On January 8, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported key 2018 priorities in conjunction with its presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 8, 2018, View Source [SID1234522985]). The presentation will outline how Agios’ clinical and research programs have the potential to provide meaningful benefit to a large number of patients. The company will webcast its presentation today at 9:30 a.m. PT (12:30 p.m. ET) at investor.agios.com.

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"We presented a significant amount of clinical data across our portfolio in 2017, including data supporting the approval of our first internally discovered medicine, which proves that our research and development engine has the ability to deliver important precision medicines from a discovery in the lab to patients as an approved drug," said David Schenkein, M.D., chief executive officer at Agios. "We are very pleased with the early launch performance of IDHIFA and expect to repeat this success with ivosidenib upon FDA approval in 2018, while continuing our label expansion opportunities for frontline AML and solid tumors and bringing our next drug candidate targeting genetically defined cancers, AG-270, into the clinic. In rare genetic diseases, AG-348 has demonstrated proof of concept in pyruvate kinase deficiency and in addition to beginning pivotal trials in this disease, we are exploring the utility of PK activation in other anemias, starting with a Phase 2 study in thalassemia."

The company plans to achieve the following key milestones in 2018:

Cancer:

Potential FDA approval and commercialization of ivosidenib for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the United States in the second half of 2018.

Plan to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ivosidenib for IDH1m R/R AML in the fourth quarter of 2018.

Support with Celgene an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib and standard induction (7+3) and consolidation chemotherapy with a primary endpoint of event free survival (EFS) in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.

Initiate a perioperative ‘window’ trial with ivosidenib and AG-881 in low-grade glioma to further investigate their effects on brain tumor tissue in the first half of 2018.

Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:

Initiate two global pivotal trials for AG-348 in PK deficiency:

ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first quarter of 2018.

ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions is expected to initiate in the second quarter of 2018.

Initiate a global registry for adult and pediatric patients with PK deficiency (PEAK) in the first quarter of 2018 to increase understanding of the long-term disease burden of this chronic anemia.

Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our latest development candidate, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
The company plans to host an analyst day in the first half of 2018 to review Agios’ commercial readiness and broad clinical development programs and highlight the depth of the research portfolio across oncology, rare genetic disease and metabolic immuno-oncology.

The company also highlighted key 2017 achievements:

Collaborated with Celgene to achieve the U.S. Food and Drug Administration (FDA) full approval and subsequent launch of IDHIFA (enasidenib) for the treatment of adult patients with R/R AML with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved diagnostic test.

Submitted a new drug application (NDA) to the FDA for ivosidenib for the treatment of patients with R/R AML with an IDH1 mutation.

Initiated a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib and VIDAZA in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy.

Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency.

Achieved FDA clearance of an IND application for AG-270, a MAT2A inhibitor, targeting MTAP-deleted tumors.
2017 Year-End Cash and Updated Guidance

Agios ended 2017 with approximately $568 million of cash, cash equivalents and marketable securities. Based on its expanded clinical and research programs announced today, the company now expects that its existing cash, cash equivalents and marketable securities as of December 31, 2017, together with anticipated interest income, anticipated expense reimbursements, and royalty payments under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through the end of third quarter of 2019.

Presentation at 36th Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 8, 2018 at 9:30 a.m. PT (12:30 p.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On January 8, 2018 Novavax, Inc. presented Investor Presentation (Presentation, Novavax, JAN 8, 2018, View Source [SID1234522978]).

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On January 8, 2018 Seattle Genetics, Inc. (NASDAQ: SGEN) reported the progress of its pipeline of antibody-drug conjugates (ADCs) at the 36th Annual J.P. Morgan Healthcare Conference (Press release, Seattle Genetics, JAN 8, 2018, View Source;p=RssLanding&cat=news&id=2325358 [SID1234523020]). Through both internal efforts and that of its collaborators, the company’s ADC technology is being employed in more than 20 programs in clinical trials, including multiple late-state development programs across hematologic malignancies and solid tumors.

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"ADCs continue to advance as an important therapeutic modality, both as single agents and as part of various combination regimens, across hematologic malignancies and solid tumors," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "We are the industry leader in ADC technology driven by our scientific expertise in monoclonal antibodies, drug payloads and stable linker technologies. Our leadership is further illustrated by the continued clinical and commercial expansion of ADCETRIS (brentuximab vedotin), progress with our late-stage programs enfortumab vedotin and tisotumab vedotin, and the breadth of our pipeline of other ADCs and empowered antibodies. In addition, our collaborators are making significant advances with several programs using our technology. ADCs are an integral part of an evolving cancer treatment paradigm, and we are committed to bringing important new treatments to patients in need."

