On November 10, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported initial clinical data from the ongoing VOYAGE Phase 1 study of FATE-NK100 as a monotherapy for the treatment of refractory or relapsed acute myelogenous leukemia (AML) (Press release, Fate Therapeutics, NOV 10, 2017, View Source [SID1234521924]). The data were presented today in a poster session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2017 Annual Meeting in National Harbor, Maryland.

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Anti-leukemia activity was observed with FATE-NK100, the Company’s first-in-class adaptive memory natural killer (NK) cell cancer immunotherapy, in each of the treated dose cohorts of the VOYAGE study. The subject in the second dose cohort (2×107 cells/kg) achieved a morphologic leukemia-free state (mLFS) following a single intravenous infusion of FATE-NK100 as a monotherapy. Prior to treatment, the subject presented in relapse, was refractory to conventional NK cell therapy and had 50% leukemic blasts in the bone marrow. At Day 14 following treatment, a bone marrow biopsy showed clearance of leukemic blasts in the marrow, and approximately 3×104 FATE-NK100 cells per mL were measured in the peripheral blood.

"These are encouraging early data for FATE-NK100 in refractory and relapsed AML, especially in patients with such high leukemic blast burden in the marrow that have exhausted all therapeutic options," said Sarah Cooley, M.D., Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation at the University of Minnesota and the clinical trial’s lead investigator. "The disappearance of all cells with morphologic characteristics of leukemia validates the in vivo anti-leukemia activity of FATE-NK100. We look forward to continuing enrollment in the VOYAGE study and to dosing the first patient with FATE-NK100 in the APOLLO study for the treatment of women with recurrent ovarian cancer."

The subject in the first dose cohort of VOYAGE (1×107 cells/kg) presented in primary induction failure with 87% leukemic blasts in the bone marrow. Two weeks following a single infusion of FATE-NK100, a bone marrow biopsy revealed a nearly 50% reduction in leukemic blasts. In addition, approximately 76% of NK cells in the peripheral blood were of FATE-NK100 origin.

"The significant reduction of leukemic blasts in the bone marrow observed in both subjects without any dose-limiting toxicities is very promising," stated Scott Wolchko, Chief Executive Officer of Fate Therapeutics. "Although the second subject’s morphologic leukemia-free state was not sustained following a single dose of FATE-NK100, the clearance of leukemia at Day 14 coupled with the presence of FATE-NK100 in circulation validates the cell product’s enhanced potency and persistence. We look forward to continuing subject enrollment and to combining FATE-NK100 with monoclonal antibody therapy for the treatment of advanced solid tumors through the launch of our DIMENSION study."

The accelerated dose-escalation design of VOYAGE is designed to evaluate the safety and determine the maximum dose of a single intravenous infusion of FATE-NK100 as a monotherapy. The three dose levels are 1×107, 2×107 and up to 1×108 adjusted to kg of body weight. FATE-NK100 has now advanced through the first two dose cohorts with no reports of dose limiting toxicities (DLTs). The third dose cohort is currently enrolling. A ten-subject expansion cohort is expected to be enrolled at the maximum dose level.

About FATE-NK100
FATE-NK100 is a first-in-class natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells, a highly specialized and functionally distinct subset of activated NK cells expressing the maturation marker CD57. Higher frequencies of CD57+ NK cells in the peripheral blood or tumor microenvironment in cancer patients have been linked to better clinical outcomes. In preclinical studies, FATE-NK100 has demonstrated enhanced anti-tumor activity across a broad range of hematologic and solid tumors, with augmented cytokine production, improved persistence and increased resistance to immune checkpoint pathways compared to other NK cell therapies that are being clinically administered today. FATE-NK100 is produced through a feeder-free, seven-day manufacturing process during which NK cells sourced from a healthy donor are activated ex vivo with pharmacologic modulators.

About VOYAGE
VOYAGE is an open-label, accelerated dose-escalation, Phase 1 clinical trial designed to evaluate the safety and determine the maximum dose of a single intravenous infusion of FATE-NK100 as a monotherapy administered after lymphodepleting chemotherapy followed by sub-cutaneous IL-2 administration in subjects with refractory or relapsed acute myelogenous leukemia (AML). One subject is being enrolled in each of the three planned dose cohorts. A ten-subject expansion cohort is expected to be enrolled at the maximum dose level. Anti-tumor activity of FATE-NK100 is being assessed by rates of complete response, disease-free survival and overall survival. The study is being conducted at the Masonic Cancer Center, University of Minnesota as an investigator-initiated study.

Acute Myelogenous Leukemia
Acute myelogenous leukemia (AML) is an aggressive cancer of the blood and bone marrow that progresses rapidly without treatment. Cancerous cells called leukemic blasts multiply and displace normal cells in the bone marrow, disrupting normal blood cell production. Each year in the United States, about 19,900 people are diagnosed with AML, and about 10,400 people die from all forms of the disease, according to the American Cancer Society. Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.

