RegulationPriority Review (United States), Breakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Rare Pediatric Disease (United States), Conditional marketing approval (China)

Structure/Sequence

Molecular FormulaC25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS Registry2447007-60-3

Clinical Trials associated with Iptacopan Hydrochloride

CTIS2026-525677-39-00

/ Not yet recruitingPhase 3

An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.

Start Date25 Nov 2026

Sponsor / Collaborator

Novartis Pharma AG

NCT07347990

/ RecruitingNot ApplicableIIT

A Prospective, Multicenter, Single-Arm Study: Safety and Efficacy of Iptacopan in the Treatment of High-Risk Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA)

The goal of this clinical trial is to evaluate the efficacy and safety of Iptacopan as a second-line treatment for high-risk hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA). Iptacopan is a selective oral small-molecule complement factor B inhibitor. It acts by inhibiting factor B, blocking the formation of C3 convertase, reducing C3b deposition, thereby suppressing C5 convertase (C3bBbC3b) and ultimately decreasing the formation of the membrane attack complex (MAC), which is expected to mitigate endothelial damage in TA-TMA pathology. The main questions this study aims to answer are:
* Does Iptacopan improve 6-month overall survival in high-risk TA-TMA patients?
* What adverse events do participants experience while taking Iptacopan?
* Does Iptacopan provide hematological response and organ function recovery in TA-TMA patients? In this prospective, multicenter, open-label, single-arm Phase II study, all participants will receive Iptacopan treatment. The primary endpoint of this study is the 6-month overall survival rate from TA-TMA diagnosis. Secondary endpoints include safety evaluation, hematological response, and organ function recovery.
During the study, participants will:
* Receive Iptacopan treatment according to protocol
* Undergo regular assessments for safety and efficacy monitoring
* Be followed for up to 24 months post-treatment initiation

Start Date25 Aug 2026

Sponsor / Collaborator

The First Affiliated Hospital, Zhejiang University Sch of Med

[+9]

CTRI/2026/07/115007

/ Not yet recruitingPhase 2

A multicenter, single arm, open label biopsy study toevaluate structural and functional changes in kidneys ofadult patients with IgA nephropathy receiving iptacopan ontop of supportive care - NIL

Start Date10 Aug 2026

Sponsor / Collaborator

Novartis Healthcare Pvt Ltd.

100 Clinical Results associated with Iptacopan Hydrochloride

100 Translational Medicine associated with Iptacopan Hydrochloride

100 Patents (Medical) associated with Iptacopan Hydrochloride

142

Literatures (Medical) associated with Iptacopan Hydrochloride

01 Feb 2027·BLOOD CELLS MOLECULES AND DISEASES

Real-world experience in management of patients with PNH treated with iptacopan: A case series

Article

Author: Gurnari, Carmelo ; Maciejewski, Jaroslaw P ; Sanikommu, Srinivasa ; Orland, Mark ; Dingli, David

This case series on managing paroxysmal nocturnal hemoglobinuria (PNH) with iptacopan focuses on scenarios in which the risk of breakthrough hemolysis (BTH) or thrombotic events is heightened. Patient 1 was a 46-year-old woman with PNH and a history of hepatic vein thrombosis. Patient 2 was a 30-year-old woman with PNH and a history of right posterior cerebral artery stroke and cerebral venous sinus thrombosis with extensive right superficial thrombophlebitis. Patient 3 was a 48-year-old woman with PNH who contracted a gastrointestinal infection 3 months after starting iptacopan. Patient 4 was a 39-year-old man with PNH who experienced serous otitis media, a complement-amplifying condition (CAC), concurrent with upper respiratory infection. Patient 5 was a 56-year-old woman with PNH who underwent radiofrequency ablation of the spine while on iptacopan. No patients experienced BTH or hospitalization while on iptacopan. No new thrombotic events were reported after initiating iptacopan. For patients experiencing CACs or surgery, no changes in PNH therapy were required. Notably, BTH did not occur in the patient with gastroenteritis, which can cause drug malabsorption. This case series suggests that iptacopan effectively maintains complement inhibition and prevents PNH-related hemolysis and thrombosis in the setting of past thromboses, gastrointestinal illness, and other CACs.

31 Dec 2026·Hematology

Improvement in anemia and symptoms after switching from crovalimab to iptacopan in paroxysmal nocturnal hemoglobinuria

Article

Author: Onizuka, Makoto ; Ogawa, Yoshiaki ; Nasukawa, Miina ; Ogiya, Daisuke ; Koyama, Seina ; Machida, Shinichiro ; Shiraiwa, Sawako ; Kawada, Hiroshi ; Tomita, Shunsuke ; Endo, Motoki ; Toyosaki, Masako

OBJECTIVES:

Paroxysmal nocturnal hemoglobinuria (PNH) may develop breakthrough hemolysis (BTH) despite C5 inhibition. Although iptacopan, an oral factor B inhibitor, has demonstrated efficacy in phase 3 trials, switching from crovalimab has not been reported. We describe the first clinical case of this switch in a real-world clinical setting.

