AVEO Receives Confirmation of Eligibility for Submission of a Tivozanib Marketing Authorization Application to the European Medicines Agency

On January 8, 2015 AVEO Oncology reported that it has received written confirmation from the European Medicine Agency (EMA) that tivozanib is eligible for submission of an application for a European Union Marketing Authorization under the Agency’s centralized procedure (Press release AVEO, JAN 8, 2015, View Source;p=RssLanding&cat=news&id=2004942 [SID:1234501284]). Confirmation of eligibility was given in response to the submission of a letter of intent enabling the Company to evaluate the opportunity for submitting a Marketing Authorization Application (MAA) for tivozanib with the EMA for the treatment of renal cell carcinoma (RCC). Tivozanib is an oral, potent, selective inhibitor of vascular endothelial growth factor tyrosine kinase inhibitor (VEGF TKI) with a long half-life and activity against all three VEGF receptors. Tivozanib has previously been granted orphan drug designation in Europe for the treatment of RCC.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The letter of intent, which must be filed at least seven months prior to submission of a MAA, initiates the process to address a number of pre-submission requirements, including the assignment of a Rapporteur and Co-Rapporteur, who are two appointed members of the Committee for Human Medicinal Products (CHMP). The CHMP is the committee responsible for preparing opinions on questions concerning human medicines. Confirmation of eligibility for submission is not predictive of the EMA’s approval of a MAA.

"Our letter of intent and the subsequent confirmation of eligibility by the EMA are first steps in evaluating the potential for submission of a tivozanib MAA in Europe for the treatment of RCC," said Michael Bailey, president and chief executive officer of AVEO. "AVEO and its global RCC clinical advisors continue to believe that tivozanib has the potential to offer RCC patients an alternative to existing therapies. With the European rights to tivozanib recently regained, we now have the opportunity to work with the EMA to fully evaluate the potential for a MAA submission."

AVEO remains encouraged by the clinical outcomes from its Phase 2 and Phase 3 studies in RCC and the efficacy and tolerability profile that tivozanib may offer to patients suffering from this challenging disease. AVEO recently entered into an option agreement with Ophthotech to investigate tivozanib for the potential treatment of non-oncologic diseases of the eye, and the Company is actively pursuing partnerships to advance the development of tivozanib in solid tumors.

8-K – Current report

On January 6, 2015 Argos Therapeutics reported a collaboration with Saint-Gobain’s Performance Plastics division, a leader in high-performance components and solutions using engineered polymers (Filing 8-K , Argos Therapeutics, JAN 7, 2015, View Source [SID:1234501282]). Under the terms of the agreement, Saint-Gobain will partner with Argos to design, integrate and scale production of a range of disposables for use in the automated manufacturing of Argos’ lead product candidate, AGS-003, currently being tested in a Phase III clinical trial for the treatment of metastatic renal cell carcinoma (mRCC).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We believe Saint-Gobain is the ideal partner to provide us with disposables that meet the technical specifications we need in the manufacturing of our personalized immunotherapies," says Jeff Abbey, president and CEO of Argos. "Their commitment to this development program and to Argos is a critical step in our effort to bring together all of the high quality resources and expertise we need to support the potential future commercialization of AGS-003. The utilization of their disposables with our automated production technology positions us to maximize throughput while processing biomaterials from multiple patients simultaneously in the same automated manufacturing suite."

"Argos’ Arcelis technology platform shows clear potential to support development of a range of autologous cell therapies that could change the future of patient care in cancer and infectious diseases. We are excited about the opportunity to partner with the Argos team to develop and supply the essential range of disposables that will be required to advance AGS-003 through late stage development and on to commercialization," says Steve Maddox, General Manager of Saint-Gobain Performance Plastics’ Life Sciences business unit.

