On December 3, 2015 Kura Oncology, Inc. (NASDAQ:KURA), a clinical stage biopharmaceutical company, reported that new data from a Phase 3 trial of tipifarnib, the company’s lead product candidate, will be presented at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in Orlando, December 5-8, 2015 (Press release, Kura Oncology, DEC 3, 2015, View Source;p=RssLanding&cat=news&id=2119854 [SID:1234508388]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The accepted abstract is listed below and is available online on the ASH (Free ASH Whitepaper) conference website: View Source

Tipifarnib As Maintenance Therapy in Acute Myeloid Leukemia (AML) Improves Survival in a Subgroup of Patients with High Risk Disease. Results of the Phase 3 Intergroup Trial E2902 (abstract # 1308)
Date & Time: Saturday, December 5, 2015 at 5:30 p.m. ET
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I

Location: Hall A, Level 2 (Orange County Convention Center)

The abstract describes results from the ECOG-ACRIN Cancer Research Group’s (ECOG-ACRIN) Phase 3 trial E2902, which compared tipifarnib as maintenance therapy to observation only with respect to disease-free survival in patients with AML in second or subsequent complete remission, in complete remission following primary induction failure, or patients 60 years of age or older in first complete remission. See www.clinicaltrials.gov using identifier NCT00093470.

Researchers in these groups designed and conducted trial E2902 independent of Kura Oncology and with sole sponsorship from the National Cancer Institute, part of the National Institutes of Health. Between August 2004 and December 2009, 144 patients with AML were enrolled in the study at multiple medical facilities (sites) across the United States, as well as one site in Israel and one site in Peru, through ECOG-ACRIN, the Alliance for Clinical Trials in Oncology and SWOG.

The median age of patients was 69 years (range 28-86), 73 patients (51%) were male and 135 patients (94%) were white. Ninety-one percent of patients were 60 years of age or older. A majority of patients enrolled on the study (70%) were in first complete remission (CR). Eighty patients (56%) had post remission chemotherapy prior to randomization. A total of 73 patients were enrolled onto the treatment arm (Arm A), while 71 patients were enrolled onto the observation arm (Arm B).

The primary endpoint of the study was an analysis of disease-free survival (DFS) on an-intent-to-treat basis. The median DFS for arms A and B was 8.87 months and 5.26 months respectively (p=0.058, HR 0.760). When restricted to eligible patients only (n=134), the DFS for arms A and B was 10.81 versus 5.26 months, respectively (p=0.019, HR 0.690). An unplanned subgroup analysis was performed by gender. The median DFS for male patients (n=73) was 5.36 versus 5.91 months for arms A and B respectively (p=0.868, HR 1.320) and 12.09 versus 3.91 months for female patients (n=71) (p=0.0002, HR=0.408). Hematological toxicity was seen in both arms but was less frequent in the observation arm.

While the primary endpoint for improved disease-free survival was not reached when evaluating all randomized patients, when restricted to eligible patients only, a statistically significant improvement in DFS was observed. Moreover, an unplanned subgroup analysis by gender demonstrated an improvement in DFS and overall survival (OS) for females who were enrolled on the study, which persists on multivariate analysis. Possible explanations for the gender differences in DFS and OS include the use of flat dosing rather than body-surface area (BSA) based dosing. Given what appears to be a potentially clinically relevant benefit, the authors concluded that further evaluation of this agent is warranted.

"The results are exciting and suggest that tipifarnib, when administered in this fashion, provides a survival benefit to a significant group of patients with AML who otherwise have a high risk of relapse," said lead investigator Selina M. Luger, MD from the University of Pennsylvania. "Further studies should be done to better understand which patients can benefit or if alternate dosing would allow this benefit to extend to a larger population."

"We are encouraged by the outcomes of this study," said Antonio Gualberto, M.D., Ph.D., Chief Medical Officer of Kura Oncology. "We are in discussions with the study investigators and other key opinion leaders to determine the appropriate next steps for development of tipifarnib in this and other indications."

The ECOG-ACRIN Cancer Research Group is a membership-based scientific organization that designs and conducts cancer research involving adults who have or are at risk of developing cancer. Its research is supported primarily by federal funding through the National Cancer Institute. www.ecog-acrin.org.

