Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Pepinemab
VX15/2503 · 4 trials · 4 indications
If primary outcome, listed below (Outcome 3), does not reach its critical p-value, the secondary outcomes will not be formally tested. If both co-primary outcomes are statistically significant, the five secondary outcomes will be formally tested following a hierarchical testing procedure.
Measured by drug related adverse event frequency and laboratory test abnormalities in all subjects.
Co-primary outcome measured by the change from baseline in the Huntington's Disease Two-item Cognitive Family (PTAP and OTS) selected from the Huntington's Disease
TEAEs are defined as Adverse events (AEs) with onset after date-time of first dose, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP.
Specimens from surgical resection will be collected under the guidance of the collaborating pathologist and tissue procurement staff at the time of surgery to capture tissue in excess of that needed for clinical staging and diagnostic purposes. Tumor specimens will be fixed in 10% formalin, embedded in paraffin and biomarkers related to activated HNSCC stroma will be assessed via immunohistochemical analysis.
Blood samples will be collected and peripheral blood mononuclear cells will be isolated from these samples and stored in -80°C freezers until analysis. Multi-parameter flow cytometry will be utilized to phenotype immune effector cells. The impact of treatment on the circulating percentage of immune suppressor subsets that may serve as predictors of response to immune checkpoint inhibition. All data will be expressed as the percentage of total circulating peripheral blood cells with each respective phenotype.
Blood samples will be collected and peripheral blood mononuclear cells will be isolated from these samples and stored in -80°C freezers until analysis. Blood will be analyzed by multi-parameter flow cytometry to identify systemic phenotypic shifts mediated by VX15/2503 in combination with immune checkpoint inhibition.
| Arm | Type | Description |
|---|---|---|
| VX15/2503 | EXPERIMENTAL | The study drug VX15/2503 will be administered via monthly intravenous infusions |
| Placebo | PLACEBO_COMPARATOR | A placebo control will be administered via monthly intravenous infusions |
| pepinemab 40mg/kg | EXPERIMENTAL | The study drug, pepinemab, will be administered via monthly intravenous infusions. |
| Group A (VX15/2503) | EXPERIMENTAL | Patients receive VX15/2503 IV over 60 minutes on day 1. Beginning days 17-36, patients undergo standard of care surgery. |
| Group B (VX15/2503, ipilimumab) | EXPERIMENTAL | Patients receive VX15/2503 IV over 60 minutes and ipilimumab IV over 90 minutes on day 1. Beginning days 17-36, patients undergo standard of care surgery. |
| Group C (VX15/2503, nivolumab) | EXPERIMENTAL | Patients receive VX15/2503 IV over 60 minutes and nivolumab IV over 60 minutes on day 1. Beginning days 17-36, patients undergo standard of care surgery. |
| Group D (nivolumab) | EXPERIMENTAL | Patients receive nivolumab IV over 60 minutes on day 1. Beginning days 17-36, patients undergo standard of care surgery. |
| Group E (ipilimumab) | EXPERIMENTAL | Patients receive ipilimumab IV over 90 minutes on day 1. Beginning days 17-36, patients undergo standard of care surgery. |
| Group F (no treatment) | NO_INTERVENTION | Patients undergo standard of care surgery. |
| Name | Type | Description |
|---|---|---|
| VX15/2503 | DRUG | VX15/2503 (pepinemab) is a humanized IgG4 monoclonal antibody and will be administered intravenously at a dose of 20 mg/kg. The antibody is formulated at 20 mg/mL in 20 mM Sodium Acetate buffer, pH 5.4, containing 130 mM Sodium Chloride and 0.02% Polysorbate 80. |
| Placebo | DRUG | Placebo consists of formulation buffer only which is 20 mM Sodium Acetate buffer, pH 5.4, containing 130 mM Sodium Chloride and 0.02% Polysorbate 80. |
| Pepinemab | DRUG | Pepinemab is a humanized IgG4 monoclonal antibody. The antibody is formulated at 20 mg/mL in 20 mM Sodium Acetate buffer, pH 5.4, containing 130 mM Sodium Chloride and 0.02% Polysorbate 80 |
| Ipilimumab | BIOLOGICAL | Given IV |
| Nivolumab | BIOLOGICAL | Given IV |
Inclusion Criteria Highlights: 1. Male or female and are at least greater than or equal to 21 years of age at Screening. 2. Must fulfill one of the following criteria at Screening: 1. Late prodromal HD as defined by a CAP score of greater than 200 and DCL 2 or 3. 2. Early manifest HD as defi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| uniQure N.V. | QURE | 2 | PHASE2 | AMT-130 |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, SPK-10001, RG6496 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |