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.

Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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