Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Ex­change con­fer­ence with biotech and phar­ma in­dus­try in­sid­ers since be­fore the pan­dem­ic be­gan, it was no sur­prise many con­ver­sa­tions in­clud­ed Covid top­ics. Yet while vac­cines and treat­ments were dis­cussed, so too were the ef­fects on drug de­vel­op­ment, fed­er­al re­spons­es, health in­equities — and what to do now and next.

George Yan­copou­los

George Yan­copou­los, chief sci­en­tist and co­founder of Re­gen­eron, opened the con­fer­ence re­spond­ing to a ques­tion from Acor­da CEO Ron Co­hen about the spot­light on the in­dus­try dur­ing Covid and some of the “flak” bio­phar­ma has tak­en in the past.

“I hope so­ci­ety rec­og­nizes that the im­pact that dis­ease can have, mea­sured as it was in the pan­dem­ic in the tril­lions, in terms of eco­nom­ics – and not even able to cal­cu­late the loss of life and suf­fer­ing as­so­ci­at­ed,” Yan­copou­los said. “I hope that makes so­ci­ety re­al­ize that per­haps shouldn’t we be in­vest­ing more than $30 mil­lion, for ex­am­ple, for NIH fund­ing, but al­so in­vest­ing a lot more in this great in­dus­try to pro­tect our­selves against these cat­a­stroph­ic loss­es. I think we’re not do­ing enough.”

He at­trib­uted Re­gen­eron’s speed in de­vel­op­ing its ear­ly mon­o­clon­al an­ti­body treat­ment RE­GEN-COV — along with work by Pfiz­er, Mod­er­na, As­traZeneca and Eli Lil­ly — build­ing on the com­pa­nies’ decades of sci­en­tif­ic de­vel­op­ment and in­vest­ing. With much more need­ed, he point­ed out.

“We have to rec­og­nize none of the ex­ist­ing so­lu­tions for dis­ease and none of the ex­ist­ing so­lu­tions for cli­mate change will save us. We need new so­lu­tions,” he said, which will on­ly come from sup­port­ing next gen­er­a­tions of tal­ent and with in­vest­ments “at much larg­er scales than we are to­day.”

Rick Bright

For­mer BAR­DA chief Rick Bright al­so talked about the need to in­vest and im­prove not on­ly the cur­rent Covid re­sponse as it be­comes en­dem­ic, but al­so the im­por­tance of trust and truth in those ef­forts. Bright head­ed BAR­DA un­der Pres­i­dent Barack Oba­ma, join­ing in 2016, but was re­moved and re­as­signed to a low­er lev­el NIH job by the Trump ad­min­is­tra­tion in April 2020. Bright sub­se­quent­ly filed a whistle­blow­er com­plaint and tes­ti­fied be­fore Con­gress about the gov­ern­ment’s chaot­ic re­sponse to the pan­dem­ic.

“We hear a lot about the ero­sion of trust and to be trust­wor­thy we have to be hon­est,” he said. “… From the out­set of this pan­dem­ic, we didn’t have a lot of truth, hon­est­ly. So we need to make sure we’re not on­ly get­ting the truth out, but make sure we’re trans­lat­ing the truth to some­thing peo­ple can un­der­stand. When they see the de­vel­op­ment of a vac­cine or mon­o­clon­al an­ti­body ther­a­peu­tic, as George said they don’t re­al­ize that it’s been a decade worth of work. That was skipped over for a po­lit­i­cal rhetoric to say, ‘Hey, I made a vac­cine in a short pe­ri­od of time.’”

Tal Zaks

Al­so tak­ing the stage on a pan­el about per­son­al­ized med­i­cine was Tal Zaks, the for­mer chief med­ical of­fi­cer at Mod­er­na and Spike­vax Covid vac­cine de­vel­op­er, who is now a part­ner at Or­biMed.

While the ideas of per­son­al­ized med­i­cines and mass mar­ket vac­cines may seem in­con­gru­ent, Zaks not­ed that “all med­i­cine has al­ways been per­son­al­ized. We go to the doc­tor for treat­ment for our­selves, not our neigh­bor.”

In Covid-19 vac­cines and treat­ments, for ex­am­ple, per­son­al­iza­tion comes in­to play for im­muno­com­pro­mised peo­ple and some can­cer pa­tients who don’t re­spond as well and need spe­cif­ic, dif­fer­ent ther­a­pies. While costs, ben­e­fits and val­ue are fa­mil­iar top­ics in per­son­al­ized med­i­cine dis­cus­sions, Zaks said the po­ten­tial size of the pa­tient pool for per­son­al­ized med­i­cines is less im­por­tant to him than what they can de­liv­er.

“Rather than look at the rise of per­son­al­ized med­i­cine as some­thing that phar­ma went to be­cause they couldn’t make mon­ey else­where, I take a dif­fer­ent view. For me, the rea­son we’re talk­ing about per­son­al­ized med­i­cine is be­cause that’s what sci­ence has un­cov­ered,” he said. “… The world of sci­ence and the world of tech­nol­o­gy is open­ing up op­por­tu­ni­ties for us to un­der­stand pop­u­la­tions bet­ter — even in preva­lent dis­eases. If you talk to phar­ma to­day, they’re ac­tu­al­ly not shy­ing away from car­dio­vas­cu­lar dis­ease or neu­ro­log­i­cal dis­ease or di­a­betes. What they’re bring­ing to the fore is a much more nu­anced way of un­der­stand­ing those pop­u­la­tions and an in­di­vid­ual’s risk fac­tor.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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BioCryst's new website for its HAE unbranded campaign encourages patients to take charge of treatment decisions.

BioCryst launch­es aware­ness cam­paign around man­age­ment of rare vas­cu­lar dis­ease

While hereditary angioedema (HAE) is rare, treatment options for the condition have become much more common. So BioCryst Pharmaceuticals is taking a new angle in its recently launched HAE awareness campaign encouraging patients to take control of their disease management.

“Hereditary angioedema (HAE) isn’t the author of your story — you are … #cHAEngetheplan. Not the goal,” the US campaign website advises.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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EMA makes new rec­om­men­da­tions for Im­bru­vi­ca, med­i­cines with ter­li­pressin over res­pi­ra­to­ry and car­dio­vas­cu­lar risks

The EMA is handing out a new recommendation surrounding J&J and AbbVie’s cancer drug Imbruvica and some medicines containing terlipressin.

On Friday, the EMA’s safety committee (PRAC) stated that it has taken new measures to reduce the risk of respiratory failure and sepsis when medicines containing terlipressin that are used to treat people with hepatorenal syndrome (HRS-1), particularly for patients with advanced acute-on-chronic liver disease or advanced kidney failure.