On October 10, 2019 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated therapeutics targeting the underlying mechanisms of cancer, reported that William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, and Jotin Marango, M.D., Ph.D., Senior Vice President and Chief Business Officer, will participate at the 5th International Conference on Acute Myeloid Leukemia "Molecular and Translational" Advances in Biology and Treatment, and present preclinical data for CG-806, its oral, first-in-class pan-FLT3/pan-BTK inhibitor, in a poster presentation on October 24th, 2019 in Estoril, Portugal (Press release, Aptose Biosciences, OCT 10, 2019, View Source [SID1234540957]).

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"We are gratified by the enthusiasm of the AML community surrounding our mutation-agnostic kinase inhibitor CG-806," said Dr. Rice. "As we approach initiation of a new clinical study of CG-806 in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), we wish to highlight at this ESH conference the ability of CG-806 to suppress multiple oncogenic signals, to potently kill a diverse range of AML cells, including those resistant to various FLT3 inhibitors, and to enhance the activity of other anticancer agents. CG-806 is currently in a Phase 1 a/b clinical trial in patients with B-cell malignancies and to date, no drug-related toxicities have been observed."

CG-806 Poster Presentation Details:

CG-806 Pan-FLT3 / Pan-BTK Inhibitor Simultaneously Suppresses Multiple Oncogenic Signaling Pathways to Treat AML
Date & Time: Thursday, October 24th, 2019, 6:40 p.m. – 8:15 p.m. WEST (1:40 p.m. – 3:15 p.m. EDT)
Session Title: Acute myeloid leukemia – Biology & Translational Research
Abstract Number: 16594
Location: Estoril Congress Center, Avenida Amaral, 2765-192, Estoril, Portugal

The poster will be available on the Aptose website at the beginning of the poster session here.

About CG-806
CG-806 is an oral, first-in-class pan-FLT3/pan-BTK multi-cluster kinase inhibitor and is in a Phase 1 clinical trial for the treatment of hematologic malignancies. This small molecule, in-licensed from CrystalGenomics Inc. in Seoul, South Korea, demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), cures animals of AML in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML and other myeloid malignancies. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser (C481S) mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B cell malignancy patients (including CLL/SLL, FL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent or other non-covalent BTK inhibitors. Because CG-806 targets key kinases/pathways operative in malignancies derived from the bone marrow, it is in development for B-cell cancers and AML.

On October 10, 2019 Geron Corporation (Nasdaq: GERN) reported that the first patient has been dosed in the IMerge Phase 3 clinical trial to evaluate imetelstat, a first-in-class telomerase inhibitor, in lower risk myelodysplastic syndromes (MDS) (Press release, Geron, OCT 10, 2019, View Source [SID1234540956]).

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"Patients with lower risk MDS become dependent on serial transfusions which leads to iron overload, heart and kidney complications, decreases in quality of life and shorter overall survival. Reducing transfusion burden and achieving transfusion independence remain significant medical needs for this disease," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "Dosing of the first patient in the IMerge Phase 3 clinical trial is an important step in developing imetelstat as a potential alternative for patients with lower risk MDS who have limited treatment options available today."

IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythropoiesis-stimulating agents (ESAs). The Phase 3 is planned to enroll approximately 170 patients in a randomized, double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of red blood cell transfusion independence (TI). A target patient population of non-del(5q) lower risk MDS patients who are naïve to treatment with hypomethylating agents (HMAs) and lenalidomide was identified in Part 1 of IMerge, or the Phase 2 portion, and will be enrolled in the Phase 3. The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia. The primary endpoint is 8-week TI rate, which is defined as the proportion of patients achieving transfusion independence during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of transfusion independence lasting at least 24 weeks, or 24-week TI rate, durability of transfusion independence and the amount and relative change in transfusions.

Recently reported Phase 2 data from Part 1 of IMerge suggested meaningful and durable transfusion independence, as well as potential disease-modifying activity and transfusion independence across different MDS patient subgroups, potentially achievable with imetelstat treatment. Many key aspects from Part 1 of IMerge remain the same for the Phase 3, including the primary and secondary endpoints, the dose and schedule of imetelstat administration, the target patient population, and a majority of the participating clinical sites.

Based upon current planning assumptions, Geron expects top-line results for the IMerge Phase 3 clinical trial to be available by mid-year 2022.

To learn more about the IMerge Phase 3 clinical trial and whether the study is enrolling patients in your area, please visit www.clinicaltrials.gov.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of diverse blood disorders that develop because bone marrow cells do not mature into healthy blood cells. Many patients develop chronic anemia, the predominant clinical problem in lower risk MDS, and become dependent on red blood cell transfusions. There are approximately 60,000 people living with the disease in the United States.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On October 10, 2019 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported that it has amended its bond financing agreement with Kreos Capital and drawn down an additional €4 million of debt under the bond financing (Press release, NicOx, OCT 10, 2019, View Source [SID1234540955]). Nicox may now draw down a further €3 million or €8 million on December 31, 2019, subject to notice to Kreos by December 16, 2019.

