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Report Summary Indigo Biosciences Study Report 110407-1 3M ToxDocs 11-108 Prepared by: John L. Butenhoff, Ph.D., D.A.B.T., C.I.H. Sue Chang, Ph.D. July, 2011 BACKGROUND: Various toxicological studies in rodents have shown that perfluoroalkyl acids (PFAs) produce responses that are consistent with activation of the xenosensor nuclear receptors peroxisome proliferator activated receptor (PPAR), constitutive androstane receptor (CAR), and pregnane X receptor (PXR) (Bjork and Wallace 2009; Curran et al. 2008; Martin et al. 2007; Pieterman et al. 2007; Ren et al. 2009; Rosen et al. 2010; Rosen et al. 2009; Shipley et al. 2004; Takacs and Abbott 2007; Vanden Heuvel et al. 2006; Wolf et al. 2008). The activations of these xenosensor nuclear receptors can lead to hepatocellular hyperplastic responses in rodents. Even though human hepatocytes contain active forms of PPAR and CAR/PXR receptors, there are significant differences exist in pharmacokinetic / pharmacodynamic profiles between the human and rodent forms. To date, a number of studies have shown that the hyperplastic component of the response to inducers of xenosensor nuclear receptors observed in rodents appears unlikely to be a component of the human hepatic response (Ross et al. 2010). In the evaluation of PFAs of various chain lengths, Wolf et al. (2008) demonstrated that activation of mouse PPAR was generally higher compared to that of human PPAR using transfected COS-1 cells. Bjork and Wallace (2009) also reported that primary rat hepatocytes were more effective in eliciting PPARrelated responses than primary human hepatocytes. Furthermore, with the 8-carbon perfluoroalkyls perfluorooctanesulfonate(PFOS) and perfluorooctanoate (PFOA), Bjork et al. (2011) showed that while multiple xenosensor nuclear receptors (i.e., PPAR, CAR, PXR) participate in the metabolic response to PFOA and PFOS exposures in both human and rat hepatocytes, however, the changes in primary human hepatocytes were more subtle and possibly reflect an adaptive metabolic response rather than an overt metabolic regulation observed in rodents. OBJECTIVE: The objective of the work reported herein was to investigate the effects of perfluorobutyrate (PFBA), PFOA, perfluorobutanesulfonate (PFBS), pherfluorohexanesulfonate (PFHxS), and PFOS on the activation of three human xenosensor nuclear receptors (thyroid receptors alpha (TR), PXR, and CAR3) using HEK-293T transactivated cells. MATERIALS AND METHODS: The test was performed by Indigo Biosciences. The compounds and concentrations evaluated are: Compound PFBA, potassium salt PFOA, ammonium salt PFBS, potassium salt PFHxS, potassium salt PFOS, potassium salt PFOS, potassium salt Lot # NB # 119868-94 (FC-143) Lot # 332 (L-7038) Lot # 2 (L-9051) NB # 118993-109 (FC-95) Lot # 217 (FC-95) TCR 00017-46 Concentrations Tested 0.41 - 100 M 0.41 - 100 M 0.41 - 100 M 0.41 - 100 M 0.41 - 100 M 0.41 - 100 M RESULTS: The full report from Indigo Biosciences is attached as Appendix I of this report. The table below summarizes the study results. Xenosensor Nuclear Receptor Compound Dose Range (M) Highest Peak Response EC50 (M) p-value T3a (+ control) 0 - 0.01 80.43 0.002123 -- PFOS 0.41 - 100 1.36 0.9528 0.0087 PFOS (lot 217) 0.41 - 100 1.475 7.466 0.0014 TR PFHxS 0.41 - 100 1.623 17.46 0.0015 PFBS 0.41 - 100 1.224 2.534 0.0035 PFOA 0.41 - 100 1.542 NAb 0.1817 PFBA 0.41 - 100 0.978 1.758 0.3298 TO-9013173c (+ control) 0 - 1 8.343 0.03377 -- PFOS 0.41 - 100 1.706 8.172 0.0032 PFOS (lot 217) 0.41 - 100 2.045 122.2 0.0018 PXR PFHxS 0.41 - 100 2.81 47.25 0.0005 PFBS 0.41 - 100 1.693 18.28 0.0008 PFOA 0.41 - 100 1.07 NA 0.0765 PFBA 0.41 - 100 0.966 NA 0.0314 CITCOd (+ control) 0 - 5 37.042 0.6444 -- PFOS 0.41 - 100 2.025 38.11 0.0001 PFOS (lot 217) 0.41 - 100 2.547 525.9 0.0021 CAR3 PFHxS 0.41 - 100 1.963 62.41 0.0516 PFBS 0.41 - 100 1.13 3.247 0.0525 PFOA 0.41 - 100 1.723 NA 0.0275 PFBA 0.41 - 