诺华 DM1 三期 HARBOR 失利，但管线与指引未被推翻

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6-K
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f6k_090826.htm
FORM 6-K
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 or 15d-16 OF
THE SECURITIES EXCHANGE ACT OF 1934
Report on Form 6-K dated September 8, 2026
(Commission File No. 1-15024)
____________________
Novartis AG
(Name of Registrant)
Lichtstrasse 35
4056 Basel
Switzerland
(Address of Principal Executive Offices)
____________________
Indicate by check mark whether the registrant files or will file annual reports under
cover of Form 20-F or Form 40-F:
Form 20-F: ☒
Form 40-F: ☐
Novartis
International AG
CH-4002
Basel
Switzerland
www.novartis.com
x.com/novartisnews
PRESS RELEASE
Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1)
Ad hoc announcement pursuant to Art. 53 LR
·
HARBOR did not meet its primary endpoint of vHOT in DM1, a progressive neuromuscular disease with high unmet need and no approved
treatment options
·
Del-desiran showed evidence of clinical activity in secondary endpoints and exploratory
analyses; full HARBOR dataset to be analyzed and appropriate development path to be determined
·
AOC pipeline continues to advance with FDA priority review granted for delpacibart zotadirsen in DMD44 and planned FDA meeting
for delpacibart braxlosiran in FSHD, based on positive Phase I/II biomarker data
·
Novartis maintains its 5-6% five-year sales CAGR guidance for 2025-2030
Basel, September 8, 2026
– Novartis today announced that the global Phase III HARBOR
study evaluating del-desiran in people living with myotonic dystrophy type 1 (DM1) did not demonstrate statistically significant
improvement versus placebo on the primary endpoint of video hand opening time (vHOT), a novel measure of hand myotonia.
1,2
Evidence of clinical activity in secondary endpoints and exploratory analyses were observed. Safety findings from HARBOR were generally
consistent with previously reported data. Novartis is evaluating the full HARBOR dataset and will engage with health authorities to determine
the most appropriate development path for del-desiran.
“Despite decades of research, there are still no approved treatment options for DM1, and
patients and caregivers continue to face a significant daily burden,” said Shreeram Aradhye, President, Development and Chief Medical
Officer, Novartis. “Developing therapies for a complex disease like DM1 remains challenging, and setbacks are part of scientific
progress. As we continue to evaluate the full HARBOR dataset, we remain committed to identifying the most appropriate development path
for the del-desiran program and advancing innovative approaches for people living with DM1 and other serious neuromuscular diseases.”
Del-desiran is one of three antibody oligonucleotide conjugate (AOC) therapies
added to the Novartis neuromuscular pipeline through the acquisition
of Avidity Biosciences. Novartis
is advancing delpacibart zotadirsen (del-zota) in patients with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping
(DMD44). The company filed del-zota for accelerated approval and was granted priority review designation by US Food and Drug Administration
(FDA). Novartis is planning to meet with the FDA on next steps for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular
dystrophy (FSHD) based on recent
positive Phase I/II biomarker data
.
About DM1
Myotonic dystrophy type 1 (DM1) is a progressive, multisystem, heterogeneous neuromuscular
disease caused by an expansion of CTG repeats in the DM1 protein kinase (
DMPK
) gene.
3,4,5
People living with
DM1 may experience a range of internal and systemic symptoms, including myotonia, muscle weakness and impaired hand function,
which can affect everyday activities, independence, and quality of life.
3
About Del-desiran
Del-desiran is an investigational antibody oligonucleotide conjugate (AOC) designed to
target the underlying cause of DM1. The therapy consists of a muscle-targeting monoclonal antibody that binds to the transferrin receptor
1 (TfR1) and is conjugated to a small interfering RNA (siRNA) designed to induce degradation of disease-causing
toxic DMPK messenger RNA (mRNA).