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As of June 30, 2019, the Nicox Group had financial debt of €7.4 million under the bond financing agreement with Kreos Capital signed in January 2019. Nicox drew down €8.0 million in January 2019.

On October 10, 2019 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that the Company will present updates of clinical data from the MANIFEST clinical trial of CPI-0610 in oral and poster presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting on December 9 (Press release, Constellation Pharmaceuticals, OCT 10, 2019, View Source [SID1234540945]). Two abstracts reflecting MANIFEST data have been accepted by ASH (Free ASH Whitepaper) and will be published on November 6. Updated data will be presented at the meeting.

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ASH Oral Presentation

Title: MANIFEST, a Phase 2 Study of CPI-0610, a Bromodomain and Extraterminal Domain Inhibitor (BETi), as Monotherapy or Add-On to Ruxolitinib, in Patients with Refractory or Intolerant Advanced Myelofibrosis

Oral Session: 634. Myeloproliferative Syndromes: Clinical: JAK Inhibitors and Combination Therapies

Date and Time: December 9, 11:15 AM

Location: Orange County Convention Center, W311EFGH, Orlando, FL

Dr. John Mascarenhas, Associate Professor of the Icahn School of Medicine at Mount Sinai and an investigator in the MANIFEST clinical trial, will give the oral presentation with interim data from MANIFEST.

ASH Poster Presentation

Title: Preliminary Report of MANIFEST, a Phase 2 Study of CPI-0610, a Bromodomain and Extraterminal Domain Inhibitor (BETi), in combination with Ruxolitinib, in JAK Inhibitor (JAKi) Treatment Naïve Myelofibrosis Patients

Session: 634. Myeloproliferative Syndromes: Clinical: Poster III

Date and Time: Monday, December 9, 2019, Poster Display: 10:00 AM-8:00 PM EST, Poster Presentation: 6:00 PM-8:00 PM EST

Location: Orange County Convention Center, Hall B, Orlando, FL

Investor Event

Constellation will host an investor meeting and webcast conference call at 12:30 PM EST on December 9 at the Rosen Centre Hotel in Orlando to discuss the interim data from the MANIFEST oral presentation and poster at ASH (Free ASH Whitepaper). Further details will be provided later. If you would like to attend, please contact Ron Aldridge, Senior Director of Investor Relations, at [email protected].

On October 10, 2019 Medivir AB (Nasdaq Stockholm: MVIR) reported that the first patient has been enrolled in a phase I study to investigate the safety and tolerability of a combination treatment of birinapant and radiation therapy in patients with recurrent Head and Neck Squamous Cell Carcinoma (HNSCC) (Press release, Medivir, OCT 10, 2019, View Source [SID1234540942]). The initiative for the study came from researchers at the US National Cancer Institute (NCI). The study is sponsored and financed as part of NCI’s Cancer Therapy Evaluation Program (CTEP). Medivir provides birinapant and will be afforded full access to all reports from the study under its Cooperative Research and Development Agreement with NCI for birinapant. The primary goals of which are to evaluate the safety of the combination treatment and to establish a maximum tolerated dose for further studies. The potential signs of a treatment response in the patients are also investigated. Medivir’s decision to support this study was based on preclinical data from NCI showing good effects from birinapant in combination with radiotherapy in models of various types of cancer of the head and neck region [1]). Further information on the study can be found at clinicaltrials.gov with the reference number NCT03803774.

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"We believe that birinapant has the potential to form part of several different combination treatments for various types of cancer," says Uli Hacksell, PhD., CEO of Medivir. "The birinapant-radiation therapy combination treatment yielded impressive results in the preclinical cancer models, and we are eagerly awaiting the results of the phase I study, which will be a good complement to the ongoing phase II combination study of birinapant and Keytruda.

For further information, please contact:
Uli Hacksell, CEO, Medivir AB, mobile: +46(0)8-5468-3100

About Head and Neck Squamous Cell Carcinoma (HNSCC)
HNSCC is diagnosed in about 550,000 individuals worldwide annually. Of these around 63,000 cases occur in the United States, where around 13,000 patients die from the disease each year. Locoregionally advanced recurrent disease in patients who have had prior chemoradiation therapy has a poor prognosis, with a two year survival rate of less than 50%.

About birinapant
Birinapant is being developed to enhance responses, and extend survival, of patients with solid tumors where existing treatments do not provide sufficient survival benefit, or where patients no longer have treatment options. Based on its unique design and mechanism, birinapant has the potential to enhance patients’ responses in combination with other treatments. Medivir’s initial focus is on developing birinapant in combination with an immuno-oncology agent.