Oral decitabine and cedazuridine (ASTX727) granted Orphan Drug Designation (ODD) by the European Commission for the treatment of Acute Myeloid Leukaemia (AML)

Oral decitabine and cedazuridine (ASTX727) granted Orphan Drug Designation (ODD) by the European Commission for the treatment of Acute Myeloid Leukaemia (AML)

LONDON, UK, 6^th January 2022 – Otsuka Pharmaceutical Europe Ltd. (“Otsuka”) and Astex Pharmaceuticals, Inc. (“Astex”) announce today that the European Commission (EC) has granted orphan-drug designation (ODD) to the oral fixed dose combination of decitabine and cedazuridine (ASTX727) for the treatment of Acute Myeloid Leukaemia (AML)¹.

ODD is granted by the EC to medicinal products intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and which affects fewer than five in 10,000 people in the European Union (EU)². The EC incentivises companies to develop medicines that provide significant benefit to those affected by rare conditions. ODD-granted therapies, such as ASTX727, entitle companies to 10 years of market exclusivity once the product is approved in the EU, among other benefits². The ODD initiative plays a key role in facilitating and encouraging the development of these important medicines to potentially improve the lives of the 30 million people in the EU suffering from a rare disease².

“The granting of ODD is a key and important step in the journey toward finding a new treatment option for patients with AML”, said Andy Hodge, CEO of Otsuka Pharmaceutical Europe Ltd. “We will continue to collaborate with all key stakeholders, including the EC, to make this treatment available to patients in need.”

Harold Keer MD, PhD, Chief Medical Officer of Astex Pharmaceuticals, Inc. said “AML continues to be a challenging disease area with high rates of relapse and low rates of five-year survival. The disease has a higher incidence rate in people aged over 60 years, which presents an important challenge as the population ages. The granting of ODD signifies that ASTX727 is considered to be a medicine that may potentially benefit those affected by this rare, life-threatening condition.”

About ASTX727

ASTX727 is currently not approved for any indication in Europe. ASTX727 is an orally administered, fixed-dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine³, an inhibitor of cytidine deaminase (CDA)^{4, 5} . By inhibiting CDA in the gut and the liver, ASTX727 is designed to allow for oral delivery of decitabine over five days in a given cycle⁶. The phase 1 and phase 2 clinical study results have been published in Lancet Haematology and Blood, respectively^{6, 7}.

Indications

ASTX727 (oral decitabine and cedazuridine) is approved under the brand name INQOVI^® in the U.S and Canada for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anaemia, refractory anaemia with ringed sideroblasts, refractory anaemia with excess blasts, and chronic myelomonocytic leukaemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups^8,9. INQOVI is approved in Australia for the treatment of adult patients with MDS intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups, and patients with CMML¹⁰.

Commercialisation of INQOVI in the U.S. and Canada is conducted by Taiho Oncology, Inc. and Taiho Pharma Canada, Inc., respectively. Astex, Otsuka and Taiho are all members of the Otsuka group of companies.

In Europe, ASTX727 is currently being evaluated within a phase 3 trial (EudraCT Number: 2018-003395-12) for the treatment of Acute Myeloid Leukemia (AML)¹¹.

About Acute Myeloid Leukaemia (AML)

AML is the most common form of acute leukaemia in adults¹². The median age at diagnosis is approximately 70 years¹³. Within Europe, there is an increase in incidence of AML; this may be attributed to the ageing population: AML incidence in Europe has risen from 3.48 in 1976 to 5.06 patients per 100,000 people in 2013¹³. Across Europe and all age groups, AML is notably more common in males than it is in females¹³. The outlook for patients diagnosed with AML has improved over time due to improved care and treatment, however between the years of 2000 and 2007, five-year survival for patients over 65 years-old was 17%¹³. After two courses of intensive induction therapy, 10 to 20% of younger and 50% of older AML patients do not achieve complete response (CR). Of those who obtain CR, 50-70% of these patients will relapse. Following relapse, prognosis for the patients is poor and treatment is challenging, with few therapeutic options available¹³.

About Astex

Astex Pharmaceuticals, Inc. is committed to the fight against cancer. Astex is developing a proprietary pipeline of novel therapies for the treatment of solid tumours and haematological malignancies. In October 2013 Astex became a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Tokyo, Japan.

About Otsuka

Otsuka Pharmaceutical is a global healthcare company with the corporate philosophy:

“Otsuka-people creating new products for better health worldwide.” Otsuka researches, develops, manufactures and markets innovative products, focusing on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health. In pharmaceuticals, Otsuka is a leader in the challenging area of mental health and also has research programs in several under-addressed diseases including tuberculosis, a significant global public health issue.

Otsuka Europe employs approximately 500 people and focuses on psychiatric and neurologic disorders, infectious disease, nephrology, oncology, and digital medicines. Otsuka Pharmaceutical Europe Ltd. is a part of Otsuka Pharmaceutical Company, Ltd., a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan.

The Otsuka group of companies employed 47,000 people worldwide with consolidated sales of approximately €11.7 billion and a spend of €1.8 billion on research and development in 2020.

For further information on Otsuka Pharmaceutical Europe Ltd., please visit www.otsuka-europe.com.

Otsuka, the Otsuka logo, Astex, the Astex logo, and INQOVI are registered trademarks of Otsuka Holdings Co., Ltd. or its subsidiaries.

MEDIA CONTACTS:
OTSUKA PHARMACEUTICAL EUROPE LTD                                                                   

Alison Ross                                                                

Otsuka Pharmaceutical Europe Ltd.               

+44 (0)776 833 7128 (mobile)                                    

ARoss@Otsuka-Europe.com