Click here to close now.

SYS-CON MEDIA Authors: Roger Strukhoff, Pat Romanski, Elizabeth White, Liz McMillan, Kelly Murphy

News Feed Item

Versartis Presents Latest Positive Results From Its Pediatric Study of Long-Acting Growth Hormone Treatment at International Endocrinology Conference

MILAN, ITALY -- (Marketwired) -- 09/22/13 -- Versartis, Inc. today released the latest positive results from the company's ongoing VERTICAL clinical trial of VRS-317, its proprietary long-acting form of recombinant human growth hormone (rhGH), at the 9th Joint Meeting of Paediatric Endocrinology in Milan. The most recent data from this trial in pediatric patients were presented by George M. Bright, MD, Versartis Vice President of Medical Affairs, in an oral discussion titled "A Phase 1b/2a Study of a New Long-acting Growth Hormone (VRS-317) in Pre-pubertal Children with Growth Hormone Deficiency (GHD)." All currently approved growth hormone drugs require daily injections and consequently pose considerable challenges to patients with GHD. In contrast, VRS-317 is being developed to provide up to once-monthly dosing and promises to improve patients' ability to adhere to their therapy regimen and to improve their overall treatment outcomes.

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. Results from the recently completed Phase 1b dose-escalating stage of the study were reported by Dr. Bright.

Dr. Bright commented, "The latest data from the Phase 1b demonstrated that a single dose of VRS-317 was very well tolerated in children with GHD and that we were able to safely raise IGF-I to the levels associated with good catch-up growth while using a reduced dosing frequency. Therefore, the data presented today provide strong support for the continued study of up to once-monthly dosing in the next stage of our trial, which was initiated earlier this month. This study will further determine the 3-month and 6-month height velocities in these GHD patients."

The VRS-317 VERTICAL Trial
The Versartis Trial In Children to Assess Long-Acting Growth Hormone (VERTICAL) study is being conducted in approximately 30 U.S. pediatric endocrinology centers and will enroll up to 72 naïve-to-treatment, pre-pubertal children with GHD that was documented by auxologic criteria and two GH stimulation tests.

In Phase 1b, the PK and PD (IGF-I) responses over a 30 day period were determined following a single, subcutaneous dose of VRS-317 at 6 ascending dose levels. Safety Review Committee meetings were held before each dose escalation to review collected data against protocol-specified stopping criteria. VRS-317 dosing began at 0.80 mg/kg, a dose shown to be safe and well tolerated in GHD adults in the previously completed trial, with dose increases to 1.20 mg/kg, 1.80 mg/kg, 2.70 mg/kg, 4.00 mg/kg and 6.00 mg/kg (equivalent to 4.8, 7.4, 11.1, 16.7, 24.7 and 37.0 mcg rhGH per kg per day taken for 30 days). In the United States, the typical dose of daily rhGH in children with GHD is approximately 40 mcg/kg/day. Thus, the VRS-317 doses studied in this trial are all below the amount of daily rhGH typically prescribed for these patients. VRS-317 dose selection for Phase 2a was based on safety and IGF-I responses from Phase 1b. Following Phase 2a dose selection, subjects are now being dosed in each of three dose cohorts for the determination of 3-month and 6-month height velocities.

Top Line Results
In the Phase 1b portion of the trial, 48 subjects (27M, 21F) with mean (SD) age 7.2 (2.2) yrs were studied in 6 dose cohorts (8 per cohort). At screening, mean (SD) HT-SDS was -2.7 (0.6), weight was 18.0 (4.6) kg and IGF-I SDS was -1.8 (0.7). VRS-317 plasma concentrations reach a maximum at a mean time of 3 days post-dose, are proportional to dose and remain detectable for up to 30 days from a single dose in all subjects tested. Maximal changes in IGF-I SDS occur between 2 to 14 days after a single dose on Day 1. The amplitude and duration of IGF-I responses increase with increasing VRS-317 dose. The increase in average IGF-1 SDS over 30 days was also proportional to dose and sufficient to support up to once-monthly dosing of VRS-317. Importantly, the prolonged IGF-I responses do not come at the expense of over-exposure to high IGF-I levels, where only a single value of IGF-I SDS in each of two patients has exceeded +2. All related adverse events that have been reported were mild and transient, with no serious or unexpected adverse events reported.

Conclusions
Single doses of VRS-317 from 0.8 to 6.0 mg/kg were safe and well tolerated when administered to 48 pre-pubertal children with GHD. In addition, dose proportional increases in VRS-317 levels and IGF-I responses were observed, indicating the flexibility for selecting doses and dose regimens of up to once-per-month dosing. Consequently, VRS-317 is a long-acting rhGH with the potential for up to monthly-dosing intervals in children with GHD.

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. VRS-317 is currently being investigated for safety and efficacy in pediatric GHD patients for up to once-monthly dosing. 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.

Contacts:
Corporate
Paul Westberg
SVP Business Development
Ph: 650 963-8585
Email: Email Contact

Media
Debra Bannister
Corporate Communications
Ph: 530 676-7373
Email: Email Contact

More Stories By Marketwired .

Copyright © 2009 Marketwired. All rights reserved. All the news releases provided by Marketwired are copyrighted. Any forms of copying other than an individual user's personal reference without express written permission is prohibited. Further distribution of these materials is strictly forbidden, including but not limited to, posting, emailing, faxing, archiving in a public database, redistributing via a computer network or in a printed form.

