On December 2, 2016 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported key presentations of clinical and scientific data related to its products at the 58th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), being held in San Diego, California, from December 3-6, 2016 (Press release, Spectrum Pharmaceuticals, DEC 2, 2016, View Source [SID1234516892]).

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For more information about the ASH (Free ASH Whitepaper) annual meeting and for a complete list of abstracts, please refer to the conference website at View Source

The following are key PTCL-related abstracts being presented at the ASH (Free ASH Whitepaper) meeting:

Abstract # Type Title First Author
Date/Time
Location
4149 Poster Case Match Control Analysis of Propel Reveals Survival Advantage for Patients with Relapsed/Refractory (R/R) Peripheral T-Cell Lymphoma (PTCL) Treated with Pralatrexate O’Connor
Monday, Dec 5,
6:00 PM-8:00 PM
Hall GH
4150 Poster Differential Outcome of Patients with Primary Refractory Vs. Relapsed Peripheral T-Cell Lymphoma: Analysis from a Prospective Multicenter US Cohort Study Lansigan
Monday, Dec 5,
6:00 PM-8:00 PM
Hall GH
1824 Poster The Pralatrexate – Romidepsin Doublet: A Well Tolerated and Highly Effective Combination for Patients with Relapsed or Refractory Peripheral T-Cell Lymphoma Amengual
Saturday, Dec 3,
5:30 PM-7:30 PM
Hall GH

The following key ZEVALIN (ibritumomab tiuxetan)-related abstract will be presented at the ASH (Free ASH Whitepaper) conference:

Abstract # Type Title First Author Location
1793 Poster Short Course of Bendamustine and Rituximab Followed By 90Y-Ibritumomab Tiuxetan in Patients with Chemotherapy-Naive Follicular Lymphoma (FOL-BRITe): Final Report of Response Rates and Progression Free Survival Costa
Saturday, Dec 3,
5:30 PM-7:30 PM
Hall GH

On December 2, 2016 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, reported that
the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of primary central nervous system lymphoma (PCNSL) (Press release, PIQUR Therapeutics, DEC 2, 2016, View Source [SID1234527272]).

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PCNSL is a very rare and aggressive form of lymphoma involving brain and its linings, eyes or spinal cord. The disease affects less than 1 person in 100,000 in the USA with approximately 7,500 new cases reported annually in the USA, Europe and Japan. Treatment options available for PCNSL are limited, and the prognosis with current therapies is poor.

"We are very pleased to receive FDA orphan drug designation for PQR309 in PCNSL. This is an important regulatory milestone for the company and a significant step towards the clinical advancement of PQR309," said Dr. Ruggero Della Bitta, Chief Medical Officer of PIQUR. "This represents an important step towards addressing a high unmet medical need and bringing a potential treatment to those with this rare and life-threatening disease."

The Orphan Drug Designation Program, administered by the FDA’s Office of Orphan Products Development, is intended to encourage companies to develop therapeutics for diseases that affect fewer than 200,000 people in the USA. The designation provides the company several benefits and incentives, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, as well as a seven-year period of market exclusivity upon regulatory product approval.

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. Preclinical and Phase 1 studies have shown PQR309 to have a favorable safety, tolerability and pharmacokinetic profile. In addition, PQR309 has shown both preclinical
activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in five Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877).

On December 2, 2016 Acetylon Pharmaceuticals reported that it has entered into an agreement to be acquired by Celgene Corporation (Press release, Acetylon, DEC 2, 2016, View Source [SID1234556812]). Prior to the consummation of the acquisition, Acetylon will spin out a new company, Regenacy Pharmaceuticals, LLC, which will focus on the development of novel drug candidates that selectively regenerate intracellular transport and upregulate gene expression to modify the course of disease. Regenacy will receive exclusive worldwide rights to Acetylon’s Phase 2 selective histone deacetylase 6 (HDAC6) inhibitor, ricolinostat (ACY-1215), for the treatment of certain non-cancer disease indications including neuropathies, as well as Acetylon’s preclinical selective HDAC1,2 inhibitor candidates and patent families for development in all human disease indications including sickle cell disease and beta-thalassemia.

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The acquisition will provide Celgene with, among other things, worldwide rights to Acetylon’s selective HDAC6 inhibitor programs and intellectual property in oncology, neurodegeneration, and autoimmune disease, including its lead drug candidates citarinostat (ACY-241) and ricolinostat (ACY-1215).

Financial terms of the acquisition are not being disclosed. The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976. BMO Capital Markets Corp. served as exclusive financial advisor to Acetylon in the transaction.

Key members of the Acetylon executive team will join Regenacy, which will operate out of Acetylon’s former headquarters in Boston’s Seaport District. Regenacy will be owned by Acetylon shareholders (excluding Celgene) and will receive net working capital in Acetylon to fund Regenacy operations.