On November 10, 2017 Alexo Therapeutics, a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint mechanism exploited by cancer cells to evade the immune system, reported data from Alexo’s first-in-human Phase 1 study of its lead candidate, ALX148, at the Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), being held in National Harbor, Maryland (Press release, Alexo Therapeutics, NOV 10, 2017, View Source [SID1234522016]). Preliminary results from the single agent portion of the trial (NCT03013218) showed that ALX148 is generally well tolerated in patients with advanced malignancy, with no dose-dependent impact on normal blood cells.

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"ALX148 is generally well tolerated and its clinical safety profile is consistent with that seen in our preclinical studies," said Sophia Randolph, M.D., Ph.D., Chief Medical Officer of Alexo. "Given the unique design of this high affinity CD47 blocker, we were able to achieve anticipated safety and pharmacokinetics, and complete CD47 target occupancy across the dosing interval. Having demonstrated single agent safety, we are encouraged to explore ALX148 in combination as a potential treatment option for cancer patients."

As of October 2017, ALX148 was intravenously administered as a single agent over a dose range of 0.3 mg/kg to 30 mg/kg in 17 patients with advanced malignancy. There was one treatment-related serious adverse event (neutropenia plus infection; 3.0 mg/kg) with no additional treatment-related events of neutropenia or infection of any grade reported. Most treatment-related adverse events were Grade 1 or 2 and occurred across eight patients, as sole events. Evaluation of the highest protocol defined dose level (30 mg/kg) is ongoing. Upon completion of the single agent portion, the second half of the trial will evaluate ALX148 in combination with checkpoint inhibitors and targeted anti-cancer antibodies.

About ALX148
ALX148 is a fusion protein that comprises an engineered high affinity CD47 binding domain of SIRPα linked to an inactive Fc region of human immunoglobulin. ALX148 potently and selectively binds CD47, blocking its interaction with SIRPα, thereby inhibiting a key immune checkpoint mechanism exploited by cancer cells. In preclinical studies, ALX148 enhances checkpoint inhibition by activating dendritic cells and reducing suppression by tumor-associated macrophages, and enhances targeted anti-cancer antibodies by maximizing phagocytosis to selectively eliminate tumor cells. ALX148 has demonstrated significant inhibition of tumor growth in these combinations with no adverse effect on CD47-expressing normal blood cells in preclinical models.

On November 10, 2017 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported that five posters based on the Company’s PD-1-directed franchise will be presented at the 2017 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in National Harbor, MD (Press release, MacroGenics, NOV 10, 2017, View Source [SID1234521925]).

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Posters will be displayed in the poster hall both Friday, November 10 and Saturday, November 11. Presentation details are provided below:

MGA012 (anti-PD-1 monoclonal antibody):

P249: "A Phase 1 Study of the Safety, Tolerability, and Pharmacokinetics (PK) of MGA012 (anti‐PD‐1 Antibody) in Patients with Advanced Solid Tumors"

P336: "Preclinical Characterization of MGA012, a Novel Clinical‐stage PD‐1 Monoclonal Antibody"

MGD013 (PD-1 x LAG-3 bispecific DART molecule):

P244: "A Phase 1, First‐in‐Human, Open‐label, Dose Escalation Study of MGD013, a Bispecific DART Protein Binding PD‐1 and LAG‐3 in Patients with Unresectable or Metastatic Neoplasms"

P337: "Preclinical Characterization of MGD013, a PD‐1 x LAG‐3 Bispecific DART Molecule"

MGD019 (PD-1 x CTLA-4 bispecific DART molecule):

P308: "A PD‐1 x CTLA‐4 Bispecific DART Protein with Optimal Dual Checkpoint Blockade and Favorable Tolerability in Non-human Primates"

The above posters are available for download from the Events & Presentations page on MacroGenics’ website at View Source

On November 10, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported data from preclinical studies with ADU-1604, the company’s humanized anti-CTLA-4 monoclonal antibody. Data from these in vitro and in vivo studies demonstrate the potency of ADU-1604 and its ability to inhibit tumor growth and enhance T cell-dependent antibody responses (Press release, Sangamo Therapeutics, NOV 10, 2017, View Source;p=RssLanding&cat=news&id=2316178 [SID1234521919]). These data, which will be highlighted later today in a poster presentation (Poster #335) at the 32ND Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), underscore the potential application of ADU-1604 for the treatment of multiple cancer types, either as monotherapy or in combination with other therapies.

