The Boomer Esiason Foundation

Category: Therapies

  • Two Phase 3 Studies of the Triple Combination of VX-445, Tezacaftor, and Ivacaftor Met Primary Endpoint of Improvement in Lung Function in People with Cystic Fibrosis

    SOURCE: VERTEX

    Two Phase 3 Studies of the Triple Combination of VX-445, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis

    • Mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline at week 4 in people with one F508del mutation and one minimal function mutation (F/MF) compared to placebo (p<0.0001)-
    • Mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 when VX-445 was added in people with two F508del mutations (F/F) already receiving tezacaftor and ivacaftor compared to control group of placebo added to tezacaftor and ivacaftor (p<0.0001)-
    • Safety and efficacy profile reported today supports potential submission of a New Drug Application for the VX-445 triple combination regimen-
    • Vertex plans to seek global regulatory approvals for either VX-659 or VX-445 triple combination regimen in both F/F and F/MF patient populations concurrently based on final 24-week data for both regimens expected in the second quarter of 2019-
    • U.S. NDA and EU MAA planned for the third and fourth quarters of 2019, respectively.

    BOSTON–(BUSINESS WIRE)–Mar. 6, 2019–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that treatment with the triple combination of the next-generation corrector VX-445, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). Data from a pre-specified interim analysis of the Phase 3 study in people with one F508del mutation and one minimal function mutation showed a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline at week 4 of treatment compared to placebo (p<0.0001). In the Phase 3 study in people with two F508del mutations, the addition of VX-445 in patients already receiving tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 of treatment compared to the control group in whom placebo was added to tezacaftor and ivacaftor (p<0.0001). The VX-445 triple combination regimen was generally well tolerated, and the safety and efficacy profile from the results released today supports the potential submission of a New Drug Application (NDA) for the VX-445 triple combination regimen.

    The Phase 3 data announced today for the VX-445 triple combination regimen follow Phase 3 data announced in late 2018 for the triple combination of VX-659, tezacaftor and ivacaftor that also showed a safety and efficacy profile supportive of a potential NDA submission. Given the similarity of the data for the 4-week primary efficacy endpoint for the VX-659 and VX-445 regimens and the near-term availability of the final 24-week data for both regimens in the second quarter of 2019, Vertex plans to utilize these final 24-week data to choose the best regimen to submit for regulatory approvals globally. Because these submissions will include the final 24-week data, Vertex will seek approval for patients ages 12 and older with one F508del mutation and one minimal function mutation and for patients with two F508del mutations concurrently. Vertex plans to submit an NDA to the U.S. FDA in the third quarter of 2019 and a Marketing Authorization Application (MAA) in Europe in the fourth quarter of 2019 for either the VX-659 or VX-445 triple combination regimen. The company also plans to disclose more detailed data from the Phase 3 studies of the selected triple combination regimen, including 24-week data from the study in patients with one F508del mutation and one minimal function mutation, in the second quarter of 2019.

    “Both the VX-659 and VX-445 triple combination regimens showed highly consistent and significant improvements in lung function across our Phase 3 programs, underscoring the important clinical benefit that a triple combination regimen may provide to patients with two F508del mutations and to those with one F508del and one minimal function mutation,” said Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We look forward to submitting global regulatory applications for one of these triple combination regimens for both patient populations later this year.”

    About the VX-445 Phase 3 Study in People with One F508del Mutation and One Minimal Function Mutation

    The data announced today for people ages 12 and older with one F508del mutation and one minimal function mutation are from an ongoing, randomized, double-blind, placebo-controlled Phase 3 study evaluating the triple combination of VX-445, tezacaftor and ivacaftor compared to triple placebo for 24 weeks. The study randomized 405 patients, and 403 patients received at least one dose of either the VX-445 triple combination regimen or triple placebo. In the U.S., the primary endpoint of the study is the mean absolute change in ppFEV1 from baseline at week 4 of triple combination treatment compared to triple placebo. The data announced today are from a pre-specified interim analysis that evaluated the primary endpoint at week 4. 402 patients had completed the week 4 visit of the study at the time of the interim analysis. The safety and efficacy profile in the interim analysis supports the potential submission of an NDA for the VX-445 triple combination regimen for patients with one F508del mutation and one minimal function mutation.

