RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Paediatric investigation plan (European Union)

Structure/Sequence

Sequence Code 66437L

Source: *****

Sequence Code 9941502H

Source: *****

Clinical Trials associated with Ravulizumab-CWVZ

NCT06333652

/ RecruitingPhase 2IIT

Clinical Trial on the Use of Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders

The researchers are testing a medication named ravulizumab for the treatment of severe preeclampsia and Hemolysis, Elevated Liver enzymes, Low Platelets (HELLP) syndrome.

Start Date01 Sep 2026

Sponsor / Collaborator

Mayo Clinic

NCT07557420

/ Not yet recruitingPhase 3

A Phase 3b, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)

The primary objective of this study is to confirm the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of ravulizumab in the treatment of Chinese adults with anti-aquaporin-4 (AQP4) antibody (Ab) + neuromyelitis optica spectrum disorder (NMOSD).

Start Date31 Aug 2026

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT07010302

/ Not yet recruitingPhase 4IIT

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune condition that mainly affects the eyes and spinal cord, causing serious symptoms such as vision loss, paralysis, and severe pain. This trial compares the effectiveness and safety of five medications commonly used to prevent NMOSD relapses: rituximab, ravulizumab, inebilizumab, satralizumab, and eculizumab.
In this study, 160 adults with NMOSD who test positive for a specific antibody (AQP4-IgG) will participate. They will be randomly assigned to receive either rituximab or one of the four other FDA-approved medications. The main goal is to find out which treatment best prevents relapses and has fewer serious side effects. The trial will also measure disability, patient satisfaction, quality of life, and biomarkers that help track disease activity.
Participants will have regular assessments, including medical exams, surveys, and tests for vision, walking ability, and brain function. They will report any side effects or health issues experienced during the study. The trial will last from one to four years for each participant.
This research aims to help patients and doctors make better-informed treatment decisions by providing clear evidence about the best available therapies for NMOSD.

Start Date01 Aug 2026

Sponsor / Collaborator

The Massachusetts General Hospital

[+1]

100 Clinical Results associated with Ravulizumab-CWVZ

100 Translational Medicine associated with Ravulizumab-CWVZ

100 Patents (Medical) associated with Ravulizumab-CWVZ

381

Literatures (Medical) associated with Ravulizumab-CWVZ

31 Dec 2026·JOURNAL OF MEDICAL ECONOMICS

Cost per responder analysis of iptacopan versus eculizumab and ravulizumab in treatment of paroxysmal nocturnal hemoglobinuria: implications for decision-making

Article

Author: Shafrin, Jason ; Muthukrishnan, Sanjana ; Kuypers, Nicholas ; Bilano, Ver ; Than, Kyi-Sin ; Paulose, Jincy

OBJECTIVE:

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. This study aimed to evaluate the economic value of iptacopan, a novel oral treatment option, compared to terminal complement inhibitors (specifically, complement component C5 inhibitor or C5i)-including eculizumab and ravulizumab-among patients with PNH who are either (i) C5i-experienced or (ii) complement-inhibitor-naïve.

METHODS:

A cost per responder analysis was conducted based on treatment efficacy from clinical trials comparing iptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion independence. Treatment costs were estimated as pharmaceutical wholesale acquisition cost and treatment administration costs, accounting for discontinuation. Outcomes evaluated included the number needed to treat to achieve a response and the cost per responder over the treatment duration of 24 weeks.

RESULTS:

Over 24 weeks, the number needed to treat to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs. 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs. 1.69 with C5is). Cost per responder was lower for iptacopan than C5i comparators for both C5i-experienced ($264,337 for iptacopan vs. $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilar with 10%-30% discount from eculizumab cost) and complement-inhibitor-naïve patients ($256,754, vs. $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilar).

CONCLUSION:

Among both C5i-experienced and complement-inhibitor-naïve patients, treatment with iptacopan resulted in higher response rates and lower cost per responder compared to C5is.

