New Potentiators
Galapagos are developing a new series of potentiators that show superior CFTR opening in comparison to Kalydeco. This superior opening was seen with class 2, 3 or 4 mutations, including F508del and G551D (see graph/image).
This new series are highly potent and have favourable PK profiles. These potentiators have a superior in vitro safety profile in comparison to Kalydeco, including a clean CYP profile (less interaction with other medications).
Galapagos plan to nominate a pre-clinical candidate by the end of 2013 and plan to start clinical trials with this potentiator by the end of 2014.

Galapagos & AbbVie Alliance
“In September 2013 Galapagos and AbbVie entered into a global alliance to discover, develop and commercialize novel therapies in cystic fibrosis (CF), an inherited chronic disease that affects 70,000 people worldwide. AbbVie and Galapagos are working collaboratively, contributing technologies and resources 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 (corrector) of 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.”

Galapagos AbbVie Cystic Fibrosis Timeline

This image, from the Galapagos website, shows the planned timeline.

Sources: October 17 and September 24 Galapagos Press Releases and Galapagos Website

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Join the conversation! 9 Comments

  1. First thank you so much for this information. I want you to know that I appreciate your research and blog so much. Thank you. I am sure you get asked this a lot, but would the new drugs help someone with only one class 2 mutation? My six month old son has deltaf508 and 1154insTC (aka c.1021_1022dupTC), so he has class 1 and class 2.

    Thanks for any insight.

    Tricia

    >

    Reply
    • Hi Tricia, People that have 2 copies of F508del produce more F508del that can be corrected and potentiated. People with 1 copy would have some improvement but there is less F508del that the meds can help. The most developed approach for heterozygotes (1 copy F508del) is the Vertex triple combination therapy, where in the lab, three meds help people with F508del to reach a higher level of improvement. Vertex plan to have a second generation corrector (part of triple therapy) in trials by the end of this year. These graphs show the in vitro (lab) improvement with triple therapy: https://sixtyfiverosesblog.wordpress.com/2013/06/13/vertex-second-generation-correctors-latest-graphs/

      Reply
  2. My 15 month old has 1154insTC and delta i507 class 1 and 2 as well so same question here.

    Reply
    • Hi Mike, I have just answered Tricia’s question which hopefully will help with yours as well. Correctors (such as VX809) are currently being tested with F508del. I507del involves the adjacent amino acid, however laboratory tests have shown that VX809 may not help I507del significantly: https://sixtyfiverosesblog.wordpress.com/2013/03/26/can-vx809-help-other-processing-mutations-besides-f508del/ Based only on that information it is hard to say whether someone with I507del could improve with VX809. It is possible that other correctors, potentially from different companies, may help I507del to a greater extent. Gen

      Reply
      • Hi, Now that some years have passed I was wondering if you know if these new triple combination therapies being developed by Vertex will help someone with I507del and 1154insTC?

  3. EXELENTES NOTICIAS . LO ULTIMO ESTA EN ESTE BLOG, POR SUPUESTO DESDE ALTINOAMRICA AL REENVIARLO SE ESTAN ENTERADO MILES Y MILES DE PACIENTES FQ . M GRACIAS ¡¡ A DISPOSICION JORGE

    Reply
  4. I wish you had access to the absurdly-expensive “Global Cystic Fibrosis Pipeline Highlights – 2014” report. It sounds like it’d be perfect for you / this site!

    Reply
    • Hi Tom, that report does sound very interesting but you are right the price is ridiculous! I will try emailing the company to see if I can get it for free, you never know, sometimes that works! Gen

      Reply
  5. Really good news

    Reply

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Category

1. Class 2 Mutations & F508del, 1. Class 3 Gating Mutations & G551D, 1. Residual Function Mutations & R117H, 3. Galapagos - GLPG1