RegulationPriority Review (US), Breakthrough Therapy (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Priority Review (CN), Breakthrough Therapy (CN), Orphan Drug (AU)

Structure

Molecular FormulaC25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS Registry2447007-60-3

Clinical Trials associated with Iptacopan

NCT06388941 / Not yet recruitingPhase 2

A Randomized, Controlled Study to Evaluate LNP023 (Iptacopan) in Patients With Active ANCA-associated Vasculitis

The purpose of this study is to evaluate the efficacy and safety of iptacopan compared to standard of care (SOC) to induce and maintain remission in study participants with active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), when used in combination with rituximab (RTX) induction. The trial will also assess the impact of iptacopan on disease relapses, evolution of renal function and proteinuria, GC side effects, patients' immune status, and QoL.

Start Date28 Aug 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

NCT06517758 / RecruitingPhase 3

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis (gMG), Followed by an Open Label Extension Phase

The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.

Start Date31 Jul 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT05795140 / RecruitingPhase 3

A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS

This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.

Start Date08 May 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

100 Clinical Results associated with Iptacopan

100 Translational Medicine associated with Iptacopan

100 Patents (Medical) associated with Iptacopan

Literatures (Medical) associated with Iptacopan

01 Jan 2024·Kidney international

Results of a randomized double-blind placebo-controlled Phase 2 study propose iptacopan as an alternative complement pathway inhibitor for IgA nephropathy

Article

Author: Junge, Guido ; Milojevic, Julie ; Rizk, Dana V ; Meier, Matthias ; Nidamarthy, Prasanna Kumar ; Sprangers, Ben ; Perkovic, Vlado ; Kashihara, Naoki ; Trimarchi, Hernán ; Rovin, Brad ; Kollins, Dmitrij ; Papachristofi, Olympia ; Zhang, Hong ; Charney, Alan ; Maes, Bart ; Barratt, Jonathan

Targeting the alternative complement pathway is an attractive therapeutic strategy given its role in the pathogenesis of immunoglobulin A nephropathy (IgAN). Iptacopan (LNP023) is an oral, proximal alternative complement inhibitor that specifically binds to Factor B. Our randomized, double-blind, parallel-group adaptive Phase 2 study (NCT03373461) enrolled patients with biopsy-confirmed IgAN (within previous three years) with estimated glomerular filtration rates of 30 mL/min/1.73 m² and over and urine protein 0.75 g/24 hours and over on stable doses of renin angiotensin system inhibitors. Patients were randomized to four iptacopan doses (10, 50, 100, or 200 mg bid) or placebo for either a three-month (Part 1; 46 patients) or a six-month (Part 2; 66 patients) treatment period. The primary analysis evaluated the dose-response relationship of iptacopan versus placebo on 24-hour urine protein-to-creatinine ratio (UPCR) at three months. Other efficacy, safety and biomarker parameters were assessed. Baseline characteristics were generally well-balanced across treatment arms. There was a statistically significant dose-response effect, with 23% reduction in UPCR achieved with iptacopan 200 mg bid (80% confidence interval 8-34%) at three months. UPCR decreased further through six months in iptacopan 100 and 200 mg arms (from a mean of 1.3 g/g at baseline to 0.8 g/g at six months in the 200 mg arm). A sustained reduction in complement biomarker levels including plasma Bb, serum Wieslab, and urinary C5b-9 was observed. Iptacopan was well-tolerated, with no reports of deaths, treatment-related serious adverse events or bacterial infections, and led to strong inhibition of alternative complement pathway activity and persistent proteinuria reduction in patients with IgAN. Thus, our findings support further evaluation of iptacopan in the ongoing Phase 3 trial (APPLAUSE-IgAN; NCT04578834).

01 Jan 2024·Kidney international

A new alternative: inhibiting complement activation in patients with IgA nephropathy

Article

Author: Floege, Jürgen

Mesangial complement C3 deposits, reflecting alternative and possibly lectin pathway activation, are characteristic in biopsies of patients with IgA nephropathy (IgAN). A recent randomized controlled trial tested the efficacy and safety of iptacopan, a factor B inhibitor, in patients with IgAN. Iptacopan dose-dependently reduced proteinuria, and there was a pronounced decrease of urinary C5b-9. This offers the perspective of "personalizing" therapy, which would be a unique feature of this novel approach to IgAN. A phase III clinical trial (APPLAUSE-IgAN) is ongoing.

04 Dec 2023·Clinical kidney journal

Targeting complement in IgA nephropathy

Review

Author: Gutiérrez, Eduardo ; Caravaca-Fontán, Fernando ; Sevillano, Ángel M ; Praga, Manuel

ABSTRACT:

Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis worldwide. Recent years have witnessed significant improvements in the understanding of the pathogenesis of IgAN and particularly, the pathogenic role of complement activation. The alternative complement pathway is the major complement cascade activator in IgAN, and glomerular C3 deposition has been shown to correlate with disease progression. In addition, several studies have provided insight into the pathogenic role of factor H–related proteins -1 and -5 in IgAN, as independent players in complement dysregulation. The lectin pathway has also been shown to be associated with the severity of IgAN. Glomerular deposition of C4d has been associated with increased histologic disease activity, faster decline in estimated glomerular filtration rate and higher risk of kidney failure. On the other hand, although overlooked in the Oxford classification, numerous studies have shown that the coexistence of thrombotic microangiopathy in IgAN is a significant indicator of a poorer prognosis. All the breakthroughs in the understanding of the contributing role of complement in IgAN have paved the way for the development of new complement-targeted therapies in this disease. Several ongoing trials are evaluating the efficacy of new agents against factor B (iptacopan, Ionis-FB-LRX), C3 (pegcetacoplan), factor D (vemircopan, pelecopan), C5 (ravulizumab, cemdisiran) and C5a receptor 1 (avacopan). In this study, we provide a comprehensive review of the role of complement in IgAN, including the emerging mechanisms of complement activation and the promising potential of complement inhibitors as a viable treatment option for IgAN.

