With a po­ten­tial cure for lyso­so­mal stor­age dis­or­ders in hand, Avro­bio rais­es $60M to back gene ther­a­py pipeline

In gene ther­a­py, re­spons­es from just one or two pa­tients can have a huge in­flu­ence on an­a­lysts’ opin­ions. Just ask the ex­ec­u­tive team at blue­bird bio, which trum­pets each pa­tient’s progress with de­light — or cal­i­brates a new de­vel­op­ment strat­e­gy when smiles turn to frowns.

So it’s no won­der that Avro­bio is so proud of its very first pa­tient.

Ge­off MacK­ay

At a sci­en­tif­ic con­fer­ence in Japan last fall, re­searchers re­viewed the case of a pa­tient with Fab­ry dis­ease whose blood lev­els of the en­zyme a-Gal A were near ze­ro at the start of treat­ment. With­in 45 days of re­ceiv­ing their gene ther­a­py AVR-RD-01, the pa­tient’s plas­ma a-Gal A ac­tiv­i­ty in­creased in­to the nor­mal range, they said. And 6 months lat­er that hadn’t changed.

In oth­er words, they got the re­sponse they want­ed and Pa­tient 1 cleared the first hur­dle on dura­bil­i­ty. All sys­tems go for a young com­pa­ny that is out to cre­ate a once-and-done fix.

At­las seed­ed the com­pa­ny — Bruce Booth is the proud chair­man — and then launched it in the spring of 2016 with a $25 mil­lion A round that backed the work of 15 staffers. Over the last few months, its staff has dou­bled. And more hires are be­ing planned for lat­er this year.

That up­beat start has helped in­spire a hefty $60 mil­lion round to back the mid-stage work that will soon be­gin for a com­pa­ny that has laid out a broad plan to fol­low some of the lead­ers in lyso­so­mal stor­age dis­or­ders with the promise of a cure that could one day rel­e­gate the cur­rent stan­dard of care for these ail­ments to an his­toric foot­note.

Bruce Booth

The Se­ries B round was co-led by Cor­morant As­set Man­age­ment and Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), and in­clud­ed Ais­ling, Brace Phar­ma Cap­i­tal, Even­tide, Morn­ing­side and Leerink Part­ners, along with ex­ist­ing in­vestors At­las Ven­ture, SV Health In­vestors and Clarus Ven­tures.

Get­ting an in­vest­ment group like Leerink in your round — not an every­day event in biotech — might make you think that there’s an IPO in the off­ing, es­pe­cial­ly with 2018 look­ing like an es­pe­cial­ly good year to take a biotech pub­lic. Gene ther­a­py pi­o­neers have al­so earned a lot of re­spect on Nas­daq, even with some tur­bu­lence for every set­back.

But Avro­bio CEO Ge­off MacK­ay is cir­cum­spect about any near-term of­fer­ings.

The com­pa­ny’s fo­cus this year is on kick­ing off a crit­i­cal Phase II tri­al for Fab­ry dis­ease, he says, to run par­al­lel to the Phase I.

There are no time­lines for the tri­als on pub­lic dis­play, though.

In gene ther­a­py, even af­ter the first US ap­proval, all com­pa­nies in the field tend to get striped with the same brush. MacK­ay’s de­sire now is to make sure that every­one knows that Avro­bio is in the lentivi­ral camp, where you’ll find lead­ers like blue­bird bio.

“AAV is a dif­fer­ent vec­tor,” he says. “It’s not fair to ex­trap­o­late to lentivi­ral.”

And at that point I hadn’t even men­tioned the James Wil­son study sound­ing the alarm around high-dose gene ther­a­pies us­ing AAV tech ear­li­er in the week.

A break­through with a gene ther­a­py would un­doubt­ed­ly have a huge im­pact on the pa­tients who de­pend on steady in­fu­sions of some high-priced ther­a­pies.

“We’re very re­spect­ful of the con­tri­bu­tion Gen­zyme and Shire’s made,” says MacK­ay. “Twen­ty years ago these pa­tients had no op­tions, then the pa­tients had an op­tion. When we look at it with fresh eyes, we see a sig­nif­i­cant op­por­tu­ni­ty to im­prove on the stan­dard of care.”

“Pa­tients now re­ly on bi­week­ly in­fu­sions of en­zymes, with se­vere chal­lenges to cost, com­pli­ance and qual­i­ty of life,” adds the CEO. “When ERT suc­ceeds it slows the pro­gres­sion of dis­ease but doesn’t halt it. The cen­tral premise is to de­liv­er via a one-time in­fu­sion for 24-7 pro­tec­tion, which we hope will halt the pro­gres­sion rather than just slow it down.”

Avro­bio has a long ways to go, but it has big plans for when it ar­rives.

Daniel O'Day [via AP Images]

UP­DAT­ED: Look­ing to re­solve lin­ger­ing doubts, Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

UP­DAT­ED: Am­gen, No­var­tis scrap Alzheimer's stud­ies — is BACE fi­nal­ly dead or will Bio­gen and Ei­sai car­ry on?

