Sandoz biosimilar filgrastim recommended for approval by FDA Oncologic Drugs Advisory Committee

From Novartis:

January 07, 2015 21:57 CET

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Sandoz biosimilar filgrastim recommended for approval by FDA Oncologic Drugs Advisory Committee

• Oncologic Drugs Advisory Committee (ODAC) votes in favor of recommending biosimilar filgrastim for approval in the US
• Biosimilar filgrastim recommended to be approved for use in all requested indications
• Committee's recommendation based on review of extensive data from analytical, non-clinical, clinical studies and post-marketing pharmacovigilance

Holzkirchen, January 7, 2014 - Sandoz, a Novartis company, announced today that US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) recommended approval of its investigational biosimilar filgrastim in the US. The Committee also recommended approval of the biosimilar for use in all indications included in the reference product's (Amgen's NEUPOGEN®) label.

"We are pleased with the ODAC's recommendation to approve our biosimilar filgrastim and we look forward to continuing to work with FDA as it completes its review of our filing," said Mark McCamish, M.D., Ph.D., Head of Global Biopharmaceutical & Oncology Injectables Development at Sandoz. "We are proud to lead the way in biosimilars globally and believe this positive recommendation brings us one step closer to delivering high-quality biosimilars to patients in the US."

The recommendation was provided after the presentation of a comprehensive package of nonclinical, clinical and post-marketing pharmacovigilance data which confirmed Sandoz' biosimilar filgrastim is highly similar to the reference product (Amgen's NEUPOGEN®). The pivotal clinical package included a pharmacokinetic and pharmacodynamics (PK/PD) study in healthy volunteers, which established bioequivalence, and a clinical efficacy and safety study in breast cancer patients which demonstrated the same clinical performance and safety as the reference product.

The clinical package is also supported by a global program including five randomized, double-blind, single and multiple dose PK/PD studies in healthy volunteers to assess pharmacokinetic and pharmacodynamic equivalence between biosimilar filgrastim and NEUPOGEN® and a European non-comparative clinical safety and efficacy study. Post-marketing pharmacovigilance data from countries outside of the US also contributed to the totality of evidence.

Under the brand name ZARZIO®, the Sandoz biosimilar filgrastim has been marketed in more than 40 countries outside the US, generating nearly 7.5 million patient-exposure days of experience. If approved in the US, Sandoz proposes to market biosimilar filgrastim under the name ZARXIOTM.

FDA frequently seeks the advice of its advisory committees as it reviews and decides whether to approve treatments, although it is not obligated to follow the recommendation.

Sandoz is the global leader with over 50 percent volume share of all biosimilars approved in the highly-regulated markets of Canada, Europe, Japan and Australia. Sandoz currently markets three biosimilars outside the US; each of which occupies the #1 biosimilar position in its respective category. Sandoz biosimilars are sold in over 60 countries and have generated over 200 million patient-exposure days in experience. Sandoz also has an unrivalled biosimilars pipeline with several molecules in various stages of development, including six molecules in Phase III clinical trials/registration - more than any other company in the industry.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by words such as "recommended," "votes in favor of recommending," "recommendation," "investigational," "look forward," "believe," "will," or similar terms, or by express or implied discussions regarding potential future product approvals, or regarding potential revenues from biosimilar filgrastim or any potential future products. 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 biosimilar filgrastim will be approved for sale in any additional markets, or at any particular time. Neither can there be any guarantee that any other potential future products will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that biosimilar filgrastim or any other potential future products will be commercially successful in the future. In particular, management's expectations regarding biosimilar filgrastim and such other potential future products 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; competition in general, including approval of additional versions of biosimilar filgrastim; the company's ability to obtain or maintain proprietary intellectual property protection, including unexpected patent litigation outcomes; government, industry and general public pricing pressures; unexpected manufacturing issues; general economic and industry conditions, 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 Sandoz
Sandoz, a Novartis company, is a global leader in the generic pharmaceutical sector. Sandoz employs over 26,500 employees across more than 160 countries, offering a broad range of high-quality, affordable products that are no longer protected by patents. With USD 9.2 billion in sales in 2013, Sandoz has a portfolio of approximately 1,100 molecules, and holds the #1 position globally in biosimilars as well as in generic injectables, ophthalmics, dermatology and antibiotics, complemented by leading positions in the cardiovascular, metabolism, central nervous system, pain, gastrointestinal, respiratory, and hormonal therapeutic areas. Sandoz develops, produces, and markets these medicines, as well as active pharmaceutical and biotechnological substances. Nearly half of Sandoz's portfolio is in differentiated products, which are defined as products that are more difficult to scientifically develop and manufacture than standard generics. In addition to strong organic growth since consolidating its generics businesses under the Sandoz brand name in 2003, Sandoz has benefitted from strong growth of its acquisitions, which include Lek (Slovenia), Sabex (Canada), Hexal (Germany), Eon Labs (US), EBEWE Pharma (Austria), Oriel Therapeutics (US), and Fougera Pharmaceuticals (US).

