This company is on the watch list for 1 reason:
1) P3 results are outside the 6-9 month time-frame we like to see in our covered biotechs. CAPR will be initiating a Phase 3 by the and of Q1 2022 (hopefully). We anticipate P3 results 1H 2023. If P3 results are positive, the SP will likely spike to 5-10x current levels. This company has huge potential in the DMD space.
Once the company is closer to P3 readout, we will consider it for inclusion in our Stock Picks list.
FN Summary: Lead asset (CAP-1002) for DMD had very good results in a small P2 trial. Drug qualified for RMAT designation. Company must do a 65 patient P3 trial, hopefully starting in by end of Q1 2022. Good cash position, but will not last long without a partner, which management says is required before starting P3. 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
Partnership Announcement - Management has not given a timeline.
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Caveat! Stock movement can be very finicky and, at times, seemingly random. This is FN's best explanation of the price movement.
Cash on Hand
Est Monthly Cash Burn
just an estimate (will increase in 2021)
Est Cash End of July 2021
Not enough if a P3 trial is needed and company does not find a partner.
Products & Sales Potential
Capricor Market Cap: ~ $102M as of Sept 15, 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.
Phase 2 Clinical Trial Results
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.
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.