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Sialic Acid · 2 trials · 3 indications
To evaluate the effect of SA-ER treatment on improvement of biochemical measures of sialylation and pathology in muscle. On mobility, strength, and function using a series of quantitative and physical performance measures and quality of life using patient-reported outcome measures.
Safety will be evaluated in terms of the incidence and frequency of treatment-emergent adverse events (TEAEs), including clinically significant changes from baseline to scheduled timepoints in clinical laboratory values, vital signs, or physical and neurologic examination findings. Medical history and reported clinical symptoms, including increasing muscle weakness, fatigue, or pain.
| Arm | Type | Description |
|---|---|---|
| 6,000 mg SA-ER | EXPERIMENTAL | Subjects will receive this dose for the duration of the study (total study duration of 48 weeks). |
| Placebo | PLACEBO_COMPARATOR | Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (48 weeks total study duration). |
| 3,000 mg SA-ER | EXPERIMENTAL | Subjects will receive this dose for the duration of the study (total study duration of 48 weeks). |
| 650 mg (single dose only) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 1,950 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 2,925 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 4,875 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 6,000 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| Name | Type | Description |
|---|---|---|
| Sialic Acid Extended Release (SA-ER) | DRUG | SA-ER will be administered in doses of 3000mg per day or 6000mg per day |
| Placebo | DRUG | Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (total study duration 48 weeks). |
| Sialic Acid Extended Release (SA-ER) Tablets | DRUG | Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis. |
| Sialic Acid Extended Release (SA-ER) Tables | DRUG | PPatients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis. |
Inclusion Criteria: 1. Must be between 18 and 65 years of age, inclusive. 2. Must be willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures. 3. Must have a documented diagnosis of GNE myopathy, HIBM...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
Sialic Acid is an investigational small molecule being developed by Ultragenyx Pharmaceutical Inc. for the treatment of GNE Myopathy and Hereditary Inclusion Body Myopathy (HIBM), rare inherited muscle disorders. It is currently in Phase 2 clinical development, meaning it is still being studied and is not yet approved for use.
Sialic Acid 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.
Sialic Acid is currently in Phase 2 clinical development. A Phase 2 study has been completed to evaluate the dose and pharmacodynamic efficacy of Sialic Acid-Extended Release (SA-ER) tablets in patients with GNE Myopathy or Hereditary Inclusion Body Myopathy. The drug remains investigational and has not received FDA approval.
Sialic Acid has been studied in two completed clinical trials. NCT01359319 was a Phase 1 safety and pharmacokinetics study in patients with Hereditary Inclusion Body Myopathy. NCT01517880 was a Phase 2 study evaluating dose and pharmacodynamic efficacy of Sialic Acid-Extended Release tablets in patients with GNE Myopathy or Hereditary Inclusion Body Myopathy.
Sialic Acid and SA-ER refer to related formulations of the same investigational drug. SA-ER stands for Sialic Acid-Extended Release, a tablet formulation used in the Phase 2 clinical trial NCT01517880. The Phase 1 trial used a different formulation of sialic acid tablets.