SEC filings and other documents

Pipeline advances and regulatory milestones drive growth, with pivotal data and approvals expected soon.

Pivotal Angelman phase III data and key gene therapy approvals expected this year.

Angelman, bone, and gene therapy programs advance with pivotal data and regulatory milestones ahead.

Revenue growth and late-stage pipeline drive path to profitability and new approvals in 2026.

Strong rare disease pipeline, revenue growth, and key data readouts expected in 2024.

Poised for profitability in 2027, driven by late-stage pipeline and strong revenue growth.

2025 revenue up 20%, restructuring targets 2027 profitability, with key launches ahead.

Phase 3 data in rare diseases and gene therapy filings are set to drive growth this year.

Imminent OI and gene therapy data, plus new launches, signal a transformative period ahead.

Q3 revenue up 15% to $160M; $400M royalty sale strengthens balance sheet ahead of pivotal data.

Strong pipeline and clinical execution position the company for profitability by 2027.

Q2 revenue up 13% to $166M; pipeline advances with key milestones and strong commercial growth.

Strong commercial growth and late-stage pipeline position the business for profitability by 2027.

Multiple rare disease launches and global expansion drive growth toward 2027 profitability.

Q1 2025 revenue up 28% year-over-year, with strong growth in Crysvita and advancing late-stage programs.

Multiple late-stage programs and strong revenue growth position for profitability by 2027.

2024 revenue up 29% to $560M; 2025 guidance targets 14–20% growth.