Upcoming Neurological Treatment Milestones in 2024 H1

Several innovative treatments are under clinical trial for a range of neurological conditions, with upcoming data releases anticipated in the fields of neurodegenerative and neuropsychiatric disorders. Here's a summary of six significant drug candidates in mid to late stages of development:

Sanofi and Denali's Collaboration on ALS Treatment
Sanofi, in partnership with Denali Therapeutics, is on the verge of revealing results from the Phase II HIMALAYA trial for their ALS treatment, SAR443820/DNL788. This compound targets RIPK1, a protein involved in inflammation and cell death. The trial, which has completed enrollment, assesses the ALS Functional Rating Scale-Revised (ALSFRS-R) to measure disease progression. The drug has received the FDA's Fast Track designation and is also being tested for multiple sclerosis in the Phase II K2 study.
Intra-Cellular Therapies' Lumateperone for MDD
Analysts are keeping a close eye on the development of treatments for major depressive disorder (MDD), with Intra-Cellular Therapies' lumateperone being a key player. The drug has already been approved for schizophrenia and for depressive episodes in bipolar disorder. Now, it is being evaluated for MDD in two Phase III studies, with data expected in the first half of 2024. The primary endpoint for these trials is the change in the Montgomery Asberg Depression Rating Scale (MADRS) after six weeks.
Annexon Biosciences' ANX005 for Guillain-Barré Syndrome
Annexon Biosciences is expecting key results from a Phase III trial of ANX005 for the treatment of Guillain-Barré syndrome (GBS), a serious autoimmune condition. ANX005 is designed to inhibit the complement pathway, and the Phase III study aims to assess its efficacy and safety. The drug has received both Fast Track and Orphan Drug designations from the FDA.
Wave Life Sciences' WVE-003 for Huntington's Disease
Wave Life Sciences is working on an antisense therapy, WVE-003, for Huntington's disease. The company is set to share data from the SELECT-HD trial, focusing on the reduction of mutant HTT (mHTT) protein levels without affecting the normal huntingtin protein (wtHTT). The data from this trial could influence a collaboration agreement with Takeda.
and 6. Praxis Precision Medicines' PRAX-628 and PRAX-562 for Epilepsy
Praxis Precision Medicines has two epilepsy treatments in the pipeline. PRAX-628, for patients with photo-paroxysmal response (PPR) epilepsy, is expected to show results from a Phase IIa study. PRAX-562, a sodium channel modulator for pediatric patients with specific developmental epilepsies, will also have Phase II results released in the first half of 2024. Both drugs are part of Praxis' Cerebrum small molecule platform, which targets neuronal excitability.

These developments are closely watched as they represent potential breakthroughs in the treatment of severe neurological conditions. The success of these trials could significantly impact the future of neurology therapeutics.