Jakob Lindberg, Oncopeptides CEO

Months af­ter pulling on­ly drug from US, On­copep­tides takes back with­draw­al re­quest — but stays mum on why

What hap­pens when you pull a re­quest to pull your drug from the mar­ket?

On­copep­tides is try­ing to find out, re­veal­ing late Fri­day that it has con­tact­ed the FDA and re­scind­ed a let­ter it sent back in Oc­to­ber, re­quest­ing vol­un­tary with­draw­al of the NDA of its mul­ti­ple myelo­ma drug, Pepax­to, in the US — the on­ly place where it’s ap­proved.

The Stock­holm-based biotech trig­gered a ma­jor melt­down with the an­nounce­ment back in Oc­to­ber, as the with­draw­al would force it to close its US and EU busi­ness units and en­act sig­nif­i­cant lay­offs.

No de­tails were giv­en for the sud­den change of mind. From a short, cryp­tic state­ment:

Fur­ther re­view and analy­ses of the het­eroge­nous Over­all Sur­vival da­ta from the phase 3 OCEAN study and oth­er rel­e­vant tri­als have led the Com­pa­ny to re­con­sid­er its pre­vi­ous vol­un­tary with­draw­al re­quest.

At the time, it seemed like On­copep­tides had lit­tle choice; Pepax­to, or mel­pha­lan flufe­namide, first won ac­cel­er­at­ed ap­proval in ear­ly 2021 as a treat­ment for re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma. But then in Ju­ly, in­ves­ti­ga­tors re­port­ed that the drug had failed the over­all sur­vival end­point in a con­fir­ma­to­ry study that pit it against Bris­tol My­ers Squibb’s Po­m­a­lyst — in fact, the risk of death was high­er for pa­tients on the Pepax­to arm.

The FDA quick­ly fol­lowed up with an alert to pa­tients and health­care providers about the safe­ty con­cern, and con­vened an ad­comm to weigh in.

But On­copep­tides stopped that dis­cus­sion from hap­pen­ing by pulling the drug days be­fore the pan­el of out­side ex­perts were set to meet.

Hav­ing stopped the mar­ket­ing of Pepax­to in the US, the com­pa­ny says it has no plans to restart “at this time.” What it will do, though, is ini­ti­ate a di­a­logue with the FDA to re­view the new da­ta.

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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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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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.

Pa­tient re­port finds con­sti­pa­tion con­di­tion not well-man­aged, open­ing door for bet­ter ed­u­ca­tion from phar­ma

Advertising for constipation treatments often uses light-hearted humor in an effort to spur open discussions about the sometimes stigmatized topic. However, that may not be enough to get people to take the condition seriously, a new patient report from Phreesia finds.

Fewer than one-fifth (17%) of patients with constipation surveyed understand the longer-term health risks of constipation such as hemorrhoids and bowel incontinence. Many are trying to manage their condition with over-the-counter medicines, but often for much longer than recommended. An equal 68% say they use home remedies or OTC meds to manage constipation. But while 90% understand that OTCs are not intended for long-term use, 50% have used an OTC constipation medicine for more than a year.

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