100 1.15 3.885 0.128 a T3, triiodothyronine, agonist for TR. b NA, not available, EC50 cannot be derived because data did not fit the curve. c TO-9013173, agonist for PXR. d CITCO, 6-(4-Chlorophenyl)imidazo[2,1-b][1,3]thiazole-5-carbaldehydeO-(3,4-dichlorobenzyl)oxime, agonist for CAR3. Human thyriod receptor R: There were statistical associations suggesting that PFBS, PFHxS, and PFOS were capable of eliciting human R activation in the assay. However, unlike the positive control triiodothyronine (T3), there were no concentration-dependent responses with any of the PFAs evaluated in the activation of human TR and the EC50's were at least several orders of magnitude higher than T3. Therefore, PFAs does not have any effect on R activation. Human pregnane X receptor (PXR): Within the limit of study design, PFOS (lot 217) and PFHxS at 33 M or higher appeared to elicit some human PXR responses, although the EC50's derived were several orders of magnitude higher than the positive control. PFBS did not have any effect on PXR activation. Even though the data obtained from PFOA and PFBA did not fit the statistical curves and no reliable EC50 could be derived, given the fact that the highest responses obtained with either PFOA or PFBA were at least an order of magnitude lower than the highest response obtained from positive control, it is unlikely that PFOA or PFBA is capable of activating human PXR. Human Constitutive androstane receptors 3 (CAR3): PFOS (both lot 217 and TCR 00017-46) at 100 M (the highest concentration tested) were capable of eliciting human CAR3 responses. Similar to PXR, the EC50's derived were several orders of magnitude higher than the positive control. PFBS and PFBA did not have any effect on human CAR3 activation. Due to the lack of concentration-dependent response, the data obtained from PFOA did not fit the statistical curves and no reliable EC50 could be derived, given the fact that the highest response obtained were much lower than the highest response obtained from positive control, it is unlikely that PFOA activates human CAR3. CONCLUSION: PFAs do not appear to activate human TR. Consistent with other study data reported by others, PFOS appears to be capable of eliciting human PXR and CAR3 responses in vitro. However, the degree of activation was several folds lower than the activation produced by TO-9013173 and CITCO (the respective controls for PXR and CAR3), and the concentrations of PFOS employed in the assay were several folds higher than the blood serum PFOS concentrations reported for general human population. PFHxS also appears to be capable of eliciting human PXR responses as well, and similar to PFOS, the extent of activation was much lower than the respective control and the concentrations of PFHxS that produced effects were much higher than the general human population. PFBS, PFOA, and PFBA did not activate human PXR and CAR3. REFERENCES: Bjork, J.A., Wallace, K.B., 2009. Structure-activity relationships and human relevance for perfluoroalkyl acid-induced transcriptional activation of peroxisome proliferation in liver cell cultures. Toxicol Sci 111, 89-99. Curran, I.,Hierlihy, S.L.,Liston, V.,Pantazopoulos, P.,Nunnikhoven, A.,Tittlemier, S.,Barker, M.,Trick, K., Bondy, G., 2008. Altered Fatty Acid Homeostasis and Related Toxicologic Sequelae in Rats Exposed to Dietary Potassium Perfluorooctanesulfonate (PFOS). J Toxicol Environ Health A 71, 1526-1541. Martin, M.T.,Brennan, R.J.,Hu, W.,Ayanoglu, E.,Lau, C.,Ren, H.,Wood, C.R.,Corton, J.C.,Kavlock, R.J., Dix, D.J., 2007. Toxicogenomic study of triazole fungicides and perfluoroalkyl acids in rat livers predicts toxicity and categorizes chemicals based on mechanisms of toxicity. Toxicol Sci 97, 595-613. Pieterman, E.,van den Hoek, A.M.,Havekes, L.M.,Ehresman, D.,Chang, S.,Butenhoff, J.L.,Princen, H.M., Cohen, L.H., 2007. Perfluorohexanesulfonate and perfluorooctanesulfonate decrease plasma cholesterol and triglycerides