Forest Hills Lab Asking to Open Phase 2 Trial of FHL-301 in Parkinson’s

Forest Hills Lab announced plans to soon apply to the U.S. Food and Drug Administration (FDA) for permission to open a Phase 2 clinical trial evaluating the tolerability and efficacy of FHL-301 in people with Parkinson’s disease.

FHL-301, or FHL CB-301, is reported to be a repurposed therapy acting as a peroxisome proliferator-activated receptor alpha (PPARa) agonist. Agonists are compounds that bind to a receptor and activate it, mimicking a biological response.

It activates PPARa that binds to specific DNA sequences, called peroxisome proliferator response elements, located in the promoter region of the target gene GDNF.

The GDNF gene encodes for a small protein known as glial cell line-derived neurotrophic factor that supports the growth, survival, and differentiation of dopaminergic neurons, which are gradually lost in Parkinson’s disease.

Plans for the Phase 2 clinical trial are based on the positive results from studies in animal models, where FHL-301 showed to be able to promote the activation (upregulation) of the GDNF gene.

“[S]cientific rationale for FHL-301 to treat Parkinson’s disease [PD] has been demonstrated with the animal disease model. It is very exciting to explore the efficacy of FH-301 with PD patients in upcoming phase 2 clinical study because FHL-301 has a unique mechanism of action which is not overlapped with other drugs,” Hahn-Jun Lee, PhD, chief science officer at Forest Hills Lab, which is based in Hong Kong, said in a press release.

“FHL-301 may be one of excellent alternative options for the treatment of Parkinson’s disease,” Lee added.

The release did not provide further details on FHL-301 as a previously approved and repurposed treatment.

Forest Hills states in its release that it is also developing other therapy candidates that target Parkinson’s disease. These potential therapies have shown relevant benefits in animals models of this disease, including anti-inflammatory effects and increased Parkinson’s-related gene expression (the process by which information in a gene is synthesized to create a protein).

The company is working with research centers in the U.S., including Rush University Medical Center, to advance these potential treatments, all for central nervous system disorders with few therapy options, with a goal of marketing them, it added.