Hemab Therapeutics ApS

HMB-001 is a novel bispecific antibody designed to be the first prophylactic treatment for Glanzmann Thrombasthenia (GT) and other debilitating bleeding disorders Phase 1 was successfully completed in the UK; Hemab plans additional sites in Europe and the U.S. for Phase 2 The U.S. FDA has cleared the Investigational New Drug (IND) application and granted Fast Track Designation to HMB-001 for the treatment of GT COPENHAGEN, Denmark and CAMBRIDGE, Mass., Dec. 11, 2023 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced today that it has completed Phase 1, the single ascending dose part, and transitioned to Phase 2, the multiple ascending dose part, of its Phase 1/2 clinical study of HMB-001 in Glanzmann Thrombasthenia (GT), a platelet disorder that causes severe, potentially life-threatening bleeding episodes. The company also announced that the U.S. Food and Drug Administration (FDA) has cleared Hemab's investigational new drug application (IND) for HMB-001 in GT, enabling enrollment in the U.S. Phase 1 of the clinical study was completed in the UK, and Phase 2 will include additional sites in Europe as well as the U.S. In addition, the FDA granted Fast Track designation to HMB-001, emphasizing the seriousness and high unmet need for treatments for GT. The Fast Track program enables Hemab to have more frequent interactions with the FDA to facilitate the development of HMB-001. "People living with Glanzmann Thrombasthenia can experience frequent, sometimes severe bleeds that can be life-threatening and compromise their quality of life. Currently, there are no prophylactic treatment options that would reduce or prevent these bleeding episodes," said Benny Sorensen, MD, PhD, CEO of Hemab. "The completion of Phase 1 on time and transitioning to Phase 2 is an important milestone for HMB-001 and Hemab. Furthermore, expanding our clinical study into the U.S., following clearance of the IND, and Fast Track designation are a testament to the importance of advancing prophylactic treatment for people living with Glanzmann Thrombasthenia." The Phase 1/2 clinical study evaluates the safety, tolerability, pharmacodynamics, and pharmacokinetics of HMB-001. Initial efficacy signals based on an assessment of changes in bleeding frequency will also be measured. The study is composed of three parts: Part A, single ascending dose, Part B, multiple ascending dose, and Part C, extended dosing. Hemab plans to report data from the Phase 1, single ascending dose portion, at an international scientific conference in early 2024. The Phase 1/2 study design was detailed in the company's poster presentation (number 1225), "A Phase 1/2, First-in-Human, Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia," at the 65th American Society of Hematology Annual Meeting and Exposition. About Glanzmann Thrombasthenia Glanzmann Thrombasthenia (GT) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann's 360 (GT360) natural history study, Hemab's research initiative in partnership with UK specialist research consultancy Haemnet, found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, and 37% of those bleeds required medical treatment. These bleeding episodes have a significant impact on the mental health and quality of life of people living with GT. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively) and 80% reported that they missed school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for people living with GT. About HMB-001 HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders. About Hemab Therapeutics Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5TM, targets the development of 5 independent assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease, and other serious disorders. Learn more at hemab.com. SOURCE Hemab Therapeutics

Clotting disorders can be treated by infusing patients with clotting proteins they lack, or with drugs that replace the function of these proteins. Gene therapies may soon become another option. Hemophilia A and B are perhaps the best-known clotting diseases, and both have benefited from decades of research while rarer bleeding disorders continue to lack treatment options, according to Benny Sorensen, CEO of Hemab Therapeutics. Hemab is already in the clinic with lead program in development for a rare clotting disorder that has no approved therapies. Now the biotech startup, which splits its operations between Boston and Copenhagen, Denmark, has $135 million to support that drug candidate as well as a pipeline of other programs aiming to bring innovation to underserved bleeding and thrombotic disorders. “That’s the vision, that’s the mission of Hemab—to leapfrog the treatment options into the 21st century,” Sorensen said. The Series B round of financing announced Tuesday was led by Access Biotechnology. Lead Hemab drug candidate HMB-001 is in development to treat Glanzmann thrombasthenia, an inherited disorder in which a genetic abnormality leads to platelets that cannot properly perform their clotting functions. Those who have the disorder tend to bruise and bleed easily. Even if these patients have platelet numbers in the range that’s considered normal, it takes them longer to stop bleeding. Standard treatment is a blood platelet transfusion, which requires patients to make regular visits to a transfusion center for the rest of their lives. One potential problem is that patients may develop antibodies against the transfused platelets, which limits their effectiveness. With HMB-001, Hemab aims to use blood clotting proteins already in the body to help improve platelet function. The drug is a bispecific antibody with one part designed to bind to and stabilize a protein called factor VII (FVIIa) and another part that binds to a target on activated platelets called TLT-1. By recruiting FVIIa to the surface of platelets, the drug is intended to facilitate clotting and also avoid clotting activity when there is no tissue damage. Hemab is developing this drug to prevent bleeding episodes in Glanzmann patients. Sorensen’s experience includes senior roles at Alnylam Pharmaceuticals and Codiak Biosciences, both of which are working with new drug modalities. Alnylam was the first company to win approval of a medicine that works by leveraging a mechanism called RNA interference. Codiak’s still experimental approach uses exosomes, tiny vesicles that shuttle between molecules between cells. By contrast, Hemab isn’t reinventing the wheel with its bleeding disorder drugs. Sorensen said the company is pursuing known targets by using antibodies, an approach validated in many diseases, including blood disorders. For example, Roche hemophilia A drug Hemlibra showed that bispecific antibodies can work to treat clotting disorders. Hemab’s scientists have deep expertise in clotting biology, with knowledge about every protein involved in coagulation and how to manipulate them, Sorensen said. What Hemab is doing is translating that knowledge into new medicines. The company rationally designs antibodies that can generate a desired mechanism of action. Depending on the disease, the company’s approach could involve a bispecific antibody or a monoclonal antibody. In preclinical research, Hemab reported that HMB-001 led to the formation of fibrin on platelets. Fibrin is a protein that is produced in response to bleeding and is a component of blood clots. The data also showed the accumulation of FVIIa. A Phase 1/2 study evaluating HMB-001 dosed its first patient last month. Preliminary data are expected in the second half of this year. HMB-001 was licensed from Novo Nordisk. The rest of the pipeline is based on Hemab’s internal research. The most-advanced of the internally developed programs, HMB-VWF, is in development for von Willebrand disease, which is characterized by low levels of the von Willebrand clotting protein. Three more preclinical programs are in the pipeline. Sorensen said Hemab’s goal is to have all five of them in the clinic by 2025. Those drug candidates all address rare conditions. Sorensen left the door open to addressing more prevalent clotting disorders, but he added that Hemab is not going to pursue hemophilia and is instead focused on diseases with unmet need. Hemab closed a $55 million Series A round of funding in 2021. The latest financing adds new investors Deep Track Capital, Avoro Capital, Invus, Rock Springs Capital, and Maj Invest Equity. They were joined by earlier investors Novo Holdings, RA Capital Management, and HealthCap. Sorensen acknowledged the challenging economic environment but he said Hemab was able to generate strong investor interest. “If you have a recognized unmet need, you‘ve got strong science, the medicines are important and you have an accomplished team, that’s the recipe for being successful,” he said.

