The agreement grants Nxera exclusive development and commercialization rights for the investigational oral Fabry disease treatment in Japan, South Korea, Taiwan and selected other Asia-Pacific markets, excluding China.
Nxera Pharma Co., Ltd. (Nxera) has exercised its option to license lucerastat from Idorsia Ltd. (Idorsia), entering into a co-development and commercialization agreement to advance the investigational treatment for Fabry disease across selected Asia-Pacific markets.
Under the agreement, Nxera will obtain exclusive rights to develop and commercialize lucerastat in Japan, South Korea, Taiwan and selected other Asia-Pacific territories, excluding China.
Financial Terms of the Agreement
Key financial and development terms include:
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Upfront Payment: Nxera will pay Idorsia CHF 7 million, in accordance with the pre-agreed option terms.
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Development Costs: Nxera will contribute to the global Phase 3 development programme.
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Royalties: Idorsia will be eligible to receive royalties on net sales generated within the licensed territories.
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Regional Responsibilities: Nxera will participate in the comparative Phase 3 kidney function study and manage clinical development activities in Japan.
Idorsia originally granted Nxera the licensing option in July 2023 as part of the acquisition of Idorsia's Asia-Pacific (excluding China) operations by Sosei Heptares, now known as Nxera Pharma.
Lucerastat: An Investigational Oral Treatment for Fabry Disease
Lucerastat is an investigational oral substrate reduction therapy (SRT) designed to treat Fabry disease independently of:
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Alpha-galactosidase A enzyme activity.
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GLA gene mutation status.
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Previous treatment with enzyme replacement therapy (ERT).
The drug works by inhibiting glucosylceramide synthase, an enzyme involved in the production of glycosphingolipids, including globotriaosylceramide (Gb3). By reducing Gb3 production, lucerastat aims to limit the accumulation of this fatty substance in cells and potentially slow disease progression.
Fabry disease is a rare, inherited X-linked lysosomal storage disorder caused by mutations in the GLA gene. The resulting deficiency or absence of alpha-galactosidase A leads to Gb3 accumulation, progressively affecting multiple organs, particularly the kidneys, heart and nervous system.
Phase 3 Clinical Development and Key Findings
Lucerastat has been evaluated in the 118-patient Phase 3 MODIFY study, which investigated its efficacy and safety in patients with Fabry disease.
Key findings reported include:
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Biomarker Reduction: Lucerastat significantly reduced plasma and urinary Gb3 levels compared with placebo.
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Kidney Function: An interim analysis of the open-label extension indicated a slower rate of kidney function decline compared with the two years before treatment.
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Long-Term Treatment: Some patients have received lucerastat for more than six years.
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Safety: The treatment was reported to be well tolerated during long-term use.
In the interim analysis of the open-label extension, the estimated eGFR decline was:
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Assessment Period |
eGFR Slope |
|---|---|
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During lucerastat treatment |
-1.64 |
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Historical period before treatment |
-3.50 |
Note: The primary endpoint of reducing neuropathic pain in the MODIFY study was not met.
Global Phase 3 Development Programme
Idorsia's global Phase 3 programme includes two key studies:
1. FAB-KLEAR Study: A kidney biopsy study involving 16 adult male patients to evaluate the treatment's effects on Fabry disease-related kidney pathology.
2. Comparative Kidney Function Study: A 74-patient study comparing lucerastat with standard-of-care enzyme replacement therapies, focusing on kidney function.
The development programme has been aligned with the US Food and Drug Administration (FDA) on a streamlined registration strategy and incorporates feedback received from the European Medicines Agency (EMA).
Nxera will participate in the comparative kidney function study and oversee clinical development activities in Japan.
Management Commentary
Christopher Cargill, President and CEO of Nxera Pharma, highlighted the clinical evidence generated so far and the potential of lucerastat as a treatment option for people living with Fabry disease. He noted that the agreement adds a late-stage rare disease asset to Nxera's growing portfolio in Japan and across the Asia-Pacific region.
Amer Joseph, MD, Chief Medical Officer and Head of Global Clinical Development at Idorsia, emphasized the sustained biomarker reductions and promising signals of slower kidney function decline observed in the clinical programme. He also acknowledged that the primary endpoint of neuropathic pain reduction was not achieved and highlighted the need for baseline-controlled data to support regulatory registration.
Fabry Disease Market Outlook
Fabry disease is frequently underdiagnosed or misdiagnosed, creating challenges in timely diagnosis and treatment.
According to DelveInsight estimates:
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Approximately 16,000 patients were diagnosed with Fabry disease across seven major markets (the US, EU4, UK and Japan) in 2020.
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The diagnosed patient population is projected to increase to approximately 21,000 by 2034.
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The global Fabry disease market is projected to reach approximately US$4 billion by 2034.
What This Agreement Means for the Companies
The agreement strengthens Nxera Pharma's presence in the rare disease segment and expands its development and commercialization opportunities across key Asia-Pacific markets.
For Idorsia, the partnership provides regional development support, an upfront payment and potential royalty revenue while enabling continued advancement of lucerastat through its global Phase 3 programme.
With Phase 3 development underway, lucerastat represents a potential new oral treatment approach for Fabry disease, with further clinical and regulatory data needed to establish its efficacy and support future approvals.
