Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
UX016 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 1g UX016 -> Extension Period | EXPERIMENTAL | Participants will be randomized 3:1 to receive UX016 or placebo. Depending on sub-cohort assignment, participants may receive two single doses of UX016 (0.5g and 1g) or one single dose (1g) before starting daily dosing, or they may proceed directly to daily dosing at the assigned 1g dose level. After completing 48 weeks of daily dosing, participants will be eligible to enter the Extension Period, during which all participants will receive UX016. |
| 2g UX016 -> Extension Period | EXPERIMENTAL | Participants will be randomized 3:1 to receive UX016 or placebo. Depending on sub-cohort assignment, participants may receive a single dose of UX016 (2g) before starting daily dosing, or they may proceed directly to daily dosing at the assigned 2g dose level. After 48 weeks of daily dosing, participants will be eligible to enter the Extension Period, during which all participants will receive UX016. |
| Placebo -> Extension Period | PLACEBO_COMPARATOR | Participants will be randomized 3:1 to receive UX016 or placebo. Depending on sub-cohort assignment, participants may receive one or more single doses of placebo before starting daily dosing or they may proceed directly to daily dosing of placebo at the assigned dose level. After 48 weeks of daily dosing of placebo, participants will be eligible to enter the Extension Period, during which all participants will receive UX016. |
| 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 distal myopathy with rimmed vacuoles \[DMRV\], hereditary inclusion body myopathy \[HIBM\], 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 |
UX016 is an investigational small molecule being developed for the treatment of GNE Myopathy, a rare inherited muscle-wasting disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in a first-in-human trial to evaluate its safety and tolerability in patients.
The specific molecular target of UX016 has not been disclosed. It is a small molecule therapeutic being developed by Ultragenyx Pharmaceutical Inc. for GNE Myopathy, a rare genetic condition. The drug's mechanism of action is not publicly detailed, and it is currently being evaluated in early-stage clinical trials.
UX016 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare diseases. The company's stock is traded under the ticker symbol RARE. Ultragenyx is conducting a Phase 1 clinical trial of UX016 in patients with GNE Myopathy in the United States.
UX016 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory body. The ongoing Phase 1 trial is a first-in-human study designed to assess the safety, tolerability, and pharmacokinetics of UX016 in patients with GNE Myopathy.
UX016 is being studied in one active Phase 1 clinical trial with the identifier NCT07511556. This first-in-human study is recruiting 24 participants with GNE Myopathy in the United States. The trial is randomized, double-blind, and placebo-controlled, enrolling adults aged 18 years and older.
No alternative names for UX016 have been reported. It is a distinct investigational small molecule being developed by Ultragenyx Pharmaceutical Inc. for GNE Myopathy. As it is still in early clinical development, it is not yet marketed under any brand name.