| | | |   | This is Meghana here welcoming you back for another week. Today we're talking about Ukraine's biochemistry sector, a pivotal hearing for Amylyx, and costly gene therapies. | | | What to know about the Amylyx ad comm An FDA advisory committee will meet at the end of the month to discuss an experimental drug from Amylyx for ALS. It's the first time the panel — known as the Peripheral and Central Nervous System Drugs Advisory Committee — will meet since the controversy over Aduhelm, Biogen’s therapy for Alzheimer's. And while the Amylyx hearing is unlikely to raise as much of a hubbub, it's expected to be closely watched.
The drug, AMX0035, is a combination of two older drugs, and designed to prevent motor neurons from dying prematurely. It’s been tested in a single Phase 2 clinical trial, and achieved its main endpoint of slowing ALS progression compared to placebo. The drug showed smaller improvements over placebo on secondary measures of muscle and respiratory function that were not statistically significant.
STAT's Adam Feuerstein lays out everything you need to know. Read more. | Ukraine’s biochemistry sector — and scientists — under attack Ivan Kondratov, a researcher at Ukraine-based medicinal chemistry firm Enamine, has had to shelve his lab work in the wake of the war with Russia. The company is one of the major providers of chemicals used in pharmaceutical labs around the world: Ukraine has been an important global supplier of unique compounds used for drug development.
“I’m pretty sure that most drug discovery projects which led to development of drug candidates and drugs during the last five years, more or less, have dealt with our products and services,” Kondratov told STAT, speaking from a town in northwest Ukraine where he’s sought refuge during the attacks. Now, instead of building upon the firm’s catalog of 20 billion synthetic compounds, he and many other scientists are under attack. “I just got the messages from them that they’re sitting in the shelters, sitting underground, listening for the bombs, and it’s terrible,” Kondratov said of friends, coworkers, and family. Read more. | Biotech Outlook 2022: Investor sentiment runs high on innovation After a historic run in 2020, biotech had a difficult year in 2021. The pendulum, however, may be ready to swing back. Innovation is expected to continue at a rapid pace. Innovation around new drug targets, design, and manufacturing, as well as delivery of advanced cell and gene therapies and expansion into new diseases particularly in the cancer, neurology and rare disease spaces, are expected to drive enthusiasm for the industry in 2022. Get the outlook. | Will Medicaid pay for gene therapies? Gene therapies are on the cusp of approval, with sickle cell treatments from Vertex Pharmaceuticals and Bluebird Bio the closest to the finish line. But they will be very expensive — and out of reach for many people if they can’t get covered by Medicaid.
The disease affects about 100,000 Americans, and is particularly prevalent among Black people. About 40% of patients are covered by Medicaid, and it’s a costly disease already — the total cost of hospital stays for the disease was $811.4 million in 2016. The hope is that a gene therapy, costly though it may be, might help defray the lifetime costs of living with sickle cell. “Sickle cell is a miserable life for patients and it would be wonderful if we have the ability to intervene,” said Gary Puckrein, leader of a nonprofit that advocates for health care equity in high-risk racial and ethnic communities. “But it could be very, very difficult for Medicaid programs to pay for these medicines.” Read more. | CMS chief calls on Congress to lower drug prices Chiquita Brooks-LaSure, the Biden-appointed official who oversees CMS, met with New York government officials and reporters to advocate for lower drug prices. She called on Congress to push through legislation that might make medication more affordable — such as the Build Back Better Act that would allow Medicare to negotiate lower drug prices. But it’s very unclear whether this sort of policy would ever pass as law. Some senators have taken issues with portions of the bill, particularly Kyrsten Sinema of Arizona. “We’ll continue to use every lever at our disposal to ensure access to affordable, comprehensive, person-centered care, including affordable medication,” Brooks-LaSure said. Read more. | More reads - Commissioner Califf needs to put the F back in FDA. (STAT)
- ‘Haven’t we learned anything?’: Experts warn of disastrous consequences if pandemic funding dries up. (STAT)
- The other Sunny Balwani. (The Information)
| Thanks for reading! Until tomorrow,  | | | |
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