Synlogic Touts Positive Data From Rare Inherited Disorder Trial, Pivotal Study To Start Next Year

Synlogic Inc SYBX 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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