RegulationPriority Review (CN), Orphan Drug (JP), Orphan Drug (KR), Orphan Drug (AU), Priority Review (AU), Breakthrough Therapy (US), Orphan Drug (US)

Gene Sequence

Sequence Code 5386H

Source: *****

Sequence Code 5425H

Source: *****

Sequence Code 5467L

Source: *****

Clinical Trials associated with Emicizumab

NCT04563520 / RecruitingPhase 3IIT

aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)

The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

Start Date01 Nov 2024

Sponsor / Collaborator

Emory University

[+1]

CTRI/2024/08/071826 / RecruitingPhase 4IIT

Outcome of Emicizumab Prophylaxis in Patients of Haemophilia A with or without Inhibitor An Early Experience from A Tertiary Care Hospital From Eastern India - NIL

Start Date15 Aug 2024

Sponsor / Collaborator-

NCT05248594 / RecruitingNot ApplicableIIT

Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

We have developed a questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

Start Date01 Aug 2024

Sponsor / Collaborator

Montefiore Medical Center

[+1]

100 Clinical Results associated with Emicizumab

100 Translational Medicine associated with Emicizumab

100 Patents (Medical) associated with Emicizumab

591

Literatures (Medical) associated with Emicizumab

31 Dec 2024·mAbs

A pivotal decade for bispecific antibodies?

Article

Author: Surowka, Marlena ; Klein, Christian

Bispecific antibodies (bsAbs) are a class of antibodies that can mediate novel mechanisms of action compared to monospecific monoclonal antibodies (mAbs). Since the discovery of mAbs and their adoption as therapeutic agents in the 1980s and 1990s, the development of bsAbs has held substantial appeal. Nevertheless, only three bsAbs (catumaxomab, blinatumomab, emicizumab) were approved through the end of 2020. However, since then, 11 bsAbs received regulatory agency approvals, of which nine (amivantamab, tebentafusp, mosunetuzumab, cadonilimab, teclistamab, glofitamab, epcoritamab, talquetamab, elranatamab) were approved for the treatment of cancer and two (faricimab, ozoralizumab) in non-oncology indications. Notably, of the 13 currently approved bsAbs, two, emicizumab and faricimab, have achieved blockbuster status, showing the promise of this novel class of therapeutics. In the 2020s, the approval of additional bsAbs can be expected in hematological malignancies, solid tumors and non-oncology indications, establishing bsAbs as essential part of the therapeutic armamentarium.

01 Dec 2024·PEDIATRIC BLOOD & CANCER

CHIEF: A retrospective self‐control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis

Article

Author: Malvar, Jemily ; Carneiro, Kendall ; Mashiach, Daniel ; Young, Guy ; Mead, Patrice ; Knight, Susan

Abstract:

Background:

Hemophilia A (HA) is an X‐linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2–6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard‐of‐care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non‐inhibitor SHA children.

Methods:

Using a retrospective electronic medical record chart review, we conducted a self‐control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.

Results:

The mean ABR on CFC and emicizumab was 1.79 and 1.13 (p = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; p = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; p = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (p < .001).

Conclusion:

Emicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.

01 Dec 2024·Blood Research

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Article

Author: Cappuccilli, Maria ; Leogrande, Melania ; Perrone, Valentina ; Esposti, Luca Degli

Abstract:

Purpose:

This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.

Methods:

From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.

Results:

Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.

Conclusion:

These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

100

News (Medical) associated with Emicizumab

05 Dec 2024

·www.globenewswire.com

FDA accepts supplemental Biologics License Application for Roche’s Columvi combination for people with relapsed or refractory diffuse large B-cell lymphoma

