LONDON – The National Health Service (NHS) in the U.K. has agreed to reimburse Zolgensma, the one-off gene therapy treatment for spinal muscular atrophy billed as the most expensive drug in the world, after a confidential deal was struck with Novartis Gene Therapies.

DUBLIN – A novel real-world study methodology that marries digital technology with a citizen science or do-it-yourself approach to “drug” procurement, formulation and administration has found – yet again – that the placebo remains one of the great wonders of the medical world.

Bionaut Labs emerged from five years in stealth mode raising $20 million to develop Bionauts, microrobots designed to deliver therapies to treat brain disorders. The financing will support the company’s therapeutic program in glioma through preclinical development and further research and development in Huntington’s disease.

Having rung the bell in phase II last summer, Ovid Therapeutics Inc. and Takeda Pharmaceutical Co. Ltd. signed a pact giving the latter global rights to develop and commercialize soticlestat, a first-in-class inhibitor of cholesterol 24-hydroxylase for the treatment of developmental and epileptic encephalopathies including Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).

A new once-daily attention deficit hyperactive disorder (ADHD) drug developed by prodrug specialist Kempharm Inc. has won FDA approval under the brand name Azstarys. The medicine combines serdexmethylphenidate, Kempharm's prodrug of the CNS stimulant dexmethylphenidate, with immediate-release d-MPH, the core medicine in the now off-patent product Focalin, a refined formulation of Ritalin.

San Diego-based Neurocrine Biosciences Inc.’s good-and-bad top-line phase II data with d-amino acid oxidase inhibitor luvadaxistat, also known NBI-1065844 and TAK-831, in schizophrenia likely came as little surprise to many, given earlier experience in the general pathway.

News of Vaccinex Inc. signing two multitarget deals with undisclosed "prominent pharmaceutical companies" sent company shares (NASDAQ:VCNX) up 70% to $4.88 on Feb. 19, even as financial terms of the deal went undisclosed. The collaborations will focus on using the company's ActivMAb antibody discovery and novel viral display platform to develop candidates against complex antigens such as G protein-coupled receptors and ion channels, the company said.

The FDA placed a partial clinical hold on atuzaginstat (COR-388) from Cortexyme Inc., of South San Francisco, stating that no new participants should be enrolled in the open-label extension portion of the phase II/III GAIN trial in mild to moderate Alzheimer’s disease.

Recently published findings in JAMA Psychiatry related to the sharply increased risk of death from COVID-19 in people with schizophrenia put the spotlight on drug development in the space, which has been steadily heating up the past few years.

As it waits on initial data from its two lead programs, in sensorineural hearing loss and multiple sclerosis, Pipeline Therapeutics Inc. raised $80 million in a series C crossover round to progress its pipeline of small-molecule drugs for neuroregeneration and to position it for a potential IPO.