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Exciting Developments in Hemophilia Treatments: Hemab Therapeutics Breakthroughs

In a notable unveiling at the 2025 EAHAD Annual Congress held in Milan, Hemab Therapeutics, a pioneering biotechnology firm, presented promising interim results from its ongoing Phase 2 study of HMB-001. This study marks a significant milestone as it evaluates the first-ever prophylactic treatment specifically for Glanzmann thrombasthenia, a rare bleeding disorder that severely impacts patients' quality of life.

Glanzmann thrombasthenia is characterized by debilitating bleeding episodes that can exceed 60-80 incidents requiring treatment each year. The presented data from the Phase 2 trial demonstrated a staggering reduction of treated bleeds by over 50% across all tested dose cohorts, highlighting HMB-001's potential to transform treatment paradigms for patients suffering this condition.

Phase 2 Study Insights

The Phase 2 trial includes a minimum of a 6-week run-in period where participants diligently record bleeds through an electronic diary, followed by a three-month treatment regimen with HMB-001. This innovative treatment, a bispecific antibody, uniquely binds to and stabilizes endogenous Factor VIIa while targeting activated platelets, thereby offering a new approach to hemostatic plug formation.

The results not only underscore the efficacy of HMB-001 but also reveal its potential to meet a critical medical need, having been granted Orphan Drug Designation by both the U.S. FDA and the UK’s Medicines and Healthcare products Regulatory Agency. This reflects a growing recognition in the medical community of the dire need for effective treatments that address the undertreated nature of bleeding disorders.

Insights from Experts

Kate Madigan, Chief Medical Officer at Hemab, emphasized that the interim Phase 2 results signal a new beginning for the Glanzmann thrombasthenia community. “These results bring us closer to a breakthrough in preventing bleeding,” stated Madigan. Alongside this, preclinical data for HMB-002, targeting Von Willebrand Disease (VWD), was also presented as a potential novel treatment option, showcasing Hemab’s commitment to advancing care for these underserved disorders.

Dr. Suthesh Sivapalaratnam, a consultant hematologist, expressed the profound impact of the findings, stating, “The Phase 2 results highlight the transformative potential of HMB-001. For those living with Glanzmann thrombasthenia, this could redefine care standards and bring renewed hope.” The significant bend in research and treatment outcomes represents a possible pivotal shift for patients, many of whom have experienced either limited options or ineffective symptom management.

HMB-002: Addressing Von Willebrand Disease

In tandem with HMB-001, Hemab's HMB-002 aims to pave the way for enhanced VWD treatments. By enhancing endogenous Von Willebrand Factor levels, this treatment seeks to address the root causes of bleeding episodes rather than merely alleviating symptoms, a common limitation of existing therapies.

Preclinical data suggest that HMB-002 may emerge as a favorable alternative, allowing for a long-lasting subcutaneous prophylactic treatment for individuals suffering from VWD. Initial findings indicate a safe and effective profile, enhancing VWF activity without compromising the protein's functionality—promising indicators for future clinical applications.

Conclusion and Future Directions

The transformative research presented by Hemab Therapeutics not only brings optimism but also highlights an urgent need for continued innovation in the treatment landscape for bleeding disorders like Glanzmann thrombasthenia and Von Willebrand Disease. As recruitment for the Phase 2 trials continues, the anticipation grows for advancements that could significantly improve the quality of life for countless patients burdened by these serious conditions.

For more information about Hemab Therapeutics and their groundbreaking work, visit hemab.com.