On December 14, 2015 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported that its San Diego diagnostic laboratory has been accredited by the College of American Pathologists (CAP) based on results of a recent on-site inspection (Press release, Ignyta, DEC 14, 2015, View Source [SID:1234508568]). This accreditation is awarded to facilities that meet the highest standards of quality in clinical laboratory services. With this recognition, Ignyta’s diagnostic laboratory joins the ranks of some of the most prominent clinical laboratories in the world.

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"We are extremely proud that our in-house diagnostic laboratory has achieved CAP accreditation, further validating our commitment to provide the highest quality laboratory services as part of our focus on helping healthcare providers take better care of patients suffering from cancer," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "This milestone is consistent with our vision of becoming the world’s leading precision medicine company, with an integrated approach to ‘Rx/Dx’ in oncology. Furthermore, we believe our diagnostic laboratory provides us with a competitive advantage. For example, in our potentially-registration-enabling Phase 2 clinical trial of entrectinib, STARTRK-2, we are using our lab as the FDA-required central lab to confirm the results of local genetic patient screening for enrollment, as well as to perform central testing for clinical sites without the means for local genetic screening to identify patients with molecular alterations eligible for this clinical trial that would otherwise not have been identified."

The U.S. Centers for Medicare & Medicaid Services (CMS) has approved CAP as an accreditation organization for clinical laboratories under CLIA (Clinical Laboratory Improvement Amendments). Ignyta had previously announced CLIA registration of its diagnostic laboratory, and CAP accreditation results in full CLIA certification for Ignyta’s diagnostic laboratory.

On December 14, 2015 Corcept Therapeutics Incorporated (NASDAQ: CORT), a pharmaceutical company engaged in the discovery, development and commercialization of drugs that treat severe metabolic, oncologic and psychiatric disorders by modulating activity at the glucocorticoid receptor (GR), reported initial efficacy data from its Phase 1/2 trial of mifepristone in combination with the chemotherapy drug eribulin to treat patients with GR-positive, metastatic, triple-negative breast cancer (TNBC) (Press release, Corcept Therapeutics, DEC 14, 2015, http://www.corcept.com/news_events/view/pr_1450104306 [SID:1234508565]). The data were presented at the 2015 San Antonio Breast Cancer Symposium (SABCS) on Saturday, December 12th, 2015.

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Trial Design and Objective

Corcept is investigating whether the addition of mifepristone, the active ingredient in Corcept’s approved medication, Korlym, will enhance the effect of eribulin in patients whose TNBC tumors express GR, one of the receptors to which Korlym binds.

The trial is being conducted in two parts. In the first phase, researchers determined the maximum tolerated combined dose of mifepristone and eribulin. This dose — 300 mg of mifepristone daily with 1.1 mg/m2 of eribulin taken on days one and eight of a three week cycle — is now being administered to patients in the trial’s second, efficacy phase, which will enroll 20 patients with GR-positive, metastatic TNBC. This phase of the trial is ongoing.

Initial Efficacy Results

At SABCS, the trial’s investigators presented efficacy data from the 15 patients enrolled to-date with GR-positive TNBC who have been treated with the recommended dose (two patients from the trial’s first phase and 13 from its second). As determined using the Response Evaluation Criteria in Solid Tumors (RECIST), initial efficacy results in this group were as follows: one patient exhibited a partial response (defined as a 30 percent or greater reduction in tumor size), seven had stable disease and five had progressive disease. Two patients were too early in their treatment to be assessed.

The combination of mifepristone and eribulin has been well-tolerated. Most adverse events have been disease-related and of mild or moderate severity, with the most common being neutropenia, fatigue, hypokalemia, nausea, hair loss and neuropathy. Neutropenia has been manageable with the administration of growth factor. No patient has suffered a serious adverse event. Pharmacokinetic data show no interaction between eribulin and mifepristone.

"We are pleased that the combination of mifepristone and eribulin appears to be active in these very ill patients," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer. "Our oncology program is based on the hypothesis that GR modulators such as mifepristone can enhance the efficacy of a variety of chemotherapeutic agents in GR-positive disease. We very much look forward to completing the trial. If the final results are sufficiently positive, we plan to initiate a Phase 3 trial of this treatment regimen next year."

Corcept’s trial builds on pre-clinical and clinical research performed by investigators at Corcept and at the University of Chicago showing that modulation of activity at GR enhances the effect of chemotherapy in the treatment of TNBC. At the 2013 SABC, University of Chicago researchers presented positive results of a trial in which six patients with metastatic, GR-positive TNBC received a combination of nab-paclitaxel and mifepristone

About Triple-Negative Breast Cancer

TNBC is a form of breast cancer in which the three receptors that fuel most breast cancer growth — estrogen, progesterone and HER-2 — are not present. Because the tumor cells lack these receptors, treatments that target estrogen, progesterone and HER-2 are ineffective. Approximately 40,000 women are diagnosed with triple-negative breast cancer each year. It is estimated that more than 75 percent of these women’s tumor cells express GR. There is no FDA-approved treatment and neither a targeted treatment nor an approved standard chemotherapy regimen for relapsed triple-negative breast cancer patients exists.

Corcept has licensed patents from the University of Chicago covering the use of GR antagonists in combination with chemotherapy to treat TNBC and castration-resistant prostate cancer.

About Korlym (mifepristone)

Korlym modulates the effects of excess cortisol in patients with Cushing’s syndrome. Since 2012, Corcept has marketed Korlym as a once-daily oral treatment of hyperglycemia secondary to endogenous Cushing’s syndrome in adult patients with glucose intolerance or diabetes mellitus type 2 who have failed surgery or are not candidates for surgery. Korlym was the first FDA-approved treatment for that illness, and the FDA has designated Korlym as an Orphan Drug for that indication.

On December 14, 2015 Celsion Corporation (NASDAQ: CLSN) reported the presentation of results from its ongoing Phase I/II US DIGNITY Study of ThermoDox in combination with mild hyperthermia in patients with recurrent chest wall (RCW) breast cancer (Press release, Celsion, DEC 14, 2015, View Source [SID:1234508564]). The data, which demonstrated a combined local response rate of 61.9% among evaluable patients treated with ThermoDox, were presented on Saturday, December 12, 2015 at the San Antonio Breast Cancer Symposium during a poster session titled New Drugs and Treatment Strategies. Celsion Corporation is a fully-integrated oncology company focused on the development of a portfolio of innovative cancer treatments, including directed chemotherapies, immunotherapies and RNA- or DNA-based therapies for the treatment of cancer and other difficult-to-treat diseases.

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"Results from this study are very encouraging and suggest that ThermoDox combined with superficial hyperthermia offers a promising and well tolerated treatment option for patients with recurrent chest wall disease from breast cancer, a highly refractory form of breast cancer associated with poor quality of life and limited treatment options," said Hope Rugo, M.D., Clinical Professor, Department of Medicine and Director, Breast Oncology Clinical Trials Program at the University of California, San Francisco, and lead investigator of the study. "These findings underscore the potential for this therapy to serve as a much needed treatment for these patients, and further define the importance of advancing development of ThermoDox in this indication," Dr. Rugo added.

In the Phase I/II trials, which were designed to evaluate the safety and anti-tumor activity of ThermoDox in combination with mild hyperthermia in RCW breast cancer, a total of 28 patients were treated at doses of either 40 or 50 mg/m2. In addition to a local response rate of 61.9% among evaluable patients, a combined local response rate was observed in 46.4% of the intent-to-treat population (13/28), notably consisting of five patients demonstrating a durable local response lasting greater than three months, including four complete responses (CR) and one partial response (PR). Patients dosed at 40 mg/m2 displayed a comparable response rate and a more favorable safety profile to that of patients receiving 50 mg/m2. As a result, 40 mg/m2 will be the recommended dose for future clinical trials in this indication.

"ThermoDox in RCW breast cancer continues to yield striking response data in this vulnerable patient population, further validating our commitment to expanding this program, including the initiation of the Euro-DIGNITY Trial, a multi-center study designed to evaluate ThermoDox’s potential to locally control chest wall lesions in earlier-stage patients," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "Additionally, we remain committed to providing patients who are suffering from this aggressive form of breast cancer with access to ThermoDox, and are continuing to work closely with myTomorrows to ensure the success of our Early Access Program in Europe for ThermoDox in RCW breast cancer."

The Company anticipates completion of the Phase II US DIGNITY trial by year-end, and plans to initiate a 70 patient Phase II study in Europe and Israel in less advanced, less heavily pretreated patients as part of the Euro-DIGNITY Trial. The Euro-DIGNITY Trial will evaluate ThermoDox plus radiation and hyperthermia in RCW breast cancer patients and is designed to support a registration filing in Europe. This study will be initiated throughout Europe and Israel and with assistance from MedLogics Corporation, an Italian-based hyperthermia device company. In addition, Celsion has a license and distribution agreement with myTomorrows to implement an Early Access Program (EAP) for ThermoDox in all countries of the European Union territory plus Switzerland for the treatment of patients with RCW breast cancer. The EAP provides physicians with access to products in later stage development demonstrating evidence of clinical benefit, with an acceptable safety profile and a quality manufacturing process in place.

The poster presentation is available on Celsion’s website at View Source

On December 14, 2015 BioLineRx Ltd. (NASDAQ/TASE:BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, reported the filing of regulatory submissions required to commence a Phase 2 trial for BL-8040 as a novel approach for mobilization and collection of bone marrow stem cells from the peripheral blood circulation (Press release, BioLineRx, DEC 14, 2015, View Source;p=RssLanding&cat=news&id=2122198 [SID:1234508562]). The trial is expected to commence shortly after receipt of regulatory approval, anticipated in the first quarter of 2016.

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The Phase 2 open-label study will be conducted as an investigator-initiated study in collaboration with Washington University School of Medicine, Division of Oncology and Hematology, and will enroll up to 24 donor/recipient pairs. The study submission was made following a meeting with the FDA in October 2015 to discuss the BL-8040 stem cell mobilization development program. The trial will evaluate the ability of BL-8040 to promote stem cell mobilization as a single agent in the allogeneic transplantation setting. Donors between 18 and 70 years of age and their HLA-matching recipients, diagnosed with advanced hematological malignancies that require stem cell transplantation, will be recruited to the study. On the donor side, the primary endpoint of the study is the ability of a single injection of BL-8040 to mobilize sufficient amounts of cells for transplantation following up to two leukapheresis collections. On the recipient side the study aims to evaluate the functionality and engraftment following transplantation of the BL-8040 collected graft.

The safety and tolerability of BL-8040 in the healthy donors will be evaluated. In addition, graft durability, the incidence of grade 2-4 acute graft versus host disease (GvHD), and other recipient related parameters will be evaluated in the patients who have undergone transplantation of hematopoietic cells mobilized with BL-8040.

Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx, stated, "Stem cell mobilization is used increasingly as a method of collecting hematopoietic stem cells for transplantation, forming part of the treatment regimen for certain types of hematological cancers, as well as for severe anemia or immune deficiency disorders. We have already completed a successful Phase 1 safety and efficacy study in healthy volunteers, supporting BL-8040 as one-day, single-dose collection regimen, with the capacity to rapidly mobilize substantial amounts of stem cells, representing a significant improvement upon the current standard of care. Following a productive meeting with the FDA regarding the development program for BL-8040 as a stem cell mobilizer for allogeneic transplantation, and in light of the fact that there are no approved drugs for stem cell mobilization to support allogeneic transplant, we are looking forward to shortly commencing yet another Phase 2 trial for our lead oncology platform."

"In parallel, BL-8040 is also undergoing a Phase 2 study for treating relapsed and refractory acute myeloid leukemia patients, and has recently initiated a Phase 2b study as an AML consolidation treatment and a Phase 1/2 study as a novel treatment for hMDS and AA, two bone marrow failure conditions. In addition, as we recently announced, we are also performing an extensive evaluation of BL-8040’s potential in the immuno-oncology space, as a combination treatment with immune checkpoint inhibitors," added Dr. Savitsky.

About BL-8040
BL-8040 is a clinical-stage drug candidate for the treatment of acute myeloid leukemia, as well as other hematological indications. It is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis (growth of new blood vessels in the tumor), metastasis (spread of the disease to other organs or organ parts) and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. BL-8040 is currently in the midst of a Phase 2 study for relapsed/refractory acute myeloid leukemia (AML) and has recently initiated a Phase 2b study as an AML consolidation treatment and a Phase 1/2 study in hMDS and AA,. In addition, in a Phase 1/2, open-label, dose escalation, safety and efficacy clinical trial in 18 multiple myeloma patients, BL-8040, when combined with G-CSF, demonstrated an excellent safety profile at all doses tested and was highly effective in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood. Additionally, in a Phase 1 stem-cell mobilization study in healthy volunteers, BL-8040 as a single agent was safe and well tolerated at all doses tested and resulted in efficient stem-cell mobilization and collection in all study participants. Importantly, the results of this study support the use of BL-8040 as one-day, single-dose collection regimen, which is a significant improvement upon the current standard of care.

BL-8040 effectively mobilizes cancer cells from the bone marrow and may therefore sensitize these cells to chemo- and bio-based anti-cancer therapy. Importantly, BL-8040 has also demonstrated a direct anti-cancer effect by inducing apoptosis. Pre-clinical studies show that BL-8040 inhibits the growth of various tumor types including multiple myeloma, non-Hodgkin’s lymphoma, leukemia, non-small cell lung carcinoma, neuroblastoma and melanoma. BL-8040 also significantly and preferentially stimulated apoptotic cell death of malignant cells (multiple myeloma, non-Hodgkin’s lymphoma and leukemia). Significant synergistic and/or additive tumor cell killing activity has been observed in vitro and in vivo when tumor cells were treated with BL-8040 together with Rituximab, Bortezomib, Imatinib, Cytarabine and the FLT-3 inhibitor AC-220 (in NHL, MM, CML, AML, and AML-FLT3-ITD models, respectively). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

On December 14, 2015 BIND Therapeutics, Inc. (NASDAQ:BIND), a clinical-stage nanomedicine company developing targeted and programmable therapeutics called ACCURINS, reported that the squamous histology non-small cell lung cancer (NSCLC) cohort of the phase 2 iNSITE 1 trial will advance to the second stage and complete enrollment to 40 patients (Press release, BIND Therapeutics, DEC 14, 2015, View Source [SID:1234508561]). The company also announced that the KRAS mutant NSCLC arm will not advance to the second stage. The rationale for these decisions is based on safety and efficacy data from the planned interim analysis of iNSITE 1 as well as updated overall survival (OS) data in the squamous cohort from the previous clinical trial in the broad NSCLC population, the BIND-014-005 trial.

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"The activity of BIND-014 as monotherapy in 2nd line NSCLC of squamous histology remains encouraging," said Hagop Youssoufian, M.D., M.Sc., chief medical officer, BIND Therapeutics. "There have been important advances in treatment strategies for NSCLC and we believe the evolving treatment landscape may benefit from chemotherapy in combination with checkpoint inhibitors. The safety profile of BIND-014, along with the potential to target disease sites with greater specificity, supports its development as a cytotoxic partner to checkpoint inhibitors and we will be exploring BIND-014 in this context."

As of October 29, 2015, both the squamous and KRAS cohorts had reached the predefined 20-patient enrollment mark for stage 1, triggering a planned evaluation of the data against pre-specified gating criteria for continuation to stage 2. The major criteria for advancing to the second stage included 6-week disease control rate (6wDCR), tolerability and, in the case of the squamous histology cohort, confirmation of the OS data from the BIND-014-005 trial.

In the squamous histology cohort, data from 20 patients in the intent-to-treat (ITT) population and 11 patients from the Per-Protocol (PP) subset demonstrated an interim 6wDCR of 25 percent (95% CI [confidence interval], 9% to 49%) and 45.5 percent (95% CI, 17% to 77%), respectively. There were no tumor responses by RECIST v1.1.

