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Sutacimig Phase 2 interim results demonstrate >50% reduction in treated bleeding event and prophylactic treatment potential in Glanzmann thrombasthenia HMB-002 clinical proof-of-mechanism data in Von Willebrand disease support its potential as a first-in-class prophylactic therapy by elevating VWF levels and extending half-life Natural history insights illuminate true patient burden and prophylactic opportunity to inform transformative, preventative solutions for underserved bleeding disorders COPENHAGEN, Denmark and CAMBRIDGE, Mass., June 24, 2025 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced clinical and preclinical results at the International Society on Thrombosis and Haemostasis (ISTH) 2025 Congress in Washington, DC. The company's 11 abstracts present breakthrough data for sutacimig (formerly HMB-001) and HMB-002, its lead therapeutic candidates, along with crucial insights from natural history studies. "The data presented at ISTH underscore the potential for Hemab's impact in addressing the severe unmet needs of people with bleeding disorders," said Kate Madigan, Chief Medical Officer at Hemab. "Our sutacimig and HMB-002 data reveal important progress towards transformative, preventative solutions for these devastating diseases. We're demonstrating the possibilities to meaningfully reduce bleeding and elevate care for people living with Glanzmann thrombasthenia and Von Willebrand disease." Highlights of Clinical Presentations Sutacimig shows promising safety and efficacy in Glanzmann thrombasthenia (GT). The Phase 2 study is fully enrolled (N=34 in Part B), and data show a clinically meaningful reduction in treated bleeding events. Clinically meaningful bleeding reduction: Sutacimig treatment in the efficacy population (n=33) demonstrated a >50% reduction in median Annualized Treated Bleeding Rate (ATBR), with the median ATBR decreasing from 21.2 to 4.61. Select patient reports indicate a reduced severity of bleeds and decreased IV rFVIIa use. Safety profile: Sutacimig demonstrated a favorable safety profile, with most adverse events (AEs) being mild to moderate in severity, and no reported thromboses or discontinuations due to AEs in ongoing dose levels of 0.3 and 0.6 mg/kg. PK/PD insights: At ongoing dose levels of 0.3 and 0.6 mg/kg, Factor VIIa (FVIIa) accumulation reached 2-4 fold above baseline. Exploratory thrombin generation assays suggested hemostatic activity, with observed improvements comparable to those seen with clinically relevant concentrations of rFVIIa. Late Breaking Abstract presentation of HMB-002 demonstrates positive proof of mechanism in Von Willebrand disease (VWD), with the first-in-human VELORA Pioneer study in Type 1 VWD patients. Favorable safety profile: No treatment-emergent AEs, injection-site reactions, hypersensitivity, or serious AEs were reported in the VELORA Pioneer Cohort A1. Encouraging endogenous Von Willebrand Factor (VWF) and FVIII accumulation: The VELORA Pioneer study demonstrated that the initial single 20mg subcutaneous dose of HMB-002 induced consistent and sustained increases in VWF and Factor VIII FVIII levels. Within 14 days, mean VWF rose >1.5-fold from baseline. Normalized APTT and improved thrombin generation: Parallel to the increases in VWF, levels of Factor VIII were corrected, resulting in normalization in APTT and thrombin generation. Preclinical data highlights Sutacimig enhances rFVIIa efficacy in in vitro studies, enabling potentially lower doses for breakthrough bleeds. Studies also show sutacimig preserves the function of stored platelet concentrates (PCs), supporting its mechanism that could reduce both rFVIIa doses and PC volumes needed for bleeding control. Nonclinical safety evaluations of HMB-002 in cynomolgus monkeys and in vitro/ex vivo assays showed no adverse effects, immunotoxicity (including complement and platelet activation or cytokine release), or off-target binding, indicating a favorable safety profile. Extended half-life and infrequent dosing: Pharmacokinetic modeling estimated a long HMB-002 half-life, supporting infrequent subcutaneous dosing. In studies with cynomolgus monkeys, HMB-002 also extended exogenously administered recombinant VWF's half-life, leading to the time-dependent accumulation of endogenous VWF and FVIII, and resulted in an approximately 3-fold prolongation of recombinant VWF in circulation. Natural history highlights GT: Recent findings complementing previous natural history data confirm the severe, often underestimated disease burden of GT. The ATHN Transcends GT study quantifies this with a mean Annualized Bleeding Rate (ABR) of 72.0, with most bleeds requiring treatment (ATBR of 51.9) and 56% of participants experiencing a severe disease burden. A separate retrospective analysis of US-based real-world patient records reveals how this frequent blood loss initiates a self-perpetuating cycle of blood loss and iron deficiency anemia that