financing
Eli Lilly invests in Flagship-founded biotech
From my colleague Allison DeAngelis: One of the biotechs founded by venture creation firm Flagship Pioneering has brought in Eli Lilly as an investor as it advances a collection of treatments for immune conditions, cancer, and obesity.
The startup, Ampersand Biomedicines, announced this morning that it raised another $65 million from Flagship, Lilly, and others. The company is pursuing preclinical studies of two drugs this year and starting up a second obesity R&D program.
This latest deal was made through Flagship's business unit, Pioneering Medicines. (Pioneering Medicines also helped broker an obesity partnership between Ampersand and Pfizer under an overarching $7 billion deal it signed with the pharmaceutical company in 2023.)
CEO Jason Gardner was mum on the differences between the two obesity programs, citing the competitive landscape, and declined to specify what Eli Lilly's interest in the company is. In case you forgot, Eli Lilly, maker of the drug Zepbound, is one of the leaders in the obesity field.
Ampersand hopes to develop medicines that more accurately work at the site of disease and minimize off-target effects. It acquired an antibody drug discovery company last year and has developed a computational platform to assess potential therapies, but hasn't revealed much more beyond that. The company has not disclosed which immune therapies or forms of cancers its two lead drug candidates are targeting.
biotech
Cargo Therapeutics ends development efforts
Cargo Therapeutics, a company focused on CAR-T therapies, said yesterday that it will shut down its development efforts and lay off 90% of its staff.
In January, Cargo said it would discontinue a Phase 2 study of its lead CAR-T candidate, after the treatment showed little long-term efficacy and significant safety concerns. At the time, Cargo said it would focus on advancing its earlier-stage programs, but it's now shutting those down, too.
The company said it will appoint Anup Radhakrishnan, currently the COO and CFO, to be interim CEO to guide the company through a reverse merger or other business combination.
Cargo was founded by two highly acclaimed cell-therapy researchers. The company raised over $500 million to develop next-generation cell therapies, particularly for patients who have relapsed after receiving one of the CAR-T treatments already on the market.
But after the lead asset failed, Cargo became what my colleague Adam Feuerstein would call a "biotech zombie," a company that has no more future but continues to exist with cash on hand. These types of companies are a widespread problem in biotech, he argues, and are contributing to the bleak state of biotech investing. Read more of what he previously wrote on that.
GENE-EDITING
Prime and Beam, despite a deal, pursue rival therapies
The gene-editing field may have yet another legal squabble on its hands — this time between two companies created by the same star biotech researcher, David Liu.
Prime Medicine yesterday announced it was developing a gene-editing treatment for a debilitating lung and liver disorder called alpha-1-antitrypsin deficiency (AATD). The issue is that six years ago, Prime appeared to sign away its rights to create AATD treatments to another company it has long collaborated with, Beam Therapeutics.
The deal was meant to prevent Prime and Beam from competing and to encourage them to use their tools as broadly as possible. Each announced large pipelines, collectively aimed at over 25 different diseases. Now, though, each appears to have AATD in its sights.
Read more from STAT's Jason Mast.
gene therapy
Patient dies on Sarepta therapy, raising questions for FDA
Sarepta Therapeutics said yesterday that a 16-year-old boy died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy. The teen, who had lost the ability to walk due to his disease, suffered liver failure, a side effect that has been seen with other gene therapy treatments. This was the first known death linked to the therapy, which has so far been given to over 800 patients.
The death renews questions around the FDA's process for approving gene therapies. Elevidys was intensely debated within the agency, with officials and reviewers disagreeing over how strong the evidence was for approval and whether it warranted making the drug available for patients facing a fatal disease with few other options.
Peter Marks, the FDA's head of biologics, ultimately overruled reviewers and lower-level officials to grant traditional approval to younger children and accelerated approval to older children who have lost the ability to walk. He did this even though Sarepta studied Elevidys largely in young children and the risks of gene therapy tend to escalate with age.
Analysts at Baird wrote that the key question for the field now is whether this will lead to a decrease in regulatory flexibility. "Now that we've seen the safety risks associated with these novel therapies rear their head yet again, we expect investor focus will be squarely on how the regulators respond moving forward," they wrote.
Cowen analyst Ritu Baral wrote that while she doesn't think this will lead to the revocation of Elevidys' accelerated approval in patients who have lost the ability to walk, she thinks it may slow uptake in that population.
Shares of Sarepta fell 27% yesterday.
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