01 Aug 2026·JOURNAL OF NEUROIMMUNOLOGY

Combined inhibition of complement C5 and neonatal Fc receptor in refractory generalized myasthenia gravis: A report of two cases

Article

Author: Tateishi, Takahisa ; Harada, Masaya ; Hoshino, Tomoaki ; Isogai, Masashige ; Irie, Ken-Ichi ; Tanaka, Yoshiko ; Koga, Tamon ; Mori, Shinichiro ; Imai, Tatsuya

In patients with refractory generalized myasthenia gravis (MG) inadequately controlled by standard immunotherapies, including intravenous immunoglobulin and intravenous methylprednisolone, molecular targeted therapies have emerged as important treatment options. Although both neonatal Fc receptor (FcRn) inhibitors and complement C5 inhibitors have demonstrated efficacy in anti-acetylcholine receptor antibody-positive generalized MG, the clinical utility of their combined use remains unclear. Here, we report two cases of refractory anti-acetylcholine receptor antibody-positive generalized MG successfully managed with combined FcRn and C5 inhibition. In Case 1, disease control was not achieved despite thymectomy, multiple standard immunotherapies, and FcRn inhibition alone. Sequential treatment with C5 inhibitors, including eculizumab, ravulizumab, and zilucoplan, was attempted; sustained stabilization occurred only after combination therapy with zilucoplan and subcutaneous efgartigimod. In Case 2, standard therapies were ineffective, and thymectomy under general anesthesia was considered unsafe because of severe disease activity. Combination therapy was therefore introduced to stabilize the disease and enable safe perioperative management. Following successful thymectomy, targeted therapies were discontinued in Case 2, whereas Case 1 required ongoing treatment. To our knowledge, this is one of the first reports demonstrating effective disease control using combined FcRn and C5 inhibition in refractory generalized MG. These cases suggest that this strategy may benefit selected patients with refractory disease, particularly when stable disease control is required; however, further studies are warranted to determine its broader applicability and long-term safety.

01 Aug 2026·JOURNAL OF NEUROIMMUNOLOGY

Monoclonal antibody efficacy in seropositive vs seronegative NMOSD: systematic review and meta-analysis

Review

Author: Daromar, Adam ; Canibano, Beatriz ; Abushalbak, Dareen Jamal ; AlHabil, Yazan ; Safan, Abeer ; Abualrob, Majd A ; Hamid, Shahd ; Zammar, Khaled

BACKGROUND AND OBJECTIVES:

Monoclonal antibodies have transformed the treatment of neuromyelitis optica spectrum disorder (NMOSD), yet their comparative efficacy by aquaporin-4 immunoglobulin G (AQP4-IgG) serostatus remains uncertain. We conducted a systematic review and meta-analysis to compare monoclonal antibody efficacy between AQP4-IgG seropositive and seronegative NMOSD patients.

METHODS:

We searched PubMed/MEDLINE, Embase, and Cochrane CENTRAL from inception through January 2025 for studies comparing monoclonal antibody efficacy outcomes (rituximab, eculizumab, satralizumab, inebilizumab, tocilizumab, or ravulizumab) between seropositive and seronegative NMOSD patients. Primary outcomes included annualized relapse rate (ARR) and relapse events. Secondary outcomes included disability progression (Expanded Disability Status Scale [EDSS]) and infectious adverse events. Random-effects meta-analysis was performed. The protocol was registered in PROSPERO (CRD1049290).

RESULTS:

Thirteen studies (4 randomized controlled trials [RCTs], 9 observational) comprising 1284 patients (1010 seropositive; 274 seronegative) were included. Seropositive patients had significantly lower relapse risk (risk ratio [RR] = 0.66; 95% CI: 0.49-0.89; p = 0.007; I² = 0%; 9 studies), indicating 34% greater efficacy in preventing relapses. An RCT-only subgroup analysis demonstrated a 59% relative risk reduction favoring seropositive patients (RR = 0.41; 95% CI: 0.25-0.69; p = 0.0008; I² = 0%). No significant differences were observed for continuous ARR (standardized mean difference [SMD] = 0.23; p = 0.61; I² = 94%), disability progression (SMD = 1.07; p = 0.32; I² = 96%), or infectious adverse events (RR = 1.13; p = 0.61; I² = 0%).

