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Living with PKU, United States
PTC Therapeutics to Host First Deep Dive Webinar on Bio-e Platform
The Bio-e platform deep dive will provide an overview of the Bio-e platform and its novel scientific approach to treating central nervous system (CNS) and non-CNS diseases. Bio-e platform technology will be reviewed in detail and prior clinical results for its lead compound PTC743 will be highlighted. Expectations for upcoming trials will be outlined, including potential registrational trials for PTC743 in refractory mitochondrial epilepsy and Friedreich ataxia (FA) and a Phase 1 trial for a second compound, PTC857, targeting GBA Parkinson's disease as its first indication.
The webinar can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the webinar and providing the passcode 3978766. A live, listen-only webcast can be accessed on the Events and Presentations page under the investor relations section of
About the Bio-e platform
The Bio-e platform is based on a family of enzyme targets, oxidoreductases, that are critical to the generation and regulation of energy key to disease pathology in the CNS and other organ systems. The platform utilizes expertise in electron-transfer chemistry to modulate key biological processes beyond the reach of current drug development approaches. The lead compounds from the Bio-e platform, PTC743 and PTC857, target the enzyme 15-lipoxygenase, a key enzymatic hub that regulates the inflammation and oxidative stress that underpin mitochondrial disease and CNS pathology. Two pivotal studies will be initiated with PTC743 in the second half of 2020: a Phase 2/3 trial in refractory mitochondrial epilepsy is expected to initiate in the third quarter of 2020 and a Phase 3 trial in FA is expected to initiate in the fourth quarter of 2020. PTC743 has been evaluated in over 400 patients with duration of exposure up to 10 years and has been reported to be safe and well-tolerated in over 450,000 cumulative dosing days. A Phase 1 trial of PTC857 targeting GBA Parkinson's disease as its first indication is expected to initiate in the third quarter of 2020.
About
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
For More Information:
Investors:
+1 (908) 912-9643
akane@ptcbio.com
Media:
+1 (908) 912-9167
jbaj@ptcbio.com
Forward-Looking Statements:
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful.
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