Hemab ApS

HMB-001 was well tolerated with no drug-related adverse or thromboembolic events; dose-dependent improvements in blood-clotting activity support the potential of HMB-001 as a first-in-class prophylactic treatment for Glanzmann Thrombasthenia and other blood clotting disorders Preclinical HMB-001 findings were published in the February 2024 issue of Nature Cardiovascular Research COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 9, 2024 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented initial results from Phase 1 of its ongoing evaluation of HMB-001. A novel bispecific antibody, HMB-001 is in development as a prophylactic treatment for the bleeding disorder Glanzmann Thrombasthenia (Glanzmann). The data were presented as one of 10 abstracts selected for the SLAM oral presentation session at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Frankfurt, Germany. "The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day," said Joe Vogel, Senior Director and Program Lead for HMB-001 at Hemab. "With Phase 2 already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients' quality of life." The Phase 1/2 first-in-human open-label study in patients with Glanzmann is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of HMB-001. As part of the UK-based Phase 1 single-ascending dose, seven patients received subcutaneous HMB-001 at 0.2 mg/kg, (n=1), 0.5 mg/kg (n=3) or 1.25 mg/kg (n=3). Over a 56-day observation period, HMB-001 was well tolerated; the most common adverse events (AEs) were mild or moderate in severity. No AEs or serious adverse events were deemed related to HMB-001. There were no dose-limiting toxicities and no thromboembolic events reported. Treatment with HMB-001 resulted in a dose-dependent pharmacodynamic effect of endogenous Factor VIIa accumulation that was associated with decreases in prothrombin time and improvement in exploratory thrombin generation analyses. The pharmacokinetic profile and half-life of HMB-001 support dosing every two weeks or a less-frequent dosing regimen. "People living with bleeding disorders like Glanzmann face devastating bleed events and considerable psychosocial impacts—from severe pain and social stigmatization to depression and isolation—but have no approved preventative treatments to turn to," said Suthesh Sivapalaratnam, MD, PhD, Queen Mary University of London, Barts Health NHS Trust. "These Phase 1 data support further investigation into the promise of HMB-001 for patients and providers across the globe who are awaiting innovation to improve the standard of care." Hemab has begun dosing and is actively enrolling in Phase 2, the multiple-ascending dose of the study, which will be expanded to sites across Europe and the U.S. For more information, visit clinicaltrials.gov (NCT06211634). In addition, Hemab announced that preclinical research findings of HMB-001 are now available online as part of the February 2024 issue of Nature Cardiovascular Research. The study results demonstrate the ability of HMB-001 to accumulate and potentiate the activity of endogenous Factor VIIa selectively on activated platelets, providing a sustained and localized procoagulant activity that may support prophylactic treatment of Glanzmann or other bleeding disorders. Read the paper here. About Glanzmann Thrombasthenia Glanzmann Thrombasthenia (Glanzmann) 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, with 37% of those bleeds requiring medical treatment. These bleeding episodes significantly impact the mental health and quality of life of people living with Glanzmann. 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 Glanzmann. About HMB-001 HMB-001 is bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa 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. The U.S. Food and Drug Administration granted Fast Track Designation to HMB-001 for the treatment of Glanzmann. 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 1-2-5™, targets the development of 5 assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com. Follow us on LinkedIn and X (formerly known as Twitter). SOURCE Hemab Therapeutics

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

BOSTON, Sept. 06, 2023 (GLOBE NEWSWIRE) -- Cerevance, a private, clinical-stage drug discovery and development company focused on advancing novel, precision neuroscience therapeutics for central nervous system (CNS) diseases using the company’s proprietary Nuclear Enriched Transcript Sort sequencing (NETSseq) platform, today announced Craig Thompson, chief executive officer (CEO) and Johnna Simões, vice president, corporate development, will participate in panel discussions during the 6th Annual LSX World Congress USA being held in Boston, MA, September 13-14th. Panel details: Title: Keynote Panel: Optimising Commercial Growth and Striving for Biotech ExcellenceDate: Thursday, September 14th Time: 9:40am ETSpeakers: Benny Sorensen, CEO, HemabGregory Verdine, CEO, LifeMine TherapeuticsEllen Donnelly, CEO, AblivaCraig Thompson, CEO, Cerevance Title: Building a Biotech Company: Key Milestones, Expert Tips and Strategies for SuccessDate: Thursday, September 14th Time: 11am ETSpeakers: Jasper Bos, General Partner, ForbionRuta Laukien, Managing Partner, Graybella CapitalJohnna Simões, Vice President, Corporate Development, Cerevance For more information and registration details, see the LSX website here. About CerevanceCerevance is focused on the development of treatments for central nervous system (CNS) disorders, prioritizing chronic neurodegenerative conditions such as Alzheimer's disease, Parkinson's disease, and Amyotrophic Lateral Sclerosis (ALS). Utilizing a large and growing collection of over 14,000 human brain tissue samples, Cerevance is generating an unprecedented level of expression and epigenetic data thereby enabling the company to identify the most promising targets for the next generation of treatments for CNS disorders. The company utilizes its proprietary NETSseq platform and advanced machine learning techniques to uncover the gene expression profiles of select cell types to identify novel targets that are uniquely expressed in relevant circuits affected by diseases or are altered in disease states. With the information obtained from its research, combined with the expertise of its team of scientists and drug developers, Cerevance is advancing multiple therapeutics that selectively modulate the discovered targets. These treatments are progressing through clinical development, with CVN424, CVN766, and CVN293 being the furthest along in the pipeline. CVN424 is a first-in-class non-dopamine therapy that shows promise in improving both motor and non-motor symptoms of Parkinson's disease and may also have disease-delaying effects. CVN766 is a potent antagonist of the orexin 1 receptor with high selectivity over the orexin 2 receptor which may benefit a variety of psychiatric conditions including schizophrenia, anxiety/panic, binge eating/obesity, substance use disorder, and Prader-Willi Syndrome. CVN293 is a novel blocker of potassium efflux in glia, regulating the inflammasome in individuals living with ALS and Alzheimer's disease. By leveraging its extensive collection of brain tissue samples, employing advanced technologies, and generating actionable data, Cerevance aims to transform the lives of patients affected by CNS diseases. Contacts Cerevance: Johnna Simoes, ir@cerevance.com Media: Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868