Investigational Medicine Venetoclax Receives Breakthrough Therapy Designation in Relapsed or Refractory Chronic Lymphocytic Leukemia in Previously Treated Patients with the 17p Deletion Genetic Mutation

— AbbVie plans to submit regulatory applications for venetoclax to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) before the end of 2015
— Venetoclax is currently in development for the treatment of relapsed or refractory chronic lymphocytic leukemia, including patients with 17p deletion

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May 6, 2015

NORTH CHICAGO, Ill., May 6, 2015 /PRNewswire/ — AbbVie (NYSE: ABBV) reported its investigational medicine venetoclax, an inhibitor of the B-cell lymphoma-2 (BCL-2) protein that is being developed in partnership with Genentech and Roche, has been granted Breakthrough Therapy Designation by the FDA for the treatment of chronic lymphocytic leukemia (CLL) in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation (Press release, AbbVie, MAY 6, 2015, View Source [SID:1234503964]).

CLL is a slow-progressing cancer of the bone marrow and blood in which the bone marrow makes too many lymphocytes, a type of white blood cell.1 CLL accounts for approximately one quarter of the new cases of leukemia diagnosed in the United States.2 Approximately 3-10 percent of CLL patients have 17p deletion at diagnosis, and it occurs in 30-50 percent of patients with relapsed/refractory CLL.3 The 17p deletion mutation is a genomic alteration in which a part of chromosome 17 is absent.4 The median life expectancy for CLL patients with 17p deletion is less than 2-3 years.5

"The Breakthrough Therapy Designation of venetoclax supports the continued development of this investigational medicine in CLL patients with 17p deletion," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "The continuing advancement of the venetoclax development program is one example of AbbVie’s focus on delivering innovative medicines that address unmet clinical needs."

According to the FDA, Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation includes preliminary clinical evidence demonstrating a drug may have substantial improvement on at least one clinically significant endpoint compared to available therapy. A Breakthrough Therapy Designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program.

Additional information regarding the venetoclax clinical trials is available on www.clinicaltrials.gov.

About Venetoclax
Venetoclax is an investigational oral B-cell lymphoma-2 (BCL-2) inhibitor being evaluated for the treatment of patients with various cancer types. The BCL-2 protein prevents apoptosis of some cells, including lymphocytes, and can be expressed in some cancer types. Venetoclax is designed to selectively inhibit the function of the BCL-2 protein. Venetoclax is being developed in collaboration with Genentech and Roche. Together, the companies are committed to BCL-2 research with venetoclax, which is currently being evaluated in Phase 2 and Phase 3 clinical trials for the treatment of CLL, along with studies in several other cancers. Venetoclax is an investigational compound and its safety and efficacy have not been evaluated by the FDA or any other health authority.

About AbbVie Oncology
AbbVie’s oncology research is focused on the discovery and development of targeted therapies that work against the processes cancers need to survive. By investing in new technologies and approaches, AbbVie is breaking ground in some of the most widespread and difficult-to-treat cancers, including multiple myeloma and chronic lymphocytic leukemia. AbbVie’s oncology pipeline includes multiple new molecules in clinical trials being studied in more than 15 different cancers and tumor types. For more information on AbbVie’s oncology portfolio, please visit View Source

Alexion to Acquire Synageva to Strengthen Global Leadership in Developing and Commercializing Transformative Therapies for Patients with Devastating and Rare Diseases

On May 6, 2015 Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) and Synageva BioPharma Corp. (Nasdaq:GEVA) reported that they have entered into a definitive agreement pursuant to which Alexion will acquire Synageva for consideration of $115 in cash and 0.6581 Alexion shares, for each share of Synageva, implying a total per share value of $230 based on the nine day volume-weighted average closing price of Alexion stock through May 5, 2015 (Press release, Alexion, MAY 6, 2015, View Source [SID:1234503620]). The acquisition strengthens Alexion’s global leadership in developing and commercializing transformative therapies for patients with devastating and rare diseases.

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The transaction has been unanimously approved by both companies’ Boards of Directors, and is valued at approximately $8.4 billion net of Synageva’s cash. The transaction is expected to accelerate and diversify Alexion’s growing revenues, and Alexion expects to achieve annual cost synergies starting this year and growing to at least $150 million in 2017. In addition, the transaction is expected to be accretive to non-GAAP earnings per share in 2018.

