News Coverage of Capricor and California Stem Cell Agency Less Than Abundant

Capricor Therapeutics' stock price retreated today after soaring 253 percent yesterday on the news that one of its products had successfully treated a small group of critically ill, Covid-19  patients.

The price closed today at 7.00, down from yesterday's close of $8.50. That was the highest price for the Beverly Hills firm since 2018.

While the news about its CAP-1002 treatment excited investors, it did not stir the news media. In the glut of hundreds of stories about the coronavirus, the mainstream media did not even mention Capricor. Nor did the Los Angeles Times, virtually the home town paper for the firm. As for the California stem cell agency, which has pumped nearly $25 million into Capricor-related research, the role of the agency was also among the missing.

The Los Angeles Business Journal did carry a tidy and straight forward story, again one that did not mention the stem cell agency, which is hoping that California voters will save its financial life next fall. That is, if they approve a proposed, $5.5 billion, ballot measure that has already missed one state-recommended deadline (April 21) for qualifying for the ballot. BioWorld also had a story that did not mention the California agency's role. 

The agency is running out of the $3 billion that voters provided for it in 2004 when they created the unprecedented state research program. It will begin closing its door in six months unless major funding is found.

The sparse news coverage of the agency will be a challenge for the agency's backers as they seek voter approval of the ballot measure, assuming it qualifies.

$20 Million, California-backed Stem Cell Trial Discloses Disappointing News

Capricor Therapeutics, Inc., today announced some bad news about its state-of-California financed clinical trial for a cardiac, stem cell therapy and said that it planned to lay off an unspecified number of employees.

Capricor's stock price plummeted 62 percent following what the company called the "unexpected" news, dropping from $1.89 to $1.16. California's stem cell agency has backed the trial with $20 million, plus an additional $7 million for earlier, related research.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said on its blog,

"Obviously this is disappointing news for everyone involved, but we know that not all clinical trials are going to be successful. CIRM supported this research because it clearly addressed an unmet medical need and because an earlier Phase 1 study had showed promise in helping prevent decline in heart function after a heart attack."

In response to a question, Kevin McCormack, senior director of communications, said the agency is talking to Capricor about the next steps. The agency regularly halts funding of awards when recipients do not meet milestones. McCormack did not respond to a question about how much money the firm had already received from CIRM.

Capricor, which is headquartered in Beverly Hills, said in a press release that an interim analysis on the phase two trial has "has demonstrated a low probability (futility) of achieving a statistically-significant difference in the 12-month primary efficacy endpoint." The company said there was "no notable difference" between treatment groups.

The company said it would cut its workforce to focus more sharply on its treatment of Duchenne muscular dystrophy, which the stem cell agency is also backing with $3.4 million. The Duchenne treatment had better news the last month, clearing its phase one trial with no adverse effects.

Timothy Henry and Rajenda Makkar of the Cedars-Sinai Heart Institute in Los Angeles are the principal investigators for the clinical trial.

The highest price for Capricor stock over the last 12 months was $5.40 and the lowest was $1.13.

Here are links to additional news stories today on Capricor: BiopharmaDive, MarketWatch, Genetic Engineering News, 

Capricor and California Stem Cell Agency Say All is Well With $27 Million Investment

One recent headline in Forbes magazine read, 

“Stem Cell Therapy To Fix The Heart: A House Of Cards About To Fall?”

Over at the Boston Globe, another story this month said,

 “A provocative new study calls into question the rationale for using stem cells to repair the heart — a much-hyped experimental therapy that grew out of insights from a groundbreaking Boston researcher’s laboratory.”

Eduardo Marban
Cedars-Sinai photo

But it appears that heart stem cell regeneration is like cheese, you might say. There are many different varieties. And in the case of a $27 million investment in heart cell regeneration by the California stem cell agency, all the players say things are just fine with their project.

The state's research effort involves a publicly traded company called Capricor Therapeutics. The Beverly Hills firm and one of its founders. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, received the $27 million from the state stem cell agency.

In the wake of the news reports involving heart stem cell regeneration, the California Stem Cell Report queried both Marban and the stem cell agency. Marban replied within 37 minutes last week. Here is the text of what he had to say in his email.

“All of the critiques have been leveled at competing technologies. Indeed, I have been among the more vocal critics (for example see http://www.tctmd.com/show.aspx?id=124193).

“Although some alarmist headlines have inappropriately questioned all cardiac stem cell work, the issues have to do with only one specific subtype of cardiac stem cell, which is marked by the expression of an antigen called c-kit. This is NOT the cell type being developed by Capricor; in fact, my laboratory has found that c-kit-positive cells play no role in Capricor’s cell product. That product, known as CAP-1002, contains a small fraction (less than 5 percent) of c-kit-positive cells which can be completely removed with no loss in potency. CAP-1002 is believed to work by indirect 'paracrine' mechanisms that differ fundamentally from those postulated for the c-kit-positive cells.

“By way of background, the critiques of c-kit-positive heart cells have come in two forms: first, in concerns expressed by the editors of the Lancet regarding data integrity in the SCIPIO trial; this was conducted by Piero Anversa and colleagues using c-kit-positive cells. Second, a recent Nature paper (attached in case you do not have a copy), of which I am an author, questions the claim that c-kit-positive cells are true cardiac progenitors.

“These concerns in no way undermine Capricor’s technology. In fact, CAP-1002 remains the only heart-derived cell product in commercial development. I am proceeding full speed ahead with mechanistic and translational work on CAP-1002, and Capricor continues to feel bullish about the product, which is now in phase 2 trials (the furthest along of any CIRM-funded project).”

Kevin McCormack, a spokesman for the stem cell agency, also said that Capricor is using a different method and a different stem cell than the one being called into question.

You can hear the May 15 Capricor conference call on its first quarter results here. Its stock price jumped from $6.15 on May 14 to as high as $7.65 during the day May 15. Its 52-week low was $1.15 and its 52-week high $17.15.

(Editor's note: If you would like a copy of the Nature paper that Marban referenced, please send an email to djensen@californiastemcellreport.com.)

Knoepfler and Capricor Score Big This Week

Capricor's stock price has soared since Monday.
Chart from Google Finance

With the new year hardly begun, UC Davis stem cell researcher and blogger Paul Knoepfler has fulfilled one of his 10 predictions for the stem cell world in 2014.

On Dec. 29, Knoepfler predicted a “big announcement from Big Pharma” in 2014. He did not have to wait long to add some shine to his crystal ball.

On Monday, he wrote about the news that Johnson&Johnson was pumping $12.5 million into Capricor Therapeutics' stem cell therapy for heart disease. Capricor could see another $325 million from J&J if all goes well in its stage two clinical trial, which is being supported by $20 million from the California stem cell agency.

“I'd call that big money,” Knoepfler wrote. He also noted in his blog he has no financial interest in either company.

Investors liked the news as well. Capricor's stock closed at $7.05 today, more than double its $3.40 close on Friday, the business day just prior to J&J announcement. 

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.

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.