in APOE*3Leiden transgenic mice. Indication for a PPAR agonist mechanism (abstract). Atherosclerosis Suppl 8, 41. Ren, H.,Vallanat, B.,Nelson, D.M.,Yeung, L.W.Y.,Guruge, K.S.,Lam, P.K.S.,LehmanMcKeeman, L.D., Corton, J.C., 2009. Evidence for the Involvement of Xenobioticresponsive Nuclear Receptors in Transcriptional Effects Upon Perfluoroalkyl Acid Exposure in Diverse Species. Reproductive Toxicology 27, 266-277. Rosen, M.B.,Schmid, J.E.,Das, K.P.,Wood, C.R.,Zehr, R.D., Lau, C., 2009. Gene Expression Profiling in the Liver and Lung of Perfluorooctane Sulfonate-Exposed Mouse Fetuses: Comparison to Changes Induced by Exposure to Perfluorooctanoic Acid. Reproductive Toxicology 27, 278-288. Rosen, M.B.,Schmid, J.E.,Corton, J.C.,Zehr, R.D.,Das, K.P., Lau, C., 2010. Gene Expression Profiling in Wild-Type and PPAR-Null Mice Exposed to Perfluorooctane Sulfonate Reveals PPAR-Independent Effects. PPAR Research 2010, 1-23. Ross, J.,Plummer, S.M.,Rode, A.,Scheer, N.,Bower, C.C.,Vogel, O.,Henderson, C.J.,Wolf, C.R., Elcombe, C.R., 2010. Human constitutive Androstane Receptor (CAR) and Pregnane X Receptor (PXR) Support the Hypertrophic but not the Hyperplastic Response to the Murine Nongentoxic Hepatocarcinogens Phenobarbital and Chlordane In Vivo. Toxicol Sci 116, 452466. Shipley, J.M.,Hurst, C.H.,Tanaka, S.S.,DeRoos, F.L.,Butenhoff, J.L.,Seacat, A.M., Waxman, D.J., 2004. trans-activation of PPARalpha and induction of PPARalpha target genes by perfluorooctane-based chemicals. Toxicol Sci 80, 151-160. Takacs, M.L., Abbott, B.D., 2007. Activation of mouse and human peroxisome proliferatoractivated receptors (alpha, beta/delta, gamma) by perfluorooctanoic acid and perfluorooctane sulfonate. Toxicol Sci 95, 108-117. Vanden Heuvel, J.P.,Thompson, J.T.,Frame, S.R., Gillies, P.J., 2006. Differential Activation of Nuclear Receptors by Perfluorinated Fatty Acid Analogs and Natural Fatty Acids: A Comparison of Human, Mouse, and Rat Peroxisome Proliferator-Activated Receptor-and -Liver X Receptor-, and Retinoid X Receptor-. Toxicological Sciences 92, 476-489. Wolf, C.J.,Takacs, M.L.,Schmid, J.E.,Lau, C., Abbott, B.D., 2008. Activation of mouse and human peroxisome proliferator-activated receptor alpha by perfluoroalkyl acids of different functional groups and chain lengths. Toxicol Sci 106, 162-171. APPENDIX I: Study Report from Indigo Biosciences Indigo Biosciences, Inc Report 110407-1 3M Summary April 7, 2011 SUMMARY Effects of test compounds on nuclear receptor activation: TRalpha: PXR: CAR3: No effects PFOS217 at 100 M; PFHxS at 33 and 100 M PFOS at 100 M; PFOS217 at 100 M EXPERIMENTAL PROCEDURE Plasmids: Two different assays are commonly used for services, a chimeric assay and a full- length (see Figure 1). Chimeric System: The ligand-binding domain of the nuclear receptors were fused to the DNA-binding domain of the yeast transcription factor Gal4 under the control of the SV40 promoter. A reporter plasmid encodes the firefly luciferase gene under the control of the Gal4 DNA response element (UAS). A transfection efficiency control vector is included in most assays (pRL-luciferase, Promega, Madison, WI). All plasmids were verified by sequencing and through examination of positive controls (Tien et al., 2006; Vanden Heuvel et al., 2006). Full Length System: The full-length cDNA of the nuclear receptor is under the control of the SV40 promoter. A reporter plasmid encodes the luciferase reporter under the control of the MMTV response element. All plasmids were verified by sequencing and through examination of positive controls. Figure 1. Design of Reporter Assays Performed by INDIGO Biosciences, Inc. Cell culture and transactivation assays: HEK 293-T fibroblasts (ATCC, Manassas, VA) were cultured in high-glucose Dulbecco's Minimal Essential Medium (DMEM) supplemented with 10% fetal bovine serum (FBS, Sigma), 0.2 mg/ml streptomycin and 200 U/ml penicillin (Gibco, Grand Island, NY). For transient transfection