Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Corporate Update (January 2023)
31 slides from Opus Genetics's corporate update, Jan 25, 2023
Slide 1: ZETA-1 APX3330 Topline Results Investor Webcast
ZETA-1 APX3330 Topline Results Investor Webcast January 25, 2023
Slide 2: Disclosures and Forward-Looking Statements
Disclosures and Forward-Looking Statements This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the success and timing of planned future clinical tria
Slide 3: Agenda and Speakers
Agenda and Speakers Topic ZETA-1 Key Takeaways and APX3330 Oral MOA ZETA-1 Trial Design and Demographics ZETA-1 Efficacy Findings ZETA-1 Safety Findings Overall Summary and Next Steps Q&A Mina Sooch, MBA Founder and Chief Executive Officer Mitch Brigell, PhD Head of Clinical Development and Strategy
Slide 4: ZETA-1 Key Takeaways and APX3330 Oral MOA
ZETA-1 Key Takeaways and APX3330 Oral MOA
Slide 5: ZETA-1 Trial: Key Takeaways
ZETA-1 Trial: Key Takeaways APX3330 achieved statistical significance on a key pre-specified secondary endpoint of preventing clinically meaningful progression of diabetic retinopathy (as defined by binocular 3 or more steps worsening on the DRSS1) after 24 weeks of treatment Trend toward more effic
Slide 6: APX3330 History and Ref-1 Inhibition Mechanism
APX3330 History and Ref-1 Inhibition Mechanism Logsdon et al (2018), Li et al (2014). Ref-1 Involved in Multiple Key Pathways that Contribute to Diabetic Retinopathy and DME Mechanism of Action – Ref-1 Inhibition Hypoxia Ref-1 HIF-1α VEGF (Signaling Cascade) Inflammation Ref-1 NF-κB Other Growth Fac
Slide 7: ZETA-1 Trial Design and Demographics
ZETA-1 Trial Design and Demographics
Slide 8: DR/DME ZETA-1 Phase 2 Design
DR/DME ZETA-1 Phase 2 Design NPDR = non-proliferative diabetic retinopathy; PDR = proliferative diabetic retinopathy ZETA-1 Clinical Trial is Sponsored by Ocuphire Pharma Randomized, Double-Masked, Placebo-Controlled 24-Week Trial (Similar To Eylea P3 DR Trial) Primary: % subjects with ≥ 2 step impr
Slide 9: Key Eligibility Criteria in ZETA-1
Source: ZETA-1 Clinical trial Key Eligibility Criteria in ZETA-1 Oral Medication Provides Binocular Treatment; DME Allowed in Fellow Eye Inclusion Exclusion Males or non-pregnant females ≥ 18 years of age At least one eye with DR DRSS 47, 53, or 61, confirmed by a central reading center) in which PR
Slide 10: ZETA-1: Demographics
ZETA-1: Demographics APX3330 n=51 Placebo n=52 Total n=103 Demographics Age (years): Mean (Range) 54.3 (26-81) 58.3 (24-78) 56.3 (24-81) Sex: Male n (%) Female n (%) 24 (47%) 27 (53%) 26 (50%) 26 (50%) 50 (49%) 53 (52%) Race: White n (%) African American n (%) Asian n (%)
Slide 11: ZETA-1: Baseline DRSS Scores Study and Fellow Eye
ZETA-1: Baseline DRSS Scores Study and Fellow Eye APX3330 n=51 Placebo n=52 Total n=103 DRSS Score – Study Eye DRSS Category (Screening) Study Eye [n (%)] 47 (Moderately severe to severe NPDR) 22 (43%) 18 (35%) 40 (39%) 53 (Moderately severe to severe NPDR) 25 (49%) 28 (54%) 53 (5
Slide 12: ZETA-1: Baseline Characteristics Study and Fellow Eye
ZETA-1: Baseline Characteristics Study and Fellow Eye APX3330 n=51 Placebo n=52 Total n=103 Baseline Characteristic BCVA letters in Study Eye Letters Read (mean) 81 78 80 (20/25 Snellen) BCVA letters in Fellow Eye Letters Read (mean) 76 77 77 (20/32 Snellen) OCT Central Subfield Thickness in Study E
Slide 13: ZETA-1 Efficacy Findings
13 HARMA ZETA-1 Efficacy Findings
Slide 14: Background on DRSS Assessment & Binocular DRSS
10 81-85 71-75 Background on DRSS Assessment & Binocular DRSS Diabetic Retinopathy Severity Scale (DRSS) The ETDRS diabetic retinopathy severity scale (DRSS) is a categorical tool for clinical trials that contains 10 discreet steps from no retinopathy to severe proliferative retinopathy derived from
Slide 15: Percent of Subjects With ≥2-Step Improvement in DRSS From Baseline
Percent of Subjects With ≥ 2-Step Improvement in DRSS From Baseline Source: ZETA-1 Clinical Trial Note: Large “N” indicates total number of participants within each arm for the mITT population. Small “n” indicates total number of evaluable eyes for each respective endpoint and arm. Note: Images from
Slide 16: Clinically Meaningful Registration Endpoints in DR
In retina, opportunity for approval to show improvement OR worsening (prevention of progression)* Precedent approvable endpoint for locally delivered drugs (non-systemic) in DR: ≥ 2-step DRSS improvement in study eye Eylea (Panorama trial) Lucentis (Rise/Ride trials) Oral/systemic drugs are differen
