Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07511556 | First-in-human Study of UX016 in GNEM | PHASE1 | NOT YET_RECRUITING | 24 | — | — | Oct 1, 2026 | Dec 1, 2028 | Apr 6, 2026 | 2 | United States |
| Arm | Type | Description |
|---|---|---|
| Lower Dose UX016 -> Extension Period | EXPERIMENTAL | Participants will be randomized 3:1. Those randomized to lower dose will receive UX016 daily following up to two single dose administrations. After 48 weeks of daily dosing, participants will be eligible to enter the extension period. |
| Higher Dose UX016 -> Extension Period | EXPERIMENTAL | Participants will be randomized 3:1. Those randomized to higher dose will receive UX016 daily following a single dose administration. After 48 weeks of daily dosing, participants will be eligible to enter the extension period. |
| Placebo -> Extension Period | PLACEBO_COMPARATOR | Participants will be randomized 3:1. Those randomized to placebo will receive placebo per the same Lower or Higher Dose UX016 cohorts. After 48 weeks of daily dosing, participants will be eligible to enter the extension period and receive UX016 at the cohort assigned dose level. |
| Name | Type | Description |
|---|---|---|
| UX016 | DRUG | Tablets for oral use |
| Placebo | OTHER | Tablets for oral use. Tablets will match the UX016 tablets, but contain no active ingredients |
Inclusion Criteria: * A confirmed diagnosis of GNEM (also known as hereditary inclusion body myopathy \[HIBM\], distal myopathy with rimmed vacuoles \[DMRV\], inclusion body myopathy 2 \[IBM2\], and Nonaka myopathy in Japan) by Clinical Laboratory Improvement Amendments (CLIA)-certified genetic tes...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |