Synlogic Announces Data Presentations at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting
These findings build on positive top-line data from Synpheny-1 presented by
“We were delighted to review these encouraging findings from our two rare metabolic disease programs among the expert metabolic clinicians who attend the SIMD meeting,” said
Highlights from the Synpheny-1 PKU Phase 2 Study Results
The Synpheny-1 results were presented by Dr.
Synpheny-1 included 20 patients with PKU. Results presented included successfully meeting the primary endpoint (change in area under the curve of D5-Phe following a meal challenge) with D5-Phe reductions of –33.8% and –42.9% for SYNB1618 and SYNB1934, respectively. The study also confirmed greater activity in PKU patients for SYNB1934, the next-generation Synthetic Biotic designed to consume Phe, as compared to first-generation candidate SYNB1618.
Highlights from results for SYNB1934 in the presentation include:
- Evidence of Phe metabolism by SYNB1934 in all patients, based on production of strain specific biomarkers (D5-TCA and D5-HA);
- Plasma Phe reduction of -53% among responders (defined as >20% reduction in Phe from baseline); response rate was 60% (3 of 5 patients);
- Data included one patient who was taking Kuvan at baseline; this patient achieved an additional reduction in plasma Phe of -80% compared to baseline; and
- Adverse events (AEs) were either mild or moderate in severity and mostly gastrointestinal in nature, consistent with those of a probiotic
“As the lead investigator for Synlogic’s PKU Phase 2 Synpheny-1 study, I was pleased to see the significant levels of interest in the additional results from the study presented during the SIMD meeting,” said
Highlights from Data Presented from the HCU Program
The company also shared additional clinical and preclinical data for SYNB1353 at the SIMD Annual Meeting. Clinical data presented included positive data from the Phase 1 study evaluating SYNB1353 in healthy volunteers using a dietary model of HCU. SYNB1353 is a Synthetic Biotic designed to consume methionine (Met), a precursor to homocysteine (HCy), as a potential treatment for HCU. A Met-restricted diet is a standard means of reducing total homocysteine (tHcy) levels, and associated risk of life-threatening complications, in patients with HCU. Study findings demonstrated proof of mechanism through the lowering of plasma Met levels and mechanistic support through the assessment of strain biomarkers of activity. Preclinical data include evidence of Met enterorecirculation and significant blunting of plasma D4-Met and plasma D4-tHCy with SYNB1353 in a mouse model of cystathionine beta-synthase (CBS) deficiency.
Posters presented at the SIMD meeting are posted on the Publications page of the
About the PKU Program & Phase 2 Synpheny-1 Study
Synlogic’s PKU program has included four clinical trials and dosing experience in 240 individuals and more than 30 PKU patients to date. The Phase 2 Synpheny-1 study was a Phase 2, open-label, 28-day study to assess safety, tolerability and efficacy of two Synthetic Biotics designed to metabolize Phe: the first-generation SYNB1618 and the next-generation SYNB1934. Synpheny-1 enrolled 20 adult patients with PKU, 11 in the SYNB1618 arm and nine in the SYNB1934 arm. The study’s objective was to confirm metabolism of Phe in PKU patients via the primary endpoint of a change in area under the curve (AUC) of plasma levels of labeled D5-phenylalanine (D5-Phe) after a meal challenge before and after the treatment period, a specific indicator of the ability of each investigational therapy to consume Phe. Dietary intake of Phe was carefully managed during the study to match patients’ usual protein and Phe intake.
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, clinical development plans, future financial position, future revenue, projected expenses, prospects, plans and objectives of management are forward-looking statements. In addition, when or if used in this press release, the words "may," "could," "should," "anticipate," "believe," "look forward, " "estimate," "expect," "intend," "on track, " "plan," "predict" and similar expressions and their variants, as they relate to Synlogic, may identify forward-looking statements. Examples of forward-looking statements, include, but are not limited to, statements regarding the potential of Synlogic's approach to Synthetic Biotics to develop therapeutics to address a wide range of diseases including: inborn errors of metabolism and inflammatory and immune disorders; our expectations about sufficiency of our existing cash balance; the future clinical development of Synthetic Biotics; the approach Synlogic is taking to discover and develop novel therapeutics using synthetic biology; and the expected timing of Synlogic's clinical trials of SYNB1934, SYNB1353, SYNB8802 and SYNB2081 and availability of clinical trial data. Actual results could differ materially from those contained in any forward-looking statements as a result of various factors, including: the uncertainties inherent in the clinical and preclinical development process; the ability of Synlogic to protect its intellectual property rights; and legislative, regulatory, political and economic developments, as well as those risks identified under the heading "Risk Factors" in Synlogic's filings with the U.S. Securities and Exchange Commission. The forward-looking statements contained in this press release reflect Synlogic's current views with respect to future events. Synlogic anticipates that subsequent events and developments will cause its views to change. However, while Synlogic may elect to update these forward-looking statements in the future, Synlogic specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Synlogic's view as of any date subsequent to the date hereof.
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Source: Synlogic, Inc.