Application is based on data from the phase III STARGLO study where Columvi plus chemotherapy showed a statistically significant and clinically meaningful improvement in overall survival1,2 This regimen could provide an off-the-shelf, fixed-duration treatment option for patients to start soon after diagnosis, which is important for those who are at high-risk of disease progressionImproving survival outcomes is needed for people with an aggressive disease like relapsed or refractory DLBCL, especially those who aren’t eligible for transplant3 Basel, 5 December 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Columvi® (glofitamab) in combination with gemcitabine and oxaliplatin (GemOx) for the treatment of people with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least one prior line of therapy and are not candidates for autologous stem cell transplant. The FDA is expected to make a decision on approval by 20 July 2025. The standard second-line therapy for R/R DLBCL patients has historically been high-dose chemotherapy followed by stem-cell transplant, however, not all patients are a candidate due to age or coexisting medical conditions. While newer therapies are becoming available, barriers remain for many and alternative treatment options are needed for these patients to improve survival outcomes.3 “For people with aggressive lymphomas like DLBCL, timely intervention with effective therapies can be crucial to reduce the risk of disease progression and improve long-term outcomes,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “We are encouraged by the overall survival benefit seen with this Columvi combination and hope it can become an important treatment option for those who are in need of alternative therapies.” The sBLA is based on results from the phase III STARGLO study, which were presented at the European Hematology Association Congress earlier this year and recently published in The Lancet.1,2 Data showed Columvi in combination with GemOx demonstrated a statistically significant and clinically meaningful overall survival (OS) improvement versus MabThera®/Rituxan® (rituximab) and GemOx (R-GemOx), making it the first CD20xCD3 bispecific antibody to show a survival benefit in DLBCL in a randomised phase III trial.1,2 Safety of the combination appeared consistent with the known safety profiles of the individual medicines.1,2 Data from the STARGLO study have been submitted to other health authorities around the world for approval consideration, including the European Medicines Agency. Columvi is part of Roche’s industry-leading CD20xCD3 bispecific antibody programme, which has seen more than 3,000 patients treated in clinical trials and more than 2,600 treated in clinical practice to date.4 Columvi was the first fixed-duration bispecific antibody to receive accelerated approval by the U.S. FDA and conditional marketing authorisation in the EU as a monotherapy to treat people with R/R DLBCL after two or more lines of systemic therapy and is currently approved in more than 50 countries around the world. As part of Roche’s efforts to elevate treatment standards in the earlier stages of DLBCL, where there is the best opportunity to improve long-term outcomes and prevent relapse, Columvi is also being investigated in combination with Polivy® (polatuzumab vedotin), MabThera/Rituxan, cyclophosphamide, doxorubicin and prednisone (R-CHP) in previously untreated DLBCL in the phase III SKYGLO study. About the STARGLO study5The STARGLO study [GO41944; NCT04408638] is a phase III, multicentre, open-label, randomised study evaluating the efficacy and safety of Columvi® (glofitamab) in combination with gemcitabine plus oxaliplatin (GemOx) versus MabThera®/Rituxan® (rituximab) in combination with GemOx (R-GemOx) in patients with relapsed or refractory diffuse large B-cell lymphoma who have received at least one prior line of therapy and who are not candidates for autologous stem cell transplant, or who have received two or more prior lines of therapy. Preclinical research indicated an increased antitumour effect when combining Columvi with GemOx over GemOx alone, so the STARGLO study was initiated to further explore the potential complementary effects of the treatment combination. Outcome measures include overall survival (OS; primary endpoint), progression-free survival, complete response rate, objective response rate, duration of objective response (secondary endpoints), and safety and tolerability. In the primary analysis (conducted after a median follow-up of 11.3 months) patients treated with Columvi plus GemOx lived significantly longer, with a 41% reduction in the risk of death (hazard ratio [HR]=0.59, 95% CI: 0.40-0.89, p=0.011) versus R-GemOx.1,2 Median OS was not reached with the Columvi regimen versus nine months for R-GemOx.1,2 Safety of the combination appeared consistent with the known safety profiles of the individual medicines.1,2 Adverse event (AE) rates were higher with the Columvi combination versus R-GemOx, noting higher median number of cycles received with the Columvi combination (11 versus 4).1,2 One of the most common AEs was cytokine release syndrome, which was generally low grade (Any Grade: 44.2%, Grade 1: 31.4%, Grade 2: 10.5%, Grade 3: 2.3%) and occurred primarily in Cycle 1.1,2 STARGLO is intended as a confirmatory study to convert the accelerated approval of Columvi in the US and conditional marketing authorisation in the EU to full approvals for people with R/R DLBCL after two or more lines of systemic therapy based on the pivotal phase I/II NP30179 study [NCT03075696]. About Columvi® (glofitamab)Columvi is a CD20xCD3 T-cell engaging bispecific antibody designed to target CD3 on the surface of T-cells and CD20 on the surface of B-cells. Columvi was designed with a novel 2:1 structural format. This T-cell engaging bispecific antibody is engineered to have one region that binds to CD3, a protein on T-cells, a type of immune cell, and two regions that bind to CD20, a protein on B-cells, which can be healthy or malignant. This dual-targeting brings the T-cell in close proximity to the B-cell, activating the release of cancer cell-killing proteins from the T-cell. Columvi is part of Roche’s broad and industry-leading CD20xCD3 T-cell-engaging bispecific antibody clinical development programme that also includes Lunsumio® (mosunetuzumab), which aims to provide tailored treatment options that suit the diverse needs, preferences, and experiences of people with blood cancers and healthcare systems. Roche is investigating Columvi as a monotherapy and in combination with other medicines for the treatment of diffuse large B-cell lymphoma and mantle cell lymphoma. About diffuse large B-cell lymphoma (DLBCL)DLBCL is the most common form of non-Hodgkin lymphoma (NHL), accounting for about one in three cases of NHL.6 DLBCL is an aggressive (fast-growing) type of NHL.6 While it is generally responsive to treatment in the frontline, as many as 40% of people will relapse or have refractory disease, at which time salvage therapy options are limited and survival is short.7 Improving treatments earlier in the course of the disease and providing much needed alternative options could help to improve long-term outcomes. Approximately 160,000 people worldwide are diagnosed with DLBCL each year.6,8 About Roche in haematologyRoche has been developing medicines for people with malignant and non-malignant blood diseases for more than 25 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), Polivy® (polatuzumab vedotin), Venclexta®/Venclyxto® (venetoclax) in collaboration with AbbVie, Hemlibra® (emicizumab), PiaSky® (crovalimab), Lunsumio® (mosunetuzumab) and Columvi® (glofitamab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibody cevostamab, targeting both FcRH5 and CD3 and Tecentriq® (atezolizumab). Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law.References[1] Abramson J, et al. Glofitamab plus Gemcitabine and Oxaliplatin (Glofit-GemOx) for Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL): Results of a Global Randomized Phase III trial (STARGLO). Presented at: EHA Hybrid Congress; 2024 Jun 3-16. Abstract #LB3438.[2] Abramson J, et al. Glofitamab plus gemcitabine and oxaliplatin (GemOx) versus rituximab-GemOx for relapsed or refractory diffuse large B-cell lymphoma (STARGLO): a global phase 3, randomised, open-label trial. Lancet 2024; 404 (10466): 1940-1954.[3] Gisselbrecht C and Van Den Neste E. How I manage patients with relapsed/refractory diffuse large B cell lymphoma. Br J Haematol 2018; 182(5): 633–643.[4] Roche data on file.[5] ClinicalTrials.gov. A Phase III Study Evaluating Glofitamab in Combination With Gemcitabine + Oxaliplatin vs Rituximab in Combination With Gemcitabine + Oxaliplatin in Participants With Relapsed/Refractory Diffuse Large B-Cell Lymphoma: NCT04408638 [Internet; cited 2024 November]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT04408638.[6] UpToDate. Patient education: Diffuse large B cell lymphoma in adults (Beyond the Basics). [Internet; cited 2024 November]. Available from: https://www.uptodate.com/contents/diffuse-large-b-cell-lymphoma-in-adults-beyond-the-basics. [7] Sehn LH, et al. Diffuse Large B-Cell Lymphoma. N Engl J Med. 2021;384(9):842-858.[8] World Health Organization. Numbers derived from GLOBOCAN 2022. Non-Hodgkin Lymphoma Factsheet [Internet; cited 2024 November]. Available from: https://gco.iarc.who.int/media/globocan/factsheets/cancers/34-non-hodgkin-lymphoma-fact-sheet.pdf. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhDPhone: +41 79 407 72 58Sileia UrechPhone: +41 79 935 81 48Nathalie AltermattPhone: +41 79 771 05 25Lorena CorfasPhone: +41 79 568 24 95Simon GoldsboroughPhone: +44 797 32 72 915Karsten KleinePhone: +41 79 461 86 83Nina MählitzPhone: +41 79 327 54 74Kirti PandeyPhone: +49 172 6367262Yvette PetillonPhone: +41 79 961 92 50Dr Rebekka SchnellPhone: +41 79 205 27 03 Roche Investor Relations Dr Bruno EschliPhone: +41 61 68-75284e-mail: bruno.eschli@roche.comDr Sabine BorngräberPhone: +41 61 68-88027e-mail: sabine.borngraeber@roche.comDr Birgit MasjostPhone: +41 61 68-84814e-mail: birgit.masjost@roche.com Investor Relations North America Loren KalmPhone: +1 650 225 3217e-mail: kalm.loren@gene.com Attachment 05122024_MR_BLA for Columvi_en

Phase 3Clinical ResultDrug ApprovalImmunotherapyAccelerated Approval

26 Nov 2024

·medcitynews.com

Roche Expands Its Scope in Allogeneic Cell Therapy With $1B Poseida Therapeutics Acquisition - MedCity News

Roche, already partnered with Poseida Therapeutics in the development of off-the-shelf cell therapies for blood cancers, is broadening its ambitions with the modality through a deal to acquire the biotech for $1 billion up front. Applying allogeneic cell therapies to solid tumors and immunological indications are among the goals Roche aims to achieve by buying San Diego-based Poseida. According to deal terms announced Tuesday, Roche will pay $9 in cash for all shares of the biotech. That price represents a 215% premium to Posedia’s closing stock price on Monday. When Poseida went public in 2020, it priced shares at $16 apiece. In addition to the upfront payment, the Swiss pharma giant has agreed to pay out up to $4 more in cash per share if certain programs achieve specified goals. These contingent value rights payments could bring the deal’s value to $1.5 billion. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News Poseida is a clinical-stage developer of cell and gene therapies. It’s part of a contingent of companies aiming to advance on the first cell therapies, which are made from a patient’s own immune cells in a lengthy and expensive manufacturing process. The cells in Poseida’s therapies are sourced from healthy donors and engineered with the company’s non-viral technologies. Such allogeneic cell therapies could lower manufacturing costs and shorten production timelines compared to the manufacturing of autologous cell therapies. In 2022, Roche paid Poseida $110 million up front for rights or options to the biotech’s blood cancer programs. Another $6 billion was tied to the achievement of milestones. Included in the deal were P-BCMA-ALLO1, an allogeneic cell therapy that targets the cancer protein BCMA to treat multiple myeloma, and P-CD19CD20-ALLO1, an allogeneic dual cell therapy that targets the proteins CD19 and CD20 to treat B-cell malignancies. Both are currently in the clinic and preclinical and early clinical data for these programs are set for presentation next month during the annual meeting of the American Society of Hematology. Roche likes what it has seen from Poseida’s research so far. “We are very encouraged by the early clinical data, and this acquisition builds on our joint progress to catalyze the development of potentially first and best-in-class cell therapies in oncology, immunology and neurology,” Levi Garraway, head of product development and chief medical officer at Roche, said in a prepared statement. Regarding immunological indications, Poseida recently submitted applications seeking FDA permission to begin clinical trials testing P-CD19CD20-ALLO1 in multiple sclerosis and systemic lupus erythematosus. Poseida’s research of non-viral delivery of genetic medicines to the liver is preclinical. One of these programs, P-FVIII-101, is an in vivo gene therapy in development for hemophilia A. Roche has a presence in this market through Hemlibra, a bispecific antibody for hemophilia A that has become a blockbuster product. Health IT Reducing Clinical and Staff Burnout with AI Automation As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers. By Dr. Michael Blackman, Chief Medical Officer at Greenway Health In a note sent to investors, William Blair analyst Sami Corwin said Roche and Poseida make for a strategic fit, given the pharma giant’s presence as an oncology leader, its investment in building a cell therapy manufacturing facility, and the potential synergies of Hemlibra with Poseida’s hemophilia A gene therapy. But the big payout to Poseida shareholders is tied to the more advanced allogeneic cell therapies. The contingent value rights outlined in the deal call for payment of $2 per share in cash when the BCMA-targeting therapy starts a pivotal study in any indication by Dec. 31, 2028. A $1 per share payment is tied to the start of a pivotal study of either the BCMA-targeting therapy or the CD19- and CD20-targeting therapy for an autoimmune disease by Dec. 31, 2034. The final contingency payment is related to a commercial milestone. Poseida shareholders will receive $1 per share when P-BCMA-ALLO1 records its first sale as a treatment for any indication by Dec. 31, 2031. Corwin said the first contingency payment will likely be achieved, given that the BCMA-targeting therapy is currently in Phase 1/2 testing and has generated encouraging clinical data, suggesting it will begin a pivotal study by the end of 2028. But the second contingency payment is higher risk given that neither candidate has reached the clinic in autoimmune disease. That said, William Blair believes 10 years is sufficient to advance from preclinical to pivotal testing if the early clinical studies are successful. Corwin said the third contingency payment is de-risked based on that program’s stage of development; William Blair models a commercial launch in 2028. Poseida was previously partnered with Takeda Pharmaceutical on the development of non-viral gene therapies. The Japanese pharma giant terminated the pact, effective July 30, 2023. Soon after, Astellas Pharma made a $50 million investment in Poseida. Under the terms of that agreement, Astellas has the right to be notified if Poseida reaches a merger deal with another company. Astellas also has the right of first refusal to make a competing offer. This right expires 18 months following the closing date of the agreement, which would be Feb. 4, 2025. The boards of both Roche and Poseida have already approved the acquisition agreement. The companies expect to complete the transaction in the first quarter of 2025.

