Karuna Therapeutics Inc. unveiled data from the phase III study called Emergent-3 with Karxt (xanomeline plus trospium) in adults with schizophrenia. The trial met its primary endpoint, with Karxt turning up a statistically significant and clinically meaningful 8.4-point reduction in Positive and Negative Syndrome Scale (PANSS) total score compared to placebo (-20.6 Karxt vs. -12.2 placebo) at the fifth week (Cohen’s d effect size of 0.60). Consistent with prior trials, Karxt yielded an early and sustained statistically significant reduction of symptoms from the second week through the end of the trial as measured by PANSS. Shares (NASDAQ:KRTX) were trading midday at $186.05, down $5.58, or 2.9%.

Biogen adcom on rare ALS drug could be a win despite phase III stumble

Despite a failed phase III study, the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee said data suggests tofersen (BIIB-067) is effective for treating the rare, genetic disease superoxide dismutase 1 amyotrophic lateral sclerosis. The intrathecally injected therapy is being developing by Biogen Inc. and Ionis Pharmaceuticals Inc. and is at the heart of the upcoming March 22 adcom meeting. The study failed to show a statistically significant difference between the tofersen and placebo groups for the primary or secondary endpoints in the prespecified primary analysis. The FDA accepted the NDA and granted priority review in July 2022, initially awarding a PDUFA action date of Jan. 25, 2023, and later extending it by three months to April 25.

WDC 2023: Entering a new era in preventing and treating dementia

If you believe the theme of the World Dementia Council (WDC) meeting in London this week, dementia is “in a new era”, where it will be possible to prevent, diagnose and treat neurodegenerative disease. That is not the case for most people living with dementia today, but the approval of the first disease-modifying drugs and the imminent arrival of new blood-based biomarkers is “a big moment,” Lenny Shallcross, executive director of WDC told the meeting on Mar. 20. For decades, Alzheimer’s and other forms of dementia have been poorly diagnosed and untreatable. To a certain extent is has been possible to manage symptoms, but not to change the trajectory of the disease.

BIO-Europe Spring 2023: Despite funding woes, deals can be made for biotechs willing to weather storm

Are deals such as M&As between biotechs and big pharma becoming a thing of the past? That was a key question posed during the opening keynote at this year’s BIO-Europe Spring conference in Basel, Switzerland. Although Susanne Kreutz, global head of corporate and business development at Novartis AG, doesn’t think this is the case, she told delegates that she believes M&A will increasingly focus in on “high-quality, high-impact, late-stage assets, where reimbursement is securable and where regulatory paths appear.” Meanwhile, in 2023 and beyond, there will be “a massive emphasis” on partnering, Kreutz believes.

AAD 2023: Takeda’s TAK-297 hits endpoints in phase IIb psoriasis trial

Takeda Pharmaceutical Co. Ltd.’s oral allosteric tyrosine kinase 2 inhibitor TAK-297 (formerly NDI-034858) met primary and secondary endpoints in a phase IIb clinical trial in patients with moderate to severe plaque psoriasis, but analysts say it may be too little too late to make a big splash compared to competitors. In the phase IIb study, 259 patients were randomized to receive one of four doses of TAK-279 once daily, or placebo for 12 weeks. A significantly greater proportion of patients achieved psoriasis area and severity index (PASI)-75 at doses greater than 5 mg at 12 weeks with TAK-279 compared to placebo.

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