117

News (Medical) associated with Iptacopan

18 Jul 2024

·www.globenewswire.com

Novartis continues to deliver strong sales growth and core margin expansion in Q2; raises FY 2024 bottom-line guidance

Ad hoc announcement pursuant to Art. 53 LR Q2 net sales grew +11% (cc1, +9% USD) with core operating income up +19% (cc, +17% USD) Sales growth driven by continued strong performance from Entresto (+28% cc), Kesimpta (+65% cc), Cosentyx (+22% cc), Kisqali (+50% cc), Leqvio (+134% cc) and Pluvicto (+44% cc)Core operating income margin 39.6%, +270 basis points (cc), mainly driven by higher net sales Q2 operating income grew +47% (cc, +43% USD) and net income up +49% (cc, +43% USD)Q2 core EPS grew +21% (cc, +17% USD) to USD 1.97Q2 free cash flow1 of USD 4.6 billion (+40% USD) driven by higher net cash flows from operating activitiesH1 sales up +11% (cc, +9% USD) and core operating income up +21% (cc, +16% USD)Q2 selected innovation milestones: Fabhalta (iptacopan) received EU, Japan and China approval for the treatment of PNH Lutathera approved by FDA for pediatric GEP-NET patients (≥12 years) Scemblix filing for 1L CML accepted by FDA; received Breakthrough Therapy designationAtrasentan FDA filing accepted for the treatment of adult patients with IgANKisqali NATALEE updated data showed continued clinical benefit with median follow-up ~4 years Full-year 2024 guidance raised for core operating income based on strong momentum2 Net sales expected to grow high-single to low double-digit (unchanged)Core operating income expected to grow mid- to high teens (from low double-digit to mid-teens) Basel, July 18, 2024 – commenting on Q2 2024 results, Vas Narasimhan, CEO of Novartis, said: “Novartis delivered a strong Q2, with net sales up 11% and core operating income margin approaching 40%. Our performance reflects continued strong momentum of our key growth drivers, both in the US and ex-US, which has allowed us to upgrade our FY2024 guidance. We also advanced our pipeline in Q2, completing submissions to the FDA for Scemblix in first-line CML and atrasentan in IgAN, generating updated data in the NATALEE study to support the strong profile of Kisqali in eBC, and executing multiple deals to expand our pipeline in RLT and prostate cancer. We remain on track to achieve our mid-term sales growth (+5% cc CAGR 2023-2028) and margin (40%+ by 2027) guidance.” Key figuresContinuing operations3 Q2 2024Q2 2023% changeH1 2024H1 2023% change USD m USD mUSDcc USD m USD mUSDccNet sales12 51211 437911 24 34122 235911Operating income4 0142 8074347 7 3875 4253643Net income3 2462 2714349 5 9344 4213443EPS (USD)1.601.094752 2.912.123747Free cash flow4 6153 29240 6 6535 97611 Core operating income4 9534 2401719 9 4908 1461621Core net income4 0083 5021418 7 6896 7351419Core EPS (USD)1.971.691721 3.773.231722 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 43 of the Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. 2. Please see detailed guidance assumptions on page 7. 3. As defined on page 33 of the Interim Financial Report, Continuing operations include the retained business activities of Novartis, comprising the innovative medicines business and the continuing corporate activities and Discontinued operations include operational results from the Sandoz business. Strategy Our focus In 2023, Novartis completed its transformation into a “pure-play” innovative medicines business. We have a clear focus on four core therapeutic areas (cardiovascular-renal-metabolic, immunology, neuroscience and oncology), with multiple significant in-market and pipeline assets in each of these areas, that address high disease burden and have substantial growth potential. In addition to two established technology platforms (chemistry and biotherapeutics), three emerging platforms (gene & cell therapy, radioligand therapy and xRNA) are being prioritized for continued investment into new R&D capabilities and manufacturing scale. Geographically, we are focused on growing in our priority geographies – the US, China, Germany and Japan. Our priorities Accelerate growth: Renewed attention to deliver high-value medicines (NMEs) and focus on launch excellence, with a rich pipeline across our core therapeutic areas.Deliver returns: Continuing to embed operational excellence and deliver improved financials. Novartis remains disciplined and shareholder-focused in our approach to capital allocation, with substantial cash generation and a strong capital structure supporting continued flexibility. Strengthening foundations: Unleashing the power of our people, scaling data science and technology and continuing to build trust with society. Financials Following the September 15, 2023, shareholder approval of the spin-off of Sandoz, Novartis reported its consolidated financial statements as “continuing operations” and “discontinued operations.” Continuing operations include the retained business activities of Novartis, comprising the innovative medicines business and the continuing corporate activities. Discontinued operations include the Sandoz Division and selected portions of corporate activities attributable to Sandoz’s business, as well as certain expenses related to the spin-off. While the commentary below focuses on continuing operations, we also provide information on discontinued operations. Continuing operations Second quarter Net sales were USD 12.5 billion (+9%, +11% cc), with volume contributing 15 percentage points to growth. Generic competition had a negative impact of 2 percentage points and pricing had a negative impact of 2 percentage points. Operating income was USD 4.0 billion (+43%, +47% cc), mainly driven by higher net sales and lower impairments, partly offset by higher R&D investments. Net income was USD 3.2 billion (+43%, +49% cc), mainly driven by higher operating income. EPS was USD 1.60 (+47%, +52% cc), benefiting from the lower weighted average number of shares outstanding. Core operating income was USD 5.0 billion (+17%, +19% cc), mainly driven by higher net sales, partly offset by higher R&D investments. Core operating income margin was 39.6% of