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2016-02-19

Novartis International AG: Novartis drug PKC412 (midostaurin) receives Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute mye

Novartis International AG / Novartis drug PKC412 (midostaurin) receives
Breakthrough Therapy designationfrom the FDA for newly-diagnosed FLT3-mutated
acute myeloid leukemia (AML) . Processed and transmitted by NASDAQ OMX
Corporate Solutions.The issuer is solely responsible for the content of this
announcement.
* PKC412 (midostaurin) significantly improved overall survival of adult
patients eligible to receive standard induction and consolidation
chemotherapy
* AML has the lowest survival rate of all adult leukemias[1] and the
treatment strategy with chemotherapy has remained unchanged for more than
25 years[2],[3]
* Currently there are no approved targeted AML treatments; global regulatory
submissions for PKC412 (midostaurin) are on track to begin in H1 2016

Basel, February 19, 2016 -
Novartis announced today that the United States Food and Drug Administration
(FDA) has granted Breakthrough Therapy designation to PKC412 (midostaurin).
PKC412 (midostaurin) is an investigational treatment for adults with
newly-diagnosed AML who are FLT3 mutation-positive, as detected by an
FDA-approved test, and who are eligible to receive standard induction and
consolidation chemotherapy.

The Breakthrough Therapy designation for PKC412 (midostaurin) is primarily
based upon the positive results from the Phase III RATIFY (CALGB 10603)
clinical trial. This study was conducted in partnership with the Alliance for
Clinical Trials in Oncology and presented during a plenary session at the
57th American Society of Hematology (ASH) Annual Meeting[4].

Patients who received PKC412 (midostaurin) and standard induction and
consolidation chemotherapy experienced a significant improvement in overall
survival (OS) (hazard ratio = 0.77,P
= 0.0074) compared to those who received standard induction and consolidation
chemotherapy alone[4]. The median OS for patients in the PKC412 (midostaurin)
treatment group was 74.7 months (95% confidence interval [CI]: 31.7, not
attained), versus 25.6 months (95% CI: 18.6, 42.9) for patients in the
placebo group[4]. No statistically significant differences were observed in
the overall rate of grade 3 or higher hematologic and non-hematologic adverse
events in the PKC412 (midostaurin) treatment group versus the placebo
group[4]. A total of 37 deaths were reported, with no difference in
treatment-related deaths observed between groups[4].

"For more than 25 years, medical developments have been limited for AML
patients and the chemotherapy treatment strategy has essentially remained
unchanged," said Alessandro Riva, MD, Global Head, Novartis Oncology
Development and Medical Affairs. "We look forward to working closely with the
FDA to bring PKC412 (midostaurin), the first potential AML targeted therapy,
to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite
the development and review of new medicines that treat serious or
life-threatening conditions, if the therapy has demonstrated substantial
improvement over an available therapy on at least one clinically significant
endpoint. The designation includes all of the Fast Track program features, as
well as more intensive FDA guidance on an efficient drug development
program[5].

This designation adds to the growing number granted to Novartis by the FDA,
illustrating the company's continued commitment to developing innovative
therapies for diseases with a significant unmet medical need.

In the US, about 20,000 people were diagnosed with AML in 2015, the majority
of whom were adults[6]. According to the latest research, approximately
one-third of AML patients also harbor a FLT3 gene mutation[7], which is
associated with worse outcomes and shorter survival than in those without the
mutation[8]. PKC412 (midostaurin) is the first drug targeting FLT3 to
demonstrate an overall survival benefit in AML[4].

Since PKC412 (midostaurin) is investigational at this time and is expected to
be submitted for FDA approval, Novartis opened a Global Individual Patient
Program (compassionate use program) and a US Expanded Treatment Protocol
(ETP) to enable PKC412 (midostaurin) access. Patients 18 years of age and
older with newly-diagnosed FLT3-mutated AML and able to receive standard
induction and consolidation therapy will be considered.

In order to help identify patients who may have a FLT3 mutation and
potentially benefit from treatment with PKC412 (midostaurin), Novartis is
collaborating with Invivoscribe Technologies, Inc. who is leading regulatory
submissions for a companion diagnostic.

About acute myeloid leukemia (AML) and the FLT3 mutation

AML is an aggressive cancer of the blood and bone marrow[9]. It prevents white
blood cells from maturing, causing an accumulation of "blasts" which do not
allow room for the normal blood cells[9]. AML is the most common acute
leukemia in adults, but also has the lowest survival rate[1]. AML accounts
for approximately 25% of all adult leukemias worldwide, with the highest
incidence rates occurring in the United States, Europe and Australia[1].

