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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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