The final median OS in nine patients with squamous histology from the BIND-014-005 trial was 11.1 months, confirming the interim median OS reported previously, with a 1-year survival rate of 44 percent. These data compare favorably with currently approved treatments. As a reference, in the CheckMate 017 trial in 2nd line NSCLC of squamous histology, a median OS of 9.2 months and 6.0 months and 1-year survival rates of 42 percent and 24 percent were reported for Opdivo (nivolumab) and docetaxel, respectively.

In the KRAS mutant cohort, data from 23 patients in ITT population and 14 patients from the PP subset demonstrated an interim 6wDCR of 17.4 percent (95% CI, 5% to 39%) and 28.6 percent (95% CI, 8% to 58%) respectively, which did not meet pre-specified criteria to move to the second stage of the iNSITE 1 trial. The overall response rate (ORR) by RECIST v1.1 was 4 percent (ITT) and 7 percent (PP). Patients currently enrolled in this cohort will continue to be followed for safety and efficacy.

Safety data in more than 200 patients treated with BIND-014 to date continue to demonstrate meaningful improvements in hematologic and non-hematologic toxicities when compared to historical docetaxel data.

"Based on preliminary data from iNSITE 1 and confirmed median overall survival data from the 005 trial, we believe that BIND-014 may be ideally suited to broaden the impact of immuno oncology approaches in the treatment of solid tumors," said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. "Our next steps are to complete enrollment in the squamous cohort of iNSITE 1 in early 2016 and, in parallel, begin designing a trial in combination with a checkpoint inhibitor that we intend to pursue contingent upon final iNSITE 1 results, potentially through new collaborations with development partners."

Enrollment is ongoing in the phase 2 iNSITE 2 trial with BIND-014 in patients with advanced cholangiocarcinoma, bladder, cervical and head and neck cancers. Topline data for the second stage of iNSITE 2 are anticipated in the first half of 2016.

About BIND Therapeutics

BIND Therapeutics is a clinical-stage nanomedicine company developing a pipeline of ACCURINS, its novel targeted therapeutics designed to increase the concentration and duration of therapeutic payloads at disease sites while reducing exposure to healthy tissue. BIND is leveraging its Medicinal Nanoengineering platform to develop a pipeline of ACCURINS targeting hematological and solid tumors and has a number of strategic collaborations with biopharmaceutical companies to develop ACCURINS in areas of high unmet need. BIND’s lead drug candidate, BIND-014, is a prostate-specific membrane antigen (PSMA) -targeted ACCURIN that contains docetaxel, a clinically-validated and widely-used cancer chemotherapy drug. BIND is currently enrolling patients in a trial with BIND-014 for non-small cell lung cancer, or NSCLC, with squamous histology. In addition, BIND is enrolling patients in a clinical trial with BIND-014 for advanced cervical, bladder, head and neck and cholangio cancers. BIND is advancing BIND-510, its second PSMA-targeted ACCURIN drug candidate containing vincristine, a potent microtubule inhibitor with dose limiting peripheral neuropathy in its conventional form, through important preclinical studies to position it for an Investigational New Drug (IND) application filing with the U.S. Food and Drug Administration. BIND is also developing ACCURINS designed to inhibit PLK1 and KSP, both of which BIND believes are promising anti-mitotic targets that have been limited in the clinic due to systemic toxicity at or below therapeutic doses.

BIND has announced ongoing collaborations with Pfizer Inc., AstraZeneca AB, F. Hoffmann-La Roche Ltd., Merck & Co., or Merck (known as Merck Sharp & Dohme outside the United States and Canada) and Macrophage Therapeutics (a subsidiary of Navidea Biopharmaceuticals) to develop ACCURINS based on their proprietary therapeutic payloads and/or targeting ligands. BIND’s collaboration with AstraZeneca has resulted in the Aurora B Kinase inhibitor ACCURIN AZD2811, which became the second ACCURIN candidate to enter clinical development. BIND’s collaboration with Pfizer has resulted in the selection of an ACCURIN candidate that is entering IND-enabling studies.