Clinical Research

Amylyx drug for ALS fails at Phase 3

Country
United States

A confirmatory Phase 3 trial of a drug for patients with amyotrophic lateral sclerosis (ALS) failed to meet its primary and secondary endpoints, raising the possibility that the therapy, originally approved in 2022, could be withdrawn from the market. The developer, Amylyx Pharmaceuticals Inc, announced the trial results on 8 March, saying that a voluntary market withdrawal had not been ruled out. The therapy, AMX0035, is a combination of two small molecules, sodium phenylbutyrate and taurursodiol, which each target different neurodegenerative pathways.

NodThera drug reverses neuroinflammation

Country
United States

NodThera Ltd said that its small molecule inhibitor of the NLRP3 inflammasome has shown evidence of reducing neuroinflammatory and inflammatory biomarkers in Parkinson’s disease. Data from a Phase 1b/2a a trial, disclosed on 7 March, showed that the drug, NT-0796, delivered mean reductions of these markers in the cerebrospinal fluid of elderly patients, bringing them to levels approximating those of healthy elderly controls.

Semaglutide reduces risk of kidney disease progression

Novo Nordisk A/S’s semaglutide (Ozempic), currently approved to reduce the risk of adverse cardiovascular events in adults with type 2 diabetes and cardiovascular disease, has achieved another milestone. This was evidence that the drug reduced the risk of kidney disease-related events by 24% in people with type 2 diabetes and chronic kidney disease. The data comes from the Phase 3 FLOW trial, which was stopped early in October 2023 due to efficacy. The headline results from this trial were released on 5 March.

Positive data for RNAi therapeutic for hypertension

Country
Switzerland

An investigational RNA interference (RNAi) treatment has delivered positive data in a Phase 2 trial of people with mild to moderate uncontrolled hypertension, according to the developers Roche and Alnylam Pharmaceuticals Inc. The two companies have now started another Phase 2 study of the drug, zilebesiran, in people with uncontrolled hypertension at high cardiovascular risk.

Vivoryon Alzheimer’s study fails

Country
Germany

A Phase 2b study of a small molecule drug for the treatment of early Alzheimer’s disease failed to meet its primary and key secondary endpoints, the Germany-based developer Vivoryon Therapeutics NV, announced on 4 March. The drug targets an enzyme, glutaminyl cyclase, which is thought to be responsible for the formation of toxic oligomers in the disease. Although the drug showed promising efficacy signals in an earlier Phase 2a trial, it did not show a statistically significant difference in change over time on cognition – the primary endpoint.

Gilead discontinues magrolimab studies

Country
United States

Gilead Sciences Inc is to discontinue studies of magrolimab, a monoclonal antibody targeting CD47 on cancer cells, following evidence that the risks associated with the drug outweigh the benefits. On 7 February the company said it was stopping a clinical programme in haematologic cancers. On 15 February it announced a partial clinical hold on four Phase 2 studies in solid tumours at the request of the US Food and Drug Administration.

MetrioPharm reports data from Covid-19 trial

Country
Switzerland

MetrioPharm AG, a Swiss biotech company, has reported positive results from a trial of its small molecule immune modulator in patients hospitalised with Covid-19. The Phase 2a study showed that the drug, MP1032, was effective when administered with a standard of care and delivered lower mortality compared with patients receiving a placebo and standard of care. According to the company, the results showed that MP1032 is comparable to remdesivir (Veklury), an approved antiviral, with additional safety benefits.

Combination effective for colorectal cancer

Country
United States

A combination of Opdivo (nivolumab) and Yervoy (ipilimumab) has been effective in reducing the risk of disease progression in patients with metastatic colorectal cancer – the first dual immunotherapy regime to show significant benefits as a first-line treatment, according to Bristol Myers Squibb Co. BMS, which developed both drugs, announced the results of the Phase 3 CheckMate-8HW trial on 20 January.

Fast track for CAR T cell therapy

Country
United States

An experimental chimeric antigen receptor (CAR) T cell therapy intended for the treatment of multiple sclerosis received a fast track designation from the US Food and Drug Administration on 19 January, indicating its potential to address a major medical need. The therapy is being developed by Kyverna Therapeutics Inc, a biotech company incorporated in 2018 and located in Emeryville, California, US.

Sanofi stops ADC programme

Country
France

Sanofi has stopped a global programme evaluating the clinical effectiveness of tusamitamab ravtansine, an antibody-drug conjugate (ADC), after a Phase 3 trial of the drug failed to improve progression-free survival in patients with a type of non-small cell lung cancer. Tusamitamab ravtansine was targeting a transmembrane cell adhesion molecule – carcinoembryonic antigen-related cell adhesion molecule 5 – which is associated with a number of cancers.