Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SA · 1 trial · 2 indications
An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE (SAE) is an AE that at any dose results in any of the following: death, a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. TEAEs include all adverse events that start on or after the first dose of study medication, or adverse events that are present prior to the first dose of study medication, but their severity or relationship increases after the first dose of study medication up to and including 30 days after the final study medication dosing date. TEAEs were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). TEAE relationship to study medication was classified as not related, possibly related, or probably related.
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
| Arm | Type | Description |
|---|---|---|
| Crossover Participants | EXPERIMENTAL | Participants completing the 48-week study (UX001-CL201; NCT01517880) were enrolled into Part I of the study: * Part I: participants continued on 6 g/day SA-ER for 12 weeks * Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment 4 times per day \[QID\]) for 36 months * Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR) * Part IV: 6 g/day or 12 g/day SA (SA-ER only) |
| Naïve Participants | EXPERIMENTAL | Treatment naïve participants with GNE myopathy were enrolled into Part II of the study: * Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment QID) for 36 months * Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR) * Part IV: 6 g/day or 12 g/day SA (SA-ER only) |
| Name | Type | Description |
|---|---|---|
| SA-ER 500 mg | DRUG | oral tablets |
| SA-IR 500 mg | DRUG | oral capsules |
Inclusion Criteria: * Enrollment in, and successful completion of the UX001-CL201 (NCT01517880) protocol OR (for 10 treatment naïve subjects): * Have a confirmed diagnosis of GNE Myopathy * Aged 18 -65 years of age, inclusive * Able to walk ≥ 200 meters and \< 80% of predicted normal during ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
SA is an investigational small molecule being studied for GNE myopathy, a rare inherited muscle disorder also known as hereditary inclusion body myopathy (HIBM). It is being developed by Ultragenyx Pharmaceutical Inc. as a potential treatment for this condition, which affects skeletal muscle function.
SA is being developed by Ultragenyx Pharmaceutical Inc., which trades on the Nasdaq under the ticker RARE. The company is the sponsor of the clinical program evaluating SA for GNE myopathy, a rare disease indication.
SA is in Phase 2 clinical development for GNE myopathy. It has not been approved by the FDA and remains investigational. The Phase 2 program has completed, with one trial conducted across sites in the United States and Israel.
SA has been evaluated in one completed Phase 2 trial, NCT01830972. This was an open label extension study of higher dose sialic acid-extended release (SA-ER) tablets and sialic acid-immediate release (SA-IR) capsules in patients with GNE myopathy, enrolling 59 participants aged 18 years and older.
SA is a small molecule studied in GNE myopathy, a condition linked to mutations in the GNE gene that encodes glucosamine (UDP-N-acetyl)-2-epimerase. The Phase 2 trial NCT01830972 evaluated sialic acid-extended release tablets and sialic acid-immediate release capsules in patients with this disorder.