British Regulators Pave Way for Pulmonary Fibrosis Patients to Gain Early Access to Prometic Therapy

British regulators have paved the way for people with severe cases of pulmonary fibrosis to gain access to Prometic Life Sciences’ tissue-scarring therapy PBI-4050 before it is approved.

The step the regulators took was to designate PBI-4050 a Promising Innovative Medicine for idiopathic pulmonary fibrosis. The next step would be for regulators to decide that patients with critical needs can obtain early access to the therapy.

It is the second Promising Innovative Medicine designation that the U.K. Medicines and Healthcare Products Regulatory Agency has given PBI-4050. The first was as a treatment for the genetic condition Alstrom syndrome.

The new designation is for PBI-4050 as an add-on to Boehringer Ingelheim’s Ofev (nintedanib) as an IPF treatment.

Prometic is developing the oral anti-scarring compound for several diseases with fibrosis components.

“We are proud to have received a second PIM designation in the UK for PBI-4050, following the designation previously received for Alström syndrome,” Pierre Laurin, Prometic’s president and CEO, said in a press release. “We believe PBI-4050 has the capabilities to address various unmet medical conditions such as IPF and Alström syndrome, for which there are severe limitations with the existing standards of care.

The latest designation opens the door to IPF patients obtaining PBI-4050 through Britain’s Early Access to Medicines Scheme.

Under the program, patients with life-threatening or severely debilitating diseases can gain access to therapies that are in late-stage clinical trial development but have yet to be approved. This gives patients who are not taking part in clinical trials access to a drug 12 to 18 months before it reaches the market.

“We look forward to continuing to collaborate with the MHRA [Medicines and Healthcare Products Regulatory Agency] with the goal of advancing both the PBI-4050 IPF and Alström syndrome clinical [trial] programs as quickly as possible,” Laurin said.

Prometic conducted a Phase 2 trial (NCT02538536)to evaluate PBI-4050 as an IPF treatment. Some participants received PBI-4050 by itself. Others received a combination of PBI-4050 and Ofev, and still others a combination of PBI-4050 and Genentech’s Esbriet (pirfenidone).

The study showed that PBI-4050 alone, or together with Ofev, slowed the progression of IPF. The yardstick researchers used was a measure of lung capability called forced vital capacity. FVC is the amount of air a person can forcibly exhale after a full breath.

Combining PBI-4050 with Esbriet appeared to lower both drugs’ effectiveness. The evidence was that patients who took the combo continued to experience lung function decline.

In the United States, PBI-4050 has received Fast Track Designation from the Food and Drug Administration as a combination therapy with Ofev. The designation can help speed up a therapy’s development and regulatory approval.

Prometic is now planning a Phase 2/3 trial of PBI-4050 and Ofev as an IPF treatment.