reporter assays, HEK 293-T ells were transfected with plasmid DNA using Lipofectamine reagent (Invitrogen, Carlsbad, CA) and following the manufacturer's recommended procedures, using HEK 293-T cells at approximately 80% confluence in 10 cm culture dishes. After 6 h, the DNA- Lipofectamine complex was removed. Following overnight culture, the media was replaced 4 h after replating with DMEM (10% FBS) containing test compounds in DMSO (0.1% final concentration). Concentrations of the chemicals are given in the figure legends. Sixteen hours after treatment, the cells were lysed with passive lysis buffer (Promega, Madison, WI) for 30 min; luciferase activity was measured using the Luciferase dual reporter assay kit (Promega, Madison, WI) and a Tecan GeniosPro (Research Triangle Park, NC) and manufacturer's recommended procedures. The fold induction of normalized luciferase activity (luciferase/ renilla) was calculated relative to vehicle-treated cells, and represents the mean of three independent samples per treatment group. Statistical Analysis: Significance of dose-dependent alterations in relative luciferase activity (p<0.05) was determined using ANOVA (p<0.05, JMP 7.0, SAS Institute, Cary,NC). Dunnett's multi-comparison t-test was used to determine doses that resulted in significant differences relative to vehicle control treated cells (JMP 7.0). Non-linear regression was performed using Prism 5.0 (Graphpad Software, La Jola, CA). References: Tien, E.S., Hannon, D.B., Thompson, J.T., and Vanden Heuvel, J.P. (2006). Examination of Ligand-Dependent Coactivator Recruitment by Peroxisome Proliferator-Activated Receptoralpha (PPARalpha). PPAR Res 2006, 69612. Vanden Heuvel, J.P., Thompson, J.T., Frame, S.R., and Gillies, P.J. (2006). Differential activation of nuclear receptors by perfluorinated fatty acid analogs and natural fatty acids: a comparison of human, mouse, and rat peroxisome proliferator-activated receptor-alpha, -beta, and -gamma, liver X receptor-beta, and retinoid X receptor-alpha. Toxicol Sci 92, 476-489. Results: (A) Human TRalpha Activity Assay RAW DATA Human TRalpha Activity Assay SUMMARIZED DATA Group Compound 1 DMSO 2 T3 3 T3 4 T3 5 T3 6 T3 7 T3 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS Dose1 0 3.2 16 80 400 2000 10000 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 Luc12 0.7829 1.3911 1.6808 2.8709 13.4186 49.8262 97.6308 1.5336 1.6208 1.7300 1.4320 2.0059 1.3598 1.4301 1.4540 1.3263 1.4341 1.1855 2.0159 1.7592 1.0655 1.3552 1.2197 Luc2 1.2260 1.4683 1.7300 2.4996 13.5485 57.6902 88.6182 1.1860 1.3368 1.6780 1.8906 1.7053 1.5801 1.4621 1.4147 1.3898 1.6616 1.6524 2.1002 1.8390 1.2300 1.1638 1.1961 Luc3 1.0826 1.2120 1.6432 2.3047 14.5054 62.8881 87.9161 1.2438 1.2753 1.6351 1.7369 1.8423 1.3423 1.1920 1.3160 1.5206 1.6981 1.6048 1.9061 1.5170 0.9756 1.2255 0.9992 Group Compound 1 DMSO 2 T3 3 T3 4 T3 5 T3 6 T3 7 T3 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS Dose Mean3 Std Err CV 0 1.0000 0.0398 3.2 1.1945 0.0668 16 1.4827 0.0221 80 2.2518 0.1462 400 12.1671 0.3016 2000 49.9929 3.3416 10000 80.4340 2.7529 0.41 0.9704 0.0790 1.23 1.0364 0.0782 3.7 1.2348 0.0202 11.1 1.2388 0.0990 33.3 1.3598 0.0638 100 1.0485 0.0562 0 1.0000 0.0626 0.41 1.0246 0.0302 1.23 1.0373 0.0420 3.7 1.1738 0.0607 11.1 1.0878 0.1090 33.3 1.4745 0.0413 100 1.2524 0.0711 0 1.0000 0.0683 0.41 1.1447 0.0517 1.23 1.0440 0.0641 6.8851 9.6853 2.5819 11.2433 4.2934 11.5774 5.9280 14.0966 13.0617 2.8275 13.8422 8.1285 9.2833 10.8415 5.0976 7.0181 8.9535 17.3490 4.8480 9.8333 11.8314 7.8284 10.6380 Stats4 0.0087 0.0014 0.0015 p-value5 0.9993 0.9978 0.1273 0.1186 0.012 0.9904 1 0.9996 0.9963 0.2795 0.837 0.0007 0.0641 1 0.7995 0.9991 Renilla6 0.90724986 1.00495683 0.92482502 0.78996843 0.5186502 0.61092297 1 1.00549124 1.06633562 0.94738703 0.82781468 0.65097695 0.63612893 1 0.98896434 0.99012433 24 PFHxS 3.7 1.02835 1.223711 1.431821 24 PFHxS 3.7 1.12618328 0.10683624 16.4312322 0.8723 