On December 11, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX), reported the presentation of six posters highlighting preclinical and clinical data sets for TGR-1202 (umbralisib), the Company’s once-daily PI3K delta inhibitor, and TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, currently being held at the Georgia World Congress Center in Atlanta, Georgia (Press release, TG Therapeutics, DEC 11, 2017, View Source [SID1234522557]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "We are very pleased by the data presented yesterday and today during the ASH (Free ASH Whitepaper) annual meeting. The preclinical data help us to better understand the difference between TGR-1202 and other agents in the class and offers a more complete rationale for the differentiated safety profile seen in the clinic. With the updated and expanded integrated safety analysis of TGR-1202 alone and in combination with other agents, we believe we have provided the long-term follow-up sufficient to allay any lingering safety concerns related to TGR-1202 caused by the toxicity profile of first generation PI3K delta inhibitors." Mr. Weiss continued, "In 2018, with registration-directed data expected in CLL and NHL, our focus will turn to showcasing the efficacy of TGR-1202 and our proprietary combination of TG-1101 plus TGR-1202, our U2 combination, ideally leading to NDA/BLA filings in CLL and NHL."

The following summarizes the highlights from each poster presented at the ASH (Free ASH Whitepaper) 2017 meeting.

Clinical Data Presentations:

An Integrated Safety Analysis of the Next Generation PI3K Delta Inhibitor Umbralisib (TGR-1202) in Patients with Relapsed/Refractory Lymphoid Malignancies

This presentation includes data that were pooled from 5 completed or ongoing Phase 1 or 2 studies containing TGR-1202, including a total of 347 patients with relapsed or refractory hematologic malignancies. Patients were heavily pretreated, with 50% of patients having seen 3 or more prior lines of therapy.

Highlights from this poster include:

● 347 patients have been treated with TGR-1202 across the 5 studies in this pooled analysis, with median duration of exposure of 6.5 months, and 176 patients on drug for 6+ months, 104 patients for 12+ months, with the longest patients on daily TGR-1202 for 4+ years
● In longer follow-up and in an expanded patient population, TGR-1202 exhibits a differentiated safety profile compared to prior generation PI3K delta inhibitors
● Discontinuations due to adverse events (AEs) were rare at under 10% for all studies
● Grade 3/4 AEs commonly associated with PI3K delta inhibitors have been rare, with pneumonitis (< 0.5%), transaminitis (~2%) and colitis (< 1%), the latter occurring with no apparent association to time on therapy
● Improved tolerability with few discontinuations due to AEs has allowed patients to remain on continuous dosing to achieve and sustain promisingly high rates of response:
o 85% Overall Response Rate (ORR) for single agent TGR-1202 in relapsed/refractory Chronic Lymphocytic Leukemia (CLL)
o 53% ORR for single agent TGR-1202 in relapsed/refractory Follicular Lymphoma (FL)

KI Intolerance Study: A Phase 2 Study to Assess the Safety and Efficacy of Umbralisib (TGR-1202) In Patients with Chronic Lymphocytic Leukemia (CLL) Who Are Intolerant to Prior BTK or PI3K-delta Inhibitor Therapy (Abstract Number 4314)

This poster presentation includes data from patients with CLL who are intolerant to prior BTK or PI3K delta inhibitor therapy who were then treated with single agent TGR-1202. To be eligible for the study patients had to have received prior treatment with a BTK inhibitor (ibrutinib, acalabrutinib) or a PI3K delta inhibitor (idelalisib, duvelisib) and discontinued therapy due to intolerance within 12 months of starting treatment on this study. Thirty-three patients were evaluable for safety (30 patients with ibrutinib intolerance, and 3 patients with idelalisib intolerance) of which 32 were evaluable for efficacy (1 patient had a confirmed Richter’s Transformation (RT) at enrollment which did not meet eligibility criteria). TGR-1202 appears to demonstrate a favorable safety profile in patients intolerant to prior ibrutinib or idelalisib, with only 2 patients (6%) discontinuing due to an adverse event, neither of which was a recurrent AE from prior TKI therapy.