    Topline Data:

    Treatment with the triple combination of VX-445, tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline at week 4 compared to triple placebo (p<0.0001), which was the primary endpoint of the study. The mean absolute within-group improvement in ppFEV1 for those who received the VX-445 triple combination regimen was 13.6 percentage points from baseline at week 4. The mean absolute within-group change in ppFEV1 for those who received triple placebo was -0.2 percentage points from baseline at week 4.

    The VX-445 triple combination regimen was generally well tolerated in this study. The safety profile reflects all available safety data for all patients at the time of the interim analysis, including 246 patients who had reached the week 12 visit (123 patients who were randomized to the VX-445 triple combination regimen and 123 patients randomized to triple placebo) and 65 patients who had completed the 24-week treatment period (29 patients randomized to receive the VX-445 triple combination regimen and 36 patients randomized to receive triple placebo).

    This study is ongoing to evaluate the VX-445 triple combination regimen for a total of 24 weeks and will generate additional safety and efficacy data and data for key secondary endpoints, including the number of pulmonary exacerbations, change in sweat chloride, change in patient-reported outcomes as measured by the respiratory domain score of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) and change in body mass index, among others.

    Open-Label Extension Study:

    All patients who complete treatment in the 24-week study, regardless of treatment assignment, are given the opportunity to enroll in a rollover study where all patients receive the VX-445 triple combination regimen. All patients who had completed the study at the time of the interim analysis elected to enter the open-label extension study.

    About the VX-445 Phase 3 Study in People with Two F508del Mutations

    The data announced today for people with two F508del mutations are from a randomized, double-blind, controlled Phase 3 study that evaluated four weeks of treatment with the triple combination of VX-445, tezacaftor, and ivacaftor compared to placebo, tezacaftor and ivacaftor. The study randomized 108 patients ages 12 years or older who have two F508del mutations. All patients received tezacaftor in combination with ivacaftor during a 4-week run-in prior to randomization, and 107 of the 108 patients who were randomized received at least 1 dose of either the triple combination of VX-445, tezacaftor and ivacaftor or placebo, tezacaftor and ivacaftor. The primary endpoint of the study was the mean absolute change in ppFEV1 from baseline (end of the 4-week tezacaftor/ivacaftor run-in) at week 4 of triple combination of VX-445, tezacaftor and ivacaftor compared to placebo in combination with tezacaftor and ivacaftor. The data announced today reflect topline data from the primary efficacy and safety analysis conducted once all (n=107) patients completed the study. The safety and efficacy profile in this study supports the potential submission of an NDA for the VX-445 triple combination regimen for patients with two F508del mutations.

    Topline Data:

    Data from this study showed a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 when VX-445 was added in patients who were already receiving tezacaftor in combination with ivacaftor compared to those in whom placebo was added to tezacaftor and ivacaftor (p<0.0001), which was the primary endpoint of the study. The mean absolute within-group improvement in ppFEV1 from baseline for those who received VX-445 in triple combination with tezacaftor and ivacaftor was 10.4 percentage points at week 4. The mean absolute within-group change in ppFEV1 from baseline for those who received placebo, tezacaftor and ivacaftor was 0.4 percentage points at week 4.

    The VX-445 triple combination regimen was generally well tolerated in this study. All of the 107 patients who received either the triple combination of VX-445, tezacaftor and ivacaftor or placebo, tezacaftor and ivacaftor completed the 4-week triple combination treatment period.

    Open-Label Extension Study:

    Similar to the study in people with one F508del mutation and one minimal function mutation, all patients who completed treatment, regardless of treatment assignment, were given the opportunity to enroll in a rollover study where all patients received the VX-445 triple combination regimen. All 107 of the patients who completed the study elected to enter the open-label extension study.

    About CF

    CF is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

    Founded in 1989 in Cambridge, Mass., Vertex’s headquarters is now located in Boston’sInnovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including being named to Science magazine’s Top Employers in the life sciences ranking for nine years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com.

    Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

    Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445 were discovered by Vertex as part of this collaboration.