METHODS:

A 72-year-old man with long-standing PNH received eculizumab followed by crovalimab. Infectious enteritis triggered BTH and worsening anemia during crovalimab therapy. Because anemia persisted after recovery, iptacopan 200 mg twice daily was initiated three weeks after the final crovalimab administration. Clinical symptoms and laboratory parameters were followed for 12 months. This case report complied with the Declaration of Helsinki.

RESULTS:

Hemoglobin increased from 9.6 to 11.6 g/dL within two weeks and remained ≥12 g/dL thereafter. Dyspnea resolved within one week, vitality improved by two weeks, and no BTH or treatment-related adverse events occurred during follow-up.

DISCUSSION:

This case demonstrates the feasibility of switching from crovalimab to iptacopan despite the absence of an established method, and pharmacokinetic considerations guided the timing.

CONCLUSION:

Switching to iptacopan led to rapid and durable improvement in anemia and symptoms, supporting proximal complement inhibition as a valuable option for PNH patients inadequately controlled with C5 inhibitors.

31 Dec 2026·Hematology

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Article

Author: Dingli, David ; Gurnari, Carmelo ; Maciejewski, Jaroslaw ; Sanikommu, Srinivasa ; Orland, Mark

OBJECTIVES:

Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

METHODS:

Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer Institute.

RESULTS:

Patient 1 is a 57-year-old woman with PNH. She experienced extravascular hemolysis (EVH) while on C5 inhibitors and breakthrough hemolysis (BTH) after switching to pegcetacoplan. Patient 2 is a 37-year-old woman seeking care for PNH, who was initiated on C5 inhibitor therapy and switched to pegcetacoplan. EVH and BTH were observed while she was on C5 inhibitor therapy, and transfusions were required with both C5 inhibitor and pegcetacoplan therapy. Patient 3 is a 58-year-old man with aplastic anemia and PNH who was initiated on C5 inhibitor therapy, then switched to pegcetacoplan. He experienced both EVH and BTH.

DISCUSSION:

All three patients switched from pegcetacoplan to iptacopan, and none experienced EVH or BTH while on iptacopan. After switching to iptacopan, all three patients improved hemoglobin levels and abating transfusion requirements.

CONCLUSION:

In this case series, three patients with PNH were successfully transitioned from pegcetacoplan to iptacopan monotherapy. No patients exhibited laboratory evidence of BTH, EVH, or intravascular hemolysis after switching to iptacopan.