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Novartis collaborates with Intellia Therapeutics and Caribou Biosciences to explore making medicines and drug discovery tools with CRISPR genome editing technology

From Novartis:

January 07, 2015 07:15 CET

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Novartis collaborates with Intellia Therapeutics and Caribou Biosciences to explore making medicines and drug discovery tools with CRISPR genome editing technology

• Collaborations give Novartis access to novel CRISPR technology platforms for the discovery and development of new medicines
• Intellia Therapeutics collaboration to explore therapeutic options for using CRISPR to engineer chimeric antigen receptor T-cells and hematopoietic stem cells
• Caribou Biosciences collaboration focused on using CRISPR as a research tool for drug discovery

Basel, Switzerland, January 7, 2015 - Novartis announced today that it has signed collaboration and licensing agreements with Intellia Therapeutics for the discovery and development of new medicines using CRISPR genome editing technology and Caribou Biosciences for the development of drug discovery tools. Intellia and Caribou are two of the leading biotechnology companies developing this novel technology.

CRISPR, an acronym that stands for clustered regularly interspaced short palindromic repeats, is an approach that allows scientists to easily and precisely edit the genes of targeted cells. In a short period of time it has proven to be a powerful tool for creating very specific models of disease for use in drug discovery and has great potential for use as a therapeutic modality for treating disease at the genetic level by deleting, repairing or replacing the genes that cause disease.

"We have glimpsed the power of CRISPR tools in our scientific programs in NIBR, and it is now time to explore how to safely extend this powerful technology to the clinic," said Mark Fishman, President of the Novartis Institutes for BioMedical Research (NIBR). " CRISPR has the potential to open a new branch of medicine, editing the genome to cure disease. Much remains to be learned, and we are delighted to explore these directions with colleagues from Intellia and Caribou. "

The alliance with Intellia combines the resources, research expertise and cell and gene therapy leadership at the Novartis Institutes for BioMedical Research with Intellia's rapidly growing CRISPR expertise and organization. Research and development activities will focus on using CRISPR ex vivo for engineering chimeric antigen receptor T-cells (CARTs) and hematopoietic stem cells (HSCs). The collaboration and licensing agreement with Caribou is focused on using Caribou's foundational CRISPR platform and intellectual property as a research tool for drug discovery.

Under the terms of the agreement with Intellia, Novartis is receiving exclusive rights to develop all collaboration programs focused on engineered CARTs and the right to develop an undisclosed number of targets for ex vivo editing of HSCs. In addition Novartis receives non-exclusive rights for limited in vivo therapeutic applications of CRISPR systems. Novartis is increasing its equity investment in Intellia, is making an upfront payment and will provide technology access fees and funding for R&D programs during the 5-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Under the terms of the agreement with Caribou, Novartis is receiving non-exclusive rights to Caribou's CRISPR platform for research conducted during the collaboration and is providing funding for the one-year research program. Novartis is also making an equity investment in the company.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by words such as "commits," "to explore," "focused on," "potential," "may," "remains," "will," "eligible," or similar terms, or by express or implied discussions regarding potential marketing approvals for products developed through the collaborations with Intellia Therapeutics and Caribou Biosciences, or regarding potential future revenues from such products, the application of the CRISPR genome editing technology, or any other technologies developed through these collaborations. 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 any products or other technologies will arise from the collaborations with Intellia Therapeutics and Caribou Biosciences. Neither can there be any guarantee that any products arising from or developed through these collaborations will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that any product or other technology arising from or developed through these collaborations will receive regulatory approval or be commercially successful in the future. In particular, management's expectations regarding the CRISPR genome editing technology, potential products and other technologies 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 manufacturing 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, cost-saving generic pharmaceuticals, preventive vaccines and over-the-counter products. Novartis is the only global company with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 130,000 full-time-equivalent associates and sell products in more than 150 countries around the world. For more information, please visit http://www.novartis.com.

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

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Rice University, IBM Partner to Bring First Blue Gene Supercomputer to Texas

University of Sao Paulo signs on as first user for Rice University's newest supercomputer

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HOUSTON - 30 Mar 2012: Rice University and IBM (NYSE: IBM) today announced a partnership to build the first award-winning IBM Blue Gene supercomputer in Texas. Rice also announced a related collaboration agreement with the University of Sao Paulo (USP) in Brazil to initiate the shared administration and use of the Blue Gene supercomputer, which allows both institutions to share the benefits of the new computing resource.

