Orphan Status for BioLineRx' Candidate


The U.S. Food & Drug Administration (:FDA) recently granted orphan drug designation to BioLineRx’ (BLRX) BL-8040 for treating people suffering from acute myeloid leukemia (:AML).

We note that orphan drug designation is given to those drugs and biologics that are meant for the safe and effective treatment, diagnosis or prevention of rare diseases that affect not more than 200,000 people in the U.S.

The orphan drug designation status for the candidate comes close on the heels of the U.S. Patent and Trademark Office issuing a new U.S. patent to BL-8040 (8,455,450). The patent, which is expected to expire in Nov 2029, will cover BL-8040 for using its composition for obtaining hematopoietic precursor cells (e.g. stem cells) for use in bone marrow transplantation.

BioLineRx’ BL-8040 is currently in a phase II study for the treatment of AML. The multicenter, open-label, phase II study is evaluating the safety and efficacy of the candidate in adults suffering from relapsed/refractory AML. The study is also assessing the pharmacokinetic profile of BL-8040 along with an efficacy evaluation.

BioLineRx is also evaluating BL-8040 for the treatment of thrombocytopenia (reduced platelet production). Last month the company reported positive results from pre-clinical trials on the candidate, where it was proved to be effective for the treatment of thrombocytopenia. Results further revealed that BL-8040 increased the number of megakaryocytes (cells that produce platelets) within the bone marrow.

We remind investors that in Sep 2012, the rights to BL-8040 were in-licensed by BioLineRx from Biokine Therapeutics Ltd.

We are encouraged by the company’s progress with its oncology candidate, BL-8040. However we note that the oncology market is extremely crowded with the presence of big companies like Roche (RHHBY).

BioLineRx presently carries a Zacks Rank #3 (Hold). Meanwhile, other companies like Actelion Ltd. (ALIOF) and Gilead Sciences Inc. (GILD) look better positioned with a Zacks Rank #1 (Strong Buy).

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