The firm’s sole nephrological asset has demonstrated proof of concept, helping advance the novel gene-targeted therapy into pivotal development early next year.

After returning to the neuroscience space in the summer through a big-money deal with Alector, GlaxoSmithKline is setting up an institute with the University of Oxford that will target neurological diseases such as Alzheimer’s, Parkinson’s, multiple sclerosis, frontal temporal dementia, ALS and pain.

A US FDA review panel has given a lukewarm reception to molnupiravir, while Inovio is assessing its vaccine candidates against the new omicron variant, J&J nears a South African vaccine deal with Aspen, and South Korea brings in new COVID-19 measures. Meanwhile, Serum exports Novavax vaccine, Bharat looks at Covaxin in Omicron and Ocugen's Covaxin trial put on hold. 

Data from SHORELINE’s 50mg cohort sparked mixed opinions from analysts, with some suggesting they still showed a risk-benefit profile that could challenge the drug commercially.

The response rate, with five complete responses, exceeds known responses to standard-of-care chemotherapy in heavily pre-treated folate receptor alpha-high patients. A US FDA filing is on track for early 2022.

The company’s lead candidate, AP1189, has shown promise in a mid-stage rheumatoid arthritis study, sparking plans for an imminent strategic business collaboration. 

A regulatory filing for Vyjuvek in the US is targeted for the first half of 2022 following the positive results of a Phase III trial, positioning Krystal for its first commercial launch.

NRx’s BriLife has demonstrated strong potential in early data from a mid-stage COVID-19 trial, spurring the company on to begin a Phase IIb/III study as soon as possible.

Despite recent setbacks for Roche and Wave's respective antisense offerings, both those two firms and a number of other companies are trying a variety of approaches to find a treatment for Huntington's.

Increases in liver enzymes and fat could dampen approval prospects despite vupanorsen meeting its mid-stage primary endpoint, some analysts have said.

Emerging Company Profile: Cybin’s novel approach to psilocybin therapeutics aims for better safety, shorter duration and less variability through the process of deuteration.

From BTK inhibitors in multiple sclerosis to candidates aiding cancer cell death, Merck KGaA is focusing on its first-in-class pipeline which it expects to make a significant impact from 2025.