REG - AstraZeneca PLC - I CAN PhIII interim analysis met primary endpoint

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RNS Number : 2013B  AstraZeneca PLC  21 April 2026

21 April 2026

 

Ultomiris demonstrated statistically significant and clinically meaningful
reduction of proteinuria in adults with immunoglobulin A nephropathy in

I CAN Phase III trial

 

Ultomiris delivered rapid reduction in proteinuria as early as week 10

 

Results show potential for terminal C5 complement inhibition with Ultomiris as
a disease-modifying treatment option for IgAN

 

Positive high-level results from a prespecified interim analysis of the I CAN
Phase III trial showed that Ultomiris (ravulizumab) met its primary endpoint,
demonstrating a statistically significant and clinically meaningful reduction
of proteinuria, based on 24-hour urine protein creatinine ratio (UPCR), at
week 34 in adults with immunoglobulin A nephropathy (IgAN) who are at risk of
disease progression. The primary endpoint of change from baseline in estimated
glomerular filtration rate (eGFR) will be measured at week 106.

 

IgAN is a rare, inflammatory disease of the kidneys that can lead to chronic
kidney disease (CKD) and progress to end-stage kidney disease (ESKD). It
begins when the body develops abnormal IgA proteins resulting in immune
complexes that are deposited in the kidneys causing damage. The deposition of
these complexes activates the complement system, leading to terminal
complement-driven inflammation. This results in damage and loss of essential
parts of the kidney, including cells in the glomeruli, the part of the kidneys
that filters and cleans the blood. Over time, this damage impacts the ability
of the kidneys to function properly.(1)

 

More than 560,000 people are diagnosed with IgAN in the US, EU5 and Japan, of
which more than 60 percent are eligible for IgAN treatment.(2-5)

 

Jonathan Barratt, MD, Mayer Professor of Renal Medicine, University of
Leicester, United Kingdom, and I CAN trial investigator, said: "Many people
living with IgAN continue to progress to kidney failure, ultimately requiring
dialysis or a transplant - outcomes that can place profound burden on
patients' daily lives - despite advances in care. The interim I CAN results
demonstrate that blocking terminal complement activation, a central driver of
kidney inflammation in IgAN, with Ultomiris may play a promising role in
reducing proteinuria. We look forward to understanding the full clinical
impact of Ultomiris in treating this disease following study completion at two
years."

 

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease,
said: "These positive data demonstrate that C5 complement inhibition with
Ultomiris results in a rapid and clinically meaningful reduction in
proteinuria as early as week 10 and underscores its potential as a
disease-modifying approach in IgAN. We look forward to filing these data with
regulatory authorities in key regions, while in parallel, advancing this Phase
III trial towards completion."

 

The safety profile observed in this trial was consistent with the known
profile of Ultomiris, with no new safety concerns identified.

 

The company will seek accelerated approval in key markets and will present
these results at a forthcoming medical meeting.

 

Notes

 

Immunoglobulin A Nephropathy (IgAN)

Immunoglobulin A nephropathy (IgAN) is a rare, inflammatory disease of the
kidneys that can lead to chronic kidney disease (CKD) and progress to
end-stage kidney disease (ESKD). It begins when the body develops abnormal IgA
proteins resulting in immune complexes that are deposited in the kidneys
causing damage. The deposition of these complexes activates the complement
system, leading to terminal complement-driven inflammation. This results in
damage and loss of essential parts of the kidney, including cells in the
glomeruli, the part of the kidneys that filters and cleans the blood. Over
time, this damage impacts the ability of the kidneys to function properly,
resulting in chronic kidney disease that can progress to end-stage kidney
disease.(1)

 

The signs and symptoms of IgAN can include blood in the urine (haematuria),
foamy urine (proteinuria), swelling in hands and feet (oedema) and high blood
pressure (hypertension).(6) Most people with IgAN do not experience symptoms
in the early stages of the disease, and therefore, it often goes undetected
until it has progressed. At diagnosis, irreversible kidney damage may have
already occurred.(5,7) Approximately half of people with IgAN who have
elevated protein levels in urine or reduced kidney function are at-risk of
progression to ESKD, or kidney failure, within 10 years of diagnosis.(8)

 

I CAN (ALXN1210-IgAN-320)

I CAN (ALXN1210-IgAN-320) is a global, Phase III, randomised, double-blind,
placebo-controlled trial evaluating the efficacy and safety of Ultomiris in
adults with immunoglobulin A nephropathy (IgAN) who are at risk of disease
progression. Participants were on stable concomitant IgAN treatment(s)
consistent with standard of care for at least three months prior to
screening.(9)

 

Participants were randomised 1:1 to receive either Ultomiris or placebo,
administered intravenously for a total of 106 weeks. Patients in the treatment
arm received a loading dose of Ultomiris on Day 1, followed by regular
weight-based maintenance dosing of Ultomiris beginning on Day 15 and then
every eight weeks through the 106-week blinded treatment period. Patients who
completed the randomised control period had the option to enter an open-label
access period.(9)

 

