In­smed fol­lows a new path­way at the FDA for an­tibac­te­ri­als, win­ning quick OK for Arikayce

The FDA grant­ed ac­cel­er­at­ed ap­proval for In­smed’s drug Arikayce for the treat­ment of a lung dis­ease caused by the bac­te­ria group My­cobac­teri­um avi­um com­plex, or MAC, in cer­tain pa­tients who have not re­spond­ed to con­ven­tion­al treat­ment. And reg­u­la­tors used the mo­ment to flag a new path­way opened at the FDA that is in­tend­ed to speed up the ar­rival of a new gen­er­a­tion of bad­ly need­ed an­tibac­te­ri­als.

Arikayce is the first drug to be ap­proved un­der the new Lim­it­ed Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, or LPAD, which al­lows for more stream­lined clin­i­cal tri­als in­volv­ing small­er pa­tient pop­u­la­tions.

Scott Got­tlieb

“As bac­te­ria con­tin­ue to grow im­per­vi­ous to cur­rent­ly avail­able an­tibi­otics, we need to en­cour­age the de­vel­op­ment of drugs that can treat re­sis­tant in­fec­tions. That means uti­liz­ing nov­el tools in­tend­ed to stream­line de­vel­op­ment and en­cour­age in­vest­ment in­to these im­por­tant en­deav­ors,” said FDA com­mis­sion­er Scott Got­tlieb in a state­ment is­sued Fri­day.

“This ap­proval is the first time a drug is be­ing ap­proved un­der the Lim­it­ed Pop­u­la­tion Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, and it marks an im­por­tant pol­i­cy mile­stone. This path­way, ad­vanced by Con­gress, aims to spur de­vel­op­ment of drugs tar­get­ing in­fec­tions that lack ef­fec­tive ther­a­pies. We’re see­ing a lot of ear­ly in­ter­est among spon­sors in us­ing this new path­way, and it’s our hope that it’ll spur more de­vel­op­ment and ap­proval of an­tibac­te­r­i­al drugs for treat­ing se­ri­ous or life-threat­en­ing in­fec­tions in lim­it­ed pop­u­la­tions of pa­tients with un­met med­ical needs,” Got­tlieb added.

Arikayce was ap­proved un­der the agency’s ac­cel­er­at­ed ap­proval path­way and has re­ceived or­phan drug sta­tus. The agency said ap­proval was grant­ed based on clin­i­cal da­ta that showed 29% of pa­tients on a mul­ti-drug an­tibac­te­r­i­al reg­i­men who took Arikayce for six months showed no growth of the bac­te­ria in their spu­tum for at least three con­sec­u­tive months com­pared with on­ly 9% of pa­tients who did not add Arikayce to their reg­i­mens.

Pos­i­tive da­ta from that on­go­ing Phase III study sent shares soar­ing in ear­ly Sep­tem­ber on hopes that it would lead to a swift FDA ap­proval.

As a con­di­tion of Arikayce’s ac­cel­er­at­ed ap­proval, In­smed will be re­quired to run an ad­di­tion­al clin­i­cal tri­al to re­ceive full ap­proval. The com­pa­ny said Fri­day that it is col­lab­o­rat­ing with the FDA on the de­sign of the tri­al, which will like­ly be a ran­dom­ized, dou­ble-blind, place­bo-con­trolled study eval­u­at­ing the ben­e­fit of Arikayce in pa­tients with Non­tu­ber­cu­lous My­cobac­te­r­i­al lung dis­ease caused by MAC.

Arikayce, al­so known as amikacin li­po­some in­hala­tion sus­pen­sion, is de­liv­ered through a neb­u­liz­er. The prod­uct will car­ry a black box warn­ing ad­vis­ing it may trig­ger bron­chospasms, hy­per­sen­si­tiv­i­ty pneu­moni­tis, the ex­ac­er­ba­tion of lung dis­ease, and spit­ting up of blood. Oth­er side ef­fects in­clude dif­fi­cul­ty speak­ing, cough, dam­aged hear­ing, up­per air­way ir­ri­ta­tion, mus­cu­loskele­tal pain, fa­tigue, di­ar­rhea and nau­sea, ac­cord­ing to the FDA.

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.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

UP­DAT­ED: 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 will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

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. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’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. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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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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Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

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.

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.

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 — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

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.