EFFECT OF FOUR SETS OF DISTINCT MODULATORS ON NON-F508DEL MUTATIONS THAT CAUSE CYSTIC FIBROSIS
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1 EFFECT OF FOUR SETS OF DISTINCT MODULATORS ON NON-F508DEL MUTATIONS THAT CAUSE CYSTIC FIBROSIS BHATT, PRIYANKA; BAILEY, VIOLAINE; DASGUPTA, AFIA; CHIN, JUSTIN; AN, WEILING; BRESILLA, CASTERA; KWOK, IRIS; COLE, BRIDGET MCCARTHY; FITZPATRICK, RICHARD; KROUSE, MAURI E I NSTITUTIONS (ALL) : FLATLEY DISCOVERY LAB, CHARLESTOWN, MA, UNITED STATES POSTER# CATEGORY: CFTR
2 BACKGROUND All this work was done in FRT cells stably expressing different CFTR mutations The cells were obtained from the Cystic Fibrosis Transmembrane Conductance Regulator Expression Core at the University of Alabama Birmingham and Directed by Dr. Jeong Hong The advantage of these cells is that all CFTR mutations (except stop mutations) express approximately the same amount of messenger RNA
3 BACKGROUND We have developed CFTR modulators categorized in distinct groups that work synergistically or additively on primary F508del CF Bronchial human epithelia MODULATOR SETS PS : CS: CS: CS3: Potentiator Set (VX-770, FD0339, FDL76) (increases channel open probability) Early Corrector Set (VX-809, VX-66, FDL30) (corrects NBD half of F508del CFTR) CFTR Stabilizer (FD0560) (improves CFTR stability in membrane) CFTR Amplifier (FD880, FDL38) (increases band B of CFTR) NOTE: The compounds within each set do not add together and all reach the same maximum efficacy
4 OBJECTIVE OF THE STUDY TO DETERMINE WHETHER ANY INDIVIDUAL COMPOUND OR COMBINATIONS WOULD HAVE AN EFFECT ON NON-F508DEL CFTR MUTATION
5 EXPERIMENTAL APPROACH ) To test all modulator sets at single concentration (Cmax in primary CFhBE cells) a) Report maximum response from each modulator set b) Single and combinations (data shown on log scale from 0.5 to 6 ms/cm ) ) Generate Dose Response Curves for all compounds in each modulator set to determine efficacy and potency a) Report maximum response from each modulator set b) Singles only (data shown on log scale from 0.5 to 6 ms/cm )
6 EFFECT OF MODULATORS ON F508DEL (CLASS II) MUTATION G (ms/cm ) Uncorrected CS CS CS3 CS + CS + CS3 Forskolin + VX-770 CFTR inh -7 3 G max (ms/cm ) Combination therapy improves response fold over uncorrected TIME (S) n= CORRECTORS n=
7 EFFECT OF MODULATORS ON R560T (CLASS II) MUTATION G max (ms/cm ) No significant effect of any corrector combination Stimulation: Forskolin Potentiation: VX-770 n= CORRECTORS
8 EFFECT OF MODULATORS ON G85E.V70 (CLASS II) MUTATION G max (ms/cm ) Effect of CFTR Stabilizer 5.3 fold over uncorrected Stimulation: Forskolin Potentiation: VX-770 n= CORRECTORS
9 EFFECT OF MODULATORS ON E9K (CLASS II) MUTATION G max (ms/cm ) Fold increase over uncorrected Stimulation: Forskolin Potentiation: VX-770 n= CORRECTORS
10 EFFECT OF MODULATORS ON N303K (CLASS II) MUTATION Stimulation: Forskolin Potentiation: VX-770 n= G (ms/cm ) Triple combination gives 6 fold increase over uncorrected CORRECTORS
11 EFFECT OF MODULATORS ON CLASS II MUTATIONS: SUMMARY. Do all class mutations tested respond to the same corrector set? NO Each different CF causing mutation appears to have it s own unique pattern of response to correctors and corrector combinations.. Following modulator /combination work in our hands F508del - Combination of Early Corrector and Stabilizer G85E.V70 - CFTR Stabilizer N303K - Triple Combination E9K - Early Corrector R560T - No tested corrector worked 3. Modulator sets giving more than fold positive response show potential and further compound optimization may be necessary within each mutation background
12 EFFECT OF MODULATORS ON R7H (CLASS IV) MUTATION fold increase over uncorrected Stimulation: Forskolin Potentiation: VX-770 n = G max (ms/cm ) CORRECTORS
13 EFFECT OF MODULATORS ON R7H (CLASS IV) MUTATION R7H can be corrected using an early corrector as recently reported by Gentzsch et al.
14 EFFECT OF MODULATORS ON G55D (CLASS III) MUTATION G max (ms/cm ) fold increase over uncorrected Stimulation: Forskolin Potentiation: VX-770 n = CORRECTORS
15 EFFECT OF MODULATORS ON WT CFTR G max (ms/cm ) Combination give fold increase in response over uncorrected CORRECTORS Stimulation: Forskolin Potentiation: VX-770 n =
16 EFFECT OF MODULATORS ON G55D MUTATION G55D - Response can be further improved with a combination of Early Corrector and Stabilizer R7H - Response can be further improved with Early Corrector WT - WT expression be further increased by Combination treatment of Early Corrector and CFTR amplifier
17 INTER MUTATION COMPARISON OF MODULATOR SETS 3 6 Untreated PS CS CS CS3 Untreated WT G max (ms/cm ) Kalydeco in G55D Orkambi in F508del N303K F508del G85E E9K R560T R7H G55D WT Class II Mutation Class IV Class III
18 USSING CHAMBERS RAW TRACES: PS RESPONSE FORSKOLIN 00 PS (3 DOSE) CFTRinh-7 Isc (µa/cm ) All cells corrected with CS 5/ Chloride gradient WT R7H G55D F508del G85E TIME (S)
19 USSING CHAMBERS RAW TRACES: PS RESPONSE Fold Increase over FSK 6 8 Low Responders Medium Responders High Responders PS includes VX-770, FD0339, FDL76
20 ACKNOWLEDGEMENTS We thank the Flatley Foundation for generous financial support All staff members EPY group Violaine Bailey Justin Chin Castera Bresilla Weiling An Dr. Jeong Hong Lab University of Alabama Birmingham
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