European Commission Approves Additional Indication for VELCADE (bortezomib) in Mantle Cell Lymphoma

On February 6, 2015 Janssen-Cilag International NV (Janssen) announced today that the European Commission has approved a variation to the terms of the marketing authorisation of VELCADE (bortezomib) in combination with rituximab, cyclophosphamide, doxorubicin and prednisone for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are unsuitable for blood stem-cell transplantation (Press release Johnson & Johnson, FEB 6, 2015, View Source [SID:1234501514]).

The decision from the European Commission follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on 18 December 2014.[1] This approval allows for the marketing of VELCADE for the above indication in all 28 countries of the European Union (EU). The approval of VELCADE in MCL is based on data from the Phase 3 study, LYM-3002.[2]

In the European Union, VELCADE is currently indicated for the treatment of multiple myeloma (MM), another rare blood-based cancer, either as monotherapy or in combination with other treatment regimens.[3]

MCL is considered a rare and aggressive type of blood cancer, which can be challenging to treat and is associated with a poor prognosis.[4],[5]

“We are delighted that the European Commission has approved extending the indications for use of VELCADE to include first-line therapy for patients with mantle cell lymphoma. We already offer IMBRUVICA as a second-line treatment in MCL and are pleased to be able to provide additional treatment options for this disease to patients and physicians,” said Thomas Stark, Vice President, Medical Affairs, Janssen Europe, Middle East and Africa (EMEA).

Study Efficacy Results

LYM-3002 was a randomised, open-label, active-controlled, multicentre, international, prospective Phase 3 study including 487 patients with newly diagnosed MCL who were ineligible, or not considered, for bone marrow transplantation.

The results showed significant benefits when treating patients with MCL using a VELCADE-based combination (VR-CAP[*]), compared to a widely used standard of care combination (R-CHOP[†]) that did not include VELCADE.[2] The VR-CAP regimen significantly improved progression-free survival (PFS), the primary endpoint, and showed improvements across a range of secondary endpoints.[2] An independent review committee reported the increase in PFS to be 59 percent (median 24.7 vs. 14.4 months; HR 0.63; p<0.001), whereas the study investigators reported the increase in PFS to be 96 percent (median 30.7 vs. 16.1 months; HR 0.51; p<0.001).[2] Study Safety Results VR-CAP was associated with additional, but manageable, toxicity when compared to R-CHOP.[2] Overall, among patients receiving VR-CAP compared to R-CHOP, serious adverse events (AE) were reported in 38 percent vs. 30 percent of patients and grade ≥3 AEs were reported in 93 percent vs. 85 percent. Discontinuations of treatment due to AEs were nine percent (VR-CAP) vs. seven percent (R-CHOP) and on-treatment drug-related deaths were two percent vs. three percent.[2] About VELCADE (bortezomib) The European Commission approval of the variation to the terms of the marketing authorisation means that VELCADE is now licensed in the EU to treat mantle cell lymphoma (MCL), a blood-based cancer, in untreated adults who are unsuitable for blood stem-cell transplantation. For MCL, VELCADE is used in combination with rituximab, cyclophosphamide, doxorubicin and prednisone.[1] VELCADE is also currently approved in the EU for the treatment of MM, another rare blood-based cancer, for the following groups of patients:[3] Previously untreated adults who are unsuitable for high-dose chemotherapy with a blood stem-cell transplant. In these patients, VELCADE is used in combination with melphalan and prednisone; Previously untreated patients who are going to receive high-dose chemotherapy followed by a blood stem-cell transplant. In this group of patients, VELCADE is used in combination with dexamethasone, or with dexamethasone plus thalidomide; Adults whose disease is getting worse after at least one other treatment and who have already had, or cannot undergo, a blood stem-cell transplant. VELCADE is either used on its own in these patients or in combination with pegylated liposomal doxorubicin or dexamethasone. VELCADE contains an active substance called bortezomib and is the first in a specific class of medicines known as proteasome inhibitors. Proteasomes are present in all cells and play an important role in controlling cell function, growth and also how cells interact with other cells around them. Bortezomib reversibly interrupts the normal working of cell proteasomes, inducing the cancerous cells, to stop growing and die.[3] VELCADE has a predictable safety profile and a favourable benefit-risk ratio, which can be used in elderly patients and people with mild to moderate renal impairment.[6] The most common side effects reported with VELCADE (bortezomib)include fatigue, gastrointestinal adverse events, transient thrombocytopenia and neuropathy.[3] VELCADE is commonly used in the treatment of various stages of MM. The product is co-developed by Millennium, the Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, and Janssen Pharmaceutical Companies. Millennium is responsible for commercialisation of VELCADE in the U.S.; Janssen Pharmaceutical Companies are responsible for commercialisation in Europe and the rest of the world. Takeda Pharmaceutical Company Limited and Janssen Pharmaceutical K.K. co-promote VELCADE in Japan. VELCADE is approved in more than 90 countries and has been used to treat more than 550,000 patients worldwide.

