Roche acquired a U.S. biotech that moves to the last clinical trial stages with a drug addressing idiopathic pulmonary fibrosis. The deal to acquire Promedior totals up to $1.4 billion including a down payment of $390 million plus up to $1 billion potential if several other conditions are met. The prized asset? PRM-151, which is said to be superior to other IPF therapies. Roche’s IPF therapy Esbriet brought just over $1billion in 2018. Promedior originated out of research from professors at Texas A&M University.
What is IPF?
A type of chronic scarring lung disease which is associated with a progressive and irreversible decline in lung function. The cause is unknown. Often the symptoms include a dry cough and shortness of breath. Risk factors can include smoking, select viral infections and a family history of the condition. A scarring of the lungs represents the underlying mechanism of the disease. Diagnosis involves ruling out other possible conditions. It is a type of interstitial lung disease (ILD). Average life expectancy after diagnosis is 4 years.
Frequency of Disease
Some benefit from pulmonary rehabilitation and supplemental oxygen. In some cases certain medications, such as pirfenidone (now part of Roche) or nintedanib (Boehringer Ingelheim) may slow progression of the disease. For some people, lung transplantation may be an option.
What is PRM-151?
PRM-151 is a recombinant human serum amyloid P/pentraxin 2 protein developed by Promedior, a biotech venture focusing on the development of therapies for fibrotic diseases using plasma-derived proteins and peptides. Pentraxin-2 is an endogenous human protein that plays an important role in regulating the response to fibrosis. It directs the immune system to naturally turn off and reverse the process of fibrosis, which occurs as a result of excess collagen secretion and cellular growth and differentiation. Other formulations stop a single target on the downstream side of fibrosis while PRM-151 works by reversing and possibly healing the fibrotic tissue.
The therapy is injected intravenously in patients and evidences promise as an effective anti-fibrotic agent that can be utilized to target several rare orphan diseases with unmet therapeutic needs, reports Pulmonary Fibrosis News.
The investigational therapy was granted Orphan Drug Designation by the U.S. FDA and the European Commission (EC). The investigational therapy entered Phase I clinical trials in patients with IPF to test safety, efficacy and tolerability of PRM-151 in mild to moderate cases and the results revealed the therapy significantly helped in increasing the Forced Expiratory Volume (FEV1) and lung capacity of patients at the end of eight weeks of the trial. The positive results were presented at the American Thoracic Society Annual Meeting on May 22, 2013. Thereafter, the sponsor (Promedior) gave the green light for Phase II planning.
Evidence of Superiority
PRM-151 has showed efficacy in Phase II clinical trials. A 117-patient Phase II trial for SAP/PTX2 (PRM-151) targeting pulmonary fibrosis patients evidenced efficacy better than the current standard of care and produced few side effects in patients. (see Raghu et al., Effect of Recombinant Human Pentraxin 2 vs Placebo on Change in Forced Vital Capacity in Patients with Idiopathic Pulmonary Fibrosis: A Randomized Clinical Trial. JAMA. 2018 Jun 12;319(22):2299-2307). The underlying dynamic of PRM-151—SAP—inhibits fibrocyte formation and hence injections of SAP appear to inhibit fibrosis.
Who is Promedior?
Founded back in 2006 and based in Malvern, PA, they have raised a total of $63 million in venture capital funding. They were founded by Richard Gomer and Darrell Pilling, both professors from Texas A&M University. Key investors include Morgenthaler, Health Care Ventures, Polaris, Forbion, Easton Hunt, Fibrotec, BioMed, and Shire.
According to the sponsor’s pipeline, PRM-151 is ready for a Phase III clinical trial for IPF. It is also being tested to address Myelofibrosis (Phase IIb); NASH/Liver fibrosis (ready for Phase II); fibrotic kidney disease (ready for Phase II); and fibroproliferative retinal diseases (preclinical).
How much is spent on Drugs?
Some analysts predict the IPF market to reach $5.9 billion by 2025. About $2 billion was spent in 2018. If Roche is successful in Phase III with PRM-151 they will essentially dominate the IPF drug market as its only sizeable competitor would be privately held Boehringer Ingelheim (Ofev).
For those struggling with IPF, PRM-151 has better odds to make it through Phase III as multi-national Roche has the capital, resources and infrastructure to drive a global Phase III study to determine if it can be approved by the FDA and EMA. So, we may just be a couple years away from having a more superior way to deal with IPF and ultimately other conditions triggering fibrosis.