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Good morning! Welcome to our overhauled, brand-new weekend edition. While it looks a little different, the best parts are the same. We’ll still deliver can’t-miss stories straight to your inbox every Saturday morning. But we’ll do it in a slightly more conversational way. As always, our goal is keeping you informed — and we’re always open to feedback. We hope you enjoy!
Let’s dive into this week’s biggest stories.
A startup founded by gene therapy pioneer Jim Wilson announced an important win in a disease area that’s been clouded by a major setback that occurred 25 years ago. The company, iECURE, claims its experimental treatment likely spared a baby boy with a debilitating genetic disease from needing a liver transplant.
Endpoints News’
Ryan Cross has the exclusive on the first-of-its-kind treatment.
Plus, Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, plans to step down after seven years at the agency. Zachary Brennan has the latest.
Several members of our team will be
on the ground
in San Francisco next week for #JPM25, so keep an eye out for their coverage. And if you spot them on a cable car, be sure to say hi! —
Nicole DeFeudis
TOP HEADLINES THIS WEEK
🧬A Jim Wilson-founded startup is claiming it has cured a young boy born with a debilitating genetic disease.
The startup, iECURE,
developed an experimental gene therapy
that edited the boy’s genome inside his body, using an enzyme to cut DNA at a safe location and then have a virus slip in a new copy of the correct gene.
What condition does the boy have?
The infant was born without an enzyme called ornithine transcarbamylase, or OTC. It’s a deficiency that affects roughly 100 newborns in the US and 1,000 babies worldwide each year, making them unable to break down protein and causing ammonia in the blood to skyrocket. It’s also the same disease that nearly derailed Wilson’s career 25 years ago, when a patient died from an immune reaction to being given an experimental gene therapy.
👶How is the boy doing now?
Clinicians administered the therapy when the boy was 6.5 months old after stabilizing him on hemodialysis, a strict diet and an ammonia-reducing drug called Ravicti. They saw signs the therapy was working after three months and began weaning him off Ravicti and introducing a regular diet. Now, six months out from treatment, his ammonia levels appear normal for his age.
Are there any caveats?
Yes, as the sample size for this treatment is still N of 1. Durability remains the biggest unknown, and iECURE still hasn’t proven that the treatment’s benefit is due to the edit integrating itself into the infant’s genome. Safety could still remain an issue, as the boy experienced a severe elevation of liver enzymes in the blood one month after the infusion. But he recovered within a month after taking immunosuppressive drugs.
💰What’s next for iECURE?
CEO Joseph Truitt said the best-case scenario is an approval in 2027, though that is an ambitious timeline. The company plans to treat
three more infants
with the same dose in the first half of this year.
👩🔬Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, is stepping down.
After seven years at the agency, Cavazzoni’s
last day will be
Jan. 18. The announcement follows the departures of device center chief Jeff Shuren and principal deputy commissioner
Namandjé Bumpus
. That means Martin Makary, president-elect Donald Trump’s
pick to lead
the FDA, would have a say in filling a handful of key roles at the agency.
Who’s stepping in?
In the meantime, Jacqueline Corrigan-Curay, CDER’s principal deputy center director, will serve as acting CDER director, pending clearance.
What’s next for Cavazzoni?
The director said her decision to leave CDER was “extremely difficult,” but that it’s time to “be more present for my family, who have taken the backseat over the past few years due to the demands of my role and our critically important public health work.”
💵Verdiva Bio broke cover on Thursday with $411 million to advance a portfolio in-licensed from Sciwind Biosciences.
The company’s
lead program
is a once-weekly oral GLP-1 candidate called ecnoglutide.
Who are the key players?
Much of Verdiva’s team hails from Aiolos Bio, which
launched
last year around an asthma drug from China-based Hengrui and was
sold to GSK
just months later for $1.4 billion. This time, Verdiva CEO Khurem Farooq says the team is in it for the long haul, noting that the startup’s investors “have deep pockets.” The company is backed by Forbion, General Atlantic, RA Capital, OrbiMed, Logos Capital, Lilly Asia Ventures and Lyfe Capital.
🇨🇳The company joins a growing list of startups developing drugs that were discovered or initially developed in China.
This week, Timberlyne Therapeutics and Ouro Medicines
also emerged
with assets from China-based Keymed Biosciences.
Experimental therapies from Google’s Calico and Denali Therapeutics both failed in mid-stage trials this week.
The drugs had been
trying to slow
patients’ ALS progression after six months. Calico’s saw some potential trends indicating it could still be tested in future studies, while Denali’s program missed all its secondary endpoints.
🧺Both drugs were being tested in a basket study at Massachusetts General Hospital.
The goal of the study is to examine more ALS drugs more quickly, but none of the seven drugs tested so far have hit their primary endpoints. Mass General plans to lengthen the primary analysis timeframe from six to nine months, believing a longer time period will be more appropriate to see whether the drugs can affect the disease.
🐢The long-struggling biotech will cleave itself in two and lay off staff,
according to a press release from this week. Both of the companies will be
publicly traded
and headcount will be reduced by 40%. Galapagos will continue to focus on advancing cell therapies for cancer, while the new company — currently called SpinCo — will attempt to spin some deals to build a pipeline in oncology, immunology and virology.
DON’T MISS
