BioMarin laid out a new corporate strategy on Wednesday, following months of reorganization and multiple staff reductions.
The new structure relies on three key business units: skeletal conditions, enzyme therapies and Roctavian, its hemophilia A gene therapy that’s being scaled back after missing initial sales forecasts. Executives said at BioMarin’s Wednesday investor day that the company has been implementing a “cost transformation program” that’s expected to continue into next year and save $500 million in operating expenses.
Over the last several months, BioMarin cut a handful of early programs and hundreds of jobs, including 225 layoffs announced last month. CFO Brian Mueller stressed on Wednesday that the cost savings program involves more than “outright cost-cutting.”
“We strategically evaluated our operating model and opportunities to achieve new cost efficiencies,” he said.
The company is also padding its balance sheet with a new $600 million revolving credit facility announced Wednesday.
When asked if more staff cuts are coming, a spokesperson told Endpoints News that “the majority of the headcount reductions related to cost transformation are behind us.”
Product portfolio
The new, leaner pipeline will allow BioMarin to focus on “high-quality assets that meet a new higher bar,” according to CEO Alexander Hardy, including the achondroplasia treatment Voxzogo.
Hardy called Voxzogo “the most exciting multi-indication product in our portfolio.” He highlighted five experimental programs beyond achondroplasia, a type of genetic condition that causes dwarfism.
Voxzogo sales totaled $470 million in 2023, but Hardy predicts BioMarin’s skeletal conditions unit could reach $5 billion in peak sales. The next most advanced Voxzogo program is in hypochondroplasia, another form of dwarfism. Phase 3 results are expected to be announced in 2026.
Stifel analysts said Wednesday that there will likely be debate about “how much ‘room'” BioMarin’s plans allow for Voxzogo competition. BridgeBio said earlier this year that it expects to complete Phase 3 enrollment for its own achondroplasia candidate infigratinib by the end of this year, and Ascendis Pharma said on a recent earnings call that it’s expecting pivotal results for its achondroplasia program “in the coming weeks.”
Executives said Wednesday that they are confident Voxzogo can “grow through competition.”
Hardy also touted BioMarin’s portfolio of enzyme therapies, which he said have been “the primary driver of growth in BioMarin’s history.” That includes its top-selling enzyme therapy Vimizim for a rare lysosomal storage disorder, which generated $701 million in 2023. Hardy said the portfolio is “very durable with low competitive threats and relatively low level of impacts from policies such as the Inflation Reduction Act.”
Across the board, BioMarin is aiming for 11 launches by 2034 and $4 billion in revenue by 2027. Hardy said the revenue guidance isn’t dependent on business development, though deals may be struck to “augment” the plan — especially now that BioMarin has brought prominent dealmaker James Sabry on board.
Business development strategy
James SabryWhile BioMarin didn’t go into much detail on its dealmaking strategy, Hardy said the company has “constantly been screening potential opportunities.”
Hardy recognized Sabry as “one of the most well-known dealmakers,” adding that his appointment “should give investors confidence that we’re going to do good deals really well.” Sabry will join BioMarin next month after a 14-year run at Roche and Genentech.
Hardy said the company will focus on deals of less than $1.5 billion in size.
“We don’t want to run before we can walk,” he said.