CORDStrom regulatory progress (PIP and MAA preparation)
Submitted Pediatric Investigation Plan (PIP) to U.K. regulator; approved for rapid assessment with response received April 9 and no substantial issues raised. Marketing Authorization Application (MAA) documentation across five sections is well underway; company expects to submit to MHRA in early Q3 and is targeting parallel EMA/FDA filings to meet the previously stated end-of-year filing timeline.
Manufacturing readiness and supply secured
Started the first of three process validation manufacturing runs on time, with two remaining runs scheduled to meet MAA deadlines. Concluded and signed an amended material transfer agreement with the Anthony Nolan U.K. Cord Blood Bank to secure long-term, high-quality umbilical cord tissue supply and permit U.S. lab testing required by the FDA (per prior Type B meeting).
Product definitions and commercialization planning
Active ingredient named by WHO as pobistrocel and company selected commercial drug name Ebstracel for the RDEB formulation; preparing regulatory and commercial documentation and engaging TMC Pharma (rare disease submission expertise) to run EMA and FDA submissions in parallel.
Progress on XPro clinical characterization
Advancing additional imaging analyses from the MINDFuL study, including MRI data focused on myelin preservation and structural integrity to further characterize XPro's potential as a disease-modifying therapy; exploring rare disease trial opportunities and partnership discussions for late-stage development.
Reduced quarterly net loss
Net loss attributable to common stockholders for Q1 2026 was $5.4 million versus $9.7 million in Q1 2025, a reduction of approximately 44.3% year-over-year, reflecting lower operating spend this quarter.
Lower R&D and stable G&A expenses; cash runway disclosed
R&D expenses decreased to $3.6 million in Q1 2026 from $7.6 million in Q1 2025 (approximately a 52.6% reduction). General and administrative expenses were roughly flat at $2.2 million versus $2.3 million (about a 4.3% decrease). Cash and cash equivalents were $21.4 million as of March 31, 2026, and management believes cash is sufficient to fund operations through Q1 2027; shares outstanding were ~26.6 million as of May 7, 2026.