On December 3, 2015 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that its collaborator, Moffitt Cancer Center, will present preclinical data for its dual-targeting bromodomain (BRD) / kinase inhibitor, MA2-014, at the 57th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition being held December 5-8, 2015, in Orlando, FL (Press release, Aptose Biosciences, DEC 3, 2015, View Source;p=RssLanding&cat=news&id=2119892 [SID:1234508437]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The dual-targeting inhibitor MA2-014 is a representative candidate from a new family of small molecule, dual-targeting BRD / kinase inhibitors licensed to Aptose from Moffitt Cancer Center. The MA2-014 program was developed to inhibit both the bromodomain 4 (BRD4) protein and the Janus kinase 2 (JAK2) for the potential treatment of various hematologic and solid tumor cancers. The data being presented by Moffitt researchers reflect in vitro activity of MA2-014 in myeloproliferative neoplasm (MPN) cell lines. MPNs are rare blood cancers including polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF).

Clinically, JAK2 inhibitors alone have demonstrated the ability to improve symptoms of MPNs, but have not been shown to induce remission. Further, combining a bromodomain inhibitor and a JAK2 inhibitor has been shown to be more effective against MPN cells than JAK2 inhibition alone. This provided rationale to develop a single molecule with potent activity against both the bromodomain family proteins and JAK2. Moffitt researchers will present data for MA2-014, a single molecule that exhibits similar anti-JAK2 activity as a known JAK2 inhibitor, TG101209, with an approximate ten-fold improvement in anti-BRD activity. Moffitt researchers also demonstrated a ten-fold improvement in the ability of MA2-014 to inhibit JAK2-V617F signaling over TG101209, and comparable to ruxolitinib. Ruxolitinib is the only FDA approved JAK inhibitor for MPNs. However, MA2-014 retained its potency against ruxolitinib-resistant cells. Moffitt researchers also determined in long-term culture assays that JAK2-V617F driven MPN Uke1 cells do not experience resistance to MA2-014 as readily as they do to TG101209 or ruxolitinib.

"We are excited to continue development of MA2-014 and other candidates in our collaboration with Moffitt and to advance highly differentiated dual-targeting BRD / kinase inhibitors for the treatment of rare blood cancers into the clinic," said William G. Rice, Ph.D., Chairman, President and CEO. "We share Moffitt’s optimism about the potential for dual-targeting BRD / kinase inhibitors as high-value epigenetic drug candidates to provide the benefits of combination therapy for hematologic cancers."

"The development and optimization of a single drug designed to simultaneously inhibit JAK2 kinase and bromodomains of the bromodomain and extraterminal proteins may improve therapy for myeloproliferative neoplasms (MPN), by delivering a potentially effective combination therapy with a single agent," said Gary Reuther, Associate Member of the Cancer Biology and Evolution Program at Moffitt. "Such dual inhibition of these targets and their respective pathways may provide a new effective MPN therapy and reduce the development of drug resistance seen with JAK2 inhibitors."

Abstract Details

Title: "Single Molecule Dual JAK2-BET Inhibition as an MPN Therapeutic"
* Date/Time: Sunday, December 6, 2015, 6:00 – 8:00 p.m.
* Location: Orange County Convention Center, Hall A, Level 2
* Abstract #: 2826
* Session: 635. Myeloproliferative Syndromes: Basic Science: Poster II

The abstract is available in the online edition of Blood, the official Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper) and on the ASH (Free ASH Whitepaper) conference website.

Aptose previously announced additional ASH (Free ASH Whitepaper) abstracts on its lead clinical candidate APTO-253:

Title: "Broad Activity of APTO-253 in AML and Other Hematologic Malignancies Correlates with KLF4 Expression Level"
* Date/Time: Saturday, December 5, 2015, 5:30-7:30 p.m.
* Location: Orange County Convention Center, Hall A
* Abstract #: 83676
* Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I

Title: "Clinical Pharmacokinetics of APTO-253 Support its Use as a Novel Agent for the Treatment of Relapsed or Refractory Hematologic Malignancies"
* Abstract #: 4934
* Location: Publication

Strategic Collaboration with Moffitt Cancer Center

Aptose recently entered into a definitive agreement with Moffitt Cancer Center for exclusive global rights to potent, multi-targeting, single-agent inhibitors for the treatment of hematologic and solid tumor cancers. These small molecule agents are highly differentiated inhibitors of the Bromodomain and Extra-Terminal motif (BET) protein family members, which simultaneously target specific kinase enzymes. The molecules developed by Moffitt exhibit single-digit nanomolar potency against the BET family members and specific oncogenic kinases which, when inhibited, are synergistic with BET inhibition. Under the agreement, Aptose gains access to the drug candidates developed by Moffitt and the underlying intellectual property covering the chemical modifications enabling potent bromodomain (BRD) inhibition on the chemical backbone of a kinase inhibitor. Aptose expects lead clinical candidates to emerge from the collaboration by late 2016.