Rice faculty will use the Blue Gene to further their own research and to collaborate with academic and industry partners on a broad range of science and engineering questions related to energy, geophysics, basic life sciences, cancer research, personalized medicine and more.

The collaborative agreement securing Brazil's share of time on Rice's Blue Gene was signed in Sao Paulo March 27 by a delegation that included Rice President David Leebron and USP President Joao Grandino Rodas. Leebron is traveling with a delegation led by Houston Mayor Annise Parker. The delegation includes Rice Provost George McLendon, Greater Houston Partnership (GHP) President and CEO Jeff Moseley and other GHP members.

"Collaboration and partnership have a unique place in Rice's history as a pre-eminent research university, and it is fitting that Rice begins its second century with two innovative partnerships that highlight the university's commitments to expanding our international reach, strengthening our research and building stronger ties with our home city," Leebron said.

USP is Brazil's largest institution of higher education and research, and Rodas said the agreement represents an important bond between Rice and USP. "The joint utilization of the supercomputer by Rice University and USP, much more than a simple sharing of high-tech equipment, means the strength of an effective partnership between both universities," he said.

Mayor Parker, a 1978 Rice alumna, said, "When I was at Rice, it looked inward. Today it looks outward through this agreement. It strengthens not only Rice University but also the city of Houston."

Rice's new Blue Gene supercomputer, which has yet to be named, is slated to become operational in May. It is based on IBM's POWER processor technology, which was developed in part at the company's Austin, Texas labs. Rice and IBM shared the cost of the system.

"High-performance computers like the IBM Blue Gene/P are critical in virtually every discipline of science and engineering, and we are grateful for IBM's help in bringing this resource to Rice," McLendon said. "For individual faculty, the supercomputer will open the door to new areas of research. The Blue Gene also opens doors for Rice as the university seeks to establish institutional relationships both in our home city and with critical international partners like USP."

Unlike the typical desktop or laptop computer, which have a single microprocessor, supercomputers typically contain thousands of processors. This makes them ideal for scientists who study complex problems, because jobs can be divided among all the processors and run in a matter of seconds rather than weeks or months. Supercomputers are used to simulate things that cannot be reproduced in a laboratory -- like Earth's climate or the collision of galaxies -- and to examine vast databases like those used to map underground oil reservoirs or to develop personalized medical treatments.

USP officials said they expect their faculty to use the supercomputer for research ranging from astronomy and weather prediction to particle physics and biotechnology.

"This significant investment by IBM is the result of a long-standing collaborative initiative with Rice where together we have developed a unique and substantial computational resource for the research community in Houston, across the country and around the world," said Tony Befi, IBM senior state executive for Texas. "This new computing capability will speed the search for new sources of energy, new ways of maximizing current energy sources, new cancer drugs and new routes to personalized medicine. So we're excited that Rice has now joined an exclusive club of the world's top research organizations who use powerful and energy-efficient Blue Gene supercomputers to solve some of the world's most pressing problems."

In 2009, President Obama recognized IBM and its Blue Gene family of supercomputers with the National Medal of Technology and Innovation, the most prestigious award in the United States given to leading innovators for technological achievement.

Including the Blue Gene/P, Rice has partnered with IBM to launch three new supercomputers during the past two years that have more than quadrupled Rice's high-performance computing capabilities. The addition of the Blue Gene/P doubles the number of supercomputing CPU hours that Rice can offer. The six-rack system contains nearly 25,000 processor cores that are capable of conducting about 84 trillion mathematical computations each second. When fully operational, the system is expected to rank among the world's 300 fastest supercomputers as measured by the TOP500 supercomputer rankings.

A image is available for download at:  http://news.rice.edu/wp-content/uploads/2012/03/0330_BLUEGENE.jpg

Located on a 300-acre forested campus in Houston, Rice University is consistently ranked among the nation's top 20 universities by U.S. News & World Report. Rice has highly respected schools of Architecture, Business, Continuing Studies, Engineering, Humanities, Music, Natural Sciences and Social Sciences and is known for its "unconventional wisdom." With 3,708 undergraduates and 2,374 graduate students, Rice's undergraduate student-to-faculty ratio is 6-to-1. Its residential college system builds close-knit communities and lifelong friendships, just one reason why Rice has been ranked No. 1 for best quality of life multiple times by the Princeton Review and No. 4 for "best value" among private universities by Kiplinger's Personal Finance. To read "What they're saying about Rice," go to www.rice.edu/nationalmedia/Rice.pdf.