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Closing of Second Financing

On October 23, 2015 SYNIMMUNE GmbH reported that it has closed a second financing round with its existing investors German Kreditanstalt für Wiederaufbau (KfW) and Synimmune Equity Ltd (Press release, Synimmune, OCT 23, 2015, View Source [SID1234552071]). The Company will establish an Advisory Board, which will be comprised of Michael Kring, CEO of High Tech Corporate Services, as Chairman, and of Prof. Hans-Georg Rammensee, Founder of SYNIMMUNE GmbH and Head of the Department of Immunology at the University of Tuebingen and a representative of Synimmune Equity. Dr. Martin Steiner, independent consultant for biotechnology companies and former founder and CEO of two biotechnology companies will join SYNIMMUNE as General Manager and CEO.

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U.S. FDA Approves YONDELIS® (trabectedin) for Soft Tissue Sarcoma (Liposarcoma or Leiomyosarcoma)

On October 23, 2015 PharmaMar reported that its licensing partner, Janssen Biotech, Inc. received marketing approval for YONDELIS (trabectedin) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with unresectable or metastatic liposarcoma (LPS) or leiomyosarcoma (LMS) who have received a prior anthracycline-containing regimen (Press release, PharmaMar, OCT 23, 2015, View Source [SID:1234507897]). LPS and LMS are among the most common types of soft tissue sarcoma and this is the first treatment to be specifically approved for LPS in the U.S.

The approval was based on the clinical efficacy and safety data from a recently published Phase 3, randomized, open-label, controlled study, ET743-SAR-3007, which evaluated YONDELIS versus dacarbazine in this patient population. This pivotal trial confirmed the results of previous clinical studies and provides strong evidence of the clinical benefit of trabectedin.

"Since YONDELIS was first approved in Europe in 2007 approximately 50,000 patients in 80 countries have benefited from this therapy across all indications," says Luis Mora, Managing Director, PharmaMar, who added that "the approval in the U.S. will allow more patients with this disease to have access to a drug that will address an unmet medical need."

For the approval of YONDELIS in the U.S., PharmaMar will receive the appropriate milestone from Janssen Products, LP.

About Liposarcoma (LPS) or Leiomyosarcoma (LMS)
LPS and LMS represent approximately 35% of all STS cases, of which there are 50 subtypes. LMS is an aggressive type of STS that occurs in smooth muscles, such as those in the uterus, abdominal cavity, or blood vessels. LPS originates in fat cells and most commonly occurs in the thigh and abdominal cavity, though it can occur in fat cells in any part of the body.

About YONDELIS (trabectedin)
YONDELIS (trabectedin) is a synthetically produced anti-tumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. It works by targeting the transcription machinery and impairing DNA repair in cancer cells, thus inducing tumor cell death. It is approved in 80 countries in North America, Europe, South America and Asia. Indications vary by country and include the treatment of advanced soft tissue sarcomas and relapsed ovarian cancer in combination with DOXIL/CAELYX (doxorubicin HCl
liposome injection). Under a licensing agreement with PharmaMar, Janssen Products, L.P. has the rights to develop and sell YONDELIS globally except in Europe, where PharmaMar holds the rights, and in Japan, where PharmaMar has granted a license to Taiho Pharmaceutical Co., Ltd.

CBMG to Present Phase IIa Results from CAR-T CD20 Immuno-Oncology

On October 23, 2015 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), a biomedicine firm engaged in the development of effective treatments for degenerative and cancerous diseases, reported that it would provide a meaningful update on a Phase IIa Trial of it’s CD20 Chimeric Antigen Receptor T-cell (CAR-T) therapy in Advanced, Refractory or Relapsed Diffuse Large B-Cell Lymphoma (DLBCL) at the upcoming 4th International Conference on Translational Medicine to be held on October 26-28, 2015 in Baltimore, Maryland, USA (Press release, Cellular Biomedicine Group, OCT 23, 2015, View Source [SID:1234507846]).

Title: Treatment of CD20-directed chimeric antigen receptor-modified T cells in patients with relapsed or refractory B-cell Non-Hodgkin’s lymphoma: An early Phase IIa trial report
Scientific Program: October 26, 2015 16:15-16:40 EDT
Location: DoubleTree by Hilton Hotel Baltimore – BWI Airport, Chesapeake Hall
Presenter: Yihong Yao, Ph.D., Chief Scientific Officer, Cellular Biomedicine Group

Full details of the presented data will be available on the Company’s website following the presentation.

Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event

On October 23, 2015 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, reported the phase 3 clinical trial designs for the luspatercept program in myelodysplastic syndromes ("MEDALIST" study) and beta-thalassemia ("BELIEVE" study), phase 1 preliminary results from the ACE-083 program, and its new IntelliTrap drug discovery platform (Press release, Acceleron Pharma, OCT 23, 2015, View Source [SID:1234507775]).

"We are extremely excited to present the plans for Acceleron’s first phase 3 clinical trials, the unprecedented increases in muscle mass demonstrated in the ACE-083 phase 1 clinical trial supporting its advancement into phase 2 trials next year and our new IntelliTrap drug discovery platform which is already generating promising new therapeutic candidates such as ACE-2494," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Acceleron is making great strides across its entire pipeline from our late stage phase 3 programs to our highly productive discovery organization and I am proud of the tremendous progress we are making."

