Sponsored by    


The Readout Damian Garde & Meghana Keshavan

Now that ESMO, Europe's largest cancer meeting, has ended, join STAT's Adam Feuerstein later today for a live conversation with oncology experts on the latest promising data, what's still to come in 2020, and any other questions you might have. Sign up here.

J&J’s Covid-19 vaccine has something its closest competitors lack

Johnson & Johnson is starting a Phase 3 trial of its Covid-19 vaccine today, making it the fourth company to do so. But unlike its nearest rivals, J&J has a vaccine that may require just one dose instead of two, which could be a differentiation in the months to come.

As the Boston Globe reports, J&J is planning to enroll up to 60,000 volunteers in the U.S. and abroad to test its vaccine, which uses a harmless virus to deliver a key protein found on the surface of SARS-CoV-2. Like its competitors, the company plans to conduct regular analyses of the vaccine’s safety and efficacy as the trial progresses, opening the door for an early conclusion if it appears especially effective.

J&J’s Phase 3 trial is about two months behind those of Moderna and the partnership of Pfizer and BioNTech, and it trails AstraZeneca’s U.S. study, which remains on hold after a safety issue. Each of those vaccines requires two administrations a few weeks apart. That means J&J’s one-dose vaccine, if it works, could inoculate twice as many people and avoid the risk that patients don’t return for a second vaccination.

Blueprint’s cancer drug might have a second life in rare disease

Blueprint Medicines has had a mixed record of clinical success with Ayvakit, an FDA-approved cancer drug. But new data suggest the treatment has a bright future as a treatment for a rare disorder that causes unpredictable allergic reactions.

In a pair of clinical trials, Avykit led to a roughly 75% response rate for patients with advanced systemic mastocytosis, a genetic disease that can cause life-threatening complications. In one study, the median length of response was more than three years. The company plans to file for an expanded FDA approval later this year, and analysts at Cowen expect Avykit to become the standard of care for advanced cases of the disease.

The news sent Blueprint’s share price up about 12% yesterday, in part because it bodes well for Avykit’s potential as a treatment for indolent systemic mastocytosis, a less severe and more common form of the disease. The drug appears safer than many investors inspected, according to SVB Leerink analyst Andrew Berens, which suggests that an ongoing study in indolent systemic mastocytosis, which uses a substantially lower dose, shouldn’t run into problems with toxicity.

Biotech’s much-debated lottery ticket got a little more complicated

Bristol Myers Squibb’s $74 billion acquisition of Celgene came with a sweetener: Every share of Celgene entitled investors to a voucher that turns into $9 if certain conditions are met and turns into dust if even one of them is not. The latest news seems evenly weighted toward profit and peril.

The key cogs are a pair of Celgene-owned CAR-T therapies, which need to win approval on certain timelines for the voucher to pay off. Yesterday brought good news for bb2121, one of the CAR-T treatments: The FDA accepted its approval application and set a decision date of March 27, which comes in right under the wire. But the news for the other treatment, which must be approved by the end of the year, was more confusing. Bristol Myers said the FDA was yet to inspect its CAR-T manufacturing facilities and hadn’t scheduled a date to do so, which could be a sign the agency is going to blow the deadline.

Those $9 vouchers are publicly traded, and the latest twists dragged their value down to around $2 each. That sets up one of the starkest risk-reward profiles in all of biotech: Investors could more than quadruple their money or walk away with absolutely nothing, and there’s no outcome in between.

Introducing ‘Vertex 3.0’

Vertex CEO Reshma Kewalramani, roughly six months into her tenure, sees the storied biotech company at an inflection point, with the chance to prove that its promise extends beyond a series of pioneering medicines for cystic fibrosis.

“Vertex 1.0,” Kewalramani said in an interview with the Boston Globe yesterday, came during the company’s salad days of trying to bend medicinal chemistry into more-effective pills. The second era came in the last decade, when Vertex successfully developed what are now that standard-of-care treatments for CF. With 2020 comes “3.0,” she said, in which “Vertex moves from a CF-only company to a CF-and company.”

The ands in question are medicines for sickle-cell disease, pain, Type 1 diabetes, and muscular dystrophy, among other investigational drugs. Each is in either early or mid-stage development, but “these diseases, with all humility, I do think we are going to transform just like we did CF,” Kewalramani said.

More reads

  • MIT researcher Regina Barzilay, who pivoted to health care after her breast cancer diagnosis, wins new $1 million AI prize. (STAT)
  • FDA to announce tougher standards for a coronavirus vaccine that make it unlikely one will be cleared by Election Day. (Washington Post)
  • Evolution of the biotech IPO markets from busted to booming. (LifeSciVC)
  • Novartis sells bonds tied to expanding access to medicines in poor countries. (STAT Plus)

Thanks for reading! Until tomorrow,

Wednesday, September 23, 2020


Facebook   Twitter   YouTube   Instagram

1 Exchange Pl, Suite 201, Boston, MA 02109
©2020, All Rights Reserved.
I no longer wish to receive STAT emails
Update Email Preferences | Contact Us
5cP.gif?contact_status=<<Contact Status>>