SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR), a biotechnology firm, announced today that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA meeting for the third quarter of 2024. This meeting aims to finalize the Biologics License Application (BLA) for deramiocel (CAP-1002), a treatment for Duchenne Muscular Dystrophy (DMD).
DMD is a severe genetic disorder that leads to progressive muscle weakness and chronic inflammation, affecting approximately 15,000 to 20,000 patients in the U.S. Deramiocel, an allogeneic cardiac-derived cell therapy, has been advancing through Phase 3 clinical development and is designed to address the consequences of DMD by providing immunomodulatory, antifibrotic, and regenerative actions.
Capricor's CEO, Linda Marbán, Ph.D., expressed the company's commitment to working with the FDA to expedite the filing and potential approval of deramiocel. She highlighted the need for multiple therapies to effectively combat DMD, suggesting that deramiocel could serve as a cornerstone treatment for patients.
The company will also present 36-month data from the HOPE-2 open label extension study at the Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference from June 27-29, 2024. This data will cover both skeletal and cardiac outcomes.
Capricor has an agreement with Nippon Shinyaku Co., Ltd., for the exclusive commercialization and distribution of deramiocel in the U.S. and Japan, pending regulatory approval. However, deramiocel is currently an Investigational New Drug and has not been approved for any indications.
The forthcoming FDA meeting is a significant step for Capricor as it seeks to bring its lead product candidate to market. The announcement of this meeting is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics announced positive three-year results from its HOPE-2 study of CAP-1002, a treatment for Duchenne muscular dystrophy (DMD). Patients showed sustained benefits in skeletal muscle function and stabilization of cardiac function. Analyst firms Oppenheimer and Ladenburg Thalmann have both expressed optimism about Capricor's developments, with Oppenheimer forecasting that CAP-1002 could achieve around $1 billion in net sales in the U.S. by 2030, resulting in approximately $310 million in revenue for Capricor.
Ladenburg Thalmann has also increased its price target for Capricor shares, reflecting positive views on the company's ongoing discussions with the FDA and potential developments. Capricor has completed enrollment for Cohort A of its HOPE-3 Phase 3 pivotal trial and is planning for potential commercialization and global expansion.
The company's financial 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. As Capricor prepares for the next steps in bringing CAP-1002 to market, the industry will be watching closely for the outcomes of their ongoing trials and regulatory discussions. These recent developments underline the progress made by Capricor in its pursuit to provide effective treatment for DMD.
InvestingPro Insights
As Capricor Therapeutics gears up for a critical Pre-BLA meeting with the FDA for its DMD treatment, deramiocel, financial metrics and analyst insights provide a broader picture of the company's market standing. With a market capitalization of 153.18 million USD, Capricor is navigating a pivotal phase in its development. Despite impressive revenue growth of 389.3% in the last twelve months as of Q1 2024, the company's financial health is under scrutiny due to concerns about its cash burn and profitability.
InvestingPro Tips highlight that while Capricor holds more cash than debt, which is a positive sign, it is also quickly burning through cash. In addition, analysts have revised their earnings downwards for the upcoming period, anticipating a sales decline in the current year. These concerns are compounded by weak gross profit margins, as evidenced by a gross profit margin of -47.2% in the same period. Furthermore, the company is expected to post a drop in net income this year, and analysts do not anticipate Capricor will be profitable within this timeframe.
The stock's performance reflects these challenges, with a 20.03% decrease over the last month and a 27.75% drop over the last three months. The price/book ratio, standing at a high 8.31, suggests that the stock is trading at a premium relative to its book value. For investors looking for additional insights, there are over 10 InvestingPro Tips available, which can be explored further at https://www.investing.com/pro/CAPR. Interested readers can use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, offering a deeper dive into Capricor's financials and future outlook.
With the FDA meeting on the horizon, Capricor's financial metrics and analyst expectations will be critical for investors to monitor as they assess the potential impact of deramiocel's approval and commercialization on the company's future performance.
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