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Vertex Presenting 15 Cystic Fibrosis Abstract at Upcoming Conference

SEPTEMBER 08, 2014
James Radke

Vertex Pharmaceuticals announced they will present a staggering 15 abstracts at the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta, GA, October 9 - 11, 2014.  

Kalydeco plus lumacoftor

Key among the presentations will be ones focused on data from the Phase 3 TRAFFIC and TRANSPORT studies of the company’s pipeline drug lumacaftor in combination with their approved drug Kalydeco (ivacaftor) in people with cystic fibrosis who have two copies of the F508del mutation.  

Right now, Kalydeco can treat about 2000 patients with cystic fibrosis and has the potential to reach about 7,000.  Vertex’s Lumacaftor or VX-809 in combination with Kalydeco is being tested on patients with  F508del homozygous who account for over 28,000 patients with cystic fibrosis.

These presentations include:
  • “Effect of lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for F508del-CFTR: Phase 3 TRAFFIC & TRANSPORT studies.” An oral presentation of these data will be delivered as part of an invited talk during Symposium Session II on October 10 at 11:30 a.m. ET.
  • “Effect of lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for F508del-CFTR: TRAFFIC Study.” Poster 249.
  • “Effect of lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for F508del-CFTR: TRANSPORT Study.” Poster 250.
Other presentation association with the combination Kalydeco and lumacaftor include:
  • “The effect of ciprofloxacin, itraconazole, and rifampin on the pharmacokinetics of lumacaftor in combination with ivacaftor in healthy individuals.” Poster 201.
  • “Effect of lumacaftor in combination with ivacaftor on FEV1 and safety measures in patients aged 6-11 years with CF who are homozygous for F508del-CFTR.” Poster 203.
  • “Effect of 8 weeks of lumacaftor in combination with ivacaftor in patients with CF and heterozygous for the F508del-CFTR Mutation.” Poster 254.
  • “Effect of bronchodilators in healthy individuals receiving lumacaftor in combination with ivacaftor.” Poster 256.

Kaledcyo monotherapy

Data from studies involving Kalydeco in different genetic mutations of cystic fibrosis are also being presented. At present, Kalydeco (150 mg tablets) is indicated for the treatment of cystic fibrosis in patients age 6 years and older who have  one of the following mutations in the  CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N,  or S549R.

Multiple Phase 2 and Phase 3 studies of Kalydeco monotherapy will be presented,  including the first presentation of the Phase 3 results of Kalydeco treatment in children ages 2 to 5. Presentations related to Kalydeco monotherapy include:
  • “R117H is a residual function mutation that is potentiated by ivacaftor.” Poster 2. An oral presentation of these data will also be delivered during Workshop Session I on October 9 at 10:25 a.m. ET.
  • “Patient-reported treatment effects of ivacaftor beyond respiratory symptoms in patients with cystic fibrosis.” Poster 98. An oral presentation of these data will also be delivered during Workshop Session II on October 10 at 3:10 p.m. ET.
  • “Effects of ivacaftor in CF patients with R117H-CFTR.” Poster 17. An oral presentation of these data will also be delivered during Workshop Session II on October 10 at 3:25 p.m. ET.
  • “Utilization of an ‘n-of-1’ study design to test the effect of ivacaftor in cystic fibrosis patients with residual CFTR function and FEV1 ≥ 40% of predicted.” An oral presentation of these data will be delivered as part of an invited talk during Symposium Session III on October 11 at 11:55 a.m. ET.
  • “Effect of ivacaftor in patients with cystic fibrosis, residual CFTR function and FEV1 ≥ 40% of predicted, N-of-1 study.” Poster 196. An oral presentation of these data will also be delivered during Workshop Session III on October 11 at 3:00 p.m. ET.
  • “An open-label study of the safety, pharmacokinetics & pharmacodynamics of ivacaftor in patients aged 2 to 5 years with CF & a CFTR Gating Mutation: The KIWI Study.” Poster 200. An oral presentation of these data will also be delivered during Workshop Session III on October 11 at 4:00 p.m. ET.
  • “Health resource utilization among patients with cystic fibrosis who initiate ivacaftor treatment.” Poster 202.
  • “The Effect of Ivacaftor on Weight Over Three Years in Patients with CF and a G551D-CFTR Mutation.” Poster 207.

Kalydeco plus VX-661

VX-661 is another corrector being tested in combination with Kalydeco that is being explored in both F508del homozygous and heterozygous patients (>45,000 patients).
  • “Phase 2 studies reveal additive effects of VX-661, an investigational CFTR corrector, and ivacaftor, a CFTR potentiator, in patients with CF who carry the F508del-CFTR mutation.” An oral presentation of these data will be delivered as part of an invited talk during Symposium Session II on October 10 at 11:55 a.m. ET.
  • “Addition of VX-661, an investigational CFTR corrector, to ivacaftor, a CFTR potentiator, in patients with CF and heterozygous for F508del/G551D-CFTR.” Poster 260.

About Cystic Fibrosis

In the United States, there are approximately 30,000 people living with cystic fibrosis (70,000 worldwide).  Cystic fibrosis is caused by a mutation in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation is  F508del; however, there are over 1500 other mutations that can produce cystic fibrosis.

Disruption of the protein CFTR leads to secretion/mucus build up in organs, especially in the lung.  Kalydeco is the only treatment currently available that addresses the underlying cause of cystic fibrosis and it is approved for persons with the G551D mutation as well as several other mutations that account for about 2,000 of the 70,000 patients with cystic fibrosis.

Patients with cystic fibrosis must regularly clear their airways to avoid mucus buildup (with physical therapy techniques and/or inhaled medications). The chronic condition also benefits from regular exercise to clear mucus in the lungs and dietary enzyme supplements to improve absorption.  For more information about cystic fibrosis, visit http://www.cff.org

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