Recent Updates
Recently added Catalysts

BIIB112

Phase 1

X-Linked Retinitis Pigmentosa | Monoclonal antibody | Ophthalmology |Biogen Inc.|Last Updated: Jan 18, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindNO_TREATMENT_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03116113A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112PHASE1 COMPLETED 50Mar 8, 2017Nov 18, 2020Jan 18, 20248 United States, United Kingdom
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs)
Up to Month 24

DLTs are defined as any of the following events considered to be related to study drug: Sustained decrease in best-corrected visual acuity (BCVA) of ≥30 letters on the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart compared to baseline (sustained is defined as lasting 48 hours or more until recovery, with recovery defined as visual acuity (VA) returning to within 10 letters of baseline VA. An exception is made for surgery-related events occurring in close temporal association {within \<24 hours} of the surgery); Vitreous inflammation, vitritis (\>Grade 3 using standardized Nussenblatt vitreous inflammation scale grading); Any clinically significant retinal damage observed that is not directly attributed to complications of surgery; Any clinically relevant suspected unexpected serious adverse reaction, with the exception of vision loss or vision threatening.

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Day 0 (surgery) in Part 1 of the study up to 24 months

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAEs are defined as the AEs starting or worsening on or after the day of the first surgery.

Part 2: Percentage of Study Eyes With ≥7 Decibels (dB) Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry
Month 12

MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labelled as '\< 0', '0', or a positive integer. The point labelled as '\< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more dBs in any 5 or more points out of the 16 central points.

Part 2: Number of Participants With TEAEs
Day 0 (surgery) in Part 2 of the study up to 12 months

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAEs are defined as AEs starting on or after the day of the first surgery.

Secondary Endpoints
Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 68 Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry
Months 1, 3, 6, 9, 12, 18, and 24
Part 1: Change From Baseline in Mean Sensitivity of the 16 Central Loci Points Assessed by MAIA Microperimetry
Baseline, Months 1, 3, 6, 9, 12, 18, and 24
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1: BIIB112 Dose 1EXPERIMENTALParticipants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0.
Part 1: BIIB112 Dose 2EXPERIMENTALParticipants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0.
Part 1: BIIB112 Dose 3EXPERIMENTALParticipants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0.
Part 1: BIIB112 Dose 4EXPERIMENTALParticipants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0.
Part 1: BIIB112 Dose 5EXPERIMENTALParticipants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0.
Part 1: BIIB112 Dose 6EXPERIMENTALParticipants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0.
Part 2: BIIB112 High DoseEXPERIMENTALParticipants will receive a single high dose of BIIB112 by sub-retinal injection.
Part 2: BIIB112 Low DoseEXPERIMENTALParticipants will receive a single low dose of BIIB112 by sub-retinal injection.
Part 2: Untreated GroupNO_INTERVENTIONParticipants will receive no intervention to allow for a controlled comparison.
Interventions
NameTypeDescription
BIIB112BIOLOGICALAdministered as specified in the treatment arm.
Unlock Study Design Details
Eligibility Criteria
Age Range10 Years — N/A
SexMALE
Healthy VolunteersNo
Study Sites8

Key Inclusion Criteria: Part 1: * Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation). * Participant with active disease clinically visible within the macular region in both eyes. Part 2: \- Participant with mean total retinal sensitivity in the study eye as assessed b...

Countries:United StatesUnited Kingdom
Unlock Eligibility Criteria