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U.S. FDA Grants Priority Review for YONDELIS® (trabectedin) for the Treatment of Patients with Advanced Soft Tissue Sarcoma

On February 3, 2015 Janssen Research & Development reported that the U.S. Food and Drug Administration (FDA) has granted Priority Review for the New Drug Application (NDA) for YONDELIS (trabectedin) to treat patients with advanced soft tissue sarcoma (STS), including liposarcoma and leiomyosarcoma subtypes, who have received prior chemotherapy including an anthracycline (Press release Johnson & Johnson, FEB 3, 2015, View Source [SID:1234501451]). Janssen submitted the NDA to the FDA on November 24, 2014.

Priority Review is a designation for a drug that treats a serious condition and may offer major advances in treatment when compared to existing options. A priority review designation means the FDA’s goal is to take action, following the two month period for the validation and acceptance of the NDA, within six months as compared to 10 months under standard review.

The filing is based on the Phase 3 randomized, open-label study ET743-SAR-3007. This trial is evaluating the safety and efficacy of trabectedin versus dacarbazine for the treatment of patients with advanced liposarcoma and leiomyosarcoma, the most common types of STS in adults, in more than 500 patients previously treated with an anthracycline and ifosfamide, or an anthracycline followed by one additional line of chemotherapy. Results of the study will be presented at a future date.

“We are excited the FDA has granted Priority Review for YONDELIS, as it is an important step forward in making this therapy available to physicians and those living with this aggressive disease,” said Peter F. Lebowitz, M.D., Ph.D., Global Oncology Head, Janssen.

CTI BioPharma Announces Independent DMC Recommendation to Continue GOG-0212 Phase 3 Study of OPAXIO™ as Maintenance Therapy in Ovarian Cancer, an NRG Oncology/GOG Study

On February 3, 2015 CTI BioPharma reported that the Gynecologic Oncology Group, now part of NRG Oncology, informed CTI BioPharma that an independent Data Monitoring Committee (DMC) recommended continuation of the GOG-0212 Phase 3 clinical study of OPAXIO (paclitaxel poliglumex) as maintenance therapy in ovarian cancer with no changes following a second of four planned interim analyses for survival and futility (Press release CTI BioPharma, FEB 2, 2015, View Source;p=RssLanding&cat=news&id=2012846 [SID:1234501445]). CTI BioPharma remains blinded to the interim analysis results. GOG-0212 is the largest maintenance study in this setting, having enrolled 1,150 patients.

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The trial is being conducted and managed by the Gynecologic Oncology Group, now part of NRG Oncology, which is one of the National Cancer Institute’s funded cooperative cancer research groups with a focus on the study of gynecologic malignancies.

"We believe that there remains a significant unmet need in keeping a patient’s cancer from returning following initial treatment for ovarian cancer," said James A. Bianco, M.D., CTI BioPharma’s President and Chief Executive Officer. "The GOG-0212 study is designed to investigate whether Opaxio, when used in a maintenance setting in ovarian cancer, could keep these women in remission and potentially extend their lives."

U.S. FDA grants Breakthrough Therapy Designation for Roche's investigational cancer immunotherapy MPDL3280A (anti-PDL1) in non-small cell lung cancer

On February 2, 2015 Roche reported that it has received a second Breakthrough Therapy Designation from the United States Food and Drug Administration (FDA) for its investigational cancer immunotherapy MPDL3280A (anti-PDL1) (Press release Hoffmann-La Roche , FEB 1, 2015, View Source [SID:1234501442]). The designation was granted for the treatment of people with PD-L1-positive (Programmed Death-Ligand 1) non-small cell lung cancer (NSCLC) whose disease has progressed during or after platinum-based chemotherapy (and appropriate targeted therapy for those with an EGFR mutation-positive or ALK-positive tumour).

“Lung cancer is the leading cause of cancer death globally, and we are pleased the FDA has granted breakthrough designation for MPDL3280A in non-small cell lung cancer,’’ said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “We are committed to personalised healthcare, developing medicines like MPDL3280A with companion tests that may help us identify those who may be appropriate candidates for our medicines. ”

This breakthrough therapy designation is based on early results of MPDL3280A in people whose NSCLC was characterised as PD-L1-positive by an investigational test being developed by Roche. All studies of MPDL3280A are prospectively evaluating PD-L1 expression. Some studies will evaluate the medicine regardless of a tumor’s PD-L1 status; other studies are evaluating the medicine only in people whose tumors are characterized as PD-L1 positive.

Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible. The FDA granted the first Breakthrough Therapy Designation for MPDL3280A in metastatic bladder cancer in 2014. Ongoing pivotal studies of MPDL3280A include lung and bladder cancer, and we plan to initiate Phase III studies in additional tumor types this year.

