Latest transcript:
Investment Highlights
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A rare disease focused clinical stage biopharmaceutical company with a pipeline of novel product candidates that are intended
to address unmet need in a variety of metabolic diseases
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ZGN 1061 for type 2 diabetes with concomitant obesity and/or NASH
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Diabetes market evolving towards therapies with enhanced A1C lowering efficacy and positive effects on common co morbidities (ob esity/NAFLD)
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Phase 2 proof of concept trial demonstrated significant A1C lowering efficacy and weight loss, with a safety and tolerability pr ofile generally
comparable to placebo
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Nonclinical ZGN 1061 studies have demonstrated significant NASH model efficacy, and complementary efficacy in combination with a GLP 1
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Next milestone: Update on development plans by end of 3Q 2019
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ZGN 1258 for rare metabolic diseases, including Prader Willi syndrome
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Prader Willi syndrome is a rare, lethal disease emerging in childhood, with ~200,000 afflicted worldwide; no current therapeutic options available
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Development plans suspended due to unexpected finding in long term toxicology studies
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ZGN 1345 for metabolic liver disease
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Multiple metabolic related liver diseases exist with high unmet medical need, from NAFLD/NASH to hepatocellular carcinoma
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Compound is an orally dosed MetAP2i with high liver concentrations but minimal to no detectable systemic exposure; once daily do sing expected
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Named as development candidate 4Q 2018; nonclinical development work underway
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March 31, 2019 cash position of $105M; runway expected to extend through at least 2020
ZGN
1061 Next Steps
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Reviewing comprehensive data sets with diabetes/NASH KOLs and potential partners
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Important progress made toward addressing the FDA Clinical Hold:
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Received FDA Type A meeting minutes to previously announced clinical hold
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FDA acknowledged newly developed in vitro assays of human plasma coagulation and tissue factor expression qualitatively
differentiate ZGN 1061
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Working with FDA to translate in vitro data and confirm relevant safety margins in an in vivo model
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Exploring a second IND in a population with higher unmet medical need
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Company expects to provide an update on development plans by end of 3Q 2019
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Submitting abstracts to present full results of the Phase 2 clinical trial at upcoming medical meetings in 2019
ZGN
1258 Summary
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Unexpected finding observed in long term toxicology studies
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Degeneration and other anomalies in muscle tissue from 4 and 6 month rodent studies
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Observed in different degrees in both vehicle and all dose arms; finding more pronounced at higher doses
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Finding never observed previously, specific to ZGN 1258
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Not seen in prior ZGN 1258 rodent studies
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Not seen in any other species with ZGN 1258
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Not seen in any other Zafgen MetAP2i program in any long term toxicology study
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Plan to file an IND suspended while finding is investigated
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Company will provide an update at a later time, if warranted, following further evaluation
PWS Commitment Continues
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PATH for PWS natural history study collaboration continues
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Study being conducted in collaboration with the Foundation for
Prader Willi Research (FPWR) and National Organization for Rare
Disorders (NORD)
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PATH for PWS designed as 4 year / 500 participant non interventional
natural history study
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Enrollment kicked off at FPWR annual conference in October; >400
enrolled as of March 2019
ZGN
1345 Oral, Liver Focused MetAP2 Inhibitor
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ZGN 1345 advanced to development candidate status in 4Q 2018
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Orally dosed MetAP2i; expected once daily dosing
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High concentrations in the liver; minimal to no detectable exposure systemically
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High MetAP2 levels strongly correlated with more severe outcomes in advanced liver disease
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Positive early data in multiple nonclinical liver disease models with high unmet medical need; further nonclinical
studies ongoing
