The Leukemia & Lymphoma Society® Releases Results of its Precision Medicine Trial in Deadly Leukemia at American Society of Hematology Annual Meeting

On November 30, 2018 The Leukemia & Lymphoma Society (LLS) reported it continues to deliver on its promise to "Beat AML," the deadliest of the blood cancers, through its groundbreaking Beat AML Master Clinical Trial, a precision medicine approach to treating these cancer patients (Press release, The Leukemia & Lymphoma Society, NOV 30, 2018, View Source;lymphoma-society-releases-results-of-its-precision-medicine-trial-in-deadly-leukemia-at-american-society-of-hematology-annual-meeting-300758232.html [SID1234531760]).

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The first results of this trial, to be announced Sunday and Monday at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, validate the feasibility of using genomic sequencing to identify the patients’ specific type of acute myeloid leukemia (AML) and give them a targeted treatment within seven days, a first-ever approach to this disease, which, until recently, had not seen treatment advances in 40 years.

As the world’s largest nonprofit dedicated to fighting blood cancers, LLS is uniquely qualified to drive the Beat AML Master Clinical Trial, an unprecedented collaboration that aims to change the paradigm of treatment through a precision medicine approach. LLS has united prominent scientists at top cancer centers, multiple pharmaceutical companies, several technology companies and the U.S. Food and Drug Administration (FDA) to bring new and better treatments to AML patients in urgent need. LLS is the first nonprofit health organization to sponsor a cancer clinical trial.

Five Beat AML Master Clinical Trial abstracts to be presented at 60th Annual ASH (Free ASH Whitepaper) Meeting:

"Initial Report of the Beat AML Umbrella Study for Previously Untreated AML: Evidence of Feasibility and Early Success in Molecularly Driven Phase 1 and 2 Studies [Abstract #559], at press briefing Sunday, December 2 at 8 a.m. PT, and in an oral scientific session on Monday, December 3 at 7 a.m.

Additional progress on Beat AML will be presented in abstracts 287, 1489, 2773, and 4053

According to Brian Druker, MD, director of OHSU Knight Cancer Institute, and a co-lead investigator for LLS’s Beat AML Master Clinical Trial, "For four decades, AML patients have been treated with a one-size-fits all approach, with most receiving a standard chemotherapy combination almost immediately upon diagnosis. But many older adults cannot tolerate the toxicity. With LLS’s vision and support, we’ve been able to bring a cutting edge medical approach to developing better treatments faster and helping more patients. With the Beat AML trial we’re doing a better job of matching patients to therapies."

More than 20,000 Americans are diagnosed with AML and 10,000 die from the cancer annually. The five-year-survival rate for older adults remains dismal at less than 20 percent.

The study, two years underway, has already shown that patients diagnosed with AML, a complex disease with multiple subtypes, benefit from having their specific genetic subtype identified quickly before receiving traditional treatment, so they can receive a targeted therapy that matches their individual cancer-driving genetic mutations. The trial has now screened more than 400 patients at 13 clinical centers across the U.S., with patients entering the trial receiving one of seven investigational therapies.

The impact of the Beat AML initiative and progress in AML in general was addressed last night at an LLS roundtable event, Innovation in Research: Blood Cancers and Beyond, moderated by NBC News Medical Correspondent John Torres, MD, and veteran healthcare journalist Ron Winslow. Advancements in immunotherapy, genomics and personalized medicine and how they are transforming cancer treatment were all part of the discussion.

LLS president and chief executive officer, Louis J. DeGennaro, PhD, set the stage for the roomful of attending researchers, treating physicians, industry executives and regulators at the Hilton Bayfront San Diego.

"AML is a formidable adversary in the medical and scientific community; this rapidly progressing disease remains one of the most lethal blood cancers," DeGennaro said. "LLS, along with our partners and the FDA, are changing the paradigm for cancer clinical trials, so we can get the right drug to the right patient at the right time."

LLS’s commitment to fighting AML will be among the news headlines of the ASH (Free ASH Whitepaper) conference, highlighted through the significant data being presented in five Beat AML abstracts. Further, LLS’s impact on AML extends beyond the clinical trial. LLS and its collaborators at Johns Hopkins University are also releasing data at the ASH (Free ASH Whitepaper) meeting through several abstracts on AML patient preferences for treatment outcomes, their worries, and their reported side effects from treatment.