ADCETRIS, which pioneered a new class of ADCs, is commercially available in 70 countries worldwide and generated more than $600 million in global sales in 2017. On January 2, 2018, the company announced that the FDA accepted for filing a supplemental Biologics License Application (BLA) for ADCETRIS in combination with chemotherapy for the frontline treatment of patients with advanced classical Hodgkin lymphoma. The FDA granted Priority Review for the application, and the Prescription Drug User Fee Act (PDUFA) target action date is May 1, 2018. The submission of the supplemental BLA is based on positive results from a phase 3 clinical trial called ECHELON-1. In October 2017, the FDA granted Breakthrough Therapy Designation (BTD) for ADCETRIS in frontline advanced Hodgkin lymphoma based on the ECHELON-1 study results.

In addition to advancing ADCETRIS, Seattle Genetics and its collaborator Astellas have initiated a pivotal phase 2 clinical trial of enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with checkpoint inhibitor (CPI) therapy. The study is designed to support potential registration under the FDA’s accelerated approval regulations. In addition, Seattle Genetics, in collaboration with its development partner Genmab, plans to initiate a phase 2 clinical trial of tisotumab vedotin for patients with recurrent and/or metastatic cervical cancer. This study is intended to support potential registration under the FDA’s accelerated approval regulations.

Seattle Genetics’ ADC technologies are also empowering several collaborator programs in late-stage clinical trials. These include:

GSK2857916, an ADC being developed by GlaxoSmithKline (GSK) for multiple myeloma. GSK recently reported encouraging data from the program at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2017;
Polatuzumab vedotin, an ADC being developed by Genentech/Roche. Positive results were presented at ASH (Free ASH Whitepaper) from a phase 2 trial in advanced-stage diffuse large B-cell lymphoma. A phase 3 trial is underway; and,
Depatuxizumab mafodotin, an ADC for glioblastoma in development by AbbVie. Encouraging data have been reported from this ADC, which is currently in a phase 3 clinical trial.
Polatuzumab vedotin and GSK2857916 have both received BTD from the FDA and PRIority MEDicines (PRIME) designations from the European Medicines Agency. These designations signify the importance of therapies such as these in addressing significant unmet medical need.

"Through our robust internal development efforts and our strong licensing and co-development agreements, we are extending the potential of ADCs globally. We look forward to future results of studies that include Seattle Genetics’ novel technologies both as monotherapies, as well as in combination with checkpoint inhibitors and other agents," said Dr. Siegall.

In 2018, Seattle Genetics anticipates several milestones, including:

Working with FDA towards the May 1 PDUFA action date for ADCETRIS in combination with chemotherapy for frontline treatment of patients with advanced classical Hodgkin lymphoma;
Reporting ECHELON-2 data of ADCETRIS in combination therapy in frontline CD30-expressing mature T-cell lymphoma (MTCL);
Continuing enrollment of the enfortumab vedotin (EV) pivotal trial in locally advanced or metastatic urothelial cancer patients previously treated with a checkpoint inhibitor;
Continuing enrollment of the EV phase 1b trial in combination with checkpoint inhibitors, for patients with locally advanced or metastatic urothelial cancer;
Initiating a phase 2 trial of tisotumab vedotin (TV) in recurrent and/or metastatic cervical cancer to potentially support registration; and,
Initiating a phase 2 trial of TV as part of a combination regimen for first-line cervical cancer and a phase 2 trial of TV in other solid tumor types;
Initiating multiple trials evaluating ladiratuzumab vedotin in combination with checkpoint inhibitors in metastatic triple negative breast cancer, as well as evaluation as a neoadjuvant therapy for early breast cancer as part of the I-SPY consortium.
ADCETRIS is currently not approved for the frontline treatment of MTCL or Hodgkin lymphoma.

On January 8, 2018 Evotec AG (Frankfurt Stock Exchange: EVT, TecDAX, ISIN: DE0005664809) and APEIRON Biologics AG, a company focused on cancer immunotherapy, reported that the companies received the first milestone payment from Sanofi under a 3-party alliance signed in August 2015 (Press release, Evotec, JAN 8, 2018, View Source [SID1234522936]). The milestone payment of EUR 3 m will be split equally between the two biotech companies. The success payment was triggered when the partners successfully advanced an undisclosed, novel immuno-oncology small molecule into late-stage pre-clinical development. Under the alliance, the three companies work together to identify small molecule leads and targets for next-generation therapies in immuno-oncology, which may complement the pre-clinical and clinical profiles of leading checkpoint inhibitors.

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Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "The Evotec and APEIRON teams are proud to have achieved our first milestone with Sanofi to discover and develop novel immuno-oncology small molecules therapies. It is a first-in-class approach with tremendous potential in combination with marketed checkpoint inhibitors but also as standalone therapy. The field of immuno-oncology will continue to evolve and at Evotec we will continue to invest in this area."

Dr Hans Loibner, Chief Executive Officer of APEIRON Biologics, said: "We are delighted by the progress made in our collaboration with Evotec and Sanofi. Based on our scientific findings and the mode of action, we jointly agreed to accelerate the research and pre-clinical development of this very promising molecule. The successful achievement of this milestone further demonstrates our abilities to drive innovation in the field of immuno-oncology."