292

News (Medical) associated with Iptacopan Hydrochloride

27 Oct 2026

·www.biospace.com

Novartis faces $1.9B hit from Avidity fail but maintains confidence in $12B buy

iStockphoto/ Afry Harvy Novartis remains “very happy” with its $12 billion acquisition of Avidity Biosciences, despite a Phase 3 disappointment for the antibody-oligonucleotide conjugate del-desiran. Novartis stands by its $12 billion takeover of Avidity Biosciences even after del-desiran, one of the main assets driving that buyout, failed a late-stage myotonic dystrophy study and spawned a $1.9 billion partial impairment charge. “We remain very, very happy with the Avidity acquisition,” Novartis CEO Vas Narasimhan told reporters during a Tuesday morning press call to present the pharma’s third quarter earnings results . “We feel confident that there’s a tremendous value we can still generate off of the platform and the three assets that we acquired.” Narasimhan attributed the write-down to “the mathematical shift of our assumption.” Novartis still believes in the asset but has adjusted its valuation to account for the need to run an additional pivotal study, which will take time and money, he said. Del-desiran is an antibody-oligonucleotide conjugate that targets transferrin receptor 1 on muscle cells and tags DMPK messenger RNA for destruction. Myotonic dystrophy type 1 is caused by mutations in the DMPK gene. In September, data from the Phase 3 HARBOR study—which enrolled 159 patients with DM1 who were randomly assigned to del-desiran or placebo—showed that the investigational therapy failed to hit the study’s primary outcome of improving hand function. Neuroscience Novartis’ $12B Avidity acquisition hits Phase 3 speedbump as dystrophy drug disappoints While Novartis’ antibody-oligonucleotide conjugate failed to significantly improve hand function in patients with myotonic dystrophy type 1, the asset nevertheless showed signs of clinical activity in secondary and exploratory measures. September 8, 2026 · 3 min read · Tristan Manalac Read more In response to the failure, Oppenheimer in a Sept. 8 note called del-desiran a “$12B disappointment.” But Narasimhan on Tuesday’s press call pointed out that “we saw evidence of clinical activity in the secondary and exploratory endpoints” in HARBOR. The pharma plans to advance del-desiran into another pivotal program and is currently evaluating what the best path forward is, he added. Narasimhan also emphasized the other clinical assets and the technology for antibody-oligonucleotide conjugates that Avidity brought to the Novartis fold. One of these candidates, called del-brax, is in Phase 3 development for facioscapulohumeral muscular dystrophy and for which the pharma is discussing with the FDA the potential for accelerated approval, Narasimhan said. Another Avidity asset, del-zota, has priority review for certain types of Duchenne muscular dystrophy. “So in our view, the Avidity acquisition remains absolutely on track,” Narasimhan said. “It’s hit a bump, which is normal, I think, when you’re trying to do pioneering science.” M&A Novartis To ‘Bolster’ Neuromuscular Pipeline With $12B Avidity Acquisition The deal, announced early Sunday afternoon, will see Novartis gain access to Avidity’s neuroscience assets, while the San Diego biotech spins out a new company to shepherd its early-stage precision cardiology programs. October 26, 2025 · 2 min read · Heather McKenzie Read more Other bumps in Novartis’ quarter Just a few days before the del-desiran announcement, the company reported that the antisense therapy pelacarsen failed to significantly lower cardiovascular risk in a late-stage trial. And these two disappointing readouts followed the news that Novartis had paused a host of mid-stage immunology and neurology studies for its CAR T therapy rapcabtagene autoleucel following three deaths. These setbacks sent the pharma’s shares down some 13% in a week . Narasimhan said on Tuesday’s call that while there have been no further safety events to report, those studies remain on hold in in U.S. and Europe. “It’s absolutely our goal to get these trials started again,” he said, highlighting the feedback the company has gotten from physicians and patients about the clinical benefits of these CAR T therapies. “We expect in the coming months to be able to provide further updates once we identify the root cause and also understand the best approach to ensure patient safety in the future.” But Novartis’ rough stretch didn’t end there. Days after news of the trial pauses broke, reports broke that the pharma had discontinued the development of the TREM2 stabilizer lifonebart in amyotrophic lateral sclerosis after the asset showed disappointing functional outcomes in a mid-stage study. Clinical research Novartis drags peers down with triple asset stumble Novartis’ shares fell 13% this week after the report of three separate late-stage asset setbacks. The impact went beyond the Swiss drugmaker, dragging peers down across the market. September 11, 2026 · 1 min read · Annalee Armstrong Read more In the third quarter, Novartis reported net sales of $14.5 billion, up 4% year-over-year at constant currencies. The anti-inflammatory drug Cosentyx was Novartis’ top-selling drug for the third quarter with nearly $1.9 billion in earnings, up 9% from the same period last year. Sales over the first nine months of the year hit $42 billion, flat versus the same period in 2025—though CFO Mukul Mehta pointed out during the press call that Novartis is “absorbing the largest patent expiry in our history.” The pharma’s Entresto lost exclusivity in summer 2025 and faced immediate generic competition in the U.S. The chronic heart failure drug made $1.16 billion in the third quarter and $3.65 billion so far this year, down 38% and 45% in constant-currency since the same periods last year. Novartis countered those decline with key growth drivers including the breast cancer drug Kisqali, which grew 37% since Q3 2025 to make $1.8 billion in the third quarter this year, and the multiple sclerosis medicine Kesimpta, which jumped 22% to hit $1.48 billion. Other high-growth assets include Scemblix, which is indicated for certain types of leukemia and surged 67% year-over-year to bring in $593 million; the cholesterol-lowering Leqvio, which saw a 63% increase to make $506 million in the quarter; and the complement blocker Fabhalta, which soared 60% to $237 million. Earlier this month, Novartis ended a Phase 2 study for Fabhalta in lupus that has inflamed the kidneys. Aspokesperson said in an email to BioSpace that “the decision was based on strategic considerations and was not driven by any interim efficacy or safety findings.” Novartis will continue to study Fabhalta—which already has approvals in a rare blood disorder called paroxysmal nocturnal hemoglobinuria as well as IgA nephropathy and complement 3 glomerulopathy—in late-stage trials for atypical haemolytic uraemic syndrome and generalized myasthenia gravis.

Phase 3AcquisitionClinical ResultOligonucleotide

27 Oct 2026

·www.biospace.com

Novartis delivered sales growth and core margin expansion in Q3; raised FY core operating income guidance

