Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone in treating Rett syndrome. The trial involves 24 female patients aged five to 12 years, receiving leriglitazone or placebo for 36 weeks.

Conducted at the Neurometabolic Disorders Unit of Hospital Sant Joan de Déu, the study aims to show improved cognition, stabilised communication skills, better behaviour and delayed neuromuscular worsening. Dr Ángeles García Cazorla leads the study.

“Following the positive results in the study NEXUS in boys with cerebral adrenoleukodystrophy (cALD), we will pursue additional orphan CNS indications with high unmet medical need,” said Marc Martinell, CEO of Minoryx. “Rett syndrome is one such indication, and we look forward to collaborating with the team of excellent physicians from Hospital Sant Joan de Déu in Barcelona.”

Preclinical studies showed leriglitazone’s recovery effects on bioenergetic alterations in human Rett fibroblasts and its anti-neuroinflammatory effect in Rett mouse models. Based on these findings, the TREE study will assess improvements in cognition, behaviour, communication skills and motor skills.

Minoryx’s Chief Medical Officer, Arun Mistry, said: “Leriglitazone has a mode of action relevant to the pathways associated with Rett syndrome. Thus far we have clinical safety and efficacy data in male paediatric patients, adult men and adult women from studies in X-ALD and Friedreich’s ataxia and the TREE study expands to paediatric female patients with Rett syndrome.”

CervoMed has announced positive results from the extension phase 2b RewinD-LB study of neflamapimod for dementia with Lewy bodies (DLB).

A new batch of neflamapimod capsules led to increased plasma drug concentrations and demonstrated improvement on the Clinical Dementia Rating Sum of Boxes (CDR-SB). Improvement was also shown on the Alzheimer’s Disease Cooperative Study – Clinical Global Impression of Change (CGIC).

Compared to old capsules or placebo, a lower incidence of falls was observed in participants receiving the new drug batch. James E Galvin MD, MPH, said: “This is a great day for patients and families impacted by DLB, as well as the DLB clinical community.” He added that neflamapimod has the potential to fundamentally change the disease course.

John Alam MD, CEO of CervoMed, said: “As presented at the recent International Lewy Body Dementia Conference, our working hypothesis for the failure of neflamapimod during the initial 16 weeks of the study is that the investigational drug capsules utilised in that phase of the trial delivered lower than expected plasma drug concentrations and effectively underdosed participants.”

He stated the new capsules led to higher drug concentration levels and improvements.
John-Paul Taylor MRCPsych, PhD, said: “Results from the extension phase of the RewinD-LB study are highly persuasive.”