DISCUSSION:

Monoclonal antibodies demonstrate significantly greater efficacy in preventing relapses in AQP4-IgG seropositive compared with seronegative NMOSD patients. Serostatus should inform treatment selection and expectations. Comparable safety profiles support continued use in seronegative patients, though further research is needed to optimize therapeutic strategies for this population.

332

News (Medical) associated with Ravulizumab-CWVZ

27 Jul 2026

·www.fiercepharma.com

AZ stays on track for $80B revenue goal as cancer meds, rare disease sales help offset Ultomiris miss

Even as trial setbacks and pipeline adjustments rear their heads, AstraZeneca feels confident that its core business is on track to support the $80 billion revenue goal it’s set for 2030. And on the more existential topics of China competition, U.S. drug pricing reform and the potential need for an external pipeline refresh, AZ’s top brass is keeping its cool. In 2026’s second quarter, the British Big Pharma generated revenue of nearly $15.4 million, growing 5% over the same period in 2025 at constant exchange rates. With the first act of 2026 now in the books, AZ is reporting total revenue of $30.7 billion, up some 6%, driven primarily by sales in oncology, rare disease and respiratory and immunology, which each grew over the first half of the year, even as total biopharma earnings dropped for the six-month period thanks to a hit in infectious disease. Among the high-grossing cancer unit, TKI lung cancer med Tagrisso remained AZ’s top earner with Q2 sales of $1.94 billion and a first-half-of-2026 haul amounting to nearly $3.8 billion, followed closely by PD-L1 blockbuster Imfinzi, which brought home $1.85 billion and $3.55 billion for Q2 and the first six months of 2026, respectively. Major multi-billion-dollar sales hauls for the second quarter and first half of the year were also posted by Farxiga in AZ’s cardiovascular category, plus Symbicort and Fasenra in the respiratory and immunology segment and Ultomiris from the rare disease portfolio, with the latter drug growing 12% to $1.31 billion for the quarter. While an improvement year-over-year, that performance fell short of analysts' expectations by around $24 million, according to a note from Jefferies Monday. Adding to that dent in Ultomiris’ quarterly report card, AZ revealed early Monday that a phase 3 trial pitting the drug against placebo in teens and adults with thrombotic microangiopathy after hematopoietic stem cell transplant had failed to hit statistical significance on the primary endpoint of event-free survival through 26 weeks. Thrombotic microangiopathies (TMAs) are rare and potentially lethal disorders that cause blood clots and damage to the walls of the smallest blood vessels in the circulatory system, and AZ had been testing Ultomiris in a type of the condition that occurs after hematopoietic stem cell transplant to treat cancer and other diseases. Still, AZ noted that its Alexion rare disease unit has nevertheless seen clinically meaningful results around overall survival in its pediatric trial and continues to advance filings on Ultomiris in that indication. The Ultomiris slip follows other phase 3 misses from AZ drugs in the period, including for Imfinzi in adjuvant hepatocellular carcinoma and Wainua in ATTR-CM. Separately, AZ revealed in its earnings report that it has scrapped two in-house antibody-drug conjugate (ADC) candidates that underperformed in the clinic. Nevertheless, executives told Fierce on a media call earlier Monday that they remain “very confident” in the company’s roster of investigational ADCs overall. When pressed on an analyst call whether AZ needs to lean on M&A activity to bolster its late-stage pipeline—especially as the company heads for a significant patent expiration period early in the next decade—CEO Pascal Soriot said in no uncertain terms that “the answer is no: We don’t need more BD to deliver.” Soriot emphasized that his confidence doesn’t come from any one single asset but the company’s phase 3 pipeline in aggregate, noting that AZ tracks its overall chance of phase 3 success at “around 60%,” which he noted was a bit lower than the industry average and below AZ’s historic track record of “75%-plus.” “The reason we don’t need more BD is really to continue planning long-term and continuously strengthen our franchises,” the CEO explained, pointing to Tagrisso as one example of a product worth investing in internally rather than rushing out to acquire a new asset. AstraZeneca’s overall second quarter earnings were in line with expectations, the Jefferies analysts pointed out Monday, noting that outsized Q2 performances from hypophosphatasia drug Strensiq (up 36% at $536 million) lymphoma and leukemia med Calquence (up 16% $1.02 billion) and asthma treatment Tezspire (up 45% $390 million) helping counter misses from Tagrisso and Lynparza, as well as AZ’s asthma rescue inhaler Airsupra ($50 million Q2), which “continues to lag,” in the Jefferies team’s estimation. With less than six months to go before the year is out, AZ is sticking to the previous guidance range, which anticipates a mid-to-high single-digit percentage revenue increase over 2025 at constant currencies. Moreover, the company reiterated that it’s confident in the $80 billion revenue target it has pledged to deliver in 2030. In reflecting on Tagrisso, Soriot also touched on the matter of China, where AZ has continued to commit resources heavily even as hawkishness around competition with the country mounts in places like the U.S. But in the CEO’s estimation, this deep relationship is exactly what positions AstraZeneca well to compete as the international biopharma landscape continues to evolve. “If you don’t have a very strong domestic business from a profit viewpoint, it’s not that simple to globalize,” Soriot said of the current China biotech dynamic. “But you have to assume they will at some point.” “What we’re doing,” he continued, “is collaborating, but we’re also competing. You saw the competition in the Tragrisso market is very, very intense, and in the ADC market very intense too. So, we learn to compete with them, and certainly we’ll take those learnings globally when they become global companies, if they do.” Soriot added that “We’re also learning from [China biotechs] in terms of how they develop products and how fast they operate. And we’ve made some changes in the way we operate.”

