Latest news with #IntraBio
Business Wire
29-07-2025
- Health
- Business Wire
AQNEURSA ® (levacetylleucine) Recommended for EU Approval by the CHMP to Treat Niemann-Pick Disease Type C
AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of AQNEURSA ® (levacetylleucine) for the treatment of Niemann-Pick disease type C (NPC). "This positive CHMP opinion represents another important milestone in expanding access to AQNEURSA to the global NPC community," said Dr. Marc Patterson, Chief Medical Officer of IntraBio. "The recommendation reflects the strength of our clinical data and the potential for AQNEURSA to be a foundational therapy for NPC, delivering meaningful benefits for patients. We are proud to work alongside the NPC community to bring this long-awaited treatment option to even more families.' NPC is a rare, inherited lysosomal disorder characterized by progressive neurological deterioration, leading to loss of motor function, difficulties with speech and swallowing, and cognitive decline. 3 NPC affects both children and adults, significantly impacting quality of life and daily functioning. 3,4 "For NPC families like mine, this positive opinion brings long-awaited hope for a treatment that can actually offer improvements," said Carmelo Fernández, President of Fundación Niemann-Pick de España. "We have waited years for a therapy that can make a meaningful difference in the lives of people with NPC, and today's announcement brings us one step closer." The positive CHMP's opinion is based on results from IntraBio's pivotal Phase III randomized, placebo-controlled, clinical trial (IB1001-301; NCT05163288), which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric and adult patients (n=60) with a confirmed diagnosis of NPC. AQNEURSA significantly improved neurological symptoms and functional abilities across its primary and all secondary endpoints within 12 weeks of treatment versus placebo, and was well tolerated throughout the development program. 1 The CHMP opinion was further supported by long-term extension phase data showing that treatment with AQNEURSA had disease-modifying and neuroprotective effects, helping to reverse disease progression over time. 2 Detailed results from the IB1001-301 trial were published in The New England Journal of Medicine in February 2024. 1 About AQNEURSA AQNEURSA was approved by the U.S. Food and Drug Administration (FDA) on 24 September 2024 for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. 5 Since approval in the U.S., AQNEURSA has experienced rapid adoption as a frontline therapy for NPC with continued growth in demand. IntraBio remains on track to proceed with additional global regulatory submissions for AQNEURSA in 2025 and beyond. IntraBio's Phase III Pivotal trial investigating N-Acetyl-L-Leucine (levacetylleucine) for Ataxia-Telangiectasia has completed recruitment in under two months, ultimately over-enrolling the trial by over 167%. Data readout is expected in Q1 of 2026. U.S. IMPORTANT SAFETY INFORMATION Embryo-Fetal Toxicity Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female's need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease. Pregnancy and Lactation For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued. There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition. Adverse Reactions The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. Drug Interactions Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy. Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized. To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or Please click here for Full Prescribing Information for AQNEURSA: About Niemann-Pick Disease Type C Niemann-Pick disease Type C (NPC) is a rare (1:100,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder. 6 The disease presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life. 3,4,7 About IB1001-301 IB1001-301 (NCT05163288) is a multinational, randomized, placebo-controlled, crossover trial that evaluates the safety and efficacy of IB1001 (AQNEURSA, levacetylleucine) in pediatric and adult patients with NPC. Patients aged 4 years and older were screened at trial sites in Australia, Europe, the United Kingdom, and the United States. Patients were assessed during a baseline period and then randomly assigned (1:1) to receive orally administered IB1001 or placebo for 12 weeks. At the end of the 12-week treatment period, patients crossed over and initiated therapy with the alternate study drug (IB1001 or placebo) over the subsequent 12-week period. Patients who completed the study had the option to participate in an open-label Extension Phase, with some patients having been dosed for over 5 years. About IntraBio IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological and neurodevelopmental diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverages the expertise of its scientific founders from the University of Oxford and the University of Munich. For more information about IntraBio, please visit the company's website at and follow on LinkedIn (@IntraBio-Inc). References 1. Bremova-Ertl T, et al. J Neurol. 2022;269(3):1651-1662 2. Patterson, Marc C., et al. "Disease-modifying, neuroprotective effect of N-acetyl-l-leucine in adult and pediatric patients with Niemann-Pick disease type C." Neurology 105.1 (2025): e213589. 3. Geberhiwot T, et al. Orphanet J of Rare Dis. 2018;13:50 4. Patterson MC, et al. Orphanet J Rare Dis. 2013;8:12; 3. NORD. NPC Signs & Symptoms. Published Dec 12, 2023. Accessed May 19, 2024. 5. AQNEURSA. Prescribing Information. IntraBio Inc 6. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab 7. Vanier MT. Orphanet J Rare Dis. 2010;5:16.
