On December 20, 2018 Immatics Biotechnologie reported The prospect of an actively personalized approach to the treatment of cancer has moved a step closer with the recent publication in Nature of data from the phase 1 study GAPVAC-101, testing a novel therapeutic concept tailored to specific characteristics of patients’ individual tumors and immune systems (Press release, Immatics Biotechnologies, DEC 20, 2018, View Source [SID1234569548]). For the first time, the feasibility of such a highly personalized form of immunotherapy has been exemplified in a multi-center, multi-national clinical setting.

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To date, glioblastoma, an aggressive form of brain cancer with poor prognosis, and other tumor types have not sufficiently benefited from recent breakthroughs with checkpoint inhibitors due to lack of high mutational load which is thought to be essential for the mode of action of this therapeutic class. Indeed, many tumor types are characterized by a low mutational load and thus only few neoantigens are targetable by the immune system. Such cancers exhibit a high unmet medical need and require additional therapeutic strategies tailored to the features of the patient’s individual tumor and appreciating the entire breadth of the cancer target repertoire.

The Glioma Actively Personalized Vaccine Consortium (GAPVAC) approach is a highly personalized method being progressed through the GAPVAC-101 first-in-human clinical trial by a European Union-funded consortium, led by Immatics Biotechnologies GmbH (Tuebingen, Germany) and BioNTech AG (Mainz, Germany).

Fifteen newly diagnosed glioblastoma patients treated at six European centers received two immunotherapies in succession; APVAC1 targeted at non-mutated antigens, followed by APVAC2 preferentially targeted at neo-antigens. Immunotherapy compositions were personalized for each patient based on analysis of the transcriptome, immunopeptidome and mutanome of the individuals’ tumors and, for APVAC1, also based on the capability of each patient to mount an immune response. Both immunotherapy types displayed favorable safety and immunogenicity. Non-mutated APVAC1 antigens induced sustained central memory CD8+ T-cell responses, whilst APVAC2 induced T helper cell type 1 (TH1) CD4+ as well as CD8+ T-cell responses against predicted neo-epitopes.

The GAPVAC-101 trial served as a blueprint for the ACTolog IMA101-101 trial sponsored by Immatics. ACTolog is the first adoptive cell therapy trial applying the concept of active personalization.

Dr. Harpreet Singh, Chief Scientific Officer of Immatics and President & CEO of Immatics US, said: "We are at a very exciting stage in the evolution of tailor-made cancer treatments based on the diseases of individual cancer patients. The ability to exploit the full repertoire of tumor antigens, including non-mutated and neoantigens, may offer more effective immunotherapies, especially for tumors with low mutational load. And there is more to come. The next step is to translate the concept of active personalization, successfully demonstrated in this study, into adoptive cell therapies ‒ which we have achieved with the ACTolog IMA101 clinical trial Immatics is currently running at MD Anderson Cancer Center."

Prof. Hans-Georg Rammensee, Head of the Department of Immunology at the University of Tuebingen, Germany, and Co-Founder of Immatics added: "I am very pleased to see that the concept of active personalization proposed by us more than a decade ago has been applied for the treatment of glioblastoma patients. GAPVAC constitutes the first clinical trial using a combination of personalized mass spectrometry, next-generation sequencing, mRNA microarray, immune repertoire analysis and peptide GMP manufacturing for every patient and delivering these complex logistics in a multi-center multi-national clinical trial."

The approach of personalization of immunotherapies was first proposed by Prof. Hans-Georg Rammensee in 2000. His department was also responsible for the APVAC "on-demand" GMP manufacturing, led by Prof. Stefan Stevanovic, in the GAPVAC trial. The University of Tuebingen also served as one of the six European clinical trial centers treating glioblastoma patients.

On December 20, 2018 GT Biopharma, Inc. (OTCQB: GTBP and Euronext Paris GTBP.PA) ("GT Biopharma" or the "Company"), an immuno-oncology biotechnology company focused on innovative treatments based on the Company’s proprietary NK-engager and Bispecific Antibody Drug Conjugate platforms, reported that Raymond W Urbanski, M.D., Ph.D., Chief Executive Officer and Chairman, will present at Biotech Showcase 2019 on Tuesday, January 8, 2019 at 11:30 AM PST in San Francisco, CA (Press release, GT Biopharma , DEC 20, 2018, View Source [SID1234539517]).

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As part of his presentation, Dr. Urbanski will provide a corporate update and discuss GT Biopharma’s pipeline of immuno-oncology products based off the Company’s proprietary Tri-specific Killer Engager (TriKE), Tetra-specific Killer Engager (TetraKE) and bi-specific Antibody Drug Conjugate (ADC) technology platforms.

The Company’s most advanced bi-specific ADC in development, GTB-1550, targets CD19+ and/or CD22+ hematological malignancies and is currently in the Phase 2 component of a Phase 1/2 Non-Hodgkin’s Lymphoma (NHL)/Acute Lymphocytic Leukemia (ALL) trial which is an open-label, investigator-led study. GT Biopharma expects to announce topline results from the Phase 2a trial of GTB-1550 in the first quarter of 2019.