ABOUT THE EVOTEC-APEIRON-SANOFI-ALLIANCE
The strategic collaboration was set up in 2015 to support the long-term pipeline building for Evotec, APEIRON Biologics and Sanofi and has a potential value of over EUR 200 m in milestone payments and significant royalties. The collaboration includes major research and development efforts to advance a first-in-class orally available small molecule approach based on a novel target to treat solid and hematopoietic cancers by enhancing the anti-tumor activity of human immune cells. All three companies are making significant contributions to this collaboration in terms of scientific expertise, technological platforms and resources. The collaboration will further enhance and complement Sanofi’s extensive oncology portfolio and enables Evotec to address the drug discovery area of immuno-oncology. Furthermore, the agreement will substantially support APEIRON’s strategy of focusing on novel and innovative checkpoint inhibiting approaches.

On January 9, 2018 Amgen Astellas BioPharma K.K. (Headquarters Tokyo; President and Representative Director Steve Sugino "Amgen Astellas BioPharma") and Astellas Pharma Inc. (Headquarters Tokyo; President and CEO Yoshihiko Hatanaka "Astellas") reported that an application was submitted in Japan for the marketing authorization for bispecific CD19-directed CD3 T cell engager (BiTE) antibody construct blinatumomab (Genetically Recombination) (generic name, development code: AMG 103, "blinatumomab") to treat relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) (Press release, Astellas, JAN 8, 2018, View Source [SID1234522970]). In Japan, blinatumomab is jointly developed by Amgen Astellas BioPharma and Astellas.

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ALL affects approximately 5,000 patients in Japan1, out of which an estimated 670 per year have relapsed or refractory ALL2,3,4. There are several limitations to current treatment options, including their limited efficacy in adult and pediatric patients with relapsed or refractory ALL and dependency on a limited number of drugs with similar mechanisms of action. Improved outcomes for relapsed or refractory ALL patients calls for the development of drugs such as blinatumomab which demonstrate efficacy as a monotherapy and have mechanisms of action dissimilar to cytotoxic agents.

The submission of application for marketing approval in Japan was based on the results from multiple global clinical studies including the Phase 3 randomized study (TOWER study), and the Japanese Phase 1b/2 study. In the TOWER study, blinatumomab was shown to extend overall survival compared to standard-of-care (SOC) chemotherapy in adult patients with relapsed or refractory ALL. Blinatumomab is considered to have the potential to address the serious unmet medical needs of ALL patients.

Blinatumomab received Orphan Drug designation from the Ministry of Health, Labour and Welfare effective September 29, 2017.

About Blinatumomab

Blinatumomab (genetically recombinant antibody) is a bispecific CD19-directed CD3 T cell engager (BiTE) antibody construct that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells. Blinatumomab was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration, and is now approved in the U.S. for the treatment of relapsed or refractory B-cell precursor ALL in adult and pediatric patients. In November 2015, the EU granted conditional marketing authorization for blinatumomab for the treatment of adults with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. AmgenInc. is seeking to gain approval for blinatumomab in countries around the world.

TOWER Study

The TOWER study was a Phase 3 randomized study investigating the efficacy of blinatumomab versus SOC chemotherapy in 405 adult patients with Ph- relapsed or refractory B-cell precursor ALL. The study enrolled a difficult-to-treat patient population, which included patients from several stages of relapse. In the blinatumomab arm, this included 35% of patients that had relapsed post-allogenic hematopoietic stem cell transplant (alloHSCT), and excluded those with late first relapse (≥ 12 months after initial remission). Patients were randomized in a 2:1 ratio to receive blinatumomab (n = 271) or one treatment with investigator’s choice out of 4 types of SOC chemotherapy regimens (n = 134). The determination of efficacy was based on overall survival. Per the recommendation of the data monitoring committee, the study was ended early for evidence of superior OS in the blinatumomab arm vs SOC chemotherapy from the pre-specified interim analysis.

These results are published in the New England Journal of Medicine.5

About BiTE Technology

Bispecific T cell engager (BiTE) antibody constructs are being investigated for fighting cancer by helping the body’s immune system to detect and target malignant cells. The modified antibodies are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE antibody constructs help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE antibody constructs are currently being investigated for their potential to treat a wide variety of cancers. For more information, visit www.biteantibodies.com.

About Amgen’s Commitment to Oncology

Amgen Oncology is committed to helping patients take on some of the toughest cancers, such as those that have been resistant to drugs, those that progress rapidly through the body and those where limited treatment options exist. Amgen’s supportive care treatments help patients combat certain side effects of strong chemotherapy, and our targeted medicines and immunotherapies focus on more than a dozen different malignancies, ranging from blood cancers to solid tumors. With decades of experience providing therapies for cancer patients, Amgen continues to grow its portfolio of innovative and biosimilar oncology medicines.