net sales, increasing 2.5 percentage points (+2.7 percentage points cc). Core net income was USD 4.0 billion (+14%, +18% cc), mainly due to higher core operating income. Core EPS was USD 1.97 (+17%, +21% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow from continuing operations amounted to USD 4.6 billion (+40% USD), compared with USD 3.3 billion in the prior-year quarter, driven by higher net cash flows from operating activities from continuing operations. First half Net sales were USD 24.3 billion (+9%, +11% cc), with volume contributing 15 percentage points to growth. Generic competition had a negative impact of 2 percentage points and pricing had negative impact of 2 percentage points. Operating income was USD 7.4 billion (+36%, +43% cc), mainly driven by higher net sales and lower impairments and restructuring charges, partly offset by a prior-year one-time income from legal matters. Net income was USD 5.9 billion (+34%, +43% cc), mainly driven by higher operating income. EPS was USD 2.91 (+37%, +47% cc), benefiting from the lower weighted average number of shares outstanding. Core operating income was USD 9.5 billion (+16%, +21% cc), mainly driven by higher net sales, partly offset by higher R&D investments. Core operating income margin was 39.0% of net sales, increasing 2.4 percentage points (+3.1 percentage points cc). Core net income was USD 7.7 billion (+14%, +19% cc), mainly due to higher core operating income. Core EPS was USD 3.77 (+17%, +22% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow from continuing operations amounted to USD 6.7 billion (+11% USD), compared with USD 6.0 billion in the prior-year period, driven by higher net cash flows from operating activities from continuing operations. Discontinued operations Discontinued operations include the Sandoz generic pharmaceuticals and biosimilars division, certain corporate activities attributable to Sandoz and certain other expenses related to the spin-off of the Sandoz business. Second quarter As the Sandoz spin-off was completed on October 3, 2023, there were no operating results in the second quarter of 2024 related to discontinued operations. In the second quarter of 2023, discontinued operations net sales were USD 2.4 billion, operating income amounted to USD 113 million and net income from discontinued operations was USD 46 million. For further details see Note 3 “Significant acquisition of businesses and spin-off of Sandoz business” and Note 11 “Discontinued operations” to the condensed interim consolidated financial statements. First half As the Sandoz spin-off was completed on October 3, 2023, there were no operating results in the first half 2024 related to discontinued operations. In the first half 2023, discontinued operations net sales were USD 5.0 billion, operating income amounted to USD 351 million and net income from discontinued operations was USD 190 million. For further details see Note 3 “Significant acquisition of businesses and spin-off of Sandoz business” and Note 11 “Discontinued operations” to the condensed interim consolidated financial statements. Total Company Second quarter Total Company net income was USD 3.2 billion in 2024, compared to USD 2.3 billion in 2023 and basic EPS was USD 1.60 compared to USD 1.11 in prior year quarter. Net cash flows from operating activities for total Company amounted to USD 4.9 billion and free cash flow amounted to USD 4.6 billion. First half Total Company net income was USD 5.9 billion in 2024, compared to USD 4.6 billion in 2023 and basic EPS was USD 2.91 compared to USD 2.20 in prior year. Net cash flows from operating activities for total Company amounted to USD 7.1 billion and free cash flow amounted to USD 6.7 billion. Q2 key growth drivers Underpinning our financial results in the quarter is a continued focus on key growth drivers (ranked in order of contribution to Q2 growth) including: Entresto(USD 1 898 million, +28% cc) sustained robust demand-led growth, with increased penetration in the US and Europe following guideline-directed medical therapy in heart failure, as well as in China with increased penetration in hypertensionKesimpta(USD 799 million, +65% cc) sales grew across all regions reflecting increased demand and strong access for a high efficacy product with convenient self-administered dosingCosentyx(USD 1 526 million, +22% cc) sales grew mainly in the US, driven by recent launches (including the HS indication and the IV formulation in the US) in addition to volume growth in core indicationsKisqali(USD 717 million, +50% cc) sales grew strongly across all regions, based on increasing recognition of its overall survival benefit in HR+/HER2- advanced breast cancer and Category 1 NCCN guidelines recommendation Leqvio(USD 182 million, +134% cc) continued to show steady growth, with a focus on increasing account and patient adoption, growing customer confidence in acquisition and access, and continuing medical educationPluvicto(USD 345 million, +44% cc) grew in the US and Europe. With supply now unconstrained, the focus is on increasing share in established RLT sites, opening new sites and referral pathways, and initiating new patientsXolair(USD 427 million, +22% cc) growth was driven mainly by emerging growth markets and Europe Ilaris(USD 368 million, +20% cc) sales grew across all regions, mainly US and EuropeScemblix(USD 164 million, +56% cc) sales grew across all regions, demonstrating the high unmet need in later lines of CML Jakavi(USD 471 million, +13% cc) sales grew across all regions, with strong demand in both myelofibrosis and polycythemia vera indicationsTafinlar + Mekinist(USD 523 million, +9% cc) sales grew in all regions, led by emerging growth markets Lutathera(USD 175 million, +17% cc) sales grew across all regions due to increased demand, following the presentation of NETTER-2 results in 1L GEP-NET Fabhalta(USD 22 million) continued to show encouraging early launch indicators in the US, as the first oral monotherapy approved for PNH patientsEmerging Growth Markets*Grew +16% (cc) overall. China grew +27% (cc) to USD 1.1 billion, mainly driven