Array BioPharma Reports Financial Results For The First Quarter Of Fiscal 2017

On November 1, 2016 Array BioPharma Inc. (Nasdaq: ARRY), a biopharmaceutical company focused on the discovery, development and commercialization of targeted small molecule cancer therapies, reported results for its first quarter of fiscal 2017 and provided an update on the progress of its key clinical development programs (Press release, Array BioPharma, NOV 1, 2016, View Source;p=RssLanding&cat=news&id=2217854 [SID1234516133]).

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Array BioPharma. (PRNewsFoto/Array BioPharma Inc.) (PRNewsFoto/)
"We were pleased to report that COLUMBUS met its primary endpoint and demonstrated a robust PFS benefit associated with the combination of binimetinib plus encorafenib versus vemurafenib in patients with BRAF-mutant melanoma," said Ron Squarer, Chief Executive Officer of Array BioPharma. "We look forward to sharing results at SMR, partnering with global regulatory authorities on planned 2017 submissions for COLUMBUS and preparing for potential commercialization."

KEY COMPANY AND PIPELINE UPDATES
Binimetinib (MEK162) and encorafenib (LGX818)
Novartis continues to substantially fund all ongoing trials with binimetinib and encorafenib that were active or planned as of the close of the Novartis Agreements in 2015, including the NEMO and COLUMBUS Phase 3 trials. Reimbursement revenue from Novartis was approximately $129.0 million for the previous 12 months, of which $31.3 million was recorded over the past quarter.

COLUMBUS: Global Phase 3 trial of binimetinib plus encorafenib versus vemurafenib in BRAF-mutant melanoma patients
In September 2016, Array announced top-line results from Part 1 of the Phase 3 COLUMBUS study evaluating LGX818 (encorafenib), a BRAF inhibitor, and MEK162 (binimetinib), a MEK inhibitor, in patients with BRAF-mutant advanced, unresectable or metastatic melanoma. The study met its primary endpoint, with the combination of encorafenib and binimetinib ("combination") significantly improving progression free survival (PFS) compared with vemurafenib, a BRAF inhibitor, alone. In the analysis of the primary endpoint, the median PFS for patients treated with the combination was 14.9 months versus 7.3 months for patients treated with vemurafenib; Hazard ratio (HR) 0.54, (95% CI 0.41-0.71, p<0.001). The combination was generally well-tolerated and reported adverse events were overall consistent with previous clinical trial results of the combination in BRAF-mutant melanoma patients. Analysis of a secondary endpoint comparing the median PFS of patients treated with the combination to patients treated with encorafenib alone showed 14.9 months versus 9.6 months, respectively; HR 0.75, (95% CI 0.56-1.00, p=0.051), which did not reach statistical significance. A complete analysis of these results will be provided to global regulatory authorities as part of planned submissions in 2017. In addition, data from Part 2 of the COLUMBUS trial are anticipated in mid-2017 and will also be provided to global health authorities as part of planned regulatory submissions seeking approval of these product candidates.

Further results from Part 1 of the COLUMBUS trial will be presented at the Society for Melanoma Research Congress on November 9, 2016. Analysis of the secondary endpoint of overall survival (OS) was not planned as part of these initial results. Binimetinib and encorafenib are investigational medicines and are not currently approved in any country.

Melanoma is the fifth most common cancer among men and the seventh most common cancer among women in the United States, with more than 76,000 new cases and over 10,000 deaths from the disease expected in 2016. Novel therapies that target the RAS/RAF/MEK/ERK pathway have a strong scientific rationale for activity in this disease, as up to 50 percent of patients with metastatic melanoma have activating BRAF mutations, the most common gene mutation in this patient population. Current marketed MEK/BRAF combination agents have a run rate forecasted to approach $1 billion in annual worldwide sales.

