20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

(Filing, Annual, Trillium Therapeutics, 2016, MAR 10, 2017, View Source [SID1234518069])

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10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-K, Athersys, MAR 10, 2017, View Source [SID1234518071])

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10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-Q, Progenics Pharmaceuticals, 2017, MAR 9, 2017, View Source [SID1234518051])

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FDA grants orphan drug designation to Boehringer Ingelheim’s investigational anti-CD33 monoclonal antibody BI 836858 for treatment of myelodysplastic syndromes

On March 9, 2017 Boehringer Ingelheim reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its anti-CD33 monoclonal antibody BI 836858 for the treatment of myelodysplastic syndromes (MDS) (Press release, Boehringer Ingelheim, MAR 9, 2017, View Source [SID1234535519]). Orphan drug designation is granted by the FDA to investigational compounds intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people.

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MDS are a group of bone marrow disorders. The bone marrow produces blood cells. In MDS, the bone marrow is characterized by cell abnormalities and ineffective blood cell production. Various subtypes of the disease exist with variable prognoses and treatment options. In some cases, MDS can progress to acute myeloid leukemia (AML), an aggressive and devastating blood cancer. According to data from the National Cancer Institute, an estimated 15,000 new cases of MDS are identified each year.

"Preclinical studies with our anti-CD33 monoclonal antibody have shown promising therapeutic potential in myelodysplastic syndromes, a rare group of bone marrow disorders," said Martina Flammer, M.D., Vice President, Clinical Development & Medical Affairs Specialty Care, Boehringer Ingelheim. "Orphan drug designation for our investigational candidate in MDS is an important recognition of our ongoing research efforts and underscores Boehringer Ingelheim’s commitment to studying treatment options for rare cancers."

A Phase I/II multi-center, open-label, dose escalation and randomized trial (NCT02240706 (link is external)) evaluating BI 836858 in patients with MDS is ongoing. BI 836858 previously received orphan drug designation for the treatment of patients with AML and is currently being evaluated as part of the Leukemia & Lymphoma Society’s (LLS) groundbreaking, first-of-its-kind Beat AML Master trial program to advance treatment for patients with AML. Using the latest genomic technology, the trial finds and matches specific AML mutations in newly-diagnosed patients over the age of 60 with an investigational drug or drugs best suited to attack the specific genetic mutations found within the cancer.

About BI 836858
BI 836858 is a monoclonal antibody that targets a protein called CD33, which is expressed on the surface of certain cancerous cells. BI 836858 has been engineered for improved antibody-dependent cell-mediated cytotoxicity (ADCC), a type of immune reaction in which a target cell or microbe is coated with antibodies and killed by certain types of white blood cells. Preclinical studies with the compound have shown promising ADCC against malignant cells.

About Boehringer Ingelheim in Oncology
Boehringer Ingelheim’s oncology research is driven by a passion to advance clinical practice and a determination to improve the lives of patients who are battling cancer. Through our own scientific innovation and partnerships, we are focused on discovering and providing novel best-in-class, breakthrough cancer medications that fit the needs of patients, caregivers and healthcare professionals. We have a clear strategy to become a leader in the field of lung cancer. Boehringer Ingelheim has successfully launched two products globally for NSCLC that have been widely adopted and established as valuable additions to current clinical practice. Continuous insights and learnings from research and development are key parts of innovation and our way forward to advance clinical practice in lung cancer and other cancer types.

OncoResponse Announces Final Closing of $22.5M Series A Financing Round

On March 9, 2017 OncoResponse, an immuno-oncology antibody discovery company, reported that it has closed its ongoing $22.5 million Series A financing round and has appointed Mike Gallatin, Ph.D., Albert Yu, M.D., and Elizabeth Jaffee, M.D. to its Scientific Advisory Board (SAB) (Press release, OncoResponse, MAR 9, 2017, View Source [SID1234522897]). The newly-appointed SAB members join original members James Welsh, M.D. and David Hong, M.D., both investigators at MD Anderson Cancer Center. Kristine Swiderek, Ph.D., CSO of OncoResponse, is chair of the SAB.

