Press Release: Novartis delivers strong full year -3-

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R&D update - key developments from the fourth quarter

New approvals

Fabhalta Approved in the US as the first oral monotherapy for

(iptacopan) the treatment of adults (both previously treated and

treatment-naïve patients) with paroxysmal nocturnal

hemoglobinuria (PNH)

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Cosentyx Approved in the US as the first new biologic therapy

for the treatment of moderate to severe hidradenitis

suppurativa (HS) in adults in nearly a decade

Approved in the US as an intravenous formulation in

three indications: psoriatic arthritis, ankylosing

spondylitis, and non-radiographic axial SpA

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Results from ongoing trials and other highlights

Scemblix Ph3 ASC4FIRST study met both primary endpoints (major

(asciminib) molecular response rate vs. imatinib or investigator-selected

tyrosine kinase inhibitors) with clinically meaningful

and statistically significant results in newly diagnosed

patients with Philadelphia chromosome-positive chronic

myeloid leukemia in chronic phase (Ph+ CML-CP). Additionally,

Scemblix showed a favorable safety and tolerability

profile. Data will be presented at an upcoming medical

conference and submitted to regulatory authorities

in 2024

Ph3 ASCEMBL study, median follow-up of almost 4 years,

in patients with Ph+ CML-CP continue to support the

efficacy, safety and tolerability profile compared

with bosutinib in 3L+ setting. Data presented at ASH

2023

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Fabhalta Ph3 APPLAUSE-IgAN study interim analysis demonstrated

(iptacopan) clinically meaningful and highly statistically significant

proteinuria reduction in patients with IgA nephropathy.

The trial met its pre-specified interim analysis (9

months) primary endpoint, demonstrating superiority

vs. placebo in proteinuria reduction, with safety

consistent with previously reported data. Novartis

plans to review interim data with regulatory authorities

for accelerated approval; study continues with final

readout at 24 months

Ph3 APPEAR-C3G study met its primary endpoint, demonstrating

superiority of iptacopan vs placebo in proteinuria

reduction at six-month analysis and provided clinically

meaningful and statistically significant proteinuria

reduction in patients with C3G on top of background

therapy. Iptacopan's safety profile was consistent

with previously reported data. Data to be presented

at an upcoming medical meeting. Study continues with

all patients receiving active therapy for six-months

Ph3 APPLY-PNH extension data showed sustained efficacy

and long-term safety of Fabhalta in adults with paroxysmal

nocturnal hemoglobinuria (PNH). Data showed sustained

clinically meaningful hemoglobin-level increases to

near-normal (>=12 g/dL), blood transfusion avoidance,

and improved patient-reported fatigue in the majority

of patients. Comparable benefits were seen in those

patients switching from anti-C5 therapy to Fabhalta.

Safety profile at 48 weeks was similar to 24 week

data. Data presented at ASH 2023

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atrasentan Ph3 ALIGN study met its primary endpoint, demonstrating

superiority of atrasentan vs placebo in proteinuria

reduction at 36-week interim analysis with clinically

meaningful and highly statistically significant reduction

in proteinuria in IgAN patients receiving supportive

care. Safety profile of atrasentan was consistent

with previously reported data. Data to be presented

at an upcoming medical meeting. Study continues with

final readout expected in 2026

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remibrutinib Ph3 REMIX-1 and REMIX-2 trials showed clinically meaningful

and statistically significant reduction in weekly

urticaria activity (UAS7), itch (ISS7) and hives (HSS7)

at Week 12 vs placebo in patients with CSU. Significant

improvement in symptom control was seen as early as

Week 2 and sustained up to Week 12. Remibrutinib was

well-tolerated and demonstrated a favorable safety

profile with rates of overall adverse events comparable

to placebo and balanced liver function tests across

both studies. Studies are ongoing with final (52-week)

readout and regulatory submissions in 2024. Data presented

at AAAI 2023

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Kisqali Final protocol-specified iDFS analysis of Ph3 NATALEE

(ribociclib) trial (with a median follow-up of 33.3 months and

78.3% of patients having completed ribociclib) reinforces

25% reduction in risk of recurrence across broad population

of patients with HR+/HER2- early breast cancer and

continues to support regulatory submissions. iDFS

benefit remains consistent across key patient subgroups,

with stability in secondary endpoints including overall

survival (OS). Among patients with stage II and stage

III tumors, ribociclib lowered risk of disease recurrence

by 30% and 24.5%, respectively. Safety profile was

in line with previously reported results. Data presented

at SABCS 2023. NATALEE data submitted to the FDA in

December 2023

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Early-stage business development in core therapeutic Cardiovascular-Renal-Metabolic:

areas and technologies

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January 31, 2024 01:00 ET (06:00 GMT)