by Entresto and Xolair *All markets except the US, Canada, Western Europe, Japan, Australia, and New Zealand Net sales of the top 20 brands in Q2 2024 Q2 2024 % changeH1 2024 % change USD mUSDccUSD mUSD ccEntresto1 89825283 7773032Cosentyx1 52620222 8522123Kesimpta79963651 4366466Kisqali71745501 3444852Promacta/Revolade544-7-51 064-6-4Tafinlar + Mekinist5235999757Jakavi4718139491215Tasigna446-6-4841-10-9Xolair42718228261518Ilaris36816207241217Sandostatin Group313-5-466813Pluvicto34544446554545Zolgensma349121464446Lucentis275-30-28589-27-26Exforge Group178-3137003Lutathera17517173441516Leqvio182133134333135137Gilenya138-49-47313-38-36Scemblix16455563006567Diovan Group16039300-41Top 20 brands total9 998151819 3261618 R&D update - key developments from the second quarter New approvals Fabhalta(iptacopan)EU, Japan and China approval for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).Lutathera(lutetium Lu 177 dotatate)FDA approval for the treatment of pediatric patients (≥12 years) with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Regulatory updates Scemblix(asciminib)FDA granted Breakthrough Therapy designation to Scemblix for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). FDA submission for first-line CML is completed and under Real-Time Oncology Review.AtrasentanFDA filing accepted for the treatment of adult patients with IgA nephropathy (IgAN).Lutathera(lutetium Lu 177 dotatate)EU filing accepted for the treatment of newly diagnosed, unresectable or metastatic, well-differentiated (G2 and G3), somatostatin receptor-positive GEP-NETs in adults. Results from ongoing trials and other highlights Scemblix(asciminib)In the Phase III ASC4FIRST study, Scemblix demonstrated superior major molecular response rates at week 48 vs investigator-selected standard-of-care tyrosine kinase inhibitors (TKIs) (67.7% vs 49.0%) and vs imatinib alone (69.3% vs 40.2%) in adults with newly diagnosed Ph+ CML-CP. Scemblix also demonstrated a favorable safety and tolerability profile. These results have been submitted to the FDA under Real-Time Oncology Review. Data presented at ASCO and EHA 2024 and published in the New England Journal of Medicine. Kisqali(ribociclib)New analyses following the end of Kisqali treatment for all patients in the Phase III NATALEE study in HR+/HER2- early breast cancer showed a continued clinically meaningful benefit with a consistent safety profile. Results to be presented at an upcoming medical meeting. In addition, a subgroup analysis from the NATALEE study at the time of final iDFS data cut-off showed the addition of Kisqali to endocrine therapy in patients with high-risk node-negative (N0) disease resulted in a 28% risk reduction in iDFS. The efficacy, safety and tolerability profile observed in the high-risk N0 subgroup is consistent with the overall NATALEE study population. Data presented at ASCO 2024.Fabhalta(iptacopan)Phase III APPEAR-C3G data showed a 35.1% proteinuria reduction vs placebo at 6 months for C3G patients treated with Fabhalta in addition to supportive care. Secondary endpoint data for estimated glomerular filtration rate showed numerical improvement over 6 months vs placebo. The study also showed Fabhalta has a favorable safety profile with no new safety signals. Submissions to the FDA and EMA for the adult C3 glomerulopathy indication are planned for H2 2024. Data presented at ERA 2024. Phase III APPLAUSE-IgAN data showed a 38.3% proteinuria reduction at nine months vs placebo for patients with IgAN. Fabhalta was well tolerated with a favorable safety profile consistent with previously reported data. Data presented at WCN 2024.AtrasentanResults from a pre-specified interim analysis of Phase III ALIGN data showed patients treated with atrasentan, in addition to supportive care with a renin-angiotensin system inhibitor, achieved a statistically significant 36.1% reduction in proteinuria vs placebo on top of supportive care at 36 weeks. Results presented at ERA 2024.RemibrutinibPhase III REMIX-1 and REMIX-2 data showed sustained efficacy and long-term safety of oral remibrutinib in chronic spontaneous urticaria (CSU) patients, with improvements in weekly urticaria activity scores observed as early as week 1 and sustained to week 52. Across both studies, remibrutinib demonstrated a favorable and consistent safety profile up to one year, including balanced liver function tests vs placebo. Novartis plans to submit remibrutinib for regulatory approval in 2025. Data presented at EAACI 2024.Coartem (artemether- lumefantrine)Phase II/III CALINA study data demonstrated that an optimized dose of Coartem developed for babies weighing <5kg with malaria has the required pharmacokinetic profile and good efficacy and safety. Data presented at the Multilateral Initiative on Malaria Pan-African Malaria Conference 2024.Deals In line with our strategic focus on oncology, Novartis acquired >90% of the total share capital of MorphoSys AG, adding to our pipeline pelabresib, a late-stage investigational BET inhibitor for myelofibrosis, and tulmimetostat, an early-stage investigational dual inhibitor of EZH2 and EZH1 for solid tumors or lymphomas. Novartis acquired Mariana Oncology, a biotech company focused on developing novel radioligand therapies (RLTs) across a range of solid tumors. The acquisition brings a robust portfolio of RLT programs, including MC-339, an actinium-based RLT being investigated in small cell lung cancer. Novartis expanded its peptide discovery collaboration with PeptiDream. Under the multi-program agreement, PeptiDream will identify and optimize novel macrocyclic peptides against targets selected by Novartis, for potential application in RLT. Novartis signed an exclusive strategic license agreement with Arvinas for the worldwide development and commercialization of ARV-766, a second generation PROTAC® androgen receptor (AR) degrader, complementing our RLT platform in prostate cancer. Capital structure and net debt Retaining a good balance between investment in the business, a strong capital structure and attractive shareholder returns remains a priority. During the