With the FDA already rejecting BioMarin Pharmaceutical Inc.'s experimental Duchenne muscular dystrophy (DMD) drug Kyndrisa (drisapersen) and now European regulators indicating they saw no chance for success, the biotech said it was ending development of the drug, plus three other first-generation follow-on products – BMN 044, BMN 045 and BMN 053 – which currently are in Phase II studies for distinct forms of the disease.

BioMarin CEO Jean-Jacques Bienaimé said the decision to terminate the R&D programs for the investigational DMD drugs was "difficult but necessary" at this time.

The move comes as DMD patients await word from the FDA on Sarepta Therapeutics Inc.'s experimental drug eteplirsen – a decision that was supposed to come several days ago, but was put off by regulators.

DMD patients have been on a rollercoaster over the past year – first watching as the FDA in January denied BioMarin permission to market Kyndrisa in the US Also see "BioMarin's Drisapersen 'Compete Response' Shows FDA Flexibility Still Limited" - Pink Sheet, 14 January, 2016., which came after an advisory committee declined to back the drug, followed by the agency's refuse-to-file action on PTC Therapeutics Inc.'s Duchenne drug Translarna (ataluren) ([A#14160223004]).

Then came the blow in April when the FDA's Peripheral and Central Nervous System Advisory Committee said it also could not back Sarepta's drug, riling up parents and patient advocates ([A#14160425005]).

BioMarin pledged to work with physicians, patient groups and regulatory authorities to develop a transition plan for those patients currently being treated with Kyndrisa, BMN 044, BMN 045 and BMN 053.

But the company insisted this is not the end of its DMD pursuit, pointing out it was continuing to "explore" its next-generation oligonucleotides as potential treatments for the rare, progressive muscle-wasting disease, which primarily affects boys, who generally don't live beyond 30 years.

"Our plan now is to invest in research of next-generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder," Bienaimé said.

Financial, Workforce Impacts

BioMarin spokeswoman Debra Charlesworth said "we expect that we are going to have to do some restructuring of parts of the organization that are focused" on the discontinued products. While she said it was too early right now to go into any specifics, "it's likely to have the greatest impact on our operations in Leiden, The Netherlands" – the former home to Prosensa, which BioMarin acquired for $680m in cash last year ([A#14141124003]).

BioMarin's acquisition of Prosensa came after GlaxoSmithKline PLC had pulled out of its deal with the Dutch firm on Kyndrisa following the failure of its Phase III DEMAND study, which showed that the antisense oligonucleotide didn't meet the primary endpoint of statistically significant improvement in the six-minute walking distance test, versus placebo.

Charlesworth said that because of the discontinuation of Kyndrisa and the other three drugs, the company expected to write off the one-time impairment of approximately $580m in the second-quarter.

She said the impact on GAAP net loss guidance was to be determined based on the final restructuring plans, which will be updated in the second-quarter financial results. "We continue to expect to achieve non-GAAP breakeven or better in 2017," Charlesworth said.

She emphasized DMD is only part of BioMarin's portfolio, which includes five FDA-approved products and a robust pipeline of other products nearing submission in the US and Europe.

Sarepta's Opportunity

RW Baird analyst Brian Skorney looked at BioMarin's abandonment of "essentially the entire Prosensa portfolio" as presenting an opportunity in Europe for Sarepta to step in.

Skorney noted that currently, expectations for European sales of eteplirsen are essentially non-existent, given the ongoing litigation between BioMarin and Sarepta over intellectual property covering exon-51 skipping for DMD.

But with BioMarin out of the race, he said Sarepta may have a shot at meaningful European DMD sales.

"Though the argument can be made that BioMarin is still in possession of Prosensa's IP, without active development of any programs covered by the IP, we do not believe it is likely to be a major roadblock and expect Sarepta to more aggressively pursue a path forward in Europe," Skorney declared. "We believe the news that Biomarin is abandoning the Prosensa programs could lead to a settlement that allows Sarepta license to the patents for a relatively low royalty."

BioMarin's Charlesworth declined to comment on whether there was a settlement in the works. "We will seek to ensure that we receive appropriate value for our IP assets," she said.

[Editor's note: This article is also published in Scrip. The Pink Sheet DAILY brings selected complementary coverage from sister publications to our subscribers.]

BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over

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