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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced interim data from part 2 portion of an ongoing dose escalation phase I/II study on investigational enzyme replacement therapy, BMN 250, currently developed for treatment of patients with Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB).

This data was presented at the International Congress of Inborn Errors of Metabolism (ICIEM).

Notably, Sanfilippo B is a disease caused by deficiency in enzyme alpha-N-acetyglucosaminidase (NAGLU), one of the four enzymes required for heparan sulfate (HS) degradation.

Shares of BioMarin have underperformed the industry year to date. The stock has increased 8.5% compared with the industry’s 15.7% rally during the period.

The phase I/II (BMN 250-201) study (n=3) was conducted in two parts. While the part 1 portion is focused on safety and pharmacodynamic activity in patients treated with higher doses (30mg, 100mg, 300mg), the part 2 portion is concerned with observing cognitive function of patients treated with 300mg dose.

Data from the trial demonstrated improvement in cognitive Development Quotient (DQ), a measure of cognitive function normalized to age, in two of the three patients compared with their pre-dose baselines. Additionally, data also showed rapid decreases in liver size to normal range from baseline in the same patients. Notably, patients with untreated Sanfilippo B usually show progressive decline in DQ.

We remind investors that in January, the company presented positive, preliminary results from the part I portion of the phaseI/II study. Data from the research demonstrated that BMN 250 reduced heparan sulfate levels to normal range in cerebral spinal fluid of MPS IIIB patients.

Significantly, BMN 250 enjoys an orphan drug status in both the U.S. and the EU for the given indication.

Per the company’s press release, approximately 2,000-3,000 patients are living with Sanfilippo B syndrome in territories where Biomarin operates. Hence, approval of the candidate will provide the company an access to the market, holding huge potential.

Apart from BMN 250, other interesting candidates include pegvaliase and BMN 270. While pegvaliase is under review in the United States for treatment of phenylketonuria (PKU), BMN 270 is being evaluated in phase I/II study for curing hemophilia A. Successful development and commercialization of these candidates will help drive long-term growth at BioMarin.

Zacks Rank & Stocks to Consider

BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the pharma sector are Akebia Therapeutics, Inc. (AKBA - Free Report) , Aduro Biotech, Inc. (ADRO - Free Report) and ACADIA Pharmaceuticals Inc. (ACAD - Free Report) , all three carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Akebia’s loss per share estimates narrowed from $4.14 to $3.85 for 2017 and from $1.98 to $1.88 for 2018 over the last 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 25.93%. Its share price soared 58.4% so far this year.

Aduro Biotech’s loss per share estimates reduced from $1.46 to $1.32 for 2017 and from $1.26 to $1.24 for 2018 over the last 30 days. The company delivered positive surprises in two of the trailing four quarters with an average beat of 2.53%.

ACADIA’s loss per share estimates narrowed from $2.80 to $2.57 for 2017 and from $2.06 to $1.90 for 2018 over the last 30 days. The company came up with positive earnings surprises in two of the last four quarters with an average beat of 7.97%.

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