On February 15, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) and Flatiron Health, Inc. reported that the two partners have signed a definitive agreement under which Roche will acquire all shares of Flatiron Health, following on from an existing equity stake of 12.6% (Press release, Hoffmann-La Roche, FEB 15, 2018, View Source [SID1234524003]). The transaction is expected to close in the first half of 2018.

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Flatiron Health, a privately held healthcare technology and services company headquartered in New York City, US, is a market leader in oncology-specific electronic health record (EHR) software, as well as the curation and development of real-world evidence for cancer research. With its large network of community oncology practices and academic medical centers across the US, Flatiron Health has created a technology platform designed to learn from the experience of every patient.

Daniel O’Day, CEO Roche Pharmaceuticals said, "This is an important step in our personalised healthcare strategy for Roche, as we believe that regulatory-grade real-world evidence is a key ingredient to accelerate the development of, and access to, new cancer treatments. As a leading technology company in oncology, Flatiron Health is best positioned to provide the technology and data analytics infrastructure needed not only for Roche, but for oncology research and development efforts across the entire industry. A key principle of this is to preserve Flatiron’s autonomy and their ability to continue providing their services to all existing and future partners."

Flatiron Health has worked with industry leaders and regulators to develop new approaches for how real-world evidence may be used in regulatory decision making, including the design and validation of novel endpoints. By working closely with its network of community practices and academic medical centers, Flatiron has also developed a suite of software products that uniquely positions the company to advance the use of real-world evidence at the point of care.

Nat Turner, Flatiron Health Co-Founder and CEO said, "Roche has been a tremendous partner to us over the past two years and shares our vision for building a learning healthcare platform in oncology ultimately designed to improve the lives of cancer patients. This important milestone will allow us to increase our investments in our provider-facing technology and services platform, as well as our evidence-generation platform, which will remain available to the entire healthcare industry."

Under the terms of the agreement, Roche will make a payment of USD 1.9 billion to Flatiron Health on a fully diluted basis, subject to certain adjustments. The closing of the transaction is subject to customary closing conditions. The parties expect that following the closing, Flatiron Health will continue its current business model, network of partnerships and overall objectives. The integrity of segregated patient protected health information will be preserved, as will dedicated sales and marketing, provider-facing and life science business activities.

On February 15, 2018 -AstraZeneca and Merck (NYSE:MRK), known as MSD outside the U.S. and Canada, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1) (Press release, Merck & Co, FEB 15, 2018, View Source [SID1234524008]).

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NF1 is an incurable genetic condition that affects one in 3,000 births with highly-variable symptoms including cutaneous (skin), neurological (nervous system) and orthopedic (skeletal) manifestations. NF1 can cause secondary complications including learning difficulties, visual impairment, pain, disfigurement, twisting and curvature of the spine, high blood pressure and epilepsy. Plexiform neurofibromas (PNs) are tumors that arise from nerve fascicles and tend to grow along the length of the nerve. PNs, a neurological manifestation of NF1,occur in approximately 20-50 percent of NF1 patients causing pain, motor dysfunction and disfigurement.

Sean Bohen, executive vice president, global medicines development and chief medical officer, AstraZeneca, said, "Neurofibromatosis type 1 is a devastating condition that can lead to life-threatening complications. There is no known cure for neurofibromatosis and there are limited treatment options to manage symptoms."

Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, "This is an important collaborative effort with our colleagues at AstraZeneca addressing an area of significant unmet medical need to potentially benefit patients with neurofibromatosis type 1."

The potential benefit of selumetinib in NF1 is being explored in the U.S. National Cancer Institute-sponsored phase 1/2 SPRINT trial in pediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018.

The FDA’s ODD program provides orphan status to medicines that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S.

In addition to NF1, selumetinib is being investigated in the phase 3 ASTRA trial of patients who are diagnosed with differentiated thyroid cancer (DTC) following surgery and treatment with radioactive iodine. Selumetinib was granted ODD by the US FDA for the adjuvant treatment of stage 3/4 DTC in 2016. It is also being explored as a monotherapy and in combination with other treatments in phase 1 trials.

