FDA or­ders a rare re­treat on Am­i­cus’ mi­gala­s­tat, drop­ping PhI­II de­mand and sig­nal­ing a ma­jor shift for drug de­vel­op­ers

Megan Crow­ley is rec­og­nized by Pres­i­dent Don­ald Trump dur­ing his first ad­dress to a joint ses­sion of Con­gress on Feb­ru­ary 28, 2017. At 15 months old, Crow­ley was di­ag­nosed with Pompe Dis­ease and not ex­pect­ed to live more than a few short years. AP Im­ages


Am­i­cus Ther­a­peu­tics $FOLD an­nounced ear­ly Tues­day that the FDA has dropped its de­mand for a new Phase III study of its Fab­ry dis­ease drug mi­gala­s­tat af­ter it was stiff-armed by un­sat­is­fied reg­u­la­tors last fall — then un­der a dif­fer­ent ad­min­is­tra­tion with a dif­fer­ent FDA com­mis­sion­er.

In­stead of pur­su­ing a new and ex­pen­sive late-stage study, Am­i­cus CEO John Crow­ley tells me the com­pa­ny has the green light to file again with the da­ta at hand. And based on dis­cus­sions with the FDA, he adds, the com­pa­ny is shoot­ing to file in the 4th quar­ter, with an ac­cel­er­at­ed ap­proval that could ar­rive in 2018, po­ten­tial­ly slic­ing years off the process.

“These are the days it’s good to be a biotech CEO,” says Crow­ley, who has been on a roller coast­er ride at the FDA over the past year af­ter be­ing forced to walk back his own plans last No­vem­ber.

John Crow­ley

There is no guar­an­tee of an ap­proval here, but the agency’s sud­den volte-face clear­ly sig­nals a new tone that vast­ly im­proves the biotech’s shot at an OK — at least in so far as we hear it from Crow­ley. And that could have a ma­jor in­flu­ence on oth­er de­vel­op­ers tack­ling the same task.

In­vestors cheered on the sud­den change in for­tunes at Am­i­cus, bid­ding up shares by 43% in pre-mar­ket trad­ing Tues­day.

Crow­ley has nev­er made a se­cret of the fact that he felt the drug — ap­proved in Eu­rope last year — was ready to go in the US af­ter it com­plet­ed the piv­otal pro­gram. The FDA, though, re­ject­ed his ap­pli­ca­tion, say­ing reg­u­la­tors want­ed more safe­ty da­ta than the Eu­ro­peans need­ed.

That re­jec­tion be­came a cause cele­bre of sorts in DC, as Pres­i­dent Don­ald Trump turned to Crow­ley’s daugh­ter, Megan, who suf­fers from Pompe dis­ease, and called out the FDA for the “slow and bur­den­some” process in­volved in new drug ap­provals dur­ing an ad­dress to Con­gress.

“Our slow and bur­den­some ap­proval process at the Food and Drug Ad­min­is­tra­tion keeps too many ad­vances, like the one that saved Megan’s life, from reach­ing those in need,” Trump said at the time. “If we slash the re­straints, not just at the FDA, but across our gov­ern­ment, then we will be blessed with far more mir­a­cles like Megan.”

Crow­ley picked up on that change in think­ing at the White House in a blog post at the time in which he said: “The FDA’s reg­u­la­tion of the or­phan de­vel­op­ment process is be­com­ing less flex­i­ble, less ef­fi­cient and less pa­tient-cen­tered.”

I asked Crow­ley what had changed be­tween the set­back last fall and the new, soft green light that the FDA has switched to.

First and fore­most, Crow­ley hit the theme that the com­pa­ny had made a con­vinc­ing case with what was avail­able. That in­cludes new da­ta and analy­sis that was made pos­si­ble af­ter the re­jec­tion. The com­pa­ny start­ed from the be­gin­ning to mar­shal the ev­i­dence need­ed to meet the agency’s gold stan­dard for an OK. That, he says, was ab­solute­ly key, ul­ti­mate­ly win­ning the day.

In ad­di­tion, he says there’s al­so a new frame­work for drug re­views that’s been gath­er­ing steam at the agency.

“I do think, can­did­ly, that there is an emerg­ing frame­work in rare dis­eases,” he says, point­ing to new ap­provals for Bio­marin on Bat­ten dis­ease as well as a nov­el la­bel ex­ten­sion for Ver­tex based on their in vit­ro as­say.

Scott Got­tlieb ap­pears be­fore a US Sen­ate sub­com­mit­tee, June 20, 2017 AP Im­ages

You can bet, though, that there will be plen­ty of sen­ti­ment sug­gest­ing that a change of ad­min­is­tra­tions and a new FDA com­mis­sion­er in Scott Got­tlieb made a telling dif­fer­ence for an abrupt about-face that is ex­tra­or­di­nar­i­ly rare to see at the FDA.

What­ev­er the cause, though, in­stead of prepar­ing to gath­er new da­ta that wouldn’t be avail­able un­til 2019, Crow­ley is back to mak­ing launch plans in the US. That will prob­a­bly re­quire a com­mer­cial group of about 50, he says, com­pared to the 80 need­ed to ad­dress a more com­plex and chal­leng­ing pay­er en­vi­ron­ment in Eu­rope.

Biotech has large­ly cheered on new FDA com­mis­sion­er Scott Got­tlieb af­ter he won con­fir­ma­tion by the Sen­ate. He promised to turn to new tech­nolo­gies to help ac­cel­er­ate new ap­provals, but vowed to stick with the in­dus­try gold stan­dard that has gov­erned reg­u­la­tors thoughts on safe­ty and ef­fi­ca­cy date.

So did the FDA just soft­en its stan­dards or wake up to the fact that the drug should nev­er have been re­ject­ed in the first place?

Let’s start the dis­cus­sion.

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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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.

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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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.

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.

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.

Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

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.