    Special Note Regarding Forward-looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kewalramani’s statements in the third paragraph, and the information provided regarding (i) the plan to seek global regulatory approvals for a triple combination regimen in both F/MF and F/F populations, (ii) the expected timing of the NDA and MAA submissions for a triple combination therapy and (iii) the plan to provide additional data regarding the selected regimen in the second quarter of 2019. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release, and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the triple combination studies are ongoing, that the company could experience unforeseen delays in submitting regulatory filings, that regulatory authorities may not approve, or approve on a timely basis, a triple-combination regimen due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

    View source version on businesswire.com: https://www.businesswire.com/news/home/20190306005389/en/

    Source: Vertex Pharmaceuticals Incorporated

    Investors:
    Michael Partridge, 617-341-6108
    or
    Eric Rojas, 617-961-7205
    or
    Zach Barber, 617-341-6470

    Media:
    617-341-6992
    mediainfo@vrtx.com

  • FDA Approves SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor)

    The U.S. Food and Drug Administration (FDA) approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) to treat cystic fibrosis (CF) in people ages 12 years and older who have two copies of the F508del mutation or one mutation that is responsive to SYMDEKO. SYMDEKO is Vertex’s third CF medicine and offers an important option for many patients, including those eligible who may be interested in a different treatment.

    Read the full press release here.

     

    SOURCE: VERTEX

  • Corbus Pharma Receives $25 Million Development Award to Support Phase 2b Clinical Study of Lenabasum

    Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) (“Corbus” or the “Company”), a clinical stage drug development company targeting rare, chronic, serious inflammatory and fibrotic diseases, announced today that it has received a Development Award for up to $25 million from the Cystic Fibrosis Foundation. The Development Award enables the Company to execute its Phase 2b study of its novel, oral, pro-resolving drug lenabasum (formerly known as anabasum) in approximately 415 people with cystic fibrosis (“CF”) who are 12 years and older and at increased risk for pulmonary exacerbations (“PEx”). Pulmonary exacerbations are severe inflammatory events in CF which are associated with acute worsening of respiratory signs and symptoms and sometimes irreversible loss of lung function.

     

    READ THE FULL PRESS RELEASE HERE

     

    SOURCE: https://ir.corbuspharma.com/press-releases/detail/261

  • Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation

    SOURCE 

     

    Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min)

    -Phase 2 data showed mean absolute improvements in ppFEV1 of 9.7 and 12.0 percentage points for VX-152 and VX-440, respectively, in triple combination with tezacaftor and ivacaftor in F508del/Min patients; Initial data from Phase 1 study showed mean absolute improvement in ppFEV1 of 9.6 percentage points with VX-659 triple combination in F508del/Min patients-

    -First data to demonstrate the potential to treat the underlying cause of CF in people with F508del/Min mutations, a severe and difficult-to-treat type of the disease-

    -Initial Phase 2 data also showed mean absolute improvements in ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation (F508del/F508del), who were already receiving tezacaftor and ivacaftor-

    -All 3 triple combination regimens were generally well tolerated across the studies-

    BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive data from Phase 1 and Phase 2 studies of three different triple combination regimens in people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min). These are the first data to demonstrate the potential to treat the underlying cause of CF in these patients, who have a severe and difficult-to-treat type of the disease. Data from the Phase 2 studies in these patients showed mean absolute improvements in percent predicted forced expiratory volume in one second (ppFEV1) of 9.7 and 12.0 percentage points from baseline for the triple combination regimens with VX-152 (200mg q12h) or VX-440 (600mg q12h), respectively. Initial data from a Phase 1 study showed a mean absolute improvement in ppFEV1 of 9.6 percentage points from baseline for the triple combination regimen of VX-659, tezacaftor and ivacaftor in people with one F508del mutation and one minimal function mutation. The company also announced today initial data showing improvements in mean absolute ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation, who were already receiving tezacaftor and ivacaftor. Vertex will host a conference call for investors today, July 18, 2017 at 5:00 p.m. EDT, to discuss these results.

    The triple combination regimens were generally well tolerated across all three studies, and the majority of adverse events were mild to moderate in severity. Across the studies, the discontinuation rate due to adverse events was low.