first half of 2024, Novartis repurchased a total of 26.7 million shares for USD 2.7 billion on the SIX Swiss Exchange second trading line. These purchases included 25.9 million shares (USD 2.6 billion) under the up-to USD 15 billion share buyback announced in July 2023 (with up to USD 10.1 billion still to be executed). In addition, 0.8 million shares (USD 0.1 billion) were repurchased to mitigate dilution related to participation plans of associates, with the remainder of repurchases for this purpose to be executed in H2 2024. Further, 1.1 million shares (for an equity value of USD 0.1 billion) were repurchased from associates. In the same period, 8.4 million shares (for an equity value of USD 0.5 billion) were delivered as a result of share deliveries related to participation plans of associates. Consequently, the total number of shares outstanding decreased by 19.4 million versus December 31, 2023. These treasury share transactions resulted in an equity decrease of USD 2.3 billion and a net cash outflow of USD 2.7 billion. As of June 30, 2024, net debt increased to USD 18.8 billion compared to USD 10.2 billion net debt at December 31, 2023. The increase was mainly due to the USD 7.6 billion annual dividend payment, net cash outflow for M&A / intangible assets transactions of USD 5.0 billion and cash outflow for treasury share transactions of USD 2.7 billion, partially offset by USD 6.7 billion free cash flow. As of Q2 2024, the long-term credit rating for the company is Aa3 with Moody’s Ratings and AA- with S&P Global Ratings. 2024 outlook Barring unforeseen events; growth vs prior year in ccPrevious guidanceNet salesExpected to grow high single to low double-digit(unchanged)Core operating incomeExpected to grow mid- to high teens(from low double-digit to mid-teens) Key assumptions: Our guidance assumes that no Entresto generics and no Promacta generics launch in the US in 2024 Foreign exchange impact If mid-July exchange rates prevail for the remainder of 2024, the foreign exchange impact for the year would be negative 2 to negative 1 percentage points on net sales and negative 3 percentage points on core operating income. The estimated impact of exchange rates on our results is provided monthly on our website. 2025 Annual General Meeting Nomination for election to the Board of Directors The Novartis Board of Directors announced today that it is nominating Elizabeth M. McNally, MD, PhD, for election to the Board. Dr. McNally is Director of the Center for Genetic Medicine at Northwestern University, Feinberg School of Medicine, and as a practicing cardiologist and renowned research leader specializing in the genetics of cardiovascular and neuromuscular disorders, her clinical and scientific expertise will add greatly to the Novartis Board of Directors. Dr. McNally completed her MD and PhD at the Albert Einstein College of Medicine, and trained in Internal Medicine and Cardiology at the Brigham and Women’s Hospital at Harvard Medical School. She is a member of the National Academy of Medicine, serves on the Board of the Muscular Dystrophy Association, and is also the Founder and CEO of Ikaika Therapeutics. Board of Directors announcements The Board also noted Charles L. Sawyers and William T. Winters will not stand for re-election at the AGM 2025 in accordance with the 12-year term limit. The Board of Directors and the Executive Committee of Novartis thank them for their outstanding contributions and many years of distinguished service. Key figures1 Continuing operations2Q2 2024Q2 2023% change H1 2024H1 2023% change USD m USD mUSDcc USD m USD mUSDccNet sales12 51211 437911 24 34122 235911Operating income4 0142 8074347 7 3875 4253643As a % of sales32.124.5 30.324.4 Net income3 2462 2714349 5 9344 4213443EPS (USD)1.601.094752 2.912.123747Cash flows from operating activities4 8753 51739 7 1406 36912 Non-IFRS measures Free cash flow 4 6153 29240 6 6535 97611 Core operating income4 9534 2401719 9 4908 1461621As a % of sales39.637.1 39.036.6 Core net income4 0083 5021418 7 6896 7351419Core EPS (USD)1.971.691721 3.773.231722 Discontinued operations2Q2 2024Q2 2023% change H1 2024H1 2023% change USD m USD mUSDcc USD m USD mUSDccNet sales 2 449nmnm 4 952nmnmOperating income 113nmnm 351nmnmAs a % of sales 4.6 7.1 Net income 46nmnm 190nmnmNon-IFRS measures Core operating income 428nmnm 935nmnmAs a % of sales 17.5 18.9 Total CompanyQ2 2024Q2 2023% change H1 2024H1 2023% change USD m USD mUSDcc USD m USD mUSDccNet income3 2462 317nmnm 5 9344 611nmnmEPS (USD)1.601.11nmnm 2.912.20nmnmCash flows from operating activities4 8753 576nmnm 7 1406 533nmnmNon-IFRS measures Free cash flow 4 6153 275nmnm 6 6535 995nmnmCore net income4 0083 811nmnm 7 6897 425nmnmCore EPS (USD)1.971.83nmnm 3.773.54nmnm nm=not meaningful 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 43 of the Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year.2. As defined on page 33 of the Interim Financial Report, Continuing operations include the retained business activities of Novartis, comprising the innovative medicines business and the continuing corporate activities and Discontinued operations include operational results from the Sandoz business. Detailed financial results accompanying this press release are included in the Interim Financial Report at the link below:https://ml-eu.globenewswire.com/resource/download/a9652d15-40e9-4a4b-b225-32014f48e99e/ DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “may,” “will,” “continue,” “ongoing,” “grow,” “launch,” “expect,” “deliver,” “focus,” “address,” “accelerate,” “deliver,” “remain,” “scaling,” “guidance,” “outlook,” “long-term,” “priority,” “potential,” “can,” “trajectory” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding results of ongoing clinical trials; or regarding potential future, pending or announced transactions, including completion of the acquisition of MorphoSys AG; regarding potential future sales or earnings; or by discussions of strategy, plans, expectations or intentions, including discussions regarding our continued investment into new R&D capabilities and manufacturing; or