On December 3, 2015 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that its collaborator, Moffitt Cancer Center, will present preclinical data for its dual-targeting bromodomain (BRD) / kinase inhibitor, MA2-014, at the 57th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition being held December 5-8, 2015, in Orlando, FL (Filing, 6-K, Aptose Biosciences, DEC 3, 2015, View Source [SID:1234508389]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The dual-targeting inhibitor MA2-014 is a representative candidate from a new family of small molecule, dual-targeting BRD / kinase inhibitors licensed to Aptose from Moffitt Cancer Center. The MA2-014 program was developed to inhibit both the bromodomain 4 (BRD4) protein and the Janus kinase 2 (JAK2) for the potential treatment of various hematologic and solid tumor cancers. The data being presented by Moffitt researchers reflect in vitro activity of MA2-014 in myeloproliferative neoplasm (MPN) cell lines. MPNs are rare blood cancers including polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF).

Clinically, JAK2 inhibitors alone have demonstrated the ability to improve symptoms of MPNs, but have not been shown to induce remission. Further, combining a bromodomain inhibitor and a JAK2 inhibitor has been shown to be more effective against MPN cells than JAK2 inhibition alone. This provided rationale to develop a single molecule with potent activity against both the bromodomain family proteins and JAK2. Moffitt researchers will present data for MA2-014, a single molecule that exhibits similar anti-JAK2 activity as a known JAK2 inhibitor, TG101209, with an approximate ten-fold improvement in anti-BRD activity. Moffitt researchers also demonstrated a ten-fold improvement in the ability of MA2-014 to inhibit JAK2-V617F signaling over TG101209, and comparable to ruxolitinib. Ruxolitinib is the only FDA approved JAK inhibitor for MPNs. However, MA2-014 retained its potency against ruxolitinib-resistant cells. Moffitt researchers also determined in long-term culture assays that JAK2-V617F driven MPN Uke1 cells do not experience resistance to MA2-014 as readily as they do to TG101209 or ruxolitinib.

"We are excited to continue development of MA2-014 and other candidates in our collaboration with Moffitt and to advance highly differentiated dual-targeting BRD / kinase inhibitors for the treatment of rare blood cancers into the clinic," said William G. Rice, Ph.D., Chairman, President and CEO. "We share Moffitt’s optimism about the potential for dual-targeting BRD / kinase inhibitors as high-value epigenetic drug candidates to provide the benefits of combination therapy for hematologic cancers."

"The development and optimization of a single drug designed to simultaneously inhibit JAK2 kinase and bromodomains of the bromodomain and extraterminal proteins may improve therapy for myeloproliferative neoplasms (MPN), by delivering a potentially effective combination therapy with a single agent," said Gary Reuther, Associate Member of the Cancer Biology and Evolution Program at Moffitt. "Such dual inhibition of these targets and their respective pathways may provide a new effective MPN therapy and reduce the development of drug resistance seen with JAK2 inhibitors."

Abstract Details

Title: "Single Molecule Dual JAK2-BET Inhibition as an MPN Therapeutic"
* Date/Time: Sunday, December 6, 2015, 6:00 – 8:00 p.m.
* Location: Orange County Convention Center, Hall A, Level 2
* Abstract #: 2826
* Session: 635. Myeloproliferative Syndromes: Basic Science: Poster II

The abstract is available in the online edition of Blood, the official Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper) and on the ASH (Free ASH Whitepaper) conference website.

Aptose previously announced additional ASH (Free ASH Whitepaper) abstracts on its lead clinical candidate APTO-253:

Title: "Broad Activity of APTO-253 in AML and Other Hematologic Malignancies Correlates with KLF4 Expression Level"
* Date/Time: Saturday, December 5, 2015, 5:30-7:30 p.m.
* Location: Orange County Convention Center, Hall A
* Abstract #: 83676
* Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I

Title: "Clinical Pharmacokinetics of APTO-253 Support its Use as a Novel Agent for the Treatment of Relapsed or Refractory Hematologic Malignancies"
* Abstract #: 4934
* Location: Publication

Strategic Collaboration with Moffitt Cancer Center

Aptose recently entered into a definitive agreement with Moffitt Cancer Center for exclusive global rights to potent, multi-targeting, single-agent inhibitors for the treatment of hematologic and solid tumor cancers. These small molecule agents are highly differentiated inhibitors of the Bromodomain and Extra-Terminal motif (BET) protein family members, which simultaneously target specific kinase enzymes. The molecules developed by Moffitt exhibit single-digit nanomolar potency against the BET family members and specific oncogenic kinases which, when inhibited, are synergistic with BET inhibition. Under the agreement, Aptose gains access to the drug candidates developed by Moffitt and the underlying intellectual property covering the chemical modifications enabling potent bromodomain (BRD) inhibition on the chemical backbone of a kinase inhibitor. Aptose expects lead clinical candidates to emerge from the collaboration by late 2016.