"Since its founding in 2008, Acetylon has made substantial progress in the development of selective HDAC inhibitors for enhanced therapeutic outcomes," said Walter C. Ogier, President and Chief Executive Officer of Regenacy. "We are excited to continue Acetylon’s legacy through the receipt of rights to many of Acetylon’s most promising compounds and the continued advancement of these clinical and preclinical programs in disease indications outside of Celgene’s areas of strategic focus, where we believe patients may especially benefit from selective HDAC inhibition."

"Acetylon has had a longstanding partnership with Celgene, and their acquisition of our HDAC6 inhibitor programs is a positive event for patients and a favorable outcome for our shareholders and employees," said Marc A. Cohen, Chairman of Acetylon. "Celgene is the optimal partner to realize the fullest potential of Acetylon’s selective HDAC6 inhibitor programs in multiple myeloma and other oncology indications. Their intimate knowledge of citarinostat and extensive experience in oncology make them uniquely qualified to continue development of these exciting programs."

About Selective HDAC Inhibition

Histone deacetylases (HDACs) comprise a family of 18 related enzymes found in most human cells, 11 of which utilize zinc atoms to catalyze the removal of acetyl groups from intracellular proteins. By this function, HDACs can induce structural changes in the DNA-histone complex to result in altered gene expression and protein synthesis. Inappropriate deacetylation can disrupt these processes and contribute to a wide range of diseases, whereas regeneration of acetylation selectively causes apoptosis (cell death) in cancer cells and also induces favorable immunomodulatory effects. Currently available HDAC drugs non-selectively affect the expression of numerous other genes in normal cells as well as disease-causing cells, which can result in side effects such as gastrointestinal dysfunction, lowered blood platelet levels and risk of hemorrhage, and profound fatigue as well as potential for significant cardiac toxicity. Selective inhibition of HDACs is anticipated to reduce or eliminate these often-severe side effects associated with non-selective HDAC inhibition and to enable the development of optimized treatment regimens, including maximally effective combination drug therapies.

On December 2, 2016 Surface Oncology, an immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that it will present preclinical data on its CD47 program at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Surface Oncology, DEC 2, 2016, View Source [SID1234516902]). The data demonstrate that SRF231, a fully human CD47 antibody, induces robust tumor cell phagocytosis and clearance, both alone and in combination with existing standard of care treatment.

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"These data highlight the strong anti-tumor activity of SRF231 in hematological disease models," said Vito Palombella, PhD, Chief Scientific Officer at Surface Oncology, "and further suggest that SRF231 has the potential to be the best-in-class CD47 antibody to help patients with a wide range of cancers."

CD47 is an important immune escape mechanism exploited by multiple tumor types, making it a target with broad therapeutic potential. CD47 acts as a macrophage checkpoint or "don’t eat me" signal that prevents cells from being eliminated by a macrophage-mediated process called phagocytosis.

The data presented at ASH (Free ASH Whitepaper) demonstrate that SRF231 enhances tumor cell phagocytosis alone and in combination with opsonizing antibodies (e.g., anti-CD20 Ab). SRF231 also leads to profound tumor growth inhibition in models of multiple myeloma and non-Hodgkin’s lymphoma.

Previously, Surface presented data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s annual meeting demonstrating that SRF231 binds with high affinity to CD47, stimulates phagocytosis of cancer cells in vitro, and has potent anti-tumor activity in multiple in vivo disease models. These data also demonstrate that SRF231 does not induce detectable hemagglutination or phagocytosis of red blood cells in vitro, a potentially important safety advantage. SRF231 is expected to enter clinical trials in 2017.

On December 2, 2016 Calithera Biosciences, Inc. (Nasdaq:CALA), a clinical stage biotechnology company focused on the discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported that preclinical data for its lead drug candidate CB-839, the company’s novel, orally bioavailable glutaminase inhibitor, will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held from December 3-6, 2016 in San Diego, California (Press release, Calithera Biosciences, DEC 2, 2016, View Source;p=RssLanding&cat=news&id=2227168 [SID1234516878]). Data to be presented include the following two poster presentations:

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Glutaminase Inhibitor CB-839 Enhances Proteasome Inhibitor Sensitivity in Multiple Myeloma Cells
Presenter: Ravyn Thompson, Nathan Dolloff, Medical University of South Carolina
Oral and Poster Abstracts Session 652: Myeloma: Pathophysiology and Preclinical Studies, Hall GH, Abstract 3294
Sunday, December 4, 2016, 6:00 p.m.-8:00 p.m. PT
Gls Inhibitor CB-839 Modulates Cellular Metabolism in AML and Potently Suppresses AML Cell Growth When Combined with 5-Azacitidine
Presenter: Tianyu Cai, Marina Konopleva, The University of Texas, MD Anderson Cancer Center
Oral and Poster Abstracts Session 616: Acute Myeloid Leukemia: Novel Therapy, Hall GH, Abstract 4064
Monday, December 5, 2016, 6:00 p.m.-8:00 p.m. PT