EX-99.2 3 a52542691ex99_2.htm EXHIBIT 99.2
Exhibit 99.2

 Deep Insights,Impactful Medicines 
 

 Disclaimers 
 

  
 

  
 

  
 

  
 

  
 

 Patients with Type 2 and 3 SMA Continue to Experience Major Functional Deficits Despite Improvement from SMN Therapy* 
 

 Non-Ambulatory Type 2/3 SMA: Majority of Patients Started on SMN Rx After Age 5 Do Not Experience Motor Function Increases* 
 

  
 

 Safety Results from TOPAZ 12-Month Top-Line Analysis Support Evaluation of Apitegromab in Phase 3 Trial 
 

 SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ 
 

 SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ 
 

 TOPAZ Age 2-12 Exploratory Analysis(Non-Ambulatory Type 2/3 SMA) 
 

 TOPAZ Age 2-12 Analysis* in Pooled Non-Ambulatory Cohorts Transformative Potential as Add-on for Apitegromab 
 

 SAPPHIRE (Phase 3) Trial Overview 
 

  
 

  
 

  
 

 Significant Interest in Potential Role of TGFβ Inhibition in Immuno-Oncology 
 

 Emerging Evidence Implicates TGFβ1 as Driving Primary Resistance to Checkpoint Inhibitors 
 

 SRK-181: Unique TGFβ1-Selective Approach to Overcoming Checkpoint Inhibitor Resistance 
 

 TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy 
 

 TGFβ1 Isoform Specificity of SRK-181 Improved Preclinical Toxicity Profile 
 

 SRK-181 Therapeutic Hypothesis: Potential Advantages of Latent TGFβ1 Inhibitor 
 

 DRAGON Phase 1 POC Trial to Evaluate SRK-181’s Ability to Overcome Primary Resistance to Checkpoint Inhibitors 
 

 DRAGON Part A: Dose Escalation Update and Safety Data 
 

 DRAGON Part A: Preliminary Pharmacokinetics (PK) Summary of SRK-181 
 

 DRAGON Part A: Preliminary Anti-Tumor Effects* 
 

 DRAGON Has Advanced to Part B to Test Proof of Concept for SRK-181 in Overcoming Anti-PD-(L)1 Resistance 
 

  
 

 Appendix 
 

 Apitegromab: Pairing the latent form with important translational insights 
 

 Apitegromab: Muscle-Directed Therapy Aimed at Complementing SMN Upregulators 
 

 Apitegromab Phase 2 Trial Design 
 

 Baseline CharacteristicsNusinersen-treated patients well into chronic maintenance phase 
 

 Non-Ambulatory Type 2 Cohort: Initiated nusinersen age <5 
 

 Non-Ambulatory Type 2/3 Cohort: Initiated nusinersen age ≥5 
 

 Majority of Ambulatory Patients Maintained or Improved in RHS Score from Baseline 
 

 Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Effects  
 

  
 

 Increases in HFMSE Not Correlated with Duration of Prior Nusinersen Treatment 
 

 WHO Motor Development Milestone Achievements Further Support Apitegromab’s Potential to Improve Motor Function  
 

 Inhibition of TGFβ1: Multipronged Approach for Immuno-Oncology 
 

 TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy