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VY-AADC01 · 3 trials · 9 indications
Grading will be assessed using NCI CTCAE, version 4.03.
Safety of VY-AADC01 will be assessed by any clinically significant abnormalities on MRI scans as compared to Baseline.
Safety of VY-AADC01 will be assessed by routine physical examinations.
Safety of VY-AADC01 will be assessed by routine clinical laboratory analysis.
C-SSRS is a standardized suicidal rating system.
Safety and Tolerability of AADC Gene Transfer assessed by Adverse Events and Serious Adverse Events.
| Arm | Type | Description |
|---|---|---|
| VY-AADC02 (NBIb-1817) | EXPERIMENTAL | Single administration of up to 3.6 x 10\^12 vector genomes (vg) of VY-AADC02 |
| Sham (Placebo) Surgery | PLACEBO_COMPARATOR | Sham surgical procedure |
| VY-AADC01 Single Dose | EXPERIMENTAL | 9.4 x 10\^12 vector genomes of VY-AADC01 |
| VY-AADC01 Dose 1 | EXPERIMENTAL | 7.5 x 10\^11 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 2 | EXPERIMENTAL | 1.5 x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 3 | EXPERIMENTAL | 4.7x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| Name | Type | Description |
|---|---|---|
| VY-AADC02 | BIOLOGICAL | Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain |
| Sham (Placebo) Surgery | OTHER | Bilateral partial burr/twist holes without dura penetration |
| VY-AADC01 | DRUG | Single dose, neurosurgically infused, bilaterally into the striatum. |
Key Inclusion Criteria: 1. Males and females, 40 to 75 years of age (inclusive) 2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria 3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale...
VY-AADC01 is an investigational gene therapy being studied for the treatment of Parkinson's disease, including idiopathic Parkinson's disease. It is administered to patients aged 40 years and older. The drug is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
VY-AADC01 is being developed by Neurocrine Biosciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol NBIX. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational gene therapy for Parkinson's disease.
VY-AADC01 is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total enrollment of 29 participants. The drug remains investigational and has not received FDA approval. All completed trials were uncontrolled, meaning they did not include a placebo or comparator group.
VY-AADC01 has been studied in two completed Phase 1 trials. NCT01973543 enrolled 15 participants with Parkinson's disease, and NCT03065192 enrolled 16 participants with advanced Parkinson's disease. Both trials were conducted in the United States and included patients aged 40 years and older.
VY-AADC01 and VY-AADC02 are separate investigational gene therapies. VY-AADC01 has been studied in two Phase 1 trials for Parkinson's disease. VY-AADC02 was evaluated in a separate Phase 1 trial (NCT03562494) for Parkinson's disease with motor fluctuations, which enrolled 14 participants.