Information from the 21/2/14 Press Release

Kalydeco recently received FDA (US) approval for eight gating mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. There are approx 150 people aged 6 and older with these mutations in the US, and 250 people aged 6 and older in Europe and Australia.

“We believe that KALYDECO has the potential to help more people with CF, and today’s approval is an important step toward that goal,” said¬†Robert Kauffman, M.D. Ph.D., Senior Vice President and Co-Chief Medical Officer at Vertex. “As we progress over the coming year, we look forward to data from multiple other ongoing studies that are designed to evaluate whether additional people with CF may benefit from KALYDECO.”

Summary of the 25/2/14 Vertex Investor Conference

Data is expected from the phase 3 VX809/Kalydeco trial in mid 2014. There is also a rollover study looking at the continued, sustained benefit.

If the phase 3 trials are successful, Vertex hope to launch the medication in 2015. If the phase 3 trials are unsuccessful, the data will be closely analysed (eg trends, efficacy, safety) and there is also the back up option of VX661.

This is a similar molecule to VX809, targeting the same folding mechanism. However, it acts in a different way, has less drug interactions (no dose adjustment needed with Kalydeco) and has different tissue penetrations.

The phase 2 VX661/Kalydeco trial will start soon. This is a 12 week study involving F508del homozygotes. VX661 is about 18 months to 2 years behind VX809.

Next Generation Correctors
It is thought that F508del heterozygous patients need three medications (triple therapy: VX661, next generation corrector, Kalydeco) in order to see a significant benefit. During the investor conference Michael Partridge mentioned ‘we don’t anticipate getting a clinical benefit with two drugs in the CF heterozygous patient population.’

Several next generation correctors are currently being investigated. Vertex aim to have one in trials by the end of this year, with combination studies in 2015. Vertex plan to use a next generation corrector that acts at a different site of the CFTR protein, providing additive and synergistic benefit with VX661.

Vertex also aim to use triple therapy to enhance the benefit in the F508del homozygous population.

Patients Taking Kalydeco
Vertex aim to enhance the benefit for patients already on Kalydeco by adding a corrector. There is currently a phase 2 trial where VX661 has been added to G551D/F508del patients who are already taking Kalydeco.

VX809/Kalydeco Pricing
Vertex believe that an approvable drug is a ‘really, really important drug.’

The collection of the data will be looked at in totality. Key endpoints include: FEV1, weight gain, exacerbations and quality of life. Vertex plan to look at ‘what is the value we are bringing to the patient?’

Sources: February Investor Conference & February Press Release


Join the conversation! 3 Comments

  1. Seem like the hetero’s keep getting farther and farther back in the herd. Quelle bummer.

    • I agree Christie. I really dislike how Vertex is structuring its trials with regard to the hetero’s. 809/661 plus Kalydeco unquestionably help hetero’s. Whether it meets some arbitrary threshold of the FDA is unknown. Vrtx should stop separating the best responders from the rest of the group because that hurts the chances of the heteros getting a drug approved. Put all together, the group average will be enough for approval and everyone can have a drug. some will be helped more than others, but everyone will be helped. They did the same with Kalydeco. They could have done the initial trial with all gating mutations. Instead they segregated out a subset of that group and, while it appears everything will work out, had the FDA not accepted N1 trials, many many gating mutations would have unnecessarily been left out. Doesn’t appear to be a patient first approach to me.

  2. does Vertex work with other country’s hand and hand for and with new break through discoveries or is this a monopoly effect to see who has successes first ,and hold back new break through informations leading up t solutions .? i would hope too think in the mwedical research field as important as this and many others is , not one to be monopolized in the sense of domonation in the market of discoveries.. I truly believe joined forces with other labs on a global scale will proove most rewarding . Stephen Earl Kirtland


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1. Class 2 Mutations & F508del, 1. Class 3 Gating Mutations & G551D, 3. Vertex - Kalydeco, 3. Vertex - Second Generation Correctors, 3. Vertex - VX809 & VX661