IPTACOPAN ASH UPDATE

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#1YYYYYYYY AYYAYYAYAY YYYYYYYY AYYAY YYLYY AYYAY Novartis Investor Relations ATTAT YYAYY AYYAY YYAYY LYYLY YYAYY AYYAY YYAYY AYYAY YYAYY LYYLY YYLYY AYYAY YYLYY LYYLY YY LY YYYYYY AYYAYYAYAY YYAYYAYYYY AYYAYYAYAY YYAYYAYYYY AY YYAYAY YYYYYYYY AYYAYYAYAY YYYYYYYY AYYAYYAYAY AYYAYYYY AYY YY YYAYAY YYYY YAYAY YYYY AYAY YAYYYY Iptacopan ASH Update Investor Presentation December 13, 2022 1 NOVARTIS | Reimagining Medicine#2Disclaimer This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "expected," "will," "planned," "pipeline," "outlook," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for iptacopan or regarding potential future revenues from iptacopan. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that iptacopan will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that iptacopan will be commercially successful in the future. In particular, our expectations regarding iptacopan could be affected by, among other things, the uncertainties 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 such as COVID-19; 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 presentation as of this date and does not undertake any obligation to update any forward-looking statements contained in this presentation as a result of new information, future events or otherwise. 2 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#3Participants David Soergel MD Global Head of Cardiovascular, Renal & Metabolism Development Reshema Kemps-Polanco Executive Vice President, Oncology US 3 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#4Iptacopan is a first-in-class, oral, selective factor B inhibitor of the alternative complement pathway ◉ Dysregulation of the complement pathway is associated with a range of rare hematologic and renal diseases As a selective factor B inhibitor, iptacopan targets the complement system proximally via the alternative pathway, leaving classical and lectin pathway signaling intact Discovered in-house at NIBR 4 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#5Opportunity to redefine care in multiple complement-driven conditions Indication 2021 2022 2023 2024 2025 2026 Market potential PNH Indication Ph3 - APPLY US prevalence thousands Ph3 APPOINT Hematology PNH <10 IgAN Ph3 APPLAUSE Nephrology Ph3 - APPEAR Ph3 - APPELHUS C3G aHUS IC-MPGN Phase 3 studies initiated or planned; additional indications are being explored IgAN ~46-551 C3G <10 aHUS <10 Ph3 IC-MPGN <10 PNH = paroxysmal nocturnal hemoglobinuria IgAN = IgA nephropathy * 9 months readout may support US submission for accelerated approval C3G = C3 glomerulopathy aHUS = atypical hemolytic uremic syndrome IC-MPGN = immune complex membranoproliferative glomerulonephritis 1. Estimated number of patients at high risk of progression with proteinuria > 1g/day (-25-30%) 5 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#6Two positive Ph3 studies in PNH are the first pivotal readouts for iptacopan Study APPLY-PNH Patient type PNH patients with residual anemia despite anti-C5 APPOINT-PNH PNH patients naive to complement inhibitor therapy Intervention Iptacopan vs. anti-C5 antibody Iptacopan, single-arm study SOCIETY AMERICAN OF 6 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 HEMATOLOG NOVARTIS | Reimagining Medicine#7Significant unmet need remains in PNH despite current standard of care anti-C5 therapy PNH prevalence 10-20 cases/million = ~6k patients in the US1 63% of patients treated with a terminal complement inhibitor showed signs of ongoing hemolysis² ~1/3 of patients reported having ≥1 RBC transfusion despite treatment with terminal complement inhibitors 3,4 >75% of patients treated with terminal complement inhibitors reported fatigue symptoms³ 1. Cançado RD, 2021 and Jalbert JJ, 2019, Mon Pere N, 2018. 2. Risitano AM et al. Blood. 2009;113(17):4094-4100. 3. Dingli D et al. Ann Hematol. 2022;101(2):251-263. 4. Kulasekararaj AG et al. Eur J Haematol. 2022;109(3):205-214. doi:10.1111/ejh.13783. 