On December 20, 2018 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immunotherapies derived from its Immulytic platform, reported that Robert Coffin, Ph.D., Chief Executive Officer and Director of Replimune, will present at the 37th Annual J.P.Morgan Healthcare Conference on Wednesday, January 9, 2019 at 8:00 AM PT at the Westin St. Francis Hotel in San Francisco, CA (Press release, Replimune, DEC 20, 2018, View Source [SID1234532180]).

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A simultaneous webcast will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.

On December 20, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that the European Commission (EC) has granted marketing authorization for a split dosing regimen for DARZALEX (daratumumab) (Press release, Genmab, DEC 20, 2018, View Source [SID1234532178]). The approval will be included in an update of the Summary of Product Characteristics in order to provide health care professionals the option to split the first infusion of DARZALEX over two consecutive days. The EC approval follows a positive opinion issued for DARZALEX by the European Committee for Medicinal Products for Human Use (CHMP) on November 18th, 2018. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"We are hopeful that the availability of this more flexible dosing option will make the first infusion of DARZALEX more convenient for European multiple myeloma patients," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

This approval was based on data from the Phase Ib EQUULEUS (MMY1001) clinical trial, which demonstrated DARZALEX pharmacokinetics (PK) concentrations were comparable regardless of whether the first dose was administered as a split infusion or as a single first infusion in patients with multiple myeloma. The safety profile of DARZALEX was comparable when administered initially as either a split or a single dose.

About the EQUULEUS (MMY1001) Study
The Phase Ib EQULLEUS open-label study includes up to 240 patients with the goal of evaluating the safety, tolerability and dose of daratumumab when administered in combination with various backbone treatment regimens for different settings of multiple myeloma.

About DARZALEX(daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.1 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United Stated, Europe and Japan. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).1,2,3,4,5

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, myelodysplastic syndromes, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On December 20, 2018 Merck KGaA, Darmstadt, Germany, a leading science and technology company, reported that through its wholly owned subsidiary, Ares Trading, it has evolved its agreement with Intrexon Corporation (NASDAQ: XON) for the development of Chimeric Antigen Receptor T-cell (CAR-T) therapies, genetically engineered T-cells with synthetic receptors that recognize a specific antigen expressed on tumor cells (Press release, Merck KGaA, DEC 20, 2018, View Source;utm_medium=email&utm_campaign=press-mailer&utm_content=en&global_redirect=1 [SID1234532177]). The agreement with Intrexon and its wholly-owned subsidiary, Precigen, Inc. enables Merck KGaA, Darmstadt, Germany, to continue to implement its focused R&D strategy, while maintaining an investment in the future potential of next-generation CAR-T development.

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Under the terms of the agreement, Merck KGaA, Darmstadt, Germany, will assign its exclusive CAR-T development rights to Intrexon. Merck KGaA, Darmstadt, Germany will receive shares of Intrexon common stock valued at $150 million in exchange for assigning Intrexon its CAR-T rights.

"Merck KGaA, Darmstadt, Germany, is excited to maintain its interest in the potential of CAR-T technology, which may offer significant future benefits to patients fighting cancer," said Belén Garijo, Member of the Executive Board and CEO Healthcare, Merck KGaA, Darmstadt, Germany. "The agreement is also illustrative of our efforts to enhance our focus on accelerating the delivery of our innovative clinical pipeline to patients."

Merck KGaA, Darmstadt, Germany, first entered into a collaboration and license agreement with Intrexon in 2015 to develop and commercialize CAR-T cancer therapies utilizing Intrexon’s proprietary RheoSwitch Therapeutic System and the Sleeping Beauty non-viral gene integration technology. The combination of these platforms enables regulation of gene expression and delivery with a non-viral approach and preclinical data indicate the potential to improve therapeutic safety and facilitate shortened manufacturing to improve time-to-treatment. As of December 31, 2017, these rights were considered intangible assets not yet available for use with a carrying amount of € 104 million.

"Merck KGaA, Darmstadt, Germany’s leading immuno-oncology research and commitment to developing innovative medicines made them an ideal partner for us in advancing targeted and controllable CAR-T therapies," said Helen Sabzevari, PhD, President of Precigen. "We look forward to continued development of these promising treatments with the goal of delivering more cost-effective, powerful, and precise therapies to patients in need."

In addition to receiving $150 million of Intrexon common stock, this agreement also includes a further $25 million investment in Intrexon. In return, Merck KGaA, Darmstadt, Germany, will receive a $25 million convertible note, providing the option to receive either Precigen or Intrexon stock. The closing of the transactions contemplated by the agreement is subject to customary closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.

On December 20, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the
development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR T)
technology and gene therapies for rare diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and incurable blood cancer with a median survival of less than 18 months and no standard of care (Press release, Mustang Bio, DEC 20, 2018, View Source [SID1234532176]).

