Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Start Date26 Jun 2023

Sponsor / Collaborator

Novo Nordisk A/S

NCT05685238

/ Active, not recruitingPhase 3

Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors

This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Start Date13 Feb 2023

Sponsor / Collaborator

Novo Nordisk A/S

NCT05681845

/ CompletedPhase 1

A Study to Demonstrate Bioequivalence of a Single Subcutaneous Administration of NNC0365-3769 (Mim8) With the DV3407-C1 Pen Injector Versus a Syringe and Enhanced Cartridge in Healthy Male Participants

Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe and cartridge. The new pen injector is intended to facilitate the administration of Mim8 for patients with haemophilia A. The participants will get Mim8 as injection under the skin (subcutaneously) of the belly using the DV3407-C1 pen injector and a needle (hereinafter referred to as pen injector) or using a needle and syringe from a cartridge (hereafter called syringe and cartridge). The participants will receive one injection with Mim8, either with the DV3407-C1 pen injector or with a syringe and cartridge. The study participation will last up to 20 weeks. Only healthy men can take part in the study.

Start Date02 Jan 2023

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Denecimig

100 Translational Medicine associated with Denecimig

100 Patents (Medical) associated with Denecimig

Literatures (Medical) associated with Denecimig

31 Dec 2025·mAbs

Antibodies to watch in 2025

Review

Author: Kapoor, Vaishali ; Kaplon, Hélène ; Crescioli, Silvia ; Reichert, Janice M. ; Visweswaraiah, Jyothsna ; Wang, Lin

The commercial development of antibody therapeutics is a global enterprise involving thousands of biopharmaceutical firms and supporting service organizations. To date, their combined efforts have resulted in over 200 marketed antibody therapeutics and a pipeline of nearly 1,400 investigational product candidates that are undergoing evaluation in clinical studies as treatments for a wide variety of diseases. Here, we discuss key events in antibody therapeutics development that occurred during 2024 and forecast key events related to the late-stage clinical pipeline that may occur in 2025. In particular, we report on 21 antibody therapeutics granted a first approval in at least one country or region during 2024, including bispecific antibodies tarlatamab (IMDELLTRA®), zanidatamab (Ziihera®), zenocutuzumab (BIZENGRI®), odronextamab (Ordspono®), ivonescimab (®), and antibody-drug conjugate (ADC) sacituzumab tirumotecan (®). We also discuss 30 investigational antibody therapeutics for which marketing applications were undergoing review by at least one regulatory agency, as of our last update on December 9, 2024, including ADCs datopotamab deruxtecan, telisotuzumab vedotin, patritumab deruxtecan, trastuzumab botidotin, becotatug vedotin, and trastuzumab rezetecan. Of 178 antibody therapeutics we include in the late-stage pipeline, we summarize key data for 18 for which marketing applications may be submitted by the end of 2025, such as bi- or multispecific antibodies denecimig, sonelokimab, erfonrilimab, and anbenitamab. Key trends in the development and approval of antibody formats such as bispecifics and ADCs, as well as clinical-phase transition and global approval success rates for these antibody formats, are reported.

01 Sep 2025·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Model-informed drug development of Mim8 – a next-generation bispecific antibody for treatment of haemophilia A

Article

Author: Kreilgaard, Mads ; Persson, Paula ; Matytsina, Irina

BACKGROUND:

Haemophilia A is a congenital bleeding disorder caused by deficiency of clotting factor VIII (FVIII). Mim8 (denecimig) is a next-generation activated FVIII mimetic bispecific antibody under phase 3 investigation for subcutaneous management of haemophilia A with or without FVIII inhibitors. Mim8 uses tiered dosing, which is a novel dosing approach in haemophilia where patients receive a fixed dose according to which body weight range they fall into and their chosen dosing frequency, reducing the need for dose calculations and potentially reducing treatment wastage.

