 |
1 Disease |  |
0 Trials |
|
0 Trials |  |
35 News |  |
- | dirocaftor (PTI-808) / Kineta, nesolicaftor (PTI-428) / Kineta, posenacaftor (PTI-801) / Kineta
New P2 trial: CHOICES: Clinical Trial to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Adults With CF (clinicaltrials.gov) - Jun 21, 2024
P2, N=52, Recruiting, Sponsor: Kors van der Ent
- | nesolicaftor (PTI-428) / Yumanity Therap
Journal: The CFTR Amplifier Nesolicaftor Rescues TGF-β1 Inhibition of Modulator-Corrected F508del CFTR Function. (Pubmed Central) - Oct 1, 2022
Finally, nesolicaftor augmented the F508del CFTR response to ETI in CFBE cells overexpressing miR-145, a negative regulator of CFTR expression. Thus, CFTR amplifiers, but only when used with highly effective modulators, may provide benefit in an inflamed environment.
- | nesolicaftor (PTI-428) / Yumanity Therap
Journal: Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene. (Pubmed Central) - Dec 16, 2021
Importantly, we never found cooperativity between the NMD inhibitor and readthrough compounds. Our results indicate that treatment of CF patients with nonsense mutations requires a precision medicine approach with the design of specific drug combinations for each mutation.
- [VIRTUAL] CFTR MODULATOR THERAPY FOR CYSTIC FIBROSIS CAUSED BY THE RARE C.3700A>G MUTATION () - Oct 7, 2020 - Abstract #NACFC2020NACFC_839;
One subject showed a 30 mM decrease in sweat chloride and symptomatic improvement on ETI. These results suggest the potential benefit of CFTR modulators, including co-potentiators, for CF caused by the c.3700A G mutation.
- dirocaftor (PTI-808) / Proteostasis, nesolicaftor (PTI-428) / Proteostasis, posenacaftor (PTI-801) / Proteostasis
[VIRTUAL] EVALUATION OF COMBINATIONS OF THE CFTR POTENTIATOR DIROCAFTOR, CORRECTOR POSENACAFTOR AND AMPLIFIER NESOLICAFTOR IN CF SUBJECTS WITH TWO COPIES OF THE F508DEL MUTATION () - Aug 26, 2020 - Abstract #NACFC2020NACFC_302;
In vitro, in human bronchial epithelial cells from F508del homozygous donors, the combination of DIR/POS/ NES increased CFTR chloride transport activity to levels comparable to that elicited by the elexacaftor/tezacaftor/ivacaftor combination... Dirocaftor, posenacaftor and nesolicaftor represent novel CFTR modulators in clinical development.
- dirocaftor (PTI-808) / Proteostasis, nesolicaftor (PTI-428) / Proteostasis, posenacaftor (PTI-801) / Proteostasis
[VIRTUAL] FIRST RESULTS OF THE HIT-CF ORGANOID STUDY: EX VIVO FORSKOLIN-INDUCED SWELLING RESPONSE TO THE CFTR POTENTIATOR DIROCAFTOR, CORRECTOR POSENACAFTOR AND AMPLIFIER NESOLICAFTOR IN ORGANOIDS DERIVED FROM CF PATIENTS WITH ULTRA-RARE MUTATIONS () - Aug 26, 2020 - Abstract #NACFC2020NACFC_293;
Dirocaftor, posenacaftor and nesolicaftor represent novel CFTR modulators in clinical development. Based on their response in the organoid FIS assay, a subset of patients will be invited to participate in a clinical trial to confirm clinical efficacy of the investigational CFTR modulators and to validate this personalized medicine approach using the organoid model strategy.
- dirocaftor (PTI-808) / Proteostasis, nesolicaftor (PTI-428) / Proteostasis, posenacaftor (PTI-801) / Proteostasis
Evaluation of combinations of the CFTR potentiator dirocaftor, corrector posenacaftor and amplifier nesolicaftor in cystic fibrosis subjects with two copies of the F508del mutation () - Jun 29, 2020 - Abstract #ECFS2020ECFS_62;
In vitro, in human bronchial epithelial cells from F508del homozygous donors, the combination of DIR/ POS/NES increased CFTR chloride transport activity to levels comparable to that elicited by the elexacaftor/tezacaftor/ivacaftor combination... Dirocaftor, posenacaftor and nesolicaftor represent novel CFTR modulators in clinical development.
