Thursday, September 17, 2015

Brain Cancer Targeted: Calabasas Biotech Firm Set to Win $20 Million in California Stem Cell Funding

John S. Yu, chairman, ImmunoCellular -- Cedars-Sinai video 
Highlights
Second CIRM bet on phase three trial
$34 million in co-funding
Application initially rejected

California’s stem cell agency next week is expected to award $20 million to a tiny California firm to help finance a phase three clinical trial of a therapy for a rare brain cancer.

The move would be the $3 billion agency’s second investment in a phase three clinical trial, the last major hurdle before a therapy can be offered for widespread public use. The first was an $18 million bet this spring on NeoStem, Inc., now known as Caladrius.

The two trials offer the best likelihood of fulfilling -- in relatively short order -- the 2004 campaign promises made to voters when they created the Golden State's stem cell research effort. 

The firm involved is ImmunoCellular Therapeutics, Ltd., a publicly traded enterprise headquartered in Calabasas, Ca., a suburb northwest of Los Angeles.  Calabasas is better known for its entertainment personalities, such as the Kardashian family, than biotech.

In addition to the state funds, the trial would be backed by $34.4 million in co-funding, according to the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. Earlier this year, ImmunoCellular estimated the total cost of the trial could be about $50 million.

The firm's proposed therapy targets glioblastoma, a fast-moving cancer that CIRM said is “resistant to current standards of care."  It has a survival rate of slightly more than one year. Immunocellular said that about 10,000 new patients are diagnosed each year in the United States.

The treatment is called ICT-107. Immunocellular reported on its Web site that the treatment is “an autologous (patient-derived) dendritic cell immunotherapeutic that targets six different antigens (peptides that are tumor markers) associated with glioblastoma.”

The CIRM summary of reviewer comments said that the phase one trial for the treatment produced a “tremendous survival response.” The summary said,
“Though the survival response observed in the Ph1(phase one) trial was not fully replicated in the Ph2 trial, results are supportive of continued clinical development and of the Ph3 trial as designed.”
ImmunoCellular has been around with its current focus since 2006 when it acquired its cellular-based technology from Cedars-Sinai in Los Angeles. John S. Yu, director of surgical neuro-oncology at Cedars, is listed as the founder of the firm. He is now chairman of the board and chief scientific officer.

The firm has four full-time employees and four part-time, including Yu, according to February 2015 filings with the Securities and Exchange Commission. The firm said,
“We have a number of consulting agreements for clinical development, regulatory affairs, investor relations and business development. We outsource all of our drug discovery research, process development, manufacturing and clinical development to third parties with expertise in those areas."
ImmunoCellular reports no revenues for the past several years. CIRM said, however, the firm has promised $34.4 million in co-funding for the clinical trial.

In an August closed-door session, CIRM’s scientific reviewers scored the ImmunoCellular application as having “exceptional merit” on a 9-0 vote. 

Ratification of that decision will occur in public next Thursday in a teleconference meeting of the agency’s 29-member governing board, which includes one representative from Cedars-Sinai.

The board almost never rejects a positive recommendation from its reviewers. The agency also does not release the names of successful applicants in advance of a board vote. The California Stem Cell Report identified ImmunoCellular through other means.

The firm's original application was deemed by CIRM reviewers to need improvement on a 1-9-2 vote and was sent back to ImmunoCellular for revision. The August review summary said,
“(R)eviewers had feasibility concerns regarding the applicant’s ability to enroll the trial and maintain the reagent supply chain to support manufacturing of the product. Reviewers also had concerns with the trial design, which centered around selection of the patient population targeted in the registration trial and the lack of immune monitoring proposed in the trial.
“The applicant was provided the opportunity to address these concerns in a revised application, and their responses and modifications to the proposal reassured reviewers that the applicant could enroll and conduct the Ph3 trial as proposed and that, if endpoints are met, the trial design could support licensing approval by FDA.”
The price of ImmunoCellular stock closed at $0.425 today, up nearly one cent. The 52-week high of the stock was $1.03 and the low 35 cents.
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