Application:
Prader-Willi Syndrome (PWS) presents significant challenges in bone health, including reduced bone density, increased fracture risk, and osteoporosis. Current treatments for osteoporosis provide limited effectiveness in PWS due to the complex genetic factors involved. This technology addresses a critical need for an effective, targeted therapeutic solution that supports bone remodeling and mass in PWS patients, leveraging advanced fatty acid amide compounds.
Our innovation:
Our research presents Fatty Acid Amides (FAAs) as innovative drug candidates, with HU-671 standing out as a promising derivative of Oleoyl Serine (OS). In preclinical models, HU-671 demonstrates enhanced efficacy compared to natural OS by resisting hydrolysis and extending its active presence in bone tissues. Unlike other osteoporosis treatments, both OS and HU-671 uniquely targets bone remodeling mechanisms, both increasing bone formation and reducing bone resorption. Additionally, HU-671 has proven effective in rescuing bone loss in mouse models for PWS, where OS levels are reduced due to genetic deletions affecting bone mass, such as MAGEL2 loss.
Advantages:
Enhanced Mechanistic Precision: HU-671’s design amplifies the natural effects of OS on bone cells, delivering superior dual-action benefits in osteoblast activation and osteoclast suppression.
Dual Action: OS & HU-671 promote osteoblast activity while inhibiting osteoclastogenesis, effectively balancing bone remodeling.
Stabilized Molecular Structure: The α-methylation modification on HU-671 protects against enzymatic degradation, extending its bioactivity and increasing its therapeutic efficacy.
Natural Origins with Enhanced Safety: As a derivative of the endogenous FAA OS, HU-671 combines natural therapeutic benefits with improved pharmacological stability, providing a safer alternative to conventional osteoporosis medications.
Opportunity:
The development of HU-671 and OS represents a significant market opportunity for addressing the high unmet medical need in PWS-related osteoporosis. HU-671, in particular, shows potential not only to transform bone health management in PWS but also to extend its therapeutic benefits to other osteoporosis indications. With its dual-action and superior efficacy, HU-671 could fill a critical gap in the current osteoporosis treatment landscape. The technology offers strong partnership opportunities in rare disease and metabolic disorder markets, with potential for early access to underserved osteoporosis segments and a first-in-class designation.
