Proceedings - Boulder Peptide Foundation

BPS 2018

TransCon technology for sustained delivery of proteins, peptides, and small molecules

Harald Rau

Chief Scientific Officer, Ascendis Pharma

ABSTRACT

Peptide and protein drug candidates often suffer from suboptimal properties such as poor bioavailability and inadequate circulation half-life due to rapid renal clearance and/or receptor- mediated clearance. The TransCon technology enables transient conjugation of a parent drug to a carrier through a linker to create a polymeric prodrug. Following injection, the proprietary linker autohydrolyzes in an enzyme-independent process based on pH and temperature, and unmodified parent drug is released at a rate fine-tuned through the design of the TransCon linker. Since drug is released in its native form, its original pharmacology is retained, with the same biodistribution, receptor affinity, and mode-of-action as the parent drug. Linkers have been developed with release rates that allow for the development of prodrugs that can deliver proteins, peptides, and small molecules with dosing frequencies ranging from days to several months. Soluble as well as insoluble carrier systems can be used to enable systemic or localized delivery. The TransCon technology has been used to develop a pipeline of rare endocrinology disease programs: TransCon Growth Hormone (in phase 3 for pediatric growth hormone deficiency), a long-acting prodrug of parathyroid hormone, TransCon PTH (in phase 1 for hypoparathyroidism), and a long-acting prodrug of C-type natriuretic peptide, TransCon CNP, (filed for initiation of clinical development in Australia for achondroplasia).

BIO

Harald Rau has served as the Chief Scientific Officer and SVP, managing the research group at Ascendis Pharma since December 2007. Prior to Ascendis Pharma, Dr. Rau served as the Chief Scientific Officer of Complex Biosystems GmbH, a biotechnology company he co-founded in 2002, which was acquired by Ascendis Pharma in December 2007. Prior to co-founding Complex Biosystems, he worked with Graffinity Pharmaceuticals, a biotechnology company, from 1998 to 2002. Dr. Rau holds a doctorate from the University of Freiburg, Germany.

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.