ReS19-T / ReMYND - LARVOL DELTA

PHASE 2A CLINICAL TRIAL SUPPORTS THERAPEUTIC EFFICACY OF SEPTIN MODULATION IN MILD-TO-MODERATE ALZHEIMER'S PATIENTS (ADPD 2025) - "Liver toxicity led to termination of study. The preliminary observations with REM0046127 warrant follow-up studies with an optimized lead compound in patients."

Clinical • P2a data • Alzheimer's Disease • CNS Disorders • Hepatology • Liver Failure

Septin regulation of Orai-mediated Ca2+ entry - a novel target for neurodegeneration. (PubMed, Cell Calcium) - "They identified a class of compounds referred to as ReS19-T that interact with Septins, previously identified as regulators of the Store-operated Ca2+ entry channel Orai...The importance of membrane contacts for maintaining precise cellular signaling has been recognized previously. However, the molecular mechanism by which Septin filaments organize the ER-PM junctions to regulate Ca2+ entry through Orai remains to be fully understood."

Journal • Alzheimer's Disease • Ataxia • CNS Disorders • Genetic Disorders • Movement Disorders • Parkinson's Disease

A Phase 2a Study to Investigate REM0046127 in Mild to Moderate Alzheimer's Disease (clinicaltrials.gov) - P2 | N=14 | Terminated | Sponsor: reMYND | N=60 ➔ 14 | Recruiting ➔ Terminated; Due to the event of repeated elevated transaminase levels in one subject - elevated ALT/AST up to 6x ULN in the absence of any other potential underlying cause - we have decided to terminate the study.

Enrollment change • Trial termination • Alzheimer's Disease • CNS Disorders

Pharmacological modulation of septins restores calcium homeostasis and is neuroprotective in models of Alzheimer's disease. (PubMed, Science) - "In amyloid-β and tau-driven mouse models of disease, ReS19-T agents restored synaptic plasticity, normalized brain network activity, and attenuated the development of both amyloid-β and tau pathology. Our findings identify the septin cytoskeleton as a potential therapeutic target for the development of disease-modifying AD treatments."

Journal • Alzheimer's Disease • CNS Disorders • Developmental Disorders

RESTORING CALCIUM HOMEOSTASIS BY PHARMACOLOGICAL MODULATION OF SEPTINS IS NEUROPROTECTIVE IN ALZHEIMER'S DISEASE (ADPD 2024) - "Our findings provide support for calcium-driven neurodegeneration and identify the septin cytoskeleton as a novel and promising therapeutic target in AD. Modulation of septin assembly by REM0046127 results in symptomatic relief and disease modification in AD preclinical models. Preliminary data from the ongoing Phase 2a study suggest these findings may translate to AD patients."

Alzheimer's Disease • CNS Disorders • SEPTIN6

A Phase 2a Study to Investigate REM0046127 in Mild to Moderate Alzheimer's Disease (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: reMYND | Phase classification: P2a ➔ P2 | Trial completion date: Jun 2023 ➔ Jun 2024 | Trial primary completion date: Jun 2023 ➔ Jun 2024

Phase classification • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

A PHASE 1 STUDY TO EVALUATE THE SAFETY AND BRAIN EXPOSURE OF REM0046127, A NOVEL, FIRST-IN-CLASS MODULATOR OF CALCIUM HOMEOSTASIS (ADPD 2023) - "Conclusions REM0046127 has a broad safety margin and is well permeable resulting in high and stable exposure in brain during the dosing period. As the effects on calcium modulation occur rapidly, therapeutic efficacy of REM0046127 can be assessed in a short Phase 2a trial, including a high dose level about 10 times the anticipated effective dose."

Clinical • P1 data

A Phase 2a, randomized, placebo-controlled, double-blind study to investigate REM0046127 in mild to moderate Alzheimer's disease Un estudio de fase 2a, aleatorizado, controlado con placebo, doble ciego para investigar REM0046127 en la enfermedad de Alzheimer de leve a moderada (clinicaltrialsregister.eu) - P2 | N=60 | Ongoing | Sponsor: reMYND

New P2 trial • Alzheimer's Disease • CNS Disorders

A Phase 2a Study to Investigate REM0046127 in Mild to Moderate Alzheimer's Disease (clinicaltrials.gov) - P2a | N=60 | Recruiting | Sponsor: reMYND

New P2a trial • Alzheimer's Disease • CNS Disorders

A Phase 1 (First in Human) Randomized, Double-blind, Placebo-controlled SAD, MAD Study With Oral REM0046127 (clinicaltrials.gov) - P1 | N=77 | Completed | Sponsor: reMYND | Recruiting ➔ Completed

Trial completion • Alzheimer's Disease • CNS Disorders

reMYND’s novel Alzheimer program reports excellent brain exposure and safety profile in Phase 1; transitioning into Phase 2a (GlobeNewswire) - P1 | N=77 | "reMYND NV...announces that its lead Alzheimer program ReS19-T has successfully completed its Phase 1 study. Topline data has demonstrated excellent brain exposure and strong safety profile....The Phase 2a results are expected by mid-2023."

New P2a trial • P1 data • Alzheimer's Disease • CNS Disorders

A Phase 1 (First in Human) Randomized, Double-blind, Placebo-controlled SAD, MAD Study With Oral REM0046127 (clinicaltrials.gov) - P1; N=78; Recruiting; Sponsor: reMYND; Trial completion date: Oct 2021 ➔ May 2022; Trial primary completion date: Sep 2021 ➔ Mar 2022

Clinical • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

A Phase 1 (First in Human) Randomized, Double-blind, Placebo-controlled SAD, MAD Study With Oral REM0046127 (clinicaltrials.gov) - P1; N=78; Recruiting; Sponsor: reMYND

Clinical • New P1 trial • Alzheimer's Disease • CNS Disorders