Hemab Therapeutics (NASDAQ:COAG) has launched a pivotal Phase 3 trial of its experimental bleeding-disorder treatment sutacimig, moving its lead program into late-stage development as it prepares additional data presentations in December and February.
On the company’s third-quarter earnings call, Chief Executive Officer Benny Sørensen said the SOLARA study will test weekly sutacimig injections in people with Glanzmann thrombasthenia, a rare bleeding disorder with no approved preventive treatment. Hemab is proceeding with a single-arm trial after the U.S. Food and Drug Administration recommended that it consider a randomized controlled study.
The company ended September with $430.2 million in cash, cash equivalents and marketable securities, down from $457.5 million at the end of June. Chief Financial Officer Mads Behrndt said Hemab continues to expect its cash to fund operations into 2029.
Phase 3 Trial Design Draws FDA Questions
SOLARA is expected to enroll approximately 75 people ages 12 to 70 who have had at least four treated bleeds in the prior 12 months. After a 24-week observation period on their current treatment, participants will receive sutacimig for 48 weeks. The main measure will compare each patient’s annualized treated bleed rate during treatment with the rate during the run-in period.
Sørensen said the FDA acknowledged Hemab’s concerns about the feasibility of randomization. The company chose the single-arm design because of variability in bleeding rates and treatment practices, as well as differences among patients, he said.
Asked whether Hemab had full alignment with the agency or was taking a regulatory risk, Sørensen said he believed the risk was “very low” and that the company would continue discussions with regulators. He cited other bleeding-disorder treatments that used single-arm studies in registration trials.
The study follows Phase 2 extension data from 34 participants. Sørensen said 92% of participants who had bled during the run-in period experienced fewer treated bleeds on sutacimig. The mean annualized rate of high-intensity treated bleeds fell 62%.
There were no treatment-related adverse events of grade three or higher, he said. Three participants had grade two thromboembolic events in higher-exposure cohorts and/or with multiple risk factors; all were managed with routine anticoagulation therapy. Hemab selected SOLARA’s dosing regimen with the goal of reducing bleeds while avoiding the high peak exposures associated with those events.
The first participants have consented and are scheduled to enter the run-in period before year-end, Sørensen said.
Further Data Expected Across Programs
Hemab plans to present updated Phase 2b sutacimig data in Factor VII deficiency at the American Society of Hematology annual meeting on December 14. As of July 24, two participants with severe congenital Factor VII deficiency had received a single dose. Both had a greater than 50% shortening of baseline prothrombin time, a measure of clotting, and adverse events were low grade and assessed as unrelated to treatment, Sørensen said.
He declined to specify how many patients would be included in the updated presentation, describing the study as a small trial conducted at one U.K. site.
For HMB-002, an experimental treatment for von Willebrand disease, Hemab expects to present new data at the EAHAD Congress in February 2027. The update will include monthly dosing data at 150 milligrams and additional single-dose data at 300 milligrams, covering patients with type 1 and type 2A disease.
Earlier Phase 1/2 results showed peak increases of approximately 2.5-fold in von Willebrand factor and factor VIII, proteins involved in clotting, Sørensen said. The single-dose portion was not designed to measure efficacy, but eight of nine evaluable patients had no treated bleeds in the 28 days after dosing. Most adverse events were mild to moderate, with no serious or thrombotic events reported.
Hemab also expects to present preclinical data on HMB-003, its candidate for heavy menstrual bleeding, on December 13. In a monkey model, the treatment reduced the proportion of severe bleeding days compared with baseline, Sørensen said. The company expects initial data from a Phase 1 trial in healthy volunteers in mid-2027.
Quarterly Loss Widens
Hemab reported a third-quarter net loss of $27.3 million, or $0.58 per share, compared with $17.1 million, or $18.04 per share, a year earlier.
Research and development expenses rose to $24.6 million from $14.6 million, primarily because of clinical program advances and higher personnel costs. General and administrative expenses increased to $5.1 million from $2.5 million.
About Hemab Therapeutics (NASDAQ:COAG)
Hemab Therapeutics is a clinical-stage biotechnology company developing treatments for serious bleeding and thrombotic disorders, with a focus on conditions that have limited treatment options. Its research centers on therapies intended to help restore or regulate blood clotting.
The company’s lead candidate, HMB-001, is an investigational bispecific antibody being developed for Glanzmann thrombasthenia, a rare inherited bleeding disorder. Hemab is also advancing research into additional disorders affecting hemostasis, the process that helps the body control bleeding.
Hemab was founded in 2020 and operates in the United States and Denmark.
