Concordia Healthcare Corp. announces FDA agreement on Special Protocol Assessment for Phase 3 Clinical trial on new therapy to treat perihilar cholangiocarcinoma

TORONTO and BANNOCKBURN, Ill., Jan. 13, 2014 /PRNewswire/ -- Concordia Healthcare Corp. ("Concordia" TSX:CXR) announced today that its subsidiary Pinnacle Biologics, Inc. ("Pinnacle"), a biopharmaceutical research and development company specializing in rare diseases, has reached an agreement with the U.S. Food and Drug Administration (the "FDA") under a special protocol assessment (the "SPA") to enroll patients with an advanced form of bile duct cancer in a pivotal Phase 3 clinical trial.  There is currently no approved therapy for this cancer type. The SPA is a written agreement between Pinnacle and the FDA regarding the design, endpoints and planned statistical analysis approach of the trial to be used in support of a potential New Drug Application submission.  The clinical trial will study the efficacy and safety of photodynamic therapy ("PDT") with PHOTOFRIN® (porfimer sodium) for injection as treatment for unresectable advanced perihilar cholangiocarcinoma ("CCA") Bismuth type III/IV.

"The agreement with the FDA to move into this Phase 3 clinical trial is a significant milestone for Pinnacle," said Mark Thompson, CEO of Concordia.  Robert S. Altman, president of Pinnacle added, "Securing this agreement reflects the depth of our experience and industry relationships with regulators, top academic institutions and clinical opinion leaders. We expect to begin enrolling patients into this study in the first half of 2014, a process, which we believe will help accelerate product development for this rare and devastating disease."

"For the first time, we have a path forward to an indication using a drug-device combination that may combat a complex cancer, which is difficult to treat and has extremely poor survival rates," said PD. Dr. med. Maria Anna Ortner, Department of Gastroenterology and Hepatology, University Hospital Zurich, and a lead study investigator.

About Photofrin

Photodynamic therapy with PHOTOFRIN® (porfimer sodium) is a two-stage process requiring administration of both drug and light. The first stage of PDT is the intravenous injection of PHOTOFRIN®. The second stage of therapy uses illumination with nonburning laser light 40–50 hours following injection with PHOTOFRIN®. When injected, PHOTOFRIN® is attracted to certain tissues, especially cancer cells. Tumor destruction results from biochemical reactions, not heat. Tumor selectivity occurs through a combination of preferential retention of PHOTOFRIN® by the tumor and selective delivery of light to the tumor site.

PHOTOFRIN® is indicated for the treatment of esophageal cancer, non-small-cell lung cancer and high-grade dysplasia in Barrett's esophagus. PHOTOFRIN® was granted orphan drug designation (ODD) by the FDA in 2004 for cholangiocarcinoma (CCA), a rare cancer in the bile ducts that carry bile from the liver to the small intestine[i], and in December 2011, the FDA granted a second ODD for PHOTOFRIN® as adjuvant therapy to surgery for the treatment of malignant pleural mesothelioma.

About Concordia

Concordia is a diverse healthcare company focused on legacy pharmaceutical products, orphan drugs and medical devices for the diabetic population.

The company's legacy pharmaceutical business consists of an ADHD-treatment drug, Kapvay® (clonidine extended release tablets), Ulesfia® (benzyl alcohol) Lotion a Head Lice Treatment, and an Asthma-related medication, Orapred ODT® (prednisolone sodium phosphate orally disintegrating tablets). Concordia's Specialty Healthcare Distribution (SHD) division (Complete Medical Homecare) distributes medical supplies targeting diabetes and related conditions. Concordia's orphan division, Pinnacle, markets Photofrin® in the United States.

Concordia operates out of facilities in Oakville, Ontario, Lenexa, Kansas (near Kansas City, Missouri), Chicago, Illinois and Bridgetown, Barbados.

Notice regarding forward-looking statements:

This release includes forward-looking statements regarding Concordia and its business, which may include, but is not limited to, statements with respect to approval of a New Drug Application for PDT with PHOTOFRIN®, the enrollment of patients into the Phase 3 clinical trial, the acceleration of product development, the ability to obtain necessary approvals, the development of PDT with PHOTOFRIN® as a new treatment for certain rare forms of cancer, the ability of PDT with PHOTOFRIN® to combat certain forms of cancer and other factors.  Often, but not always, forward-looking statements can be identified by the use of words such as "plans", "is expected", "expects", "scheduled", "intends", "contemplates", "anticipates", "believes", "proposes" or variations (including negative variations) of such words and phrases, or state that certain actions, events or results "may", "could", "would", "might" or "will" be taken, occur or be achieved. Such statements are based on the current expectations of Concordia's management, and are based on assumptions and subject to risks and uncertainties.  Although Concordia's management believes that the assumptions underlying these statements are reasonable, they may prove to be incorrect. The forward-looking events and circumstances discussed in this release may not occur by certain specified dates or at all and could differ materially as a result of known and unknown risk factors and uncertainties affecting the company, including risks regarding the pharmaceutical industry, the receipt of approval of PDT with PHOTOFRIN® as a New Drug Application, the ability to enrol patients into the Phase 3 clinical trial, the ability to accelerate product development, the development of PDT with PHOTOFRIN® to treat certain rare forms of cancer, the ability of PDT with PHOTOFRIN® to combat certain forms of cancer, the ability to further develop Concordia's products, the failure to obtain regulatory approvals, economic factors, market conditions, the equity markets generally, risks associated with growth and competition and many other factors beyond the control of Concordia.  Although Concordia has attempted to identify important factors that could cause actual actions, events or results to differ materially from those described in forward-looking statements, there may be other factors that cause actions, events or results to differ from those anticipated, estimated or intended. No forward-looking statement can be guaranteed. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and Concordia undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise.

[i].  http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm

SOURCE Concordia Healthcare Corp.