Pulmonary arterial hypertension (PAH) is characterized by high blood pressure in the lungs due to obstruction of small lung arteries. The reduced blood flow makes it harder for the right side of the heart to pump blood to the lungs, leading to heart failure. While the current standard-of-care (SoC) drugs treat some of features of PAH, they do not treat the underlying cause and do not offer a cure. These SoC drugs carry serious side-effects, further reducing patient quality-of-life. The clinical features of PAH also occur in other related heart and lung (cardiopulmonary) diseases, including more common conditions, such as heart failure and lung fibrosis. Like PAH, these diseases are poorly served by the SoC drugs used to treat them.
ATXA Therapeutics has developed NTP42, a disruptive new drug that directly treats all clinical hallmarks of PAH in both the lungs and heart. NTP42 blocks the thromboxane receptor, a target at the root cause of PAH. By treating all clinical hallmarks in the lungs and heart, NTP42 offers a game-changing solution for PAH, leading to its rapid adoption once marketed. It may also potentially treat other cardiopulmonary diseases with similar clinical features. Previously, ATXA’s Phase I clinical trial in healthy men showed that NTP42, when given as an oral suspension of the formulated drug (NTP42:KVA4), is safe & highly efficacious at low doses. The next step is to test NTP42 in PAH patients.
Thus, with the support of EIC Blended Finance and VC equity co-investment, the objectives of PAH-Advance are to complete the necessary pre-trial work needed for regulatory approval and to undertake the Phase II clinical trial itself. On Trial completion, ATXA will be well positioned to secure a licensing deal with a pharma company who will then bring NTP42 to the market, making it accessible to PAH patients and offering their healthcare providers new options for this devasting disease.
In this grant-funded part of PAH-Advance, the aim was to develop a capsule form of NTP42 to make it easy for patients to take the drug at home in a non-clinical setting. Before we test NTP42 capsules in the Phase II trial in PAH patients, it must be tested in a Bridging clinical trial in healthy volunteers, to ensure that it is safe and effective. We also aimed to complete key activities, such as drug manufacture and drug safety studies, that will help us secure approval for our Phase II trial.
In addition, a key aim of the PAH-Advance program was to demonstrate that NTP42 is effective in other cardiopulmonary diseases. This is important for the patients affected by this devastating disease and their doctors. It will also demonstrate to potential investors and licensees that NTP42 has potential benefits beyond PAH, making ATXA a more attractive option for pharma companies in a licensing deal.
In the second part of PAH-Advance, to be funded by EIC and VC equity co-investment, ATXA will complete all trial-enabling activities, including capsule manufacture. We will also seek regulatory approval to carry out the Phase II clinical trial in PAH patients in Europe and US. On approval, we will undertake the Trial with >100 PAH patients who will receive either a placebo or NTP42 capsules for 24-weeks, with patients offered the opportunity to take the drug longer. Patients will be monitored for safety of the drug as well as its ability to treat PAH. In parallel, ATXA will engage with pharma companies to secure a licensing deal on completion of the Phase II trial. Successful completion of the Trial and securement of a licensing deal or other business alignment will greatly advance NTP42’s route to the market. With the proceeds of the licensing deal, we will continue to develop NTP42 and related pipeline drugs to treat other priority target diseases, further diversifying the uses of our drugs. This will also enable company growth, transforming ATXA into a major European pharma with a diversified diseases and drugs portfolio.
