A year af­ter stun­ning Chi­na OK, Shang­hai Alzheimer's drug­mak­er set to en­roll first US pa­tient — and the stakes are huge

Just as ex­perts are set to de­bate whether Bio­gen’s ad­u­canum­ab should be­come the first Alzheimer’s drug to be ap­proved in the US in al­most two decades, a ther­a­py that seized a con­di­tion­al OK in Chi­na late last year — shock­ing sci­en­tists around the world — is fi­nal­ly mak­ing head­way in the clin­ic.

The US arm of Shang­hai Green Val­ley’s glob­al Phase III tri­al has iden­ti­fied its first pa­tient, Bloomberg re­port­ed, and dos­ing will be­gin in four weeks.

Even the ear­li­est da­ta like­ly won’t be­come avail­able un­til 2026, as Green Val­ley es­ti­mates pri­ma­ry com­ple­tion to be De­cem­ber 2025 on its clin­i­cal­tri­als.gov post­ing. But the re­sults will be high-stakes as an in­di­ca­tion of not just Chi­na’s drug R&D prowess but its reg­u­la­to­ry stan­dards. Did an ob­scure Chi­nese biotech tru­ly de­liv­er an ef­fec­tive treat­ment for a dis­ease that’s de­fied all West­ern so­lu­tions? Or did reg­u­la­tors, in­ten­tion­al­ly or un­in­ten­tion­al­ly, clear a use­less place­bo in ser­vice of boast­ing rights?

Clar­i­ty Clin­i­cal Re­search, an Alzheimer’s fo­cused group based out of New York state, is help­ing screen pa­tients for the study — which is de­signed to re­cruit 2046 par­tic­i­pants across the US, Chi­na and Eu­rope.

Green Val­ley is re­serv­ing $600 mil­lion for the Phase III tri­al, Bloomberg not­ed, as part of a $3 bil­lion in­vest­ment in­to elu­ci­dat­ing and prov­ing the un­der­ly­ing mech­a­nisms of GV-971.

Hav­ing launched the drug last De­cem­ber at $134.52 per box, in Ju­ly the com­pa­ny be­gan con­struc­tion on a $180 mil­lion new man­u­fac­tur­ing fa­cil­i­ty in Shang­hai ded­i­cat­ed to ex­ports and built-in ac­cor­dance with GMP re­quire­ments in Chi­na, the US and Eu­rope. It ex­pects the site to come on­line by the end of 2024.

The ini­ti­a­tion has been a long way com­ing. As soon as it scored con­di­tion­al ap­proval in Chi­na last year, the biotech had said it would launch a glob­al pro­gram in ear­ly 2020. But while the FDA green­light­ed the tri­al in April, amid the Covid-19 pan­dem­ic Green Val­ley was ap­par­ent­ly un­able to meet its stat­ed start date in Oc­to­ber.

Giv­en as oral cap­sules, the drug con­sists of oligo­man­nate, an oligosac­cha­ride de­rived from sea­weeds. While the re­searchers had ini­tial­ly thought it may work by al­ter­ing amy­loid be­ta — the dom­i­nant tar­get that crit­ics say should be dead by now — they ul­ti­mate­ly pro­posed that “GV-971 ther­a­peu­ti­cal­ly har­ness­es the ab­nor­mal pro­duc­tion of amino acids, in­fil­tra­tion of im­mune cells to the brain, and in turn neu­roin­flam­ma­tion via re­mod­el­ling the gut mi­cro­bio­ta.”

Based on its first Phase III, which re­cruit­ed pa­tients in Chi­na on­ly, Green Val­ley claimed that it im­proved cog­ni­tive func­tions in pa­tients with mild-to-mod­er­ate Alzheimer’s.

It had some promi­nent al­lies in its cor­ner. Er­ic Reiman, ex­ec­u­tive di­rec­tor of Ban­ner Alzheimer’s In­sti­tute, is a sci­en­tif­ic ad­vis­er along­side renowned re­searchers Jef­frey Cum­mings and Philip Schel­tens, ap­plaud­ing the safe­ty pro­file of the drug and how it di­ver­si­fies the port­fo­lio. IQVIA and Sig­nant Health lend­ed some se­ri­ous CRO mus­cle to the orig­i­nal late-stage tri­al sup­port­ing the OK.

Oth­er re­ac­tions ranged from cau­tious­ly op­ti­mistic to skep­ti­cal. The fi­nal, sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment on the sole pri­ma­ry end­point of ADAS-Cog12 was hit, in part, be­cause the place­bo group saw a sud­den de­cline at week 24.

None of the sec­ondary end­points were hit — echo­ing a failed Phase II pre­sent­ed in 2014. Some called the re­sults mod­est at best and mean­ing­less at worst.

Howard Fil­lit

“We have sort of in­con­sis­tent re­sults, they’re not the kind of ro­bust re­sults that I think we would want to see here in the Unit­ed States to demon­strate ef­fi­ca­cy,” Howard Fil­lit, the found­ing ex­ec­u­tive di­rec­tor and CSO of the Alzheimer’s Drug Dis­cov­ery Foun­da­tion, said in an in­ter­view with End­points News fol­low­ing the Chi­nese OK. He added: “Maybe the Chi­nese reg­u­la­to­ry agency has a dif­fer­ent kind of risk tol­er­ance and since this is a safe drug, they’re al­low­ing pa­tient ac­cess while more re­search is be­ing done.”

The way Green Val­ley’s new tri­al is de­signed, GV-971 would need to meet two met­rics to be de­clared suc­cess­ful: ADAS-cog/11 (fo­cus­ing on cog­ni­tion) and AD­CS-CG­IC (clin­i­cian’s over­all as­sess­ment of to­tal change).

Dubbed GREEN MEM­O­RY, the study will eval­u­ate a slew of oth­er end­points as well as lev­els of GV-971 in the body, and whether it af­fects brain struc­ture neu­rode­gen­er­a­tion.

So­cial im­age cred­it: Zhangjiang Hi-Tech Park

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

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Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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