Item 8.01 Other Events
On December 16, 2019, the Company announced the submission of a New Drug Application, or NDA, to the U.S. Food and Drug Administration (FDA) for
ripretinib, the Companys investigational broad-spectrum KIT and PDGFRα inhibitor, for the treatment of patients with advanced gastrointestinal stromal tumors (GIST) who have received prior treatment with imatinib, sunitinib and
regorafenib. The NDA is being reviewed by the FDA under its Oncology Center of Excellence Real-Time Oncology Review, or RTOR, pilot program. The pilot program aims to explore a more efficient review process to ensure that safe and effective
treatments are available to patients as early as possible, while maintaining and improving review quality.
This Report contains forward-looking
statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding our expectations regarding our goal of bringing ripretinib to patients with advanced GIST, the
potential for ripretinib to serve as an important new treatment option for people with advanced GIST, working with the FDA through its review of our NDA application via the FDAs Real-Time Oncology Review pilot program and the possible benefits
of that pilot program and breakthrough therapy designation, preparing for the potential launch of ripretinib in the United States, if approved, our readiness for commercial launch, and the estimated incidence and prevalence of GIST and related
patient populations. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate,
predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these
identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results
to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the delay of any current or planned clinical studies or the
development of our product candidates, including ripretinib, our ability to successfully demonstrate the efficacy and safety of our product candidates including in later-stage studies, the preclinical and clinical results for our product candidates,
which may not support further development of such product candidates, the possibility that results experienced in early, preliminary, top-line or initial data may not be indicative of the results experienced
in final data, our ability to work with the FDA under its RTOR pilot program and timely respond to information requests or requirements in connection with our recently-filed NDA for ripretinib in advanced GIST, that acceptance into the RTOR pilot
program does not guarantee or influence approvability of our NDA for ripretinib in advanced GIST, which is subject to the standard benefit-risk evaluation by FDA, and that we may not derive any benefit from inclusion in the RTOR pilot program,
including, but not limited to, a more efficient review process compared to investigational drugs evaluated without this pilot program or under standard FDA procedures, the fact that the RTOR program is a pilot program being tested by FDA, is not a
formal regulatory pathway with regulatory process, regulations or procedures, and may be suspended or halted at any time, including, without limitation, because FDA decides not to continue the pilot, or because FDA determines that our application no
longer meets its criteria for inclusion in the RTOR pilot program, the fact that receipt of a breakthrough therapy designation for a product candidate, such as ripretinib, may not result in us receiving any of the benefits of such