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In October 2015, OncoResponse secured an initial closing of its Series A financing co-led by ARCH Venture Partners, Canaan Partners, and MD Anderson, with William Marsh Rice University and Alexandria Real Estate Equities also participating. In May 2016, OncoResponse secured an additional investment by Baxalta (now Shire), which was followed by supplemental investments from GreatPoint Ventures and the Helsinn Investment Fund in October 2016. The recent investment by current insiders and HT Family Office brings the total Series A financing to $22.5 million, which will be used to support OncoResponse’s ongoing efforts to interrogate the humoral response of elite responders to cancer immunotherapy to identify antibodies and potential targets for novel therapeutic development.

OncoResponse utilizes a clinically validated platform technology to rapidly screen antibodies made by the human immune system and identify those with exceptional reactivity to cancer. The Company has a strategic alliance with MD Anderson Cancer Center, which provides continuous access to patient samples across multiple cancer indications and to oncology and translational medicine expertise including clinical and regulatory input.

"HT Family Office is an excellent addition to our solid investor base and we warmly welcome them along with our new Scientific Advisory Board members, who bring extensive industry and oncology expertise to our team," said Clifford J. Stocks, CEO of OncoResponse. "With the final closing of our Series A, we look ahead to the continued development of our research programs that aim to expand the promise of immuno-oncology by identifying therapeutically relevant antibodies from patients with elite response to cancer immunotherapy in a number of oncology indications."

"OncoResponse’s approach to addressing the unmet need in patients who are partial or non-responders to immunotherapy is harmonized with our investment strategy," said Alvin Syh, General Manager of HT Family Office. "We are honored to be able to support the advancement of this potentially transformative technology at such a crucial time in the Company’s development."

New members of the OncoResponse Scientific Advisory Board include:

Mike Gallatin, Ph.D.

Dr. Gallatin is a Senior Advisor on the Frazier Healthcare Partners Life Sciences team and brings over 35 years of experience as a scientist and executive to the OncoResponse Scientific Advisory Board. He co-founded Calistoga Pharmaceuticals (acquired by Gilead) and Stromedix Pharmaceuticals (acquired by Biogen Idec) and served as President of Calistoga, which was the first company to demonstrate the clinical benefit of an isoform selective PI3K (idelalisib) inhibitor in hematologic malignancies. Dr. Gallatin was one of the founding scientists at ICOS Corporation (acquired by Eli Lilly and Company), where he served as VP and Scientific Director. Earlier in his career, Mike developed expertise in the fields of immunology/inflammation and oncology while on the faculty at the Fred Hutchinson Cancer Center. Dr. Gallatin received his Ph.D. from the University of Alberta Department of Immunology and was a Damon Runyon-Walter Winchell and American Cancer Society fellow at Stanford University in the laboratory of Dr. Irving Weissman.

Albert Yu, M.D.

Dr. Yu has over 20 years of clinical drug development experience and expertise. He previously served as Vice President of Clinical Development at Omeros Corporation and as Chief Medical Officer and Vice President of Clinical Affairs at Calistoga Pharmaceuticals. At Calistoga, he played a key role in the development of Zydelig for the treatment of non-Hodgkin lymphoma and chronic lymphocytic leukemia. Prior to that, he was Senior Director of Clinical Operations, Research & Affairs at ICOS Corporation, where he was instrumental in the development of Cialis for the treatment of erectile dysfunction. Dr. Yu received his B.S. in biology from Massachusetts Institute of Technology and his M.D. from the University of Washington. He is trained in internal medicine, critical care medicine and pulmonary disease.

Elizabeth M. Jaffee, M.D.

Dr. Jaffee is an international leader in the development of immune based therapies for pancreatic and breast cancers. She is currently Deputy Director of the Sidney Kimmel Comprehensive Cancer Center and Professor of Oncology at Johns Hopkins University, where her research focuses on evaluating mechanisms of immune tolerance to cancer, the identification of human tumor antigens recognized by T cells, and the analysis of antitumor immune responses in breast and pancreatic cancers. Prior to joining Johns Hopkins, she served as a research fellow and principal investigator at the University of Pittsburgh. Dr. Jaffee received her B.A. in biology and immunology magna cum laude from Brandeis University and her M.D. from New York Medical College. She completed her medical residency at Presbyterian-University Hospital in Pittsburgh, Pennsylvania.