Keros Therapeutics, Inc. Announces Completion of Dosing of Planned Cohorts in KER-047 Phase 1 Trial and Provides Program Update

LEXINGTON, Mass., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Keros Therapeutics, Inc. (“Keros” or the “Company”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematological and musculoskeletal disorders with high unmet medical need, today announced the completion of the planned cohorts in a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of its lead small molecule product candidate, KER-047, in healthy volunteers.

“We are pleased to report the topline findings from the completed cohorts in our Phase 1 clinical trial demonstrating target engagement and further delineating the relationship of activin receptor-like kinase-2 (“ALK2”) inhibition and hepcidin suppression. We believe the translation of biology from rodents to humans provides a strong rationale to evaluate KER-047 in anemias due to elevated levels of hepcidin and in fibrodysplasia ossificans progressiva (“FOP”), a rare musculoskeletal disorder resulting from aberrant ALK2 signaling,” said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer of Keros.

Keros has performed a preliminary analysis of single ascending and multiple ascending dose cohorts completed to date and plans to present the results from this Phase 1 clinical trial at a major medical conference later in 2020.

• The objectives of the Phase 1 clinical trial were to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of single and multiple ascending dose levels of KER-047 in healthy volunteers. In the multiple ascending dose cohorts, KER-047 was administered as daily doses ranging from 50-350 mg for up to 7 days. The tolerability profile in healthy volunteers has been characterized in this Phase 1 clinical trial.
• There were no serious adverse events reported in either part of the trial. The most common adverse events observed in healthy volunteers in this trial were headache, nausea, vomiting, diarrhea, gastroenteritis, chills, pyrexia, myalgia, decreased appetite, lymphopenia, neutropenia, and liver enzyme increases. Dose-related decreases in lymphocytes were observed following peak increases in serum iron at the highest doses, which we believe is consistent with KER-047’s mechanism of action and suggestive of excessive mobilization and subsequent depletion of iron.
• Multiple pharmacodynamic biomarkers were included to assess KER-047's inhibition of ALK2. A reduction in hepcidin was observed following 7 days of dosing in the multiple ascending dose cohorts. Additionally, the reduction in hepcidin, increases in serum iron and increases in reticulocyte hemoglobin observed in healthy volunteers administered KER-047 is supportive of iron mobilization from tissue stores.

Based upon its preliminary analysis, Keros expects to expand its Phase 1 clinical trial to evaluate an additional two cohorts of healthy volunteers, which will help to further define dosing regimens to inform the design of upcoming Phase 2 clinical trials. Keros continues to expect commencement of a Phase 2 clinical trial in patients with iron-refractory iron deficiency anemia and other anemias with elevated hepcidin, including myelofibrosis, and a Phase 2 clinical trial in patients with FOP, both in the first half of 2021.