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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

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