Cell TherapyAcquisitionGene TherapyASH

06 Nov 2024

·endpts.com

Novo unveils new cardiometabolic data as it reports solid third-quarter profits

Novo Nordisk disclosed several key updates from its cardiometabolic and hematology pipelines Wednesday as the drugmaker revealed its third-quarter profits edged out expectations. First up, the Danish pharma giant said semaglutide met the primary endpoint of a Phase 3b trial in people with peripheral arterial disease (PAD) and type 2 diabetes. At 52 weeks, 13% of people given semaglutide saw a “statistically significant” improvement in their maximum walking distance compared with placebo, according to a Wednesday release . Novo said it plans to file for approval of Ozempic in PAD in the first half of next year. Also in the diabetes space, the company’s DNA immunotherapy candidate has a safe and well-tolerated profile based on a Phase 1 test in August. The treatment is designed to preserve the body’s natural production of insulin and thus slow down or stop type 1 diabetes in people at high risk of developing it. The company has yet to spell out what it plans to do with the asset. On the hematology side, the Danish drugmaker said it is gearing up to file for approval for its hemophilia A prophylactic therapy next year. The treatment, dubbed Mim8, led to better annualized bleeding rates in a Phase 3b trial comparing it with standard-of-care emicizumab. Novo also said it terminated overall development of its mineralocorticoid receptor antagonist, named ocedurenone, after stopping a Phase 3 trial in chronic kidney disease earlier this year. The trial came to an end in June after it was deemed unable to meet its endpoints. Elsewhere, the Danish drugmaker’s Q3 performance appears to be better than Eli Lilly’s, with net profits coming in just ahead of the Wall Street consensus of DKK 27 billion ($3.9 billion). By contrast, Lilly’s total Q3 revenues of $11.4 billion missed its $12.2 billion consensus forecast. Novo’s shares $NVO rallied as much as 4% premarket Wednesday. The drugmaker narrowed its expectations for 2024 sales growth to between 23% and 27% from an original range of 22% to 28%. The revision comes after the company trimmed its guidance for profit growth in the second quarter. Both Novo and Lilly are facing competitive pressures from companies making compounded versions of their blockbuster diabetes and weight loss drugs. But Novo, during its earnings call with the media, underscored safety issues with these compounded drugs. “We have been looking at a number of compounded products in the market and identified multiple safety concerns and also noted more than 100 hospitalizations and 10 deaths based on compounded semaglutides,” CFO Karsten Munk Knudsen said. Last month, a trade group of compounding pharmacies sued the FDA for taking Eli Lilly’s tirzepatide off its shortage list despite some evidence that the drug was still in short supply.