Medscape
25-07-2025
- Health
- Medscape
EMA Recommends Aqneursa for Niemann-Pick Type C Disease
At its July 2025 meeting, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended granting a marketing authorization in the European Union for Aqneursa (IntraBio Ireland Ltd) for the treatment of neurologic manifestations of Niemann-Pick type C (NPC) disease. The treatment can be used in combination with miglustat, or as a monotherapy for patients in whom miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg, the CHMP said. It also pointed out that miglustat is the only medicine authorized to treat NPC disease and has been shown to slow the general progression of neurologic symptoms in patients. NPC disease is a rare, progressive, and fatal genetic disorder caused by mutations encoding lysosomal proteins that are essential for the intracellular transport and metabolism of body fats, including cholesterol. Over time, the cells of the central nervous system and the body organs stop working. There are no curative therapies for NPC disease. The course of the disease varies highly depending on the age of onset, but most patients with NPC disease are children and die before the age of 20. Improvement in Neurologic Signs, Symptoms, and Functioning The active substance of Aqneursa is levacetylleucine, a modified form of the amino acid leucine that targets underlying processes of neurologic dysfunction. Although the mechanism of action of levacetylleucine is not yet fully understood, nonclinical studies have demonstrated that it corrects energy metabolism. This includes improved production of adenosine triphosphate, the main source of energy for cerebellar tissues and cells. A randomized, double-blind, placebo-controlled, two-period crossover phase 3 study demonstrated that levacetylleucine offered improvement in neurologic signs, symptoms, and functioning — measured using the Scale for the Assessment and Rating of Ataxia — in patients with NPC disease after 12 weeks of treatment compared with placebo. For the study, 60 patients aged 4 years or older with a confirmed diagnosis of NPC disease and at least mild disease-related neurologic symptoms were randomized in a 1:1 ratio to receive either levacetylleucine or placebo for 12 weeks. For the second 12 weeks, patients were switched to the opposite: either placebo or levacetylleucine. At the end of the first 12 weeks, patients treated with levacetylleucine demonstrated a statistically significant improvement compared with those treated with placebo. At the end of the second 12 weeks, patients who switched from levacetylleucine to placebo experienced a significant worsening of symptoms. The only adverse event causally related to treatment with levacetylleucine is flatulence. The drug will be available as 1-g granules for oral suspension. Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.
Techday NZ
12-05-2025
- Business
- Techday NZ
EY & SAP launch integrated finance service to drive growth
EY has announced the launch of a new Integrated Finance Managed Service solution in collaboration with SAP, aimed at helping organisations accelerate their enterprise transformation. The service model operates using SAP's cloud solutions and covers multiple core business functions, including human resources, payroll, finance operations, controllership, financial planning and analysis, treasury, tax, and application management. EY's offering incorporates SAP S/4HANA Cloud together with EY's domain and industry experience. According to the company, the solution is designed to help high-growth businesses create value and bolster relationships with investors, regulators, and clients. EY has become a managed services provider with a "Run" focus as part of the SAP PartnerEdge programme. Through this ongoing collaboration with SAP, EY intends to help clients more rapidly achieve both strategic and operational goals. The Integrated Finance Managed Services model is built on a robust data infrastructure and utilises an ecosystem of alliances. It is intended to provide rapid scalability and agility, as well as flexibility and resilience to support organisations as they transform and grow. The model also aims to promote improved governance and enhanced efficiency through automation, while relieving businesses of the burden of building, maintaining, and financing advanced technology, with the aim of freeing up capital for future innovation initiatives. One of the early adopters is IntraBio, which used Integrated Finance Managed Services as part of its efforts to realign financial, human resources, and accounting processes around its core goal of developing and commercialising new products. By outsourcing finance processes, IntraBio was able to maintain its focus on bringing drugs to patients in need. This approach supported IntraBio's successful launch of AQNEURSA, a treatment for Niemann-Pick Disease Type C, following its approval by the U.S. Food and Drug Administration in 2024. Marie-Laure Delarue, EY Global Vice Chair - Assurance, commented: "Businesses and their finance functions face intense challenges at the best of times, as they strive for growth in hugely competitive markets. In today's volatile economic and geopolitical climate, these challenges have only intensified." "Through Integrated Finance Managed Services, we're able to lift some of the burden that so often hinders business transformation efforts, meaning they can turn their attention to the real drivers of success. As IntraBio's story clearly shows, companies that can redirect their time and energy toward their priorities can reap incredible rewards in terms of innovation, product development, and ultimately their competitive edge." In addressing the operational considerations faced by businesses, Raj Sharma, EY Global Managing Partner - Growth and Innovation, said: "The world's most innovative companies with bold growth and transformation plans are often weighed down by non-core tasks, diverting valuable executive time and resources. Well-structured enterprise functions are critical to scaling and sustaining success, but without the right support, it can become a distraction from the strategic priorities that drive real value." "The Integrated Finance Managed Services solution can provide companies with a new model, helping businesses operate more efficiently and enabling leaders to focus on what matters most. We believe this solution can fundamentally change how clients think about their enterprise functions." Thomas Saueressig, Member of the Executive Board of SAP SE, Customer Services & Delivery, added: "Through this collaboration with EY, we are excited to combine SAP's leading cloud suite, enriched with Business AI capabilities, and EY teams' deep domain and industry experience to help deliver better insights and drive better outcomes for customers." EY's solution is positioned as an option for organisations seeking to shift focus from operational management to strategic business objectives, responding to increasing pressure in finance and other business-critical domains in the current economic and regulatory environment.