7 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#8Complement regulation in PNH is impaired 1,2 Alternative pathway (Tick-over) C3 Factor B C3(H2O) Factor D CD55 C3 convertase Factor B Factor D C3 CD55 C3b C3 convertase C3a C3b Proximal pathways Terminal pathway C5 convertase C5 inhibitors C5 C6, C7, C8, C9 C5a C5b Extravascular hemolysis via C3 opsonization and phagocytosis CD59 MAC Intravascular hemolysis → PNH is a rare, chronic hematological disorder characterized by intravascular hemolysis (IVH), thrombophilia and bone marrow failure 1,2 → Caused by an acquired mutation in hematopoietic stem cells, which results in a lack of complement- regulatory proteins, leading to IVH → Targeting the terminal complement pathway at C5 can address IVH, reduce thrombosis and improve overall survival 3-9 → However, up to 2/3 of patients have clinically meaningful residual anemia, largely because of emerging extravascular hemolysis 1,10 C = complement component; CD = cluster of differentiation; GPI = glycosylphosphatidylinositol; MAC = membrane attack complex; PNH = paroxysmal nocturnal hemoglobinuria; SoC = standard of care. 1. Risitano AM et al. Front Immunol 2019;10:1157. 2. Risitano AM, Peffault de Latour R. Br J Haematol 2022;196:288-303. 3. Hillmen P et al. N Engl J Med 2006;355:1233-43. 4. Kelly RJ et al. Blood 2011;117:6786-92. 5. Brodsky RA et al. Blood 2008;111:1840-7. 6. Hillmen P et al. Blood 2007;110:4123-8. 7. Loschi M et al. Am J Hematol 2016; 91:266-70. 8. Kulasekararaj AG et al. Blood 2019;133:540-9. 9. Lee JW et al. Blood 2019;133:530-9. 8 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 10. Risitano AM et al. Blood 2009;113:4094-100 NOVARTIS | Reimagining Medicine#9Iptacopan, a first-in-class, oral, selective factor B inhibitor, targets the complement system proximally via the alternative pathway1 Alternative pathway (Tick-over) C3 Iptacopan Factor B C3(H2O) Factor D C3 convertase Iptacopan binds to the active site of factor B, Iptacopan inhibiting the activity of C3 convertase¹ Factor B Factor B Factor D C3 C3 C3b convertase C3a C3b Proximal pathways --- Terminal pathway C5 convertase C5 C6, C7, C8, C9n C5a C5b No C3 opsonization Iptacopan controlled intra- and extravascular hemolysis in 10 patients with a sub-optimal response to eculizumab, leading to transfusion independence and an improved quality of life² MAC THE LANCET Haematology 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 Material from The Lancet Haematology is used with permission. 1. Schubart A et al. Proc Natl Acad Sci USA 2019;116:7926-31. 2. Risitano AM et al. Lancet Haematol 2021;8:e344-54. 9 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#10APPLY-PNH is a randomized Ph3 trial investigating iptacopan monotherapy in PNH patients with residual anemia despite SoC Study design DATA PRESENTED UP TO 24 WEEKS Iptacopan 200mg BID (n=62) Continue with iptacopan 200mg BID Anti-C5 antibody Anti-C5 antibody (n= 35) Switch to iptacopan 200mg BID Up to 8 weeks 24 weeks 24 weeks D-60 Screening period D1 D168 Randomized treatment period Treatment extension period D336 EoS PNH = Paroxysmal Nocturnal Hemoglobinuria Hb Hemoglobin RBC = Red Blood Cell BID twice a day EOS End of Study 10 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 Population (n = 97) Adult PNH patients with residual anemia (Hb 10g/dL) on a stable regimen of anti-C5 therapy 6 months prior to randomization Primary endpoints Superiority for proportion of patients achieving increase in Hb ≥ 2g/dL from baseline in the absence of RBC transfusion Superiority for proportion of patients achieving Hb ≥ 12g/dL in the absence of RBC transfusion NOVARTIS | Reimagining Medicine#11Iptacopan was superior to SoC for both primary endpoints; majority of iptacopan patients achieved more normal Hb levels vs. 0 on SoC Observed Increase from baseline in Hb of ≥ 2 g/dL in the absence of RBC transfusions 51/601 patients treated with iptacopan 0/35 patients treated with SoC Hb ≥ 12 g/dL in the absence of RBC transfusions 42/601 0/35 patients treated patients treated with iptacopan with SoC Population estimate Estimated proportion² of patients, % (95% CI) 100 90 80 70 60 10 222223220 50 82.3% 40 Difference = 80.3% (95% CI 71.3, 87.6) P<0.00013 2.0% Estimated proportion² of patients, % (95% CI) 822222220 100 68.8% Difference = 67.0% (95% CI 56.3, 76.9) P<0.00013 1.8% 1. 2/62 patients in the iptacopan arm had missing data between Days 126 and 168 so were not evaluable based on observed data. 2. Marginal proportions, differences in marginal proportions and 95% Cls were calculated using a logistic regression model using the Firth correction that adjusted for baseline covariates and accounted for missing data and the possibility of no patients meeting the primary endpoint criteria in the SoC arm; consequently, the treatment effect of iptacopan is underestimated and the treatment effect of SoC is overestimated. Marginal proportions reflect the population average probability of a patient meeting the primary endpoint criteria. 3. P values are two-sided and unadjusted. Cl, confidence interval 11 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#12Iptacopan monotherapy was superior to SoC for transfusion avoidance Observed Population estimate Transfusion avoidance Estimated proportion¹ of patients, % (95% CI) 100 22843220 60/62 14/35 patients treated patients treated with iptacopan with SoC 96.4% Difference = 70.3% (95% CI 52.6, 84.9) P<0.00012 26.1% Of the patients treated with anti-C5 therapy who received transfusions, they received on average more than double the number of transfusions vs. the few patients on iptacopan A post hoc sensitivity analysis using a different approach for handling missing data confirmed the significance of the pre-specified analysis: 96.7% (95% CI 91.3, 100.0) 38.9% (95% CI 23.1, 55.8) Difference = 57.8% (95% CI 39.8, 74.2), P<0.00012 1. Marginal proportions, differences in marginal proportions and 95% CIs were calculated using a logistic regression model that adjusted for baseline covariates and accounted for missing data. Marginal proportions reflect the population average probability of a patient meeting the endpoint criteria. 2. P values are two-sided and unadjusted 12 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#1312 g/dL Iptacopan monotherapy was superior to SoC at increasing Hb level from baseline Mean Hb (SD) over time during the 24-week randomized treatment period³ Mean Hb level, g/dL (SD) 15- 14- 13- 12 11 10 + 0 8 BL Wk1 Wk2 Wk4 Wk6 Wk8 Wk12 Wk16 Wk18 Wk20 Wk22 Wk24 Patients with available data Iptacopan 62 61 59 60 Anti-C5 SoC 35 29 27 32 59 32 59 31 Study visit 60 33 59 59 59 57 28 34 32 32 61 34 7 NOVARTIS | Reimagining Medicine Adjusted mean Hb change from baseline¹ (95% CI) was +3.59 (3.32, 3.86) g/dL for iptacopan vs -0.04 (−0.42, 0.35) g/dL for SoC, with a difference of +3.63 (3.18, 4.08) g/dL (P<0.00012). 1. Between Days 126 and 168 (excluding values within 30 days of RBC transfusion). 2. A repeated measures model, adjusting for covariates including baseline Hb, was used for comparisons between the treatment arms. P value is two-sided and unadjusted. 3. Includes post-transfusion data. 2/62 patients in the iptacopan arm and 21/35 patients in the SoC arm had RBC transfusions between Days 14 and 168. BL = baseline Wk = week 13 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022#14Iptacopan monotherapy was superior to SoC at reducing patient- reported fatigue from baseline Mean FACIT-Fatigue score (SD) during the 24-week randomized treatment period 50 45- 40- 35 Mean FACIT-Fatigue score (SD) 25 88 30 25 20- Wk18 Wk20 Wk22 Wk24 15. BL Wk1 WK2 Wk6 Patients with available data Iptacopan 62 60 57 Anti-C5 SoC 33 27 28 61 32 Wk12 Study visit 57 29 58 59 56 60 29 28 28 30 Adjusted mean change from baseline¹ in FACIT-Fatigue score (95% CI) was +8.59 (6.72, 10.47) for iptacopan vs +0.31 (-2.20, 2.81) for SoC, with a difference of +8.29 (5.28, 11.29) (P<0.00012) 1. Between Days 126 and 168. 2. A repeated measures model, adjusting for covariates including baseline FACIT-Fatigue score, was used for comparisons between the treatment arms. P value is two-sided and unadjusted. 14 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#15Iptacopan monotherapy was superior to SoC at reducing absolute reticulocyte count from baseline Mean absolute reticulocyte count (SD) during the 24-week randomized treatment period Mean absolute reticulocyte count, 109/L (SD) 325- 275 225- 175- 125- 75- 25- 0. ULN LLN BL Wk1 WK2 Wk4 Wk6 Wk8 Wk12 Wk16 Wk18 Wk20 Wk22 Wk24 Patients with available data Study visit Iptacopan 62 61 58 Anti-C5 SoC 35 28 27 60 32 59 31 59 31 60 33 Adjusted mean change from baseline¹ in absolute reticulocyte count (95% CI) was -115.89 (-126.49, -105.30) x 109/L for iptacopan vs +0.37 (-13.03, 13.77) x 109/L for SoC, with a difference of -116.26 (-132.17, -100.36) x 109/L (P<0.00012). 56 59 56 56 58 27 34 32 32 34 1. Between Days 126 and 168. 