Biotech vet MacK­ay grabs a $25M A round to back a gene ther­a­py up­start

Ge­off McK­ay

Af­ter com­plet­ing an 11-year stint as CEO of Organo­gen­e­sis, Ge­off MacK­ay gath­ered a small, ex­pe­ri­enced team to­geth­er and be­gan a glob­al search for new gene ther­a­pies at Avro­Bio. Ear­li­er this year, they land­ed seed fi­nanc­ing from At­las Ven­ture af­ter ze­ro­ing in on the work of a pair of sci­en­tists in Toron­to. And to­day he land­ed a $25 mil­lion A round to get his two lead pro­grams in­to the clin­ic lat­er this year.

MacK­ay be­lieves that this is ex­act­ly the right time to get in­to gene ther­a­py, af­ter pi­o­neers like Blue­bird have es­tab­lished how the tech­nol­o­gy can work safe­ly. And he’s putting that to the test with a new gene ther­a­py aimed at rare cas­es of Fab­ry dis­ease.

“We like that the con­cept has been val­i­dat­ed,” says the biotech vet, who al­so ex­pects to be able to move quick­ly now that the A round is in place. MacK­ay says that if all goes ac­cord­ing to plan, the first round of safe­ty and ef­fi­ca­cy da­ta from small stud­ies should show up next year. But he’s not try­ing to rush things.

For the Fab­ry pro­gram, which will look to sub­sti­tute en­zyme re­place­ment ther­a­pies with a once-and-done cu­ra­tive treat­ment, in­ves­ti­ga­tors are us­ing an ex vi­vo ap­proach: ex­tract­ing CD34+ hematopo­et­ic stem cells from pa­tients and then us­ing a lentivi­ral vec­tor to reengi­neer them to ex­press the nor­mal gene be­fore in­fus­ing them back in­to the pa­tient.

Avro­Bio al­so has an en­try in the fast-grow­ing im­munother­a­py are­na for acute myeloid leukemia. It’s ex­tract­ing can­cer cells and en­gi­neer­ing them to ex­press IL-12 in or­der to spur a durable T cell as­sault on the can­cer.

“IL-12 not a new con­cept or cy­tokine,” says the CEO. “It’s been stud­ied for 20 years.” It’s al­so been too tox­ic to use sys­tem­i­cal­ly, so Avro­Bio is us­ing its tech­nol­o­gy to get right in­to the tu­mor mi­croen­vi­ron­ment, where it can do all the dam­age it can af­ter cap­tur­ing all the neoanti­gens in the can­cer cell.

The Avro­Bio team is work­ing with two sci­en­tif­ic co-founders, Jef­frey Medin, who un­til re­cent­ly was a pro­fes­sor at the Uni­ver­si­ty of Toron­to along with Christo­pher Paige. Avro­Bio is based in Cam­bridge, home turf to MacK­ay, with a fa­cil­i­ty in Toron­to where it can re­main close to its sci­en­tif­ic home.

At­las Ven­ture, Clarus and SV Life Sci­ences co-led the A-round, con­tribut­ing their part­ners to the board. Bruce Booth at At­las is sign­ing on as chair­man while Scott Re­quadt, a man­ag­ing di­rec­tor at Clarus, and Josh Resnick at SV Life Sci­ences join the board.

Right now, the team con­sists of a core group of 8, which MacK­ay says is set to grow.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.