Scholar Rock Holding Corporation · NASDAQ:SRRK

Scholar Rock — ESMO Targeted Anticancer Therapies (TAT) 2023

13 slides from Scholar Rock's conference presentation, March 2023

  1. Slide 1: Safety and Efficacy Results of SRK-181, a Latent TGFβ1 Inhibitor, from a Phase 1 Trial

    SAFETY AND EFFICACY RESULTS OF SRK-181, A LATENT TGFβ1 INHIBITOR, FROM A PHASE 1 TRIAL (DRAGON TRIAL) Timothy A. Yap1, Justin F. Gainor2, Meredith McKean3, Bruno Bockorny4, Minal Barve5, Randy Sweis6, Ulka Vaishampayan7, Ahmad Tarhini8, Deepak Kilari9, Arati Chand10, Raghad Karim11, David Park12, Su

  2. Slide 2: Declaration of Interests

    Declaration of Interests Dr. Timothy A. Yap  Employment: University of Texas MD Anderson Cancer Center, where I am Medical Director of the Institute for Applied Cancer Science, which has a commercial interest in DDR and other inhibitors (IACS30380/ART0380 was licensed to Artios)  Advisory/Consulti

  3. Slide 3: SRK-181: Unique Latent TGFβ1 Selective Approach to Overcoming Checkpoint Inhibitor Resistance

    SRK-181: Unique Latent TGFβ1 Selective Approach to Overcoming Checkpoint Inhibitor Resistance SRK-181: Latent TGFβ1 Inhibitor Targets TGFβ1 Potential to SRK-181 inhibits the TGFβ1 implicated overcome in check point inhibitor resistance CPI resistance Selective to Highly selective Increases therapeut

  4. Slide 4: Selectivity of SRK-181 Offers Potential to Avoid Toxicity and Dose-limiting Challenges

    Selectivity of SRK-181 Offers Potential to Avoid Toxicity and Dose-limiting Challenges of Non-selective TGFβ Inhibition Toxicology: Unremarkable Slight Non-selective TGFβ Inhibitors Selective TGFβ1 Inhibitor Toxicity: Minimal, slight and moderate Toxicity: Minimal Minimal Moderate CONTROL LY2109761

  5. Slide 5: DRAGON Phase 1 POC Trial to Evaluate SRK-181's Ability to Overcome Resistance

    DRAGON Phase 1 POC Trial to Evaluate SRK-181’s Ability to Overcome Primary/Acquired Resistance to Checkpoint Inhibitors Part A1: SRK-181(IV) Part A2: SRK-181(IV) + Anti-PD-(L)1 Part A Endpoints Advanced solid tumors that failed Non-Responders to prior Anti-PD-(L)1; Primary: SOC treatment (completed)

  6. Slide 6: DRAGON Part A: Demographics and Disposition

    DRAGON Part A: Demographics and Disposition Category Part A1 Part A2 Category Part A1 Part A2 Monotherapy Combination Monotherapy Combination Treatment Treatment N 19 15 Age, median (range) 66(41 - 79) 65 (32 - 75) Enrolled 19 15 Gender (F/M) 8/11 3/12 On Study 0 0 Ethnicity Stopped Treatment 19 15

  7. Slide 7: DRAGON Part A: Safety

    DRAGON Part A: Safety PART A1 Monotherapy No DLTs No Grade 4 or 5 were observed up to Treatment-Emergent AEs Related to SRK-181, All Grades >10% 3000 mg q3w and treatment-related AEs occurred 2000 mg q2w 80 240 800 1600 2400 3000 2000 All Dose (MG) N=1 N=1 N=3 N=4 N=3 N=3 N=4 N=19 Treatment-related

  8. Slide 8: Dragon Part A: Preliminary Efficacy Data

    Dragon Part A: Preliminary Efficacy Data Duration of Treatment Part A Duration of Treatment Part A1 Monotherapy(n=19) 8 patients had a best response of stable disease (SD) All 3 patients with ovarian cancer were stable for 6-10 month Ovarian SRK-181 Age Lines of Duration of Best Patient Dose Prior T

  9. Slide 9: Dragon Part B (Combination Treatment) Update

    Dragon Part B (Combination Treatment) Update  20 patients dosed across multiple cohorts • Cohort ccRCC: 9 patients • Cohort NSCLC: 3 patients • Cohort UC: 4 patients • Cohort Melanoma: 1 patient • Cohort Any Other: 3 patients (Enrollment closed)  SRK-181 was generally well tolerated in combination

  10. Slide 10: Summary of ccRCC Patients (Part A2 and Part B, Combination Treatment)

    Summary of ccRCC Patients (Part A2 and Part B, Combination Treatment)  As of Dec 02, 2022, total 11 ccRCC patients are enrolled in Part A2 and Part B • N=2 in Part A2 (all discontinued from study) and N=9 in Part B (5 pts remain on study) • Enrollment continues  3 confirmed PRs have been observed

  11. Slide 11: Summary of ccRCC Patients with PR in Dragon

    Summary of ccRCC Patients with PR in Dragon (Part A2 and Part B, Combination Treatment) Responded SRK-181 Dose Age (Year)/ Lines of IMDC Score Metastatic Sites Pt Status Duration of Best % Change in Pts (mg, Q3W) Gender Prior Therapy at Screening at Screening Treatment (wks) SOD* from Baseline Pt #1

  12. Slide 12: Conclusion

    Conclusion As of Dec 02, 2022 • SRK-181 was generally well tolerated in combination with anti-PD-(L)1 at all doses • No DLTs were observed up to 3000mg q3w/2000mg q2w as monotherapy and up to 2400mg q3w as combination treatment. • Three confirmed PR were observed in patients with anti-PD-1 resistant

  13. Slide 13: Acknowledgment

    Acknowledgment Authors would like to appreciate everyone who takes part in the study. • Thanks to the patients and their families for their participation • Thanks to the investigators, co-investigators and their study team for their time and effort • Thanks to the advisory committees for their expe

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