Update from the AAN Annual Meeting: Progress Toward the First Potential Approved Treatment for APBD
April 24, 2026
We are pleased to share that researchers and partners advancing APBD treatment development participated in the American Academy of Neurology (AAN) Annual Meeting, held April 18-22, 2026, in Chicago, IL. As the premier global conference in neurology, AAN attracts over ten thousand attendees from around the world each year. Bringing together neurologists, clinical and academic researchers, and industry professionals, the conference provides an important opportunity to shine the spotlight on APBD research and treatment development.
From left to right: Or Kakhlon, PhD; Yossi Gilgun-Sherki, PhD (GHF); Michio Hirano, MD; and Yaniv Glazer (GHF)
This year’s AAN convening marked a significant milestone for APBD drug development and Golden Heart Flower (GHF), with two abstracts accepted showcasing the progress of the GHF-201 program, a novel small-molecule therapy designed to reduce polyglucosan body accumulation, the underlying pathogenic factor in APBD.
Dr. Kakhlon’s talk focused on the outcomes of the Urgent Compassionate Use Program in Israel
Dr. Hirano’s poster presentation focused on the proposed framework for a future Phase II/III clinical trial of GHF-201 in APBD patients.
Or Kakhlon, PhD (Hadassah Medical Center), an APBD Research Foundation SMAB member, presented his talk titled “GHF-201, A Novel Oral Compound for Adult Polyglucosan Body Disease, Demonstrates Promising Safety and Efficacy in a Compassionate Use Program,” on behalf of Alexander Lossos, MD, who is in charge of this program. The presentation highlighted outcomes from the ongoing compassionate use program in Israel. Data from treated patients demonstrated clinically meaningful improvements in motor function, including muscle strength, along with reductions in polyglucosan accumulation and supportive biomarker changes. These findings were further reinforced by favorable safety and tolerability data from a Phase 1 study in healthy volunteers, supporting continued clinical development.
In parallel, Michio Hirano, MD (Columbia University), Principal Investigator of the planned clinical study, presented “A Phase 2/3 Adaptive Design Study of GHF-201 as the First Potential Treatment of Adult Polyglucosan Body Disease” outlining an innovative, pivotal clinical study design. This study is intended to evaluate both the safety and efficacy of GHF-201 in patients through a two-stage approach. Phase 2 is planned to support a New Drug Application submission to the FDA for conditional approval of the drug, followed by a confirmatory Phase 3 clinical study to measure clinical outcomes. The design incorporates advanced methodologies and has been developed in alignment with the FDA, with the goal of advancing GHF-201 as the first approved therapy for APBD.
The acceptance of these abstracts at AAN represents more than a scientific update; it reflects growing recognition of the importance of this work and the momentum toward bringing a meaningful treatment option to the APBD community.
What’s Next?
GHF and their collaborators are now focused on finalizing the study design and advancing regulatory discussions to ensure alignment with the FDA and other regulatory bodies. Enrollment for the Phase 2/3 study is anticipated to begin later this year, pending readiness and funding.
Yaniv Glazer, GHF’s CEO said of the upcoming APBD clinical trials, “Additional updates will be shared in the coming months, including information on participating clinical centers and patient eligibility. We remain committed to advancing this important mission and deeply appreciate the continued support of the APBD community.”