NEMO: Global Phase 3 trial of binimetinib versus dacarbazine in NRAS-mutant melanoma patients
In September 2016, Array announced that the FDA has accepted its NDA for binimetinib in NRAS-mutant melanoma with a target action date under the Prescription Drug User Fee Act (PDUFA) of June 30, 2017. Also, the binimetinib Marketing Authorization Application (MAA) submitted by Pierre Fabre was validated and is currently under evaluation by the Committee for Medicinal Products for Human Use (CHMP). The FDA indicated that it plans to hold an advisory committee meeting (ODAC) in the first half of 2017 as part of the review process.

Activating NRAS mutations are present in up to 20 percent of patients with metastatic melanoma, and are a poor prognostic indicator for these patients. Treatment options for this population remain limited beyond immunotherapy, and these patients face poor clinical outcomes and high mortality.

BEACON CRC: Global Phase 3 trial of binimetinib, encorafenib and Erbitux (cetuximab) versus Erbitux in BRAF-mutant colorectal cancer (CRC) patients
Array is advancing BEACON CRC, a global Phase 3 trial of encorafenib and Erbitux (cetuximab), with or without binimetinib, versus standard of care in patients with BRAF-mutant CRC who have previously received first-or second-line systemic therapy. In September 2016, Array announced that it has reached agreement with the FDA regarding a Special Protocol Assessment (SPA) related to BEACON CRC. The SPA acknowledges that the design and planned analysis of BEACON CRC adequately address the objectives necessary to support a regulatory submission for the approval of the doublet regimen of encorafenib and Erbitux. The FDA also communicated that sharing evidence from the study that the triplet regimen (encorafenib, Erbitux and binimetinib) both met its primary endpoint (Overall Survival) as compared to the control arm, and demonstrated a clinically meaningful benefit as compared to the doublet regimen, would provide support for approval of the triplet regimen and that determination regarding approval of the triplet regimen will consider the totality of the data. Array expects to have data from the safety lead-in portion of the study in 2017.

BEACON CRC was initiated based on results from a Phase 2 study of the combination of encorafenib and cetuximab, with or without alpelisib, a selective PI3K alpha inhibitor, in patients with advanced BRAF-mutant CRC, which were presented at the 2016 ASCO (Free ASCO Whitepaper) meeting. Data from this study suggest that mOS for these patients may exceed one year, which is more than double several historical published benchmarks for this population.

Colorectal cancer is the third most common cancer among men and women in the United States, with more than 134,000 new cases and nearly 50,000 deaths from the disease projected in 2016. In the United States, BRAF mutations occur in 8 to 15 percent of patients with colorectal cancer and represent a poor prognosis for these patients.

ARRY-382
Phase 1/2 dose escalation study advancing with ARRY-382, a colony-stimulating factor-1 receptor (CSF-1R) inhibitor, in combination with pembrolizumab, a PD-1 antibody, for the treatment of patients with advanced solid tumors

Array is advancing a Phase 1/2 dose escalation immuno-oncology trial of ARRY-382 in combination with pembrolizumab (Keytruda), a Programmed Cell Death Receptor 1 (PD-1) antibody, in patients with advanced solid tumors. ARRY-382 is a wholly-owned, highly selective and potent, small molecule inhibitor of CSF-1R kinase activity.

The study will enroll up to 18 patients with selected advanced solid tumors to determine the maximum tolerated dose and/or recommended Phase 2 dose of the combination. In addition, the safety profile, pharmacodynamic effects, and preliminary assessment of activity of the combination will be assessed. With appropriate results, Array has the option to advance the combination into expansion cohorts of patients with metastatic melanoma or advanced non-small cell lung cancer (NSCLC). Array expects to have data from the dose-escalation portion of the study in 2017.

Results from a prior Phase 1 study designed to assess the safety, pharmacokinetics and pharmacodynamics of ARRY-382 for treating patients with cancer have been presented and a dose and schedule that demonstrates target engagement based on multiple pharmacodynamic biomarkers was identified for further study. ARRY-382 is an investigational new drug and is not currently approved for any use in any country.

ARRY-797 (ARRY-371797)
Phase 2 trial in patients with LMNA A/C-related dilated cardiomyopathy (LMNA-related DCM)
In August 2016, Array announced results from a Phase 2 study of ARRY-797, an oral, selective p38 mitogen-activated protein kinase inhibitor, in patients with LMNA-related DCM, a rare, degenerative cardiovascular disease caused by mutations in the LMNA gene and characterized by poor prognosis. The trial results were presented at the European Society of Cardiology Congress 2016. The results demonstrated an absolute mean change from baseline of 69 meters on the six-minute walk test (6MWT) at week 12, the study’s primary endpoint (baseline 6MWT ranged from 246 to 412 meters). This magnitude of improvement exceeded historical benchmarks for 6MWT that have served as the basis for recent approvals of other drugs in other rare diseases. ARRY-797 administration also resulted in sustained improvements in N-terminal pro-brain natriuretic peptide (NT-proBNP), functional capacity and cardiac function through 48 weeks in LMNA-related DCM patients. Patients who rolled over to a continuing treatment protocol maintained improvements in the 6MWT and NT-proBNP levels through 72 weeks of treatment. Other secondary endpoints measured including echocardiographic measures of left and right ventricular function and patient-reported outcomes using the Kansas City Cardiomyopathy Questionnaire (KCCQ), and both mirrored the improvements seen with the 6MWT. ARRY-797 was well tolerated with most patients experiencing mild to moderate adverse events, including stomatitis, acne and upper respiratory tract infection.

Taken together, the data to date suggest a path forward for this program, and Array has met with regulators to discuss the design of a study that could be the basis for marketing approval. Array is evaluating different options to advance the ARRY-797 program, including advancing it on its own, partnering the program for further development and commercialization or creating a separate company based on this asset.

LMNA-related DCM is estimated to occur in about 6,000 – 10,000 patients in the US. The number of patients currently identified with a molecular diagnosis is likely to be less than this estimate because of underutilization of genetic testing. Patients with LMNA-related DCM typically begin experiencing symptoms in their twenties or thirties and by age 45, nearly 70 percent of patients have had a heart transplant, have experienced a major cardiac event or have died. By comparison, only 25 percent of DCM patients who do not have LMNA mutations experience similar events by age 45. Currently, there are no disease-specific treatments approved for LMNA-related DCM. ARRY-797 is an investigational new drug and is not currently approved for any use in any country.

FINANCIAL HIGHLIGHTS
Raised $132.25 million in public offering
Array completed an underwritten public offering of 21,160,000 shares of its common stock at a price of $6.25 per share on October 3, 2016. The total gross proceeds from the offering are $132.25 million, before underwriting discounts and commissions and offering expenses.

First Quarter of Fiscal 2017 Compared to Fourth Quarter of Fiscal 2016 (Sequential Quarters Comparison)

Revenue for the first quarter of fiscal 2017 was $39.3 million, compared to $43.2 million for the prior sequential quarter, mainly driven by reduced Novartis reimbursed activity.
Cost of partnered programs for the first quarter of fiscal 2017 was $8.8 million, compared to $5.4 million for the prior quarter. The increase in cost of partnered programs was primarily due to the initiation of the BEACON CRC trial.
Research and development expense was $46.6 million, compared to $49.5 million in the prior quarter. The slight decrease in research and development expense is primarily related to the ongoing transition of binimetinib and encorafenib trials from Novartis to Array.
Net loss for the first quarter was $28.6 million, or ($0.20) per share, and was $25.0 million, or ($0.17) per share in the prior quarter.
First Quarter of Fiscal 2017 Compared to First Quarter of Fiscal 2016 (Prior Year Comparison)

Revenue for the first quarter of fiscal 2017 increased $23.1 million compared to the same quarter of fiscal 2016. The increase was primarily due to reimbursement revenue from Novartis.
Cost of partnered programs increased $2.6 million compared to the first quarter of fiscal 2016. The increase was primarily due to the BEACON CRC trial.
Research and development expense increased $25.6 million, compared to the first quarter of fiscal 2016. The increase was due to binimetinib and encorafenib expenses as we transitioned activity from the "Novartis Agreements."
Net loss for the first quarter of fiscal 2017 was $28.6 million, or ($0.20) per share, and was $21.0 million, or ($0.15) per share, for the same quarter in fiscal 2016.