Phase 3ADC

27 Jul 2026

·www.businesswire.com

AstraZeneca results: H1 and Q2 2026

CAMBRIDGE, England--(BUSINESS WIRE)--AstraZeneca: Revenue and EPS summary H1 2026 % Change Q2 2026 % Change $m Actual CER1 $m Actual CER - Product Sales 28,896 8 5 14,510 5 4 - Alliance Revenue 1,699 31 29 874 34 33 Product Revenue 30,595 9 6 15,384 6 5 Collaboration Revenue 77 (6) (9) - n/m n/m Total Revenue 30,672 9 6 15,384 6 5 Reported EPS ($) 3.60 4 3 1.61 2 (2) Core2 EPS ($) 5.21 12 11 2.63 21 18 Key performance elements for H1 2026 (Growth numbers at constant exchange rates) Total Revenue up 6%, with double-digit growth in Oncology and Rare Disease offsetting headwinds from Farxiga US loss of exclusivity and China volume-based procurement Core Operating profit and Core EPS increased 11% Interim dividend increased 3 cents to $1.06 per share (79.5 pence, 10.32 SEK) 30 approvals in major regions since Q4 2025 results Pascal Soriot, Chief Executive Officer, AstraZeneca, said: "In the first half we saw strong performance and continued pipeline delivery, including six key positive Phase III programmes and eight first approvals in major markets, including in the US for Baxfendy, our first-in-class medicine for hypertension. While we are disappointed by the CARDIO-TTRansform outcome, we are on track to deliver our $80bn Total Revenue ambition, which assumes successes and setbacks. We remain confident in the strength of our pipeline and have more than twenty high-value readouts due over the next 18 months. We continue to invest at pace in our transformative technologies, and in our commercial execution to bring our innovative medicines to patients around the globe and drive growth beyond 2030." Guidance AstraZeneca reconfirms Total Revenue and Core EPS guidance3 for FY 2026 at CER, based on the average foreign exchange rates through 2025. Total Revenue is expected to increase by a mid-to-high single-digit percentage Core EPS is expected to increase by a low double-digit percentage The Core Tax rate is expected to be between 18-22% If foreign exchange rates for July 2026 to December 2026 were to remain at the average rates seen in June 2026, it is anticipated that Total Revenue in FY 2026 would benefit from a low single-digit percentage positive impact (unchanged) compared to the performance at CER, and Core EPS growth would be broadly similar (unchanged) to the growth at CER. Results highlights Table 1: Milestones achieved since the prior results announcement Phase III and other registrational data readouts Medicine Trial Indication Event Imfinzi VOLGA MIBC not candidates for cisplatin Primary endpoint met Imfinzi EMERALD-2 Adjuvant HCC Primary endpoint not met Imfinzi NILE 1L bladder cancer Primary endpoint met sone-ve CLARITY-Gastric01 2L+ Cldn18.2+ gastric/GEJ cancer Primary endpoint met Wainua CARDIO-TTRansform ATTR-CM Primary endpoint not met Ultomiris TMA-313 HSCT-TMA (adults) Primary endpoint not met Ultomiris ALXN1210-MG-319 gMG (paediatric) Primary endpoint met Regulatory approvals Medicine Trial Indication Region Calquence AMPLIFY 1L CLL (fixed duration) JP Datroway TROPION-Breast02 1L TNBC for patients where immunotherapy is not an option US Enhertu DESTINY-Breast05 High-risk HER2+ early breast cancer (post-neoadjuvant) US Enhertu DESTINY-Breast11 Neoadjuvant HER2+ Stage II or III breast cancer US Enhertu DESTINY-PanTumor02 / DESTINY-Lung01 / DESTINY-CRC02 HER2-positive solid tumours EU Etcamah (camizestrant) SERENA-6 ESR1m HR+ HER2- 1L locally advanced or metastatic breast cancer EU, JP Imfinzi POTOMAC NMIBC US Imfinzi MATTERHORN Resectable gastric/GEJ cancer JP Orphathys NCT04923932 3L+ MET+ gastric/GEJ cancer CN Truqap CAPItello-281 PTEN-deficient mHSPC US Baxfendy BaxHTN Hypertension US Fasenra NATRON Hypereosinophilic syndrome US, EU, JP, CN Regulatory submissions or acceptances* in major regions Medicine Trial Indication Region Baxfendy BaxHTN / Bax24 / BaxAsia Hypertension JP tozorakimab OBERON / TITANIA / MIRANDA / PROSPERO COPD EU, CN Ultomiris I CAN IgAN US, JP efzimfotase alfa MULBERRY / CHESTNUT / HICKORY HPP JP * US, EU and China regulatory entries in this table denote filing acceptance Other pipeline updates For recent trial starts and anticipated timings of key trial readouts, please refer to the Clinical Trials Appendix document in the financial results section of the AstraZeneca investor relations website: www.astrazeneca.com/investor-relations.html Table 2: Key elements of financial performance: Q2 2026 For the quarter Reported Change Core Change ended 30 June $m Act CER $m Act CER Product Revenue 15,384 6 5 15,384 6 5 * See Tables 3, 7, 23, 24 and 25 for further details of Product Revenue, Product Sales and Alliance Revenue Collaboration Revenue - n/m n/m - n/m n/m * See Tables 4 and 26 for further details of Collaboration Revenue Total Revenue 15,384 6 5 15,384 6 5 * See Tables 5 and 6 for Total Revenue by Therapy Area and by region Gross Margin (%) 84 +1pp - 84 +1pp +1pp + Variations in Gross Margin can be expected between periods due to various factors, including fluctuations in foreign exchange rates, product seasonality and Collaboration Revenue − Pricing headwinds, including those driven by loss of exclusivity and VBP in China R&D expense 4,053 14 13 3,662 6 5 * Core R&D: 24% of Total Revenue + Increasing number of trials, and patients in those trials + Investments in transformative technologies + Addition of R&D projects from business development + Positive data readouts for high value pipeline opportunities that have ungated large late-stage trials SG&A expense 5,651 16 14 4,050 7 4 * Core SG&A: 26% of Total Revenue + Investment to support ongoing and future launches Other operating income and expense4 152 92 93 152 >2x >2x + Various partner milestones Operating profit 3,164 (10) (13) 5,158 12 10 Operating Margin (%) 21 -4pp -4pp 34 +2pp +2pp Net finance expense 355 (4) (8) 340 13 8 + Lower interest income on short-term deposits − Reported Net finance expense benefitted from a lower discount unwind on contingent consideration liabilities Tax rate (%) 10 -11pp -11pp 15 -6pp -6pp − Benefit from adjustments to deferred tax assets, as a result of certain internal legal entity changes. * Variations in the tax rate can be expected between periods EPS ($) 1.61 2 (2) 2.63 21 18 For dollar values in this table, the unit of change is percent. For Gross Margin, Operating Margin and Tax rate, the unit of change is percentage points (pp). In the table above, R&D expense, SG&A expense and Net finance expense are displayed as positive numbers. The plus and minus symbols next to comments denote the directional impact of the item being discussed. For example, a plus symbol next to a comment about an R&D item indicates that the item increased R&D expenditure relative to the prior year period. Corporate and business development Dizal Pharmaceutical Co In July 2026, AstraZeneca