5 Things You Need to Know From BioMarin's R&D Day

Orphan drugs and more orphan drugs were on everyone's minds at BioMarin's (NASDAQ: BMRN  ) Research and Development Day held on Tuesday. Here are the highlights from this event.

1. Abuzz over Vimizim
The biggest buzz of the day focused on Vimizim, which targets treatment of Morquio A syndrome (also known as mucopolysaccharidosis IV type A, or MPS IVA). Biomarin submitted the drug for regulatory approval in the U.S. and in Europe earlier this year. The company anticipates a decision by Europe's Committee for Medicinal Products for Human Use, or CHMP, by the end of 2013. An FDA advisory committee is scheduled to discuss Vimizim on Nov. 19, with a decision by the U.S. Food and Drug Administration scheduled for February.  

The FDA advisory committee will focus on three key areas: clinical meaningfulness of results, statistical robustness of results, and duration of effect. BioMarin's team went into detail about why the company thinks Vimizim should fare well in all of those areas.

BioMarin is already working with payers to clear the way for reimbursement, although no price has been set yet. The company expects to hit the market in the U.S. with Vimizim within 30 days after FDA approval.

2. Optimistic about cancer drug
PARP inhibitors weren't viewed too positively after Sanofi's inaparib failed to meet its primary endpoint in a phase 3 study in 2011. However, subsequent analysis suggested that the drug didn't actually inhibit the PARP enzyme very well. Biomarin is optimistic that its PARP inhibitor, BMN673, could achieve success in fighting cancer.

The company presented information that leads the company to believe that BMN673 is "far more potent" than any other PARP inhibitor on the scene currently. BioMarin compared its drug against rivals including AstraZeneca's (NYSE: AZN  ) olaparib, Clovis Oncology's (NASDAQ: CLVS  ) rucaparib, and AbbVie's (NYSE: ABBV  ) veliparib.  

However, one drug not mentioned was Tesaro's (NASDAQ: TSRO  ) PARP inhibitor niraparib, which could present the biggest threat for BMN673. BioMarin and Tesaro are both conducting phase 3 studies this year. Clovis is in phase 1 with rucaparib, while AbbVie and AstraZeneca are in phase 2 with their PARP inhibitors.

 3. Glad about Pompe disease drug endpoint
BioMarin plans to kick off a phase 3 study of experimental drug BMN701 for the treatment of Pompe disease later this year. The company is in talks with the FDA and European regulators about the design of this study.

The late-breaking news for BMN701 was that the FDA accepted maximal inspiratory pressure, or MIP, as the primary endpoint for the phase 3 study. This was clearly good news for BioMarin, because there is a good chance that BMN701 will produce significant improvement in MIP over the duration of the six-month trial. MIP has not been used as a primary endpoint in previous studies.

4. Anticipating possible accelerated process for PEG-PAL
Presenters at the R&D day also talked briefly about PEG-PAL, or pegylated recombinant phenylalanine ammonia lyase. The drug targets treatment of phenylketonuria, or PKU, a rare genetic disorder caused by a deficiency of the phenylalanine hydroxylase, or PAH, enzyme.

Biomarin has a phase 3 study under way for PEG-PAL as a second-line treatment for patients who don't respond to the company's currently available drug, Kuvan. Results from the study are expected by the fourth quarter of 2014. Suyash Prasad, BioMarin's senior group medical director of clinical sciences, said that the company has a good shot at gaining accelerated approval from the FDA.

5. BMN111
Last, but not least, BioMarin addressed what's going on with BMN111. The experimental drug targets the treatment of achondroplasia, which is the most frequently occurring form of dwarfism.

The FDA has previously placed a partial clinical hold on moving forward with a phase 2 study of BMN111. However, that hold has now been lifted. BioMarin expects to begin phase 2 testing in late 2013 or early 2014.

Looking ahead
Overall, Biomarin's update at the R&D day was positive. The company seems poised to do well in the future. I expect Vimizim will gain approval on both sides of the Atlantic. Its pipeline looks solid.

The one concern that investors might have is that most of the good news that Biomarin could get in the immediate future might already be priced into the stock. Shares have climbed more than 46% so far in 2013.

That being said, Biomarin's focus on orphan diseases is smart and could make the company an attractive candidate for an acquisition some time down the road. This is certainly a stock to keep on your radar screen.

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