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 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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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