GigaGen Announces $1.37M National Cancer Institute (NCI) Grant Award for Discovery and Development of New Oncology Therapies

On November 1, 2016 GigaGen Inc., a biopharmaceutical company with patented technology for discovery of T cell receptor (TCR) and antibody drugs from immune repertoires, reported that it has been awarded a $1.37 million Phase II grant from the National Cancer Institute (NCI) through the NIH Small Business Innovation Research (SBIR) program (Press release, GigaGen, NOV 1, 2016, View Source [SID1234520617]). The grant supports discovery and development of oncology drugs from mouse and human B cell repertoires using GigaGen’s novel massively parallel drug discovery technology.

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Cancer still accounts for 25% of US deaths despite an enormous investment in novel therapies. Monoclonal antibodies (mAbs) are now the biological agents of choice for cancer therapy. Today, most mAb discovery programs use either phage or yeast display, or mouse immunization followed by hybridoma isolation. Although these technologies have had success, antibody discovery and development is still slow and expensive. R&D programs need faster, deeper, and more efficient antibody discovery technologies to find the next effective cancer treatment.

GigaGen has the only technology that enables massively parallel antibody screening, engineering, and development by capturing millions to billions of antibody-encoding DNA sequences from a sample, and then expressing the DNA sequences as antibodies for affinity screening and antigen discovery. The NCI grant funds efforts to discover therapeutic antibodies produced by the immune systems of cancer survivors who beat their disease. In parallel, the NCI grant funds an improved approach to discovering checkpoint inhibitor antibodies in mouse models.

"We are pleased that the NCI has recognized the enormous potential of our technology to increase the speed and decrease the cost of oncology drug discovery," said Dave Johnson, Ph.D., CEO of GigaGen. "Using GigaGen’s novel technology, one technician competes favorably with dozens of technicians who are stuck using conventional technology. We are able to understand immune repertoires more deeply than ever before, leading to novel therapies in the exciting, emerging field of immune modulation of cancer."

Gilead Sciences Announces Third Quarter 2016 Financial Results

On November 1, 2016 Gilead Sciences, Inc. (Nasdaq: GILD) reported its results of operations for the third quarter ended September 30, 2016 (Press release, Gilead Sciences, NOV 1, 2016, View Source [SID1234516203]).

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The financial results that follow represent a year-over-year comparison of third quarter 2016 to the third quarter 2015. Total revenues were $7.5 billion in 2016 compared to $8.3 billion in 2015. Net income was $3.3 billion or $2.49 per diluted share in 2016 compared to $4.6 billion or $3.06 per diluted share in 2015. Non-GAAP net income, which excludes amounts related to acquisition-related, up-front collaboration, stock-based compensation and other expenses, was $3.7 billion or $2.75 per diluted share in 2016 compared to $4.8 billion or $3.22 per diluted share in 2015.


Three Months Ended Nine Months Ended
September 30, September 30,
(In millions, except per share amounts) 2016 2015 2016 2015
Product sales $ 7,405 $ 8,211 $ 22,737 $ 23,742
Royalty, contract and other revenues 95 84 333 391
Total revenues $ 7,500 $ 8,295 $ 23,070 $ 24,133

Net income attributable to Gilead $ 3,330 $ 4,600 $ 10,393 $ 13,425
Non-GAAP net income* $ 3,677 $ 4,836 $ 12,128 $ 14,285

Diluted earnings per share $ 2.49 $ 3.06 $ 7.59 $ 8.73
Non-GAAP diluted earnings per share* $ 2.75 $ 3.22 $ 8.87 $ 9.29

* Non-GAAP net income and non-GAAP diluted earnings per share exclude acquisition-related, up-front collaboration, stock-based compensation and other expenses. A reconciliation between GAAP and non-GAAP financial information is provided in the tables on pages 7 and 8.