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Martina Sersch, M.D., Ph.D., Chief Medical Officer of Mustang, said, "We are pleased to receive Orphan Drug
Designation for MB-102, which has shown the potential to address an area of high unmet medical need. This significant milestone for Mustang will provide additional market exclusivity and financial benefits to advance MB-102, which we believe is an important new treatment for patients with BPDCN. Based on the Phase 1 data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in November 2018, we expect to initiate a multicenter Phase 1/2 clinical trial in patients with acute myeloid leukemia (AML), BPDCN and high-risk myelodysplastic syndrome in 2019."

MB-102 is currently being studied in a single center, first-in-human Phase 1 dose-escalation clinical trial evaluating the
safety and anti-tumor activity of escalating doses of MB-102 in patients with relapsed or refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a single dose of MB-102 with an option for a second infusion if they continue to meet safety and eligibility criteria and still have CD123+ disease. MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities.

The FDA grants Orphan Drug Designation to drugs and biologics that are 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. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.

About Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and incurable blood cancer with a median survival of less
than 18 months and no standard of care. High levels of CD123 expression is one of the diagnostic hallmarks of BPDCN, making CD123 an attractive target for T cell-based adoptive immunotherapy.

About MB-102 (CD123 CAR T)
MB-102 (CD123 CAR T) is a CAR T cell therapy that is produced by engineering patient T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with myelodysplastic syndromes, as well as in hematologic malignancies including AML, B-cell acute lymphoblastic leukemia, hairy cell leukemia, BPDCN, chronic myeloid leukemia and Hodgkin’s lymphoma.

In the first-in-human clinical trial at City of Hope (NCT02159495), MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in November 2018. Dose escalation continues at City of Hope in both indications.

On December 19, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP) and TriArm Therapeutics, Ltd, reported they will launch Eden BioCell, Ltd. to lead clinical development and commercialization of Sleeping Beauty-generated CAR-T therapies in the People’s Republic of China (including Macau and Hong Kong), Taiwan and Korea (Press release, Ziopharm, DEC 19, 2018, View Source [SID1234573334]). TriArm is a cell therapy company with operations in Germany, China and the United States that was formed by Panacea Venture Healthcare, a fund co-founded and managed by James Huang, Managing Partner of Kleiner Perkins Caufield & Byers China (KPCB China).

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For the territory of China, Taiwan and Korea, Ziopharm will license the rights to Eden BioCell for third-generation Sleeping Beauty-generated CAR-T therapies targeting the CD19 antigen. Eden BioCell will be owned 50-50 by Ziopharm and TriArm. TriArm has committed up to $35 million to this joint venture. Under the terms of the agreement, Eden BioCell has rights in the region to CAR-T cells very rapidly manufactured in two days or less using the Sleeping Beauty platform to express a CD19-specific CAR and membrane-bound interleukin-15, or mbIL15, along with a kill switch. Ziopharm CEO Laurence Cooper, M.D., Ph.D., and Panacea Venture Healthcare Managing Director James Huang will serve on Eden BioCell’s Board of Directors and each party will share decision-making authority.

"Since 2017, we have been doing diligence on ways to address the many obstacles that exist in the production and commercialization of CAR-T immunotherapy. Viral-based CAR-T therapies already are facing cost constraints and limited commercial success in U.S. and EU markets due to the expense and complexity in manufacturing, and this problem will be exacerbated in China, with its healthcare system, reimbursement structure and large patient population," said Mr. Huang. "I am excited to be working with Ziopharm’s Sleeping Beauty platform, which, as the most clinically-advanced non-viral approach to genetical modification of T cells, offers the best chance to simplify manufacturing, reduce costs, and most importantly, help the many patients who need access to these T-cell therapies."

"Advancing our Sleeping Beauty platform in the China region is a key part of both our business development and clinical development strategies," said Dr. Cooper. "James Huang has an outstanding track record of creating value in China, and he and the team at TriArm are ideal partners because they have entrenched relationships with front-line physicians and officials at leading hospitals and regulatory bodies, a commitment to conduct high-quality trials, and state-of-the-art facilities with good manufacturing practices."

TriArm will manage all clinical development to execute trials in China for Eden BioCell. The TriArm team has considerable experience in all areas of drug development, including scientific research, clinical and regulatory areas, as well as significant laboratory and manufacturing know-how regarding T-cell therapies.

Griffin Securities, Inc. acted as the financial advisor for Ziopharm for this licensing agreement.

Conference Call
Ziopharm will host a webcast and conference call on Wednesday, Dec. 19, at 8 a.m. ET to discuss this new joint venture. The call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 3272014. To access live webcast or the subsequent archived recording, visit the "Investors Events and Presentations" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.