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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.