Sanofi’s Promising Cancer Drug Fails Intermediate Clinical Trial
Sanofi had forecast the medicine to be one of its upcoming "blockbusters," but a testing setback disappoints hopes. In other pharma news: Medicaid coverage for sickle cell treatment; how the war in Ukraine disrupts drug development; gene-editing technology; and more.
Bloomberg:
Sanofi Cancer Drug Fails Test In Blow To Possible Blockbuster
Sanofi’s experimental cancer medicine amcenestrant failed in an intermediate clinical test for breast cancer, dealing a blow to one of the French drugmaker’s potential future blockbusters. The patients who got the drug, rather than endocrine treatment, didn’t live longer without the disease progressing -- a key benefit measure known as progession-free survival -- in the phase 2 trial, Sanofi said Monday. The stock fell as much as 6.2% in Paris trading, the steepest drop in almost two years. (Fourcasde and Loh, 3/14)
In other pharmaceutical and biotech industry news —
Stat:
Will Medicaid Patients Be Able To Access Gene Therapies For Sickle Cell?
Gary Puckrein sees a dilemma coming that has him excited, and at the same time, worried. In the next year or so, U.S. regulators may approve gene therapies – one developed by Vertex Pharmaceuticals, and another by Bluebird Bio – to treat sickle cell disease, an inherited group of blood disorders that affect an estimated 100,000 Americans. The condition is particularly prevalent among Black people, with 1 of every 365 Black individuals in the U.S. born with the condition, which can cause significant pain, infections, and lead to an early death. Any treatment would be welcome news, especially since a gene therapy holds the promise of eradicating the illness with potentially just one treatment. But gene therapies are widely expected to come with a high price tag — and it’s estimated that roughly 40% of sickle cell patients are covered by Medicaid. That is raising concern that state Medicaid officials may soon face a struggle for coverage. (Silverman, 3/14)
Stat:
In Ukraine, He Was Creating The Building Blocks For Drug Developers
It was just last month, with the Covid-19 situation having calmed down in Kyiv, that Ivan Kondratov had been able to return to his office a couple days per week. He managed a slew of medicinal chemistry projects, including the building of “target libraries” for clients, mostly large biopharma companies around the world. His employer, Enamine, had become a pillar of global drug development, its Rolodex of clients steadily growing along with its catalog of chemical compounds for drugmakers to test in creating potential treatments. But on Feb. 24, all of that came to a halt as Kondratov and hundreds of thousands of other Ukrainians were stunned awake by the sound of explosions. And everything changed for the chemists and scientists. (Cueto, 3/14)
CNBC:
30-Year-Old Female Founder's Billion-Dollar Bet On CRISPR Gene Editing
Along Highway 101 north of the San Francisco Airport, a break-out biotech start-up named Mammoth Biosciences co-founded by Nathan Chen’s sister Janice in 2018 is fast emerging in the revolutionary field of CRISPR technology. While not high profile like her gold medal-winning, ice skating brother — or Mammoth co-founder Jennifer Doudna, who won a Nobel Prize in chemistry for her work on CRISPR — Chen’s bioscience work in gene editing technology is in the forefront of medical discoveries from identifying bacterial and viral infections to early cancer detection. (Fannin, 3/12)
Stat:
What To Know About Amylyx's ALS Drug And A Closely Watched Meeting
Amylyx Pharmaceuticals is one heck of an encore. On March 30, the Food and Drug Administration is bringing together outside experts in neurology to review an experimental drug from Amylyx for the treatment of amyotrophic lateral sclerosis, or ALS. The hearing is expected to be closely watched by ALS patients and their advocates, given the significant need for new treatments for the disease. But the hearing is likely to garner extra attention because it’s the first meeting of the FDA advisory group since it met in November 2020 and voted unanimously against the approval of Aduhelm, Biogen’s drug for Alzheimer’s disease. The FDA later ignored that recommendation and approved the medication, leading to the resignation of three members of the panel and an uproar over whether the agency had compromised its standards. (Feuerstein, 3/14)
And in news from the health care industry —
Modern Healthcare:
Jury Sides With Sutter Health In Federal Antitrust Case
A jury sided with Sutter Health on Friday in the long-running federal lawsuit accusing the health system of anticompetitive business practices that drove up healthcare costs by more than $400 million. The unanimous verdict means Sacramento, California-based Sutter has successfully defended against claims that it illegally forced insurers to include all 24 of its hospitals in their contracts, a practice known as tying. The 10-member jury also found Sutter did not force health plans into contracts that prevented them from steering patients to lower-cost, non-Sutter hospitals. (Bannow, 3/11)
AP:
IU Health, Facing Profit Questions, Gives Med School $416M
Indiana University Health quietly donated $416 million to the Indiana University School of Medicine — a move that comes as the state’s largest hospital system faces pressure to lower its prices and profits. The donation wasn’t announced by either institution, which are separate organizations, but was noted as a “contribution to a related entity” made Dec. 30 in IU Health’s financial statements issued this month, the Indianapolis Business Journal reported. (3/13)
WJCT News:
Legislature Earmarks $80 Million For Renovation Of UF Health Jax Trauma Center
The Legislature is set to give $80 million to UF Health Jacksonville for "critical updates" to its trauma center. The allocation is part of the proposed $112.1 billion budget for the 2022-23 fiscal year that will receive a final vote from both chambers Monday before heading to the governor. The city-owned facility, which is nearly four decades old, partly houses the hospital's emergency department and acts as the only safety net hospital on the First Coast. (Troncoso, 3/13)