Cystic Fibrosis (CF) Drug Development Milestones and Newsflow
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Past data announcements
- $PTI provides top-line phase 1 data for PTI-428 in healthy volunteers [8/2016 PR]
- $VRTX phase 3 trial of VX-661 / IVA fails futility analysis in F508del / minimal function population[8/2016 PR]
- $GLPG C1 corrector '2222 phase 1 is done with "favorable data" [9/2016 slide deck, linked below]
Corporate slide decks
3Q-2016
- $VRTX complete enrollment in VX-661 phase 3 trial in F508del / residual function patients in September [8/2016 PR]
- $CRBP complete enrollment in phase 2 resunab study in all-comer CF patients [6/2016 Slide Deck]
October 27-29, 2016: North American Cystic Fibrosis Conference #NACFC
- $PRQR QR-010 data from Nasal Potential Difference (NPD) study in 8 heterozygous and 8 homozygous F508del CF patients [PR. Reiterated 8/2016]
- $PRQR QR-010 data for phase 1b single ascending dose (SAD) cohorts in homozygous F508del CF patients [PR.Reiterated 8/2016]
- $PRQR update on status / data for QR-010 phase 1b multiple ascending dose (MAD) cohorts in homozygous F508del CF patients [PR]
- $GLPG $ABBV present phase 1 SAD and MAD data in healthy volunteers with corrector '2222 [6/2016 R&D Day]
- $GLPG $ABBV present phase 2 potentiator data
- $PTI present phase 1 data for PTI-428
4Q-2016
- $VRTX complete enrollment in VX-661 + ivacaftor phase 3 trial in F508del / residual function mutation CF patients [PR, delayed from prior mid-2016 guidance at #NACFC15 and size decreased from N=300 to 200, reiterated 7/2016]
- $VRTX phase 1 healthy volunteer data for VX-152 and VX-440, then start phase 2 triple combination study (ivacaftor + VX-661 + corrector TBD) [4/2016 earnings and 7/2016 earnings says "2h17"]
- $GLPG $ABBV phase 2 data in G551D CF patients for potentiator '1837 [6/2016 R&D Day, reiterated 9/2016 slide deck]
- $GLPG $ABBV start phase 1 healthy volunteer trials for backup C1 corrector '2851 and lead C2 corrector '2737 [6/2016 R&D Day, timing reiterated 7/2016 CC. Reiterated 9/2016 slide deck]
- $GLPG $ABBV start phase 1 healthy volunteer trial for potentiator TBD + corrector '2222 [6/2016 R&D Day]
- $PTI start 14-day phase 2 study of '428 +ORKAMBI [5/2016 10-Q. Reiterated 8/2016 slide deck, link above]
- $PTI file IND for PTI-801 and PTI-808 for phase 1 development [8/2016 PR "by year-end"]
- $PTI 7-day FEV1 data for PTI-428 [8/2016 slide deck says "around year-end"]
- $NVLS phase 2 data from N91115 (cavosonstat) + ORKAMBI study in homozygous F508del CF patients [7/2016 PR: enrollment complete, data by year-end 2016. Reiterated 8/2016]
- $CNCE open IND and initiate phase 2 study of CTP-656 (deuterated ivacaftor) [6/2016 PR. Reiterated 8/2016]
- $PLX phase 2 data for inhaled AIR DNase study in CF patients [7/2016 PR: enrollment complete in 2h2016, data around year-end 2016]
1Q-2017
- $VRTX complete enrollment in VX-661 + ivacaftor phase 3 trial in F508del / gating mutation CF patients [4/2016 PR says late 2016 or early 2017. Reiterated 8/2016. Delayed from prior mid-2016 guidance at #NACFC15]
- $GLPG $ABBV phase 1 healthy volunteer data for backup potentiator '2451 [7/2016 CC. Delayed from prior "4q16" guidance at 6/2016 R&D Day. 9/2016 slide deck says "1h17"]
- $GLPG $ABBV start phase 1 healthy volunteer triple combination trial (components TBD) [6/2016 R&D Day]
- $PTCT phase 3 TRANSLARNA (ataluren) ACT study data [PR]
- $CRBP phase 2 data from resumab study in all-comers CF patients [8/2016 10-Q] Delayed from prior guidance 4q16 [6/2016 Slide Deck]
- $PTI start dosing in 3-month phase 2 study of PTI-428 + ORKAMBI [8/2016 slide deck, linked above]
- $PTI start phase 1 PTI-BC-733 triple combo healthy volunteer study [8/2016 slide deck]
2Q-2017
- $PTI phase 2 POC data from PTI-428 + ORKAMBI trial [5/2016 10-Q and Baird/RBC reports. Delayed from prior guidance of 4q-2016 in 2/2016 IPO roadshow]
- $PTI phase 1 data for PTI-801 and PTI-808 [8/2016 slide deck link above]
- $PTI report top-line data PTI-NC-733 phase 1 triple combination study (potentiator, corrector, amplifier) in 1h-2017 [RBC initiation report. Reiterated 8/2016 slide deck]
- $NVLS phase 2 data for N91115 (cavosonstat) + KALYDECO study in F508del / gating mutation CF patients [Initiated 5/2016. Reiterated 8/2016]
- $VRTX European regulatory application for ORKAMBI age 6-11 years population [5/2016 10-Q, reiterated 7/2016]
- $VRTX top-line data phase 3 VX-661 + ivacaftor in homozygous F508del CFTR [7/2016 earnings and 8/2016 PR says 1h17. Delayed from prior guidance of 1q17 in 4/2016]
- $VRTX top-line data from phase 3 VX-661 + ivacaftor in F508del / residual function [8/2016 PR]
3Q-2017
- $GLPG $ABBV start triple combination phase 2 trial in CF patients [6/2016 R&D Day. "mid-2017" reiterated 9/2016 slide deck, linked above]
- $PTI start POC / dose-ranging studies of PTI-801 and PTI-808 [8/2016 slide deck, linked above]
4Q-2017
- $CNCE phase 2 data for CTP-656 [6/2016 PR says "2h 2017". Reiterated 8/2016]
- $VRTX planned NDA filing for VX-661 + ivacaftor [7/2016 earnings. Reiterated 8/2016]
- $PTI start phase 3 with PTI-428 [8/2016 slide deck link above]
- $PTI start POC study with triple combo PTI-NC-733 [8/2016 slide deck]
1H-2018
- PTI-NC-733 proof of concept data and phase 3 initiation [8/2016 slide deck, link above]
2H-2018
- $PTI PTI-428 phase 3 data [8/2016 slide deck, link above]
No guidance yet on the following data readouts
- $VRTX ORKAMBI clinical trial in homozygous F508del patients 2-5 years old is ongoing.
- $VRTX CRISPR and Moderna collaborations "three years or more" from being in the clinic [7/2016 CC]
- $PTCT European decision on TRANSLARNA (ataluren) MAA [8/2016 earnings PR revokes prior guidance of mid-2016 decision, ACT study data now likely needed for approval]