Abeona Therapeutics (ABEO) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Clinical and patient impact

• pz-cel addresses major unmet needs in RDEB by providing durable wound healing and pain relief, supported by long-term clinical data and strong patient testimonials.

• Phase I/II-A and pivotal phase III VIITAL studies show lasting benefits, with many patients expressing high satisfaction and willingness for repeat treatments.

• Ongoing studies and patient advocacy highlight demand for broader applications, including for hand surgeries and other wound types.

• Complementary positioning with other therapies is reinforced by physician and patient feedback, emphasizing the need for multiple treatment modalities.

• Patient excitement and advocacy are driving awareness and interest in the therapy.

Regulatory and manufacturing progress

• After receiving a CRL in April focused on CMC issues, nearly all FDA requirements have been addressed, with only rapid sterility testing remaining before resubmission.

• Close, collaborative engagement with the FDA, aided by multiple rare disease designations, has accelerated feedback and alignment on regulatory expectations.

• Manufacturing capacity at launch will support about 120 patients per year, with plans to expand as demand and patient identification increase.

• Five to seven geographically dispersed centers of excellence are being prepared for launch, with strong operational and clinical enthusiasm.

• Expansion of manufacturing and treatment sites will be guided by real-world demand and evolving epidemiological data.

Market access, pricing, and commercial outlook

• Payers, including major commercial insurers and Medicaid, show willingness to defer clinical decisions to physicians due to the ultra-rare nature and high unmet need.

• Budget impact analyses suggest a single treatment episode could be cost-effective compared to ongoing standard care, with pricing expected to align with other one-time gene therapies.

• Peak sales are projected at $500 million+, assuming manufacturing and site expansion to meet demand for 300-350 patients annually.

• Patient willingness to travel and referral networks support robust uptake at launch centers.

• No significant payer-imposed step edits or restrictions are anticipated due to the non-systemic nature and high disease burden.