MapLight’s ML-004 Autism Extension Study: What Ongoing Safety Data Mean for Investors

MapLight Therapeutics, Inc. (MPLT) announced an update on their ongoing clinical study.

Study Overview
MapLight Therapeutics is running an extension study called “An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).” The goal is to track long‑term safety and tolerability of ML-004 in about 120 people with autism who finished a prior trial, helping investors gauge how durable and scalable this drug program might be if it moves toward late-stage development.

Intervention/Treatment
The study tests ML-004, an experimental daily tablet developed by MapLight. It is a combination immediate-release and extended-release pill designed to keep drug levels steady over the day, with doses ranging from 12 mg to 72 mg. The aim is to see if ML-004 can be used safely over longer periods in adolescents and adults with autism.

Study Design
This is an interventional Phase 2 trial using a single treatment group, so everyone receives ML-004 and there is no placebo arm. The design is open-label, meaning both doctors and participants know they are taking the drug, and the main purpose is treatment-focused, with safety and tolerability as the key measures. The study is enrolling by invitation only, limited to participants from the earlier ML-004-002 trial.

Study Timeline
The study was first submitted on May 22, 2023, marking the formal regulatory start of the program. It is currently listed as “enrolling by invitation,” indicating active recruitment from the prior study population. The most recent update was posted on December 19, 2025, showing the sponsor is still refining or adding information as data accumulates. Final primary and overall completion dates are not yet posted, signaling that investors should view this as an ongoing mid-stage project rather than a near‑term approval catalyst.

Market Implications
For MapLight Therapeutics, this extension study is mainly about de-risking ML-004’s safety profile in autism, a large and underserved market where there are limited approved treatments and high unmet need. Positive long-term safety data could support future, larger trials and may boost investor confidence in the company’s pipeline value and partnering potential. In the broader neurodevelopmental space, established players with marketed psychiatric and neurology drugs may face new competition if ML-004 progresses, while early-stage peers in autism-focused biotech could see sentiment readthrough based on how investors respond to MapLight’s updates. For now, the update confirms continued progress but does not by itself change near-term revenue expectations, so share-price impact is likely to depend on subsequent data readouts and any strategic deals linked to this program.

This clinical study remains active and updated, with more details available on the ClinicalTrials.gov portal under its registered identifier.

To learn more about MPLT’s potential, visit the MapLight Therapeutics, Inc. drug pipeline page.