OBJECTIVES:

This study aimed to establish the early population pharmacokinetics (PK) and PK/pharmacodynamic (PD) properties of Mim8 using both biomarker and clinical endpoint data. Models were used to inform drug-development decisions using trial simulations and transform the dosing strategy in haemophilia to a novel simplified treatment option using tiered dosing in paediatric and adult populations.

PATIENTS/METHODS:

Data from 129 participants exposed to Mim8 in the phase 1 PK trial (NCT0512747) and the FRONTIER1 trial (NCT04204408) were used to develop population PK and PK/PD models for thrombin generation and treated bleeds. The models were used for trial simulations to optimise dosing regimens evaluating Mim8 exposure, biomarker and clinical endpoint targets on a population level (paediatric and adolescent/adult) and on a typical subject level for the range of body weight (BW) in the trial. Clinical endpoint targets were set to: (1) at least 70 % of patients without treated bleeds in a 6-month trial on a population level and >95 % reduction in relative bleed risk on a typical subject level across all weight ranges and dosing frequencies, and (2) sustained peak Mim8 exposure below highest exposure observed in phase 1/2 trials.

RESULTS:

The population PK of Mim8 were best described with a structural two-compartment model with baseline BW as the most significant covariate for Mim8 exposure. Both peak thrombin response and Mim8 effect on bleed risk were best described with a direct link PK/PD model with an E_max response. Model-estimated EC₅₀ was 2.03 µg/mL for peak thrombin and 0.07 µg/mL for bleed risk. Using trial simulations, tiered dosing in three weight ranges (<15, 15-<45, and ≥45 kg) was identified to reach the clinical endpoint targets and maximum desired exposure for all three dosing frequencies: once every week, once every 2 weeks, and once every month. This was done with an initial one-time loading dose establishing steady-state conditions within the first day, followed by maintenance doses at the desired dosing frequency. Loading and maintenance doses for the nine combinations of weight ranges and dosing frequencies were covered by five dose presentations. Ongoing phase 3 trials have been based on the dosing strategy presented here.

CONCLUSIONS:

Model-informed drug development was used to design a novel treatment paradigm for patients living with haemophilia A by utilising a tiered-dosing strategy with Mim8, to be evaluated in phase 3 trials.

01 Jul 2025·THROMBOSIS RESEARCH

In vitro effects of Mim8 and combined Mim8-bypassing therapy on thrombin generation, thromboelastography and fibrin clot ultrastructure

Article

Author: Dargaud, G Yesim ; Nougier, Christophe ; Lund, Jacob ; Josset, Laurie ; Rezigue, Hamdi

INTRODUCTION:

Mim8, a fully human bispecific IgG4 antibody, FVIIIa mimetic, which emerges as a promising prophylactic treatment option for patients with hemophilia A (HA).

AIM:

This study investigates the in vitro hemostatic activity of Mim8 in blood samples from six patients with severe hemophilia A using a thrombin generation assay (TGA) and thromboelastography. The results were compared to those obtained with emicizumab.

MATERIALS AND METHODS:

TG was assessed in both platelet-poor (PPP) and platelet-rich plasma (PRP). Fibrin clots formed during TG were collected, and clot ultrastructure was examined using scanning electron microscopy (SEM). Whole blood samples were analyzed using ROTEM-NATEM.

RESULTS:

Mim8 significantly improved the TG capacity of severe HA plasma samples. Mim8 6 μg/mL demonstrated higher TG capacity compared to emicizumab 50 μg/mL. In PRP samples fortified with Mim8 6 μg/mL and rFVIIa 90 μg/kg together, ETP levels were fully normalized. No synergistic effect was observed when FVIII concentrate was combined with Mim8 at therapeutic doses due to the higher affinity of FVIII for FIXa and FX compared to Mim8. ROTEM-NATEM in whole blood confirmed the TGA results. SEM showed a more robust fibrin clot structure with thinner fibrin fibers in the presence of Mim8 compared to emicizumab.

