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Return of the biotech mega-round, contending weight-loss pills, & Intercept on the brink

May 23, 2023
National Biotech Reporter
Hello, all. Damian here with news of a rarified biotech fundraise, the brewing competition among obesity treatments, and a pioneering company at a crossroads.

The need-to-know this morning

  • Wave Life Sciences is halting development of a gene-silencing treatment called WVE-004 due to negative results from a placebo-controlled study that showed no clinical benefit for patients with a genetic form of ALS and/or frontotemporal dementia. 

  • Affimed, a German biotech developing cell therapies for cancer, laid off 25% of its workforce as part of a restructuring plan.

Venture Capital

The dream of 2021 is alive in biotech

While biotech venture capital investments are trending at a six-year low, it's still possible to raise a $300 million Series A round.

As STAT's Allison DeAngelis reports, ReNAgade Therapeutics has done just that, with MPM Capital's BioImpact Capital and F2 Ventures co-leading what is the biggest single biotech Series A investment so far this year. 

As ReNAgade's name suggests, the focus is on RNA medicines. Since its inception, the company has been developing novel lipid nanoparticles to ferry RNA strands into specific tissues, working to overcome the limitations of current technologies. The company has also started a joint venture with another of MPM's portfolio companies called Orna Therapeutics, which is developing a new form of genetic medicine called circular RNA.

Read more.



Obesity

Novo Nordisk can't make enough semaglutide

And the problem will only get worse if the company wins FDA approval for an oral version of its blockbuster weight-loss drug.

Yesterday, the company said a tablet formulation of Wegovy led to roughly 15% weight loss over 17 months in a study enrolling people diagnosed with obesity. That effect is comparable to weekly injections of the drug, and Novo Nordisk expects to submit the oral version for U.S. and EU approvals later this year.

The problem is that Novo Nordisk is struggling to meet demand for Wegovy as it is, which could make commercializing oral semaglutide impossible in the short term. Further complicating matters, it takes a lot of semaglutide to replicate Wegovy's effects with a tablet. The weekly injection uses just 2.4 mg of the active ingredient, while the oral one requires a daily dose of 50 mg. 

Read more.


In the lab

And that could benefit Pfizer

As Novo Nordisk struggles to meet demand for Wegovy, Pfizer is making progress on a pill that promises to be similarly effective and markedly easier to make.

It's called danuglipron, and unlike semaglutide, a peptide treatment that requires specialized manufacturing, it's an old-fashioned small-molecule drug designed to do the same thing. And it seems to be working. Yesterday, Pfizer published results from a study enrolling about 400 patients with Type 2 diabetes in which high doses of danuglipron led to reductions in blood sugar and body weight comparable to semaglutide.

Pfizer's stock price rose 5% on the data, adding more than $10 billion to the company's valuation. The company still has a lot of work ahead before danuglipron could win approval, first for diabetes and then obesity. But the pill's very existence, alongside potential Wegovy competitors from Amgen and Eli Lilly, makes clear that Novo Nordisk's continued dominance of the market is hardly a given.


Biotech

Should Intercept just give up on NASH?

A single press release made Intercept Pharmaceuticals the hottest biotech company in the world. In 2014, a week before the J.P. Morgan Healthcare Conference, Intercept said its treatment for the liver disease NASH had worked so well that its mid-stage clinical trial was stopped early. The stock price quadrupled, and so began a pharmaceutical gold rush to find what would be the first treatment for the increasingly prevalent disease.

Nine years and a 98% stock price decline later, it's becoming clear Intercept's drug will not achieve that milestone. After Friday's decisively negative public hearing, the FDA seems almost certain to reject Intercept's application for accelerated approval next month, meaning the company would have to spend the next three years collecting enough data to apply for a traditional approval.

And that might not be worth the wait, Intercept CEO Durso said. On a conference call with analysts yesterday, Durso said Intercept is "prepared to pivot to profitability" if it doesn't win approval in June, meaning the company that put NASH on the biotech map might stop investing in the disease altogether. 


More around STAT
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More reads

  • Achieve Life Sciences drug helps smokers quit in trial but would face stiff competition if approved STAT
  • What does generative AI mean for health care? We asked the experts, STAT
  • Novo gains after doctors find heart risk drops on Wegovy, Bloomberg
  • Seagen criticized by shareholder advisory firm for 'problematic' executive compensation ahead of Pfizer acquisition, STAT
  • Hepion's NASH drug meets main goal in mid-stage trial, Reuters

Thanks for reading! Until tomorrow,


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