An FDA High Sign for a California Stem Cell Agency Bet: San Diego Biotech Business Earns FDA Speed-Up Approval

A San Diego firm backed by nearly $24 million from the California stem cell agency scored this week with a special designation from the federal government that could help speed approval of its therapy for a type of blood cancer. 

The firm is Poseida Therapeutics, Inc., which is testing the safety of a therapy for a type of blood cancer in a phase one clinical trial. 

The firm announced yesterday that the Food and Drug Administration (FDA) had granted Regenerative Medicine Advanced Therapy (RMAT) status for the adult stem cell-connected treatment. The designation is intended to expedite development of the multiple myeloma therapy. 

Eric Ostertag, CEO of the firm, said in a news release, that Poseida's potential treatment, P-BCMA-101, "is the first anti-BCMA CAR-T therapy to receive RMAT designation from the FDA and underscores the urgent need for new treatment options for multiple myeloma." 

He continued, 

“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population...."

Ostertag said the firm expects to have more data by the end of the year. 

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has invested in 49 clinical trials. Five have garnered RMAT designation, which can significantly speed development of a commercial product. As of September, the FDA had granted only 24 RMAT designations nationally. 

Last Sepetember, Geoff Lomax, CIRM senior officer for medical affairs and strategic centers, wrote in cell&gene that the two-year-old, RMAT program streamlines therapy development by enabling possible priority FDA review and accelerated federal approval. 

Longitude Capital of Menlo Park, Ca., is a major investor in the firm, pumping in tens of millions of dollars.  

Stem Cell 'Ethical Tensions,' Recouping the Public Investment, Affordability and Much More

"Staggering" amounts of public money have been spent on stem cell research, and "care must be taken" to assure that commercialization does not exact an excessive "human or monetary price," according to an article this week in the journal Science.

The cautionary note was sounded in the prestigious publication while the world's largest stem cell gathering is underway in Australia with more than 2,500 researchers and others in attendance. Plus next week, the $3 billion California stem cell agency convenes its directors to mull over its own programs and give away more millions. 

Written by Douglas Sipp, a researcher at RIKEN in Japan, Megan Munsie of University of Melbourne and Jeremy Sugarman of John Hopkins University, the Science article said

"Ethical tensions related to stem cell clinical translation and regulatory policy are now center stage...."

They said that expedited government procedures for use of stem cell treatments have been set up in the United States, Japan and Italy. At the same time, they wrote, 

"A staggering amount of public money has been spent on stem cell research globally."

The article declared, 

"The state and the taxpaying public's interests should arguably be reflected in the pricing of stem cell products that were developed through publicly funded research and the regulatory subsidies. Detailed programs for recouping taxpayers' investments in stem cell research and development must be established."

They warned, 

"Care must be taken to ensure that entry of stem cell–based products into the medical marketplace does not come at too high a human or monetary price."

Another journal article this week also sounded cautionary notes. Written by Paul Knoepfler of UC Davis, it was titled: 

"Too Much Carrot and Not Enough Stick in New Stem Cell Oversight Trends"

Knoepfler addressed the Food and Drug Administration's efforts to speed use of stem cell therapies. He wrote in Cell Stem Cell and also on his blog, saying,

"It’s been remarkable to see the FDA approve up to 20 regenerative medicine advanced therapy(RMAT) designations in just over a year. However, I think there’s a strong possibility the agency has swung too far from the too slow review of stem cell and regenerative medicine investigational therapies in the past to now going at warp speed. Since none of the current 20 designated RMAT products had any kind of prior expedited review designation given, is it reasonable to think all 20 now meet rigorous enough standards and all because of new data? It’s hard to say, but there’s likely a spectrum of existing data behind these RMAT designated studies.

"We won’t have an overall RMAT verdict for years as the RMAT trials play out. However, I predict that the agency has lowered the bar too far. There are also concerns that the conditional approval system in Japan is too liberal, as evidenced by discussion over the approval of a recent IPS cell cardiovascular study. Taken together this is what I mean by 'too much carrot.'  Another issue with RMAT is that the criteria by which the designations are given (or not) are not clear." 

Next Thursday, the governing board of California's $3 billion stem cell agency is scheduled to meet to give away more millions for stem cell research in the Golden State. The agency will also be examining the non-scientific considerations that it uses in deciding which applications to fund. It may be that some of the issues raised by these four researchers will also come into play.