The Demoucelle Parkinson Charity has decided to support further in-patient testing of a drug targeting the buildup of protein alpha-synuclein in the brain, hoping that it will ultimately prove successful both as a treatment and a cure.
After encouraging safety and tolerability results in their phase 1 trial, German biotech company MODAG will now conduct another small-scale but longer trial (phase 1b), for which 24 patients will receive a higher dose of MODAG’s anle138b drug or a placebo. This should provide important additional data to prepare for a larger Phase 2 trial.
The Demoucelle Parkinson Charity is working together with the Michael J. Fox Foundation for Parkinson’s Research to support this extension trial.
For more information about the research, please read the text from the Michael J. Fox Foundation below.
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MODAG Anle138b Phase 1b 28-day Efficacy Extension Cohort
Study Rationale: The Michael J. Fox Foundation (MJFF) is committed to finding a cure and better treatments for Parkinson’s disease (PD). The most urgent unmet medical need in PD is a treatment to slow or stop the progression of the disease. Pathological aggregates of the protein alpha-synuclein — a hallmark of PD — are promising targets for such interventions. With generous donor support, MJFF funded the Phase Ib trial of anle138b, an oral inhibitor of alpha-synuclein aggregation from German biotechnology company MODAG. That study administered lower dose therapy (6 volunteers), higher dose (6) or placebo (4) over one week to participants with moderate to later-stage Parkinson’s disease. Findings indicated safety as well as a signal of potential symptomatic benefit. In October 2021, MODAG announced a licensing agreement for anle138b with pharmaceutical company Teva. With the infrastructure from the initial Phase Ib study, MODAG is now planning an add-on Phase Ib trial.
Hypothesis: More data and sample analysis from a larger cohort treated with the higher drug dose for a longer period may provide more insight into the potential of anle138b for PD, inform future trial design and provide additional safety/tolerability data over a longer dosing period. These additional data may enable acceleration of a Phase II trial, advancing this treatment closer to patient hands.
Study Design: The Phase Ib extension trial will randomize 24 participants with moderate to later-stage PD to the higher dose (300 mg/day) of the drug or to placebo. Treatment will last 28 days, and volunteers will complete assessments for six weeks after the last dose. Leveraging the Phase I trial sites and study teams hastens the start of this trial — and delivery of results — rather than building this work into a new Phase II study. Analysis will review safety and tolerability as well as impact on biological markers of disease progression and on PD symptoms.
Next Steps: This Phase Ib expansion will serve as a bridge to Phase II. Findings could influence dose selection, participant eligibility and outcome measures. Moreover, this new set of data may enable “breakthrough designation” status by the U.S. Food and Drug Administration, which would enable faster review and greater coordination with the regulatory agency around anle138b development.
Impact on Treatment of Parkinson’s Disease: While our primary goal is a cure for Parkinson’s, MJFF is also committed to treatments that ease disease symptoms. Initial results from anle138b show this therapy may have potential for both. MJFF believes that with the right tools and partners, treatments such as this have a real chance at making it to market and changing the future of care and cures for Parkinson’s disease.