Ad hoc announcement pursuant to Art. 53 LR Third quarter Q3 net sales grew +4% (cc ¹ , +4% USD), with core operating income ¹ +10% (cc, +11% USD) Sales growth driven by priority brands including Kisqali (+37% cc), Kesimpta (+22% cc), Scemblix (+67% cc), Leqvio (+63% cc) and Cosentyx (+9% cc) Core operating income margin ¹ was 41.6%, +2 percentage points (cc) Q3 operating income declined -61% (cc, -59% USD) mainly due to a partial impairment of the del-desiran asset; net income down -71% (cc, -70% USD) Q3 core EPS ¹ grew +8% (cc, +9% USD) to USD 2.46 Q3 free cash flow ¹ was USD 5.1 billion (-18% USD) 9 months net sales (0% cc, +2% USD) and core operating income (-2% cc, -1% USD) broadly in line with PY Q3 selected innovation milestones: Remibrutinib positive PhIII REMODEL-1 and -2 results in RMS Rhapsido FDA approval in most prevalent form of CIndU Pluvicto FDA and CN approvals in mAPMN/S prostate cancer Pelabresib EU submission completed in MF Del-zota FDA priority review granted for DMD44 Full-year 2026 guidance² raised for core operating income Net sales expected to grow low single-digit (unchanged) Core operating income expected to be in line to grow low single-digit (from decline low single-digit) Basel, October 27, 2026 – Commenting on Q3 2026 results, Vas Narasimhan, CEO of Novartis, said: “Novartis delivered solid sales growth in Q3, as priority brands and launches more than offset generic erosion, demonstrating the replacement power of the business. Combined with productivity efforts, this drove strong bottom-line performance and allowed us to upgrade our full-year guidance. At the same time, we continued to advance our pipeline, with positive Phase III results for remibrutinib in RMS, label expansions for Rhapsido and Pluvicto in the US, and the first submission for pelabresib in the EU. While the del-desiran readout in DM1 was not what we had hoped, we saw evidence of clinical activity in the data and are working through the development path for this medicine. Importantly, our programs in FSHD and DMD continue to progress well. With continued momentum across the business and pipeline, we remain well on track to deliver our mid- and long-term outlook.” Key figures Q3 2026 Q3 2025 % change 9M 2026 9M 2025 % change USD m 3 USD m 3 USD cc USD m 3 USD m 3 USD cc Net sales 14 522 13 909 4 4 42 043 41 196 2 0 Operating income 1 843 4 501 -59 -61 10 828 14 028 -23 -24 Net income 1 196 3 930 -70 -71 7 609 11 563 -34 -35 EPS (USD) 0.63 2.04 -69 -70 4.01 5.94 -32 -33 Free cash flow 5 111 6 217 -18 14 002 15 941 -12 Core operating income 6 036 5 460 11 10 16 873 16 960 -1 -2 Core net income 4 655 4 330 8 6 13 027 13 522 -4 -5 Core EPS (USD) 2.46 2.25 9 8 6.85 6.94 -1 -3 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 45 of the Condensed 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. USD millions unless indicated otherwise. Strategy Our focus Novartis is a “pure-play” innovative medicines company. 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. Strengthen foundations : Unleashing the power of our people, scaling data science and technology, and continuing to build trust with society. Financials Third quarter Net sales were USD 14.5 billion (+4%, +4% cc), with volume growth contributing 14 percentage points, partly offset by 9 percentage points from generic competition. Pricing had a negative impact of 1 percentage point, and currency had no impact. Operating income was USD 1.8 billion (-59%, -61% cc), declining mainly due to higher R&D expenses, including a USD 1.9 billion partial impairment charge of the del-desiran IPR&D asset, and higher other expense, partly offset by higher net sales. Net income was USD 1.2 billion (-70%, -71% cc), declining mainly due to lower operating income and higher interest expense. EPS was USD 0.63 (-69%, -70% cc). Core operating income was USD 6.0 billion (+11%, +10% cc), mainly driven by higher net sales. Core operating income margin was 41.6% of net sales, increasing 2.3 percentage points (2.0 percentage points in cc). Core net income was USD 4.7 billion (+8%, +6% cc), driven by higher core operating income, partly offset by higher interest expense. Core EPS was USD 2.46 (+9%, +8% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow amounted to USD 5.1 billion (-18%), declining due to lower net cash flows from operating activities. Nine months Net sales were USD 42.0 billion (+2%, 0% cc), with volume growth contributing 14 percentage points, partly offset by 12 percentage points from generic competition. Pricing had a negative impact of 2 percentage points, and currency had a positive impact of 2 percentage points. Operating income was USD 10.8 billion (-23%, -24% cc), declining mainly due to higher R&D expenses, including a USD 1.9 billion partial impairment charge of the del-desiran IPR&D asset, and higher other expense. Net income was USD 7.6 billion (-34%, -35% cc), mainly due to lower operating income, higher interest expense and higher income taxes. EPS was USD 4.01 (-32%, -33% cc). Core operating income was USD 16.9 billion (-1%, -2% cc), declining mainly due to higher R&D expenses and higher other expense, partly offset by lower SG&A expenses. Core operating income margin was 40.1% of net sales, decreasing 1.1 percentage points (0.8 percentage points in cc). Core net income was USD 13.0 billion (-4%, -5% cc), mainly due to lower core operating income and higher interest expense. Core EPS was USD 6.85 (-1%, -3% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow amounted to USD 14.0 billion (-12%), declining due to lower net cash flows from operating activities. Q3 priority brands Underpinning our financial results in the quarter is a continued focus on key growth drivers (ranked in order of contribution to Q3 growth) including: Kisqali (USD 1 821 million, +37% cc) continued to grow strongly across all regions, with market share growth in early breast cancer and leadership in metastatic breast cancer. Kesimpta (USD 1 484 million, +22% cc) sales showed continued strong momentum globally, driven by increased demand and broad access. Scemblix (USD 593 million, +67% cc) sales grew across all regions, with continued strong momentum in the early-line setting in the US, Japan and Germany. L eqvio (USD 506 million, +63% cc) sales grew across all regions, with continued uptake in China following NRDL inclusion. Cosentyx (USD 1 857 million, +9% cc) sales grew in most markets, driven by the HS and SpA indications. US growth benefited from prior-year revenue deduction adjustments. P luvicto (USD 674 million, +20% cc) sales showed continued strong demand driven by the mAPMR¹ setting, with US and China approvals in mAPMN/S² in Q3, and ongoing access expansion ex-US. Zolgensma Group (USD 404 million, +35% cc) sales grew strongly, reflecting continued launch momentum from Itvisma in the US, Japan and other ex-US countries. R hapsido (USD 101 million) continued to show strong launch uptake in the US, supported by sustained prescription growth and a free drug program facilitating patient access. Ex-US, sales were mainly driven by early launch uptake in Germany and China. F abhalta (USD 237 million, +60% cc) sales grew, with expansion in PNH and renal indications. 