In the pipeline: Drugs for a devastating condition

Whitney Weldon is one of about 800 people in the world with a rare disease known as FOP. (ALICE PROUJANSKY FOR STAT)

For decades, the rare disease known as fibrodysplasia ossificans progressiva, or FOP, was a medical curiosity, a condition without a treatment or even a biological explanation. A small community of patients simply adjusted to grim realities: Their tissue would turn to bone, their joints would freeze, and their life expectancy would be short.
 
Now, however, after decades of work by researchers, three companies may be on the brink of leaping a “tough scientific hurdle,” as one CEO put it, and bringing treatments to market. The most advanced drug could win Food and Drug Administration approval next year.
 
STAT’s Damian Garde has the story of a patient group, some dedicated researchers, and a fair amount of scientific serendipity.

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FDA approves Sage’s postpartum depression drug 

Postpartum depression is the most common complication of childbirth. Yesterday the FDA approved brexanalone, the first drug to treat the condition, which affects 400,000 American women every year but often goes untreated because new mothers can be stigmatized about reporting symptoms.

It’s also the first-ever drug developed by and approved from Sage Therapeutics. But as STAT’s Adam Feuerstein notes, there are challenges that might limit use of the drug, called Zulresso: It's administered via an intravenous infusion over 60 hours and women must be treated at an inpatient facility or hospital where they can be monitored.

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In a 'Vant' first, urology drug looks like a winner  

One star in the "Vant" constellation of companies is suddenly shining brighter. Urovant, the urology spinout from hedge fund manager turned biotech entrepreneur Vivek Ramaswamy, said its overactive-bladder drug hit all its goals in a late-stage trial.

That’s a remarkable turn of events, five years after a spectacular Alzheimer’s drug blowup from Axovant, STAT’s Adam Feuerstein reports. And although Urovant’s lead pipeline drug, vibregon, treats a less common and arguably less challenging condition, there is money to be made: Myrbetriq, marketed by Astellas, hauled in $750 million in sales last year.

Next up for Urovant: revealing more details at a medical meeting, gaining FDA approval, and convincing insurers not to go with a cheaper generic.

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Biotech’s first cryptocurrency is a hard sell

When we last checked in with Agenus at the end of February, the tiny biotech company was pitching investors on a sale of “Biotech Electronic Security Tokens” to finance clinical trials for AGEN2034, an experimental cancer immunotherapy. The buyers of said tokens would be entitled to royalties on future sales of AGEN2034, assuming its approval at some later date.

So, how’s that bio-crypto fundraising effort working out, Agenus?

“We announced an anticipated closing date in March 2019 and the possibility for us to raise up to a maximum of $100 million in the offering. As of the date of this filing, we have not closed on the sale of any tokens,” Agenus disclosed in its annual report filed with the SEC on March 18.

No tokens taken. Perhaps what’s missing is the blockchain.