CHICAGO and RARITAN, NJ, June 1, 2015 – Janssen Research & Development, LLC (Janssen) announced data from the Phase 3 multicenter study SAR3007, which demonstrated a significant improvement in progression-free survival (PFS) with trabectedin (YONDELIS®) compared to dacarbazine in patients with advanced liposarcoma (LPS) or leiomyosarcoma (LMS) previously treated with an anthracycline and at least one additional chemotherapy regimen. SAR3007 is the largest randomized Phase 3 study ever conducted in this patient population. These data were presented today in an oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL.
Soft tissue sarcoma (STS) is a type of cancer originating in the soft tissues that connect, support and surround other body structures, such as muscle, fat, blood vessels, nerves, tendons and the lining of joints., LPS and LMS are among the most common types of STS in adults and represent approximately 40-50% of all STS cases.,
“Advanced soft tissue sarcomas represent a complex set of rare diseases which, when advanced, are life threatening. Our patients need new treatment options that are effective and reasonably well tolerated, as the treatment landscape has been relatively stagnant for decades,” said George Demetri, M.D., Director, Center for Sarcoma and Bone Oncology at the Dana-Farber Cancer Institute and Professor at Harvard Medical School. “In soft tissue sarcomas, disease stabilization is an important metric for evaluating treatment success in patients with advanced disease. The safety data from this trial were consistent with the well-defined adverse events observed in previous clinical trials of trabectedin and in clinical use outside the United States where trabectedin has been approved to treat these aggressive diseases.”
Trabectedin is approved in 77 countries in North America, Europe, South America and Asia under the trade name YONDELIS® for the treatment of advanced STS as a single agent. Janssen submitted a New Drug Application for YONDELIS®to the U.S. Food and Drug Administration on November 24, 2014, which was granted Priority Review on February 3, 2015.
“The data presented at ASCO underscore the value of trabectedin as a potentially important treatment option for patients with LPS and LMS subtypes of soft tissue sarcoma,” said Craig Tendler, M.D., Vice President, Late Development and Global Medical Affairs, Oncology, Janssen. “This latest research underlines our long-standing commitment to patients with advanced soft tissue sarcoma. To date, more than 50,000 patients worldwide have been treated with YONDELIS®, including approximately 3,000 patients in our Expanded Access Program in the United States.”
In this randomized, active-controlled Phase 3 study in patients with advanced LPS or LMS, trabectedin significantly reduced the risk of disease progression or death by 45% compared with those who received dacarbazine (hazard ratio [HR] = 0.550; P< 0.0001; median [M] 4.2 vs. 1.5 months, respectively), with results validated through an audit by independent radiologists. The improved PFS benefit with trabectedin treatment was consistently observed across all clinically relevant subgroups and was further supported by an increased objective response rate (ORR), a longer duration of response (DOR), and a higher clinical benefit response rate as compared to dacarbazine. At the interim analysis for overall survival (OS), the trial had not met the primary endpoint of OS. The study is ongoing to determine the final OS results, which will be presented at a future meeting. The results for the secondary efficacy endpoints are mature.
Safety findings were consistent with the well-characterized safety profiles of both agents, with the most common Grade 3-4 toxicities in the trabectedin versus the dacarbazine groups being decreased absolute neutrophil count (40% vs. 25%), decreased platelets (19% vs. 20%), and transient increases in liver transaminases, including alanine transaminase (ALT) (29% vs. 1%). Drug-related deaths occurred in 2.1% of patients in the trabectedin group versus 0% of patients in the dacarbazine group.
The Phase 3 multicenter study SAR3007 compared trabectedin with dacarbazine in patients with advanced LPS or LMS previously treated with an anthracycline and at least one additional chemotherapy regimen. The primary endpoint is OS and secondary endpoints included PFS, time to progression (TTP), ORR, DOR, symptom severity and safety. Both treatments were administered via an IV infusion every three weeks with the trabectedin dose of 1.5 mg/m2 given over 24 hours versus dacarbazine dose of 1 g/m2 given over 20-120 minutes. Ninety-four percent of study participants were in the United States.
