BioMarin Drops Pompe Drug In Hunt For Profitability

BioMarin continues to pare down its portfolio in order to focus its cash on higher value assets in an effort to reach profitability.

Less than two weeks after BioMarin Pharmaceutical Inc. opted to discontinue the entire development of the Prosensa portfolio, the company mentioned it would be abandoning another mid-stage asset for Pompe disease as it tries to be more prudent about spending.

BioMarin has said publicly that it hopes to break even or better in 2017, but that goal is going to require hitting certain revenue goals as well as tightening spending.

"There's spending controls. That's one of the drivers behind the recent announcement on the DMD program, we're going to be dialing that back significantly. And in addition to that, the 701 program which we like quite a lot. We've decided that we will not be taking that forward ourselves into a Phase III, and instead we'll be searching for a partner to take the program on and so that's just all part of the same thing which is managing our R&D investment to allow us to build a great portfolio and drive to profitability," said BioMarin Chief Financial Officer Daniel Spiegelman at the Goldman Sachs investor conference on June 9.

BMN-701 is a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease, a rare disease that causes muscle weakness and eventually effects the heart, lungs and nervous system.

"It's really overall portfolio management and prioritization. And the fact that now, we are – this year, we're passing $1 billion in revenues. We want to focus on projects that will add significant top line here, without too much of an R&D investment. And I would say now, our decisions regarding our product portfolio, whether it's late-stage, preclinical or clinical, I think we decided that any product that cannot reach at least $500m of peak sales is probably not something we want to get involved in," added CEO Jean-Jacques Bienaime, who estimates BMN-701 could only reach peak sales of $400m.

BioMarin acquired the asset in August 2010 when it bought its developer ZyStor Therapeutics for $22m upfront and $93m in development milestones. [See Deal]

Biomedtracker analysts believe that the likelihood of approval for the drug was 29%, 5% above the average.

Leerink Swann analyst Joseph Schwartz believes the discontinuation of BMN-701 was a sensible move for the company. "We believe BMRN's decision to discontinue its development programs underscores its discipline for achieving break-even/profitability (non-GAAP) by FY2017," he wrote in a June 9 note to investors. "Cutting losses early to reinvest in more promising assets, like BMN250 (MPS IIIB/Sanfilippo B), seems prudent to us."

Schwartz points out that both Sanofi and Amicus Therapeutics Inc. have drugs in development for Pompe disease that could benefit from the exit of competition.

The company abandoned the development of its Duchenne muscular dystrophy programs at the beginning of June after FDA made it clear that its lead program, Kyndrisa (drisapersen) was unapprovable. The drug had previously been shot down by an advisory committee, despite strong support from the DMD community. (Also see "BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over" - Scrip, 1 Jun, 2016.)

The biotech also chose to discontinue development of three earlier follow-on assets to Kyndrisa -- BMN 044, BMN 045 and BMN 053 – which were in Phase II studies for distinct forms of the disease. All four drugs were acquired by BioMarin in its November 2014 takeover of Prosensa Holding BV for $691m in cash and $160m in earn-outs. [See Deal] BioMarin has now abandoned virtually the entire Prosensa portfolio.