On September 3, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that it has successfully completed the dose-escalation part of the Phase 1/2a clinical study for AGI-134, a novel compound that evokes a direct anti-tumor response, as well as a vaccine effect, via a unique, multi-arm mechanism that targets patient-specific tumor neoantigens (Press release, BioLineRx, SEP 3, 2019, View Source [SID1234539206]). AGI-134 was found to be safe and well tolerated, with no serious drug-related adverse events or dose-limiting toxicities reported. The maximal tolerated dose was not reached and the recommended dose for part 2 of the study was determined.

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The ongoing Phase 1/2a study is a multicenter, open-label study expected to take place at approximately 15 sites in the US, UK and Israel. The objectives of the study are to evaluate the safety and tolerability of AGI-134 at the recommended dose in multiple solid tumor types, to evaluate a wide array of biomarkers, and to validate AGI-134’s mechanism of action. Furthermore, efficacy will be assessed by clinical and pharmacodynamic parameters. The dose-expansion part 2 of the study is expected to commence shortly.

Prof. Mark Middleton of the University of Oxford, the study’s principal investigator, stated, "We are pleased with these initial safety results of the first-in-human clinical trial assessing AGI-134 for the treatment of solid tumors. AGI-134 represents a new mechanistic class of cancer immunotherapies, with a unique and highly differentiated mode of action, harnessing pre-existing immune machinery to trigger a systemic anti-tumor response and create a pro-inflammatory tumor microenvironment. We expect the mechanistic assessments performed during the study to further elucidate and confirm AGI-134’s activity. These assessments are ongoing and will be extended during part 2 of the study."

"We are excited with the positive results of the first part of the Phase 1/2a of our second lead oncology asset," said Philip Serlin, Chief Executive Officer of BioLineRx. "Numerous pre-clinical studies to date have demonstrated that treatment with AGI-134 leads to regression of established primary tumors, prevents growth of untreated distal secondary tumors, and triggers a vaccine effect that may prevent the development of future metastases. Following the FDA’s recent IND approval for AGI-134, we plan to add sites in the US to the study, which is currently being conducted in the UK and Israel, by the first half of 2020. We are looking forward to initiating part 2 of the study shortly, with initial results expected by year-end 2020."

"In addition, we are also waiting with great anticipation for the upcoming top-line data for BL-8040’s COMBAT/KEYNOTE-202 trial in pancreatic cancer, which is running according to schedule and is expected to read out by the end of the year," added Mr. Serlin.

About AGI-134

AGI-134 is a synthetic alpha-Gal glycolipid in development for solid tumors that is highly differentiated from other cancer immunotherapies. AGI-134 is designed to label cancer cells with alpha-Gal via intra-tumoral administration, thereby targeting the body’s pre-existing, highly abundant anti-alpha-Gal (anti-Gal) antibodies and redirecting them to treated tumors. Binding of anti-Gal antibodies to the treated tumors results in activation of the complement cascade, which destroys the tumor cells and creates a pro-inflammatory tumor microenvironment that also induces a systemic, specific anti-tumor (vaccine) response to the patient’s own tumor neo-antigens.

AGI-134 has been evaluated in numerous pre-clinical studies. In a mouse melanoma model, treatment with AGI-134 led to regression of established primary tumors and suppression of secondary tumor (metastases) development. Synergy has also been demonstrated in additional pre-clinical studies when combined with an anti-PD-1 immune checkpoint inhibitor, offering the potential to broaden the utility of such immunotherapies, and improve the rate and duration of responses in multiple cancer types. AGI-134 was obtained by BioLineRx through the acquisition of Agalimmune Ltd.

On September 3, 2019 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its participation in the 21st Annual Global Investment Conference sponsored by H.C. Wainwright & Co. LLC (Press release, ProMIS Neurosciences, SEP 3, 2019, https://www.prnewswire.com/news-releases/promis-neurosciences-to-present-at-hc-wainwright-investment-conference-300909802.html [SID1234539223]). The conference is being held September 8-10, 2019 at the New York Palace Hotel in New York.

