Hemab Secures $135M for Neglected Bleeding Disorders

2023-02-21

基因疗法上市批准

Courtesy of Getty Images Hemab Therapeutics aims to bring new treatment options for lesser-known bleeding disorders. On Tuesday, the Boston- and Copenhagen-based biotech announced its oversubscribed Series B round closed with $135 million. Hemophilia A and B have historically soaked up the lion's share of R&D focus. CSL Behring and partner uniQure set records last fall with the first gene therapy approved for hemophilia B. With a $3.5 million price tag, Hemgenix is the most expensive medicine in the world. Meanwhile, BioMarin is awaiting FDA approval of its gene therapy, already available in the EU, for severe hemophilia A. Lesser-known severe bleeding and thrombotic disorders have “been neglected,” Hemab CEO Benny Sorensen told BioSpace. The Hemab team hopes to “leapfrog” options for these patients who haven’t seen true treatment advancement in decades. Benny Sorensen Its lead asset, HMB-001, is being assessed in the Phase I/II study for Glanzmann Thrombasthenia. The bispecific antibody binds to, stabilizes and recruits factor VIIa at the site of vascular injury to help the blood clot. If successful, HMB-001, along with Hemab’s other pipeline programs, will be the first prophylactic therapeutics for these bleeding and thrombotic disorders. Initial data is anticipated during the second half of 2023. Patients with Glanzmann bleed ten times more frequently than those with hemophilia A and B, Sorensen said. More than half of patients have one bleed event per day, which leads to iron deficiency, fatigue and low mood. The condition is particularly difficult for girls and women of menstrual age who carry the heaviest disease burden. A second program, HMB-VWF, targets the most common bleeding disorder, Von Willebrand disease. VWD has also seen little to no innovation for half a century, Sorensen said. HMB-VWF is still in the preclinical stage but slated for the clinic in 2024. A prophylactic approach has the potential to reduce bleed events significantly. Sorensen, who has been focused on thrombosis and hemostasis for 20 years, said that “every single bleed counts” and every reduction in bleed events is transformative to a patient’s quality of life. The team at Hemab is small – 15 full-time staff – yet chock-full of experience. Sorensen and his group have been involved with more than 50% of all drugs developed in this space. For investing partner Access Therapeutics, the team's experience and the chance to be involved in something that could meet a high unmet need in an underserved population “checked all the boxes,” managing director Dan Becker told BioSpace. Becker will join Hemab’s board as part of the transaction. In addition to Access, current investors Novo Holdings, RA Capital Management and HealthCap were joined in this round by Deep Track Capital, Avoro Capital, Invus and more.

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