NOTES TO EDITORS

About neurofibromatosis type 1 (NF1)

The NF1 gene provides instructions for making a protein called Neurofibromin. The disease is associated with many symptoms, including soft lumps on and under the skin (subcutaneous neurofibromas), skin pigmentation (cafe au lait spots) and, in 20-50 percent of patients, tumors on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause morbidities such as pain, motor dysfunction and disfigurement. Patients with NF1 may experience a number of other complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. People with NF1 also have an increased risk of developing other cancers, including malignant brain and peripheral nerve sheath tumors, and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.

About selumetinib

Selumetinib, is an investigational MEK 1/2 inhibitor licensed by AstraZeneca from Array BioPharma Inc. in 2003.

The NF1 gene codes for a protein called Neurofibromin. This protein negatively regulates the RAS/MAPK pathway, which helps to control cell growth, differentiation and survival. Mutations in the NF1 gene may result in dysregulation in RAS/RAF/MEK/ERK signaling, which can cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumor growth. Selumetinib inhibits the MEK enzyme in this pathway, potentially leading to inhibition of tumor growth.

On February 15, 2018 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported that it will host a webcast and conference call to discuss the Company’s financial results for the fourth quarter and full year ended December 31, 2017, and provide a general business overview on Wednesday, February 28, 2018, at 4:30 p.m. ET (1:30 p.m. PT) (Press release, Portola Pharmaceuticals, FEB 15, 2018, View Source;p=RssLanding&cat=news&id=2332869 [SID1234524013]).

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Conference Call Details
The live conference call on Wednesday, February 28, 2018, at 4:30 p.m. ET, can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 4893459. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

On February 15, 2018 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that it will host a conference call at 1:30 p.m. PST/4:30 p.m. EST on Thursday, March 1, 2018 following release of its fourth quarter and full year 2017 financial results (Press release, Puma Biotechnology, FEB 15, 2018, http://investor.pumabiotechnology.com/press-release/puma-biotechnology-host-conference-call-discuss-fourth-quarter-and-full-year-financial [SID1234524014]).

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The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international). Please dial in at least ten minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on the company’s website for 90 days.

On February 15, 2018 Celularity reported that it has been created through the contribution and acquisition of extensive intellectual property, clinical-stage assets, basic and clinical research, and product development expertise including (Press release, Celularity, FEB 15, 2018, View Source [SID1234530906]):

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First proprietary allogeneic "off-the-shelf" immunotherapeutic platform
Proprietary clinical stage Placental Natural Killer (PNK) Cell Program
IND-ready CD38 CAR-T program
Innovative allogeneic CD38 CAR NK program
Phase 3-ready placental adherent cell products for serious Crohn’s Disease
Sorrento Therapeutics G-MAB Library of 50+ fully-human antibody-CAR constructs
Unparalleled IP position: 800+ issued patents in cell therapy and regenerative medicine; dominating position worldwide for placental stem cells
World-class cell and biomaterials manufacturing capabilities
Commercial-stage functional regeneration products Biovance and Interfyl, and late-stage pipeline assets across broad disease indications
LifeBankUSA, pioneering stem cell biobanking business with two decades’ operating expertise
Celularity, a biotechnology company founded by stem cell pioneer Robert Hariri, MD, PhD, is announcing the formation of the company with $250M in funding with contributions from Celgene (NSDQ: CELG), United Therapeutics (NSDQ: UTHR), Sorrento Therapeutics (NSDQ: SRNE), Human Longevity, Inc., Genting Group, the Dreyfus Family Office, Section 32, and Heritage Group. Celularity is co-founded with Vice Chairman, Peter H. Diamandis, MD, alongside Dr. Hariri, and has a board of industry luminaries including Vice Chairman, John Sculley, formerly of Apple and Pepsi-Cola, Bill Maris of Section 32 (previously founder and CEO of Google Ventures), and Andrew von Eschenbach, former commissioner of the US Food and Drug Administration.

