On February 15, 2018 AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known as MSD outside the US and Canada) reported that the US 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, AstraZeneca, FEB 15, 2018, View Source [SID1234523985]).

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NF1 is an incurable genetic condition that affects one in 3,000 births,[i] with highly-variable symptoms, including cutaneous (skin), neurological (nervous system) and orthopaedic (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 a neurological manifestation of NF1 and arise from nerve fascicles that tend to grow along the length of the nerve. PNs occur in approximately 20-50% of NF1 patients causing pain, motor dysfunction and disfigurement.[ii]

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer, at 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."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, at MSD Research Laboratories, said: "We’re looking forward to working with our colleagues at AstraZeneca to develop selumetinib and understand how it may benefit patients with NF1."

The potential benefit of selumetinib in NF1 is being explored in the US National Cancer Institute-sponsored Phase I/II SPRINT trial in paediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018.

The FDA’s ODD programme 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 US.

In addition to NF1, selumetinib is being investigated in the Phase III 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 III/IV DTC in 2016. It is also being explored as a monotherapy and in combination with other treatments in Phase I trials.

NOTES TO EDITORS
About neurofibromatosis type 1 (NF1)

NF1 is caused by a spontaneous or inherited mutation in the NF1 gene. 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% of patients, tumours 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 tumours, and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.[iii]

About selumetinib

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

The NF1 gene provides instructions for making a protein called Neurofibromin, which negatively regulates the RAS/MAPK pathway, helping to control cell growth, differentiation and survival. Mutations in the NF1 gene may result in dysregulations in RAS/RAF/MEK/ERK signalling, which can cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumour growth. Selumetinib inhibits the MEK enzyme in this pathway, potentially leading to inhibition of tumour growth.

About the AstraZeneca and MSD Strategic Oncology Collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza (olaparib), the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. The collaboration is based on increasing evidence that PARP and MEK inhibitors can be combined with PD-L1/PD-1 inhibitors for a range of tumour types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as a monotherapy. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death

On February 15, 2018 Cambrex Corporation (NYSE:CBM), a leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), reported that Steven Klosk, President and Chief Executive Officer, will participate in a fireside chat at the 2018 RBC Capital Markets Global Healthcare Conference on February 22, 2018 at 3:05 p.m. EST in New York City (Press release, Cambrex, FEB 15, 2018, View Source [SID1234524000]).

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The live audio webcast and slide presentation can be accessed from the Cambrex website at www.cambrex.com in the Investors section under "Webcasts & Presentations", and a replay will be available for 90 days after the live event concludes.

On February 13, 2018 Advaxis, Inc. (NASDAQ:ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of cancer immunotherapies, reported that it has submitted a conditional Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the company’s lead Lm Technology product candidate, axalimogene filolisbac, for the treatment of adult women who progress beyond first-line therapy of persistent, recurrent or metastatic carcinoma of the cervix (PRmCC) (Press release, Advaxis, FEB 15, 2018, View Source [SID1234523996]).

"The submission of the MAA represents a significant regulatory milestone for Advaxis and the ongoing development of our Lm Technology Platform," stated Anthony Lombardo, interim Chief Executive Officer of Advaxis. "The submission is based on the improvement in 12-month survival rates observed in the Phase 2 GOG-0265 study. We feel that these data support axalimogene filolisbac as a potential therapeutic option for patients living with PRmCC who are in desperate need of new treatment options beyond first-line therapy," added Lombardo.

The MAA submission is built around data from the GOG-0265 study which examined overall survival rates in 50 women and showed a 12-month overall survival rate (primary efficacy endpoint) of 38% (n=19/50) in women with PRmCC, representing a 55% improvement over an expected, model-predicted,12-month survival rate of 24.5%.2 More than 50% of treated women in this study had previously received multiple prior lines of therapy including treatment with bevacizumab and subsequently experienced progression of their disease.2

"Despite the availability of preventative measures, metastatic cervical cancer continues to be a major public health concern associated with high mortality rates within Europe," said Mansoor Mirza, M.D., Chief Oncologist at the Copenhagen University Hospital in Denmark and Medical Director of the Nordic Society of Gynaecological Oncology (NSGO). "The results from GOG-0265 are encouraging and could represent a meaningful step forward in the care of women suffering from PRmCC, which has seen very little innovation in almost 30 years."

In the GOG-0256 study, axalimogene filolisbac was generally well-tolerated with mostly Grade 1 and 2 flu-like adverse events associated with cytokine release which were managed with standard medical care. This safety profile is consistent with the ongoing clinical experience of axalimogene filolisbac across all clinical trials.

The EMA will evaluate the totality of the data, including results from GOG-0265 as well as supportive data from other clinical trials evaluating axalimogene filolisbac. In parallel with the MAA review process, the company will continue assessing partnership opportunities for the potential commercialization of axalimogene filolisbac in Europe.