worsens complications for all patients. This cycle creates a disproportionate unmet need for women, who report high rates of hemorrhage (75%) and anemia (23%), often triggered by heavy menstrual bleeding. Men are also heavily burdened, with significantly higher rates of gastrointestinal bleeding (36%) and hematomas (30%). A systematic care gap compounds the clinical crisis: management is mainly limited to on-demand treatments administered post-bleed, and a large proportion of patients are cared for outside of specialized hematology centers, contributing to ongoing underdiagnosis and underestimation of disease prevalence. This highlights a critical need for improved access to expert care and effective prophylactic therapies for all individuals with GT. VWD: New lived-experience data from the VWD360 study reveals a much higher than previously reported unmet need in VWD, highlighting a significant socioeconomic and psychological burden, especially the disproportionate impact on women suffering from heavy menstrual bleeding. The study found that participants averaged 1.33 bleeds weekly, with 71% missing work and high rates of low mood (73%). Crucially, data from a VWD360 substudy and VELORA Discover challenge historical assumptions, showing Type 1 VWD bleeding rates are comparable to, or even higher than, other subtypes, revealing a major care gap for this neglected patient population. Benny Sorensen, MD, PhD, CEO of Hemab, stated, "Our multiple ISTH presentations underscore Hemab's commitment to leveraging the biotechnological revolution and deep hemostasis expertise to develop innovative prophylactic therapies for patients with bleeding disorders. Our dedication to always listening to the people living with these diseases has generated extensive natural history data revealing substantially greater unmet medical need than previously recognized, driving our continued clinical advancement." About Glanzmann Thrombasthenia Glanzmann thrombasthenia (GT) is a severe bleeding disorder marked by debilitating, sometimes life-threatening bleeding episodes. Results from an international Glanzmann's 360 (GT360) natural history study revealed the substantial burden of this disease: 88% of the 117 participants reported at least one bleed in the previous week, with 34% of those bleeds requiring medical treatment. These bleeding episodes significantly impact patients' mental health and quality of life, with 67% reporting low mood, 52% reporting emotional problems, and 46% experiencing social isolation. Additionally, 81% of participants reported missing school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for GT. About Sutacimig (formerly HMB-001) Sutacimig is a subcutaneously administered 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 mechanism allows for the accumulation of endogenous Factor VIIa in the body and recruitment of Factor VIIa directly to the surface of the activated platelets, where it facilitates hemostatic plug formation. Sutacimig is designed to be a first-in-class prophylactic treatment for Glanzmann thrombasthenia (GT) with the potential to treat other debilitating bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation and Orphan Drug Designation to sutacimig for the treatment of GT while the UK Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway (ILAP). For more information, please visit clinicaltrials.gov (NCT06211634). About Von Willebrand Disease Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. The severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function. About HMB-002 HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Clinical and nonclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.gov (NCT06610201 and NCT06754852). About Hemab Therapeutics Hemab is a multiple clinical-asset biotechnology 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 innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5TM, targets building a pipeline of development programs to deliver long-awaited innovation for patients with high unmet need diseases like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com. Follow us on LinkedIn, Facebook, Instagram, and X. Media Contact: Peg Rusconi [email protected] 617-910-6217 SOURCE Hemab Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Primary Endpoint Met: Statistically significant reduction in heavy menstrual bleeding achieved in all dosage of Merigolix groups compared to placebo Broader Therapeutic Potential: Positive Phase 2 results in both uterine fibroids and endometriosis could place Merigolix as a strong best-in-class candidate for novel drug approval and global licensing opportunities Multi-Indication Development: Clinical trials are underway for three indications enhancing the asset value-uterine fibroids, endometriosis, and assisted reproductive technology (ART), the latter in collaboration with Hansoh Pharma in China SEONGNAM, South Korea, May 7, 2025 /PRNewswire/ -- TiumBio (KRX: 321550.KQ), a clinical-stage biopharmaceutical