Regulatory
The FDA's next neuroscience challenge might be on the way
PTC Therapeutics' treatment for Friedreich's ataxia, a rare neuromuscular disorder, didn't meet its primary endpoint in a Phase 3 study, the company said yesterday. But PTC, citing "signals of clinical benefit," hasn't ruled out submitting it for approval, setting up what could be another thorny debate for the FDA's neuroscience division.
The news is that vatiquinone, an oral medicine, did not significantly improve patients' scores on a rating scale of Friedreich's ataxia symptoms compared to placebo, a failure that sent PTC's stock price down about 20% in after-hours trading. But the study, which enrolled 146 patients, found some benefits on individual symptoms including upright stability and fatigue, and the overall data look much better when you isolate the patients who received the drug for the entire length of the trial, according to PTC.
PTC now has to decide whether to take its chances and file for FDA approval, which would make vatiquinone the latest debatably effective medicine for a serious disease to come before the agency's neuroscience division. What might complicate PTC's case is the February approval of Reata Pharmaceuticals' Skyclarys, a Friedreich's ataxia drug that met its primary goal in a pivotal study, albeit with modest benefits.
Biotech
Novo Nordisk is getting into CRISPR
Novo Nordisk has been making headlines — and ocean liners of cash — from its diabetes and obesity drugs Ozempic and Wegovy. But over the last couple years, the Danish drugmaker, long known for its metabolic disease research, has quietly invested in genetic medicine, buying into experimental treatments for hemophilia and sickle cell disease.
The company is now pairing those two interests. On Wednesday, Novo announced a deal with Life Edit Therapeutics to develop gene editing therapies for both rare genetic disorders and "more prevalent cardiometabolic diseases." The deal, which will focus on base editors that can turn one DNA letter into another, is part of a broader push to bring CRISPR-based technologies, first deployed for cancer and rare conditions, into research for more common ailments.
It also raises an obvious question: Would Novo try to use gene editing for the disease it is most closely associated with, obesity? Karina Thorn, Novo's head of RNA and gene therapies, wouldn't rule it out — or in, for that matter. "We are looking to apply this technology across all of Novo Nordisk's therapeutic interests," she said. "At this point, I can't comment on which exact target or which exact disease."
Millennials
Martin Shkreli's in the news
"How Vivek Ramaswamy helped make Martin Shkreli the 'pharma bro,'" reads this Politico headline, under which we learn that Shkreli has been telling people Ramaswamy was a friend and key investor, while Ramaswamy says the investment in question was merely his former hedge fund holding shares in one of the Shkreli companies that didn't do any crimes.
Each player hits his marks in this story. First Ramaswamy pivots the discussion of Shkreli to the ills of major pharmaceutical firms that operate under "the veneer of 'do-good stakeholder capitalism.'" Then Shkreli enters in his familiar joking-but-also-not-joking character to ask Politico for money in exchange for an interview before offering his own money in exchange for positive coverage of his software company.
All that aside, between Elizabeth Holmes' prison sentence, Shkreli's second act in tech, and Ramaswamy's quest for the White House, what a year it has been for biotech's banner millennial entrepreneurs.
No comments