Hemab Therapeutics Details Post-IPO Pipeline Push, Sutacimig Phase III Plans

Key Points

• Hemab Therapeutics said its lead drug sutacimig is on track to enter a pivotal Phase III trial in the second half of this year after Phase I/II results in Glanzmann’s thrombasthenia showed up to an 87% reduction in annualized treated bleed rate.
• The company is also advancing HMB-002 in von Willebrand disease, with additional dose data due at ISTH and early multiple-ascending-dose efficacy results expected in late 2026 or early 2027.
• Hemab said it raised $347 million in its IPO and, together with existing cash, now has a runway into 2029 under current plans.

Hemab Therapeutics NASDAQ: COAG Chief Executive Officer Benny Sorensen outlined the company’s development plans following its recent initial public offering during a fireside chat with Jefferies biotech analyst Maury Raycroft.

Sorensen said Hemab is pursuing a strategy to build “a multiple product, exclusive coagulation franchise company” focused on blood coagulation disorders. The company’s lead program, sutacimig, has completed a Phase I/II study in Glanzmann’s thrombasthenia and is expected to enter a pivotal Phase III trial in the second half of this year, he said.

Sutacimig is also being studied in Factor VII deficiency, with initial Phase II data expected in 2026 or early 2027. Hemab’s second clinical-stage asset, HMB-002, is in a Phase I/II first-in-human trial in von Willebrand disease, with initial multiple-ascending-dose efficacy data also projected for 2026 or early 2027. Sorensen added that the company plans to present data on HMB-003 at the International Society on Thrombosis and Haemostasis meeting in July in Paris.

Sutacimig Phase III Planning

Sorensen said all sutacimig data to be presented at ISTH are already included in Hemab’s S-1 filing and that no new data will be disclosed at the meeting. The presentation will include complete Part B multiple-ascending-dose data and data from Part C, the open-label extension.

In Glanzmann’s thrombasthenia, Sorensen said Hemab observed up to an 87% reduction in annualized treated bleed rate in the low-dose weekly dosing regimen. He described annualized treated bleed rate as a regulatory-endorsed primary endpoint. Among patients who had experienced life-threatening bleed events in the prior 12 months, he said the company saw a 100% reduction in severe bleed events in Part B, with the reduction remaining “way over 60%” in the open-label extension.

Sorensen said the safety data support moving into a pivotal study, while noting that Hemab identified a risk of “exaggerated pharmacology” at high exposure, including Grade 2 venous thrombotic events. He said the company believes it can mitigate that risk in Phase III through dose selection and investigator education.

“The data are sufficient to transition to pivotal,” Sorensen said, citing feedback from regulatory authorities. He said Hemab has Breakthrough Therapy designation, allowing for ongoing dialogue with the U.S. Food and Drug Administration.

According to Sorensen, Hemab has aligned with regulators on primary and secondary endpoints, an open-label study design, the primary statistical analysis and safety mitigation strategy. Remaining discussions include whether the pivotal trial should be single-arm or an open-label randomized controlled trial, as well as final dose-regimen alignment.

Sorensen said the company’s base case is a single-arm trial, similar to the Phase I/II study, but Hemab is exploring whether a randomized controlled design could strengthen the study without materially changing its execution. He said the study size, statistical analysis plan and powering would not change under either design. The Phase III trial is expected to include a run-in period of up to six months to prospectively collect annualized treated bleed rate data.

Hemab expects the pivotal study to require 75 to 100 patients to build a sufficient safety database, Sorensen said. The company has not yet provided guidance on geographic enrollment proportions, though Sorensen said patients are expected to be enrolled across the United States, Europe and likely the Gulf States and Japan.

Factor VII Deficiency and Market Opportunity

For Factor VII deficiency, Sorensen said Hemab will provide more detail after the summer on the number of patients in its ongoing proof-of-concept work. He said the indication has clear biomarkers, including prothrombin time and Factor VII levels.

“If we reduce the prothrombin time and see increases in VII, we have a proof of concept,” Sorensen said, adding that Hemab would not need “a hell of a lot of patients” to reach that conclusion.

Sorensen said Hemab has conducted a Factor VII Deficiency 360 natural history study with about 100 patients, giving the company insight into bleed frequency. He said the natural history study is not likely to be sufficient as a control and would be supplemented with prospectively collected data, similar to the Glanzmann program.

Discussing market size, Sorensen said Hemab knows of at least 10,000 diagnosed patients with Glanzmann’s thrombasthenia and Factor VII deficiency across the U.S., Europe, Gulf States, Japan and a small set of other markets that use specialty drugs. He said there may be additional undiagnosed or miscategorized patients, but he declined to estimate the number.

HMB-002 in Von Willebrand Disease

Sorensen also discussed HMB-002, Hemab’s program for von Willebrand disease. He said the company has presented data from 20-milligram and 50-milligram fixed doses, showing favorable safety and tolerability as well as a 1.5-fold increase in von Willebrand factor and Factor VIII, which he described as Hemab’s predefined success criteria.

At ISTH, Hemab plans to present data for 20-milligram, 50-milligram and 150-milligram doses. Sorensen characterized the update as incremental because the company has already reached its 1.5-fold increase target. Hemab expects to transition to multiple ascending dosing, with early efficacy data expected at either ASH in December or EAHAD in February.

Sorensen said HMB-002 is designed to work in Type 1 and Type 2A, 2M and 2N von Willebrand disease, representing about 98% to 99% of patients. He said the drug is unlikely to have a significant monotherapy effect in Type 3 disease, though physicians have suggested it could potentially be used with concentrates to reduce dosing frequency.

Hemab is collecting prospective run-in data in von Willebrand disease using an electronic bleed diary. Sorensen said more than 80 patients are currently in at least a four-month prospective run-in period, which Hemab intends to use to generate baseline annualized treated bleed rates before chronic treatment with HMB-002.

HMB-003 and Cash Runway

On HMB-003, Sorensen said abstracts will be released at the end of June, with full data to follow at ISTH. He described the program as targeting “another extremely high unmet need subset” and said it uses a validated technology. Hemab has previously disclosed that HMB-003 is a fatty acid-conjugated peptide, which Sorensen likened to a “GLP-1-like molecule applied in hemostasis.”

Sorensen closed by discussing Hemab’s financial position. He said the company raised $347 million in its IPO and, combined with existing cash, has runway into 2029 under current plans.

About Hemab Therapeutics NASDAQ: COAG

Hemab Therapeutics is a clinical-stage biotechnology company focused on developing novel therapies for people with serious bleeding and thrombotic disorders. The company's research is centered on treatments designed to address diseases of the blood and coagulation system, with the goal of improving outcomes for patients who may not be adequately served by existing options.

Hemab's pipeline includes drug candidates aimed at rare hematologic conditions, including factor XI-related approaches and other programs in inherited bleeding disorders.

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