Janet Woodcock and Francis Collins

Janet Wood­cock and Fran­cis Collins dish on lessons learned from the pan­dem­ic

With al­most 550,000 Covid-19 deaths in the US, it might seem hard to fath­om any sil­ver lin­ings from such a tragedy. But NIH di­rec­tor Fran­cis Collins and FDA act­ing com­mis­sion­er Janet Wood­cock did their best on Wednes­day to ex­plain what they’ve seen and learned and what the coun­try needs to do to pre­pare for the next pan­dem­ic.

For Wood­cock, who led the ther­a­peu­tics side of Op­er­a­tion Warp Speed be­fore re­turn­ing to lead the FDA, Covid-19 clin­i­cal tri­al in­ef­fi­cien­cies and a dearth of com­mu­ni­ty-based re­search in­fra­struc­ture sit atop her list.

While point­ing to her pub­li­ca­tion last month in Na­ture Re­views Drug Dis­cov­ery, she ex­plained how just 5% of the al­most 3,000 Covid-19 ther­a­peu­tic tri­als were ac­tu­al­ly ran­dom­ized and ad­e­quate­ly pow­ered to re­veal ac­tion­able da­ta.

“There was a tremen­dous amount of du­pli­ca­tion, and a lot of small tri­als that yield­ed tan­ta­liz­ing in­for­ma­tion that sent peo­ple off to use treat­ments that were not use­ful and po­ten­tial­ly harm­ful,” Wood­cock said at the Milken In­sti­tute event. “In a glob­al emer­gency we need to have more cen­tral co­or­di­na­tion be­cause we should re­al­ly ran­dom­ize the first pa­tient.”

On the lack of re­search in­fra­struc­ture, Wood­cock ex­plained how com­mu­ni­ties with­out aca­d­e­m­ic med­ical cen­ters or ad­e­quate­ly trained per­son­nel were stuck with­out any op­tions for en­rolling peo­ple in tri­als. “You can’t just build up in­fra­struc­ture ca­pac­i­ty in the mid­dle of a pan­dem­ic,” she said. “We had tens of thou­sands of peo­ple dy­ing from Covid and we couldn’t get enough pa­tients en­rolled in some tri­als.”

Collins, mean­while, point­ed to three lessons learned. The first is to en­sure NIH and the phar­ma in­dus­try main­tain its pub­lic-pri­vate part­ner­ship, known as AC­TIV, which bet­ter co­or­di­nat­ed and stream­lined bio­med­ical re­search re­sources for test­ing new and re­pur­posed com­pounds to treat Covid-19.

“If we can do it in two weeks, as we did with AC­TIV, we can prob­a­bly do it again and make these kinds of part­ner­ships,” Collins said.

Sec­ond­ly, he point­ed to the dis­con­nect in the clin­i­cal tri­al net­works in the US, not­ing Wood­cock’s com­ments. While he said the du­plica­tive and po­ten­tial­ly un­help­ful tri­als weren’t re­ceiv­ing funds from NIH, they may have been us­ing NIH-fund­ed in­fra­struc­ture.

“We can­not let this clin­i­cal tri­al ecosys­tem fall back in­to this scat­ter­shot ap­proach or we’ll be sor­ry. We need a much more rig­or­ous ap­proach. If we need­ed the ev­i­dence, we now have it,” Collins said.

And third­ly, he point­ed to the need for con­tin­u­ing NIH’s ven­ture cap­i­tal-like strat­e­gy used in its RADx com­pe­ti­tion to speed new Covid-19 tests to mar­ket.

“There’s a lot of tech­nolo­gies out there,” he added, not­ing the Shark Tank ap­proach may be bet­ter suit­ed for many of the ones in de­vel­op­ment, as op­posed to the NIH grant process, which can take more time.

Last­ly, both Wood­cock and Collins point­ed to the need to in­crease the di­ver­si­ty of clin­i­cal tri­als, which Collins not­ed was a heavy lift for some of the Covid-19 vac­cine de­vel­op­ers.

“The way in which bio­med­ical re­search works is con­t­a­m­i­nat­ed by struc­tur­al racism,” Collins said.

Wood­cock echoed that sen­ti­ment, ex­plain­ing how of­ten tri­als are run in dif­fer­ent coun­tries but they don’t nec­es­sar­i­ly re­flect the di­ver­si­ty of the US pop­u­la­tion.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Achiev­ing Dig­i­tal Trans­for­ma­tion: Un­lock­ing Cost Re­duc­tion, Clin­i­cal Ex­cel­lence & Pre­ci­sion Ther­a­peu­tics Man­u­fac­tur­ing

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

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