1 Metastatic androgen pathway modulation-resistant prostate cancer (mAPMR), also known as metastatic castration resistant prostate cancer (mCRPC). ² Metastatic androgen pathway modulation-naïve or sensitive prostate cancer (mAPMN/S), also known as metastatic hormone-sensitive prostate cancer (mHSPC). Net sales of the top 20 brands in the third quarter and nine months Q3 2026 % change 9M 2026 % change USD m USD cc USD m USD cc Cosentyx 1 857 9 9 5 247 8 6 Kisqali 1 821 37 37 5 032 45 44 Kesimpta 1 484 21 22 4 072 27 26 Entresto 1 165 -38 -38 3 651 -44 -45 Pluvicto 674 20 20 1 967 42 41 Jakavi 564 5 5 1 697 9 6 Tafinlar + Mekinist 570 4 4 1 644 -2 -4 Ilaris 591 25 26 1 616 18 17 Scemblix 593 66 67 1 588 78 78 Leqvio 506 64 63 1 438 67 63 Zolgensma Group 404 34 35 1 071 16 13 Xolair 331 -25 -25 1 061 -21 -23 Sandostatin Group 311 3 3 900 -2 -4 Lutathera 219 3 3 655 7 6 Fabhalta 237 59 60 631 80 79 Exforge Group 183 4 1 577 6 2 Promacta/Revolade 142 -61 -61 505 -64 -65 Diovan Group 137 -4 -6 447 0 -3 Tasigna 141 -36 -37 438 -53 -54 Piqray/Vijoice 122 36 37 326 8 8 Top 20 brands total 12 052 7 7 34 563 3 1 R&D update – key developments from the third quarter New approvals Rhapsido (remibrutinib) In October, FDA approved Rhapsido as the first treatment for adults with symptomatic dermographism (SD) inadequately controlled by H1 antihistamines, expanding its use beyond chronic spontaneous urticaria (CSU) into chronic inducible urticaria (CIndU). Novartis plans to submit the full RemIND dataset to health authorities globally. Pluvicto (lutetium Lu177 vipivotide tetraxetan) FDA and China’s NMPA approved Pluvicto in combination with an androgen receptor pathway inhibitor (ARPI) for patients with PSMA+ metastatic androgen pathway modulation-naive/sensitive (mAPMN/S) prostate cancer¹. Pluvicto is now approved across all stages of PSMA+ metastatic prostate cancer. Fabhalta (iptacopan) Japan’s MHLW approved Fabhalta for the treatment of IgA nephropathy (IgAN), making it the first approved treatment for this indication in Japan. Additionally, FDA granted traditional approval of Fabhalta in IgAN. ¹ Also known as metastatic hormone-sensitive prostate cancer (mHSPC). Regulatory updates Cosentyx (secukinumab) CHMP adopted a positive opinion recommending approval for Cosentyx in polymyalgia rheumatica (PMR), based on positive Phase III REPLENISH data. If approved, Cosentyx will be the first IL-17A inhibitor approved in Europe for PMR. DAK539 (pelabresib) Regulatory submission for pelabresib in myelofibrosis was completed in the EU, based on the positive Phase III MANIFEST-2 study, which showed deep and durable disease control, with a safety pro to ruxolitinib alone, including numerically fewer deaths and disease progressions in the pelabresib arm. KPE179 (del-zota) Del-zota was granted priority review by FDA for people living with Duchenne muscular dystrophy who have a genetic variant that may be amenable to exon 44 skipping (DMD44). Del-zota previously received FDA Breakthrough Therapy designation. Leqvio (inclisiran) FDA updated the Leqvio label to include language recognizing that lowering LDL-C reduces the risk of major adverse cardiovascular events (MACE) in adults at increased cardiovascular risk. This update reflects established evidence from statins and monoclonal antibody PCSK9 inhibitors, supporting LDL-C reduction as a key component of cardiovascular risk management. Vanrafia (atrasentan) Regulatory submissions for Vanrafia in IgAN were completed in Japan and China. KLU156 (ganaplacide + lumefantrine) Regulatory submission for KLU156 in uncomplicated malaria was completed in the US and received priority review. Results from ongoing trials and other highlights LOU064 (remibrutinib) Positive results from the Phase III REMODEL-1 and -2 trials showed oral remibrutinib met its primary endpoint in relapsing multiple sclerosis (RMS), significantly reducing annualized relapse rate (ARR) versus teriflunomide. Remibrutinib also showed superiority on all key secondary endpoints, including MRI lesion reduction, and a clinically meaningful delay in disability progression. The safety pro favorable and consistent with remibrutinib in CSU, with no liver safety signal. Data were presented at MSToronto2026 in October. EWF980 (del-desiran) In the Phase III HARBOR study, del-desiran did not meet its primary endpoint of video hand opening time (vHOT) versus placebo in myotonic dystrophy type 1 (DM1). Evidence of clinical activity was observed in secondary endpoints and exploratory analyses. Novartis is evaluating the full HARBOR dataset and will engage with health authorities to determine the most appropriate development path. Data will be presented at an upcoming medical congress. TQJ230 (pelacarsen) In the Phase III HORIZON trial, pelacarsen did not meet its primary endpoint of reducing the risk of cardiovascular events versus placebo in patients with elevated Lp(a) and established cardiovascular disease (CVD), despite reducing Lp(a) levels in this study population. The findings provide important new evidence on the relationship between Lp(a) lowering and cardiovascular outcomes and may help inform future approaches to cardiovascular risk management. Data will be presented at an upcoming medical congress. Kisqali (ribociclib) The six-year follow-up analysis of the Phase III NATALEE trial showed a clinically meaningful overall survival (OS) benefit with Kisqali plus endocrine therapy (ET) versus ET alone in the broadest HR+/HER2- early breast cancer (eBC) population at high risk of recurrence, reducing the risk of death by nearly 20%. Patients had been off Kisqali treatment for a median of 38 months, with clinical benefit continuing to deepen after treatment completion. The safety pro consistent with previous analyses. Data were presented at ESMO 2026. LOU064 (remibrutinib) Novartis has discontinued the Phase III study of remibrutinib in generalized myasthenia gravis (gMG) following a futility analysis, which indicated the treatment was unlikely to meet its efficacy objectives. No safety concerns were identified, and there is no impact on remibrutinib studies in other indications. Selected transactions Novartis completed the acquisition of Myricx Bio, strengthening the oncology pipeline with a new class of antibody-drug conjugates (ADCs), including two lead assets targeting B7-H3 and HER2, as well as a broader payload platform with potential impact across multiple solid tumor settings. Novartis exercised its exclusive option to acquire full global rights to Sironax's proprietary Brain Delivery Module (BDM) platform, a next-generation blood-brain barrier crossing technology with the potential to transform the treatment of central nervous system diseases. Novartis entered into an exclusive global licensing agreement with BoomRay Pharmaceuticals for an undisclosed preclinical radioligand therapy asset, further strengthening its radioligand therapy portfolio. In October, Novartis entered into a strategic licensing and option agreement with Abogen Biosciences to advance RNA-encoded therapeutics, including an exclusive worldwide license to ABO2203, a novel mRNA-encoded CD19xCD3 T-cell engager with the potential to transform the treatment landscape for autoimmune diseases. The transaction is subject to the completion of customary closing conditions. 