Regulatory momentum toward U.S. resubmission and 2026 launch
Company reaffirmed guidance to resubmit the BLA and target a U.S. launch upon approval in 2026. Since the November Type A meeting, FDA engagement has been active (including an FDA field team visit to the Catalent, IN site) and the FDA indicated intent to reinspect the Catalent facility after routine manufacturing resumed in late February. Management is prepared to resubmit the BLA quickly following a successful reinspection.
European MAA review progressing
The Marketing Authorization Application (MAA) with the EMA is under review and management expects a decision in mid-2026, enabling potential launch sequencing in Europe beginning with Germany in H2 2026.
Pipeline progress — multiple programs advancing
Phase 2 OVAL (ipilimumab/epitogromab) continues enrolling infants/toddlers (<2 years) including SMN-treated patients. FSHD IND cleared and FORGE Phase 2 (randomized, placebo-controlled, ~60 patients) is on track to start mid-2026. SRK‑439 (high‑potency myostatin inhibitor) is dosing in Phase 1 with topline data expected in H2 2026. Subcutaneous formulation Phase 1 showed 800 mg subQ produced overlapping PD with 800 mg IV; further clinical/regulatory strategy planned.
Commercial preparedness and community engagement
U.S. commercial team deployed: engaging ~140 SMA treatment centers and ~2,600 prescribing physicians; expanded specialty pharmacy network for continuity of care; home infusion network with >10,000 affiliated nurses; disease awareness campaign 'Life Takes Muscle' launched; active payer engagement (national/regional, Medicare/Medicaid).
Strengthened balance sheet and financing flexibility
Ended 2025 with $368,000,000 in cash and cash equivalents. Q4 included $60,400,000 from warrant exercises (≈16% of year-end cash). Secured a new debt facility with Blue Oak Capital for up to $550,000,000 (≈149% of year-end cash): $100,000,000 available at close (used to repay prior facility), additional $100,000,000 expected this quarter, option to draw $150,000,000 following FDA approval, and incremental option up to $200,000,000 by mutual consent.
Operating spend transparency and prioritization
Q4 2025 operating expenses were $91,900,000 (including $19,400,000 non‑cash stock-based compensation). Excluding stock comp, Q4 operating expenses were $72,500,000. Full year 2025 operating expenses were $384,600,000 (including $75,600,000 stock comp); ex-stock comp FY2025 op ex = $309,000,000. Non‑cash stock compensation represented ~21.1% of Q4 op ex and ~19.7% of FY op ex.