On March 30, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported a corporate update and announced 2016 financial results (Press release, Mateon Therapeutics, MAR 30, 2017, View Source [SID1234518379]).

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Corporate Achievements During 2016

Changed company’s name to Mateon Therapeutics, Inc. to reflect focus on combination vascular targeted therapy in orphan oncology indications
Re-invigorated Board of Directors with complete changeover of independent members during 2016, adding three experienced biopharmaceutical industry leaders: Donald R. Reynolds, Bobby W. Sandage, Jr., Ph.D., and Simon C. Pedder, Ph.D.
"2016 was a key year for execution of the business plans that we developed in the months after I started as CEO in mid-2015, as we initiated our important FOCUS study, advanced the PAZOFOS study and moved our OXi4503 study in AML into its combination-treatment phase," stated William D. Schwieterman, M.D., Mateon’s President and Chief Executive Officer. "We also rebranded the company and freshened our board, bringing in new outside board members with deep industry expertise as we made meaningful changes based on our new direction. Now, as the first quarter of 2017 nears completion, we are very encouraged by the early data in AML. We are also eagerly awaiting next month the first randomized clinical trial data from the company in years, as we conduct a scheduled interim analysis of FOCUS in platinum-resistant ovarian cancer."

Key 2016 and Recent Developments

CA4P in combination with bevacizumab for Treatment of Ovarian Cancer

• Presented new data reflecting improved survival outcomes for CA4P-treated patients from Study GOG-0186I, an open-label randomized Phase 2 clinical trial in recurrent ovarian cancer

Data demonstrated an improvement of 5.6 months in overall survival and 3.7 months in progression-free survival in patients with measurable disease
• Published positive results from Study GOG-0186I in Journal of Clinical Oncology, official journal of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). Study was funded by National Cancer Institute, conducted by Gynecologic Oncology Group (GOG).

• Initiated the FOCUS Study, a Phase 2/3 clinical trial of CA4P in combination with bevacizumab (Avastin) and chemotherapy in platinum-resistant ovarian cancer

Currently have 25 sites actively recruiting patients
CA4P in combination with pazopanib for Treatment of Recurrent Ovarian Cancer

• Initiated enrollment in Phase 2 portion of the PAZOFOS Study, a Phase 1b/2 clinical trial of CA4P in combination with pazopanib (Votrient)

CA4P in combination with bevacizumab for Treatment of Glioblastoma Multiforme

• Received Orphan Drug Designation for CA4P for the treatment of glioma from U.S. Food and Drug Administration (FDA)

CA4P for Treatment of Neuroendocrine Tumors (NETs)

• Received Orphan Drug Designation for CA4P for the treatment of NETs from both the FDA and the European Commission

• Presented data from monotherapy study in NETs and announced an investigator-sponsored study in NETs using CA4P in combination with everolimus (AFINITOR)

OXi4503 in combination with cytarabine for Treatment of Relapsed/Refractory Acute Myeloid Leukemia (AML)

• Completed enrollment of first three cohorts and initiated the fourth cohort of OX1222, an open-label Phase 1b dose-ranging study of OXi4503 in combination with cytarabine

• Presented data from OX1222 at 58th Annual Meeting of American Society of Hematology (ASH) (Free ASH Whitepaper)

• Presented updated data from OX1222 at the 29th Annual ROTH Conference

Highlights include 3 patients, one from each dose cohort, experiencing complete remission
Additional Pipeline Developments

• Announced encouraging preliminary data from four syngeneic mouse models evaluating CA4P in combination with checkpoint inhibitors

Most compelling results were found combining CA4P with an anti-CTLA4 antibody in an EMT-6 mammary model – 7 of 8 mice receiving combination were tumor free at study’s completion, compared to 1 of 8 in CA4P monotherapy arm and 2 of 8 in anti-CTLA4 antibody monotherapy arm
Financial Results

For the year ended December 31, 2016, Mateon reported a net loss of $13.7 million, similar to the net loss for the year ended December 31, 2015. R&D expenses decreased to $8.8 million in 2016 compared to $9.1 million in 2015, while general and administrative expenses increased to $5.0 million in 2016 compared to $4.6 million in 2015.