10-Q – Quarterly report [Sections 13 or 15(d)]

ImmunoGen has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing 10-Q , ImmunoGen, FEB 5, 2015, View Source [SID1234501489]).

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RXi Pharmaceuticals Announces Closing of the Exclusive Global Licensing Agreement to Samcyprone™ with Hapten Pharmaceuticals

On February 5, 2015 RXi Pharmaceuticals reported the closing of its previously disclosed exclusive global license agreement for the therapeutic use of Samcyprone with Hapten Pharmaceuticals, LLC (Press release, RXi Pharmaceuticals, FEB 5, 2015, View Source;FID=27469217 [SID:1234503089]).

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Under the terms of the license agreement, Hapten received a one-time upfront cash payment of $100,000 and 200,000 shares of RXi common stock. Hapten will also be entitled to receive future milestone payments tied to the achievement of certain clinical and commercial objectives, such as the enrollment of the first patient in a Phase 3 clinical trial and regulatory approval, and escalating royalties based on product sales.

Synta Outlines New Corporate Strategy

On February 5, 2015 Synta Pharmaceuticals reported an outlined a new corporate strategy aimed at focusing the Company’s resources on achieving key value creating milestones in 2015 and 2016, and transforming the organization into a leading oncology biopharmaceuticals company (Press release Synta Pharmaceuticals, FEB 5, 2015, View Source;p=RssLanding&cat=news&id=2014219 [SID:1234501488]).

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Synta’s core strengths in oncology research and development are exemplified by the discovery and rapid development of ganetespib, its Phase 3, novel, potent, small molecule inhibitor of Heat Shock Protein 90 (Hsp90), and its Hsp90 Drug Conjugate (HDC) platform, which includes a broad library of potential product candidates. As part of its new strategy, the Company will align its talent and resources around these core strengths, focusing on several key areas:

Maximizing the value of ganetespib for patients and shareholders

Synta will focus its clinical development activities on ganetespib. Ganetespib is in development for the treatment of a broad range of cancers, with its lead program in non-small cell lung cancer (NSCLC) in the company sponsored pivotal Phase 3 GALAXY-2 clinical trial. Interim analysis from this study is expected in the second half of 2015 and final analysis is expected in the first half of 2016. In addition, ganetespib is also in a Phase 3 clinical trial for acute myeloid leukemia (AML), and Phase 2 clinical trials for ovarian and breast cancer, each sponsored through cooperative groups. As part of its development plan for ganetespib, Synta is exploring certain biomarkers, which it expects may guide future development of the product, pending confirmation of their prognostic and predictive potential via results of ongoing studies.

Advancing candidates from our Hsp90 Drug Conjugate (HDC) platform into the clinic

Synta has initiated IND-enabling studies for its lead HDC drug candidate, STA-12-8666, with an expectation to file an Investigational New Drug (IND) application by the first quarter of 2016. STA-12-8666 is a conjugate of an Hsp90 inhibitor bound to SN-38, the active metabolite of irinotecan. STA-12-8666 has demonstrated significant activity in patient derived xenograft (PDX) models of pancreatic cancer and small cell lung cancer. STA-12-8666 also showed improved tolerability compared to irinotecan in preclinical models. In addition to STA-12-8666, Synta expects to identify one additional HDC drug candidate to nominate for preclinical development by the end of 2015. Synta intends to continue pursuing partnerships and business development activities to advance other candidates within its HDC platform.

