IntraBio is on a roll with its expansion plans for chemically altered amino acid, Aqneursa.
The Texas-based pharma just secured FDA approval to expand the label of Aqneursa, which is now the first treatment indicated for a rare and prematurely fatal neurodegenerative disease called ataxia-telangiectasia (A-T).
The positive ruling was based on data from IntraBio’s phase 3 IB1001-303 phase 3 data announced earlier this year. In the crossover study, which included 73 A-T patients ages 4 to 50, Aqneursa significantly improved ataxia symptoms and provided functional benefits for everyday life compared with placebo within 12 weeks of starting treatment.
Data showed a nearly 2-point improvement in the Scale for the Assessment and Rating of Ataxia (SARA), a tool used to measure ataxia patient symptoms. The FDA prioritized “functional SARA score”, a modified metric focused on gait, sitting, stance, and speech. In this measure, Aqneursa offered a 0.6-point advantage. There were no serious adverse events, and no patients stopped treatment due to negative side effects.
“This is a historic day for the A-T community and the patients and caregivers who have coped for decades without a treatment approved specifically for A-T,” said Brad Margus, founder of the A-T Children's Project.
Driven by a mutation in the ATM gene, ataxia-telangiectasia is marked by a progressive loss of balance, coordination and fine motor skills, as well as immunodeficiency, speech issues and increased cancer risk. It typically manifests in early childhood and affects an estimated 1 in 40,000 people worldwide.
The FDA cleared Aqneursa, given as an oral suspension, for both children and adults with A-T, as long as they are over 15 kilograms (33 pounds).
IntraBio CEO, Mallory Factor, said the rare disease pharma is “committed to making sure that families who could benefit from Aqneursa are able to access it.” The drugmaker manages a financial support program to help families offset the out-of-pocket costs of Agneursa, which can cost $15,000 every one to two weeks.
A-T marks Aqneursa’s second indication. The drug was originally approved by the FDA in 2024 to treat lysosomal storage disorder, Niemann-Pick disease type C. And in early 2026, it snagged the same approval from the European Commission.
How Aqneursa treats either neurological condition is still unclear. IntraBio suspects the drug works by correcting energy metabolism in the brain rather than treating any one specific genetic defect.
The privately held pharma is putting that theory to test in a third indication. IntraBio is currently wrapping up recruitment for a phase 3 testing Aqneursa in a group of disorders caused by mutations in the CACNA1A gene. CACNA1A disorders affect 1 in 11,700—about 30,000 people in the U.S. alone—and, like A-T, there is no approved therapy for these patients, according to the company. IntraBio expects to be over-enrolled and moving forward with data collection by October.