UshStat (SAR421869) / Oxford Biomedica, Sanofi - LARVOL DELTA

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: Sanofi | Trial completion date: Jun 2032 ➔ Jun 2031 | Trial primary completion date: Jun 2032 ➔ Jun 2031

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

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: Sanofi | Trial completion date: Nov 2033 ➔ Jun 2031 | Trial primary completion date: Nov 2033 ➔ Jun 2031

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

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial primary completion date: Apr 2033 ➔ Apr 2034

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial completion date: Jan 2036 ➔ Nov 2033 | Trial primary completion date: Jan 2036 ➔ Nov 2033

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

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: Sanofi | N=18 ➔ 9

Enrollment change • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Oxford BioMedica | Trial completion date: Nov 2030 ➔ Jan 2032

Trial completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial primary completion date: Apr 2034 ➔ Mar 2035

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1 | N=18 | Enrolling by invitation | Sponsor: Oxford BioMedica

New P1 trial • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial primary completion date: Jan 2032 ➔ Jun 2022

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: Sanofi | Trial completion date: Jun 2031 ➔ Jun 2032 | Trial primary completion date: Jun 2031 ➔ Jun 2032

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

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial primary completion date: Oct 2032 ➔ Apr 2033

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P2 | N=9 | Active, not recruiting | Sponsor: Sanofi | Phase classification: P1/2 ➔ P2

Phase classification • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial completion date: Feb 2035 ➔ Jan 2036 | Trial primary completion date: Feb 2035 ➔ Jan 2036

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

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Sanofi | Trial primary completion date: Jun 2022 ➔ Oct 2032

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Oxford BioMedica | Trial primary completion date: May 2030 ➔ Jan 2032

Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: Oxford BioMedica | Phase classification: P1 ➔ P1/2

Phase classification • Inherited Retinal Dystrophy • Ophthalmology

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: Sanofi | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Inherited Retinal Dystrophy • Ophthalmology

Six-year results of a Phase I/IIa gene therapy trial in patients with Usher Syndrome Type-1B (USH1B) (ARVO 2024) - P1/2 | "Lower doses of EIAVMYO7A appear to be well-tolerated, but there were 2 SAEs in the highest dose cohort. Larger studies would be required to fully assess the potential of lentivirus-based retinal gene therapy, however SAR421869 was discontinued early due to the risk/benefit profile."

Clinical • Gene therapy • P1/2 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • Uveitis

Oxford Biomedica: Jefferies Healthcare Conference (Oxford Biomedica) - Anticipated end of P1/2 trial (NCT01505062) in Usher syndrome type 1B in 2017/2018

Anticipated trial completion date • Ophthalmology

Study of SAR421869 in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2; N=9; Terminated; Sponsor: Sanofi; Trial completion date: Apr 2017 ➔ Aug 2019; Trial primary completion date: Apr 2017 ➔ Aug 2019

Clinical • Trial completion date • Trial primary completion date

SAR421869: Data from P1/2a trial (NCT01505062) for Usher syndrome type 1B in 2021 (Sanofi) - Q3 2019 Results: Data from P2b of P1/2 trial (NCT02065011) for Usher syndrome type 1B in 2033

P1/2 data • P2 data

Study of SAR421869 in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B (clinicaltrials.gov) - P1/2; N=9; Terminated; Sponsor: Sanofi; N=27 ➔ 9; Recruiting ➔ Terminated; Study stopped not for safety reasons. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.

Clinical • Enrollment change • Trial termination

The Usher Syndrome, a Human Ciliopathy (PubMed, Klin Monbl Augenheilkd) - "Currently, gene-based therapy concepts, such as gene addition, applications of antisense oligonucleotides and TRIDs ("translational readthrough inducing drugs") for the readthrough of nonsense mutations are preclinically evaluated. For USH1B/MYO7A the UshStat gene therapy clinical trial is ongoing."

Journal

SAR421869: Data from P1/2a trial (NCT01505062) for Usher syndrome type 1B in 2021 (Sanofi) - Q1 2019 Results: Data from P2b trial (NCT02065011) for Usher syndrome type 1B in 2033

P1/2 data • P2b data

Oxford BioMedica provides update on programmes outlicensed to Sanofi (Oxford Biomedica) - "Oxford BioMedica...has been informed that as part of a company-wide portfolio review, Sanofi intends to seek a new partner for OXB’s outlicensed ophthalmology gene therapies, SAR422459 for Stargardt diseaseand SAR421869 for Usher’s Syndrome type 1b...Sanofi has confirmed that it will continue with its ongoing Phase I/II proof-of-concept study with SAR422459, while it will not continue to develop SAR421869. Sanofi is currently in discussions with potential external partners to continue the further development of both of these programmes."

Licensing / partnership