CONCLUSIONS:

Mim8 significantly improves TG in vitro in both PPP and PRP from patients with severe HA, with ETP levels comparable to those of FVIII at 100 IU/dL. The TGA can effectively monitor the combined treatment with Mim8 and either rFVIIa or APCC, which is not possible with currently available routine laboratory tests.

News (Medical) associated with Denecimig

05 Dec 2025

·www.biopharmadive.com

Hemophilia gene therapies are struggling on the market, even as innovation soars

Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from the market while two are fighting an uphill battle for reimbursement and a solid footing, according to Michael Recht, chief medical and scientific officer for the National Bleeding Disorders Foundation.Analysts predicted Biomarin Pharmaceuticals hemophilia A gene therapy Roctavian would hit peak sales of $2.2 billion. Instead, Biomarin is now looking to offload the drug due to weak commercial uptake.In a similar position, Pfizer pulled its hemophilia B treatmentBeqvez from the market while CSL Behrings Hemgenix, which was first to market, is also significantly underperforming.Dismal sales came as a surprise to many who saw hemophilia gene therapies as a major scientific leap forward and a promising one-time alternative that could free patients from frequent clotting factor infusions or injections.But gene therapies come with a high price tag. Hemgenix became the worlds most expensive drug when it was approved in 2022, with a list price of $3.5 million per dose. Still, treatment could be worth the sticker shock, and CSL estimates that Hemgenix could save up to $5.8 million per person by eliminating the need for once- or twice-weekly clotting factor injections.Its a value-laden intervention, Recht said. Its just shockingly expensive.Reimbursement hurdlesPayers have balked at the bill for gene therapy treatments, although Recht said all of his interested patients have been able to get them.As part of an advocacy organization, when weve spoken to the payers, what we've heard is, Well, the average time someone is on a specific health plan, since the Affordable Care Act has been around, is under two years. So, why should we pay for lifetime therapy when that person might be on our plan for under two years? Recht said.Still, an NBDF data analysis found that rapid plan hopping is not as common as insurers claim. Since the introduction of the Affordable Care Act, people typically only change plans when they change jobs, he said.Im very hopeful there will be products to address the unmet needs of the inheritable bleeding disorders population.Dr. Michael RechtChief medical and scientific officer, NBDFBeyond reimbursement, structural disincentives further complicate adoption. The Hemophilia Treatment Center 340B Factor Program offers certified organizationsdiscounts for medications and supplies. The centers can then reinvest those savings back into facility programs.The money for gene therapy, however, typically gets paid to a hospital, according to Recht.So the hospital would get all the income from the gene therapy, and the treatment center could lose $500,000 to $750,000 a year in revenues from someone on prophylaxis.This discourages partaking in gene therapy, he added.To me, that is not a compelling reason at all, and potentially an unethical reason if this is the right drug [for a patient], Recht said. But this is what I've heard from some of my colleagues.Making the gene therapy leapWhile gene therapy isnt the best option for all patients, it could be for a subset. But many can afford to take a wait-and-see approach because other treatments are effective.There are some really great non-gene therapy options for people with hemophilia A, Recht said. [Hemlibra] is a great drug, and has changed the way Ive treated people.Many patients also dont qualify for treatment. Hemgenix targets hemophilia B, which makes up only about 15% of overall cases, and some patients, including children and those with certain liver conditions, are excluded.For Beqvez, uptake obstacles proved too difficult to overcome.Beqvez was a perfectly good product, Recht said. But as the second to market with the results of their Phase 3 maybe not as robust as the Hemgenix clinical trial, I think that played a big role.Roctavian, a hemophilia A option, targets a broader pool of patients, but has also faced challenges.The durability is the biggest issue, Recht said, noting that its still uncertain how long the treatment will last, although recent data showed it still worked after five years. To get 10 years of not having hemophilia, that's still 10 years of not having hemophilia.The markets futureDespite the market struggles, the future for hemophilia patients is promising.I'm very hopeful there will be products to address the unmet needs of the inheritable bleeding disorders population, Recht said.Novo Nordisk, for example, recently submitted anapplication for Mim8, a drug that Recht predicts could be as safe and effective as Hemlibra, but potentially more appealing to patients because it uses a pen injector.Interest in gene therapy hasnt waned, and companies are still looking for a way in. Be Biopharma is in Phase 1 with BE-101, a cell therapy with a twist.Theyre putting the vector into a persons own B cells, Recht said. The fascinating part about this is if this is safe and it works, it will be re-dosable because you wont have an immune response like you do with an adeno-associated virus vector.Startups are also testing gene editing for hemophilia. However, many of these treatments are years from reaching patients.While determining the best uses for all the existing medications remains a challenge, there are more options than ever.Where we are today in the 30-ish years of my career is a miracle, he said. '