    “These safety and efficacy data are clear and compelling, indicating significant potential benefit for people with CF from each of these three different triple combination regimens,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We will be collecting and evaluating additional data from these and other studies and will make a decision on which regimen(s) to take forward into pivotal program(s), which we expect to begin in the first half of 2018.”

    Vertex has established a Steering Committee of global CF experts and clinical trial investigators to support the design, conduct and execution of the triple combination pivotal study program. This committee is co-chaired by Steven M. Rowe, M.D., M.S.P.H., Professor of Medicine, Pediatrics, and Cell, Developmental and Integrative Biology, Director of the Gregory Fleming James Cystic Fibrosis Research Center, Nancy and Eugene Gwaltney Endowed Chair for Medical Research, University of Alabama at Birmingham, and Jennifer Taylor-Cousar, M.D., Associate Professor, Departments of Medicine and Pediatrics, Pulmonary Divisions, Medical Director of Clinical Research Services and Co-Director and Director of the CF Therapeutics Development Network, Adult CF Program, National Jewish Health, Colorado.

    “Patients with minimal function mutations have been waiting for a medicine to treat the underlying cause of their disease, which makes these data showing pronounced improvements in lung function particularly important,” said Dr. Rowe. “It’s also encouraging to see that the addition of a next-generation corrector may lead to substantial additional benefits for patients with two copies of the F508del mutation, who were already receiving tezacaftor and ivacaftor.”

     

    FINISH READING THE ORIGINAL PRESS RELEASE HERE

  • PTC Therapeutics Announces Results from Pivotal Phase 3 Clinical Trial of Ataluren

    SOURCE: PRESS RELEASE VIA PTS THERAPEUTICS

    PTC Therapeutics Announces Results from Pivotal Phase 3 Clinical Trial of Ataluren in Patients Living with Nonsense Mutation Cystic Fibrosis

     

    – ACT CF trial missed primary and secondary endpoints –

    – Company to host conference call today, March 2nd at 9:00 am ET –

     

    SOUTH PLAINFIELD, N.J., March 2, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, which has now been used by more than 1,000 patients across multiple indications. PTC plans to discontinue current clinical development of ataluren in cystic fibrosis, close ongoing extension studies and withdraw its application for marketing authorization in cystic fibrosis in Europe.

     

    “We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause of nonsense mutation cystic fibrosis, one of the most difficult forms to treat,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We are particularly grateful to patients and investigators who participated in our trials. We remain committed to patients receiving ataluren in other indications.”

     

    ACT CF was a double-blind, placebo-controlled, 48-week clinical trial comparing ataluren to placebo in nmCF patients six years of age or older not receiving chronic inhaled aminoglycosides. The Phase 3 study, conducted in 16 countries, enrolled 279 patients who were randomized to receive either ataluren or placebo. In the intent-to-treat population, the primary endpoint of lung function as measured by absolute change in percent-predicted FEV1 (forced expiratory volume in one second), over 48 weeks from baseline, there was a 0.6% difference in favor of ataluren versus placebo (-1.4% change on ataluren versus -2.0% change on placebo; p=0.534). For the secondary endpoint of rate of pulmonary exacerbations, there was a trend in favor of ataluren, with the rate in the ataluren group being 14% lower than the placebo group (p=0.401). The results were not statistically significant. 

     

    The safety profile of ataluren in the ACT CF study was consistent with previous studies and no new safety signals were identified.

     

    About Cystic Fibrosis

    Cystic fibrosis is among the most common life-threatening genetic disorders worldwide. It is caused by defects in a single gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR. The CFTR gene encodes the CFTR protein, which is used by the body to transport chloride across cell membranes. Genetic mutations that result in the loss of function of the CFTR protein cause the body to produce abnormally thick and sticky mucus that clogs multiple organs, including the lungs, pancreas and liver. In particular, the absence or very low levels of CFTR leads to progressive loss of lung function, potentially life-threatening lung infections, permanent pancreatic damage and malnutrition because digestive enzymes from the pancreas do not reach the intestines to help break down and absorb food. The average age of death for CF patients is in their mid-thirties.

