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EryDex Low dose DSP · 1 trial · 2 indications
The International Cooperative Ataxia Rating Scale (ICARS) was an assessment of the degree of impairment in patients with cerebellar ataxia and was administered in its entirety; however, the primary efficacy assessment was based on the modified (m)ICARS, which excluded the Oculomotor domain (items 17 to 19) and items 8 to 12 of the Kinetic Functions domain of the ICARS. The mICARS was a 54 points maximum score (min 0) questionnaire divided into 3 sections: * Posture and Gait Disturbance section-7 items (min score 0, max score 34) * Kinetic Function-2 items (min score 0, max score 12) * Speech Disorder- 2 items (min score 0, max score 8). The assessment was designed to be completed within 30 minutes, and higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score.
| Arm | Type | Description |
|---|---|---|
| EryDex Low Dose DSP | EXPERIMENTAL | EDS-EP dose range of \~5-10 mg DSP/infusion. This study treatment was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). Other Names: EryDex System end product Low dose DSP |
| EryDex High Dose DSP | EXPERIMENTAL | EDS-EP dose range of \~14-22 mg DSP/infusion. This study treatment was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). Other Names: EryDex System end product High dose DSP |
| Placebo | PLACEBO_COMPARATOR | Patients were treated with autologous erythrocytes prepared with the EDS process using a placebo solution. Placebo was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). |
| Name | Type | Description |
|---|---|---|
| EryDex Low dose DSP | DRUG | EDS-EP dose range of \~5-10 mg DSP/infusion |
| EryDex High dose DSP | DRUG | EDS-EP dose range of \~14-22 mg DSP/infusion |
| Pooled Placebo | DRUG | EDS processed autologous erythrocytes using a sodium chloride \[NaCl\] solution. |
Inclusion Criteria: 1. Patient met clinical criteria for diagnosis of A-T. The neurological signs of A-T (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must have been documented. Such signs of A-T illustrated the body sys...
EryDex Low dose DSP is an investigational small molecule being developed for the treatment of ataxia telangiectasia, a rare genetic nervous system disease. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
EryDex Low dose DSP is a formulation of dexamethasone sodium phosphate encapsulated in red blood cells. The drug is designed to deliver the corticosteroid dexamethasone intracellularly, potentially reducing systemic side effects while targeting the neurological manifestations of ataxia telangiectasia.
EryDex Low dose DSP is being developed by Quince Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol QNCX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with ataxia telangiectasia.
EryDex Low dose DSP is in Phase 3 clinical development. A Phase 3 trial has been completed, but the drug remains investigational and is not yet approved for commercial use. The completed trial enrolled 176 participants across multiple countries.
EryDex Low dose DSP has one completed Phase 3 clinical trial, identified as NCT02770807, titled 'Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients.' The randomized, double-blind, placebo-controlled study enrolled 176 patients aged 6 years and older across 12 countries.
EryDex Low dose DSP is a specific formulation of dexamethasone sodium phosphate encapsulated within red blood cells. This encapsulation approach is designed to alter the drug's distribution and potentially improve tolerability compared to standard dexamethasone, though both contain the same active pharmaceutical ingredient.