regarding our capital structure; or regarding the consequences of the spin-off of Sandoz and our transformation into a “pure-play” innovative medicines company. Such forward-looking statements are based on the current beliefs and expectations of management 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. You should not place undue reliance on these statements. There can be no guarantee that the investigational or approved products described in this press release 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 such products will be commercially successful in the future. Neither can there be any guarantee expected benefits or synergies from the transactions described in this press release will be achieved in the expected timeframe, or at all. In particular, our expectations could be affected by, among other things: uncertainties regarding the success of key products, commercial priorities and strategy; uncertainties in the research and development of new products, including clinical trial results and additional analysis of existing clinical data; uncertainties regarding the use of new and disruptive technologies, including artificial intelligence; global trends toward healthcare cost containment, including ongoing government, payer and general public pricing and reimbursement pressures and requirements for increased pricing transparency; uncertainties regarding our ability to realize the strategic benefits, operational efficiencies or opportunities expected from our external business opportunities; our ability to realize the intended benefits of our separation of Sandoz into a new publicly traded standalone company; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products; uncertainties in the development or adoption of potentially transformational digital technologies and business models; uncertainties surrounding the implementation of our new IT projects and systems; uncertainties regarding potential significant breaches of information security or disruptions of our information technology systems; uncertainties regarding actual or potential legal proceedings, including regulatory actions or delays or government regulation related to the products and pipeline products described in this press release; safety, quality, data integrity, or manufacturing issues; our performance on and ability to comply with environmental, social and governance measures and requirements; major political, macroeconomic and business developments, including impact of the war in certain parts of the world; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; and other risks and factors referred to in Novartis AG’s most recently filed Form 20-F and in subsequent reports filed with, or furnished to, 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 as a result of new information, future events or otherwise. All product names appearing in italics are trademarks owned by or licensed to Novartis. This communication is neither an offer to purchase nor a solicitation of an offer to sell shares of MorphoSys. The final terms and further provisions regarding the delisting purchase offer are available in the offer document published by Novartis BidCo AG (formerly known as Novartis data42 AG) (the “Bidder”). The offer document has been approved by the BaFin and has been filed with the U.S. Securities and Exchange Commission (the “SEC”). The solicitation and offer to buy shares of MorphoSys is only being made pursuant the offer document. In connection with the Offer, the Bidder and Novartis AG have filed Tender Offer Statement on Schedule TO with the SEC (together with the offer document, an Offer to Purchase including the means to tender and other related documents, the “Offer Documents”), the management board and supervisory board of MorphoSys have issued a joint reasoned statement in accordance with sec. 27 of the German Securities Acquisition and Takeover Act and MorphoSys has filed a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC (together with the joint reasoned statement, the “Recommendation Statements”). THE MORPHOSYS SHAREHOLDERS AND OTHER INVESTORS ARE URGED TO READ THE OFFER DOCUMENTS AND THE RECOMMENDATION STATEMENTS BECAUSE THEY CONTAIN IMPORTANT INFORMATION WHICH SHOULD BE READ CAREFULLY BEFORE ANY DECISION IS MADE WITH RESPECT TO THE OFFER. The Offer Documents and the Recommendation Statements have been distributed to all stockholders of MorphoSys in accordance with German and U.S. securities laws. The Tender Offer Statement on Schedule TO and the Solicitation/Recommendation Statement on Schedule 14D-9 are available for free at the SEC’s website at www.sec.gov. Additional copies may be obtained for free by contacting the Bidder or MorphoSys. Free copies of these materials and certain other offering documents are available on the Bidder’s website at www.novartis.com/investors/morphosys-acquisition or by contacting the Bidder’s investor relations department at +41 61 324 7944. 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 LinkedIn, Facebook, X/Twitter and Instagram. Novartis will conduct a conference call with investors to discuss this news release today at 14:00 Central European time and 8:00 Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar. Detailed financial results accompanying this press release are included in the condensed interim financial report at the link below. Additional information is provided on our business and pipeline of selected compounds in late-stage development. A copy of today's earnings call presentation can be found at https://www.novartis.com/investors/event-calendar. Important dates October 29, 2024 November 20-21, 2024 January 31, 2025 Third quarter & nine months 2024 resultsMeet Novartis Management 2024 (London, UK)Fourth quarter & full year 2024 results Please find full media release in English attached and on the following link:Media Release (PDF) Further language versions are available through the following links: German version is available through the following link:Medienmitteilung (PDF) French version is available through the following link:Communiqué aux médias (PDF)