2. A repeated measures model, adjusting for covariates including baseline absolute reticulocyte count, was used for comparisons between the treatment arms. P value is two-sided and unadjusted. LLN = lower limit of normal ULN upper limit of normal 15 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#16There was no significant difference between iptacopan monotherapy and SoC for change from baseline in LDH level Mean LDH level (SD) during the 24-week randomized treatment period Mean LDH, U/L (SD) 900 800 700 600 500 400 300 200 100 H I 1.5 x ULN ULN ■ As expected, with all patients having been treated with anti-C5s prior to entering the study, IVH was well controlled and LDH levels <1.5x ULN in the vast majority of patients 0 BL Wk1Wk2 Wk4 Wk6 Wk8 Wk12 Patients with available data Iptacopan 62 61 61 Anti-C5 SoC 35 28 28 Study visit Wk16 Wk18 Wk20 Wk22 Wk24 ■ No difference in LDH levels shows that iptacopan maintains IVH control 61 62 62 34 34 34 62 34 61 59 59 57 61 34 35 33 32 35 Adjusted geometric mean ratio to baseline¹ in log-transformed LDH (95% CI) was 0.96 (0.90, 1.03) for iptacopan vs 0.98 (0.89, 1.07) for SoC, equating to a reduction of 1.15% (95% CI -10.18, 11.32) with iptacopan vs SoC (P=0.83452). 1. Between Days 126 and 168. 2. A repeated measures model, adjusting for covariates including baseline LDH, was used for comparisons between the treatment arms. P value is two-sided and unadjusted. ULN = upper limit of normal 16 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#17Iptacopan monotherapy was superior to SoC for annualized rate of clinical breakthrough hemolysis¹ Adjusted annual Rate ratio Arm n/N² rate, % (95% CI) (95% CI)³ P value³ Rate of clinical breakthrough hemolysis1 Iptacopan 2/62 0.07 (0.02, 0.31) 0.10 0.0118 (0.02, 0.61) Anti-C5 SoC 6/35 0.67 (0.26, 1.72) ■ Rate ratio of 0.10 means 10-fold lower rate of annualized clinical breakthrough hemolysis 1. Events that met the protocol-specified criteria for clinical breakthrough hemolysis. All hemolytic events were also reported as TEAEs, even if they did not meet the criteria for clinical breakthrough hemolysis. 2. n=number of patients with at least one event, N=overall number of patients. 3. A negative binomial model was used for the comparison between treatment arms. P value is two-sided and unadjusted. TEAE = treatment-emergent adverse event. 17 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#18There was no significant difference between iptacopan monotherapy and SoC for the annualized rate of MAVES Adjusted annual Arm n/N1 rate, % (95% CI) Rate ratio (95% CI)² P value² Iptacopan 1/62 0.03 (0.00, 0.25) Rate of MAVES Not estimable 0.3173 Anti-C5 SoC 0/35 0 ■ Serious TEAE of transient ischemic attack, considered by the investigator to be unrelated to iptacopan ■ The patient had a concomitant serious TEAE of sick sinus syndrome and is continuing to receive iptacopan treatment MAVE = major adverse vascular event 1. n=number of patients with at least one event, N=overall number of patients. 2. A negative binomial model was used for the comparison between treatment arms. P value is two-sided and unadjusted. TEAE = treatment-emergent adverse event. 18 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#19Iptacopan monotherapy was well tolerated and had a favorable safety profile Most common TEAEs (24 patients in either arm)1 n (%) Iptacopan 200mg bid N=62 Any TEAE 51 (82.3) Anti-C5 SoC N=35 28 (80.0) No deaths No discontinuations due to TEAES Mild/Moderate / Severe, % 32.3/45.2/4.8 37.1 34.3/8.6 Headache 10 (16.1) 1 (2.9) Diarrhea 9 (14.5) 2 (5.7) Nasopharyngitis 7 (11.3) 2 (5.7) Nausea 6 (9.7) 1 (2.9) COVID-19 5 (8.1) 9 (25.7) Urinary tract infection 5 (8.1) 1 (2.9) Arthralgia 5 (8.1) 1 (2.9) Abdominal pain 4 (6.5) 1 (2.9) Increased blood LDH 4 (6.5) 3 (8.6) Dizziness 4 (6.5) 0 Serious TEAES: 9.7% vs 14.3% Hemolysis serious TEAEs: Iptacopan: None SoC: Breakthrough hemolysis (n=1) and extravascular hemolysis (n=1) No serious infections caused Breakthrough hemolysis 2 (3.2) 6 (17.1) by encapsulated bacteria TEAE = treatment-emergent adverse event 1. Organized by descending frequency in the iptacopan arm 19 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#20Two positive Ph3 studies in PNH first pivotal readouts for iptacopan Study APPLY-PNH Patient type PNH patients with residual anemia despite anti-C5 APPOINT-PNH PNH patients naive to complement inhibitor therapy Intervention