On June 23, 2016 Mylan N.V. (NASDAQ, TASE: MYL), one of the world’s leading pharmaceutical companies, reported a €1 million donation to the Princess Máxima Center for Pediatric Oncology, a new national cancer center currently being built in Utrecht, the Netherlands (Press release, Mylan, JUN 23, 2016, View Source [SID:1234513522]).

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The donation from Mylan will support the Princess Máxima Center in its mission of treating and curing children with cancer by enhancing research efforts and supporting patients and their families while at the center for treatment.

Mylan Executive Chairman Robert J. Coury commented, "Mylan’s mission is to set new standards in healthcare and provide access to high quality medicine to patients around the world; however, our mission starts with the communities in which we operate, and we have had the privilege to serve customers and patients in the Netherlands for more than 17 years. The mission of the Princess Máxima Center is closely aligned with Mylan’s and I would like to commend Prof. Pieters for his vision and leadership in raising the standard of care for cancer in the Netherlands and ensuring children with cancer have equal access to the best care available."

Mylan CEO Heather Bresch added, "Every two days a child dies from cancer in the Netherlands and one in four children does not survive their fight with this disease. Through our commitment to providing access to oncology medications to patients around the world, we strive to do our part to help treat and cure cancer, and we believe our partnership with The Princess Máxima Center is just one of the ways we can go beyond medicine to deliver better health for a better world."

Professor Pieters, member of the board of the Princess Máxima Center commented, "We are grateful to Mylan for their generous support of the Princess Máxima Center, which is focused on treating children with cancer using the best possible methods, while also preventing the development of negative side effects in young patients, both now and over the long term. By bringing together expertise in care and research in the Princess Máxima Center, the Netherlands will become a leader in the field of pediatric oncology both in Europe and globally. It is a position that will help us fulfill our ambition: to cure every child with cancer. I am delighted that our collaboration with Mylan will further support the care we provide and our continued research."

Matean Niël, Mylan country manager in the Netherlands added: "I am thrilled to see Mylan supporting a cause in the Netherlands that puts people and patients first. Mylan’s support of the Princess Máxima Center creates a lot of pride among our hundreds of employees based in Bunschoten, Weesp and Zwolle. This initiative reflects our local commitment to institutional care in the Netherlands."

On June 22, 2016 IFM Therapeutics, a biopharmaceutical company developing a portfolio of first-in-class small molecules targeting the innate immune system, reported the closing of a $27 million Series A financing led by Atlas Venture and
Abingworth, with participation from Novartis. In conjunction with the funding Jean-François Formela and Vincent Miles, Partners at Atlas and Abingworth respectively, have joined IFM’s CEO, Gary D. Glick, on the board of directors, with Dr. Formela serving as chair of the board (Press release, IFM Therapeutics, JUN 22, 2016, View Source [SID1234520287]).

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IFM Therapeutics, incubated as a part of the Atlas Venture seed program, is developing modulators of novel targets that either enhance innate immune responses for treating cancer, or dampen certain immune responses that drive many inflammatory diseases. The company
will use the proceeds of the financing to advance and expand its early-stage portfolio and begin clinical development of its most advanced product candidate, a selective activator of a novel target, for treating solid tumors.

"While proteins in the innate immune system represent an attractive landscape of therapeutic targets, they have been notoriously difficult to drug," said Dr. Formela from Atlas. "During the brief period since its founding, IFM has made excellent progress on several of these targets, reflecting its exceptional team of experienced scientists and executives, possessing expertise in medicinal chemistry, a deep understanding of the relevant biology, and relationships with academic thought leaders in the areas of immunology and immune oncology."

"IFM’s programs have the potential to make a major difference in the lives of patients with serious, and sometimes fatal, diseases," said Abingworth’s Dr. Miles. "We look forward to helping the team build on their strong start to advance these programs into clinical development."

"This financing is an important validation of the IFM team and technology," said Dr. Glick, IFM’s Co-founder and CEO. "The company is fortunate to be working with a talented and experienced group of investors. Their expertise in building world-class biopharmaceutical
companies will be invaluable as we grow the company."

On June 22, 2016 Nymox Pharmaceutical Corporation (NASDAQ:NYMX) reported results from the Company’s 7 year prospective placebo controlled double blind studies of treatment of 995 U.S. men with the Company’s lead drug fexapotide (Press release, Nymox, JUN 22, 2016, View Source;fvtc=4&fvtv=6907 [SID:1234513492]). Men who received fexapotide showed a major reduction in the incidence of prostate cancer, compared to placebo and compared to the known and expected normal incidence of the disease.

