Amylyx and the ALS aftermath

Friday was an unfortunate, ground-shifting day for the ALS community. The failure of Amylyx Pharmaceuticals' drug Relyvrio to show any benefit over a placebo in a large clinical trials will almost certainly lead to the withdrawal of the medicine from the market.

The disappointment of Relyvrio's failure is another painful reminder of just how few effective therapies exist for ALS. Physicians are now scrambling to advise patients about what to do with a medicine that doesn't work. And then there are broader questions: Did the FDA extend its policy of "regulatory flexibility" too far in approving Relyvrio on equivocal data? How, if at all, will this setback affect the approval of drugs for other rare diseases? And what happens to Amylyx now that its only source of revenue and profit appears to be going away?

autoimmune disease

After fundraise, Alumis drug does well in psoriasis

Right on the heels of closing an impressive $259 Series C, San Francisco startup Alumis reported that its lead drug candidate ESK-001 helped alleviate psoriasis in a Phase 2 trial. The data, presented at a late-breaking session of this week's American Academy of Dermatology conference in San Diego, showed that patients taking the highest dose of the drug saw a 75% improvement in their psoriasis compared to placebo.

The drug, an allosteric tyrosine kinase 2 inhibitor, performed comparably to Sotyktu, a similar medicine made by Bristol Myers Squibb that was approved in 2022, FierceBiotech writes. In a few months, Alumis will use its new funding to launch Phase 3 studies for ESK-001 in psoriasis; it's also testing the drug in lupus and an inflammatory eye disease called uveitis.

biosecurity

Preventing the weaponization of AI-designed proteins

Researchers are calling for artificial intelligence-mediated protein engineering to be handled in a safe and ethical manner, Nature writes. There's concern that they might be used as bioweapons — though for now, the potential benefits outweigh the dangers.

"The best way of defending against AI-generated threats is to have AI models that can detect those threats," one biosecurity expert said. More than 100 scientists have signed the manifesto, which is called "Responsible AI x Biodesign." It commits signatories to 10 tenets, which include conducting research solely for the benefit of society, and creating better DNA synthesis screening so that hazardous biomolecules can be detected before they are manufactured.

animal models

New online marketplace to sell lab animal parts

A new online marketplace called aRukon is set to launch globally this year to help researchers sell unused animal samples to other labs, Science writes. The idea is to potentially reduce waste, save money, and prevent the unnecessary death of animals.

The platform was conceived by Javier Burgos, a biomedical researcher at Jaume I University in Spain. After researching neurodegenerative disease for 20 years, he found himself "in front of an open freezer, faced with the challenging decision of determining which samples to discard," he said. "Panic sets in when the freezer reaches full capacity."

aRukon, which will be free to access, is launching with funding from Spain's State Research Agency. Researchers will be able to list animal parts for sale, from cerebrospinal fluid to whole organs. Sellers can set their own prices, but the concept is to discourage profit.

multiple system atrophy

Lundbeck's MSA drug is promising, analysts say

Lundbeck's experimental anti-alpha-syn monoclonal antibody drug slowed disease progression of multiple system atrophy up to 27%, data presented at this week's AD/PD conference in Portugal show. Leerink analysts consider the data promising — even though the study missed the primary endpoint. Volumetric MRI analysis showed that the loss of white matter volume slowed down, which is encouraging in an aggressive, neurodegenerative disease.

"Overall, the consistent improvements across different outcomes suggest a real efficacy signal and support advancement of the drug into Phase 3 development in this area of high unmet need with no drug approved," they write.

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