What's To Come At BioMarin

| About: BioMarin Pharmaceutical (BMRN)

Biomarin (BMRN) is a company that specializes in developing treatments for rare diseases. They currently have 4 approved drugs in Kuvan for PKU, Naglazyme for MPS VI, Aldurazyme for MPS I, Firdapse (approved in the EU only) for LEMS. The majority of their current revenue comes from sales of Naglazyme, with 2012 revenue guidance of $250 to $265 million. Total revenue guidance for 2012 is between $475 to $510 million. Biomarin gets interesting in the second part of this year because of several pending trial readouts.

Upcoming data

  • 3Q 2012: Results for Phase II trial for PEG-PAL for PKU
  • 3Q 2012: Results for Phase I trial for BMN-111 for achondroplasia in healthy volunteers
  • 2H 2012: Results for Phase I/II trial for BMN-673 for solid tumors
  • 4Q 2012: Results for Phase III trial for [BMN-110]GALNS for MPS IVA
  • 4Q 2012: Results for Phase I/II trial for BMN-701 for Pompe disease
  • 4Q 2012/1Q 2013: Initiation of Phase II trial for BMN-111 for achondroplasia in patients
  • 1Q 2013: Market authorization application filings for GALNS[BMN-110] for MPS IVA
  • 1Q 2013: Results for Phase I trial for BMN-673 for hematological malignancies
  • 1Q 2013: Potential initiation of Phase III trial for PEG-PAL for PKU
  • 1Q 2013: IND filing for BMN-190 for LINCL [Batten disease]
  • Mid-2013: Results for PKU-016 Kuvan neurocognitive study
  • 2H 2013: Potential initiation of Phase III trial for BMN-673 for solid tumors or hematological malignancies
  • 4Q 2013: Potential initiation of Phase III trial for BMN-701 for Pompe disease
  • 4Q 2013: Anticipated FDA approval of GALNS[BMN-110] for MPS IVA

The most important of these results are the BMN-110 Phase 3 data in MPS IVA. Patients with MPS IVA are missing the enzyme called galactose 6-sulphatase; currently, there are no specific treatments available for this disease. Note, Biomarin is also running 3 other studies of BMN-110.

This trial completed enrollment in early March. Top-line results are not expected until the fourth quarter of 2012 with tentative plans to file regulatory applications in the first quarter of 2013. Due to physician and patients, the trial overenrolled with 176 patients (vs. 162 planned). Biomarin's trial has 3 arms: placebo, BMN-110 weekly and BMN-110 every other week. BMN-110 is given via intravenous infusion at a dose of 2.0 mg/kg. The trial is scheduled to complete around the end of August. Primary endpoint is change from baseline in endurance as measured by the 6-minute Walk Test at 12-weeks and 24-weeks. The secondary endpoints are change from baseline in urine keratan sulfate (KS) and change from baseline in endurance as measured by the 3-minute Stair-Climb Test. Biomarin previously reported Phase 1/2 data for BMN-110 in MPS IVA. Presentation slides can be found here.

The data show encouraging signs of activity, given the decreases in KS levels and improvements. This study was also a dose-escalation study, patients started at 0.1mg/kg for 12 weeks, then 1.0 mg/kg for 12 weeks and 2.0mg/kg for 12 weeks. The 2.0mg/kg had the best response, it's also the dose being used in the Phase 3 study. The improvements in endurance were similar to those observed in pivotal studies of approved enzyme replacement therapies[like Biomarin's Naglazyme for MPS VI].

The other 2 readouts that will be closely watched are the early results from the Phase 1/2 trial of BMN-673 for solid tumors and the Phase 1/2 results from the BMN-701 for Pompe disease. Biomarin is testing BMN-673, a PARP Inhibitor, for genetically defined cancers. BMN-701 is their attempt at entering the Pompe disease market where Genzyme's Myozyme reigns supreme. Biomarin's preclinical work has shown BMN-701 to have more efficient delivery/superior uptake. Their Phase 1/2 results will need to show results superior to Myozyme to push this forward into Phase 3.

Clearly, Biomarin is trying to carve out a little niche in oncology using their expertise, but PARP inhibitors have had some issues recently. Last year, Sanofi (SNY) announced Iniparib [BSI-201] failed to prolong survival in its first phase 3 trial in metastatic, triple-negative breast cancer (TNBC) and Astra-Zeneca (AZN) followed up by saying they wouldn't pursue olaparib (AZD-2281) in hereditary BRCA1- and BRCA 2-associated breast cancer. Biomarin is evaluating cancer patients whose malignancies have BRCA1 and BRCA2 mutations and other DNA abnormalities. Biomarin believes BMN-673 "to be more selectively cytotoxic with a longer half-life and better bioavailability," whether or not this is true is still to be seen.


Biomarin's recent capital raise should net them roughly $250 million, putting their cash balance over $500 million. It was a wise move to raise capital, since much of their future value rides on some of these pending catalysts, like BMN-110 for GALNS. Their shares could be volatile going into these results. Biomarin has shown expertise in lysosomal storage diseases, so there is a reasonably good chance that their GALNS trial could pan out favorably. Failure of this trial would certainly warrant a pullback. Recent M&A rumors have driven shares more than 10% higher over the past few weeks. It will take a sizable deal to get Biomarin's execs interested in selling.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.