VP-001 / PYC Therap - LARVOL DELTA

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