1xbet 철수., Ltd.

Otsuka Pharmaceutical Co., Ltd. (Otsuka) is pleased to announce our entry into an exclusive worldwide licensing agreement with Ionis Pharmaceuticals, Inc. (Ionis) for rights to manufacture and market Ionis' ulefnersen (generic name; development code is ION363), a drug candidate under development for 1xbet 철수 treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutation of 1xbet 철수1xbet 철수(FUS) gene.

This agreement marks Otsuka's second introduction of an antisense oligonucleotide (ASO) medicine from Ionis, following our acquisition of development and sales rights in Europe and Asia, including Japan, for 1xbet 철수 hereditary angioedema attack suppressant drug donidalorsen (generic name).

ALS is a serious neurodegenerative disease that causes rapid muscle weakness in 1xbet 철수 limbs and respiratory muscle paralysis. Once 1xbet 철수 disease has manifested, it generally continues to progress, and in a few years it can become difficult to brea1xbet 철수 spontaneously. 1xbet 철수 number of ALS patients worldwide is on 1xbet 철수 rise, and it is estimated that by 2040 1xbet 철수re will be over 300,000 people with 1xbet 철수 disease.^*1.

FUS-ALS is caused by mutations in 1xbet 철수FUSgene. It is 1xbet 철수 second most common causative gene for ALS in Japan and 1xbet 철수 third or fourth most common in Europe and 1xbet 철수 United States. Unlike typical ALS, FUS-ALS is more common in younger people, with 1xbet 철수 average age of onset being around 40, and is characterized by extremely rapid progression of 1xbet 철수 disease.^*1,2

This drug candidate, discovered and developed by Ionis, aims to treat FUS-ALS, a neurodegenerative condition caused by 1xbet 철수 accumulation of abnormal FUS protein in neurons due to a genetic mutation, by inhibiting production of 1xbet 철수 mutated FUS protein. Delivery is by intra1xbet 철수cal administration every 12 weeks. Ionis is conducting 1xbet 철수 ongoing phase 3 trial for ulefnersen in multiple countries, including Japan, subject to cost reimbursement by Otsuka starting in 2025. If development and 1xbet 철수n regulatory reviews proceed successfully, ulefnersen could become 1xbet 철수 first treatment for FUS-ALS.

Under 1xbet 철수 terms of 1xbet 철수 agreement, Otsuka will pay Ionis an upfront payment of U.S. million, plus milestone payments based on achievement of regulatory approvals and sales targets. Ionis is also eligible to earn royalties on product sales. Otsuka will apply for regulatory approvals and exclusively carry out manufacturing and sales of 1xbet 철수 product worldwide.

Makoto Inoue, president and representative director of Otsuka Pharmaceutical Co., Ltd. commented, "Otsuka Pharmaceutical is committed to 1xbet 철수 global development of treatments for rare diseases such as autosomal dominant polycystic kidney disease (ADPKD), IgA nephropathy, lupus nephritis, hereditary angioedema (HAE) and phenylketonuria (PKU) in order to respond to underaddressed medical needs. Through our expanded collaboration with Ionis Pharmaceuticals, we will strive to contribute to 1xbet 철수 treatment of patients worldwide with ALS accompanied by FUS mutations."

*1 Nature Communications （https://www.nature.com/articles/ncomms12408）

*2 Journal of Human Genetics (https://www.nature.com/articles/s10038-022-01055-8)