Synlogic Announces Publication of Preclinical and Clinical Data for SYNB1353 as a Potential Treatment for Classical Homocystinuria
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Insights
The recent findings regarding SYNB1353 represent a significant step forward in the treatment of classical homocystinuria (HCU), a rare metabolic disorder. The ability of SYNB1353 to degrade methionine effectively and prevent its conversion to homocysteine could be a game-changer for patients with HCU, who currently rely on strict dietary restrictions to manage their condition. The implications of these findings are substantial, as lowering plasma homocysteine levels is crucial to reducing the risk of thromboembolism, a common and serious complication of HCU.
From a medical research perspective, the tolerability of the treatment is equally noteworthy. The similarity in gastrointestinal adverse events reported between the SYNB1353 and placebo groups suggests that the treatment may have a side effect profile that is manageable in a clinical setting. This is particularly important for long-term therapies targeting chronic diseases. However, it is imperative to monitor long-term safety and efficacy in a larger, more diverse patient population to fully understand the therapeutic potential and any unforeseen risks.
For investors and stakeholders in the biopharmaceutical sector, the development of SYNB1353 by Synlogic could represent a significant advancement in the company's portfolio, particularly given the unmet need in the HCU market. The successful preclinical and early clinical results may position Synlogic as a leader in rare metabolic disease treatment, potentially leading to increased investor confidence and positive stock market activity surrounding the company's shares.
Nevertheless, it is important to consider the commercial potential of SYNB1353. Given the rarity of HCU, the market size is limited, which could affect the overall financial impact of the drug. However, Synlogic's approach to leveraging its platform technology across multiple rare metabolic diseases could enhance the value proposition for investors. The scalability of such a platform may lead to efficiencies in research and development, potentially resulting in a more robust pipeline and diversified risk.
In terms of health economics, the introduction of SYNB1353 could have a meaningful impact on the healthcare system by potentially reducing the long-term costs associated with the management of classical HCU. Current management strategies, which include lifelong dietary restrictions, are not only burdensome for patients but also come with significant healthcare and societal costs. A therapeutic option that can safely lower homocysteine levels could decrease the frequency of medical interventions and complications, such as thromboembolism, thus leading to improved quality of life and reduced healthcare spending.
However, the cost-effectiveness of SYNB1353 will need to be evaluated against existing management strategies. The pricing strategy for SYNB1353 will be a critical factor in its adoption. If priced appropriately, SYNB1353 could provide a cost-effective solution for HCU patients and payers, facilitating market penetration and uptake.
Findings support positive proof of mechanism, through successful degradation of methionine, precursor to homocysteine, using dietary disease model in preclinical models and healthy volunteer study
SYNB1353 was well-tolerated, with similar proportions in both the placebo and active arms reporting mild-moderate gastrointestinal adverse events
CAMBRIDGE, Mass., Feb. 02, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, announced today the publication of a manuscript detailing the development and activity of SYNB1353 in preclinical models and demonstrating safety, tolerability, and clinical proof of mechanism in healthy volunteers through the successful lowering of methionine (Met), a precursor to homocysteine, in a dietary model of classical homocystinuria (HCU).
Among findings outlined in the manuscript, SYNB1353 efficiently degraded both dietary and entero-recirculating methionine to prevent its absorption and subsequent conversion to homocysteine in preclinical models, suggesting that SYNB1353 should result in lowering of plasma homocysteine levels in HCU patients. In addition, in results from the clinical study in healthy volunteers, SYNB1353 was generally well-tolerated and adverse events were mild to moderate, transient, and predominantly gastrointestinal in nature. The proportion of subjects reporting gastrointestinal events were similar for the SYNB1353 and placebo cohorts (
“Given the significant disease burden and acute need for new treatment options for those affected by classical HCU, we are pleased to share these findings that highlight the potential of our novel approach, which targets methionine, an amino acid that has long been validated as a therapeutic target in classical HCU through the use of dietary restrictions,” said Mylène Perreault, Ph.D., Head of Research at Synlogic. “Importantly, the SYNB1353 program has built on learnings and progress from our other rare metabolic disease programs, including the design of the potential therapeutic, the preclinical research and the advancement in clinical development.”
The publication, entitled “The Live Biotherapeutic SYNB1353 Decreases Plasma Methionine via Directed Degradation in Animal Models and Healthy Volunteers,” and published in the peer-reviewed journal, Cell Host & Microbe, is now available online at https://www.sciencedirect.com/science/article/pii/S193131282400009X.
About Classical Homocystinuria (HCU) & SYNB1353
Classical homocystinuria (HCU) is a rare metabolic disease characterized by extreme levels of homocysteine caused by an inherited deficiency in the cystathionine beta-synthase (CBS) enzyme. When CBS is absent, homocysteine builds up, putting patients at risk of multisystem complications, including potentially life-threatening, acute thromboembolic events, optical damage from lens dislocation, skeletal deficiencies, and neurocognitive impairments. Methionine (Met), an essential amino acid in dietary protein, is a precursor to homocysteine, and a restrictive, low-Met diet is a standard treatment for lowering total homocysteine (tHcy). SYNB1353 is a novel, orally administered, non-systemically absorbed drug candidate designed to consume Met in the gastrointestinal tract, thereby lowering homocysteine levels in patients with HCU. It is the first drug candidate developed through a research collaboration between Synlogic and Ginkgo Bioworks and the first investigational medicine developed on Ginkgo's platform to enter the clinic. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation, Fast Track designation and Orphan Drug Designation (ODD) to SYNB1353 for the potential treatment of HCU. Synlogic holds worldwide development and commercialization rights to SYNB1353.
About Synlogic
Synlogic is a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases in need of new treatment options. The Company focuses on rare metabolic diseases, with its lead program, labafenogene marselecobac (SYNB1934), currently being studied in Synpheny-3, a global, pivotal Phase 3 study for patients with phenylketonuria (PKU), and SYNB1353, a potential treatment for classical homocystinuria (HCU). Both PKU and HCU are caused by inborn errors of metabolism, and present significant need for innovation due to limitations of today’s medical treatment options.
Synlogic’s early-stage pipeline includes research and development on product candidates addressing medical needs in enteric hyperoxaluria, gout, cystinuria, as well as inflammatory bowel disease (IBD). The Company’s productivity is fueled by a reproducible, proprietary approach that creates new enzymatic pathways designed to consume or produce specific biological targets provided in GI-restricted, orally administered biopharmaceuticals. Synlogic designs, develops and manufactures these drug candidates, which are produced by applying precision genetic engineering to well-characterized probiotics. For more information, please visit www.synlogictx.com or follow us on Twitter, LinkedIn, Facebook, Instagram, and YouTube.
Forward Looking Statements
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," “focused on,” "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 labafenogene marselecobac (previously known as 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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