On December 3, 2015 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported that Daniel Levitt, MD, PhD, Executive Vice President and Chief Medical Officer, will present a corporate update at the 26th Annual Oppenheimer Healthcare Conference on Tuesday, December 8th at 3:55p.m. Eastern Time (Press release, CytRx, DEC 3, 2015, View Source;p=RssLanding&cat=news&id=2119926 [SID:1234508391]). The conference will take place at the Westin Grand Central Hotel in New York, NY.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live and archived webcast of the presentation will be available on the Company’s website at www.cytrx.com/investors/presentations.

About Soft Tissue Sarcoma

Soft tissue sarcoma is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, and can arise anywhere in the body at any age. According to the American Cancer Society, there are approximately 50 types of soft tissue sarcomas. In 2013 more than 11,400 new cases were diagnosed in the U.S. and approximately 4,400 Americans died from this disease. In addition, approximately 40,000 new cases and 13,000 deaths in the U.S. and Europe are part of a growing underserved market.

About SCLC

An estimated 1.6 million new cases of lung cancer are diagnosed worldwide each year. In the Western world, approximately 13-15% of cases are SCLC, a deadly form of lung cancer associated with tobacco use. The five year survival rate is less than 7%, in part because an estimated 70% of patients have extensive disease at diagnosis. According to the National Cancer Institute, more than 30,000 new cases will be diagnosed in the USA in 2014. The estimated 2014 SCLC incidences for Europe and Asia are over 58,000 and 136,000, respectively.

About Glioblastoma Multiforme

Glioblastoma is the most common and most malignant primary brain tumor in adults and afflicts more than 12,000 new patients in the U.S. annually. The median survival after diagnosis is approximately 14 months, despite patients subsequently receiving surgical resection, radiotherapy and chemotherapy. Limited efficacy of chemotherapeutic agents has been attributed to several contributing factors including insufficient drug delivery to the tumor site through the blood / brain barrier.

About Aldoxorubicin

The widely used chemotherapeutic agent doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. This allows for greater doses (3 ½ to 4 times) of doxorubicin to be administered while reducing its toxic side effects. In studies thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2,000 mg/m2.

On December 3, 2015 Sunesis Pharmaceuticals, Inc. (NASDAQ:SNSS) reported that it has initiated a Compassionate Use Program for vosaroxin. The Compassionate Use Program will be made available to eligible patients in the U.S. and selected European countries diagnosed with relapsed or refractory acute myeloid leukemia (AML) and will be managed by Clinigen Group plc’s (AIM:CLIN) Idis Managed Access (MA) division (Press release, Sunesis, DEC 3, 2015, View Source;p=RssLanding&cat=news&id=2119850 [SID:1234508393]). Clinigen Group plc’s (AIM:CLIN) Idis Managed Access (MA) division.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Vosaroxin is an investigational treatment and is currently not approved for use by any regulatory agency. Compassionate use programs are put in place to provide access to medicines for patients who have serious, or immediately life-threatening illnesses, and for whom no alternative treatment options are available. Access is provided in response to an unsolicited request from a physician for his/her patient with an unmet medical need.

"Sunesis is committed to providing patients and healthcare providers around the globe with more options for treating relapsed and refractory AML, a disease for which the standard of care has changed little in the last four decades," said Par S Hyare, VP Global Oncology Operations, Sunesis. "We are pleased to be working with Idis MA, a recognized leader in providing ethical access to medicines that address unmet needs and will be working towards gaining approval for vosaroxin in the U.S. and Europe for the treatment of relapsed and refractory AML."

Simon Estcourt, Managing Director of Idis Managed Access, Clinigen Group said: "AML is the most common acute leukemia affecting adults, with a very low survival rate, so there is a real need for new treatment options in these patients. By using our global logistical and regulatory expertise we will work with Sunesis and the AML community to provide ethical access to vosaroxin to help eligible patients who have no alternative treatment options."

For more information about Idis’ services and its Managed Access Programs, healthcare professionals may contact Idis via telephone on +44 (0)1932 824 135, fax on +44 (0)1932 824 335. To contact Idis by e-mail, U.S. physicians only use [email protected]. Physicians from all other countries please use [email protected].

About vosaroxin
Vosaroxin is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed or refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.