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ProMIS’ Executive Chairman, Eugene Williams will provide overviews of its novel drug discovery and development programs for Alzheimer’s disease, Parkinson’s disease and ALS (amyotrophic lateral sclerosis) Tuesday, September 10th at 10:25 am ET. The audio webcast and slides of Mr. Williams’ presentation will be archived and available on ProMIS’ web site and through the following link View Source

On September 3, 2019 Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Tokyo, Japan, reported that the US Food & Drug Administration (FDA) has granted orphan drug designation for the company’s orally administered fixed-dose combination of cedazuridine and decitabine (ASTX727 or C-DEC) for the treatment of myelodysplastic syndromes (MDS) including chronic myelomonocytic leukemia (CMML) (Press release, Astex Pharmaceuticals, SEP 3, 2019, View Source [SID1234539239]). The designation follows on from the recent announcement that the phase 3 ASCERTAIN study of ASTX727 in adults with intermediate and high-risk MDS or CMML met the primary endpoint of decitabine exposure equivalence of total 5-day dosing between oral ASTX727 and IV decitabine. The data from the study will be presented at an upcoming scientific meeting. The designation provides for seven years of marketing exclusivity in the US following product approval, as well as certain tax incentives and grants.

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"We are delighted that the FDA has granted orphan drug designation for the fixed dose combination of cedazuridine with decitabine," said Mohammad Azab, MD, Astex Pharmaceuticals’ president and chief medical officer. "Subject to regulatory review and approvals, ASTX727 could bring a new treatment option to patients with these deadly diseases. We are extremely grateful to all the patients, caregivers, partner research and manufacturing organizations, as well as the healthcare professionals who contributed to the clinical development program of ASTX727."

ASTX727 is an investigational compound and is not currently approved in any country. Astex plans to file an NDA with the US FDA by the end of 2019.

Astex’s parent company, Otsuka Pharmaceutical Co., Ltd., and Taiho Pharmaceutical Co., Ltd. previously announced that, subject to regulatory approvals, commercialization of ASTX727 in the US and Canada will be conducted by Taiho Oncology, Inc. and Taiho Pharma Canada, Inc. respectively. Astex, Otsuka and Taiho are all members of the Otsuka group of companies.

About Cedazuridine and Decitabine Fixed-Dose Combination (ASTX727)

ASTX727 is a novel, orally administered fixed dose combination of cedazuridine, an inhibitor of cytidine deaminase,1 with the anti-cancer DNA hypomethylating agent, decitabine.2 By inhibiting cytidine deaminase in the gut and the liver, ASTX727 is designed to allow for oral delivery of the approved DNA hypomethylating agent, decitabine, at exposures which emulate exposures achieved with the approved intravenous form of decitabine administered over 5 days.3

ASTX727 has been evaluated in a phase 1/2 pharmacokinetics-guided dose escalation and dose confirmation study in patients with MDS and CMML (see View Source NCT02103478) and a pivotal phase 3 study (ASCERTAIN) (see View Source NCT03306264) conducted at investigator sites in the US and Canada and designed to confirm the results from the phase 1/2 study. The phase 3 study is being extended to include patients with acute myeloid leukemia (AML) unsuitable to receive intensive induction chemotherapy.

The concept of using cedazuridine to block the action of cytidine deaminase is also being evaluated in a low dose formulation of cedazuridine and decitabine for the treatment of lower risk MDS (see View Source NCT03502668).

About Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Myelodysplastic syndromes are a heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias affecting one or more of the three lineages. US incidence of MDS is estimated to be 10,000 cases per year, although the condition is thought to be under-diagnosed.4,5 The prevalence has been estimated to be from 60,000 to 170,000 in the US.6 MDS may evolve into acute myeloid leukemia (AML) in one-third of patients.7 The prognosis for MDS patients is poor; patients die from complications associated with cytopenias (infections and bleeding) or from transformation to AML. CMML is a clonal hematopoietic malignancy characterized by accumulation of abnormal monocytes in the bone marrow and in blood. The incidence of CMML in the US is approximately 1,100 new cases per year,8 and CMML may transform into AML in 15% to 30% of patients.9 The hypomethylating agents decitabine and azacitidine are effective treatment modalities for hematologic cancers and are FDA-approved for the treatment of higher risk MDS and CMML. These agents are administered by IV infusion, or by large-volume subcutaneous injections.

On September 3, 2019, AIM ImmunoTech Inc. (the "Company") reported its corporate presentation (Presentation, Hemispherx Biopharma, SEP 3, 2019, View Source [SID1234539394]).

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On September 3, 2019 Alector, Inc. (Nasdaq: ALEC), a clinical stage biotechnology company pioneering immuno-neurology, reported that Arnon Rosenthal, Ph.D., chief executive officer of Alector will participate in a fireside chat at the 17th Annual Morgan Stanley Global Healthcare Conference on Tuesday, Sept. 10, at 12:55 p.m. ET in New York City (Press release, Alector, SEP 3, 2019, View Source [SID1234551116]).

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To access the audio portion of the fireside chat, please visit the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 90 days following the conference.