Highlights from this poster include:

● 94% (30 of 32) of patients remain progression-free
● Median time on study at the data cut off was approximately 6 months with the majority of patients continuing on study and follow-up ongoing
● No patient discontinued TGR-1202 due to a recurrent AE which led to discontinuation from their prior kinase inhibitor

Phase I/II Study of Pembrolizumab in Combination with Ublituximab (TG-1101) and Umbralisib (TGR-1202) in Patients with Relapsed/Refractory CLL (Abstract Number 3010)

This presentation includes data from patients with relapsed or refractory Chronic Lymphocytic Leukemia (CLL) or Richter’s Transformation (RT) treated with the triple combination of TG-1101, TGR-1202, and pembrolizumab. Eleven patients were evaluable for safety (9 CLL patients and 2 RT patients) and 10 were evaluable for efficacy (9 CLL and 1 RT), with one patient too early to evaluate.

Highlights from this poster include:

● One AE of increased LFTs was observed which met criteria for DLT; patient was re-challenged and remains on study treatment with TGR-1202 maintenance now 15+ months
● 78% (7 of 9) ORR in patients with relapsed/refractory CLL
● 75% (3 of 4) ORR in BTK refractory CLL patients
● Responses have been durable with the first patient progression-free for 24+ months

Preclinical Data Presentations:

Differential Regulation of T Cells By PI3K Delta Inhibitors in a CLL Murine Model (Abstract Number: 3009)

This poster presentation included preclinical data describing the differential regulation of human T cells by TGR-1202 in a preclinical CLL murine model.

Highlights from this poster include:

● TGR-1202 oral treatment induced less incidence of toxicity in CLL mice compared to other PI3K delta inhibitors
● TGR-1202 relatively preserved Treg quantity and function in a dose dependent manner compared to other PI3K delta inhibitors in normal and murine CLL T cells
● Inhibition of casein-kinase 1 epsilon (CK1e) by TGR-1202 may explain the relative preservation of Treg cells in these in-vivo models

Umbralisib/TGR-1202 As a Novel Dual PI3K/CK1 Inhibitor Has a Unique Therapeutic Role in Silencing Oncogenes in Aggressive Lymphomas (Abstract Number 2809)

This poster presentation expanded on existing preclinical data demonstrating that TGR-1202 is synergistic with carfilzomib in certain aggressive lymphoma cell lines.

Highlights from this poster include:

● TGR-1202 is highly synergistic with the proteasome inhibitor carfilzomib in cell line models of double hit lymphoma and mantle cell lymphoma
● Based on this preclinical work, a Phase 1 clinical study to evaluate the safety and efficacy of TGR-1202 in combination with carfilzomib is currently enrolling patients

PI3K Delta Inhibitors Induce Primary Monocyte Cytotoxicity but Do Not Alter Monocyte Differentiation (Abstract Number 4284)

This poster presentation included preclinical data exploring the effect of PI3K delta inhibitors on monocyte activity.

Highlights from this poster include:

● The clinical benefit and initial lymphocytosis seen with PI3K delta inhibitors in CLL may be related in part to direct effects on monocyte derived cells
● Idelalisib and TGR-1202 differed in the extent of monocyte cytotoxicity induced and inhibition of pAKT
● The direct effects of PI3K delta inhibitors on monocytes suggests these drugs may have efficacy beyond B-cell malignancies, including in monocytic neoplasms or other malignancies with monocyte derived cells in the tumor microenvironment

The above referenced presentations, are available on the Publications page of the Company’s website at www.tgtherapeutics.com.

On December 11, 2017 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported data highlighting results from three preclinical studies of its controllable switch technology for T cell immunotherapies at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) in Atlanta, Georgia (Press release, Bellicum Pharmaceuticals, DEC 11, 2017, View Source;p=RssLanding&cat=news&id=2322102 [SID1234522549]).

"These data continue to support our excitement over the technology’s potential to make cell therapies safer and more effective in more tumor types," said Rick Fair, Bellicum’s President & Chief Executive Officer. "We are currently validating our platform in the clinic in three different product candidates, and look forward to reporting results on these programs in 2018. With the most advanced controllable cell technologies in our industry, we believe we are well positioned to move additional preclinical CAR-T projects into clinical trials that have the potential to be best-in-class."