0.99138284 25 PFHxS 11.1 1.392872 1.459168 1.302646 25 PFHxS 11.1 1.27011069 0.04160013 5.67301272 0.2744 1.02595598 26 PFHxS 33.3 1.623792 1.799535 1.884285 26 PFHxS 33.3 1.6225666 0.07035373 7.51009139 0.0028 0.79998882 27 PFHxS 100 1.75969 1.39303 2.142978 27 PFHxS 100 1.61892442 0.1985641 21.243926 0.0029 0.54180085 28 PFBS 0 1.035781 1.261699 1.234255 28 PFBS 0 1 0.06045835 10.4716941 0.0035 1 1 29 PFBS 0.41 1.004704 1.079429 1.217323 29 PFBS 0.41 0.9347972 0.05289827 9.80132274 0.9307 0.8384736 30 PFBS 1.23 0.7317497 1.077492 1.036778 30 PFBS 1.23 0.8058418 0.0926712 19.9184531 0.1556 0.66056222 31 PFBS 3.7 1.280438 1.577579 1.464072 31 PFBS 3.7 1.22378633 0.07353596 10.4077002 0.0849 0.83033015 32 PFBS 11.1 1.188956 1.45895 1.279778 32 PFBS 11.1 1.11211175 0.06737708 10.4935965 0.6166 0.92584538 33 PFBS 33.3 1.347046 1.306689 1.344105 33 PFBS 33.3 1.13197621 0.01103419 1.68835449 0.4681 0.85057157 34 PFBS 100 1.301196 1.379461 1.349161 34 PFBS 100 1.14103068 0.01935382 2.93785192 0.4068 0.69897459 35 PFOA 0 1.208199 1.082189 1.120016 35 PFOA 0 1 0.03283914 5.68790617 0.1817 1 1 36 PFOA 0.41 1.162493 1.788925 1.337093 36 PFOA 0.41 1.25747888 0.16418436 22.6147452 0.5882 1.11237482 37 PFOA 1.23 1.45962 1.604089 1.301355 37 PFOA 1.23 1.27992578 0.07690176 10.4066779 0.5121 1.24511386 38 PFOA 3.7 1.917233 1.412162 1.929093 38 PFOA 3.7 1.54189592 0.14986618 16.8348481 0.0555 1.1221669 39 PFOA 11.1 1.41738 1.93721 1.387912 39 PFOA 11.1 1.3905983 0.15692358 19.5455164 0.2224 1.15636803 40 PFOA 33.3 1.250512 1.199197 1.943313 40 PFOA 33.3 1.28812364 0.21106931 28.3810304 0.4853 0.98125502 41 PFOA 100 1.214905 1.29889 1.253875 41 PFOA 100 1.10475768 0.02134525 3.34653057 0.9835 0.67508295 42 PFBA 0 1.498251 1.936381 1.693365 42 PFBA 0 1 0.07413945 12.84133 0.3298 1 1 43 PFBA 0.41 1.694401 1.747789 1.292758 43 PFBA 0.41 0.92335233 0.08401431 15.7596455 0.8183 1.01709165 44 PFBA 1.23 1.639416 1.554529 1.692198 44 PFBA 1.23 0.95283656 0.02345958 4.26444437 0.9727 1.14804843 45 PFBA 3.7 1.778415 1.648278 1.589801 45 PFBA 3.7 0.97825603 0.03261079 5.77390251 0.9995 1.02335903 46 PFBA 11.1 1.413671 1.450736 1.361416 46 PFBA 11.1 0.82406893 0.01515706 3.18575257 0.1511 1.00881868 47 PFBA 33.3 1.434516 1.443324 1.613287 47 PFBA 33.3 0.87580531 0.03403547 6.73108011 0.4261 1.12207964 48 PFBA 100 1.662825 1.405156 1.822196 48 PFBA 100 0.95362322 0.07107962 12.9100795 0.9747 1.094378 1 Dose in pM for T3 and uM for client's compounds 3 Expressed relative to DMSO control 6 Relative Renilla activity (toxicity marker) 2 Expressed as (Luciferase RLU/Renilla RLU) 4 Oneway Anova, Dose x Relative Luciferase, Prob > F Relative renilla below 50% (0.5) indicates overt toxicit 5 Dunnett's t-test, DMSO control versus each sample Curve Fitting Data T3 PFOS PFOS217 PFHxS PFBS PFOA PFBA Bottom -0.07873 0.9065 0.9974 1.028 0.9063 Ambiguous 0.9826 Top 98.35 1.233 1.366 1.779 1.167 0.894 LogEC50 3.327 -0.02099 0.8731 1.242 0.4038 0.245 EC50 2123 0.9528 7.466 17.46 2.534 1.758 Span 98.43 0.327 0.369 0.7513 0.261 -0.08855 Statistical Analysis P-value Peak4 LSSD5 4 Highest observable 5 Lowest statistically NA, not applicable PFOS PFOS217 PFHxS 0.0087 0.0014 0.0015 1.360 1.475 1.6225666 33.3 33.3 33.3 response (relative to DMSO control) significant dose (p<0.05, Dunnett's) PFBS 0.0035 1.2237863 N/A PFOA 0.1817 1.5418959 N/A PFBA 0.3298 0.8240689 N/A Relative Luciferase Activity Relative Luciferase Activity 100 T3 80 60 40 20 0 0 1 2 3 4 5 Log(Dose) pM 3 PFOS PFOS217 2 PFHxS PFBS PFOA 1 PFBA 0 -1 0 1 2 Log(Dose) M (B) Human PXR Activity Assay RAW DATA Group Compound 1 DMSO 2 TO-9013173 3 TO-9013173 4 TO-9013173 5 TO-9013173 6 TO-9013173 7 TO-9013173 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS 24 PFHxS 25 PFHxS 26 PFHxS 27 PFHxS 28 PFBS 29 PFBS 30 PFBS 31 PFBS 32 PFBS 33 PFBS 34 PFBS 35 PFOA 36 PFOA 37 PFOA 38 PFOA 39 PFOA 40 PFOA 41 PFOA 42 PFBA 43 PFBA 44 PFBA 45 PFBA 46 PFBA 47 PFBA 48 PFBA Dose1 0 0.32 1.6 8 40 200 