Strong Revenue Growth
Q4 product revenue of $21.3M, up 83% year-over-year (Q4 2024: $11.6M); company more than doubled revenue in 2025 versus 2024 and provided 2026 guidance of revenue expected to exceed $110M.
Improving Profitability and Margins
Adjusted EBITDA margin improved to 29% in 2025 from 18% in the prior-year period; GAAP net income of $1.5M in Q4 and non-GAAP net income of $5.4M for 2025. Adjusted gross profit was $15.5M (73%) in 2025 versus $6.8M (59%) in prior year; company expects adjusted EBITDA margin of at least 30% in 2026 and adjusted gross margin comfortably above 70% (ramping to 75%–80%).
Desmota FDA Approval and Early Commercial Traction
Desmota (oral liquid desmopressin) FDA-approved in Feb 2026 and launched within two weeks; offers unique liquid formulation with clean label (no age restriction) addressing pediatric and adult central diabetes insipidus. Management estimates average net revenue of ~ $80,000 per patient per year and maintains $30M–$50M potential peak sales guidance; multiple patents extend to 2044.
Successful Product Relaunches and Patient Growth (Incrolex / Incralex)
Incrolex patient count increased from 67 at acquisition (Dec 2024) to over 100 on therapy with a goal of 120 by year end; company pursuing label harmonization study (open-label ~30 patients, first patient expected in Q3) which could increase the U.S. market opportunity up to fivefold if successful.
Alkindi & Kindivy Momentum
Alkindi and Kindivy franchise showing strong growth; Alkindi had its strongest year in 2025 in patient count and referrals. Management estimates target pediatric market ~5,000 children under age eight, has captured ~12% to date, and believes ~20% market share would support at least $50M peak annual sales.
Hemangiol Acquisition and Rare-Disease Commercial Strategy
Acquired Hemangiol for $14M in cash, relaunch planned May 1 with seven experienced commercial hires; Hemangiol treats an estimated 5,000–10,000 infants annually and management expects to optimize distribution, reduce gross-to-net, introduce zero copay, and position Hemangiol as a major product by 2027.
Galzin (Galcen) Growth and ET700 Opportunity
Galzin reached ~300 active patients one year after relaunch with a sizable addressable population still using OTC zinc; ET700 extended-release program to begin PET proof-of-concept in April with topline later in the year and management projecting ET700 peak potential in excess of $100M if successful.
Robust Pipeline and Ambitious Long-Term Targets
Busy clinical year planned (Candivy, Incrolex label harmonization, ET700, PK studies for AMGLIDIA and ET800). NDA plans include AMGLIDIA by year-end and ET800 in 2027. Company announced new long-term goals: build largest U.S. rare disease portfolio (13–14 products), exit 2027 at $200M revenue run rate, 50% adjusted EBITDA margin by 2028, and $500M revenue by 2030.