Ignyta Unveils “5X5” Goal and Announces 2016 Strategic Priorities

On January 11, 2016 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported its aspirational "5×5" Goal and announced its strategic priorities for 2016 (Press release, Ignyta, JAN 11, 2016, View Source [SID:1234508751]).

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"2015 was yet another transformational year for Ignyta. With the successful acquisition or licensing of six new assets, three of which were either in the clinic or entered the clinic in 2015, Ignyta’s development stage pipeline quadrupled – undergoing an impressive transformation in less than one year," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "Based on our successes in 2015, we believe that Ignyta is uniquely positioned among companies of our size to develop meaningful treatment options for the benefit of cancer patients. We are relentlessly driven by this patient focus, which underlies our previously announced overarching, ‘audacious’ goal of eradicating residual disease in precisely defined cancer patient populations by 2030."

"In 2016, we want to continue to advance our robust pipeline of targeted first-in-class and best-in-class product candidates in pursuit of this goal, and also to more closely tie development successes from our pipeline to this goal," continued Dr. Lim. "That is why we have set an aspirational ‘5×5’ Goal for the company, which means the achievement of five new product or indication approvals in five years, by the end of 2020. This is certainly a very high bar, but we are impatient to make new therapies available to patients, and are building on a strong foundation of four clinical stage assets, three of which have already demonstrated clinical proof-of-concept. We believe that the best way to make meaningful progress in improving the lives of cancer patients is through the pursuit of audacious goals."

Ignyta’s strategic priorities for 2016 will include:

With respect to entrectinib, the company’s novel, orally available, selective tyrosine kinase inhibitor targeting tumors that harbor activating alterations to NTRK1/2/3 (encoding TrkA/TrkB/TrkC), ROS1 or ALK, continuing to initiate new clinical sites and enroll patients in its STARTRK-2 global, potentially registration-enabling clinical trial of this product candidate;

With respect to taladegib, the company’s novel, orally available, hedgehog/ smoothened inhibitor, initiating one or more pivotal Phase 2 clinical trials in advanced basal cell carcinoma, as well as potentially initiating a Phase 1b basket study for patients with other solid tumors harboring a hedgehog pathway alteration;

With respect to RXDX-105, Ignyta’s multikinase inhibitor with potent activity against such targets as RET and BRAF, achieving clinical proof of concept in patients with activating RET alterations and initiating the Phase 1b portion of the ongoing clinical trial in patients with solid tumors harboring RET or BRAF alterations;

With respect to RXDX-107, the company’s new chemical entity comprising an alkyl ester of bendamustine encapsulated in human serum albumin to form nanoparticles, completing the Phase 1a portion of the ongoing clinical trial and identifying the recommended Phase 2 dose for this product candidate;

With respect to RXDX-106, the company’s potent, pseudo-irreversible inhibitor of TYRO3, AXL and Mer (TAM) and cMET with potential application in immuno-oncology settings, filing an IND to begin clinical evaluation of this product candidate; and
With respect to Ignyta’s other programs, conducting all activities necessary to advance them toward the clinic, as warranted by the data that Ignyta generates.

Eagle Pharmaceuticals and AMRI Announce Agreement to Jointly Develop, Manufacture and Commercialize Parenteral Drug Products

On January 11, 2016 Eagle Pharmaceuticals ("Eagle" or the "Company") (NASDAQ:EGRX) and AMRI (NASDAQ:AMRI) reported that they have entered into an agreement to jointly develop and manufacture several select and complex parenteral drug products for registration and subsequent commercialization in the United States, which will significantly expand Eagle’s portfolio of existing products and product candidates targeting therapeutic areas including oncology, critical care and orphan diseases (Press release, Eagle Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508715]).

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Under terms of the agreement, AMRI will develop and initially provide cGMP manufacturing and analytical support for the registration of the new product candidates. Eagle will be responsible for advancing the product candidates through clinical trials and regulatory submissions.

AMRI will be reimbursed certain costs for formulation activities, process and analytical development and manufacture of regulatory submission batches. Following U.S. Food and Drug Administration ("FDA") approval, AMRI will supply the products to Eagle pursuant to a commercial supply agreement and receive payments based on Eagle’s sales of the products. Eagle will be responsible for U.S. commercial distribution of all approved products, once approved by the FDA.

"As we commercialize our FDA approved products and bring additional products pending approval to market in 2016, this agreement will allow Eagle to expand our portfolio of injectable therapies in a focused and efficient manner by coupling our internal expertise in clinical, regulatory and commercial execution with AMRI’s unique depth of experience with aseptic formulation development, working with complex Active Pharmaceutical Ingredients (API) and handling highly-specialized manufacturing requirements. With this agreement, we expect to more efficiently scale our development infrastructure and speed our market entry with new products, ultimately building long term value for customers and shareholders alike," stated Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals.

"We are very pleased to partner with Eagle as they look to expand their portfolio of innovative products that address unmet needs. We are very impressed with what Eagle is preparing to bring to market and look forward to playing a key role in the advancement of their product portfolio," said Bill Marth, AMRI’s President and CEO.

