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Agios Outlines Key Clinical Development & Research Strategies

On October 16, 2015 Agios Pharmaceuticals (NASDAQ:AGIO) reported its clinical development strategy for the company’s lead cancer metabolism and rare genetic metabolic disorders programs, along with insights into emerging research at its R&D Day today (Press release, Agios Pharmaceuticals, OCT 16, 2015, View Source [SID:1234507725]).

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"With our lead IDH programs progressing through Phase 1 expansion cohorts and the initiation of the Phase 3 AG-221 study with our collaboration partner Celgene, we are moving closer to our goal of providing people with advanced AML with transformational new medicines as quickly as possible," said David Schenkein, M.D., chief executive officer at Agios. "We will share our long-term vision for these programs at today’s R&D Day, as we hope to one day provide benefit to every patient diagnosed with an IDH mutant-positive cancer."

"At our core, Agios is a research-driven organization, and I’m proud that our scientists have discovered AG-519, a novel PK activator, which represents our fifth new investigational medicine in just seven years. We continue to conduct research that we believe will enable us to make important advances for patients," Dr. Schenkein continued.

IDH Program Updates

In clinical studies to date, AG-221 and AG-120, which target mutated IDH2 and IDH1, respectively, have demonstrated positive clinical single-agent activity with durable complete and partial responses and manageable safety profiles in patients with AML. Together with our collaboration partner Celgene, Agios remains committed to bringing AG-221 and AG-120 to patients as quickly and efficiently as possible by leveraging a clinical development strategy that maximizes both speed and breadth.

Agios reported clinical development updates for AG-221 and AG-120 in AML, including:

Initiation of the AG-221 Phase 3 Study: The IDHENTIFY study of AG-221 is a Phase 3, international, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of AG-221 versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy. Additional details can be found below and on www.clinicaltrials.gov. This study is being conducted by Celgene.

Novel Design of the AG-221 and AG-120 Frontline Trials in AML:
For Newly Diagnosed AML Patients Eligible for Intensive Chemotherapy: A Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy is planned for initiation by the end of 2015.

For Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy: A Phase 1/2 combination study of either AG-221 or AG-120 with VIDAZA (azacitidine) is planned for initiation in the first quarter of 2016. This study has a Phase 1 component to determine the safety of the combinations, followed by a Phase 2 randomized component evaluating the safety and clinical activity of each investigational combination versus single-agent VIDAZA using a primary endpoint of overall response rate.
ASH Data Presentations: New data from the ongoing Phase 1 dose-escalation and expansion studies of AG-221 and AG-120 in advanced hematologic malignancies have been accepted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exhibition taking place December 5-8, 2015 in Orlando.

EORTC-NCI-AACR Data Presentation: The first data from the ongoing Phase 1 trial of AG-120 in advanced IDH1-mutant positive solid tumors have been accepted for oral presentation at EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) taking place November 5-9, 2015 in Boston.
Pyruvate Kinase (PK) Deficiency Program Updates

Agios is pioneering the development of its small molecule enzyme activators in PK deficiency, a rare genetic metabolic disorder with no disease-altering therapies.

AG-348: AG-348, a first-in-class orally available, potent, selective small molecule activator of pyruvate kinase-R (PKR), is on track and enrolling in DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency.

AG-519: Agios announced today the development of its second PKR activator, AG-519. This program provides clinical development optionality for our PK activator portfolio and potentially opportunities in other hemolytic anemias where PK activation may be therapeutic. The development plan for AG-519 includes a placebo-controlled Phase 1 study in healthy volunteers, which is planned for the first quarter of 2016. This study will be an integrated single ascending dose (SAD) and multiple ascending dose (MAD) trial.
Research Program Updates

Agios has advanced and led the emerging field of cancer metabolism with its novel IDH1 and IDH2 programs, demonstrating significant potential benefit for AML patients whose cancers carry these mutations. Agios’ work in IDH exemplifies the company’s strategy of targeting metabolic vulnerabilities. The company continues to discover novel metabolic targets that meet a high bar for future development. Today at its R&D day, Agios will describe:

Its precision medicine strategy to discover novel cancer metabolism targets.
Novel research approach to rare genetic diseases using allosteric modulation to correct the underlying dysfunction in metabolic pathways.

The potential for PKR activators to provide clinical benefit in other indications, such as beta-thalassemia.

Webcast

A live webcast of the company’s R&D Day will begin today at 9:00 a.m. ET and can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for 30 days following the presentation.