6
Del-desiran received Orphan Drug, Fast Track and Breakthrough Therapy Designations
from the US Food and Drug Administration (FDA), and Orphan Medicinal Product Designation in the European Union (EU).
About the HARBOR study
HARBOR (NCT06411288) is a global Phase III, randomized, double-blind, placebo-controlled study evaluating
the efficacy and safety of del-desiran over 54 weeks in approximately 150 people living with DM1. Participants
were randomized to receive del-desiran or placebo every eight weeks. The study assesses the impact of del-desiran across
multiple functional aspects of DM1. The primary endpoint is vHOT and key secondary endpoints include muscle
strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, activities of daily living as measured by
DM1-Activ, and mobility and physical function as measured by the 10-meter walk/run test (10mWRT).
1
Novartis in Neuroscience
Neurological diseases are deeply personal, affecting people of any age, from newborns to seniors,
often striking in the prime of life. At Novartis, we are doubling down on our commitment to neurology, expanding our legacy of innovation
in spinal muscular atrophy (SMA) and multiple sclerosis (MS) to work in neuroimmunology, neurodegeneration, and neuromuscular diseases.
Our goal is to protect people’s health across their lifespan, developing more treatment options that lead to better outcomes.
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,” “progressive,” “continues,” or similar
expressions, or by express or implied discussions regarding: potential new products including del-desiran, del-zota or del-brax; potential
new indications for existing products; potential product launches or potential future revenues from del-desiran, del-zota or del-brax;
results of ongoing clinical trials; or potential future, pending or announced transactions; or potential future sales or earnings from
del-desiran, del-zota or del-brax. You should not place undue reliance on these statements. Such forward-looking statements are based
on the current beliefs and expectations of management regarding future events, and are subject to significant known
and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions
prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no
guarantee that del-desiran, del-zota or del-brax will be submitted or approved for sale or for any additional indications
or labeling in any market, or at any particular time. Nor can there be any guarantee that del-desiran, del-zota or del-brax will
be commercially successful in the future. In particular, our expectations could be affected by, among other things, uncertainties
concerning: global healthcare cost containment, including ongoing government, payer and general public pricing and reimbursement pressures
and requirements for increased pricing transparency; the success of our key products, commercial priorities and strategy; research
and development of new products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain
or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent
protection and exclusivity on key products; our ability to realize the strategic benefits, operational efficiencies or opportunities expected
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Form 20-F and in subsequent reports filed with, or furnished to, 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 as
a result of new information, future events or otherwise.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine
to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face
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References
1.
ClinicalTrials.gov. Available at: https://clinicaltrials.gov/study/NCT06411288. Accessed August 2026.
2.
Duong T, de Monts C, McIntyre M, et al. 444P Exploring hand myotonia: assessing hand opening and individual finger movements through
machine learning. Neuromuscular Disorders. 2024;43(Suppl 1):104441.
3.
Muscular Dystrophy Association. Myotonic Dystrophy (DM). Available at: https://www.mda.org/disease/myotonic-dystrophy. Accessed August 2026.
4.
Kwan TT, Meng Q, Delos Santos N, et al. Delpacibart etedesiran improves the molecular pathology of myotonic dystrophy type 1
in the phase 1/2 MARINA study. Molecular Therapy. 2026;34(5). doi:10.1016/j.ymthe.2026.03.013.
5.
National Human Genome Research Institute. About Myotonic Dystrophy. Available at: https://www.genome.gov/Genetic-Disorders/Myotonic-Dystrophy.
Accessed August 2026.
6.
Johnson NE, Tai LJ, Hamel JI, et al. An antibody-oligonucleotide conjugate for myotonic dystrophy type 1. N Engl J
Med. 2026;394:717-730. PMID: 41707138.
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SIGNATURES
Pursuant to
the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
Novartis AG
Date: September 8, 2026
By:
/s/ PAUL
PENEPENT
Name:
Paul Penepent
Title:
Head of Financial Reporting and Accounting