"Synageva is an ideal strategic and operational fit for Alexion that aligns with what we know well and do well — providing life-transforming therapies to an increasing number of patients with devastating and rare diseases," said David Hallal, Chief Executive Officer of Alexion. "With strong ongoing Soliris growth in PNH and aHUS worldwide, and the anticipated 2015 global launches of Strensiq and Kanuma, we will accelerate and diversify our revenue growth. We are excited to create the most robust rare disease pipeline in biotech across a range of therapeutic modalities. Synageva is an outstanding company that shares Alexion’s commitment to serving patients with rare diseases, and together we will create increasing value for our stakeholders."

"Alexion is uniquely suited to advance Synageva’s mission to deliver life-saving therapies to patients whose diseases were once considered too rare for developing treatments," said Sanj K. Patel, President and Chief Executive Officer of Synageva. "As Kanuma moves closer toward patients who suffer from LAL Deficiency, and the other pipeline programs continue to progress, I am confident that this transaction will help continue to improve the lives of patients with LAL Deficiency and other devastating, rare diseases for years to come."

The addition of Kanuma expands Alexion’s premier global metabolic rare disease franchise. Alexion will leverage its proven expertise in rare disease education and diagnostics, and its 50-country operating platform, to maximize the opportunity to serve patients suffering from LAL-D. The Company expects that these efforts will result in more infants, children and adults with LAL-D receiving a rapid and accurate diagnosis and, following regulatory approvals for Kanuma, enable physicians to make better informed treatment decisions for their patients. Kanuma is under Priority Review with the U.S. Food and Drug Administration (FDA) and has been granted accelerated assessment of its Marketing Authorization Application (MAA) by the European Medicines Agency (EMA). Kanuma has been granted Breakthrough Therapy Designation by the FDA for LAL Deficiency presenting in infants. Regulatory decisions in the U.S. and Europe are expected in the second half of 2015.

Alexion developed Soliris (eculizumab) from the laboratory through regulatory approvals, and currently provides Soliris to patients around the world with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. Since its launch in 2007, Soliris has grown to more than $2 billion in revenues in 2014, with additional growth anticipated as the Company has consistently identified significant numbers of new patients with PNH and aHUS each year. With Soliris, and following the anticipated approvals of Kanuma and Strensiq, Alexion will have three highly innovative and transformative therapies serving patients with four devastating and rare diseases in 2015.

"By every measure, Alexion is at the strongest and most promising point in our history given the strength of our clinical, commercial, and operational performance and the depth of our team," said Leonard Bell, M.D., Chairman of Alexion’s Board of Directors. "These strengths will enable us to accelerate the transformation of the lives of patients suffering from LAL-D around the world. Also, I am personally very pleased that Dr. Felix Baker, a deeply experienced board member and leader in the biopharmaceutical industry, will join the Alexion Board of Directors when our transaction is completed. I look forward to working with Felix as we pursue our ambitions to serve more patients with more severe and rare disorders."

"This transaction provides Synageva shareholders with immediate value and the opportunity to participate in Alexion’s long-term growth potential," said Felix Baker, Ph.D., Chairman of Synageva’s Board of Directors. "I am excited to be joining the board of Alexion, a leading, global biotechnology company that is aligned with the mission that Synageva was founded upon – to serve patients who would otherwise be left behind."

Acquisition Creates Most Robust Rare Disease Pipeline in Biotech; Expands Manufacturing Capabilities

Synageva’s pipeline is complementary to Alexion’s growing portfolio of highly innovative product candidates for patients with devastating and rare diseases. Alexion will have a robust clinical pipeline with eight product candidates in clinical trials for eleven indications. The programs include Synageva’s SBC-103, an investigational enzyme replacement therapy in an ongoing Phase 1/2 trial for patients with mucopolysaccharidosis IIIB (MPS IIIB), a genetic and progressive rare metabolic disease. SBC-103 was granted Fast Track designation by the FDA in January 2015.

In addition, Alexion will have more than 30 diverse pre-clinical programs across a range of therapeutic modalities, including 12 from Synageva’s novel drug discovery platform. At least four pre-clinical candidates from the combined pipelines are expected to enter the clinic by year-end 2016.

Alexion will also have expanded manufacturing capabilities with three Synageva upstream facilities. Synageva brings to Alexion a proprietary manufacturing technology, known as the expression platform, an integrated system of proprietary vectors that can be used to produce proteins with human-like glycosylation patterns, creating additional therapies with better targeting capabilities and the potential for greater efficacy.

Terms of the Transaction

Alexion will acquire all of the outstanding shares of common stock of Synageva through an exchange offer, followed by a second-step merger, with each share receiving $115 in cash and 0.6581 shares of Alexion stock. The stock portion of the consideration is expected to be tax-free to Synageva stockholders.

