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One-time gene therapy could improve wet AMD

European Pharmaceutical Review

REGENXBIO’s Phase I/IIa trial investigated the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for wet age-related macular degeneration (wet AMD). The two-year data showed that ABBV-RGX-314 provided stable or improved visual acuity and retinal thickness in patients.

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BMS strikes $14bn deal for Karuna Therapeutics

European Pharmaceutical Review

KarXT targets both the M1 and M4 muscarinic receptors, resulting in a differentiated safety and efficacy profile. The drug is also undergoing trials both for adjunctive therapy to existing standard of care agents in schizophrenia and for the treatment of psychosis in patients with Alzheimer’s disease.

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NewAmsterdam Pharma enters merger deal with Frazier Lifesciences

Pharmaceutical Technology

This pro forma cash balance of nearly $470m is anticipated to offer a cash runway for the merged company through 2026. An oral, low-dose and once-a-day CETP inhibitor, obicetrapib demonstrated promising safety and >50% LDL-reducing efficacy when given along with high-intensity statins in dyslipidemia patients in a Phase IIb trial.

Pharma 59
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Janssen leads effort to cut waste in digital health devices

pharmaphorum

The project started on 1 October and is scheduled to complete in September 2026. Devices such as smart pill boxes, electronic blood collection devices, wearable monitors, and intelligent labels are increasingly being used to improve healthcare efficiency and conditions for patients, but present an “expanding e-waste challenge.”

Leads 121
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First multiple sclerosis biosimilar approved in Europe

European Pharmaceutical Review

This was in addition to Phase I PK/PD study results and a confirmatory Phase III Antelope study in RRMS patients. Both studies confirmed that the biosimilar matched the reference biologic for pharmacokinetics as well as efficacy, safety and immunogenicity. The site is planned to be completed by 2026.

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Biologics – manufacturing trends in a modern facility

European Pharmaceutical Review

‘From bench to bedside’ is a well-known aphorism that merely hints at the arduous journey drug treatments must undergo from their position as a candidate molecule or soon to be explored pathway, to becoming a tablet, injection, or infusion administered to patients worldwide. How does it do this?

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NewAmsterdam goes public to advance drug in abandoned class

pharmaphorum

Merck’s exit came after other companies including Pfizer, Eli Lilly and Roche all scrapped CETP development programmes following safety or efficacy issues. BROADWAY and BOOKLYN will generate data in 2024, with PREVAIL set to read to in 2026, and if the results are positive will be followed by filings in the US, Europe, China and Japan.