The company has also decided to align and simplify its strategy by using axalimogene filolisbac exclusively in all ongoing and planned HPV-related cancer clinical trials, including the upcoming ADVANCE trial, previously planned with ADXS-DUAL. The strategic decision to harmonize all trials to axalimogene filolisbac is based on its clinical profile to date in over 250 patients, and its demonstration of similar activity in both HPV 16 and 18 subtypes in GOG-0265. The company believes that harmonizing to a single product candidate for all HPV-related programs will streamline developmental, regulatory and commercialization strategies.

About Axalimogene Filolisbac

Axalimogene filolisbac is a targeted Listeria monocytogenes (Lm)-based investigational immunotherapy that attacks HPV-associated cancers by altering a live strain of Lm bacteria to generate cancer-fighting T cells against cancer antigens while neutralizing the tumor’s natural protections that guard the tumor microenvironment from immunologic attack.

Axalimogene filolisbac has already achieved multiple regulatory milestones, including classification as an EMA advanced therapy-medicinal product for the treatment of cervical cancer, receipt of the U.S. Food and Drug Administration (FDA) Fast Track Designation as an adjuvant therapy for treating high-risk, locally advanced cervical cancer (HRLACC), receipt of a Special Protocol Assessment agreement with the FDA for the Phase 3 AIM2CERV trial, and orphan drug designations in three HPV-associated indications (PRmCC, head and neck, and anal cancer). In addition, axalimogene filolisbac will be studied in combination with nivolumab in the ADVANCE trial, a potential registrational trial for patients with PRmCC, which is planned to begin in 2018.

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 GT Biopharma, Inc. (OTCQB: GTBP) (GTBP.PA) ("GT" or the "Company"), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel cancer immunotherapy products as well as central nervous system treatments, reported that Shawn M. Cross will become Chairman and Chief Executive Officer effective February 15, 2018 (Press release, GT Biopharma , FEB 15, 2018, View Source [SID1234539533]).

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Mr. Cross has held the position of President and Chief Operating Officer of GTBP since November 16, 2017. The plan to add The Chairman of the Board and CEO positions to Mr. Cross’ duties as President/COO was initiated by the Company’s board of directors to prepare the company for its next phase of growth. Mr. Cross will lead GTBP through NASDAQ capital markets listing process, attract institutional investment and analyst coverage to the company, and guide development of its neurology portfolio and its first TriKE into human clinical testing in 2018.

Dr. Kathleen Clarence-Smith will become Vice Chairwoman of the Board of Directors and President of the Neurology Division to focus her efforts on creating value in GTBP’s central nervous system portfolio and drive the company’s intellectual property developments.

Anthony J. Cataldo will remain as a member of the Board of Directors and become a consultant to the company.

Mr. Cross is an internationally recognized healthcare investment banker specializing in the biopharmaceutical sector with over 20 years of experience and has completed dozens of capital markets and strategic transactions for growth-oriented biopharmaceutical companies. He joined GTBP as President and Chief Operating Officer of the Company. Previously he was Managing Director, Head of Biotechnology Investment Banking at Deutsche Bank Securities Inc. and Managing Director and Head of Biopharmaceutical Investment Banking at Wells Fargo Securities, LLC where he completed dozens of capital markets and strategic transactions for growth-oriented biopharmaceutical companies.

GT Biopharma Executive Chairman Anthony J. Cataldo said, "It is now time to move GT Biopharma to an institutional quality biotech company. I created GTBP using the same model I used when creating, what is now known as, Iovance Biotherapeutics, Inc. (IOVA); an approximately $1.5 billion market cap company. The leadership provided by Shawn Cross, Dr. Kathleen Clarence-Smith and Dr. Ray Urbanski, our Chief Medical Officer will be invaluable to moving these assets towards clinical and commercial success. I am excited for our shareholders and look forward to helping the board and management progress GT Biopharma to becoming a significant biotech company."

GT Biopharma Chief Executive Officer Dr. Kathleen Clarence-Smith said, "Having known Shawn for over a decade, I’m confident that Shawn will be a very effective CEO for GTBP while I focus on maximizing the value of our neurology portfolio. I look forward to moving the company to the next level."

GT Biopharma President and COO Shawn M. Cross said, "I continue to see a great deal of promise in GT Biopharma’s pipeline of immuno-oncology and neurology therapeutics and am honored to become Chairman and Chief Executive Officer of such a dynamic company. I look forward to announcing additional experienced members to our management team and board in the coming weeks. I also am excited to work closely with Dr. Urbanski as we prepare to enter our first TriKE into human clinical testing as well as with Dr. Clarence-Smith in creating value from our neurology portfolio."