On August 6, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and The University of Texas MD Anderson Cancer Center reported a five-year collaboration agreement with a goal of evaluating therapies for multiple hematologic malignancies, including acute myeloid leukemia (AML) and myelodysplastic syndromes (Press release, Jazz Pharmaceuticals, AUG 6, 2018, View Source;p=RssLanding&cat=news&id=2362297 [SID1234528511]).

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The joint effort brings together MD Anderson’s translational medicine and clinical research capabilities with Jazz’s hematology/oncology portfolio, including its FDA-approved medicines as well as current and potential future investigational therapies.

"This collaboration represents a significant opportunity to efficiently develop innovative therapies and therapeutic combinations," said Tapan Kadia, M.D., associate professor of Leukemia at MD Anderson. "Our aim is to always provide leading-edge care for our leukemia patients, and it is our hope that this joint effort will result in new treatment solutions."

Jazz and MD Anderson will pursue research opportunities in areas of high unmet need. The initial focus of the collaboration is to evaluate and generate additional data for Vyxeos (daunorubicin and cytarabine) liposome for injection, in new patient populations and in combination with other therapies.

"Jazz is committed to providing meaningful medicines for people with hematologic cancers, particularly those with serious unmet clinical needs," said Allen Yang, M.D., Ph.D., vice president and acting chief medical officer of Jazz Pharmaceuticals. "We look forward to collaborating with MD Anderson to help advance treatment options for patients as part of our growing hematology oncology therapeutic area."

Vyxeos received FDA approval in August 2017 for the treatment of adults with newly-diagnosed therapy-related (t-AML) or AML with myelodysplasia-related changes (AML-MRC), which represents part of high-risk or secondary AML populations. AML-MRC is more common in older patients who often do not respond well to intensive chemotherapy; while t-AML can occur as a result of previous chemotherapy or radiation therapy.

About Vyxeos
Vyxeos is a liposome formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion. Vyxeos is indicated for the treatment of adults with newly-diagnosed t-AML or AML-MRC. For more information about Vyxeos in the United States, please visit View Source

Important Safety Information
Vyxeos has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute Vyxeos for other daunorubicin- and/or cytarabine- containing products.

Vyxeos should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine or any of its ingredients.

Vyxeos can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with Vyxeos. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

Vyxeos can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles or legs
unusual tiredness
Vyxeos may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
Vyxeos contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

Vyxeos can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site. Vyxeos can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving Vyxeos. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of Vyxeos.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Please see full Prescribing Information for Vyxeos including BOXED Warning at View Source

On August 6, 2018 amcure, a biopharmaceutical company developing first-in-class cancer therapeutics, reported the dosing of the first patient in the extension cohort of its ongoing phase I/Ib study evaluating AMC303, amcure’s lead compound (Press release, amcure, AUG 6, 2018, View Source [SID1234528453]). The decision to move into the second part of the clinical trial protocol was based on positive safety and pharmacokinetic data obtained in the dose escalation part and a recommendation by the data safety monitoring board. amcure will present top-line data from the dose-escalation part of the trial in an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, October 19-23 in Munich/Germany.

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AMC303 targets CD44v6, a key cell membrane molecule in pathways of several receptor-tyrosine-kinases, such as c-MET, VEGFR-2 and RON, which are involved in tumor growth and metastases. This approach provides a potential novel mechanism for the treatment of patients with advanced and solid tumors that have already begun to spread throughout the body.

The current study, which is being conducted in Belgium and Spain, is designed to assess the safety, tolerability and pharmacokinetics of multiple and increasing doses of AMC303 as monotherapy in patients with advanced metastatic malignant solid tumors of epithelial origin. The study also includes a comprehensive biomarker program. With the extension cohort, amcure is focusing its patient selection on patients with a moderate to high expression of the target molecule CD44v6 in four specific tumor types of squamous tumors: head and neck squamous cell carcinoma (HNSCC), squamous non-small-cell lung carcinoma (NSCLC), esophageal and cervical tumors.

