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HedgePath Pharmaceuticals' Pathway for Filing SUBA BCCNS NDA Clarified in Meeting with FDA

HedgePath Pharmaceuticals' Pathway for Filing SUBA BCCNS NDA Clarified in Meeting with FDA Key components of SUBA BCCNS NDA agreed to in FDA meeting minutes Expanded efficacy and safety modules required, which is anticipated to push proposed NDA submission into Q1 2019

PR Newswire

TAMPA, Fla., Oct. 9, 2018

TAMPA, Fla., Oct. 9, 2018 /PRNewswire/ -- HedgePath Pharmaceuticals, Inc. (OTCQB:HPPI) announced today it has gained additional clarity regarding its clinical program and the anticipated timing for filing of its New Drug Application (NDA) for SUBA™-Itraconazole as a treatment for Basal Cell Carcinoma in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS) based on meeting minutes received by HPPI from the U.S. Food and Drug Administration (FDA) relating to HPPI's face-to-face meeting with FDA on September 25, 2018.

During the meeting, HPPI discussed the requirements for filing its SUBA BCCNS NDA, and in the meeting minutes, FDA confirmed that it has agreed with HPPI's interpretation of 8 of the 11 NDA requirements discussed at the meeting. For the remaining three items, FDA has given HPPI guidance on certain items which, if lacking from the NDA submission, would lead the FDA to not accept the filing.  First, FDA has instructed HPPI to update its efficacy and safety information to include more recent data than its proposed cutoff date of December 2017 in order to provide additional data on the ten remaining patients who were still receiving therapy beyond December 2017.  FDA also indicated that HPPI must provide an analysis of basal cell carcinoma tumor burden responses via independent analysis of tumor photographs (which HPPI has already undertaken) to confirm results reported by the clinical investigators.  FDA has also requested that HPPI submit an Integrated Safety Summary (ISS) that includes data not only from HPPI's clinical trial, but all human trials of SUBA-Itraconazole (the active ingredient in SUBA BCCNS) regardless of strength and indication. 

Nicholas J. Virca, HPPI's President and Chief Executive Officer, stated that "We are pleased with the outcome of this meeting with FDA as we have gained important additional clarity on what is required for our SUBA BCCNS NDA.  The consequence of FDA requiring the completion of the ISS module is that HPPI will require more time than we previously anticipated to submit our NDA, resulting in a revised anticipated NDA submission date of sometime in the first quarter of 2019.  We are currently in the process of performing the requested updates, which we believe will enhance our data package."

As previously announced, under HPPI's supply and license agreement with Mayne Pharma, if the SUBA BCCNS NDA is not accepted for filing by December 31, 2018 (subject to limited extension if the NDA is filed in December), Mayne Pharma may elect to take back the SUBA-Itraconazole product for BCCNS in the United States (including by way of an exclusive license from HPPI of its clinical data) in exchange for a royalty on any future net sales.  HPPI and Mayne Pharma have commenced discussions on this important matter.

Readers are cautioned that no assurances can be given that the matters referenced herein, if addressed, will be found by FDA to be sufficient for acceptance of an NDA filing (including the risk that even if such matters are addressed, that the NDA may still not be accepted for filing for these or other reasons) or, even if the NDA is accepted for filing that it will be ultimately be approved by FDA. 

About SUBA-Itraconazole

HPPI's lead drug candidate, SUBA-Itraconazole, is a patent-protected formulation of itraconazole, an approved oral antifungal drug that has been in use for over 25 years.  HPPI is the exclusive U.S. licensee (through Mayne Pharma, the majority stockholder of HPPI) of SUBA-Itraconazole for the treatment of cancer.  Prior to research at Johns Hopkins University, itraconazole was not known to have any target in mammalian cells. Investigators at Johns Hopkins discovered that itraconazole inhibits the hedgehog pathway by binding to a surface receptor in the pathway called Smoothened.  Unlike generic itraconazole, that has poor and unpredictable bioavailability, SUBA-Itraconazole can be dosed at half the level of the generic formulation due to its superior bioavailability, which exceeds 90%.  As such, HPPI believes that generic itraconazole cannot be substituted for SUBA-Itraconazole.


HPPI's initial indication is for the orphan disease BCCNS. SUBA-Itraconazole has qualified under the FDA's Orphan Drug Designation Program as a potential therapy for BCCNS.  There is no approved pharmaceutical therapy for this familial cancer syndrome.  There are estimated to be 10,000 patients in the U.S. with BCCNS.  This is an autosomal dominantly inherited defect in the hedgehog pathway that causes the pathway to be up-regulated, resulting in hundreds or even thousands of basal cell carcinomas developing over the lifetime of the affected patients.  In many types of cancers, the hedgehog pathway is basically hijacked by the cancer cells to assist their growth and metastatic spread, but in the case of basal cell carcinomas, whether in this hereditary syndrome or in the much more common, sporadic basal cell carcinomas, the hedgehog pathway has a mutation that makes it the sole driver of the development of BCC tumors.  Inhibition of the pathway, then, can inhibit the appearance of new tumors, shrink existing tumors, and even cause some tumors to disappear altogether.

Cautionary Note Regarding Forward Looking Statements

This press release and any statements of representatives and partners of HedgePath Pharmaceuticals, Inc. (the "Company") related thereto contain, or may contain, among other things, certain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995.  Such forward-looking statements involve significant risks and uncertainties.  Such statements may include, without limitation, statements with respect to the Company's plans, objectives, projections, expectations and intentions and other statements identified by words such as "projects," "may," "will," "could," "would," "should," "believes," "expects," "anticipates," "estimates," "intends," "plans," "potential" or similar expressions.  These statements are based upon the current beliefs and expectations of the Company's management and are subject to significant risks and uncertainties, including those detailed in the Company's filings with the Securities and Exchange Commission.  Actual results (including, without limitation, the actual timing for, or actual results of, the Company's clinical trial described herein, the Company's meeting with FDA or the FDA's review of any trial data or New Drug Application submitted by the Company to FDA as described herein) may differ significantly from those set forth or implied in the forward-looking statements (and may further differ from the interim study results previously disclosed by the Company).  These forward-looking statements involve numerous risks and uncertainties that are subject to change based on various factors (many of which are beyond the Company's control).  The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. 

For more information:

Nicholas J. Virca, President and CEO

Investor Relations Contact:
Garrison Hasara, CFO and Treasurer

© 2018 HedgePath Pharmaceuticals, Inc.  All rights reserved.

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Last Updated: 09-Oct-2018