Versartis’ VRS-317 6-Month Data Published in The Journal of Clinical Endocrinology & Metabolism
Results Show Positive Safety and Efficacy Data With Similar Outcomes Across Three Dosing Regimens
MENLO PARK, Calif., Dec. 16, 2015 (GLOBE NEWSWIRE) — Versartis, Inc. (NASDAQ:VSAR), an endocrine-focused biopharmaceutical company that is developing somavaratan (VRS-317), a novel, long-acting form of recombinant human growth hormone (rhGH) for growth hormone deficiency (GHD), today announced that results of a Phase 1b/2a pediatric trial of the investigational drug, somavaratan, were published online in The Journal of Clinical Endocrinology & Metabolism (JCEM) entitled “A Randomized Safety and Efficacy Study of somavaratan, a Long-Acting rhGH, in Pediatric Growth Hormone Deficiency” .
The trial was designed to determine the optimal somavaratan dose regimen to normalize insulin-like growth factor-I (IGF-I) in pediatric GHD patients, and to evaluate safety and efficacy of the drug over six months.
Highlights from the Trial
Over 6 months, somavaratan demonstrated clinically meaningful improvements in height velocity and IGF-I levels in pre-pubertal GHD patients with no significant differences between the monthly, twice-monthly or weekly dosing regimens.
A single dose of somavaratan showed sustained IGF-I response for up to one month with no accumulation of drug or IGF-I with repeat dosing.
Jay Shepard, Chief Executive Officer, commented, “We are very pleased to have the safety and efficacy data from our six-month study of somavaratan published in JCEM, one of the most highly-regarded journals in the field of endocrinology. The publication underscores the physician and patient interest in a long-acting treatment for children with GHD. Given the safety and efficacy data reported to date, we believe somavaratan may provide an enhanced option for these patients and their parents or caregivers.”
Bradley S. Miller, M.D., Ph.D., Associate Professor in the Department of Pediatric Endocrinology at the University of Minnesota Masonic Children’s Hospital and one of the investigators in the trial stated, “The less frequent dosing profile of somavaratan at different dosing regimens in pediatric patients achieved similar height velocities and IGF-I levels across the subsets of patients. It is exciting to see that there were no significant differences in the outcomes from monthly, twice-monthly and weekly dosing for patients, which is in line with results the team hoped to demonstrate. These results using a less frequent dosing regimen indicate an important advancement for pediatric patients with GHD.”
The multi-center, open-label Phase 1b/2a trial consisted of two phases: an initial Phase 1b, 30-day single ascending dose finding phase followed by a Phase 2a, 6-month, randomized, safety and efficacy phase comparing treatment effects of three dosing regimens. In Phase 1b, the results demonstrated that somavaratan pharmacokinetics (PK) was linearly proportional to the dose level, and that dose-dependent increases in the magnitude and duration of IGF-I responses enabled weekly, twice-monthly or monthly dosing. Additionally, a single dose of somavaratan showed sustained IGF-I responses for up to one month. Therefore, patients were randomized to somavaratan 1.15 mg/kg weekly, 2.5 mg/kg twice-monthly, or 5.0 mg/kg monthly in the Phase 2a portion of the trial. Mean annualized height velocities for somavaratan administered monthly, twice-monthly, or weekly were similar between groups at 7.86 ± 2.5, 8.61 ± 2.7, and 7.58 ± 2.5 cm/year, respectively. No somavaratan or IGF-I accumulation occurred with repeat dosing. There were no related serious adverse events or unexpected adverse events. Adverse events reported were mostly mild and transient in nature.
Patients completing the Phase 1b/2a trial reported here were continued into an ongoing Phase 2b Extension Study to assess long-term safety of the investigational product. To date, the Company has reported 12-month and 18-month data from this Extension Study demonstrating a continued safety and efficacy profile that is comparable to daily rhGH therapy. Based on the results published here and from the ongoing Extension Study, Versartis initiated a global Phase 3 registration trial, VELOCITY, investigating the safety and efficacy of twice-monthly dosing in GHD children.
About Versartis, Inc.
Versartis, Inc. is an endocrine-focused biopharmaceutical company initially developing somavaratan (VRS-317), a novel, long-acting form of recombinant human growth hormone in late-stage clinical trials for the treatment of growth hormone deficiency (GHD) in children and adults. Somavaratan is intended to reduce the burden of daily injection therapy by requiring significantly fewer injections, potentially improving compliance and, therefore, treatment outcomes. Versartis’ clinical trials can be found at www.versartistrials.com. For more information on Versartis, visit www.versartis.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions or current expectations concerning, among other things, plans and timing of our clinical trials and the potential for eventual regulatory approval of somavaratan. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: our success being heavily dependent on somavaratan; somavaratan being a new chemical entity; the risk that somavaratan may not have favorable results in clinical trials or receive regulatory approval; potential delays in our clinical trials due to regulatory requirements or difficulty identifying qualified investigators or enrolling patients; the risk that somavaratan may cause serious side effects or have properties that delay or prevent regulatory approval or limit its commercial potential; the risk that we may encounter difficulties in manufacturing somavaratan; if somavaratan is approved, risks associated with its market acceptance, including pricing and reimbursement; potential difficulties enforcing our intellectual property rights; our reliance on our license of intellectual property from Amunix Operating, Inc. and our need for additional funds to support our operations. We discuss many of these risks in greater detail under the heading “Risk Factors” contained in our Annual Report on Form 10-K for the year ended December 31, 2014, and in our Quarterly Report on Form 10-Q for the three months ended September 30, 2015, which are on file with the Securities and Exchange Commission (SEC). Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
Contacts: Corporate & Investors: Joshua Brumm Chief Financial Officer (650) 963-8582 IR@versartis.com Investors: Nick Laudico/David Burke The Ruth Group (646) 536-7030/7009 email@example.com firstname.lastname@example.org Media: Debra Bannister Corporate Communications (530) 676-7373 email@example.com