Slide 1: Beacon Therapeutics
Beacon Therapeutics z4 SYNCONA INVESTMENT MANAGEMENT
Slide 2: SIML's differentiated approach to value creation
ach to value creation SIML’s differentiated approach to value creation
Slide 3: Building Beacon
Building Beacon Building a successful Phase III late-stage clinical company in <4 years Targeting X-linked retinitis pigmentosa (XLRP), a blinding disease with large unmet need and no current treatment options VALUE CREATION JOURNEY INSIGHT & STRATEGY & TEAM & CAPITAL CLINICAL FOUNDATIONAL RESTRUCTU
Slide 4: A high unmet medical need
=4 SYNCONA INVESTMENT MANAGEMENT A high unmet medical need
Slide 5: XLRP: inherited blindness with no treatment options
XLRP: inherited blindness with no treatment options Progressive disease, primarily in men, leads to blindness in their 40s1 >70% of cases have a defect in the RRPGR gene, leading to rod and cone degeneration2,3 Rare disease, affecting 280k patients globally (10-20% of all RP)2* No treatment, no way
Slide 6: Beacon is led by a team with deep heritage in gene therapy
Beacon is led by a team with deep heritage in gene therapy and its application in ocular disease Lance Baldo Dan Chung Ryan Robinson Abraham Scaria Natasha Jarrett Brian Mullan Chief Executive Officer Chief Medical Officer Chief Financial Officer Chief Scientific Officer SVP Regulatory Affairs SVP T
Slide 7: A potentially first and best-in-class therapy
n-class therapy A potentially first and best- in-class therapy
Slide 8: Laru-zova has the potential to restore natural function of rods and cones
Laru-zova has the potential to restore natural function of both rods and cones by delivering the full-length RPGRORF15 protein Laru-zova is injected sub-retinally and delivers a working copy of the faulty gene Needle Treatment • In XLRP, a faulty RPGR gene leaves rods and cones without working admin
Slide 9: Laru-zova is measured against LLVA and microperimetry (LLVA)
Laru-zova is measured against LLVA and microperimetry to support approval and adoption Low luminance visual acuity (LLVA): Standard chart lighting Low lighting (LLVA) • LLVA is a test that measures how well a person can read letters on an eye chart at a lower light level compared to standard chart l
Slide 10: Laru-zova is measured against LLVA and microperimetry (microperimetry)
Laru-zova is measured against LLVA and microperimetry to support approval and adoption Microperimetry: Readings at each test point Readings combined into a map • Microperimetry measures visual function across the macula using retinal image tracking to measure light sensitivity at specific points in
Slide 11: 10 years ago, images like these convinced Syncona of the approach
10 years ago, images like these convinced Syncona that this medical approach could be transformational for XLRP patients 11
Slide 12: FDA-endorsed primary endpoint met in pivotal study
point met in pivotal study FDA-endorsed primary endpoint met in pivotal study
Slide 13: VISTA study design randomised males with XLRP to two doses of laru-zova
The VISTA study design randomised 85 males with XLRP to two doses of laru-zova or untreated control, with the primary readout at month 12 Phase 2/3 randomised, controlled, masked, multicentre study of the efficacy, safety and tolerability of two dose levels of laru-zova versus an untreated control g
Slide 14: Both doses improved LLVA at month 12
Both doses improved LLVA at month 12, with up to 31% of patients gaining 15 or more letters versus none in the control group 3-line response, primary endpoint: 2-line response: Participants gaining ≥15 ETDRS letters from baseline Participants gaining ≥10 ETDRS letters from baseline p=0.0002 p<0.0001
Slide 15: Laru-zova demonstrated totality of data with favourable safety
Laru-zova demonstrated compelling totality of data with favourable safety and tolerability profile Key efficacy endpoints at month 12: Safety and tolerability: • LLVA ≥3-line gain, ITT: 31.0% with high dose (p=0.0019) and 24.1% with • Ocular TEAEs were balanced across low dose (p=0.0106), versus 0%
Slide 16: US and Europe contribute almost equally to a $15B+ revenue opportunity
The US and Europe contribute almost equally to a $15B+ revenue opportunity from around 11,400 eligible patients United States EU4 + UK 100k patients with RP $8.0B $6.9B 96k patients with RP 14,000 13,400 XLRP XLRP 9,800 RPGR XLRP $15B+ 9,100 RPGR XLRP 5,880 5,500 Eligible patients* Eligible patients
Slide 17: Beacon now moves to global filings
Beacon now moves to global filings, which could make laru-zova the first approved treatment for XLRP What happens next: Sept 2026 Oct 2026 Late 2026 Through year 5 VISTA met its primary Further VISTA data at Rolling BLA submission Long-term follow-up of endpoint at month 12 AAO Retina Subspecialty t
Slide 18: A portfolio poised to deliver value
value A portfolio poised to deliver value
Slide 19: Key value inflection points ahead across broader portfolio
Key value inflection points ahead across broader portfolio With the potential to deliver significant NAV growth Key value inflection point (KVIP) B E S T I DE A S P R E - CLI NI CA L CLI NI CA L LA TE - STAGE BLA COMMERCIAL N E X T E X P E C T E D KVIP CLI N I CA L ON THE MARKET Autolus Therapeutics