In­dus­try lead­ers need to speak out now more than ever

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

The House im­peach­ment man­agers pre­sent­ed a grip­ping, vis­cer­al case two weeks ago in pros­e­cut­ing the 45th pres­i­dent for in­cite­ment of mob vi­o­lence to stop the cer­ti­fi­ca­tion of a free and fair US elec­tion.

Yet nev­er has a case so damn­ing pre­saged an ac­quit­tal so fore­gone. Sev­en brave sen­a­tors crossed the aisle to do the right thing and vote for a re­pub­lic, if we can keep it.

Just be­cause the im­peach­ment tri­al is over does not mean the threat to our de­mo­c­ra­t­ic norms is. The Sen­ate ac­quit­tal comes with a long-term cost: the hy­per­vig­i­lance of the Amer­i­can peo­ple. It is up to all of us to de­ter­mine what the for­mer pres­i­dent’s con­duct means for our na­tion’s fu­ture.

In biotech­nol­o­gy, our day job is to dis­cov­er and de­vel­op in­no­v­a­tive, ground­break­ing ther­a­pies and see that they get de­liv­ered fair­ly to the world’s pa­tient pop­u­la­tions. My com­pa­ny, Nkar­ta Ther­a­peu­tics, is com­mit­ted to re­al­iz­ing the po­ten­tial of the body’s nat­ur­al killer cells to treat can­cer. As CEOs, in­ten­sive fo­cus on our day jobs does not ex­cuse us from the role that our boards and col­leagues re­ly on us to play, which is to lead. Lead­er­ship, at its core, means call­ing out bad be­hav­ior and stand­ing up for what is right.

In the 2020 elec­tion, the Amer­i­can peo­ple vot­ed for a new ad­min­is­tra­tion com­mit­ted to sci­en­tif­ic in­tegri­ty, so­cial jus­tice, racial rec­on­cil­i­a­tion and eco­nom­ic re­new­al. How­ev­er, I be­lieve that the last four years un­leashed malev­o­lent forces in our so­ci­ety; how quick­ly, and if, Pan­do­ra’s box can be closed is an open ques­tion.

Emerg­ing biotech com­pa­nies re­cruit top sci­en­tists from every race, cul­ture and back­ground. For me, build­ing an in­clu­sive cul­ture that tru­ly cel­e­brates our dif­fer­ences means that si­lence is not an op­tion. I once had an in­vestor call me and fret that he had Googled my com­pa­ny name and saw as many ar­ti­cles on the first screen about so­cial jus­tice as he did about break­through sci­ence. I asked him if he had any doubts that I am do­ing every­thing in my pow­er to suc­ceed sci­en­tif­i­cal­ly and fi­nan­cial­ly. “None what­so­ev­er,” he said.

“That makes me hap­py to hear,” I replied. “Those ar­ti­cles you saw — and the com­pa­ny val­ues they rep­re­sent — are a big rea­son why the team is work­ing so hard on the sci­ence and feels as in­vest­ed in this com­pa­ny as you do.”

His­tor­i­cal­ly, many bio­phar­ma­ceu­ti­cal CEOs have been care­ful to watch our words so we do not alien­ate the par­ty that be­lieves in the free mar­kets up­on which our suc­cess ul­ti­mate­ly de­pends. How­ev­er, I have be­come in­creas­ing­ly out­spo­ken in my be­lief that biotech CEOs need to be more out­spo­ken in their be­liefs.

In to­day’s po­lit­i­cal en­vi­ron­ment, I be­lieve it is more im­por­tant than ever that we show the par­ty in pow­er our hearts, our true val­ues and our com­mit­ment to pa­tients. For me, that starts with speak­ing out against a new breed of cult-of-per­son­al­i­ty pop­ulism that dis­dains sci­en­tif­ic knowl­edge, traf­fics in racism and vi­o­lent ri­ots and un­der­mines democ­ra­cy.

Amer­i­can lead­ers across gov­ern­ment, in­dus­try, me­dia, ed­u­ca­tion and be­yond have a spe­cial re­spon­si­bil­i­ty to use our voic­es to de­fend shared val­ues that should tran­scend par­ti­san af­fil­i­a­tion. The free­doms too many Amer­i­cans take for grant­ed can be in­stant­ly lost, and tra­di­tion­al al­lies of con­ser­v­a­tives like the bio­phar­ma­ceu­ti­cal in­dus­try are unique­ly sit­u­at­ed to de­mar­cate un­cross­able lines.

Why take the risk? Be­cause si­lence is a greater risk. Our in­dus­try de­pends on de­mo­c­ra­t­ic sta­bil­i­ty and the mar­ket con­fi­dence it in­spires. Sta­ble mar­kets are why biotech en­tre­pre­neurs can raise the enor­mous cap­i­tal re­quired to bring long­shot break­throughs to pa­tients. For this rea­son and many more, we can­not turn our heads at threats to the foun­da­tion of the U.S. po­lit­i­cal and eco­nom­ic sys­tem.

