Through the activities of the SMEI funded program, ATXA have successfully secured Clinical Trial Authorisation (CTA) approval, the ultimate objective of PAH-HOPE and a major milestone for the company. Granting of CTA, by the UK’s Medicines and Health Regulatory Authority (MHRA), marks NTP42’s entry into clinical development, putting ATXA on an exciting journey to realise its mission of bringing new hope to the PAH patient.
The achievement of the CTA is a direct result of the successful completion of a comprehensive program of technical, regulatory, commercialization and KOL engagement work packages, overseen by effective program management and governance. Through PAH-HOPE, the team have successfully completed the scaled-up manufacture generating high quality pharmaceutical grade NTP42 that exceeds pre-approved specifications while also developing a unique and highly efficient, robust manufacturing process (Drug Substance Manufacture). Following on from scale-up manufacture, the PAH-HOPE team have developed and manufactured a formulated drug product that is suitable for use an oral medicine (Formulation and Packaging). Moreover, ATXA’s drug has been evaluated in a formal Drug Safety and Toxicology program with no adverse events identified that would prevent its use in humans. With the successful completion of these technical activities, the PAH-HOPE team completed the dossier of regulatory documents required for the CTA filing, including preparing and finalizing the first formal drafts of the Investigational Medicinal Product Dossier (IMPD) and Investigator’s Brochure (IB). These documents were included in the CTA submission to the MHRA, which following their review was approved, paving the way for ATXA’s First-in-Human (FIH) Phase I clinical trial in healthy volunteers.
In parallel to the technical and regulatory work packages, the PAH-HOPE management team advanced the business and commercial objectives of the program and company, where a robust, yet adaptable business plan has been generated along with other supporting business and/or promotional material to meet the demands of current and future investors. Furthermore, the scientific and management team have made substantial progress in activities generally associated with Engagement & Communication, disseminating the mission, objectives, results and achievements of PAH-HOPE to all stakeholders, be they the PAH patient, our ultimate customer, key opinion leaders (KOLs) expert in pharmaceutical development, business strategy, fundraising or patient treatment. This has involved a variety of platforms, be it through scheduled face-to-face or virtual meetings, attendance or podium presentations at clinical or investor conferences, press releases, media coverage, formal peer-review manuscript publications or through on-line portals such as the dedicated PAH-HOPE website (
https://www.atxatherapeutics.com/PAH-HOPE(se abrirá en una nueva ventana)) social media or video (
https://youtu.be/Um0QEQwiY4A(se abrirá en una nueva ventana)) and has resulted in significant outreach and positive feedback while socializing the benefits of NTP42 for the PAH patient and the overarching ambition of ATXA and PAH-HOPE. Each of these activities and achievements have been made possible by ensuring excellence in Governance and Management of the PAH-HOPE program at ATXA, instigated through effective internal and external communication and reporting structures between all parties be they dedicated staff, scientific or clinical advisors or specialist sub-contractors, as well as putting in place a secure and efficient IT infrastructure.
