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  • Microbiota-Based MaaT013 Shows Strong Efficacy in Refractory GI-aGVHD | Targeted Oncology

    Microbiota-Based MaaT013 Shows Strong Efficacy in Refractory GI-aGVHD | Targeted Oncology

    Primary analysis data from the phase 3 ARES trial (NCT04769895) show that the microbiota-based therapeutic MaaT013 (Zervyteg) generated compelling efficacy signals and acceptable safety in patients with acute graft-vs-host disease with gastrointestinal involvement (GI-aGVHD) who are refractory to corticosteroids and ruxolitinib (Jakafi).1 Results were presented by Florent Malard, MD, PhD, professor of Hematology at Saint Antoine Hospital and Sorbonne University, at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida.

    As of the data cutoff on November 11, 2024, the study met its primary end point with a GI overall response rate (GI-ORR) of 62% (95% CI, 49%–74%) among 66 patients treated with MaaT013 on day 28 of treatment, significantly exceeding the historical control of 22% (P <.0001). The all-organ ORR was 64% (95% CI, 51%–75%). Notably, both GI and all-organ responses exhibited a similar average duration of response (DOR) of 6.4 months (95% CI, 4.8–8.0).

    Furthermore, data describing key secondary end points revealed that responses were maintained through later time points. At day 56 and month 3, the GI-ORRs were 49% and 44%, respectively; likewise, all-organ ORRs were 48% and 44%. Responses across all time points showed exceptionally high rates of complete response and very good partial response.

    Importantly, the deep and durable responses translated into promising survival benefits. A Kaplan-Meier graph displayed a separation of curves between responders and nonresponders beginning shortly after the first administration of MaaT013, widening substantially over the course of 1 year with responders maintaining a clear survival advantage over time. Median overall survival (OS) data were immature at the time of analysis, but the estimated probability of survival at 1 year was 54%, a clinically meaningful improvement for this patient population with historically poor clinical outcomes.

    Safety and Tolerability

    MaaT013’s safety profile was determined to be acceptable. There were 157 serious treatment-emergent adverse events (AEs) reported across 76% of patients, with the most common being escherichia sepsis, general physical health deterioration, and septic shock.

    There was a total of 34 treatment-related AEs across 29% of patients, which mainly comprised various bacterial infections and GI disorders. Of these events, 7 were considered serious. There were 26 fatal AEs, with 1 event of septic shock determined to be related to MaaT013 by investigators.

    About MaaT013 and the ARES Trial

    The phase 3 ARES trial is a single-arm, multicenter, open-label trial in Europe investigating MaaT013, a pooled allogeneic fecal microbiotherapy, as salvage therapy in adult patients with refractory GI-aGVHD.2 The primary end point of the trial is GI-ORR at day 28, assessed by an independent review committee (IRC); secondary end points include GI-ORR at day 56 and month 3, all-organ ORR at day 28, and DOR per IRC and investigator assessment, as well as OS. In the study, MaaT013 was administered as a rectal suspension as a 150-mL enema.

    Patients were included if they had undergone allogeneic hematopoietic stem cell transplant, experienced an aGVHD episode with lower GI symptoms per MAGIC guidelines, and were resistant to systemic steroids and either resistant or intolerant to ruxolitinib. Key exclusion criteria included having active cytomegalovirus colitis, lines of aGVHD treatment other than steroids and ruxolitinib, hyperacute GVHD, and active uncontrolled infection.

    Of the 66 total patients, the majority (77%) had aGVHD with involvement limited to the GI tract. Others had GI and skin involvement (17%), GI and liver involvement (3%), and involvement of all 3 organs (3%). Regarding the hypothesized mechanisms of action driving responses in the skin and liver, Malard offered some preliminary insights based on earlier research.

    “So far, based on this study, we don’t have the translational data to investigate how this is working, but from the previous [phase 2] HERACLES study [NCT03225937], we already have some data on the fact that we have some systemic immunomodulatory effect of the drug, with some decrease in the proinflammatory cytokine at the systemic level, and also an increase in essential fatty acid… in the serum of the patient,” Malard explained in the question-and-answer session. “We are also going to evaluate in another study all the immune cell subsets, in particular Tregs and so on, to find if this is how it’s working.”

    MaaT013 is currently under regulatory review by the EMA following submission of a marketing authorization application in June 2025, with a decision regarding approval anticipated in the second half of 2026.3 If approved, MaaT013 would become the first microbiome-based therapy for the treatment of this high-need disease.

    DISCLOSURES: Malard declared receiving honoraria from Priothera, Jazz Pharmaceuticals, Therakos, Sanofi, Novartis, AstraZeneca, and MSD.