Product Sales

Total product sales for the third quarter of 2016 were $7.4 billion compared to $8.2 billion for the same period in 2015. Product sales for the third quarter of 2016 were $5.1 billion in the United States, $1.4 billion in Europe, $452 million in Japan and $479 million in other locations. Product sales for the third quarter of 2015 were $5.6 billion in the United States, $1.7 billion in Europe, $454 million in Japan and $504 million in other locations.

Antiviral Product Sales

Antiviral product sales, which include primarily products in Gilead’s HIV and liver disease areas, were $6.8 billion for the third quarter of 2016 compared to $7.7 billion for the same period in 2015.

HIV and other antiviral product sales were $3.5 billion compared to $2.9 billion for the same period in 2015. The increase was primarily due to the continued uptake of our tenofovir alafenamide (TAF) based products, Genvoya (elvitegravir 150 mg/cobicistat 150 mg/emtricitabine 200 mg/tenofovir alafenamide 10 mg), Descovy (emtricitabine 200 mg/tenofovir alafenamide 25 mg) and Odefsey (emtricitabine 200 mg/rilpivirine 25 mg/tenofovir alafenamide 25 mg).
HCV product sales, which consist of Harvoni (ledipasvir 90 mg/sofosbuvir 400 mg), Sovaldi (sofosbuvir 400 mg) and Epclusa (sofosbuvir 400 mg/velpatasvir 100 mg), were $3.3 billion compared to $4.8 billion for the same period in 2015. The decline was due to lower sales of Harvoni and Sovaldi, partially offset by sales of Epclusa, which was launched in the United States and Europe in June and July 2016, respectively.
Other Product Sales

Other product sales, which include Letairis (ambrisentan), Ranexa (ranolazine) and AmBisome (amphotericin B liposome for injection), were $564 million for the third quarter of 2016 compared to $509 million for the same period in 2015.

Operating Expenses


Three Months Ended Nine Months Ended
September 30, September 30,
(In millions) 2016 2015 2016 2015
Research and development expenses (R&D) $ 1,141 $ 743 $ 3,890 $ 2,257
Non-GAAP research and development expenses*
$ 981 $ 713 $ 2,790 $ 2,066

Selling, general and administrative expenses (SG&A) $ 831 $ 903 $ 2,406 $ 2,360
Non-GAAP selling, general and administrative expenses* $ 780 $ 850 $ 2,256 $ 2,211

* Non-GAAP R&D and SG&A expenses exclude acquisition-related, up-front collaboration, stock-based compensation and other expenses. A reconciliation between GAAP and non-GAAP financial information is provided in the tables on pages 7 and 8.

During the third quarter of 2016, compared to the same period in 2015:

Research and development expenses and non-GAAP research and development expenses* increased primarily due to the overall progression of Gilead’s clinical studies, including a $200 million milestone expense associated with Gilead’s purchase of Nimbus Apollo, Inc.
Selling, general and administrative expenses and non-GAAP selling, general and administrative expenses* decreased primarily due to lower branded prescription drug fee expense.
Cash, Cash Equivalents and Marketable Securities

As of September 30, 2016, Gilead had $31.6 billion of cash, cash equivalents and marketable securities compared to $24.6 billion as of June 30, 2016. This increase was primarily due to the issuance of $5.0 billion aggregate principal amount of senior unsecured notes in September 2016. Cash flow from operating activities was $4.3 billion for the quarter. During the third quarter and the first nine months of 2016, Gilead utilized $1.0 billion and $10.0 billion on stock repurchases, respectively.

