Levacetylleucine

The Austin, TX-based biotech told Endpoints News it plans to submit an application to the FDA in April that would seek approval for its drug, Aqneursa. Known generically as N-acetyl-L-leucine, Aqneursa was first approved in 2024 to treat neurological symptoms related to another rare disease called Niemann-Pick disease type C. There are currently no approved treatments specifically for ataxia-telangiectasia. Seventy-three patients with the disorder were enrolled as part of the Phase 3 trial, and they received either Aqneursa or placebo every day. After 12 weeks of treatment, patients who received Aqneursa saw a -1.92-point improvement on a measure of motor function called the Scale for the Assessment and Rating of Ataxia, according to the results reported Wednesday. Meanwhile, patients who received placebo saw a -0.14-point score change. That difference was statistically significant with a p-value of p Ataxia-telangiectasia is a progressive disease characterized by ataxia — a loss of muscle control — and tiny red blood vessels called telangiectasias on the skin and in the eyes. The disease starts in childhood, gets worse over time and can affect many organs of the body. IntraBio said the study also met secondary endpoints and that the drug was “safe and well-tolerated” in the study. The biotech also said Wednesday that European authorities granted marketing authorization to Aqneursa for Niemann-Pick disease type C. It’s also planning to seek European approval for the treatment of ataxia-telangiectasia. The content above comes from the network. if any infringement, please contact us to modify.

The company is heading to global regulators "immediately" based on the results of its phase 3 study of Aqneursa in Ataxia-Telangiectasia. As IntraBio works to grow the global treatment market for Aqneursa in Niemann-Pick disease type C, the company is already carving the path to a second potential use for the drug.The Texas-based pharma is planning to “immediately” advance regulatory bids with the FDA, the European Medicines Agency (EMA) and other global regulators based on the results of its phase 3 study of Aqneursa (levacetylleucine) in Ataxia-Telangiectasia (A-T)—a progressive neurodegenerative disease that impacts about 1 in 70,000 people and has no approved treatments, IntraBio said in a Wednesday press release.In the study, treatment with Aqneursa prompted a statistically significant and clinically meaningful -1.88-point improvement on the Scale for the Assessment and Rating of Ataxia (SARA) over placebo after 12 weeks, the company said. The trial’s secondary endpoints were also met based on positive showings on other clinical measures of ataxia, IntraBio added.The results represent a “major scientific and clinical milestone,” trial investigator Franziska Hoche, M.D., said in the release, offering “compelling evidence that levacetylleucine has a meaningful impact on A-T patients’ lives.”A-T typically manifests in early childhood with symptoms that include progressive loss of coordination, impaired speech and eye movements and wheelchair dependence as the brain’s cerebellum degenerates. Many patients also experience visible blood vessel changes, immune system deficiencies, lung disease and a significantly increased risk of cancer, the company said.“This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T,” Brad Margus, who founded the A-T Children’s Project nonprofit, added in the release. “We look forward to continuing to collaborate with IntraBio to help ensure levacetylleucine is rapidly approved by the FDA and made available for patients in our community, given their urgent need for effective, approved treatments.” IntraBio partnered up on the study with key opinion leaders and A-T patient organizations to evaluate symptomatic effects and longer-term clinical outcomes, the company noted. Elsewhere, Aqneursa is also in late-stage development for a group of rare neurological conditions linked to mutations in the CACNA1A gene.Niemann-Pick disease type C market gainsIt wasn't long ago that IntraBio and Aqneursa entered the commercial frontier with an FDA nod in lysosomal storage disorder Niemann-Pick disease type C (NPC), quickly becoming the second therapy approved for the condition just days after Zevra Therapeutics’ Miplyffa crossed the regulatory finish line in 2024.IntraBio made its treatment available immediately upon approval, effectively erasing the short head start Miplyffa had as the latter contender took a couple extra weeks to get off the ground. Compared to Miplyffa’s dosing regimen that includes Johnson & Johnson’s enzyme inhibitor Zavesca, Aqneursa is the U.S.’s first stand-alone therapy that can treat the adult and pediatric patient populations, IntraBio points out. As of Jan. 21, Aqneursa is already making broader global headway than its rival thanks to a new approval from the European Commission. In Europe, the drug must be taken alongside Zavesca or by itself in patients who can't tolerate the enzyme inhibitor, according to the company's Wednesday release.Miplyffa, meanwhile, has not yet snagged its coveted European approval. At the recent J.P. Morgan Healthcare Conference in San Francisco, Zevra execs placed a high emphasis on the sizeable commercial opportunity that the European market presents, given higher diagnoses rates there.IntraBio, a private company, has not disclosed sales figures for Aqneursa, but Zevra has secured 137 prescription enrollment forms since its NPC launch as of the third quarter. Analysts at Delveinsight expect Aqneursa to generate $500 million in peak U.S. sales in NPC.