PTC, Dyne, Rapport, Cytokinetics news

May 20, 2024

77 4 minutes read

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Hello! Today, we talk about how Bernie Sanders might be hitting a wall with the drug industry, see a glimmer of potential for HIV vaccines, and introduce a new Monday fundraising feature.

Also, please tell us what you think of The Readout.

The need-to-know this morning

PTC Therapeutics said the European Commission has requested a re-evaluation of a previous decision made by European drug regulators to remove its treatment for Duchenne muscular dystrophy, called Translarna, from the market.
Dyne Therapeutics reported clinical updates on two drugs being developed to treat myotonic dystrophy type 1 and Duchenne muscular dystrophy, respectively.
Rapport Therapeutics, a developer of drugs for neurological diseases, filed for an initial public offering.

Bernie Sanders vs. Novo. Who wins?

Bernie Sanders may finally be hitting a limit in his considerable efforts to badger the pharmaceutical industry about high drug prices. As the head of a Senate health committee that focuses on the subject, he’s now demanding that Novo Nordisk cut prices for its blockbuster GLP-1 drugs. And that seems unlikely.

“There is absolutely no reason to think that these manufacturers are going to respond to Sanders’ criticisms,” one conservative-leaning economic policy expert told STAT’s Rachel Cohrs Zhang.

On a recent investor call, Novo executives said the full value of medicines like Ozempic hasn’t been realized yet, and that prices would naturally decline as competition heats up. Sanders recently wrote an op-ed in a Danish newspaper asking the country to crack down on Novo Nordisk, and released a report on how much its weight-loss drug would cost if more adults would start taking it. But the bully pulpit “can’t do everything,” a pricing advocate told STAT.

Cytokinetics wants to stay independent

Cytokinetics CEO Robert Blum is committed to independently commercializing aficamten, should the heart drug achieve approval. Phase 3 results showed the drug’s efficacy in treating obstructive hypertrophic cardiomyopathy — perhaps even more so than a similar treatment made by Bristol Myers Squibb, STAT’s Elaine Chen writes. This has led to speculation about whether the company will be acquired, or if it will license the medicine to larger pharma players that have more resources.

Speaking at a talk during STAT’s Breakthrough Summit West, however, Blum said the plan remains “to build a commercial business. And that’s not something new. That’s not something that’s the result of a failed M&A dialogue.”

Animal studies show potential for HIV vaccine strategy

Resilience and mutability have allowed HIV to dodge efforts to develop an effective vaccine for decades. But four Science studies describe new efforts at teaching the immune system — in mice and monkeys — to produce strong antibody responses that block many viral strains by exposing them to different vaccines.

In one study, researchers used engineered proteins to activate specialized B cells in rhesus monkeys so they produced antibodies that could latch onto the HIV virus. It’s unclear whether these antibodies could actually protect against infection and disease — but it’s an encouraging first step.

“In most vaccines, you’re trying to get a common antibody response, it’s kind of like if there’s a jar of jelly beans and it has one gold — and that’s the one you’re trying to get,” one of the study’s authors told STAT. “But for a hard problem like HIV, instead of a jelly bean jar, you’re trying to find that one gold jelly bean in a swimming pool.”

What’s new in fundraising

As a new Monday feature, we’re highlighting interesting fundraises we found while trawling recent SEC filings.

Here’s one: A stealth Boston-area startup called Progentos Therapeutics has raised an impressive $68.6 million. The CEO on file is Christopher Loose, a Yale entrepreneurship lecturer. Notably, he cofounded MIT spinout Frequency Therapeutics, which focused on reverse hearing loss, and also catheter company Semprus BioSciences with MIT’s Robert Langer.

Progentos may be focused on multiple sclerosis, according to another SEC filing in March — showing that Progentos acquired the MS program from Frequency Therapeutics, which merged last year with Korro Bio. Loose was chief scientific officer of Frequency, which also had a preclinical remyelination program underway for multiple sclerosis.

Also of interest? Looks like Breakout Ventures is raising its third fund. We don’t yet know how much, but the company has backed several founder-led biotech companies. Its second fund rolled out in 2021, using its $112.5 million to focus on “creative biosciences startups.” Its first was in 2017, for $60 million.