Phase 3Clinical ResultImmunotherapyPhase 1Financial Statement

100 Deals associated with Emicizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D10821	Emicizumab	B02BX06	DB13923

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Coagulation Protein Disorders	JP	Chugai Pharmaceutical Co., Ltd.	23 Mar 2018
Hemophilia A	US	Genentech, Inc.	16 Nov 2017

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Factor 8 Deficiency, Acquired	Phase 2	AT	Hoffmann-La Roche, Inc.	23 Mar 2021
Factor 8 Deficiency, Acquired	Phase 2	DE	Hoffmann-La Roche, Inc.	23 Mar 2021
Von Willebrand Disease, Type 3	Phase 1	US	Genentech, Inc.	01 Nov 2022
Hemophilia	Phase 1	CN	F. Hoffmann-La Roche Ltd.	12 Mar 2018
Hemophilia	Phase 1	CN	Roche China Holding Ltd.	12 Mar 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Hemophilia A	-	Emicizumab	crynbrazzr(dusoimkhmn) = ocpghzhsoo sjrqsvobdd (wwhkvxxvej, 1.1) View more	-	09 Dec 2024
NCT04431726 (HAVEN 7, ASH2024)	Phase 3	Hemophilia A FVIII inhibitors	55	Emicizumab	jliokelzob(phfehwqjhc) = cuyxgmbrtk yomsrekkex (bbcfzosjnu )	Positive	08 Dec 2024
				Factor VIII	jliokelzob(phfehwqjhc) = vjdrdubdfl yomsrekkex (bbcfzosjnu )
ASH2024	Not Applicable	-	-	Emicizumab	ukvctlmeyh(dtymzkkbzc) = iohtvwoybp gkztcmpodt (gbiyxofzpu ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Hemophilia A	-	Emicizumab	ktynggoele(gfmdlbnszh) = ehuxpegbea xdnieipfxi (sliobvwgrw )	-	07 Dec 2024
				Comparators	ktynggoele(gfmdlbnszh) = wqsvelortq xdnieipfxi (sliobvwgrw )
NCT04431726 (HAVEN 7, CTgov)	Phase 3	Hemophilia A	55	Emicizumab	rgdvklzees(ognaepmwgx) = mqdwkkesnw hnsctsrblp (xqweqmmsnx, dgflrkbabs - qdtrksuqgc) View more	-	14 Jun 2024
EHA2024	Not Applicable	-	25	octocog alfa	ctzkthsuaw(rnuncikfgo) = vavpxbhtxv bvfmhzonjs (vxsibnsrtp, 38)	-	14 May 2024
				emicizumab	ctzkthsuaw(rnuncikfgo) = ctsjktfnsc bvfmhzonjs (vxsibnsrtp, 9)
EHA2024	Not Applicable	Hemophilia A	16	Emicizumab	xxigiqeglu(mvojoikrtx) = efrxhtzptw ccqfrqhnzi (ddpbzilyvl )	Negative	14 May 2024
				FVIII replacement	xxigiqeglu(mvojoikrtx) = kzuxadnwth ccqfrqhnzi (ddpbzilyvl )
NCT03191799 (STASEY, Pubmed \| TH Open) Manual	Phase 3	Hemophilia A	56	Emicizumab prophylaxis with additional hemostatic medication	kxfrngeevk(gxeivuytrg) = tfbbxvqmpd mumbhroyly (qigeteifxd ) View more	Positive	12 Jan 2024
				Emicizumab prophylaxis without additional hemostatic medication	kxfrngeevk(gxeivuytrg) = foqyakvxsn mumbhroyly (qigeteifxd )
ASH2023	Not Applicable	Hemophilia A	-	Emicizumab	msdgswjnlt(puitigupcj) = sbaylekzdv iyaodbmdct (cdftwwapjl )	-	11 Dec 2023
				Efanesoctocog alfa	msdgswjnlt(puitigupcj) = tapmmzwlvb iyaodbmdct (cdftwwapjl )
ASH2023	Not Applicable	Factor 8 Deficiency, Acquired	-	Emicizumab	jrcspozyzl(avscrvxwlk) = xmsxqnyabx kvqdvsgqrk (odvmhiygul ) View more	Positive	10 Dec 2023
				Immunosuppressive therapy (IST)	jrcspozyzl(avscrvxwlk) = oaqblpskbq kvqdvsgqrk (odvmhiygul ) View more

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