Mutations in specific genes are found in many cases of AML, and biomarker
testing is considered standard of care for newly-diagnosed patients to help
determine the best possible treatment option[7]. FMS-like tyrosine kinase-3
(FLT3) is a receptor tyrosine kinase, a type of cell-surface receptor, which
plays a role in the proliferation, or increase, in the number of certain
blood cells[10].

About PKC412 (midostaurin)

PKC412 (midostaurin) is an investigational, oral, multi-targeted kinase
inhibitor in development for the treatment of patients with AML with a FLT3
mutation. The safety and efficacy profile has not been fully established.
There is no guarantee that PKC412 (midostaurin) will become commercially
available.

PKC412 (midostaurin) is also being investigated for the treatment of
aggressive systemic mastocytosis/mast cell leukemia.

Disclaimer

The foregoing release contains forward-looking statements that can be
identified by words such as "Breakthrough Therapy," "on track,"
"investigational," "look forward," "potential," "commitment," "expected,"
"will," "potentially," "being investigated," or similar terms, or by express
or implied discussions regarding potential marketing approvals for PKC412
(midostaurin), or regarding potential future revenues from PKC412. 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 PKC412 will be submitted or approved for sale
in any market, or at any particular time. Nor can there be any guarantee that
PKC412 will be commercially successful in the future. In particular,
management's expectations regarding PKC412 could be affected by, among other
things, the uncertainties inherent in research and development, including
unexpected clinical trial results and additional analysis of existing
clinical data; unexpected regulatory actions or delays or government
regulation generally; the company's ability to obtain or maintain proprietary
intellectual property protection; general economic and industry conditions;
global trends toward health care cost containment, including ongoing pricing
pressures; unexpected safety issues; unexpected manufacturing or quality
issues, and other risks and factors referred to in Novartis AG's current Form
20-F 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 contained
in this press release as a result of new information, future events or
otherwise.

About Novartis

Novartis provides innovative healthcare solutions that address the evolving
needs of patients and societies. Headquartered in Basel, Switzerland,
Novartis offers a diversified portfolio to best meet these needs: innovative
medicines, eye care and cost-saving generic pharmaceuticals. Novartis is the
only global company with leading positions in these areas. In 2015, the Group
achieved net sales of USD 49.4 billion, while R&D throughout the Group
amounted to approximately USD 8.9 billion (USD 8.7 billion excluding
impairment and amortization charges). Novartis Group companies employ
approximately 119,000 full-time-equivalent associates. Novartis products are
available in more than 180 countries around the world. For more information,
please visithttp://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis
athttp://twitter.com/novartis.

References

[1] Deschler B, Lübbert M. Acute myeloid leukemia: epidemiology and etiology.
Cancer. 2006;107(9):2009-2107.
[2] NCCN Clinical Practice Guidelines In Oncology (NCCN Guidelines®) Version
1. 2015 Acute Myeloid
Leukemia.http://www.nccn.org/professionals/physician_gls/pdf/aml.pdf.
Accessed October 2015.

[3] Lin TL, Levy MY. Acute myeloid leukemia: focus on novel therapeutic
strategies. Clinical Medicine Insights: Oncology. 2012;6:205-217.
[4] Stone RM, et al. The Multi-Kinase Inhibitor Midostaurin (M) Prolongs
Survival Compared with Placebo (P) in Combination with Daunorubicin
(D)/Cytarabine (C) Induction (ind), High-Dose C Consolidation (consol), and
As Maintenance (maint) Therapy in Newly Diagnosed Acute Myeloid Leukemia
(AML) Patients (pts) Age 18-60 with FLT3 Mutations (muts): An International
Prospective Randomized (rand) P-Controlled Double-Blind Trial (CALGB
10603/RATIFY [Alliance]). Presented at the 57th Annual Meeting of the
American Society of Hematology.
[5] US Food and Drug Administration. Frequently Asked Questions: Breakthrough
Therapies.http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmen....
Accessed February 2016.

[6] Siegel RL, Miller KD, Jemal A. Cancer statistics, 2015. Ca Cancer J Clin.
2015;65(1):5-29.
[7] Levis M. FLT3 mutations in acute myeloid leukemia: what is the best
approach in 2013? Hematology Am Soc Hematol Educ Program. 2013;2013:220-6.
[8] Yanada M, Matsuo K, Suzuki T, Kiyoi H, Naoe T. Prognostic significance of
FLT3 internal tandem duplication and tyrosine kinase domain mutations for
acute myeloid leukemia: a meta-analysis. Leukemia. 2005;19(8):1345-1349.
[9] National Institute of Health (NIH) National Cancer Institute (NCI). Adult
Acute Myeloid Leukemia Treatment
(PDQ®)http://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq.
Accessed February 2016.

[10] Gilliland DG, Griffin JD. The roles ...

Författare WKR

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