We’re excited to share that Golden Heart Flower, Ltd. (GHF) has just launched a Phase I clinical trial in Europe to study GHF-201 in adult healthy volunteers. GHF-201 is a therapy designed to reduce polyglucosan body accumulation — the underlying cause of key symptoms, including bladder dysfunction and progressive loss of mobility — in Adult Polyglucosan Body Disease (APBD) patients.
Yaniv Glazer, CEO of GHF, shared, “This milestone marks a significant step forward for us, as we work to advance our mission to develop therapies for APBD and other rare but more prevalent diseases.”
Phase I clinical trials are conducted with the goal of evaluating the safety, tolerability, and exposure of a new treatment by administering single and multiple increasing doses to healthy volunteers.
If the Phase I clinical trial is successful, GHF plans to engage with regulatory agencies and discuss its next step in a clinical development program. Such a program is essential in demonstrating that a treatment is both safe and effective for its intended use and is typically required by agencies like the US Food and Drug Administration (FDA) before a new drug can be brought to the market.
Who is Golden Heart Flower?
GHF was founded by a family personally impacted by APBD and has since become a leader in the research and development of therapies targeting this disease area. The company’s name – drawn from a Hebrew fairy tale that symbolizes love and healing – reflects its deep commitment to changing the narrative for patients. For GHF, “There’s nothing else to try,” is unacceptable. The company’s lead compound, GHF-201, is a small molecule therapy developed in collaboration with Prof. Or Kakhlon (Hadassah Medical Center) and Prof. Miguel Weil (Tel Aviv University).
How Does GHF-201 Work?
GHF-201 works by degrading polyglucosan bodies, the harmful build-ups in the body that cause the symptoms of APBD. In APBD mouse models, GHF-201 has shown promising results, including reduced polyglucosan build-up, improved motor function, and extended lifespan. GHF has been running an Urgent Compassionate Use Program at Hadassah Medical Center for nearly four years, under the supervision of Prof. Alexander Lossos. With approval from Israel’s Ministry of Health, the program has provided treatment to three APBD patients using GHF-201.
Data from the Phase I clinical trial in healthy volunteers, as well as from the Urgent Compassionate Use program in APBD patients and pre-clinical studies, will help to inform the doses to be used in the next clinical pivotal phase(s) of development.
In late 2023, the FDA granted Orphan Drug Designation (ODD) to GHF-201, helping to accelerate the development and approval of drugs for rare diseases. The designation also impacts the drug’s market protection in the treatment of glycogen storage disease type IV, which includes APBD. Recently, GHF-201 also received Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA). The two designations provide key development and protection incentives for GHF-201 as a rare disease therapy and underscore its potential to meet an unmet medical need in the APBD and possibly the broader GSD communities.
Shlomi Schneider, GHF’s Co-Founder stated, “We’re raising an investment round to advance GHF-201 into the pivotal phase. Now is the time to join the mission and make a lasting impact on the future of APBD treatments.”
According to Jeff Leveson, DDS, Co-President of the APBD Research Foundation, “I am proud that our Foundation supported the groundbreaking research of Prof. Kakhlon and Prof. Lossos over many years — work that has laid the foundation for this exciting therapeutic initiative. We are deeply grateful to GHF for its professional, rigorous, and visionary efforts in helping bring the promising treatment one step closer to APBD and allied disease patients.”