On May 17, 2018 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported two data presentations supporting plans for rapid development of avapritinib, a selective KIT and PDGFRα inhibitor, in patients with gastrointestinal stromal tumors (GIST) and systemic mastocytosis (SM) (Press release, Blueprint Medicines, MAY 17, 2018, View Source;p=RssLanding&cat=news&id=2349637 [SID1234526761]).

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Data from a retrospective natural history study of previously treated patients with advanced PDPGFRα D842V-driven GIST will be presented in a poster session at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois on June 1-5, 2018. In addition, updated data from Blueprint Medicines’ Phase 1 EXPLORER trial of avapritinib in patients with advanced SM will be presented in a poster session at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden on June 14-17, 2018.

The accepted abstracts are listed below and are now available online on the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) conference websites, respectively: View Source and View Source

2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting

Presentation Title: A retrospective natural history study of patients (pts) with PDGFRα D842V mutant advances gastrointestinal stromal tumor (GIST) previously treated with a tyrosine kinase inhibitor (TKI)
Session Title: Sarcoma
Session Date & Time: Saturday, June 2, 2018 from 8:00 a.m. – 11:30 a.m. CT (9:00 a.m. – 12:30 p.m. ET)
Abstract Number: 11533
Poster Board Number: 278
Location: Hall A, McCormick Place

23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper)

Presentation Title: Avapritinib (BLU-285), a Selective KIT Inhibitor, is Associated with High Response Rate and Tolerable Safety Profile in Advanced Systemic Mastocytosis (AdvSM): Results of a Phase 1 Study
Session Title: Myeloproliferative neoplasms – Clinical
Session Date & Time: Friday, June 15, 2018 from 17:30 – 19:00 CEST (11:30 a.m. – 1:00 p.m. ET)
Abstract Number: PF612
Location: Poster Area, Stockholmsmässan

On May 17, 2018 Obsidian Therapeutics, Inc., a biotechnology company dedicated to the development of next-generation cell and gene therapies with pharmacologic operating systems, reported that the company presented preclinical data on its regulated IL12 and IL15 programs at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Chicago, IL (Press release, Obsidian Therapeutics, MAY 17, 2018, View Source [SID1234526779]).

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Obsidian is developing CAR-T therapies that incorporate Destabilizing Domains (DDs) to regulate expression of immune cytokines, thereby providing pharmacologic control over these potent but potentially toxic molecules. DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein that is engineered into a cell or gene therapy product. IL12 and IL15 are two important immune cytokines that play important roles in tumor response to adoptive cell therapy but which require precise control to optimize their therapeutic benefit.

"IL12 and IL15 are critical factors that promote CAR-T cell expansion, persistence, and penetration into solid tumors," said Vipin Suri, Ph.D., Vice President of Discovery of Obsidian. "However, unregulated expression of these cytokines by CAR-T or other adoptively-transferred cells can potentially compromise safety and efficacy. Obsidian’s technology allows the treating physician to control expression of IL12 and IL15 via the use of safe, FDA-approved small-molecule drugs, and the preclinical data we present today demonstrate the elegance and effectiveness of this approach."

Highlights of the two preclinical presentations follow:

Abstract number 113: Exogenous In Vitro and In Vivo Regulation of Interleukin-12 Secretion from T Cells Using Destabilizing Domain Technology
Presenter: Dexue Sun

Session: Cancer – Immunotherapy, Cancer Vaccines I

Construction of a single-chain regulated IL12 using a DD derived from FKBP
Demonstration of small molecule-regulated expression of IL12 in a variety of cell types including primary human T cells
Use of different promoters to tune expression level of the regulated cytokine
Demonstration of in vivo regulation of IL12 in adoptively transferred T cells
Development of a CD19 CAR construct co-expressing regulated IL12, with in vitro data showing effective performance of the regulated cytokine cassette
Abstract number 133: Dose dependent exogenous regulation of membrane bound Interleukin-15-Interleukin-15 receptor alpha fusion protein for adoptive T-cell therapy
Presenter: Christopher Reardon
Session: Cancer – Targeted Gene & Cell Therapy I

Design of regulated membrane-bound IL15-IL15 Receptor Alpha (mbIL15) fusion construct incorporating DDs for pharmacologic control with small molecule ligands
Construction of mbIL15-DD construct incorporating human DDs, regulated by FDA-approved small-molecule drugs
Dose- and time-dependent regulation of mbIL15 expression in multiple cell types
Regulation of mbIL15 expression on primary human T cells in vivo
About Destabilizing Domains

Obsidian uses Destabilizing Domains (DDs) to enable pharmacologic regulation of protein activity for next-generation cell and gene therapies. Obsidian’s DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein. In the absence of a specific small-molecule ligand, the fusion protein is rapidly degraded, whereas in the presence of the ligand, the fusion protein becomes stable and functional. Obsidian uses this approach to equip engineered cells with controllable functions that can be precisely tuned by the administration of non-immunosuppressive, small-molecule medicines that are readily available and dispensed by the treating physician.

On May 16, 2018 Kiadis Pharma N.V. ("Kiadis Pharma" or the "Company") (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing a T-cell immunotherapy product candidate designed to reduce Graft versus Host Disease (GVHD) and relapse after hematopoietic stem cell transplantations (HSCT), reported that it is scheduled to attend the following investor conferences in May and June 2018 (Press release, Kiadis, MAY 16, 2018, View Source [SID1234526672]):

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Bio€quity Europe
May 14-16, 2018, Het Pand, Ghent, Belgium
Arthur Lahr, Chief Executive Officer, will present on Wednesday May 16, 2018 at 2:40 p.m. CEST

Jefferies 2018 Global Healthcare Conference
June 5-8, 2018, Grand Hyatt, New York, USA
Arthur Lahr, Chief Executive Officer, will present on Thursday June 7, 2018 at 2:00 p.m. EDT

On May 16, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that new data from its isocitrate dehydrogenase (IDH) programs will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held June 1-5, 2018 in Chicago (Press release, Agios Pharmaceuticals, MAY 16, 2018, View Source [SID1234526695]).

