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Surface Oncology Announces Global Strategic Collaboration to Advance Next‐Generation Immunotherapies

On January 11, 2016 Surface Oncology (Surface), a leader in the development of next‐generation cancer immunotherapies, reported that it has entered into a broad strategic collaboration with Novartis to develop immunotherapeutics targeting the tumor microenvironment and the interface of innate and adaptive immunity (Press release, Surface Oncology, JAN 11, 2016, View Source [SID1234527366]). "This strategic collaboration reflects our shared commitment to innovation in cancer immunotherapy beyond checkpoint inhibitors," said Detlev Biniszkiewicz, CEO of Surface. Biniszkiewicz added "It fuels our pipeline, provides the option to co‐develop and cocommercialize half of the partnership programs, and allows us to further build value as an independent company as we also pursue new programs outside of the collaboration." Under the terms of the agreement, Surface is eligible to receive up to $170M in upfront, equity, and near‐term milestone payments. Novartis will gain exclusive access to Surface’s current pipeline of novel cancer immunotherapies, including an exclusive worldwide license to its leading program and options to license up to three additional programs from the existing portfolio, exercisable at IND. Surface is eligible to receive clinical and commercial milestones and up to double‐digit royalties on product sales. Surface also has the option to retain U.S. development and commercialization rights for at least half of the collaboration’s programs. Mark C. Fishman, M.D., President of the Novartis Institutes for BioMedical Research, commented, "We are very excited to enter into this collaboration with Surface. This alliance is another building block in our strategy to develop a portfolio of programs that we believe will
lead the next wave of immuno‐oncology medicines."

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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.

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.

Tensha Therapeutics To Be Acquired By Roche

On January 11, 2016 Tensha Therapeutics, a privately-held company based in Cambridge, MA, reported it will be acquired by Roche (Press release, Tensha Therapeutics, JAN 11, 2016, View Source [SID1234518479]). Founded by James E. Bradner, MD, of the Dana-Farber Cancer Institute, and managed and funded by HealthCare Ventures, Tensha has developed a pioneering epigenetic technology that disrupts bromodomain and extra terminal domain (BET) proteins in order to develop potential treatments for cancer. The lead product, TEN-010, is a small molecule BET inhibitor that is currently in two Phase 1b clinical trials for the treatment of patients with cancer.

"BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC."
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Under the terms of the agreement, Tensha’s shareholders will receive an upfront cash payment of $115 million, plus additional contingent payments of up to $420 million based on the achievement of certain predetermined clinical and regulatory milestones. The transaction is subject to customary closing conditions and anticipated to close in the first quarter of 2016.

"BET proteins are a highly promising class of therapeutic targets in cancer," said James Bradner, MD, Founder of Tensha, Associate Professor at Harvard Medical School, and Investigator at the Dana-Farber Cancer Institute. "BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC."

"We selected TEN-010 as a highly selective, potent BET inhibitor, and we moved rapidly and strategically to advance its development," said Steven Landau, MD, Chief Medical Officer of Tensha and Director of Clinical and Scientific Analysis for HealthCare Ventures. "Our initial clinical data demonstrating the potential of TEN-010 in patients with NUT midline carcinoma was presented at the AACR (Free AACR Whitepaper)/NCI/EORTC conference in November."

"We are very excited about this acquisition, as it moves TEN-010 into the pipeline of a world leader in the development of novel cancer therapeutics. With leadership in solid and hematological tumors and deep understanding of BET biology, Roche is the ideal company to explore the broad clinical potential of TEN-010," said Douglas E. Onsi, Chief Executive Officer of Tensha and Managing Director of HealthCare Ventures. "We appreciate the work of the Bradner lab, the Tensha and HealthCare Ventures employees, and the clinical teams at our trial sites for their important roles in bringing TEN-010 to studies in patients."

About BET Proteins

Bromodomain and extra terminal domain (BET) proteins are central mediators of gene control and cellular memory. In cancer, BET proteins activate growth and survival genes. Further, they contribute to cancer cell memory by binding to the genome as molecular bookmarks. Tensha’s Founder, James Bradner, MD, Associate Professor at the Dana-Farber Cancer Institute and Harvard Medical School, was the first to recognize the broad potential of BET inhibitors as anti-cancer agents. The Bradner lab first reported BET inhibitors in 2010 in Nature magazine, and established the potential of BET inhibitors in solid tumors and blood cancers. With chemist Jun Qi, Bradner thereafter invented TEN-010, a best-in-class BET inhibitor, for clinical development. BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC. BET inhibition represents a new paradigm of targeting cellular memory, or epigenetics, in cancer, inflammation, and fibrosis.

AstraZeneca and Moderna Therapeutics Announce New Collaboration to Co-Develop and Co-Commercialize Immuno-Oncology mRNA Therapeutics

On January 11, 2016 AstraZeneca, along with its global biologics research and development arm, MedImmune, and Moderna Therapeutics reported a new collaboration to discover, co-develop and co-commercialize messenger RNA (mRNA) therapeutic candidates for the treatment of a range of cancers (Press release, Moderna Therapeutics, JAN 11, 2016, View Source [SID1234517365]). The collaboration is in addition to the agreement announced by the companies in 2013 to develop mRNA Therapeutics for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.

The collaboration will combine MedImmune’s protein engineering and cancer biology expertise with Moderna’s mRNA platform. mRNA-based therapies are an innovative treatment approach that enables the body to produce therapeutic protein in vivo, opening up new treatment options for a wide range of diseases that cannot be addressed today using existing technologies.

