Clinical Research

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.

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.

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 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 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.

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.

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 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. 

argenx SE reported the second failure of a trial involving its antibody fragment efgartigimod alfa on 20 December following unexpectedly positive results from the placebo arm of the study. Called ADDRESS, the Phase 3 trial was investigating a subcutaneous formulation of efgartigimod (efgartigimod alfa and hyaluronidase) in adults with two types of pemphigus, an autoimmune disease that affects the skin and mucous membranes. 

A small molecule drug in development by Vertex Pharmaceuticals Inc has reduced pain in patients with diabetic peripheral neuropathy, a type of nerve damage that can occur with the disease. A Phase 2 trial of VX-548 which inhibits NaV1.8, a voltage-gated sodium channel, showed a statistically significant reduction in a measure of pain, Vertex announced on 13 December 2023. Patients with diabetic peripheral neuropathy are often treated with anti-seizure drugs or antidepressants, according to the Mayo Clinic. Opioid analgesics can also be used as second or third-line agents.