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Also known as DYNE-101, zeleciment basivarsen (DYNE-101)
Zeleciment basivarsen · 2 trials · 5 indications
| Arm | Type | Description |
|---|---|---|
| Placebo-Controlled Period: DYNE-101 | EXPERIMENTAL | Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. |
| Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. |
| Long-Term Extension Period: DYNE-101 | EXPERIMENTAL | Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks. |
| MAD Cohort: Placebo-Controlled Period: DYNE-101 | EXPERIMENTAL | Participants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks. |
| MAD Cohort: Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks. |
| MAD Cohort: Treatment Period: DYNE-101 | EXPERIMENTAL | Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks. |
| MAD Cohort: Long-Term Extension Period: DYNE-101 | EXPERIMENTAL | Participants will receive DYNE-101, Q4W or Q8W for up to 208 weeks. |
| Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101 | EXPERIMENTAL | Participants will receive DYNE-101, Q8W for up to 24 weeks. |
| Dose Expansion Cohort: Placebo-Controlled Period: Placebo | EXPERIMENTAL | Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks. |
| Dose Expansion Cohort: Treatment Period: DYNE-101 | EXPERIMENTAL | Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks. |
| Dose Expansion Cohort: Long-Term Extension Period: DYNE-101 | EXPERIMENTAL | Participants will receive DYNE-101, Q8W for up to 208 weeks. |
| Name | Type | Description |
|---|---|---|
| zeleciment basivarsen (DYNE-101) | DRUG | Administered by IV infusion |
| Placebo | DRUG | Administered by IV infusion |
| DYNE-101 | DRUG | Administered by IV infusion |
Inclusion Criteria: * Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable. * Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the...
Zeleciment basivarsen is being developed for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare genetic disorder also known as Steinert disease. It is an investigational RNA therapy and has not been approved by the FDA. It is currently in Phase 3 clinical development.
Zeleciment basivarsen targets DMPK, the gene that produces the dystrophia myotonica protein kinase. By targeting DMPK RNA, the therapy aims to reduce the toxic RNA species that cause the symptoms of Myotonic Dystrophy Type 1. It is an RNA-based therapeutic.
Zeleciment basivarsen is being developed by Dyne Therapeutics, Inc., which trades on the Nasdaq under the ticker DYN. Dyne Therapeutics is a clinical-stage biopharmaceutical company focused on rare diseases.
Zeleciment basivarsen is in Phase 3 clinical development for Myotonic Dystrophy Type 1. A Phase 3 trial is currently recruiting, and a Phase 1 trial is active but not recruiting. The drug has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA.
Zeleciment basivarsen is being evaluated in two clinical trials. The Phase 3 trial, NCT07486934, is recruiting 150 participants with DM1 across multiple countries. The Phase 1 trial, NCT05481879, has completed enrollment with 127 participants and is active but not recruiting.
Yes, Zeleciment basivarsen is also known as DYNE-101. DYNE-101 is the investigational code name used by Dyne Therapeutics for this RNA therapy targeting DMPK for Myotonic Dystrophy Type 1. Both names refer to the same drug candidate.