Slide 17: Potential Phase 3 Endpoints as an Oral Drug; Results Improve with Time
Source: ZETA-1 Clinical Trial Note: Large “N” indicates total number of participants within each arm for the mITT-LOCF population. Small “n” indicates total number of evaluable eyes for each respective endpoint and arm. Note: Images from Central Reading Center will be reviewed prior to EOP2 FDA meet
Slide 18: Percent of Subjects With Binocular Worsening in DRSS of ≥3-Step
Percent of Subjects With Binocular Worsening in DRSS of ≥ 3-Step Selected Primary Registration Endpoint for Phase 3, To Be Formally Confirmed at EOP2 FDA Meeting p=0.04 p=0.07 Percent of Subjects With Worsening in DRSS of ≥3 Steps From Baseline by Visit Binocular Eyes (mITT-LOCF) n=49 n=45 n=49 n=45
Slide 19: Waterfall by Subject Binocular Change in DRSS
Waterfall by Subject Binocular Change in DRSS at Week 24 Source: ZETA-1 Clinical Trial Note: Images from Central Reading Center will be reviewed prior to EOP2 FDA meeting 8 Subjects in Placebo and 0 in APX3330 had a 3-Step DRSS Worsening at Week 24 ≥ 3 Steps Worsening n=8 ≥ 3 Steps Worsening n=0 Wat
Slide 20: Historic Data for Diabetic Patients on DR Progression
Historic Data for Diabetic Patients on DR Progression Source - Frequency of Evidence-Based Screening for Retinopathy in Type 1 Diabetes. N Engl J Med. 2017 Apr 20;376(16):1507-1516. doi: 10.1056/NEJMoa1612836. PMID: 28423305; PMCID: PMC5557280 The Worse the DRSS, the Higher the Risk of Vision Threat
Slide 21: ZETA-1 Safety Findings
21 HARMA ZETA-1 Safety Findings
Slide 22: PK/Safety Data Findings
PK/Safety Data Findings APX3330 PK/serum levels as predicted at 600 mg/day Serum levels of APX3330 are consistent with previous findings in hepatitis and oncology trials Fewer subjects lost 5 or more letters at week 24 with APX3330 compared to placebo Limited treatment related AEs (mostly mild and t
Slide 23: Treatment Emergent Adverse Events
Treatment Emergent Adverse Events APX3330 Safety Similar To or Better Than Placebo 0 Treatment-Related AEs involving liver, heart, kidney, brain, lung, or vital signs Oral APX3330 safety profile consistent with that seen in prior trials 14 SAEs (in 11 Subjects) 3 unrelated SAEs in APX3330 11 unrela
Slide 24: Summary and Next Steps
24 HARMA Summary and Next Steps
Slide 25: APX3330 Product Candidate Profile for Multiple Retinal Indications
APX3330 Product Candidate Profile for Multiple Retinal Indications Source: ZETA-1 Clinical Trial; *1 subject had vision blur thought to be related by investigator in ZETA-1 Oral, First-In-Class Ref-1 Inhibitor with Favorable Human Safety Data from 12 Completed Trials Novel MOA for Treating Retina ↓
Slide 26: Broad Opportunities to Treat Retinal Diseases with APX Platform
Diabetic Retinopathy PDR(DRSS >60) Moderate to Severe NPDR (DRSS 43-53) Mild NPDR 34 Million Diabetics in US 10M 6M 1M 1M ~8M+DR Patients Broad Opportunities to Treat Retinal Diseases with APX Platform APX3330 May Treat Patients Across Retinal Diseases as Single Agent or Adjunctive Therapy Anti-VEGF
Slide 27: Landscape of Systemic Therapies for Diabetic Retinopathy
Landscape of Systemic Therapies for Diabetic Retinopathy Ocuphire’s APX3330 is the Most Advanced Oral Drug Moving into EOP2 Mtg and Phase 3 www.clinicaltrials.gov Company Drug Target/MOA Indication Route of Administration Phase 1 Phase 2 Phase 3 Primary Endpoint/ Secondary Endpoints LY333531 Protein
Slide 28: APX3330 has Potential to be Early Preventative Therapy for DR Patients
APX3330 has Potential to be Early Preventative Therapy for DR Patients Eylea® label; APX3330 Investigator Brochure, ZETA-1 clinical trial Clinically meaningful decrease in diabetic retinopathy severity OR Early intervention with oral may prevent progression of DR to vision loss Efficacy Signal Intra
Slide 29: Key Takeaways and Next Steps
Key Takeaways and Next Steps APX3330 is the most advanced oral program in development for diabetic eye disease APX3330 demonstrated favorable safety with compelling potential to slow progression of diabetic retinopathy ZETA-1 statistically significant results on ‘binocular 3-step worsening DRSS' end
Slide 30: Corporate Highlights
Corporate Highlights Ocuphire PharmaNasdaq: OCUP Upcoming Catalysts: Topline Results APX3330 ZETA-1 P2b trial for DR/DME (Early 2023) EOP2 FDA Meeting for APX3330 (2H 2023) Pivotal Phase 3 Trials for Nyxol in Presbyopia with 1st Data Readouts (Late 2023) Potential Approval of 1st Nyxol NDA (Late 202
Slide 31: Thank You and Q&A
www.ocuphire.com ir@ocuphire.com Ocuphire Pharma Thank You and Q&A