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A collection of recent regulatory approvals and other actions globally

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Mid-February saw Valeant Pharmaceuticals International Inc. announce that the U.S. Food and Drug Administration (FDA) had approved the Biologics License Application (BLA) for Siliq (brodalumab) injection—a monoclonal antibody that targets the IL-17 receptor for patients with moderate-to-severe plaque psoriasis—for subcutaneous use. Siliq is indicated for the treatment of moderate-to-severe plaque psoriasis in adult patients who are candidates for systemic therapy or phototherapy and have failed to respond or have lost response to other systemic therapies. Valeant expects to commence sales and marketing of the therapeutic in the United States in the second half of 2017.
“We believe Siliq fulfills a significant unmet medical need, and I am proud of our team’s success in developing and bringing to market this treatment for patients with moderate-to-severe plaque psoriasis,” said Joseph C. Papa, chairman and CEO of Valeant. “We are pleased that Siliq will soon be available to help treat the suffering of adults who live with this debilitating, incurable condition, and further our mission to improve people's lives with our healthcare products.”
Siliq has a Black Box Warning for the risks in patients with a history of suicidal thoughts or behavior because suicidal ideation and behavior have been reported in trials. Siliq was approved with a risk evaluation and mitigation strategy involving a one-time enrollment for physicians and one-time informed consent for patients. The most common adverse reactions, however, have been headache, arthralgia, fatigue, oropharyngeal pain and diarrhea. Siliq is contraindicated in patients with Crohn’s disease. Use of Siliq in patients with a chronic infection or a history of recurrent infection should be handled with caution because of the risk of severe infection from use of the drug in such patients. Patients should be evaluated for tuberculosis infection prior to initiating treatment.
Plaque psoriasis is the most common type of psoriasis and is a chronic, noncommunicable skin disease. The disease alters the life cycle of skin cells, causing them to build up rapidly on the surface of the skin. Siliq works by binding to IL-17RA with high affinity, therefore blocking the inflammatory downstream activity of IL-17A, IL-17F, IL-17A/F heterodimer and IL-17E. By targeting the IL-17 receptor, SILIQ prevents skin cells from accumulating. In three clinical studies that have been completed, more than half of patients who used Siliq achieved total skin clearance within a year.
EU approval of Suliqua triggers milestone payment to Zealand
COPENHAGEN, Denmark—Early this year, Zealand Pharma announced a $10-million milestone payment following Sanofi’s announcement of European Commission marketing authorization in Europe for Suliqua, the once-daily titratable fixed-ratio combination of basal insulin glargine 100 units/mL and GLP-1 receptor agonist lixisenatide for the treatment of adults with type 2 diabetes. Suliqua is authorized for use in combination with metformin to improve glycemic control when this has not been provided by metformin alone, metformin combined with another oral glucose-lowering medicinal product or with basal insulin.
“We welcome the approval by the European Commission of Suliqua in the EU. The product will be marketed by Sanofi and the first launch is planned for Q2 2017. We look forward to see this treatment option made available to people with diabetes in the EU, helping to achieve glycemic control without increasing the risk of hypoglycemia and without additional weight gain,” said Britt Meelby Jensen, president and CEO of Zealand. “Following the recent launch of Soliqua 100/33 in the U.S., this is another important milestone for us, which will provide significant revenue to Zealand in many years to come.”
The approval is based on data from two Phase 3studies, LixiLan-O and LixiLan-L, which enrolled more than 1,900 adults with type 2 diabetes worldwide to evaluate the efficacy and safety of the fixed-ratio combination when used in patient populations insufficiently controlled after oral antidiabetic drugs or after basal insulin therapy.
FDA grants Cellectis IND approval for UCART123
NEW YORK—Cellectis, a biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T cells (UCART), has received an Investigational New Drug approval from the U.S. Food and Drug Administration (FDA) to conduct Phase 1 clinical trials with UCART123, the company’s most advanced, wholly owned TALEN gene-edited product candidate, in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). This marks the first allogeneic, “off-the-shelf” gene-edited CAR T cell product candidate that the FDA has approved for clinical trials. Cellectis intends to initiate the trials in the first half of 2017.
UCART123 targets CD123, an antigen expressed at the surface of leukemic cells in AML and tumoral cells in BPDCN. 
“The FDA’s approval of Cellectis’ UCART123 is a major milestone not only for the company but also for the medical community, global biotech and pharmaceutical industries at large,” said Dr. Loan Hoang-Sayag, Cellectis’ chief medical officer. “Cellectis’ allogeneic UCART products have the potential to create an important shift with regard to availability, and cost-effectiveness, to make these therapies widely accessible to patient population across the world.”
“After the National Institutes of Health’s Recombinant DNA Advisory Committee’s unanimous approval of two Phase 1 study protocols for Cellectis’ UCART123 in December 2016, the FDA’s approval of Cellectis’ IND is a new major regulatory milestone achieved, for having UCART123 proceed into clinical development and reaching cancer patients in need,” added Stephan Reynier, chief regulatory and compliance officer for Cellectis.
FDA approves immunotherapy drug for advanced bladder cancer
HOUSTON—Bladder cancer patients with advanced disease have a new option for treatment after the FDA recently approved a drug that launches an immune response against the disease. The FDA approval of the checkpoint inhibitor nivolumab, known commercially as Opdivo, was based on a Phase 2 multicenter clinical trial led by Dr. Padmanee Sharma at MD Anderson Cancer Center.
“Nearly 20 percent of patients had their tumors shrink substantially, with some complete responses, which is a significant improvement over other second-line options for these patients,” Sharma said. 
Under its Breakthrough Therapy designation and Priority Review status, the FDA granted accelerated approval of nivolumab for patients with locally advanced urothelial carcinoma whose disease progresses during or after platinum-based chemotherapy treatment, which remains frontline standard of care.
Onxeo receives USPTO Notice of Allowance
PARIS—Onxeo S.A., a clinical-stage biotechnology company specializing in the development of innovative drugs for the treatment of orphan diseases, in particular in oncology, announced in February that the U.S. Patent and Trademark Office (USPTO) had issued a Notice of Allowance for a new patent (No. 15/232,844)  relating to the company’s candidate AsiDNA, a first-in-class product that prevents tumor cells from repairing their DNA.
AsiDNA is composed of 64 nucleotides consisting of two strands of 32 nucleotides of complementary sequence, with a particular sequence, and is already protected internationally, including in the United States.
This new patent protects any related compounds comprised of between 40 and 400 nucleotides in any sequence, as well as related pharmaceutical composition and related methods for treating cancer. It provides Onxeo with a very broad scope of protection in this class of compounds.
According to the company, the allowance of this patent, a few months after its filing in August 2016, confirms the innovative science behind the AsiDNA  technology, that uses a “bait” to block the tumor DNA repair pathways.
This newly allowed patent will expire in mid-2031. This term could be further extended to 2036 through the patent term extension.
“Obtaining this new patent demonstrates that we are able to generate IP rights over broad claims. This is strategically important for Onxeo as it significantly reinforces our unique competitive position. AsiDNA is a breakthrough technology for the treatment of cancer and the expansion of the intellectual property related to AsiDNA is paramount to realizing its full potential and value to our shareholders,”  commented Judith Greciet, CEO of Onxeo.

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