1000 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 Luc12 0.2699435 1.423668 2.186356 2.075191 4.315969 7.138336 6.916809 1.451643 1.790085 1.87229 1.613219 2.22789 2.704702 1.913311 1.616936 1.555113 2.453635 1.455274 2.625709 3.184645 1.519225 1.513617 1.227581 0.9261827 2.194648 2.512845 2.747372 1.234832 2.301728 2.631239 1.931713 1.639419 1.359096 1.097309 2.475862 0.8823314 2.016315 1.732709 1.730784 1.061295 3.558488 1.737606 1.564718 1.315794 1.205181 1.082178 1.451322 1.919343 Luc2 0.7931641 2.339388 1.62824 2.24288 4.973182 6.818035 6.96418 1.376961 1.547474 2.037152 3.017765 2.980243 3.347376 1.918895 1.877738 2.457464 2.57857 2.340294 3.331317 3.218487 1.242754 1.4925 1.156105 1.522918 2.146304 2.365294 2.917912 1.597554 2.410897 1.983705 1.97877 1.663126 1.397776 1.191628 2.712058 1.735937 1.389305 2.11884 1.94652 1.556958 2.108 1.728782 1.921795 1.614791 1.655908 1.196779 1.487678 1.283868 1 Dose in nM for TO901317 and uM for client's compounds 2 Expressed as (Luciferase RLU/Renilla RLU) Human PXR Activity Assay SUMMARIZED Luc3 0.9932408 1.035379 1.885377 2.516727 4.966083 7.072407 8.114037 1.484295 1.778647 1.930471 2.610429 2.454123 2.587978 1.231107 1.365523 2.152485 2.489945 2.048763 1.913984 3.950592 0.9651626 1.631546 1.445242 1.737681 1.748068 2.881724 4.808423 1.371582 2.404659 1.791228 1.221857 1.736557 1.278307 1.527382 1.992268 1.606417 0.9668043 1.785514 2.246388 2.084046 2.014824 1.955788 1.171592 1.825245 1.55435 1.458556 1.59692 2.034257 Group Compound 1 DMSO 2 TO-9013173 3 TO-9013173 4 TO-9013173 5 TO-9013173 6 TO-9013173 7 TO-9013173 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS 24 PFHxS 25 PFHxS 26 PFHxS 27 PFHxS 28 PFBS 29 PFBS 30 PFBS 31 PFBS 32 PFBS 33 PFBS 34 PFBS 35 PFOA 36 PFOA 37 PFOA 38 PFOA 39 PFOA 40 PFOA 41 PFOA 42 PFBA 43 PFBA 44 PFBA 45 PFBA 46 PFBA 47 PFBA 48 PFBA Dose 0 0.32 1.6 8 40 200 1000 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 Mean3 1 1.82006755 2.16202489 2.59246901 5.40707309 7.97630819 8.34281032 0.85179388 1.01044632 1.15337784 1.43017289 1.51328903 1.70640369 1 0.95988476 1.21759449 1.48561821 1.15425039 1.55451776 2.04485166 1 1.24429482 1.02730951 1.12332241 1.63369699 2.08198771 2.81011781 1 1.69299195 1.52383938 1.22083232 1.19865375 0.95985008 0.90778974 1 0.58838089 0.6089568 0.78508571 0.82500514 0.65489915 1.0697926 1 0.85908407 0.87710727 0.81432971 0.68930131 0.83654975 0.96593471 Std Err 0.06773475 0.43987464 0.18352244 0.14642964 0.24794841 0.11111887 0.44540175 0.01882165 0.04682685 0.02860172 0.24719367 0.13204547 0.13988599 0.1352895 0.08761379 0.1570151 0.0219844 0.1542821 0.24242037 0.1480452 0.12874006 0.03482091 0.06998658 0.19539558 0.11388829 0.12361465 0.53158522 0.07547117 0.02525888 0.18131024 0.17471967 0.02086622 0.02511526 0.09313316 0.08850901 0.11096929 0.12738341 0.05050336 0.062464 0.12337667 0.2088036 0.04107671 0.1198679 0.08177771 0.07552401 0.06162773 0.02420658 0.12910719 3 Expressed relative to DMSO control 4 Oneway Anova, Dose x Relative Luciferase, Prob > F 5 Dunnett's t-test, DMSO control versus each sample DATA CV Stats4 p-value5 Renilla6 11.7320024 41.8602721 14.7024301 9.78308966 7.94254565 2.41293994 9.24698541 3.82722316 0.0032 0.9165 0.907249863 8.02679824 1 1.004956829 4.29517767 0.9048 0.924825023 29.9370795 0.1429 0.789968427 15.113403 0.0644 0.518650202 14.1988466 0.009 0.610922973 23.4328283 0.0018 1 1 15.8093494 1 1.005491244 22.3356905 0.7988 1.066335615 2.56311428 0.1484 0.947387028 23.1513406 0.9419 0.827814675 27.0105882 0.0838 0.650976949 12.539873 0.0011 0.636128925 22.2984328 0.0005 1 1 4.84704883 0.9358 0.98896434 11.799785 1 0.990124327 30.1280445 0.9976 0.991382842 12.0744727 0.2659 1.025955982 10.283771 0.0233 0.799988824 32.7649113 0.0004 0.541800852 13.0719904 0.0008 1 1 2.58416215 0.0021 0.838473597 20.6083762 0.0173 0.660562222 24.7882807 0.5181 0.830330148 3.01516253 0.6136 0.925845375 4.53205125 0.9997 0.850571568 17.7696828 0.9732 0.69897459 