FDA slaps a hold on Sol­id Bio's gene ther­a­py for Duchenne MD in wake of safe­ty alert

Just a few weeks af­ter Sol­id Bio­sciences $SLDB put out an 11th hour no­tice that its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy had been put on a par­tial clin­i­cal hold due to safe­ty con­cerns, just ahead of its $125 mil­lion IPO, the biotech is back Wednes­day evening with the news that the agency has now halt­ed the study in the wake of an ad­verse re­ac­tion to the ther­a­py by the first pa­tient in the tri­al.

The stock plunged 54% af­ter the news hit, sig­ni­fy­ing that this time around in­vestors aren’t quite so san­guine about safe­ty is­sues. But the pain didn’t end there. JP­Mor­gan, which helped on the IPO, turned bear­ish on the news and cau­tioned on the com­pa­ny’s prospects in the wake of a safe­ty is­sue that raised se­ri­ous doubts about Sol­id’s ex­ec­u­tive team. By mid-af­ter­noon on Thurs­day, the stock was down 63%, shear­ing off more than $600 mil­lion in val­ue.

“In our view, it will like­ly take com­pelling clin­i­cal da­ta to re­gain con­fi­dence in man­age­ment (not ex­pect­ed in the near-term),” said JP Mor­gan’s Anu­pam Ra­ma, ac­cord­ing to a Bloomberg re­port.  Ra­ma set the new price at $9, a sick­en­ing drop from the $16 price they launched at.

Sol­id Bio CEO Ilan Gan­ot

This is from Sol­id’s state­ment:

The first pa­tient dosed in the clin­i­cal tri­al was a non-am­bu­la­to­ry ado­les­cent who re­ceived 5E13 vg/kg of SGT-001 on Feb­ru­ary 14, 2018. Sev­er­al days af­ter ad­min­is­tra­tion the pa­tient was hos­pi­tal­ized due to lab­o­ra­to­ry find­ings that in­clud­ed a de­crease in platelet count fol­lowed by a re­duc­tion in red blood cell count and ev­i­dence of com­ple­ment ac­ti­va­tion. The pa­tient showed no signs or symp­toms of co­ag­u­lopa­thy (bleed­ing dis­or­der) and no rel­e­vant changes from base­line in liv­er func­tion tests. The pa­tient re­spond­ed well to med­ical treat­ment and is cur­rent­ly asymp­to­matic. All lab­o­ra­to­ry pa­ra­me­ters have ei­ther im­proved or re­turned to nor­mal, and he is con­tin­u­ing out­pa­tient as­sess­ments per pro­to­col.

The FDA is like­ly on high alert on Sol­id af­ter James Wil­son, a pi­o­neer in gene ther­a­py who de­vel­oped the AAV de­liv­ery tech that Sol­id is us­ing, re­signed from the ad­vi­so­ry board af­ter an an­i­mal study raised a red flag about po­ten­tial safe­ty is­sues when used at a high dose. Wil­son was alarmed af­ter a mon­key was killed by liv­er tox­i­c­i­ty af­ter be­ing ex­posed to a high dose AAV treat­ment, with ev­i­dence of mo­tor neu­ron dam­age in piglets. A num­ber of com­pa­nies use AAV tech in-li­censed for their work. But none of the oth­ers has been hit with a hold.

Sol­id CEO Ilan 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.

Some an­a­lysts sup­port­ing Sarep­ta $SRPT in the race to de­vel­op a gene ther­a­py for DMD are like­ly to glee­ful­ly sug­gest that Sol­id’s trou­bles are Sarep­ta’s boon. Sarep­ta has dosed a cou­ple of pa­tients now, look­ing for a one-time ther­a­py to help save boys from a dev­as­tat­ing ill­ness.

Gan­ot’s de­ci­sion to keep the par­tial hold out of the news while it was do­ing a road show on the IPO rais­es ques­tions about the lev­el of trans­paren­cy – or lack it – that al­lows pri­vate drug de­vel­op­ers to op­er­ate in se­cre­cy. Aside from no­ti­fy­ing re­searchers, reg­u­la­tors and pa­tients, a pri­vate de­vel­op­er is not re­quired to air safe­ty is­sues. The FDA cer­tain­ly won’t say any­thing. And we saw the ex­act same thing play out re­cent­ly when Unum filed their IPO, re­veal­ing two pa­tient deaths months ear­li­er and a clin­i­cal hold on the high dose of their lead cell ther­a­py, which they sub­se­quent­ly dumped.