- dirocaftor (PTI-808) / Proteostasis, nesolicaftor (PTI-428) / Proteostasis, posenacaftor (PTI-801) / Proteostasis
Initial evaluation of the ex vivo response to the CFTR potentiator dirocaftor, corrector posenacaftor and amplifier nesolicaftor in organoids derived from cystic fibrosis subjects with ultra-rare mutations () - Jun 29, 2020 - Abstract #ECFS2020ECFS_59;
Dirocaftor, posenacaftor and nesolicaftor represent novel CFTR modulators in clinical development. 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.
- ||| dirocaftor (PTI-808) / Kineta
Trial completion: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Apr 22, 2020
P1/2, N=179, Completed, Sponsor: Proteostasis Therapeutics, Inc.
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. Active, not recruiting --> Completed
- || nesolicaftor (PTI-428) / Kineta
Trial completion: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO (clinicaltrials.gov) - Mar 26, 2020
P1, N=15, Completed, Sponsor: Proteostasis Therapeutics, Inc.
Active, not recruiting --> Completed Recruiting --> Completed
- || dirocaftor (PTI-808) / Kineta
Enrollment closed: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Jan 13, 2020
P1/2, N=180, Active, not recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Recruiting --> Completed Recruiting --> Active, not recruiting
- 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.
- Symdeko (tezacaftor/ivacaftor) / Vertex, PTI-428 / Proteostasis
INITIAL RESULTS EVALUATING THE FIRST-IN-CLASS CFTR AMPLIFIER, PTI-428, IN SUBJECTS WITH CF ON BACKGROUND TREATMENT WITH TEZACAFTOR/ IVACAFTOR () - Nov 11, 2019 - Abstract #NACFC2019NACFC_24;
Treatment with PTI-428 led to an increase in CFTR protein production of approximately 50% in subjects with CF on background treatment with tezacaftor/ivacaftor, similar to that seen with PTI-428 plus lumacaftor/ivacaftor. Subject selection may explain the differential impact of PTI-428 on ppFEV1 on backgrounds of lumacaftor/ivacaftor versus tezacaftor/ivacaftor, and thus provides an important example of possible selection bias in the current era of the availability of multiple CFTR modulators.
- 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.
- Symdeko (tezacaftor/ivacaftor) / Vertex, PTI-428 / Proteostasis
INITIAL RESULTS EVALUATING THE FIRST-IN-CLASS CFTR AMPLIFIER, PTI-428, IN SUBJECTS WITH CF ON BACKGROUND TREATMENT WITH TEZACAFTOR/ IVACAFTOR () - Nov 11, 2019 - Abstract #NACFC2019NACFC_7;
Treatment with PTI-428 led to an increase in CFTR protein production of approximately 50% in subjects with CF on background treatment with tezacaftor/ivacaftor, similar to that seen with PTI-428 plus lumacaftor/ivacaftor. Subject selection may explain the differential impact of PTI-428 on ppFEV1 on backgrounds of lumacaftor/ivacaftor versus tezacaftor/ivacaftor, and thus provides an important example of possible selection bias in the current era of the availability of multiple CFTR modulators.
- ||| dirocaftor (PTI-808) / Kineta
Phase classification, Enrollment change, Trial completion date, Trial primary completion date: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Jun 17, 2019
P1/2, N=180, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Subject selection may explain the differential impact of PTI-428 on ppFEV1 on backgrounds of lumacaftor/ivacaftor versus tezacaftor/ivacaftor, and thus provides an important example of possible selection bias in the current era of the availability of multiple CFTR modulators. Phase classification: P1 --> P1/2 | N=135 --> 180 | Trial completion date: Jun 2019 --> Jan 2020 | Trial primary completion date: Jun 2019 --> Jan 2020
- ||| PTI-428 / Proteostasis
Orphan drug: $PTI Receives Orphan Drug Designation in the EU for PTI-428 for the Treatment of Cystic Fibrosis (Twitter) - Jun 4, 2019
- || nesolicaftor (PTI-428) / Kineta
Trial completion: Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Apr 17, 2019
P2, N=40, Completed, Sponsor: Proteostasis Therapeutics, Inc.