About Soft Tissue Sarcoma
Soft tissue sarcoma is a type of cancer originating in the soft tissues that connect, support and surround other body structures, such as muscle, fat, blood vessels, nerves, tendons and the lining of joints.1,2 In the United States, nearly 12,000 people will be diagnosed and approximately 4,870 are expected to die of soft tissue sarcomas in 2015. Leiomyosarcoma is an aggressive type of soft tissue sarcoma that occurs in smooth muscles, such as those in the uterus, gastrointestinal tract or lining of blood vessels. Liposarcoma 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 novel, multimodal, synthetically produced antitumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. The anti-cancer medicine works by preventing the tumor cells from multiplying and is approved in 77 countries in North America, Europe, South America and Asia for the treatment of advanced soft tissue sarcoma as a single agent, and in 70 countries for relapsed ovarian cancer in combination with DOXIL®/CAELYX® (doxorubicin HCl liposome injection).
Under a licensing agreement with PharmaMar, a wholly owned member of the Zeltia Group, Janssen Products, LP has the rights to develop and sell YONDELIS® globally except in Europe, where PharmaMar SA holds the rights, and in Japan, where PharmaMar has granted a license to Taiho Pharmaceuticals Co., Ltd. If approved in the United States, YONDELIS® would be commercialized by Janssen Biotech, Inc.
About Janssen Research & Development, LLC
At Janssen, we are dedicated to addressing and solving some of the most important unmet medical needs of our time in oncology, immunology, neuroscience, infectious diseases and vaccines, and cardiovascular and metabolic diseases. Driven by our commitment to patients, we develop innovative products, services and healthcare solutions to help people throughout the world. Janssen Research & Development, LLC; Janssen Products, LP; and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson. Please visithttp://www.janssenrnd.com for more information.
Janssen in Oncology
In oncology, our goal is to fundamentally alter the way cancer is understood, diagnosed and managed, reinforcing our commitment to the patients who inspire us. In looking to find innovative ways to address the cancer challenge, our primary efforts focus on several treatment and prevention solutions. These include a focus on hematologic malignancies, prostate cancer and lung cancer; cancer interception with the goal of developing products that interrupt the carcinogenic process; biomarkers that may help guide targeted, individualized use of our therapies; as well as safe and effective identification and treatment of early changes in the tumor microenvironment. Please visit oncology.janssenrnd.com.
 Mayo Clinic. Disease Conditions: Soft tissue sarcoma.
Available at: http://www.mayoclinic.org/diseases-conditions/soft-tissue-sarcoma/basics/definition/con-20033386. Accessed: May 2015.
 American Cancer Society. What is a soft tissue sarcoma? Available at: http://www.cancer.org/cancer/sarcoma-adultsofttissuecancer/detailedguide/sarcoma-adult-soft-tissue-cancer-soft-tissue-sarcoma. Accessed May 2015.
 American Cancer Society. What are the key statistics about soft tissue sarcomas?
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 Toro JR, et al. Int J Cancer. 2006;119:2922-2930.
 American Cancer Society: Cancer Facts and Figures 2015. Atlanta, Ga: American Cancer Society, 2015
 The Liddy Shriver Sarcoma Initiative. An Introduction to Leiomyosarcoma of the Bone and Soft Tissue.
Available at: http://sarcomahelp.org/leiomyosarcoma.html. Accessed: May 2015.
 The Liddy Shriver Sarcoma Initiative. What is Liposarcoma? Available at: http://sarcomahelp.org/liposarcoma.html. Accessed: May 2015.
 University of Rochester Medical Center. Liposarcoma.
Available at: http://www.urmc.rochester.edu/encyclopedia/content.aspx?ContentTypeID=134&ContentID=221. Accessed: May 2015.