"Our Phase I/Ib study with AMC303 is progressing as planned, demonstrating the safety and linear pharmacokinetic properties in a heavily pre-treated patient population. As drug combinations become increasingly the standard in today’s oncology practice, having a safe therapeutic option with a unique and additive mechanism of action would be an attractive asset. We look forward to continuing the development of AMC303 towards this goal with more preclinical data on the drug’s potential and on CD44v6’s disease biology and first clinical data being presented at the upcoming ESMO (Free ESMO Whitepaper) congress," said Klaus Dembowsky, CEO of amcure GmbH.

For more information on the trial please visit View Source

About AMC303

amcure’s lead compound, AMC303, is being developed as a potential treatment for patients with advanced and metastatic epithelial tumors, e.g. pancreatic cancer, head and neck cancer, gastric cancer, colorectal cancer, breast cancer and lung cancer. AMC303 has a high specificity for inhibiting CD44v6, a co-receptor required for signaling through multiple cellular pathways (c-Met, VEGFR-2, RON) involved in tumor growth, angiogenesis and the development and regression of metastases. AMC303 has demonstrated strong effects in various in vitro and in vivo assays.

On August 6, 2018 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that Jeff Baxter, President and CEO, will present at the Canaccord Genuity 38th Annual Growth Conference in Boston, MA, on Wednesday, August 8, 2018, at 9:30 AM ET (Press release, VBI Vaccines, AUG 6, 2018, View Source [SID1234528474]).

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A live webcast of the presentation and a subsequent replay may be accessed by visiting the Investors page of VBI’s website at: View Source A replay of the webcast will be archived on the company’s website for 90 days following the presentation.

Event: Canaccord Genuity 38th Annual Growth Conference
Date: Wednesday, August 8, 2018
Time: 9:30 – 9:55 AM ET
Event Website: View Source

On August 6, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported it has signed a License, Collaboration and Distribution Agreement with CMS Medical Venture Investment Limited ("CMS Medical") for the commercialization of Can-Fite’s Piclidenoson for the treatment of rheumatoid arthritis and psoriasis and Namodenoson for the treatment of advanced liver cancer and NAFLD/NASH in China (including Hong Kong, Macao and Taiwan) (Press release, Can-Fite BioPharma, AUG 6, 2018, View Source [SID1234528454]).

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Under the terms of the agreement, CMS Medical is making an upfront payment of $2,000,000 to Can-Fite and is required to pay to Can-Fite milestone payments of up to $14,000,000 upon the achievement of certain regulatory milestones and payments of up to $58,500,000 upon the achievement of certain sales milestones. In addition, the agreement provides for double-digit royalty payments on net sales.

CMS Medical is a wholly-owned subsidiary of China Medical System Holdings Limited ("CMS"; SEHK: 867), a specialty pharmaceutical company based in China, focusing on marketing, promotion and sales of prescription drugs and other medicinal products to therapeutic departments in hospitals. CMS builds up its product portfolio for its target markets by asset acquisition, equity investment, licensing and distribution as well as in-house R&D. CMS is listed on the Hong Kong Stock Exchange with a current market capitalization of approximately HK$34 billion as at August 1, 2018.

According to the agreement, CMS will be responsible for the development of Piclidenoson and Namodenoson to obtain regulatory approval in China and shall be further responsible for obtaining and maintaining regulatory approval in China for the indications described above. Can-Fite may, at the option of CMS, supply finished product to CMS.

"We are very excited to enter into this agreement with CMS, with their broad and professional experience in development, registration and marketing of diverse drugs in the Chinese market and believe that they are the right partner for us to penetrate this big market," stated Dr. Sari Fishman, VP Business Development of Can-Fite. "We believe that CMS’s commitment to us is strong validation of our development efforts to date."