     

    About ataluren (Translarna™)

    Ataluren (brand name: Translarna™), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Ataluren is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States. The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

     

    About PTC Therapeutics

    PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.

     

    SOURCE: PRESS RELEASE VIA PTS THERAPEUTICS

  • Vertex Announces Planned Initiation of Phase 2 Studies Evaluating Next-Generation Correctors

    FULL PRESS RELEASE HERE

    Vertex Announces Planned Initiation of Phase 2 Studies Evaluating the Next-Generation Correctors VX-440 and VX-152 in Triple Combination Regimens to Treat the Underlying Cause of Cystic Fibrosis

    • VX-440 to be evaluated as part of 4-week triple combination dosing with tezacaftor (VX-661) and ivacaftor; VX-152 to be evaluated as part of 2-week triple combination dosing
    • Studies to enroll people with cystic fibrosis who have one copy of the F508del mutation and a minimal function mutation and also people with two copies of the F508del mutation
    • Additional next-generation correctors advancing into Phase 1 development; VX-659 Phase 1 clinical study expected to begin in 2016 and will enroll healthy volunteers and CF patients

    FULL PRESS RELEASE HERE

  • AbbVie and Galapagos to Co-Develop CF Therapies

    Sep 24, 2013

    SOURCE: ABBVIE 

    NORTH CHICAGO, Ill. and MECHELEN, Belgium, Sept. 24, 2013 /PRNewswire/ — Galapagos NV (Euronext: GLPG) and AbbVie (NYSE: ABBV) announced today that they have entered into a global alliance to discover, develop and commercialize novel potentiator and combination therapies in cystic fibrosis (CF), an inherited chronic disease that affects 70,000 people worldwide.

    AbbVie and Galapagos will work collaboratively to contribute technologies and resources in order to develop and commercialize oral drugs that address the main mutations in CF patients, including F508del and G551D.  The goal of the collaboration is to identify compounds that correct defects in expression of (corrector) and/or increase the activity (potentiator) of the main mutations in the cystic fibrosis transmembrane regulator (CFTR) protein, including the F508del mutation, which is the most common with 90 percent prevalence among patients with CF.

    In the alliance, AbbVie and Galapagos will develop potentiators and correctors discovered by Galapagos and expand the range of molecules, with the aim to initiate Phase 1 clinical studies at the end of 2014.  Following successful clinical development and regulatory approval, AbbVie will be responsible for commercial activities, with Galapagos retaining exclusive rights in China and South Korea and co-promotion rights in Belgium, the Netherlands, and Luxembourg. 

    Under the terms of the agreement, AbbVie will make an initial upfront payment of $45 million to Galapagos for rights related to the global alliance.  Upon successful completion of pre-determined success milestones, AbbVie and Galapagos will share responsibility and funding for Phase III clinical development.  Galapagos is eligible to receive up to $360 million in total additional payments for developmental and regulatory milestones, sales milestones upon the achievement of minimum annual net sales thresholds and additional double-digit royalty payments on net sales.

    “Galapagos is very pleased to join forces with AbbVie in this exciting new area of CF.  Our programs in CF show promise.  Partnering with AbbVie allows us to ramp up our commitment significantly, share development risk and expertise, and increase our chances of bringing best-in-class therapies to CF patients,” said Onno van de Stolpe, chief executive officer, Galapagos.

    “We’re pleased to enhance our partnership with Galapagos to include research in cystic fibrosis, a debilitating disease with significant unmet medical need. Our knowledge of the patient experience, combined with innovative advances in the understanding of disease etiology, offers the potential for new transformational treatments,” said Jim Sullivan, Ph.D., vice president, pharmaceutical discovery, AbbVie.

    Galapagos initiated its research in CF in 2005 as part of a collaboration with the Cystic Fibrosis Foundation.  In 2010, Galapagos decided to pursue CF as the first orphan disease in which the company is attempting to discover, develop and launch its own medicines.  Galapagos has developed small molecule therapies that can restore the function of the defective CF protein (CFTR).  The first pre-clinical candidate is expected to be nominated this year, with the first clinical trials starting at the end of 2014.

