FDA ac­cepts pri­or­i­ty re­view for Re­gen­eron's evinacum­ab, which could join Pralu­ent in cho­les­terol mar­ket

Al­most a year ago to the day, Re­gen­eron an­nounced sol­id Phase III test­ing re­sults for evinacum­ab, a com­pound that’s de­signed to low­er LDL cho­les­terol in pa­tients who need treat­ment be­yond a PC­SK9 in­hibitor. As that an­niver­sary ap­proach­es, the biotech is po­ten­tial­ly one step clos­er to putting it on the mar­ket.

The FDA has grant­ed pri­or­i­ty re­view and ac­cept­ed a BLA for evinacum­ab as a sup­ple­ment to oth­er lipid-low­er­ing ther­a­pies in in­di­vid­u­als with ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia, Re­gen­eron an­nounced Wednes­day morn­ing. Re­gen­eron’s tar­get ac­tion date is next Feb­ru­ary 11.

Evinacum­ab is still un­der re­view in Eu­rope, with the EMA hav­ing rec­om­mend­ed an ac­cel­er­at­ed as­sess­ment.

Re­gen­eron al­ready has a PC­SK9 in­hibitor on the mar­ket in Pralu­ent, orig­i­nal­ly ap­proved back in 2015. Pralu­ent and Am­gen’s Repah­ta, the oth­er big PC­SK9 drug on the mar­ket, en­gaged in a spar­ring match with in­sur­ers af­ter low­er-than-ex­pect­ed adop­tion due to stick­er shock. Their prices was slashed to $5,850 in Oc­to­ber 2018 af­ter in­sur­ers won that bat­tle.

HoFH is a rare con­di­tion in which pa­tients have mu­ta­tions in both copies of ei­ther PC­SK9, LDLR or APOB genes. This can in­crease their risk for pre­ma­ture ath­er­o­scle­rot­ic dis­ease and car­diac events as ear­ly as their teenage years.

Those with the dis­ease typ­i­cal­ly do not re­spond well to the stan­dard PC­SK9 in­hibitors and oth­er lipid-low­er­ing treat­ments like statins. The Phase III re­sults from last year showed that in­di­vid­u­als on evinacum­ab showed a 49% re­duc­tion in LDL cho­les­terol from base­line com­pared to just 2% in the place­bo group, which was still tak­ing oth­er med­ica­tions to low­er cho­les­terol.

At the time, an­a­lysts not­ed that evinacum­ab could be a boon for in­di­vid­u­als who tried drugs like Pralu­ent or Repatha and saw lit­tle ef­fect. Cowen an­a­lyst Yaron Wer­ber wrote that peak sales could reach $200 mil­lion to $400 mil­lion in the US with sim­i­lar num­bers in Eu­rope.

Evinacum­ab it­self tar­gets an­giopoi­etin-like pro­tein 3, or ANGPTL3, which in­hibits lipopro­tein li­pase and en­dothe­lial li­pase and plays a key role in lipopro­tein me­tab­o­lism. The com­pound is al­so be­ing stud­ied in pa­tients with re­frac­to­ry hy­per­c­ho­les­terolemia and se­vere hy­per­triglyc­eridemia, both in Phase II.

Should the com­pound be ap­proved, it could fea­si­bly join a long line of Re­gen­eron’s ef­fec­tive an­ti­body treat­ments. Drugs such as Dupix­ent, Kevzara, Lib­tayo and Pralu­ent have all come from the same ge­net­i­cal­ly-en­gi­neered mouse plat­form that the com­pa­ny used to de­vel­op evinacum­ab. Re­gen­eron not­ed, how­ev­er, that the com­pound has yet to be ful­ly eval­u­at­ed for safe­ty and ef­fi­ca­cy by any reg­u­la­to­ry au­thor­i­ty.

Those four drugs pulled in a com­bined $1 bil­lion-plus for the biotech in the sec­ond quar­ter of 2020, though the vast ma­jor­i­ty came from Dupix­ent sales. The eczema drug, which was re­cent­ly ap­proved for chil­dren ages six to 11 with mod­er­ate-to-se­vere atopic der­mati­tis, net­ted the com­pa­ny $945 mil­lion in the quar­ter.

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

Jef­frey Hat­field takes over from Diego Mi­ralles as CEO of Vi­vid­ion; Drag­on­fly scores a new ex­ec with COO Alex Lu­gov­skoy

→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.