biotech
Vertex stumbles as it looks to new phase of growth
Vertex Pharmaceuticals reported disappointing first-quarter earnings yesterday. It saw weaker-than-expected sales of its cystic fibrosis drugs and little or no contributions from a gene therapy for sickle cell disease and a recently launched pain medicine.
Vertex also said it paused an early study involving a closely watched, inhaled, mRNA-based therapy for cystic fibrosis due to a “tolerability issue.”
The results suggest that the company, which has been trading near its all-time, will have a longer way to go before its next leg of growth.
Read more.
artificial intelligence
Recursion cuts nearly half of its pipeline
From my colleague Allison DeAngelis: When AI drug company Recursion Pharmaceuticals merged with Exscientia last year, it gave the company an influx of cash — always handy to have, in this languorous biotech winter — and a pipeline of 11 drugs in clinical and preclinical development. But yesterday, Recursion revealed that it's culling nearly half of its pipeline.
The company axed three clinical programs — one of which, a drug for cerebral cavernous malformation, underwhelmed in Phase 2 data reported last year. The company had steered investors' focus to its next-generation compounds, including a treatment for the common bacterial infection known as C. difficile. But that drug was also discontinued yesterday, along with a drug for neurofibromatosis and a preclinical drug.
Recursion is also pausing work on a drug for solid tumors.
“Since we did the integration, the portfolio has grown and we said that we would actually make disciplined decisions to sharpen our focus,” the company's R&D and commercial chief, Najat Khan, said during the company’s first quarter earnings call. “These moves reflect a clear commitment to a high bar on differentiated medicine… while also contributing to capital efficiency by reallocating these precious resources towards the highest potential opportunities.”
Investors reacted negatively to the news. The company’s stock fell more than 16% yesterday. It’s also a setback in Recursion's ambition to develop 100 drugs — a goal the CEO stood by last year.
biotech
Another gene therapy for vision loss fails
From my colleague Jason Mast: J&J said late last week that a gene therapy it was developing for a rare form of vision loss failed in a clinical trial. Although patients with X-linked retinitis pigmentosa who received the therapy were, after a year, better able to navigate a maze than those who received a sham injection, the difference was not statistically significant, the company said in an update to providers.
The failure for J&J, which bought the treatment from a biotech for $130 million in “upfront and near-term milestone payments” in 2023, adds to a growing list of disappointments for eye disease gene therapies.
When Spark Therapeutics' Luxturna, designed to treat another form of hereditary blindness, was OK’d in 2017 as the first FDA-approved gene therapy, it sparked hopes that similar technology could be used to treat potentially hundreds of different eye disorders. Yet today Luxturna remains the only ocular gene therapy on the market.
Biogen spent $800 million in 2019 and then failed in the same disease, along with a condition called choroideremia. A gene-editing treatment from Editas failed. Other drugs have shown some promising early data, but investment has been limited by the rarity of most inherited forms of vision loss.
science
Biopharma execs stress the need for NIH funding
As the Trump administration makes cuts both to the funding that NIH gives out to institutions and to the NIH itself, several biopharma executives yesterday spoke of the importance of maintaining robust public funding to ensure the U.S. maintains competitive in biomedical innovation.
Amgen's chief scientific officer, Jay Bradner, said on a panel at the the Milken Institute Global Conference that funding from the NIH “is the very foundation that all of our medicines are built from.” It fuels basic science research, and so it acts as “a force multiplier in a way that research and development funding and private industry really can't be,” he said.
“At this moment of unprecedented opportunity, we can't take our foot off the gas pedal. We'll lose our competitive edge,” Bradner added.
Neil Kumar, CEO of BridgeBio, said he thinks it's worth reassessing how NIH funding is distributed to different universities, given that it seems more funding has been concentrated in select institutions. But, he also said, “we need the magnitude of capital to remain the same. Opportunities from it will be profound.”
These comments came as researchers experience disruptions at the NIH Clinical Center, a hospital that aims to run trials for the hardest-to-treat diseases, my colleagues report. Read more on that here.
regulation
Gottlieb's plan to stem Chinese drug licensing deals
U.S. drugmakers are increasingly licensing drugs from China, and in order to stop this shift, U.S. regulators should ease the requirements for companies to be able to start clinical testing, Scott Gottlieb, former FDA commissioner, writes in a new opinion piece.
One of the biggest hurdles for drug companies is the animal testing that the FDA requires before Phase I studies, Gottlieb says. While he supports the recent move by current commissioner Marty Makary to phase out animal testing, he also expresses concern that the agency won't be able to develop new alternative tools to assess drugs given recent cuts to the FDA's workforce.
“The imperative to modernize early-stage drug development — to ensure that groundbreaking drug discovery remains in the U.S. rather than migrating to China — is colliding head-on with an impulse to slash the very government workforce capable of spearheading these reforms,” he writes.
Read more.