Phase classification: P1 --> P1/2 | N=135 --> 180 | Trial completion date: Jun 2019 --> Jan 2020 | Trial primary completion date: Jun 2019 --> Jan 2020 Recruiting --> Completed
- | nesolicaftor (PTI-428) / Kineta
New P2 trial: Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Jul 18, 2018
P2, N=40, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
- ||| dirocaftor (PTI-808) / Kineta
Enrollment open: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Jul 3, 2018
P1, N=135, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Recruiting --> Completed Active, not recruiting --> Recruiting
- ||| nesolicaftor (PTI-428) / Kineta
Trial completion: Study to Assess the Safety, Tolerability, PK, ECG Effects, Food Effect, and Drug-drug Interaction (DDI) of Hormonal Contraceptives of PTI-428 in Healthy Female Volunteers (clinicaltrials.gov) - May 29, 2018
P1, N=94, Completed, Sponsor: Proteostasis Therapeutics, Inc.
Active, not recruiting --> Recruiting Recruiting --> Completed
- || dirocaftor (PTI-808) / Kineta
Enrollment closed: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Apr 25, 2018
P1, N=70, Active, not recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Recruiting --> Completed Recruiting --> Active, not recruiting
- nesolicaftor (PTI-428) / Kineta
Trial completion date, Trial primary completion date: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO (clinicaltrials.gov) - Apr 17, 2018
P1, N=16, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Recruiting --> Active, not recruiting Trial completion date: Nov 2017 --> Dec 2018 | Trial primary completion date: Oct 2017 --> Dec 2018
- || dirocaftor (PTI-808) / Kineta
Trial completion date, Trial primary completion date: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Apr 5, 2018
P1, N=66, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Trial completion date: Nov 2017 --> Dec 2018 | Trial primary completion date: Oct 2017 --> Dec 2018 Trial completion date: Nov 2017 --> Jun 2019 | Trial primary completion date: Nov 2017 --> Feb 2019
- |||| nesolicaftor (PTI-428) / Kineta
Trial completion, Enrollment change: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Jan 11, 2018
P1/2, N=56, Completed, Sponsor: Proteostasis Therapeutics, Inc.
Trial completion date: Nov 2017 --> Jun 2019 | Trial primary completion date: Nov 2017 --> Feb 2019 Recruiting --> Completed | N=136 --> 56
- | nesolicaftor (PTI-428) / Kineta
New P1 trial: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO (clinicaltrials.gov) - Aug 22, 2017
P1, N=16, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
- ||| dirocaftor (PTI-808) / Kineta
New P1 trial: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis (clinicaltrials.gov) - Aug 15, 2017
P1, N=60, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
- ||| nesolicaftor (PTI-428) / Kineta
Phase classification, Enrollment change, Trial initiation date, Trial primary completion date: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Apr 28, 2017
P1/2, N=136, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Recruiting --> Completed | N=136 --> 56 Phase classification: P1 --> P1/2 | N=36 --> 136 | Initiation date: Mar 2016 --> Jul 2016 | Trial primary completion date: Dec 2016 --> Dec 2017
- nesolicaftor (PTI-428) / Kineta
New P1 trial: Study to Assess the Safety, Tolerability, PK, ECG Effects, Food Effect, and Drug-drug Interaction (DDI) of Hormonal Contraceptives of PTI-428 in Healthy Female Volunteers (clinicaltrials.gov) - Jul 27, 2016
P1, N=88, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
- nesolicaftor (PTI-428) / Kineta
Enrollment open: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Apr 22, 2016
P1, N=36, Recruiting, Sponsor: Proteostasis Therapeutics, Inc.
Phase classification: P1 --> P1/2 | N=36 --> 136 | Initiation date: Mar 2016 --> Jul 2016 | Trial primary completion date: Dec 2016 --> Dec 2017 Not yet recruiting --> Recruiting
- nesolicaftor (PTI-428) / Kineta
New P1 trial: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Mar 24, 2016
P1, N=36, Not yet recruiting, Sponsor: Proteostasis Therapeutics, Inc.