CSL's new CEO Paul McKenzie charts course for growth amid expansions in gene therapy, kidney diseases

With a new CEO, a new subsidiary, a newly approved gene therapy and three R&D centers under construction on three different continents, change is coming fast for CSL Limited.

Established more than a century ago in Australia as Commonwealth Serum Laboratories, CSL has held a relatively low profile compared to other companies in its global orbit.

But those days may be over for the world’s largest collector of plasma. The $11.7 billion acquisition of kidney disease specialist Vifor, which it completed eight months ago, adds diversification.

Additionally, by serving as the commercial partner for uniQure’s hemophilia B gene therapy Hemgenix, CSL is set to face a test in the burgeoning field. The drug is the most expensive in the world and carries a list price of $3.5 million per treatment.

“It’s a portfolio both commercially and in R&D that’s well positioned for continuing the journey of sustainable and profitable growth,” CEO Paul McKenzie said in an interview with Fierce Pharma.

McKenzie took over as CEO less than a month ago, moving up from the chief operating officer post to replace Paul Perreault. McKenzie came to CSL four years ago after stints at Merck, Biogen, Bristol Myers Squibb and Johnson & Johnson.

“It is an organization where having internal successors in the key positions is critically important because it is a unique mix of businesses,” McKenzie said.

The global CSL conglomerate consists of plasma biotech CSL Behring, CSL Vifor and CSL Seqirus, its flu vaccine business.

CSL’s head of R&D and chief medical officer Bill Mezzanotte said that the company’s plasma franchise puts it in an advantageous position compared to other major players in the industry. 

“Immunoglobulin has been on the market in some form or another for over 40 years and it still hasn’t hit peak sales and so that’s a pretty unique situation compared to a company that almost eight or nine years away is starting to panic about what are they going to do when something goes off patent,” Mezzanotte said. “It allows us the runway to methodically diversify and take the other risks we're taking.”

Working through the pandemic was challenging for CSL. Plasma donations were down and the company’s COVID-19 vaccine, developed along with the University of Queensland, was abruptly scrapped in December 2020 after trial participants developed an immune response to an HIV protein fragment.

The failure of the vaccine was former CEO Perreault's biggest regret in his 10 years as the CSL's chief exec, he told the Australian Financial Review two months ago.

But the acquisition of Vifor and partnering opportunities—like the one CSL struck with uniQure for Hemgenix—have the company considering an exciting future.

CSL hopes to launch Hemgenix in the United States “very soon,” McKenzie said. The gene therapy also sports approvals in Europe and the U.K.

“We have a number of us physicians that have written prescriptions, but there's a process to go through of course between payers and procedurally to be ready to dose somebody,” McKenzie said.

Amid the expansion efforts, CSL is investing heavily in its R&D operations. The company has research sites under construction in Marburg, Germany; Melbourne, Australia; and Waltham, Mass.

Last week, CSL celebrated the opening of the Waltham site, which will serve as its center for vaccine development. Four months ago. CSL partnered with Arcturus Therapeutics to gain access to its mRNA vaccine technology platform.

“R&D is the business of hope,” Mezzanotte added about the Waltham site. “And nothing screams hope more than a brand-new building with a brand-new purpose.”