Full Year 2016 Guidance Reiterated

Gilead reiterates its full year 2016 guidance, as revised on July 25, 2016:


(In millions, except percentages and per share amounts) Updated July 25, 2016
Reiterated November 1, 2016
Net Product Sales $29,500 – $30,500
Non-GAAP*
Product Gross Margin 88% – 90%
R&D Expenses $3,600 – $3,800
SG&A Expenses $3,100 – $3,300
Effective Tax Rate 18.0% – 20.0%
Diluted EPS Impact of Acquisition-related, Up-front Collaboration, Stock-based Compensation and Other Expenses $1.47 – $1.53

* Non-GAAP Product Gross Margin, R&D and SG&A expenses and effective tax rate exclude acquisition-related, up-front collaboration, stock-based compensation and other expenses. A reconciliation between GAAP and non-GAAP full year 2016 guidance is provided in the tables on page 9.

Corporate Highlights

Announced that Kelly A. Kramer was appointed to the company’s Board of Directors and Audit Committee. Ms. Kramer is currently Executive Vice President and Chief Financial Officer of Cisco Systems, Inc.
Announced that Gilead entered into a partnership with the World Health Organization (WHO) to provide $20 million in funding and drug donations over five years to expand access to diagnostic services and treatment for visceral leishmaniasis (VL). As part of this collaboration, Gilead will donate 380,000 vials of AmBisome to meet the needs of WHO to treat VL in key endemic countries, including Bangladesh, Ethiopia, India, Nepal, South Sudan and Sudan.
Product and Pipeline Updates announced by Gilead during the Third Quarter of 2016 include:

Announced that the European Commission granted marketing authorization for once-daily Truvada (emtricitabine 200 mg/tenofovir disoproxil 245 mg) in combination with safer-sex practices to reduce the risk of sexually acquired HIV-1 infection among uninfected adults at high risk, a strategy known as pre-exposure prophylaxis, or PrEP. Truvada was approved by the European Medicines Agency in 2005 for use in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults aged 18 years and over, and is currently the most prescribed antiretroviral medicine in Europe as part of combination therapy.
Announced that the European Commission granted marketing authorization for Epclusa, the first pan-genotypic, single tablet regimen for the treatment of adults with genotype 1-6 chronic hepatitis C virus (HCV) infection. Epclusa for 12 weeks was authorized for use in patients without cirrhosis or with compensated cirrhosis (Child-Pugh A), and in combination with ribavirin (RBV) for patients with decompensated cirrhosis (Child-Pugh B or C). Epclusa is also the first single tablet regimen approved for the treatment of patients with HCV genotype 2 and 3, without the need for RBV. Physicians also have the flexibility to consider the addition of RBV for genotype 3 infected patients with compensated cirrhosis. The marketing authorization followed an accelerated review procedure by the European Medicines Agency, reserved for medicinal products expected to be of major public health interest.
Non-GAAP Financial Information

The information presented in this document has been prepared by Gilead in accordance with U.S. generally accepted accounting principles (GAAP), unless otherwise noted as non-GAAP. Management believes non-GAAP information is useful for investors, when considered in conjunction with Gilead’s GAAP financial information, because management uses such information internally for its operating, budgeting and financial planning purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Gilead’s operating results as reported under GAAP. Non-GAAP measures may be defined and calculated differently by other companies in the same industry. A reconciliation between GAAP and non-GAAP financial information is provided in the tables on pages 7, 8 and 9.

Medivir’s nucleotide polymerase inhibitor for the treatment of liver cancer, MIV-818, enters non-clinical development

On November 1, 2016 Medivir AB (Nasdaq Stockholm: MVIR) reported that MIV-818 has been selected as a candidate drug (CD) from its nucleotide DNA polymerase inhibitor project for the treatment of hepatocellular carcinoma (HCC), and has now entered non-clinical development.

Liver cancers are orphan indications in North American and Western European markets. However they are the second leading cause of cancer-related death worldwide, and one of the fastest growing forms of cancer in the US, based on incidence and mortality. Hepatocellular carcinoma (HCC) is the most common cancer of the liver. Despite improvements in the detection and management of the disease, the 5-year survival rate for patients in the USA who are diagnosed with liver cancer remains below 20%. While curative surgical treatments are available to HCC patients who are diagnosed early in their disease, the prognosis for all inoperable HCC cases remains poor. There is consequently an urgent need for improved treatments, particularly for patients with advanced stages of HCC and other forms of liver cancer.

