US Biotech Co. Targets Muscuar Dystrophy with First-In-Class Drug

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1. Ownership and Share Structure Information

The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.

On May 12, 2026, Capricor Therapeutics Inc. (CAPR:NASDAQ) announced financial results for Q1 2026 and provided significant corporate updates. Capricor is a pre-commercial biotech company focused on developing "transformative cell and exosome-based therapeutics for the treatment of rare diseases. Specifically, the company is currently developing Dermiocel (CAP-1002), a drug intended to treat Duchenne Muscular Dystrophy (DMD).

DMD is a severe, X-linked genetic disorder characterized by worsening muscle degeneration that affects the skeletal, respiratory, and cardiac muscles. According to the company, "It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited."

The company's flagship drug, Dermiocel, consists of allogeneic cardiosphere-derived cells (CDCs), which are a rare population of cardiac cells that have shown potent immunomodulatory and anti-fibrotic actions during clinical and preclinical studies. CDCs excrete exosomes, which target macrophages and alter their expression profile to adopt a healing phenotype.

So far, Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA.

Capricor's Q1 financial highlights include:

• Cash Position: Cash, cash equivalents, and marketable securities totaled approximately US$278.6 million as of March 31, 2026, compared to approximately US$318.1 million as of December 31, 2025.
• Revenues: There was no revenue recognized for the first quarter of 2026 or 2025.
• Costs and Expenses: Total operating expenses for Q1 2026 were approximately US$36.8 million, compared to approximately US$25.0 million for Q1 2025.
• Net Loss: The company reported a net loss of approximately US$33.9 million, or US$0.59 per share, for Q1 2026, compared to a net loss of approximately US$24.4 million, or US$0.53 per share, for Q1 2025.
• Financial Outlook: The company believes that, based on the current operating plan and financial resources, its available cash, cash equivalents, and marketable securities will be sufficient to cover anticipated expenses and capital requirements through Q4 2027. This outlook excludes any potential revenue from product sales, the potential monetization of a PRV, if received, or other non-operating sources of capital.

Capricor has packed the first quarter of 2026 with numerous operational highlights. Some include:

• Capricor's Biologics License Application (BLA) for Deramiocel is currently under review by the U.S. Food and Drug Administration.
• Cardiac MRI analyses showed a significant reduction in myocardial fibrosis by late gadolinium enhancement (LGE) versus placebo, a clinically meaningful finding given that fibrosis is cumulative and irreversible. The Duchenne Video Assessment (DVA) "eat 10 bites" measure, a home-based, caregiver-captured assessment of upper limb function and daily living activities, demonstrated statistically significant improvement versus placebo, directly correlated with patient independence and quality of life.
• The company's manufacturing facility in San Diego successfully completed an FDA Pre-License Inspection. The facility is operational and positioned to support initial commercial launch. Second-floor expansion is underway, with the goal of scaling to approximately 2,000–2,500 patients per year, roughly 10,000 doses annually, at full capacity.
• Capricor has secured Michael Maurer as its Chief Commercial Officer (CCO) with direct DMD commercial experience.
• Approximately 90 patients are currently enrolled across Capricor's collective open-label extension studies, with several patients receiving continuous intravenous infusions of Deramiocel for up to five years. Deramiocel has maintained a consistent, well-tolerated safety profile across more than 800 infusions.
• Upon potential approval, Capricor expects to be eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV). PRVs are transferable and can be monetized through sale, representing a meaningful potential source of non-dilutive capital.

Linda Marbán, Ph.D., and CEO of Capricor, said in the release: "Capricor enters this pivotal moment with important regulatory and clinical momentum as we work toward potential approval of Deramiocel for the treatment of Duchenne muscular dystrophy. With the FDA review of our BLA underway and a PDUFA target action date of August 22, 2026, our highest priority is execution: working closely with the agency, preparing for potential launch, and continuing to build the capabilities of a commercial-stage company. We remain focused on ensuring that Deramiocel reaches every eligible patient as quickly as possible…"

"Our thesis is straightforward: a potential first-in-class approval in a defined rare disease population, proprietary in-house manufacturing, a growing pipeline, and a leadership team with the conviction and the capital to execute," continued Dr. Marbán. "We are building something that will create meaningful and durable value for patients, for this company, and for the shareholders who have believed in this mission."

