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The Readout Damian Garde & Meghana Keshavan

Huntington's families pinned hopes on new drugs. Two failed in a matter of days

Last month, Roche announced it was cutting off the dosing of its experimental therapy for Huntington’s disease. Days later, Wave Life Sciences said it was abandoning two Huntington’s therapies in earlier stage trials.

The pair of setbacks have hit the Huntington’s community hard, STAT’s Andrew Joseph reports. As one woman put it, “It was like a sucker punch twice within one week.” There are no treatments for the devastating, progressive disease, and Roche’s treatment, which targeted the underlying cause of Huntington’s, was seen as the most promising medicine in development.

The hopeful news is that there are other experimental therapies in trials or in preclinical studies, and researchers have also vowed to use the information gleaned from the recently failed studies to make progress moving forward.

Read more.

Sage’s tremor drug meets its goal, but not without caveats

Sage Therapeutics said yesterday that its experimental treatment for essential tremor outperformed placebo in a mid-stage trial, but the highest dose of the drug brought intolerable side effects, raising questions about its future.

As STAT’s Adam Feuerstein reports, Sage’s drug led to a 36% reduction in the severity of upper-limb tremor, a statistically significant benefit over the 21% reduction in the placebo group. But Sage’s starting dose of 60 mg was hard to tolerate, with 62% of patients needing to get 45 mg or 30 mg instead and nearly 40% of patients discontinuing altogether.

The results were positive enough to convince Sage and partner Biogen to keep developing the drug, called SAGE-324. Under a deal struck last year, the two companies would split the profits of SAGE-324 and a depression treatment called zuranalone, which is currently in late-stage development.

Read more.

Some stability at CDER, at last

The FDA has now been without a permanent commissioner for a full year. But the agency’s all-important Center for Drug Evaluation and Research now has its permanent leader.

Patrizia Cavazzoni, who was serving as acting director of CDER, was named the permanent director yesterday, officially replacing her predecessor, Janet Woodcock, who is of course acting director of the agency. Cavazzoni, as STAT’s Nicholas Florko noted in a recent profile, has kept a low public profile but has repeatedly demonstrated her willingness to take on some of the agency’s most perplexing problems.

Woodcock’s future remains blurry as ever. Long considered to be in the running for a nomination to the top job, it’s unclear whether she’ll remain at the FDA should she not get it, particularly now that her old one is taken.

Read more.

PhRMA is willing to give an inch when it comes to drug prices

The influential lobbying group PhRMA is trying out a new tack in Washington: Maybe some drug pricing reform is OK.

As STAT’s Nicholas Florko reports, the organization is breaking with tradition by endorsing several policy changes that will actually affect the drug industry’s profits. Those tweaks fall far short of the reforms put forth by some Democratic lawmakers, but PhRMA’s embrace of even several minor changes is unprecedented.

The policy ideas come alongside a new seven-figure PhRMA ad campaign, focused on print and digital platforms, urging Congress not to take the drug industry for granted. The historic speed at which companies developed Covid-19 vaccines has been a boon to pharma’s flagging reputation, and the trade group is looking to leverage some of that goodwill.

Read more.

More reads

  • Europe tries to lower drug prices with small doses of transparency. (STAT+)
  • India approves Russia's Sputnik V Covid-19 vaccine. (Reuters)
  • Microsoft to buy Nuance Communications for $19.7 billion. (Boston Globe)
  • Gene therapies for neurodegenerative diseases: Pfizer, Novartis, Biogen and others seek more from FDA. (Endpoints)
Correction: Yesterday's edition misstated the lead investigator in Regeneron Pharmaceuticals' Covid-19 study. It is Myron Cohen, director of the Institute for Global Health and Infectious Diseases at the University of North Carolina.

Thanks for reading! Until tomorrow,

Tuesday, April 13, 2021

STAT

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