Healthcare Industry News:  Bronchiectasis 

Biopharmaceuticals

 News Release - June 10, 2016

Raptor Pharmaceutical Announces Presentation of New Data Analyses from a Phase 3 Study of QUINSAIR(TM) at the 39th European Cystic Fibrosis Conference

NOVATO, Calif., June 10, 2016 -- (Healthcare Sales & Marketing Network) -- Raptor Pharmaceutical Corp. (RPTP), a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, today announced new data analyses of its Phase 3 clinical study (MPEX-209) comparing QUINSAIRTM to inhaled tobramycin solution in patients with cystic fibrosis and chronic Pseudomonas aeruginosa infections that were presented at the 39th European Cystic Fibrosis Conference in Basel, Switzerland.

“Patients with cystic fibrosis have few approved inhaled antimicrobial options for the treatment of chronic Pseudomonas infection and in many cases, continue to have pulmonary exacerbations despite available therapies,” said Krishna R. Polu, M.D., Chief Medical Officer of Raptor. “We believe it is critically important to provide physicians with additional data that suggest that QUINSAIR may reduce the number of pulmonary exacerbations in patients with a history of frequent pulmonary exacerbations. Further analyses suggest that QUINSAIR administration did not increase the risk for colonization with new pathogens or lead to increased antimicrobial susceptibilities.”

History of Pulmonary Exacerbations (PEx) as a Predictor of Response to Nebulized Levofloxacin Compared with Nebulized Tobramycin, P.A. Flume – Poster number 38.

Key findings:
  • MPEX-209 was a randomized, controlled, open-label study of levofloxacin inhalation solution, also known as MP-376/QUINSAIR, versus tobramycin inhalation solution over three 28-day cycles alternating with intervals of 28 days off treatment.
  • Subjects were categorized by prior-year pulmonary exacerbation (PEx) history as zero, one to two, or three or more. Study subjects were analyzed by these categories for PEx occurring during the study, which were defined as events resulting in treatment with antibiotics among patients meeting =4 of 12 respiratory signs and symptoms.
  • The analyses showed that subjects with three or more prior year PEx who were randomized to receive QUINSAIR had a significantly lower incidence of PEx when compared with their peers who were randomized to receive the active comparator, tobramycin inhalation solution (p=0.026).
  • The most frequently reported adverse reactions were cough/productive cough, dysgeusia and fatigue/asthenia.

    Microbiologic Changes Observed over 6 months in a Randomized, Open-Label Comparison of Inhaled Levofloxacin and Inhaled Tobramycin in Persons with CF and Chronic P. aeruginosa (Pa) Airway Infection, D.R. VanDevanter – Poster number 37.

    Key findings:

  • In MPEX-209, over the course of the three on/off cycle 168-day study of inhaled tobramycin or levofloxacin, no significant changes within treatment groups or differences between treatment groups were observed by selective culture with respect to: the proportion of P. aeruginosa (Pa) isolates susceptible to levofloxacin, tobramycin, aztreonam, or meropenem using EUCAST breakpoints and the proportion of Pa isolates not susceptible to at least 3 of these 4 classes.
  • The prevalence of CF bacterial opportunists cultured from study patients did not change significantly within treatment groups or between treatment groups over the course of the study.
  • The most frequently reported adverse reactions were cough/productive cough, dysgeusia and fatigue/asthenia.
About QUINSAIRTM (levofloxacin inhalation solution)

QUINSAIRTM is a proprietary inhaled formulation of levofloxacin, a fluoroquinolone antibiotic, which is approved in the EU and Canada for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with CF. Administration of QUINSAIRTM with the high efficiency Zirela® Nebulizer System (PARI Pharma GmbH) allows for the delivery of high concentrations of active drug directly to the site of infection in approximately five minutes. QUINSAIRTM is contraindicated in patients with hypersensitivity to levofloxacin, a history of tendon disorders related to fluoroquinolones, or epilepsy and also to patients who may be pregnant or breast feeding. QUINSAIRTM's safety was evaluated in two double-blind, placebo-controlled studies and an active comparator study in which the most frequently reported adverse reactions were cough/productive cough, dysgeusia and fatigue/asthenia.

About Raptor Pharmaceutical

Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of transformative therapeutics for rare, debilitating and often fatal diseases. Raptor is leading the global commercialization of two products for orphan diseases, including PROCYSBI®, for the management of nephropathic cystinosis in adults and children ages two years and older, and QUINSAIRTM, an inhaled fluoroquinolone antibiotic for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis (CF). Raptor's R&D pipeline includes RP103, known commercially as PROCYSBI®, for Huntington's disease and mitochondrial disorders, including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for nephropathic cystinosis in the U.S. and EU. The pipeline also includes MP-376, known commercially as QUINSAIRTM, which has Qualified Infectious Disease Product (QIDP) designation for three distinct indications: the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with CF and in patients with non-cystic fibrosis Bronchiectasis (BE), and in patients with nontuberculous mycobacteria (NTM). Raptor holds orphan drug designation in the U.S. for MP-376 for the treatment of CF, which, when added to the five years of exclusivity associated with QIDP designation, would confer 12 years of regulatory exclusivity upon FDA approval. For additional information, please visit www.raptorpharma.com.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are indicated by words or phrases such as "believes," "expects," "anticipates," "estimates," "plans," "continuing," "ongoing," "projected" and similar words or phrases and relate to future events, including statements regarding: , anticipated upcoming milestones, PROCYSBI® as a treatment option for patients with nephropathic cystinosis and RP103 as a treatment option for patients with Huntington's disease and mitochondrial disorders, including Leigh syndrome, orphan drug and regulatory exclusivity for MP-376 therapy in the U.S., Raptor's plans and timing to develop MP-376 in cystic fibrosis in the U.S., Bronchiectasis not associated with cystic fibrosis and potentially also nontuberculous mycobacteria and Raptor's other development programs. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause the company's actual results to be materially different from these forward-looking statements. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Factors which may contribute to differences in actual results include, among others: Raptor's ability to market and sell QUINSAIRTM; continued and increased market acceptance and sales of PROCYSBI® in the U.S. and other territories; Raptor's ability to expand the use of RP103 and MP-376 and to receive regulatory approval for other indications; Raptor's reliance on single active pharmaceutical ingredient suppliers for PROCYSBI and QUINSAIRTM and other third parties in connection with drug product development; compliance with healthcare regulations, ongoing regulatory requirements and potential penalties; any serious adverse side effects associated with PROCYSBI®, QUINSAIRTM or any other future products; any product liability claims; third-party payor coverage, reimbursement and pricing for PROCYSBI®, QUINSAIRTM and future products; enacted and future healthcare legislation; Raptor's ability to obtain and maintain orphan drug or other regulatory exclusivity for PROCYSBI®, QUINSAIRTM or any other future products; the integration of European operations with U.S. operations; relationships with key scientific and medical collaborators; intellectual property protection and claims and continued license rights; and Raptor's ability to fund its operations and make required payments on its debt. Certain of these risks, uncertainties and other factors are described in greater detail in the company's filings from time to time with the Securities and Exchange Commission (SEC), which Raptor strongly urges you to read and consider, including: Raptor's annual report on Form 10-K for the twelve months ended December 31, 2015 filed with the SEC on February 26, 2016, as amended by Amendment No. 1 to Form 10-K filed with the SEC on April 29, 2016 and Raptor's other periodic reports filed with SEC, all of which are available free of charge on the SEC's web site at http://www.sec.gov. Subsequent written and oral forward-looking statements attributable to Raptor or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in Raptor's reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements except as may be required by law.


Source: Raptor Pharmaceutical

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