Dive Brief:

While the loss of the deal has made a hole on the company's value, Capricor is looking on the bright side.

"Over the last few years, and during the term of the Janssen option period, we believe that significant value for our CAP-1002 asset has been created through the demonstration of clinical proof-of-concept to treat Duchenne muscular dystrophy (DMD) and also from the progress that has been made towards the development of a commercial-scale manufacturing process for the cells," said Linda Marbn, Capricor's president and CEO.

The company also suggested that a potential upside of the loss of the agreements is that it "resolves uncertainty concerning the scope of the license for CAP-1002 and provides Capricor the freedom to enter into new licensing and/or business development opportunities."

Although, as most investors know, it's generally a bad sign when your big pharma partner bails and, typically, hurts prospects for gaining another commercialization partner.

Capricor has faced some challenges in 2017. In February, it pulled out of an agreement with the Mayo Clinic, which included scrapping development of a Phase 2 heart failure drug, cenderitide, in order to focus on cell and exosome-based therapeutics. And then in May, it faced problems with CAP-1002 in the ALLSTAR Phase 1/2 trial. These topline results showed that CAP-1002 had only a small chance of meeting the primary endpoint of significantly reducing cardiac scarring in adults who had had a major heart attack. This resulted in a reduction in the scope of the company's options, including its workforce size.

The focus for this product, which is manufactured from donated heart tissue, is now in young men with Duchenne muscular dystrophy-associated cardiomyopathy, and the HOPE Phase 1/2 trial is ongoing. Six-month results were presented late last month at the 2017 Patient Project Muscular Dystrophy (PPMD) Annual Connect Conference, showing improved cardiac systolic wall thickening, and improved performance of upper limb in treated patients.

"We discussed potential product registration strategies for this indication at our recent meeting with the U.S. Food and Drug Administration. We expect to commence a randomized, double-blind, placebo-controlled clinical trial of repeat administrations of intravenous CAP-1002 in boys and young men with DMD in the second half of this year, subject to regulatory approval," said Marbn.

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J&J drops stem cell partner Capricor - BioPharma Dive