Proceedings - Boulder Peptide Foundation

BPS 2017

Discovery and Clinical Development of Elamipretide

Hazel Szeto

Scientific Founder, Stealth Biotherapeutics

ABSTRACT

Mitochondria play a central role in energy generation in the cell, providing ATP to carry out essential biological functions. As energy output declines, the most energetic tissues are preferentially affected, including the skeletal muscles, heart and retina. Age-related decline in mitochondrial function is associated with numerous chronic degenerative diseases. This talk will describe the discovery of a family of mitochondria-targeted tetrapeptides (SS peptides) that preserve mitochondrial structure and restore cellular bioenergetics by targeting cardiolipin, a unique phospholipid on the inner mitochondrial membrane. Preclinical and clinical development of the first drug candidate (Elamipretide) for rare genetic mitochondrial diseases (myopathy, Leberâ€™s hereditary optic neuropathy, Barth Syndrome) and common age-related mitochondrial diseases (heart failure, macular degeneration, muscle weakness) will be presented.

BIO

Dr. Hazel H. Szeto recently left Weill Cornell Medical College where she was Professor of Pharmacology and Director of the Research Program in Mitochondrial Therapeutics. Dr. Szeto pioneered the development of mitochondria-targeted peptides that protect mitochondrial cristae structure and restore cellular bioenergetics by targeting cardiolipin on the inner mitochondrial membrane. Mitochondrial dysfunction and bioenergetics failure is a hallmark of rare genetic diseases and common age-associated chronic diseases. Preclinical studies have demonstrated remarkable efficacy of these compounds in cardiorenal and metabolic diseases, neurodegenerative diseases, skeletal muscle weakness, and neuropathy. These compounds are now in clinical development sponsored by Stealth Biotherapeutics, a company founded by Dr. Szeto in 2006. The first drug candidate (elamipretide) is currently undergoing multiple Phase 2 clinical trials for mitochondrial diseases, heart failure, muscle weakness, and age-related macular degeneration. The US Food & Drug Administration granted Fast Track designation for elamipretide for primary mitochondrial myopathy. Dr. Szeto received her M.D. and Ph.D. (Pharmacology) from Cornell University Medical College.

Oral Presentations in Scientific Sessions

The session topics for Scientific Program are Peptides in the Clinic, Drug Delivery, Chemistry of Complex Peptides, Spotlight on Discovery, and Peptide Showcase. Abstracts may be submitted via the Apply to Present page on the website. Submissions will be reviewed by the Scientific Advisory Board on a monthly basis and the speaker will be notified of a decision.

Presentation length: Format is 25-30 minutes slide presentation with 5-10 minutes for Q&A, with the exception of Peptide Showcase talks which are 10 minute slide presentations with 5 minutes for Q&A. Please confirm with your session chair to confirm the exact presentation length.

Invited Speakers: Speakers are requested to supply their presentation details 30 days after receiving an invitation to present.

Program Book Deadline: Final abstract and title must be provided no later than September 1st. No changes to the program are possible after this date.

Presentation Format: Oral presenters please provide your presentation slides to the A/V table on the day of your presentation.

Lodging: All speakers are requested to reserve their own room. Discount rates at the conference hotel are available until August 16th.

Registration: All speakers, excluding sponsored presentation speakers, receive a complimentary registration upon approval of the abstract.

Peptide Showcase Description

The Peptide Showcase is an opportunity for an individual or company to “showcase” new ideas, technology and pipeline assets. Priority shall be given to presentations from biotech and startup companies. Service providers and vendors are not eligible. Speakers in the session receive a complimentary event registration.

Posters

Posters can be presented on any topic relevant to peptides including original research, innovative products and technologies.

Eligibility- Any attendee (from academia, industry, biotech or vendor/sponsor) can present a poster. The presentation must contain original scientific thinking. To submit a poster go to the Submit a Poster link from the meeting menu. All submissions are reviewed on a rolling basis and will be notified of approval. September 1st is the last day to submit a poster abstract. Approved posters are published on the BPF website.

A 30 ” x 40″ board shall be provided with thumb tacks. Board will be on an easel and can be rotated for either landscape or portrait formats. Once you apply for a poster, you will get email confirmation that your poster was accepted. The poster session and number assignments will be emailed one week before the Symposium.

Poster boards shall be available for display from 8am to 8pm the day of your assigned poster session. See the program agenda for the poster session schedule.

Public Release

Every presentation at the Boulder Peptide Symposium is automatically recorded. Speakers are required to give consent for sharing of the presentation video with the peptide community.