Shares of Capricor Therapeutics CAPR gained 8.4% on Thursday following the completion of the submission of a biologics license application (BLA) to the FDA seeking full approval for deramiocel to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. CAPR’s lead product candidate and only clinical pipeline asset, deramiocel, is an investigational, allogeneic cardiac-derived cell therapy. A final decision of the FDA is anticipated in the second half of 2024.
Capricor initiated its rolling submission process for the deramiocel BLA in October 2024, based on a successful pre-BLA meeting with the FDA. The completion of the submission process has also triggered a $10 million milestone payment from its distribution partner, Nippon Shinyaku, under the terms of the companies’ U.S. Commercialization and Distribution Agreement. The impending influx of cash is set to strengthen CAPR’s cash position.
The company has requested the FDA to grant priority review to the BLA. A filing accepted under the regulatory body’s Priority Review pathway reduces the review period to six months from the standard 10 months. This status is given to treatments that the FDA believes can significantly improve existing options or provide solutions where no approved therapy exists.
In the past three months, shares of Capricor have plunged 30.9% compared with the industry’s 2.7% decline.
Zacks Investment Research
Image Source: Zacks Investment Research
More on CAPR’s BLA Submission for Deramiocelto Treat DMD
Capricor’s BLA for deramiocel to treat DMD cardiomyopathy is supported by its cardiac data from its phase II HOPE-2 and HOPE-2 Open Label Extension (OLE) studies compared to natural history data.
In June 2024, the company announced favorable three-year results from the HOPE-2 OLE study of deramiocel for the DMD indication. Per the data readout, a statistically significant reduction in the decline of upper limb function (PUL 2.0) and stabilization of left ventricular ejection fraction among deramiocel-treated patients was observed over three years. This contrasts sharply with the expected decline observed in untreated DMD patients, suggesting that deramiocel can potentially slow disease progression and improve quality of life.
Deramiocel also demonstrated a well-tolerated safety profile throughout the study. Deramiocel enjoys the Orphan Drug designation in the United States and the EU for the treatment of DMD. Additionally, subject to the FDA approval of the deramiocel BLA for DMD cardiomyopathy, Capricor would be eligible to receive a Priority Review Voucher based on its previous receipt of a rare pediatric disease designation.
CAPR’s Next Steps in the Deramiocel Developmental Program
Capricor plans to later expand the label of deramiocel to include the treatment of skeletal muscle myopathy. To achieve the same, CAPR expects to report clinical data on skeletal muscle myopathy by combining Cohorts A and B from the phase III HOPE-3 study as part of a post-approval study.
Additionally, the HOPE-3 study results may support marketing approvals outside the United States, if needed. Capricor has already started regulatory processes in the EU and Japan and will collaborate with health authorities to streamline the approval process for deramiocel in these regions.
DMD is a serious genetic disorder showing progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. Per Capricor, DMD occurs in approximately one in every 3,500 male births and the patient population is estimated to be approximately 15,000-20,000 alone in the United States. Present treatment options are limited and there is no cure for the disease.
According to a Mordor Intelligence report, the DMD treatment market size is estimated at $4.07 billion in 2025 and is expected to reach $9.78 billion by 2030, witnessing a CAGR of 19.13%. Thus, the DMD market offers a lucrative opportunity.
Capricor Therapeutics, Inc. Price and Consensus
Capricor Therapeutics, Inc. Price and Consensus
Capricor Therapeutics, Inc. price-consensus-chart | Capricor Therapeutics, Inc. Quote
CAPR’s Zacks Rank & Stocks to Consider
Capricor currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the sector are Castle Biosciences CSTL, CytomX Therapeutics CTMX and Spero Therapeutics SPRO. While CSTL and SPRO sport a Zacks Rank #1 (Strong Buy) each, CTMX carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, 2024 estimates for Castle Biosciences have improved from a loss of 59 cents per share to earnings of 34 cents. During the same timeframe, loss per share estimates for 2025 have narrowed from $2.15 to $1.84. In the past three months, shares of Castle Biosciences have lost 4.5%.
CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.
In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. Estimates for 2025 loss per share have narrowed from $1.54 to 79 cents during the same timeframe. In the past three months, Spero’s shares have lost 16.9%.
SPRO’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 94.42%.
In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents during the same timeframe. In the past three months, CTMX stock has lost 7.8%.
CytomX’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 115.70%.
Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
