On November 23, 2015 Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for use of ONCASPAR as a component of antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients (Press release, Baxalta, NOV 23, 2015, View Source [SID:1234508319]). Schedule your 30 min Free 1stOncology Demo! The CHMP’s positive opinion will now be referred to the European Commission (EC), which grants marketing authorization for medicines in the European Union. Pending EC approval, Baxalta will be authorized to market ONCASPAR in the 28 member countries of the European Union (EU), as well as Iceland, Liechtenstein and Norway.
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Pending EC approval, ONCASPAR will provide an important treatment option for more European patients with this rapidly progressing cancer of the white blood cells responsible for more than 80 percent of childhood leukaemia cases2 – the most common type of childhood cancer.
"We are pleased to receive a positive CHMP opinion for ONCASPAR as part of a multi-agent chemotherapy regimen in paediatric and adult populations; this is a significant milestone in increasing patient access to this important biologic treatment for patients impacted by ALL," said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. "At Baxalta, we are committed to expanding the availability of ONCASPAR globally, and this decision makes a curative treatment available to more patients across the world."
Today, children in the U.S. diagnosed with ALL have a survival rate of more than 90 percent – a direct result of multi-agent chemotherapy treatments. ONCASPAR is a key component of these curative therapies. Currently, there have been national licenses granted to market ONCASPAR in Argentina, Belarus, Germany, Kazakhstan, Poland, Russia, Ukraine and the United States.
About ONCASPAR (pegaspargase)
In the U.S., ONCASPAR (pegaspargase) is indicated as a component of a multi-agent chemotherapeutic regimen for the first–line treatment of patients with acute lymphoblastic leukaemia (ALL) and for the treatment of patients with ALL and hypersensitivity to native forms of L-asparaginase.
Important Safety Information for ONCASPAR
ONCASPAR is contraindicated in patients with a history of serious allergic reactions to pegaspargase, and in patients with a history of pancreatitis, serious thrombosis, or serious hemorrhagic events with prior L-asparaginase therapy.
The most common adverse reactions with ONCASPAR (=2%) are allergic reactions (including anaphylaxis), hyperglycemia, pancreatitis, central nervous system (CNS) events, thrombosis, coagulopathy, hyperbilirubinemia, and elevated transaminases. Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.
Patients should be observed for one hour after administration as anaphylaxis or serious allergic reactions can occur. Discontinue ONCASPAR in patients that develop pancreatitis, serious allergic reactions, or serious thrombotic events. Patients with abdominal pain should be evaluated for evidence of pancreatitis.
Serum glucose should be monitored as irreversible glucose intolerance can occur in some cases. Coagulopathy and hepatotoxicity can occur; appropriate monitoring should be performed.
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About Acute Lymphoblastic Leukaemia
Acute lymphoblastic leukaemia (ALL) is a rare, fast-growing cancer of the white blood cells, and each year there are approximately 4,000-5,000 new cases in Europe and the United States, respectively. The disease is the most common childhood cancer and is responsible for more than 80 percent of childhood leukaemia cases. The five-year paediatric survival rate has climbed to 90 percent with modern therapies.
Author: [email protected]
Aduro Biotech Receives Orphan Drug Designation in the European Union for CRS-207 for the Treatment of Mesothelioma
On November 23, 2015 Aduro Biotech, Inc. (Nasdaq:ADRO) reported that the European Medicines Agency (EMA) granted Orphan Drug Designation to CRS-207 for the treatment of malignant pleural mesothelioma (MPM) (Press release, Aduro BioTech, NOV 23, 2015, View Source [SID:1234508317]). Schedule your 30 min Free 1stOncology Demo! "The receipt of Orphan Drug Designation in the European Union (EU) for CRS-207 for the treatment of MPM marks a significant regulatory milestone for Aduro, as we expand our operations in Europe and advance our therapies closer to the commercial marketplace," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We look forward to working with the EMA to expeditiously advance CRS-207 through development with the goal of bringing this potential therapy to patients throughout Europe suffering from mesothelioma."
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To receive Orphan Drug Designation from the EMA, a therapy must be intended for the treatment of a life-threatening or chronically debilitating rare condition with a prevalence of less than five in 10,000 in the European Union. Orphan Drug Designation provides incentives designed to facilitate development, including protocol assistance and scientific advice and importantly, may provide up to ten years of market exclusivity in the EU following product approval.
