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BioMarin’s Strong Pipeline Could Be a Long-Term Growth Driver

Daniel Collins - Author

Sep. 12 2017, Updated 7:37 a.m. ET

Clinical trials with BMN 270

After success in the company’s phase 1/2 trial with BMN 270, an investigational gene therapy for hemophilia A, BioMarin Pharmaceuticals (BMRN) is expected to start phase 3 trials. In August 2017, BioMarin Pharmaceuticals announced its plans to start two separate phase 3 trials for 4e13 vg/kg dose and 6e13vg/kg dose.

In the phase 1/2 trial in November and December, at week 32, all three patients who were administered 4e13 vg/kg dose achieved a normal range of factor VIII activity levels, demonstrating median and mean factor VIII levels of 51%. Also, among the three patients who were administered 4e13 vg/kg dose in February and March 2017, everyone’s Factor VIII activity levels had progressed to a mild range. Two of the three patients continued to show an upward trend of factor VIII activity levels.

The above chart shows BioMarin Pharmaceuticals’ pipeline portfolio.

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Clinical trial with BMN 250

In September 2017, BioMarin Pharmaceuticals presented interim statistics from the dose escalation arm of phase 1/2 trial with BMN 250. BMN 250 is an enzyme replacement therapy under investigation that utilizes a novel fusion of recombinant human Alpha-N-acetylglucosaminidase (or NAGLU) with a derived peptide of insulin-like growth factor 2 (or IGF2) for the treatment of individuals with Sanfilippo B syndrome or mucopolysaccharidosis IIIB (or MPS IIIB). The phase 1/2 trial demonstrated tolerability of ICV-administered BMN 250 in Sanfilippo B patients. The primary aim of the study was to evaluate the safety of BMN 250 in Sanfilippo B patients.

BioMarin Pharmaceuticals’ peers in the hemophilia drug market include Pfizer (PFE), Bioverativ (BIVV), Bayer (BAYZF), Novo Nordisk (NVO), CSL, and others. The iShares Russell Mid-Cap Growth ETF (IWP) invests ~0.55% of its total portfolio holdings in BioMarin Pharmaceuticals.


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