    Webcast presentation
    Galapagos will hold an audio webcast presentation for journalists, analysts, and investors today at 15.00 CET/9 am ET /6 am PT, viewable at http://www.glpg.com

    .

    About Cystic Fibrosis
    Cystic fibrosis (CF) is a hereditary disease of the entire body which leads to severe disability and early death in many cases.  Symptoms include frequent lung infections, sinus infections, poor growth, and diarrhea.  The cause is a defect in the gene which encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein which regulates components of sweat, mucus, and digestive juices.  CF affects approximately 70,000 people worldwide.  Patient symptoms are treated with antibiotics and other medicines.  There currently is no cure for the disease, and the predicted median age of survival is in the late 30s.

    About AbbVie
    AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott.  The company’s mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world’s most complex and serious diseases.  In 2013, AbbVie employs approximately 21,000 people worldwide and markets medicines in more than 170 countries.  For further information on the company and its people, portfolio and commitments, please visit http://www.abbvie.com.  Follow @abbvie on Twitter or view careers on our Facebook or LinkedIn page.

    About Galapagos 
    Galapagos (Euronext: GLPG; OTC: GLPYY) is specialized in novel modes-of-action, with a large pipeline of four clinical, six pre-clinical, and 20 discovery small-molecule and antibody programs in CF, inflammation, antibiotics, metabolic disease, and other indications.
    AbbVie and Galapagos signed an agreement in CF where they work collaboratively to develop and commercialize oral drugs that address two mutations in the CFTR gene, the G551D and F508del mutation.  In the field of inflammation, AbbVie and Galapagos signed a worldwide license agreement whereby AbbVie will be responsible for further development and commercialization of GLPG0634 after Phase 2B.  GLPG0634 is an orally-available, selective inhibitor of JAK1 for the treatment of rheumatoid arthritis and potentially other inflammatory diseases, currently in Phase 2B studies in RA and about to enter Phase 2 studies in Crohn’s disease.

    The Galapagos Group, including fee-for-service companies BioFocus, Argenta and Fidelta, has 800 employees and operates facilities in five countries, with global headquarters in Mechelen, Belgium.  Further information at: http://www.glpg.com

    Galapagos forward-looking statements

    This release may contain forward-looking statements, including, without limitation, statements containing the words “believes,” “anticipates,” “expects,” “intends,” “plans,” “seeks,” “estimates,” “may,” “will,” “could,” “stands to,” and “continues,” as well as similar expressions. Such forward-looking statements may involve known and unknown risks, uncertainties and other factors which might cause the actual results, financial condition, performance or achievements of Galapagos, or industry results, to be materially different from any historic or future results, financial conditions, performance or achievements expressed or implied by such forward-looking statements. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. Galapagos expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless required by law or regulation.

    AbbVie forward-looking statements
    Some statements in this news release may be forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995.  The words “believe,” “expect,” “anticipate,” “project” and similar expressions, among others, generally identify forward-looking statements.  AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements.  Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.  Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie’s operations is set forth in Item 1A, “Risk Factors,” in our 2012 Annual Report on Form 10-K/A, which has been filed with the Securities and Exchange Commission.  AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.

    SOURCE AbbVie

    For further information: AbbVie, Media, Adelle Infante, 847-938-8745, or Investors, Liz Shea, 847-935-2211; Galapagos NV, Onno van de Stolpe, Chief Executive Officer, Tel. +31 6 2909 8028, or Elizabeth Goodwin, Director Investor Relations, Tel: +31 6 2291 6240, ir@glpg.com

  • Cornerstone Therapeutics Inc. announced it is now actively marketing PERTZYE® in the US

    SOURCE

    Cornerstone Therapeutics Initiates PERTZYE(R) (pancrelipase) Sales and Marketing Efforts for the Treatment of Exocrine Pancreatic Insufficiency in Patients With Cystic Fibrosis
    CARY, NC–(Marketwired – Jul 29, 2013) – Cornerstone Therapeutics Inc. (NASDAQ: CRTX) announced today it is now actively marketing PERTZYE® (pancrelipase) in the U.S. for the treatment of Exocrine Pancreatic Insufficiency (EPI) due to cystic fibrosis. PERTZYE is the only U.S. Food and Drug Administration (FDA) approved pancreatic enzyme replacement therapy (PERT) containing bicarbonate-buffered, enteric-coated microspheres.1 It is estimated that about 90% of patients with cystic fibrosis require PERT.2

