On September 28, 2017 Baxter International Inc.(NYSE:BAX), reported that it will host a conference call to discuss its third quarter 2017 financial results on Wednesday, October 25, 2017, at 7:30 a.m. Central Standard Time (Press release, Baxter International, SEP 28, 2017, View Source [SID1234520893]).

To access the call, dial 844.886-1929 (domestic) or 225.239.4663 (international). Please dial into the call at least 10 minutes prior to the start to connect. The Conference ID is 68201200.

This call is also being webcast by Nasdaq OMX and can be accessed at Baxter’s website at www.baxter.com. The conference call will be recorded by Baxter and is copyrighted material. It cannot be recorded or rebroadcast without Baxter’s permission.

On September 28, 2017 Galena Biopharma, Inc. (NASDAQ: GALE), a biopharmaceutical company developing hematology and oncology therapeutics that address unmet medical needs, reported that two clinical trials evaluating NeuVax (nelipepimut-S) in combination with trastuzumab (Herceptin; Genentech/Roche) for the prevention of recurrence in breast cancer patients have enrolled the protocol-defined number of patients to complete enrollment (Press release, Galena Biopharma, SEP 28, 2017, View Source [SID1234520707]). The milestones were reported by the clinical research organization conducting both trials – a Phase 2b clinical trial in HER2 1+/2+ patients and a Phase 2 clinical trial in HER2 3+ patients.

“Completing enrollment in both of these trials represents a major milestone for NeuVax development,” said Bijan Nejadnik, M.D., Executive Vice President and Chief Medical Officer of Galena. “The combination of trastuzumab and NeuVax has been shown to be synergistic in preclinical investigation, and we believe could be an effective treatment to prevent breast cancer recurrence in patients with no other treatment options. We would like to thank our investigators and patients who are participating in these trials as we look forward to the interim results next year for the Phase 2b trial and the primary endpoints for both trials in 2019.”

Phase 2b Clinical Trial in HER2 1+/2+ Patients

The Phase 2b clinical trial has enrolled the necessary 300 patients to complete enrollment. The clinical trial is a randomized, multicenter, investigator-sponsored, study enrolling HER2 1+ and 2+, HLA A2+, A3+, A24 and/or A26, node positive, and high-risk node negative breast cancer patients. Eligible patients are randomized to receive NeuVax + GM-CSF + trastuzumab or trastuzumab + GM-CSF alone. Once enrolled, all patients receive the standard trastuzumab dosing for 12 months. One cohort also receives six doses of NeuVax given as a primary vaccine series starting with the third dose of trastuzumab and then goes on to receive a NeuVax booster inoculation once every six months for up to 36 months. The next milestone for the trial will be the interim efficacy analysis that is scheduled to be performed by the Data Safety Monitoring Board (DSMB) in the first quarter of 2018. The primary endpoint of the study is disease-free survival after 24 months, with results expected from that milestone in the fourth quarter of 2019. Genentech/Roche is providing the trastuzumab and partial funding for this trial.

Data presented in October 2016 demonstrated that this novel combination of trastuzumab and NeuVax with HER2 low-expressing patients is well tolerated and the cardiac effects of trastuzumab are not impacted by the addition of NeuVax. In February 2017, the DSMB reported that there were no safety concerns with the trial and the trial is not futile. The recommendation from the DSMB was to continue the trial with one revision to the statistical analysis plan regarding the timing of the pre-specified interim analysis. Given the lengthy duration of enrollment for the trial, the DSMB determined that the pre-specified interim efficacy analysis be moved up from 12 months to 6 months after the last patient is enrolled. Therefore, the DSMB expects to perform the interim efficacy analysis in the first quarter of 2018.

Phase 2 Clinical Trial in HER2 3+ Patients

The Phase 2 clinical trial has enrolled the necessary 100 patients to complete enrollment. This multi-center, prospective, randomized, single-blinded trial enrolled patients with a diagnosis of HER2 3+ breast cancer who are HLA A2+ or HLA A3+ and are determined to be at high-risk for recurrence. High-risk is defined as having received neoadjuvant therapy with an approved regimen that includes trastuzumab but not obtaining a pathological complete response at surgery, or underwent surgery as a first intervention and was found to be pathologically node-positive (³4 positive lymph nodes, or having 1-3 positive lymph nodes (pN1) if hormone receptor negative). These high-risk patients are known to have higher recurrence rates than other HER2 3+ breast cancer patients. Eligible patients are randomized to receive NeuVax + GM-CSF + trastuzumab or trastuzumab + GM-CSF alone. Once enrolled, all patients receive the standard trastuzumab dosing for 12 months. One cohort also receives six doses of NeuVax given as a primary vaccine series starting with the third dose of trastuzumab and then goes on to receive a NeuVax booster inoculation once every six months for up to 36 months. The primary endpoint of the study is disease-free survival after 24 months, with results expected from that milestone in the fourth quarter of 2019. Partial funding for this trial was awarded through the Congressionally Directed Medical Research Program funded through the Department of Defense, via a Breast Cancer Research Program Breakthrough Award.

In February 2017, the DSMB reported that there were no safety concerns with the trial and the trial is not futile. The pre-specified interim safety analysis was completed on n=50 patients and demonstrated that the agent is well tolerated with no increased cardiotoxicity associated with giving NeuVax in combination with trastuzumab. The recommendation from the DSMB was to continue the HER2 3+ trial unmodified.

About NeuVax (nelipepimut-S)

NeuVax (nelipepimut-S) is a first-in-class, HER2-directed cancer immunotherapy under evaluation to prevent breast cancer recurrence after standard of care treatment in the adjuvant setting. It is the immunodominant peptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut-S sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTLs) following binding to specific HLA molecules on antigen presenting cells (APC). These activated specific CTLs recognize, neutralize and destroy, through cell lysis, HER2 expressing cancer cells, including occult cancer cells and micrometastatic foci. The nelipepimut-S immune response can also generate CTLs to other immunogenic peptides through inter- and intra-antigenic epitope spreading. In clinical studies, NeuVax is combined with recombinant granulocyte macrophage-colony stimulating factor (GM-CSF).

NeuVax is currently in two breast cancer studies in combination with trastuzumab (Herceptin; Genentech/Roche): a Phase 2b trial in node positive and triple negative HER2 IHC 1+/2+ (clinicaltrials.gov identifier: NCT01570036); and, a Phase 2 trial in high risk, node positive or negative HER2 IHC 3+ patients (clinicaltrials.gov identifier: NCT02297698). A Phase 2 clinical trial is also ongoing with NeuVax in patients with ductal carcinoma in situ (DCIS) (clinicaltrials.gov identifier: NCT02636582), and a Phase 2 trial is planned in patients with gastric cancer.

On September 28, 2017 The U.S. Food and Drug Administration reported that they approved Verzenio (abemaciclib) to treat adult patients who have hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer that has progressed after taking therapy that alters a patient’s hormones (endocrine therapy). Verzenio is approved to be given in combination with an endocrine therapy, called fulvestrant, after the cancer had grown on endocrine therapy. It is also approved to be given on its own, if patients were previously treated with endocrine therapy and chemotherapy after the cancer had spread (metastasized) (Press release, US FDA, SEP 28, 2017, View Source [SID1234520694]).

“Verzenio provides a new targeted treatment option for certain patients with breast cancer who are not responding to treatment, and unlike other drugs in the class, it can be given as a stand-alone treatment to patients who were previously treated with endocrine therapy and chemotherapy,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

Verzenio works by blocking certain molecules (known as cyclin-dependent kinases 4 and 6), involved in promoting the growth of cancer cells. There are two other drugs in this class that are approved for certain patients with breast cancer, palbociclib approved in February 2015 and ribociclib approved in March 2017.

Breast cancer is the most common form of cancer in the United States. The National Cancer Institute at the National Institutes of Health estimates approximately 252,710 women will be diagnosed with breast cancer this year, and 40,610 will die of the disease. Approximately 72 percent of patients with breast cancer have tumors that are HR-positive and HER2-negative.

The safety and efficacy of Verzenio in combination with fulvestrant were studied in a randomized trial of 669 patients with HR-positive, HER2-negative breast cancer that had progressed after treatment with endocrine therapy and who had not received chemotherapy once the cancer had metastasized. The study measured the length of time tumors did not grow after treatment (progression-free survival). The median progression-free survival for patients taking Verzenio with fulvestrant was 16.4 months compared to 9.3 months for patients taking a placebo with fulvestrant.

The safety and efficacy of Verzenio as a stand-alone treatment were studied in a single-arm trial of 132 patients with HR-positive, HER2-negative breast cancer that had progressed after treatment with endocrine therapy and chemotherapy after the cancer metastasized. The study measured the percent of patients whose tumors completely or partially shrank after treatment (objective response rate). In the study, 19.7 percent of patients taking Verzenio experienced complete or partial shrinkage of their tumors for a median 8.6 months.

Common side effects of Verzenio include diarrhea, low levels of certain white blood cells (neutropenia and leukopenia), nausea, abdominal pain, infections, fatigue, low levels of red blood cells (anemia), decreased appetite, vomiting and headache.

Serious side effects of Verzenio include diarrhea, neutropenia, elevated liver blood tests and blood clots (deep venous thrombosis/pulmonary embolism). Women who are pregnant should not take Verzenio because it may cause harm to a developing fetus.

The FDA granted this application Priority Review and Breakthrough Therapy designations.

The FDA granted the approval of Verzenio to Eli Lilly and Company.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On September 29, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Perjeta (pertuzumab), in combination with Herceptin (trastuzumab) and chemotherapy (the Perjeta-based regimen), for adjuvant (after surgery) treatment of HER2-positive early breast cancer (eBC) (Press release, Hoffmann-La Roche, SEP 28, 2017, View Source [SID1234520698]). The FDA is expected to make a decision on approval by 28 January 2018. The sBLA is based on results of the phase III APHINITY study. A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.

“We are pleased to receive Priority Review for the Perjeta-based regimen for the adjuvant treatment of HER2-positive early breast cancer,” said Sandra Horning, M.D., Roche’s Chief Medical Officer and Head of Global Product Development. “The goal of treating breast cancer early is to provide people with the best chance for a cure. Despite advances in the treatment of this disease, many people treated with the current standard of care still see their cancer return.”

The combination of Perjeta, Herceptin and chemotherapy is licensed as a neoadjuvant (before surgery) treatment for people with HER2-positive eBC in more than 85 countries worldwide following approvals by the European Medicines Agency (EMA) and the US FDA. In the US, the regimen is currently available under the FDA Accelerated Approval Program. This sBLA seeks to convert the current accelerated approval to full approval in the US. Additionally, the APHINITY trial reflects the commitment to evaluate the Perjeta-based regimen as part of a complete treatment approach for eBC. Perjeta in combination with Herceptin and docetaxel chemotherapy is also approved in the US and the European Union for people with previously untreated HER2-positive metastatic breast cancer.

About APHINITY1
APHINITY (Adjuvant Pertuzumab and Herceptin IN Initial TherapY in Breast Cancer, NCT01358877/ BO25126/ BIG 4-11) is an international, phase III, randomised, double-blind, placebo-controlled, two-arm study evaluating the efficacy and safety of Perjeta plus Herceptin and chemotherapy compared to Herceptin and chemotherapy as adjuvant therapy in 4,805 people with operable HER2-positive eBC. The primary efficacy endpoint of the APHINITY study is invasive disease-free survival (iDFS), which in this study is defined as the time a patient lives without return of invasive breast cancer at any site or death from any cause after adjuvant treatment. Secondary endpoints include cardiac and overall safety, overall survival, disease-free survival and health-related quality of life.

On September 28, 2017 CBT Pharmaceuticals (CBT), a biopharmaceutical company focused on developing innovative oncology therapeutics harnessing the immune system and targeting specific molecular pathways to tame cancer, reported that the first subject has been dosed in the Phase 1 multi-center study, CBT-101-01 (Press release, CBT Pharmaceuticals, SEP 28, 2017, View Source [SID1234520681]). CBT-101 is a specific oral inhibitor of the c-Met kinase. This is the company’s first clinical trial in the United States (NCT03175224). Following identification of a dose for CBT-101, the trial is planned to expand into tumor specific cohorts.

“This is an important milestone for CBT and its employees. We are also extremely grateful to the patients and clinical researchers at premier U.S. institutions that will contribute to the evaluation of CBT-101,” said Sanjeev Redkar, Ph.D., President and Chief Executive Officer. “CBT-101 has demonstrated strong inhibition of tumor growth in cell lines and patient derived models across multiple tumor types with c-Met fusions, mutations or amplifications. We anticipate the clinical results so that we can explore single agent development as well as combination approaches.”

“The aberrant activation of c-Met has been demonstrated to be highly correlated with outcome in many cancer indications, including kidney, lung, gastric, esophageal and brain cancer and plays a major role in cancer pathogenesis including tumor growth, angiogenesis and metastasis, as well as the suppression of cell death,” said Anthony El-Khoueiry, M.D., Associate Professor of Clinical Medicine and Phase 1 Program Director at USC Norris Comprehensive Cancer Center. “Through our research collaboration with CBT Pharmaceuticals and the cadre of investigators on this trial, we look forward to characterizing CBT-101 safety profile and dose, and defining a path for further development.”

CBT-101-01 Clinical Trial

This is a Phase 1, multi-center, open-label, 2-part Dose Escalation Segment with a Dose and Disease Expansion Cohort study to assess the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of CBT-101 monotherapy in subjects with advanced solid tumors with c-Met dysregulation. These findings will be used to establish a recommended Phase 2 dose for future clinical trials. A total of approximately 68 subjects will be enrolled.

There are two other Phase 1 trials with CBT-101 (PLB1001) ongoing in China – one in Met-positive Advanced Non-Small Cell Lung Cancer and the other in PTPRZ1-MET Fusion Gene Positive Recurrent High-Grade Gliomas.

More information on all trials is available at ClinicalTrials.gov.

CBT-101 Oral Capsule (PLB1001)

CBT-101 is a novel small molecule drug that targets the epithelial to mesenchymal transition (EMT) pathway that is dysregulated in several tumors. It is a specific inhibitor of the c-Met receptor. CBT-101 has demonstrated tumor inhibitory effect in a variety of human primary c-Met amplified gastric, hepatic, pancreatic and lung cancer xenograft animal models with c-Met fusions, mutations or amplifications (AACR, 2017).