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Welcome back to another edition of Endpoints Weekly! We hope you all have been staying safe indoors as the Canadian wildfires blanketed much of the US Northeast this week. Hopefully things calm down over the weekend!
Jumping into the headlines, we saw Biogen report data for its tau-targeting Alzheimer’s drug at a conference in London this week, showing some promising signals on a difficult target despite the trial technically failing. An upcoming Phase 3 program will be closely watched across the industry, as the mid-stage data raised many interesting questions. Elsewhere, two notable biotechs debuted this week, there was another Eli Lilly deal, and we saw some activity at the FDA. Be sure to also check out our Q&A with Betty Tijms, head of science at the Alzheimer Center Amsterdam. Have a great weekend! — Max Gelman |
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Max Gelman |
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Senior Editor, Endpoints News |
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Biogen’s mixed Alzheimer’s data |
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🧠Many biopharma companies are researching whether targeting a protein in the brain called tau can result in benefits for Alzheimer’s disease patients. Individuals with both tau pathology and buildup of amyloid plaque almost always progress to having Alzheimer’s, but tau has been more difficult to drug. In London this week at the annual Alzheimer’s Association International Conference, Biogen presented some of the strongest data yet for experimental anti-tau
drugs.
But the results were marred by the strange lack of a dose response, senior science correspondent Ryan Cross writes from across the pond. Patients who received the lower dose of Biogen’s drug, called diranersen or BIIB080, slowed cognitive decline by 26% compared to placebo. Those who got the larger dose only had their disease slowed by 14%. Both treatment groups received diranersen every six months via spinal injection. When the higher-dose cohort got the injection every three months, the slowing of cognitive decline was only 9%.
None of these results were statistically significant, and the trial was a technical failure. However, the lower dose’s response was comparable to the benefits shown by approved anti-amyloid drugs Leqembi and Kisunla (cross-trial caveats notwithstanding). Despite this, there were too few patients in that group for the result to reach significance, as only 60 patients received it compared to 115 in each of the higher-dose groups.
So what’s next? Biogen and its partner Ionis both said diranersen appears to be hitting tau harder than any prior experimental drug, and they called it the first drug to show that lowering tau can have clinical benefit. But this week’s results will likely delay a Phase 3 program, as Biogen had fast-tracked the Phase 2 trial around the higher dose. Neither company shared specific details about plans for the Phase 3 yet. Read more from Ryan here.
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Two new startups launch |
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💰The first is led by the former team behind Akero Therapeutics. The company, called Avere Therapeutics, unveiled on Tuesday with a pipeline drug from China and a reverse merger to go public on the Nasdaq. It’s a familiar strategy: Find a drug candidate in China, court an experienced executive team, and win over big-name investors to back the effort, Kyle LaHucik writes. Avere is focused on a clinical-stage oral IL-23 receptor antagonist
(AVR-001) licensed from China’s Hansoh Pharmaceutical for $120 million upfront. It hopes to create a once-weekly oral IL-23 to compete with Johnson & Johnson’s once-daily Icotyde.
Meanwhile, a secretive startup that’s working on a new generation of lipid nanoparticles raised $89 million. Until recently, LNPs were only proven to work as local injections for vaccine delivery or as an infusion that shuttled drugs to the liver. Nava Therapeutics has been creating and screening new libraries of lipids that could deliver medicines to other parts of the body. The startup is led by Federico Mingozzi, the former chief science and technology officer of Spark
Therapeutics — the first company to win FDA approval of a gene therapy. He said he wants to bring LNPs “to places where they haven’t been before.” Nava’s first destination is the in vivo CAR-T field, where it hopes to launch its first clinical trial in autoimmune disease in the first half of 2027. You can read more from Ryan Cross here. |
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A Q&A with Betty Tijms |
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🔎The head of science at the Alzheimer Center Amsterdam believes it’s time to stop treating Alzheimer’s as a single condition. Tijms discovered five distinct subtypes by analyzing thousands of proteins found in the spinal fluid of people with the disease, and presented an update to that ongoing work this week during a plenary talk at the Alzheimer’s Association International Conference in London. She told Ryan Cross that she hopes her work will lead to better treatments — if drugmakers are willing to grapple with the complexity of the disease. “The idea that you can have one disease but different underlying mechanisms, that’s very new,” she told Ryan. You can read the full conversation here. |
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Lilly acquires AtaiBeckley for $2.8B upfront |
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🤝Eli Lilly continued its dealmaking spree by diving head-first into the world of psychedelic treatments. The company announced this week that it would acquire AtaiBeckley for $2.8 billion upfront, a 40% premium to AtaiBeckley’s average share price over the previous 30 days. The biotech’s lead drug for treatment-resistant depression has started Phase 3 “activities,” Lilly said. AtaiBeckley shareholders are also eligible
for up to $1 billion for certain milestones.
The move bolsters Lilly’s neuroscience portfolio, which features its Alzheimer’s treatment Kisunla and its recent purchase of the sleep disorder biotech Centessa. For years, Lilly had a significant presence in the antidepressant arena with Prozac, but the drug has been generic for some time. Former Denali Therapeutics executive Carole Ho joined Lilly in November last year as president of its neuroscience division. Read more here.
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FDA acting chief walks back practice of policymaking through journals |
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The FDA said two medical journal articles published over the last year did not constitute agency policy. The about-face formally addresses one of the main criticisms of former Commissioner Marty Makary and his practice of instituting policy by posts, podcasts and articles rather than formal rulemaking, Max Bayer wrote this week. In written responses to questions from Rep. Diana DeGette (D-CO) obtained by Endpoints, Acting Commissioner Kyle Diamantas said that the two articles, written by agency officials — including one co-authored by
Makary — were not actually guidance. One of those articles, written by Makary and former CBER chief Vinay Prasad in the New England Journal of Medicine, detailed the plausible mechanism pathway. The agency has since released formal guidance on that pathway, stemming from a Rare Disease Innovation Hub workshop the same month. |
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