Under the terms of the new agreement, AstraZeneca and Moderna, a pioneer of mRNA Therapeutics, have agreed to collaborate on two specific immuno-oncology programs, based on promising pre-clinical data, including pharmacology in tumor models. Moderna will fund and be responsible for discovery and preclinical development of product candidates, with the aim of delivering one Investigational New Drug (IND) application-ready molecule for each of the two programs. Moderna’s efforts will be led by its oncology-focused venture, Onkaido. AstraZeneca will be responsible for early clinical development, led by MedImmune, and Moderna and AstraZeneca will share the costs of late-stage clinical development. The two companies will co-commercialize resulting products in the US under a 50:50 profit sharing arrangement. AstraZeneca will lead ex-US commercialization efforts, with Moderna receiving tiered royalties up to substantial double digits on ex-US sales.

Pascal Soriot, Chief Executive Officer, AstraZeneca, said: "We’re pleased to be expanding our relationship with Moderna with this new collaboration, to advance the potential of pioneering messenger RNA technology in developing game-changing new treatments for cancer patients."

"Since our companies’ original strategic agreement in March 2013, Moderna’s relationship with AstraZeneca has been very fruitful. This new agreement with AstraZeneca demonstrates the effectiveness of our existing relationship and the power of our mRNA technology," said Stéphane Bancel, Chief Executive Officer of Moderna. "We’re gratified to deepen our relationship with AstraZeneca and MedImmune with this major initiative, and we look forward to getting underway immediately with our new joint immuno-oncology programs."

Under the companies’ original strategic agreement, AstraZeneca holds exclusive access to select any target of its choice in cardiometabolic diseases, as well as select targets in oncology, over a period of up to five years for subsequent development in mRNA. Several projects are progressing towards clinical development under the arrangement, and a first-in-human study is expected to commence in late 2016.

With its novel technology to enable mRNA as a drug, Moderna is building a fully scaled drug discovery and development platform centered on the rapid and low-cost design, delivery and production of mRNA drug candidates. Moderna is also advancing an innovative business model built on the decentralization of drug development activities. With an ecosystem of in-house concept development, ventures focused on therapeutic areas and a cluster of major pharma and biotech partners, Moderna is enabling more than 90 discovery and preclinical programs today across oncology, infectious diseases, rare diseases and cardiovascular diseases. Moderna’s pipeline also covers a broad expanse of novel drug modalities, each representing a distinct approach to using the company’s novel mRNA expression platform to encode proteins that achieve a therapeutic benefit.

Ionis Pharmaceuticals Revises 2015 Financial Guidance and Provides Pipeline Update in Conjunction with J.P. Morgan Conference

On January 11, 2016 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported that the Company expects to significantly improve upon its financial guidance for 2015 (Press release, Isis Pharmaceuticals, JAN 11, 2016, View Source;p=RssLanding&cat=news&id=2128373 [SID:1234508772]). The Company expects to end 2015 with a pro forma net operating loss (NOL) in the low $20 million range and more than $775 million in cash.

"Over the past several years, we have created a strong and consistent financial foundation. Our corporate partnerships have produced increasing levels of revenue as our partnered programs have matured and expanded. We have been able to support the advancement of our very large pipeline with modest expense growth due to the efficiency of our technology platform as well as the significant in-kind contributions of our partners. With three potentially transformational medicines close to commercialization, we look forward to adding product revenues and royalties to our revenue base over the next few years," said Elizabeth L. Hougen, chief financial officer of Ionis Pharmaceuticals. "We expect to end 2015 with a pro forma NOL in the low $20 million range, which represents a 60% improvement over our original guidance. In addition, we expect to end 2015 with more than $775 million in cash, substantially exceeding our 2014 year-end cash balance and our 2015 cash guidance. Importantly, our significant increase in revenue over the last several years reflects the successes in all of our collaborations as the drugs in those collaborations advanced."

The Company intends to provide financial guidance for 2016 in connection with its year-end 2015 financial results in February.

Pipeline Update
Ionis management will present an update today on its drug development pipeline at the J.P. Morgan conference, including an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones. The data reported today show continued increases in median event-free survival and muscle function scores. The safety and tolerability profile of nusinersen to date continues to support further development.

In 2015, Ionis added nine new drugs to its pipeline. Already in 2016, the company has added two new drugs to its pipeline: IONIS-BIIB5Rx and IONIS-BIIB6Rx. Both new drugs are being developed together with Biogen and are designed to address undisclosed targets for the treatment of patients with neurodegenerative diseases. Ionis currently has a total of 38 drugs in its pipeline.

"We believe we are just beginning to realize the value we have created. We have three potentially groundbreaking medicines for which we have completed target enrollment in the respective pivotal Phase 3 trials and preparations are underway for each of these drugs for the necessary regulatory filings for marketing authorization. In addition, we and our partners are well along in preparing to commercialize these medicines. As our pipeline continues to mature, we have many other medicines coming along behind these three late-stage drugs," said B. Lynne Parshall, chief operating officer at Ionis. "We have also demonstrated in 2015 that the advances we are making in our technology continue to make better and better medicines, which allows us to expand the application of our drugs to new tissues, new targets and new diseases. All of this sets us up for an exciting and event-filled 2016."

In January 2016, Ionis terminated its license agreement with Sanofi Genzyme for KYNAMRO (mipomersen sodium).

"We are disappointed that we have had to terminate our contract with Genzyme to commercialize KYNAMRO," said Ms. Parshall. "We know that KYNAMRO is an important drug for patients with homozygous FH. Genzyme is continuing to support patients and physicians as we explore other potential commercialization options."

In addition, Ionis announced that results from the Phase 1/2 study with IONIS-AR-2.5Rx in heavily pretreated, late-stage prostate cancer patients demonstrated encouraging data, including several durable reductions in PSA levels. The drug also exhibited a good safety and tolerability profile supportive of continued development. Data from this study will be presented later in the year. Ionis plans to continue the development of IONIS-AR-2.5Rx independent of AstraZeneca.