Syncona Ltd GBP · LSE:SYNC
Syncona Ltd GBP — Syncona — Investor Presentation (October 2024)
27 slides from Syncona Ltd GBP's investor presentation, Oct 30, 2024
Slide 1: Syncona Investor Webinar: Beacon Therapeutics
Synconaltd.com Syncona Investor Webinar: Beacon Therapeutics 30 October 2024
Slide 2: Cautionary statement
Cautionary statement This presentation has been prepared and published solely for informational purposes. Nothing expressed or implied from the forward-looking statements. contained in this presentation is intended to constitute an offer, invitation or inducement to engage in an investment activity.
Slide 3: Overview of today's session
Overview of today’s session Beacon is a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases Chris Hollowood, PhD, SIML CEO Market backdrop and Syncona update AGTC acquisition and the subsequent formation of Beacon Lance Bal
Slide 4: Syncona: Chris Hollowood, SIML CEO
Synconaltd.com Syncona: Company update Chris Hollowood, SIML CEO
Slide 5: Market recovery continues
Market recovery continues Average Enterprise Value of a Biotech listed on US exchanges by stage of development 1 Recovery continues to be weighted towards late-stage assets $1,200.00 $1,000.00 Public and private financing environment improving $800.00 Inflation has reduced since its peaks in 2022, a
Slide 6: Clinical and operational progress across the portfolio
Clinical and operational progress across the portfolio Continue to see strong momentum across our increasingly maturing portfolio Recent clinical progress Recent financings Expected upcoming milestones Anaveon entered the clinic with its July 2024 - Beacon’s £134m Series B Beacon to release data fro
Slide 7: Building a global leader in ophthalmic gene therapy
Building a global leader in ophthalmic gene therapy Strong momentum following recent Series B financing Acquisition of a potentially best-in-class asset Acquired AGTC-501 through the $23.3m (£20.8m) take-private of NASDAQ-listed AGTC in November 2022 AGTC-501 a clinical-stage gene therapy for X-Link
Slide 8: Beacon Therapeutics
Beacon Therapeutics Pipeline, clinical progress and milestones © Beacon Therapeutics 2024 All rights reserved
Slide 9: Beacon Therapeutics – Making Remarkable Happen
Beacon Therapeutics – Making Remarkable Happen OUR MISSION: TO SAVE AND RESTORE VISION IN PATIENTS WITH BLINDING RETINAL DISEASES Proprietary pipeline Late-stage clinical program Global research and Experienced management addressing rare and prevalent for XLRP, a devastating disease development foot
Slide 10: Experienced Leaders in Ophthalmic Gene Therapy
Experienced Leaders in Ophthalmic Gene Therapy PROVEN RECORD OF SUCCESS IN TRANSLATIONAL AND CLINICAL DEVELOPMENT Dr. Lance Baldo Dr Abraham Scaria Tom Biancardi Natasha Jarrett Chief Executive Officer Chief Scientific Officer Chief Financial Officer SVP, Regulatory Affairs • 20 years of biopharma e
Slide 11: Proprietary Pipeline of Innovative Gene Therapies
Proprietary Pipeline of Innovative Gene Therapies PROGRAMS SPANNING EARLY TO LATE-STAGE DEVELOPMENT, WITH VALIDATING DATA IN HAND, ADDRESSING RARE & PREVALENT MARKETS WITH HIGH UNMET NEED Asset Indication Preclinical Phase I Phase II Phase III X-Linked AGTC-501 Retinitis Pigmentosa (XLRP) Phase 2/3
Slide 12: Robust Clinical Development Program for AGTC-501
Robust Clinical Development Program for AGTC-501 2023 2024 2025 2026 2027 2028 Ph 1/2 HORIZON LPI 2020 (29 pts) 5-yr follow-up Ph 2 SKYLINE 5-yr follow-up LPI 2022 (14 pts) AGTC-501 Ph 2 DAWN 5-yr follow-up FPI 2023 (up to 24 pts) Ph 2/3 VISTA 5-yr follow-up FPI 2024 (75 pts) © Beacon Therapeutics 2
Slide 13: X-Linked Retinitis Pigmentosa (XLRP)
X-Linked Retinitis Pigmentosa (XLRP) PROGRESSIVE PHOTORECEPTOR DEGENERATION THAT LEADS TO BLINDNESS WITH NO TREATMENT OPTIONS Severe, aggressive, inherited retinal Normal Retina Retinitis Pigmentosa disease characterized Childhood 20-30s 40-50s by progressive photoreceptor degeneration1 Optic Disc F
Slide 14: AGTC-501 24-Month Interim Results of Phase 2 SKYLINE Trial
Subretinal AGTC-501 Gene Therapy for XLRP: 24-Month Interim Results of the Phase 2 SKYLINE Trial Data presented at American Academy of Ophthalmology Annual Meeting October 2024 © Beacon Therapeutics 2024 All rights reserved
