City Therapeutics (CTY) investor relations material

Pipeline and near-term catalysts

• Three clinical-stage programs use GalNAc-delivered siRNA; Factor XI is in Phase I, RBP4 began Phase I dosing, and a CTA for anemia of chronic disease is planned by year-end 2026.

• Factor XI Phase I data are planned for ASH in December 2026; RBP4 Phase I data are expected mid-2027, with Part A and B data targeted by year-end 2027.

Stargardt and RBP4

• Stargardt affects ~30,000 U.S. patients; ABCA4 mutations drive toxic vitamin A derivative buildup, and tinlarebant showed 36% slowing of lesions.

• Proposed cityRBP4 profile is a physician-administered subcutaneous dose every six months; Phase I will assess safety and PK/PD, including RBP4 knockdown and correlated vitamin A delivery.

• Two dose levels will target RBP4 inhibition comparable to tinlarebant's average inhibition and a higher level; Part A is a single-ascending-dose study in healthy participants, followed by Part B in Stargardt patients.

• Part C multiple-ascending-dose study and an open-label extension are planned; follow-up is expected to generate efficacy and imaging data over the next three years.

• Delayed dark adaptation was characterized as a modest, incremental effect; Belite reported roughly five additional minutes for full dark adaptation, and two Phase III discontinuations were unrelated to it.

Factor XI

• Target is genetically validated; the proposed positioning is for patients unable to take ELIQUIS due to comorbidities, rather than an initial head-to-head effort against it.

• Target profile is 300 mg subcutaneous dosing every six months with at least 95% Factor XI inhibition; Phase I data at ASH will show progress toward that knockdown goal.

• Phase II is planned as a total knee arthroplasty trial starting mid-2027.

• A partnership is being considered within the next year; potential structures include milestones and royalties, a NewCo spinout, or a 50/50 model, with substantial capital needed for multiple Phase III trials.