Strong Atruby commercial performance
Atruby net product revenue of $146,000,000 in Q4 2025 and $362,400,000 for full year 2025; total company revenue of $154,200,000 in Q4 and $502,100,000 for FY2025 (FY2025 revenue up ~$280.2M, ~126% year-over-year). Company reported 35% quarter-over-quarter growth in net product revenue (Q4) and >25% NBRx share as of 12/31/2025. As of Feb 20, 2026, Atruby had 7,804 unique patient prescriptions written by 1,856 unique prescribers, demonstrating accelerating new patient growth and prescriber depth.
Three positive late-stage readouts across pipeline
Company announced positive top-line Phase 3 results for infigratinib in achondroplasia and positive Phase 3 for Encalarec NADH1 (BBP-418) in LGMD2I, plus other late-stage successes, marking three successful late-stage readouts and signaling portfolio maturation and de-risking.
Robust Phase 3 efficacy and clean safety for infigratinib (achondroplasia)
Phase 3 met primary endpoint: change from baseline in average height velocity at week 52 (p < 0.0001) with a mean treatment difference of +2.1 cm/year versus placebo. Key secondary: statistically significant improvement in body proportionality (LS mean difference -0.05, p < 0.05 in children <8 years) and height z-score increase of +0.41 SD (p < 0.0001). Safety: well tolerated with no discontinuations or SAEs related to study drug; three mild/transient hyperphosphatemia events (4% on active) with no dose reductions.
Commercial and launch readiness for additional programs
Company building commercial leadership for LGMD2I and preparing for ADH1 launch activities; identified >1,700 unique patients in claims data for ADH1. Management anticipates launches for Encalorate and BBP-418 in late 2026 or early 2027 and has a roadmap for global launch footprints based on Atruby launch playbook.
Improving cash profile and runway
Ended 2025 with $587,500,000 in cash, cash equivalents, and marketable securities and completed issuance of $632,500,000 aggregate principal amount of 2033 convertible notes in January 2026, providing significant runway. Management expects cash burn to roughly hold steady through 2026 then decline, with the pipeline projected to begin generating cash in late 2027 and become a cash-generating engine by 2028 (company projects >$600,000,000 in profit in 2028 from four post–Phase 3 assets).
Potential non-dilutive value via Priority Review Vouchers (PRVs)
Three programs already have Rare Pediatric Disease designation and could be eligible for PRVs on approval: limb-girdle (LGMD2I), infigratinib (achondroplasia), and Canavan gene therapy—representing potential non-dilutive asset value (PRV market observed around ~$200–$300M each).
Operational efficiency in R&D
Management highlighted the ability to advance programs from preclinical to Phase 3 at under $300,000,000 in many cases and emphasized an R&D engine with higher-than-average probability of technical success, supporting scalable organic growth.