According to Amy Burd, PhD, LLS vice president of research strategy and the organization’s lead for the Beat AML initiative, "Taken together, these five Beat AML abstracts tell a story of how, through collaboration, we have put in motion a dynamic trial model that is already having an impact on patients and how doctors approach treatment of AML."

More Progress in AML

Breakthroughs in AML have occurred rapidly over the past two years after a 40-year drought. From the start of 2017 to date, nine AML therapies have received approval from the U.S. Food and Drug Administration (FDA). All have been advanced with LLS support.

Three just received approval this month:

gilteritinib (XOSPATA), developed by Astellas Pharma, received FDA approval on November 28, to treat relapsed and refractory AML patients with a genetic mutation called FLT3, which are seen in approximately one-third of patients with AML. It is the second FLT3 inhibitor to receive FDA approval since 2017; the other, midostaurin (Rydapt ), was approved in April 2017, for newly diagnosed FLT3 AML patients. Gilteritinib is also part of the Beat AML Master Clinical Trial. While this recent approval is for patients who have failed previous treatments, it is being tested in Beat AML as a frontline treatment. Another FLT3 inhibitor, quizartinib is under review with a decision anticipated by Spring.
venetoclax (Venclexta ), developed by Abbvie and Genentech, received approval on November 21, in combination with a chemotherapy – azacitidine, decitabine or low-dose cytarabine (LDAC), in newly diagnosed patients who are aged 75 years and older or are ineligible for intensive chemotherapy due to coexisting medical conditions. This therapy targets the cellular pathway that regulates natural cell death. When this process goes awry as it often does in cancer the body is unable to rid itself of unhealthy cells.
glasdegib (Daurismo ), was also FDA approved on Nov. 21. Pfizer’s AML drug targets a cell signaling pathway called Hedgehog, which is critical for the development of immature cells into cells with more specialized functions. It is approved for patients who are age 75 or older, or who have other chronic health conditions or diseases that might preclude the use of intensive chemotherapy.
One of last night’s panelists, Erkut Bahceci, M.D., vice president, medical sciences-hematology, Astellas Pharma, discussed the approval of gilteritinib.

"We have learned from past failures of prior drugs targeting this same mutation and designed this drug to help overcome those weaknesses," Bahceci said. "In collaboration with the FDA, we were able to speed innovation and achieve this approval quickly. It only took five years since the first patient was treated with gilteritinib."

Other AML therapies approved in 2017 and 2018 are: midostaurin (Rydapt ); enasidenib (Idhifa);(CPX-351) (Vyxeos ); gentuzumab ozogamicin (Mylotarg); arsenic trioxide (Trisenox ); ivosedinib (Tibsovo). LLS supported research for each of these therapies.

LLS’s Commitment to AML

LLS has long been committed to leading the offensive on AML, with more than one-quarter of our research budget dedicated to finding new and better treatments. And we continue to provide lifesaving support and advocacy for AML patients and their caregivers. Among our most recent round of grants are multiple projects targeting AML, including a Specialized Center of Research grant led by Craig Jordan, Ph.D., University of Colorado, and his colleagues who are taking a multi-pronged approach to eradicate cancer stem cells in AML patients. The team was pivotal in demonstrating that the aforementioned, recently approved venetoclax can produce long-term disease control in patients with AML, and appears to be a game-changer for the future therapy of this and other diseases.

John Byrd, M.D., of The Ohio State University Comprehensive Cancer Center, and a co-lead investigator of the Beat AML trial, commented that with so many new options now for AML the key will be finding the right combinations.

"If you think of a cake and you only have flour and maybe a little sugar you won’t be able to make a very good cake," Byrd said. "I think now for subsets of AML we probably have the right ingredients to completely change the natural history of this disease. We just need to come up with the recipe for how to combine things."

Onco360® Selected to Dispense XOSPATA® (Gilteritinib)

On November 30, 2018 Onco360, the nation’s largest independent Oncology Pharmacy, reported that it was selected as a specialty pharmacy network partner for Astellas’ new product XOSPATA (gilteritinib) for the treatment of patients with relapsed or refractory FMS-like Tyrosine Kinase 3 (FLT3) mutation-positive acute myeloid leukemia (AML) (Press release, Onco360, NOV 30, 2018, View Source [SID1234531759]).

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"Onco360 is excited to be selected as one of only three specialty pharmacy providers for XOSPATA patients," said Paul Jardina, President and CEO, Onco360. "The recent approval of XOSPATA unlocks a new treatment option for AML patients with extremely limited treatment alternatives. As a provider of this key treatment, Onco360 can support the highly specialized needs of AML patients and their physicians across the United States."

