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 the treatment of GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM). It is designed to address the underlying sialic acid deficiency in this rare genetic muscle disorder. SA is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
SA targets the sialic acid pathway, providing sialic acid to compensate for the deficiency caused by mutations in the GNE gene. This deficiency leads to the muscle weakness and wasting characteristic of GNE Myopathy. By supplementing sialic acid, SA aims to restore normal cellular function and potentially slow disease progression.
SA is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare diseases. Ultragenyx is publicly traded on the NASDAQ under the ticker symbol RARE. The company is conducting clinical trials to evaluate the safety and efficacy of SA in patients with GNE Myopathy.
SA is in Phase 2 clinical development for the treatment of GNE Myopathy. It is an investigational drug, meaning it has not been approved by the FDA or any other regulatory agency. The Phase 2 trial has been completed, and the data will inform further development decisions.
SA has been studied in one clinical trial, NCT01830972, an open-label Phase 2 extension study. This trial evaluated higher doses of SA extended-release tablets and SA immediate-release capsules in patients with GNE Myopathy. The study enrolled 59 participants in the United States and Israel and has been completed.
SA is a form of sialic acid, specifically developed as a therapeutic agent. The clinical trial evaluated both sialic acid extended-release (SA-ER) tablets and sialic acid immediate-release (SA-IR) capsules. These formulations are designed to deliver sialic acid to patients with GNE Myopathy who have a deficiency in this compound.