Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Al­though Thurs­day’s Sen­ate health com­mit­tee hear­ing was fo­cused on how for­eign coun­tries and ad­ver­saries might be try­ing to steal or neg­a­tive­ly in­flu­ence bio­med­ical re­search in the US, the on­ly coun­try men­tioned by the sen­a­tors and ex­pert wit­ness­es was Chi­na.

Com­mit­tee chair Pat­ty Mur­ray (D-WA) made clear in her open­ing re­marks that the US can­not “let the few in­stances of bad ac­tors” over­shad­ow the hard work of the many im­mi­grant re­searchers in the US, many of which have won No­bel prizes for their work. But she al­so said, “There is more the NIH can be do­ing here.”

The hear­ing fol­lows a se­ries of high pro­file con­vic­tions of sci­en­tists for steal­ing in­tel­lec­tu­al prop­er­ty from top bio­med­ical re­search in­sti­tu­tions.

Just this week, Yu Zhou of Ohio was sen­tenced to 33 months in prison af­ter he plead­ed guilty to steal­ing sci­en­tif­ic trade se­crets re­lat­ed to ex­o­somes and ex­o­some iso­la­tion from Na­tion­wide Chil­dren’s Hos­pi­tal’s Re­search In­sti­tute, which he sought to sell in Chi­na.

Song Guo Zheng, A pro­fes­sor of in­ter­nal med­i­cine and re­searcher at The Ohio State Uni­ver­si­ty and Penn­syl­va­nia State Uni­ver­si­ty, al­so plead­ed guilty in late 2020 to mak­ing false state­ments to fed­er­al au­thor­i­ties as part of an im­munol­o­gy re­search grant fraud scheme. He ad­mit­ted to ly­ing on ap­pli­ca­tions in or­der to use more than $4 mil­lion in NIH grants to de­vel­op Chi­na’s ex­per­tise around rheuma­tol­ogy and im­munol­o­gy, ac­cord­ing to writ­ten tes­ti­mo­ny Thurs­day from Gary Cantrell, deputy in­spec­tor gen­er­al at HHS’ Of­fice of In­spec­tor Gen­er­al.

“The threat is sig­nif­i­cant,” Michael Lauer, deputy di­rec­tor for ex­tra­mur­al re­search at NIH, told the sen­a­tors. He said NIH has iden­ti­fied more than 500 sci­en­tists of con­cern, and reached out to the in­sti­tu­tions where over 200 of them work, al­though each one re­quires a tremen­dous amount of work.

But Lauer al­so not­ed in­stances where re­search in­sti­tu­tions are dis­cov­er­ing prob­lems on their own. For in­stance, Alan List, pres­i­dent and CEO of the Mof­fitt Can­cer Cen­ter in Flori­da, re­signed in late 2019 af­ter the cen­ter con­duct­ed an in­ter­nal re­view on col­lab­o­ra­tions with re­search in­sti­tu­tions in Chi­na.

Can­dice Wright, act­ing di­rec­tor of sci­ence, tech­nol­o­gy as­sess­ment, and an­a­lyt­ics at GAO, al­so ex­plained how non-fi­nan­cial con­flicts, such as ac­cess to cer­tain re­search labs or bi­o­log­ic ma­te­ri­als, can pose risks too. “Non-fi­nan­cial con­flicts can be great risks and we don’t see a lot of at­ten­tion paid to those,” she said.

But progress has been made in re­cent years to be more vig­i­lant of po­ten­tial crimes, even as crit­ics have raised ques­tions about whether this crack­down has un­fair­ly tar­get­ed Chi­nese re­searchers. Last year, two rep­re­sen­ta­tives, Jamie Raskin (D-MD) and Judie Chu (D-CA) wrote let­ters to the FBI and NIH, rais­ing con­cerns that in­no­cent Chi­nese sci­en­tists were be­ing pro­filed, and asked for in­for­ma­tion on the de­mo­graph­ic make­up of sci­en­tists un­der in­ves­ti­ga­tion.

Sen. Richard Burr (R-NC) con­ced­ed that the NIH has come a long way since 2018, when NIH Di­rec­tor Fran­cis Collins sent a let­ter to more than 10,000 re­search in­sti­tu­tions, urg­ing them to en­sure NIH grantees were re­port­ing their links with for­eign gov­ern­ments. But he and oth­er sen­a­tors specif­i­cal­ly took is­sue with Chi­na.

“It’s a con­cert­ed ef­fort from those in Chi­na, backed by their gov­ern­ment, to bring back any­thing they can learn, store or steal,” Burr said. Oth­er Re­pub­li­cans on the com­mit­tee like Sen. Bill Cas­sidy (LA) ques­tioned whether 23andMe’s op­er­a­tions in Chi­na might be wor­ri­some, but none of the wit­ness­es on the pan­el of­fered any specifics.

As an­ti-Asian sen­ti­ment has reached a fever pitch in re­cent months in the US, Lauer al­so sought to make clear that the over­whelm­ing ma­jor­i­ty of Chi­nese-born sci­en­tists work­ing in the US are not bad ac­tors. He said NIH has iden­ti­fied crim­i­nals who are Amer­i­cans too.

“We can­not re­ject bril­liant minds that are work­ing hon­est­ly,” Lauer added. “Le­git­i­mate in­ter­na­tion­al col­lab­o­ra­tions are great, and this is ex­treme­ly im­por­tant, but that’s dif­fer­ent from ly­ing, cheat­ing and steal­ing.”

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).

BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: In­ter­nal Trump-era emails re­veal a pletho­ra of celebs, com­pa­nies vy­ing for FDA’s at­ten­tion

The FDA on Thursday publicly released a trove of about 500 pages of internal and heavily redacted emails, showing how celebrities like Dr. Oz and Laura Ingraham vied for the FDA’s attention on Covid-related issues that ultimately proved to be a waste of time.

Many of the conversations, obtained via the Freedom of Information Act, involve major Trump-era health officials who, in retrospect, made crucial and often ill-advised decisions on increasing access to the over-hyped hydroxychloroquine.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.