On June 13, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported the presentation of data by Onconova collaborators from the U.S. and Europe from the ongoing Phase 2 clinical trial of oral rigosertib in combination with azacitidine in patients with either first- or second-line higher-risk myelodysplastic syndromes (HR-MDS) at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Copenhagen, Denmark, which took place June 9 — 11, 2016 (Press release, Onconova, JUN 13, 2016, View Source [SID:1234513246]).

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The results of this study are being finalized to initiate end of Phase 2 discussions with U.S. and European regulatory agencies to define the next steps in the development plan for this combination therapy. These interim results were initially presented at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015.

"We are encouraged by the interim overall response rate results of this Phase 2 trial, which demonstrated that 23 of 30 patients, or 77%, responded to treatment, and anticipate further updates to the data set," stated Steve Fruchtman, M.D., Chief Medical Officer of Onconova. "Of note, 84% of HMA-naïve patients responded to this novel combination. We look forward to discussing these results, and the longer-term follow-up from this study, with U.S. and European regulatory agencies in order to define the next step of development for this combination therapy."

A full copy of the EHA (Free EHA Whitepaper) poster entitled, "Results from Phase I/II Study of the Combination of Oral Rigosertib and Azacitidine in Patients with Myelodysplastic Syndromes (MDS)," may be accessed by visiting "Posters" in the Investors and Media section of Onconova’s website at www.onconova.com.

On June 13, 2016 GlycoMimetics, Inc. (NASDAQ:GLYC) reported that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its novel E-selectin antagonist GMI-1271 for treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) and elderly patients aged 60 years or older with AML (Press release, GlycoMimetics, JUN 13, 2016, View Source [SID1234617424]).

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"We believe GMI-1271 has the potential to address important unmet needs for individuals with relapsed or refractory AML, as well as for older AML patients, for whom the standard of care often fails to provide outcomes as positive as those seen in other patient groups," said Helen Thackray, M.D., Chief Medical Officer of GlycoMimetics. "The FDA’s granting of a Fast Track designation for GMI-1271 to treat AML is an important step in advancing this drug candidate through the regulatory process, and if successful, in bringing a novel drug to patients in an expedited time frame."

The Fast Track Designation is designed to facilitate development and expedite review of experimental therapies that address the unmet medical needs of patients with serious conditions.

AML is a cancer of immature white blood cells that starts in the bone marrow but can quickly spread into the blood, lymph nodes, liver, spleen, central nervous system, and testicles. Each year in the United States, about 19,900 people (usually older than 45 years of age) are diagnosed, and about 10,400 people die from all forms of the disease, according to the American Cancer Society. Chemotherapeutic methods among patients with refractory and relapsed AML have low remission rates, between 25 and 30 percent.

GlycoMimetics announced on Friday, June 10, presentation of data from the Phase 1 portion of its on-going Phase 1/ 2 clinical trial testing GMI-1271 combined with induction chemotherapy, in patients with relapsed/refractory acute myeloid leukemia (AML). Data was reported at the European Hematology Association (EHA) (Free EHA Whitepaper)21stCongress in Copenhagen, Denmark in a poster entitled "Results of a Phase 1 study of GMI-1271, a potent E-selectin antagonist in combination with induction chemotherapy in relapsed/refractory AML: a novel, well-tolerated regimen with a high remission rate."

In addition, GlycoMimetics recently announced that the first patient with relapsed or refractory AML has been dosed in the company’s Phase 2 portion of the ongoing Phase 1/2 clinical trial of GMI-1271. This clinical trial is a multinational open-label study evaluating endpoints for safety, pharmacokinetics (PK) and efficacy of GMI-1271 in combination with induction chemotherapy in patients with high-risk AML. This trial is being conducted at a number of academic medical institutions in the United States, Ireland, and Australia. While the primary objective is to assess safety, additional endpoints include overall response rate, biomarkers of activity, durability of response and overall survival. The Phase 2 portion of the study is expected to include approximately 25 participants with relapsed or refractory AML and approximately 25 participants who are newly diagnosed.

About GMI-1271

GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with AML cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper), GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.

On June 13, 2016 MabVax Therapeutics Holdings, Inc. (OTCQB: MBVX), a clinical-stage oncology drug development company, reported the initiation of a second investigational Phase I site for MVT-5873 (HuMab-5B1) with Sarah Cannon Research Institute through its network of research sites (Press release, MabVax, JUN 13, 2016, View Source [SID:1234513257]). The product is for patients with locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) or other CA19-9 positive malignancies. In addition to the Sarah Cannon Research Institute at Tennessee Oncology site in Nashville, Tenn., patient enrollment in the Phase I trial is underway within Sarah Cannon Research Institute at Florida Cancer Specialists in Sarasota, Fla.

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The Phase I trial is evaluating the safety, tolerability and pharmacokinetics of MVT-5873 as a single agent or in combination with the current standard-of-care chemotherapy regimen in patients with metastatic pancreatic cancer. The first group of patients are being enrolled in a traditional dose escalation regimen to assess safety and determine the optimal dose of the antibody. A second patient group will establish the safety and recommended dose of the antibody when administered with standard-of-care chemotherapy. Lead investigators at Sarah Cannon’s research sites are Todd M. Bauer, MD, associate director of drug development in Nashville, and Judy Wang, MD, associate director of drug development in Sarasota.

