IntraBio encroaches on Zevra's domain after lysosomal storage disorder approvals

IntraBio’s recent FDA approval for its drug Aqneursa represents a significant advancement in the treatment of Niemann-Pick disease type C (NPC), just days after Zevra Therapeutics achieved a historic milestone with the approval of Miplyffa. NPC is a rare lysosomal storage disorder that disrupts the body's ability to transport cholesterol and other lipids within cells, leading to harmful accumulations in organs such as the liver, spleen, and lungs. This accumulation results in severe organ damage, and the average lifespan for individuals with NPC is around 13 years. Furthermore, the disease adversely affects speech, cognition, swallowing, movement, and fine motor skills.

Last week, Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for NPC, requiring co-administration with the enzyme inhibitor miglustat. However, Zevra projected a market availability timeline of eight to twelve weeks for Miplyffa. In contrast, IntraBio's Aqneursa, approved on Tuesday, is already available as a standalone therapy for both adults and children weighing at least 15 kg (approximately 33 pounds).

Aqneursa’s approval followed IntraBio’s pivotal trial IB1001-301, which examined its efficacy in treating neurological symptoms and functioning in patients over the age of four with a confirmed NPC diagnosis. The trial achieved its primary efficacy endpoint and all secondary endpoints across the different patient groups receiving Aqneursa. Within 12 weeks, Aqneursa significantly improved neurological signs and symptoms of NPC, contributing to better functional outcomes. The FDA's evaluation focused on a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), known as the functional SARA (fSARA), which measures gait, stability, speech, and limb coordination. Patients treated with Aqneursa showed notable improvements in fSARA scores compared to those given a placebo.

Common side effects reported for Aqneursa include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting. The drug’s prescribing information includes a warning about potential embryo-fetal harm if administered during pregnancy, advising patients to inform their healthcare providers of any known or suspected pregnancies. Aqneursa is administered orally up to three times daily, with or without food, and the dosing depends on the patient's body weight.

Originating from the U.K., IntraBio has recently moved its headquarters to Austin, Texas, following a $40 million funding round. Aqneursa is the company's first commercial product, and IntraBio is ambitious about its future potential. CEO Mallory Factor indicated that the company plans to explore Aqneursa’s application for other rare and common neurodegenerative and neurodevelopmental disorders based on their clinical research.

To ensure patients have access to Aqneursa, IntraBio has introduced a support program called Aqneursa Cares. This initiative is designed to provide financial assistance to minimize or eliminate out-of-pocket costs for eligible patients and connect them with third-party resources. Additionally, Aqneursa Cares offers educational resources and a dedicated team of specialists to support patients.

In summary, IntraBio's rapid approval and launch of Aqneursa provide a new, readily available treatment option for NPC, offering a competitive edge over Zevra Therapeutics' Miplyffa. This development not only disrupts the NPC treatment landscape but also holds promise for broader applications in neurodegenerative and neurodevelopmental disorders.