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 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.

On June 12, 2016 Pfizer Inc. today reported the publication of findings from the Phase 3 INO-VATE ALL study in the online issue of The New England Journal of Medicine (Press release, Pfizer, JUN 12, 2016, View Source [SID:1234513240]). The study, also known as Study 1022, is an open-label, randomized, Phase 3 study evaluating the safety and efficacy of inotuzumab ozogamicin as compared with investigator-choice chemotherapy in 326 adult patients with relapsed or refractory CD22-positive acute lymphoblastic leukemia (ALL). Results showed improvement over chemotherapy on a number of measures including complete hematologic remission and progression-free survival (PFS). Updated results and newly available overall survival (OS) data were also presented as a late-breaking oral presentation (#LB2233) today at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) 2016 Annual Meeting in Copenhagen, Denmark.

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"Relapsed or refractory ALL is an aggressive leukemia in urgent need of new treatment options as about half of adult patients will not respond to chemotherapy or will see their disease return," said Hagop M. Kantarjian, M. D., lead study investigator and professor, The University of Texas MD Anderson Cancer Center. "The efficacy results seen in patients treated with inotuzumab ozogamicin in this study are impressive, particularly median progression-free survival, high rates of hematological remission and absence of minimal residual disease. These results suggest inotuzumab ozogamicin, if approved, could be a valuable new addition to currently available treatment options for ALL patients, including as a bridge to stem cell transplantation, which is the best chance for a cure at this stage of the disease."

The INO-VATE ALL study had two independent primary endpoints, complete response with or without hematologic remission and OS. INO-VATE ALL met its first primary endpoint of complete response, which was significantly better with inotuzumab ozogamicin compared to chemotherapy (80.7% [95% CI, 72%-88%] vs. 29.4% [95% CI, 21%-39%], P<0.001). Inotuzumab ozogamicin also significantly extended PFS compared to chemotherapy (HR: 0.45 [97.5% CI, 0.34-0.61], P<0.001; median PFS, 5.0 vs. 1.8 months, in their respective arms). The second primary endpoint of OS showed a strong trend toward longer OS for patients treated with inotuzumab ozogamicin compared to chemotherapy, but did not reach the level of statistical significance (p < 0.0104) for the trial (HR: 0.77 [97.5% CI, 0.58-1.03], one-sided P=0.0203; median OS, 7.7 months [95% CI, 6.0-9.2] vs. 6.7 months [95% CI, 4.9-8.3]). The two-year OS rate for inotuzumab ozogamicin was 23 percent (95% CI, 16%‒30%) compared to chemotherapy at 10 percent (95% CI, 5%‒16%).

"Adult patients with relapsed or refractory ALL have a five-year survival rate of less than 10 percent, making these patients particularly difficult to treat. To see remission rates and two-year survival rates that are more than doubled compared to standard of care chemotherapy is very gratifying. We believe these data add to the growing body of evidence that supports inotuzumab ozogamicin as an important potential treatment option in adults with relapsed or refractory ALL," said Mace Rothenberg, MD, Chief Development Officer, Oncology, Pfizer Global Product Development.

Results from INO-VATE ALL also showed patients treated with inotuzumab ozogamicin achieved high rates of minimal residual disease (MRD) negativity (78.4% [95% CI, 68%-87%; P<0.001]), and experienced a duration of response (DOR) of 4.6 months (95% CI, 3.9-5.4; HR: 0.55; P<0.034). In comparison, 28.1 percent (95% CI, 14%-47%; P<0.001) of patients treated with chemotherapy achieved MRD negativity and median DOR was 3.1 months (95% CI, 1.4-4.9; HR: 0.55; P<0.034). More patients also proceeded to stem-cell transplant with inotuzumab ozogamicin compared to standard chemotherapy (41% vs. 11%, P<0.001).

The most common adverse events (AEs) observed for both inotuzumab ozogamicin and chemotherapy were cytopenias, including febrile neutropenia (16% vs. 22%). Common nonhematologic treatment-emergent AEs with inotuzumab ozogamicin included nausea (32%), headache (28%) and pyrexia (27%). Patients in the chemotherapy arm experienced nausea (47%), pyrexia (43%) and diarrhea (40%).

Additionally, any-grade veno-occlusive liver disease (VOD) occurred more frequently in patients treated with inotuzumab ozogamicin compared to chemotherapy (11% vs. 1%). Five patients taking inotuzumab ozogamicin developed VOD during treatment and 10 patients developed VOD after subsequent stem cell transplant. Among those taking chemotherapy, one patient developed VOD after transplant; no cases of VOD occurred during treatment with chemotherapy.

Inotuzumab ozogamicin received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for ALL in October 2015. Pfizer is working closely with the FDA and other regulatory authorities with the aim of making inotuzumab ozogamicin available for adult patients with relapsed or refractory CD22-positive ALL.

About Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL) is an aggressive type of leukemia with a poor prognosis in adults.1 The current foundational treatment is intensive, long-term chemotherapy.2 In 2016, it is estimated that 6,590 cases of ALL will be diagnosed in the United States, with about 2 in 5 cases in adults.3 Approximately 20 to 40 percent of newly diagnosed adults with ALL are cured with current treatment regimens.4 For patients with relapsed or refractory adult ALL, the five-year overall survival rate is less than 10 percent.5

About Inotuzumab Ozogamicin

Inotuzumab ozogamicin is an investigational antibody-drug conjugate (ADC) comprised of a monoclonal antibody (mAb) targeting CD22, a cell surface antigen found on cancer cells in almost all B-ALL patients, linked to a cytotoxic agent. 1,6 When inotuzumab ozogamicin binds to the CD22 antigen on malignant B-cells, it is thought to be internalized into the cell, where the cytotoxic agent calicheamicin is released to destroy the cell.7

Inotuzumab ozogamicin originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing and clinical development activities for this molecule.