Merck announced the initiation of Shorespan-007, a pivotal Phase 3 clinical trial evaluating bomedemstat, an investigational orally available lysine-specific demethylase 1 (LSD1) inhibitor, for the treatment of patients with essential thrombocythemia (ET) who have previously not received cytoreductive therapy. Essential thrombocythemia is a chronic, rare blood disorder and is the most common type of myeloproliferative neoplasm. Global recruitment of the Shorespan-007 trial has begun, with patients now enrolling.
“The standard of care in essential thrombocythemia has remained unchanged for decades, and patients are in need of new options that have the potential to not only improve disease control, but also improve their quality of life,” said Dr. Gregory Lubiniecki, vice president, global clinical development, Merck Research Laboratories. “We are rapidly advancing our clinical development programs with the goal of helping to address these unmet needs and bring more options to patients living with myeloproliferative neoplasms.”
Shorespan-007 is a Phase 3, randomized, double-blind, active comparator-controlled clinical trial (NCT06456346) evaluating bomedemstat compared to the current standard of care chemotherapy, hydroxyurea, for treatment of patients with ET who have previously not received cytoreductive therapy. The trial will enroll approximately 300 patients globally. The primary endpoint of the study is durable clinicohematologic response rate (CHR). Key secondary endpoints include Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) individual fatigue symptom item score, Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a total fatigue score and MFSAF v4.0 total symptom score. Additional secondary endpoints include duration of hematologic remission, event-free survival and disease progression rate.
As previously announced, in addition to Shorespan-007, bomedemstat is also being investigated in Shorespan-006, a Phase 3, global, randomized, open-label, active comparator-controlled clinical trial (NCT06079879), evaluating bomedemstat compared to best available therapy as treatment in approximately 300 patients with ET who have an inadequate response to or are intolerant of hydroxyurea.
Bomedemstat has U.S. Food and Drug Administration Orphan Drug and Fast Track Designations for the treatment of ET and myelofibrosis (MF), Orphan Drug Designation for the treatment of acute myeloid leukemia and Priority Medicines scheme designation by the European Medicines Agency for the treatment of MF. Merck presented updated data from the Phase 2b Shorespan-003 trial, including first time genomic data, at the American Society of Hematology (ASH) Annual Meeting in December 2023. The Shorespan-003 trial is one of multiple Phase 2 clinical trials where bomedemstat is being evaluated alone and in combination for the treatment of MPNs such as ET, MF and polycythemia vera (PV).