The platform Regeneron co-founder George Yancopoulos helped build yielded the first successful Ebola drug. Now, they face an even bigger test in Covid-19. (Regeneron)

With all eyes on their Covid-19 drug, Re­gen­eron wins a land­mark ap­proval in Ebo­la

For the first time, the FDA has ap­proved a ther­a­py for Ebo­la, hand­ing a land­mark OK to Re­gen­eron at the same mo­ment that the New York biotech awaits word from the agency on a sim­i­lar drug they de­vel­oped for Covid-19.

Re­gen­eron de­vel­oped the drug, a cock­tail of 3 neu­tral­iz­ing Ebo­la an­ti­bod­ies now known as In­mazeb, dur­ing the 2014-2016 West African out­break, and when cas­es rose again in the DRC in 2018, the NIH se­lect­ed it as one of four drugs to be test­ed in a large, first-of-its-kind clin­i­cal tri­al. The study was halt­ed 12 months lat­er af­ter two drugs showed over­whelm­ing ef­fi­ca­cy. About a third of Ebo­la pa­tients who re­ceived the Re­gen­eron drug died, com­pared with just over 50% for the an­ti­body ZMapp.

Along­side the ap­proval of Mer­ck’s Ebo­la vac­cine, the drug was a huge phar­ma­ceu­ti­cal achieve­ment and pub­lic health vic­to­ry against a dis­ease that doc­tors have strug­gled to treat or pre­vent since it was first iden­ti­fied in 1976. But it al­so quick­ly proved to be a test run for Re­gen­eron’s next an­tivi­ral pro­gram, as they em­barked bare­ly 5 months lat­er on build­ing a sim­i­lar cock­tail of neu­tral­iz­ing an­ti­bod­ies for what was then known sim­ply as the nov­el coro­n­avirus.

BAR­DA has heav­i­ly sup­port­ed both pro­grams. Re­gen­eron co-founder and CSO George Yan­copou­los high­light­ed the sim­i­lar­i­ties in his state­ment on the ap­proval, echo­ing pre­vi­ous com­ments from An­tho­ny Fau­ci and oth­er sci­en­tists.

“This is the first time the FDA has ap­proved a treat­ment specif­i­cal­ly for Ebo­la, which has caused a num­ber of dead­ly out­breaks,” Yan­copou­los said. “As we ap­ply the same so­phis­ti­cat­ed tech­nolo­gies and man­u­fac­tur­ing ca­pa­bil­i­ties against COVID-19, we hope this will be one of many demon­stra­tions of how the pow­er of sci­ence can be suc­cess­ful­ly de­ployed against dan­ger­ous in­fec­tious dis­eases.”

Re­gen­eron an­nounced their first batch of hu­man da­ta for the Covid-19 ther­a­py late last month and soon there­after found them­selves in the na­tion­al spot­light when White House doc­tors de­cid­ed to give a new­ly-di­ag­nosed Pres­i­dent Trump a high dose of the an­ti­body cock­tail. Trump sub­se­quent­ly promised an EUA for the ther­a­py and a sim­i­lar one in de­vel­op­ment with Eli Lil­ly, tout­ing them as “like a cure” and a “mir­a­cle.” The FDA is still re­view­ing Re­gen­eron’s ap­pli­ca­tion.

There is, of course, no or­di­nary mar­ket for an Ebo­la drug, par­tic­u­lar­ly not in the US. But BAR­DA has al­ready agreed to pur­chase an undis­closed num­ber of dos­es of the drug for $10 mil­lion in 2021 and $67 mil­lion over the fol­low­ing 5 years. The treat­ment has al­so been used in the cur­rent DRC out­break un­der com­pas­sion­ate use.

“Re­gen­eron is ac­tive­ly work­ing with non-gov­ern­men­tal or­ga­ni­za­tions and pub­lic health agen­cies to en­sure con­tin­ued ac­cess to In­mazeb in low- and mid­dle-in­come coun­tries,” the com­pa­ny added in a state­ment.

In ex­change for suc­cess­ful­ly bring­ing an Ebo­la drug to mar­ket, Re­gen­eron will re­ceive a pri­or­i­ty re­view vouch­er, which they can use to ac­cel­er­ate FDA re­view or sell to an­oth­er com­pa­ny. These vouch­ers can fetch up­wards of $50 mil­lion on the open mar­ket; Sanofi once spent $245 mil­lion to ac­quire one.

The oth­er drug that proved ef­fec­tive in the large Ebo­la study, a sin­gle an­ti­body dis­cov­ered with tech­nol­o­gy now owned by the Glax­o­SmithK­line-part­nered Covid-19 de­vel­op­er Vir, is al­so now sit­ting be­fore the FDA. It is owned by Ridge­back Ther­a­peu­tics, a tiny Flori­da-based biotech.

Mapp Bio­phar­ma­ceu­ti­cals, the com­pa­ny that de­vel­oped and pro­duced ZMapp, has dis­con­tin­ued the drug.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

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Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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