Slide 1: A New Path to Medicine Company Reception
A New Path to Medicine Company Reception May 16, 2022 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements The following slides and any accompanying oral presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “should,” “expect,”
Slide 3: Efzofitimod: A Novel Mechanism to Treat Lung Inflammation and Fibrosis
Efzofitimod: A Novel Mechanism to Treat Lung Inflammation and Fibrosis 3 Representative histology showing immune cell infiltrate and collagen content in a rat model of bleomycin induced lung fibrosis presented at the ATS annual meeting 2018. Placebo treated Efzofitimod treated Healthy lung Inflammat
Slide 4: Foundational Science: Novel Functions of Extracellular tRNA Synthetases
Foundational Science: Novel Functions of Extracellular tRNA Synthetases 4 tRNA synthetases are secreted extracellularly and occur in novel forms which lose their canonical function Extracellular tRNA synthetase disruption (genetic/ autoimmune) is associated with disease in humans Validated synthetas
Slide 5: Efzofitimod: A Novel Immunomodulator for Fibrotic Lung Disease
Efzofitimod (ATYR1923, KRP-R120) A Novel Immunomodulator for Fibrotic Lung Disease
Slide 6: Efzofitimod: First-in-Class Therapy for Fibrotic Lung Disease
Efzofitimod: First-in-Class Therapy for Fibrotic Lung Disease 6 MOA = mechanism of action
Slide 7: Efzofitimod Therapeutic Goal: Restore Immune Balance and Prevent Fibrosis
7 Efzofitimod Therapeutic Goal: Restore Immune Balance and Prevent Fibrosis
Slide 8: First Efzofitimod Indication: Pulmonary Sarcoidosis
First Efzofitimod Indication: Pulmonary Sarcoidosis Multisystem inflammatory disorder of unknown etiology, characterized by the formation of granulomas (clumps of immune cells) Lung affected in ~90% of all patients ~5x increased mortality in advanced disease SOC: corticosteroids; cytotoxic immunosup
Slide 9: Results from Phase 1b/2a Study in Pulmonary Sarcoidosis
Efzofitimod (ATYR1923, KRP-R120) Results from Phase 1b/2a Study in Pulmonary Sarcoidosis Peter H. S. Sporn, M.D. Professor of Medicine (Pulmonary and Critical Care); Cell and Developmental Biology; and Medical Education Northwestern University Feinberg School of Medicine
Slide 10: Phase 1b/2a Trial Design
Phase 1b/2a Trial Design 10 FVC = Forced Vital Capacity; PFTs = Pulmonary Function Tests
Slide 11: Phase 1b/2a Inclusion / Exclusion Criteria
Phase 1b/2a Inclusion / Exclusion Criteria Inclusion Histologically proven diagnosis of pulmonary sarcoidosis Stable treatment with 10 -25 mg/day oral corticosteroid oral immunomodulator allowed Symptomatic/active disease at baseline FVC ≥ 50% percent predicted MRC Dyspnea Scale score (>= 1) Exclusi
Slide 12: Phase 1b/2a Study Schema
Phase 1b/2a Study Schema 12 (1) subtherapeutic dose
Slide 13: Phase 1b/2a Disposition
Phase 1b/2a Disposition mITT = randomized and dosed; *Defined as receiving at least 5 doses and having an end of study assessment Treated (mITT) Prematurely Discontinued Completed* Table 14.1.1.1 23Aug2021 13
Slide 14: Baseline Demographics and Disease Characteristics Generally Balanced
Baseline Demographics and Disease Characteristics Generally Balanced 14
Slide 15: Monthly Dosing of Efzofitimod was Safe and Well-Tolerated
Monthly Dosing of Efzofitimod was Safe and Well-Tolerated No new or unexpected findings with repeat dosing No dose-response relationship observed Most frequent AEs were consistent with underlying disease: Cough, fatigue, wheezing No signal of immunogenicity No drug related SAEs No deaths 15 Safety p
Slide 16: Dose-dependent Reduction in Steroid Utilization Compared with Placebo
Dose-dependent Reduction in Steroid Utilization Compared with Placebo 58% overall steroid reduction from baseline and 22% relative reduction compared to placebo in steroid utilization post taper in the 5 mg/kg treatment group 33% of patients able to taper off of steroids completely in 5 mg/kg treatm
Slide 17: Dose-dependent Improvement in FVC % Predicted Compared to Placebo
Dose-dependent Improvement in FVC % Predicted Compared to Placebo 17 Differences represent from MMRM adjusting for baseline FVC % Predicted, mean (95% CI)
Slide 18: Higher Efzofitimod Doses Show Trends of Improvement in FVC % Predicted
Higher Efzofitimod Doses Show Trends of Improvement in FVC % Predicted 18
Slide 19: Dose-dependent Improvements in PFTs Over Time Compared with Placebo
Dose-dependent Improvements in PFTs Over Time Compared with Placebo 19 PFTs = Pulmonary Function Tests; CFB = Change From Baseline Where is week 24 data?
