Algernon Pharmaceuticals Inc. (CSE:AGN, OTCQB:AGNPF) has shared positive topline data showing it has met the co-primary endpoint in the Phase 2 proof of concept study evaluating its repurposed drug NP-120 (Ifenprodil) for the potential treatment of idiopathic pulmonary fibrosis (IPF), a disease that causes scarring (fibrosis) of the lungs and chronic cough.
In the Vancouver-based clinical-stage pharma company’s study, 65% of the 20 patients had stable or improved forced vital capacity (FVC) over the 12-week treatment period with statistical significance when compared to an anticipated placebo effect of 40%. FVC is the amount of air that can be forcibly exhaled from one’s lungs after taking the deepest breath possible.
To evaluate the efficacy of NP-120 in IPF patients, lung function in the trial was measured by FVC, which was taken for each patient at baseline, and then again at 12 weeks. Patients whose FVC declined were classified as non-responders, while those whose FVC improved or remained stable were classified as responders. The primary endpoint of the IPF part of the study was the proportion of patients who responded.
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Opinion leaders advising Algernon, as well as historical data, indicated that 30 to 40% of IPF patients will experience no decline in FVC if they were dosed with a placebo over the 12-week period, demonstrating that Ifenprodil showed promising IPF efficacy in the trial.
In addition, Algernon reported reduction in many of the serum markers that were tested, including proC3, C3M, C6M, reC1M, proC8 and ELP-3, although the data did not reach statistical significance. Elevation of these markers has been associated with increased mortality and risk of disease progression in previous research studies.
“The IPF data looks quite good,” Dr Martin Kolb, Professor of Respirology at McMaster University said in a statement. “I was very surprised to see the data achieve statistical significance with such a small study size when you consider the original goal of the study was to try to identify a signal. As a result, I am confident that the company should begin planning a sufficiently powered Phase 2b study to investigate Ifenprodil as a possible new treatment for IPF patients, including those who have associated cough.”
Chronic cough data set
For the chronic cough part of the study’s primary endpoint, 30% of subjects achieved the endpoint of a 50% reduction in the average number of coughs per hour over 24 hours from baseline to week 12. While the primary cough study endpoint did not achieve statistical significance when compared to an anticipated placebo effect of 25%, the secondary endpoint of actual changes (reduction) in cough counts did.
“These data are quite compelling,” said Dr Jacky Smith, Professor of Respiratory Medicine at the University of Manchester, and an honorary consultant at Manchester University NHS Foundation Trust. “Although the primary cough endpoint does not reach statistical significance, the reductions in cough counts, particularly the median cough counts, are suggestive of a beneficial effect.”
Although Ifenprodil has been used in Japan for decades to treat vertigo, this is the first study in an IPF population. Algernon said "no new safety concerns were identified."
Full data set
Algernon expects to receive the full data set in August 2022 and will present the results of the study at the 21st International Colloquium on Lung and Airway Fibrosis in Reykjavik, Iceland in October this year.
“Simply put, the IPF data is better than we could have imagined,” said Algernon CEO Christopher J. Moreau. “The outlook for patients with IPF remains dismal, with 50% mortality expected within three to four years ... new drugs are desperately needed.”
The Algernon CEO noted that the IPF market is expected to hit $4.2 billion by 2030.
“While the cough data is also promising, we will wait until we have the full data set before making any final decisions on pursuing both IPF and chronic cough in separate Phase 2b clinical studies or focussing on just IPF patients with associated cough,” he added.
In the meantime, based on the positive data, the company plans to file a pre-IND application with the US Food and Drug Administration (FDA) for a Phase 2b IPF study. Algernon plan to switch to a new once-a-day formulation of Ifenprodil from the three-times daily dosing currently being used.
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
Follow her on Twitter: @UttaraProactive