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

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Frank Zhang (AP Images)

Rocked by cus­toms in­ves­ti­ga­tion, Leg­end's CFO takes over as CEO Frank Zhang placed un­der house ar­rest

When Frank Zhang stepped down from GenScript — the contract research group he’s run for 18 years — to take up the CEO post at its CAR-T focused spinout Legend Biotech, he assured analysts that he was in for the long haul.

Just 49 days later, though, he’s been forced to hand back the title.

In a dramatic turn of events, Legend disclosed that Zhang is under house arrest in China as part of a customs investigation involving GenScript. While he remains the chairman, CFO Ying Huang has been tapped to double as interim CEO.

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UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”