In an end-of-year IPO blitz, 4 biotechs pitch new of­fer­ings to­tal­ing $370M

An im­muno-on­col­o­gy com­pa­ny fo­cused on a more po­tent T cell at­tack. Two biotechs that grabbed as­sets off the re­main­dered shelves at Big Phar­ma. And one gene ther­a­py up­start look­ing to cure Duchenne mus­cu­lar dy­s­tro­phy, one of the most con­tro­ver­sial fields in bio­phar­ma R&D.

Al­to­geth­er, the group of 4 mount­ed a last minute wave of IPO fil­ings dur­ing the wan­ing hours of 2017. And if they come even close to rais­ing the $370 mil­lion pen­cilled in for their S-1s we’ll have a good idea whether the ven­ture-backed brigade of pri­vate biotechs can con­tin­ue a sol­id run that sparked up the sec­ond half of last year.

Or not.

The phar­ma as­sets were ob­tained for lit­tle or no cash down, ask­ing in­vestors to pay for the re­search that will be need­ed to prove whether they are ac­tu­al­ly new drugs in the mak­ing. Duchenne mus­cu­lar dy­s­tro­phy has been both a mine field and a trea­sure map. And im­muno-on­col­o­gy rep­re­sents one of the most in­tense­ly com­pet­i­tive fields in drug R&D to­day.

So in case you were ask­ing, there are no sure things in biotech. Here’s the break­down.

Ar­mo Bio sets the stage for a piv­otal I/O pitch with $86M IPO

Pe­ter Van Vlas­se­laer

Com­pa­ny: Ar­mo Bio­Sciences
Ini­tial IPO tar­get: $86 mil­lion
CEO + stake: Pe­ter Van Vlas­se­laer (8.1%)
Found­ed: 2012
Based: Red­wood City, CA

Ar­mo Bio­Sciences’ $67 mil­lion mez­za­nine round at the end of last Au­gust set the stage for this $86 mil­lion IPO.

Cel­gene and Google’s GV came back along­side Klein­er Perkins and Or­biMed as well as a broad­er syn­di­cate in­clud­ing Chi­na’s Qim­ing Ven­ture Part­ners for the C round back­ing a new tech­nol­o­gy aimed at whip­ping up a more po­tent CD8-pos­i­tive T cell at­tack on can­cer cells — ground ze­ro in the cur­rent on­slaught of can­cer R&D pro­grams.

Ar­mo is fol­low­ing what has be­come a well trav­eled path­way in can­cer drug de­vel­op­ment, piv­ot­ing from ear­ly stage to late-stage stud­ies. And it’s sell­ing the po­ten­tial of a lead ther­a­py that has gar­nered some pos­i­tive re­sults in a hand­ful of pa­tients. The key pitch in the S-1:

The com­bi­na­tion with AM0010 and FOL­FOX showed in PDAC (pan­cre­at­ic duc­tal ade­no­car­ci­no­ma) pa­tients with a me­di­an num­ber of two pri­or ther­a­pies, a mPFS of 2.6 months. The mOS for the com­bi­na­tion of AM0010 and FOL­FOX is 10.2 months with a me­di­an fol­low-up time of 20.3 months with a range be­tween 15.8 and 25.9 months as of Oc­to­ber 2017. At that time, the one year sur­vival rate was 42.9%. These re­sults are of par­tic­u­lar in­ter­est com­pared to a mOS of 4.3 months, mPFS of 1.7 months and one-year sur­vival of 18.5% re­port­ed in a study of FOL­FOX in the sec­ond-line set­ting.

The first in­ter­im analy­sis of the Phase III is due in “ear­ly 2018,” with a 2020 read­out on the sec­ond in­ter­im analy­sis that could trig­ger a mar­ket­ing ap­pli­ca­tion. There’s al­so a pro­gram for non-small cell lung can­cer.

Klein­er Perkins con­trols about 20% of the shares now, trailed by Or­biMed at 19% and DAG Ven­tures at 11%. GV is in for 5.6% of the eq­ui­ty. The stock will be list­ed un­der the sym­bol $AR­MO.


An­oth­er drug off of No­var­tis’ back shelf trig­gers a quick­ie $85M IPO from PureTech

Chen Schor

Com­pa­ny: resTOR­bio
Ini­tial IPO tar­get: $85 mil­lion
CEO + stake: Chen Schor (8.8%)
Found­ed: 2017
Based: Cam­bridge, MA

Nine months af­ter PureTech ramped up a new com­pa­ny called resTOR­bio and then picked up a pair of drugs from No­var­tis’ out­li­cens­ing shelf, the biotech has giv­en birth to an $85 mil­lion IPO pitch.

ResTOR­bio’s for­tunes will rest on mTORC1 in­hibitors with a po­ten­tial to treat a va­ri­ety of ag­ing-re­lat­ed ail­ments, start­ed with res­pi­ra­to­ry tract in­fec­tions for the el­der­ly. A Phase IIb study cov­er­ing the south­ern and north­ern hemi­spheres is ex­pect­ed to read out in the sec­ond half of this year.