Iptacopan vs. anti-C5 antibody Iptacopan, single-arm study SOCIETY AMERICAN OF 20 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 HEMATOLOG NOVARTIS | Reimagining Medicine#21APPOINT-PNH study is a single-arm Ph3 trial investigating iptacopan monotherapy in treatment-naive patients with PNH Study design iptacopan 200mg BID Time Up to 8 weeks 24 weeks Duration D-58 D1 D168 24 weeks Study period Screening period Core treatment period Treatment extension period BID = twice a day EOS - End of Study ULN = upper limit of normal 21 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 D336 EoS Population (n = 40) Adult PNH patients with hemolysis (LDH > 1.5x ULN) and anemia (Hb < 10g/dL) naive to complement inhibitor therapy Primary endpoint Proportion of patients achieving a sustained increase in Hb of ≥ 2g/dL, in the absence of transfusions NOVARTIS | Reimagining Medicine#22Iptacopan monotherapy achieved clinically meaningful increases in hemoglobin levels in treatment-naive patients with PNH APPOINT-PNH met primary endpoint of proportion of patients achieving a sustained increase in Hb of ≥ 2g/dL, in the absence of transfusions, at 24 weeks Safety profile consistent with previously reported data Data to be presented at upcoming medical meeting 22 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#23As a potent, selective factor B inhibitor, iptacopan has the potential to be practice-changing, the new standard of care in PNH Addresses both intravascular and extravascular hemolysis Superior in PNH patients with residual anemia despite prior anti-C5 treatment (APPLY) ■ Normalized hemoglobin levels ■ Reduced need for transfusions ■ Reduced patient-reported fatigue ■ Favorable safety with no serious breakthrough hemolysis Clinically meaningful Hb increases in treatment- naive patients (APPOINT) Safety profile consistent with APPLY-PNH Significant QoL benefits as the first oral monotherapy 23 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#24Global regulatory filings starting in H1 2023 Selected iptacopan PNH submissions FDA submission expected H1 2023 EMA submission expected H1 2023 PMDA submission expected H2 2023 Orphan Drug Designation FDA Apr. 2020, EMA Jul. 2020 Breakthrough Therapy Designation FDA Dec. 2020 24 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 China FDA submission expected H2 2023 NOVARTIS | Reimagining Medicine#25Participants David Soergel MD Global Head of Cardiovascular, Renal & Metabolism Development Reshema Kemps-Polanco Executive Vice President, Oncology US 25 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS Reimagining Medicine#26The path to PNH diagnosis and treatment involves many steps and can take months to years Delays in diagnosis and treatment of PNH Up to 3 years to diagnose PNH (avg. 7-9 months) ■ Median age at disease onset 36 years¹ ■ Common symptoms (e.g., fatigue, hemoglobinuria) can have multiple causes ■ "Watch & Wait" for disease progression before treatment is initiated ■ Patients still experiencing symptoms and may be receiving transfusions Increase urgency to intervene earlier Limited options available for treatment ■ Until 2021, only anti-C5's available for treatment ■ 4-6 weeks to determine if treatment is working ■ Some patients unwilling to commit to regular infusions More choice in first line and switch Need to make treatment for life manageable ☐ Regular monitoring with stable patients every 3 months but unstable patients as often as weekly ■ "Good enough" patient outcomes accepted Managed by hematologists Unburden patients from infusions and expect more from treatments 1. 5. Schrezenmeier H et al. Ann Hematol. 2020;99(7):1505-1514. Source: Patient journey market research 2022 26 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#27Opportunity to redefine PNH treatment paradigm ~6k Prevalent¹ PNH patients in US 30% ~400 Incident² PNH patients/year in US Treated with complement inhibitor³ Untreated 70% • ■ ■ Current market -USD 2bn WW (USD 1bn US)4 ■ Of C5-treated patients, -80% have Hb < 12g/dL5 Still experiencing symptoms - - Managing life around infusion schedule Some still receiving transfusions Varying views of when treatment should be started Heterogeneous presentation of symptoms Some unwilling to commit to regular infusions Some still receiving transfusions Displace Anti-C5 Potentially increase treatment rate Start appropriate patients on iptacopan 1. Prevalence: 12-18 per million individuals in the US (Jalbert JJ, 2019, Mon Pere N, 2018). 