SAN FRANCISCO, CA, USA I June 16, 2016 I Medivation, Inc. (NASDAQ: MDVN) today announced results from a Phase I/II study of pidilizumab, an investigational antibody with immune-mediated anti-tumor effects, that demonstrated potential clinical benefit in pediatric patients with diffuse intrinsic pontine glioma (DIPG). The study, which was exploratory in nature, was designed to assess the safety and tolerability of pidilizumab, as well as key clinical outcomes, such as event-free and overall survival, in this pediatric population. Preliminary data were presented this week in an oral symposium at the International Symposium on Pediatric Neuro-Oncology (ISPNO) by the study’s lead investigator Iris Fried, M.D., Attending Physician, Pediatric Hemato-oncology, Hadassah Medical Center, Jerusalem, Israel.

Diffuse intrinsic pontine glioma (DIPG) is a rare and aggressive pediatric cancer which is responsible for the highest brain tumor mortality in children.1 Approximately 300-400 pediatric brain stem tumors are diagnosed per year in the United States, approximately 75%-80% of which are DIPGs.2 Children with DIPG experience a median overall survival between 9-12 months and a two-year survival rate of less than 10%.1

Data from nine pediatric patients with DIPG who were treated with pidilizumab following completion of standard radiation therapy were presented. The median age of the study population was 6.5 years (range: 3-19 years): eight patients had intermediate risk features and one patient had high risk features. The reported mean event-free and overall survival estimates were 12 and 15.6 months, respectively. Three patients with DIPG remained progression-free at 16.3, 22, and 24 months following diagnosis, with one patient experiencing a partial response. Adverse events of any grade reported in at least one treatment cycle include neutropenia, fatigue, loss of appetite, hypertension, nausea, and lymphopenia; only neutropenia and hypertension were reported as grade 3 adverse events. The study continues to enroll patients.

“Separate studies have revealed the potential of pidilizumab in hematological malignancies, and these results, while in a small cohort in a rare disease, suggest its potential activity in other conditions,” said David Hung, M.D., Founder, President and Chief Executive Officer of Medivation. “We are especially encouraged by these results in a devastating disease that typically leads to such rapid and certain mortality, and we remain committed to advancing the clinical development of pidilizumab as we continue to characterize its unique and differentiated mechanism of action.”

Despite more than 30 years of clinical research, there have been no improvements in clinical outcomes and there are no approved treatments for DIPG.1,2

About Medivation, Inc.

Medivation, Inc. is a biopharmaceutical company focused on the development and commercialization of medically innovative therapies to treat serious diseases for which there are limited treatment options. Medivation aims to transform the treatment of these diseases and offer hope to critically ill patients and their families. For more information, please visit at

Forward-Looking Statement

Certain of the statements made in this press release are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are subject to risks and uncertainties which may cause actual results to differ significantly from those expressed or implied by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, the clinical benefit and safety shown in this exploratory Phase I/II clinical trial may not be reproduced or confirmed in subsequent controlled or pivotal clinical trials; unexpected adverse events or adverse results from clinical trials involving pidilizumab could cause delay or discontinuation of Medivation’s ongoing or planned development activities for pidilizumab; further investigation of pidilizumab in non-hematological malignancies, such as DIPG, may not be supported or undertaken by Medivation; Medivation’s ability to identify and confirm the binding target for pidilizumab; and other risks detailed in Medivation’s filings with the Securities and Exchange Commission, or SEC, including its quarterly report on Form 10-Q for the quarter ended March 31, 2016, which was filed on May 5, 2016. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date of this press release. Medivation disclaims any obligation or undertaking to update, supplement or revise any forward-looking statements contained in this press release.


1. Bartels U, Hawkins C, Vézina G, Kun L, Souweidane M, Bouffet E. Proceedings of the diffuse intrinsic pontine glioma (DIPG) Toronto Think Tank: advancing basic and translational research and cooperation in DIPG. Neurooncol. 2011 Oct;105(1):119-25.
2. National Cancer Institute. Childhood Brain Stem Glioma Treatment (PDQ®)-Health Professional Version. Available at:

SOURCE: Medivation