AML is the most common form of acute leukemia in adults in the United States (U.S.). Only 27 percent of patients diagnosed with AML survive for five years following initial diagnosis. FLT3 mutations occur in about 33 percent of AML patients and is an indicator of a poor prognosis. The American Cancer Society estimates that in 2018, approximately 19,500 new patients will be diagnosed with AML in the U.S.1 Previously, there were no U.S. Food and Drug Administration (FDA)-approved products for patients with relapsed or refractory FLT3 mutation-positive AML.

XOSPATA is a manufactured by Astellas Pharma Inc. and was approved by the U.S. FDA on November 28, 2018 based on positive clinical trial results.2 Full prescribing information can be found at www.xospata.com.

XOSPATA is immediately available to order through Onco360. Referral forms and information on how to order can be found at Onco360.com/Order.

QIAGEN and NeoGenomics collaborate to offer cancer patients Day-One access to innovative companion diagnostics for newly approved drugs

On November 30, 2018 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) and NeoGenomics, Inc. (NASDAQ: NEO) reported a master service agreement to accelerate the availability of innovative companion diagnostics that enable precision medicine for cancer patients (Press release, Qiagen, NOV 30, 2018, View Source [SID1234531758]). The partnership between QIAGEN and NeoGenomics, a leading provider of cancer-focused genetic testing services, will ensure Day-One patient access to FDA-approved molecular tests paired with newly approved drugs for cancer.

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Building on the U.S. Food and Drug Administration’s modernized regulatory approach to advanced diagnostics, especially next-generation sequencing (NGS) tests, the collaboration with NeoGenomics will allow QIAGEN and pharmaceutical partners to streamline the development and launch of targeted drugs and companion diagnostics to guide treatment decisions. The partnership offers flexible pathways leading to introduction of FDA-approved companion diagnostics simultaneously with launch of new therapies.

QIAGEN and NeoGenomics will discuss their efforts to expedite access for precision medicine solutions at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting and Exposition from December 1-4, 2018, in San Diego.

"As a leading provider of oncology testing for both clinical trials and patient care, NeoGenomics is uniquely positioned to assist pharmaceutical and biotech companies to develop and commercialize companion diagnostic tests. Our collaboration with QIAGEN will ensure that patients have access to the most advanced companion diagnostics to target new cancer medicines, as soon as those medicines are approved. We are excited to work with QIAGEN to deploy cutting edge technologies to remain at the forefront of precision medicine" said Douglas M. VanOort, Chairman and CEO of NeoGenomics.

"We are excited to collaborate with NeoGenomics to ensure immediate availability of QIAGEN companion diagnostics during clinical trials and upon approval by the FDA, supporting synchronized launches of new oncology drugs, to make a difference for patients," said Peer Schatz, CEO of QIAGEN. "Together with our Pharma partners, we are now planning to provide investigational use only (IOU) tests to NeoGenomics and other labs, enabling them to verify, set up and run our companion diagnostics in clinical trials and in anticipation of regulatory approval. The companion diagnostic services can be promoted by NeoGenomics through their national commercial teams starting with their FDA approval, facilitating the rapid adoption of innovative targeted therapies which can deliver meaningful benefits to patients. We look forward to discussing this approach further at ASH (Free ASH Whitepaper) 2018, and demonstrating our rapidly evolving Sample to Insight solutions to the world’s top hematology and oncology experts."

NeoGenomics has a national footprint and broad customer reach in cancer-related genetic testing services and one of the most comprehensive oncology-focused test menus. The master service agreement provides a flexible framework with multiple options for co-development, verification, setup, and launch of new companion diagnostics, including next-generation sequencing tests, for biomarker profiling paired with new targeted drugs.

As QIAGEN collaborates with pharma and biotech partners, co-development progresses from drug discovery and creation of a biomarker test, to clinical development evaluating the proposed drug and test, to validation for clinical use and then commercialization of the new drug and companion diagnostic. Commercial alignment and launch readiness for companion diagnostics at the time of drug approval have become increasingly important for QIAGEN´s pharmaceutical partners. For more information, please visit View Source

Crescendo Biologics announces early licensing by Takeda of first oncology-targeted Humabody®

On November 30, 2018 Crescendo Biologics Ltd (Crescendo), the drug developer of novel, targeted T-cell enhancing therapeutics, reported that Takeda Pharmaceutical Company Limited (Takeda), has exercised an option under its existing, multi-target collaboration and license agreement (Press release, Crescendo Biologics, NOV 30, 2018, View Source [SID1234531757]). Takeda has taken an exclusive licence to Humabodies directed to one of its oncology targets.

