Press Release: FDA approves Novartis Piqray(R) - the first and only treatment specifically for patients with a PIK3CA mutation in HR+/HER2- advanced breast c...

Novartis International AG / FDA approves Novartis Piqray(R) - the first

and only treatment specifically for patients with a PIK3CA mutation in

HR+/HER2- advanced breast cancer. Processed and transmitted by West

Corporation. The issuer is solely responsible for the content of this

announcement.

-- Piqray (alpelisib, formerly BYL719) plus fulvestrant nearly doubled

median PFS (11.0 vs 5.7 months) in HR+/HER2- advanced breast cancer

patients with a PIK3CA mutation compared to fulvestrant alone in the

SOLAR-1 clinical trial[1],[2],[3],[4]

-- 40% of HR+/HER2- advanced breast cancer patients may face worse disease

prognosis due to presence of PIK3CA mutations in their tumors[5],[6],[7],

[8],[9]

-- Piqray was the first new drug application approved under the FDA Oncology

Center of Excellence Real-Time Oncology Review pilot program

The digital press release with multimedia content can be accessed here:

https://novartis.gcs-web.com/FDA-approves-Novartis-Piqray-the-first-and-only-treatment-specifically-for-patients-with-a-PIK3CA-mutation-in-HRHER2-advanced-breast-cancer

Basel, May 24, 2019 - Novartis today announced the US Food and Drug

Administration (FDA) has approved Piqray(R) (alpelisib, formerly BYL719)

in combination with fulvestrant for the treatment of postmenopausal

women, and men, with hormone receptor positive, human epidermal growth

factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or

metastatic breast cancer, as detected by an FDA-approved test following

progression on or after an endocrine-based regimen[1].

PIK3CA is the most commonly mutated gene in HR+/HER2- breast cancer;

approximately 40% of patients living with HR+/HER2- breast cancer have

this mutation[8],[10]. PIK3CA mutations are associated with tumor growth,

resistance to endocrine treatment and a poor overall prognosis[11],[12].

Piqray targets the effect of PIK3CA mutations and may help overcome

endocrine resistance in HR+ advanced breast cancer.

"The FDA approval of Piqray, which was discovered at the Novartis

Institutes for BioMedical Research, marks the first ever treatment

specifically for HR+/HER2- advanced breast cancer with a PIK3CA

mutation. We are proud to offer a new treatment option that specifically

addresses the needs of the patients living with this mutation," said

Susanne Schaffert, PhD, CEO, Novartis Oncology. "We are grateful to our

researchers' bold and unrelenting pursuit of a first-in-class treatment

for this incurable disease, and to the patients, investigators and

administrators who participated in the clinical trials leading to this

remarkable milestone."

FDA approval is based on results of the Phase III trial, SOLAR-1, that

showed Piqray plus fulvestrant nearly doubled median progression-free

survival (PFS) compared to fulvestrant alone in HR+/HER2- advanced

breast cancer patients with a PIK3CA mutation (median PFS 11.0 months vs

5.7 months; HR=0.65, 95% CI: 0.50-0.85; p<0.001)[2]. Piqray provided

consistent PFS results across pre-specified subgroups, including among

patients previously treated with a CDK4/6 inhibitor[2],[3].

Overall response rate (ORR), an indicator of the proportion of patients

who experience at least a 30% reduction in overall tumor size (in

patients with measurable disease), was more than doubled when Piqray was

added to fulvestrant in patients with a PIK3CA mutation, (ORR= 35.7% vs

16.2% for fulvestrant alone, p=0.0002)[2]. Piqray and its associated

companion diagnostic test from QIAGEN N.V. was the first combination

product approved under the FDA Oncology Center of Excellence Real-Time

Oncology Review pilot program.

"Today's approval is expected to change the way we practice medicine in

advanced breast cancer. For the first time, physicians can test for

PIK3CA biomarkers and develop a treatment plan based on the genomic

profile of a patient's cancer," said Fabrice André, MD, PhD,

research director and head of INSERM Unit U981, professor in the

Department of Medical Oncology at Institut Gustave Roussy in Villejuif,

France, and global SOLAR-1 principal investigator. "In the SOLAR-1 Phase

III trial, alpelisib plus fulvestrant nearly doubled median PFS and more

than doubled overall response rate in patients with a PIK3CA mutation,

offering them new hope for longer life without progression."

Patients with HR+/HER2- advanced breast cancer can be selected for

treatment with Piqray based on the presence of PIK3CA mutations.

Concurrent with the approval of Piqray, the therascreen(R) * PIK3CA

companion diagnostic test from QIAGEN was also approved by the FDA and

is now available for patient testing.

