US Food and Drug Administration Approves Signifor® Long-Acting Release (LAR) (Pasireotide) for Injectable Suspension
Dec 16 14
Novartis announced that the US Food and Drug Administration has approved Signifor® long-acting release (LAR) (pasireotide) for injectable suspension, for intramuscular use, for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option. The approval of Signifor LAR, a next-generation somatostatin analog (SSA), helps address a critical unmet need among the acromegaly patient population. Signifor LAR has been studied and found effective in both medically naïve patients with acromegaly who have had prior surgery or for whom surgery was not an option, as well as patients whose disease is not fully controlled on first generation SSAs2. Acromegaly is a rare, debilitating endocrine disorder caused by the excess production of growth hormone (GH) and insulin-like growth factor-1 (IGF-1). In the majority of cases, the disease is caused by a non-cancerous tumor on the pituitary gland. Prolonged exposure to GH and IGF-1 may cause patients to experience extreme physical changes including the enlargement of hands, feet and facial features1. Acromegaly is also associated with two- to three-fold increased mortality rates and serious health complications, including heart disease, hypertension, diabetes, arthritis and colon cancer1,4,5. In fact, heart disease is responsible for approximately 60% of deaths among people with acromegaly6. This FDA approval was based on two multicenter Phase III studies, C2305 and C2402, which respectively examined medically naive patients who have had prior surgery or for whom surgery was not an option and patients with acromegaly inadequately controlled on first generation SSAs. In both studies, higher rates of full biochemical control defined as mean GH level The C2305 study was a multicenter, randomized, double-blind study in patients with active acromegaly who were not previously treated with medication (medically naïve), and had persistent disease despite prior surgery or were ineligible for surgery.
BioLineRX Signs Novartis Strategic Cooperation Deal
Dec 16 14
BiolineRX Ltd. signed a strategic business cooperation agreement with pharmaceutical multinational Novartis. Under the agreement, Novartis will invest $10 million in BioLineRX at a 22% premium on 's market price, for a 12.8% stake, and the two companies will search for joint development projects in Israel. BioLineRX's share price shot up following the announcement and trading had to be suspended in the share. Under the plan, three projects will be selected and developed by BioLineRX, and Novartis will have the option of developing them in cooperation with BioLineRX. Exercising the option will involve a $5 million payment, plus half of the project's development cost, which will be provided as an investment in BioLineRX, also at a premium on the market price. BioLineRX VP business development David Malek explains that at the end of the joint development process, Novartis can purchase the rights to these projects on predetermined terms. The search is not restricted to any particular segments in the pharmaceutical industry.
Novartis AG Highlights New CTL019 Clinical Data Showing Complete Remissions in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia
Dec 6 14
Novartis AG announced new CTL019 clinical data showing complete remissions in children and young adults with relapsed/refractory acute lymphoblastic leukemia. Findings from continued clinical studies of investigational chimeric antigen receptor (CAR) therapy, CTL019, demonstrate its potential role in the treatment of certain types of lymphocytic leukemia. In one long-term study of pediatric patients with acute lymphoblastic leukemia (ALL), results showed that 36 of 39 pediatric patients with relapsed/refractory (r/r) ALL, or 92%, experienced complete remissions (CR) with CTL019(). These results, which will be presented in an oral session at the 56th American Society of Hematology (ASH) annual meeting in San Francisco, continue to increase scientific understanding of CTL019 (Abstract #380, December 8, 10:45 AM)() . Additional abstracts will be presented at ASH that evaluate the efficacy and safety of CTL019 in the treatment of B cell cancers including ALL, chronic lymphocytic leukemia (CLL) and B cell non-Hodgkin lymphoma (NHL). Additional highlights of the pediatric r/r ALL study include findings that patients have ongoing CR. Median follow-up was 6 months. Sustained remissions were achieved up to one year or more with 6-month event-free survival of 70% and overall survival of 75%, in most cases without further therapy() . The probability of six-month CTL019 persistence was 68%, which was accompanied by B cell aplasia, a pharmacodynamic marker of CTL019 persistence and function() . Persistence of CTL019 cells detected by flow cytometry and/or qPCR, and accompanied by B cell aplasia, continued for up to 30 months after infusion in patients with ongoing responses. All responding patients developed cytokine release syndrome (CRS) at peak T cell expansion. Treatment for CRS was required for hemodynamic or respiratory instability in 33% of patients and CRS was managed with an IL-6 receptor antagonist, together with corticosteroids in five patients. These events were delayed, and few patients experienced infusional toxicities, including infusion-associated fever. Also included among the presentations at ASH is a study investigating CTL019 in the treatment of individuals with CD19+ B cell lymphomas that reveals complete responses in patients with advanced, relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma. In addition, data will be presented on three cases of refractory cytokine release syndrome (CRS) in adult patients with ALL. Additional CTL019 Highlights at ASH include: Randomized, Phase II Dose Optimization Study of Chimeric Antigen Receptor Modified T Cells Directed Against CD19 (CTL019) in Patients with Relapsed, Refractory CLL. Cytokine Release Syndrome (CRS) after Chimeric Antigen Receptor (CAR) T Cell Therapy for Relapsed/Refractory (R/R) CLL. Humoral Immunity and Plasma Cell Changes in Patients Responding to CD19-Specific Chimeric Antigen Receptor (CAR)-Modified T-cell Adoptive Immunotherapy. Novel Chimeric Antigen Receptor T cells for the Treatment of CD19-negative Relapses Occurring after CD19-targeted Immunotherapies. Novel Chimeric Antigen Receptor T Cells for the Treatment of Hodgkin Lymphoma. CTL019 uses CAR technology to reprogram a patient's own T cells to "hunt" cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient's blood; they proliferate and bind to the targeted CD19+ cancer cells and potentially kill these tumor cells. Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world. After CTL019 infusion, cytokine release syndrome (CRS) occurs when the engineered cells become activated and multiply in the patient's body resulting in the release of cytokines. During CRS, patients typically experience varying degrees of flu-like symptoms with high fevers, nausea, muscle pain, and in some cases, low blood pressure and breathing difficulties. CRS severity correlates with disease burden. Additionally, CRS also can occur in other non-CAR therapy settings including some monoclonal antibodies.