What It's Like To Have Dinner With BioMarin Executives

After having dinner with BioMarin’s management team, Schoenebaum shared six takeaways:

1) Confidence In Drisapersen

Management, one again, said it is confident that drisapersen could be potential approved in the United State. before the end of the year.

The team said a few recent interactions it had with the FDA since it closed Prosensa in January were the main reasons behind its increased confidence “from the time of the deal announcement last November, when BMRN only had the FDA meeting minutes (which alone were enough to drive the purchase of Prosensa).”

Related Link: UBS Analysts Might've Found Another BioMarin Catalyst

2) Design Options For Drisapersen Study

The FDA seems to be open to a variety of designs for the drisapersen confirmatory study, including a “pbo-controlled study of drisapersen in exon 51 – a study that management believes could be enrolled ex-US and would have important commercial implications with payers.”

The company is waiting until it receives the final guidelines from the FDA to commence with the confirmatory study, which it think will be finalized post-AdCom.

3) Possible Prevention Of Sarepta Marketing

Management think that, if drisapersen is approved in the EU in 2016, its orphan designation could possibly prevent Sarepta Therapeutics Inc (NASDAQ: SRPT) from marketing; “whereas in the US the bar for orphan exclusivity is lower and both BMRN and SRPT should be able to market. About ~30 percent of the global addressable market for drisapersen is in EMA governed territories (and the US only represents ~15 percent of the market).

“Recall, BMRN is expecting a CHMP opinion for drisapersen in 1Q 2016,” the research note added.

4) Hemophilia A Gene Therapy

BioMarin will also evaluate Hemophilia A gene therapy. The company will dose the first patient very soon, and the analysts at Evercore ISIS believe data could be released by the end of the year.

5) PEG-PAL Study

For PEG-PAL, the experts anticipate “data from the phase 3 randomized discontinuation study [is] set to read out late Q1 next year. Based on recent discussions with the FDA, management believes that only Phe lowering will be required for approval, yet a statistically significant result in neurocog will be useful commercially,” the report noted.

6) Non-GAAP Profitability

Management reiterated the company’s commitment to its non-GAAP profitability guidance for 2017, if drisapersen is approved in either the United States or the EU.

The team also thinks the balance sheet has "considerable" flexibility to do another deal. The analysts noted that BioMarin “would, in theory, do a ‘compelling’ BD deal that would delay 2017 profitability, yet the bar would be exceptionally high and that is not base case expectation.”

Disclosure: Javier Hasse holds no stakes in any of the securities mentioned.

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