Chi­nese health au­thor­i­ty erects new gold stan­dard in on­col­o­gy R&D, spook­ing in­vestors in boom­ing field

The US has long been the leader in re­search and de­vel­op­ment in on­col­o­gy, but home­grown Chi­nese drug­mak­ers have jumped one hur­dle af­ter an­oth­er in mak­ing a name for them­selves. Now, the Chi­nese gov­ern­ment is erect­ing new hur­dles for its na­tive in­dus­try to leap, and — at least for now — it’s putting a slight chill on the field.

The Chi­nese health au­thor­i­ty NM­PA’s Cen­ter for Drug Eval­u­a­tion re­leased new guid­ance on on­col­o­gy R&D late last week, of­fer­ing a new gold stan­dard for how home­grown drug­mak­ers should de­ter­mine clin­i­cal ef­fi­ca­cy amid a boom­ing field for drug in­no­va­tion, ac­cord­ing to draft doc­u­ments.

The guid­ance broad­ly calls on Chi­nese drug­mak­ers to fo­cus on a pa­tient-cen­tric R&D ap­proach, fold­ing chang­ing stan­dards of care in­to clin­i­cal tri­als and sharp­en­ing re­search around ar­eas of un­met need. The newest roadmap comes amid a wave of Chi­nese in­vest­ment in drug de­vel­op­ment, par­tic­u­lar­ly on­col­o­gy, which has scored some mas­sive val­u­a­tions on the Hong Kong Ex­change and earned big-name part­ners in some of the largest multi­na­tion­al phar­mas.

As biotech in­vestor Brad Lon­car not­ed in a tweet, the draft doc­u­ments are most no­table for their in­sis­tence on us­ing best stan­dard of care as con­trol in late-stage clin­i­cal tri­als, rais­ing the bar high­er for on­col­o­gy drugs to show clin­i­cal ben­e­fit.

“When plan­ning to choose a place­bo or BSC as the con­trol drug, you should en­sure that there is no stan­dard treat­ment for this in­di­ca­tion in clin­i­cal prac­tice; when BSC is avail­able, BSC should be pre­ferred [as] a con­trol, not a place­bo,” ac­cord­ing to a trans­la­tion of the doc­u­ments.

As Lon­car notes, the guid­ance could go a long way to­ward rais­ing the qual­i­ty of Chi­nese on­col­o­gy R&D but could al­so have a sti­fling ef­fect on small­er play­ers look­ing for a mar­ket to ap­proval for their own drugs. In the mean­while, Chi­nese in­vestors ap­pear to have tak­en the guid­ance as a gen­er­al neg­a­tive on the field as a whole.

On Mon­day, ma­jor on­col­o­gy play­ers such as BeiGene (-1.93%), Hutchmed (-5.21%), Zai Lab (-3.49%) and Jun­shi Bio­sciences (-4.79%) were all down, po­ten­tial­ly un­der­scor­ing some con­cern over how the new guid­ance could make late-stage de­vel­op­ment more dif­fi­cult.

Where that ef­fect could be most ap­par­ent is in the PD-1 class, an area where Chi­na has shown a deep pipeline as late-stage en­trants in­to the glob­al and US mar­kets. All four drug­mak­ers list­ed above have at least one PD-1 can­di­date in the pipeline, with BeiGene — which has head­quar­ters in both Bei­jing and Cam­bridge, MA — part­ner­ing with phar­ma gi­ant No­var­tis on a glob­al li­cens­ing deal.

Chi­nese play­ers have not on­ly looked to get the eighth PD-1 across the fin­ish line in the US but are al­so look­ing to take an in­side track on the Chi­nese mar­ket as well. Jun­shi, which is part­nered glob­al­ly with Co­herus on PD-1 tori­pal­imab, re­cent­ly re­leased win­ning Phase III da­ta for its drug in na­sopha­ryn­geal can­cer, a con­di­tion that af­fects South­east Asian pa­tients at a dis­pro­por­tion­ate rate. Da­ta from that piv­otal study could un­der­gird an ap­proval in the US, which would be a first for the FDA and a sign of the grow­ing in­flu­ence of Chi­nese R&D on the glob­al drug mar­ket.

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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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.

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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Renhong Tang, Simcere co-CEO

Almi­rall part­ners up with Sim­cere in po­ten­tial $500M+ deal — with plans to take IL-2 can­di­date glob­al

A Chinese pharma is looking to go international with one of its preclinical candidates, and it’s teaming up with a Spanish company in a new pact potentially worth half a billion dollars to do just that.

Simcere and Almirall announced Thursday that the two companies had reached a deal for Simcere’s IL-2 mutant fusion protein drug candidate, called SIM0278. According to a statement, Almirall gets an exclusive right to develop and commercialize the drug candidate in all indications and markets outside of China, Hong Kong, Taiwan and Macau.

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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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