California Stem Cell Agency to Award More Than $7 Million for Duchenne and 'Bubble Boy' Afflictions

Highlights
Capricor, UCSF win
Duchenne, "bubble boy" affliction targeted
Board linkage to recipient enterprises

The California stem cell agency is set next week to make two awards totaling $7.4 million to a Beverly Hills stem cell firm and to scientists at UC San Francisco for late stage research into therapies for rare diseases.

The largest award, $4.3 million, appears to be going to a team in San Francisco that has already received $3.9 million for its research. The lead scientists on that effort were Morton Cowan and Jennifer Puck. 

The latest award involves the "bubble boy" immunodeficiency disease. The agency's summary of the application review said that the research "could lead to a lasting cure" for that version of the affliction. 

The review said the treatment would modify a gene "to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene (Art)." The goal of the grant is to complete nonclinical efficacy studies and set the stage for a clinical trial in 18 months.

Meeting behind closed doors earlier this year, the agency's blue-ribbon, out-of-state grant reviewers narrowly approved the application on 8-6-1 vote, meaning eight favored the award,  six thought the application needed improvement and one voted for denial. The governing board of the agency, formally known as the California Institute for Regenerative Medicine (CIRM), has a decade-long record of going along with its reviewers' positive funding decisions. Reviewers are not required to publicly disclose their economic or professional interests.

Also approved by reviewers was a $3.4 million award to Capricor, Inc., a firm that has already received $19.8 million from the agency to develop stem cell heart treatments, The $19.8 million came on top of earlier, related funding for research at Cedars-Sinai that hit $7 million. The lead scientist on those efforts was Eduardo Marban, who co-founded the firm with his wife, Linda. She is now president of the firm. He is chairman of the scientific advisory board.  

Capricor will add $2.3 million in matching funds to what CIRM provides to finance a clinical trial of the firm's treatment for Duchenne muscular dystrophy cardiomyopathy using a product called CAP-1002. The review summary said the treatment is "intended to stop fibrosis and potentially initiate regeneration following administration."

No therapies exist for treatment of cardiomyopathy for persons with Duchenne, according to the review summary.

The reviewers voted 12-1-0 to approve the award. 

Capricor is publicly traded. Its stock closed today at $2.18. Its 52-week high was $10.68 and its low was $1.88.

CIRM's governing board includes representatives from Cedars-Sinai and UC San Francisco. About 90 percent of the $1.9 billion awarded by CIRM has gone to enterprises with links to past or present board members.

CIRM does not disclose the names of award recipients until after the full board acts. The California Stem Cell Report identified the applicants on the basis of publicly available information. 

California Stem Cell Merger: Capricor and Niles Therapeutics

Capricor, Inc., a Beverly Hills company benefiting from $27 million from the California stem cell agency, this week announced that it is merging with Niles Therapeutic, Inc., of San Mateo.

Linda Marban
Capricor photo

The Capricor story and its treatment for heart disease have been highlighted (see here and here) by the $3 billion state research agency, which is partially funding a clinical trial for the firm. The firm sprang from work by Eduardo Marban of Cedars-Sinai in Los Angeles, one of Capricor's founders. He received $6.9 million for his early and current work. Capricor was awarded $19.8 million more.

Capricor, a privately held firm, and the publicly traded Niles announced on Monday that they were merging. The new company will be known as Capricor Therapeutics, Inc., and will be based in San Mateo.

The new firm will be publicly traded with Capricor CEO Linda Marban as the new CEO.

The new board of directors will have two members from Niles and seven from Capricor, including its executive chairman, Frank Litvack, who was an unsuccessful candidate for chairman of the stem cell agency board in 2011.

The merger press release said that the new company “should have better access to capital, more potential for steady pipeline development and more risk diversification."

On completion of the merger, a joint press release said,

“Nile will issue to Capricor stockholders shares of Nile common stock such that Capricor stockholders will own approximately 90% of the combined company's outstanding shares, and Nile stockholders will own approximately 10%, calculated in each case on a fully-diluted basis assuming the issuance of shares underlying options and warrants. Options of Capricor will be assumed by Nile and become options to acquire stock of Nile.”

Linda Marban said,

"Capricor's and Nile's product portfolios complement each other well, as our therapies will address both the underlying causes and debilitating effects of heart disease. Capricor's CDCs are allogeneic cardiac derived stem cells that aim to attenuate and potentially improve damage to the heart that can result in heart failure, while Nile's cenderitide is intended to treat patients following hospital discharge from an acute episode of heart failure."

Niles' stock price stood at $0.04 recently. Its 52 week high was $0.20 and the 52-week low was $0.02.