The completion of the exchange offer and the merger are subject to customary closing conditions, the tender of a majority of the outstanding shares of Synageva common stock and receipt of required regulatory approval. The transaction is expected to close mid-2015. The merger agreement provides that Alexion may, in certain circumstances, determine to alternatively effect the transaction through a one-step merger, in which case a meeting of Synageva stockholders would be held to vote on the transaction.

In connection with the Transaction, Synageva shareholders, including affiliates of Baker Brothers Investments, have entered into voting and support agreements with Alexion covering approximately 33.5% of Synageva’s outstanding shares.

Alexion has received committed financing of $3.5 billion from Bank of America Merrill Lynch and J.P. Morgan in connection with the transaction.

Lazard and J.P. Morgan are acting as the financial advisors to Alexion, and Wachtell, Lipton, Rosen & Katz is serving as legal counsel. Goldman Sachs & Co. is acting as the financial advisor to Synageva, and Sullivan & Cromwell LLP and Ropes & Gray LLP are serving as legal counsel.

For additional details on the transaction, please visit www.alexion-synagevatransaction.com.

Share Repurchase Authorization

The Company also announced that its Board of Directors has increased the size of the Company’s share repurchase authorization to a total of $1 billion. The Board’s authorization is open-ended and does not establish a timeframe for the purchases; however, no repurchases will be made during the pendency of the transaction.

Juno Therapeutics and Fate Therapeutics Announce Strategic Research Collaboration to Improve the Therapeutic Profile of Engineered T Cell Immunotherapies

On May 6, 2015 Juno Therapeutics, Inc. (Nasdaq:JUNO) and Fate Therapeutics, Inc. (Nasdaq:FATE) reported that they have executed a strategic research collaboration and license agreement to identify and utilize small molecules to modulate Juno’s genetically-engineered T cell product candidates to improve their therapeutic potential for cancer patients (Press release, Fate Therapeutics, MAY 6, 2015, View Source [SID1234526874]). The collaboration brings together Juno’s industry-leading expertise in the development of chimeric antigen receptor (CAR) and T cell receptor (TCR) based cellular immunotherapies and Fate’s innovative platform for programming the biological properties and in vivo therapeutic potential of hematopoietic cells.

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"A deep understanding of T cell biology is the basis of Juno’s approach to creating best-in-class cellular immunotherapies," said Hans Bishop, Chief Executive Officer of Juno Therapeutics. "Partnering with Fate Therapeutics, and accessing its strong science and leading platform for modulating the properties of immunological cells, enables interrogation of new avenues of T cell manipulation and provides an opportunity to enhance the therapeutic profile of our genetically-engineered T cell product candidates."

Through the four-year research and development collaboration, Fate will be responsible for screening and identifying small molecules that modulate the biological properties of engineered T cells. Juno will be responsible for the development and commercialization of engineered T cell immunotherapies incorporating Fate’s small molecule modulators. Juno has the option to extend the exclusive research term for two years through an additional payment and continued funding of collaboration activities.

"We are excited to establish this strategic alliance with Juno, a company that shares our deep commitment to developing transformative cellular therapeutics for patients afflicted with life-threatening disorders," said Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "This partnership exemplifies the extension of our small molecule programming platform to additional hematopoietic cell types, such as T cells, as we continue to build and advance our innovative pipeline of programmed hematopoietic cellular therapeutic candidates."

Financial terms of the agreement include an upfront payment to Fate of $5 million and the purchase by Juno of one million shares of Fate common stock at $8.00 per share. Juno will fund all mutual collaboration activities for an exclusive four-year research term. For each product developed by Juno that incorporates modulators identified through the collaboration, Fate is eligible to receive approximately $50 million in target selection fees and clinical, regulatory and commercial milestones, as well as low single-digit royalties on sales. Fate retains exclusive rights to its intellectual property for all purposes outside of programmed CAR and TCR immunotherapies.

About Chimeric Antigen Receptor (CAR) Technology

Juno’s chimeric antigen receptor (CAR) technology genetically engineers T cells to recognize and kill cancer cells. Juno’s CAR T cell technology inserts a gene for a particular CAR into the T cell, enabling it to recognize cancer cells based on the expression of a specific protein located on the cell surface. When the engineered T cell engages the target protein on the cancer cell, it initiates a cell-killing response against the cancer cell.

