April 29, 2024

The need-to-know this morning

Ono Pharmaceutical of Japan is buying Boston-based Deciphera Pharmaceuticals, a maker of cancer drugs, for $2.4 billion.
Daiichi Sankyo and AstraZeneca said their targeted cancer drug Enhertu showed a statistically significant and clinically meaningful slowing of tumor progression in a randomized, Phase 3 study involving patients with hormone-positive, HER2-low metastatic breast cancer.
The FDA approved a new treatment from X4 Pharmaceuticals for the treatment of WHIM Syndrome, an immune disorder that leads to serious and frequent infections. The new drug will be marketed under the brand name Xolremdi.
Repertoire Immune Medicines, a startup backed by Flagship Pioneering, announced a research partnership with Bristol Myers Squibb to develop therapeutic vaccines for autoimmune diseases.
Prime Medicine said it received FDA clearance to start the first clinical trial of a therapy based on prime editing, an emerging gene-editing technology. The drug, called PM359, will be investigated in a Phase 1-2 study of chronic granulomatous disease.

Exclusive

Safety issues emerge with MorphoSys blood cancer drug

There's concern over the safety of pelabresib, an experimental treatment made by MorphoSys, STAT's Adam Feuerstein reports. The drug, which is being studied in myelofibrosis, is the linchpin of a $3 billion acquisition by Novartis. Physicians are reporting that patients given pelabresib have rapidly progressed from myelofibrosis, a bone marrow cancer, to acute myeloid leukemia, a more aggressive and deadly blood cancer.

There have been six of these "AML transformation" safety events among 212 patients in the pelabresib arm, compared to two similar events among 214 patients in the control arm. The FDA has been informed of this imbalance in severe cancer progressions, and is monitoring the issue. Regulators have issued a warning letter to all the physicians involved in the study.

It's not yet clear whether this emerging safety issue will impact the drug's submission to the FDA, or if it might lead Novartis to delay or even terminate its acquisition of MorphoSys.

clinical trials

Doctors Without Borders amps up clinical trial transparency

Doctors Without Borders has released the financial details of a tuberculosis clinical trial — a move that it says is unprecedented move and that seems to be a challenge to the pharmaceutical industry. It cost $36 million to test the best treatment approach for multi-drug resistance in tuberculosis in more than 500 patients in three countries.

The charity said that releasing the costs is meant to prevent drugmakers from claiming that high research costs justify high drug prices.

"We know that clinical trials are the most expensive part of R&D," a policy adviser for the nonprofit told STAT's Ed Silverman. "Overall, this is just a first step taken… to show that clinical trial — and full R&D — cost transparency is critically important for pricing and access of medical products. And we have to start somewhere."

Doctors Without Borders has been actively campaigning for drugmakers to lower their TB treatment prices — particularly for bedaquiline, a drug that's become vital to treat the disease.

pandemic preparedness

Intellectual property transparency a priority for pandemic agreement

Countries are in the final stages of negotiating an agreement meant to prevent the next pandemic, but the current draft has watered down IP obligations, opines Loyola University intellectual property expert Cynthia M. Ho. She says that temporarily removing IP barriers could help maximize manufacturing efficiency when another pathogen inevitably sweeps the globe.

"Without that, people around the world will remain unnecessarily vulnerable to forthcoming pandemics — and leaders will be doomed to repeat past mistakes," she writes.

Congress

House bill aimed at Chinese biotechs is advancing

A House committee plans on soon marking up legislation that would prevent U.S. biotechs from working with China's WuXi AppTec, five lobbyists told STAT's John Wilkerson. WuXi, which is specifically named in the legislation, helps many U.S. biotech companies to develop their medicines. But proponents of the legislation say WuXi or any other firm identified as a Chinese "biotechnology company of concern" might threaten national security.

The BIOSECURE Act wouldn't sanction companies that already have contracts with WuXi, nor are the consequences of future business dealings clear. But some think it would prevent companies from Medicare and Medicaid, which would be too big a risk to take.

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