Press Release: Novartis investigational iptacopan -2-

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References

1. Novartis data on file.

2. Novartis iptacopan meets primary endpoints in Phase II study in rare

kidney disease C3 glomerulopathy (C3G). Novartis. Accessed December 6,

2023.

https://www.novartis.com/news/media-releases/novartis-iptacopan-meets-primary-endpoints-phase-ii-study-rare-kidney-disease-c3-glomerulopathy-c3g

https://www.novartis.com/news/media-releases/novartis-iptacopan-meets-primary-endpoints-phase-ii-study-rare-kidney-disease-c3-glomerulopathy-c3g

3. Wong E, Nester C, Cavero T, et al. Efficacy and Safety of Iptacopan in

Patients With C3 Glomerulopathy. Kidney Int Rep. 2023;8:2754-2764.

4. Schena FP, Esposito P, Rossini M. A Narrative Review on C3

Glomerulopathy: A Rare Renal Disease. Int J Mol Sci. 2020;21(2):525.

5. Smith RJ, Alexander J, Barlow PN, et al. New Approaches to the Treatment

of Dense Deposit Disease. J Am Soc Nephrol. 2007;18(9):2447-2456.

6. Martin B, Smith RJH. In: Adam MP, Ardinger HH, Pagon RA, et al. C3

Glomerulopathy. GeneReviews(R) [Internet]. Updated 2018. University of

Washington, Seattle; 1993-2022.

7. Smith RJH, Appel GB, Blom AM, et al. C3 Glomerulopathy -- Understanding a

Rare Complement-driven Renal Disease. Nat Rev Nephrol.

2019;15(3):129-143.

8. Goodship TH, Cook HT, Fakhouri F, et al. Atypical Hemolytic Uremic

Syndrome and C3 Glomerulopathy: Conclusions from a "Kidney Disease:

Improving Global Outcomes" (KDIGO) Controversies Conference. Kidney Int.

2017;91(3):539-551.

9. Kidney Disease: Improving Global Outcomes (KDIGO) Glomerular Diseases

Work Group. KDIGO 2021 Clinical Practice Guideline for the Management of

Glomerular Diseases. Kidney Int. 2021;100(4S):S1-S276.

10. Novartis investigational iptacopan Phase III study demonstrates

clinically meaningful and highly statistically significant proteinuria

reduction in patients with IgA nephropathy (IgAN). Novartis. Accessed

December 6, 2023.

https://www.novartis.com/news/media-releases/novartis-investigational-iptacopan-phase-iii-study-demonstrates-clinically-meaningful-and-highly-statistically-significant-proteinuria-reduction-patients-iga-nephropathy-igan

https://www.novartis.com/news/media-releases/novartis-investigational-iptacopan-phase-iii-study-demonstrates-clinically-meaningful-and-highly-statistically-significant-proteinuria-reduction-patients-iga-nephropathy-igan

11. Novartis Pharmaceuticals. Study of Efficacy and Safety of LNP023 in

Primary IgA Nephropathy Patients (APPLAUSE-IgAN). clinicaltrials.gov.

Accessed December 6, 2023. https://clinicaltrials.gov/study/NCT04578834

https://clinicaltrials.gov/study/NCT04578834

12. Novartis Pharmaceuticals. Study of Efficacy and Safety of Iptacopan in

Patients With C3 Glomerulopathy. (APPEAR-C3G). clinicaltrials.gov.

Accessed December 6, 2023. https://clinicaltrials.gov/study/NCT04817618

https://clinicaltrials.gov/study/NCT04817618

13. Novartis Pharmaceuticals. Efficacy and Safety of Iptacopan (LNP023) in

Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to

Complement Inhibitor Therapy (APPELHUS). clinicaltrials.gov. Accessed

December 6, 2023. https://clinicaltrials.gov/study/NCT04889430

https://clinicaltrials.gov/study/NCT04889430

14. Novartis Pharmaceuticals. Study of Efficacy and Safety of Iptacopan in

Participants With IC-MPGN (APPARENT). clinicaltrials.gov. Accessed

December 6, 2023. https://clinicaltrials.gov/study/NCT05755386

https://clinicaltrials.gov/study/NCT05755386

15. Bomback AS, Kavanagh D, Vivarelli M, et al. Alternative Complement

Pathway Inhibition With Iptacopan for the Treatment of C3

Glomerulopathy-Study Design of the APPEAR-C3G Trial. Kidney Int Rep.

