Nuformix’s NXP002 Candidates Show Potential Against IPF in ‘In-Patient’ Preclinical Model

Preclinical data show that treatment with Nuformix’s investigational candidates within the NXP002 program can prevent fibrosis progression with enhanced efficiency compared to the current standard of care, the company announced.

The study, conducted in collaboration with researchers at Newcastle University, U.K., revealed that the new therapeutic agents could act on key inflammatory targets involved in idiopathic pulmonary fibrosis (IPF), and inhibit fibrosis’ underlying mechanisms.

“Few patients respond to current treatments and have to tolerate severe side effects that dramatically impact quality of life — severe vomiting on one therapy or severe diarrhea on the other,” Dan Gooding, CEO of Nuformix, said in a press release. “This promising data gives us confidence in our ability to both inhibit fibrosis and attenuate inflammation in patients without these side effects.”

Taking advantage of cocrystal technology, Nuformix reshapes the chemical structure of known compounds while retaining their therapeutic activity. With this approach, the company can reduce the time and cost of achieving better clinical success compared to traditional biotech development strategies.

NXP002 candidates are re-engineered crystalline forms of known therapeutic agents that are expected to hold therapeutic activity for a broad range of fibrotic diseases.

To explore the potential of NXP002 program candidates, researchers used a new preclinical model developed at Newcastle University. This validated experimental model uses fibrotic lung tissue collected from patients who have had lung transplants, making it a unique ‘in-patient’ model that cannot be re-created in current preclinical models.

The fibrotic tissue is kept alive and functional in the lab in specific conditions that preserve the disease mechanisms, creating a useful model to accurately study disease progression. Because only patients with severe and late-stage disease are proposed for transplant, this new strategy allows detailed study of the impact of therapies on this population.

Using this specific preclinical model, researchers could demonstrate that NXP002 candidates had  strong inhibitory activity against fibrosis, even in severely fibrotic tissue, and acted on key inflammatory targets.

The team also showed NPX002’s enhanced efficacy in treating IPF compared to Genentech’s approved therapy Esbriet (pirfenidone).

“Newcastle University use a highly innovative new human tissue model, which has the potential to become the new gold standard for pre-clinical studies,” Gooding said. “These findings are important for IPF patients and show our NXP002 program can play an important role in improving on current treatment options and extending both life and its quality.”

Nuformix plans to further develop one candidate of the NXP002 program, and optimize its delivery to maximize efficacy and tolerability before starting clinical studies in patients.