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BioMarin to Begin Early-Stage Study on Gene Therapy for PKU

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has granted the company Investigational New Drug status for its gene therapy candidate, BMN 307 in patients with Phenylketonuria (“PKU”), a rare disease affecting the brain. The company has received approval to its Clinical Trial Application for BMN 307 permitting it to initiate a clinical study in the United Kingdom. The candidate also enjoys Orphan Drug status in the United States and Europe.

BioMarin’s shares have declined 5.2% in the past year compared with the industry’s decrease of 1.2%.

The company is planning to initiate dosing in a phase I/II study — PHEARLESS — in the first quarter of 2020.The study will evaluate single dose administration of BMN 307 for normalize blood phenylalanine (Phe) concentration levels, restoration of natural Phe metabolism and enable a normal diet in PKU patients. The company is already enrolling PKU patients in an observational clinical study, PHENOM, evaluating BMN 307 to measure both established and new markers of disease and clinical outcomes over time.

According to current treatment guidelines, PKU patients need to control their Phe levels throughout their lifetime. This requires lifelong treatment. A successful development of single dose administration of BMN 307 should be a major breakthrough in the PKU treatment field.

The company already has two approved PKU treatments in its commercial portfolio — Kuvan and Palynziq (pegvaliase-pqpz). We note that Kuvan is set to face generic competition beginning October 2020 in the U.S. market. Although Palynziq, an enzyme therapy, is witnessing strong commercial uptake in the United States, it is available under PALYNZIQ REMS, a restricted distribution program. A successful development of BMN 307, a novel gene therapy for treatment of PKU patients, may help the company to offset any loss of sales due to generic competition for Kuvan and restricted access to Palynziq.

Meanwhile, a few other companies are also developing treatments for PKU in early-stage studies, which include Synlogic’s SYBX small molecule candidate, SYNB1618; Homology Medicines’ FIXX gene therapy, HMI-102; and Rubius Therapeutics’ RUBY red cell therapeutic candidate, RTX-134.

Apart from BMN 307, BioMarin has another gene therapy candidate, valoctocogene roxaparvovec, in its pipeline. It is developing the candidate as a potential treatment for severe hemophilia A. The candidate is under review in Europe while the company has submitted a biologics license application seeking approval for valoctocogene roxaparvovec to the FDA.

Zacks Rank

BioMarin carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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