Synlogic Reports Positive Top-line Data from Phase 1/2a Study of SYNB1618 in Patients with Phenylketonuria and Guides to Next Phase of Development.
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- US-based clinical stage company Synlogic, Inc., (NASDAQ: SYBX) has received positive top-line clinical data from patient cohorts of a randomised, double-blind, placebo-controlled Phase 1/2a study of SYNB1618, which is being developed for the treatment of phenylketonuria, the company said.
The study's primary objectives were to evaluate safety and tolerability of an early liquid formulation. Exploratory outcomes were related to the assessment of the pharmacodynamic effects of SYNB1618, including measurement of previously identified biomarkers related to SYNB1618's engineered ability to consume phenylalanine.
A statistically significant increase in biomarkers of SYNB1618 activity was observed in SYNB1618-treated subjects but not in those treated with placebo.
The full data set from the Phase 1/2a study will be discussed in an oral presentation at the upcoming annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) to be held in Rotterdam, September 3-6, 2019.
Synlogic has regulatory alignment for plans to initiate a randomised, double-blind, placebo controlled bridging study of a new solid oral formulation of SYNB1618 in healthy volunteers.
In this bridging study, Synlogic plans to select optimal doses of SYNB1618 for an efficacy study in patients with PKU designed to evaluate blood Phe-lowering. Synlogic has developed a robust and reproducible process and manufactured a new solid formulation of SYNB1618 that maintains strain viability and activity.
The solid oral formulation with improved quality attributes may enable dosing to higher activity. In addition, the convenience of a solid SYNB1618 formulation enables a larger out-patient efficacy study in patients. Synlogic expects to initiate the SYNB1618 efficacy study in the first half of 2020.
Synlogic's Phase 1/2a trial was a randomised, double-blind, placebo-controlled study of an orally administered early liquid formulation of SYNB1618.
The study had two parts; the first evaluated ascending doses of SYNB1618 or placebo administered on a single day and multiple ascending doses administered over seven days in healthy volunteers. The second part evaluated SYNB1618 as a single dose and as multiple doses in patients with PKU.
The study's primary objectives were to evaluate safety and tolerability of SYNB1618. Exploratory endpoints assessed the pharmacodynamic effects of SYNB1618, including measurement of biomarkers related to SYNB1618's engineered ability to consume Phe. The Phase 1/2a study was not designed or powered to measure Phe-lowering in SYNB1618-treated subjects.
The results of the HV part of this study were reported in September 2018. These data demonstrated that SYNB1618 was safe and established a dose that was well-tolerated for evaluation in patients with PKU.
The secondary objective was to characterize the microbial kinetics of SYNB1618 in feces, as measured by qPCR. Exploratory outcomes were related to the assessment of the pharmacodynamic effects of SYNB1618, including measurement of biomarkers related to SYNB1618 activity and, importantly, confirming that both pathways engineered to consume Phe are operational within the human GI tract.
Synlogic is developing a novel class of living medicines, Synthetic Biotic medicines, based on its proprietary drug development platform.
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|Article Type:||Clinical report|
|Date:||Jul 16, 2019|
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