Am­i­cus CEO Crow­ley bags a gene ther­a­py com­pa­ny, a new pipeline — and a great pho­to op — for $100M in cash

Am­i­cus CEO John Crow­ley has struck a deal to buy up a port­fo­lio of gene ther­a­pies for an ex­treme­ly rare dis­ease — bag­ging a small biotech for $100 mil­lion in cash plus hun­dreds of mil­lions in mile­stones. And it in­cludes some great op­tics.

Crow­ley, who’s been fond of high­light­ing a biotech ca­reer in­spired by a daugh­ter af­flict­ed with Pompe dis­ease, is ac­quir­ing a com­pa­ny launched by Hol­ly­wood pro­duc­er Gor­don Gray and his wife, Kris­ten, whose own daugh­ters suf­fer from Bat­ten dis­ease.

Bri­an Kas­par

The key fig­ure you shouldn’t lose sight of here is Ce­lenex co-founder Bri­an Kas­par of Na­tion­wide Chil­dren’s Hos­pi­tal in Colum­bus, who spear­head­ed the sci­ence work on AveX­is’ gene ther­a­py for spinal mus­cu­lar at­ro­phy — since bought out by No­var­tis for $8.7 bil­lion. Kas­par, Kathrin Mey­er and Arthur Burgh­es of Ohio State did the pre­clin­i­cal and ear­ly Phase I work on a slate of gene ther­a­pies for Bat­ten dis­ease that now go in­to the pipeline at Am­i­cus.

With this much cash up­front. Crow­ley is keep­ing the de­vel­op­ment mile­stones lean, point­ed­ly lim­it­ing that part to $15 mil­lion in po­ten­tial pay­ments. There’s an­oth­er $262 mil­lion in mile­stones on the ta­ble for sub­mis­sions and ap­provals.

Am­i­cus, which on­ly re­cent­ly land­ed a con­tro­ver­sial OK for their new Fab­ry dis­ease drug Galafold, is tak­ing on $150 mil­lion in debt to pay for the deal. That mon­ey is com­ing from Bio­Phar­ma Cred­it, an in­vest­ment fund man­aged by Phar­makon Ad­vi­sors.

The Gray fam­i­ly. (Im­age: Char­lotte and Gwyneth Gray Foun­da­tion)

Click on the im­age to see the full-sized ver­sion

Matthew Her­p­er at Forbes got the fam­i­ly por­trait sto­ry, which in­clud­ed a chat with Crow­ley, who al­ways en­joys spot­light­ing his pas­sion for R&D in­spired by sick kids.

Crow­ley in­clud­ed a pledge along­side the mon­ey, telling Her­p­er: “I promised Gor­don I would treat his chil­dren and the oth­er kids as if they were my own.”

Im­age: John Crow­ley. AM­I­CUS

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where will they look for an FDA hus­tle up?

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

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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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