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 nine months of 2026, Novartis repurchased 30.9 million shares for USD 4.7 billion on the SIX Swiss Exchange second trading line. These repurchases included 18.8 million shares (USD 2.8 billion) under the up-to USD 10 billion share buyback announced in July 2025 (with up to USD 4.9 billion still to be executed). In addition, 12.1 million shares (USD 1.8 billion) were repurchased to mitigate the anticipated full-year dilution related to participation plans of employees. Further, 2.0 million shares (USD 0.3 billion) were repurchased from employees. During the same period, USD 0.9 billion of equity-based compensation plan expenses were recognized to equity and 13.3 million shares were delivered to employees related to equity-based compensation plans from prior years. As a result, the total number of shares outstanding decreased by 19.6 million compared with December 31, 2025. These treasury share transactions resulted in an equity decrease of USD 4.1 billion and a cash outflow of USD 4.8 billion. Net debt increased to USD 37.4 billion at September 30, 2026, compared with USD 21.9 billion at December 31, 2025. The increase was mainly due to the free cash flow of USD 14.0 billion being more than offset by the net cash outflow for M&A, intangible assets transactions and other acquisitions of USD 16.7 billion, the USD 9.1 billion annual dividend payment and cash outflows for treasury share transactions of USD 4.8 billion. As of Q3 2026, the long-term credit rating for the company is Aa3 with Moody’s Ratings and AA- with S&P Global Ratings. 2026 outlook Barring unforeseen events; growth vs. prior year in cc Net sales Expected to grow low single-digit Core operating income Expected to be in line to grow low single-digit (from decline low single-digit) Foreign exchange impact If late-October exchange rates prevail for the remainder of 2026, the foreign exchange impact for the year would be positive 1 percentage point on net sales and positive 1 percentage point on core operating income. The estimated impact of exchange rates on our results is provided monthly on our website. 2027 Annual General Meeting Nomination for election to the Board of Directors The Novartis Board of Directors announced today that it is nominating Rajesh Jha for election to the Board. During a 36-year career at Microsoft, Mr. Jha held a series of senior leadership roles across software engineering, productivity platforms and enterprise technologies, and served on the company's Senior Leadership Team from 2016 until his retirement. Most recently, he served as Executive Vice President, Experiences + Devices, where he led the product teams for Microsoft 365, Office, Teams, Windows + Devices, and Dynamics, and played a key role in the integration of artificial intelligence across Microsoft's product portfolio. His extensive experience in technology, AI and large-scale digital platforms would add significantly to the Novartis Board of Directors. Key figures¹ Q3 2026 Q3 2025 % change 9M 2026 9M 2025 % change USD m² USD m² USD cc USD m² USD m² USD cc Net sales 14 522 13 909 4 4 42 043 41 196 2 0 Operating income 1 843 4 501 -59 -61 10 828 14 028 -23 -24 As a % of sales 12.7 32.4 25.8 34.1 Net income 1 196 3 930 -70 -71 7 609 11 563 -34 -35 EPS (USD) 0.63 2.04 -69 -70 4.01 5.94 -32 -33 Net cash flows from operating activities 5 503 6 571 -16 15 061 16 880 -11 Non-IFRS measures Free cash flow 5 111 6 217 -18 14 002 15 941 -12 Core operating income 6 036 5 460 11 10 16 873 16 960 -1 -2 As a % of sales 41.6 39.3 40.1 41.2 Core net income 4 655 4 330 8 6 13 027 13 522 -4 -5 Core EPS (USD) 2.46 2.25 9 8 6.85 6.94 -1 -3 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 45 of the Condensed Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. 2. USD millions unless indicated otherwise. Detailed financial results accompanying this press release are included in the Condensed Interim Financial Report at the link below: Disclaimer This communication 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 “expected,” “anticipated,” “can,” “will,” “continue,” “ongoing,” “growth,” “launch,” “expanding,” “deliver,” “accelerate,” “guidance,” “outlook,” “priority,” “potential,” “momentum,” “on track,” “pipeline,” “evidence,” “progress,” “emerging,” or similar expressions, or by express or implied discussions regarding: potential new products, potential new indications for existing products, potential product launches or potential future revenues from any such products; or results of ongoing clinical trials; potential future, pending or announced transactions; potential future sales or earnings; strategy, plans, expectations or intentions, including discussions regarding our continued investment into new R&D capabilities and manufacturing; our capital structure. You should not place undue reliance on these statements. 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. There can be no guarantee that the investigational or approved products described in this communication 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 that the expected benefits or synergies from the transactions described in this communication will be achieved in the expected timeframe, or at all. In particular, our expectations could be affected by, among other things, uncertainties concerning: global healthcare cost containment, including ongoing government, payer and general public pricing and reimbursement pressures and requirements for increased pricing transparency; the success of key products, commercial priorities and strategy; the research and development of new products, including clinical trial results and additional analysis of existing clinical data; 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; our ability to realize the strategic benefits, operational efficiencies or opportunities expected from our external business opportunities; the development or adoption of new technologies, including artificial intelligence, and new business models; the implementation of our new IT projects and systems; potential significant breaches of information security or disruptions of our information technology systems; actual or potential legal proceedings, including regulatory actions or delays or government regulation related to the products and pipeline products described in this communication; safety, quality, data integrity, or manufacturing issues; our performance on and ability to comply with environmental, social and governance measures and requirements; major macroeconomic and geo- and socio-political developments, including the impact of any potential tariffs on our products or the impact of war in certain parts of the world; future global exchange rates; 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 communication 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. 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 300 million people worldwide. Reimagine medicine with us: Visit us at 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 13:00 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 . 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 . Important dates November 18-19, 2026 Meet Novartis Management 2026 (London, UK) February 3, 2027 Fourth quarter & full year 2026 results March 5, 2027 Annual General Meeting # # # Novartis Media Relations E-mail: media.relations@novartis.com Novartis Investor Relations Central investor relations line: +41 61 324 7944 E-mail: investor.relations@novartis.com 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)