Funding enables completion of ongoing Phase 1/2 study of bispecific antibody HMB-001 in Glanzmann Thrombasthenia, initiation of pivotal studies, start and conclusion of Phase 1/2 study of HMB-VWF in von Willebrand Disease, as well as future pipeline evolution. COPENHAGEN, Denmark and BOSTON, Feb. 21, 2023 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the closing of an oversubscribed $135 million Series B financing. Access Biotechnology led the round, with participation from new investors Deep Track Capital, Avoro Ventures, Invus, Rock Springs Capital, and Maj Invest Equity, as well as all current investors Novo Holdings, RA Capital Management, and HealthCap. "Hemab is fundamentally reimagining the treatment paradigm for underserved bleeding and thrombotic disorders. This financing will allow us to progress our clinical programs for the first prophylactic treatments for Glanzmann Thrombasthenia and von Willebrand Disease, delivering functional cures for patients in need," said Benny Sorensen, MD, PhD, CEO & President of Hemab. "We're grateful for this robust syndicate of investors who support our approach of leveraging validated advanced technologies and deep insights into the biology of clotting to overcome decades of scientific stagnation." The financing will support Hemab's scientific and corporate growth plans through 2025, including completion of an ongoing Phase 1/2 clinical study of lead candidate HMB-001 in Glanzmann Thrombasthenia, initiation of pivotal studies, start and completion of Phase 1/2 clinical evaluation for HMB-VWF in von Willebrand disease, and future pipeline evolution in accordance with the company's Hemab 1-2-5™ strategic guidance—targeting development of 5 clinical assets by 2025. "Strong investor confidence—in this case, an upsized round and more than $200 million in demand—is a testament to the expertise of the Hemab team, their validated scientific approach, and the opportunity to bring long-overdue innovation to patients living with severe bleeding and thrombotic diseases," said John Maraganore, PhD, Chair of Hemab's Board of Directors. "As founding investor and the company's largest shareholder, we are excited to support Hemab in the next phase of its plan to build the ultimate clotting company, with a Nordic foundation and global footprint," said Jørgen Søberg Petersen, MD, PhD, MBA, Partner at Novo Holdings. Hemab also announced Dan Becker, MD, PhD, Managing Director at Access, and Uya Chuluunbaatar, PhD, Partner at Avoro Ventures, will also join as Directors. Christine Borowski, PhD, Vice President at Access, will join as a Board Observer. "We are thrilled to partner with Hemab's exceptional leadership to advance a pipeline of transformative new medicines for areas of high unmet need in hemostatic disorders," said Dr. Becker. About Hemab Therapeutics Hemab is a clinical-stage biotech company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in the US and Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5TM, targets the development of 5 clinical assets by 2025 to deliver long-awaited innovation for patients with Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease, Hereditary Hemorrhagic Telangiectasia (or Osler-Weber-Rendu disease), Congenital Antithrombin III Deficiency, and other serious disorders. Learn more at hemab.com. About HMB-001 HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and TLT-1 on activated platelets with the other arm. This allows for accumulation of FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 was designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with potential for other debilitating rare bleeding disorders. It entered Phase 1/2 clinical evaluation in late 2022, with initial data expected 2H 2023. SOURCE Hemab Therapeutics