On January 11, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported an update on the progress of its continued transformation and growth today at the virtual 39th Annual J.P. Morgan Healthcare Conference (Press release, Takeda, JAN 11, 2021, View Source [SID1234573854]). President and Chief Executive Officer, Christophe Weber, shared details on Takeda’s portfolio and pipeline strategy and financial outlook, including key programs expected to contribute to the company’s revenue growth over the next decade.

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"In 2020, Takeda demonstrated the resilience of our business model, the depth of our portfolio and the commitment of our employees as we continued to serve patients and communities globally while overcoming challenges posed by the COVID-19 pandemic," said Christophe Weber, Takeda president and chief executive officer. "As a values-based and R&D-driven biopharmaceutical company celebrating 240 years in 2021, we remain focused on bringing life-transforming treatments to patients worldwide by delivering on our highly innovative pipeline and our continued commitment to patients, our people and the planet."

Highly Innovative Pipeline Supports Sustained Growth

Takeda has built a world-class, state-of-the-art, externally-facing R&D engine and has generated an innovative and modality diverse pipeline of approximately 40 clinical-stage new molecular entities (NMEs). Takeda’s pipeline portfolio has the potential to contribute significantly to revenue growth and the company has a goal to reach JPY5 trillion ($47 billion) revenue by FY20301, representing 50% growth from FY2019.

The majority of revenue growth is expected to come from the company’s Wave 1 pipeline, which includes 12 unique NMEs, representing potential best-in-class/first-in-class therapies and its existing 14 global brands.

Takeda’s Wave 1 programs include five that have received a Breakthrough Therapy designation and three that were granted fast track designation by the U.S. Food and Drug Administration (FDA). In addition, one program was designated under the SAKIGAKE Designation System by the Japanese Ministry of Health, Labour and Welfare and another program was the first breakthrough designation granted by the Chinese Food and Drug Administration to a multinational biopharmaceutical company. Twelve pivotal milestones, including five pivotal data readouts, are expected through fiscal year 2022 with additional near-term development milestones expected across all Wave 1 programs.

Beyond the Wave 1 pipeline, Takeda’s research engine, which comprises internal research capabilities and more than 200 active partnerships, is rapidly advancing a steady stream of next-generation therapies in Wave 2 of our pipeline that will provide sustained growth in FY2025 and beyond. These Wave 2 early-clinical and preclinical programs are designed to provide transformative or curative potential for targeted populations with high unmet need across core therapeutic areas. They are based on targets with strong human validation, represent diverse modalities and leverage new platform capabilities in cell therapy, gene therapy and data sciences.

Key Wave 1 Pipeline Assets Have Significant Market Potential

TAK-003
Takeda’s tetravalent dengue vaccine candidate (TAK-003) has the potential to help address the massive global burden of dengue including key priorities for dengue control such as protection of seronegative individuals (persons not previously exposed to dengue) and prevention of hospitalization. TAK-003 is based on a live-attenuated dengue serotype 2 virus, which provides the genetic "backbone" for all four vaccine viruses. The TAK-003 development program includes the pivotal Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES), a double-blind, randomized, placebo-controlled trial evaluating the safety and efficacy of two doses of TAK-003 in the prevention of laboratory-confirmed symptomatic dengue fever of any severity and due to any of the four dengue virus serotypes in children and adolescents. The TIDES trial is continuing, and safety and efficacy will be assessed over a total of four and a half years. Dengue is the fastest spreading mosquito-borne viral disease and was recognized by WHO to be one of the top ten threats to global health in 2019. Approximately half of the world’s population now lives under the threat of dengue, which is estimated to cause 390 million infections each year.

TAK-755
Takeda’s TAK-755 has the potential to be a transformative therapy for thrombotic thrombocytopenic purpura (TTP), a rare and life-threatening clotting disorder defined by low or absent circulating ADAMTS13 activity (an enzyme essential for regulation of normal blood clotting). There is very high unmet need for new therapies for both congenital and immune-mediated TTP, and TAK-755 is the first and only ADAMTS13 replacement therapy currently in development for both types of TTP. If approved, TAK-755 could be the only replacement therapy to rapidly and completely correct ADAMTS13 levels, and positively impact morbidity and mortality. If approved, TAK-755 has the potential to be first-in-class and the therapeutic choice for prophylaxis in congenital TTP, and a best-in-class therapy for the treatment of immune-mediated TTP. TAK-755 could simplify treatment, avoid adverse events related to plasma-derived therapy and plasma exchange, and provide the potential for at-home therapy. Data readouts are currently expected in 2021 for a phase 2 trial in immune-mediated TTP and in 2022 for a pivotal Phase 3 trial in congenital TTP.

TAK-007
Developed in collaboration with the University of Texas MD Anderson Cancer Center, Takeda’s TAK-007 is a chimeric antigen receptor (CAR) natural killer (NK) cell therapy ‘armored’ with IL-15 targeting CD19 positive B-cell malignancies. The vision is for TAK-007 to be an allogeneic therapy, meaning the NK cells are taken from a non-related healthy donor rather than the patient themselves. As a result, TAK-007 has the potential to be manufactured in advance and stored for off-the-shelf-use. MD Anderson’s CAR-NK CD19 is being studied in a phase 1/2 trial in patients with relapsed or refractory non-Hodgkin’s lymphoma (NHL) and chronic lymphocytic leukemia (CLL). In the ongoing phase 1/2 trial, CAR-NK therapy has not been associated with the severe cytokine release syndrome (CRS) or neurotoxicity observed with existing CAR-T cell therapies and has the potential to be administered in an outpatient setting. A TAK-007 pivotal study is expected to begin enrolling patients with relapsed or refractory B-cell malignancies in 2021. There are currently no approved CAR NK therapies and no allogeneic cell therapies approved to treat cancer.

TAK-994 & TAK-925
TAK-994 is the first oral selective orexin 2 receptor agonist to enter clinical development for the treatment of narcolepsy type 1 (NT1), a rare neurologic condition characterized by excessive daytime sleepiness, cataplexy (signs and symptoms of the disease) and is due to a loss of orexin producing neurons. TAK-994 is currently being evaluated in an ongoing Phase 2 clinical trial in narcolepsy (SPARKLE-1501). If approved, TAK-994 may be the first treatment to address the underlying biology of the disease. TAK-925 (IV formulation) has published proof-of-concept data in NT1, narcolepsy type 2 (NT2), and shift work sleep disorder. Data for idiopathic hypersomnia and obstructive sleep apnea will be disclosed in the future.

Financial Strength
Takeda is committed to maintaining investment grade credit ratings and remains on track towards its medium-term target of 2x Net Debt/adjusted EBITDA ratio within the fiscal years 2021 to 2023, with rapid de-leveraging driven by strong cash flow and proceeds from non-core asset divestitures. In the first half of FY2020, Takeda exceeded its $10B non-core asset divestiture target with 11 deals and up to ~$11.6 billion of non-core disposals announced since January 2019, while further de-leveraging in H1 FY2020 led to a 3.7x net debt/adjusted EBITDA ratio at the end of the period.

Takeda has solid growth momentum and potential for accelerated underlying growth over the medium term. Takeda is also on track to achieve our targeted annual run rate of $2.3 billion in cost synergies by the end of FY2021, further supporting margin performance to meet its medium-term underlying core operating profit margin target in the mid-30s. Takeda remains committed to shareholder returns with a well-established dividend policy of 180 yen per share annually.

Slides from the J.P. Morgan Healthcare Conference presentation and a link to the audio webcast can be accessed on Takeda’s website at: View Source