Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 gene therapy being developed for X-linked retinitis pigmentosa, a rare inherited retinal disease that causes progressive vision loss. It is designed to address the underlying genetic cause of the condition. BIIB112 is currently in clinical development and has not been approved by regulatory authorities.
BIIB112 is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. Biogen is conducting clinical research to evaluate the safety and efficacy of BIIB112 in patients with X-linked retinitis pigmentosa.
BIIB112 is in Phase 1 clinical development. A Phase 1 trial of BIIB112 has been completed, and the drug remains investigational. It has not received regulatory approval and is not yet available as a marketed therapy.
BIIB112 has been studied in a completed Phase 1 clinical trial registered as NCT03116113. This trial evaluated retinal gene therapy for X-linked retinitis pigmentosa and enrolled 50 male participants aged 10 years and older at sites in the United States and United Kingdom.
BIIB112 is a retinal gene therapy specifically developed for X-linked retinitis pigmentosa. The completed Phase 1 trial NCT03116113 was titled 'A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112,' confirming that BIIB112 is the gene therapy being tested for this condition.