In mul­ti­ple myelo­ma show­down, J&J adds an­oth­er feath­er to Darza­lex's cap

Sanofi’s Sar­clisa burst on­to the mul­ti­ple myelo­ma scene last year to chal­lenge J&J’s CD38 heavy­weight Darza­lex. But J&J isn’t go­ing down with­out a fight, and now it’s tout­ing an­oth­er ap­proval for the hard-to-treat blood can­cer — along with an eas­i­er route of ad­min­is­tra­tion that could save pa­tients hours of their time.

The FDA has giv­en Darza­lex Faspro the thumbs-up as a sec­ond-line treat­ment for adults with mul­ti­ple myelo­ma in com­bi­na­tion with po­ma­lido­mide and dex­am­etha­sone (Pd), Janssen said Mon­day. The ap­proval marks the sixth in­di­ca­tion for Darza­lex Faspro, which is co-for­mu­lat­ed with Halozyme’s re­com­bi­nant hu­man hyaluronidase PH20, a type of en­zyme that cat­alyzes the degra­da­tion of hyaluron­ic acid.

Ac­cord­ing to J&J, the drug is now the first sub­cu­ta­neous CD38 an­ti­body ap­proved in com­bi­na­tion with Pd — shav­ing hours off of the time it would take to de­liv­er it via IV.

“With this ap­proval, we are now able to com­bine po­ma­lido­mide and dex­am­etha­sone with a dara­tu­mum­ab sub­cu­ta­neous op­tion that can be ad­min­is­tered in min­utes rather than the hours need­ed for in­tra­venous ad­min­is­tra­tion,” prin­ci­pal in­ves­ti­ga­tor Mele­tios Di­mopou­los said in a state­ment.

The ap­proval was based on the APOL­LO study, which showed that Darza­lex Faspro and Pd re­duced pa­tients’ risk of dis­ease pro­gres­sion or death by 37% com­pared to Pd alone (p=0.0018). Mean pro­gres­sion-free sur­vival in the treat­ment arm was 12.4 months, com­pared to 6.9 months in the con­trol arm, J&J said. And 25% of pa­tients on Darza­lex Faspro achieved com­plete re­spons­es, com­pared to just 4% in the Pd arm, ac­cord­ing to the phar­ma.

The treat­ment does come with some risks, as half of pa­tients ex­pe­ri­enced a se­ri­ous ad­verse event, some of the most com­mon be­ing pneu­mo­nia and res­pi­ra­to­ry tract in­fec­tion. Fa­tal side ef­fects oc­curred in 7% of pa­tients.

J&J inked a $1.1 bil­lion agree­ment with Gen­mab to de­vel­op, man­u­fac­ture and com­mer­cial­ize Darza­lex back in 2012. It shelled out $55 mil­lion up­front, with tiered “dou­ble dig­it” roy­al­ties. Last year, the block­buster raked in about $4.19 bil­lion.

Sar­clisa was ap­proved in March 2020 to treat mul­ti­ple myelo­ma in com­bi­na­tion with po­ma­lido­mide and dex­am­etha­sone. And ear­li­er this year, it was OK’d for mul­ti­ple myelo­ma pa­tients who’ve re­ceived one to three lines of ther­a­py in com­bi­na­tion with carfil­zomib and dex­am­etha­sone. Re­searchers say the cock­tail re­duced the risk of dis­ease pro­gres­sion or death by 45% com­pared to stan­dard of care.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

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.

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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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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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.

CMS spent more than $18B in four years on ac­cel­er­at­ed ap­provals with in­com­plete con­fir­ma­to­ry tri­als, in­spec­tor gen­er­al finds

The battle over whether and how to reform the FDA’s accelerated approval pathway is heating up again, just as the Senate punted any talks until the lame duck session just before the end of the year.

On Thursday, HHS’ inspector general released a new report reiterating concerns, also noted recently by the FDA’s Oncology Center of Excellence, about delayed or slowed confirmatory trials that are necessary to prove that the accelerated approvals were worth their salt in the first place.

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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.

Paul Hudson, Sanofi CEO (Photographer: Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi, Re­gen­eron’s Dupix­ent scores an­oth­er in­di­ca­tion with first-ever ap­proval for nodu­lar skin dis­or­der

Sanofi chief executive Paul Hudson told investors earlier this year that the Big Pharma was going to emphasize its sales kingpin Dupixent moving forward.

He wasn’t joking — the megablockbuster drug and sales king, recording just shy of $2 billion in sales this past quarter, has now officially secured its fifth indication from the FDA.

Sanofi and Regeneron, who jointly work on Dupixent development and commercialization, announced the new development on Thursday, saying that the FDA gave the all-clear to Dupixent to treat patients with prurigo nodularis, a rare autoimmune disorder characterized by a persistent, severe itch — and also visualized by hard, extremely itchy bumps known as nodules that form on the skin. The FDA noted in its announcement that it is the agency’s first approval for the disease.

Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA (Sipa via AP Images)

Sen­ate pass­es bill to re­work an­i­mal test­ing re­quire­ments for drug de­vel­op­ers

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would alter a federal mandate for animal testing on new drugs, but stops short of removing animal testing entirely.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.