ABEONA THERAPEUTICS INC. (ABEO)·Q4 2024 Earnings Summary

Executive Summary

• FDA review of pz-cel remained on track with an April 29, 2025 PDUFA date; management received a marked-up draft USPI on March 14 and expects first U.S. patient treated in 3Q 2025 if approved .
• Commercial readiness advanced: five U.S. EB centers are being onboarded as qualified treatment centers (QTCs); payer engagements exceed 40 PIIEs; revenue recognition will occur upon administration (surgery) .
• Manufacturing plan: supply-gated launch with initial capacity ~4 treatments/month, ramping to ~6 by late 2025/early 2026 and a maximum ~10/month in 1H 2026; annual maintenance shutdown planned in Dec–Jan to protect quality systems .
• Financials: 2024 R&D $34.4M, G&A $29.9M, net loss $63.7M; cash and investments $98.1M at year-end, with runway into 2026 (pre any pz-cel or PRV proceeds) .
• Potential catalysts: FDA approval and possible PRV award; management noted recent PRV sales “north of $150M” and intends to optimize timing and price rather than sell immediately .

What Went Well and What Went Wrong

What Went Well

• Regulatory momentum: “We received from the FDA a marked-up draft USPI to initiate label discussions… no big surprises in the big ticket items” .
• Commercial readiness: five EB centers in onboarding; time from biopsy to administration ~25 days; payer education (>40 PIIEs) and dual procurement models (buy-and-bill or via specialty pharmacy) to facilitate access .
• Reimbursement groundwork: CMS granted an ICD-10-PCS procedure code and assigned pz-cel to Pre-MDC MS-DRG 018, among the highest inpatient reimbursement categories for cell and gene therapies .

What Went Wrong

• Launch to be supply-gated: management explicitly expects a patient backlog until manufacturing ramps; initial output ~4 treatments/month .
• Continued operating losses and elevated G&A driven by pre-commercial investment; 2024 net loss $63.7M and G&A rose to $29.9M on launch preparation costs .
• Operational complexity: autologous process requires extensive coordination and site activation (~3 months post-approval), potentially elongating early revenue ramp .

Financial Results

Quarterly snapshot (USD Millions; $ in mm; EPS in $)

Notes: Q4 figures are derived from FY minus nine-month (9M) results, both reported by the company.

Full-year comparison (USD Millions; $ in mm)

No segments are reported.

Operational KPIs

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• “We’re less than six weeks away from our PDUFA date… we received… a marked-up draft USPI… [and] there are no big surprises [on] the label” — CEO Vish Seshadri .
• “With an average of two treatments per patient and about 750 patients, we foresee about 1,500 treatment opportunities… even with a conservative floor of $1.5 million per treatment, we believe pz-cel can have a cumulative revenue potential of more than $2 billion in the U.S. alone” — CCO Madhav Vasanthavada .
• “We currently estimate our initial manufacturing capacity during the early launch phase to be about four treatments per month… ramp… to six… and… a maximum capacity of 10 monthly treatments in the first half of 2026” — CTO Brian Kevany .
• “Revenue recognition occurs upon pz-cel administration… [we] are planning for commercial launch in the third quarter of this year if pz-cel is approved” — CFO Joseph Vazzano .
• “We do not anticipate any potential factors that may preclude a PRV… recent PRVs… sold for north of $150 million… we will optimize pricing over speed” — CEO Vish Seshadri .

Q&A Highlights

• CMC remediation status: Company addressed all 12 CRL items; multiple FDA interactions support confidence heading into action date (review ongoing, cannot pre-judge outcome) .
• Launch dynamics/backlog: Expect early backlog given supply-gated launch and strong physician interest; slot allocation will prioritize QTCs as they activate .
• Physician/patient activation: Tight-knit community and advocacy partners; plan to balance digital and field presence while gating promotion to match supply .
• PRV: Satisfies key eligibility criteria (rare pediatric designation, priority review, first-time product); management tracking resale market and timing .
• Ex-U.S.: Evaluating EU pathways (e.g., Austria/Germany centers of excellence) and logistics vs local mfg; U.S. approval/launch is the near-term priority .
• Treatment throughput: Up to 12 keratinocyte sheets per treatment (40 cm² each; ~480 cm² total); many patients may require ~2 rounds to address large chronic wound burden .

Estimates Context

• Wall Street consensus (S&P Global) for Q4 2024 revenue/EPS was unavailable in this session due to data access limits; the company reported no revenue in 2024 and did not disclose standalone Q4 EPS (only FY EPS). Values retrieved from S&P Global were unavailable for this request.
• Implications: Near-term Street models are typically pre-revenue until FDA approval; post-approval, models should incorporate a supply-gated 2H’25 ramp, revenue recognition upon administration (~25 days after biopsy), and manufacturing cadence constraints (4→6→10/month) .

Key Takeaways for Investors

• Binary catalyst: FDA action on April 29, 2025; label alignment appears constructive; approval could unlock PRV and set up a 3Q’25 launch .
• Launch will be supply‑gated: Track QTC activations and monthly capacity progression (4→6→10 treatments/month) as primary drivers of 2H’25–2026 revenue trajectory .
• Access set-up looks favorable: CMS coding and DRG 018 assignment plus >40 payer exchanges reduce reimbursement friction; dual procurement offers site flexibility .
• Early backlog likely: Expect measured uptake as centers gain experience; backlog clearing depends on manufacturing ramp and site throughput .
• PRV optionality: If granted, potential non-dilutive cash (mgmt notes recent resales “north of $150M”); management will optimize price/timing .
• Financial runway into 2026: Sufficient to execute launch and capacity ramp before any pz-cel cash flows or PRV monetization .
• Medium-term thesis: Durable efficacy profile, concentrated prescriber base, and high willingness-to-travel in RDEB support focused center strategy; execution risk centers on CMC reliability, site activation pacing, and scaling manufacturing .