Gene TherapyPhase 3Phase 1Cell Therapy

03 Dec 2025

·www.prnewswire.com

Hemophilia A Market to Exhibit Growth at a CAGR of 2.9% During the Forecast Period (2025-2034) Owing to the Rising Hemophilia Prevalence, Strong Uptake of New Drugs, and Emergence of High-cost Gene Therapies | DelveInsight

The dynamics of the hemophilia A market are expected to change mainly due to the increase in prevalence and the launch of upcoming therapies such as Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others during the forecast period. LAS VEGAS, Dec. 3, 2025 /PRNewswire/ -- DelveInsight's Hemophilia A Market Insights report includes a comprehensive understanding of current treatment practices, hemophilia A emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophilia A Market Summary The market size for hemophilia A was found to be USD 12.2 billion in the leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] in 2024. The United States accounted for the largest hemophilia A treatment market size, approximately 48% of the total market size in the 7MM in 2024, compared to other major markets, including the EU4 countries, the United Kingdom, and Japan. HEMLIBRA's growth curve (US Q1 2025 year-over-year sales) in the United States was flat, indicating increased competition from newer therapies. The total number of diagnosed prevalent cases of Hemophilia A in the 7MM was around 49,500 in 2024. Leading hemophilia A companies, such as Novo Nordisk, Ultragenyx Pharmaceutical, Sangamo Therapeutics, ASC Therapeutics, Roche, Chugai, and others, are developing new hemophilia A treatment drugs that can be available in the hemophilia A market in the coming years. The promising hemophilia A therapies in clinical trials include Mim8, DTX201 (Peboctocogene camaparvovec, BAY2599023), Giroctocogene fitelparvovec (SB-525), ASC618, NXT007/RG6512, and others. Discover the new hemophilia A treatment @ Hemophilia A Treatment Market Key Factors Driving the Growth of the Hemophilia A Market Rising Hemophilia A Target Patient Pool The diagnosed prevalence of Hemophilia A is expected to rise from 49K in 2024 to 50K by 2034. Improved diagnostic capabilities and increased disease awareness mainly drive this increase in cases. Advancements in Gene Therapy Gene therapy has emerged as a transformative approach in hemophilia A treatment. Notably, BioMarin's ROCTAVIAN has been approved in the U.S. and EU, offering a one-time infusion that provides long-term correction of the factor VIII deficiency. Pfizer's gene therapy giroctocogene fitelparvovec for hemophilia A has also demonstrated promising results in late-stage trials, significantly reducing bleeding episodes and outperforming traditional treatments. Regulatory Approvals and Hemophilia A Market Expansion Recent regulatory approvals have bolstered the availability of hemophilia A treatments. For instance, the U.S. FDA approved Pfizer's once-weekly injection, HYMPAVZI (in October 2024), for hemophilia A patients aged 12 and above, aiming to prevent or reduce bleeding episodes. Similarly, Sanofi's QFITLIA (in March 2025), a subcutaneous treatment administered every two months, was approved for patients with Hemophilia A or B aged 12 and older. Emergence of Non-Factor Hemophilia A Therapies Expanding beyond traditional therapies, hemophilia A care now includes two cutting-edge non-factor approaches: anti-TFPI therapies (Pfizer's HYMPAVZI and Novo Nordisk's ALHEMO) and siRNA therapies (Sanofi's QFITLIA). Robust Hemophilia A Clinical Trial Activity Several hemophilia A companies are actively developing emerging therapies, including Novo Nordisk (Mim8, ALHEMO), Sangamo Therapeutics (Giroctocogene fitelparvovec), Roche/Chugai (NXT007), ASC Therapeutics (ASC-618), Ultragenyx (DTX201), and others. Hemophilia A Market Analysis The primary goal of current treatment strategies for hemophilia A is to minimize complications caused by bleeding into joints, tissues, or organs. With ongoing technological advancements and improved medical understanding, patients now have access to a range of therapeutic options. Treatment is typically administered either "on-demand" or as "prophylaxis", with prophylactic therapy increasingly becoming the preferred approach. The U.S. FDA has approved several recombinant Factor VIII products for hemophilia A management, including HELIXATE FS, RECOMBINATE, KOGENATE FS, ADVATE, REFACTO, ELOCTATE, NUWIQ, ADYNOVATE, KOVALTRY, JIVI, and XYNTHA. Additionally, plasma-derived Factor VIII products, such as MONARC-M, MONOCLATE-P, HEMOFIL M, and KOATE-DVI, remain available. The latest addition, QFITLIA (fitusiran), a siRNA-based therapy approved in 2025, works by lowering antithrombin levels to enhance clot formation. It offers the convenience of only six injections per year and is effective for both inhibitor and non-inhibitor patients. HEMLIBRA has emerged as a first-line prophylactic therapy for Hemophilia A patients with inhibitors, although immune tolerance induction (ITI) therapy continues to be the gold standard. For those who encounter difficulties with ITI, HEMLIBRA provides an alternative, requiring less frequent FVIII dosing during ITI. Looking ahead, hemophilia management is shifting toward extended half-life factor therapies and cutting-edge modalities, including siRNA agents, bispecific antibodies, and gene therapy. The competitive landscape is expected to expand with the introduction of new long-acting factor products and next-generation therapeutic technologies. Hemophilia A Competitive Landscape The hemophilia A clinical trial landscape features several drugs in mid- and late-stage development that are expected to be approved in the near future. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others. Novo Nordisk's Mim8 is an advanced FVIIIa-mimicking bispecific antibody designed to provide sustained hemostatic control with convenient once-weekly or once-monthly prophylactic dosing for individuals with Hemophilia A, regardless of inhibitor status. Delivered subcutaneously, Mim8 functions by bridging activated Factors IXa and X (FIXa/FX) upon activation, effectively compensating for the absence of FVIII. This restores normal thrombin generation and promotes effective blood clotting. Ultragenyx Pharmaceutical's DTX201 represents the first clinical-stage gene therapy for Hemophilia A utilizing an Adeno-associated Virus (AAV) vector derived from the AAVhu37 serotype. It is a non-replicating AAV vector carrying a single-stranded DNA genome that encodes a B-domain–deleted FVIII, driven by a liver-specific promoter and enhancer sequence optimized for strong transgene expression. The AAVhu37 capsid, part of the hepatotropic clade E family, was chosen for its demonstrated ability in preclinical studies to enable efficient liver-targeted FVIII gene delivery, optimal biodistribution, and long-lasting FVIII expression. The anticipated launch of these emerging therapies are poised to transform the hemophilia A market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hemophilia A market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about the FDA approved gene therapy hemophilia A @ FDA Approved AAV Gene Therapy Hemophilia A Recent Developments in the Hemophilia A Market In July 2025, Novo Nordisk announced that the FDA has approved ALHEMO injection for once-daily prophylactic use to prevent or reduce bleeding episodes in adults and children aged 12 years and older with hemophilia A or B (HA/HB) without inhibitors. This approval