The BACE theory of controlling Alzheimer’s died with failed pivotal projects at Merck, Eli Lilly and their partners at AstraZeneca. Now Amgen and Novartis have come along to bulldoze it under a mound of safety threats — leaving only Biogen and Eisai to carry on with a less than zero chance of success — with the notable addition that they may actually be doing harm to patients.

After the market closed Thursday, Amgen and Novartis announced that they were dumping two pivotal programs underway with the Banner Alzheimer’s Institute on their BACE drug CNP520 (umibecestat) after an independent review of the data indicated that patients’ cognitive abilities were actually worsening at a faster pace than the placebo arm.

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Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Fresh out of Eli Lil­ly, Christi Shaw sur­faces as Daniel O'­Day's new CEO at CAR-T pi­o­neer Kite

Well, that didn’t take long. 

We found out Thursday evening that Christi Shaw has given up her top post as the head of the Bio-Medicines group at Eli Lilly for the helm at CAR-T pioneer Kite. New Gilead CEO Daniel O’Day, a Roche veteran, had made finding a Kite CEO a top priority on his arrival at Gilead. And he went right for a headliner.

O’Day was clearly excited about the coup.

“We conducted an extensive search for a new leader at Kite and we believe that Christi’s unique set of skills will allow us to continue to build on our leadership position in cell therapy,” he said in a prepared statement. “Christi’s vast experience across complex therapeutic areas, and particularly in oncology, will serve Kite very well. She is clearly a leader who will bring teams and individuals together and I am confident she will build upon the entrepreneurial spirit at Kite as we seek to help more people with cancer around the world.”

Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Eli Lil­ly's Christi Shaw bows out of top post at the Bio-Med­i­cines unit

Less than 3 years after Eli Lilly CEO David Ricks recruited Novartis vet Christi Shaw to run their big Bio-Medicines business, she’s out.

In a statement put out Thursday morning, Lilly said that Shaw’s last day will come at the end of August. Patrik Jonsson, currently president and general manager of Lilly Japan, will succeed Shaw once he gets the paperwork sorted out.

Lilly’s shares dropped 4% on the news.

Jeff Poulton

Al­ny­lam’s Maraganore switch­es ‘per­haps the best CFO in mid-cap biotech’ with Shire vet Jeff Poul­ton

There’s a new CFO taking charge of the numbers at RNAi pioneer Alnylam.

Alnylam chief John Maraganore says that CFO Manmeet S. Soni is leaving in the proverbial pursuit of new opportunities. And he’s being replaced by ex-Shire CFO Jeff Poulton, not long after the Takeda takeover obliterated that position.

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Analy­sis: In most of the Big Phar­ma world, R&D spend­ing tow­ers over rev­enue from new drugs. Guess who beat the odds

It’s always been the case that there’s no medicine less useful than one that doesn’t make it to patients (unless you regard the task of R&D to be perpetually learning about swimming, while under water…). Yet, launching new medicines that physicians want, that payers will cover, and that patients will take, is a discipline that is unevenly distributed among the big players.

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President Donald Trump at State of the Union. AP Images

White House changes course to kill re­bate re­form

So what exactly is the White House’s plan to tackle rising drug prices? It doesn’t look like we will get definitive answers anytime soon. On the heels of President Donald Trump’s surprise revelation that an executive order is in the works to implement a “favored nations clause,” his administration is now putting out word that it has abandoned an earlier proposal to overhaul rebates.

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The FDA's Janet Wood­cock talks about some big changes she's push­ing for in drug de­vel­op­ment, and agency re­views

Janet Woodcock is perhaps the most influential regulator at the FDA. And when the head of CDER talks about the changes being made at the agency when it comes to clinical trial designs, or the need to reorganize for a specific disease arena, an assessment of the expansion of gene therapy or I/O, common development mistakes, and so on, you can be sure the industry pays attention to every word.

So it was with some eagerness that I opened up Geoffrey Porges’ summary of their recent conversation about the FDA. And I wasn’t disappointed. In a wide-ranging exchange with the SVB Leerink analyst, Woodcock discussed the growing importance of patient-reported outcomes in clinical trials, a campaign underway now to see if CROs would help spur more basket studies to compare drugs head-to-head, and much, much more.

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Ex-DARPA di­rec­tor pur­sues all-in-one can­cer pill as NED CEO; Karyopharm los­es com­mer­cial chief ahead of drug roll­out

“Why not try?”

That’s what Geoffrey Ling told me over the phone when asked about what led him to his journey to the position of CEO at NED Biosciences — a company with a lofty goal of creating an all-in-one oral drug to treat all types of cancer and making this drug available to not only developed nations, but also the developing world. 

Ling comes from an extensive background in medicine and the government. He is the co-leader of The Brain Health Project, a professor of neurology and an attending neurocritical care physician at John Hopkins University and Hospital, as well as the assistant director for Medical Innovation of the Science Division in president Obama’s White House Office of Science and Technology Policy (OSTP).