Research Update

The Science of Adding Tomorrows
By Andrea N. Flynn, PhD
Associate Research Scientist
New York University

Earlier this summer, Vertex Pharmaceuticals released the results of its Phase 2 clinical trial of Kalydeco™, the first ever FDA approved therapy to target the root cause of CF, in combination with VX-809, a CF drug currently in development.  The study lasted 56 days and examined the effects of the combination therapy in 109 individuals 18 years old or older with two copies of Delta F508, the most common CF mutation.  The study found individuals who received the highest dose (600 mg) of VX-809 in combination with Kalydeco had a significant improvement in lung function.  Based on these promising results, Vertex plans to begin pivotal trials in early 2013.  Pivotal trials are those that collect data the FDA can use to decide whether to approve a drug. Approximately 50% of individuals with CF have two copies of Delta F508, while another 40% have a single copy. Vertex also plans to conduct additional studies of the VX-809/Kalydeco combination therapy in individuals with a single copy of Delta F508 over the coming year.

PTC Therapeutics has also announced the results of their Phase 3 clinical trial of Ataluren, a therapy being developed to treat nonsense mutations.  Nonsense mutations result in the production of a shortened CF protein with reduced function, and are present in about 10% of individuals with CF. Ataluren is designed to facilitate the production of a full-length, functional CF protein.  The 48 week study included 230 volunteers, 6 years or older, in North America, Europe, and Israel.  Participants had a reduction in the decline of lung function and a lower rate of pulmonary exacerbations.  PTC Therapeutics is currently analyzing the data with clinical researchers and consulting with advocacy groups while it determines the next step.

Even more good news comes from Gilead Sciences, Inc.  Restrictions on the inhaled antibiotic Cayston®, which had been in place since early this year, have now been lifted.  Prescriptions previously on hold began shipping on September 4th and Cayston will once again be available through specialty pharmacies, as it was before the restriction.  Gilead has also discontinued its separate medical necessity exemption program.  Patients with questions about the availability of Cayston can call the Cayston Access Program at 1-877-7CAYSTON, visit www.Cayston.com/supply or email Caystoninfo@gilead.com.

An independent FDA advisory panel has voted to back a new, inhaled form of TOBI® (tobramycin solution for inhalation).  This outside panel decided the TOBI inhaled powder (TIP) is safe and effective for the treatment of Pseudomonas aeruginosa, a bacteria that is the leading cause of lung damage in individuals with CF.  Unlike TOBI, which is taken with a nebulizer, TIP can be taken with an inhaler, making it much more convenient.  Following this encouraging outside panel decision, Novartis Pharmaceutical Corporation is currently seeking FDA approval for TIP.

All of these therapies received significant support from the Cystic Fibrosis Foundation and underscore the Foundation’s commitment to find a cure for CF for all people with the disease as soon as possible.  Stay up to date on all the therapies in the Drug Development Pipeline at www.cff.org/research/DrugDevelopmentPipeline.

On Wednesday, October 17th, Credit Suisse hosted the Greater New York Chapter’s 2012 Scientific Update.  Dr. Bob Beall, President and CEO of the CF Foundation was the keynote speaker, and Katie McGee, Health and Wellness Reporter for WLNY 10/55’s “Live from the Couch” and Entertainment Reporter for CBS-2, was the evening’s moderator.  Before the presentation, guests enjoyed cocktails and many personally met Dr. Beall.  Following the cocktail hour, Katie McGee commented on the CF Foundation’s success as well as the Foundation’s long-standing relationship with CBS-2 before introducing Dr. Beall.  Dr. Beall captivated the audience as he shared research updates and medical advancements the Foundation has made over the past year. 

We are delighted to be able to share with you Dr. Beall’s Scientific Update presentation.  Please click here to view.