About the IDHENTIFY Phase 3 Study of AG-221

The Phase 3, international, multicenter, open-label, randomized clinical trial is designed to compare the efficacy and safety of AG-221 versus conventional care regiments in subjects 60 years or older with IDH2 mutant-positive AML refractory to or relapsed after second- or third-line therapy. Patients will be randomly assigned to receive either AG-221, 100 mg orally once a day for 28 days, or one of the conventional care regiments. The conventional treatment options include best supportive care only, azacitidine, low-dose cytarabine or intermediate-dose cytarabine. The primary endpoint of the trial is overall survival. The study is expected to enroll approximately 280 patients and is being conducted by Celgene. Please refer to www.clinicaltrials.gov for additional clinical trial details.

About Acute Myelogenous Leukemia (AML)

AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to the American Cancer Society, the median age of onset is 66. Less than 10 percent of U.S. AML patients are eligible for bone marrow transplant and the vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH1 and IDH2 mutations are present in about 15 to 23 percent of AML cases.

About AG-348 and PK Deficiency

PKD is a rare inherited disease resulting from mutations in the PKR enzyme that result in hemolytic anemia, which is the accelerated destruction of red blood cells. The mutations in the PKR enzymes cause a deficit in cellular energy within the red blood cell, as evidenced by a decline in the energy metabolite ATP and a build-up of the metabolite 2,3-DPG. Agios scientists have previously reported that AG-348 is a potent activator of the wild-type and mutated PKR enzymes, resulting in restoration of ATP levels and a decrease in 2,3-DPG levels in patient blood ex vivo. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment- and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PKD. AG-348, a first-in-class orally available, potent, selective small molecule activator of PKR, was discovered by Agios scientists, and the company retains worldwide development and commercialization rights.

10-Q – Quarterly report [Sections 13 or 15(d)]

Burzynski Research Institute has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

CU biotech start-up gets $1.5 million contract to develop head and neck cancer treatment

On October 15, 2015 SuviCa Inc. of Boulder, a University of Colorado Boulder biotech start-up company reported that it has been awarded roughly $1.5 million in federal funding to develop novel treatments for head and neck cancer (Press release, suvica, OCT 15, 2015, View Source [SID1234517395]).

The award to SuviCa is a Phase II Small Business Innovation Research (SBIR) contract from the National Cancer Institute, part of National Institutes of Health and the Department of Health and Human Services. SuviCa was founded in 2010 based on a novel drug-screening technology developed by CU-Boulder Professor Tin Tin Su, SuviCa co-founder and chief scientific officer.

The funding will allow SuviCa to continue development of the drug SVC112, a compound shown to enhance the anti-tumor effects of radiation in animal models of human head and neck cancer, said SuviCa CEO Judy Hemberger. While radiation is a regular therapy used to treat such cancers, it also is associated with potentially devastating side effects and tumor recurrence, she said.

New treatments under development by Su and her colleagues involve small molecules that work together with currently used cancer therapeutics to destroy cancerous tumors, said Hemberger. The lead candidate for treatment currently is a "radiation enhancer" molecule that inhibits a specific process that cancer cells rely on to recover from radiation damage.

The project is being directed by Su and Bert Pronk, SuviCa’s vice president of preclinical development. The team also includes clinicians and scientists at CU-Boulder, the University of Colorado Cancer Center and Colorado State University.

CU’s Technology Transfer Office (TTO) exclusively licensed SVC112, along with related technology, to SuviCa in 2011 and 2012.

"We have very talented researchers at each of these institutions that together are tackling the challenges involved with successfully developing novel treatment options for head and neck cancer," said Su, a professor in CU-Boulder’s Department of Molecular, Cellular and Developmental Biology.

"We are excited to receive continued support from the National Cancer Institute," said Hemberger. "Our goal as a company is to generate innovative ways to improve how we treat cancer patients, and we are pleased that the scientific and business communities recognize that we are working with an important technology."

The small molecules developed by SuviCa target ribosomes, which synthesize proteins inside of cells, said Hemberger. Ribosomes are becoming increasingly recognized as a potential therapeutic target in cancer treatment.

TTO pursues, protects, packages and licenses intellectual property generated from research at the university. Tech Transfer provides assistance to faculty, staff and students, as well as to businesses looking to license or invest in CU technology.

Open Monoclonal Technology and ARMO BioSciences Announce Anti-PD1 Antibody and OmniAbTM Platform License Agreement

On October 15, 2015 ARMO BioSciences, Inc. (ARMO), a leading developer of immuno-oncology therapeutics and Open Monoclonal Technology, Inc. (OMT ) reported a licensing agreement providing ARMO with exclusive rights to OMT’s Programmed Cell Death Protein 1 (PD-1) assets as well as unlimited access to OMT’s proprietary OmniRat, OmniMouse and OmniFlic human antibody generation platforms (Press release, ARMO BioSciences, 15 15, 2015, View Source [SID:1234513063]).

