orphan

Aptorum Group Announces Significant Progress of Repurposed Drug Candidate, SACT-1 for Neuroblastoma Targeting IND Submission in H2 2020

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Aptorum Group Limited (Nasdaq: APM) (“Aptorum Group”), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, announces positive data and development in relation to its repurposed drug candidate, SACT-1, for the treatment of neuroblastoma, a rare type of childhood cancer that develops in infants and young children. Subject … Skaityti toliau

AOP Orphan, AOP Orphan IP AG and orphanix GmbH join forces to develop RetinolX to prevent bronchopulmonary dysplasia

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Vienna (pts036/13.05.2019/13:30) – AOP Orphan IP AG and AOP Orphan Pharmaceuticals AG have entered into a partnership with orphanix GmbH to jointly develop RetinolX, an innovative medicine for the prevention of Bronchopulmonary Dysplasia (BPD). BPD is a life-threatening lung disease affecting very preterm infants. Around 1 % of births are very preterm and potentially affected … Skaityti toliau

AOP Orphan announces EU Marketing Authorization for BESREMi® (Ropeginterferon alfa-2b) for rare blood cancer (PV)

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Vienna (pts022/20.02.2019/15:20) – * BESREMi® is approved as first-line monotherapy in adults for the treatment of Polycythaemia vera (PV) without symptomatic splenomegaly (1) * BESREMi® showed high and durable hematologic response, molecular response and disease modifying capabilities without leukemogenic or carcinogenic potential (2),(3) * BESREMi® was safe, well tolerated and will be available as a … Skaityti toliau

AOP ORPHAN AND CHORD ANNOUNCE A PARTNERSHIP TO DEVELOP CRD1 (CLADRIBINE) FOR TREATMENT OF ORPHAN AUTOIMMUNE DISEASES

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Vienna and Geneva (pts017/03.01.2019/15:00) – AOP Orphan Pharmaceuticals AG (AOP Orphan) and Chord Therapeutics (Chord) step into a new partnership to develop CRD1 for treatment of orphan autoimmune diseases. CDR1 is a new formulation of cladribine, an immunosuppressant with promising activity against autoimmune diseases. Autoimmune neurological diseases are a family of severe disorders that result … Skaityti toliau

AOP Orphan announces positive CHMP opinion for Ropeginterferon alfa-2b/BESREMi®

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Vienna (pts010/17.12.2018/11:20) – * AOP Orphan´s EMA marketing authorization application for Ropeginterferon alfa-2b/BESREMi® resulted in a positive opinion adopted by EMA´s CHMP and is expected to receive formal approval within the EU in Q1 2019 * BESREMi® is indicated as a monotherapy in adults for the treatment of Polycythaemia vera without symptomatic splenomegaly * BESREMi® … Skaityti toliau

AOP Orphan announces three-year results on Ropeginterferon alfa-2b in Polycythemia Vera at ASH

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Vienna (pts046/03.12.2018/19:30) – * High rates of durable hematological response and symptom control with good tolerability were reconfirmed with Ropeginterferon alfa-2b after 36 months of treatment. * Disease Modification by Ropeginterferon alfa-2b was illustrated by high molecular response rates, associated with the ability to reduce allelic burden of both mutant JAK2 and importantly also non-JAK2 … Skaityti toliau