Stemline Therapeutics Announces SL-701 Granted Orphan Drug Designation for the Treatment of Glioma

On January 30, 2015 Stemline Therapeutics reported that SL-701 has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of glioma (Press release Stemline Therapeutics, JAN 30, 2015, View Source [SID:1234501432]).

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SL-701 is an enhanced immunotherapy designed to activate the immune system to kill malignant gliomas, which are aggressive malignancies of the brain that arise in both adults and children. A multicenter Phase 2 clinical trial is currently evaluating the antitumor activity of SL-701 in adult patients with second-line glioblastoma multiforme (GBM), a particularly aggressive type of glioma. An earlier version of the therapy demonstrated clinical activity, including durable complete responses (CRs) and partial responses (PRs) as well as prolonged disease stabilizations and an overall survival signal, in both adults and children with malignant glioma.

"We are pleased with the FDA’s decision to grant Orphan Drug designation to SL-701 as it provides Stemline with a number of benefits through development and commercialization of this novel therapy," noted Eric K. Rowinsky, M.D., Stemline’s Chief Medical Officer and Head of Research and Development. He continued, "GBM is a highly aggressive disease with few effective treatment options and remains a major unmet need. Instead of targeting a single component of the cancer, SL-701 is designed to activate and direct the immune system against multiple targets overexpressed on glioma cells. We are very pleased about the high level of interest in the study from patients and clinicians alike."

OSE Pharma appoints Simbec-Orion to conduct its Tedopi® Phase III international clinical trial in advanced lung cancer patients

On January 29, 2015 OSE Pharma reported that they have entered into a collaboration agreement to conduct the upcoming Tedopi Phase III pivotal trial in HLA-A2 positive advanced non-small cell lung cancer (NSCLC) patients who have failed on previous therapy (Press release, OSE Pharma, JAN 29, 2015, View Source [SID:1234502959]).

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The protocol of this pivotal Phase III trial, which will treat advanced invasive (stage IIIb) or metastatic (stage IV) NSCLC patients who express the HLA-A2 receptor (approximately 45% of the NSCLC population), has been recently approved both by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency’s (EMA). Simbec-Orion will manage this multi-centre, multi-country study involving up to 70 sites and 500 patients in the United States and Europe.

Simbec-Orion has started the feasibility study for the planned study with international clinical experts and patient enrolment is planned for the second half of 2015. Simbec-Orion will be responsible for site selection, patient enrolment, clinical monitoring, data management, statistical analysis and regulatory affairs.

Simbec-Orion has been selected because of its expertise in oncology and rare diseases offering medical and operational strengths, flexibility, commercial insight and a shared commitment to patients. As part of the collaboration agreement, Simbec-Orion has accepted warrants giving right to equity as part of payment for a portion of its fees. OSE Pharma and Simbec-Orion believe that this will closely align both parties’ interests.

"We are delighted to be collaborating with OSE Pharma in this pivotal step prior to registration of OSE Pharma’s Tedopi," said Ronald Openshaw, Chief Executive Officer of Simbec-Orion. "We have combined the development expertise of OSE Pharma with our broad clinical know-how to create a true strategic partnership."

"After an extensive global review of potential clinical research organisations, we selected Simbec-Orion as a strategic partner for our Phase III programme. This partnership provides clinical development support for Tedopi and will help us accelerate in the race for registration of immunotherapies", said Dominique Costantini CEO of OSE Pharma.

"Although pricing was an important consideration in our evaluation process, Simbec-Orion’s understanding of our mission was crucial and weighed heavily in our final decision. We are very pleased to have this team involved in such an important aspect of OSE Pharma’s future. We believe this Phase III study conducted with Simbec-Orion is an important step to validate the results of earlier studies and demonstrate the risk/benefit ratio of Tedopi alone.

"We believe that the combinatorial approach of Tedopi with other immune-oncology therapies on which we are also currently working will enable to deliver synergies and increase the duration of patients’ response."

About OSE Pharma

OSE Pharma is a European cancer immunotherapy company with a multi-epitope technology named Memopi that directs the body’s immune system to generate a specific cytotoxic T response to prevent cancer cell growth.

OSE Pharma’s lead product, OSE-2101, Tedopi combines 10 "neo-epitopes" directed against five tumour associated antigens. In its most advanced application, it is about to enter a pivotal Phase III study in patients with advanced non-small cell lung cancer (NSCLC) who express HLA-A2 and failed first line therapy. Tedopi has orphan drug status in the USA and is considered as personalized medicine in Europe in HLA-A2 positive patients.

OSE Pharma is also planning a new Phase II clinical trial in combination with another immunotherapy treatment in NSCLC.

Tedopi targets five tumour associated antigens (TAA), selected because their presence is linked to a poor prognosis and the severity of various cancers. Tedopi contains ten optimized epitopes, or "neo- epitopes", designed on the binding of HLA-A2 and TCR,. These neo-epitopes generate strong specific T cytotoxic responses that fight cancer and prevent tumour escape..