On January 19, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported that the Company has reached a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) for the design of Delcath’s new Phase 3 clinical trial of Melphalan Hydrochloride for Injection for use with the Delcath Hepatic Delivery System (Melphalan/HDS) to treat patients with hepatic dominant ocular melanoma (Press release, Delcath Systems, JAN 19, 2016, View Source [SID:1234508808]).

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 SPA provides agreement that the Phase 3 trial design adequately addresses objectives that, if met, would support the submission for regulatory approval of Melphalan/HDS.

The agreement also represents the satisfactory resolution of a substantial number of the FDA’s issues in the Complete Response Letter (CRL) issued in September 2013. These issues were related to safety of a previous generation of the Melphalan/HDS device and procedure. Delcath completed the work necessary to satisfy these requirements prior to submitting its request for the SPA agreement.

The new pivotal trial, the FOCUS Clinical Trial for Patients with Hepatic Dominant Ocular Melanoma (the FOCUS trial), will evaluate the safety and efficacy profile of the Melphalan/HDS versus best alternative care. The primary endpoint will be overall survival, and secondary endpoints will include progression-free survival, overall response rate and quality-of-life measures. Full details of the Phase 3 clinical trial will be made public upon the launch of the study and will be available at www.clinicaltrials.gov.

"This agreement marks a major milestone for Delcath," said Jennifer K. Simpson, Ph.D., MSN, CRNP, President and Chief Executive Officer of Delcath. "Under this SPA our new FOCUS trial, if successful, will provide a clear pathway to an indication in hepatic dominant ocular melanoma for Melphalan/HDS. Additionally, through the dedicated work of our team and in close collaboration with the FDA, we have satisfied a substantial number of the requirements of the FDA’s 2013 CRL. Based on our commercial experience in Europe, the continued support and enthusiasm from Key Opinion Leaders and the clinical data that have been presented and published recently, we have confidence that our FOCUS trial can demonstrate the efficacy and safety necessary for a positive benefit/risk profile for Melphalan/HDS, and that the study’s objectives can be met. There is strong interest from leading cancer centers in the U.S. and Europe to participate in this study and we look forward to beginning enrollment in this registrational trial."

About Special Protocol Assessments
The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and written guidance on the design and size of proposed protocols that are intended to form the basis for a new drug application. Final marketing approval depends on the results of efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in the Phase 3 clinical program. The SPA agreement may only be changed through a written agreement between the sponsor and the FDA, or if the FDA becomes aware of a substantial scientific issue essential to product efficacy or safety.

On January 19, 2016 Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, reported that the European Commission has granted Marketing Authorization for use of ONCASPAR as a combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients (Press release, Baxalta, JAN 19, 2016, View Source [SID:1234508806]).

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!

With this approval, Baxalta is authorized to market ONCASPAR in the 28 member countries of the European Union (EU), as well as Iceland, Liechtenstein and Norway. The drug is already licensed to market in Argentina, Belarus, Germany, Kazakhstan, Poland, Russia, Ukraine and the United States.

"ONCASPAR has been used as an integral component of the treatment regimen for paediatric and adult patients with ALL for many years, in Europe, and worldwide," said Prof. Dr. med. Martin Schrappe, director of the department of general paediatrics at the Schleswig-Holstein University Hospital in Kiel, Germany. "Today’s marketing authorization will ensure that more patients across the EU will benefit from access to ONCASPAR as part of a standard of care regimen."

With this authorization, ONCASPAR will provide an important treatment option for more European patients with this rapidly progressing cancer of the white blood cells responsible for up to 80 percent of childhood leukaemia cases – the most common type of childhood cancer. However, ALL is not only a childhood cancer but can also occur in adults. Adult ALL accounts for approximately 40 percent of the annual incidence.2

"For more than two decades, ONCASPAR has fulfilled a clear need for an effective and well-tolerated treatment for ALL patients worldwide. This European marketing authorization allows Baxalta to expand the use of ONCASPAR, improving treatment outcomes for all patients in the EU," said David Meek, executive vice president and president, Oncology, Baxalta. "This approval is important as we strive to make a difference in the lives of people living with cancer in all parts of the world."

About ONCASPAR (pegaspargase) in the EU

ONCASPAR is indicated as a component of antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients.3

Contraindications3

Hypersensitivity to the active substance or to any of the excipients
Severe hepatic impairment (bilirubin > 3 times upper limit of normal [ULN]; transaminases > 10 times ULN)
History of serious thrombosis with prior L-asparaginase therapy
History of pancreatitis, including the related to previous asparaginase therapy
History of serious hemorrhagic events with prior L-asparaginase therapy
EU Important Safety Information

ONCASPAR is indicated as a component of antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients.

