The U.S. FDA wants more data on PTC-518 before PTC Therapeutics Inc.'s phase II study of Huntington’s disease can continue enrollment. While stopped in the U.S., the study of the oral, small-molecule splicing modifier still is enrolling participants at sites in several European countries and in Australia.

“Epilepsy is really a classical neurological disorder,” Lars Pinborg told the audience at the European College of Neuropsychopharmacology (ECNP) annual conference on Sunday. “Or is it?” Pinborg, of Rigshospitalet's The Neuroscience Center in Denmark, was chairing a session dedicated to an alternative hypothesis, summed up in the session title: “Is epilepsy a psychiatric disorder?”

Relmada Therapeutics Inc. is scratching its collective chin as it sifts through data from the failed phase III Reliance III study of REL-1017 (esmethadone) in treating major depressive disorder (MDD). A higher-than-expected placebo response, however, prompted the company to label the study’s results as “paradoxical.”

Bloomsbury Genetic Therapies Ltd. has raised £5 million (US$5.5 million) in a seed round, to take four gene therapy programs based on research carried out by the scientific founders at University College London into clinical development.

Ascidian Therapeutics Inc. secured $50 million in series A funding from its founder Apple Tree Partners to take a therapy based on its RNA exon editing technology into a first-in-human trial in ABCA4 retinopathy and to advance additional programs in neuromuscular, neurological, and rare disease indications.

Researchers at the University of Cincinnati have published data showing that in patients with dominantly inherited Alzheimer’s disease-causing mutations, high levels of soluble amyloid-β42 (Aβ42) in the cerebrospinal fluid predicted a reduced risk of developing dementia over three years.

A new analysis of Actinogen Medical Ltd.’s phase II Xanadu trial showed that in biomarker-positive patients with mild Alzheimer’s disease there was a clear clinical effect with lead compound Xanamem not seen in the earlier trial.

Despite clinical responses undermining expectations for the placebo arm of its phase III Alzheimer's disease (AD) study, Lucidity, Taurx Pharmaceuticals Ltd. executives Oct. 6 said they still see the data as supporting their ability to pursue regulatory submissions for hydromethylthionine mesylate (HMTM), an oral tau aggregation inhibitor. For people with mild cognitive impairment especially, they said, "HMTM treatment resulted in sustained improvement in cognition over pretreatment baseline, and normalization of brain atrophy to a rate similar to healthy individuals."

Cerecin Inc. has successfully closed the first tranche of a series IIB financing with investors from South Korea, with SK Securities Co. Ltd. and KNT Investment LLC leading the round. Proceeds from the financing, of undisclosed value, will help the company advance clinical studies of its CER-0001 (tricaprilin) in migraine and infantile spasm.