Latest Stories
Since 2008 and for the first time in history, more than half of humans live in urban areas, urging cities to become “smart.” Today, cities can leverage the wide availability of smartphones combined with new technologies such as Beacons or NFC to connect their urban furniture and environment to create citizen-first services that improve transportation, way-finding and information delivery. In her session at @ThingsExpo, Laetitia Gazel-Anthoine, CEO of Connecthings, will focus on successful use c...
The recent trends like cloud computing, social, mobile and Internet of Things are forcing enterprises to modernize in order to compete in the competitive globalized markets. However, enterprises are approaching newer technologies with a more silo-ed way, gaining only sub optimal benefits. The Modern Enterprise model is presented as a newer way to think of enterprise IT, which takes a more holistic approach to embracing modern technologies.
DevOps Summit, taking place Nov 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 17th Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The widespread success of cloud computing is driving the DevOps revolution in enterprise IT. Now as never before, development teams must communicate and collaborate in a dynamic, 24/7/365 environment. There is no time to wait for long developmen...
Security can create serious friction for DevOps processes. We've come up with an approach to alleviate the friction and provide security value to DevOps teams. In her session at DevOps Summit, Shannon Lietz, Senior Manager of DevSecOps at Intuit, will discuss how DevSecOps got started and how it has evolved. Shannon Lietz has over two decades of experience pursuing next generation security solutions. She is currently the DevSecOps Leader for Intuit where she is responsible for setting and driv...
The explosion of connected devices / sensors is creating an ever-expanding set of new and valuable data. In parallel the emerging capability of Big Data technologies to store, access, analyze, and react to this data is producing changes in business models under the umbrella of the Internet of Things (IoT). In particular within the Insurance industry, IoT appears positioned to enable deep changes by altering relationships between insurers, distributors, and the insured. In his session at @Things...
“Oh, dev is dev and ops is ops, and never the twain shall meet.” With apoloies to Rudyard Kipling and all of his fans, this describes the early state of the two sides of DevOps. Yet the DevOps approach is demanded by cloud computing, as the speed, flexibility, and scalability in today's so-called “Third Platform” must not be hindered by the traditional limitations of software development and deployment. A recent report by Gartner, for example, says that 25% of Global 2000 companies will b...
Software-driven innovation is becoming a primary approach to how businesses create and deliver new value to customers. A survey of 400 business and IT executives by the IBM Institute for Business Value showed businesses that are more effective at software delivery are also more profitable than their peers nearly 70 percent of the time (1). DevOps provides a way for businesses to remain competitive, applying lean and agile principles to software development to speed the delivery of software that ...
Big Data is amazing, it's life changing and yes it is changing how we see our world. Big Data, however, can sometimes be too big. Organizations that are not amassing massive amounts of information and feeding into their decision buckets, smaller data that feeds in from customer buying patterns, buying decisions and buying influences can be more useful when used in the right way. In their session at Big Data Expo, Ermanno Bonifazi, CEO & Founder of Solgenia, and Ian Khan, Global Strategic Positi...
JFrog on Thursday announced that it has added Docker support to Bintray, its distribution-as-a-service (DaaS) platform. When combined with JFrog’s Artifactory binary repository management system, organizations can now manage Docker images with an end-to-end solution that supports all technologies. The new version of Bintray allows organizations to create an unlimited number of private Docker repositories, and through the use of fast Akamai content delivery networks (CDNs), it decreases the dow...
More organizations are embracing DevOps to realize compelling business benefits such as more frequent feature releases, increased application stability, and more productive resource utilization. However, security and compliance monitoring tools have not kept up and often represent the single largest remaining hurdle to continuous delivery. In their session at DevOps Summit, Justin Criswell, Senior Sales Engineer at Alert Logic, Ricardo Lupo, a Solution Architect with Chef, will discuss how to ...
Thanks to Docker, it becomes very easy to leverage containers to build, ship, and run any Linux application on any kind of infrastructure. Docker is particularly helpful for microservice architectures because their successful implementation relies on a fast, efficient deployment mechanism – which is precisely one of the features of Docker. Microservice architectures are therefore becoming more popular, and are increasingly seen as an interesting option even for smaller projects, instead of bein...
Over the last few years the healthcare ecosystem has revolved around innovations in Electronic Health Record (HER) based systems. This evolution has helped us achieve much desired interoperability. Now the focus is shifting to other equally important aspects - scalability and performance. While applying cloud computing environments to the EHR systems, a special consideration needs to be given to the cloud enablement of Veterans Health Information Systems and Technology Architecture (VistA), i.e....
17th Cloud Expo, taking place Nov 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, will feature technical sessions from a rock star conference faculty and the leading industry players in the world. Cloud computing is now being embraced by a majority of enterprises of all sizes. Yesterday's debate about public vs. private has transformed into the reality of hybrid cloud: a recent survey shows that 74% of enterprises have a hybrid cloud strategy. Meanwhile, 94% of enterprises a...
One of the biggest impacts of the Internet of Things is and will continue to be on data; specifically data volume, management and usage. Companies are scrambling to adapt to this new and unpredictable data reality with legacy infrastructure that cannot handle the speed and volume of data. In his session at @ThingsExpo, Don DeLoach, CEO and president of Infobright, will discuss how companies need to rethink their data infrastructure to participate in the IoT, including: Data storage: Understand...
The truth is, today’s databases are anything but agile – they are effectively static repositories that are cumbersome to work with, difficult to change, and cannot keep pace with application demands. Performance suffers as a result, and it takes far longer than it should to deliver new features and capabilities needed to make your organization competitive. As your application and business needs change, data repositories and structures get outmoded rapidly, resulting in increased work for applica...