At December 31, 2016, Mateon had cash and short-term investments of $12.0 million.

On March 2017 Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE:OXB), a leading gene and cell therapy group, reported the announcement by Novartis that the US Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) (Press release, Oxford BioMedica, MAR 30, 2017, View Source [SID1234518317]). The priority review designation is expected to shorten the anticipated review time by the FDA.

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Oxford BioMedica is the sole manufacturer of the lentiviral vector expressing CTL019 for Novartis. As announced in October 2014, Oxford BioMedica will also receive undisclosed royalties on potential future sales of Novartis CAR-T products.

John Dawson, Chief Executive Officer of Oxford BioMedica, commented: "The news that the FDA has accepted the BLA for CTL019 and granted it priority review is an important development for Oxford BioMedica. We continue to work closely with Novartis in delivering the lentiviral vector expressing CTL019, a product described earlier this year by Novartis as having "blockbuster" potential."

On March 30, 2018 Redx Pharma reported that it has been awarded aUS$1 million grant by CARB-X, one of the world’s largest public-private partnerships, launched in July 2016 to accelerate global antibacterial innovation and research (Press release, Redx Pharma, MAR 30, 2017, View Source [SID1234524747]). The 11 successful projects were selected through a competitive process from 168 applications from around the world. The awarded grants were based on the merits of each of the company’s research proposals, as evaluated by the CARB-X Advisory Board and the CARB-X team.

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Today’s announcement by CARB-X showcases the first projects selected for the Powered byCARB-X portfolio, spotlighting Redx as a highly-respected partner of choice on a global stage. Redx will receive US$1 million over 18 months, with an option for future tiered milestone payments, to drive scientific progress against globally challenging drug resistant Gram-negativebacteria. This collaboration with CARB-X enables Redx to move its Gram-negative program into the next stage of development with a prospective partner.

Dr Neil Murray, Chief Executive Officer of Redx Pharma, said: We are delighted to be collaborating with CARB-X, a truly innovative initiative that is targeting the development of new life-saving antibacterials and diagnostics. Receiving this grant today is testament to the great science that exists at Redx. This grant cements a path forward for our recently announced strategy, to secure external partners to ensure that our high quality anti-infective science will continue whilst we sharpen our focus on the clinical development of our oncology and immunology programs.

On March 30, 2017 Third Rock Ventures, LLC reported the launch of Tango Therapeutics, a new cancer therapeutics company discovering and developing novel medicines designed to target cancer vulnerabilities beyond mutated oncogenes to deliver transformational new therapies for patients (Press release, Tango Therapeutics, MAR 30, 2017, View Source [SID1234520724]).

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Tango was launched with a $55 million Series A investment from Third Rock Ventures. The company has established a robust product engine that leverages advances in DNA sequencing and CRISPR-based target discovery to generate breakthrough medicines that will provide deeper, more sustained benefit than today’s targeted therapies, and extend the benefit of available immuno-oncology agents.

"Cancers are complex genetic diseases marked by multiple lesions in each tumor. These include genes that are turned on to drive cancer growth and those that are inactivated and thus, unable to function as tumor suppressors," said Barbara Weber, M.D., Tango’s interim Chief Executive Officer and a Venture Partner at Third Rock Ventures. "Loss of tumor suppressor genes is a hallmark of cancer, but the genes, themselves, are not tractable targets for drug discovery. The availability of comprehensive DNA sequencing, coupled with CRISPR-enabled target discovery, provides us with new paths to identify novel drug targets and combinations that take advantage of vulnerabilities created by loss of tumor suppressor gene function — something we have been unable to do effectively in the past. With the sophisticated genomics tools now available, the time is right for Tango to take on this challenge and focus on patients without effective treatment options."

Tango is focused on three areas of drug development, each in well-defined patient populations currently lacking effective treatment options, and each with hallmarks of cancer that have not been targeted yet. These include:

Loss of tumor suppressor gene function: A universal feature of cancer is the inactivation of genes which normally protect against tumor development. Tango is working to turn tumor suppressor gene loss from cancer’s strength into a weakness by identifying associated targetable vulnerabilities, an effect known as synthetic lethality. Drugs against synthetic lethal targets have the potential added benefit of effectiveness against cancer cells without damaging normal cells.