    REFERENCES
    1. Malard, F. MaaT013 for ruxolitinib-refractory acute graft-versus-host disease with gastrointestinal involvement: Results from the ARES phase III trial. Presented at: 67th American Society of Hematology Annual Meeting and Exposition; December 6–9, 2025; Orlando, Florida. Abstract 817.‌
    2. MaaT013 as salvage therapy in ruxolitinib-refractory GI-aGVHD patients (ARES). ClinicalTrials.gov. Updated October 17, 2024. Accessed December 6, 2025. https://clinicaltrials.gov/study/NCT04769895
    3. November 3, 2025: Maat Pharma announces positive phase 3 results evaluating Xervyteg® (MaaT013) in acute graft-versus-host disease selected for oral presentation at ASH Congress 2025. News release. MaaT Pharma. November 3, 2025. Accessed December 7, 2025. https://tinyurl.com/su2wha2y

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  • IIHF Statement on Ice Hockey Rink

    IIHF Statement on Ice Hockey Rink

    Below is the statement published by the International Ice Hockey Federation regarding the Ice Hockey rink dimensions for Milano Cortina 2026:

    The IIHF can confirm that the ice surfaces for the Milano Cortina 2026 Olympic Winter Games will measure…

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  • Large Earthquake Strikes Japan: The Latest

    Large Earthquake Strikes Japan: The Latest

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    Earthquake, Tsunami Warning Rocks North Japan Coast

    A large earthquake struck near Japan’s east coast late Monday night local time, triggering tsunami waves and injuring several people nearby.

    The quake was given a preliminary rating of 7.6,…

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  • Peru reports first case of baby born after ectopic pregnancy in liver

    Peru reports first case of baby born after ectopic pregnancy in liver

    Baby Aylin is held by her mother, Valeria Vela, at Cayetano Heredia Hospital in Lima, Peru, on Wednesday. Aylin is only the fourth case in the world of a successful hepatic ectopic pregnancy, where a mother was able to carry the pregnancy to term…

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  • New USEFP building symbolises ‘spirit of collaboration’ in Pak-US educational ties: minister

    New USEFP building symbolises ‘spirit of collaboration’ in Pak-US educational ties: minister

    Minister of State for Education Wajiha Qamar (left) and US Charge d’Affaires Natalie A Baker inaugurate new purpose-built headquarters of United States Educational Foundation in Pakistan (USEFP) in Islamabad on…

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  • Pharmacy Deserts: A Crisis Health Care Providers Can Help Solve with Technology and Teamwork

    Pharmacy Deserts: A Crisis Health Care Providers Can Help Solve with Technology and Teamwork

    A quiet emergency is taking shape across America’s health care landscape, one defined not by new diseases, but by disappearing access. Pharmacy deserts are an escalating public health crisis in the US. Nearly 46% of US counties lack convenient pharmacy access, leaving over 16 million Americans without nearby options for essential medications. This access gap reflects a deeper inflection point: the traditional retail pharmacy model is no longer sustainable in many communities, pushing health care providers and technology partners to rethink how and where care is delivered.

    As major retail pharmacy chains shutter thousands of stores, people in affected areas must travel over an hour to get prescriptions, vaccines, or basic health care needs. These closures are caused by a combination of financial pressure, industry consolidation, and changing consumer expectations, all of which creates a perfect storm that could leave millions of people behind.

    This growing inaccessibility has a tangible cost. Medication nonadherence, often driven by lack of pharmacy access, adds $290 billion annually in avoidable healthcare expenses, including $100 billion in preventable hospitalizations.3 Rising drug costs and fragmented care coordination are now the leading forces behind declining adherence, intensifying disparities in chronic disease management and preventive care. When patients can’t access or refill prescriptions easily, the consequences ripple across the healthcare system, leading to poorer outcomes and higher costs.

    Technology: Bridging the Accessibility and Care Gap

    Digital innovation is essential to reversing this trend. Telepharmacy, mobile pharmacy apps, and artificial intelligence (AI)-enabled dispensing can dramatically extend reach, bringing pharmacists to patients virtually, 24/7. An analysis by Accenture shows telehealth visits are now 38 times higher than prepandemic levels, demonstrating that patients are ready for digital engagement.4

    Meanwhile, 50% of OTC sales are projected to occur online within the next 3 to 4 years, and mobile pharmacy purchases are growing at a 19% compound annual growth rate through 2026. These shifts, along with home delivery, offer a blueprint for equitable access beyond physical locations. Yet technology alone isn’t the answer. Its impact depends on how providers integrate digital tools into care coordination and patient engagement models.

