San Diego's Viacyte Scoring Again with State Funding; Total Now Hits $55 Million

California’s stem cell research agency appears ready to pump another $16.6 million into a diabetes therapy effort that has already received $38.5 million from the Golden State.

At a total of more than $55 million, it is likely the single largest direct investment that the state of California has ever made in a business. The cash is expected to be approved by directors of the $3 billion stem cell agency at their meeting Wednesday in Berkeley.

The agency has a policy of secrecy concerning the names of those seeking public money and did not identify the firm.  However, it was apparent from documents on the Web site of the California Institute for Regenerative Medicine (CIRM), as the agency is known, that the recipient is Viacyte, Inc., of San Diego, Ca. The firm is the most heavily backed business by the stem cell agency. Second is Capricor, Inc., of Beverly Hills, Ca., at $19.8 million.

Viacyte is working on a diabetes treatment that it has described as a “virtual cure” for type 1 diabetes. The company describes the device as akin to a “flat tea bag” that would be implanted beneath the skin.

Last month, the firm announced it had received $20 million (see here and here) from Janssen Pharmaceuticals, a subsidiary of Johnson and Johnson. Viacyte also last month announced it has received a federal go-ahead to begin a clinical trial for its human embryonic stem cell treatment.

Only about one out 10 traditional drugs emerge into the marketplace from clinical trials. No therapies involving human embryonic stem cells (hESC) have successfully run the clinical trial gauntlet in the U.S. and secured approval for widespread use.

This week’s funding for Viacyte involves two awards in application AP1-08039 in the agency’s new, $200 million “accelerated development pathway,” which is designed to speed research into the clinic and marketplace. The agency’s blue-ribbon reviewers examined the application behind closed doors last spring but said in a summary posted publicly last week that the therapy had the potential to “transform” diabetes treatments.

More than two million persons suffer from diabetes in the California and roughly 370 million worldwide.

Only four other applicants were considered in the “pathway” round. Viacyte was the only business. The other applications came from Henry Klassen at UC Irvine, Thomas Kipps of UC San Diego, Donald Kohn of UCLA and Clive Svendsen of Cedars of Sinai. All the institutions have representatives on the CIRM board of directors, but were rejected by reviewers.  As mentioned above, none was identified by the agency but all could be recognized by other means.

All of the agency’s comments and summary of reviewer comments can be found on this document. Klassen’s application is DR2A-05379. Kipps’ application is AP1-08043.  Svendsen’s application is AP1-08047. Kohn’s application is AP1-08048.

  

Rejected applicants will have an opportunity to apply again in later offerings in the $200 million acceleration pathway program, which is aimed at producing major scientific results by 2017.

Viacyte had applied for $25 million, the upper limit in this week’s awards. However, the reviewers rejected two “modules” in the application worth $8.4 million, suggesting that they were not timely and could be reworked incorporating reviewer suggestions.

California's New $200 Million Stem Cell Spark

Directors of the $3 billion California stem cell agency this month will attempt to supercharge some of their major research programs and push stem cell therapies more rapidly into the marketplace and the clinic.

Sir John Bell
Academy of Medical Sciences photo

The move is part of a $200 million effort approved only last December and was recommended by the agency’s blue-ribbon scientific advisory board, chaired by Sir John Bell of the University of Oxford in the United Kingdom. 

Awards in the first phase of the program – dubbed an “Accelerated Development Pathway” -- will range up to $25 million each. The effort will also involve more assistance and streamlined procedures from the California Institute of Regenerative Medicine (CIRM), as the agency is formally known.

The awards are scheduled to be approved publicly on Sept. 10 at the directors’ meeting at the Claremont Hotel in Berkeley, Ca. Winners will be culled from a select group of researchers already funded by the agency.

The number of applicants was not immediately available from CIRM, but they were limited to individuals and institutions that have already received the agency’s signature disease team or strategic partnership awards. Also unknown was whether any businesses were among the applicants. Applicants rejected in next week’s round will have a chance to apply in upcoming, new “pathway” rounds.

According to the request for applications (RFA) on the CIRM Web site, the agency is seeking “high potential” research that can achieve “clinical demonstration of an acceptable safety profile and proof of concept during or before 2017.”

The RFA continued,

“For example, a team could propose additions to an ongoing CIRM-funded clinical trial that would accelerate development decisions such as including the testing of a biomarker they identified in correlated research work or adding a patient group based on data from an unblinded safety study. Other examples of accelerating activities could include changes in manufacturing processes or delivery devices, based on novel ideas that emerged from the activities of the initial award or related research that could require comparability studies to facilitate development of the candidate therapeutic. In addition, the Accelerated Development Pathway would also consider the possibility of funding a future clinical trial to demonstrate clinical proof of concept in the approved therapeutic indication, ‘subject to satisfaction of milestones and conditions.’”

The “pathway” program was approved by CIRM directors after its new Scientific Advisory Board recommended its creation. The advisors said the agency should move “at speed” to turn research into treatments.

Creation of the panel of advisors was recommended by the Institute of Medicine (IOM) in a $700,000 study funded by CIRM. The IOM report said the panel would be invaluable in helping the agency to “make fundamental decisions about dealing with challenges that cut across particular diseases, decide which discoveries should progress toward the clinic and determine how best to engage industry partners in developing new therapies.”

(See here for the names of the members of the advisory panel. The full text of their report can be found at the end of this link.)

The panel has held only one meeting, and that was behind closed doors.