R&D Event Firmed Up Timelines and Laid Out the De-Risked Pipeline. September BEST1 Cohort 1 Readout Likely Next Big Catalyst.

研报英文原文证据摘录

R&D Event Firmed Up Timelines and Laid Out the De-Risked Pipeline. September BEST1 Cohort 1 Readout Likely Next Big Catalyst.

Investment Thesis

Opus genetics uses validated gene therapy approaches to treat and correct inherited retinal disease (IRD). There are hundreds

of IRDs, but Opus Genetics targets indications where the biology is well validated by animal models considered representative of

human disease. Two key programs should provide steady news flow beginning in mid26 with P1 data that could be highly de-

risking in BEST1 disease and potentially pivotal data in LCA5 around YE26. The Opus approach looks risk-reduced as it employs

well understood sub-retinal delivery of AAV-family therapeutics many of which were developed by the same scientists responsible

for the first approved gene therapy LUXTURNA.BIOTECHNOLOGY Upcoming Catalysts Price Performance

■ BEST1 P1/2 Trial Full Cohort data- September 2026 $6

■ LCA5 P3 dosing initiation - 4Q26 $5

■ RDH12 clinical study initiation - 4Q26

$4EQUITY ■ MERTK clinical study initiation - 1Q27

■ RHO clinical study initiation - 2H27 $3

$2

Base Case Assumptions: $12 Price Target $1

■ Early BEST1 readouts suggest activity. LCA5 readout is very positive with all or $0RESEARCH nearly all patients benefitting from treatment Jul-25 Aug-25 Sep-25 Oct-25 Nov-25 Dec-25 Jan-26 Feb-26 Mar-26 Apr-26 May-26 Jun-26 ■ OPGx-LCA5 - 70% PoS. 2028 market entry. Peak cumulative penetration of 100%.

Price of $1,750,000 per patient with prices increasing 3% annually. IRDS&P 500 (rebased)

Source: FactSet ■ OPGx-BEST1 - 75% PoS. 2028 market entry. Peak cumulative penetration of 80%.

Price of $1,750,000 per patient with prices increasing 3% annually.

■ OPGx-RHO - 30% PoS. 2030 market entry. Peak cumulative penetration of 80%. Company Description