U.S. Food and Drug Administration Grants Breakthrough Therapy Designation to Takeda’s Investigational Compound, TAK-994, an Oral Orexin Agonist in Clinical Development for Narcolepsy Type 1 (NT1)

– If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to TAK-994,¹ its Phase 2 investigational oral orexin agonist, which is designed to selectively target orexin 2 receptors. TAK-994 is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1),² a chronic neurological disorder that alters the sleep-wake cycle.³ EDS is a hallmark symptom of NT1 and is characterized by a person’s inability to stay awake and alert throughout the day, and falling asleep unintentionally or at inappropriate times on a daily basis.^3,4

The FDA’s Breakthrough Therapy Designation process is designed to expedite the development and review of a drug that is intended to treat a serious condition, for which preliminary clinical evidence exists indicating it may demonstrate a substantial improvement over available therapies on at least one clinically significant endpoint.⁵

The TAK-994 BTD was based, in part, on early phase and preliminary clinical data that indicates Takeda’s investigational oral orexin agonist may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients.¹ Currently, TAK-994 is being studied in an ongoing Phase 2 (TAK-994-1501) study.² Data from the completed Phase 2 study will be presented at a future scientific conference after the study’s conclusion.

“Individuals with narcolepsy type 1 suffer from excessive daytime sleepiness, which might mean routinely falling asleep at work or in school. If approved, investigational TAK-994 has the potential to transform the way we currently treat NT1 by addressing the underlying orexin deficiency central to the disease,” said Sarah Sheikh, Head, Neuroscience Therapeutic Area Unit at Takeda. “Orexin plays a critical role in regulating a person's sleep wake cycle, and supports the body's natural wake promoting pathways in the brain.”

Investigational TAK-994, which was discovered at Takeda’s laboratories in Shonan, Japan, part of the open innovation center at the Shonan Health Innovation Park, is the lead candidate in Takeda’s pioneering multi-asset orexin-focused franchise. This designation marks a key milestone in Takeda’s accelerated development efforts to bring this important potential therapy to patients with NT1.

Takeda’s Commitment to Neuroscience

Takeda Neuroscience is driven by the unmet need of patients with neurologic and psychiatric diseases. Our mission is to bring innovative and potentially disease-modifying medicines to these individuals. Our dedication extends beyond our marketed products and research efforts. We are committed to raising awareness for these conditions, building strategic partnerships with both industry and advocacy, educating patients and physicians, and broadening access to therapies.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

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References

1. Data on File. Takeda Pharmaceuticals. 2021.
2. A Study of TAK-994 in Participants With Narcolepsy With or Without Cataplexy (Narcolepsy Type 1 [NT1] or Narcolepsy Type 2 [NT2]). ClinicalTrials.gov. Published April 1, 2021. https://clinicaltrials.gov/ct2/show/NCT04096560
3. Ruoff C, Rye D. The ICSD-3 and DSM-5 guidelines for diagnosing narcolepsy: clinical relevance and practicality. Curr Med Res Opin. 2016;32(10):1611-1622. doi:10.1080/03007995.2016.1208643
4. Brown J, Makker H. An approach to excessive daytime sleepiness in adults. BMJ. 2020;(368):m1047.
5. Breakthrough Therapy. FDA. Published online November 3, 2018. Accessed July 23, 2021. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy

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