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

Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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UP­DAT­ED: Am­gen tops cost watch­dog's price gougers list based on 'un­sup­port­ed' in­creas­es for En­brel with­out mean­ing­ful da­ta

In a top 10 ranking of the most egregious price gougers from 2019, Amgen’s Enbrel topped US cost watchdog ICER’s naughty list with “unsupported” markups that added as much as $403 million to the nation’s drug spend during that time.

Price increases for some of pharma’s most popular drugs have long been a focus of consumer ire, but the industry has argued those increases are routine and meant to cover the cost of R&D innovation. Without meaningful guidance at the state or federal level, ICER looked to connect how much a drug had progressed in the clinic compared with its increase in both wholesale and net price in 2019.

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Cog­nate dou­bles man­u­fac­tur­ing ca­pac­i­ties in Mem­phis, Eu­rope, as de­mand for cell and gene ther­a­pies sky­rock­ets

The marketplace for gene and cell manufacturing therapeutics continues to be scorching.

Cognate BioSciences, a leading CDMO specializing in gene and cell therapy technologies, announced plans Friday that will double its total manufacturing capacities at sites in both the US and Europe — in direct response to a “great demand of commercial capacity within the biologics industry.”

The company provided most details for its US expansion, which will take place at its current headquarters in Memphis, Tennessee near the Memphis International Airport — crucial, it said, as Memphis is one of the world’s busiest cargo airports. Cognate will add two separate facilities totaling 250,000-square-feet: a GMP distribution center to manage global supply chain needs, and a third site for commercial manufacturing.

Eli Lil­ly re-ups di­ver­si­ty pledge, pitch­ing in $30M to ven­ture fund for mi­nor­i­ty-owned health­care firms

The fight against racial injustice spurred by a series of high-profile shootings of Black men by police earlier this year put Big Pharma and healthcare — industries targeted for their lack of diversity — in the hot seat. Eli Lilly made an early pledge to change its ways and put more back into the community, and now it’s continuing to make good on that commitment.

Lilly will infuse $30 million into the Unseen Capital Health Fund, a venture fund looking to invest in early-stage minority-owned healthcare companies that have been historically “unseen” by the investment community, the pharma said Friday.

CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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Stefan Oelrich (Bayer)

What can a 157-year-old phar­ma gi­ant bring to the ta­ble of cell and gene ther­a­pies? Quite a bit, Bay­er says

By now, Bayer has sketched out in bold strokes some grand plans for cell and gene therapy, cemented by big-dollar acquisitions of platform companies.

But just how do you stitch together a new unit bursting with the newest ideas within a storied pharma?

Stefan Oelrich, the head of Bayer’s pharma division, briefly lifted the curtain and spotlighted three key factors as he took the stage on a virtual media day, flanked by Emile Nuwaysir and Sheila Mikhail, the chiefs of BlueRock and AskBio, who each introduced their work in a way you’d expect from a biotech CEO.