Clinical ResultDrug ApprovalPhase 3Breakthrough TherapyFinancial Statement

28 May 2024

·www.fiercepharma.com

Novartis rides IgAN trioka to 2 trial wins in rare kidney diseases

Novartis is developing three drugs against the kidney disease IgAN. In recent days, the company shared updates on two of them. Novartis is eyeing an FDA filing for a second drug in immunoglobulin A nephropathy (IgAN), as the Swiss pharma goes all-in on the rare autoimmune kidney disease from three different angles. Novartis plans to submit atrasentan to the FDA by the end of June for a potential accelerated approval in IgAN based on positive data from the phase 3 ALIGN trial. In the study, atrasentan led to a significant 36.1% relative reduction in proteinuria compared with placebo after 36 weeks of treatment, according to results presented at the European Renal Association Congress. Atrasentan could be a potential competitor to Travere Therapeutics’ rival drug Filspari (sparsentan). But as analysts from William Blair and Leerink Partners suggested, the Novartis med does not seem to have the upper hand. An IgAN showdown Heading into atrasentan’s 36-week readout, investors were expecting to see at least a 40% placebo-adjusted urine protein reduction because of earlier readouts based on small patient populations and shorter treatment periods, Leerink analysts pointed out in a Monday note. The Novartis drug pulled off a 38.1% reduction in proteinuria, as measured by urine protein to creatinine ratio (UPCR), versus 3.1% for placebo. Both groups also received standard supportive care. By comparison, in the phase 3 PROTECT trial, Travere’s Filspari was associated with an average reduction in proteinuria of 49.8% after 36 weeks, compared with 15.1% for off-label irbesartan. This translates into a 41% relative reduction for Filspari. The two drugs’ trials enrolled roughly similar populations, with some slight differences such as baseline UPCR that are not likely to significantly change the outcome or interpretation of results, William Blair analysts wrote in a Tuesday note. However, a notable difference in the percentage of Asian patients might be a confounding factor for a cross-trial comparison, the team added. Despite the differences between the trials, atrasentan does not appear to be differentiated versus Filspari, Leerink’s analysts said. A recent Leerink survey suggested that doctors would split their use of Filspari and atrasentan equally. Travere’s Filspari may have two advantages. First of all, Filspari already boasts an accelerated approval in IgAN since February 2023, and Travere has already filed for a full approval with a target decision date in September. That gives Filspari a roughly two-year head start against Novartis’ atrasentan, the William Blair team noted. Secondly, Filspari could reduce the number of pills patients have to take, which is an important consideration for many with IgAN, Leerink analysts noted. Filspari is a dual endothelin angiotensin receptor antagonist, while atrasentan inhibits the endothelin A receptor alone. Filspari’s mechanism eliminates the need for an angiotensin receptor blocker such as irbesartan, which is part of the standard of care for IgAN. However, having separate pills allows doctors to adjust the dose of each therapy for a patient, the Leerink team added. In the end, “choosing between the two will likely come down to a case-by-case decision, as well as which therapy is covered by payers/has an easier path to authorization,” the Leerink analysts said. Filspari’s early launch has been progressing nicely, with about 2,000 patient start forms received by the end of March. The drug missed statistical significance on its confirmatory trial endpoint—change in estimated glomerular filtration rate (eGFR) slope, a measure of kidney function—in the PROTECT trial. But Travere believes the results observed in the study could convince the FDA and doctors of its benefits. The confirmatory evidence flop leaves a window open for Novartis’ atrasentan to potentially differentiate itself with an eGFR win. Atrasentan’s ALIGN study remains ongoing and blinded toward its final eGFR analysis, which is expected in 2026. Plus, Filspari is still only penetrating a very small corner of the IgAN at this stage, and there remains significant room for a competitor, Willima Blair analysts aid. IgAN troika Atrasentan, which Novartis believes carries blockbuster potential, is one of the three drugs that the company is advancing in IgAN. Fabhalta, an oral factor B inhibitor, recently showed a 38.3% relative proteinuria reduction versus placebo in the phase 3 APPLAUSE-IgAN trial. The drug got its initial FDA approval in December for paroxysmal nocturnal hemoglobinuria (PNH), and Novartis has filed the IgAN indication with a decision now under priority review. At the ERA Congress, Novartis also shared phase 3 results for Fabhalta in an ultra-rare kidney disease called C3 glomerulopathy. In the APPEAR-C3G study, patients who got Fabhalta achieved a 35.1% reduction in proteinuria compared with placebo after six months of treatment. Novartis plans to file for the indication based on that surrogate endpoint in the second half of this year. At this point, on the eGFR endpoint, Fabhalta showed a numerical improvement of 2.2mL/min/1.73m2. Novartis has estimated Fabhalta could reach over $3 billion in peak sales across PNH, IgAN and C3G. The IgAN candidates target the kidney disease differently, and their ultimate positioning, including sequencing or combinations, will partly be defined by the data they generate, Shreeram Aradhye, M.D., said in a January interview with Fierce Pharma. With Fabhalta, Novartis is targeting the alternative complement pathway. Overactivation of the pathway and deposition of the complement protein could cause kidney damage. So Fabhalta is intervening through that pathogenic mechanism of the injury, Aradhye explained. Atrasentan, by targeting the endothelin receptor, inhibits excess proliferation and inflammatory signaling in mesangial cells, which are involved in IgAN. The third agent, zigakibart, is a subcutaneous anti-APRIL antibody. Aradhye said the drug has the potential to be disease-modifying because it intervenes at an early stage when abnormal IgA is formed. Its phase 3 IgAN readout is expected in 2026, and Novartis also has blockbuster hopes for that drug.

Clinical ResultPhase 3Drug ApprovalAccelerated ApprovalPriority Review

28 May 2024

·www.biopharmadive.com

Roche’s Sabry retires; Duchenne drug fails confirmatory test

BioPharma Dive is testing out a new format rounding up smaller updates from around the industry. Have thoughts on what could make this type of story better? Drop us a line!Today, a brief rundown of news from Merck & Co. and biotechnology startup Cartography Biosciences, as well as updates from Roche, Novartis, Novo Nordisk and Nippon Shinyaku that you may have missed over the holiday weekend.Merck & Co.s drug Keytruda helped people with a hard-to-treat form of breast cancer live longer when used first as a neoadjuvant, or preoperative, therapy, and then as an adjuvant therapy. The study, called Keynote-522, compared Keytruda- and chemotherapy-based regimens to neoadjuvant chemo alone in early-stage triple-negative breast cancer thats considered high risk. Merck, which won U.S. approval for the regimen in July 2021 based on earlier data, said Tuesday the improvement in survival was statistically significant and clinically meaningful. Ned PagliaruloCartography Biosciences will receive $20 million from Gilead Sciences in a research collaboration to discover drug targets for cancers of the breast and lungs. Cartography, which raised $57 million in a Series A venture financing two years ago, touts a pair of computational and genomics platforms that it says can map novel tumor-selective antigens. Ned PagliaruloJames Sabry, who has led pharma partnering for Roche, is retiring after fourteen years with the Swiss drugmaker, the company said Monday. He will be replaced by Boris Zatra, who will on July 1 become head of group business development, a new role that combines several dealmaking functions.Zatra will report to Roche CEO Thomas Schinecker as part of an enlarged executive committee. Ned PagliaruloPreliminary results from a confirmatory clinical trial of Nippon Shinyakus Viltepso for Duchenne muscular dystrophy showed treatment didnt lead to a meaningful benefit over a placebo. The study, which enrolled 77 boys with Duchenne who could still walk, measured the time it took participants to stand from lying down. After 48 weeks, both groups showed a trend of increased velocity from baseline, with no statistically significant difference between them. The study was meant to confirm Viltepsos accelerated approval, which the Food and Drug Administration granted in 2020. Nippon Shinyaku said it is conducting further analyses of the results and working closely with regulators. Ned PagliaruloNovartis on Monday announced positive Phase 3 results for two kidney disease drugs its developing. One, called atrasentan and acquired through a buyout of Chinook Therapeutics, significantly reduced protein in urine compared to a placebo among people with a rare condition known as IgA nephropathy. The other, an approved medicine Novartis sells as Fabhalta, also lowered proteinuria versus placebo in treating C3 glomerulopathy, an even less common disease. Proteinuria reduction is considered a surrogate marker of benefit in certain kidney conditions, and Novartis plans to submit both drugs for regulatory approvals in IgA nephropathy and C3 glomerulopathy, respectively. Ned PagliaruloA panel of FDA advisers voted 7-4 against recommending approval of a weekly insulin injection from Novo Nordisk in people with Type 1 diabetes, according to Reuters. The shot, called insulin icodec, could offer a more convenient alternative than current daily insulin injections. But FDA staff flagged a higher rate of hypoglycemia in participants given Novos experimental drug in testing, compared to those who received a comparator treatment. The agency usually, though not always,follows its advisers recommendations. In Europe, regulators recently authorized insulin icodec for sale there. Ned Pagliarulo '

