June 12, 2013

June Vertex Update

Kalydeco Monotherapy Beyond G551D

Kalydeco is available for G551D patients in the US and many European Countries
There are about 1000 G551D patients aged over 6 in the US and 1000 in Europe (3-4% of CF population)

Potential label expansion:
– other gating mutations
– residual function, including R117H- where channel is at the surface
– Data expected from these studies in the second half of this year
– Hoping for label expansion in 2014

Expand from 3-4% to 10-15% of the 70,000 CF patients worldwide = 7000 – 10000 patients

 VX809 Breakthrough Designation

Allows ongoing discussions with FDA, more resources from FDA and allows “ongoing dialogues of how we may get the drug to the patient as fast as possible, pending clinical results.”

Phase 3 VX809/Kalydeco

– Potential to treat another 50% of the 70,000 (homozygous F508del)
– Harder to treat as the channel is not at the surface- need a trafficking molecule such as VX809
– Kalydeco can then help it to open
– Encouraging phase 2 results
– Currently enrolling patients in the two phase 3 programs, 500 patients each
– Anticipate data next year (first or second half). Will update timeframe later this year when the trials have finished enrolling
– Study design- 2 different doses and placebo, 6 months duration. Endpoints: FEV1 (primary), pulmonary exacerbations, weight gain & how the patient feels.

Upcoming data presented at European Cystic Fibrosis Conference

Presenting 3 pieces of data
1. VX809: Additional analysis from Cohort 3 (BID dosing)
2. VX661: Additional data.
3. Next generation correctors: In vitro data. Plan to enhance benefit by approaching the folding in a different way with a combination of first and second generation correctors

VX661 and VX983 Next Steps

VX809 is 1 year to 18 months ahead of VX661. VX809 has a PK (drug interaction) with Kalydeco which means dosing needs to be increased. VX661 and VX983 are similar (but different to VX809). They do not interact with Kalydeco.

Plan: Progress both VX661 and VX983, then choose one (based on safety/benefit), then look at how they combine with second generation correctors, then trials with triple combination therapy (first & second generation corrector with Kalydeco, for both F508del homozygotes and heterozygotes)

– VX983/Kalydeco- 1-month study later this year, data next year
– VX661-  ongoing discussions with FDA
– Second generation corrector- hope to be in trials by end of 2014

Current Pricing

Based on value and benefit to patient (both Kalydeco and future combination therapy)

Europe- challenging, more financial restrictions, many countries with different reimbursement processes. Pricing based on value and benefit.

Sources: June Vertex Investor Conference (no longer available) and Seeking Alpha Transcript


Join the conversation! 7 Comments

  1. I don’t see mention of trials for 2-5 y/o g551d for Kalydeco expansion. Do you have any information on expected results of that ongoing trial subset? When does Vertex expect initial data and foresee its availability to these young children?

  2. Hi Kelly, the 2-5 year old trial was not mentioned during the latest vertex presentation, but has been mentioned in most of the recent presentations. In April the trial was still in the enrolling phase. I am not sure of any more details regarding the timeline.

  3. Hopefully the label expansion will bring down the price.. grrr…

  4. Thanks for the info! If they are still “enrolling,” in the 2-5 y/o trial on Clinicaltrials.gov, why is the primary outcome measure expected in October 2013? I would love to know if there are children in this age group participating in the trials now 🙂

    • Hi Kelly, I think your question was probably answered with my latest vertex update (31/7/13): there are two parts to the 2-5 yo trial and part 1 has finished, which would be the results expected in Oct 2013. Part 2 is enrolling and results are expected mid 2014.


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1. Class 2 Mutations & F508del, 1. Class 3 Gating Mutations & G551D, 1. Residual Function Mutations & R117H, 3. Vertex - Kalydeco, 3. Vertex - Second Generation Correctors, 3. Vertex - VX809 & VX661