About Cell Programming

Since its founding, Fate Therapeutics has been dedicated to programming the function of cells ex vivo to improve their therapeutic potential. Using advanced molecular characterization tools and technologies, Fate’s platform enables the identification of small molecule or biologic modulators that promote rapid and supra‑physiologic activation or inhibition of therapeutically‑relevant genes and cell‑surface proteins, such as those involved in the homing, proliferation and survival of hematopoietic stem cells or those involved in the persistence, proliferation and reactivity of immunological cells. Fate utilizes its deep understanding of the hematopoietic system to rapidly assess and quantify the therapeutic potential of programmed hematopoietic cells in vivo, and applies its modulators to maximize the safety and efficacy of hematopoietic cellular therapeutics.

8-K – Current report

On May 6, 2015 Juno Therapeutics and Fate Therapeutics reported that they have executed a strategic research collaboration and license agreement to identify and utilize small molecules to modulate Juno’s genetically-engineered T cell product candidates to improve their therapeutic potential for cancer patients (Filing, 8-K, Fate Therapeutics, MAY 6, 2015, View Source [SID:1234503625]). The collaboration brings together Juno’s industry-leading expertise in the development of chimeric antigen receptor (CAR) and T cell receptor (TCR) based cellular immunotherapies and Fate’s innovative platform for programming the biological properties and in vivo therapeutic potential of hematopoietic cells.

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"A deep understanding of T cell biology is the basis of Juno’s approach to creating best-in-class cellular immunotherapies," said Hans Bishop, Chief Executive Officer of Juno Therapeutics. "Partnering with Fate Therapeutics, and accessing its strong science and leading platform for modulating the properties of immunological cells, enables interrogation of new avenues of T cell manipulation and provides an opportunity to enhance the therapeutic profile of our genetically-engineered T cell product candidates."

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Through the four-year research and development collaboration, Fate will be responsible for screening and identifying small molecules that modulate the biological properties of engineered T cells. Juno will be responsible for the development and commercialization of engineered T cell immunotherapies incorporating Fate’s small molecule modulators. Juno has the option to extend the exclusive research term for two years through an additional payment and continued funding of collaboration activities.

"We are excited to establish this strategic alliance with Juno, a company that shares our deep commitment to developing transformative cellular therapeutics for patients afflicted with life-threatening disorders," said Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "This partnership exemplifies the extension of our small molecule programming platform to additional hematopoietic cell types, such as T cells, as we continue to build and advance our innovative pipeline of programmed hematopoietic cellular therapeutic candidates."

Financial terms of the agreement include an upfront payment to Fate of $5 million and the purchase by Juno of one million shares of Fate common stock at $8.00 per share. Juno will fund all mutual collaboration activities for an exclusive four-year research term. For each product developed by Juno that incorporates modulators identified through the collaboration, Fate is eligible to receive approximately $50 million in target selection fees and clinical, regulatory and commercial milestones, as well as low single-digit royalties on sales. Fate retains exclusive rights to its intellectual property for all purposes outside of programmed CAR and TCR immunotherapies.

About Chimeric Antigen Receptor (CAR) Technology

Juno’s chimeric antigen receptor (CAR) technology genetically engineers T cells to recognize and kill cancer cells. Juno’s CAR T cell technology inserts a gene for a particular CAR into the T cell, enabling it to recognize cancer cells based on the expression of a specific protein located on the cell surface. When the engineered T cell engages the target protein on the cancer cell, it initiates a cell-killing response against the cancer cell.

ARIAD Announces Commercial Distribution Agreement for Iclusig (ponatinib) in Turkey

On May 6, 2015 ARIAD Pharmaceuticals reported that ARIAD has granted Gen Ilac exclusive rights to distribute Iclusig (ponatinib) in Turkey for patients with Philadelphia-positive leukemias(Press release, Ariad, MAY 6, 2015, View Source [SID:1234503619]).

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Gen Ilac was granted the exclusive right to sell Iclusig as an investigational product in Turkey on a named-patient basis and will provide associated medical affairs and regulatory support. ARIAD retains the option to file for marketing authorization at a later date and, if approved, to hold the Marketing Authorization for Iclusig in Turkey. Gen Ilac has the exclusive right then to market and commercialize the product upon approval.

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"This agreement with Gen Ilac furthers our mission to provide Iclusig to patients in geographies beyond our core commercial footprint," said Marty J. Duvall, executive vice president and chief commercial officer of ARIAD. "We are excited by Gen Ilac’s experience and proven capabilities in this area, which makes them an ideal partner to introduce Iclusig to patients and physicians in Turkey."

"We are excited by our partnership with ARIAD and the hope it provides to refractory CML patients in Turkey," said Abidin Gulmus, chief executive officer of GEN ILAC. "This partnership aligns well with our company mission and our expertise in providing treatments in the field of hematology."

About Iclusig (ponatinib)

Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.