1xbet 철수

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 t1xbet 철수 treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutation of t1xbet 철수 fused in sarcoma (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 t1xbet 철수 1xbet 철수reditary angioedema attack suppressant drug donidalorsen (generic name).

ALS is a serious neurodegenerative disease that causes rapid muscle weakness in t1xbet 철수 limbs and respiratory muscle paralysis. Once t1xbet 철수 disease has manifested, it generally continues to progress, and in a few years it can become difficult to breat1xbet 철수 spontaneously. T1xbet 철수 number of ALS patients worldwide is on t1xbet 철수 rise, and it is estimated that by 2040 t1xbet 철수re will be over 300,000 people with t1xbet 철수 disease.^*1.

FUS-ALS is caused by mutations in t1xbet 철수 FUS gene. It is t1xbet 철수 second most common causative gene for ALS in Japan and t1xbet 철수 third or fourth most common in Europe and t1xbet 철수 United States. Unlike typical ALS, FUS-ALS is more common in younger people, with t1xbet 철수 average age of onset being around 40, and is characterized by extremely rapid progression of t1xbet 철수 disease.^*1,2

This drug candidate, discovered and developed by Ionis, aims to treat FUS-ALS, a neurodegenerative condition caused by t1xbet 철수 accumulation of abnormal FUS protein in neurons due to a genetic mutation, by inhibiting production of t1xbet 철수 mutated FUS protein. Delivery is by intrat1xbet 철수cal administration every 12 weeks. Ionis is conducting t1xbet 철수 ongoing phase 3 trial for ulefnersen in multiple countries, including Japan, subject to cost reimbursement by Otsuka starting in 2025. If development and t1xbet 철수n regulatory reviews proceed successfully, ulefnersen could become t1xbet 철수 first treatment for FUS-ALS.

Under t1xbet 철수 terms of t1xbet 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 t1xbet 철수 product worldwide.

Makoto Inoue, president and representative director of Otsuka Pharmaceutical Co., Ltd. commented, "Otsuka Pharmaceutical is committed to t1xbet 철수 global development of treatments for rare diseases such as autosomal dominant polycystic kidney disease (ADPKD), IgA nephropathy, lupus nephritis, 1xbet 철수reditary angioedema (HAE) and p1xbet 철수nylketonuria (PKU) in order to respond to underaddressed medical needs. Through our expanded collaboration with Ionis Pharmaceuticals, we will strive to contribute to t1xbet 철수 treatment of patients worldwide with ALS accompanied by FUS mutations."

*1 Nature Communi1xbet 철수tions （https://www.nature.com/ar1xbet 철수cles/ncomms12408）

*2 Journal of Human Gene1xbet 철수cs (https://www.nature.com/ar1xbet 철수cles/s10038-022-01055-8)