Takeda Announces Positive Topline Results from Phase 2 Study Evaluating Mezagitamab (TAK-079), a Potential Best-in-Class Anti-CD38 Monoclonal Antibody, for Primary Immune Thrombocytopenia

Rhea-AI Summary

Takeda announces positive Phase 2 results for mezagitamab in patients with primary immune thrombocytopenia, showing safety, tolerability, and efficacy. Plans for a global Phase 3 trial in fiscal year 2024. Mezagitamab demonstrates promising platelet response rates and potential disease remission in ITP patients.

Insights

Medical Research Analyst

The recent announcement from Takeda regarding mezagitamab's positive interim Phase 2 results for treating primary immune thrombocytopenia (ITP) is a significant development in the field of immunotherapy. ITP is an autoimmune disorder where the body's immune system attacks and destroys platelets, leading to an increased risk of bleeding. Current treatments focus on platelet count increase and immunosuppression, but mezagitamab offers a novel approach by targeting CD38 expressing cells, which are implicated in the autoimmune process of ITP.

From a medical research perspective, the dose-dependent increase in platelet count and the rapid and sustained response are promising indicators of mezagitamab's potential efficacy. The drug's progression to Phase 3 trials will be closely monitored, as it could represent a new class of disease-modifying treatments for ITP. The Orphan Drug and Fast Track Designations by the FDA underscore the therapeutic potential and the unmet medical need in this space. It is also noteworthy that approximately 20% of ITP patients do not respond adequately to existing therapies, highlighting the importance of this research.

Financial Analyst

The implications of Takeda's announcement for investors center on the company's late-stage pipeline growth, with mezagitamab becoming one of five new molecular entities entering Phase 3 development in fiscal year 2024. The progression of mezagitamab into Phase 3 trials is a significant milestone that can positively influence Takeda's valuation due to the potential market expansion into the ITP therapeutic area. The Fast Track Designation is particularly relevant for investors as it may expedite the review process and reduce the time to market, potentially leading to earlier revenue generation.

However, it's important to note that the results from the Phase 2 trial have been stated to have no impact on the full year consolidated forecast for the fiscal year ending March 31, 2024. This indicates that any potential financial benefits from mezagitamab will likely affect future fiscal periods. Investors should consider the inherent risks of clinical trials and the fact that success in Phase 2 does not guarantee successful outcomes in Phase 3 or regulatory approval.

Market Research Analyst

In the context of market dynamics, the development of mezagitamab by Takeda taps into a niche but growing market for ITP treatments. Given the unmet need for more effective and tolerable treatments, a successful Phase 3 trial could position Takeda as a leader in this space. The market research angle would focus on the potential patient population size, competitive landscape and pricing strategies. With ITP being a rare disease, mezagitamab's pricing will likely reflect orphan drug status, which traditionally commands a premium price point.

Moreover, the global Phase 3 trial indicates Takeda's commitment to broad market entry, which could have a substantial impact on the company's international market share. It is also essential to monitor competitor activities and any parallel advancements in the treatment of ITP that could affect mezagitamab's market position post-approval.

− Pre-Specified Interim Analysis Shows Positive Safety and Efficacy Results; Mezagitamab Safe and Well Tolerated

− Takeda Intends to Initiate a Global Phase 3 Trial of Mezagitamab in Patients with Primary Immune Thrombocytopenia in Fiscal Year 2024

− Late-Stage Pipeline Continues to Gain Momentum With Five New Molecular Entities in Phase 3 Development in Fiscal Year 2024

OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP). Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb) with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells) resulting in their depletion.

The TAK-079-1004 trial (NCT04278924) evaluated three different doses of subcutaneous mezagitamab vs placebo, given once weekly for eight weeks in patients with chronic (more than one year in duration) or persistent (3-12 months in duration) primary ITP. An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results. Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy.

Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024. When mezagitamab advances to Phase 3 in ITP Takeda will have five new molecular entities in Phase 3 development in fiscal year 2024 including TAK-279 for the treatment of psoriasis and psoriatic arthritis, TAK-861 for the treatment of narcolepsy type 1, soticlestat for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, and fazirsiran for the treatment of α1-antitrypsin associated liver disease.

ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening. The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable.^1,2

“These Phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP. There remains considerable unmet need among ITP patients who may not respond or have inadequate response to prior treatment. In addition, some patients who do respond to available therapies struggle to manage side effects or relapse despite treatment,” said Chinwe Ukomadu, Head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda. “We look forward to initiating the Phase 3 trial and presenting these data at an upcoming scientific congress.”

Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP from the U.S. Food and Drug Administration and the program recently received Fast Track Designation.

Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

About Takeda

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience, and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.

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Medical Information

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References

1. Provan D, Donald A, et al. Blood Advances. 2019;26;3(22):3780-3817.
2. Rodeghiero F. International Journal of Hematology. 2023;117:316–33.

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Media Contacts:

Japanese Media

Yuko Yoneyama

yuko.yoneyama@takeda.com

+81 70-2610-6609



U.S. and International Media

Chris Stamm

chris.stamm@takeda.com

+1 617-374-7726

Source: Takeda Pharmaceutical Company Limited

FAQ

What are the positive results announced by Takeda regarding mezagitamab in the Phase 2 study for primary immune thrombocytopenia?

Takeda reported positive topline results from the Phase 2 study, demonstrating safety, tolerability, and efficacy of mezagitamab in patients with persistent or chronic primary immune thrombocytopenia.

What is the plan of Takeda regarding a global Phase 3 trial for mezagitamab in fiscal year 2024?

Takeda intends to initiate a global Phase 3 trial for mezagitamab in patients with primary immune thrombocytopenia in fiscal year 2024.

What is the mechanism of action of mezagitamab in treating primary immune thrombocytopenia?

Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody that depletes CD38 expressing cells, resulting in promising platelet response rates in patients.

What are the key features of ITP, the disease targeted by mezagitamab?

ITP is a rare autoimmune disease characterized by the accelerated destruction of platelets, leading to decreased platelet count and increased risk of bleeding.

What designations has mezagitamab received for the treatment of ITP?

Mezagitamab has received Orphan Drug Designation from the U.S. FDA and Fast Track Designation for the treatment of ITP.