The Company’s novel technology platform is designed to enable full control over the activation, persistence, and elimination of cell therapies to safely elicit the full effect of CAR-T and TCR activity in the body. Unlike traditional approaches, Bellicum’s controllable CAR-T and TCR constructs are designed to provide anti-tumor surveillance, even in the absence of cancer antigen. The switch technologies covered in the posters include:

Technology Description
GoCAR-T CAR-T cells incorporated with the inducible MyD88/CD40 (iMC) costimulatory switch to provide ligand-regulated control over the activation and persistence of cells
CIDeCAR CAR-T construct that includes the MC costimulatory domain with the CaspaCIDe safety switch
Dual-Switch CAR-T CAR-T cells with both the iMC costimulatory switch and CaspaCIDe safety switch to provide greater control over the activation and persistence of therapeutic cells, as well as the ability to rapidly eliminate them by activating the safety switch
8Summary of Study Results

"Dual-Switch CAR-T cells: Orthogonal Molecular Switches to Control Activation and Elimination of CAR-T Cells to Target CD123+ Cancer" (Abstract 3184)

Researchers targeted CD123—which is highly expressed in acute myeloid leukemia (AML) and leukemic stem cells—with a novel construct consisting of a first-generation CAR combined with regulated activation and apoptotic signaling elements. T cell costimulation was controlled by rimiducid, and a rapamycin-controlled pro-apoptotic safety switch was designed to induce rapid dimerization of caspase-9 to mitigate possible CAR-T cell toxicity. Results demonstrate that when combined with a first-generation CD123-specific CAR, these molecular switches enable controlled, robust expansion of engineered T cells to control tumor growth in vitro and in vivo, and provide a rapid and efficient safety mechanism to block excessive cytokine release.

"Inducible MyD88/CD40 (iMC) Costimulation Enhances Polyclonal Epstein-Barr Virus-Specific Cytotoxic T Lymphocyte (EBV-CTL) Proliferation and Anti-Tumor Activity" (Abstract 3337)

Using peripheral blood mononuclear cells from healthy donors, researchers generated EBV-specific T cells, which were genetically modified with iMC. They concluded that modifying EBV-CTL with iMC resulted in increased T cell proliferation and persistence and improved anti-tumor efficacy, suggesting that iMC may have broad applications, such as modifying tumor-infiltrating lymphocytes, virus-specific T cells and other polyclonal T cell products to increase their potency.

"MyD88/CD40 enhanced CD19-specific CAR-T cells maintain therapeutic efficacy following resolution of cytokine-related toxicity using inducible caspase-9" (Abstract 4615)

Scientists demonstrated that CD19-specific CAR-T cells modified with a constitutively active form of the potent fusion protein MC were effective at eliminating aggressive tumors, with efficacy associated with cytotoxic cytokine release. However, this toxicity was effectively resolved with rimiducid-mediated activation of co-expressed iC9 or by selecting distinct T cell populations without affecting long-term efficacy of the CAR-T treatment.

The presentations can be found in the Investors & Media section of the Company’s website.

On December 11, 2017 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") reported the data of the investigational agent gilteritinib from the ongoing, open-label, dose escalation/expansion Phase 1 study (NCT02236013) in newly diagnosed patients with acute myeloid leukemia (AML) (Press release, Astellas, DEC 11, 2017, View Source [SID1234522548]). The data are being presented today in an oral presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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"These initial data shed encouraging light on the safety and tolerability of gilteritinib when combined with intensive chemotherapy for newly diagnosed AML patients," said Keith W. Pratz, M.D., of John Hopkins Sidney Kimmel Comprehensive Cancer Center, who is the principal investigator for the study. "In addition, while evaluating antitumor effects is an exploratory goal, the response rates in FLT3mut+ patients are promising and warrant expanded investigation of gilteritinib in this upfront treatment setting. Continuing research to evaluate the potential role for a FLT3 inhibitor in newly diagnosed patients and other stages of AML should continue to be a priority in our collective efforts to improve outcomes for patients."

The primary objective of this Phase 1 study is to assess the safety/tolerability profile, including dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD), of gilteritinib when combined with 7+3 induction (cytarabine and idarubicin) and high-dose cytarabine (HiDAC) consolidation chemotherapy, followed by single agent maintenance therapy in patients 18 years of age and older who have been newly diagnosed with AML. Assessment of antitumor effects of this combination therapy is an exploratory objective.