2. Incidence: 1.0-1.5 per million individuals (Hill A, 2017). 3. Treated with anti-C5 or anti-C3 4. Based on C5i therapies only 5. Debureaux PE et al. Bone Marrow Transplant 2021;56:2600-2 Source: Patient journey market research 2022 27 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#28Significant experience in non-malignant hematology and rare disease provides strong foundation for launch Track record of execution in rare hematology / oncology conditions → Promacta market leadership in SAA (ultra-rare) and ITP (rare) based on deep understanding of HCP insights and patient needs / motivations; also an oral option in originally infusion-driven market → Building on rare disease playbook from Vijoice and Afinitor TSC launches, including early and critical focus on patient engagement and advocacy Existing → ~2.5k hematologists / oncologists seeing PNH patients relationships with → PNH treaters Rare disease, but treated and managed not just by experts in large centers, but also community HCPs 28 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 → Strong existing customer relationships with majority of PNH treaters → Top medical experts engaged in either clinical studies or advisory capacity → Account profiling underway to identify individual success levers NOVARTIS | Reimagining Medicine#29Launch readiness to support rare disease success is on track Patient engagement and activation Relationships built with key patient advocacy groups ■ Focus on educating PNH patients on available therapies and activating them to seek treatment that's right for their life Disease state education Launched PNH disease education campaign at ASH 2022 to raise awareness of high burden of disease and unmet need Ongoing digital engagement Patient support services Detailed mapping of patient journey to minimize friction for new and switch patients Leveraging experience in Rare Disease, Oncology and MS to ensure best practice Comprehensive evidence generation Ph3 program covering broad spectrum of PNH patients: treatment naïve (APPOINT) and switch (APPLY) Patient registries with key stakeholder organizations Field medical and field sales teams already in place 29 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#30On track to launch a potential new standard-of-care in PNH 1 Iptacopan could be practice-changing: ✓ Superior efficacy to anti-C5 therapy ✓ Significant QoL benefits Oral option in infusion market Potential new standard of care 2 On track with launch readiness: Playbook for rare disease launches ✓ Existing relationships with PNH HCPS. Disease education campaign launched ☐ Medical and sales teams in place 30 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine#31YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAY AYY YYA YYA YAY AYYYYYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYAYYA YAYYAY YAYYA YAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYA AYYAY Q&A AYY YYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYAYYA YAYYAY YYAYYA YAYYAY YAYYA NOVARTIS | Reimagining Medicine#32YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAY AYY YYA YYA YAY AYYYYYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYAYYA YAYYAY YAYYA YAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYA AYYAY Appendix AYY YYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY AYYAYYAYYA YYAYYAYYAY YYAYYA YAYYAY YYAYYA YAYYAY YAYYA NOVARTIS | Reimagining Medicine#33Iptacopan has the potential to become the new SoC in a well established and growing global PNH market PNH complement inhibitors market size is estimated at ~USD 2bn¹ PNH market is expected to grow at 7.6% CAGR over the next 10 years in the 7 major markets² driven by new entrants and increased penetration of complement inhibitors³ Current total C5 inhibitor sales in PNH roughly evenly split between US and ex-US1 Significant opportunities ex-US, including China, where until recently there were no complement inhibitors available 1. Evaluate Pharma Dec 2022. 2. 7 Major Markets: US, Germany, France, UK, Italy, Spain and Japan. 3. Delveinsights PNH market report 2022. 33 NOVARTIS | Reimagining Medicine

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BioAtla Investor Presentation Deck

Healthcare