Additionally, the Company recently announced its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) is now open and it is authorized to initiate a first-in-human Phase 1 study with GTB-3550 (formerly OXS-3550), its first-in-class (TriKE), for the treatment of acute myelogenous leukemia (AML), myelodysplatic syndrome (MDS) and mastocytosis. The study, which is expected to commence in the first half of 2019, will be led by Principal Investigator, Sarah A. Cooley, MD, MS, Associate Professor, Division of Hematology, Oncology and Transplantation at Masonic Cancer Center, University of Minnesota. The Company believes that GTB-3550 could serve as a relatively safe, cost-effective, and easy-to-use therapy for refractory/relapsed AML, high-risk MDS and advanced systemic mastocytosis and could also be combined with chemotherapy and/or other agents as frontline therapy thus targeting a much larger patient population.

In addition to the presentation, Dr. Urbanski will also be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference. For more information about the conference, please click here to visit the conference website.

A live audio webcast of the presentation will be available on the Events page of the Investors section of the Company’s website (www.gtbiopharma.com). A webcast replay will be accessible for 90 days following the live presentation.

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 20, 2018 Endocyte, Inc. (Nasdaq:ECYT) ("Endocyte"), a biopharmaceutical company developing targeted therapeutics for cancer treatment, reported that at its Special Meeting of Stockholders held earlier today, Endocyte’s stockholders approved the adoption of the merger agreement pursuant to which Novartis AG will acquire Endocyte for $24 per share, or a total equity value of approximately $2.1 billion, in cash (Press release, Endocyte, DEC 20, 2018, View Source [SID1234532192]).

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Approximately 99.8% of the votes cast at the meeting voted to approve the adoption of the merger agreement, representing approximately 69.5% of Endocyte’s outstanding common stock as of the record date for the Special Meeting of Stockholders.

Subject to the satisfaction of customary closing conditions, the proposed merger is expected to close on or about December 21, 2018.

On December 20, 2018 Merus N.V. (Nasdaq:MRUS), a clinical-stage immuno-oncology company developing innovative bispecific antibody therapeutics (Biclonics), reported a settlement of all pending patent litigation and administrative proceedings between Merus and Regeneron Pharmaceuticals, Inc. pertaining to certain antibody generation platforms of each company (Press release, Merus, DEC 20, 2018, View Source;p=RssLanding&cat=news&id=2381425 [SID1234532208]).

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As part of the settlement, both parties have signed a global patent cross-license agreement, and Regeneron has agreed to provide an investment in Merus of $15 million through the purchase of 600,000 common shares at a price of $25 per share representing a premium of 118% from the close of trading on December 20, 2018. The cross-license and stock purchase are being made in conjunction with the dismissal of all claims to approximately $10.5 million for the reimbursement of attorney fees and other expenses, plus interest, awarded to Merus by the trial court on July 10, 2018. The settlement marks the end of all adversarial proceedings.

"This settlement marks the conclusion of a multi-year and multi-jurisdiction dispute," said Ton Logtenberg, Ph.D., President and Chief Executive Officer of Merus. "Today’s agreement to resolve these matters in an amicable manner is a positive result, and we believe, continues to ensure that Merus is able to further advance our Biclonics platform to discover and develop differentiated bispecific antibody therapeutics for cancer patients in need."

Under the terms of settlement, all worldwide patent proceedings related to the parties’ respective antibody generation platforms have been resolved. Under the global patent cross-license agreement, the parties have granted certain royalty-free rights to each other, while maintaining the uniqueness of their respective platform technologies. Neither party is licensed to exploit the other party’s products.

The settlement also resolves a pending Dutch proceeding of a related counterpart patent, which has been stayed since 2014, and a number of opposition proceedings in multiple jurisdictions the parties have outstanding.

"Merus’ platform technology and associated patents reflect the Company’s ongoing groundbreaking work in the field of bispecific antibody generation and development," said Peter B. Silverman, J.D., EVP and General Counsel of Merus. "This settlement further validates the strength of our IP portfolio and our ability to develop innovative Biclonics candidates."

Both parties have agreed to keep details relating to the global settlement confidential, other than what is disclosed in this press release or is otherwise required to be disclosed by law.

About U.S. Patent No. 8,502,018 (‘018 patent) Litigation
In March 2014, Regeneron filed a complaint against Merus alleging that it infringed one or more claims in the U.S. Patent No. 8,502,018 (‘018 patent), entitled "Methods of Modifying Eukaryotic Cells." Merus filed counterclaims seeking, among other things, a declaratory judgment that Merus did not infringe the ‘018 patent, that the ‘018 patent was invalid and a declaratory judgment of unenforceability of the ‘018 patent on the basis that the patent was procured by inequitable conduct. Merus prevailed at the trial court on these counterclaims. Regeneron stipulated to non-infringement and invalidity of the patent following the district court’s decision on claim construction. And after a trial in June 2015, the trial court concluded that Regeneron’s ‘018 patent was unenforceable. On July 27, 2017, the Federal Circuit affirmed. On December 26, 2017, the full Federal Circuit denied Regeneron’s request to rehear the matter, and the Supreme Court denied Regeneron’s petition for certiorari on October 1, 2018, ending the case in favor of Merus.

On March 26, 2018, the trial court granted Merus’ motion for attorneys’ fees, expert fees, and costs, and on July 10, 2018, granted Merus’ motion for approximately $10.5 million plus interest. Regeneron appealed the decision to the Federal Circuit. The parties have now agreed to dismiss the appeal and the claim for fees as a result of the settlement and Regeneron’s $15 million investment in Merus.