A schizophrenia drug in Cerevel Therapeutics Inc.’s lineup understandably stole much of the thunder during coverage of Abbvie Inc.’s takeover late last year to the tune of $8.7 billion, but much further back in the pipeline awaits another potentially lucrative prospect: a kappa opioid receptor antagonist (KORA) for major depressive disorder (MDD).

Asceneuron SA has raised $100 million in an oversubscribed series C to take its lead small molecule, ASN-51, into phase II, with aim of demonstrating it prevents the formation of tau tangles and slows the progression of Alzheimer’s disease.

The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg this week. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier.

Interim data from two early stage Friedreich’s ataxia (FA) cardiomyopathy studies from Lexeo Therapeutics Inc. hit the mark by reducing heart muscle thickness, a key cause of death among patients with the rare disease. The results came from the Sunrise-FA phase I/II study and an investigator-initiated phase Ia study of LX-2006, an adeno-associated virus-mediated gene therapy encoding the human frataxin gene. The drug is designed to improve frataxin protein expression to improve mitochondrial cell function.

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

Uniqure NV shares (NASDAQ:QURE) closed July 9 at $6.67, up $2.89, or 76%, after the firm made public updated interim data including up to 24 months of follow-up findings from 29 treated patients enrolled in the ongoing U.S. and European phase I/II trials of AMT-130 for Huntington’s disease (HD).

On the heels of positive phase II results of its extended-release ketamine (R-107) in treatment-resistant depression, Douglas Pharmaceuticals Ltd. is gearing up to begin phase III trials of its ketamine treatment that is safe enough to take at home without clinical supervision.

Less than a month after the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee handed down a unanimous vote in favor of Eli Lilly and Co.’s Alzheimer’s disease candidate (AD), donanemab, the agency approved the drug as a once-monthly injection for adults with early symptomatic disease. Branded Kisunla, the beta-amyloid antagonist marks the second approved AD drug that has demonstrated in clinical trials an ability to slow cognitive decline, going up against Leqembi (lecanemab) from Biogen Inc. and Eisai Co. Ltd., which won full approval in July 2023, only six months after nabbing an accelerated nod.

Following a data-cleaning delay and mixed results in Alzheimer’s disease, Annovis Bio Inc. reported phase III Parkinson’s disease data showing treatment with buntanetap stops cognitive decline and improves motor and other functions, including postural instability and gait difficulty, meeting all primary and secondary endpoints.

In the first five months of 2024, the BioWorld Neurological Diseases Index (BNDI) saw a 10.35% decline, underperforming both the Nasdaq Biotechnology Index (NBI), which rose by 0.7%, and the Dow Jones Industrial Average (DJIA), which increased by 2.64%. This represents an additional drop for BNDI, which closed February with a 4.1% decline. In contrast, in 2023, the BNDI closed with a 4.36% increase, outperforming the NBI, which rose by 3.74%, but not matching the 13.7% rise seen in the DJIA.