Company: SUPN
Filing Date: 2025-02-25
Form Type: 10-K
Source: 0001356576-25-000017
Chunk: 603

Company: SUPERNUS PHARMACEUTICALS, INC.
Filing Date: 2025-02-25
Form: 10-K
Item: Item 1
Chunk 603
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 placebo (n=51) with 0.9 hours. The reduction in daily "OFF" time was accompanied by a similar significant increase in daily GOOD ON time (2.8 hours for ONAPGO-treated patients compared to 1.1 hours for the placebo group; p=0.0188). In addition, numerically greater improvements in daily "OFF" time and daily GOOD ON time were seen as early as the first week of treatment and were maintained throughout all measured timepoints. Additionally, ONAPGO-treated patients more frequently reported improvement in their state of general health compared with placebo-treated patients (PGIC: 79% vs. 24%; p<0.0001). The most common adverse events (≥10% incidence) were infusion-site nodule, nausea, somnolence, infusion-site erythema, dyskinesia, headache, and insomnia.

SPN-817 (huperzine A)

SPN-817 represents a novel MOA for an anticonvulsant. SPN-817 is a novel synthetic form of huperzine A, whose MOA includes potent acetylcholinesterase inhibition, with pharmacological activities in CNS conditions such as epilepsy. The development will initially focus on the drug's anticonvulsant activity, which has been shown in preclinical models to be effective 

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for the treatment of partial seizures and Dravet Syndrome. SPN-817 has received Orphan Drug Designation for both Dravet Syndrome and Lennox-Gastaut Syndrome from the FDA.

We are focused on completing and optimizing the synthesis process of the synthetic drug as well as developing a novel dosage form. Given the potency of SPN-817 (huperzine A), a novel extended-release oral dosage form is critical to the success of this program because initial studies with the immediate-release formulations of non-synthetic SPN-817 (huperzine A) have shown serious dose-limiting, side effects.

We are conducting an open-label Phase 2a clinical study of SPN-817 in patients with treatment-resistant seizures. The first part of this study (Stage A) has completed dosing in the titration and maintenance  periods. We have completed enrollment of Stage A and announced in     November 2024 the topline data from all subjects with focal seizures who received the 3mg and 4mg twice daily doses, completed the maintenance period (n=10), and enrolled in the post-maintenance extension