Luspatercept Phase 3 Clinical Trials in Myelodysplastic Syndromes (MDS) and Beta-Thalassemia

Acceleron announced that the phase 3 MDS trial will be a double-blind, randomized, placebo-controlled study of luspatercept in 210 very low to intermediate risk MDS patients (the "MEDALIST" study). The primary endpoint is the proportion of patients that become red blood cell transfusion independent (≥ 8 weeks) during the first 24 weeks of the study.

The phase 3 beta-thalassemia trial will be a double-blind, randomized, placebo-controlled study of luspatercept in 300 regularly transfused beta-thalassemia patients (the "BELIEVE" study). The primary endpoint is the proportion of patients with ≥ 33% reduction in transfusion burden from weeks 13 to 24 compared to the 12 weeks preceding treatment.

Both the MEDALIST and BELIEVE studies are planned to begin by the end of the year.

ACE-083 Phase 1 Preliminary Results

Acceleron reported positive top-line data from the phase 1 randomized, double-blind, placebo-controlled, dose-ranging study in healthy volunteers. ACE-083 is designed to selectively increase muscle mass and strength in the muscles in which the drug is administered. The results showed a dose dependent increase in muscle volume, assessed by MRI, with the highest dose level generating a 14% increase in volume of the injected muscle, the rectus femoris, in the thigh.

Based on these exciting results, Acceleron announced it intends to advance ACE-083 into a phase 2 clinical trial in patients with facioscapulohumeral muscular dystrophy in mid-2016.

IntelliTrap Drug Discovery Platform

Acceleron introduced its new IntelliTrap drug discovery platform from which it is creating a large and diverse library of new, selective therapeutic candidates targeting the TGF-beta superfamily. This platform has already generated several new therapeutic candidates including ACE-2494, a systemic muscle therapeutic and the first clinical candidate to emerge from this platform. Acceleron aims to initiate its first clinical trial of ACE-2494 by the end of 2016.

A replay of the live webcast of the Research and Development Day event will be accessible from the "Investors & Media" section of the company’s website, www.acceleronpharma.com.

FDA approves new therapy for certain types of advanced soft tissue sarcoma

On October 23, 2015 The U.S. Food and Drug Administration reported that it has approved Yondelis (trabectedin), a chemotherapy, for the treatment of specific soft tissue sarcomas (STS) – liposarcoma and leiomyosarcoma – that cannot be removed by surgery (unresectable) or is advanced (metastatic) (Press release, , OCT 23, 2015, View Source [SID:1234507774]). This treatment is approved for patients who previously received chemotherapy that contained anthracycline.

According to the National Cancer Institute, STS is a disease in which cancer cells form in the soft tissues of the body, including the muscles, tendons, fat, blood vessels, lymph vessels, nerves and tissues around joints. Liposarcoma and leiomyosarcoma are specific types of STS that occur in fat cells (liposarcoma) or smooth muscle cells (leiomyosarcoma). STS can form almost anywhere in the body, but is most common in the head, neck, arms, legs, trunk and abdomen. In 2014, an estimated 12,000 cases of STS were diagnosed in the United States.

"The treatment of advanced or metastatic soft tissue sarcoma represents a difficult challenge with few effective therapeutic choices available for patients," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Today’s approval of Yondelis provides a treatment option for advanced or metastatic liposarcoma and leiomyosarcoma."

The effectiveness and safety of Yondelis were demonstrated in 518 clinical trial participants with metastatic or recurrent leiomyosarcoma or liposarcoma. Participants were randomly assigned to receive either Yondelis (345 patients) or dacarbazine (173 patients), another chemotherapy drug. Participants who received Yondelis experienced a delay in the growth of their tumor (progression-free survival), which occurred on average about 4.2 months after starting treatment, compared to participants assigned to dacarbazine, whose disease progressed an average of 1.5 months after starting treatment.

The most common side effects among participants who received Yondelis were nausea, fatigue, vomiting, diarrhea, constipation, decreased appetite, shortness of breath (dyspnea), headache, tissue swelling (peripheral edema), a decrease in infection-fighting white blood cells (neutropenia), low blood platelet counts (thrombocytopenia), low red blood cell count (anemia), elevated liver enzymes and decreases in albumin, a protein found in blood.

Yondelis carries a warning alerting health care providers of the risk of severe and fatal blood infections (neutropenic sepsis), muscle tissue breakdown (rhabdomyolysis), liver damage (hepatotoxicity), leakage around the vein or catheter (extravasation), tissue necrosis (breakdown) and heart failure (cardiomyopathy). Patients with known hypersensitivity to trabectedin, a drug used to treat cancer, should not take Yondelis.

Health care providers are also encouraged to advise women of potential risks to a developing fetus when taking Yondelis. Women who are taking Yondelis should not breastfeed.

Yondelis is marketed by Janssen Products of Raritan, New Jersey.