The Cam­bridge, MA-based biotech says it’s work­ing with the FDA on get­ting the hold lift­ed.

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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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three ma­jor buy­outs an­nounced: Take­da/Shire; Bris­tol-My­ers/Cel­gene and now Ab­b­Vie/Al­ler­gan. And with this lat­est deal it’s in­creas­ing­ly clear that the sharp fall from grace suf­fered by high-pro­file play­ers which have seen their share prices blast­ed has cre­at­ed an open­ing for the growth play­ers in big phar­ma to up their game — in sharp con­trast to the pop­u­lar bolt-on deals that have been dri­ving the growth strat­e­gy at No­var­tis, Mer­ck, Roche and oth­ers.

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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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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.

SQZ, Ery­tech kick off $57M cell ther­a­py part­ner­ship; Jean-Paul Kress lands new CEO gig at Mor­phoSys

→ In a mar­riage of two tech­nolo­gies meant to make cell ther­a­pies more pow­er­ful, SQZ Biotech is team­ing up with France’s Ery­tech Phar­ma for a col­lab­o­ra­tion, with $57 mil­lion re­served for the first project and $50 mil­lion for each sub­se­quent ap­proval (prod­uct or in­di­ca­tion). Hav­ing ac­cess to Ery­tech’s method of fash­ion­ing ther­a­peu­tics from red blood cells, the Cam­bridge, MA-based com­pa­ny said, will am­pli­fy SQZ’s cell en­gi­neer­ing ca­pa­bil­i­ties and al­low them to de­vleop a new class of im­munomod­u­la­to­ry ther­a­pies. Its own tech — so far ap­plied in can­cer but al­so has po­ten­tial in di­a­betes — tem­po­rary dis­rupts the cell mem­brane by squeez­ing the cell, thus cre­at­ing a brief win­dow for tar­get ma­te­ri­als such as anti­gens to en­ter.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

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.

With 4 more biotech IPOs due to wrap up Q2, how is the class of 2019 far­ing?

With 22 biotech IPOs on the books and four more set to price in the last week of June, in­vest­ment ad­vis­er Re­nais­sance Cap­i­tal has tak­en the pulse of the re­cent rush.

By the IPO ex­perts’ count, 25 out of 32 health­care of­fer­ings this year have been from biotechs — dif­fer­ing slight­ly from Brad Lon­car’s tal­ly — and the over­all pic­ture is one of un­der­per­for­mance. While they av­er­aged a first-day re­turn of 9.0%, col­lec­tive­ly they have trad­ed down to a 5.9% re­turn. Turn­ing Point $TP­TX and Cor­texyme $CRTX emerged on top at the half-year mark, ris­ing 135% and 109% re­spec­tive­ly.

Eye­ing a $500M peak sales pot, Almi­rall dou­bles down on le­brik­izum­ab as Der­mi­ra lines up PhI­II

With eyes on what it be­lieves is a $500 mil­lion peak rev­enue op­por­tu­ni­ty in Eu­rope, Barcelona-based Almi­rall has stepped up with $50 mil­lion in cash to take up the op­tion on Der­mi­ra’s IL-13 an­ti-in­flam­ma­to­ry drug le­brik­izum­ab just ahead of the start of Phase III. And there’s an­oth­er $30 mil­lion due as the late-stage pro­gram gets geared up.

That shouldn’t be long from now, as Der­mi­ra ex­pects to be­gin the late-stage tri­al work for atopic der­mati­tis be­fore the end of this year as it fol­lows a trail that ex­ecs in­sist leads to block­buster re­turns. Along the way, they’ll need to take on the 600-pound go­ril­la in atopic der­mati­tis: the IL-13/IL-4 drug Dupix­ent, from Re­gen­eron and Sanofi. Ri­vals al­so in­clude Leo Phar­ma, in its piv­otal with tralok­izum­ab, and Anap­tys­Bio in the hunt with a mid-stage pro­gram for etokimab, pre­vi­ous­ly re­ferred to as ANB020.