November 20, 2024

The need-to-know this morning

Argenx said it will advance a subcutaneously injected form of its drug Vyvgart into Phase 3 studies for the treatment of myositis, an autoimmune disease that attacks muscles. The decision was based on positive results from a mid-stage study.
AI drug developer Recursion Pharmaceuticals closed its merger with Exscientia.
Sage Therapeutics said it is discontinuing the development of SAGE-718, the only remaining drug in its clinical research pipeline, following negative results from a study to treat cognitive impairment in patients with Huntington's disease.
Hoth Therapeutics bought up to $1 million in bitcoin as a treasury reserve asset.

pharma

Pfizer named a new Chief Scientific Officer

Pfizer on Wednesday promoted its Chief Oncology Officer Chris Boshoff to head all R&D at the company, STAT’s Matthew Herper writes. Boshoff is taking over for Mikael Dolsten, who served as chief scientific officer for 15 years and announced his retirement in July.

Some investors may be disappointed that Pfizer went in-house for a top executive, instead of recruiting a fresh voice from the outside. Boshoff has been at Pfizer for 11 years, and has been the face of the company’s cancer franchise — including its $43 billion acquisition of Seagen last year.

“Chris has a compelling vision for the future of R&D at Pfizer and deep knowledge of our entire pipeline and R&D organization that positions him well to succeed,” CEO Albert Bourla said, in a statement.

cardiovascular

Investors tell Silence Therapeutics to shush

From STAT’s Matthew Herper: On Tuesday, Silence Therapeutics presented what seemed like positive data on zerlasiran, its experimental drug for reducing cardiovascular risk, at the annual meeting of the American Heart Association. Then, its stock fell 36%.

Investors had several worries. Zerlasiran is an RNA-silencing drug that reduced levels of Lp(a), a cholesterol-related compound thought to increase the risk of heart attacks and strokes, by 80%, according to the study data presented. It is given by injection.

But at the same AHA meeting, Eli Lilly presented positive study results on its own Lp(a)-lowering drug. That drug, called muvalapin, is given orally — a potential advantage.

Two patients in the zerlasiran also had elevated liver enzyme levels, although levels of bilirubin, another blood test result used in measuring liver safety, were not elevated. A risk of liver toxicity weighing on zerlasiran might make it more challenging for Silence to find a Big Pharma partner willing to help pay for the expensive development of the drug.

Analysts at BMO Capital Markets defended Silence, writing, “We believe the stock reaction is overdone and see no safety or competitive threat concerns.” On Wednesday, at least, investors disagreed.

government

Pharma execs unruffled by RFK, Jr.

Pharma executives at the Jefferies London Healthcare Conference seemed nonplussed over President-elect Trump choosing Robert F. Kennedy, Jr. as health secretary. For example, Bristol Myers Squibb CFO David Elkins said that “what we do as an industry is we work with both sides,” and that “it doesn’t matter who’s in office.”

The incoming Trump administration will likely be beneficial for many companies, just as its first iteration was. Even vaccine makers aren’t particularly concerned: “We have to have our eyes open when it comes to risk assessment,” one BioNTech exec said, in regards to RFK Jr.’s views on vaccines. “But we don’t see any direct policy proposal or direct threat to the Covid business.”

drug discovery

Schrödinger CEO calls AI drug discovery ‘nonsense’

Schrödinger CEO Ramy Farid says his company isn’t just another AI company. Founded back in 1990, he says it’s a physics-based molecular modeling outfit that uses machine learning — but doesn’t rely on it exclusively. The company just secured a $2.3 billion deal with Novartis, a testament to how lucrative its physics-forward approach happens to be.

“You cannot build training sets for machine learning only using experiments,” he told STAT’s Brittany Trang — adding that it’s “actually nonsense” to rely solely on AI for drug discovery.

“If you can build a large enough training set, machine learning will work, and the only way to build a large enough training set is using physics,” he said.