May 28, 2024

The need-to-know this morning

An experimental drug from Insmed successfully reduced lung problems among patients with an airway disease in a closely watched Phase 3 trial, sending the company's share price soaring early this morning.
Seeking to build up its pharma business, the Japanese conglomerate Asahi Kasei is buying Swedish biotech Calliditas Therapeutics in a nearly $1.1 billion cash deal.
Agios Pharmaceuticals announced a $905 million purchase agreement with Royalty Pharma for rights to royalties on sales of the cancer drug vorasidenib.
An inhaled, mRNA-based drug from Arcturus Therapeutics improved lung function in patients with cystic fibrosis, according to a preliminary results from a Phase 1 study.
Johnson & Johnson is paying $1.25 billion to acquire an experimental bispecific antibody under development for atopic dermatitis from privately held Numab Therapeutics.

ASCO

ASCO studies to watch

The annual meeting of ASCO starts later this week, and the glut of new data can be a bit overwhelming. So we're highlighting some studies that are of particular interest, as we await news from STAT's Adam Feuerstein, Matthew Harper, and Angus Chen, who will be on site in Chicago. (Sign up for our daily "ASCO in 30 seconds" newsletter here.)

We're interested to learn more about some impressive CAR-T results from AstraZeneca and Chinese partner AbelZeta in patients with advanced liver cancer. There's also data from Australia showing that clinicians may able to bypass chemotherapy in some patients with stage 2 colorectal cancer. We're intrigued by an AI-based patient navigator that helps patients access colonoscopy. And more!

gene therapy

Will gene therapy 'endanger' the deaf community?

The deaf community is closely knit and known for its vibrance. But now that gene therapies are being introduced that can cure certain forms of congenital hearing loss, an old question is reemerging: Do deaf people want a "cure?"

Existing gene therapies only exist for a small slice of the deaf population, but some experts wager they could ultimately be used in perhaps half of all congenitally deaf people. And that concerns some bioethicists and members of the community.

"For the signing and deaf community to continue to exist, we need to have a critical mass of people," one deaf philosophy professor told STAT's Timmy Broderick. "What happens if the number of deaf people dwindles to a low enough point? What is the impact of that technology, then?"

rare disease

A Duchenne confirmatory trial fails for exon-skipper

Duchenne muscular dystrophy drug Viltepso didn't meet its primary endpoint in a confirmatory trial. Although the children who were given the medicine could stand up faster from the floor at the end of the study, the children in the placebo arm of the trial could do so as well — with no statistically significant difference between the groups.

The treatment, made by Japanese drugmaker Nippon Shinyaku, is part of the controversial group of drugs called exon-skippers. They're designed to increase the amount of dystrophin proteins in these patients, who lack the ability to produce them on their own. Sarepta's exon-skipper drug, Exondys 51, was first approved in 2016 thanks to tireless lobbying efforts from parents of children with the disease — but has yet to complete confirmatory trials for the drug.

asco

Merus drug, with immunotherapy, boosts tumor response in patients with head and neck cancer

Merus said this morning that the combination of its experimental drug petosemtamab with the checkpoint inhibitor Keytruda shrank tumors in 62% of patients with head and neck cancer, according to an interim analysis of an ongoing mid-stage clinical trial.

The new efficacy results, derived from a larger number of patients, look similar to an initial disclosure made last week that triggered a 36% increase in Merus' stock price.

Petosemtamab has generated considerable attention, particularly from biotech investors, due to its potential to improve the treatment of head and neck cancer, the sixth most-common cancer worldwide. Currently, patients with metastatic but newly diagnosed head and neck cancer are typically treated with Keytruda, the anti-PD-1 immunotherapy made by Merck, or a combination of Keytruda and chemotherapy.

fundraising

New financings on our radar

Just highlighting a few interesting fundraises from the past week:

Halda Therapeutics is two-thirds of the way into raising a $36 million round, regulatory filings show. The Yale spinout is developing what it has trademarked "RIPTAC" bifunctional molecules for cancer, which use ligands to link proteins together to target tumor cells and "hold and kill" them. Halda came out of stealth a year ago with $76 million in financing.

We've also learned of a $22 million Series A round from Valar Labs, which uses AI to predict responses to cancer therapies. The funding was co-led by Andreessen Horowitz and DCVC; earlier this month, Valar released data showing that its histology test could predict a patient's response to BCG in high-risk non-muscle invasive bladder cancer.

Some larger rounds we noted: Pheon Therapeutics raised a $120 million Series B for its antibody-drug conjugate drugs for cancer. And another obesity player, SixPeaks Bio, came out of stealth with $110 million in funding — with potentially $80 million from AstraZeneca.

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