broadens the drug's indication beyond its initial December 2024 approval for patients with HA/HB with inhibitors. In June 2025, Pfizer Inc. reported positive topline findings from its Phase 3 BASIS trial (NCT03938792), which evaluated HYMPAVZI (marstacimab) in adults and adolescents with hemophilia A or B who have inhibitors. In May 2025, the FDA approved Antihemophilic factor (recombinant), PEGylated-aucl (Jivi; Bayer) for treating pediatric patients aged 7 and older with hemophilia A. The approval was supported by results from the Alfa-PROTECT and PROTECT Kids trials. In March 2025, Alnylam Pharmaceuticals announced the FDA's approval of Qfitlia™ (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S. It is the first and only treatment to lower antithrombin (AT), aiming to promote thrombin generation, rebalance hemostasis, and prevent bleeds. What is Hemophilia A? Hemophilia A is an inherited bleeding disorder caused by a deficiency or low levels of clotting factor VIII. For many years, the standard treatment has been replacement therapy with FVIII. This began with FVIII obtained from donated whole blood, later advanced to plasma-derived FVIII, and is now primarily managed with recombinant human FVIII (rFVIII) products that have transformed hemophilia care. While the condition is typically diagnosed at birth, it can also be acquired later in life when the immune system develops antibodies that neutralize clotting factors, a rare condition known as acquired hemophilia. Hemophilia A Epidemiology Segmentation The hemophilia A epidemiology forecast section provides insights into the historical and current hemophilia A patient pool and forecasted trends for the leading markets. In the 7MM, the highest diagnosed prevalent cases of Hemophilia A were in the United States, accounting for nearly 14,900 cases in 2024. These cases are anticipated to increase in the upcoming years. The hemophilia A treatment market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into: Total Diagnosed Prevalent Cases of Hemophilia A Diagnosed Prevalent Cases of Hemophilia A with Inhibitors or without Inhibitors Age-Specific Diagnosed Prevalent Cases of Hemophilia A Severity-specific Diagnosed Prevalent Cases of Hemophilia A Scope of the Hemophilia A Market Report Therapeutic Assessment: Hemophilia A current marketed and emerging therapies Hemophilia A Market Dynamics: Key Market Forecast Assumptions of Emerging Hemophilia A Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Hemophilia A Market Access and Reimbursement Download the report to evaluate the hemophilia A treatment company HEMLIBRA on hemophilia in women @ Royal Hemophilia A or B Table of Contents Related Reports Hemophilia A Clinical Trial Analysis Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Hemophilia A companies, including Jiangsu Gensciences Inc., Takeda, Gritgen Therapeutics Co., Ltd., Suzhou Alphamab Co., Ltd., Novo Nordisk, Pfizer, SK Plasma Co., Ltd., Hoffmann-La Roche, Shanghai Belief-Delivery BioMed Co., Ltd, ASC Therapeutics, Biocad, Expression Therapeutics, LLC, GCBiopharma, Amarna Therapeutics, Cabaletta Bio, among others. Acquired Hemophilia A Market Acquired Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key acquired hemophilia A companies, including Pfizer, Novo Nordisk, Hema Biologics, Hoffman-La-Roche, Takeda, among others. Hemophilia Market Hemophilia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia companies, including Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, among others. Hemophilia B Market Hemophilia B Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, UniQure Biopharma B.V., ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo - SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo

Drug ApprovalPhase 3Clinical ResultImmunotherapy

29 Sep 2025

·www.prnewswire.com

Novo Nordisk submits Biologics License Application (BLA) to FDA for Mim8; an investigational, prophylaxis treatment for people living with hemophilia A with or without inhibitors

If approved, Mim8 (denecimig) would offer the flexibility of treatment with a single-use, prefilled disposable pen injector with dosing options including once every month, once every two weeks, and once every week for people living with hemophilia A In hemophilia A, denecimig is designed to help the body form blood clots more effectively and was studied as part of the FRONTIER program across different dosing frequencies, age groups, severities, and with or without inhibitors1 Novo Nordisk is advancing its long-standing commitment to people living with hemophilia A, a potentially life-threatening condition BAGSVÆRD, Denmark and PLAINSBORO, N.J., Sept. 29, 2025 /PRNewswire/ -- Today, Novo Nordisk announced Mim8 (denecimig) has been submitted for review to the US Food and Drug Administration (FDA) through a Biologics License Application (BLA), a formal request to evaluate a biologic medicine. Denecimig is an investigational, next-generation bispecific antibody Factor VIIIa (FVIIIa) mimetic, designed as a preventive bleed routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital FVIIIa deficiency), with or without inhibitors.2 If approved, denecimig will be the first FVIIIa mimetic with flexible once every month, once every two weeks, and once every week dosing in a pre-filled, single-use pen, catering to the diverse needs of people living with hemophilia A, with or without inhibitors.1 "It is important that we continue to drive scientific advances in hemophilia A, and the FRONTIER program supports the potential of denecimig as a prophylaxis bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity, regardless of inhibitor status," said Anna Windle, Senior Vice President, Clinical Development, Medical & Regulatory Affairs at Novo Nordisk. "A cornerstone of hemophilia treatment is to help prevent bleeding episodes, and this submission is another step forward in our long-standing mission to help improve care. If approved, denecimig's single-dose prefilled injector pen and flexible dosing schedule may better fit into the lives of people living with this challenging condition." The FDA submission is grounded in the results from the FRONTIER study program, a comprehensive program of studies designed to establish the efficacy and safety profile of denecimig as a prophylaxis treatment to prevent or reduce the frequency of bleeding episodes in people with hemophilia A, with or without inhibitors.1,3-6 About Mim8 (denecimig) Mim8 (denecimig) is an investigational FVIIIa mimetic bispecific antibody designed with the aim to deliver once every month, once every two weeks, or once every week prophylaxis for people living with hemophilia A, with or without inhibitors.3-6 Denecimig, which is administered under the skin, "mimics" the role of FVIIIa by bridging Factor IXa and Factor X.7 This action replaces FVIIIa function, which helps restore the body's thrombin generation capacity, helping blood to clot.8 The use of denecimig in people living with hemophilia A is investigational and not approved by any regulatory authorities worldwide. About Hemophilia A Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.9 According to the World Federation of Hemophilia, it is estimated to affect approximately 836,000 people worldwide and hemophilia A is estimated to account for 80-85% of all hemophilia cases.10 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.6 Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII).9 Some people with hemophilia A may develop inhibitors, an immune system response to the clotting factors used in replacement therapy, which can cause treatment to become ineffective.11 It has been estimated that approximately 30% of people living with hemophilia A have inhibitors.11 About the FRONTIER Clinical Program The FRONTIER clinical program includes FRONTIER1-5 and investigates denecimig as a preventive bleed treatment across pediatric and adult populations with hemophilia A, with or without inhibitors. 1,3-5,12,13 FRONTIER2, FRONTIER3 and FRONTIER4 formed the basis of the denecimig BLA submission. FRONTIER2 evaluated denecimig treatment once every month and once every week in adults and adolescents 12 years of age and older; FRONTIER3 evaluated denecimig treatment once every month and once every week in children below the age of 12; and FRONTIER4 was an open label extension trial evaluating the efficacy of denecimig once every two weeks (Q2W) as well as investigating the long-term safety of denecimig across all dosing regimens (once every month, once every two weeks, and once every week) in subjects with hemophilia A, with or without inhibitors.3-5 Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 78,400 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn, and YouTube. Contacts for further information References Novo Nordisk Data on File. Bowyer AE, Hickey K, Kitchen S, Ezban M. A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non-factor VIII related haemostasis assays. Haemophilia. 2023;29(6):1633-1637. ClinicalTrials.gov. A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4). Last accessed September 2025. Available at . Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138(14):1258-1268. Lentz S, Chowedary P, Gil L, et al. FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic. J Thromb Haemost. 2024;22(4): 990-1000. U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed August 2025. Available at . MedlinePlus. Hemophilia. Last accessed September 2025. Available at . World Federation of Hemophilia. World Federation of Hemophilia Report on the Annual Global Survey 2023. Last accessed September 2025. Available at . Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209. ClinicalTrials.gov. A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1). Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5). Last accessed September 2025. Available at . Novo Nordisk is a registered trademark of Novo Nordisk A/S. All other trademarks, registered or unregistered, are the property of their respective owners. © 2025 Novo Nordisk All rights reserved. US25HRBD00364 September 2025 Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical Result

