SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR) has announced significant regulatory progress for its leading drug candidate, Deramiocel, also known as CAP-1002, intended for the treatment of Duchenne muscular dystrophy (DMD).
Following a recent Type-B meeting with the U.S. Food and Drug Administration (FDA), the agency has granted a pre-Biologics License Application (BLA) meeting based on clinical data from the HOPE-2, HOPE-2 open label extension (OLE), and available data from HOPE-3 studies.
The FDA's agreement paves the way for Capricor to begin a rolling BLA submission expected to start in the third quarter of 2024. This submission approach allows the company to submit individual sections of the BLA as they are completed, potentially expediting the review process.
Capricor CEO Linda Marbán, Ph.D., expressed optimism about the development and the company's ongoing preparations for potential commercialization, including the finalization of a commercial manufacturing facility. Dr. Marbán also highlighted the upcoming presentation of additional 3-year data from the HOPE-2 OLE study at the PPMD conference, which is anticipated to add further support for the efficacy and safety of Deramiocel.
Deramiocel has received Orphan Drug Designation and is supported by the Regenerative Medicine Advanced Therapy (RMAT) Designation. If the FDA approves Deramiocel for DMD treatment, Capricor could receive a Priority Review Voucher due to the drug's rare pediatric disease designation.
Duchenne muscular dystrophy is a genetic disorder marked by progressive muscle weakness and chronic inflammation, affecting approximately 15,000-20,000 patients in the U.S. Currently, there is no cure for DMD, and treatment options are limited.
Capricor is also engaged in preclinical development of exosome-based therapeutics through its proprietary StealthX™ platform, focusing on vaccinology and targeted delivery of therapeutics for various diseases.
The company's announcement is based on a press release statement and reflects the ongoing commitment to address the unmet medical needs in rare diseases, particularly DMD.
In other recent news, Capricor Therapeutics has reported positive results from its HOPE-2 open-label extension study of CAP-1002, a treatment for Duchenne muscular dystrophy (DMD). The study showed sustained benefits in skeletal muscle function and stabilization of cardiac function in patients with DMD. These findings were shared with the FDA during a Type-B meeting, as the company prepares for a potential Biologics License Application and commercialization.
In financial news, Capricor's position appears stable, with a cash runway until Q1 2025, bolstered by a milestone payment from Nippon Shinyaku and funds raised through their at-the-market program. On the analyst front, Oppenheimer initiated coverage on Capricor, assigning an Outperform rating and expressing optimism about the company's lead candidate, CAP-1002. Ladenburg Thalmann also updated its outlook on Capricor, increasing the stock's price target to $25 from $24, while reiterating a Buy rating.
In terms of upcoming events, Capricor plans to present detailed results at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference and expects to announce topline results from the Phase 3 HOPE-3 pivotal trial in the fourth quarter of 2024. These are the latest developments in the company's ongoing efforts to advance its DMD therapy and prepare for potential commercialization.
InvestingPro Insights
As Capricor Therapeutics (NASDAQ: CAPR) makes strides with its drug candidate Deramiocel, investors may be curious about the financial health and market performance of the company. According to recent data from InvestingPro, Capricor holds a market capitalization of $172.1 million USD.
Despite the promising clinical advancements, the company's financial metrics indicate challenges. Capricor's revenue has grown significantly over the last twelve months as of Q1 2024, with a remarkable increase of 389.3%. Still, this has not translated into profitability, with a gross profit margin of -47.2% and an operating income margin of -96.53% during the same period.
InvestingPro Tips highlight several concerns for Capricor, including a high Price / Book multiple of 9.34 and the company's quick cash burn rate. Moreover, Capricor does not pay a dividend to shareholders, which might be a consideration for income-focused investors. On a positive note, the company has experienced a sizeable price uptick over the last six months, showing a 49.86% total return, which could capture the interest of growth-oriented investors.
For those interested in a deeper dive into Capricor's financials and future prospects, InvestingPro offers additional insights. There are 11 more InvestingPro Tips available for Capricor, which could provide valuable guidance for investors considering this stock. To explore these tips and gain a comprehensive understanding of Capricor's investment potential, visit https://www.investing.com/pro/CAPR and remember to use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription. This could be a strategic resource for investors looking to make informed decisions in the biotech sector.
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