Phase 3Clinical ResultDrug ApprovalBreakthrough TherapyLicense out/in

23 Oct 2026

·www.fiercebiotech.com

Roche puts numbers behind phase 3 kidney disease win amid blockbuster ambitions

Roche's Genentech has put numbers behind its phase 3 success in IgA nephropathy (IgAN), reporting that antisense oligonucleotide sefaxersen reduced a key marker of kidney damage by 43.1% compared with placebo. In the late-stage trial, called IMAgINATION, patients receiving the drug candidate demonstrated a significant reduction in proteinuria—the presence of excess protein in urine that signals kidney damage—at 37 weeks. Among patients with blood in their urine at baseline, a greater proportion of those receiving sefaxersen experienced resolution of the condition compared with placebo, the company reported. The drug was well tolerated, with no new safety signals and a safety profile consistent with an earlier phase 2 trial. Roche presented the detailed results at the American Society of Nephrology's Kidney Week meeting in Denver, a month after first announcing that the trial had met its primary endpoint . The company plans to continue the study to measure kidney function over two years while sharing data with regulatory agencies "with the goal of bringing this treatment to patients as soon as possible." Sefaxersen, designed to be administered through a once-monthly subcutaneous injection, inhibits the production of complement factor B, a protein involved in the immune system's alternative complement pathway. Overactivation of this pathway contributes to kidney damage in patients with IgA nephropathy. "By inhibiting complement factor B in the liver, sefaxersen may offer a novel approach to slow disease progression in IgAN, potentially helping patients delay or avoid long-term dialysis or kidney transplantation," Genentech Chief Medical Officer and Head of Global Product Development Levi Garraway, M.D., Ph.D., said in the release. Roche licensed sefaxersen, previously called IONIS-FB-LRx, from Ionis Pharmaceuticals in 2022, building on an option agreement signed in 2018. Expecting the IgAN market to grow rapidly through the end of the decade, Roche has projected peak sales of 1 billion to 2 billion Swiss francs for sefaxersen. But Roche's drug won't be the only option in town. Novartis gained accelerated FDA approval for its own factor B inhibitor, Fabhalta, in 2024, followed by full approval in July 2026. The twice-daily oral medication could have an advantage over Roche's once-monthly injection when it comes to ease of administration. However, Fabhalta has been associated with increased cholesterol levels, while Roche's drug showed no such effect in a previous phase 2 study.