Effective therapies for patients with inoperable intermediate HCC include the delivery of drugs directly to the cancer tumour through the liver’s blood supply, which is technically challenging and for which many patients are ineligible. In contrast, most anticancer drugs that are widely distributed throughout the body are ineffective. Sorafenib is the only orally administered drug used to treat liver cancer. It is approved for use in patients with advanced HCC but confers only modest survival benefits. Despite these limitations, sorafenib has achieved over $1B in annual worldwide sales across several cancer indications, with the majority of sales from treatment of HCC.

Medivir has developed substantial capabilities to selectively deliver the active metabolites of nucleoside and nucleotide analogues to the liver, based on its long-standing interests in discovering improved treatments for chronic hepatitis B virus and hepatitis C virus infections. MIV-818 is a potent and selective inhibitor of the proliferation of liver cancer cell lines that has been designed to deliver high levels of the active drug selectively to the liver. MIV-818 has the potential to become the first liver-targeted orally administered drug to address HCC and other liver cancers. Medivir expects to communicate the preclinical antitumour and pharmacokinetic profile of MIV-818 at major scientific meetings in 2017.

"MIV-818 is unique in that it is an orally administered chemotherapeutic that will be developed exclusively for liver cancers. Many treatments that were successful in other cancers have failed to provide benefits to liver cancer patients, often because systemic toxicity prevents effective drug concentrations from being reached at the tumour site. We have designed MIV-818 to be liver-directed in order to overcome these limitations, and we look forward to advancing it into clinical trials as rapidly as possible." said Richard Bethell, Chief Scientific Officer, Medivir AB. "We are delighted to have delivered the first CD from our internal portfolio of early-stage anti-cancer and immuno-oncology projects since it represents an important milestone in our transition to being an exclusively oncology-focused pharmaceutical company."

Astex Achieves Milestone on US FDA Filing of New Drug Application (NDA) for LEE011 (Ribociclib) Plus Letrozole as a First-Line Treatment for HR+/HER2- Advanced Breast Cancer

On November 1, 2016 Astex Pharmaceuticals, a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics for oncology and diseases of the central nervous system, reported that it has received a milestone payment from Novartis in relation to the US FDA NDA filing by Novartis for LEE011 (ribociclib) plus letrozole as a first-line treatment for HR+/HER2- advanced breast cancer (Press release, Astex Pharmaceuticals, NOV 1, 2016, View Source [SID1234516166]).

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Novartis also announced that it had received FDA Priority Review for the NDA application of LEE011 as first-line treatment of postmenopausal women with hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer in combination with letrozole.

LEE011 (ribociclib) was developed by the Novartis Institutes for BioMedical Research (NIBR) under a research collaboration with Astex. Under the collaboration, which commenced in 2005, NIBR scientists worked with Astex on a programme of early drug discovery research resulting in the discovery of LEE011. Novartis then led LEE011 into preclinical and later clinical development. Under terms of the agreement, Astex is eligible to receive further milestone payments in respect of additional regulatory filing and approvals in Europe and Japan, as well as royalty payment on annual sales of ribociclib should the drug be approved.

Harren Jhoti, President and CEO of Astex, said, "We are absolutely delighted that Novartis has reached such a significant stage in the development of LEE011. If the product is approved, it will provide an important treatment option for many patients with advanced disease. We congratulate Novartis for an excellent job in developing LEE011 and on the achievement of US FDA Priority Review of the NDA filing."

About LEE011 (ribociclib)

LEE011 (ribociclib) is a selective cyclin dependent kinase inhibitor, a class of drugs that help slow the progression of cancer by inhibiting two proteins called cyclin dependent kinase 4 and 6 (CDK4/6). These proteins, when over-activated in a cell, can enable cancer cells to grow and divide too quickly. Targeting CDK4/6 with enhanced precision may play a role in ensuring cancer cells do not grow uncontrollably.