The two-part trial first enrolled patients to successive cohorts to determine the MTD. Successive cohorts received gilteritinib doses of 40, 80 or 120 mg/day. Dose escalation decisions were made based on DLTs that occurred following the first dose of gilteritinib during induction. Patients in the dose expansion cohort received gilteritinib at the recommended expansion dose established during dose escalation. Patients also received gilteritinib during consolidation, and then received maintenance therapy with once-daily gilteritinib over a 28-day cycle for up to 26 cycles.

"We are very encouraged by this initial data from our ongoing study of gilteritinib in combination with intensive chemotherapy in newly diagnosed AML patients, and pleased that it earned selection for oral presentation at ASH (Free ASH Whitepaper)," said Steven Benner, M.D., senior vice president and global therapeutic area head, Oncology Development, Astellas. "Mutations of FLT3 in AML are associated with a poor prognosis across the course of disease treatment and, through our comprehensive clinical development program, Astellas is committed to understanding how selective inhibition by gilteritinib might be beneficial to as many patients as possible."

As of July 9, 2017, 50 patients (n=17, dose escalation cohort; n=33, dose expansion cohort) had been enrolled in this ongoing study and 49 had received at least one dose of gilteritinib. Of the 48 patients with documented FLT3 mutation status, 23 (47.9%) were FLT3mut+, of whom 13 (56.5%) had internal tandem duplications (ITD).

Additional key findings include:

During dose escalation, two subjects in the 40 mg/day cohort who had received gilteritinib on days 1-14 experienced DLTs (neutropenia, thrombocytopenia and decreased ejection fraction). After gilteritinib induction schedule modification, no additional DLTs were observed.
The maximum tolerated dose was not reached; gilteritinib 120 mg/day was chosen as the recommended expansion dose.
Grade ≥ 3 treatment-emergent adverse events (TEAEs) occurring in ≥ 10% of subjects were febrile neutropenia (36.7%), thrombocytopenia (18.4%), neutropenia (16.3%) and decreased platelet count (12.2%).
Serious drug-related TEAEs occurring in >1 subject were febrile neutropenia (n=8), sepsis (n=2), small intestinal obstruction (n=2), lung infection (n=2), and decreased ejection fraction (n=2).
In FLT3mut+ and FLT3 wild type subjects, end-of-treatment CRc rates were 100% and 60.9%, respectively.
About Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is a cancer that impacts the blood and bone marrow, and its incidence increases with age. The American Cancer Society estimates that in 2017, approximately 21,000 new patients will be diagnosed with AML in the United States and about 10,000 cases will result in death.

About Gilteritinib

Gilteritinib is an investigational compound that has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) as well as FLT3 tyrosine kinase domain (TKD), two common types of FLT3 mutations that are seen in approximately one-third of patients with AML. Further, gilteritinib has also demonstrated inhibition of the AXL receptor in AML cell lines, which has been reported to be associated with therapeutic resistance. Astellas is currently investigating gilteritinib in various AML patient populations through several additional Phase 3 trials. Visit d to learn more about ongoing gilteritinib clinical trials.

Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and potentially commercialize gilteritinib. Gilteritinib has been granted Orphan Drug designation and Fast Track designation by the U.S. FDA, and SAKIGAKE designation by the Japan Ministry of Health, Labor and Welfare.

The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for the uses being investigated. Information about pharmaceutical products (including products currently in development), which is included in this press release are not intended to constitute an advertisement or medical advice.

On December 11, 2017 The OHSU Knight Cancer Institute and Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported the presentation of preclinical data demonstrating that CG’806, a pan-FLT3/pan-BTK inhibitor, has broad and potent drug activity against acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL) and other hematologic disease subtypes (Press release, Aptose Biosciences, DEC 11, 2017, View Source [SID1234522544]). The data were highlighted in a poster presentation on Monday, December 11, 2017 at the American Society of Hematology (ASH) (Free ASH Whitepaper) 59th Annual Meeting & Exposition, being held December 9-12 in Atlanta, GA.

