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11.12.2023 01:29:44

Press Release: Novartis presents new 48-week -2-

inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis

Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.

Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on https://www.linkedin.com/company/novartis/ LinkedIn, https://www.facebook.com/novartis/ Facebook, https://twitter.com/Novartis X/Twitter and https://instagram.com/novartis?igshid=MzRlODBiNWFlZA==__;!!N3hqHg43uw!pjp8z253J5NjaOYrW65UbAAlHeHRdQ-w0m4ezZxEQEl0ptafXN2M99VRIk39pf49PAc8NbK93Pxp3uaSBQkAf8oEnzWXG8Sk$ Instagram.

References

1. Risitano AM, Kulasekararaj A, R th A, et al. Factor B Inhibition with

Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and

Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal

Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from

the Multicenter, Phase III APPLY-PNH Trial. Presented at: 65th American

Society of Hematology Annual Meeting & Exposition (ASH); December 9-12,

2023; San Diego, CA

2. Risitano AM, R th A, Kulasekararaj A, et al. Oral Iptacopan Monotherapy

Has Superior Efficacy to Anti-C5 Therapy in Patients with Paroxysmal

Nocturnal Hemoglobinuria and Residual Anemia: Results From the Phase III

APPLY-PNH Study. Presented at: 49th Annual Meeting of the European

Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023;

Paris, France.

3. Risitano AM, Han B, Ueda Y, et al. Oral Complement Factor B Inhibitor

Iptacopan Monotherapy Improves Hemoglobin to Normal/Near-Normal Levels in

Paroxysmal Nocturnal Hemoglobinuria Patients Naïve to Complement

Inhibitors: Phase III APPOINT-PNH Trial. Presented at: 49th Annual

Meeting of the European Society for Blood and Marrow Transplantation

(EBMT); April 23-36, 2023; Paris, France.

4. 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

5. Jang H, 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

6. 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

7. 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

8. 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

9. 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

10. 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

11. Novartis Pharmaceuticals. A Randomized, Multicenter, Active-Comparator

Controlled, Open-Label Trial to Evaluate Efficacy and Safety of Oral,

Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia,

Despite Treatment With an Intravenous Anti-C5 Antibody.

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

https://clinicaltrials.gov/ct2/show/NCT04558918

12. 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

13. 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

14. 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

15. McKinley CE, Richards SJ, Munir T, et al. Extravascular Hemolysis Due to

C3-Loading in Patients with PNH Treated with Eculizumab: Defining the

Clinical Syndrome. Blood. 2017;130(Supplement 1):3471.

doi:10.1182/blood.V130.Suppl_1.3471.3471

16. Barratt J, Rovin B, Zhang H, et al. POS-546 EFFICACY AND SAFETY OF

IPTACOPAN IN IgA NEPHROPATHY: RESULTS OF A RANDOMIZED DOUBLE-BLIND

PLACEBO-CONTROLLED PHASE 2 STUDY AT 6 MONTHS. Kidney Int Rep.

2022;7(2):S236. doi:10.1016/j.ekir.2022.01.577

17. Rizk DV, Rovin BH, Zhang H, et al. Targeting the Alternative Complement

Pathway With Iptacopan to Treat IgA Nephropathy: Design and Rationale of

the APPLAUSE-IgAN Study. Kidney Int Rep. 2023;8(5):968-979.

doi:10.1016/j.ekir.2023.01.041

18. 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

19. 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

20. 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

21. 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

22. 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-investigation

al-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-design

ation-pnh-and-rare-pediatric-disease-designation-c3g

23. Novartis. Data on File.

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December 10, 2023 19:30 ET (00:30 GMT)

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