Industry Programs

PIPELINE RESEARCH SESSION ON MDS AND MYELOID MALIGNANCIES

Thursday, 9 May | 11:45 - 12:30 | Tivoli Congress Hall

Chair: Prof.  Guillermo Sanz


11:45-12:00

Astex Pharmaceuticals, Inc.

TITLE: REVIEW AND PROGRESS IN THE DEVELOPMENT OF HYPOMETHYLATING AGENTS AT ASTEX PHARMACEUTICALS

Speaker: Mohammad Azab, MD, M Sc., President and Chief Medical Officer, Astex Pharmaceuticals

Abstract:

Since the development and approval of decitabine IV by FDA in 2006 and EMA in 2012 in selected patients with higher risk MDS or elderly AML not candidates for intensive chemotherapy, Astex Pharmaceuticals have continued to develop new molecules to improve on decitabine for treatment of myeloid malignancies. We will review the recent clinical data from those molecules.

 

12:00-12:15

Syros Pharmaceuticals

TITLE: SY-1425: LEVERAGING RARa TARGETING IN AML AND MDS

Speaker: David A. Roth, M.D., Chief Medical Officer, Syros

Abstract:

SY-1425 is an oral, first-in-class selective RARa agonist that is currently in a Phase 2 trial in genomically defined subsets of patients with RARA pathway activation. Early data from the ongoing Phase 2 trial show SY-1425 in combination with azacitidine has high response rates and rapid onset of action in newly diagnosed unfit AML patients positive for RARa and IRF8 biomarkers of RARa pathway activation. Importantly, the combination was generally well-tolerated with no evidence of increased myelosuppression. Data from the trial also show that about approximately a third of AML and MDS patients are positive for the RARa and IRF8 biomarkers. These data, combined with 1425's unique mechanism of action and preclinical synergy with a number of AML and MDS therapies, suggest 1425 may have broad potential as a combination agent in RARa and IRF8 biomarker-positive AML and higher-risk MDS patients.

 

12:15-12:30

Takeda

TITLE: A PRAGMATIC PATIENT-REPORTED OUTCOME STRATEGY FOR RARE DISEASE CLINICAL TRIALS: APPLICATION OF THE EORTC ITEM LIBRARY

Speaker: Jill Bell PhD, Global Outcomes Research, Takeda

Abstract:

Measuring health-related quality of life (HRQOL) in rare disease clinical trials can be challenging. Generic patient-reported outcome (PRO) instruments may lack the sensitivity required to demonstrate clinical change brought about by new therapies. To improve the ability of the EORTC-QLQ-C30 to detect clinically meaningful treatment benefit in MDS/CMML and AML, supplemental items were integrated to enhance conceptual coverage in the context of these rare diseases.

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IMPROVING PATIENT OUTCOMES IN MYELODYSPLASTIC SYNDROMES

SYMPOSIUM IS ORGANISED AND SUPPORTED BY Novartis

Thursday, 9 May | 18:00 - 19:00 | Tivoli Congress Hall

Chair: Andreas Glenthøj (Denmark)

18:00 - 18:05  WELCOME AND INTRODUCTIONS
                       Andreas Glenthøj (Denmark)

18:05 - 18:20  WHY SHOULD WE MANAGE IRON OVERLOAD IN PATIENTS WITH                             MYELODYSPLASTIC SYNDROMES?

                       Emanuele Angelucci (Italy)

18:20 - 18:35  HOW CAN WE OPTIMIZE THE BENEFITS OF IRON CHELATION                                   THERAPY IN CLINICAL PRACTICE?

                       Norbert Gattermann (Germany)

18:35 - 18:50  FUTURE DIRECTIONS FOR TREATMENT OF MYELODYSPLASTIC                                   SYNDROMES

                       David Valcarcel (Spain)

18:50 - 19:00  Q&A - MEETING CLOSE

                       Andreas Glenthøj (Denmark)

 

NAVIGATING THE PATHWAYS TO INEFFECTIVE ERYTHROPOIESIS IN MDS

SYMPOSIUM IS ORGANISED AND SUPPORTED BY Celgene

Friday, 10 May | 12:00 - 13:15| Tivoli Congress Hall

Chair: Pierre Fenaux (France)

12:00 - 12:10  WELCOME AND INTRODUCTIONS
                       Pierre Fenaux (France)

12:10 - 12:30  GENETIC CHANGES UNDERLYING INEFFECTIVE ERYTHROPOIESIS

                       Rafael Bejar (USA)

12:30 - 12:50  THE SIGNIFICANCE OF THE MICROENVIRONMENT IN                                                 INEFFECTIVE  ERYTHROPOIESIS

                       Marc Raaijmakers (The Netherlands)

12:50 - 13:10  TGF-Β SUPERFAMILY/SMAD SIGNALLING: A PATHWAY TO                                         INEFFECTIVE ERYTHROPOIESIS

                       Uwe Platzbecker (Germany)

13:10 - 13:15  CLOSING REMARKS AND TAKE-HOME MESSAGES

                       Pierre Fenaux (France)


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