Press Release: Novartis investigational iptacopan -2-

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References

1. Novartis data on file.

2. Cançado RD, Araújo A da S, Sandes AF, et al. Consensus

statement for diagnosis and treatment of paroxysmal nocturnal

haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348.

doi:10.1016/j.htct.2020.06.006

3. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients

with paroxysmal nocturnal hemoglobinuria receiving treatment with the

C5-inhibitors eculizumab or ravulizumab: results from a US patient

survey. Ann Hematol. 2022;101(2):251-263. doi:10.1007/s00277-021-04715-5

4. Debureaux PE, Kulasekararaj AG, Cacace F, et al. Categorizing

hematological response to eculizumab in paroxysmal nocturnal

hemoglobinuria: a multicenter real-life study. Bone Marrow Transplant.

2021;56(10):2600-2602. doi:10.1038/s41409-021-01372-0

5. Debureaux PE, Cacace F, Silva BGP, et al. Hematological Response to

Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel

Classification to Identify Unmet Clinical Needs and Future Clinical

Goals. Blood. 2019;134(Supplement_1):3517-3517.

doi:10.1182/blood-2019-125917

6. Risitano AM, Röth A, Soret J, et al. Addition of iptacopan, an oral

factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal

haemoglobinuria and active haemolysis: an open-label, single-arm, phase

2, proof-of-concept trial. Lancet Haematol. 2021;8(5):e344-e354.

doi:10.1016/S2352-3026(21)00028-4

7. Jang JH, Wong L, Ko BS, et al. Iptacopan monotherapy in patients with

paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label

proof-of-concept study. Blood Adv. 2022;6(15):4450-4460.

doi:10.1182/bloodadvances.2022006960

8. Novartis investigational oral monotherapy iptacopan demonstrates

clinically meaningful superiority over anti-C5 treatment in Phase III

APPLY-PNH study. Novartis. Accessed November 4, 2022.

https://www.novartis.com/news/media-releases/novartis-investigational-oral-monotherapy-iptacopan-demonstrates-clinically-meaningful-superiority-over-anti-c5-treatment-phase-iii-apply-pnh-study

9. Novartis Pharmaceuticals. A Multicenter, Randomized, Double-Blind,

Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and

Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.

clinicaltrials.gov; 2022. Accessed September 20, 2022.

https://clinicaltrials.gov/ct2/show/NCT04817618

10. Novartis Pharmaceuticals. A Multi-Center, Randomized, Double-Blind,

Placebo-Controlled, Parallel Group, Phase III Study to Evaluate the

Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients.

clinicaltrials.gov; 2022. Accessed September 21, 2022.

https://clinicaltrials.gov/ct2/show/NCT04578834

11. Novartis Pharmaceuticals. A Multicenter, Single-Arm, Open Label Trial to

Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult AHUS

Patients Who Are Naive to Complement Inhibitor Therapy.

clinicaltrials.gov; 2022. Accessed September 20, 2022.

https://clinicaltrials.gov/ct2/show/NCT04889430

12. Novartis Pharmaceuticals. A Multicenter, Single-Arm, Open-Label Trial to

Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH

Patients Who Are Naive to Complement Inhibitor Therapy.

clinicaltrials.gov; 2022. Accessed September 21, 2022.

https://clinicaltrials.gov/ct2/show/NCT04820530

13. Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal

haemoglobinuria. Nat Rev Dis Primer. 2017;3(1):17028.

doi:10.1038/nrdp.2017.28

14. Schrezenmeier H, Röth A, Araten DJ, et al. Baseline clinical

characteristics and disease burden in patients with paroxysmal nocturnal

hemoglobinuria (PNH): updated analysis from the International PNH

Registry. Ann Hematol. 2020;99(7):1505-1514.

doi:10.1007/s00277-020-04052-z

15. Schubart A, Anderson K, Mainolfi N, et al. Small-molecule factor B

inhibitor for the treatment of complement-mediated diseases. Proc Natl

Acad Sci. 2019;116(16):7926-7931. doi:10.1073/pnas.1820892116

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

Breakthrough Therapy Designation for PNH and Rare Pediatric Disease

Designation for C3G. Novartis. Accessed September 22, 2022.

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

17. Novartis data on file.

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

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

Novartis. Accessed September 22, 2022.

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

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

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

22, 2022.

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

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(END) Dow Jones Newswires

December 08, 2022 01:15 ET (06:15 GMT)