Kad­mon bags a quick win for its graft-ver­sus-host dis­ease drug ahead of Au­gust PDU­FA date

About a year and a half af­ter Kad­mon trum­pet­ed “knock-your-socks-off” re­sults for its chron­ic graft-ver­sus-host dis­ease (GVHD) treat­ment, the FDA has fol­lowed through with a speedy ap­proval six weeks be­fore the agency was sup­posed to an­nounce a de­ci­sion.

The FDA has ap­proved Rezurock (belu­mo­sudil) as a third-line treat­ment in adult and pe­di­atric pa­tients 12 years and old­er, based on piv­otal re­sults from the ROCK­star tri­al show­ing a 75% over­all re­sponse rate and a 69% par­tial re­sponse rate.

Like the acute form of the dis­ease, chron­ic GVHD can oc­cur af­ter am stem cell trans­plant when im­mune cells from the donor at­tack the re­cip­i­ent, caus­ing in­flam­ma­tion and fi­bro­sis in tis­sues like the skin, eyes, joints, liv­er and lungs. The con­di­tion af­fects about 14,000 pa­tients in the US, ac­cord­ing to Kad­mon. Rezurock is de­signed to tamp down that in­flam­ma­to­ry re­sponse by block­ing Rho-as­so­ci­at­ed coiled-coil ki­nase 2 (ROCK2). It’s now the first ap­proved ther­a­py tar­get­ing that pro­tein.

Kad­mon’s stock $KDMN jumped on the news, up 20.56% late Fri­day af­ter­noon.

Of the 65 pa­tients in the ROCK­star study giv­en 200mg of Rezurock twice dai­ly, just un­der half of them had four or more or­gans af­fect­ed by the dis­ease.

“We be­lieve that this is the most ad­vanced, most se­vere pop­u­la­tion ever stud­ied in this dis­ease,” San­jay Ag­gar­w­al, SVP of clin­i­cal de­vel­op­ment, said back in May 2020.

Six per­cent of pa­tients achieved a com­plete re­sponse, Kad­mon said, and 62% of re­spon­ders didn’t re­quire new sys­temic ther­a­py for at least 12 months. The me­di­an time to first re­sponse was 1.8 months.

“Pa­tients re­ceiv­ing REZUROCK re­port­ed sig­nif­i­cant im­prove­ments in cGVHD symp­toms, show­ing that not on­ly did treat­ment re­sult in or­gan re­spons­es, but it al­so made peo­ple feel bet­ter,” said Stephanie Lee, re­search di­rec­tor of the long-term fol­low-up pro­gram at the Fred Hutchin­son Can­cer Re­search Cen­ter.

Up­on re­leas­ing the first glimpse at piv­otal da­ta back in 2019, Kad­mon framed their drug as one of a grow­ing num­ber of op­tions pa­tients might use, not­ing that many of the peo­ple in this tri­al had didn’t re­spond to In­cyte’s Jakafi or Ab­b­Vie and J&J’s Im­bru­vi­ca.

The com­pa­ny was found­ed by Sam Wak­sal, the en­tre­pre­neur who was con­vict­ed of in­sid­er trad­ing in 2003. He found­ed the com­pa­ny af­ter his re­lease, then hand­ed the reins to his broth­er Har­lan be­fore its pub­lic de­but as his sen­tence barred him from head­ing a pub­lic com­pa­ny.

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

BREAK­ING: 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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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.

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.

Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

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.