Nxera Pharma (4565) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic vision and business model

• Focused on structure-based drug discovery using the NxWave platform, leveraging AI and proprietary data to deliver 30 drug candidates in 12 years.

• Japanese commercial business modeled on U.S. go-to-market, targeting specialty and rare diseases with bilingual teams.

• Aims for JPY 50 billion annual revenue and 30%+ operating margins by 2030, with launches from wave one before 2030 and wave two in 2030-2035.

• Commercial business in Japan provides stable revenue, while U.K. research generates milestones and royalties.

• Ongoing business development to add rare and specialty care products to the Japanese portfolio.

Commercial performance and product portfolio

• PIVLAZ holds over 70% market share for aneurysmal subarachnoid hemorrhage in Japan, three and a half years post-launch.

• QUVIVIQ, for insomnia, has seen sales accelerate after the two-week prescription limit was lifted.

• Vamorolone, a next-generation corticosteroid for Duchenne muscular dystrophy, was recently in-licensed and will be rapidly advanced in Japan.

• Vamorolone is expected to improve standard of care due to better safety and tolerability, with a potential launch in 2027 if Phase III is skipped, or 2029 otherwise.

• Acquisition of Idorsia's Japan business and partnership with Shionogi have exceeded expectations, with both products now profitable.

R&D pipeline and partnering strategy

• NxWave platform supports 15 clinical programs with partners across multiple therapeutic areas.

• Portfolio refocused on high-potential oral small molecules for chronic weight management, obesity, and endocrine disorders.

• Two Phase II-ready assets (GPR52 agonist for schizophrenia and EP4 agonist for IBD) are being prepared for competitive partnering.

• Strong data for both assets, with GPR52 showing novel mechanism and EP4 demonstrating gut restriction and target engagement.

• Track record of successful out-licensing and repartnering, with confidence in securing new deals.