2022;7(10):2150-2159.

16. Novartis receives FDA approval for Fabhalta(R) (iptacopan), offering

superior hemoglobin improvement in the absence of transfusions as the

first oral monotherapy for adults with PNH. Novartis. Accessed December

6, 2023.

https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh

https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh

17. Iptacopan Prescribing Information. East Hanover, NJ: Novartis

Pharmaceuticals Corp; December 2023.

18. Medjeral-Thomas NR, O'Shaughnessy MM, O'Regan JA, et al. C3

Glomerulopathy: Clinicopathologic Features and Predictors of Outcome.

Clin J Am Soc Nephrol. 2014;9(1):46-53.

19. Ravindran A, Fervenza FC, Smith RJH, Sethi S. C3 Glomerulopathy

Associated with Monoclonal Ig is a Distinct Subtype. Kidney Int.

2018;94(1):178-186.

20. Caravaca-Fontán F, Lucientes L, Cavero T, Praga M. Update on C3

Glomerulopathy: A Complement-Mediated Disease. Nephron.

2020;144(6):272-280.

21. Servais A, Noël LH, Roumenina LT, et al. Acquired and Genetic

Complement Abnormalities Play a Critical Role in Dense Deposit Disease

and Other C3 Glomerulopathies. Kidney Int. 2012;82(4):454-464.

22. Zand L, Lorenz EC, Cosio FG, et al. Clinical Findings, Pathology, and

Outcomes of C3GN after Kidney Transplantation. J Am Soc Nephrol.

2014;25(5):1110-1117.

23. Regunathan-Shenk R, Avasare RS, Ahn W, et al. Kidney Transplantation in

C3 Glomerulopathy: A Case Series. Am J Kidney Dis. 2019;73(3):316-323.

24. Caravaca-Fontán F, Polanco N, Villacorta B, et al. Recurrence of

Immune Complex and Complement-mediated Membranoproliferative

Glomerulonephritis in Kidney Transplantation. Nephrol Dial Transplant.

2023;38(1):222-235.

25. Novartis investigational oral therapy iptacopan (LNP023) receives FDA

Breakthrough Therapy Designation for PNH and Rare Pediatric Disease

Designation for C3G. Novartis. Accessed December 6, 2023.

https://www.novartis.com/news/media-releases/novartis-investigational-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-designation-pnh-and-rare-pediatric-disease-designation-c3g

https://www.novartis.com/news/media-releases/novartis-investigational-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-designation-pnh-and-rare-pediatric-disease-designation-c3g

26. Novartis announces European Medicines Agency (EMA) has granted orphan

drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN).

Novartis. Accessed December 6, 2023.

https://www.novartis.com/news/media-releases/novartis-announces-european-medicines-agency-ema-has-granted-orphan-drug-designation-iptacopan-lnp023-iga-nephropathy-igan

https://www.novartis.com/news/media-releases/novartis-announces-european-medicines-agency-ema-has-granted-orphan-drug-designation-iptacopan-lnp023-iga-nephropathy-igan

27. Novartis received European Medicines Agency (EMA) PRIME designation for

iptacopan (LNP) in C3 glomerulopathy (C3G). Novartis. Accessed December

6, 2023.

https://www.novartis.com/news/media-releases/novartis-received-european-medicines-agency-ema-prime-designation-iptacopan-lnp-c3-glomerulopathy-c3g

https://www.novartis.com/news/media-releases/novartis-received-european-medicines-agency-ema-prime-designation-iptacopan-lnp-c3-glomerulopathy-c3g

28. Novartis data on file.

29. National Kidney Foundation. Global Facts: About Kidney Disease. Accessed

December 6, 2023.

https://www.kidney.org/kidneydisease/global-facts-about-kidney-disease

https://www.kidney.org/kidneydisease/global-facts-about-kidney-disease

30. NHS. Kidney Disease. Accessed December 6, 2023.

https://www.nhs.uk/conditions/kidney-disease/

31. National Kidney Foundation. Chronic kidney disease (CKD). Accessed

December 6, 2023.

https://www.kidney.org/atoz/content/about-chronic-kidney-disease

https://www.kidney.org/atoz/content/about-chronic-kidney-disease

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(END) Dow Jones Newswires

December 11, 2023 01:15 ET (06:15 GMT)