BioMarin disclosed late Wednesday that longtime CEO JeanJacques (JJ) Bienaimé is retiring effective December 1 and will be succeeded by Alexander Hardy, who is currently the CEO of Genentech at Roche. 

Bienaimé has led the San Rafael, California-based biotech for 18 years and was named chairman in 2015. 

Hardy, for his part, has helmed South San Francisco-based Genentech for more than four years and brings more than three decades of industry experience. Richard Meier, current lead independent director at BioMarin, will become board chair as part of the leadership team update.

“I deeply admire the strong foundation and purpose-driven culture that JJ and the BioMarin team have built and am honored to lead BioMarin at such a pivotal time in the company’s history,” Hardy stated

The C-suite shuffle, which coincided with another big life sciences leadership succession — Thursday’s announcement by Bristol Myers Squibb that Chris Boerner is assuming the CEO reins from Giovanni Caforio – was announced the same day BioMarin reported its third-quarter earnings. 

Revenue of $581 million fell short of Wall Street consensus of $608 million, partly due to new hemophilia A gene therapy Roctavian’s sales miss. 

That said, analysts had nothing but praise for the managerial aspect of BioMarin’s big news day.

“We view [Hardy] as a logical choice,” for BioMarin’s next phase, wrote Leerink analyst Joseph Schwartz in an investor note Thursday. 

“Some could view the timing of the announcement as curious,” he added, “given Roctavian is still in the early stages of launch – though we believe that this was long in the making and Hardy is a capable operator.”

Roctavian’s third-quarter sales of $800,000 fell short of even the stingiest of projections. The company, which had initially set the drug’s fiscal year 2023 sales guidance in the range of $50 million to $150 million, lowered that guidance Thursday to less than $10 million. That was a bigger downward revision than many anticipated.

Still, the drug, approved in June and priced at $2.9 million for a single dose, was expected to have a slower launch trajectory, given the time required to funnel patients through to treatment: It took a year after Roctavian’s EMA approval for a single German patient to get the therapy, and so far just two EU patients have been dosed.

BioMarin expects sales to ramp up next year after Roctavian’s final price is published in Germany and site-readiness and administrative delays are resolved in the U.S., where three patients have completed all pre-dosing tests. Those tests can take between two to five months to complete.

“The gene therapy launch continues to experience some inertia and is progressing much slower than expectations,” Schwartz observed. “The company has made incremental progress in the U.S. to align with stakeholders, though the first patient has still not been dosed.”

Negotiations over how 340(b) prescription drug discounts will be shared by certain parties at the provider level are another reason for the slow build, according to analysts. Unique one-time payment and reimbursement hurdles have added to the complex administrative tasks required of hemophilia treatment centers. 

However, progress is being made. Nearly two-thirds of covered U.S. lives have been issued Roctavian coverage policies. Additionally, signed outcomes-based warranties are also in place with more than 95 million lives, whereby BioMarin will refund up to 100% of the cost, on a prorated basis, if there is any lack of response during the warranty period. 

On a brighter note, BioMarin posted another solid quarter for Voxzogo. The achondroplasia drug’s revenue of $123.1 million beat sales forecasts, prompting the firm to raise guidance for the third time this year — to a range of $435 million to $455 million. 

The company also made headway in relieving Voxzogo’s tight supply situation. Those issues are expected to dissipate by the middle of next year, and BioMarin foresees having about 2,600 patients on the drug by the end of 2023. 

Which bodes well for Voxzogo’s expansion into other short-stature indications. A hypochondroplasia Phase 3 trial is set to start in 2024, with plans to test the drug in idiopathic and polygenic short stature, as well. 

In addition, expansion into achondroplasia patients under five and two years old in the U.S. and EU, respectively, should add roughly 1,000 addressable patients to BioMarin’s territories.

All told, BioMarin’s third-quarter sales grew 15% year-on-year, primarily driven by Voxzogo and enzyme product Palynziq, while enzyme drug Aldurazyme came in below expectations. The company also slightly refined its total revenue guidance, but said it still expects just under $2.5 billion in total revenues and just under $1.8 billion from its enzyme products.

When Bienaimé joined BioMarin in 2005, it had a single marketed product and about $26 million in revenue. The CEO not only steered the company to profitability with major expansion of its commercial portfolio. 

He also oversaw a major expansion of the workforce, from about 300 employees only in the U.S. to more than 3,000 people in almost 80 countries today.

“It has been a privilege leading BioMarin’s exceptionally talented team over the past 18 years, ” Bienaimé stated.

BioMarin said Bienaimé will remain a member of the board until the company’s 2024 annual shareholder meeting and will serve as an advisor through the end of next year.