On January 6, 2019 Ascentage Pharma Group, Inc., and The University of Texas MD Anderson Cancer Center reported a five-year strategic collaboration agreement to advance the development of five potential new cancer therapies (Press release, Ascentage Pharma, JAN 6, 2019, View Source [SID1234532492]).

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The alliance is aimed at developing novel cancer therapeutics based upon Ascentage’s proprietary Protein-Protein Interaction drug discovery technology platform. MD Anderson’s leukemia team will join in efforts to advance the clinical development of Ascentage’s apoptosis-targeted and tyrosine kinase inhibitor candidates, including Bcr-Abl inhibitor HQP1351, Bcl-2/xL inhibitor APG-1252, Bcl-2 selective inhibitor APG-2575, IAP inhibitor APG-1387 and MDM2-p53 inhibitor APG-115. These compounds will be studied as single-agent therapies, as well as in combination with approved or investigational therapeutics against various forms of hematologic malignancies.

The strategic alliance leverages MD Anderson’s translational research and clinical expertise to help accelerate the development of the select candidates, chosen for their potential to target cancer cell apoptosis and other pathways for treating life-threatening cancers, such as Acute Myelocytic Leukemia (AML), Chronic Myeloid Leukemia (CML), Acute Lymphoblastic Leukemia (ALL), Myeloproliferative Neoplasms (MPN), and Myelofibrosis. The alliance will be led by Hagop Kantarjian, M.D., Chair of Department of Leukemia at MD Anderson, whose research and collaborations were the basis for the FDA approvals of over 20 drugs in leukemia.

"MD Anderson is highly dedicated to developing and providing more effective therapies for patients. This strategic alliance is important for our work towards finding cures to treat cancers. We will be investigating this pipeline of candidate therapies, and we are interested in the novel mechanism of their actions," said Dr. Kantarjian.

"We are pleased to announce this important partnership with MD Anderson," said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. "We look forward to working closely with the investigators in MDACC in the hopes of accelerating the clinical development of these important candidates to provide new treatment options for cancer patients in the US and worldwide."

On January 6, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported an update on its business performance, including preliminary net product sales results for 2018, and key clinical development programs for 2019 (Press release, Neurocrine Biosciences, JAN 6, 2019, View Source [SID1234532491]). Kevin Gorman, Chief Executive Officer of Neurocrine, will discuss these updates as part of a webcast presentation at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, Jan. 7 at 2:30 p.m. PT (5:30 p.m. ET).

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"Our fourth quarter and 2018 results reflect the dedication of our team in educating healthcare providers about tardive dyskinesia and the benefit INGREZZA can bring to patients. We still have a lot of work to do as many people suffering from tardive dyskinesia remain undiagnosed and untreated, and we remain committed to helping the lives of patients through our recently expanded field sales team and disease awareness education," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2019, we will remain focused on reaching more patients with INGREZZA and making progress in our clinical development programs, including the planned submission of AbbVie’s new drug application for uterine fibroids and our planned submission for Parkinson’s disease, as well as advancing our congenital adrenal hyperplasia program."

Preliminary Fourth Quarter and Full-Year 2018 Net Product Sales Results (Unaudited)

Based on preliminary unaudited financial information, the Company expects net product sales for the three months and full-year ended December 31, 2018 to be approximately $130 million and $409 million, respectively, compared to $64.5 million and $116.6 million for the same periods in 2017.

2019 Expected Milestones

INGREZZA (valbenazine) for Tardive Dyskinesia

"Talk About TD" disease state awareness campaign
Execution of post-marketing clinical studies, including RE-KINECT, the largest real-world study in patients with possible tardive dyskinesia
Presentations at key scientific annual meetings, including American Academy of Neurology (AAN), American Psychiatric Association (APA), International Parkinson and Movement Disorder Society (MDS)
Elagolix in Collaboration with AbbVie

Continued launch of ORILISSA (elagolix) to treat endometriosis by AbbVie
Elagolix for uterine fibroids: planned 2019 New Drug Application (NDA) submission
Opicapone for Parkinson’s Disease

NDA submission in Q2 2019
Preparation for 2020 opicapone commercial launch
Presentations at key scientific annual meetings, including AAN, MDS
Congenital Adrenal Hyperplasia (CAH)/NBI-74788 (In Development)

Phase IIa data for CAH (adults) in Q1 2019
Phase IIa initiation for CAH (pediatric) in Q2/Q3 2019
Pivotal study initiation for CAH (adults) in 2H 2019, pending U.S. Food and Drug Administration (FDA) discussion in Q2
Early Stage Programs

Investigational New Drug submission and initiation of a Phase I trial for a new, internally discovered program
About INGREZZA (valbenazine) Capsules
INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications
INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

On January 6, 2019 The University of Texas MD Anderson Cancer Center and 4D pharma reported a strategic collaboration to evaluate 4D’s live biotherapeutic oncology pipeline across a range of cancer settings (Press release, MD Anderson, JAN 6, 2019, View Source [SID1234553989]).

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The alliance brings together MD Anderson’s translational medicine and clinical research capabilities with 4D’s expertise in the discovery and development of live biotherapeutics. The collaboration will initially assess the 4D’s lead oncology candidate, MRx0518, as a potential treatment for solid tumors.