entered into an exclusive license agreement with Dizal Pharmaceutical Co (Dizal), Ltd for Zegfrovy (sunvozertinib), a novel oral irreversible EGFR inhibitor for patients with lung cancer. AstraZeneca will acquire worldwide rights to develop and commercialise Zegfrovy, which is approved in the US and China for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, whose disease has progressed on or after platinum-based chemotherapy. AstraZeneca will make an upfront payment to Dizal of $600m and additional payments of up to $900m upon achievement of specific development, regulatory and sales-related milestones. Additionally, Dizal will receive tiered royalties on the global sales of Zegfrovy. The transaction is expected to close in the second half of 2026, subject to customary closing conditions and regulatory clearances. Sino Biopharmaceutical In July 2026, AstraZeneca and Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (CTTQ), a subsidiary of Sino Biopharmaceutical Limited, entered into an exclusive licence agreement for the development, manufacturing and commercialisation of CTTQ's PDE3/4 inhibitor, TQC3721, which is being developed for respiratory indications. Sino Biopharmaceutical Limited is eligible to receive an upfront payment of $200m, with additional development, regulatory and sales milestones up to $1.9bn, as well as tiered royalties ranging up to double-digit percentages based on the annual net sales of TQC3721 products. The agreement is subject to customary closing conditions, including regulatory clearances. Sustainability highlights In July 2026, AstraZeneca hosted a call for investors to discuss the latest developments in its Sustainability strategy. A replay of the call is available on astrazeneca.com. Reporting calendar The Company intends to publish its 9M and Q3 2026 results on 30 October 2026. Conference call A conference call and webcast for investors and analysts will begin today, 27 July 2026, at 11:45 UK time. Details can be accessed via astrazeneca.com. Notes Constant exchange rates. The differences between Actual Change and CER Change are due to foreign exchange movements between periods in 2026 vs. 2025. CER financial measures are not accounted for according to generally accepted accounting principles (GAAP) because they remove the effects of currency movements from Reported results. Core financial measures are adjusted to exclude certain items. The differences between Reported and Core measures are primarily due to costs relating to the amortisation of intangibles, impairments, legal settlements and restructuring charges. A full reconciliation between Reported EPS and Core EPS is provided in Tables 10 and 11 in the Financial Performance section of this document. The Company is unable to provide guidance on a Reported basis because it cannot reliably forecast material elements of the Reported results, including any fair value adjustments arising on acquisition-related liabilities, intangible asset impairment charges and legal settlement provisions. Please refer to the Cautionary statements section regarding forward-looking statements at the end of this announcement. Income from disposals of assets and businesses, where the Group does not retain a significant ongoing economic interest, is recorded in Other operating income and expense in the Group's financial statements. To read AstraZeneca's H1 & Q2 2026 Financial Results press release in full, read here .