Slide 15: Phase 2 SKYLINE Study Design
Phase 2 SKYLINE Study Design Randomized, controlled, multicenter study to evaluate the safety, efficacy, and tolerability of AGTC-501 in patients with XLRP caused by mutations in the RPGR gene Primary Objective • Proportion of response by microperimetry** 12M Endpoint 6.8 E+11 vg/eye n=8 between stu
Slide 16: Phase 2 SKYLINE Endpoints
Phase 2 SKYLINE Endpoints Primary Efficacy Endpoint • Proportion of response by microperimetry between study and fellow eye at Month 12: – Response defined as ≥ 7 dB improvement in ≥ 5 loci (microperimetry via MAIA) Secondary Endpoints Images adapted from Josan1 • Change from baseline (CFB) at Month
Slide 17: Phase 2 SKYLINE Efficacy Summary at Month 24
Phase 2 SKYLINE Efficacy Summary at Month 24 Greater response rate seen in the high dose study eyes compared to low dose and fellow eyes, consistent from Month 12 to Month 24 Responder Rate Month 12 Responder Rate Month 24 Patients (%) Achieving a ≥7 dB Improvement from Baseline Patients (%) Achievi
Slide 18: Phase 2 SKYLINE Interim Results
Phase 2 SKYLINE Interim Results • Robust improvement in retinal sensitivity in the Early and robust improvement in retinal sensitivity seen with high dose high dose group* vs. the low dose group† and group with durability maintained to month 24 untreated eyes1 ,2 • There were no ocular serious adver
Slide 19: Conclusions Phase 2 SKYLINE 24-Month Interim Analysis
Conclusions Phase 2 SKYLINE 24-Month Interim Analysis Data show continued robust improvements in visual function AGTC-501 was generally safe and well-tolerated • To date, AGTC-501 data show robust improvements in retinal sensitivity as assessed by MAIA microperimetry • No Ocular SAEs were deemed rel
Slide 20: Currently enrolling clinical trials
Currently enrolling 2 clinical trials Phase 2/3 VISTA and Open Label Phase 2 DAWN Now Enrolling DAWN • Open label Phase 2 study comparing two doses of AGTC-501 in the fellow eye of previously treated male participants with XLRP caused by mutations in the RPGR gene • Primary Objective: evaluate the s
Slide 21: Beacon Therapeutics – Making Remarkable Happen
Beacon Therapeutics – Making Remarkable Happen OUR MISSION: TO SAVE AND RESTORE VISION IN PATIENTS WITH BLINDING RETINAL DISEASES Proprietary pipeline Late-stage clinical program Global research and Experienced management addressing rare and prevalent for XLRP, a devastating disease development foot
Slide 22: Thank you
Thank you For further enquiries, please use the below contact details. Lance Baldo, MD CEO Beacon Therapeutics lbaldo@beacontx.com +1 (650) 922-6440 © Beacon Therapeutics 2024 All rights reserved
Slide 23: Appendix
beacen therapeutics Appendix
Slide 24: Overview of AGTC-501 Gene Therapy for XLRP
Overview of AGTC-501 Gene Therapy for XLRP Proprietary capsid designed for high transduction of codon-optimized, full-length transgene delivered subretinally Image created by R. Sisk in BioRender CAPSID PROMOTER CODON-OPTIMIZED GENE Proprietary capsid AAV2tYF Photoreceptor specific GRK1 Codon-optimi
Slide 25: Phase 2 SKYLINE Demographics and Baseline Characteristics
Phase 2 SKYLINE Demographics and Baseline Characteristics Groups were well matched N = 14 100% males Low Dose High Dose Age (7.5 E+10 vg/eye) (6.8 E+11 vg/eye) (N=6) (N=8) 8-17 Years Old 18-50 Years Old Endpoints 100% SE FE SE FE 90% 80% 68.3 (3.20) 73.2 (1.72) 66.5 (6.52) 71.1 (5.14) 70% BCVA (ETDR
Slide 26: Ocular Serious Adverse Events (SAEs) at Month 24
Ocular Serious Adverse Events (SAEs) at Month 24 24-month analysis indicated AGTC-501 was generally safe and well-tolerated, with no clinically significant safety events associated with treatment Ocular Serious Adverse Events Low Dose High Dose All Patients (SAE) (7.5 E+10 vg/eye) (6.8 E+11 vg/eye)
Slide 27: Ocular Treatment-emergent Adverse Events Related to AGTC-501 at Month 24
Ocular Treatment-emergent Adverse Events (TEAEs) Related to AGTC-501 at Month 24 Ocular Treatment-emergent Low Dose High Dose All Patients Adverse Event (TEAE) (7.5 E+10 vg/eye) (6.8 E+11 vg/eye) (n=14) (n=6) (n=8) Study Eye Fellow Eye Study Eye Fellow Eye Study Eye Fellow Eye # of Patients with Any