Insmed, Asahi Kasei, Agios, Royalty Pharma

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Hello, biotech friends. As we prepare for ASCO, we’re thinking about some interesting questions. For example, gene therapy has great potential to cure certain forms of congenital deafness, but does the community actually want that? We also see an affirmative process fail in Duchenne, and more.

The must-know this morning

• An experimental drug from Insmed has successfully reduced lung problems in patients with respiratory disease in a closely watched Phase 3 trial, sending the company’s stock price soaring early this morning.
• The Japanese conglomerate is trying to build up its pharmaceutical business Asahi Kasei buys Swedish biotech Calliditas Therapeutica in a cash deal worth nearly $1.1 billion.
• Agios pharmaceutical products one announced $905 million purchase agreement of 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 preliminary results from a phase 1 study.
• Johnson & Johnson pays $1.25 billion for this acquire a privately owned experimental bispecific antibody in development for atopic dermatitis Numab therapies.

ASCO studies to watch

ASCO’s annual meeting starts later this week and the abundance of new data can be a bit overwhelming. That’s why we’re highlighting some studies of particular interest while we await news from STAT’s Adam Feuerstein, Matthew Harper and Angus Chen, who will be on the ground in Chicago. (Sign up here for our daily newsletter ‘ASCO in 30 seconds’.)

We would like to know more about some impressive CAR-T results from AstraZeneca and its Chinese partner AbelZeta in patients with advanced liver cancer. There is also data from Australia showing that doctors may be able to avoid 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!

Read more.

Will gene therapy ‘endanger’ the deaf community?

The deaf community is closely connected and known for its vibrancy. But as gene therapies are introduced that can cure some forms of congenital hearing loss, an old question is resurfacing: Do deaf people want a “cure”?

Existing gene therapies only exist for a small portion of the deaf population, but some experts are betting that they could eventually be used in perhaps half of all congenitally deaf people. And that worries some bioethicists and community members.

“For the signing and deaf community to survive, we need a critical mass of people,” a deaf philosophy professor told STAT’s Timmy Broderick. “What happens if the number of deaf people drops to a low enough point? What is the impact of that technology?”

Read more.

Confirmatory trial against Duchenne fails for exon skipper

The Duchenne muscular dystrophy drug Viltepso failed to meet its primary endpoint in a confirmatory trial. Although the children who received the drug were able to get up from the floor more quickly by the end of the study, the children in the placebo arm of the study were also able to do so – 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 are designed to increase the amount of dystrophin proteins in these patients, who are unable to produce them themselves. Sarepta’s exon-skipper drug, Exondys 51, was first approved in 2016 thanks to tireless lobbying efforts by parents of children with the disease, but has yet to complete confirmatory trials for the drug.

Read more.

The Merus drug, combined with immunotherapy, increases 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-phase clinical trial.

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

Petosemtamab has received significant attention, especially from biotech investors, for its potential to improve the treatment of head and neck cancer, the sixth most common cancer in the world. Currently, patients with metastatic but newly diagnosed head and neck cancer are typically treated with Keytruda, Merck’s anti-PD-1 immunotherapy, or a combination of Keytruda and chemotherapy.

Read more.

New financing on our radar

Just to highlight a few interesting fundraising events from the past week:

Halda Therapeutics is two-thirds of the way to increasing a $36 million round, according to the registration files. The Yale spinout is developing bifunctional cancer molecules, which it has trademarked “RIPTAC,” which use ligands to link proteins together to target tumor cells and “hold and kill” them. Halda emerged from stealth a year ago with $76 million in funding.

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

We noted some larger rounds: Pheon Therapeutics brought one Series B of $120 million for its antibody-drug conjugate drugs for cancer. And another obesity player, SixPeaks Bio, came out of stealth mode $110 million in funding – with a possible $80 million from AstraZeneca.

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• Takeda lays off 641 employees in Massachusetts Boston sphere
• FDA panel votes against Novo Nordisk’s weekly insulin in patients with type 1 diabetes, Reuters
• Even after a sharp decline in sales, Pfizer sees the bright side with Paxlovid’s priority review voucher, FiercePharma