Here are the highlights from Vertex's CF R&D programs presented at the NACFC.
1. Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements in lung function through 48 weeks of treatment with lumacaftor in combination with ivacaftor in people with two copies of the F508del mutation.
> So not much new information from the Kalydeco combination trials for DelF508 except that sustained improvement is seen.
2. VX-661 in Combination with Ivacaftor (Kalydeco). VX-661 is Vertex's second CFTR corrector and is being developed to play a role in multiple combinations of CFTR modulators aimed at treating people with CF who have one or two copies of the F508del mutation. VX-661 is currently being evaluated in combination with ivacaftor as part of a 12-week Phase 2b study in people ages 18 and older who have two copies of the F508del mutation. Vertex today announced that this study is fully enrolled and that data will be available in early 2015.
> Great to see new clinical drugs in the pipeline.
3. New Drug Application submitted for approval of ivacaftor (Kalydeco) in children ages 2 to 5 with specific mutations in the CFTR gene who have one of the following nine mutations in the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.
> Great news for the little toddlers needing Kalydeco!
4. Triple Combination of VX-661, Ivacaftor and a Next-Generation Corrector: Vertex has multiple next-generation correctors in the lead-optimization stage of research and expects to begin clinical development of a next-generation corrector in 2015. In vitro data showed that a triple combination of VX-661, ivacaftor and a next-generation corrector resulted in increased chloride transport in human bronchial epithelial cells with one or two copies of the F508del mutation, as compared to the use of a single corrector in combination with ivacaftor.
> First triple combination data. I wander what the price of treatment will be if 2 additional drugs are used on top of Kalydeco?
There are approximately 22,000 people ages 12 and older who have two copies of the F508del mutation in North America, Europe and Australia, including approximately 8,500 in the United States and approximately 12,000 in Europe. So we can only assume that the idea of combination trials is to increase the market size which will in turn decrease the price of Kalydeco in combination with other drugs.
All-in-all, nothing ground-breaking to come out of NACFC from Vertex except that progress is being made and the pipeline of drugs and trials is growing to include more delF508 focus (60-70% of the CF population) and attempts to cater for children ages 2-5 with Kalydeco.
Source: http://t.co/KbciPoBqPl
- Chris Baldwin
1. Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements in lung function through 48 weeks of treatment with lumacaftor in combination with ivacaftor in people with two copies of the F508del mutation.
> So not much new information from the Kalydeco combination trials for DelF508 except that sustained improvement is seen.
2. VX-661 in Combination with Ivacaftor (Kalydeco). VX-661 is Vertex's second CFTR corrector and is being developed to play a role in multiple combinations of CFTR modulators aimed at treating people with CF who have one or two copies of the F508del mutation. VX-661 is currently being evaluated in combination with ivacaftor as part of a 12-week Phase 2b study in people ages 18 and older who have two copies of the F508del mutation. Vertex today announced that this study is fully enrolled and that data will be available in early 2015.
> Great to see new clinical drugs in the pipeline.
3. New Drug Application submitted for approval of ivacaftor (Kalydeco) in children ages 2 to 5 with specific mutations in the CFTR gene who have one of the following nine mutations in the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.
> Great news for the little toddlers needing Kalydeco!
4. Triple Combination of VX-661, Ivacaftor and a Next-Generation Corrector: Vertex has multiple next-generation correctors in the lead-optimization stage of research and expects to begin clinical development of a next-generation corrector in 2015. In vitro data showed that a triple combination of VX-661, ivacaftor and a next-generation corrector resulted in increased chloride transport in human bronchial epithelial cells with one or two copies of the F508del mutation, as compared to the use of a single corrector in combination with ivacaftor.
> First triple combination data. I wander what the price of treatment will be if 2 additional drugs are used on top of Kalydeco?
There are approximately 22,000 people ages 12 and older who have two copies of the F508del mutation in North America, Europe and Australia, including approximately 8,500 in the United States and approximately 12,000 in Europe. So we can only assume that the idea of combination trials is to increase the market size which will in turn decrease the price of Kalydeco in combination with other drugs.
All-in-all, nothing ground-breaking to come out of NACFC from Vertex except that progress is being made and the pipeline of drugs and trials is growing to include more delF508 focus (60-70% of the CF population) and attempts to cater for children ages 2-5 with Kalydeco.
Source: http://t.co/KbciPoBqPl
- Chris Baldwin