That’s why the bio­phar­ma­ceu­ti­cal in­dus­try de­cid­ed to re­con­sid­er po­lit­i­cal con­tri­bu­tions to mem­bers of Con­gress who sup­port­ed over­turn­ing the re­sults of a con­sti­tu­tion­al­ly prop­er and ex­haus­tive­ly ad­ju­di­cat­ed elec­tion. As the vice chair of the Biotech­nol­o­gy In­no­va­tion Or­ga­ni­za­tion (BIO), the largest biotech in­dus­try group, I was heart­ened that so many of my col­leagues on both sides of the aisle agreed that this was the right course of ac­tion.

But the best way to com­bat dis­crim­i­na­tion and in­val­i­date white su­prema­cy is to em­brace racial di­ver­si­ty and to reck­on with past so­ci­etal trans­gres­sions. In the health­care sys­tem, that means ac­knowl­edg­ing lega­cies of mis­treat­ment and mis­trust that have made mi­nori­ties dis­pro­por­tion­ate­ly skep­ti­cal about vac­cines and clin­i­cal tri­al par­tic­i­pa­tion.

Pfiz­er and Mod­er­na en­rolled an im­pres­sive 42% and 37% mi­nori­ties, re­spec­tive­ly, in their Phase III COVID-19 vac­cine clin­i­cal tri­als. Me­dia and pa­tient ad­vo­cates want to know if this is a flash in the pan or an on­go­ing com­mit­ment. CEOs must con­tin­ue to speak out and act af­fir­ma­tive­ly if we are to main­tain cred­i­bil­i­ty and car­ry that mo­men­tum over to di­ver­si­fy tri­als in oth­er dis­ease ar­eas. Pro­grams like BIO Equal­i­ty rep­re­sent a long-term, in­dus­try­wide ef­fort to fight for di­ver­si­ty, ac­cess and af­ford­abil­i­ty for all pa­tients. Nkar­ta is proud to be a BIO mem­ber.

My ap­proach to drug de­vel­op­ment is in­formed by my own ex­pe­ri­ence liv­ing with Crohn’s dis­ease and run­ning a pa­tient ad­vo­ca­cy group for chil­dren with bow­el and blad­der con­di­tions. I can speak the heresy that drug costs are, in fact, too high, be­cause I’m talk­ing about low­er­ing pa­tient out-of-pock­et costs, not slash­ing in­sur­ance and gov­ern­ment re­im­burse­ment rates that have en­abled us to in­no­vate life-sav­ing vac­cines and an­ti­bod­ies against COVID-19 with record speed.

I can agree with HHS Sec­re­tary De­signee Xavier Be­cer­ra that health care is a fun­da­men­tal right but strong­ly dis­agree that in­val­i­dat­ing patents is a smart strat­e­gy to help pa­tients ac­cess med­i­cine. I can be­lieve in the so­cial con­tract that al­lows new med­i­cines — whether small mol­e­cules, pro­teins, an­ti­bod­ies or cell ther­a­pies — to be priced for a re­turn on in­vest­ment ini­tial­ly so long as gener­ics en­ter the mar­ket with­out de­lay, un­nec­es­sary ne­go­ti­a­tion or com­pli­ca­tion once patents ex­pire.

I can agree that Rep. Katie Porter (D-CA) is a pa­tri­ot but dis­agree with her state­ment that Big Phar­ma com­pa­nies buy­ing up small­er ones some­how de­val­ues them. Ac­tu­al­ly, this dy­nam­ic churn frees sci­en­tists to start new com­pa­nies and in­no­vate anew, while large com­pa­nies — with their glob­al sales teams and armies of com­mer­cial pro­fes­sion­als and drug de­vel­op­ers — sell, mar­ket and dis­trib­ute new med­i­cines.

Fight­ing for de­mo­c­ra­t­ic ideals doesn’t mean fi­deli­ty to politi­cians from any par­tic­u­lar par­ty. For me, in these tu­mul­tuous times, it is about hav­ing a moral com­pass, do­ing what’s right and speak­ing up for pa­tients and sci­en­tists who rep­re­sent the glo­ri­ous di­ver­si­ty and great hope of our plan­et.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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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.”

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Why re­mote drug man­u­fac­tur­ing eval­u­a­tions won't re­al­ly re­duce FDA's back­log of in­spec­tions

For the first several months of the pandemic last spring, the FDA continued to plow through its user fee-enabled work on new drug and biologic applications, meeting nearly all of its goal dates.

But by last fall and into the winter, complete response letters and other delays began arriving in companies’ mailboxes as the agency struggled to catch up to a growing backlog of both domestic and foreign drug manufacturing inspections.

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