Approval Probability
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Adjusted LOA
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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 genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
UX016 targets sialic acid, a molecule involved in cellular processes. By modulating sialic acid, the drug aims to address the underlying deficiency associated with GNE Myopathy, though its exact mechanism is still under investigation in 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 on the NASDAQ exchange.
UX016 is currently in Phase 1 clinical development. It is an investigational drug and has not yet received regulatory approval. The ongoing Phase 1 trial is designed to evaluate the drug's safety, tolerability, and pharmacokinetics in patients with GNE Myopathy.
UX016 is being studied in a Phase 1 first-in-human trial with the identifier NCT07511556. This randomized, double-blind, placebo-controlled study is recruiting approximately 24 adult participants with GNE Myopathy in the United States.
No alternative names for UX016 have been disclosed. It is identified solely by its development code UX016 and is being investigated as a distinct therapeutic candidate for GNE Myopathy.