Aduro is conducting a Phase 1b study of CRS-207 in combination with standard of care chemotherapy in patients with unresectable malignant pleural mesothelioma. The company plans to advance directly to a Phase 3 clinical trial with CRS-207 in combination with standard-of-care chemotherapy in patients with unresectable MPM in the first half of 2016. In addition to the newly granted Orphan Drug Designation from the EMA, CRS-207 received Orphan Status for the treatment of mesothelioma from the U.S. Food and Drug Administration in March 2015.
About CRS-207
CRS-207 is one of a family of product candidates based on Aduro’s live-attenuated, double-deleted (LADD) Listeria monocytogenes immunotherapy platform that induces a potent innate and T cell-mediated adaptive immune response. CRS-207 has been engineered to express the tumor-associated antigen mesothelin, which is over-expressed in many cancers including mesothelioma and pancreatic, non-small cell lung, ovarian, endometrial and gastric cancers.
Roche presents early data on investigational cancer immunotherapy atezolizumab in combination with Zelboraf in patients with BRAFV600 mutant-positive metastatic melanoma
On November 23, 2015 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported results from a phase Ib study of the investigational cancer immunotherapy atezolizumab (MPDL3280A), used in combination with the BRAF inhibitor Zelboraf (vemurafenib) for previously untreated BRAFV600 mutation-positive unresectable or metastatic melanoma (Press release, Hoffmann-La Roche , NOV 23, 2015, View Source [SID:1234508314]). Schedule your 30 min Free 1stOncology Demo! Adverse events (AEs) were manageable and generally reversible. The study also showed that the combination resulted in an objective response rate [ORR] of 76% (95% confidence interval [CI:] 50.1%–93.2%) of people (n=17), including three complete responders (CR). The data were presented at the Society of Melanoma Research (SMR) 2015 International Congress.1
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"These early efficacy results encourage us to further evaluate combination strategies of atezolizumab and targeted therapies like Zelboraf in people living with advanced melanoma, a disease which is still associated with a poor prognosis", Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development.
Roche is also investigating a triplet regimen with atezolizumab plus the established targeted therapy combination of Zelboraf and Cotellic (cobimetinib), a selective MEK inhibitor, in a phase Ib study.
About the phase Ib study of atezolizumab in combination with Zelboraf
This phase Ib, open-label study aimed to evaluate the safety and pharmacology of atezolizumab in combination with Zelboraf in patients with previously untreated BRAFV600 mutation-positive metastatic melanoma
17 patients were evaluable for safety and efficacy at the time of this data cut
Patients received atezolizumab combined with Zelboraf concurrently (Cohort 1 [C1], n=3) or after a run-in period with Zelboraf alone of 56 days (Cohort 2 [C2]; n=8) or 28 days (Cohort 3 [C3]; n=6)
Patients were given atezolizumab intravenously every 3 weeks at 20 mg/kg (C1) or 15 mg/kg (or 1,200 mg fixed; C2/C3). Oral Zelboraf was given twice daily at 960 mg during the run-in period and at 720 mg during atezolizumab and Zelboraf combined treatment
PD-L1 expression was centrally assayed using immunohistochemistry (IHC; SP142 assay)
About BRAFV600 mutation-positive metastatic melanoma
Melanoma is less common, but more aggressive and deadlier, than other forms of skin cancer.2,3 A mutation of the BRAF protein occurs in approximately half of melanomas, and a test can be used to determine who can be treated with a BRAF-inhibitor.4 When melanoma is diagnosed early, it is generally a curable disease,5,6 but most people with advanced melanoma have a poor prognosis.3 More than 232,000 people worldwide are currently diagnosed with melanoma each year.7 In recent years, there have been significant advances in treatment for metastatic melanoma, and people with the disease have more options. However, it continues to be a serious health issue with a high unmet need and a steadily increasing incidence over the past 30 years.8
About atezolizumab
Atezolizumab (also known as MPDL3280A) is an investigational monoclonal antibody designed to interfere with a protein called PD-L1. Atezolizumab is designed to target PD-L1 expressed on tumour cells and tumour-infiltrating immune cells, preventing it from binding to PD-1 and B7.1 on the surface of T cells. By inhibiting PD-L1, atezolizumab may enable the activation of T cells, restoring their ability to effectively detect and attack tumour cells.
About Zelboraf
Zelboraf was the first approved treatment for patients with unresectable or metastatic melanoma with BRAFV600 mutation as detected by a validated test, such as Roche’s cobas 4800 BRAF Mutation Test. Zelboraf is not indicated for use in patients with wild-type BRAF melanoma.