15.3302107 0.0765 1 1 32.666671 0.1224 1.112374818 36.2315586 0.1506 1.245113855 11.1420164 0.6516 1.122166898 13.1139568 0.8018 1.156368033 32.6301639 0.2345 0.981255019 33.8064073 0.9963 0.675082953 7.11469436 0.031409 1 1 24.1672842 0.702 1.017091648 16.1488967 0.798 1.148048428 16.0636932 0.4594 1.023359028 15.4855875 0.0864 1.008818678 5.01189864 0.5767 1.122079641 23.1506552 0.9995 1.094378002 6 Relative Renilla activity (toxicity marker) Relative renilla below 50% (0.5) indicates overt toxicity Curve Fitting Data TO901317 Bottom 1.449 Top 8.773 LogEC50 1.529 EC50 33.77 Span 7.325 PFOS 0.8992 1.741 0.9124 8.172 0.8413 PFOS217 1.111 3.179 2.087 122.2 2.068 PFHxS 1.044 3.636 1.674 47.25 2.591 PFBS PFOA 1.401 Interrupted 0.7898 1.262 18.28 -0.6114 PFBA Interrupted Statistical Analysis P-value Peak4 LSSD5 4 Highest observable 5 Lowest statistically NA, not applicable PFOS PFOS217 PFHxS 0.0032 0.0018 0.0005 1.706 2.045 2.8101178 100 100 33.3 response (relative to DMSO control) significant dose (p<0.05, Dunnett's) PFBS 0.0008 1.692992 0.41 PFOA 0.0765 1.0697926 N/A PFBA 0.031409 0.6893013 N/A Relative Luciferase Activity Relative Luciferase Activity 10 TO901317 8 6 4 2 0 -1 0 1 2 3 4 Log(Dose) pM 4 PFOS PFOS217 3 PFHxS PFBS 2 PFOA PFBA 1 0 -1 0 1 2 Log(Dose) M (C) Human CAR3 Activity Assay RAW DATA Group Compound 1 DMSO 2 CITCO 3 CITCO 4 CITCO 5 CITCO 6 CITCO 7 CITCO 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS 24 PFHxS 25 PFHxS 26 PFHxS 27 PFHxS 28 PFBS 29 PFBS 30 PFBS 31 PFBS 32 PFBS 33 PFBS 34 PFBS 35 PFOA 36 PFOA 37 PFOA 38 PFOA 39 PFOA 40 PFOA 41 PFOA 42 PFBA 43 PFBA 44 PFBA 45 PFBA 46 PFBA 47 PFBA 48 PFBA Dose1 0 1.6 8 40 200 1000 5000 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 Luc12 0.2167248 1.506333 1.661328 1.616416 6.079296 26.20632 29.71467 1.2954 1.48921 1.943437 2.207644 2.357472 3.579363 1.912326 1.317282 1.540737 1.812527 1.414215 3.215519 3.44258 1.810927 1.250442 1.558483 0.65403 2.704604 1.915768 2.305565 1.079713 1.028022 0.280539 1.663123 1.293376 1.251064 0.8910954 1.089725 1.243024 1.937257 2.095318 2.081964 1.414356 1.591633 1.811601 2.717717 2.286602 1.951135 1.444449 1.750552 2.031571 Luc2 0.6569583 2.135472 1.75017 1.645852 7.039314 22.4475 30.46399 1.461348 1.626203 2.614328 2.396115 2.187248 3.781329 1.875983 2.546865 1.696937 2.455303 2.01943 2.594508 3.481387 1.347109 1.364983 1.117709 1.186113 1.69808 2.378524 1.811519 1.296352 1.083755 0.5447473 1.508355 1.477396 1.159395 1.369629 1.238347 1.567674 1.951602 1.740867 1.814638 1.651225 1.223631 2.122233 1.987632 2.237881 2.10414 1.721626 1.918734 2.429702 1 Dose in nM for TO901317 and uM for client's compounds 2 Expressed as (Luciferase RLU/Renilla RLU) Human CAR Activity Assay SUMMARIZED Luc3 0.9400501 1.036676 1.654854 2.143853 6.824971 22.89514 31.20507 1.263326 1.499532 2.015931 2.389978 2.338161 2.666823 1.163002 1.013073 1.672663 1.985599 1.870738 2.204319 5.685531 1.06981 1.598152 1.35386 1.338007 1.317618 1.878478 4.181846 1.287055 0.9279157 1.172045 0.978928 1.23914 1.332643 1.274268 1.168205 1.695682 0.9804638 2.189428 1.857409 1.860067 1.308074 2.217719 1.445104 2.522597 2.45519 1.657182 1.753327 2.009645 Group Compound 1 DMSO 2 CITCO 3 CITCO 4 CITCO 5 CITCO 6 CITCO 7 CITCO 8 PFOS 9 PFOS 10 PFOS 11 PFOS 12 PFOS 13 PFOS 14 PFOS217 15 PFOS217 16 PFOS217 17 PFOS217 18 PFOS217 19 PFOS217 20 PFOS217 21 PFHxS 22 PFHxS 23 PFHxS 24 PFHxS 25 PFHxS 26 PFHxS 27 PFHxS 28 PFBS 29 PFBS 30 PFBS 31 PFBS 32 PFBS 33 PFBS 34 PFBS 35 PFOA 36 PFOA 37 PFOA 38 PFOA 39 PFOA 40 PFOA 41 PFOA 42 PFBA 43 PFBA 44 PFBA 45 PFBA 46 PFBA 47 PFBA 48 PFBA Dose 0 1.6 8 40 200 1000 5000 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 0 0.41 1.23 3.7 11.1 33.3 100 Mean3 1 1.89641876 2.05364197 2.19136708 8.08411556 29.0023171 37.0423262 0.81192113 0.93206527 1.32766776 1.41250206 1.39011284 2.02522423 1 0.98503609 0.99172462 1.26298449 1.07130879 1.61863111 2.54669884 1 0.996625 0.95321637 0.75171849 1.35300624 