BIND Therapeutics Provides Clinical Update for BIND-014 and 2015 Strategic Overview

On January 7, 2015 BIND Therapeutics reported its 2015 strategic overview and enrollment of the first patient expressing a KRAS mutation in a global, multicenter two-tiered phase 2 trial with BIND-014 in non-small cell lung cancer (NSCLC) patients with KRAS mutant tumors (mutated Kirsten ras oncogene homolog) or squamous histology (Press release BIND Therapeutics, JAN 7, 2015, View Source [SID:1234501285]). The trial was driven by positive results from the phase 2 trial in NSCLC presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Annual Symposium in Barcelona on November 19, 2014, with a confirmed objective response rate of 22 percent (n=9); one KRAS mutant NSCLC PR was also seen in the phase 1 trial with BIND-014, yielding a combined total response rate of 30 percent (n=10). Results from the phase 2 trial also suggested meaningful differentiation in NSCLC patients with squamous histology when compared to historical docetaxel results. BIND-014 results presented at EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) also demonstrated a disease control rate of 66 percent and overall survival of 11.1 months (n=9) in NSCLC patients with squamous histology. BIND intends to begin accruing NSCLC patients with squamous cell histology in the two-tier phase 2 trial in 1Q 2015.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to see these phase 2 data for BIND-014 demonstrating that our Accurin technology is providing on-target anti-tumor activity with meaningful reductions in the incidence and severity of side-effects expected from conventional drugs," said Hagop Youssoufian, M.D., chief medical officer at BIND Therapeutics. "Accordingly, we have devised a development plan in 2nd line NSCLC that we believe allows us to rapidly advance BIND-014 to phase 3. In addition, we are devoting significant resources to the advancement of BIND-014 in carefully selected tumor types with significant unmet need and a strong rationale for BIND-014 activity. Cytotoxic and anti-mitotic agents remain an integral part of therapy for most patients with cancer. We believe that these agents’ proven benefits can be significantly enhanced with our Accurin platform by achieving greater potency and less off-target toxicity. This is in addition to potential applications of the Accurin technology to other targets in cancer and non-cancer therapeutic areas, including many that are intractable with currently available approaches."

The company also announced topline data from its ongoing phase 2 trial with BIND-014 in metastatic castration resistant prostate cancer (mCRPC). The primary endpoint of the study was to determine the efficacy of BIND-014 as measured by radiographic progression-free survival (rPFS) in patients with chemotherapy-naïve metastatic CRPC. The trial enrolled 42 patients, 31 of whom had been treated with androgen inhibitors prior to enrolling in the study. As of December 15, 2014, BIND-014 demonstrated a median rPFS of 8.1 months with three patients currently on treatment and 60 percent of the patients enrolled attained a rPFS of 6 months or greater. Safety and tolerability were also promising, with notable reductions in adverse effects that often limit dosing of conventional docetaxel, including hematologic and non-hematologic toxicities. As of December 15, 2014, three patients continue on treatment and 24 patients continue to be followed for overall survival. Complete results of the mCPRC trial will be presented at an upcoming medical meeting.

"The efficacy and tolerability profile of BIND-014 in mCRPC, particularly in this later stage population with prior androgen inhibitor treatment, serve to reinforce the promise of the Accurin platform," said Scott Minick, chief executive officer, BIND Therapeutics. "Given the rapidly evolving prostate cancer treatment landscape, we believe that there are more promising opportunities at this time for the Accurin platform within our development portfolio and are excited about the opportunities for BIND-014 in NSCLC and other difficult to treat areas."

BIND’s 2015 key business priorities are aimed at supporting the ongoing development of BIND-014 in areas of high unmet need with the potential for high activity, with additional investment in key research, development and collaboration programs.

"We made substantial progress in 2014, including significant advances in our BIND-014 program and exciting new collaborations that further validate and advance our Accurin platform," said Andrew Hirsch, BIND’s chief operating officer. "As we continue our evolution to a multi-product drug development platform company, we expect 2015 to be a particularly active period, with the initiation of additional BIND-014 clinical programs in six indications with data readouts that present multiple opportunities for expedited regulatory review. In addition, we are initiating manufacturing scale-up and investigational new drug-enabling studies for BIND-510, our PSMA-targeted vincristine Accurin, and expecting important collaboration milestones in areas of high unmet need that may benefit from our Accurin technology."