Infinity Provides Key 2016 Goals and Financial Guidance

On January 11, 2016 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported 2016 anticipated milestones for duvelisib, an oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, and provided financial guidance for 2016 (Press release, Infinity Pharmaceuticals, JAN 11, 2016, View Source;p=RssLanding&cat=news&id=2128081 [SID:1234508752]). Infinity expects to report topline data from DYNAMO, a Phase 2 study of duvelisib in patients with refractory indolent non-Hodgkin lymphoma (iNHL), early in the third quarter of 2016. Infinity also anticipates completing an interim analysis of DUO, a Phase 3 study of duvelisib in patients with relapsed/refractory chronic lymphocytic leukemia (CLL), in the second half of 2016. The company expects marketing applications, if supported by these data, to be submitted to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in the fourth quarter of 2016. These updates were made in conjunction with the 34th Annual J.P. Morgan Healthcare Conference that begins today in San Francisco. Infinity’s chair, president and chief executive officer, Adelene Perkins, will discuss the company’s continued execution of its corporate strategy and 2016 priorities as part of a live presentation on Wednesday, January 13, at 2:00 p.m. PT (5:00 p.m. ET). The presentation will be webcast on Infinity’s website, www.infi.com.

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"In 2016, we are focused on three key strategic objectives designed to bring duvelisib to patients: submitting applications for regulatory approval based on anticipated positive duvelisib data; preparing for the launch of duvelisib together with our partner, AbbVie; and, advancing clinical studies designed to differentiate duvelisib, with the ultimate aspiration of offering a potential cure to patients with hematologic malignancies," said Ms. Perkins.

The Phase 1b/2 clinical study of duvelisib in combination with venetoclax, AbbVie’s first-in-class investigational B-cell lymphoma-2 (BCL-2) selective inhibitor, is expected to begin this month. This study is designed to evaluate the safety and efficacy of duvelisib in combination with venetoclax in approximately 174 patients with relapsed or refractory CLL, small lymphocytic lymphoma, iNHL or aggressive NHL.

"The fourth strategic objective for Infinity is to advance our second development candidate, IPI-549, and we are pleased to announce the Phase 1 study is now under way," Ms. Perkins continued. "IPI-549 represents an important extension of our oncology portfolio to include development candidates directed at both hematologic malignancies and solid tumors."

2016 Duvelisib Goals

Infinity expects to achieve the following duvelisib milestones in 2016:

Report topline DYNAMO data in 3Q16
Report topline DUO data in 2H16*
Submit a New Drug Application (NDA) for iNHL and CLL in 4Q16*
AbbVie submission of Marketing Authorization Application (MAA) for follicular lymphoma and CLL in 4Q16*
Report initial data from CONTEMPO, a Phase1b/2 study in treatment-naïve patients with follicular lymphoma, in 2H16
Advance Phase 1b/2 study of duvelisib in combination with venetoclax
*CLL NDA filing and follicular lymphoma/CLL MAA filing predicated on DUO interim analysis.

2016 Financial Guidance

Infinity ended 2015 with approximately $245 million in cash and investments (unaudited) and plans to report its fourth quarter and full-year 2015 financial results in late February. The company is providing the following guidance today with respect to its 2016

financial outlook:

Revenue: Infinity expects revenue for 2016 to range from $225 million to $245 million, assuming the achievement of $200 million in anticipated regulatory milestones under the company’s collaboration with AbbVie: $125 million associated with the acceptance of the first NDA submission and $75 million associated with the acceptance of the first MAA submission, both expected in the fourth quarter of 2016.

Net Income: Infinity expects net income for 2016 to range from $15 million to $35 million.

Cash and Investments: Infinity expects to end 2016 with a year-end cash and investments balance ranging from $45 million to $65 million. The company expects to earn $200 million in regulatory milestones, as noted above, in the fourth quarter of 2016 with payment by AbbVie anticipated in the first quarter of 2017.

About Duvelisib
Duvelisib is an oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, two proteins with predominantly non-overlapping roles known to support the growth and survival of malignant B-cells.[i] Preclinical data suggest that PI3K-delta signaling can lead to the proliferation of malignant B-cells, and both PI3K-gamma and PI3K-delta play a role in the formation and maintenance of the supportive tumor microenvironment.[ii] Duvelisib is the only investigational PI3K-delta,gamma inhibitor in Phase 3 clinical development and has the potential to be a first-in-class treatment for certain types of hematologic malignancies, or blood cancers. AbbVie and Infinity Pharmaceuticals, Inc. are jointly developing duvelisib in oncology.

