As Hutchmed awaits FDA ap­proval for su­r­u­fa­tinib, drug gets the go-ahead in Chi­na; SCO­TUS knocks back last Amarin patent bid

Chi­nese reg­u­la­tors on Fri­day signed off on the sec­ond ap­proval for Hutchmed’s on­col­o­gy drug su­r­u­fa­tinib af­ter a suc­cess­ful Phase III tri­al.

Known com­mer­cial­ly as Su­lan­da in Chi­na, the drug re­cent­ly ap­proved for ad­vanced pan­cre­at­ic neu­roen­docrine tu­mors al­so won ap­proval in Chi­na 7 months ago for the treat­ment of non-pan­cre­at­ic neu­roen­docrine tu­mors.

Hutchmed CEO Chris­t­ian Hogg said in a state­ment, “Since its launch in Jan­u­ary this year, pa­tients with ep­NETs have ben­e­fit­ed from treat­ment with su­r­u­fa­tinib through its unique mode of ac­tion by both in­hibit­ing an­gio­gen­e­sis and pro­mot­ing the body’s im­mune re­sponse against tu­mor cells.”

Hutchmed re­cent­ly filed for a $603 mil­lion IPO in Hong Kong, which will serve as a home­com­ing for the com­pa­ny as it has been list­ed on the Nas­daq since 2016. The com­pa­ny is cur­rent­ly await­ing FDA re­view of su­r­u­fa­tinib, which was filed in May. Hutchmed was the first Chi­nese com­pa­ny to bring an in-house on­col­o­gy drug to mar­ket in that coun­try with fruquin­tinib, which was ap­proved as a third-line ther­a­py for metasta­t­ic CRC. — Josh Sul­li­van

SCO­TUS de­nies Amarin’s last bid to save patent

The Supreme Court has shot down Amarin’s last bid to win back its patents on what would have been a block­buster heart drug.

The court de­nied the Irish drug­mak­er’s re­quest to re­view an ap­peal’s court de­ci­sion that found key patents over its fish oil-based drug were in­valid. That rul­ing, com­ing near­ly a year af­ter the com­pa­ny showed their omega-3 tablets could pre­vent heart at­tacks and strokes, had turned what could have been an in­stant block­buster in­to a mod­est com­mer­cial prod­uct that, in the US, would have to com­pete with gener­ic com­peti­tors.

The ap­peals court rul­ing up­held a low­er court de­ci­sion from March 2020 that found that, al­though ri­val drug­mak­ers would in­fringe on sev­er­al claims in Amarin’s patents by mar­ket­ing gener­ics, those claims were “ob­vi­ous” and in­valid. — Ja­son Mast

Chi­na gives third OK to BeiGene’s BTK in­hibitor

BeiGene is stack­ing up the Chi­nese ap­provals for Brukin­sa.

The Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion has cleared the BTK in­hibitor for pa­tients with Walden­ström’s macroglob­u­line­mia who have re­ceived at least one pri­or ther­a­py, now the third B-cell ma­lig­nan­cy it’s ap­proved to treat.

The biotech tout­ed car­dio­vas­cu­lar safe­ty ad­van­tages on top of “deep and durable re­spons­es.” Even though it failed to beat J&J and Ab­b­Vie’s Im­bru­vi­ca on com­plete re­sponse rates in the head-to-head AS­PEN tri­al in­volv­ing 229 pa­tients, reg­u­la­tors de­cid­ed last Oc­to­ber that the low­er rate of side ef­fects (plus oth­er signs of ef­fi­ca­cy) were enough to war­rant a pri­or­i­ty re­view.

“Brukin­sa was specif­i­cal­ly de­signed by BeiGene sci­en­tists to lim­it off-tar­get ef­fects seen with first-gen­er­a­tion BTK in­hibitors and we have built a broad clin­i­cal de­vel­op­ment pro­gram to as­sess clin­i­cal ben­e­fit, in­clud­ing the head-to-head AS­PEN tri­al,” Jane Huang, BeiGene’s CMO in hema­tol­ogy, said in a state­ment.

Out­side of Chi­na, the FDA has ap­proved Brukin­sa for man­tle cell lym­phoma, while BeiGene is look­ing to carve out mar­ket share from Im­bru­vi­ca on oth­er fronts like re­lapsed/re­frac­to­ry CLL or small lym­pho­cyt­ic lym­phoma. — Am­ber Tong

Rafael Phar­ma­ceu­ti­cals gears for merg­er

A late-stage on­col­o­gy play­er is look­ing to back­flip on­to the New York Stock Ex­change, as Rafael Phar­ma­ceu­ti­cals makes plans to merge with Rafael Hold­ings $RFL.

The com­pa­nies shared the news of their im­pend­ing merg­er on Mon­day morn­ing. Rafael’s lead can­di­date de­vimi­s­tat, de­signed to tar­get can­cer meta­bol­ic path­ways, is cur­rent­ly in Phase III tri­als for metasta­t­ic pan­cre­at­ic can­cer and re­lapsed or re­frac­to­ry acute myeloid leukemia.

CEO Ameet Mallik, for­mer ex­ec­u­tive VP and head of No­var­tis’ US on­col­o­gy busi­ness, is ex­pect­ed to steer the com­pa­ny to Phase III read­outs in metasta­t­ic pan­cre­at­ic can­cer at the end of this year, ac­cord­ing to a state­ment. The drug has re­ceived fast-track des­ig­na­tion for that in­di­ca­tion, as well as AML.

“I am tru­ly ex­cit­ed to lead the Com­pa­ny at this piv­otal time as we ad­vance de­vimi­s­tat, a pipeline in a mol­e­cule, through two Phase 3 reg­is­tra­tional clin­i­cal tri­als and sev­er­al Phase 1 and Phase 2 clin­i­cal tri­al pro­grams in ad­di­tion­al in­di­ca­tions,” he said. — Nicole De­Feud­is

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.

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.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

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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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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