Biotech Sector Demands Funding

In February 2026, Iqvia discussed the global pharma market projection for 2026, noting that total drug usage is expected to surpass four trillion doses daily by 2030. They wrote, "The largest drivers of medicine spending growth through the next five years will continue to be the use in developed markets of innovative therapeutics, especially in oncology, immunology, diabetes, and obesity."

Pharma sector funding fell between 2024 and 2025, according to a March 26, 2026, article for Fierce Biotech by Nick Paul Taylor. He wrote that pharma funding had fallen from 2024 but noted that, "2025 was still the third-best year of the past decade. Similarly, overall funding was well above the pre-pandemic norm and only topped by 2020, 2021, and 2024."

At the same time, J. Edward Moreno of Sherwood News reported contradictory news on January 14, 2026, writing that, "In 2025, announced global biotech deals totaled US$228.4 billion, up from US$132.3 billion in 2024, data from Dealogic shows . . . Just two weeks into 2026, US$9.2 billion in deals have been announced."

According to Moreno, movement is expected in the sector: "As some of the most lucrative drugs lose exclusivity in the next few years, pharmaceutical giants are increasingly shopping around for biotechs to add to their portfolios — and they are more than happy to pay a hefty premium for the right company . . . For some Big Pharma companies, business development spending is now about equal to, or more than, research and development."

This statement could explain the emerging drop in research and development spending at biotech and pharma companies. On March 25, 2026, BioSpace's Annalee Armstrong reported that, "R&D spending at the top 16 pharmaceutical companies declined by 3.6% overall in 2025, as many aggressively cut spending and refocused pipelines."

BCG talked about trends biopharma companies need to be aware of in 2026 in order to stay competitive, saying, "Near term, companies need to continue to innovate to decrease the complexity and cost of these therapies, and governments can find ways to incentivize and pay for them. The longer-term challenge for companies is to factor operational and economic considerations into R&D decision making earlier, ensuring that trial designs match real-world usage, indication sequences match opportunity, and endpoints enable market access."

"Buy" Ratings All Around

Several analysts issued or reiterated ratings and target prices after Capricor announced its Q1 2026 financials. For Oppenheimer, Leland Gershell, MD, Ph.D., gave the company an "Outperform" rating, with a 12-18-month price target of US$54.00 on May 12, 2026. Gershell wrote, "Following recent Phase 3 success for lead candidate Deramiocel in Duchenne Muscular Dystrophy (DMD), we anticipate FDA approval in 3Q. We forecast ~US$1.1B in 2031 sales by commercial partner NS Pharma, of which we model ~US$315M net share to CAPR that year. We believe Deramiocel's growing revenue stream will support advancement of the company's preclinical exosome technology, a cell-free platform with broad therapeutic and vaccine potential that could generate lucrative industry deals over time."

The following day, on May 13, 2026, Joseph Pantiginis, Ph.D., gave the company a "Buy" rating for H.C. Wainwright & Co. With a price target of US$60, Pantiginis said, "Our price target is based on our clinical net present value (NPV) model, which is driven entirely by the company's lead asset, Deramiocel, in DMD patients. There is no contribution from added Deramiocel indications or future potential exosomes candidates, which we believe represent potential upside to our valuation." 

Finally, Boobalan Pachaiyappan, Ph.D., managing director and senior research analyst for Roth, weighed in after Capricor announced Mr. Maurer's addition to the team. He wrote, " We have assessed Capricor Therapeutics using a discounted cash flow (DCF) methodology by employing an 85% POA, 9% discount rate, and a 0% terminal growth rate, yielding a market value of US$2.07 million. Our estimates suggest that Deramiocel could be launched in the U.S. in 2027, with potential loss of exclusivity beginning in 2039."

A Multi-Drug Pipeline

Capricor awaits full facility validation, with FDA approval targeted for the first half of 2027.

The company's StealthX™ platform is advancing several therapeutic programs, with a focus on muscle-targeted delivery of siRNA, proteins, and small molecules. Capricor is also laying the groundwork for the expansion of Deramiocel into Becker muscular dystrophy and engaging regulatory authorities in Europe and Japan.

Ownership & Share Information¹

Capricor Therapeutics Inc. has a market cap of US$1.74 billion, with 57.91 million shares outstanding. The company's 52-week range is US$4.30-US$40.37.

Institutions own 59.72% of shares, while Strategic Investors own 8.54%. Management & Insiders own 0.94% of shares, and the remaining 30.8% of shares are held by Retail.