Shares in Finnish biotech Faron Pharma are in free fall this morning after a late-stage trial of its lead drug Traumakine for acute respiratory distress syndrome (ARDS) comprehensively failed.
The result took the company and investors by surprise, coming just a few weeks after Faron had been trumpeting its preparations for regulatory filings for Traumakine in Europe and the U.S. at its R&D day in London, including a ramp-up in manufacturing capacity.
The company’s shares on the London Stock Exchange were down 83% after the announcement, reversing a protracted period of growth largely on the promise of Traumakine for ARDS, a life-threatening condition in which fluid builds up in the lungs. It usually occurs in patients ill with another disease or major trauma and currently has no approved pharmaceutical treatment. It is estimated that approximately 200,000 people develop ARDS in the U.S. each year and around 170,000 in Europe.
Faron CEO Markku Jalkanen, Ph.D., said the biotech is “incredibly disappointed and surprised by these results,” adding that it will now take some time to explore why the result differed from its earlier results in ARDS, including a very positive phase 1/2 trial published in The Lancet Respiratory Medicine in 2014 which showed an 81% reduction in mortality.
Traumakine failed on all the outcome measures in the INTEREST study, including mortality rates and the number of ventilator-free days. All-cause mortality at day 28 was 26.4% for Traumakine and 23% for placebo, and at 90 days came in at 32.6% and 31.6%, respectively. Moreover, the number of ventilator-free days in the first four weeks was 10 with Traumakine and 8.5 in the control group.
Jalkanen suggested that the 300-patient trial could have been affected by an “unusually low mortality rate observed in the placebo arm.” Typically, between 30% and 45% of patients who develop ARDS die, suggesting that the fatality rate in the study was at the low end of expectations. However, it’s worth noting that the placebo mortality rate in the Lancet study was similarly low at around 32.2%, versus 8.1% for Faron’s drug.
Traumakine (also known as FP-1201) is a recombinant human interferon beta-1a in an intravenous formulation which is thought to work by up-regulating CD73 and stimulating anti-inflammatory adenosine. That in turn prevents vascular leakage, which is the main feature of ARDS, according to the company.
All eyes will now be on a Japanese phase 3 trial of Traumakine that is due to read out in the third quarter, but prospects for the program are now looking dim. If the situation for the drug is terminal, Faron’s next most advanced pipeline project isanti-Clever-1 antibody Clevegen, in phase 1/2 testing for cancers of the ovaries, pancreas and other organs associated with high numbers of Clever-1 positive immune-suppressive macrophages.
“We will continue our plans to advance … Clevegen into clinical development in solid cancers later this year,” commented Jalkanen.