Analyst Conference Summary

biotechnology

conference date: August 2, 2017 @ 1:30 PM Pacific Time
for quarter ending: June 30, 2017 (second quarter, Q2 2017)

Forward-looking statements

I missed the live conference because of a conflict, and at this point they have not bothered to post the replay, and I have to get up early for a heavy day on Thursday, so I'll just leave the data gleaned from the press release below.

Overview: Revenue growing surprisingly slowly (to me), but gene-therapy announcement is promising.

Basic data (GAAP):

Revenue was $317.4 million, up 4% sequentially from $304 million, and up 6% from $300.1 million year-earlier.

Net income was negative $36.8 million, down sequentially from negative $16 million, but up from negative $419 million year-earlier.

Diluted EPS was negative $0.21, down sequentially from negative $0.09 and up from negative $2.58 year-earlier.

Guidance:

Conference Highlights:

Jean-Jacques Bienaimé, Chairman and CEO of BioMarin said, "We expect that trajectory to continue during the second half of 2017 as we execute our Phase 3 program developing both the 4e13 vg/kg and 6e13 vg/kg doses in two separate studies with BMN 270; filing the MAA for pegvaliase in Europe; and providing updates across our development pipeline at R&D Day on October 18. With these investments in our pipeline and commercial organization, while we expect to incur a GAAP net loss for 2017, we expect to achieve non-GAAP income for the full-year 2017, as planned."

Based on updated Phase 1/2 data from 6 patients, BioMarin will initiate trials for BMN 270 for hemophilia A at two doses: 6e13vg/kg and 4e13vg/kg.

In April the FDA approved Brineura for CLN2, late-infantile form of Batten disease. Europe followed suit in June.

BioMarin settled with Par Pharmaceutical, essentially allowing them to market Kuvan starting October 1, 2020. BioMarin's European patent is valid until 2024.

Sarepta bought EXONDYS 51 rights for $35 million plus possible further milestone paments and royalties.

Non-GAAP net income was $26.6 million, down 24% sequentially from $35 million, and up 56% sequentially from $17.0 million year-earlier. Stock-based compensation excluded was $ million.

Cash and equivalents ended at $1.21 billion, flat sequentially from $1.21 billion. Long term convertible debt was $676 million.

Total operating costs (GAAP) were $356 million, consisting of: cost of sales $56 million, research and development $143 million, selling, general and administrative $144 million, and intangible amortization & contingent consideration of $13 million. Loss from operations was $39 million. Other expense net $7 million. Income tax benefit $9 million.

Brineura (Cerliponase alfa) for CLN2, late-infantile form of Batten disease BLA was approved by the FDA in April 27. Commercial launch, but because children with CLN2 die quickly, there is not a relatively large pool of diagnosed patients waiting. Also recommended for approval by the CHMP in Europe. The U.S. launch has begun.

Pegvaliase for phenylketonuria (PKU) Phase 3 results met the primary endpoint. Biogen submitted a Biologics License Application (BLA) to U.S. FDA in

BMN 270 gene therapy product for hemophilia A: interim results established proof of concept. Further data released in January were positive. Biomarin plans a Phase 2b study designed to be registration enabling, possibly in Q3. 6 patients have been dosed so far. Began building the manufacturing facility for this.

BMN 250 for MPS IIIB (Sanfilippo Syndrome Type B) preliminary Phase 1/2 data released in January showed reduced heparan sulfage biomarker at 30 mg. Newer patients are safely at 300 mg. Study will now move to the expansion phase.

Vosoritide for achondroplasia Phase 3 study was initiated in December. Primary endpoint is growth velocity in children.

Q&A:

[note this is a summary, not a transcript. And very selective, given the length of this particular Q&A session]

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