License out/inFinancial StatementDrug ApprovalPhase 3Clinical Result

27 Jul 2026

·firstwordpharma.com

AstraZeneca looks to restore confidence following rare pipeline setbacks

Seeking to rebound from a surprise setback for one of its key pipeline assets, AstraZeneca on Monday — alongside reporting second-quarter financial results — reaffirmed confidence in its forecast for the current year, as well as its outlook through 2023. "We are on track to deliver our $80 billion total revenue ambition, which assumes successes and setbacks," said CEO Pascal Soriot, adding "we remain confident in the strength of our pipeline."AstraZeneca and partner Ionis Pharmaceuticals announced earlier this month that the Phase III CARDIO-TTRansform study of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet its primary endpoint, sending the UK drugmaker's shares down as much as 9%. For further analysis, read — Spotlight On: AstraZeneca investors wake up to surprise setback and Spotlight On: Alnylam's whiplash over AstraZeneca's TTRansformative miss.Expectations for CARDIO-TTRansform had been high, both from AstraZeneca and the scientific community. "It should have worked," Soriot said Monday, with the findings, showing that eplontersen — already on the market as Wainua and Wainzua — didn't work in conjunction with a stabiliser therapy, representing a "very important learning for the cardiology community." Separately on Monday, AstraZeneca reported a disappointing outcome for Ultomiris (ravulizumab) in adults and adolescents with thrombotic microangiopathy, as well as a positive Phase III read out for its Claudin 18.2-targeting antibody-drug conjugate sonesitatug vedotin in patients with advanced or metastatic gastric cancer. "Biology is biology, it doesn't always deliver what you expect," Soriot said.Oncology drugs growIn the second quarter, AstraZeneca reported that sales rose 6% to $15.4 billion, boosted by double-digit growth in oncology and rare diseases, which offset headwinds from loss of exclusivity to Farxiga in the US — overall revenue from the product slipped 16% to $1.8 billion.Quarterly sales of oncology drugs jumped 27% to $7.3 billion, with revenue from Imfinzi growing 12% to $1.9 billion and Enhertu surging 33% to $888 million. Net income in the three-month period lifted 2% to $2.5 billion.Commenting on the results, Shore Capital analyst Sean Conroy said "the balance between revenue growth and earnings progression is now less favourable than in prior years, given the increasing levels of R&D investment needed in order for it to grow beyond 2030."AstraZeneca also reiterated its outlook for the current year of a mid-to-high single-digit percentage increase in revenue, with core earnings per share growing by a low-double-digit percentage.

Phase 3Financial StatementADCClinical Result

100 Deals associated with Ravulizumab-CWVZ

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KEGG	Wiki	ATC	Drug Bank
-	Ravulizumab-CWVZ	L04AA43	DB11580

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Neuromyelitis Optica	European Union	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	Iceland	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	Liechtenstein	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	Norway	Alexion Europe SAS	12 Jul 2023
Thrombotic Microangiopathies	South Korea	AstraZeneca Korea Co. Ltd.	21 May 2020
AQP4-IgG positive Neuromyelitis optica spectrum disorder	Australia	Alexion Pharmaceuticals Australasia Pty Ltd.	17 Oct 2019
Myasthenia Gravis	Australia	Alexion Pharmaceuticals Australasia Pty Ltd.	17 Oct 2019
Atypical Hemolytic Uremic Syndrome	European Union	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	Iceland	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	Liechtenstein	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	Norway	Alexion Europe SAS	02 Jul 2019
Hemoglobinuria, Paroxysmal	United States	Alexion Pharmaceuticals, Inc.	21 Dec 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Henoch-Schonlein Purpura Nephritis	Phase 3	United States	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	China	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	Japan	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	Italy	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	South Korea	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	Spain	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Henoch-Schonlein Purpura Nephritis	Phase 3	Taiwan Province	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Purpura, Schoenlein-Henoch	Phase 3	United States	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Purpura, Schoenlein-Henoch	Phase 3	China	Alexion Pharmaceuticals, Inc.	14 Jun 2025
Purpura, Schoenlein-Henoch	Phase 3	Japan	Alexion Pharmaceuticals, Inc.	14 Jun 2025