Celularity sources, develops, and deploys transformative therapies derived from the placenta for treatment of complex medical conditions including hematological and solid tumors, autoimmune disease, diabetes, as well as degenerative effects of aging. By combining synergistic assets from Celgene, United Therapeutics, Sorrento Therapeutics, and Human Longevity Inc., Celularity is accelerating the development of cell and tissue regenerative products to address unmet medical needs. These treatments have the potential to reverse life-threatening diseases and extend the healthy human lifespan.

"My goal is to make it so the next generation grows up in a world where cancer is managed just like the common cold, and the body’s natural regenerative engine remains empowered throughout our lives," said Dr. Hariri, Founder, Chairman and Chief Executive Officer of Celularity. "Celularity is a new biotechnology company model founded to harness the placenta as a platform for discovery and therapeutics, ultimately with a goal of amplifying the body’s ability to fight disease, restore function and extend the healthy lifespan. It is my vision that the cellular medicines we derive from the placenta will lead to abundant and affordable treatments."

Celularity has assembled an industry-leading intellectual property portfolio of more than 800 issued patents, which attain a dominating position around placental-derived stem and progenitor cells. Celularity is also the first to own and deploy the full value chain ranging from sourcing placental stem cells to delivering patient treatment. The company is built on three key pillars: Cell Therapy, Functional Regeneration, and Biosourcing:

Cell Therapy: Celularity has developed five clinical stage cell therapy candidates currently being evaluated for cancer, and immunological and degenerative diseases
Functional Regeneration: Celularity is addressing serious wounds, burns, orthopedic, and other surgical indications including reconstructive and aesthetic applications with a broad range of placental biomaterials. Derived from placental tissue, Celularity’s BIOVANCE and Interfyl were the first biomaterial products introduced to the market to enhance the body’s regenerative processes
Biosourcing: Celularity owns and operates LifeBankUSA, the world’s only repository that allows families to bank their newborn’s placental cells and biomaterials for future therapeutic and regenerative use
Celularity is the only company with an allogeneic placental cell platform. Placental stem cells are uniquely immunoprivileged, therefore treatments do not require cells to be engineered or matched for each individual patient. Celularity is focused on this highly-scalable platform of technology to optimize economics and access to these cutting-edge therapies, including immuno-oncology. These placental stem cells allow for unprecedented scalability for Celularity’s CAR-T and CAR-NK platforms.

"Celularity’s technology has the potential to augment human immunity and longevity," said Dr. Peter H. Diamandis, Co-Founder and Vice-Chairman of Celularity. "The company’s ultimate vision is to make 100 years old the new 60, providing people with maximal cognition, mobility and aesthetics as they age. The 20 years of science, research, and intellectual property pioneered by Dr. Bob Hariri has the highest potential to become the cornerstone for this vision."

Andrew von Eschenbach, MD, among the founding members of the Celularity Board of Directors, and the former United States Food and Drug Administration (FDA) Commissioner and Director of the National Cancer Institute, said, "The pioneering work of Celularity founder Bob Hariri has unleashed the unique properties of placental derived stem cells, which have renewed hope for creating safe and effective therapies for the most challenging degenerative diseases." Dr. von Eschenbach added, "Celularity, with its focus on accelerating innovation in regenerative medicine, can become the leading catalyst for cell therapy to address many of the world’s unmet medical needs."

"Our investment in Celularity brings together its proprietary allogeneic placental platform and Sorrento’s best in class CAR-T products and cGMP manufacturing capabilities," said Henry Ji, another founding member of Celularity’s Board of Directors and Chairman, President and CEO of Sorrento Therapeutics. "Together, we can deliver these health solutions at scale to treat exponentially more people than we ever thought possible."