About Cotellic plus Zelboraf
Cotellic and Zelboraf are used in combination to treat unresectable or metastatic melanoma with BRAFV600 mutation. Found in approximately half of melanomas, mutated BRAF causes abnormal signalling inside certain cancer cells leading to tumour growth. Zelboraf binds to mutant BRAF, to interrupt abnormal signalling that can cause tumours to grow.9,10 Cotellic is designed to inhibit some forms of MEK. Both BRAF and MEK are proteins in a cell signalling pathway that help control cell growth and survival. When used in combination, Cotellic and Zelboraf are thought to reduce cancer cell growth longer than Zelboraf alone. A patient’s healthcare provider will perform a test to make sure Cotellic and Zelboraf are right for the patient. It is not known if Cotellic and Zelboraf are safe and effective in children under 18 years of age.
Cotellic was approved in Switzerland in August 2015 and in the US in November 2015. In September, the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) issued a positive opinion for Roche’s marketing authorisation application for Cotellic in the European Union. A decision from the European Commission is expected before the end of 2015.
Updated data showed Cotellic in combination with Zelboraf helped people with a specific type of advanced melanoma live significantly longer than with Zelboraf alone
On November 23, 2015 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported data from the pivotal coBRIM study, which showed that Cotellic (cobimetinib) in combination with Zelboraf (vemurafenib) helped people with BRAF V600E and V600K mutation-positive unresectable or metastatic melanoma live significantly longer (overall survival; OS) than with Zelboraf alone (Press release, Hoffmann-La Roche , NOV 23, 2015, View Source [SID:1234508313]).1 Schedule your 30 min Free 1stOncology Demo! Cotellic plus Zelboraf reduced the risk of death by 30 percent compared to Zelboraf alone and helped people live a median of nearly two years (median OS 22.3 months vs. 17.4 months, hazard ratio [HR]=0.70, 95 percent CI: 0.55–0.90, p=0.005).1 Ongoing study monitoring did not identify any new safety signals. The final coBRIM OS results were presented during the 12th International Congress of the Society for Melanoma Research (SMR) held in San Francisco, California from 18 – 21 November.
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"With about half of the people taking Cotellic and Zelboraf alive after two years, these data underscore the progress being made in cancer research towards better patient outcomes." said Sandra Horning, M.D., Chief Medical Officer and Head of Global Product Development. "Five years ago, the survival rate for BRAF mutation-positive advanced melanoma was measured in months, and now we are measuring it in years."
This final analysis of the OS data from coBRIM showed that with the combination of Cotellic and Zelboraf, 74.5 percent of people with BRAF V600 mutation-positive advanced melanoma in the study were alive at one year and 48.3 percent were alive at two years.
The data were presented in an oral session presentation by Dr. Victoria Atkinson, Medical Oncologist at Princess Alexandra Hospital, Queensland, Australia on 21 November.
The announcement follows the U.S. Food and Drug Administration (FDA) approval of Cotellic for the treatment of people with BRAF V600E or V600K mutation-positive unresectable or metastatic melanoma in combination with Zelboraf. A decision from the European Commission is expected before the end of 2015. The final OS results are being submitted to both of these health authorities for consideration.
About the coBRIM study
CoBRIM is an international, randomised, double-blind, placebo-controlled Phase III study evaluating the safety and efficacy of 60 mg once daily of Cotellic plus 960 mg twice daily of Zelboraf compared to 960 mg twice daily of Zelboraf alone. In the study, 495 patients with BRAF V600 mutation-positive unresectable locally advanced or metastatic melanoma (detected by the cobas 4800 BRAF Mutation Test) and previously untreated for advanced disease were randomised to receive Zelboraf every day on a 28-day cycle plus either Cotellic or placebo on days 1-21. Treatment was continued until disease progression, unacceptable toxicity or withdrawal of consent. Investigator-assessed PFS is the primary endpoint. Secondary endpoints include PFS by independent review committee, objective response rate, overall survival, duration of response and other safety, pharmacokinetic and quality of life measures.2
About Cotellic plus Zelboraf
Cotellic and Zelboraf are prescription medicines used in combination to treat melanoma that has spread to other parts of the body or cannot be removed by surgery, and that has a certain type of abnormal "BRAF" gene. Found in approximately half of melanomas3, mutated BRAF causes abnormal signaling inside cancer cells leading to tumor growth4,5. Zelboraf is designed to inhibit some mutated forms of BRAF and Cotellic is designed to inhibit some forms of MEK6. Both BRAF and MEK are proteins in a cell signaling pathway that help control cell growth and survivals7. When used in combination, Cotellic and Zelboraf are thought to reduce cancer cell growth longer than with Zelboraf alone. A patient’s healthcare provider will perform a test to make sure Cotellic and Zelboraf are right for the patient. Cotellic and Zelboraf are not used to treat melanoma with a normal BRAF gene. It is not known if Cotellic and Zelboraf are safe and effective in children under 18 years of age.
