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.

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Can an FDA hus­tle up on trastuzum­ab be far be­hind?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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