Japan’s Otsuka Pharmaceutical announces the signing of an exclusive worldwide licensing agreement with US-based Ionis Pharmaceuticals for the manufacturing and marketing rights to Ionis’ ulefnersen, a drug candidate in development to treat patients with amyotrophic lateral sclerosis caused by the mutation of the Fused gene in sarcoma (Fus). This agreement - explains the Japanese company – represents the second introduction by Otsuka of an antisense oligonucleotide drug (Aso) from Ionis, after the acquisition of the development and marketing rights in Europe and Asia, including Japan, of donidalorsen, drug for the suppression of hereditary angioedema attacks.
ALS (ALS in the Anglo-Saxon world) is a serious neurodegenerative disease that causes rapid muscle weakness in the limbs and paralysis of the respiratory muscles, recalls a note. The number of ALS patients in the world is increasing and it is indeed estimated that by 2040 there will be over 300 thousand people affected globally.Fus-Als is caused by mutations in the Fus gene, the second most common gene causing ALS in Japan and the third or fourth most common in Europe and the USA. Unlike typical ALS, fus-Als is more common in younger people, with an average age of onset of around 40 years, and is characterized by extremely rapid disease progression. The basis of Fus-Als is the accumulation of abnormal Fus protein in neurons and ulefnersen, discovered and developed by Ionis, aims to treat this form of ALS by inhibiting the production of mutated fus protein. It is taken via intrathecal administration every 12 weeks. Ionis is conducting the Phase 3 study in several countries, including Japan, subject to cost reimbursement from otsuka starting in 2025. If development and subsequent regulatory assessments proceed successfully, ulefnersen could become the first treatment for the Fus-Als.
Under the terms of the agreement, Otsuka will pay Ionis an upfront payment of US$10 million, as well as subsequent payments based on the achievement of regulatory approvals and sales targets. Ionis is also entitled to receive royalties on product sales. Otsuka will apply for regulatory approvals and will exclusively manufacture and sell the product worldwide.
“Otsuka Pharmaceutical is committed to the 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), to respond to little-regarded medical needs. By expanding our collaboration with Ionis Pharmaceuticals, we will strive to help treat patients around the world with ALS associated with Fus mutations.” states Makoto Inoue, President and Representative Director of Otsuka Pharmaceutical Co., Ltd.
“Rare diseases represent unique and complex challenges for both patients and their families. The acquisition of the rights to ulefnersen is a meaningful step forward in our mission to offer innovative therapeutic solutions for people affected by amyotrophic lateral sclerosis caused by the mutation in the Fus gene. Thanks to the collaboration with Ionis Pharmaceuticals, with ulefnersen we are opening a new path and we are proud to lead innovation in RNA-targeted drugs, identifying treatments that can really make a difference in patients’ lives”, he comments Alessandro Lattuada, CEO of Otsuka italia.
How does ulefnersen work to target the genetic mutations responsible for Fus-ALS?
Interview with Dr. Emily Chen, ALS Research Expert
Time.news Editor: Thank you for joining us today, Dr. Chen. We’re discussing Otsuka Pharmaceutical’s recent licensing agreement with Ionis Pharmaceuticals regarding the drug candidate ulefnersen for treating amyotrophic lateral sclerosis (ALS) caused by Fus gene mutations. Can you explain the meaning of this agreement?
Dr. Emily Chen: certainly! This agreement is a pivotal move in the fight against Fus-ALS,a especially aggressive form of ALS. Otsuka’s exclusive licensing rights allow them to manufacture and market ulefnersen globally.This drug represents a beacon of hope for patients who are currently navigating a very challenging prognosis, as it targets the underlying genetic mutation responsible for the disease.
Time.news Editor: You mentioned Fus-ALS being particularly aggressive. Can you elaborate on what makes this form of ALS different from typical ALS?
Dr. Emily Chen: Unlike typical ALS, which often affects older adults, Fus-ALS has an earlier onset, generally around age 40. It also progresses much more rapidly.This rapid progression is due to the accumulation of mutated Fus protein in neurons, which leads to accelerated muscle weakness and paralysis.Understanding these differences underscores the urgent need for targeted therapies like ulefnersen.
Time.news Editor: Ulefnersen is still in Phase 3 clinical trials. What makes you optimistic about its potential approval and effectiveness?
Dr.Emily Chen: The advancement of ulefnersen represents a novel approach in treating ALS—it is an antisense oligonucleotide therapy that directly inhibits the production of the mutated protein.With increasing global patient numbers—expected to reach over 300,000 by 2040—there’s a significant medical need for effective treatments. If the clinical data supports its efficacy and safety, I believe regulatory bodies will be inclined to approve it.
Time.news Editor: Otsuka has made clear its commitment to addressing rare diseases. how does this initiative fit into broader industry trends?
Dr. Emily Chen: There’s a growing trend in the pharmaceutical industry to focus on rare diseases, which often receive less attention and funding. This approach not onyl addresses unmet medical needs but also encourages innovation in treatments. As companies like Otsuka collaborate with specialized firms like Ionis, we’re likely to see more breakthroughs in drug development that can significantly improve patients’ lives.
Time.news Editor: What advice would you give to individuals or families affected by Fus-ALS as they await new treatments like ulefnersen?
Dr. Emily Chen: My primary advice would be to stay informed about developments in research and treatment options. Patients and families may also find support through local ALS organizations, which can provide resources and connect them with clinical trials. Engaging in open discussions with healthcare providers can help manage symptoms and improve quality of life while waiting for new therapies.
Time.news Editor: Thank you, Dr. Chen, for your insights on the implications of Otsuka and Ionis’s agreement for treating Fus-ALS. We appreciate your expertise as we look to the future of rare disease treatments.
Dr. Emily Chen: Thank you for having me! I’m hopeful that discussions like this can raise awareness and ultimately lead to better support and treatment for those affected by ALS.