Ascendis Pharma A/S Announces Publication of Comprehensive Results from Randomized, Active-Controlled Phase 2 Trial of Once-Weekly TransCon Growth Hormone in Pediatric Patients

Friday, February 17, 2017

Ascendis Pharma A/S a biopharmaceutical company that utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases, today announced the publication of its previously presented pediatric phase 2 trial of TransCon Growth Hormone in the Journal of Clinical Endocrinology and Metabolism.

The authors concluded that once-weekly, sustained release TransCon Growth Hormone was comparable to daily growth hormone in terms of efficacy, safety and tolerability in children with growth hormone deficiency (GHD). The publication is available online at: https://academic.oup.com/jcem/article/2996234/A-Randomized-Phase-2-Study-of-Long-Acting-TransCon.

GHD in children is a serious orphan disease, which results in short stature, metabolic abnormalities, and cognitive deficiencies, often leading to decreased quality of life.1 Current GHD treatment requires daily growth hormone injections for many years. However, the administrative burden often results in at least one missed injection per week, contributing to suboptimal outcomes.2 Currently, there are no long-acting growth hormone treatment options commercially available in the United States or Europe.

"Suboptimal outcomes due to poor compliance remain a significant barrier in management of GHD," said Professor Pierre Chatelain, M.D., lead author of the manuscript from Université Claude Bernard Lyon 1. "The data suggest that TransCon Growth Hormone has the potential to address the significant need for a long-acting growth hormone therapy without compromising efficacy, safety and tolerability."

TransCon Growth Hormone is designed to release unmodified growth hormone that diffuses freely into the body's tissues, where it may carry out the effects of native growth hormone with easy-to-remember weekly dosing.

Separately, results of a Phase 2 study evaluating the safety, pharmacokinetics, and pharmacodynamics of TransCon Growth Hormone in adult patients with GHD were published online in the journal Endocrine Connections. The publication is available at: http://m.endocrineconnections.com/content/early/2017/02/14/EC-17-0007.

Based on the positive Phase 2 results in pediatric patients, Ascendis has an ongoing global Phase 3 trial of TransCon Growth Hormone compared to daily growth hormone. The randomized, active-controlled heiGHt Trial aims to enroll 150 pediatric patients with GHD by the end of 2017. Healthcare professionals may access additional information regarding TransCon Growth Hormone and the heiGHt Trial at www.heighttrial.com.

About Ascendis Pharma A/S

Ascendis Pharma is applying the TransCon technology platform to build a leading rare disease commercial company. The company utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases by improving clinically validated parent drugs and creating therapies with potential for best-in-class efficacy, safety and/or convenience.

Ascendis Pharma has a wholly-owned pipeline of rare disease endocrinology programs, including once-weekly TransCon Growth Hormone, which is currently being evaluated in the Phase 3 heiGHt Trial for children with growth hormone deficiency (GHD), TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism, and TransCon CNP, a long-acting prodrug of C-type natriuretic peptide for achondroplasia. Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology.

For more information, please visit www.ascendispharma.com.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding our future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) our product pipeline, (ii) whether TransCon Growth Hormone's has the potential to address the significant need for a long-acting growth hormone therapy without compromising efficacy, safety and tolerability, (iii) whether TransCon Growth Hormone can carry out the effects of native growth hormone with easy-to-remember weekly dosing, (iv) our aim to enroll 150 pediatric patients with GHD by the end of 2017 in our Phase 3 heiGHt trial, (v) our ability to apply the TransCon technology platform to build a leading rare disease commercial company, and (vi) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that we make, including the following: unforeseen safety or efficacy results in our TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs, general and administrative expenses, other research and development expenses and our business generally; delays in the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug; for planned clinical studies; and our ability to obtain additional funding, if needed, to support our business activities. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to our business in general, see our current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including our Report on Form 6-K filed with the SEC on October 18, 2016. Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments we may enter into or make. We do not assume any obligation to update any forward-looking statements, except as required by law.

References

1 BMC Endocrine Disorders 2012, 12:26 2 PLoS ONE 2011, 6(1), e16223

Internal contact:                                    
Scott T. Smith                       
Chief Financial Officer             
(650) 352-8389                       
ir@ascendispharma.com          

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