NAYA Biosciences is expanding its clinical portfolio through the acquisition of an innovative gene therapy program from Florida Biotechnologies focused on treating rare mitochondrial diseases. This binding deal demonstrates NAYA’s strategy to rapidly build pipeline assets in high-potential areas like gene therapy.
NAYA has entered into a binding letter of intent to acquire Florida Biotechnologies for $20 million in NAYA shares, with potential milestone payments up to $5 million more. The deal specifically targets Florida Biotech’s clinical-stage gene therapy for Leber’s Hereditary Optic Neuropathy (LHON).
LHON is a rare mitochondrial genetic disease leading to progressive vision loss and blindness. There are currently no approved treatments. Florida Biotech’s AAV gene therapy aims to deliver functional copies of the mutated gene to restore cellular function.
Encouraging Safety Data
This therapeutic has already demonstrated encouraging safety data in a 28-patient Phase 1 trial conducted by Florida Biotech and the University of Miami’s Bascom Palmer Eye Institute. Building on this initial study, NAYA aims to accelerate Phase 2 development and provide early access to patients.
The therapy delivers the gene of interest inside an adeno-associated virus (AAV) vector containing a mitochondrial targeting sequence. This sequence enables delivery specifically to mitochondria, the key site of pathology in LHON and many other mitochondrial diseases.
Broad Applicability
While the lead indication is LHON, NAYA highlights this approach has potential for various mitochondrial orphan diseases. The platform’s strong intellectual property covers use of different AAV capsids and routes of administration.
This flexibility and the high unmet need enables a streamlined regulatory path, including Regenerative Medicine Advanced Therapy and priority review voucher opportunities. The technology has already received over $6 million in grant funding to date.
The asset aligns with NAYA’s focus on innovative regenerative medicine approaches with near-term clinical potential. Gene therapy represents a key component of NAYA’s strategy to build a pipeline addressing major unmet needs.
Expertise in Developing Gene Therapies
NAYA has extensive in-house expertise to advance the acquired AAV gene therapy through late-stage trials toward commercialization. For example, NAYA Chief Medical Officer Dr. Fred Grossman previously led development of the first approved gene therapy, Glybera, during his time at uniQure.
Florida Biotech co-founder Dr. Peter Kash also joins NAYA’s board, bringing over 36 years of biotech leadership experience, including developing and financing gene therapy pioneer Kite Pharma. This expertise will prove valuable for unlocking the platform’s full potential.
Execution of Broader Growth Strategy
NAYA Biosciences was formed earlier in 2023 through the merger of Clinigence Holdings and 4Front Biotech. This deal created a holding company structure to facilitate pipeline expansion through additional strategic acquisitions.
The Florida Biotechnologies program represents the next step in this growth strategy. NAYA plans to continue acquiring high-potential clinical assets to build a robust portfolio spanning gene therapy, oncology, fertility and other areas.
These efforts support NAYA’s overall mission of providing patients earlier access to transformative therapies. The company aims to take an entrepreneurial approach to aggressively advance new technologies through development.
Pending Merger to Unlock Public Markets
NAYA’s deal to acquire Florida Biotechnologies is contingent upon the completion of NAYA’s previously announced merger with fertility company INVO Bioscience, expected in Q1 2024.
The merger will create a publicly traded life sciences company under the INVO name, providing access to capital to support NAYA’s development programs and commercialization. The combined company is valued at over $100 million.
Conclusion
In summary, NAYA Bioscience’s move to acquire Florida Biotechnologies’ AAV gene therapy strengthens its pipeline and aligns with its strategy to focus on high-potential clinical assets. Leveraging its development expertise, NAYA is positioned to accelerate this innovative therapy toward commercialization and fill an important unmet need for patients with mitochondrial diseases.