Versartis, Inc. Receives Orphan Drug Status for Its Novel Long-Acting Growth Hormone Treatment From European Commission (EC)

REDWOOD CITY, CA--(Marketwired - September 13, 2013) - Versartis, Inc. today announced that the European Commission has granted Orphan Medicinal Product Designation for VRS-317, the company’s proprietary long-acting form of recombinant human growth hormone (rhGH) for the treatment of growth hormone deficiency (GHD). VRS-317 is currently being investigated for safety and efficacy in pediatric GHD patients for up to once monthly dosing. At the present time, all marketed growth hormone treatments require daily injections.

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union. It also provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, a 10 year market exclusivity period after approval in the European Union and certain financial incentives such as fee reductions and waivers for companies developing orphan product candidates.

Versartis Chief Executive Officer Jeffrey L. Cleland, PhD, commented, “European orphan drug designation is the latest in a series of milestones for Versartis and underscores the potential of VRS-317 to address the significant unmet need for improved therapies for children and adults with GHD. In addition, we look forward to providing an exciting clinical update later this month in Europe, where we also will be meeting with an international group of leading investigators from the EU, US and Japan to plan our Phase 3 program.

The latest results from the company’s ongoing Versartis Trial In Children to Assess Long-Acting Growth Hormone (VERTICAL) study will be presented at the 9th Joint Meeting of Paediatric Endocrinology in Milan on Sunday, September 22, by Versartis Vice President of Medical Affairs George M. Bright, MD. The VERTICAL trial of VRS-317 consists of two stages: a single ascending dose stage (Phase 1b) to determine the safety, PK and PD of VRS-317 doses and to enable selection of dose regimens used in the repeat dose stage (Phase 2a) to obtain 6-month height velocity results. The oral presentation will include data from the Phase Ib dose-escalating stage of the study, which was conducted in approximately 30 pediatric endocrinology centers in the United States. The results from this stage support initiation of the repeat dose stage (Phase 2a) of the trial.

Presentation Details:

Abstract Title: “A Phase Ib/2a study of a new long-acting growth hormone (VRS-317) in pre-pubertal children with growth hormone deficiency (GHD)”

Presentation number: FC16-214
Session: Novel Growth-promoting Treatment
Date: Sunday, September 22
Time: 7:30 - 8:30 am

About Versartis
Versartis, Inc. is a biotechnology company developing therapeutics for the treatment of endocrine disorders. The company’s lead product candidate is VRS-317, a novel long-acting form of human growth hormone. Versartis is pursuing the development of new therapeutic proteins utilizing the proprietary Amunix half-life extension technology (XTEN). XTEN is a novel hydrophilic sequence of natural amino acids and is expressed as a fusion protein with a therapeutically active peptide or protein. New compounds developed by Versartis using the XTEN technology are expected to provide improved therapeutic outcomes such as enhanced efficacy/compliance, fewer side effects, prolonged half-life (up to monthly dosing), as well as low-cost production and enhanced stability. Further information on Versartis can be found at www.versartis.com. Versartis regulatory representative in Europe is Larode Ltd, United Kingdom.