Risk Factors Update Summary

• Litigation update: Answered invalidity contentions in March 2023. No trial date set.
• Expanded orphan drug designations for pralsetinib in various cancer treatments, including metastatic RET fusion-positive NSCLC and thyroid cancer.
• The company exited early-stage research to focus resources on mid to late-stage programs.
• The FDA approved REZLIDHIA capsules for adult patients with IDH1 mutation AML.
• Expanded commercialization of GAVRETO (pralsetinib) in the US with a purchase price of $15.0 million.
• Kissei launched TAVALISSE in Japan and obtained national reimbursement in February 2023.
• An additional reduction in workforce was made, primarily in the research organization.
• The FDA approved the continuation of the Phase 2 trial for R289 in lower-risk MDS.
• Olutasidenib was designated as an orphan drug for AML, providing market exclusivity until September 2031.
• Medison Israel made its first commercial sale of TAVALISSE and received national reimbursement.
• Risks associated with financial institution failures impacting liquidity and business operations.
• Expanded product liability claims risk due to safety issues post-litigation by customers.
• Increased focus on commercializing products globally, with potential risks due to evolving global tensions.
• Potential delays in regulatory submissions or product development due to evolving FDA standards and clinical trial requirements.
• Increased net product sales of REZLIDHIA (olutasidenib) from $0.9 million to $10.6 million.
• Knight submitted MAA for regulatory approval in Mexico, Colombia, and Brazil for fostamatinib.
• Emphasis on the importance of market acceptance and factors influencing the commercial potential of products.
• Concerns about shareholder activism and private securities-related litigation disrupting business operations.
• Strategic Collaboration Agreement with MD Anderson for the evaluation of REZLIDHIA in AML and other hematologic cancers.
• The FDA approved REZLIDHIA capsules for R/R AML, adding a second commercial product.
• Regulatory changes: New Windsor Framework impacts UK market from January 1, 2025.
• The DSMB recommended ceasing enrollment in the fostamatinib study arm due to low likelihood of benefits.
• Entered into an Asset Purchase Agreement with Blueprint to purchase GAVRETO assets.
• Growing emphasis on ESG factors impacting investor decisions and corporate responsibility ratings.
• Phase 2 clinical trial with CONNECT to evaluate REZLIDHIA in combination with temozolomide for high-grade glioma.
• Financial impact: Loss from operations increased to $20.5 million in 2023.
• Debt update: $60.0 million term loan balance as of December 31, 2023.
• The company may expand clinical testing efforts beyond current product candidates.
• Entry into a Global Strategic Partnership with Lilly for the development of R552, a RIPK1 inhibitor.
• Regulatory compliance: New SEC Cyber Rule effective from September 5, 2023.
• Completion of enrollment in the FORWARD study for fostamatinib in wAIHA.
• The company faces risks related to privacy laws, including the CPRA and EU GDPR.
• Commercialization license agreement with Grifols for exclusive rights to fostamatinib in Europe/Turkey.
• Market access: New International Recognition Procedure for Great Britain launched on January 1, 2024.
• The company is subject to evolving cybersecurity requirements and potential breaches.