Aboveallislove
Super Moderator
http://investors.vrtx.com/releasedetail.cfm?ReleaseID=1033559,
A wonderful day.
A wonderful day.
Vertex Triple works for het-mins and improves ddf508 better!
- Thread starter Aboveallislove
- Start date
Thanks for sharing Aboveallislove!
These are incredible results! Happy tears
Summary of Results:
In the Phase 2 clinical trial of VX-440 in combination with ivacaftor and tezacaftor (VX-661), the 18 participants in the study with a single F508del mutation who received the 600 mg dose of VX-440 had a 12 percent increase in lung function, substantial improvement in their quality of life, and a decrease in sweat chloride of 33.1 after four weeks of treatment. The 20 participants in the study with two copies of the F508del mutation who received the 600 mg dose of VX-440 had a 9.5 percent increase in lung function and a decrease in sweat chloride of 31.3 after four weeks of treatment.
In the Phase 2 clinical trial of VX-152 in combination with ivacaftor and tezacaftor (VX-661), the 10 participants in the study with a single F508del mutation who received the 200 mg dose of VX-152 had a 9.7 percent increase in lung function and a decrease in sweat chloride of 14.1 after two weeks of treatment. The 10 participants in the study who have two copies of the F508del mutation who received the 200 mg dose of VX-152 had a 7.3 percent increase in lung function and a decrease in sweat chloride of 20.9 after two weeks of treatment.
In the Phase 1 clinical trial of VX-659 in combination with ivacaftor and tezacaftor (VX-661), participants in the study with a single F508del mutation who received the 120 mg q12 dose of VX-659 had a 9.6 percent increase in lung function and decrease in sweat chloride of 41.6 after two weeks of treatment.
Pregnancy Risks
Women of childbearing age in the VX-440 study were required to use contraception because of worries about the drug’s effects on fetuses, which may argue in favor of VX-152, where the same risk wasn’t seen, according to Jennifer Taylor-Cousar, a cystic fibrosis doctor at National Jewish Health in Denver and investigator in one of the studies who is consulting for Vertex on its final-stage study plans.
“To have to decide not to get pregnant would be very difficult for our patients,” Taylor-Cousar said by phone. “As women are healthier and healthier with CF and living longer they definitely want to have families.”
Patients generally tolerated the drugs well, according to Vertex, though some side effects cropped up. One patient taking a triple combination with VX-440 had higher-than-normal liver enzymes, which can suggest liver damage, and stopped taking treatment. Another taking VX-152 stopped taking the drug cocktail after contracting pneumonia.
These are incredible results! Happy tears
Summary of Results:
In the Phase 2 clinical trial of VX-440 in combination with ivacaftor and tezacaftor (VX-661), the 18 participants in the study with a single F508del mutation who received the 600 mg dose of VX-440 had a 12 percent increase in lung function, substantial improvement in their quality of life, and a decrease in sweat chloride of 33.1 after four weeks of treatment. The 20 participants in the study with two copies of the F508del mutation who received the 600 mg dose of VX-440 had a 9.5 percent increase in lung function and a decrease in sweat chloride of 31.3 after four weeks of treatment.
In the Phase 2 clinical trial of VX-152 in combination with ivacaftor and tezacaftor (VX-661), the 10 participants in the study with a single F508del mutation who received the 200 mg dose of VX-152 had a 9.7 percent increase in lung function and a decrease in sweat chloride of 14.1 after two weeks of treatment. The 10 participants in the study who have two copies of the F508del mutation who received the 200 mg dose of VX-152 had a 7.3 percent increase in lung function and a decrease in sweat chloride of 20.9 after two weeks of treatment.
In the Phase 1 clinical trial of VX-659 in combination with ivacaftor and tezacaftor (VX-661), participants in the study with a single F508del mutation who received the 120 mg q12 dose of VX-659 had a 9.6 percent increase in lung function and decrease in sweat chloride of 41.6 after two weeks of treatment.
Pregnancy Risks
Women of childbearing age in the VX-440 study were required to use contraception because of worries about the drug’s effects on fetuses, which may argue in favor of VX-152, where the same risk wasn’t seen, according to Jennifer Taylor-Cousar, a cystic fibrosis doctor at National Jewish Health in Denver and investigator in one of the studies who is consulting for Vertex on its final-stage study plans.
“To have to decide not to get pregnant would be very difficult for our patients,” Taylor-Cousar said by phone. “As women are healthier and healthier with CF and living longer they definitely want to have families.”
Patients generally tolerated the drugs well, according to Vertex, though some side effects cropped up. One patient taking a triple combination with VX-440 had higher-than-normal liver enzymes, which can suggest liver damage, and stopped taking treatment. Another taking VX-152 stopped taking the drug cocktail after contracting pneumonia.
Aboveallislove
Super Moderator
Yes happy tears here too. There are 2 more drugs in the ring still and they are both BETTER in vitro. And hopefully don't have the same ris\k re pregnancy.
Aboveallislove
Super Moderator
The study tested CFers with one copy of df508 and one copy of another gene not expected to benefit from Orkambi/next generation because it has minimal function (so some class 1). We're getting closer. So the tripple wor\ks for anyone with at least one df508 and with close to |Kalydeco FEV improvement
Incredible results!
Does anyone know if they also intend to study the triple combo in people with gating and residual function mutations, plus one copy of df508? I realize the het/min population is the more urgent priority, but it seems to me the triple combo will likely be beneficial to all with at least one copy of df508, regardless of what the other mutation is. I listened to the investor call yesterday but it wasn't mentioned.
I also wonder whether they are using a specific list of minimal function mutations, or if they're basically just saying any mutation BUT the gating and residual function ones already identified. I have concerns for people like myself with ultra-rare mutations if they are restricting to a particular list of mutations.
Does anyone know if they also intend to study the triple combo in people with gating and residual function mutations, plus one copy of df508? I realize the het/min population is the more urgent priority, but it seems to me the triple combo will likely be beneficial to all with at least one copy of df508, regardless of what the other mutation is. I listened to the investor call yesterday but it wasn't mentioned.
I also wonder whether they are using a specific list of minimal function mutations, or if they're basically just saying any mutation BUT the gating and residual function ones already identified. I have concerns for people like myself with ultra-rare mutations if they are restricting to a particular list of mutations.
Aboveallislove
Super Moderator
The strategy is to have one triple for anyone with df508 or where \known benefit of Or\kambi and one for those with 551 and the second a het-min. How they test that will be difficult to \know and will depend on the FDA. My "gut" will be they run a trial for the triple that involves one arm with het-mins and one with dd508 and maybe a third with 2 mutations already approved for Orkambi (so how that was defined) that don't have df508. My gut is based on what the FDA is li\kely to want to see evidence of and the strategy Vertex gave yesterday of one triple for all but those with 551 and het-min.
Romina Primavera
New member
Hi, im new in this website, I would like to ask you all about new information about Vertex Triple. Im from Uruguay, and I have CF, I have 27 years old. is available this new treatment?
I have df508 and another unknown mutation. In which country is available this treatment? Im sorry for my bad English but I have to put it into practice.
I have df508 and another unknown mutation. In which country is available this treatment? Im sorry for my bad English but I have to put it into practice.
Aboveallislove
Super Moderator
Romina: Your English was great! These medicines was still in trials but as long as you have one copy of df508 they should work for you if the trials go well. I do not know if they do trials in your country but can you check with you doctors to find out? The trials will likely begin mid 2018.