Synlogic Touts Positive Data From Rare Inherited Disorder Trial, Pivotal Study To Start Next Year
- Synlogic Inc announced topline data from the Phase 2 Synpheny-1 study in phenylketonuria (PKU), a rare inherited disorder that causes an amino acid called phenylalanine to build up in the body.
- Based on the results, the company also confirmed that SYNB1934 will be the drug candidate progressing to the Phase 3 registrational study expected to begin in H1 2023.
- The Phase 2 study enrolled 20 patients with PKU; 11 were in the SYNB1618 arm, and nine were in the SYNB1934 arm.
- Both strains demonstrated clinically meaningful reductions in fasting plasma Phe. On an "all comers" basis, the day 14 mean change from baseline in fasting plasma Phe was -20% for SYNB1618 and -34% for SYNB1934.
- Results were consistent and positive across all measured activity indicators for both drug candidates, consistent with previously shared results in healthy volunteers.
- Results from patients already taking sapropterin (Kuvan) at baseline and then receiving SYNB1618 and SYNB1934 were consistent with the overall efficacy profile, demonstrating the potential for adjunctive use.
- All adverse events were mild or moderate in severity and predominantly gastrointestinal (GI). There were no serious adverse events (SAEs).
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