Cotellic is also being investigated in combination with several investigational medicines, including an immunotherapy, in several tumour types such as non-small cell lung cancer and colorectal cancer. Cotellic was discovered by Exelixis Inc. and is being developed by Roche in collaboration with Exelixis.
About melanoma
Melanoma is less common, but more aggressive and deadlier than other forms of skin cancer.8,9 When melanoma is diagnosed early, it is generally a curable disease,10,11 but most people with advanced melanoma have a poor prognosis.8 More than 232,000 people worldwide are currently diagnosed with melanoma each year12 In recent years, there have been significant advances in treatment for metastatic melanoma and people with the disease have more options. However, it continues to be a serious health issue with a high unmet need and a steadily increasing incidence over the past 30 years.13
CTI BioPharma Initiates Rolling Submission of U.S. New Drug Application for Pacritinib for the Treatment of Patients with Myelofibrosis
On November 23, 2015 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) reported the initiation of its rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R (Press release, CTI BioPharma, NOV 22, 2015, View Source;p=RssLanding&cat=news&id=2114525 [SID:1234508312]). As part of the application, CTI BioPharma and its partner, Baxalta Incorporated (Baxalta), are seeking accelerated approval and priority review for pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/uL). If approved for the requested indication, pacritinib would be the first JAK2 inhibitor approved for the treatment of patients with myelofibrosis with platelet counts of less than 50,000/uL – a specific patient population for which there are currently no approved drugs. The rolling NDA allows completed portions of an NDA to be submitted and reviewed by the FDA on an ongoing basis. CTI BioPharma and Baxalta plan to complete the submission before the end of 2015.
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Myelofibrosis (a type of myeloproliferative neoplasm) is a rare, but serious and life-threatening chronic bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. Myelofibrosis is associated with significantly reduced quality of life and shortened survival, can affect patients of all ages (with a median affected age being 65 years) and an estimated prevalence in the United States of approximately 18,000 patients.
"We believe the initiation of the rolling NDA submission represents a major step forward toward potentially offering pacritinib as a next generation JAK2/FLT3 inhibitor to patients with this rare and chronic type of blood cancer," said James A. Bianco, M.D., President and CEO of CTI BioPharma. "We are excited to have achieved this milestone and look forward to working with the FDA during the review process, with the goal of bringing this important treatment to market – which we hope will fill an unmet need for many patients whose lives are profoundly impacted by myelofibrosis."
The submission includes data from the PERSIST-1 Phase 3 trial – as well as data from Phase 1 and 2 studies of pacritinib. Submission of an NDA after a single Phase 3 trial under accelerated approval, instead of waiting to complete two Phase 3 trials, could potentially reduce time to market by up to 14 months. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high-risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment emergent thrombocytopenia on another JAK2 therapy; or patients who are intolerant to or whose symptoms are not well controlled (or sub-optimally managed) on another JAK2 therapy.
About Myelofibrosis and Myeloproliferative Neoplasms
Myelofibrosis is a one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of hematological blood cancers. The three main types of MPNs are myelofibrosis, polycethemia vera, and essential thrombocythemia.1 Myelofibrosis is a serious and life-threatening chronic bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue, and pain.
The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S. of which myelofibrosis accounts for approximately 18,000 patients.2 In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis2 and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia.3 The median survival for high-risk patients is less than one and a half years; median survival for myelofibrosis patients overall is approximately six years.4
About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis, including but not limited to patients with disease-related thrombocytopenia, patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy, or patients who are intolerant of, or whose symptoms are sub-optimally managed on other JAK2 inhibitor therapy. The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Pacritinib does not have regulatory approval and is not commercially available.
CTI BioPharma and Baxalta (NYSE:BXLT) are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Baxalta will jointly commercialize pacritinib in the U.S. while Baxalta has exclusive commercialization rights for all indications outside the U.S.