Clinical ResultPhase 3Drug ApprovalExecutive Change

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Approved

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Indication	Country/Location	Organization	Date
Hemoglobinuria, Paroxysmal	US	Novartis Pharmaceuticals Corp.	05 Dec 2023

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Glomerulonephritis, IGA	NDA/BLA	US	Novartis AG	15 Apr 2024
Myasthenia Gravis	Phase 3	US	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	GR	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Glomerulonephritis, Membranoproliferative	Phase 3	-	Novartis Pharmaceuticals Corp.	06 Mar 2023
Atypical Hemolytic Uremic Syndrome	Phase 3	US	Novartis Pharmaceuticals Corp.	17 Jan 2022
Atypical Hemolytic Uremic Syndrome	Phase 3	CN	Novartis Pharmaceuticals Corp.	17 Jan 2022
Atypical Hemolytic Uremic Syndrome	Phase 3	JP	Novartis Pharmaceuticals Corp.	17 Jan 2022
Atypical Hemolytic Uremic Syndrome	Phase 3	JP	Novartis Pharma Produktions GmbH	17 Jan 2022
Atypical Hemolytic Uremic Syndrome	Phase 3	JP	China Novartis Institutes for BioMedical Research Co., Ltd.	17 Jan 2022
Atypical Hemolytic Uremic Syndrome	Phase 3	JP	Novartis Pharma AG	17 Jan 2022

Clinical Result

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Phase

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04154787 (CTgov)	Phase 2	Idiopathic Membranous Glomerulonephritis	37	Rituximab	nksocvvbwk(pmwoouicse) = fivcjabvxm bhcnthhhtc (lpaobypcbb, fcukefrcma - bljhkqhdkf) View more	-	05 Jul 2024
NCT04817618 (APPEAR-C3G, PRNewswire) Manual	Phase 3	C3 glomerulopathy	-	Fabhalta (iptacopan)	gszsobrhhp(jofyevypup) = qzruophtri ziqttbxnyi (ihsetmspkw )	Positive	25 May 2024
				Placebo	-
NCT04558918 (APPLY-PNH, EHA2024)	Phase 3	Hemoglobinuria, Paroxysmal C3 fragment deposition	95	Iptacopan 200 mg twice daily	trpzsptqgr(ftboeuxfkd) = qfzkoxujtb miwpjkyijo (vwmuxkevhj, -16.1%) View more	Positive	14 May 2024
				Anti-C5 inhibitors	trpzsptqgr(ftboeuxfkd) = lzaynxxyti miwpjkyijo (vwmuxkevhj, -16.9%) View more
NCT04747613 (APPOINT-PNH \| APPLY-PNH \| roll-over extension, EHA2024)	Phase 3	Hemoglobinuria, Paroxysmal	61	Iptacopan	bprqfhjpij(opdzgygcwc) = dcqzhrhdep uyarabxffh (csclotpwpt )	Positive	14 May 2024
NCT04578834 (APPLAUSE-IgAN, PRNewswire) Manual	Phase 3	Glomerulonephritis, IGA	443	Fabhalta	fhimzxpjmn(ouzlgzynpk) = nhkxxjfzqo cwwsfasaxj (crxvjqnycw )	Positive	15 Apr 2024
NCT04578834 (APPLAUSE-IgAN, ISN WCN2024)	Phase 3	Glomerulonephritis, IGA proteinuria \| eGFR	443	Iptacopan 200 mg	fmnfbdnejw(wmuwujbbvw) = fhysuzuazr vrujfcocco (siortuwjbd, 1.4 - 2.7)	Positive	01 Apr 2024
				Placebo	fmnfbdnejw(wmuwujbbvw) = kjuzdhzyym vrujfcocco (siortuwjbd, 1.5 - 2.8)
Pubmed	Phase 3	Hemoglobinuria, Paroxysmal lactate dehydrogenase (LDH)	-	Iptacopan	cnyuzukorw(mddykzyrsd) = Headache was the most frequent adverse event with iptacopan gikbwmtdhu (llpggnzwpa ) View more	-	14 Mar 2024
				Placebo
NCT03439839 (CLNP023X2201, CTgov)	Phase 2	Hemolysis \| Hemoglobinuria, Paroxysmal	16	Standard of Care+iptacopan (Cohort 1: LNP023 200mg Bid + SoC)	mbhhevequz(lgzgcblkrm) = tkkyjxooqd vssltkccxh (kkutkfqccb, mqhattnbkt - azwtsfpakk) View more	-	03 Jan 2024
				Standard of Care+iptacopan (Cohort 2: LNP023 50mg/200mg Bid + SoC)	mbhhevequz(lgzgcblkrm) = pomburzrsp vssltkccxh (kkutkfqccb, eemtonpoff - ycmyiovgau) View more
NCT04817618 (APPEAR-C3G, GlobeNewswire) Manual	Phase 3	C3 glomerulopathy	-	iptacopan	yhfoqdmbum(yfbhotoxgj) = The study met its primary endpoint, with iptacopan (200 mg twice daily) demonstrating superiority compared to placebo in providing clinically meaningful and statistically significant proteinuria (protein in urine) reduction on top of background therapy at six months. ytgnmjtorq (awtgjuhxjo )	Positive	11 Dec 2023
				Placebo

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