Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Merger or Acquisition (October 2024)
27 slides from Opus Genetics's merger presentation, Oct 22, 2024
Slide 1: Delivering on the Promise of Ophthalmic Gene Therapy for Rare Inherited Retinal Diseases
Delivering on the Promise of Ophthalmic Gene Therapy for Rare Inherited Retinal Diseases October 2024 Braydon, RDH12 patient
Slide 2: 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 expectations regarding our cash runway, data from and future enrollment for our clinical trials, ou
Slide 3: Agenda – Acquisition of Opus Genetics
Agenda – Acquisition of Opus Genetics Opening Remarks & Transaction Overview George Magrath, MD Chief Executive Officer Opus Genetics Introduction Ben Yerxa, PhD President Pipeline Overview Ash Jayagopal, PhD Chief Scientific & Development Officer Q& A Opus Genetics Management 1 2 3 4 3
Slide 4: Acquisition of Opus Genetics
Acquisition of Opus Genetics Experienced management team with significant ophthalmology and gene therapy experience Approximate cash balance of $37M (September 30, 2024)* Value potential of Phentolamine Ophthalmic Solution 0.75% franchise milestones and royalties World-class science from gene therap
Slide 5: 2026 Cash Runway Extends Past Four Clinical Trial Readouts in 2025
Q1 2025 Q2 2025 Q3 2025 Q4 2025 Phentolamine Ophthalmic Solution 0 . 75 % Q1: P DLD T h 3 LYNX-2 LD 1H: P Presb h 3 VEGA-3 yopia TLD OPGx- LCA 5 Q3: Ph 1/2 Pediatric data OPGx- BEST1 Q4: Ph 1/2 data 5 BEST1, bestrophin 1; DLD, dim light disturbances; IRD, inherited retinal disease; LCA5, Leber conge
Slide 6: Transaction Overview
Ocuphire has completed acquisition of Opus in all-stock transaction Pro forma ownership of 58% Ocuphire / 42% Opus Company will trade under new ticker of “IRD” Pro forma estimated cash and cash equivalents is $37M as of September 30, 2024, with an expected runway into 2026 Not including additional n
Slide 7: Established Technology with Clinical Proof of Concept, Efficient Development Pathway, and Significant Commercial Value
Established Technology with Clinical Proof of Concept, Efficient Development Pathway, and Significant Commercial Value Luxturna® is a registered trademark of Spark Therapeutics, Inc. AAV, adeno-associated virus; FDA, Food and Drug Administration; IRD, inherited retinal disease; NIH, National Institu
Slide 8: Fully Integrated Leadership Team with Decades of Expertise
Fully Integrated Leadership Team with Decades of Expertise and Successful Track Record of Development and Commercialization 8 George Magrath, MD, MBA, MS Chief Executive Officer Nirav Jhaveri, MBA Chief Financial Officer Joseph Schachle, MBA Chief Operating Officer Jean Bennett, MD, PhD Scientific A
Slide 9: Efficient IRD Pipeline with Multiple Near-Term Value Inflection Points
Efficient IRD Pipeline with Multiple Near-Term Value Inflection Points and Expected Meaningful Cashflow from Phentolamine Franchise *Orphan Drug Designation and Rare Pediatric Disease Designation received from the U.S. FDA for OPGx-LCA5; All remaining candidates are eligible for Orphan Drug and Rare
Slide 10: OPGx-LCA5 Phase 1/2 Gene Therapy for LCA5
OPGx-LCA5 Phase 1/2 Gene Therapy for LCA5 Alan, LCA5 patient LCA5, Leber congenital amaurosis 5.
Slide 11: LCA5 is an Early-Onset, Severe Hereditary Retinal Degeneration
P r e v a l e n c e ~200 patients in the U.S.1,2 LCA5 represents ~2% of all LCA cases3 C l i n i c a l C h a r a c t e r i s t i c s Patients typically present in 1st year of life with nystagmus and vision loss3,4 Early loss of rod-mediated peripheral vision results in constricted visual fields3,4 V
Slide 12: OPGx-LCA5 Restores Structure and Function in Photoreceptors
Lebercilin is a ciliary protein critical for protein trafficking in photoreceptor inner and outer segments1 In LCA5, photoreceptor function is severely impaired due to absence of the structural protein lebercilin1 − However, photoreceptors persist in LCA5 patients through the third decade of life, s
Slide 13: OPGx-LCA5 Improved Visual Function in Three Legally Blind Adult Patients
First-in-human, open label, dose-escalation study − First three low-dose adult patients completed in 2024 All three patients demonstrated visual improvement at 6 months: − Improvement in the FST functional test of light sensitivity − Significant improvement in VR-guided mobility testing − Greater th
Slide 14: All 3 Patients Demonstrated Improved Number of Objects Recognized
3/3 Patients Showed Improved Visual Function through 6 Months Videos colorized for presentation purposes. BL, baseline; VR, virtual reality. 1. Luxturna Prescribing Information. Spark Therapeutics. Note: Subject 01-03 and 01-04 are cone-mediated disease and Subject 01-01 is rod-mediated disease. Not
Slide 15: 3/3 Patients Showed Improved Retinal Sensitivity as Assessed by FST
15 3/3 Patients Showed Improved Retinal Sensitivity as Assessed by FST Subject 01-03 Subject 01-01 Subject 01-04 FST improvement observed for cone-mediated (red) and rod-mediated (blue outlined) vision Significant gains in retinal sensitivity and comparable to adult patients dosed with Luxturna Note
Slide 16: OPGx-LCA5 Phase 1/2 Next Steps: Ready to Enroll Pediatric Subjects
Ready for enrollment of pediatric patients in Q1 2025, with preliminary data expected in Q3 2025 FDA Office of Orphan Drug Products grant awarded to support Phase 1/2 trial Rare Pediatric Disease Designation and Orphan Drug Designation received from the FDA, which confers eligibility for Priority Re
Slide 17: OPGx-BEST1 Phase 1/2-Ready Gene Therapy for BEST1-associated Disease
OPGx-BEST1 Phase 1/2-Ready Gene Therapy for BEST1-associated Disease BEST1, bestrophin 1.