The first clinical trial, an open label Phase I study of MRx0518 in combination with Keytruda and conducted in collaboration with Merck & Co., Inc., Kenilworth, N.J., has been initiated and is due to open shortly. The study will enroll up to 132 patients with metastatic cancer across multiple histologies who progressed on prior anti-PD-1 therapy.

Subsequent studies are also being planned under the collaboration, including using MRx0518 in combination with stereotactic body radiotherapy (SBRT) for the treatment of pancreatic cancer.

"This alliance, with one of the leading oncology research centers in the world, will provide a strong and long-term foundation for the development of 4D’s live biotherapeutics in cancer," said Duncan Peyton, 4D’s chief executive officer. "The current study, which is one of the first live biotherapeutic programs to reach the clinic in the immuno-oncology space, represents an important step forward in the development of MRx0518 and 4D’s broader oncology franchise. We look forward to continuing to work with our partners at MD Anderson to progress this study and help bring this therapy to patients."

On January 6, 2019 GE Healthcare and Vanderbilt University Medical Center (VUMC) reported a five-year partnership to enable safer and more precise cancer immunotherapies (Press release, GE Healthcare, JAN 6, 2019, View Source [SID1234532487]). Multiple diagnostic tools will be developed to help predict both the efficacy of an immunotherapy treatment and its adverse effects for a specific patient before the therapy is administered. This would allow physicians to better target immunotherapies to the right patients and avoid potentially damaging, ineffective and costly courses of treatments.

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Immunotherapies use the immune system to recognize and attack cancer cells and can be more effective than traditional treatments, but response rates are often low and side effects can be severe1. GE Healthcare and VUMC will retrospectively analyze and correlate the immunotherapy treatment response of thousands of VUMC cancer patients, with their anonymized demographic, genomic, tumor, cellular, proteomic and imaging data. They will then develop AI-powered apps that draw on this data to help physicians identify the most suitable treatment for each individual patient.

Simultaneously, GE Healthcare and VUMC will develop new positron-emission tomography (PET) imaging tracers, which together with the apps, will help physicians to stratify cancer patients for clinical trials. It currently takes an average of 12 years2 and costs almost $2bn3 to bring a drug to market. In many cases, inappropriate patients are recruited to participate in immunotherapy trials, incurring unnecessary expense and slowing down approvals of new therapies. It is hoped that the PET tracers will ultimately also be used to monitor the efficacy of immunotherapies in everyday practice.

"Immunotherapy offers tremendous promise but given the current unpredictability of some patients’ reactions to treatments, it is also associated with increased morbidity and cost. This partnership provides the opportunity to leverage strengths of both of our organizations to further personalize cancer care by creating new tools that allow clinicians to more accurately predict how patients will respond to a specific therapy," said Jeff Balser, MD, PhD, President and Chief Executive Officer, Vanderbilt University Medical Center and Dean of the Vanderbilt University School of Medicine.

"GE Healthcare and Vanderbilt will combine their data science, genomic, imaging and cellular analysis capabilities to help improve clinical decision making. This partnership is a great example of the increasing convergence of the tools, technologies and data used by therapy innovators and healthcare providers," said Kieran Murphy, President and Chief Executive Officer, GE Healthcare.

GE Healthcare and the Vanderbilt-Ingram Cancer Center, a world-renowned stem cell transplant facility, will also collaborate on methods to improve productivity, efficiency and cost of stem cell transplant processing operations by automating processes, digitizing workflows, improving throughput and industrializing operations.

The first analytics application prototype will be available by the end of 2019 and the PET tracer proof-of-concept by the end of 2020.

On January 6, 2019 Samsung Bioepis Co., Ltd. reported that its rapidly growing biosimilar business will expand into mainland China through a licensing agreement with 3SBio Inc (Press release, Samsung Bioepis, JAN 6, 2019, View Source [SID1234532488]). The agreement covers multiple biosimilar candidates from Samsung Bioepis, including SB8, a biosimilar candidate referencing AVASTIN 1 (bevacizumab).

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Under the agreement, Samsung Bioepis and 3SBio will collaborate across a number of areas, including clinical development, regulatory registration and commercialization in China. Samsung Bioepis will receive upfront and milestone payments, as well as royalties on sales. Additional financial details were not disclosed.

"We are very excited to expand our biosimilar business into China, where we hope to see our biosimilars play an important role in widening patient access to high-quality healthcare. We are confident we will achieve this goal through our partnership with 3SBio, which brings together Samsung Bioepis’ proven development platform with 3SBio’s strong commercialization platform," said Christopher Hansung Ko, President and Chief Executive Officer, Samsung Bioepis. "At Samsung Bioepis, we will continue to demonstrate our enduring commitment to biosimilars by further strengthening our pipeline and widening their availability for patients and healthcare systems across the world."

Established in February 2012, Samsung Bioepis currently has four biosimilars approved and marketed across Europe, which include the anti-TNF trio of BENEPALI (etanercept), FLIXABI (infliximab) and IMRALDI (adalimumab), as well as an oncologic biosimilar, ONTRUZANT (trastuzumab). In the United States, the company has one biosimilar approved and marketed, RENFLEXIS (infliximab), with both SB5 (adalimumab) and SB3 (trastuzumab) biosimilar candidates currently under regulatory review.

In total, over 100,000 patients across the world are currently under treatment with Samsung Bioepis’ biosimilars, with over 6 million doses administered.