Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as AAV8-RPGR
BIIB112 · 1 trial · 1 indication
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.
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.
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.
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.
| Arm | Type | Description |
|---|---|---|
| Part 1: BIIB112 Dose 1 | EXPERIMENTAL | Participants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0. |
| Part 1: BIIB112 Dose 2 | EXPERIMENTAL | Participants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0. |
| Part 1: BIIB112 Dose 3 | EXPERIMENTAL | Participants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0. |
| Part 1: BIIB112 Dose 4 | EXPERIMENTAL | Participants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0. |
| Part 1: BIIB112 Dose 5 | EXPERIMENTAL | Participants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0. |
| Part 1: BIIB112 Dose 6 | EXPERIMENTAL | Participants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0. |
| Part 2: BIIB112 High Dose | EXPERIMENTAL | Participants will receive a single high dose of BIIB112 by sub-retinal injection. |
| Part 2: BIIB112 Low Dose | EXPERIMENTAL | Participants will receive a single low dose of BIIB112 by sub-retinal injection. |
| Part 2: Untreated Group | NO_INTERVENTION | Participants will receive no intervention to allow for a controlled comparison. |
| Name | Type | Description |
|---|---|---|
| BIIB112 | BIOLOGICAL | Administered as specified in the treatment arm. |
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...
BIIB112 is an investigational monoclonal antibody being developed by Biogen Inc. for X-Linked Retinitis Pigmentosa, a rare inherited retinal disease. It has been studied as a retinal gene therapy approach in a Phase 1 clinical trial. BIIB112 is not approved and remains in clinical development.
BIIB112 is being studied for the treatment of X-Linked Retinitis Pigmentosa, a rare genetic eye condition that causes progressive vision loss. The Phase 1 trial evaluated the therapy in male patients aged 10 years and older with this condition. It is investigational and has not been approved for any use.
BIIB112 is developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen sponsored the Phase 1 clinical trial of BIIB112 in X-Linked Retinitis Pigmentosa. The company is the sole developer named for this asset.
BIIB112 is in Phase 1 clinical development. One Phase 1 trial has been completed, and no active trials are currently listed. BIIB112 is investigational and has not received FDA approval. It remains in early-stage clinical development for X-Linked Retinitis Pigmentosa.
BIIB112 has been studied in one clinical trial, NCT03116113, titled A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112. The Phase 1 trial enrolled 50 participants across the United States and the United Kingdom and is now completed. It was randomized, double-blind, and no-treatment controlled.
The Phase 1 trial NCT03116113 enrolled 50 male participants aged 10 years and older with X-Linked Retinitis Pigmentosa. It was a randomized, double-blind, no-treatment controlled study conducted in the United States and the United Kingdom. Healthy volunteers were not eligible, and the trial is completed.