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4D MOLECULAR THERAPEUTICS, INC.

(FDMT)
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4D Molecular Therapeutics : ASRS 2021 Annual Meeting – 4D-125 for XLRP Phase 1/2 Clinical Data – Dr. Cagri Besirli

10/10/2021 | 04:02pm EST

Phase 1/2 Clinical Trial of Intravitreal 4D-125 AAV Gene Therapy in Patients with Advanced XLRP:

Interim Safety & Preliminary Clinical Activity

CG Besirli, MT Mathias, PJ Francis, S Honarmand,

MS Ip, RY Kim, DH Kirn, and DG Birch

FINANCIAL DISCLOSURES

  • Cagri G Besirli, MD, PhD (presenter)
  1. Research: 4DMT, MeiraGTx, Janssen Pharmaceuticals, Spark Therapeutics, Regeneron, Genentech
  1. Consultant: MeiraGTx, Janssen Pharmaceuticals, iRenix Medical

o Equity ownership/Royalty: iRenix Medical, ONL Therapeutics

2

Key Takeaways for 4D-125 Preliminary Phase 1/2 Clinical Data

DATA CUT-OFF: SEPTEMBER 1, 2021

  • 4D-125was well-tolerated
  • No dose-limiting toxicities, serious adverse events or chronic inflammation
  • Evidence of clinical activity observed in the treated eye vs. the untreated eye in evaluable patients on three clinical activity endpoints:
  1. Increased mean retinal sensitivity by microperimetry in treated vs. untreated eye
  1. Increased number of loci gaining ≥ 7 dB sensitivity by microperimetry in treated vs. untreated eye

o Increased preservation of ellipsoid zone area by SD-OCT in treated vs. untreated eye (progression decreased)

3

4D-125: R100 Capsid for IVT Delivery of RPGR Transgene

INTRAVITREAL DELIVERY ENABLES TREATMENT OF BROAD RANGE OF PATIENTS, INCLUDING EARLIER STAGE

Photoreceptor-

specific RPGR

PROMOTER TRANSGENE

PRODUCT DESIGN

  • Vector: R100
  • Transgene: RPGR
  • Promoter: Photoreceptor-specific

Blocked by

Limited retinal

Conventional

barrier (e.g., ILM)

transduction

naturally

Retina

occurring

vectors

Heparan

Inner

sulfate

retina

Cytosol

Vitreous

body

Eye cross section

Inner retina

Photoreceptors RPE

Overcome

Enhanced retinal

barrier (e.g., ILM)

transduction

R100

Abbreviations: ILM, inner limiting membrane; RPE, retinal pigment epithelium.

4

4D-125 Study Design

OPEN-LABEL, PHASE 1/2 TRIAL IN ADULTS WITH XLRP

STUDY DESIGN

DOSE ESCALATION

DOSE EXPANSION

COHORT 1

COHORT 2

EXPANSION

3E11 vg/eye

1E12 vg/eye

1E12 vg/eye

n = 3

n = 3

n = 6-12

DSMC review

ASSESSMENT SCHEDULE

Biomarkers assessed by Independent Reading Center

KEY INCLUSION CRITERIA

  • Male 18 years of age
  • Hemizygous RPGR mutation
  • Clinical diagnosis of non-syndromic retinitis pigmentosa
  • Measurable ellipsoid zone line (EZL) on macular SD-OCT
  • >1 nonzero point on microperimetry (dose-escalation)OR
  • 1dB mean retinal sensitivity on microperimetry (dose- expansion)

PRIMARY ENDPOINT

  • Incidence and severity of TEAEs and SAEs, including clinically significant changes in safety parameters

KEY SECONDARY ENDPOINTS

  • Change from baseline in EZ area loss by SD-OCT over 12 months
  • Change from baseline in visual field sensitivity by microperimetry over 12 months

5

This is an excerpt of the original content. To continue reading it, access the original document here.

Disclaimer

4D Molecular Therapeutics Inc. published this content on 10 October 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 10 October 2021 20:01:02 UTC.


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