SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotechnology company with a market capitalization of $627 million, has announced the completion of its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for deramiocel, a cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. This submission could potentially lead to the first approved therapy for this aspect of DMD if the FDA grants approval. According to InvestingPro data, the company's stock has surged nearly 192% over the past six months, reflecting growing investor optimism about this development.
The BLA is the result of comprehensive clinical trials, including Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) studies. These trials have indicated that deramiocel may help manage cardiac complications associated with DMD, a genetic disorder characterized by muscle degeneration and premature death. The company has requested a priority review from the FDA, which could shorten the standard 10-month review period to six months.
Capricor's CEO, Linda Marbán, Ph.D., expressed optimism about the strength of the application and the potential benefits for patients in need. The therapy has received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA) and is supported by RMAT designation in the U.S. and ATMP designation in Europe.
As part of this milestone, Capricor is set to receive a $10 million payment from Nippon Shinyaku Co., Ltd., its distribution partner, under the terms of their U.S. Commercialization and Distribution Agreement. Moreover, FDA approval could make Capricor eligible for a Priority Review Voucher due to the therapy's rare pediatric disease designation. While the company currently maintains strong liquidity with more cash than debt on its balance sheet, InvestingPro analysis reveals challenging fundamentals with a -95% gross profit margin and negative EBITDA of -$34.8 million in the last twelve months.
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have demonstrated immunomodulatory, antifibrotic, and regenerative effects in clinical studies. Over 200 patients have been treated with CDCs, which are also the subject of over 100 peer-reviewed scientific publications.
DMD affects approximately one in every 3,500 male births in the U.S., with an estimated patient population of 15,000-20,000. The disorder leads to progressive muscle damage and heart failure, the latter being the leading cause of death in DMD patients. Current treatments are limited, with no cure available.
This news is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics has maintained its Buy rating and $40.00 price target from Jones Trading, despite market fluctuations triggered by an FDA warning letter sent to a researcher involved with the company's deramiocel HOPE-3 study. The company is projected to complete the rolling Biologics License Application (BLA) submission by the end of 2024, with potential FDA approval in the second half of 2025.
Capricor has also expanded its distribution agreement with Nippon Shinyaku, now covering Europe, with potential sales milestones up to $715 million. The company reported a net loss of approximately $12.6 million for the third quarter of 2024, but has a cash balance of approximately $165 million, supporting the upcoming commercial launch and manufacturing expansion.
In other developments, the European Medicines Agency (EMA) awarded Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to deramiocel, potentially accelerating its development and market exclusivity in Europe. Piper Sandler reaffirmed its Overweight rating and $35.00 price target for Capricor, highlighting the potential of deramiocel in treating Duchenne muscular dystrophy (DMD) cardiomyopathy.
These are among the recent developments at Capricor Therapeutics, reflecting the ongoing progress and strategic moves made by the company.
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