100 Deals associated with Denecimig

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia A	NDA/BLA	United States	Novo Nordisk A/S	30 Sep 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04204408 (FRONTIER1, Pubmed \| Res Pract Thromb Haemost)	Phase 1/2	Hemophilia A	41	Mim8 maintenance doses	azmzrgxoci(cekhowntyp) = wnedzahkgj rbpdsfjunn (yxjldtqphj ) View more	Positive	01 Oct 2025
FRONTIER3 (GlobeNewswire) Manual	Phase 3	Hemophilia A	70	Mim8	kgesfyuqip(adgrunppdp) = xlgonxdzjp yxpwmsuyga (qtofliolbo ) View more	Positive	07 Feb 2025
NCT05685238 (FRONTIER4, ASH2024)	-	Hemophilia A	37	Mim8	rgkqevqupl(iqadyjjvzh) = gyjqslszhq dmdatuwvrj (umthnyyncr )	Positive	09 Dec 2024
NCT05053139 (Frontier 2, Corporate Publications) Manual	Phase 3	Hemophilia A	254	Mim8 once-weekly prophylaxis (no prior prophylaxis treatment)	yilwunwdkw(adjkagedas) = pvubcdqdid vfbtctofjk (jorzqskzbj ) View more	Positive	23 Jun 2024
NCT05053139 (Frontier 2, Corporate Publications) Manual	Phase 3	Hemophilia A	254	Mim8 once-monthly prophylaxis (no prior prophylaxis treatment)	yilwunwdkw(adjkagedas) = ckbrzljipo vfbtctofjk (jorzqskzbj ) View more	Positive	23 Jun 2024
NCT05053139 (FRONTIER 2, GlobeNewswire) Manual	Phase 3	Hemophilia A	254	Mim8 once-weekly	bonbapcppa(tbywisxthh) = obcmbgswws vaosrxtnlw (wkrwtvkrpl ) Met View more	Positive	13 May 2024
NCT05053139 (FRONTIER 2, GlobeNewswire) Manual	Phase 3	Hemophilia A	254	Mim8 once-monthly	bonbapcppa(tbywisxthh) = ahzbihnvfy vaosrxtnlw (wkrwtvkrpl ) Met View more	Positive	13 May 2024
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER 1, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Hemophilia A	42	Mim 8	htmadgbggt(symiovjnzc) = drftglagui nejtuqcjqh (idyoohcbvp ) View more	Positive	15 Nov 2022
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER1, ISTH2022) Manual	Phase 1/2	-	-	Mim 8 (SAD phase)	yjvbshlytu(wxgjvowasp) = Mim8 was well tolerated following both single and multiple dosing, and no thromboembolic events or related serious adverse events were reported msvrzmapot (gufgsonwox )	Positive	11 Jul 2022
	Phase 1/2	-	-	Mim 8 (MAD phase)		Positive	11 Jul 2022
NCT04204408 (FRONTIER1, ISTH2022)	Phase 1/2	Hemophilia A	-	Mim8	gclklalcbn(zhzopcrvzr) = xmobwykqps zxnbimtobr (vwneucdmtg ) View more	-	09 Jul 2022

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis