Novartis halts mid-stage IPF trial, continues PAH drug testing

Novartis has decided to terminate its mid-stage trial of an oral treatment for idiopathic pulmonary fibrosis (IPF), as confirmed by the company. The termination, described as a “sponsor decision,” was officially recorded last Thursday in the federal trials registry. A spokesperson stated that Novartis will continue to evaluate the future potential of treatments for IPF.

The study was focused on testing LTP001, an experimental medicine, against a placebo. This once-daily medication was being evaluated for its efficacy in treating IPF, a condition characterized by lung scarring that impairs breathing and can be fatal within a few years of diagnosis. In some cases, participants in the trial were also administered one of two FDA-approved standard treatments for IPF: Roche’s Esbriet and Boehringer Ingelheim’s Ofev, both approved in 2014. Generic versions of Esbriet are available, and Roche announced in February that it is exploring “potential options for Esbriet,” including a possible sale.

Several other biopharmaceutical companies are actively working on potential IPF treatments. Noteworthy among these are Pliant, PureTech, and Avalyn Pharma. Boehringer Ingelheim plans to submit another IPF treatment, nerandomilast, for FDA approval, following positive Phase 3 trial results.

Additionally, LTP001 is being investigated for its potential in treating pulmonary arterial hypertension (PAH), a rare lung condition that causes high blood pressure in the lungs and heart, affecting around 40,000 people in the United States. Merck’s Winrevair recently received approval for treating PAH. In Novartis' July earnings presentation, PAH was highlighted as the primary indication for LTP001. Subsequently, the company concluded a placebo-controlled Phase 2 and an extension study of LTP001 in PAH.

A Novartis spokesperson reiterated on Monday that the company is committed to advancing LTP001 for PAH treatment. The spokesperson also noted that there have been no new or concerning safety signals observed in the trials to date. LTP001 functions as an inhibitor of SMURF1, a protein that has been found to be elevated in pulmonary arteries in animal studies.

In the broader PAH treatment field, Johnson & Johnson received FDA approval earlier this year for a single-tablet combination therapy for PAH. Meanwhile, Aerovate’s treatment candidate failed a mid-stage trial, resulting in layoffs. Other companies, including Gossamer Bio and Diagonal Therapeutics, are also exploring treatments for PAH.