VP-001 / PYC Therap - LARVOL DELTA

DINGO: A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 (clinicaltrials.gov) - P1/2 | N=16 | Recruiting | Sponsor: PYC Therapeutics | N=12 ➔ 16 | Trial completion date: Aug 2027 ➔ Mar 2028 | Trial primary completion date: Jun 2027 ➔ Jan 2028 | Not yet recruiting ➔ Recruiting

Enrollment change • Enrollment open • Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

FDA ALIGNMENT ON REGISTRATIONAL TRIAL DESIGN IN BLINDING EYE DISEASE (PYC Therapeutics Press Release) - "Key outcomes of the meeting included confirmation by the FDA that: PYC’s proposal to include a sham control arm within the registrational trial is acceptable; PYC’s proposed inclusion and exclusion criteria for the study are acceptable; Either endpoint PYC is tracking in the ongoing Phase 1/2 studies can be nominated as the primary endpoint in the registrational study; and 24-months or more of data from the registrational study will be required to support the NDA...PYC will use this guidance to finalise its proposed registrational study design prior to seeking endorsement of the protocol by the FDA in H2 2025 and initiating the trial...Once final alignment with the FDA has been achieved, PYC will initiate the registrational trial for VP-001"

FDA event • New P2/3 trial • Retinitis Pigmentosa

Platypus: SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: PYC Therapeutics | Trial completion date: Mar 2025 ➔ Aug 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025

Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • PRPF31

A Phase 1B Multiple Ascending Dose Study of VP-001; a peptide conjugate of oligonucleotide designed to treat PRPF31-related Retinitis Pigmentosa (ARVO 2025) - "VP-001 is safe and well tolerated in participants that received 3 repeat intravitreal injections. Participants also showed improvement in macular sensitivity and low luminance visual acuity."

P1 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • PRPF3 • PRPF31

MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: PYC Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

DINGO: A Study of Two Doses of VP-001 Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy Previously Treated with VP001 (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: PYC Therapeutics

New P1/2 trial • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • PRPF31

Platypus: SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: PYC Therapeutics | Recruiting ➔ Active, not recruiting | Trial completion date: May 2024 ➔ Mar 2025 | Trial primary completion date: May 2024 ➔ Mar 2025

Enrollment closed • Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • PRPF31

MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby) (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: PYC Therapeutics

New P1 trial • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Phase 1 First in Human Study of VP-001; a peptide conjugate of oligonucleotide designed to treat Retinitis Pigmentosa Type 11 patients (ARVO 2024) - "A single intravitreal injection of VP-001 was safe and well tolerated at 3 µg and 10 µg doses. A 30-µg dose is also administered, and if shown safe at week 4, a 75-µg dose cohort may be added to the study based on recommendation by the SRC. Data from all 3 cohorts will be presented."

Clinical • P1 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • CNOT3 • PRPF3 • PRPF31

A first-in-class therapeutic for the treatment of visual loss in Retinitis Pigmentosa Type 11 patients (ARVO 2024) - "The therapeutic action and mutation agnostic nature of VP-001 has been validated at both the gene modulation and cellular level in RP11 patient-derived target cells. VP-001 is found to be safe, tolerable, and durable in preclinical animal studies. Integration of preclinical pharmacodynamic and pharmacokinetic data indicates VP-001 may elicit therapeutic benefit in RP11 patients."

Clinical • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • CNOT3 • PRPF3 • PRPF31