biotech
The gene therapy field can't escape FDA concerns
At a conference held by the Alliance for Regenerative Medicine in Rome last week, the focus was supposed to be on opportunities for new therapies in Europe, but instead, the regulatory instability in the U.S. was top of mind.
It shows how much the field has been shaken by the rejections and reversals made under former gene therapy regulator Vinay Prasad.
While the FDA rejection rate for cell and gene therapies was 18% from 2020 to 2024, it rose to 38% over the following 15 months, Tim Hunt, CEO of the Alliance, said during his opening remarks.
The shift at the FDA “really puts an extreme, academic, methodological purity over the needs of the patient community, patients that are gravely ill, and really ignores regulatory flexibility and a lot of the tools that Congress has pulled forward,” he said.
Read more from STAT's Drew Joseph.
pharma
Industry's reputation clouded by pricing concerns
The pharma industry's overall reputation among patient groups ticked up last year, but patients have become more concerned about how sufficiently drugmakers are addressing patient needs, according to an annual survey conducted by a research group called PatientView.
Even though a slightly greater percentage of patients groups reported that drugmakers had an “excellent” or “good” reputation this year compared with last year, the groups expressed greater concern over access to medicines, innovation, and generating products that benefit patients.
Read more from STAT's Ed Silverman.
financing
Cancer biotech raises $220M for myeloma drug
CellCentric said today that it has raised an oversubscribed $220 million Series D round to fund the development of its candidate for multiple myeloma.
The British biotech's drug, called inobrodib, is an oral p300/CBP inhibitor. The financing will support an ongoing Phase 2 study and a Phase 3 trial the company plans to start in the second half of this year.
About a year ago, CellCentric had also raised a significant amount — $120 million in a Series C round.
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