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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.

Tae Han Kim, Samsung Biologics CEO (SeongJoon Cho/Bloomberg via Getty Images)

Sam­sung Bi­o­log­ics nets $330M+ deal from As­traZeneca ahead of 'Su­per Plan­t' con­struc­tion

Just a few weeks after announcing plans to construct a $2 billion “Super Plant,” Samsung Biologics is keeping its foot on the gas.

The Korean CDMO has inked a $330.8 million manufacturing contract with AstraZeneca, the companies announced Monday evening, providing the British drugmaker the ability to expand production capabilities in the Asia market. Per terms of the deal, the partnership could be increased to $545.6 million.

Francesco De Rubertis

Medicx­i's David Grainger and Francesco De Ru­ber­tis pump €200M in­to six com­pa­nies and what they say is a first-of-its kind fund

In what they’re billing as a first for biotech, David Grainger, Francesco De Rubertis and their team at Medicxi have put down a €200 million to sweep up stakes in six companies from their predecessor VC and pump new money into them.

Medicxi didn’t disclose which companies it was investing in but the portfolio draws from Index Ventures Life VI, one of the last funds the Medicxi team launched while they were still part of the multinational, tech-focused VC firm Index Ventures. That team kept advising Index on their life sciences portfolio even after they spun out to form their own firm in the middle of 2016.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Scripps reach­es $10M set­tle­ment with gov­ern­ment over al­le­ga­tions NIH grants weren't prop­er­ly ac­count­ed for

Scripps Research Institute has settled a case with the Justice Department alleging claims of misappropriated funds, the US attorney for the district of Maryland announced late last week.

Prosecutors said the institute improperly used NIH-funded research grants for non-grant related activities, including working on new grant applications, teaching activities and other administrative tasks. As part of the settlement, Scripps has agreed to pay $10 million.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.