Healthcare Industry News:  neuroblastoma 

Biopharmaceuticals Oncology Regulatory

 News Release - December 8, 2015

Ignyta Receives Orphan Drug Designation in the European Union for Entrectinib for the Treatment of Neuroblastoma

SAN DIEGO--(Healthcare Sales & Marketing Network)--Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, today announced that the European Medicines Agency (EMA) has granted orphan drug designation for Ignyta’s product candidate entrectinib for the treatment of neuroblastoma.

“We are pleased that the EMA has provided this designation for entrectinib, adding to our orphan designations in the U.S. for the treatment of neuroblastoma and the treatment of TrkA-, TrkB-, TrkC-, ROS1- or ALK-positive non-small cell lung cancer and colorectal cancer,” said Jonathan Lim, M.D., Chairman and CEO of Ignyta. “Entrectinib has the potential to address unmet needs of patients with rare cancers, and we will continue to aggressively pursue our clinical development program for entrectinib in solid tumors for the benefit of these patients.”

About Orphan Drug Designation

To receive orphan drug designation from the EMA, a medicinal product must be intended for the treatment of a life-threatening or a chronically debilitating rare disease affecting not more than 5 in 10,000 individuals in the European Union (EU), and have the potential to be of significant benefit to those affected by that condition. Orphan drug designation provides incentives designed to facilitate development, including protocol assistance, reduced fees for regulatory activities and up to ten years of market exclusivity in the EU upon marketing approval for the designated indication.

About Entrectinib

Entrectinib is a novel, orally available, selective tyrosine kinase inhibitor targeting tumors that harbor activating alterations to NTRK1/2/3 (encoding TrkA/ TrkB/TrkC), ROS1 or ALK. Entrectinib is the most potent Trk inhibitor in the clinic, without undesirable off-target activity, and the only Trk inhibitor with clinically demonstrated activity against CNS metastases. This product candidate is in a Phase 2 clinical trial called STARTRK-2, which is the second of the “Studies of Tumor Alterations Responsive to Targeting Receptor Kinases.” The trial is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types and molecular targets.

About Ignyta, Inc.

At Ignyta, we fight cancer – a formidable opponent that manifests as thousands of different molecularly defined diseases and takes away millions of lives globally, every year. In this fight, our Big Hairy Audacious Goal (BHAG) is not just to shrink tumors but to eradicate residual disease – the source of cancer relapse and recurrence – in precisely defined patient populations by 2030. We will work tirelessly to achieve this BHAG by pursuing an integrated therapeutic (Rx) and companion diagnostic (Dx) strategy for treating cancer patients. Our Rx efforts are focused on discovering, in-licensing or acquiring, then developing and commercializing, molecularly targeted therapies; cancer stem cell/dormant tumor cell targeted therapies; novel chemotherapies/cell cycle inhibitors; and cancer immunotherapies – four therapeutic cornerstones that, sequentially or in combination, are foundational for eradicating residual disease. Our Dx efforts aim to pair these product candidates with biomarker-based companion diagnostics that are designed to precisely identify, at the molecular level, the patients who are most likely to benefit from the monotherapies and polytherapies we develop. We believe that only through this integrated Rx/Dx approach can we succeed in this fight. For more information, please visit: www.ignyta.com.

Forward-Looking Statements

This press release contains forward-looking statements about Ignyta as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, references to the potential for Ignyta to receive regulatory approval for entrectinib, the potential for entrectinib to help patients with neuroblastoma or other rare cancers and the potential benefits of orphan drug designation. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies and operating as a development stage company; Ignyta’s ability to develop, initiate or complete preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates; changes in Ignyta’s plans to develop and commercialize its product candidates; the potential for final results of the ongoing clinical trials of entrectinib or other product candidates, or any future clinical trials of entrectinib or other product candidates, to differ from preliminary or expected results; Ignyta’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; Ignyta’s ability to obtain and maintain intellectual property protection for its product candidates; the risk that orphan drug exclusivity may not effectively protect a product from competition and that such exclusivity may not be maintained; the potential for the company to fail to maintain the CLIA registration of its diagnostic laboratory or to fail to achieve full CLIA accreditation of such laboratory; the loss of key scientific or management personnel; competition in the industry in which Ignyta operates; and market conditions. These forward-looking statements are made as of the date of this press release, and Ignyta assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents the company files with the SEC available at www.sec.gov, including without limitation Ignyta’s Annual Report on Form 10-K for the year ended December 31, 2014 and subsequent Quarterly Reports on Form 10-Q.


Source: Ignyta

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