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In total, five abstracts led by Agios describing new data from the company’s IDH programs have been accepted for presentation at ASCO (Free ASCO Whitepaper), as well as one abstract led by Celgene Corporation. IDHIFA (enasidenib) is being commercialized in collaboration with Celgene.

The accepted abstracts are listed below and are available online on the ASCO (Free ASCO Whitepaper) conference website: View Source

Oral presentations by Agios:

Title: Phase 1 Study of AG-881, an Inhibitor of Mutant IDH1/IDH2, in Patients with Advanced IDH-mutant Solid Tumors, Including Glioma
Date & Time: Friday, June 1, 2018 from 3:09-3:21 p.m. CT
Oral Abstract Session: Central Nervous System Tumors
Abstract: 2002
Location: S102
Presenter: Ingo K. Mellinghoff, M.D., Memorial Sloan Kettering Cancer Center

Title: Ivosidenib (IVO; AG-120) in Mutant IDH1 Relapsed/Refractory AML: Results of a Phase 1 Study
Date & Time: Saturday, June 2, 2018 from 3:00-3:12 p.m. CT
Oral Abstract Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Abstract: 7000
Location: E450
Presenter: Daniel Aaron Pollyea, M.D., M.S., University of Colorado School of Medicine
This abstract has been selected as part of the "Best of ASCO (Free ASCO Whitepaper)" program to be presented in cities across the country. "Best of ASCO (Free ASCO Whitepaper)" features the top abstracts, highlighting the most cutting-edge science and education from the annual meeting.

Poster presentations by Agios and/or Celgene:

Title: Mutant IDH (mIDH) Inhibitors, Ivosidenib or Enasidenib, with Azacitidine (AZA) in Patients with Acute Myeloid Leukemia (AML)
Poster Session Date & Time: Monday, June 4, 2018 from 8:00-11:30 a.m. CT
Poster Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Abstract: 7042
Poster Board: 102
Poster Location: Hall A
Author: Courtney Denton DiNardo, M.D., University of Texas MD Anderson Cancer Center

Title: AGILE: A Phase 3, Multicenter, Randomized, Placebo-Controlled Study of Ivosidenib in Combination with Azacitidine in Adult Patients with Previously Untreated Acute Myeloid Leukemia with an IDH1 Mutation
Poster Session Date & Time: Monday, June 4, 2018 from 8:00-11:30 a.m. CT
Poster Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Abstract: TPS7074
Poster Board: 133b
Poster Location: Hall A
Author: Eytan Stein, M.D., Memorial Sloan Kettering Cancer Center

Title: Pharmacokinetics/pharmacodynamics (PK/PD) of Ivosidenib in Patients with IDH1-mutant Advanced Solid Tumors from a Phase 1 Study
Poster Session Date & Time: Monday, June 4, 2018 from 8:00-11:30 a.m. CT
Poster Session: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics
Abstract: 2577
Poster Board: 403
Poster Location: Hall A
Author: Bin Fan, Ph.D., Agios Pharmaceuticals

Title: Clinical pharmacokinetics/pharmacodynamics (PK/PD) of Ivosidenib in Patients with IDH1-mutant Advanced Hematologic Malignancies from a Phase 1 Study
Poster Session Date & Time: Monday, June 4, 2018 from 8:00-11:30 a.m. CT
Poster Session: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics
Abstract: 2581
Poster Board: 407
Poster Location: Hall A
Author: David Dai, Ph.D., Agios Pharmaceuticals

On May 16, 2018 Moleculin Biotech, Inc., (Nasdaq:MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that a second U.S. site, located in Lubbock, Texas, has qualified for its clinical trial to study Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML") (Press release, Moleculin, MAY 16, 2018, View Source [SID1234526711]).

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UMC Southwest Cancer Center has now qualified as the second U.S. site for Moleculin’s clinical trial of Annamycin. Dr. Sanjay Awasthi, Division Chief of Hematology/Oncology at Texas Tech University will serve as the site’s Principal Investigator.

"We are extremely pleased to announce our second site, as every new site opened for this clinical trial expands our ability to recruit patients and moves us one step closer to generating the data we are all eager to see," commented Walter Klemp, Moleculin’s Chairman and CEO. "Annamycin represents a unique treatment opportunity to address a critical unmet need in relapsed/refractory AML. Currently approved first-line therapy fails to help a majority of AML patients because of multidrug resistance and the limitations presented by the inherent damage those drugs can do to a patient’s heart ("cardiotoxicity"). Unfortunately, there is no effective second-line therapy for a majority of those patients. Preclinical studies have shown that Annamycin has an ability to avoid the multidrug resistance mechanisms that defeat currently approved AML drugs and is designed to have little to no cardiotoxicity. We hope to reproduce this data in our current Phase I/II clinical study and that Annamycin can finally provide a second-line treatment option for the majority of AML patients."

UMC Southwest Cancer Center

The UMC Southwest Cancer Center in Lubbock, Texas is a nationally recognized leader in the fight against cancer. Their multi-disciplinary approach combines the expertise of Texas Tech Physicians with UMC’s compassionate care and technological advancements. Comprehensive cancer treatment and care is available in one facility, close to home.