FN Summary: Lead asset (CAP-1002) had very good results in a small P2 trial. Drug qualified for RMAT designation. This allows them to possibly file for a BLA using their P2 results. If they can’t (definitely a possibility), then they must do a P3 trial. Good cash position, but will not last if P3 is required. No recent insider buying on the open market.
Table of Contents
This company hits most of the right spots!
RMAT designation for their flagship drug!!
Good Phase 2
Plenty of Cash
Recent Insider Buying
Waiting on discussion results (either proceed with BLA, or a P3 trial) with the FDA! By end of 3rd Q
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Cash on Hand
Est Monthly Cash Burn
just an estimate (will increase in 2021)
Est Cash End of June 2021
Enough until FDA makes their decision, but not enough if a P3 trial is needed.
Products & Sales Potential
Capricor Market Cap: ~ $112M as of July 22, 2021
Yes, we get it. This is the most dry part of the page. But, it is also the most important. Read this: Importance of Management
- Dr. Marbán is currently serving as CEO and has served in that capacity and on the Board since November 2013.
- As co-founder of Capricor, Inc., our wholly-owned subsidiary, Dr. Marbán has been with Capricor, Inc. since 2005 and became its Chief Executive Officer in 2010. She combines her background in research with her business experience to lead the Company and create a path to commercialization for its novel therapies.
- Dr. Marbán was the lead negotiator in procuring the license agreements that are the foundation of the Company’s intellectual property portfolio.
- Under her direction as CEO, the Company has been awarded approximately $30.0 million in non-dilutive grant awards which have funded our research and development programs, and a loan award that funded Capricor, Inc.’s ALLSTAR clinical trial.
- Dr. Marbán’s deep knowledge of the cardiac space, in particular, allows her to provide unique direction for the Company’s development and growth. From 2003 to 2009, Dr. Marbán was with Excigen, Inc., a biotechnology start-up company, where she was responsible for business development, operations, pre-clinical research, and supervising the development of gene therapy products in a joint development agreement with Genzyme Corp. While at Excigen, she also negotiated a joint development and sublicense agreement with Medtronic Corp. utilizing Excigen’s technology and supervised the building of a lab in which the work was to be performed.
- Dr. Marbán began her career in academic science, first at the Cleveland Clinic Foundation working on the biophysical properties of cardiac muscle. That work continued when she moved to a postdoctoral fellowship at Johns Hopkins University (“JHU”). While at JHU, she advanced to the rank of Research Assistant Professor in the Department of Pediatrics, continuing her work on the mechanism of contractile dysfunction in heart failure.
- Dr. Sudhir Borgonha serves as the VP of Clinical Development. Dr. Borgonha has held senior leadership positions in the industry across multiple geographies. Most recently, he was the Director of Translational Medicine at the Fanconi Anemia Research Fund, where he led efforts to propel a spectrum of approaches including gene therapy to treat cancer in rare diseases.
- As the Medical Director of Strand Genomics, he oversaw the development of new technologies such as liquid biopsy and customized cancer panels, while he oversaw clinical reporting for over 5,000 patients a year (1,000 of them were rare diseases).
- As the Medical Director of Martindale Pharma, he oversaw clinical and medical affairs of over 100 products in more than 12 countries and successfully designed, developed, and implemented multiple clinical trials at Translational Medicine Inc.
- He was a co-founder of Angstrom Medica (Acquired by Pioneer Surgical), a biomaterials science company which he co-founded with fellow students while at MIT. Dr. Borgonha is a co-founder of ten3T, a remote cardiac monitoring company.
Clinical Trial for DMD
Understanding the Phase 2 trial is crucial to anticipate the success of a possilbe Phase 3 trial.
Capricor is in discussions with the FDA to see if they can proceed from their Phase 2 results straight to a BLA. If they can, it will be a huge boost the the share price.
The results of the Phase 2 trial are shown below.
Due to the impressive results of CAP-1002, it qualified for RMAT! The company is currently in discussion with the FDA to determine if they can proceed with a BLA or need to conduct a Phase 3 trial.
CAPR is also doing an open label extension trial, which began in July 2020. The trial is estimated to end in Oct 2021.
(FN posts only the most recent documentation)
CAPR Discussion Forums (listed in order of best DD potential)
Q. What is RMAT Designation?
A. RMAT stands for “regenerative medicine advanced therapy”. This designation provides expedited programs available to sponsors of regenerative medicine therapies for serious conditions, such as DMD. Source
Q. What does it mean for Capricor?
A. It means more frequent FDA meetings, eligibility for rolling review & priority review and accelerated approval. Most importantly, it might allow the company to skip the P3 trial and submit a BLA! If that is permitted it will save the company hundreds of millions of dollars.
Q. Does the FDA have any guidance for developing drugs for DMD?
A. Yes it does. Here is the document.
Q. Why is the FDA willing to approve select DMD drugs after only a Phase 2 trial?
A. DMD is a horrible disease. It can be summed up in this chart.
If you had a child with this disease, would you want to wait 2-3 years for sponsors to finish a Phase 3 trial? No, you would take whatever drug shows promise.