The third quarter of 2025 was marked by significant operational progress as we continue to scale the ZEVASKYN commercial launch to meet growing patient demand. We're seeing growing patient demand for ZEVASKYN, the first and only autologous cell-based gene therapy for the treatment of adult and pediatric patients with recessive dystrophic EB, or RDEB. We are happy to also report that demand for ZEVASKYN continues to grow.
In summary, we are very encouraged by the growing patient demand, patients actively progressing toward treatment, continued growth of the QTC site network, and a favorable market access landscape for ZEVASKYN. The current cash position, without accounting for anticipated revenue from ZEVASKYN, is expected to be sufficient to fund current and planned operations for over two years. This increase reflects the reclassification of R&D expenses as noted, along with increased headcount and professional costs associated with the commercial launch of ZEVASKYN.
Gao brings over 20 years of industry experience and is a recognized expert in gene therapy, especially in ophthalmology, which will be valuable as we advance our pipeline. Can you remind us then, in terms of revenue recognition from the time that you dose these patients, how long until revenue recognition? The revenue is recognized when the product is applied on the patient from an accounting standpoint.
| Metric | Period | Current guidance |
|---|---|---|
| First commercial treatment | Q4 2025 | Shifted to Q4 2025 following release-assay optimization; biopsies resumed November 2025 |
| Company-wide profitability | 1H 2026 | Still first half of 2026; management sees no significant impact from the first-treatment delay |
| ZPOFs / demand | Current | 12 signed ZEVASKYN Product Order Forms; identified QTC patients more than doubled to ~30 |
| Market access | Current | >80% of commercial lives covered by published policies; all 51 Medicaid programs plus Puerto Rico effective Oct 1, 2025; CMS J-code effective Jan 1, 2026 |
| Year-end plant shutdown | Dec 2025-Jan 2026 | FDA-mandated maintenance/recalibration shutdown from mid-December for about a month |
| Metric | YoY | Note |
|---|---|---|
| R&D expense | $4.2M vs $8.9M | Costs capitalized into inventory and select production/engineering-run costs reclassified to SG&A after approval. |
| SG&A expense | $19.3M vs $6.4M | Reclassification of R&D plus increased headcount and professional costs for the commercial launch. |
| Net loss | -$5.2M vs -$30.3M | Lower operating loss year over year; no PRV gain in the quarter (that was recognized in Q2). |
| EPS | -$0.10 vs -$0.63 | Narrower net loss. |
| Cash and investments | $207.5M (Sept 30, 2025) | More than two years of runway without ZEVASKYN revenue. |
| Identified QTC patients | ~30 (from ~12) | Growing demand at the QTCs, excluding the larger non-QTC referral pool; 12 ZPOFs signed. |
| Topic | Previous mention | Current period | Trend |
|---|---|---|---|
| Manufacturing / sterility assay | On track | A false-positive on a newly required rapid sterility assay forced a lot rejection and a temporary biopsy pause; assay optimized, validated and submitted, biopsies resumed November 2025, pushing first treatment to Q4. | — |
| Commercial demand | ~50 identified | QTC-identified patients doubled to ~30; 12 ZPOFs signed; management expects high conversion given motivated, severe patients. | — |
| Market access | Early wins | All major commercial payers have published policies (>80% of commercial lives); Medicaid baseline coverage across all states effective Oct 1; permanent CMS J-code effective Jan 1, 2026. | — |
| QTC network | 2 centers | Third center (Children's Hospital Colorado) activated; several more in onboarding. | — |
| Pipeline / team | - | ABO-503 (XLRS) selected for FDA RDEA pilot program; Dr. James A. Gao appointed SVP, Head of Clinical Development and Medical Affairs. | — |