Abeona Therapeutics (ABEO) Jefferies Global Healthcare Conference 2025 summary

Company overview and recent developments

• Transitioned to commercial-stage with ZEVASKYN approval for RDEB, receiving a $155M PRV sale and funding for eight quarters.

• Anticipates profitability in early 2026, with strong partner programs and upcoming data from Ultragenyx.

• Market cap is around $300M, considered undervalued by management.

Product launch and patient access

• Lurie Children's Hospital is the first activated treatment center, with positive feedback from stakeholders.

• Five qualified treatment centers (QTCs) targeted for activation by year-end, with sufficient patient demand to meet 2024 and early 2025 goals.

• Patient interest is high, with no refusals reported and active engagement through multiple channels.

• Initial treatment goal is 10-14 patients in 2024, supported by manufacturing capacity and patient inquiries.

Manufacturing and operational planning

• Manufacturing slots are sufficient for 2024 goals, with a December shutdown planned to minimize disruption.

• Capacity to scale from 4-6 slots/month in 2024 to 10/month by mid-2026, with staff hiring and training underway.

• Facility expansion is planned on the same floor, with $25-30M investment over 24 months, targeting late 2027 for additional capacity.

• Regulatory processes for facility changes are on track, with FDA discussions and change control protocol submission planned.