company focused on discovering and developing innovative therapeutics for patients with rare and incurable disease, today announced that Merigolix (code name: TU2670, Daewon's code name: DW4902) has successfully met the primary endpoint of reducing heavy menstrual bleeding in a Phase 2 clinical trial for uterine fibroids. In this trial, Merigolix demonstrated statistically significant improvements across all dosage groups compared to placebo in patients with uterine fibroids. Merigolix is an investigational, once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist. Unlike traditional GnRH agonists, which require injection and often lead to an initial hormone surge, Merigolix offers a more patient-friendly oral administration without an initial hormonal flare, providing a faster onset of action and improved treatment adherence. The Phase 2 clinical trial of Merigolix in uterine fibroids was conducted by TiumBio's partner, Daewon Pharmaceutical, and enrolled a total of 71 women diagnosed with uterine fibroids. Participants were randomly assigned to receive high, medium, or low doses of Merigolix, or placebo, administered orally once daily for 12 weeks, followed by a 12-week observation period. The results showed that Merigolix led to statistically significant improvements in heavy menstrual bleeding (HMB)—a validated clinical endpoint—across all dosage groups compared to the placebo. In addition to the primary endpoint, Merigolix demonstrated statistically and clinically meaningful improvements in several key secondary endpoints, including fibroid size reduction, increased hemoglobin levels (indicating improved anemia), and relief of pelvic pain. The safety and tolerability profile of Merigolix was consistent with prior clinical data, with no new safety signs. The mechanism of action of Merigolix can be used in various disorders, including endometriosis, uterine fibroids, ART, precocious puberty, and prostate cancer. Notably, in a separate Phase 2 clinical trial for endometriosis completed in 2024, Merigolix also showed excellent therapeutic efficacy and safety, further reinforcing its potential as a promising new treatment. "We are very excited that Merigolix has demonstrated excellent therapeutic efficacy not only in endometriosis but also in uterine fibroids," said Hun-Taek Kim, Ph.D., MBA, CEO of TiumBio. "successfully achieving both efficacy and safety in Phase 2 significantly increases the probability of regulatory approval and licensing opportunities. We plan to accelerate development and expand into the global uterine fibroid treatment market," he added. "Uterine fibroids are a condition that causes significant suffering for many women, and we believe Merigolix has the potential to become an important new oral treatment that addresses this unmet medical need," said Jonathan In Baek, President of Daewon Pharmaceutical. "We are pleased with the positive results in uterine fibroids and will continue our efforts to provide patients with a convenient and effective new treatment option," he continued. Uterine fibroids are benign tumors that develop in the uterus, primarily affecting women of reproductive age, particularly those in their 30s and older. The condition has a high prevalence rate of approximately 20% among women over the age of 35. The most common symptom is heavy menstrual bleeding, but it can also cause abnormal uterine bleeding, pelvic pain, and anemia. Meanwhile, TiumBio entered into a licensing agreement with Daewon Pharmaceutical in 2019, granting Daewon the rights to develop and commercialize Merigolix in South Korea. About TiumBio Co., Ltd. TiumBio (KRX: 321550.KQ) is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative therapeutics for patients with rare and incurable diseases. Its mission is to expand the hope and happiness of mankind through our science. TiumBio has three leading pipeline assets: Merigolix (code name: TU2670), TU2218, and TU7710, all in various stages of clinical development. Merigolix is a once-daily, oral GnRH receptor antagonist being developed for the treatment of endometriosis, uterine fibroids, and assisted reproductive technology. TU2218 is a first-in-class oral immune-oncology therapy targeting TGF-β and VEGF pathways to promote response rates in cancer patients when used in combination with immune checkpoint inhibitors. TU7710 is a novel rFVIIa designed to extend its half-life in order to provide more clinical benefits to hemophilia patients with inhibitors. With its expertise in drug development, TiumBio is committed to the discovery and development of innovative treatments to ease the burden of debilitating diseases. For further information, visit our website at and connect with us on LinkedIn. Contacts: Junseok Jang, Head of Corporate Communications & Investor Relations [email protected] Da-ye Song, Manager, Corporate Communications & Investor Relations [email protected] SOURCE TiumBio WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED