Clinical Research

ADC Therapeutics SA of Switzerland reached agreement with investors on 6 May to sell shares and warrants valued at $105 million to support its portfolio of antibody-drug conjugates for cancer. The new financing will lengthen the company’s cash runway enabling it to advance new indications for Zynlonta (loncastuximab tesirine), a marketed drug, and clinical-stage assets in its portfolio. ADC is developing antibody-drug conjugates for both haematological cancers and solid tumours. On 31 March it had cash and cash equivalents of $234.3 million compared with $278.6 million on 31 December 2023.

A gene therapy being developed by Regeneron Pharmaceuticals Inc has enabled a young child, suffering from a congenital hearing loss, to overcome deafness. The child, dosed at 11 months of age, is one of two to have responded to the experimental therapy which uses gene replacement to address mutations in the OTOF gene – the cause of the disease. A second child, who received the therapy at the age of four, showed initial improvements after dosing.

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its primary and secondary endpoints, raising the possibility that the therapy, originally approved in 2022, could be withdrawn from the market.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 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.

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.