"This is an important transaction for ARMO as we transition to our next stage of corporate growth. In particular, securing a PD-1 targeting agent provides us with a complete, efficient and cost- effective combination therapy solution for our next generation cytokine therapies," commented Peter Van Vlasselaer, Ph.D., President and Chief Executive Officer of ARMO BioSciences.

The addition of an anti-PD-1 program to ARMO’s pipeline of cytokine immunotherapies reinforces the company’s commitment to the development of safe and effective treatments for cancer. ARMO’s clinical stage PEGylated form of recombinant human IL-10, known as AM0010, and anti- PD-1 antibodies work through complementary but distinct mechanisms on CD8+ T cells so the combination maximizes the activation, proliferation and survival of intratumoral, tumor-reactive, cytotoxic CD8+ T cells, which are considered to be central to clinical response. In ongoing clinical studies, AM0010 has already shown activity as a monotherapy and in combination with anti-PD-1 agents in immune-sensitive tumors such as melanoma, RCC, NSCLC and others not previously thought to be sensitive to immunotherapy such as colorectal and pancreatic cancers. Early results from these trials are suggestive of AM0010’s role in augmenting activated tumor infiltrating T cells and PD-1 expression, thereby bestowing anti-PD-1 sensitivity on tumors, including those that had been refractory to anti-PD-1 treatment.

Dr. Roland Buelow, OMT founder and CEO, said, "ARMO’s focus on discovery and development of novel therapies based on proprietary insights of dysregulated immune responses in cancer and atherosclerotic, fibrotic and inflammatory diseases are particularly amenable to human therapeutic antibodies. We are pleased to enable ARMO to access both OMT’s anti-PD1 antibodies and mono- and bi-specific antibody platforms to further the company’s therapeutic objectives."

Sunesis Pharmaceuticals Announces Oral Presentation of VALOR Analysis at the 77th Annual Meeting of the Japanese Society of Hematology

On October 15, 2015 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported that data from the company’s VALOR trial evaluating vosaroxin in older patients with acute myeloid leukemia (AML) will be presented at the AML Clinical Trial Oral Session of the 77th Annual Meeting of the Japanese Society of Hematology (JSH), taking place in Kanazawa, Japan (Press release, Sunesis, OCT 15, 2015, View Source;p=RssLanding&cat=news&id=2097654 [SID:1234507723]). The results are being presented Friday, October 16th at 8:30 a.m. Japanese Standard Time by Robert Stuart, M.D., Professor of Medicine, Division of Hematology/Oncology, Department of Medicine, Medical University of South Carolina.
The presentation (Abstract OS-1-82), titled "Patients Age ≥60 Yrs With First Relapsed/Refractory AML Treated With Vosaroxin/Cytarabine vs Placebo/Cytarabine," will be the first presentation in the session, which will take place in Session Room 15 (Emerald A) in Hotel Kanazawa 4F. Detailed results of the VALOR trial were presented in the "Late Breaking Abstracts" session of the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2014. The data presented today at the JSH Annual Meeting were first presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June 2015.

"Globally, patients with relapsed or refractory AML are highly underserved, particularly older patients whose prognoses are far poorer," said Shuichi Miyawaki, MD, PhD., Division of Hematology, Tokyo Metropolitan Ohtsuka Hospital, and former vice-president of the Japan Adult Leukemia Study Group. "As in the U.S. and Europe, treatment standards in Japan have not changed significantly in the past several decades. The data from VALOR in patients age 60 years and older show a compelling survival benefit, durable responses and tolerability profile that strongly support the potential for the vosaroxin/cytarabine combination as an important new treatment option for this highly underserved population."

Sunesis recently announced that the company intends to submit a Marketing Authorization Application (MAA) for vosaroxin as a treatment for patients age 60 years and older with relapsed/refractory AML with the European Medicine Agency (EMA) by the end of 2015.

About QINPREZO (vosaroxin)
QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed or refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.
The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.

About AML
AML is a rapidly progressing cancer of the blood characterized by the uncontrolled proliferation of immature blast cells in the bone marrow. The American Cancer Society estimates that there will be approximately 20,830 new cases of AML and approximately 10,460 deaths from AML in the U.S. in 2015. Additionally, it is estimated that the prevalence of AML across major global markets (U.S., France, Germany, Italy, Spain, United Kingdom and Japan) is over 75,000. AML is generally a disease of older adults, and the median age of a patient diagnosed with AML is about 67 years. AML patients with relapsed or refractory disease and newly diagnosed AML patients over 60 years of age with poor prognostic risk factors typically die within one year, resulting in an acute need for new treatment options for these patients.