New hope for patients with lung scarring disease

Research has shown a new medicine could help patients with a fatal lung condition.

Dr Sophie Fletcher led Southampton’s involvement in the global trial. She recruited patients with a rare lung disease called pulmonary fibrosis, which causes scarring in the lungs.

It is the second of two groundbreaking trials. The first was published last year. It found the medicine nerandomilast slows the progression of idiopathic pulmonary fibrosis (IPF).

Now, this trial shows nerandomilast is also effective at slowing progressive pulmonary fibrosis (PPF). If approved, the drug could be used to treat patients with both conditions.

The results have been published in the New England Journal of Medicine.

Providing more options

IPF and PPF are forms of pulmonary fibrosis, also known as interstitial lung disease (ILD).

They both cause inflammation and scarring in the lungs. In IPF the cause is unknown, whereas for PPF there is usually a known disease causing the condition.

Although considered a rare disease, PPF is estimated to affect up to 5.6 million people worldwide. It gets worse over time until the lungs stop working. Symptoms include shortness of breath, fatigue and a persistent cough.

There is currently only one approved treatment, nintedanib, that can slow this progression. However, this can have severe side effects. These include diarrhoea, feeling and being sick, abdominal pain, loss of appetite and weight loss. This can prevent patients from taking it.

Nerandomilast is being tested in this trial by Boehringer Ingelheim. It could provide an alternative treatment with fewer side effects.

Dr Fletcher is a Consultant Respiratory Physician and part of the Research Leaders Programme at University Hospital Southampton. She is also Honorary Associate Professor of Respiratory Medicine at the University of Southampton.

“We are delighted to be part of a global study for this rare group of devasting progressive lung disease,” she said. “The results from this work give hope for more therapeutic options for our patients.”

Slowing down the disease

The FIBRONEER-ILD trial involved patients with PPF from 403 sites, including University Hospital Southampton. It was a global trial, involving 1,176 patients from 44 countries.

Patients in the trial were randomly assigned to take 18 mg nerandomilast twice a day, 9 mg nerandomilast twice a day, or a placebo. They did not know which group they were in.

Their lung function was assessed after a year (52 weeks), by measuring their forced vital capacity (FVC). This is the amount of air they can forcefully exhale after taking a deep breath.

All patients had worse lung function at 52 weeks. However, this was much greater for those taking the placebo (−165.8 ml) than those taking either 18 mg (-98.6 ml) or 9 mg (−84.6 ml) of nerandomilast. The treatment, at both doses, slowed the progression of the disease.

Importantly, the numbers of patients who stopped taking the treatment due to side effects was similar in the treatment and placebo groups. This suggests that nerandomilast has less severe side effects than nintedanib, meaning it may provide a good alternative option for patients.

Boehringer Ingelheim are now seeking approval for nerandomilast to become a new treatment for IPF and PPF in the US, China and the EU, with plans for other countries to follow.

Nerandomilast must be approved by the National Institute for Health and Care Excellence (NICE) before it can be made available on the NHS. However, patients at UHS who participated in the trial can access it now as part of the FIBRONEER-ON study.

“Idiopathic pulmonary fibrosis and progressive pulmonary fibrosis are devastating conditions, with one in two people dying within five years of IPF diagnosis,” said Shashank Deshpande, Head of Human Pharma and Member of the Board of Managing Directors at Boehringer Ingelheim.

“Despite this stark reality, ongoing research may provide new possibilities for patients, as there remains a need for additional therapies. The latest efficacy, safety and tolerability data on nerandomilast points to its potential in addressing the needs of those impacted by IPF and PPF.”