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.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,500+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,500+ biopharma pros reading Endpoints daily — and it's free.

Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,500+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,500+ biopharma pros reading Endpoints daily — and it's free.