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06578949 (CTgov)	Phase 3	Hemoglobinuria, Paroxysmal	18	Ravulizumab	mgvcijzcwo(nzzrcsguvu) = btecprntog ezygwvclrr (chgrdlosbu, twjkkbdpoj - qglpogusop) View more	-	05 Jun 2026
EHA2026	Not Applicable	Hemoglobinuria, Paroxysmal	1,328	Ravulizumab	- View more	Positive	12 May 2026
				Eculizumab
EHA2026	Not Applicable	Hemoglobinuria, Paroxysmal	53	Ravulizumab (Complement inhibitor-experienced patients)	pbcbpbrqmx(hozwlwnsvj) = jihzlmupdt qhrnjowqdd (wbxosjoibx ) View more	Positive	12 May 2026
				Ravulizumab (Complement inhibitor-naïve patients)	pbcbpbrqmx(hozwlwnsvj) = pnwdjmwonr qhrnjowqdd (wbxosjoibx ) View more
EHA2026	Not Applicable	Hemoglobinuria, Paroxysmal	10	EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan	cvzgervbns(cxcnuimtui) = During a median follow-up of 49.5 months, 3/10 patients required therapy modification due to persistent anemia compatible with EVH. tympagoyue (fvsomcmusn )	Positive	12 May 2026
NCT04557735 (ALXN1210-TMA-314, EBMT2026)	Phase 3	Thrombotic Microangiopathies	41	Ravulizumab plus best supportive care	arxkvfzolx(toxztknmtw) = fyjqhyshgm nfxeygzbao (qwuezdqafi ) View more	Positive	22 Mar 2026
EBMT2026	Not Applicable	Thrombotic Microangiopathies	3	Ravulizumab	qmmpxnlmxf(axwycublqi) = tkdgzxssmh xpeftzcdre (biuhqnilrx )	Positive	22 Mar 2026
NCT03920293 (CHAMPION MG, MDA2026)	Phase 3	Myasthenia Gravis	113	Ravulizumab	byohfgpwyr(atbarikppe) = ixwftaguyd rfdninsbgv (mkvzerydyz ) View more	Positive	08 Mar 2026
				Placebo	byohfgpwyr(atbarikppe) = qfrvrscgat rfdninsbgv (mkvzerydyz )
NCT04557735 (ALXN1210-TMA-314, Tandem Meetings ASTCT and CIBMTR2026)	Phase 3	Thrombotic Microangiopathies	41	Ravulizumab	asrwmkarvp(rfinbprquo) = btnmtbjozu rzmblifpsf (bxcsigftdq ) View more	Positive	04 Feb 2026
NCT04557735 (ALXN1210-TMA-314, CTgov)	Phase 3	Thrombotic Microangiopathies \| Hematopoietic stem cell transplantation	41	Ravulizumab (Ravulizumab)	afjlyrotvo = eaxvtydqzs etwjhzehzy (heqcqpihnf, istjzbjmwe - mnpsyruara) View more	-	12 Jan 2026
				Ravulizumab (Treatment Period: Ravulizumab)	iujynbtdii(gnphnolvpg) = dylpjmnwjp dvyxlfrucl (ibqlcsdbql, ohewcvfkpn - gvshhehdhh) View more
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	87	eculizumab	cbngbdoicj(twfytpijed) = xnaaxmfxob xbrfizqxct (hothpqoiph ) View more	Positive	06 Dec 2025
				ravulizumab	cbngbdoicj(twfytpijed) = rpfdyjkkht xbrfizqxct (hothpqoiph ) View more

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