BIND THERAPEUTICS 2015 CLINICAL OVERVIEW:

BIND-014 (PSMA-targeted Accurin docetaxel) in solid tumor malignancies

BIND is currently enrolling patients in a global, multicenter, two-tier phase 2 trial with BIND-014 in patients with KRAS mutant NSCLC, which accounts for approximately 20 percent of all NSCLC, and squamous NSCLC, which accounts for approximately 25 percent of all NSCLC.
KRAS mutant NSCLC: Meaningful differentiation from docetaxel in KRAS mutant NSCLC was seen in the phase 1 and 2 studies, with an objective response rate of 30 percent seen across both studies. There are currently no treatments specifically approved for KRAS mutant NSCLC and anticipated enrollment in this study is 40 patients, with preliminary data anticipated as early as 2H 2015.
Squamous histology NSCLC: Meaningful differentiation from docetaxel in squamous histology NSCLC was seen in the phase 2 study, with disease control rate of 66 percent and interim overall survival of 11.1 months as of October 23, 2014. BIND anticipates enrolling approximately 40 patients, with first patient enrollment in 1Q 2015 and preliminary data in late 2015.
BIND expects to report final overall survival data from its ongoing phase 2 trial with BIND-014 in the NSCLC broad patient population in 1H 2015. Phase 2 data to date demonstrate substantial clinical activity as monotherapy in advanced 2nd line NSCLC patients, with 15 percent partial response rate (n= 40; 5 confirmed, 1 unconfirmed) and meaningful reduction of docetaxel-related toxicities. The increased anti-tumor activity at a lower dose than conventional docetaxel provides important validation of the Accurin platform.
BIND is initiating global, multicenter, two-stage, phase 2, multi-tumor trial with BIND-014 in patients with cholangiocarinoma, cervical cancer, bladder cancer and head and neck cancers, with anticipated first patient enrollment in 2Q 2015. These areas of high unmet need offer potential opportunities for more rapid development with anticipated stage 1 data readout in early 2016.
Topline phase 2 data from BIND-014 in mCRPC demonstrate promising efficacy and tolerability profile, with rPFS of 8.1 months as of December 15, 2014 and significant reductions in adverse effects that often limit dosing of conventional docetaxel. These data further serve to validate the Accurin platform. BIND anticipates overall survival data from the ongoing phase 2 trial of BIND-014 in mCRPC in 1H 2015 with full data presentation at an upcoming medical meeting.

BIND-510 (Accurin vincristine) in solid and hematological malignancies

BIND plans to initiate investigational new drug (IND)-enabling toxicology studies and manufacturing scale-up for its second proprietary product candidate, BIND-510, in 1H 2015. Based on encouraging preclinical results, BIND plans to file an IND application with the U.S. Food and Drug Administration (FDA) for BIND-510 in 2016.

BIND THERAPEUTICS BUSINESS OVERVIEW:

In December 2014, BIND achieved a development milestone as part of its global collaboration agreement with Pfizer Inc. to develop and commercialize Accurins utilizing select small molecule targeted therapies. The collaboration aims to employ BIND’s Medicinal Nanoengineering platform to impart tissue and cellular targeting to molecularly targeted drugs. Pfizer has responsibility for development and commercialization of the selected Accurins. BIND received an upfront payment of $4.0 million in 2013 and has the potential to receive payments up to $89.5 million upon the achievement of specified development and regulatory events. BIND may also receive additional payments up to $110 million for specified commercial events as well as royalties in the low-single to high-single digit percentages on potential future sales of each Accurin commercialized, if any.
BIND has initiated initial formulation efforts with Merck’s novel polo-like kinase 1 (PLK1) and kinesin spindle protein (KSP) inhibitors, and BIND expects to apply its Accurin technology which it believes has the potential to preserve the strong preclinical activity of these compounds while reducing observed toxicity issues. The collaboration agreement includes the option to incorporate additional Merck compounds in the future. BIND will fund and conduct research and development activities to advance Accurin product candidates based on these agents through first-in-human clinical studies, after which Merck and BIND will alternate in choosing whether or not to further develop and commercialize the Accurin products. If BIND opts in, in most scenarios there will be no payments made to Merck beyond a royalty on future product sales. If Merck opts in, it will pay BIND a fee based on a multiple of BIND’s research and development expenses, plus a royalty on future product sales.
BIND’s collaboration with AstraZeneca has achieved positive results with the Accurin nanoparticle AZD2811, demonstrating improved efficacy and enhanced trafficking to tumor sites in multiple preclinical models while displaying minimal bone marrow toxicity. AZD2811 is an Accurin containing AZD1152-hQPA, the active metabolite of the prodrug barasertib (AZD1152), a potent and selective inhibitor of aurora B kinase. BIND and AstraZeneca are working toward an IND application with the U.S. FDA for AZD2811. AstraZeneca has the exclusive right to lead development and commercialization and BIND will lead manufacturing during the development phase. BIND received an upfront payment of $4.0 million in 2013 and has the potential to receive payments totaling up to $193 million for specified clinical, regulatory and commercial milestones. BIND will also receive tiered single to double-digit royalties on future sales, if any.
BIND expects that one of its collaboration partners will file an IND application with the U.S. FDA for an Accurin therapeutic by mid-2015.
BIND ended 2014 with approximately $41 million (unaudited) in cash, cash equivalents and short-term investments.