Clinical ResultPhase 3Phase 2Drug ApprovalOligonucleotide

100 Deals associated with Iptacopan Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D12251 D12252	Iptacopan Hydrochloride	L04AJ08	DB16200

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
glomerulonephritis chronic complement component 3 glomerulopathy	Canada	Novartis AG	04 May 2026
Complement Factor H Deficiency	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	24 Dec 2025
C3 glomerulopathy	United States	Novartis Pharmaceuticals Corp.	20 Mar 2025
Glomerulonephritis, IGA	United States	Novartis Pharmaceuticals Corp.	07 Aug 2024
Hemoglobinuria, Paroxysmal	United States	Novartis Pharmaceuticals Corp.	05 Dec 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Myasthenia Gravis	Phase 3	United States	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	United States	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	China	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Japan	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Japan	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Argentina	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Australia	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Brazil	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Denmark	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Denmark	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASN2026	Phase 3	Glomerulonephritis, IGA	1,714	targeted-release budesonide	yysqlhoscp(bzkcchizjj) = ttbqfijzkp ivhgdmdqdn (obayuuopqs )	Positive	24 Oct 2026
				methylprednisolone	yysqlhoscp(bzkcchizjj) = jmgyocyfze ivhgdmdqdn (obayuuopqs )
NCT03955445 (APPEAR-C3G, ASN2026)	Phase 3	C3 glomerulopathy	57	Iptacopan 200 mg b.i.d.	rchyhxjjct(gonejmhxat) = a reduction of 45% (95% CI: 23%, 61%) from baseline in FMV UPCR (n=45) awppfxdqhw (vsniyzqdvb )	Positive	24 Oct 2026
ASN2026	Not Applicable	Glomerulonephritis, IGA	34	Budesonide	cnosbfuixk(shddcanuvw) = gqqhtjcwqg ldtfkohnjc (kaowyshfsy ) View more	Negative	24 Oct 2026
				Sparsentan	oxlbzzkusu(umobvmgeoy) = jrtusrsnhc nnnoagdayq (hlaugqjccw )
NefIgArd (ASN2026)	Phase 3	Glomerulonephritis, IGA	1,361	Nefecon	gyynelcfhl(rzksxaclor) = All treatments reduced UPCR versus control groups: 27% for Nefecon at 9 months, 51.2% for sibeprenlimab at 9 months, 38.3% iptacopan at 9 months, 36.1% for atrasentan at 36 weeks, and 41% for sparsentan at 36 weeks (all 95% confidence intervals exclude zero). eyjdtvkyur (irvlgcdotj ) View more	Positive	24 Oct 2026
				Iptacopan
ASN2026	Not Applicable	Glomerulonephritis, IGA	310	IgAN-specific therapies	jpaljfqyub(qtxhyezqcj) = wgmiatwqta toqxcjdlmy (xzlxkmsozu ) View more	Positive	24 Oct 2026
ASN2026	Not Applicable	C3 glomerulopathy	27	Iptacopan	qqvscpfyfe(vvaxiphpzg) = rrjvwayfjc vhzlvcqcav (gqpxhyhcgx ) View more	Positive	23 Oct 2026
ASN2026	Not Applicable	Glomerulonephritis, IGA	58	Iptacopan	cgmhakozfn(gznpsqlkns) = owyauqujqf awbvwzioxm (gmeyxeiudn ) View more	Positive	22 Oct 2026
NCT04557462 (APPLAUSE-IgAN, ASN2026)	Phase 3	Glomerulonephritis, IGA	368	Iptacopan 200 mg twice daily + supportive care	ajydopffky(eyofaoyomj) = one death due to acute pancreatitis was unrelated to ipta ttvetrspvp (zqslyfqglf )	Positive	22 Oct 2026
				Placebo + supportive care
NCT04578834 (APPLAUSE-IgAN, Pubmed \| N Engl J Med)	Phase 3	Glomerulonephritis, IGA	477	Iptacopan 200 mg	bbomscbclb(lxybochiwy) = jtszeyepdz bznvrnummv (uiibbathze ) View more	Positive	30 Jul 2026
				Placebo	bbomscbclb(lxybochiwy) = rqaogwaogf bznvrnummv (uiibbathze ) View more
ISTH2026	Not Applicable	Hemoglobinuria, Paroxysmal	240	Proximal complement inhibitors	xofdwlcadr(tspjxkwoaa) = nvaekrqomb kxmsgcxoxr (vmdqntuniw ) View more	Positive	13 Jul 2026
				C5 inhibitors	-

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