    Reimagining Pharmacies as Frontline Care Hubs

    For the pharmacies that remain, they must evolve from the last line of defense to the front line of care. Integrating pharmacies earlier in the care journey and connecting patients, payers, providers, and pharmacists creates a unified ecosystem that supports adherence and preventive health. Research shows that patients who receive pharmacy-based medical interventions have 3% higher medication adherence and 2.7% fewer emergency visits than those who do not.3

    For providers, stepping directly into pharmacy access offers both necessity and opportunity. As value-based care expands, providers are increasingly accountable for outcomes that hinge on medication adherence. Direct partnerships, or even owned digital pharmacy models, allow them to close last-mile access gaps, reduce readmissions, and deliver continuous care outside traditional settings.

    By expanding pharmacists’ roles to include services such as chronic disease monitoring, vaccinations, and medication therapy management, we can build stronger, value-based care models rooted in accessibility and trust.

    Feeding the Appetite for Change

    This type of change requires collaboration between providers, pharmacists, and technology innovators. Encouragingly, Accenture research shows nearly 80% of US adults say they are willing to share data within connected health systems to gain better access and coordinated experiences.5 The urgency is clear: Access is declining fastest where social determinants already strain health equity. Providers who act now can redefine their role not only as caregivers but also as enablers of consistent, affordable access to medication.

    Ultimately, pharmacy deserts are a symptom of systemic fragmentation, and the cure lies in coordinated, technology-enabled care models that meet patients where they are. By transforming pharmacies into digital-first health access points, we can bridge gaps, improve outcomes, and build a healthier, more equitable future for all.

    REFERENCES
    1. Study finds 46 percent of US counties have pharmacy deserts. News release. National Community Pharmacists Association. August 28, 2024. Accessed December 8, 2025. https://www.ncpa.org/newsroom/qam/2024/08/28/study-finds-46-percent-us-counties-have-pharmacy-deserts
    2. Nowosielski B. Pharmacy deserts prominent in areas of high social vulnerability. Drug Topics. August 23, 2024. Accessed December 8, 2025. https://www.drugtopics.com/view/pharmacy-deserts-prominent-in-areas-of-high-social-vulnerability
    3. Kwan N. The impact of pharmacy deserts. US Pharm. 2024;49(4):32-36.
    4. Bestesennyy O, Gilbert G, Harris A, Rost J. Telehealth: a quarter-trillion-dollar post-COVID-19 reality? McKinsey & Company. July 9, 2021. Accessed December 8, 2025. https://www.mckinsey.com/industries/healthcare/our-insights/telehealth-a-quarter-trillion-dollar-post-covid-19-reality
    5. Accenture study finds growing demand for digital health services revolutionizing delivery models: patients, doctors + machines. News release. Accenture. March 6, 2018. Accessed December 8, 2025. https://newsroom.accenture.com/news/2018/accenture-study-finds-growing-demand-for-digital-health-services-revolutionizing-delivery-models-patients-doctors-machines

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  • Tern leukemia drug results likely to catch investor attention

    Tern leukemia drug results likely to catch investor attention

    Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

    ORLANDO, Fla. — Terns Pharmaceuticals reported an update Monday on its targeted leukemia drug that maintained and even boosted molecular response rates in advanced-stage patients. 

    The study results, while still early, are likely to draw even more positive attention from investors who already view the Terns drug as a potential successor to a commercial blockbuster from Novartis. 

    At 24 weeks, four escalating doses of the Terns drug, called TERN-701, achieved a major molecular response of 64% in patients with chronic myeloid leukemia, a slow-growing cancer that starts in myeloid cells. The 28 patients evaluable had already experienced treatment with a median of three drugs.

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  • Teenage Afghan asylum seekers who abducted and raped girl, 15, sentenced | Warwickshire

    Teenage Afghan asylum seekers who abducted and raped girl, 15, sentenced | Warwickshire

    Two teenage Afghan asylum seekers who abducted and raped a 15-year-old girl have been given custodial sentences.

    Jan Jahanzeb and Israr Niazal, both 17, face possible deportation and were ordered to register as sex offenders following the…

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  • Hemab Therapeutics Announces Positive Complete Phase 2 Data for Sutacimig in Glanzmann Thrombasthenia at ASH 2025; Plans to Advance to Pivotal Phase 3 Study

    Hemab Therapeutics Announces Positive Complete Phase 2 Data for Sutacimig in Glanzmann Thrombasthenia at ASH 2025; Plans to Advance to Pivotal Phase 3 Study

    Consistent and robust efficacy demonstrated across bleed types, locations, and dose cohorts

    The weekly dosing cohort achieved an estimated 87% reduction in annualized treated bleeding rate (ATBR)

    Results validate potential as the first prophylactic therapy to address the heavy physical and psychosocial burden of Glanzmann thrombasthenia; Phase 3 registration study planned for 2026

    CAMBRIDGE, Mass. and COPENHAGEN, Denmark, Dec. 8, 2025 /PRNewswire/ — Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced positive results from its completed Phase 2 multiple ascending dose (MAD) portion of the CL-101 study of sutacimig for the prophylactic treatment of Glanzmann thrombasthenia (GT).