No­var­tis let go of resTOR­bio’s lead drug — RTB101 — for a batch of stock for NI­BR ac­count­ing for 9.4% of the shares, with no mon­ey down. A suc­cess­ful de­vel­op­ment pro­gram could trig­ger up to $24 mil­lion in mile­stones with roy­al­ties due on any sales, and the biotech has so far front­ed a to­ken $300,000 for their progress to date.

PureTech, which is run by Daphne Zo­har, owns 44.4% of the com­pa­ny with an­oth­er 20% held by Or­biMed.

PureTech ex­ec and resTOR­bio’s CEO Chen Schor holds 8.8% of the stock, which will be list­ed as $TORC.


Can Men­lo’s $1M drug deal with Mer­ck in­spire a $98M IPO to pay for Phase III?

Steven Bas­ta

Com­pa­ny: Men­lo Ther­a­peu­tics
Ini­tial IPO tar­get: $98 mil­lion
CEO + stake: Steven Bas­ta (3.7%)
Found­ed: 2011 (as Tiger­cat)
Based: Red­wood City, CA

Can a low-pro­file biotech with a sin­gle as­set picked up for $1 mil­lion up­front paid to Mer­ck pull off a $98 mil­lion IPO in ear­ly 2018?

Red­wood City, CA-based Men­lo Ther­a­peu­tics in­tends to find out.

The biotech, which had $75 mil­lion in cash in the bank at the end of Sep­tem­ber, has com­plet­ed a suc­cess­ful Phase II for pru­ri­tus. The group that found­ed the com­pa­ny picked up ser­lop­i­tant back in 2012 for what amounts to lunch mon­ey in this busi­ness, bag­ging rights to a drug (MK0594) that Mer­ck had put through a cou­ple of tri­als for al­co­hol de­pen­dence (ter­mi­nat­ed) and over­ac­tive blad­der.

The drug is an NK-1 re­cep­tor an­tag­o­nist, which the biotech’s ex­ecs be­lieve is a key me­di­a­tor to the urge to scratch or cough. Tamp that down and Men­lo be­lieves its drug can help peo­ple with a va­ri­ety of ail­ments tied to pru­ri­tus as well as chron­ic cough­ing. And once it goes in­to Phase III, Men­lo says it will owe Mer­ck an­oth­er $3 mil­lion along with up to $25 mil­lion in de­vel­op­ment and reg­u­la­to­ry mile­stones with an­oth­er $50 mil­lion in goal cash built on top of that — pro­vid­ed they can steer it to the mar­ket.

The S-1 pitch is es­sen­tial­ly based on da­ta from two Phase II stud­ies that were re­port­ed out last Sep­tem­ber. In­ves­ti­ga­tors re­port­ed a bor­der­line suc­cess (p<0.05) on the re­duc­tion from base­line VAS pru­ri­tus score and a change from base­line in av­er­age-itch VAS score at 8 weeks (p<0.001).

Vi­vo Cap­i­tal is the big share­hold­er, with 25% of the eq­ui­ty. Remedi­tex Ven­tures weighs in at 18% and Pre­sidio Part­ners con­trols 16.5% of the stock.

Bag­ging dis­cards off Big Phar­ma’s back shelves — or their re­jects — has be­come a pop­u­lar, though not nec­es­sar­i­ly suc­cess­ful, strat­e­gy. Vivek Ra­maswamy is build­ing a mul­ti­fac­eted en­ter­prise us­ing that ap­proach, though his first for­ay out with Ax­o­vant ran in­to a brick wall of fail­ure.

Men­lo plans to trade as $MN­LO.


Can a gene ther­a­py up­start fo­cused on a con­tro­ver­sial tar­get like Duchenne MD win over in­vestors to the tune of $100M?

Ilan Gan­ot

Com­pa­ny: Sol­id Bio­Sciences
Ini­tial IPO tar­get: $100 mil­lion
CEO + stake: Ilan Gan­ot (N/A)
Found­ed: 2013
Based: Cam­bridge, MA

It was clear at the end of Q1 last year that Sol­id’s $50 mil­lion round was aim­ing at a crossover to the pub­lic mar­kets, and the biotech did not dis­ap­point, post­ing its $100 mil­lion IPO on the last busi­ness day of the year.

Found­ed by Duchenne dad Ilan Gan­ot, the com­pa­ny has gained the sup­port of RA Cap­i­tal and its close con­fed­er­ates at Bain for a gene ther­a­py aimed at noth­ing less than a cure of DMD. By in­tro­duc­ing a syn­thet­ic dy­s­trophin trans­gene con­struct, called mi­crody­s­trophin, via a vi­ral vec­tor, the com­pa­ny hopes to prove it can do what Sarep­ta and oth­ers have been grop­ing for with one de­ci­sive in­ter­ven­tion.

The fact that not every­one in the field — in­clud­ing some at the FDA — is con­vinced that dy­s­trophin is the right bio­mark­er for ad­dress­ing this dis­ease, there’s ob­vi­ous­ly a long way to go in prov­ing it works.

The S-1 lists the in­vestors, but doesn’t spell out what per­cent­ages of the eq­ui­ty they own — a miss­ing fea­ture which will need to be patched in.

The biotech plans to list as $SLDB.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.