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    Glaucoma drug granted orphan status

    The European Medicines Agency (EMA) has granted orphan drug designation to ISTH0036, a locked nucleic acid-modified antisense oligonucleotide under development by Munich-based Isarna Therapeutics, for the prevention of scarring after glaucoma filtration surgery (GFS). The company now is eligible for development fee reductions and incentives, including 10-year market exclusivity for the drug.

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    “Currently, patients with advanced-stage glaucoma have limited long-term treatment options,” Philippe Calais, PhD, president and chief executive officer of the company, said in a statement. “The EMA’s recognition of the potential of ISTH0036 to help protect glaucoma patients’ vision in this stage of their disease with orphan drug status is a significant step forward for the ongoing development of this novel therapy.”

    The news follows preclinical studies of the drug in animals and the launch of a Phase I trial in humans.

    Transforming growth factor beta 2 (TGF-ß2) is substantially elevated in the eyes of people with glaucoma and has been identified as playing a critical role in the pathophysiology of glaucoma because it:

    (1) Affects changes in the trabecular meshwork that can lead to open-angle glaucoma.

    (2) Has a direct pathophysiologic effect on the optic nerve head.

    (3) Leads to so-called “bleb closure” post-GFS by driving the fibrosis/scarring process.

    GFS often is the last line of treatment for patients who have advanced-stage glaucoma, but postoperative scarring can cause the drainage canal that was opened during surgery to close, leading to surgery to be unsuccessful and causing IOP to increase again. Isarna Therapeutics, known as Antisense Pharma GmbH until late 2013, says that ISTH0036 is the only compound under clinical development that directly targets TGF-ß2.

    In April, the company began a first in-human Phase 1 trial of the drug in patients with advanced-stage glaucoma post-GFS. Conducted at the University Hospitals of Mainz and Tuebingen, Germany, the trial is expected to enroll about 24 to 30 patients, and they will be treated with escalating doses of ISTH0036. In addition to testing safety, researchers will monitor with an eye toward patients for IOP and visual field preservation.

    Next: Trial analysis

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