    The data, presented today in an oral session at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, demonstrate clinically meaningful efficacy that was consistent across bleed locations, bleed types (spontaneous and traumatic), and dose cohorts evaluated. Based on these results, Hemab plans to advance sutacimig into a pivotal Phase 3 registration study in 2026.

    “These Phase 2 results represent transformational potential for people living with Glanzmann thrombasthenia, who have waited a lifetime for a modern prophylactic treatment,” said Benny Sorensen, MD, PhD, Chief Executive Officer of Hemab. “The clinically meaningful reductions in bleeding demonstrated across this study provide compelling evidence that sutacimig could shift the treatment paradigm from reactive crisis management to prevention. We are moving forward with urgency to bring this therapy to patients who have been overlooked for far too long.”

    “What stands out in these results is the reduction of the most severe bleeding events requiring intensive interventions,” said Paul Saultier, MD, PhD, Head of the French Platelet Reference Center, APHM Hospital de la Timone. “These are the bleeds that bring patients to the hospital and create the greatest burden. Combined with the reductions we saw across different bleed types and anatomical locations, these data suggest sutacimig could provide meaningful benefit for GT patients.”

    Phase 2 Clinical Data Highlights: Hemab’s Phase 2 study of sutacimig (N=34) is intended to address a profound gap in care for GT as there are currently no effective prophylactic treatment options. Sutacimig was assessed at varying doses to determine the optimal regimen for Phase 3. Key findings include:

    • Consistent and clinically meaningful reductions in bleeding: Sutacimig demonstrated robust and clinically meaningful reductions in ATBR across dose cohorts, with an approximate 50% reduction in mean ATBR in the overall efficacy population (N=31). The weekly dosing cohort achieved an estimated 87% reduction in ATBR (95% CI: 80%, 92%). Importantly, efficacy was consistent across all major bleed locations including nose, gum/mouth, and gastrointestinal sites, and demonstrated robust activity against both traumatic and spontaneous bleeding events.
    • Reduction of bleeds requiring high intensity treatment: Participants experienced a 100% reduction in mean ATBR of bleeding events requiring high intensity treatment (defined as those requiring recombinant factor VIIa, platelet transfusions, plasma, cryoprecipitate, or medical procedures) during the treatment period. This represents a meaningful reduction of the most clinically consequential acute bleeding events.
    • Dosing schedule optimization: Analyses indicate that weekly dosing provides consistent exposure across the dosing interval, resulting in optimal clinical response.
    • Safety and tolerability: Overall sutacimig was well tolerated. Adverse events were primarily mild to moderate and either non-specific or typical for patients with GT, with a single related serious adverse event (grade 2 DVT) occurring at the highest dose level (0.9 mg/kg). Anti-drug antibodies impacting PK/PD were observed in five participants; however, titers resolved in one participant with continued dosing, and no associated safety events were reported.
    • Retention: Underscoring the perceived benefit, 82% of participants elected to enter the ongoing long-term extension study.

    Presentation Details

    • Title: Sutacimig, a Novel Bispecific Antibody for Prophylactic Treatment of Glanzmann Thrombasthenia: Analysis of a Phase 2 Study
    • Session: OCCC – W304EFGH
    • Presenter: Paul Saultier, MD, PhD, APHM Hospital de la Timone, France

    *Data as of July 1, 2025.

    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 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 1-2-5™, targets building a pipeline of development programs to deliver long-awaited innovation for people 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:
    Deerfield
    Peg Rusconi
    [email protected]

    Investors:
    Hemab Therapeutics
    Mads Behrndt
    [email protected]

    SOURCE Hemab Therapeutics


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  • Jessie Buckley on Golden Globes ‘Hamnet’ Scene and Unscripted Finale

    Jessie Buckley on Golden Globes ‘Hamnet’ Scene and Unscripted Finale

    [This story contains spoilers from Hamnet.]

    In Hamnet, Jessie Buckley plays a real historical figure, Agnes or Anne Hathaway, known as the wife of William Shakespeare. 

    But rather than delving into the historical research,…

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