Optimizing our pipeline and research agenda

Synta performed a comprehensive review of the Company’s drug candidate portfolio, development programs and research agenda in order to optimize the allocation of its limited resources. This review has led to a rationalization of the Company’s portfolio and research activities, demonstrated by the divestiture of Synta’s IL-12/23 inhibitor program and its CRAC ion channel inhibitor program in 2014. As part of this effort, the company reduced its headcount in 2014 by a total of 20 people and reallocated resources to increase support of the ongoing ganetespib development program and HDC platform. Going forward, Synta intends to build its pipeline through a focused, internal research agenda, complemented by external partnerships and business development activities. To support the future pipeline development and research agenda, Synta has named Dr. Neil Spector, Sandra P. Coates Chair Breast Cancer Research, Duke University Medical Center, Scientific Advisor to the Company. Dr. Spector will retain his full-time faculty appointment and research/clinical responsibilities at Duke, while dedicating a percentage of his consulting time to Synta activities.

Adapting our structures to achieve our goals and strengthen our balance sheet

Synta is adapting its operating model to better suit its future strategy needs. Rather than focusing primarily on building internal capabilities across discovery, development and commercialization, this new model will reflect a leaner, more agile organization that leverages internal strategic capabilities with the expertise of external capabilities, as-needed.

As part of this change in Synta’s operating model, the company announced today a reduction in force of approximately 20%. Synta expects to realize cost savings from this downsizing in 2015. On a cumulative basis, headcount has been reduced from 133 at the beginning of 2014 to 90 today. The company will continue to seek productivity improvements and increased operating efficiencies as the new operating model for the organization is put in place. This will include a consolidation of Synta’s office and laboratory facilities in 2016.

Upcoming Milestones

The company plans to achieve the following key milestones by the end of 2016:

Completion of interim analysis of GALAXY-2 in 2H 2015 with final analysis in 1H 2016
Pending a successful outcome of GALAXY-2, filing of a New Drug Application (NDA) for ganetespib in NSCLC in 2016
Submission of an IND for STA-12-8666 by Q1 2016
Initiating IND enabling studies for an additional HDC drug candidate in 2016

Anne Whitaker, President and Chief Executive Officer of Synta, commented, "Our ambition is to bring to market novel cancer medicines to treat patients battling cancer by leveraging our internal strengths in oncology research, development and commercialization, as well as external relationships with academia, investigators, and partners. The next several quarters hold tremendous potential for bearing out the value of our pipeline. The structural changes we are making within our organization, while difficult decisions, are necessary to help us realize this potential. We are extremely grateful for the contributions of those who will be leaving the Company, and we all wish them much success in their future endeavors."

Dendreon Enters into Amended Agreement with Valeant in Connection with Court-Supervised Sales Process

On February 5, 2015 Dendreon reported that it has entered into an amended agreement with Valeant Pharmaceuticals International in connection with the previously announced court-supervised sales process (Press release Dendreon, FEB 5, 2015, View Source [SID:1234501485]). Under the terms of the amended agreement, subject to bankruptcy court approval, Valeant would acquire the world-wide rights of PROVENGE (sipuleucel-T) and certain other Dendreon assets for $400 million, subject to higher and better bids.

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As previously announced, the bid deadline for interested parties to submit qualified bids to participate in an auction for the Company’s assets is scheduled for February 10, 2015 at 5:00 p.m. Eastern Time. Assuming additional qualified bids are submitted, an auction would be held on February 12, 2015. Under the terms of the amended agreement, Valeant will serve as the "stalking horse" bidder in conjunction with the court-supervised auction.

The Company is operating in the ordinary course and continues to produce and deliver PROVENGE without interruption.