"If you are facing a complex disease like metastatic breast cancer, you

want to follow a path that is specific to your type of disease," said

Shirley Mertz, President, Metastatic Breast Cancer Network. "Finding the

right treatment team and getting the right tests, like testing for the

PIK3CA mutation, will help your healthcare team identify accurate,

precise treatment options for your disease."

Novartis is committed to providing patients with access to medicines, as

well as resources and support to address a range of needs. The Novartis

Oncology Patient Support Program is available to help guide eligible

patients through the various aspects of getting started on treatment,

from providing educational information to helping them understand their

insurance coverage and identify potential financial assistance options.

For more information, patients and healthcare professionals can call

1-800-282-7630.

Full prescribing information for Piqray can be found at

https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/piqray.pdf.

About Piqray(R) (alpelisib)

Piqray is a kinase inhibitor approved in combination with fulvestrant

for the treatment of postmenopausal women, and men, with HR+/HER2-,

PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an

FDA-approved test following progression on or after endocrine-based

regimen[1].

Approximately 40% of HR+ advanced breast cancer patients have a mutation

that may activate the PI3K-alpha isoform, called PIK3CA mutations[5],[6],

[7],[8]. These mutations are associated with resistance to endocrine

therapy, disease progression and a poor prognosis[11],[12]. Piqray works

by inhibiting the PI3K pathway, predominantly the PI3K-alpha isoform, to

address the effect of PIK3CA mutations.

About SOLAR-1

SOLAR-1 is a global, Phase III randomized, double-blind,

placebo-controlled trial studying Piqray in combination with fulvestrant

for postmenopausal women, and men, with PIK3CA-mutated HR+/HER2-

advanced or metastatic breast cancer that progressed on or following

aromatase inhibitor treatment with or without a CDK4/6 inhibitor[1],[2],

[3]. SOLAR-1 is the pivotal Phase III trial that supported this

approval.

The trial randomized 572 patients. Patients were allocated based on

central tumor tissue assessment to either a PIK3CA-mutated cohort

(n=341) or a PIK3CA non-mutated cohort (n=231). Within each cohort,

patients were randomized in a 1:1 ratio to receive continuous oral

treatment with Piqray (300 mg once daily) plus fulvestrant (500 mg every

28 days + Cycle 1 Day 15) or placebo plus fulvestrant. Stratification

was based on visceral metastases and prior CDK4/6 inhibitor treatment[1],

[2],[3]. Patients and investigators are blinded to PIK3CA mutation

status and treatment.

The primary endpoint is local investigator assessed PFS using RECIST 1.1

for patients with a PIK3CA mutation. The key secondary endpoint is

overall survival, and additional secondary endpoints include, but are

not limited to, overall response rate, clinical benefit rate,

health-related quality of life, efficacy in PIK3CA non-mutated cohort,

safety and tolerability[1],[2],[3]. SOLAR-1 is ongoing to assess overall

survival and other secondary endpoints.

About Novartis in Advanced Breast Cancer

For more than 30 years, Novartis has been tackling breast cancer with

superior science, great collaboration and a passion for transforming

patient care. With one of the most diverse breast cancer pipelines and

one of the largest numbers of breast cancer compounds in development,

Novartis leads the industry in discovery of new therapies and

combinations, especially in HR+ advanced breast cancer, the most common

form of the disease.

Indication

PIQRAY(R) (alpelisib) tablets is a prescription medicine used in

combination with the medicine fulvestrant to treat women who have gone

through menopause and men who have hormone receptor (HR)-positive, human

epidermal growth factor receptor 2 (HER2)-negative advanced breast

cancer or breast cancer that has spread to other parts of the body

(metastatic), with an abnormal phosphatidylinositol-3-kinase catalytic

subunit alpha (PIK3CA) gene, and whose disease has progressed on or

after endocrine therapy. Your health care provider will test your cancer

for an abnormal "PIK3CA" gene to make sure that PIQRAY is right for you.

It is not known if PIQRAY is safe and effective in children.

Important Safety Information

Patients should not take PIQRAY if they have had a severe allergic

reaction to PIQRAY or are allergic to any of the ingredients in PIQRAY.

PIQRAY may cause serious side effects. PIQRAY can cause severe allergic

reactions. Patients should tell their health care provider or get

medical help right away if they have trouble breathing, flushing, rash,

fever, or fast heart rate during treatment with PIQRAY. PIQRAY can cause

severe skin reactions. Patients should tell their health care provider

or get medical help right away if they get severe rash or rash that

keeps getting worse, reddened skin, flu-like symptoms, blistering of the

lips, eyes or mouth, blisters on the skin or skin peeling, with or

without fever. PIQRAY can cause high blood sugar levels (hyperglycemia).

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