A new drug for pulmonary fibrosis has reduced disease progression by more than 50% over a 52-week treatment in two clinical trials.

Pulmonary fibrosis is a very serious disease, with a life expectancy of 3 to 5 years after diagnosis. The new drug, nerandomilast (Boehringer Ingelheim), could revolutionise the treatment of the disease after several failures of all potential new drugs tested for this condition in the last decade that have failed in phase 2 or phase 3 studies.

Positive results of two phase 3 studies of nerandomilast were presented at the American Thoracic Society's annual congress (ATS, San Francisco, 16–21 May). The trials were conducted with idiopathic pulmonary fibrosis patients (FIBRONEER-IPF study) and in secondary progressive pulmonary fibrosis (FIBRONEER-ILD study).

The new drug is an orally administered selective inhibitor of phosphodiesterase 4B, which can also be combined with existing drugs (nintedanib or pirfenidone). The trials’ results will be published in the NEJM on Sunday, 18 May. Luca Richeldi, full professor of diseases of the respiratory system at the Catholic University of the Sacred Heart and director of the UOC of Pneumology at Fondazione Policlinico Gemelli IRCCS, is the global principal investigator (PI) and first author of the study on idiopathic pulmonary fibrosis, confirming his position as one of the world's leading experts on the subject.

He is also the author of the study on secondary fibrosis as a member of the steering committee. Professor Richeldi said: “The publication of these two studies represents a breakthrough for the disease because it opens up a new generation of drugs for these patients, WHO have so far had very limited therapeutic options available to them.

From now on, they will also be able to benefit from combined treatments with several drugs. Nerandomilast has shown clear efficacy in slowing the progression of IPF when used alone and has fewer side effects than previous therapies (the most common side effect was diarrhoea). In IPF, the drug reduced disease progression by more than 50% over the 52-week trial duration (the trial randomised 1,177 patients treated with neramidilast 9 or 18 mg daily or placebo).

In non-idiopathic forms of fibrosis, it was also shown to produce a reduction in mortality. This dual indication (for both idiopathic and secondary forms of fibrosis) is a novelty because it will allow the treatment of a broader spectrum of pathologies compared to the narrow spectrum of IPF. Non-idiopathic forms include, for example, pulmonary diseases secondary to autoimmune diseases, exposure, or drug-related diseases.”

Professor Richeldi added: “A single drug, nerandomilast, is effective in treating pathologies previously considered very different. While nerandomilast does not cure pulmonary fibrosis (once established, lung damage does not regress), it represents a major step forward for the treatment of this condition. It is also important to note that the new drug delays the start of oxygen therapy, which, in my experience, is extremely disabling for these patients, limiting (and sometimes wiping out) their social life and leading to significant negative effects on their quality of life.”

Professor Richeldi concluded: “The next step we are working on is a two-year study in patients with the earliest form of the disease, known as ILA (interstitial lung abnormalities), treated with nerandomilast to understand whether treating preclinical or subclinical phases of the disease can further slow its progression and even prevent the emergence of pulmonary fibrosis symptoms.”