Strong Revenue Growth
Total net revenue of $36.2M in Q1 2026, a 78% increase versus $20.4M in Q1 2025 (incremental $15.8M). Composition: $24.6M MIPLYFFA U.S. net sales, $10.2M net reimbursements from the global EAP (Arimoclomol), $1.1M royalty revenue and $0.3M from OLPRUVA.
Profitability Driven by Strategic Transaction
Reported net income of $37.9M (EPS $0.62 basic, $0.60 diluted) versus a net loss of $3.1M a year ago. This includes a one-time gain of approximately $43.3M from the SDX portfolio sale; excluding one-time items and related tax provision, adjusted quarterly net income was $11.5M (approximately $0.18 per diluted share).
Solid Balance Sheet and Debt Elimination
Cash, cash equivalents and investments of $236.8M as of March 31, 2026. Company is debt-free after early debt retirement, which management says will save ~ $8M per year in interest expense. Cash decreased modestly by $2.1M (-0.9%) versus Dec 31, 2025 primarily due to deleveraging.
Commercial Launch Traction for MIPLYFFA
170 prescription enrollment forms for MIPLYFFA from launch through March 31, 2026 (9 received in Q1). Management estimates this reaches roughly half of the currently diagnosed U.S. NPC patient population (300–350 diagnosed). Patient mix is ~50% adults and 50% children.
Clinical and Guideline Validation
MIPLYFFA added to NPC clinical practice guidelines; company highlights >5 years of data across >270 NPC patients from trials, open-label extension, global EAP and pediatric substudy. MIPLYFFA + miglustat is described as the first disease‑modifying therapy shown to halt progression at 12 months in a randomized trial, with benefits as early as 12 weeks and durable effects >5 years.
Global Access Progress
122 patients enrolled in the global Expanded Access Program across multiple geographies. EMA Marketing Authorization Application for Arimoclomol is under review; responses to the 120-day list were submitted within the clock stop period and review is progressing.
Late-Stage Pipeline Advancement
Phase III DiSCOVER trial for Celiprolol (VEDS) has enrolled 62 patients (10 added in Q1). Study is event-driven; 2 confirmed events recorded toward 28 required for the interim analysis. Management pursuing additional FDA engagement in H2 2026 to explore acceleration pathways.
Portfolio Optimization Monetization
Sale of SDX portfolio to Commave Therapeutics for $50M (net proceeds $45M); $40.5M received in Q1 with remaining $4.5M received in April. Aquestive entitled to 10% ($5M) per contractual obligation. Transaction provided non-dilutive capital and a one-time gain recognized.