Slide 18: BEST1 Mutations are Associated with Retinal Degeneration
P r e v a l e n c e ~9,000 patients the U.S.1 Accounts for ~3.5% of all IRDs2 C l i n i c a l C h a r a c t e r i s t i c s Mutations in BEST1 have been associated with at least five clinically distinct retinal degenerative diseases3 Bestrophinopathy is characterized by retinal lesions, with symptom
Slide 19: Compelling Safety and Efficacy Data from IND-Enabling Canine Study
19 Compelling Safety and Efficacy Data from IND-Enabling Canine Study Robust restoration of RPE- photoceptor interface demonstrated in canine models of autosomal recessive BEST1 disease Treated cBEST1 models exhibit reversal of lesions and retinal microdetachments, which are hallmarks BEST1 disease
Slide 20: Data from OPGx-BEST1 Phase 1/2 Study Expected in Q4 2025
20 OPGx-BEST1 well-tolerated in toxicology studies; human dose and NOAEL established BEST1 AAV GMP drug product ready for human dosing; subject to regulatory clearance Positive feedback from EU regulators for Phase 1/2 clinical trial design in Germany Ready to initiate Phase 1/2 clinical trial in Ge
Slide 21: Valuable Gene Therapies Create Robust Commercial Potential
21 Valuable Gene Therapies Create Robust Commercial Potential $850,000 $2,254,412 $2,800,000 $3,000,000 $3,200,000 $3,000,000 $2,500,000 $2,000,000 $1,500,000 $1,000,000 $500,000 $0 $3,500,000 WAC ($) Product L u x t u r n a ® * Z o l g e n s m a ® Z y n t e g l o S k y s o n a E l e v i d y s Indic
Slide 22: Candidates that Fuel Our Future Growth
Candidates that Fuel Our Future Growth Phentolamine Ophthalmic Solution 0.75% Franchise and APX3330
Slide 23: Global Partnership for Phentolamine Ophthalmic Solution 0.75% Franchise
LASIK, laser assisted in situ keratomileusis. 1. Ryzumvi. Prescribing Information. Ocuphire Pharma, Inc.; 2023. 2. Wilson FA, et al. J Ophthalmol. 2015;2015:435606. 3. Berdahl J, et al. Clin Ophthalmol. 2020;14:3439-3450. 4. Lindstrom RL. Millennials will be the next target for laser vision correcti
Slide 24: Completed Phase 2 Study of Oral APX3330 Shows Promising Safety and Efficacy
24 Completed Phase 2 Study of Oral APX3330 Shows Promising Safety and Efficacy D i a b e t i c R e t i n o p a t h y M a r k e t i s L a r g e a n d U n d e r s e r v e d DR is the leading cause of blindness in working age adults, impacting 10M patients in the U.S.1,2 Most have early-stage disease o
Slide 25: APX3330 is Available for Partnering
W h y P a r t n e r i n g : Due to capital requirements and development timelines for APX3330, we determined that future clinical development of a late-stage DR program would be best suited for a partner Redirecting our spend towards more capital-efficient gene therapy programs E f f o r t S u p p o
Slide 26: Acquisition Creates Leading IRD Franchise with Multiple Near-Term Milestones
A c q u i s i t i o n Combines partnered asset in Phentolamine with Opus’s cutting-edge, rare IRD gene therapy portfolio Cash runway expected to extend into 2026 Experienced management team U p c o m i n g M i l e s t o n e s * OPGx-LCA5: Ready for enrollment of pediatric subjects in Phase 1/2 study
Slide 27: Every patient's eyes tell a story
Every patient’ s eyes tell a story Images of real patients with IRDs.
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