 billion to $5.2 billion globally by 2031. The U.S. is a significant contributor to this market, accounting for 94.2% of sales in 2021 and 96.4% in 2031 (reaching $1.5 billion and $5.0 billion, respectively).

Capricor Therapeutics (NASDAQ: CAPR) is poised for significant future revenue growth over the next 2-3 years, driven primarily by the potential commercialization of its lead product candidate, Deramiocel (CAP-1002), for Duchenne Muscular Dystrophy (DMD), alongside the advancement of its innovative exosome platform.

Here are the key expected drivers of future revenue growth:

1. Regulatory Approval and Commercial Launch of Deramiocel (CAP-1002) for Duchenne Muscular Dystrophy: Capricor's primary revenue driver hinges on the anticipated regulatory approval and subsequent commercialization of Deramiocel for DMD-cardiomyopathy. The company submitted a Biologics License Application (BLA) to the FDA, which has been granted priority review with a Prescription Drug User Fee Act (PDUFA) action date set for August 31, 2025. Capricor has entered into an exclusive commercialization and distribution agreement for Deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Upon FDA approval, an $80 million milestone payment from Nippon Shinyaku is expected. The Duchenne muscular dystrophy market is projected to reach approximately $19.46 billion by 2034, indicating a substantial market opportunity. The company is actively preparing for potential commercial launch, including the completion of its commercial manufacturing facility.
2. Expansion of Deramiocel (CAP-1002) Patient Population and Indications: While the initial focus for approval is DMD-cardiomyopathy, Deramiocel has demonstrated broad immunomodulatory and anti-fibrotic actions, preserving cardiac and skeletal muscle function in muscular dystrophies. Clinical studies, such as the HOPE-2 open-label extension, have shown benefits in both ambulatory and non-ambulatory Duchenne patients. This suggests potential for future label expansion to a wider range of DMD patients or additional DMD-related indications, thereby increasing the addressable market and revenue.
3. Advancement of the StealthX™ Exosome Platform into New Therapeutic Areas: Capricor is actively developing its proprietary StealthX™ exosome platform for applications beyond DMD, specifically in vaccinology and the targeted delivery of oligonucleotides, proteins, and small molecule therapeutics. A significant step in this direction is the planned initiation of a Phase 1 clinical trial for a StealthX™ exosome-based vaccine in the third quarter of 2025, conducted by NIAID under Project NextGen. This platform offers a pathway to expand into new therapeutic markets, such as oncology and infectious diseases, providing additional, independent revenue streams in the future.

Share Issuance

• In October 2024, Capricor Therapeutics priced an underwritten public offering of 4,412,000 shares of common stock at $17.00 per share, with an option for underwriters to purchase an additional 661,800 shares.
• The company closed this public offering on October 18, 2024, generating approximately $86.3 million in gross proceeds, including the full exercise of the underwriters' over-allotment option for a total of 5,073,800 common stock shares.
• The net proceeds from the October 2024 offering are intended for continued product candidate development, manufacturing, working capital, and general corporate purposes.

Inbound Investments

• Capricor has a strategic collaboration with Nippon Shinyaku Co., Ltd. for the exclusive commercialization and distribution of its lead product candidate, deramiocel, in the United States and Japan.
• Prior to December 31, 2024, Capricor recognized $40.0 million from upfront and first development milestone payments and an additional $10.0 million from a second development milestone payment as primary revenue from the Nippon Shinyaku U.S. Distribution Agreement.
• Capricor has raised approximately $300 million in equity capital (both privately and publicly) and around $100 million in non-dilutive funding from collaboration partners, including Nippon Shinyaku, and government sources like the NIH and the California Institute for Regenerative Medicine (CIRM).

Capital Expenditures

• Capricor's cash, cash equivalents, and marketable securities totaled approximately $122.8 million as of June 30, 2025, which is expected to cover anticipated expenses and capital requirements into the fourth quarter of 2026.
• The proceeds from the October 2024 public offering are designated for ongoing product candidate development, manufacturing activities, working capital, and general corporate purposes, which implicitly includes capital expenditures related to manufacturing and development.
• Total operating expenses for the second quarter of 2025 were approximately $27.7 million, an increase from approximately $15.6 million in the second quarter of 2024, driven primarily by increased research and development costs associated with late-stage clinical development and manufacturing scale-up.