- |||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, posenacaftor (PTI-801) / Yumanity Therap
Comparison of cystic fibrosis transmembrane conductance regulator rescue in intestinal organoids with Fair Therapeutics triple combination with elexacaftor/tezacaftor/ivacaftor (Exhibition Hall) - Oct 8, 2022 - Abstract #NACFC2022NACFC_1186;
One of thesealternatives includes the triple modulator combination by FairTherapeutics (FT) consisting of a CFTR corrector (posenacaftor), potentiator(dirocaftor) and messenger ribonucleic acid amplifier (nesolicaftor). This project is in progress, but preliminary data showpromising results for the FT triple-combination treatment.643 Delivery of gp64-pseudotyped lentivirus carrying codon-optimizedcystic fibrosis transmembrane conductance regulator provides betterfunctional restoration in human cystic fibrosis airway epithelialcultures
- |||||||||| Symdeko (tezacaftor/ivacaftor) / Vertex, dirocaftor (PTI-808) / Yumanity Therap, exaluren (ELX-02) / Eloxx Pharma
Identification of organoid responders to CFTR modulators in the HIT-CF Europe project -Underlying the need for new treatment strategies for people with ultra-rare mutations (119 A) - Aug 5, 2022 - Abstract #NACFC2022NACFC_461;
Intestinal organoids of PwCF withultra-rare mutations were screened with compounds that had alreadypassed phase I and II clinical trials (dirocaftor (DIR)/posenacaftor (POS)/nesolicaftor (NES) and ELX-02)...In the DIR/POS/NES screen, PDOs were incubated for 24 hours withDIR/POS using a PDO from a F508del/F508del donor incubated for 24 hourswith tezacaftor/ivacaftor as the positive control...Based on organoid responsiveness to ELX-02 and DIR/POS, upto 78% of PwCF that carry ultra-rare mutations could benefit fromupcoming CFTR modulating therapies. Although we have taken a greatstep forward in personalized medicine for PwCF, there is still a large unmetneed for those who haveCFTRclass VII (unrescuable) mutations or variantsthat cannot be restored by the tested CFTR modulating therapies.
- |||||||||| posenacaftor (PTI-801) / Kineta
Trial completion: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Apr 30, 2020
P1, N=171, Completed,
Based on their response in the organoid test assay a subset of subjects will be invited to participate in a study to confirm clinical efficacy. Recruiting --> Completed
- |||||||||| posenacaftor (PTI-801) / Kineta
Trial completion date, Trial primary completion date: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Jan 23, 2020
P1, N=180, Recruiting,
Active, not recruiting --> Completed Trial completion date: Dec 2019 --> Mar 2020 | Trial primary completion date: Dec 2019 --> Mar 2020
- |||||||||| PTI-801 / Proteostasis
CURRENT STATUS OF THE PROTEOSTASIS THERAPEUTICS CFTR MODULATOR DEVELOPMENT PROGRAM (205) - Nov 11, 2019 - Abstract #NACFC2019NACFC_50;
P1/2
Similarly, in vitro CFTR activity of the triple combination of PTI 801 and PTI 808 with the PTI 428 amplifier is superior to that seen with tezacaftor/ivacaftor in combination with VX-659 (corrector) in homozygous and heterozygous F508del cell cultures (data to be presented)...A phase 2 study is currently ongoing to evaluate the effects of PTI 808 in combination with PTI 801, with or without PTI 428, over a 28-day treatment period in CF subjects who are either homozygous or heterozygous for the F508del CFTR genotype. The goal is to initiate a phase 3 study in 2020.
- |||||||||| PTI-808 / Proteostasis, PTI-428 / Proteostasis, PTI-801 / Proteostasis
EVALUATION OF NOVEL CFTR MODULATOR COMBINATIONS OF THE CORRECTOR PTI-801, POTENTIATOR PTI-808, AND AMPLIFIER PTI-428 IN CF SUBJECTS () - Nov 11, 2019 - Abstract #NACFC2019NACFC_8;
In vitro, in human bronchial epithelial cells from F508del homozygous donors, the combinations of PTI-801+PTI-808 and PTI-801+PTI-808+PTI-428 increased CFTR chloride transport activity by 193% and 369%, compared to that of tezacaftor+ivacaftor, respectively, suggesting a superior in vitro response to a currently approved modulator combination. PTI-801, PTI-808 and PTI-428 represent novel CFTR modulators in clinical development.
- |||||||||| posenacaftor (PTI-801) / Kineta
Trial completion date, Trial primary completion date: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Sep 9, 2019
P1, N=180, Recruiting,
PTI-801, PTI-808 and PTI-428 represent novel CFTR modulators in clinical development. Trial completion date: Jun 2019 --> Dec 2019 | Trial primary completion date: Jun 2019 --> Dec 2019
- |||||||||| dirocaftor (PTI-808) / Kineta
Trial completion, Enrollment change, Combination therapy: Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Sep 5, 2019
P1/2, N=12, Completed,
Trial completion date: Jun 2019 --> Dec 2019 | Trial primary completion date: Jun 2019 --> Dec 2019 Recruiting --> Completed | N=32 --> 12
- |||||||||| posenacaftor (PTI-801) / Kineta
Enrollment change, Trial completion date, Trial primary completion date: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Jan 22, 2019
P1, N=180, Recruiting,
Phase classification: P1 --> P1/2 | N=135 --> 180 | Trial completion date: Jun 2019 --> Jan 2020 | Trial primary completion date: Jun 2019 --> Jan 2020 N=125 --> 180 | Trial completion date: Aug 2018 --> Jun 2019 | Trial primary completion date: Aug 2018 --> Jun 2019
- |||||||||| dirocaftor (PTI-808) / Kineta
Trial completion date, Trial primary completion date, Combination therapy: Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Nov 15, 2018
P1, N=32, Recruiting,
Phase classification: P1 --> P1/2 Trial completion date: Nov 2018 --> May 2019 | Trial primary completion date: Oct 2018 --> Apr 2019
- |||||||||| posenacaftor (PTI-801) / Kineta
Trial completion date, Trial primary completion date: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Feb 22, 2018
P1, N=120, Recruiting,
Trial completion date: Nov 2017 --> Jun 2019 | Trial primary completion date: Nov 2017 --> Feb 2019 Trial primary completion date: Apr 2018 --> Aug 2018 | Trial completion date: Jul 2018 --> Aug 2018
- |||||||||| posenacaftor (PTI-801) / Kineta
Enrollment change, Trial primary completion date: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Aug 7, 2017
P1, N=120, Recruiting,
Trial primary completion date: Apr 2018 --> Aug 2018 | Trial completion date: Jul 2018 --> Aug 2018 N=60 --> 120 | Trial primary completion date: Jul 2017 --> Apr 2018
- |||||||||| posenacaftor (PTI-801) / Kineta
New P1 trial: Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis (clinicaltrials.gov) - May 4, 2017
P1, N=60, Recruiting,
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