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     141  0 Kommentare Retrophin Announces Agreement to Acquire Orphan Technologies

    Agreement adds OT-58, a novel enzyme replacement therapy in Phase 1/2 development for the treatment of classical homocystinuria

    Compelling strategic fit that will expand pipeline of potential first-in-class therapies targeting rare diseases

    Retrophin to host conference call and webcast today at 5:00 p.m. ET

    SAN DIEGO, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ: RTRX) today announced that it has entered into a definitive agreement to acquire Orphan Technologies Limited, a privately held, clinical-stage biopharmaceutical company focused on the development of product candidate OT-58 for the treatment of classical homocystinuria (HCU). OT-58 is a novel investigational enzyme replacement therapy being evaluated in Phase 1/2 development for the treatment of classical HCU, a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to life-threatening thrombotic events such as stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options, including heavy dietary restrictions and supplemental use of vitamin B6 and betaine, are often ineffective in managing homocysteine levels and a significant unmet need remains.

    “Many people with HCU face a continuous risk of developing life-threatening complications because current treatment options are largely ineffective in managing homocysteine levels,” said Eric Dube, Ph.D., chief executive officer of Retrophin. “OT-58 has demonstrated an ability to meaningfully reduce homocysteine levels in preclinical models and has the potential to ultimately become the first disease modifying therapy for HCU. This promising, novel development candidate fits directly with our mission to identify, develop and deliver life-changing therapies to people living with rare disease and brings exciting growth potential to Retrophin.”

    OT-58 is a PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical HCU — a deficiency in the naturally occurring enzyme cystathionine beta synthase (CBS). A deficiency in CBS prevents regular metabolism from occurring and results in elevated levels of homocysteine. In preclinical studies, OT-58 has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Specifically, dosing of OT-58 in mouse models corrected metabolite levels, including up to 90% reduction in homocysteine levels in plasma and tissues, and appeared to prolong survival, prevent osteoporosis and rescue ocular structure. OT-58 is currently advancing in a Phase 1/2 dose escalation study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. OT-58 has been granted Rare Pediatric Disease and Fast Track designations by the US Food and Drug Administration (FDA), as well as Orphan Drug designation in the US and Europe.

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    Retrophin Announces Agreement to Acquire Orphan Technologies Agreement adds OT-58, a novel enzyme replacement therapy in Phase 1/2 development for the treatment of classical homocystinuria Compelling strategic fit that will expand pipeline of potential first-in-class therapies targeting rare diseases …