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The poster CG’806, a First-in-Class Pan-FLT3/BTK Inhibitor, Exhibits Potent Growth Inhibition as a Single Agent and in Combination with a BET Bromodomain Inhibitor and a Bcl2 Inhibitor Against AML and CLL Patient Samples, evaluated the activity of CG’806 on various hematologic malignancy cell lines and patient primary bone marrow specimens through the Beat AML Initiative. CG’806 exhibited broad and potent activity against primary patient samples over a diverse range of hematologic malignancy subtypes, including AML, CLL, myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN), and acute lymphoblastic leukemia (ALL).

The poster presentation can be accessed on the Events & Presentations section of the Aptose website at the following link.

"In patient samples and cultured cell lines, CG’806 demonstrated potent and broad inhibitory activity against hematologic malignancies alone and in combination," said Stephen E. Kurtz, Ph.D., lead author and Research Assistant Professor at the OHSU Knight Cancer Institute. "Both AML and CLL are in urgent need of effective therapies that provide more durable clinical responses. CG’806 represents a potential new treatment approach that warrants further development."

OHSU researchers used an ex vivo drug sensitivity assay to determine the activity of CG’806 as a single agent and in combination with the BET bromodomain inhibitor OTX-015 or the Bcl2 inhibitor venetoclax on freshly isolated primary patient samples. Across the four general subtypes of hematologic malignancies in the dataset with patient samples, there was broad sensitivity to CG’806, with 55% (90/164) AML, 48% (46/96) CLL, 22% (6/27) ALL, and 53% (14/26) MDS/MPN cases exhibiting an IC50 < 100nM. CG’806 demonstrated median IC50 values of 70nM and 140nM against primary AML and CLL cells, respectively. CG’806 also exerted potent picomolar to low-nanomolar IC50 anti-proliferative activity against human AML, B-ALL, mantle-cell lymphoma, Burkitt’s lymphoma, and diffuse large B-cell lymphoma cell lines. CG’806 in combination with OTX-015 demonstrated median IC50 values of 20nM and 40nM against primary AML and CLL cells, respectively. CG’806 in combination with venetoclax demonstrated median IC50 values of 20nM and 10nM against primary AML and CLL cells, respectively.

"Collaborating with OHSU and the Beat AML initiative has provided us an exceptional opportunity to explore the activity of CG‘806 against a large and diverse set of freshly isolated patient bone marrow samples from patients with AML, CLL and other hematologic malignancies," commented William G. Rice, Ph.D., Chairman and Chief Executive Officer of Aptose. "CG’806 continues to reveal compelling preclinical results that are superior to other FLT3 or BTK inhibitors, and we are eagerly preparing CG’806 for clinical studies and look forward to an IND submission in 2018."

Separately, Aptose and The University of Texas MD Anderson Cancer Center researchers also presented new data on CG’806 at ASH (Free ASH Whitepaper) (see press release here).

In addition to the abstracts that were presented at ASH (Free ASH Whitepaper), two additional abstracts on CG’806 and two abstracts on APTO-253, Aptose’s small molecule c-Myc Inhibitor, have been published on the ASH (Free ASH Whitepaper) abstracts site. All abstracts will become part of the permanent ASH (Free ASH Whitepaper) and Blood abstracts archive.

For more information on Beat AML refer to View Source

About CG’806

CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK inhibitor. This small molecule demonstrates potent inhibition of all wild type and mutant forms of FLT3 tested (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates AML tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with FLT3-driven AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinases operative in B cell malignancies, suggesting CG’806 may be developed for CLL and MCL patients that are resistant/refractory/intolerant to covalent BTK inhibitors.

On December 11, 2017 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported the presentation of preclinical data from research led by The University of Texas MD Anderson Cancer Center demonstrating that CG’806, a highly potent pan-FLT3/pan-BTK inhibitor, exerts a profound anti-leukemia effect in human and murine leukemia cell lines harboring FLT-3 ITD mutations, mutations that are usually associated with very poor prognoses in leukemia patients. In addition, CG’806 demonstrates apoptosis, or programmed cell death, in AML patient samples by several mechanisms and is able to overcome resistance that is seen with other FLT3 inhibitors (Press release, Aptose Biosciences, DEC 11, 2017, View Source [SID1234522545]). The data were highlighted in poster presentations on Sunday and Monday, December 10 and 11, 2017 at the American Society of Hematology (ASH) (Free ASH Whitepaper) 59th Annual Meeting & Exposition, being held December 9-12 in Atlanta, GA.