"We are very excited to engage with Sarah Cannon’s world-class clinical research organization and are highly encouraged by progress with patient enrollment to date," said David Hansen, MabVax President and CEO. "Pancreatic cancer is very difficult to treat and patients with advanced disease have few therapeutic options. We are delighted to be working with site staff at these research sites to evaluate MVT-5873 as a potential new therapeutic option for these patients."

About HuMab-5B1:

MabVax’s HuMab-5B1 antibody is fully human and was discovered from the immune response of cancer patients vaccinated with an antigen-specific vaccine during a Phase I trial at Memorial Sloan Kettering Cancer Center. In preclinical research, the 5B1 antibody has demonstrated high specificity and affinity, and has shown potent cancer cell killing capacity and efficacy in animal models of pancreatic, colon and small cell lung cancers. The antigen the antibody targets is expressed on more than 90% of pancreatic cancers making the antibody potentially broadly applicable to most patients suffering from this type of cancer.

On June 13, 2016 Perrigo Company plc (NYSE: PRGO; TASE) reported that it has filed Abbreviated New Drug Applications (ANDAs) with the U.S. Food and Drug Administration (FDA) for ingenol mebutate gel (0.015% and 0.05%), the generic versions of Picato gel (0.015% and 0.05%) (Press release, Perrigo Company, JUN 13, 2016, View Source [SID:1234514915]). Perrigo has notified Leo Pharma A/S and Leo Laboratories, Ltd., the owners of the New Drug Applications (NDAs) and patents listed in FDA’s Orange Book, of its filings.

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On June 10, 2016, Leo Pharma A/S, Leo Laboratories Limited and Leo Pharma, Inc. filed suit in the United States District Court for the District of Delaware alleging patent infringement. This action formally initiates the litigation process under the Hatch-Waxman Act. Perrigo believes that it is a first filer for this opportunity, making it eligible for 180 days of generic exclusivity.

Picato gel (ingenol mebutate gel) is a prescription medicine indicated for the topical treatment of actinic keratosis. Branded sales for the twelve months ending April 2016 were approximately $69 million.

Perrigo’s CEO John T. Hendrickson stated, "This filing illustrates Perrigo’s commitment to providing Quality Affordable Healthcare Products. The Rx team continues to invest in the development of important products that strengthen our extended topicals portfolio and drive savings for our customers and consumers."

On June 13, 2016 The Multiple Myeloma Research Foundation (MMRF), a leader in precision medicine, reported that its founder, Kathy Giusti, whom Fortune recently named as one of three business leaders who are disrupting medicine, has been appointed faculty co-chair of the Kraft Precision Medicine Accelerator at Harvard Business School (HBS) (Press release, Multiple Myeloma Research Foundation, JUN 13, 2016, View Source [SID:1234513283]).

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Giusti, an alumna of HBS, will lead the Harvard Business School Kraft Precision Medicine Accelerator with HBS faculty member and co-chair Richard Hamermesh under the umbrella of the School’s Health Care Initiative. She will continue to act as a member of the MMRF Board of Directors and take an active role in the organization’s leadership.

The HBS Kraft Precision Medicine Accelerator is made possible by a $20M endowment from the Kraft Family Foundation, under the leadership of foundation President, Robert K. Kraft. The Kraft family, through its family foundation, is committed to giving back to the community. The foundation’s primary mission includes supporting education, healthcare, science, and the needs of underserved individuals.

"Precision medicine has the potential to revolutionize the way we prevent, diagnose, treat, and, ultimately, cure cancer and other devastating diseases. I look forward just as much to sharing the MMRF model as I do to learning best practices from other world-class organizations focused on this promising approach," said Ms. Giusti, who is also a multiple myeloma patient.

"The promise of precision medicine will only be realized if we abandon a siloed approach to research and work collaboratively toward a greater good – two approaches Kathy Giusti has embraced and advocated in her nearly two decades of service to the cancer community. I am certain her vision and leadership of the HBS/Kraft Precision Medicine Accelerator will greatly improve the lives of people with cancer and other diseases," said Robert Kraft.

The mission of the HBS/Kraft Precision Medicine Accelerator is to speed innovation and medical breakthroughs in precision medicine, the process by which genomic information and other unique characteristics of a person’s disease are used to predict which treatments will be most effective. The Accelerator will convene best-in-class leaders from the business, medical, scientific, and technological communities to identify and solve challenges slowing the advancement of precision medicine, disseminate best practices and models to overcome these challenges, and, ultimately, enable the faster commercialization of high-impact innovations.

"Many of the biggest challenges in advancing breakthroughs promised by precision medicine are business challenges—how to best develop and commercialize medical solutions for public benefit, and how to optimally share data, for example. HBS is uniquely positioned to address these challenges by drawing on the ingenuity and expertise of our faculty, alumni, and students, and by convening the leaders who can develop innovative new models," said HBS Dean Nitin Nohria.