Oxford BioMedica notes acceptance by FDA of a Biologics License Application (BLA) filing for CTL019

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."

Redx Pharma awarded US$1 million grant by CARB-X

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.

Third Rock Ventures Launches Tango Therapeutics with $55 Million Series A Investment

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.

U.S. FDA Division of Hematology Products Lifts Partial Clinical Hold on Karyopharm’s Selinexor Clinical Trials

On March 30, 2017 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) Division of Hematology Products has lifted the partial clinical hold placed on the clinical trials for selinexor (KPT-330), enabling patient enrollment and dosing of new patients in the Company’s clinical trials of selinexor in hematological malignancies (Press release, Karyopharm, MAR 30, 2017, View Source [SID1234518346]). The partial clinical hold was not the result of any patient death or any change in the safety profile of selinexor. Enrollment may now resume in all selinexor studies in hematologic malignancies, including the STORM study in refractory multiple myeloma, the SADAL study in relapsed/refractory diffuse large B-cell lymphoma (DLBCL), and the STOMP study of selinexor and backbone therapies in multiple myeloma. In addition, Investigator Sponsored Trials in hematologic malignancies with selinexor may resume accruing patients.

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Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm, stated, "The Karyopharm team worked diligently to update and submit the required documents to the FDA, which allowed the hematology divison to expeditiously remove the partial clinical hold. We anticipate that the solid tumor divisions will follow suit shortly. Patient enrollment is again underway in our hematologic oncology studies. Our previously disclosed enrollment rates and timelines for both ongoing and planned trials are not expected to be materially impacted."

Agenus Restructures Business to Sharpen Focus on Clinical Development of Cancer Therapies

On March 30, 2017 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with a clinical stage pipeline of immune checkpoint antibodies and cancer vaccines, reported that it is reorganizing its business and operations to sharpen its focus on clinical development of its two checkpoint inhibitor antibodies and vaccine program (Filing, Agenus, MAR 30, 2017, View Source [SID1234518345]). Agenus plans to close its Basel site and consolidate key functions to its Cambridge, UK and Lexington, MA facilities, and phase out approximately 50 positions across the organization. Additionally, Robert Stein, M.D., Ph.D., President of R&D, will retire to become a senior R&D advisor exclusive to Agenus.

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Agenus’ goals for this realignment are to:

Accelerate development and commercialization of its product portfolio to drive shareholder value
Further extend the Company’s cash runway beyond the impact from the recently amended Incyte partnership, which strengthened the balance sheet by $80 million and reduced development expenses
Consolidate operations to improve R&D efficiencies
Ensure commercial readiness and manufacturing
Prioritized programs include combination therapies targeting CTLA-4 and PD-1. In addition, Agenus will continue to drive its innovative immuno-oncology portfolio towards clinical development with two preclinical antibodies targeting 4-1BB and TIGIT, as well as AutoSynVax, a clinical-stage neoantigen cancer vaccine. The Company is exploring combination studies with AutoSynVax and Agenus’ checkpoint antibodies. Substantial focus will also be placed on the Company’s manufacturing operations in Berkeley, CA to ensure GMP readiness. This is particularly pertinent as Agenus progresses its clinical registration trials with an intent to commercialize within the next four years.

As part of the restructuring, approximately 50 positions are planned to be phased out within the next six months. In addition, the Company will transition or consolidate certain key management positions, with the objective of streamlining leadership and reducing costs.

"These changes to our organizational structure make us a leaner and more focused organization, which is critically important for our next phase of advancement towards commercial readiness. We will also maintain a focused R&D effort to rapidly generate and develop best of breed novel immuno-oncology candidates. It is important to indicate that as an agile and efficient company we aim to rapidly deliver effective treatments at affordable prices," commented Dr. Armen.

Having built an extraordinary R&D capability for Agenus and spearheaded the advancement of five programs from discovery to clinical stage in the last three years, Dr. Robert Stein will be retiring from his current role as President of R&D and will become a senior R&D advisor to Agenus. The current R&D leadership, which has been assembled under his tutelage, will continue to have access to Dr. Stein for strategic R&D guidance.

"We are grateful to Dr. Stein for his outstanding leadership and contributions in defining and building our research engine," said Dr. Armen. "I look forward to continuing to work with him very closely in the future. I would also like to acknowledge the contributions of our other colleagues who will be departing the Agenus organization."