Can-Fite is currently enrolling patients for its Phase III ACRobat trial of Piclidenoson for the treatment of rheumatoid arthritis and plans to shortly initiate patient enrollment for its Phase III Comfort trial of Piclidenoson for the treatment of psoriasis. The rheumatoid arthritis and psoriasis therapeutic market is dominated by biological drugs that are primarily administered via intravenous injection (IV) and have potential side effects. Rheumatoid arthritis and psoriasis are huge unmet need markets, where rheumatoid arthritis is estimated to reach $35B in 2020 and psoriasis is forecast to reach $9B in 2018.

Phase II studies with Namodenoson for the treatment of advanced liver cancer (hepatocellular carcinoma, Child Pugh B) and NAFLD/NASH are currently ongoing. The last patient for the advanced liver cancer trial was enrolled in August 2017, and treatment of remaining patients is still ongoing.

On August 6, 2018 bluebird bio, Inc. (NASDAQ: BLUE) and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported a collaboration to apply their respective technology platforms to the discovery, development and commercialization of novel immune cell therapies for cancer (Press release, bluebird bio, AUG 6, 2018, View Source [SID1234528455]). The collaborators will specifically leverage Regeneron’s VelociSuite platform technologies for the discovery and characterization of fully human antibodies, as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and cell therapy.

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"The collaboration with Regeneron complements bluebird bio’s growing immuno-oncology development portfolio, which includes clinical and pre-clinical CAR T and T cell receptor programs," said Philip Gregory, D.Phil., Chief Scientific Officer of bluebird bio. "With Regeneron’s proven targeting technologies, in combination with our deep expertise in cell biology and vector technology, as well as clinical experience with leading CAR T cell drug products, we hope to rapidly advance novel cellular therapies with the potential to transform the lives of people with cancer."

"Like Regeneron, bluebird is a science-focused company looking to push the limits of what novel technologies can do in drug discovery and development," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "We believe that the tremendous synergies between Regeneron’s proven technologies and bluebird’s toolbox of advanced cell and gene therapy technologies create a promising opportunity to help people with cancer by developing innovative new treatments. This collaboration adds yet another dimension to our rapidly advancing portfolio of immuno-oncology candidates and combination approaches."

The collaborators have jointly selected six initial targets and will equally share the costs of research and development up to the point of submitting an Investigational New Drug (IND) application. Additional targets may be selected over the five-year research collaboration term. When an IND is submitted for a potential cell therapy product, Regeneron will have the right to opt-in to a co-development/co-commercialization arrangement for certain collaboration targets, with 50/50 cost and profit sharing. If Regeneron does not opt-in, the company is eligible to receive milestone payments and royalties from bluebird bio on any potential resulting products.

Regeneron will also make a $100 million investment in bluebird bio common stock at a price of $238.10 per share, which represents a premium of 59 percent over the $150 closing price on August 3, 2018. This approximately $37 million premium will be credited against Regeneron’s initial 50 percent funding obligation for basic collaboration research, after which the collaborators will fund ongoing research equally. The transaction is subject to preclearance by the Federal Trade Commission under applicable antitrust laws.

Cell-based immunotherapies such as chimeric antigen receptor T cells (CAR Ts) use human immune cells (typically T cells derived from the patient with cancer) that are modified and returned to the patient to serve as therapeutic agents that specifically target and kill cancer cells. In advanced clinical studies, researchers have shown that modified T cells are highly active therapies in patients with a variety of blood cancers even after other treatment approaches have failed, and there are existing FDA-approved medicines that utilize this approach.

bluebird bio’s technologies use a customized lentiviral vector to modify T cells so that they can recognize tumor-specific proteins expressed by cancer cells and kill them upon engagement. Regeneron’s VelociSuite technologies, including VelocImmune and Veloci-T, enable the creation of fully-human antibodies and T cell receptors. These complementary technologies have the potential to expand the types of tumors that modified T cells can safely and effectively target by enabling the T cells to reach both extracellular and intracellular tumor antigens.