Duchenne MD dad piv­ots from last-minute alert on FDA's par­tial hold to $125M IPO for Sol­id Bio

In most IPOs, risk fac­tors can range from any­thing from “our lead drug may not work” to the threat of famine and plague — they’re in­tend­ed to cov­er vir­tu­al­ly every con­tin­gency.

Ilan Gan­ot

But as Ilan Gan­ot was out drum­ming up sup­port for his $125 mil­lion IPO for Sol­id Bio­sciences in re­cent months, there was one key risk fac­tor that hadn’t been in­clud­ed. The FDA had put its lead drug on par­tial clin­i­cal hold in No­vem­ber, with reg­u­la­tors re­fus­ing to al­low re­searchers to start test­ing the high dose.

On­ly the low dose was green-light­ed for the tri­al, the com­pa­ny dis­closed in a tardy amend­ment to the IPO to­day, but the high dose was stymied un­til Sol­id could “sub­mit ad­di­tion­al CMC in­for­ma­tion that demon­strates that man­u­fac­tur­ing ca­pac­i­ty and prod­uct at­trib­ut­es can sup­port the high-dose group.”

And that’s not all.

Ear­li­er this month the biotech not­ed that gene ther­a­py pi­o­neer James Wil­son from Penn had re­signed from their sci­en­tif­ic ad­vi­so­ry board due to ris­ing safe­ty con­cerns re­lat­ed to high dos­ing us­ing the vec­tor he had de­vel­oped.

Re­cent­ly, James M. Wil­son, M.D., Ph.D., re­signed from our Sci­en­tif­ic Ad­vi­so­ry Board cit­ing emerg­ing con­cerns about the pos­si­ble risks of high sys­temic dos­ing of AAV. If in the fu­ture we are un­able to demon­strate that any such ad­verse events were not caused by the ad­min­is­tra­tion process or re­lat­ed pro­ce­dures, the FDA, the Eu­ro­pean Com­mis­sion, the EMA or oth­er reg­u­la­to­ry au­thor­i­ties could or­der us to cease fur­ther de­vel­op­ment of, or de­ny ap­proval of, SGT-001 or our oth­er prod­uct can­di­date for any or all tar­get­ed in­di­ca­tions.

The news, though, didn’t hit un­til Sol­id Bio was wrap­ping up the pric­ing. Sol­id has been plan­ning to sell about 6 mil­lion shares at $16 to $18 a share, with a mar­ket val­u­a­tion of rough­ly $550 mil­lion. Thurs­day evening, Sol­id put out an an­nounce­ment that they had sold 7.8 mil­lion shares at $16 apiece.

Their suc­cess comes amid a wave of fresh hits on the IPO front. Men­lo Ther­a­peu­tics bagged $119 mil­lion in an up­sized of­fer­ing to get the sea­son un­der­way on Thurs­day. And right on the heels of the move by Sol­id Bio, resTOR­bio raised $85 mil­lion and Ar­mo gar­nered $86 mil­lion, bring­ing the to­tal haul from 4 IPOs in two days to $415 mil­lion, which is sure to in­spire plen­ty more new IPOs in Q1.

Gan­ot — a for­mer JP Mor­gan in­vest­ment banker — has made much of the fact that he’s a Duchenne MD dad out to find a gene ther­a­py that could cure the lethal, rare dis­ease. 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. And he had at­tract­ed some heavy­weight back­ers, in­clud­ing RA Cap­i­tal and their col­leagues at Bain.

Now their new risk fac­tor in­cludes the note that the drug may fail if they can’t get the FDA to lift the hold, which they were in­formed of at least two months ago.

I not­ed at the be­gin­ning of Jan­u­ary that the IPO Gan­ot filed al­so failed to lay out ex­act­ly who owned what in the com­pa­ny, an odd omis­sion for some­one shoot­ing to raise that much cash.

The lat­est up­dates in­clude the fact that Gan­ot owns 4.3% of the com­pa­ny with JPMC Strate­gic In­vest­ments in for 9% and Per­cep­tive at 8.7%.

Gan­ot him­self will earn a base salary of $450,000 this year, with a shot at a $200,000 bonus. And his wife is al­so work­ing for the com­pa­ny, with a salary that’s un­der $200,000.

The stock will trade as $SLDB, with every­one look­ing to see where it heads on Fri­day.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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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.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.

Tasly Bio­phar­ma pitch­es long-await­ed IPO — will it trig­ger an­oth­er $1B gold rush on HKEX?

In the run up to the Hong Kong stock ex­change’s an­tic­i­pat­ed rule change — open­ing the door for Chi­nese pre-rev­enue biotechs to go pub­lic clos­er to home — more than a year ago, Tasly Bio­phar­ma was one of the big play­ers whose ru­mored in­ter­est helped stoke en­thu­si­asm for the new list­ing venue. The com­pa­ny has since kept the drum­roll rum­bling in the back­ground, rais­ing a pre-IPO round and con­vinc­ing part­ner Trans­gene to swap own­er­ship in a joint ven­ture for eq­ui­ty. Now the oth­er shoe has fi­nal­ly dropped as ex­ecs out­line plans for a pipeline dom­i­nat­ed by car­dio­vas­cu­lar drugs.