PERTH, Australia – Neuren Pharmaceuticals Ltd.’s stock nearly doubled on positive top-line phase III results for trofinetide in Rett syndrome, meeting all primary and secondary endpoints in the pivotal phase III trial run by partner Acadia Pharmaceuticals Inc.

Far from the Groundhog Day spectacle of amyloid-targeting antibodies that do little to nothing for patients in trial after trial, researchers have reported success in reducing the risk of Alzheimer’s disease (AD) in unexpected ways. Researchers at the Cleveland Clinic Foundation have discovered that a Viagra prescription was associated with a roughly 70% reduced risk of being diagnosed with AD. And investigators from the University of Washington reported that in the prospective Adult Changes in Thought study, a long-term study into risk and protective factors for dementia, persons who underwent cataract surgery reduced their risk of AD by 30%, an effect that lasted for 10 years after surgery.

LONDON – Aviadobio Ltd. has raised $80 million in a series A round to take a precision microdosed gene therapy for treating familial frontotemporal dementia into a phase I/II clinical trial.

Novartis AG, keen to expand its central nervous system franchise as generics chase down crown jewel Gilenya (fingolimod), has secured rights from UCB SA to co-develop and commercialize two potential Parkinson's disease therapies for $150 million up front and milestone payments approaching $1.5 billion.

LONDON – Newco Loqus23 Therapeutics Ltd. has closed a £7 million (US$9.4 million) extension of its seed round, providing the means to progress its lead small-molecule program in Huntington’s disease through lead optimization. The extended round, from the Novartis Venture Fund and the U.K. government-backed Dementia Discovery Fund, follows an initial seed investment of £4.5 million by DDF in 2019, that was not made public at the time.

LONDON – Sosei Heptares is to get $100 million up front in a potential $2.6 billion deal with Neurocrine Biosciences Inc., in which the pair will develop muscarinic receptor agonists in the treatment of schizophrenia, dementia and other neuropsychiatric disorders. San Diego-based Neurocrine is taking rights to a portfolio of small molecules against specific subtypes of the muscarinic receptor family, targeting M1 and M4 alone, plus a dual M1/M4 agonist.

A European approval for Alzheimer’s drug aducanumab by year-end looks even more in doubt. A week after being called in before the EMA’s Committee for Medicinal Products for Human Use (CHMP) for an oral explanation of the trial data, Biogen Inc. said it received a “negative trend vote” on the marketing authorization application. A formal opinion by the CHMP is expected at its December meeting, but analysts are not optimistic.

European regulators have put off a decision on Biogen Inc and Eisai, Co. Ltd.’s controversial Alzheimer’s drug, aducanumab, for likely another month, after the companies announced further supportive data from a follow-up under review by the FDA.

PERTH, Australia – Australian researchers have debunked previous research that suggests autism spectrum disorder behavior may be caused by differences in the composition of the gut microbiota.