staynafloat
2 months ago
--FDA Grants Pre-BLA Meeting Request and Rolling BLA Submission after Review of HOPE-2 and HOPE-2 OLE 3-Year Results--
SAN DIEGO, June 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Additionally, Deramiocel has been selected as the proposed International Nonproprietary Name (INN) for CAP-1002 by the World Health Organization.
โThe last year has been transformative for Capricor as we have moved rapidly towards potential approval for CAP-1002 for the treatment of DMD,โ said Linda Marbรกn, Ph.D., Capricorโs chief executive officer. โThe most recent meeting had several very important outcomes; the first of which was that the FDA has agreed to a pre-BLA meeting based on their review of our clinical data from HOPE-2, HOPE-2 open label extension (OLE) and available data from HOPE-3. This key agreement will allow us to begin our BLA filing with a rolling submission planned to initiate in the third quarter of 2024.โ
The rolling submission process allows for the submission of pre-agreed components of the BLA to be submitted as they are completed, which can streamline the regulatory review process and de-risk the BLA. Capricor plans to further outline the submission schedule with the FDA in the coming months.
Dr. Marbรกn continued, โWe continue to make significant progress on this program as we are awaiting final data in the fourth quarter of 2024 from our already enrolled HOPE-3 pivotal trial and prepare for potential commercialization, including completion of our commercial manufacturing facility and actively preparing for our pre-BLA meeting and rolling BLA submission. Furthermore, we are looking forward to our presentation of additional 3-year data from our HOPE-2 OLE study at the upcoming PPMD conference which we believe will add further evidence of the efficacy and safety of CAP-1002.โ
Deramiocel (CAP-1002) for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for this drug is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for Deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received.
https://www.capricor.com/investors/news-events/press-releases/detail/281/capricor-therapeutics-announces-positive-type-b-meeting
GreedyAgorist
5 months ago
How did that work out for you, ed? Did you get some 5's assigned to you last month? I only picked up another 100 shares in February, after doing the same in January. It was December when I increased my position here by 60%. Now all those shares are up for grabs at 5. We will find out in 10 days if that is a bargain or not. At worst it will be a 25% gain in 3 months.
I only have one position in cash covered puts. Those will also expire on the 15th. It will be like scoring a 20% dividend out of something that does not pay a dividend.* Wish I had more put positions; I will the next time this "pos" dips. BTW, I trade this in an IRA, so no Wash Sale worries. I won't presently become a millionaire like Volcano. But maybe future me will.
Obvious statement: I am not ed3/6me's financial advisor or anyone else's. Just demonstrating how you can milk a ticker for almost monthly income without it being a blue chip dividend payer. I can't say it removes those anxious feelings you get when it dumps (or pumps), but it will remove some cash off the table.
*I could use more such scores to keep up with inflation; you know, just in case Biden scapegoating evil corporations doesn't do the trick.
staynafloat
5 months ago
Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy
SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- โ Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March. In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission. The Company has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in Duchenne muscular dystrophy (DMD) and these designations enable sponsors to work closely with the FDA and receive its guidance on potential expedited pathways for approval.
โAt this time, with our pivotal Phase 3, HOPE-3 trial fully enrolled in Cohort A and top-line data expected in the fourth quarter of this year, we believe that discussing our CMC plans for potential commercialization with the FDA will allow us the opportunity to bring CAP-1002 to patients in the most expeditious manner possible for patients in need,โ said Linda Marbรกn, Ph.D., Capricorโs chief executive officer. โAt Capricor, we remain focused on getting to approval as quickly as possible and we look forward to providing more details after this meeting.โ
https://www.capricor.com/investors/news-events/press-releases/detail/271/capricor-therapeutics-announces-upcoming-type-b-meeting