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Good morning, everyone. Damian here with some hotly anticipated news from Vertex Pharmaceuticals, a closer look at the latest FTC controversy, and a detailed look at what it’s like to actually receive a gene therapy.
The need-to-know this morning
- Pfizer provided financial guidance for 2024 that fell well below Wall Street expectations. Shares are trading lower in the pre-market session.
- Alnylam Pharmaceuticals is meeting with investors and analysts today to discuss the company’s R&D programs and outlook.
Vertex non-opioid drug reduces chronic nerve pain
Vertex Pharmaceuticals said Wednesday that an experimental drug reduced pain in people with diabetes who have chronic nerve pain — mid-stage study results that support the biotech company’s efforts to develop an effective painkiller without the addictive potential of opioids.
The drug, a pill called VX-548, is designed to precisely block pain-sensing neurons from signaling the brain. It’s the most important, and closely followed, drug candidate in Vertex’s research pipeline because pain is both crippling and common. It’s the leading cause of disability worldwide, and chronic pain affected a fifth of U.S. adults in 2021, according to the Centers for Disease Control and Prevention.
If VX-548 is approved for both acute and chronic pain, it could become a future blockbuster, adding billions of dollars in revenue on top of Vertex’s successful and highly profitable stable of cystic fibrosis treatments.
Maze CEO: FTC challenge ‘frustrating and disappointing’
Jason Coloma, CEO of Maze Therapeutics, found out about the demise of his company’s partnership with Sanofi the same way most people else did: by reading the French company’s press release on Monday.
As STAT’s Adam Feuerstein reports, Coloma said he was “personally disappointed” in Sanofi’s decision to back out of the agreement after the FTC challenged its deal with Maze. The two companies were moving forward with an early-stage treatment for Pompe disease, a rare disorder for which Sanofi markets two approved medicines.
“I came from pharma so I understand there are business decisions that need to be made and a rationale that we’re not going to be privy to,” Coloma said. “I’m not angry, per se, just more disappointed that we didn’t get to see it through.”
Meanwhile, at ASH
The annual meeting of the American Society of Hematology just finished up in San Diego, where STAT’s Angus Chen and Jonathan Wosen reported from the ground. You can find all of their work here, and don’t miss these highlights from the meeting:
- Johnson & Johnson’s Darzalex, already a blockbuster therapy in multiple myeloma, succeeded in a study enrolling newly diagnosed patients, improving results over the course of four years compared to a standard three-drug treatment. “This is now going to affect the community quite considerably,” said Joseph Mikhael, chief medical officer of the International Myeloma Foundation. “I think this will clearly become the standard of care.”
- A targeted therapy from Syndax showed positive results for patients with a hard-to-treat form of leukemia. The drug, revumenib, charted a 63% response rate for patients with relapsed or refractory leukemia with KMT2A genetic rearrangements. Many of those patients were able to receive a potentially curative stem cell transplant later on, which is often the ultimate goal for patients with relapsed or refractory patients.
A plot to solve the ‘whack-a-mole’ of drug discovery
Turning a promising idea into a drug that might work for people is an iterative process, requiring chemists to make scores of tiny tweaks to a given compound and then run test after test to see how it’s working, a process that can cost millions of dollars and a number of years.
Inductive Bio, a startup making its public debut today, wants to streamline that process with the help of technology. The company has amassed a trove of data, from public sources and proprietary work, on how small chemical tweaks affect the metabolism, absorption, and toxicity of investigational drugs. Inductive trained its machine learning platform on those data, and the product is a software that can predict the results of key assays and address “the nitty-gritty whack-a-mole problem” of drug discovery, said Josh Haimson, Inductive’s co-founder and CEO.
“The value comes in saying, rather than making 2,000 compounds to get to your development candidate, we can use these machine learning models to stack the deck in your favor, and maybe you can make 600 or 1,000 compounds instead,” Haimson said.
The company has about $4 million in seed funding from a syndicate that includes Andreessen Horowitz and Lux Capital. Inductive has signed deals with research organizations and biotech companies, including Denali Therapeutics, and is in the process of pitching more. That means explaining clearly what Inductive’s technology can do while steering clear of the hype that often accompanies machine learning.
“There is a true revolution happening in these technologies, but it’s not a silver bullet,” he said. “Nothing is. Drug discovery is a really hard business.”
What it’s like to get a gene therapy
Gene therapy is, technically, a one-time medicine. But that doesn’t account for the weeks of pretreatment, prolonged hospitalization, and months of recovery required for what can ultimately be a curative therapy.
As STAT’s Brittany Trang reports, the Campbell family has been living through that process since their daughter, Shelby, became one of the first people in the U.S. to receive a gene therapy for the rare blood disorder beta thalassemia. “We have hope that this is a miracle cure,” Shelby’s mom, Michelle Campbell, said a few months before the administration, “but I’m not exactly looking forward to it.”
By the time Shelby’s “one-time” treatment has concluded, she’ll have spent seven weeks in the hospital, received toxic chemotherapy to wipe out the defective cells, lost her hair, suffered high fevers and mouth sores, and been isolated from friends and unable to attend school for more than seven months while her immune system recovers.
More reads
- Could sickle cell someday be treated not with CRISPR, but with a pill? STAT
- AstraZeneca to pay up to $1.1 billion for vaccine developer, Bloomberg
- Bone marrow transplant advance could widen use of this less buzzy cure for sickle cell disease, STAT
- Moderna commercial officer steps down, CEO to look over vaccine sales, Reuters