Press Release: Novartis continues strong momentum -3-

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New approvals

Scemblix FDA granted accelerated approval to Scemblix for adult

(asciminib) patients with newly diagnosed Philadelphia chromosome-positive

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

The FDA also broadened the indication for Scemblix

to include adult patients with previously treated

Ph+ CML-CP.

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Kisqali EC approved Kisqali as an adjuvant treatment in combination

(ribociclib) with an aromatase inhibitor for patients with HR+/HER2-

early breast cancer (eBC) at high risk of recurrence

regardless of nodal status, nearly doubling the eligible

population.

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Regulatory updates

Fabhalta Submission for the treatment of C3 glomerulopathy

(iptacopan) (C3G) was completed in the US, and the FDA granted

Priority Review status to Fabhalta in this indication.

The FDA also confirmed no need for an Advisory Committee

meeting.

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Results from ongoing trials and other highlights

OAV101 IT Novartis announced positive topline results from the

(onasemnogene abeparvovec) Phase III STEER study. This pivotal trial assessed

the efficacy and safety of investigational intrathecal

OAV101 in treatment-naïve patients with spinal

muscular atrophy (SMA) Type 2, aged two to less than

18 years who are able to sit but have never walked

independently. The study met its primary endpoint

showing an increase from baseline across the study

population in total Hammersmith Functional Motor Scale

- Expanded (HFMSE) scores. HFMSE is a gold standard

for SMA-specific assessment of motor ability and disease

progression.

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Pluvicto Final overall survival (OS) analysis in the Phase

(lutetium Lu177 vipivotide tetraxetan) III PSMAfore study in pre-taxane mCRPC demonstrated

an OS hazard ratio less than 1.0 (HR<1.0) in the intent-to-treat

(ITT) population unadjusted for cross-over. These

results have been shared with the FDA as part of their

ongoing review of Pluvicto in this indication.

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Kisqali Results from an updated analysis of the pivotal Phase

(ribociclib) III NATALEE trial of Kisqali plus endocrine therapy

(ET) in patients with HR+/HER2- stage II and III eBC

showed a sustained reduction in distant recurrence

of 28.5% compared to ET alone, underscoring Kisqali's

extended efficacy beyond its 3-year treatment duration.

No new safety signals were identified. Data presented

at SABCS.

In addition, Kisqali was recognized by NCCN Guidelines(R)

as a Category 1 preferred therapy in combination with

an aromatase inhibitor for patients with HR+/HER2-

eBC. Kisqali is the only Category 1 preferred CDK4/6

inhibitor recommended for both all node-positive disease

as well as node-negative disease with high-risk disease

characteristics. Kisqali also achieved the highest

score (A) on the European Society for Medical Oncology-Magnitude

of Clinical Benefit Scale (ESMO-MCBS) for eBC, while

maintaining a rating of 5 and 4 in the mBC setting.

In January 2025, Novartis settled compound patent

litigation with a generic manufacturer, supporting

Kisqali US patent protection until at least Q1 2031.

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Scemblix 96-week results from the Phase III ASC4FIRST trial

(asciminib) with Scemblix showed sustained superior major molecular

response vs. all investigator-selected standard-of-care

TKIs (imatinib, nilotinib, dasatinib and bosutinib)

and vs. imatinib alone in adult patients with newly

diagnosed Ph+ CML-CP. Fewer treatment-related grade

>=3 AEs and half the rate of AEs leading to treatment

discontinuation were reported for Scemblix vs. both

imatinib and second-generation TKIs. Data presented

at ASH.

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Fabhalta In the Phase III APPEAR-C3G study, patients with C3G

(iptacopan) treated with oral Fabhalta in addition to supportive

care experienced clinically meaningful proteinuria

reduction sustained at 12 months. In addition, in

the open-label period of the study, proteinuria reduction

was seen in participants switched to Fabhalta, and

improvement in estimated glomerular filtration rate

(eGFR) slope was observed upon Fabhalta initiation

compared to patients' historic rapid decline. Fabhalta

continued to show a favorable safety profile. Data

presented at ASN.

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Selected transactions Novartis entered into a global license and collaboration

agreement with PTC Therapeutics for PTC518, a HTT

mRNA splice modulator with the potential to become

the first oral disease-modifying therapy for Huntington's

disease. Under the agreement, Novartis will assume

responsibility for PTC518's development, manufacturing

and commercialization following the completion of

the placebo-controlled portion of the ongoing Phase

II PIVOT-HD study, expected in H1 2025.

Novartis acquired Kate Therapeutics, a preclinical-stage

biotechnology company focused on developing adeno-associated

virus (AAV)-based gene therapies to treat genetically

defined neuromuscular diseases. The acquisition will

strengthen Novartis' efforts to advance gene therapies

and neuroscience innovation and includes enabling

technology platforms and several preclinical therapeutic

candidates.

Novartis entered into a worldwide licensing and collaboration

agreement with Ratio Therapeutics for a next-generation

SSTR2-targeting radiotherapeutic candidate. The collaboration

focuses on preclinical research and selection of an

SSTR2-targeting development candidate, after which

Novartis will lead development, manufacturing and

commercialization.

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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.

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January 31, 2025 01:00 ET (06:00 GMT)