Big Pharma's J&J Buys Into California Stem Cell Therapy Research for Heart Disease

California's $27 million investment involving a Beverly Hills stem cell firm is paying off once again as the business attracted a major, financial vote of confidence from Big Pharma's Johnson&Johnson.

The firm, Capricor Therapeutics, last month received approval for a phase two clinical trial for its heart disease therapy. Yesterday, it announced that the Janssen Biotech arm of Johnson&Johnson was immediately pumping $12.5 million into the product with the potential of $325 million more, depending on the outcome of the phase two trial.

Linda Marban
Capricor photo

John Carroll of Fierce Biotech wrote that the move marked a “rare Big Pharma gamble on a field that is trying hard to mount a comeback.” The California stem cell agency, which is funding the phase two trial, said the news was “heartening”and a represented a “very good start” to the year for Capricor.

Linda Marban, CEO of Capricor, told Fierce Biotech that J&J had been probing Capricor for a year. She said, 

“One of the reasons why I was motivated to work on this deal is because of the statement it makes in the field. It says, OK, somebody very large and powerful is taking a look at this technology and saying there's something there, and that's the most exciting thing for me."

Interest from the “large and powerful” is of major importance not only to Capricor but to the stem cell agency, which runs out of state money for new research grants in 2017. It is slowly trying to develop other sources of revenue, and it has yet to bring a therapy to market despite promises to voters during the 2004 ballot campaign that created the agency. Votes of confidence from Big Pharma will go a long way in encouraging investment in the agency and the stem cell field generally.

Investors indeed were more than encouraged by the yesterday's news, which sent Capricor's stock soaring 48 percent. It closed at $5.06 yesterday, up from $3.40 on Friday. Marban has said the firm hopes to be profitable by 2018.

Johnson&Johnson's investment is not the first tied to a key executive at Capricor, Frank Litvack, who is executive chairman of the firm. Litvack, who unsuccessfully ran in 2011 against Jonathan Thomas for the chairmanship of the stem cell agency, sold Conor MedSystems to J&J for $1.6 billion in 2006.

Bradley Fikes of the San Diego U-T discussed the Capricor research late last month in some detail. One of the phase one trial sites was at Scripps Health. Fikes wrote,

“Mark Athens received Capricor’s treatment on Sept. 25, about a month after having a moderate heart attack. The Encinitas resident was the last treated under Phase 1, said Scripps cardiologist Richard Schatz, who performed the procedure. It will take about six months to know whether the treatment worked, Schatz said.”

Fikes continued,

“'All their previous work showed that the scar got smaller and the muscle tissue around it got more robust,' Schatz said. 'So two things happened: The viable tissue got bigger and the scar got smaller. And that should translate into some sort of clinical benefit down the road.'”

Here are links to the press release from Capricor and other stories in story Genetic Engineering and Biotechnology News and PharmaTimes.

Golden State Milestone: Stem Cell Agency Announces its First Phase Two Clinical Trial

The California stem cell agency today scored a first with the announcement that one of its projects – a heart disease therapy -- has now advanced to a phase two clinical trial after successfully completing a phase one safety trial.

In a press release, Jonathan Thomas, chairman of the $3 billion research effort, hailed the Allstar trial by Capricor Therapeutics, Inc., of Beverly Hills. He declared,

"This is a highly significant announcement for us at CIRM as it's the first time we have funded a therapy into a Phase 2 clinical trial.

"Heart disease claims around 600,000 American lives every year, so clearly there is a huge need for new approaches and more effective therapies. We are hopeful this is the first of many treatments to turn the tide against this disease, and that this will be the first of many projects we are funding to get to a Phase 2 trial."

The adult stem cell therapy “uses unrelated donor-derived stem cells, called cardiosphere-derived cells, that are then infused into a patient’s artery with the aim of reducing scarring caused by heart attacks,” CIRM said.

Capricor Therapeutics, which has its roots in Cedars-Sinai in Los Angeles, describes itself as as “a diversified heart failure biotechnology company.” It came into being last summer as the result of the merger of Capricor and Niles Therapeutics. The company has benefited from about $27 million in support from the stem cell agency, including $6.9 million for early work by one of its founders Eduardo Marbán at Cedars-Sinai.

The company's stock price closed at $2.48 today, down 17 cents. Its 52-week range is from $2.48 to $2.65.

The firm's CEO, Linda Marbán, said the move into phase two was a “giant leap” for the firm and the heart therapy field.

The executive chairman of the firm is Frank Litvack, a heart surgeon who was the only other candidate for chairmanship of the stem cell agency when Thomas won the job in 2011.

The announcement today is a milestone for the stem cell agency, which is aggressively seeking results that will help to generate financial support after 2017, when its current state funding runs out.

The CIRM news release said,

“The next phase will involve an estimated 300 patients who have had heart attacks, and they will be evaluated in a double-blind, randomized, placebo-controlled trial.  This will be further broken down into two groups: one will include patients 30-90 days post attack, the second will be 91 days to one year after the incident.”

The phase two trial is to determine the therapy's effectiveness and further study its safety. If successful, it can move into a phase three trial to confirm its effectiveness, monitor side effects, compare it to common treatments and collect information that will allow the drug or treatment to be used safely.

Here is a CIRM video in which Eduardo Marbán discusses some of the issues involved in the therapy. Here is a link to a 2011 presentation to the CIRM governing board about the early research – a presentation that generated some excitement on the board.