1.46002716 1.96292155 1 0.82980975 0.54525413 1.13302485 1.09467121 1.02183439 0.96502228 1 1.28890817 1.39271654 1.72343696 1.64575376 1.40882659 1.17935106 1 0.99982118 1.14557739 1.05834494 0.78407144 0.88150309 1.05191616 Std Err 0.10351685 0.3870763 0.03739328 0.20808699 0.35379451 1.4415003 0.52319677 0.03718058 0.02668682 0.1288026 0.03746052 0.03260492 0.20772368 0.14780522 0.28408237 0.02940407 0.11634902 0.11032706 0.17839661 0.44913387 0.15340346 0.07259294 0.09036442 0.14714271 0.29359727 0.11412028 0.51231352 0.05791329 0.03733765 0.21651598 0.16964702 0.05904785 0.040982 0.11976036 0.03683287 0.11559589 0.27573495 0.11718022 0.07113687 0.11047504 0.09549803 0.05978845 0.17980789 0.04287573 0.07276453 0.04084049 0.02711702 0.06657416 3 Expressed relative to DMSO control 4 Oneway Anova, Dose x Relative Luciferase, Prob > F 5 Dunnett's t-test, DMSO control versus each sample DATA CV Stats4 p-value5 Renilla6 17.9296436 35.3527305 3.15376591 16.4471411 7.58017458 8.60880098 2.44639979 7.93163981 0.0001 0.6929 0.907249863 4.95919338 0.9951 1.004956829 16.8033495 0.2118 0.924825023 4.59351654 0.0839 0.789968427 4.06250254 0.1081 0.518650202 17.7653397 0.0001 0.610922973 25.6006153 0.0021 1 1 49.9519863 1 1.005491244 5.135432 1 1.066335615 15.9560482 0.91 0.947387028 17.8372535 0.9999 0.827814675 19.0897107 0.2754 0.650976949 30.5463162 0.0014 0.636128925 26.5702592 0.0516 1 1 12.6160452 1 0.98896434 16.4197521 1 0.990124327 33.9034695 0.9467 0.991382842 37.5848512 0.8067 1.025955982 13.5382502 0.6075 0.799988824 45.2057319 0.064 0.541800852 10.0308766 0.0525 1 1 7.79343841 0.8134 0.838473597 68.7783281 0.075 0.660562222 25.9338761 0.922 0.830330148 9.34288534 0.9822 0.925845375 6.9466146 1 0.850571568 21.4949473 0.9999 0.69897459 6.37964081 0.0275 1 1 15.5339193 0.5002 1.112374818 34.2917544 0.2323 1.245113855 11.7765893 0.0104 1.122166898 7.48670126 0.0224 1.156368033 13.5821104 0.2032 0.981255019 14.0252924 0.8577 0.675082953 10.3556633 0.128 1 1 31.1492104 1 1.017091648 6.48257753 0.6792 1.148048428 11.9083911 0.9909 1.023359028 9.02185598 0.3261 1.008818678 5.32817916 0.8218 1.122079641 10.9618831 0.995 1.094378002 6 Relative Renilla activity (toxicity marker) Relative renilla below 50% (0.5) indicates overt toxicity Curve Fitting Data CITCO Bottom 0.7363 Top 42.9 LogEC50 2.809 EC50 644.4 Span 42.16 PFOS 0.9705 2.328 1.581 38.11 1.357 PFOS217 1.009 10.65 2.721 525.9 9.645 PFHxS 0.9236 2.601 1.795 62.41 1.677 PFBS PFOA 0.8209 Ambiguous 1.053 0.5115 3.247 0.2322 PFBA 1.054 0.9223 0.5894 3.885 -0.1318 Statistical Analysis P-value Peak4 LSSD5 4 Highest observable 5 Lowest statistically NA, not applicable PFOS PFOS217 PFHxS 0.0001 0.0021 0.0516 2.025 2.547 1.9629215 100 100 N/A response (relative to DMSO control) significant dose (p<0.05, Dunnett's) PFBS 0.0525 0.5452541 N/A PFOA 0.0275 1.723437 3.7 PFBA 0.128 0.7840714 N/A Relative Luciferase Activity Relative Luciferase Activity 40 CITCO 30 20 10 0 -1 0 1 2 3 4 Log(Dose) pM 4 PFOS PFOS217 3 PFHxS PFBS 2 PFOA PFBA 1 0 -1 0 1 2 Log(Dose) M Statistical Summary: TRalpha Statistical Analysis PFOS PFOS217 PFHxS PFBS PFOA PFBA P-value 0.0087 0.0014 0.0015 0.0035 0.1817 0.3298 Peak1 1.360 1.475 1.6225666 1.2237863 1.5418959 0.8240689 LSSD2 33.3 33.3 33.3 N/A N/A N/A Statistical Analysis PFOS PFOS217 PFHxS PFBS PFOA PFBA PXR P-value Peak1 0.0032 1.706 0.0018 0.0005 2.045 2.8101178 0.0008 0.0765 0.031409 1.692992 1.0697926 0.6893013 LSSD2 100 100 33.3 0.41 N/A N/A CAR3 Statistical Analysis PFOS PFOS217 PFHxS PFBS PFOA PFBA P-value 0.0001 0.0021 0.0516 0.0525 0.0275 0.128 Peak1 2.025 2.547 1.9629215 0.5452541 1.723437 0.7840714 LSSD2 100 100 N/A N/A 3.7 N/A Relative Luciferase Activity 3 TR 2 1 PFOS PFOS217 PFHxS PFBS PFOA PFBA Relative Luciferase Activity 0 -1 0 1 2 Log(Dose) M 4 3 PXR 2 1 PFOS PFOS217 PFHxS PFBS PFOA PFBA 0 -1 0 1 2 Log(Dose) M 4 3 CAR3 2 1 PFOS PFOS217 PFHxS PFBS PFOA PFBA 0 -1 0 1 2 Log(Dose) M Relative Luciferase Activity