-- Phase II primary endpoint results for investigational iptacopan in IgAN 
      demonstrated effective and clinically meaningful reduction of 
      proteinuria1 -- a key risk predictor in kidney disease progression2 
 
   -- Iptacopan also showed a trend toward stabilization of kidney function1; 
      Phase III clinical trial APPLAUSE is underway 
 
   -- There are no currently approved treatments for IgAN -- a rare and often 
      progressive kidney disease that mainly affects young adults and can 
      progress to kidney failure3-7 
 
   -- Iptacopan is in development for several complement-driven renal diseases 
      (CDRDs), including IgAN and C3 glomerulopathy (C3G), and the blood 
      disorder paroxysmal nocturnal hemoglobinuria (PNH), targeting a key 
      driver of these diseases 
 
 
   Basel, June 06, 2021 -- Novartis today announced Phase II primary 
endpoint data showing investigational iptacopan (LNP023) -- a 
first-in-class, oral, targeted factor B inhibitor -- reduced protein in 
the urine (proteinuria), an increasingly recognized surrogate marker 
correlating with progression to kidney failure(2) , and showed promise 
in stabilizing kidney function in patients with IgA nephropathy 
(IgAN)(1) . The data were presented at the 58(th) ERA-EDTA Congress held 
virtually from June 5--8, 2021. 
 
   In the Phase II study (NCT03373461), patients (n=112) with IgAN were 
randomized to placebo or different doses of iptacopan(1) . The primary 
endpoint was met with a statistically significant (p=0.038) dose 
response effect on reduction in proteinuria (as measured by 24-hour 
urinary protein to creatine ratio [UPCR 24h]) with iptacopan vs. placebo, 
at 90 days(1) . At the highest dose of 200mg twice daily a 23% reduction 
in proteinuria was predicted, compared with placebo, at 90 days(1) . 
 
   "IgAN is a devastating disease with no currently approved treatments. 
These efficacy data, seen after 90 days of treatment, along with the 
safety profile, offer hope that inhibition of the alternative complement 
pathway with iptacopan may be an effective way to delay IgAN disease 
progression," said study lead author Jonathan Barratt, Professor of 
Renal Medicine, University of Leicester and nephrology consultant, 
Leicester General Hospital. "These data highlight the ability of 
iptacopan to address one of the key drivers for this disease and its 
potential to provide a much-needed, targeted treatment for people living 
with IgAN." 
 
   Iptacopan also demonstrated a trend towards stabilizing kidney function, 
as assessed by estimated glomerular filtration rate (eGFR)(1) : a key 
measure of kidney clearance function that estimates the rate of blood 
passing through and being filtered by the kidneys(8) . Additionally, 
iptacopan showed a favorable safety and tolerability profile(1) . 
 
   "Complement-driven renal diseases, such as IgAN, are devastating and 
mostly affect young adults, imposing a high disease burden. These new 
data in IgAN add to the growing body of evidence around the potential of 
iptacopan to target a key driver in these rare renal diseases," said 
John Tsai, Head of Global Drug Development and Chief Medical Officer at 
Novartis. "Conscious of the significant patient need for 
disease-modifying treatment options, we are rapidly advancing clinical 
development of iptacopan with the Phase III IgAN trial APPLAUSE already 
underway." 
 
   Iptacopan is the most advanced asset in the company's nephrology 
pipeline and targets the alternative complement pathway, a key driver of 
complement-driven renal diseases (CDRDs). New data from an interim 
analysis of a Phase II study of iptacopan in C3 glomerulopathy (C3G) -- 
another CDRD -- will also be presented at the congress on Monday 07 June 
2021 at 12:30 p.m. CEST(9) . Novartis has plans to initiate additional 
Phase III studies in other renal indications. 
 
   Iptacopan is also in development for a life-threatening blood disorder, 
paroxysmal nocturnal hemoglobinuria (PNH). Based on disease prevalence 
and positive data from Phase II studies, iptacopan has received EMA 
orphan drug designation in IgAN(1) (0) , orphan drug designations from 
the FDA and EMA in C3G and PNH(1) (1) , FDA Breakthrough Therapy 
Designation in PNH(1) (2) , and EMA PRIME designation for C3G(1) (3) . 
 
   About the studies 
 
   NCT03373461 is a Phase II randomized, double-blind, placebo-controlled, 
dose-ranging, parallel-group adaptive design study to investigate the 
efficacy and safety of iptacopan in primary IgAN(1) . It is the first 
study to report the efficacy and safety of selective inhibition of the 
alternative complement pathway in IgAN(1) . The primary endpoint, and 
the primary aim of the interim analysis presented at the 2021 ERA-EDTA 
Congress, was to evaluate the dose response effect of iptacopan versus 
placebo on the reduction in urinary protein to creatinine ratio (UPCR 
24h) at 90 days of treatment(1) . Secondary endpoints include safety and 
tolerability of iptacopan, eGFR, and biomarkers reflecting activity of 
the alternative complement pathway(1) . 
 
   About iptacopan 
 
   Iptacopan is an investigational, first-in-class, orally administered 
factor B inhibitor of the alternative complement pathway, targeting one 
of the key drivers of these diseases(14-16) . It has the potential to 
become the first targeted therapy to delay progression to dialysis in 
IgAN. Discovered at the Novartis Institutes for BioMedical Research, 
iptacopan is currently in development for a number of CDRDs where 
significant unmet needs exist, including IgAN, C3G, atypical hemolytic 
uremic syndrome (aHUS), and membranous nephropathy (MN), as well as the 
blood disorder PNH. 
 
   While Novartis has a 35-year history in kidney transplantation 
treatments, iptacopan is the first treatment in the nephrology pipeline 
addressing CDRDs. Our aim is to transform treatment by targeting one of 
the key drivers of these rare and often progressive diseases(15) and, in 
doing so, potentially extend dialysis-free life for people with CDRDs. 
 
   About complement-driven renal diseases (CDRDs) 
 
   CDRDs, which include IgAN, are thought to be partly caused by an 
overactivation of the alternative complement pathway -- part of the 
immune system -- creating an inflammatory response, which can lead to 
kidney damage(15) (,17-) (20) . CDRDs mainly affect young adults, and 
can often lead to kidney failure which requires dialysis or 
transplantation and can lead to premature death(3-7) . 
 
   IgAN patients with persistent proteinuria levels of >=1 g/day are at 
higher risk of disease progression, with about 30% progressing to kidney 
failure within 10 years(21-23) . Corticosteroids are often used to treat 
IgAN, as there are no approved treatment options. However, data on their 
efficacy have been inconsistent and this class of drugs has well-known 
side effects, which can be severe(24-26) . 
 
   There is a need for effective and well-tolerated, targeted therapies for 
IgAN that can delay disease progression. 
 
   Disclaimer 
 
   This press release contains forward-looking statements within the 
meaning of the United States Private Securities Litigation Reform Act of 
1995. Forward-looking statements can generally be identified by words 
such as "potential," "can," "will," "plan," "may," "could," "would," 
"expect," "anticipate," "seek," "look forward," "believe," "committed," 
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or implied discussions regarding potential marketing approvals, new 
indications or labeling for the investigational or approved products 
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from such products. You should not place undue reliance on these 
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this press release will be submitted or approved for sale or for any 
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on file with the US Securities and Exchange Commission. Novartis is 
providing the information in this press release as of this date and does 
not undertake any obligation to update any forward-looking statements 
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events or otherwise. 
 
   About Novartis 
 
   Novartis is reimagining medicine to improve and extend people's lives. 
As a leading global medicines company, we use innovative science and 
digital technologies to create transformative treatments in areas of

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