Ther­a­peu­tic­sMD flags “de­fi­cien­cies” in NDA; CRUK, NCI col­lab­o­rate on RAS in­hibitor re­search

→ Bo­ca Ra­ton, FL-based Ther­a­peu­tic­sMD $TXMD is flag­ging some se­ri­ous prob­lems for their new drug ap­pli­ca­tion at the FDA. In a state­ment to­day the com­pa­ny said reg­u­la­tors had iden­ti­fied “de­fi­cien­cies” in their ap­pli­ca­tion, though it’s not ex­act­ly sure what those de­fi­cien­cies are. They’re seek­ing a mar­ket­ing ap­proval for TX-004HR, “an in­ves­ti­ga­tion­al bio-iden­ti­cal 17β-estra­di­ol vagi­nal soft­gel cap­sule for the treat­ment of mod­er­ate-to-se­vere vagi­nal pain dur­ing sex­u­al in­ter­course (dys­pare­u­nia), a symp­tom of vul­var vagi­nal at­ro­phy (VVA) in post­menopausal women.” The biotech says it plans to find out what the de­fi­cien­cies are and re­solve them AS­AP.

→ Can­cer Re­search UK and the Can­cer Re­search Tech­nol­o­gy Re­search Fund are chip­ping in £2.5 mil­lion to fund a col­lab­o­ra­tion with the Na­tion­al Can­cer In­sti­tute to test ex­per­i­men­tal RAS in­hibitors, one of the tough­est tar­gets in can­cer re­search. RAS dri­ves some par­tic­u­lar­ly lethal can­cers, like pan­cre­at­ic and lung can­cer.

Em­maus Life Sci­ences says the On­co­log­ic Drug Ad­vi­so­ry Com­mit­tee will re­view its NDA for the oral L-glu­t­a­mine prod­uct En­dari, for the treat­ment of sick­le cell dis­ease.

→ San Car­los, CA-based Li­on Biotech­nolo­gies $LBIO is team­ing with the MD An­der­son Can­cer Cen­ter on a mul­ti-year pact in­volv­ing clin­i­cal tri­als to eval­u­ate TIL ther­a­py in ovar­i­an can­cer, var­i­ous sar­co­mas, and pan­cre­at­ic can­cer. In ad­di­tion, pre­clin­i­cal re­search will ex­plore the ex­pan­sion of TIL in oth­er rare tu­mor types.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene requires the divestiture of the blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay. That’s delaying the completion of the buyout, which the pharma giant says now may not be done before early 2020, rather than Q3 as promised. And the news quickly blew a hole in its stock price as investors reacted to the surprise switch up in plans.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. But Bristol’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. And Bristol-Myers is sticking with its experimental drug, discounting Otezla’s future.

That’s not what the analysts were expecting.

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Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

Mike Grey. Mirum

In $86M IPO pitch, Mirum spells out plans to turn Shire dis­cards in­to or­phan liv­er drug suc­cess­es

Mike Grey doesn’t have any time to waste. Hav­ing re­gained con­trol of two liv­er dis­ease drugs from Shire and po­si­tioned them for piv­otal stud­ies — five years af­ter first hand­ing them off in a deal to sell Lu­me­na, where he was CEO — Grey is steer­ing Mirum straight in­to an IPO with a $86 mil­lion ask.

Not that Mirum has spent much of its $120 mil­lion Se­ries A cash since launch­ing last No­vem­ber. Ac­cord­ing to the S-1, the Cal­i­forn­ian biotech has burned through $23.3 mil­lion as of March, but ex­pects ex­pens­es to pick up once their clin­i­cal work gath­ers steam.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Evotec CEO Werner Lanthaler, File Photo

Ox­ford, Evotec ramp up LAB10x with AI ex­perts at Sen­syne — fo­cused on biotech spin­outs

Ox­ford is al­ly­ing it­self with Evotec and ar­ti­fi­cial in­tel­li­gence out­fit Sen­syne Health to ramp up some new biotech spin­outs while look­ing to “ac­cel­er­ate da­ta-dri­ven drug dis­cov­ery and de­vel­op­ment.”

The big idea here is that Ox­ford sci­en­tists — some of the best drug hunters in the world — can uti­lize Sen­syne’s AI plat­form for their work, re­ly­ing on the chemists and hands-on de­vel­op­ers at Evotec to push ahead to a crit­i­cal proof of con­cept mo­ment. And they’ll do it through a project leader called LAB10x, which gets £5 mil­lion over the next three years to fund the work.