Upcoming Neurological Trial Results in H1 2024

Several innovative treatments are under clinical trials for a range of neurological conditions, with upcoming data releases anticipated in the neurodegenerative and neuropsychiatric fields. Six notable candidates in mid to late stages of testing are being closely monitored.

Sanofi and Denali's Collaborative ALS Therapy
In the fight against Amyotrophic Lateral Sclerosis (ALS), Sanofi, in collaboration with Denali Therapeutics, is on the cusp of revealing preliminary findings from the Phase II HIMALAYA trial for SAR443820/DNL788. This compound acts as an inhibitor of RIPK1, a protein pivotal in inflammatory and cell death pathways, potentially mitigating neuroinflammation and cell death linked to neurodegeneration. The HIMALAYA trial, which has enrolled 305 participants, is a two-part study. The first segment examines changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), while the second part, an open-label extension, focuses on the combined assessment of function and survival (CAFS) score. The treatment has received the FDA's Fast Track designation and is also under investigation for multiple sclerosis in the Phase II K2 study.

Intra-Cellular Therapies' Lumateperone for MDD
The year holds significant potential for advancements in treating major depressive disorder (MDD), with Intra-Cellular Therapies' lumateperone being a key player. Already approved for schizophrenia and depressive episodes in bipolar disorder, lumateperone is now being evaluated for MDD in two Phase III studies expected to release data in the first half of 2024. The medication, part of a three-part Phase III program, is being tested on adult patients with inadequate responses to existing antidepressants. The primary outcome measure is the change in the Montgomery Asberg Depression Rating Scale (MADRS) total score after six weeks.

Annexon Biosciences' ANX005 for GBS
Annexon Biosciences is poised to unveil critical data from a Phase III trial of ANX005, intended for the treatment of Guillain-Barré syndrome (GBS), a severe autoimmune disorder of the nervous system. With no FDA-approved treatments currently available for GBS, ANX005, designed to inhibit the complement pathway, has received both Fast Track and Orphan Drug designations from the FDA. A Phase III study assessing the efficacy and safety of this monoclonal antibody is set to release data in the first half of 2024.

Wave Life Sciences' WVE-003 for Huntington's Disease
Wave Life Sciences is challenging Huntington's disease with WVE-003, an antisense therapy currently in the Phase Ib/IIa SELECT-HD trial. The company plans to share data from the trial's 30 mg multi-dose cohort and all single-dose data in the second quarter of 2024. Huntington's disease is caused by a mutation in the huntingtin (HTT) gene, leading to progressive cognitive and motor symptoms. WVE-003 aims to reduce levels of mutant HTT protein while preserving the beneficial huntingtin protein.

5 and 6. Praxis Precision Medicines' PRAX-562 and PRAX-628 for Epilepsy
Praxis Precision Medicines is expecting to announce results for two of its epilepsy treatments. PRAX-628, a next-generation small molecule targeting sodium-channels in the brain, is in Phase IIa trials for photo-paroxysmal response (PPR) epilepsy, with topline results expected in the first quarter of this year. Additionally, the Phase II EMBOLD study for PRAX-562, a sodium channel modulator for pediatric patients with specific developmental epilepsies, is anticipated to release findings in the first half of 2024.

These developments represent significant strides in the quest for effective treatments for a variety of neurological conditions, offering hope for improved patient outcomes in the near future.