Infinity and AbbVie are conducting a broad clinical development program evaluating duvelisib in patients with hematologic malignancies. In addition to DYNAMO and DUO, ongoing studies include BRAVURA, a Phase 3, double-blind, placebo-controlled study in patients with relapsed iNHL; FRESCO, a Phase 2 study in patients with relapsed/refractory follicular lymphoma; CONTEMPO, a Phase 1b/2 study in treatment-naïve patients with follicular lymphoma, and SYNCHRONY, a Phase 1b study in CLL patients whose disease is refractory to or has relapsed while receiving a Bruton’s tyrosine kinase (BTK) inhibitor. AbbVie is also conducting a clinical study in duvelisib in combination with venetoclax in patients with relapsed or refractory CLL, small lymphocytic lymphoma, iNHL or aggressive NHL, as well as a Phase 1 study of duvelisib in Japanese subjects with relapsed or refractory lymphoma. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About IPI-549
IPI-549 is an orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 inhibits immune suppressive macrophages within the tumor microenvironment, whereas other immunotherapies such as checkpoint modulators more directly target immune effector cell function. As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

Duvelisib and IPI-549 are investigational compounds and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

Advanced Accelerator Applications Signs Exclusive License Agreement With Johns Hopkins University to Develop PSMA Receptor Ligand in Prostate Cancer

On January 11, 2016 Advanced Accelerator Applications S.A. (NASDAQ:AAAP) ("AAA" or "the Company"), an international specialist in molecular nuclear medicine, reported an exclusive license agreement with Johns Hopkins University in Baltimore, Maryland to develop and market PSMA-SR6, a receptor ligand of Prostate-Specific Membrane Antigen (PSMA) for clinical therapeutic and diagnostic purposes (Press release, Johns Hopkins University, JAN 11, 2016, View Source [SID1234524452]). AAA will focus on developing this treatment and its companion diagnostic for prostate cancer through novel molecular nuclear medicine techniques similar to those implemented for the development of Lutathera and Somakit. Prostate cancer affects nearly 1 in 7 men during their lifetime worldwide.

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The terms of the agreement include payment to Johns Hopkins of an upfront licensing fee, as well as certain milestone and royalty payments.

"This licensing agreement is the first step toward broadening our development pipeline by leveraging a formula that we have already successfully applied to develop our lead therapeutic and diagnostic candidates Lutathera and Somakit. We plan to radiolabel PSMA-SR6, to develop a 177Lu-PSMA-SR6 to treat and monitor prostate cancer and a 68Ga-PSMA-SR6, which will help to diagnose and stage disease. The PSMA expression pathway has been widely investigated with labelled antibodies, but we believe that a small molecule, with very high specificity and rapid uptake into tumors and clearance from non-targeted organs could be the ideal candidate for a full theragnostic approach. We are very pleased to partner with the Johns Hopkins University as they have been pioneering and leading this new field for many years," says Stefano Buono, Chief Executive Officer of AAA.

"Our license agreement with AAA extends Johns Hopkins University’s research leadership in PSMA to benefit patients," says Neil Veloso, Executive Director of Johns Hopkins Technology Ventures. "We are very pleased that AAA has selected PSMA-SR6 for full development for commercial applications in an area of significant patient need."

AAA is planning to support a proof-of-concept study in humans that may start in 2016 for both diagnostic and therapeutic applications of PSMA-SR6.

PSMA-SR6 is a unique second-generation selective prostate cancer PSMA receptor ligand developed by Dr Martin Pomper at Johns Hopkins University. PSMA-SR6 has a unique structure and is selective for PSMA expressed on prostate cancer tumor cells. It belongs to a new class of PSMA receptor ligands with high potential as diagnostic and therapeutic markers for prostate cancer. Studies have consistently demonstrated PSMA expression in all types of prostate tissue and an increased PSMA expression in cancer tissue.

Spectrum Pharmaceuticals Signs a Strategic Partnership With Servier Canada

On January 11, 2016 Spectrum Pharmaceuticals, Inc. (NasdaqGS:SPPI), a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology and Servier Canada Inc., an affiliate of Servier a research-oriented pharmaceutical company which is pioneering new therapies primarily for cancer and cardiovascular diseases, reported the signing of a strategic partnership (Press release, Spectrum Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508757]). As part of this partnership, Spectrum will grant the exclusive rights to develop and commercialize in Canada a franchise of four Spectrum hemato-oncology drugs: ZEVALIN (ibritumomab tiuxetan) Injection for intravenous use, Folotyn(pralatrexate injection), Beleodaq (belinostat) for Injection and Marqibo (vinCRIStine sulfate LIPOSOME injection) for intravenous infusion. Spectrum will receive $6 million in upfront payments, plus development milestone payments and royalties based on net product sales.

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"We are pleased to announce this strategic partnership with Servier, a leading company in Canada," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "This deal allows us to continue to focus on our core priorities including SPI-2012, Poziotinib, Apaziquone and Evomela. This year we plan to initiate two key trials with drugs that target blockbuster markets and we are looking forward to hearing from the FDA on two of our NDA submissions. We believe that our pipeline has never been as strong as it is today, and we continue to focus on executing on our key priorities."

"This strategic partnership between Spectrum Pharmaceuticals and Servier Canada will contribute to consolidate our global strategy in Oncology. Our ambition is to become a benchmark player in this field," said Frédéric Sesini, Executive Vice-President International Operations of Groupe Servier. "We are fully committed in providing Canadian patients with innovative treatment options. With this key partnership, Servier Canada Oncology will start operating and will work to make these treatments available soon," underlined Frederic Fasano, Chief Executive Officer of Servier Canada Inc.