Slide 20: Dose-dependent Symptom Improvements Compared with Placebo
Dose-dependent Symptom Improvements Compared with Placebo 20 *p<0.05 on difference between adjusted means from MMRM; Pbo = Placebo MCIDs: TDI - Witek 2003; LCQ – Raj 2009; FAS - de Kleijin 2011 (Negative score is better for Fatigue); KSQ Lung – Baughman 2021; KSQ Lung – Baughman 2021 = clinically me
Slide 21: Dose-dependent Control of Key Disease and Inflammatory Biomarkers
Dose-dependent Control of Key Disease and Inflammatory Biomarkers 21 Graphs represent fold change at Week 24 compared to Baseline IFNg IP-10 / CXCL10 MCP-1 / CCL2 IL-2Ra
Slide 22: Phase 1b/2a Data Supports Proof-of-Concept and Clinical Advancement
Phase 1b/2a Data Supports Proof-of-Concept and Clinical Advancement Efzofitimod was safe and well-tolerated Strong suggestion of efficacy: Dose-response and consistent positive trends across key efficacy endpoints and multiple analysis populations Clinically meaningful symptom improvements in dyspne
Slide 23: Efzofitimod Phase 3 EFZO-FIT Study
Efzofitimod (ATYR1923, KRP-R120) Phase 3 EFZO-FIT™ Study Robert P. Baughman, M.D. Emeritus Professor of Medicine University of Cincinnati
Slide 24: Dose-dependent Reduction in Steroid Utilization Compared with Placebo
Dose-dependent Reduction in Steroid Utilization Compared with Placebo 58% overall steroid reduction from baseline and 22% relative reduction compared to placebo in steroid utilization post taper in the 5 mg/kg treatment group 33% of patients able to taper off of steroids completely in 5 mg/kg treatm
Slide 25: Dose-dependent Symptom Improvements Compared with Placebo
Dose-dependent Symptom Improvements Compared with Placebo 25 *p<0.05 on difference between adjusted means from MMRM; Pbo = Placebo MCIDs: TDI - Witek 2003; LCQ – Raj 2009; FAS - de Kleijin 2011 (Negative score is better for Fatigue); KSQ Lung – Baughman 2021; KSQ Lung – Baughman 2021 = clinically me
Slide 26: EFZO-FIT: Phase 3 Study of Efzofitimod in Pulmonary Sarcoidosis
EFZO-FITTM : Phase 3 Study of Efzofitimod in Pulmonary Sarcoidosis 26
Slide 27: Trial Design
Trial Design 27 Still under discussion with Kyorin clinical team
Slide 28: Major Efficacy Endpoints
Major Efficacy Endpoints Primary Endpoint Change from baseline in mean daily OCS dose post-taper Secondary Endpoints Annual rate of change in absolute value of FVC Percent change from baseline in mean daily OCS dose post-taper at Week 48 Change from baseline in KSQ-Lung score at Week 48 28
Slide 29: Key Inclusion / Exclusion Criteria
Key Inclusion / Exclusion Criteria Inclusion Adults ages 18-75, inclusive Diagnosis of pulmonary sarcoidosis for ≥6 months Requiring stable treatment with ≥7.5 but ≤25 mg/day oral corticosteroids Medical Research Council (MRC) Dyspnea Scale ≥ 1 KSQ Lung Score ≤ 70 Exclusion Extent of fibrosis > 20%
Slide 30: Phase 3 Study Schema
Phase 3 Study Schema 30 OCS = oral corticosteroids
Slide 31: Multi-center Trial with Sites in North America, Europe and Japan
Multi-center Trial with Sites in North America, Europe and Japan 31 Anticipating 60-80 centers in 10 countries
Slide 32: Thank You
alyr Thank You