June 30, 2023

The need-to-know this morning

French drugmakers Ipsen and Genfit announced positive results from a Phase 3 study of elafibranor in participants with primary biliary cholangitis, a rare liver disease.
Bausch + Lomb is paying $1.75 billion to Novartis to acquire Xiidra, an approved treatment for dry eye disease, the Wall Street Journal reports. It's the first big transaction by Bausch CEO Brent Saunders since he returned to the eye-care product company in March.
Aurinia Pharmaceuticals, maker of an approved treatment for lupus, said its board will explore "strategic alternatives to maximize shareholder value."

gene therapy

The 'lost heroes' of Duchenne muscular dystrophy

It's been bittersweet for many families ever since Sarepta won approval for its groundbreaking gene therapy for Duchenne muscular dystrophy. Some have advocated for decades to find new treatments for the disease — knowing fully well that their own children would never see the benefit.

These "legacy moms" have children who have died, or whose disease has progressed past the point where new therapies might help. Many, however, spent their lives in research studies to help treatments like Sarepta see approval. Sometimes the studies were successful, but oftentimes not. Despite fervent efforts from families to help children with this debilitating disease, many times, it just didn't work out.

"It's a lot of joy, a lot of pride," one mother and Duchenne advocate said, describing to her emotions. "But I didn't pay the ultimate price, my son did. And life is getting hard for him."

gene therapy

Biomarin's hemophilia gene therapy finally wins approval

The FDA yesterday approved Roctavian, BioMarin's gene therapy for hemophilia A. The treatment was rejected by U.S regulators in 2020, despite data showing that it reduced bleeding episodes and helped patients have fewer blood transfusions. But the FDA wanted more data on the one-time infusions' durability and safety. European regulators, on the other hand, approved Roctavian last August.

"Today's approval of Roctavian builds on BioMarin's proven track record of advancing treatments that target the underlying cause of life-threatening genetic conditions," Biomarin's CEO said in a statement.

proxy fight

Alkermes shareholders vote against activist investor's board plans

Alkermes shareholders have voted to re-elect all of the company's current directors — ending its proxy battle with a hedge fund that wanted to choose three of its own. The result is a setback for Sarissa Capital Management, which owns 8.5% of the drugmaker and is owned by activist investor Alex Denner. Sarissa accused Alkermes leadership of running the company poorly and cratering shareholder value.

Denner wanted not his own seat on the board, but to choose two other members, to wrest more control of the company. But shareholders clearly don't agree with his plan.

podcast

Can Novavax finally get it right?

What's a "triple-G" drug? And is Novo Nordisk losing ground? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Elaine Chen joins us to explain how the era of Wegovy could be short-lived in light of powerful new weight-loss medicines from Eli Lilly. Then, John Jacobs, the new CEO of Novavax, stops by to explain his plan to right the ship at a company that has struggled to make the most of its potent Covid-19 vaccine.

Listen here.

insider trading

Pfizer statistician indicted over Paxlovid-based insider trading

Pfizer statistician Amit Dagar has been charged by the SEC with insider trading, based on his early knowledge of the "game changer" results of a Paxlovid trial for Covid-19. Dagar, who had been a senior statistical program lead for the trial, allegedly traded Pfizer stock before results became public. The results showed that Paxlovid reduced hospitalization or death by 89% compared to placebo in high-risk adults — leading to Pfizer's stock skyrocketing 11% — its largest single-day price move since 2009.

Dagar, who worked at Pfizer from 2017 through January of this year, brought Pfizer call options, including some that expired the next day. He also tipped off a friend, who did much the same. This allowed Dagar to earn nearly $214,400 in profit, and his friend gained $60,300.

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