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"These data from preclinical studies of ADU-1604, a novel anti-CTLA-4 product candidate derived from our proprietary B-select antibody platform, are encouraging and provide support to file an Investigational New Drug Application to advance ADU-1604 into clinical studies," stated Andrea van Elsas, Ph.D., chief scientific officer of Aduro Biotech. "As a company with multiple programs and proprietary technology platforms, we are well positioned to leverage our product candidates, as monotherapies and in rational combinations, to develop new treatment options for patients in need."

Presentation Title: Characterization of a novel differentiated anti-CTLA-4 antibody (ADU-1604) in vitro and in vivo
Researchers conducted in vitro and in vivo studies comparing ADU-1604 to benchmark anti-CTLA-4 antibodies 10D1 (‘ipilimumab’) and CP-675,206 (‘tremelimumab’). Data from these studies demonstrate that ADU-1604 binds to a unique epitope on a human CTLA-4 (hCTLA-4) and is at least comparable to benchmarks in functionality. Data from in vivo studies using a well-established humanized mouse model of non-small cell lung cancer and a non-human primate model, demonstrate that ADU-1604 inhibits tumor growth and enhances T cell responses, respectively. Further, proof of concept studies in syngeneic mouse models demonstrate that anti-CTLA-4 further enhances anti-tumor activity when used in combination with ADU-S100 (also known as MIW815), Aduro’s lead investigational STING agonist, and in combination with Aduro’s proprietary immunotherapy platform of live-attenuated double-deleted Listeria monocytogenes strains (LADD).

About CTLA-4
Cytotoxic T‐lymphocyte‐associated protein 4 (CTLA‐4) is a negative regulator of T‐cell responses and is an immune checkpoint. Blocking CTLA-4 using antibodies may produce an anti-tumor response by enhancing T cell activation and their cancer cell killing activity in the tumor. This therapeutic target has been clinically validated by others in advanced melanoma. Aduro is developing a proprietary humanized anti-CTLA-4 antibody (ADU-1604) that binds to a unique epitope and its potency has been demonstrated in vitro and in vivo. Based on preclinical studies, Aduro believes that ADU-1604 when combined with innate and adaptive immune cell stimulators, such as STING agonists and cancer vaccines, can display an amplified anti-tumor effect against poorly immunogenic tumors. Aduro’s CTLA-4 antibody is being advanced through IND-enabling studies.

On November 10, 2017 Nymox Pharmaceutical Corporation (NASDAQ:NYMX) reported that a Symposium on Fexapotide Triflutate studies will be held at the Annual Meeting of the American Urological Association North Central Section, in Scottsdale AZ next week (Press release, Nymox, NOV 10, 2017, View Source;fvtc=4&fvtv=6907 [SID1234521932]). The symposium, "Fexapotide Triflutate: Long-Term BPH Studies 2009-2017" will be chaired by Mohamed Bidair MD of San Diego CA. The other panel members at the Symposium will be Alan Hay MD FACS of Salem OR, Stephen Richardson MD of Salt Lake City UT, and Barton Wachs MD of Long Beach CA.

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Nymox’s lead drug Fexapotide has been in development for over 10 years and has been tested by expert clinical trial investigative teams in over 70 distinguished clinical trial centers throughout the US, and has been found after 7 years of prospective placebo controlled double blind studies of treatment of 995 U.S. men with prostate enlargement to not only show clinically meaningful and durable relief of BPH symptoms, but also to show a major reduction in the incidence of prostate cancer, compared to placebo and compared to the known and expected normal incidence of the disease. The same clinical program has also shown in a long-term blinded placebo crossover group study an 82-95% reduction in the number of these patients who required surgery after they received crossover Fexapotide in the trial, as compared to patients who did not receive Fexapotide but instead received crossover conventional approved BPH treatments (p<.0001).

The Symposium will present detailed clinical data on the Phase 3 clinical trials that have been completed for Fexapotide and that have shown excellent safety and significant efficacy for the treatment of BPH. In addition, scientific data supporting the safety and efficacy from non-clinical and laboratory testing and analysis will be demonstrated. The main presentation will be followed by a panel discussion and by an interactive question and answer session with the specialist doctors in attendance.

For more information please contact [email protected] or 800-936-9669.

Forward Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding Nymox, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements regarding the need for new options to treat BPH and prostate cancer, the potential of Fexapotide to treat BPH and prostate cancer and the estimated timing of further developments for Fexapotide. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development program, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, including the regulatory approval process, the timing of Nymox’s regulatory filings, Nymox’s substantial dependence on Fexapotide, Nymox’s commercialization plans and efforts and other matters that could affect the availability or commercial potential of Fexapotide. Nymox undertakes no obligation to update or revise any forward looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Nymox in general, see Nymox’s current and future reports filed with the U.S. Securities and Exchange Commission, including its Annual Report on Form 20-F for the year ended December 31, 2016, and its Quarterly Reports.