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The poster The Pan-FLT3/BTK Multi-Kinase Inhibitor CG’806 Induces AML Killing in FLT-Mutant and Wild Type Cells, and Exerts Synergistic Pro-Apoptotic Effects with Concomitant Targeting of Anti-Apoptotic Bcl-2 and/or Mcl-1 demonstrated pronounced anti-leukemia activity of CG’806 against a broad array of AML cells, including those with FLT3-wild type, FLT3 with single mutations, or with FLT3 harboring dual ITD plus D835 or ITD plus F691 mutations, and it demonstrated synergistic effects in combination with Bcl-2 or Mcl-1 inhibitors even in FLT3 mutated AML cells. CG’806 elicited its broad spectrum killing of AML cells through its ability to suppress the FLT3 pathway as well the BTK, AURK, AKT and ERK signaling pathways that are differentially operative in different AML cells. Notably, CG’806 maintained cytotoxic activity against AML cells in the presence of FLT3 ligand and bone marrow stromal cells, and CG’806 demonstrated dose-dependent in vivo antitumor activity in a circulating AML murine model.

The poster CG’806, a Novel Pan-FLT3/BTK Multi-Kinase Inhibitor, Induces Cell Cycle Arrest, Apoptosis or Autophagy in AML Cells Depending on FLT3 Mutation Status further elucidated the anti-leukemia effect of CG’806. CG’806 exerted profound suppression of cell proliferation through G1 cell cycle arrest and induction of apoptosis in FLT3-mutant AML cells, which is associated with inhibition of mutant FLT3 signaling and the downstream p-AKT/p-mTOR/cyclin D1/p-Rb signaling axis. In contrast, CG’806 exerted a G2/M arrest in FLT3-wildtype (WT) cells through inhibition of aurora (AURK) and BTK kinases and induction of non-apoptotic cell death (autophagy or polyploidy). CG’806 sensitized AML cells to standard chemotherapeutic agents cytarabine and idarubicin and significantly enhanced pro-apoptotic effects. Taken together, these data support the development of CG’806 for a diverse set of AML patients with FLT3-ITD, FLT3-ITD plus additional TKD/gatekeeper mutations, as well as FLT3-WT.

Data were presented by members of the research team led by Michael Andreeff, M.D., Ph.D., Professor of Medicine, Haas Chair in Genetics, Department of Leukemia, at The University of Texas MD Anderson Cancer Center.

The poster presentations can be accessed on the Events & Presentations section of the Aptose website at the following link.

"As our mechanistic understanding of CG’806 grows, we are beginning to construct a framework of how a single molecule can inhibit specific clusters of kinases and kill a heterogeneous group of AML cells without observed toxicity to normal cells," commented William G. Rice, Ph.D., Chairman and Chief Executive Officer of Aptose. "As a pan-FLT3/pan-BTK multi-kinase inhibitor, CG’806 has the ability to kill a broad range of AML cells through inhibition of multiple oncogenic pathways that are differentially expressed in subgroups of cells. It appears to overcome the limitations of competitive FLT3 inhibitory agents, to enhance the AML cell killing effects of certain other chemotherapies, and to exhibit a robust therapeutic index. We look forward to initiating clinical trials of CG’806 in 2018."

Separately, Aptose and Oregon Health & Science University Knight Cancer Center researchers also announced new data on CG’806 presented at ASH (Free ASH Whitepaper) (see press release here).

In addition to the abstracts that were presented at ASH (Free ASH Whitepaper), two additional abstracts on CG’806 and two abstracts on APTO-253, Aptose’s small molecule c-Myc Inhibitor, have been published on the ASH (Free ASH Whitepaper) abstracts site. All abstracts will become part of the permanent ASH (Free ASH Whitepaper) and Blood abstracts archive.

About CG’806

CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK inhibitor. This small molecule demonstrates potent inhibition of all wild type and mutant forms of FLT3 tested (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates AML tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with FLT3-driven AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinases operative in B cell malignancies, suggesting CG’806 may be developed for CLL and MCL patients that are resistant/refractory/intolerant to covalent BTK inhibitors.