Nas­daq wraps up Q3 with IPO pitch­es from Gami­da Cell, NGM, Arog to­talling $219M

Nas­daq’s bub­bling biotech week did not just end with Gri­s­tone’s top-of-the-range raise, which brought the IPO tal­ly to $520 mil­lion. Late Fri­day, three more biotechs lined up to en­ter the club, seek­ing a col­lec­tive $219 mil­lion to fu­el mid- and late-stage en­deav­ors on can­cer and NASH.

As we en­ter Q4, it re­mains to be seen whether 2018 — at 49 com­plet­ed deals — will sur­pass the 66 bio­phar­ma IPO record set in 2014. But it is with­in range.

While the year has seen a num­ber of pre­clin­i­cal biotechs reap big wind­fall on their pub­lic de­but, Gami­da Cell, NGM Bio and Arog Phar­ma all have some hu­man da­ta — and for two of them, big name back­ers or part­ners — to show for their pitch­es.

Ju­lian Adams

Biotech vet­er­an Ju­lian Adams made his IPO am­bi­tions clear on his very first day as CEO of Gami­da Cell. For the Jerusalem-based com­pa­ny to go all the way through Phase III, build out man­u­fac­tur­ing and ex­e­cute a planned mar­ket launch of its uni­ver­sal cell graft, he told End­points News last No­vem­ber, they need­ed much more than the sup­port al­ready pro­vid­ed by No­var­tis, Shav­it Cap­i­tal, VMS In­vest­ment Group, the Is­rael Biotech Fund, Clal Biotech­nol­o­gy In­dus­tries and Is­rael Health­Care Ven­tures.

NiCord, their lead prod­uct com­pris­ing a mix of stem cells, prog­en­i­tor cells as well as den­drit­ic cells, is at the very be­gin­ning of a piv­otal Phase III tri­al for pa­tients with blood can­cers. The clin­i­cal team is al­so in­ves­ti­gat­ing the drug’s ef­fec­tive­ness in treat­ing bone mar­row fail­ure dis­or­ders, as an al­ter­na­tive for pa­tients who ex­pe­ri­ence graft re­jec­tion with con­ven­tion­al trans­plant op­tions.

To wrap all that, and for the build­out of their man­u­fac­tur­ing in Kiry­at Gat, Is­rael, Gami­da Cell is seek­ing $69 mil­lion. They plan to list as $GM­DA.

David Wood­house

NGM Bio, mean­while, is ask­ing for $75 mil­lion on the promise of its NASH ther­a­py, which comes with a slate of Phase II da­ta re­port­ed in April 2017, where in­ves­ti­ga­tors re­port­ed a hit for the pri­ma­ry end­point as well as some im­prove­ments in bio­mark­ers.

“NG­M282 is the first agent I’ve test­ed that holds the po­ten­tial to com­plete­ly re­verse steato­sis in as short as 12 weeks of ther­apy,” an Ox­ford re­searcher said at the time.

That tri­al is still on­go­ing, and a Phase IIb is ex­pect­ed to start in ear­ly 2019 un­der the lead­er­ship of new CEO David Wood­house — a for­mer Gold­man Sachs banker who joined the com­pa­ny as CFO in 2015. He’s re­plac­ing William Rieflin, who’s been bumped up to the ex­ec­u­tive chair­man seat. Pres­i­dent Jeff Jonker, mean­while, is on his way out to a new ex­ec­u­tive po­si­tion. We don’t know where.

While ad­vanc­ing its in-house drug, NGM Bio — go­ing by $NGM — has al­so been work­ing close­ly with Mer­ck, which struck a $450 mil­lion re­search deal in 2015 for ac­cess to its growth dif­fer­en­ti­a­tion fac­tor 15 pro­gram. Col­lab­o­ra­tion rev­enue reg­is­tered at $40.7 mil­lion for the first half of 2018.

Scott Sal­ka

Dal­las-based Arog Phar­ma is the youngest of the three, found­ed in 2010 by on­col­o­gist Vinay Jain af­ter he li­censed the lead drug, crenolanib, from Pfiz­er for a cheap up­front li­cense fee “in the low sev­en fig­ures” with reg­u­la­to­ry and com­mer­cial­iza­tion mile­stones to­tal­ing $12.5 mil­lion.

In their fil­ing, the com­pa­ny — now led by ex-Am­bit chief Scott Sal­ka — ex­plains why it is so ex­cit­ed about the drug, which has re­ceived fast track des­ig­na­tion at the FDA as a ther­a­py for acute myeloid leukemia:

The clin­i­cal de­vel­op­ment of crenolanib at Pfiz­er had fo­cused on the in­hi­bi­tion of PDGFR in sol­id tu­mors and had demon­strat­ed fa­vor­able safe­ty and tol­er­a­bil­i­ty in over 100 pa­tients in two clin­i­cal tri­als. Af­ter ob­tain­ing rights to crenolanib, we dis­cov­ered its po­tent ac­tiv­i­ty against FLT3, a now val­i­dat­ed tar­get; such ac­tiv­i­ty against FLT3 was pre­vi­ous­ly un­known. In ad­di­tion, we dis­cov­ered that op­ti­miz­ing the dos­ing sched­ule to three times dai­ly al­lows for greater sus­tained tar­get in­hi­bi­tion, a crit­i­cal at­tribute of ef­fec­tive ty­ro­sine ki­nase in­hibitor, or TKI, ther­a­pies.

If the IPO goes ac­cord­ing to plan, Arog will get a $75 mil­lion in­fu­sion of cash to take this and a sec­ond li­censed pro­gram for­ward un­der the sym­bol $AROG.

Oric CEO Ja­cob Chacko and Dean Fer­rig­no, a for­mer ex­ec at Sor­ren­to, are among the in­sid­ers cur­rent­ly fund­ing the com­pa­ny, though their stakes are not spelled out in the fil­ing.

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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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.

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.

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.

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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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.