ONCASPAR is contraindicated in patients with severe hepatic impairment (defined as bilirubin > 3 times upper limit of normal [ULN]; transaminases > 10 times ULN), a history of serious thrombosis with prior L asparaginase therapy, a history of pancreatitis, including the related to previous asparaginase therapy and those with a history of serious hemorrhagic events with prior L asparaginase therapy.

Anaphylaxis or serious allergic reactions can occur; therefore, patients should be observed for one hour after administration. Discontinue ONCASPAR in patients with serious allergic reactions. Patients with abdominal pain should be evaluated for evidence of pancreatitis. Discontinue ONCASPAR in patients with pancreatitis. ONCASPAR should also be discontinued in patients with serious thrombotic events.

Glucose intolerance, in some cases irreversible, can occur; serum glucose should be monitored. Coagulopathy and hepatotoxicity can occur; appropriate monitoring should be performed.

The most common adverse reactions with ONCASPAR (=2%) are allergic reactions (including anaphylaxis), hyperglycemia, pancreatitis, central nervous system (CNS) toxicity, thrombosis, coagulopathy, hyperbilirubinemia and elevated transaminases.

Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.

Oncaspar may possess immunosuppressive activity. It is therefore possible that use of this medicinal product promotes infections in patients.

Combination therapy with Oncaspar can result in severe hepatic toxicity and central nervous system toxicity. Caution is required when Oncaspar is given in combination with other hepatotoxic substances, especially if there is preexisting hepatic impairment. In this case, patients should be monitored for liver impairment.

In the presence of symptoms of hyperammonemia (e.g. nausea, vomiting, lethargy, irritation), ammonia levels should be monitored closely.

Labeling may differ by country registration. Please refer to your country specific labeling for detailed information.

About Acute Lymphoblastic Leukaemia1

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 more than 80 percent with modern therapies.

On January 19, 2016 Advaxis, Inc. (NASDAQ:ADXS), a clinical stage biotechnology company developing cancer immunotherapies, reported that it has added two more patents to its growing patent portfolio (Press release, Advaxis, JAN 19, 2016, View Source [SID:1234508805]). The first patent, European Patent No. 1804831, expands the composition of matter claims covering HER-2 tumor antigens. The second patent, U.S. Patent No. 9,226,958, expands the use of the Company’s Lm Technology beyond oncology, specifically to induce an immune response in parasitically infected patients.

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!

"These patents broaden our protection in HER2 and expand our reach beyond oncology, into parasitically infected patients," said Daniel J. O’Connor, President and Chief Executive Officer of Advaxis. "We are continuing to work diligently on our patent portfolio, currently with 90 granted patents and 111 pending applications, worldwide."

Advaxis is the exclusive licensee of both of these patents. European Patent No. 1804831 will expire on September 14, 2025 and U.S. Patent 9,226,958 will expire on October 3, 2031. The company has an extensive patent portfolio that protects its product candidates and Listeria monocytogenes (Lm) based immunotherapy technology. The Company currently owns or has rights to more than 200 patents and applications globally, which are owned, licensed from, or co-owned with the Trustees of the University of Pennsylvania, Merck, Sharpe & Dohme BV, the National Institutes of Health, the University of Georgia Research Foundation, Inc., and Georgia Regents University. Its patents currently extend protection through 2032.

About HER2 Expressing Solid Tumor Cancers

Human epidermal growth factor receptor 2 (HER2) is overexpressed in a percentage of solid tumors such as breast, gastric, bladder, brain, pancreatic, ovarian and pediatric bone cancer (osteosarcoma). The American Cancer Society estimates that in 2015 in the United States alone there will be 231,840 new cases of invasive breast cancer; 24,590 new cases of gastric cancer; 74,000 new cases of bladder cancer; 22,850 new cases of brain/spinal cancer; 48,960 new cases of pancreatic cancer; 21,290 new cases of ovarian cancer; and 207 new cases of pediatric osteosarcoma. HER2 expression is associated with more aggressive disease, increased risk of relapse and decreased overall survival, and is an important target for immunotherapy.

About ADXS-HER2

ADXS-HER2 is an Lm Technology immunotherapy product candidate being developed by Advaxis to target HER2 expressing cancers. ADXS-HER2 has received orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of osteosarcoma. Advaxis is developing ADXS-HER2 for both human and animal health, and has seen encouraging data in canine osteosarcoma, which is considered a model for human osteosarcoma. Advaxis has licensed ADXS-HER2 and three other immunotherapy constructs to Aratana Therapeutics, Inc. for the development of pet therapeutics.