Exelixis and Swedish Orphan Biovitrum AB (Sobi) Extend and Restructure Distribution Agreement for COMETRIQ® for Medullary Thyroid Cancer

On January 7, 2015 Exelixis reported that it has extended and restructured its agreement with Swedish Orphan Biovitrum AB (Sobi) to support the distribution and commercialization of COMETRIQ (cabozantinib) for progressive, unresectable, locally advanced or metastatic medullary thyroid cancer (MTC) in the European Union (EU), Switzerland, Norway, Russia, and Turkey (Press release Exelixis, JAN 7, 2015, View Source;p=RssLanding&cat=news&id=2004606 [SID:1234501288]). The agreement, which was established in February 2013 and due to expire on December 31, 2015, will now extend to December 31, 2019. Moreover, the payment structure of the partnership will transition from fixed fees paid by Exelixis to Sobi to support initial build out of COMETRIQ European commercial infrastructure to a sales margin-based approach. Exelixis continues to maintain commercial rights for all other potential cabozantinib oncology indications on a global basis.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our amended agreement with Sobi will continue to allow Exelixis to provide COMETRIQ to MTC patients outside the United States," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "Over the course of this coming year, we anticipate reaching several key milestones for the company, including receipt of top-line data from the METEOR phase 3 trial of cabozantinib in metastatic renal cell carcinoma in the second quarter, and regulatory progress with our partnered compound cobimetinib for metastatic melanoma in both the United States and European Union."

"We are very pleased to extend the agreement with Exelixis and proud to support access to COMETRIQ in Europe. The evolution of our partnership with Exelixis will continue to bring significant value to patients," said Anders Edvell, Vice President and Head of Sobi Partner Products at Sobi.

Sobi exclusively markets, sells, and distributes COMETRIQ for its MTC indication in the covered territory of the European Union, Switzerland, Norway, Russia, and Turkey. In parts of the territory where COMETRIQ is not approved, Sobi administers a Named Patient Use program. Exelixis is responsible for regulatory approvals in the covered territory, and the company retains the ability to terminate the agreement at will at any time upon payment of certain pre-determined fees.

Five-Year Collaboration Focused on CART and Hematopoetic Stem Cell Applications across a Range of Therapeutic Areas

On January 7, 2015 Intellia Therapeutics, a leader in the development of therapeutic products using CRISPR/Cas9 technology for gene editing and repair, reported a five-year research and development collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells (CARTs) and hematopoetic stem cells (HSCs) (Press release, Intellia Therapeutics, JAN 7, 2015, View Source [SID1234533042]). This collaboration comes only three months after Intellia was launched by Atlas Venture and Caribou Biosciences, providing an important validation of Intellia’s team and capabilities.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications, including blood disorders, cancer and other genetic based diseases. It has been shown to be an efficient and precise method for gene editing across multiple cell and tissue types, making it an ideal platform for ex vivo applications, such as CART- and HSC-based therapies, as well as in vivo applications.

Under the terms of the agreement, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CARTs. Within HSCs, Novartis and Intellia will jointly advance multiple programs, and have agreed to a process for assigning development and ownership rights, which will enable Intellia to develop its own proprietary internal HSC pipeline.

In addition to increasing its equity holding in Intellia, Novartis is making an upfront payment, and providing technology access fees and funding for R&D programs during the five-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline. Intellia also reserves the right to pursue additional enabling partnerships in other areas of therapeutic interest.

"Our collaboration with Novartis is an important building block for Intellia that will greatly accelerate our effort to translate the promise of CRISPR/Cas9 into meaningful advances for 2 patients," said Nessan Bermingham, Ph.D., Chief Executive Officer and co-founder of Intellia. "CARTs and HSCs represent two of the most immediate opportunities for CRISPR therapeutic development, and Novartis, as a leader in this space, is the ideal partner with which to develop strong product pipelines in these areas."