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The men in the study received fexapotide or placebo for the treatment of their prostate enlargement (BPH) symptoms. All men were thoroughly evaluated to exclude any prostate cancer prior to qualifying for enrollment in the studies. The participants were enrolled at over 70 top well-known U.S. urological investigational centers, and were followed for up to 7 years (median of 5 years) after treatment. The study analyzed all cases of prostate cancer that were subsequently diagnosed. The expected rate of new prostate cancer in the U.S. general male population in this age group is in the 5-20% range after 7 years. In the BPH population in published large trials of drugs for the prevention of prostate cancer, the incidence of new prostate cancer cases after 4-7 years has been reported in major studies to be 20-25%. The new data analysis from the Nymox fexapotide study has now shown the statistically significant and very low incidence of 1.3% for prostate cancer in this comparable fexapotide treated BPH population.

"These results are astonishingly good. Other drug treatments and controls tested in similar studies have been associated with a prostate cancer incidence 10 times higher than the results reported today by Nymox for fexapotide. This is truly good news. The data strongly indicate that in addition to benefit for BPH symptoms, fexapotide will also help to prevent cancer in these patients," said Dr. Ronald Tutrone, one of the Principal Investigators in the Nymox Fexapotide Prostate Cancer and BPH studies. Dr. Tutrone is Chief of the Division of Urology, Greater Baltimore Medical Center; Medical Director of Chesapeake Urology Research Associates and Chairman of the William E. Kalhert Endowment for Urological Research.

Fexapotide is a safe and painless single injection treatment given in the urologist’s office. The drug is in Phase 3 for BPH and Phase 2 for prostate cancer. It has been tested in over 1700 drug and placebo treatment administrations in the U.S. As a treatment for BPH, fexapotide shows long-term efficacy without the safety risk and side effect concerns or added cancer risk associated with currently approved BPH treatments. As a treatment for prostate cancer fexapotide was found to lead to highly statistically significant reduction in disease progression in a large 147 patient multi-year Phase 2 study of U.S. men with low grade cancer.

Dr. Paul Averback, CEO of Nymox said, "The new results now add a third dimension to fexapotide utility: clinical prostate cancer prevention. The drug has now demonstrated statistically significant prospective long-term outcome data showing dramatic reduction in the incidence of newly diagnosed prostate cancer after minimal BPH treatment with fexapotide. Nymox announced in Q3 last year that it will seek regulatory approvals for fexapotide for BPH based on the long-term BPH safety and efficacy data announced Q3 last year. We believe that the exciting new prostate cancer prevention results reported today will add to the evidence in fexapotide’s favor towards our goal of widespread major benefit for middle-aged and elderly men."

Dr. Averback added, "We are extremely grateful to the thousands of people who have been part of these clinical trials. The Company also thankfully acknowledges our shareholders for their long-term commitment that supports these studies."

On June 22, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company reported dosing of the first patient in a Phase 1 clinical trial evaluating KPT-9274, an oral, first-in-class, dual-acting p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT) inhibitor, in patients with advanced solid malignancies (including sarcoma, colon and lung cancer) or non-Hodgkin’s lymphoma (NHL) whose disease has relapsed after standard therapy(s) (NCT02702492) (Press release, Karyopharm, JUN 22, 2016, View Source [SID1234517065]).

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This first-in-human, multi-center, open-label, dose-escalation trial is expected to enroll up to 175 patients. The primary endpoints of this study are to determine the recommended Phase 2 dose (RP2D) and the maximum tolerated dose (MTD) of KPT-9274 administered alone and with extended release niacin, and to evaluate safety and tolerability. The key secondary endpoint is to assess anti-tumor activity in patients predicted to be more sensitive to PAK4/NAMPT inhibition, including those with NAPRT-deficient tumors and tumors harboring IDH1 mutations.

"Our first-in-class, oral, small molecule PAK4 and NAMPT modulator, KPT-9274, has synergistic anti-tumor effects through several mechanisms including immune cell activation by inhibiting ß-catenin, reduction of NAD levels which tumor cells use as a key energy source, blockade of DNA damage repair mechanisms, and induction of tumor cell apoptosis," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "We are encouraged by the preclinical profile of KPT-9274, highlighted at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) and 2016 American Association of Cancer Research annual meetings, which support the novel mechanism and demonstrated encouraging preclinical anti-tumor activity. To our knowledge, we are the only company with a compound in clinical development that directly targets both PAK4 and NAMPT. This important Phase 1 study will continue to build upon the body of scientific evidence supporting KPT-9274’s safety and efficacy, and we look forward to reporting top-line data next year."

