European Union Horizon 2020 research & innovation ProgrammE
SME Instruments Phase 2 Development Programme
ATXA Therapeutics Limited secured €2.5 million funding under EU Horizon 2020 SME Instrument Phase 2 Development Programme to complete the pre-clinical development of its drug, NTP42 and to secure Clinical Trial Authorisation (CTA) approval for ATXA’s first-in-human (FIH) Phase I clinical trial. The program, referred to as PAH-HOPE, commenced in October 2018 and was completed in March 2021. Through this, the PAH-HOPE team at ATXA successfully completed all activities and achieved the ultimate milestone, securement of CTA approval from the Medicines & Health Regulatory Authority (MHRA; 8th March 2021). By paving the way for ATXA’s Phase I clinical trial, the CTA approval marks the company’s entry into clinical development with NTP42.
This project received funding from the European Union Horizon 2020 research and innovation programme under Grant Agreement No 822258.
New Hope for the PAH Patient: A Novel Therapeutic for Pulmonary Arterial Hypertension (PAH) - PAH-HOPE
PAH is a debilitating lung & heart disease with an urgent need for new medicines targeting different pathways than current drugs. These drugs do not effectively treat the disease, they carry serious side-effects, are poorly tolerated and only extend life-expectancy from 2.8 years (untreated) to 3.6 years (treated).
ATXA has developed novel therapeutic drugs to treat PAH. ATXA’s drug target is the human Thromboxane Receptor, a pathway involved in all the clinical features of PAH. Based on evaluations in 2 independent animal models of PAH, ATXA’s lead NTP42 is predicted to be a truly disruptive disease-modifying drug, treating all aspects of PAH and greatly extending life expectancy. ATXA’s NTP42 fits with the current focus on value-based healthcare delivery, resulting in improved healthcare outcomes that matter to patients, physicians, payors but at lower cost of delivery.
The objective of PAH-HOPE was to advance ATXA’s drug NTP42 into clinical development, starting from scaled up drug manufacture, oral drug formulation through to securing regulatory CTA. Through the four major activities or Work Packages (WPs), along with associated management, governance and communication programmes, ATXA has successfully delivered on and achieved all objectives of PAH-HOPE. The company have completed the scaled-up manufacture of its drug, producing a high-quality pharmaceutical grade NTP42. Following on from this, ATXA have developed and manufactured a formulated drug product that is suitable for use an oral medicine and has been shown to be efficacious in animal models of PAH. 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 these technical activities completed, the dossier of regulatory documents required for the CTA filing were finalised, including the Investigational Medicinal Product Dossier (IMPD) and Investigator’s Brochure (IB). Following review of the CTA submission, the MHRA granted approval, paving the way for ATXA’s First-in-Human (FIH) Phase I clinical trial in healthy volunteers.
Securement of the CTA has positioned ATXA to advance NTP42 into its Phase I clinical trial and into clinical development, leading the way for Phase II/III trials through to marketing authorization and product launch in 2027.
About SME Instrument
The EIC Accelerator (previously know as SME Instrument) is part of the European Innovation Council (EIC) pilot that supports top-class innovators, entrepreneurs, small companies and scientists with funding opportunities and acceleration services.
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