The primary endpoints are change from baseline in proteinuria based on 24-hour
urine protein creatinine ratio (UCPR) at week 34 and change from baseline in
estimated glomerular filtration rate (eGFR) at week 106, assessed at the
interim analysis and final analysis, respectively. Key secondary endpoints for
the final analysis include reduction in 24-hour UPCR ≥ 50% from baseline at
week 34, change from baseline in proteinuria based on 24-hour UPCR at week 10,
time to sustained ≥ 30% eGFR decline up to week 106 and time to first
occurrence of composite kidney event up to week 106. The trial was designed to
enrol approximately 510 participants from 28 countries across North America,
South America, Europe, Asia and Australia.(9)

 

Ultomiris

Ultomiris (ravulizumab), the longest-acting C5 complement inhibitor, provides
immediate, complete and sustained complement inhibition. The medication works
by inhibiting the C5 protein in the terminal complement cascade, a part of the
body's immune system. When activated in an uncontrolled manner, the complement
cascade over-responds, leading the body to attack its own healthy
cells. Following a loading dose, Ultomiris is administered intravenously
every eight weeks in adults, or every four or eight weeks in paediatric
patients (based on body weight).

 

Ultomiris is approved in the US, EU, Japan and other countries for the
treatment of certain adults with paroxysmal nocturnal haemoglobinuria (PNH)
and is also approved for certain children with PNH in the US, EU and other
countries.

 

Ultomiris is also approved in the US, EU, Japan and other countries for the
treatment of certain adults and children with atypical haemolytic uraemic
syndrome (aHUS).

 

Additionally, Ultomiris is approved in the US, EU, Japan, China and other
countries for the treatment of certain adults with generalised myasthenia
gravis (gMG).

 

Further, Ultomiris is approved in the US, EU, Japan, China and other countries
for the treatment of certain adults with neuromyelitis optica spectrum
disorder (NMOSD).

 

Ultomiris is being assessed as a treatment for additional indications as part
of a broad development programme.

 

Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients and families
affected by rare diseases and devastating conditions through the discovery,
development and delivery of life-changing medicines. A pioneering leader in
rare disease for more than three decades, Alexion was the first to translate
the complex biology of the complement system into transformative medicines,
and today it continues to build a diversified pipeline across disease areas
with significant unmet need, using an array of innovative modalities. As part
of AstraZeneca, Alexion is continually expanding its global geographic
footprint to serve more rare disease patients around the world. It is
headquartered in Boston, US.

 

AstraZeneca (https://www.astrazeneca.com/)

AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical
company that focuses on the discovery, development, and commercialisation of
prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals,
including Cardiovascular, Renal & Metabolism, and Respiratory &
Immunology. Based in Cambridge, UK, AstraZeneca's innovative medicines are
sold in more than 125 countries and used by millions of patients worldwide.
Please visit astrazeneca.com (http://www.astrazeneca.com/) and follow the
Company on Social Media @AstraZeneca
(https://www.linkedin.com/company/astrazeneca) .

 

Contacts
For details on how to contact the Investor Relations Team, please click here
(https://www.astrazeneca.com/investor-relations.html#Contacts) . For Media
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.

 

References

1.   Cheung CK, et al. The pathogenesis of IgA nephropathy and implications
for treatment. Nat Rev Nephrol. 2025;21(1):9-23.
doi:10.1038/s41581-024-00885-3. Epub 2024 Sep 4.

2.   DeCongelio M, et al. The incidence and prevalence of immunoglobulin A
nephropathy in the United States. Clin Nephrol. 2025;103(1):19-25.

3.   Buisker J, et al. The prevalence of immunoglobulin A nephropathy in the
European Union and the impact of the COVID-19 pandemic: an estimation approach
utilizing the kidney biopsy frequency. Clin Kidney J. 2025; 18(4):sfaf068.

4.   Sugiyama H, et al. Japan Renal Biopsy Registry and Japan Kidney Disease
Registry: Committee Report for 2009 and 2010. Clin Exp Nephrol. 2013;17(2):
155-173.

5.   Kidney Disease: Improving Global Outcomes (KDIGO) IgAN and IgAV Work
Group, et al. KDIGO 2025 Clinical Practice Guideline for the Management of
Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV).
Kidney Int. 2025; 108(4S): S1-S71.

6.   Rajasekaran A, et al. IgA Nephropathy: An Interesting Autoimmune Kidney
Disease. Am J Med Sci. 2021; 361(2):176-194.

7.   Stamellou E, et al. IgA nephropathy. Nat Rev Dis Primers. 2023;9(1):67.
doi:10.1038/s41572-023-00476-9.

8.   Wong K, et al. Effects of rare kidney diseases on kidney failure: a
longitudinal analysis of the UK National Registry of Rare Kidney Diseases
(RaDaR) cohort. Lancet. 2024;403(10433):1279-1289.

9.   ClinicalTrials.gov. A Phase 3, Randomized, Double-Blind,
Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in
Adult Participants With Immunoglobulin A Nephropathy (IgAN). NCT Identifier:
NCT06291376. Available here (https://clinicaltrials.gov/study/NCT06291376) .
Accessed April 2026.

 

 

Matthew Bowden

Company Secretary

AstraZeneca PLC

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