Multiple oncogenic drivers: To address the multiple genetic changes that give rise to cancer, Tango is working to identify novel targets for rational combinations that will offer more robust treatment effects than is possible with single-agent approaches. Decades of cancer research have shown that with the right drug combinations in the right patients, cancers can be curable.

Immune evasion: Complementing current, immune-cell-directed cancer therapies, Tango is working to identify and target the genetic alterations in cancer cells responsible for helping them avoid immune destruction. Drugs against these targets could substantially increase the benefit of current immuno-oncology drugs without increasing immune damage to normal tissues.

Tango product engine: Putting patient selection first to accelerate discovery

What fuels each of Tango’s programs is an increasingly sophisticated ability to utilize synthetic lethality, the interaction between two genes that causes cell death when both are inactivated. In cancer cells, one of these genes is inactivated by mutation; the other will be inactivated by a drug. This approach leaves normal cells largely unaffected, with the potential to greatly enhance anti-tumor efficacy and reduce associated toxicity.

The first FDA-approved example of synthetic lethality in cancer treatment is the use of PARP inhibitors for BRCA-mutant ovarian cancer. Alan Ashworth, Ph.D., a scientific Founder of Tango, discovered that combining a BRCA1/2 mutation and PARP inhibition creates synthetic lethality. Recent data show that continuing treatment with a PARP inhibitor after chemotherapy in patients with BRCA-mutant ovarian cancer extends progression-free survival for more than two years, compared to placebo. The Tango product engine is designed to systematically discover other such context-specific vulnerabilities, uncovering weaknesses created by genetic alterations in cancer and targeting them for therapeutic benefit.

In executing this strategy, Tango will upend the traditional paradigm by doing target discovery in cancer subgroups with a pre-defined patient selection approach. The company will use data from DNA sequencing of patient-derived tumor samples to define genetic contexts, and deploy CRISPR-based target discovery techniques in those cancer subtypes to identify novel drug targets.

Tango’s success will be driven by its depth of understanding of the genetic subtypes of cancer, and corresponding insights into novel drug targets and combinations uniquely relevant to each subtype. By shaping discovery efforts in this way, Tango has the potential to reach the clinic quickly, and with a clear plan for identifying the patients most likely to benefit from each new treatment, an approach that will increase both speed and probability of success in translating novel target discoveries into transformational new medicines for patients.

World-leading team spanning diverse disciplines

The Tango management team includes recognized leaders in target discovery, cancer biology, functional genomics, translational medicine and company building. Company leaders include Barbara Weber, M.D., interim Chief Executive Officer; Cary Pfeffer, M.D., interim Chief Business Officer; Daniella Beckman, C.P.A., Chief Financial Officer; Alan Huang, Ph.D., Senior Vice President, Head of Biology; John Maxwell, Ph.D., Vice President, Head of Chemistry; and Janid Ali, Ph.D., Vice President, Head of Biochemistry.

The Tango founders are a group of internationally recognized scientists and clinicians who have shaped the current state of knowledge and practice in cancer biology and genetics, translational medicine and CRISPR technology:

Alan Ashworth, Ph.D., FRS, President of the University of California San Francisco Helen Diller Family Comprehensive Cancer Center

José Baselga, M.D., Ph.D., Physician-in-Chief at Memorial Sloan Kettering Cancer Center

Levi Garraway, M.D., Ph.D., Senior Vice President of Global Oncology at Eli Lilly and Company

William Kaelin, M.D., Professor in the Department of Medicine at the Dana-Farber Cancer Institute, Harvard Medical School and a Howard Hughes Medical Institute Investigator

Timothy K. Lu, M.D., Ph.D., Associate Professor of Biological Engineering, Electrical Engineering and Computer Science at the Massachusetts Institute of Technology

Antoni Ribas, M.D., Ph.D., Professor of Medicine, Surgery, and Molecular and Medical Pharmacology at the University of California Los Angeles

About Tango Therapeutics

Tango Therapeutics is a biotechnology company developing novel medicines for patients by discovering and drugging context-dependent vulnerabilities in cancers. Tango was launched in 2017 by Third Rock Ventures and is headquartered in Cambridge, Mass.