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This licence option exercise comes substantially earlier than planned and marks the highly successful delivery and further pre-clinical evaluation by Takeda of Humabody leads meeting its stringent criteria.

Dr Peter Pack, CEO of Crescendo, commented:

"The team at Crescendo has made great progress on our Humabody programmes, working closely with the Takeda team. To date, we have met all the technical milestones on time or earlier than planned, which is proof of our excellent collaboration. We are delighted that the option to license has been taken by Takeda ahead of schedule and look forward to further future successes."

Chris Arendt, Head, Oncology Drug Discovery Unit & Immunology Unit, Takeda, commented:

"At Takeda, we continue to research diverse modalities to bring transformative treatments to patients with cancer. Our decision to exercise the licence was based on the quality of the Humabody leads and the potential we see to develop improved and differentiated immuno-oncology therapies."

Takeda’s option is part of the existing multi-target collaboration and licence agreement announced in October 2016 where Takeda received the right to develop and commercialise Humabody-based therapeutics resulting from the collaboration. Under the agreement, Crescendo is eligible to receive clinical development, regulatory and sales-based milestone payments of up to $754 million plus royalties on Humabody-based product sales by Takeda.

Turnstone Biologics Unveils Novel Vaccinia Oncolytic Viral Immunotherapy Platform

On November 30, 2018 Turnstone Biologics, a clinical-stage immuno-oncology company leading the next generation of oncolytic viral therapies, reported its pre-clinical data today supporting the development of a new Vaccinia therapeutic platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Tumor Immunology and Immunotherapy Meeting in Miami (Press release, Turnstone Biologics, NOV 30, 2018, View Source [SID1234531756]). This platform was developed by Dr. John Bell and his colleagues at The Ottawa Hospital Research Institute and the University of Ottawa, and engineered to be potent, highly selective and immune-stimulatory. Furthermore, the versatility of the platform enables additional therapeutic agents to be encoded in the virus and produced at tumor sites. This single platform virus has the potential to create a portfolio of diverse products. Turnstone has exclusively licensed this technology.

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This proprietary Vaccinia virus can be delivered systemically and is designed to target and kill cancer cells throughout the body, modulate the tumor microenvironment and stimulate both innate and adaptive immune responses. The virus also contains a very large transgene capacity, allowing for the insertion of multiple therapeutic agents. These agents will be produced selectively at tumor sites as the virus replicates only in the tumor cells, effectively enabling a local combination therapy from a single therapeutic promoting cancer cell killing. Turnstone is conducting IND-enabling studies on TBio-6517, its lead product candidate from this platform, consisting of the Vaccinia virus expressing three potent immune modulators, and anticipates initiating clinical studies in 2019.

"The decades-long search for immunotherapies and combinations that are safe and effective for a range of cancers has proven challenging," said Dr. Bell. "While previous Vaccinia therapies have shown promise, we believe that we have selected and designed significant improvements into this platform, potentially allowing us to safely and effectively combine multiple modes of action into a single therapy."

"This is an exciting time for Turnstone as we unveil another novel viral immunotherapy platform technology in our continued pursuit to develop transformative technologies that advance the treatment of cancer," said Mike Burgess, MBChB, Ph.D., President, Research and Development at Turnstone Biologics. "We are rapidly advancing our lead candidate from this proprietary platform, TBio-6517, to the clinic with the goal of achieving effective and durable outcomes for a greater number of cancer patients."

The oral presentation entitled "Utilizing Novel Oncolytic Vaccinia Virus for Selective Expression of Immunotherapeutic Proteins in Metastatic Tumors" includes key proof-of-concept data:

Cancer Cell Selectivity: The Turnstone Vaccinia virus was shown to rapidly kill more than 80% of cancer cells across multiple cell lines while sparing normal cells, exhibiting high tumor specificity in mouse models.
Potent Oncolytic Activity: The Turnstone Vaccinia virus replicated in human lung, sarcoma, melanoma, ovarian, gastic and thymic tumor explants, and showed efficacy with improved safety in the HT29 colon cancer tumor model.
Transgene Expression: TBio-6517 demonstrated selective expression of its three encoded immunomodulators at the sites of tumors in preclinical models, with no evidence for exposure in the peripheral blood.