| |   | Good morning, all. Damian here with some promising developments in immunotherapy, a vexing regulatory saga, and the latest frontier for Right To Try. | | | What does it mean when the FDA says no? For all of 2022, the investor debate around Axsome Therapeutics has focused not on whether its drugs work but on how to interpret what seem like unpredictable twists in the FDA approval process. And the latest one adds yet more uncertainty to Axsome’s volatile year.
The news is that Axsome now expects the FDA to reject AXS-07, its experimental migraine treatment, later this month after getting word that the agency has some unanswered questions about how the drug is produced. Analysts expect only middling revenue from Axsome’s migraine drug, but the company’s share price fell about 20% yesterday on fears that the company’s FDA problems could affect a different and potentially more lucrative treatment in its pipeline.
AXS-05, Axsome’s therapy for major depressive disorder, has been through a back-and-forth FDA saga of its own and could win approval this quarter. The company has repeatedly said that there’s no read-through between each drug’s rocky path through the regulatory process, but the market apparently thinks otherwise. We’ll find out soon enough. | Promising early results for a new way to hit ‘reset’ on the immune system Doctors have treated leukemia and lymphoma patients with bone marrow transplants for more than 50 years. But these cancer-killing infusions come at a cost: Donor immune cells sometimes attack a patient’s healthy cells and tissues, a response known as graft-versus-host-disease. In some cases, that friendly fire is deadly.
California-based Orca Bio has come up with a strategy it thinks is safer and more effective. The company’s experimental therapy, known as Orca-T, includes T regulatory cells, which control and dampen runaway inflammation.
Yesterday, the biotech announced fresh results, reporting that 5% of trial participants given the therapy had moderate-to-severe graft-versus-host disease within a year of their transplant, compared to 38% of patients given a traditional transplant. And 90% of patients who got Orca-T were still alive a year later, compared to 68% of those who didn't. The company’s now launching a randomized Phase 3 trial to see if those findings hold up. | Join STAT on May 3 in Boston to explore the challenges, and the unique opportunities for new approaches, solutions, and partnerships, of the drug development process. This in-person program will bring together a diverse group of individuals to share their perspectives about the drug development process. Stay once the conversation is complete for a networking and cocktail reception with STAT and your fellow attendees. Get your ticket now (and save 40% as a STAT+ subscriber). | Pfizer, Valneva to advance Lyme vaccine in adults, kids Pfizer and partner Valneva announced yesterday that their experimental Lyme disease vaccine, the only such vaccine in development, will be tested in children at the same time as it is tested in adults.
The companies said that a Phase 2 study in children between the ages of 5 and 17 showed the three-dose vaccine was as safe in children as it had been in adults, and that it generated an even stronger immune response. The companies plan to start a large Phase 3 study aimed at showing that the vaccine prevents volunteers from being infected with the Borrelia burgdorferi bacteria, which causes Lyme.
About 30,000 cases of Lyme are reported annually to the Centers for Disease Control and Prevention, but the agency says based on other methods that the annual number of cases in the U.S. may top 500,000. The B. burgdorferi bacterium is spread through the bite of blacklegged ticks. Symptoms include fever, headache, and a characteristic skin rash. Untreated, the bacteria can damage the joints, the heart, and the nervous system.
The Pfizer-Valneva vaccine is composed of proteins manufactured in e. Coli that generate an immune response to six of the most common types of the bacterium. Unlike most vaccines, this one, called VLA15, does not affect the B. burgdorferi bacteria when it is in the human body, Annaliesa Anderson, Pfizer’s chief scientific officer for bacterial vaccines, said in an interview. Instead, the antibodies produced by the vaccine become active only when the tick takes its blood meal. It is only then that the vaccine’s target, outer surface protein A (OspA), is vulnerable. Pfizer and Valneva’s Phase 3 test will be the first test of the approach in two decades. The vaccine would likely need to be given annually, because immunity would wane, Anderson said. | The next Right To Try battleground is ultra-orphan drugs The libertarian think tank behind the federal Right to Try law has chosen a new target: ultra-orphan drugs designed to treat a single patient’s rare genetic disorder. But it remains unclear whether current regulations are actually standing in the way of so-called N-of-1 therapies.
As STAT’s Nicholas Florko reports, the latest legislation, soon to become law in Arizona, would give patients immediate access to such customized medicines regardless of whether they’ve been tested in clinical trials, doing away with what proponents say is a slow and unwieldy process.
But experts in the field say it sounds like a solution in search of a problem. Stanley Crooke, the founder of Ionis Pharmaceuticals who now leads an N-of-1-focused nonprofit, said that while the current process could move faster, there’s no need for such deregulation.
Read more. | More reads - Pharma R&D payments to European researchers are like ‘dark money.' STAT+
- Black Diamond lays off 30% of its workforce and culls a drug from its 'MasterKey' chest. Endpoints
- FDA approves Gilead's Covid-19 drug remdesivir for young children. Reuters
| Thanks for reading! Until tomorrow,  | | | |
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