This Phase 1 clinical study is supported by extensive preclinical data demonstrating KPT-9274’s anti-cancer activity against hematological and solid tumors while showing minimal toxicity to normal cells in vitro. Preclinical studies demonstrate that by blocking PAK4, KPT-9274 potently inhibits ß-catenin as well as certain Ras oncogene-dependent pathways. ß-catenin is believed to be a key mediator of immune suppression, including resistance to immune-activating therapies. In addition, both ß-catenin and Ras are key growth signaling pathways for many common tumors such as colon and lung cancers. NAMPT, which can be found in a complex with PAK4 within the cell, is a pleiotropic protein with intra- and extra-cellular functions as an enzyme, cytokine, growth factor and hormone, and is thought to play a role in cellular energy metabolism. Hematologic and solid tumor cells become dependent on both PAK4 and NAMPT pathways and are therefore susceptible to single-agent cytotoxicity by KPT-9274. In mouse and rat xenograft studies, orally administered KPT-9274 showed robust anti-cancer activity with favorable tolerability. Based on in vitro and in vivo activity, Karyopharm believes KPT-9274 holds significant potential for the treatment of a wide variety of both solid and hematological cancers.

About KPT-9274

KPT-9274 is a first-in-class, orally bioavailable, small molecule immunometabolic modulator that works through non-competitive dual inhibition of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT). Co-inhibition of these targets is believed to lead to synergistic anti-tumor effects through suppression of ß-catenin by blocking PAK4, leading to both immune cell activation and inhibition of tumor growth, energy depletion through NAMPT inhibition, blockade of DNA repair, cell cycle arrest and ultimately apoptosis. KPT-9274 may therefore have both immune-activating and direct antitumor effects. In contrast, normal cells are less sensitive to inhibition by KPT-9274 due in part to their relative genomic stability and lower metabolic demands.

On June 21, 2016 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) ("Aptose" or the "Company") reported a corporate update in advance of the Company’s Annual General Meeting of shareholders to be held today at 10:00 AM ET in Toronto, Ontario, Canada (Press release, Aptose Biosciences, JUN 21, 2016, View Source;p=RssLanding&cat=news&id=2178995 [SID:1234513471]).

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Aptose previously announced on November 20, 2015 that an earlier formulation of APTO-253 drug product caused filter clogging during a Phase Ib clinical trial. Aptose voluntarily suspended dosing of patients and contacted the FDA, after which APTO-253 was placed on clinical hold for the filter clogging event. Subsequent activities sought to develop a new formulation in which APTO-253 would be soluble and stable, and to evaluate the formulation in mock infusion studies as part of a submission to the FDA.
In studies performed to date, Aptose has shown positive results with a new prototype formulation for APTO-253. The prototype formulation utilizes the same excipients that were used in the earlier drug formulation, except the methodology and order of addition of excipients were modified to create potentially a more soluble and stable formulation of APTO-253. In these exploratory studies, the prototype formulation was evaluated during mock infusion procedures at multiple dose levels, and no filter clogging was observed. Upon completion of formal studies, Aptose plans to collect and analyze all data and submit this analysis to the FDA for evaluation.

On June 8, 2016 Aptose and CrystalGenomics, Inc. announced an exclusive global option and license agreement focused on the development of CG026806 (CG’806), a first-in-class, highly potent, non-covalent small molecule inhibitor of the Bruton’s tyrosine kinase (BTK), FMS-like tyrosine kinase 3 (FLT3) and the Aurora kinases (AURK). Further to the execution of the agreement, Aptose expects to undertake Investigational New Drug (IND) enabling studies in the near future, and, if it exercises its option under the agreement, to initiate a Phase 1 clinical trial by mid-2017. The combination of CG’806’s potency and in vivo safety profile suggest that CG-806 may serve as an important therapeutic option for patients with AML, CLL and other malignancies.
In addition to APTO-253 and CG’806, in November, 2015 Aptose announced collaborations with Moffitt Cancer Center, and the medicinal chemistry organization, Laxai Avanti Life Sciences (LALS), to create and develop dual mechanism cancer agents to simultaneously target the epigenetic bromodomain 4 (BRD4) motif of BET proteins and the kinase active site of certain oncogenic enzymes. The program exists in the discovery / preclinical stage and is expected to deliver one or more new agents to the Aptose pipeline in the future.

"This past year has been a time of repair and growth for our drug product pipeline. Clearly, we faced the need to solve manufacturing issues related to APTO-253 and deliver a new formulation that can yield reliable clinical material to support the ongoing clinical trial in patients with AML. In addition, we halted the majority of legacy programs, while adding the CG’806 and the BRD4-kinase inhibitor programs to our pipeline. These actions were taken with the aim of creating shareholder value and developing highly differentiated drugs that can improve the lives of cancer patients. We are proud of our progress, and we look forward to advancing these programs in the coming years," commented William G. Rice, Ph.D., Chairman, President and CEO of Aptose Biosciences, Inc.

About Aptose
Aptose Biosciences is a clinical-stage biotechnology company committed to discovering and developing personalized therapies addressing unmet medical needs in oncology. Aptose is advancing new therapeutics focused on novel cellular targets on the leading edge of cancer research coupled with companion diagnostics to identify the optimal patient population for our products. The company’s small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. For further information, please visit www.aptose.com. Aptose Biosciences Inc. is listed on NASDAQ under the symbol APTO and on the TSX under the symbol APS.