    In healthy individuals, the pancreas produces bicarbonate that helps regulate the pH of the small intestine to hydrolyze fat, support sufficient lipase bioactivity, and optimize nutrient absorption.3-5 Patients with CF who have EPI may have a more acidic duodenal pH environment because the pancreas does not produce the bicarbonate-rich secretion required to neutralize gastric acid.3,5 PERTZYE’s unique formulation, containing pancreatic enzymes buffered with bicarbonate, is designed to simulate normal pancreatic function in CF patients with EPI. 1,3-5

    “We are pleased to now be able to provide patients a unique PERT formulation to help manage this complex condition,” said Craig A. Collard, Cornerstone’s Chief Executive Officer. “PERTZYE is the first of two FDA approved cystic fibrosis therapies that Cornerstone plans to offer this year. PERTZYE, together with BETHKIS® (tobramycin inhalation solution) when it launches this fall, will deliver on our goal to build a portfolio of cystic fibrosis treatments and educational support for people living with this disease.”

    Cornerstone Therapeutics licensed exclusive U.S. marketing rights to PERTZYE for the treatment of EPI in patients with cystic fibrosis from Digestive Care, Inc. (DCI) in May 2013. DCI’s sales and marketing efforts for PERTZYE will continue to focus on meeting the needs of patients with EPI due to conditions other than cystic fibrosis in the U.S. market.

    ABOUT PERTZYE (pancrelipase) DELAYED-RELEASE CAPSULES

    PERTZYE is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Exocrine Pancreatic Insufficiency due to cystic fibrosis or other conditions. PERTZYE is not interchangeable with other pancrelipase products. PERTZYE is the only FDA approved PERT containing bicarbonate-buffered, enteric-coated microspheres. This unique formulation was designed to optimize the pH environment for enzyme activity and nutrient absorption.1 PERTZYE is currently available in two strengths: 8,000 and 16,000 USP units of lipase/capsule.

    Please read the rest of the press release here. 

  • FDA Approves New Powder form of Novartis TOBI

    A dry powder form of the antibiotic TOBI – TOBI Podhaler (tobramycin inhalation powder) – used to treat lung infections caused by the bacteria Pseudomonas aeruginosa has been approved by the US Food and Drug Administration (FDA).

    People with CF ages six and older who have Pseudomonas aeruginosa  and whose lung function (FEV1) is between 25 percent and 80 percent predicted can use the new Novartis treatment. 

    TOBI Podhaler does not require a nebulizer, does not need to be stored in a refrigerator, and does not require a power source to operate the inhaler. Administered with a pocket-sized inhaler, the new dry powder shortens treatment time by about 70 percent.  

    Novartis anticipates the availability of TOBI Podhaler in the United States in the second quarter of 2013. 

    Read the full Novartis press release here. 

  • PTC Therapeutics Announces Promising Results from Phase 3 Trial of Ataluren

    Source: Cystic Fibrosis Foundation

    PTC Therapeutics Inc. recently announced results from a Phase 3 clinical trial of ataluren, a potential oral drug aimed at treating the underlying cause of cystic fibrosis.

    The 48-week study tested ataluren in people with CF ages six and older who have what is known as a “nonsense mutation.”

    The results showed that study participants who received ataluren had a lower decline in lung function and a lower rate of pulmonary exacerbations, compared with those who took a placebo.

    PTC Therapeutics said it will be discussing the data with its clinical researchers and with advocacy groups to determine the next steps.

    About 10 percent of people with CF have nonsense mutations, which interrupt the production of the CFTR protein — the key protein associated with CF — causing it to be too short and not function normally. Ataluren is designed to enable the production of a full-length and functional CFTR protein.

    The Phase 3 study enrolled more than 230 volunteers at multiple sites in North America, Europe and Israel.