Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 bil­lion Vi­for buy­out, the com­pa­ny post­ed a dip in prof­its, due in part to a drop in plas­ma do­na­tions amid the pan­dem­ic.

How­ev­er, CEO Paul Per­reault as­sured in­vestors and an­a­lysts on the full-year call that the team has left “no stone un­turned” when as­sess­ing op­tions to grow plas­ma vol­umes. The chief ex­ec­u­tive al­so spelled out pos­i­tive re­sults for the com­pa­ny’s mon­o­clon­al an­ti­body garadacimab in hered­i­tary an­gioede­ma (HAE), though he isn’t re­veal­ing the ex­act num­bers just yet.

“Garadacimab has the po­ten­tial to be an­oth­er flag­ship mol­e­cule for CSL,” Per­reault said on the call, adding in a news re­lease that garadacimab met its pri­ma­ry and sec­ondary end­points and showed “fa­vor­able safe­ty and tol­er­a­bil­i­ty.”

The com­pa­ny’s stock on the Aus­tralian Se­cu­ri­ties Ex­change was down 1.3% on Wednes­day, pric­ing at around $202.

CSL prof­its were down rough­ly 6% for the four quar­ters end­ing June 30, Per­reault re­vealed on Wednes­day. A drop in plas­ma do­na­tions in FY21 con­strained sales of the com­pa­ny’s core plas­ma ther­a­pies in FY22, giv­en the long man­u­fac­tur­ing cy­cle, he said.

“The pan­dem­ic has put us two years be­hind pro­ject­ed growth in plas­ma col­lec­tions – which is sub­op­ti­mal for pa­tient care,” Per­reault said in a news re­lease.

CSL’s plas­ma do­na­tions are used to pro­duce ther­a­pies that treat bleed­ing dis­or­ders from HAE to Von Wille­brand dis­ease. CSL’s im­munoglob­u­lin and al­bu­min fran­chis­es re­ly heav­i­ly on do­na­tions.

Though the com­pa­ny has tak­en mea­sures to grow its plas­ma col­lec­tion, that comes at a high­er cost. All in all, plas­ma col­lec­tion is up 24% over the pre­vi­ous fis­cal year, as the re­sult of 27 new plas­ma col­lec­tion cen­ters open­ing. The com­pa­ny al­so aims to launch its plasma­phere­sis plat­form in FY23, which promis­es to re­duce the av­er­age do­na­tion time by about 30%.

“I will say Covid has pre­sent­ed chal­lenges for pa­tients. They have had lim­it­ed ac­cess to treat­ment and di­ag­no­sis. There has al­so been an en­vi­ron­ment of con­strained sup­ply,” Per­reault said on the call. “As the glob­al sup­ply re­cov­ers, we are an­tic­i­pat­ing a strong growth re­bound in the Ig port­fo­lio, with op­por­tu­ni­ties in core in­di­ca­tions of pri­ma­ry im­mune de­fi­cien­cy, sec­ondary im­mune de­fi­cien­cy and CIDP.”

As for garadacimab, Per­reault said full re­sults will be pre­sent­ed at an up­com­ing con­fer­ence, and CSL hopes to be­gin fil­ing with glob­al health au­thor­i­ties by the end of the cur­rent fis­cal year.

The CEO is wait­ing un­til Oc­to­ber to re­veal more in­for­ma­tion about the in­te­gra­tion of Vi­for. The com­pa­nies re­ceived reg­u­la­to­ry clear­ance just a cou­ple of weeks ago — months af­ter ini­tial­ly an­nounc­ing the deal.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

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Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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