Gene ther­a­pies for the cats and dogs you love? Penn-part­nered Scout Bio just raised $20M to make it a re­al­i­ty

The gene ther­a­py rev­o­lu­tion that’s been sweep­ing through biotech isn’t just about hu­mans any­more. A Penn-part­nered biotech called Scout Bio has just picked up a $20 mil­lion round to step up its work on once-and-done ther­a­pies for com­mon pet ail­ments.

Co-found­ed by leg­endary gene ther­a­py pi­o­neer and Penn in­ves­ti­ga­tor James Wil­son, Scout is us­ing some fa­mil­iar AAV tech to go af­ter dis­eases like chron­ic kid­ney dis­ease, chron­ic pain and the in­flam­ma­to­ry skin con­di­tion atopic der­mati­tis.

Cindy Cole

“In the Unit­ed States alone, there are 90 mil­lion dogs and 94 mil­lion cats, un­der­scor­ing the op­por­tu­ni­ty in a new gen­er­a­tion of med­i­cines that dra­mat­i­cal­ly im­prove stan­dards of care for ma­jor chron­ic con­di­tions,” said Cindy Cole, Tech­ni­cal Part­ner at Dig­i­tal­is Ven­tures.

Need­less to say, Scout need nev­er go be­yond an­i­mal stud­ies for an ap­proval to mar­ket these drugs to vets — though pre­sum­ably the cost of these ther­a­pies will be dra­mat­i­cal­ly low­er than the high 6-fig­ure and pro­posed 7-fig­ure price tags that have at­tract­ed so much dis­cus­sion.

I queried Scout about that and heard back that the com­pa­ny be­lieves it can mar­ket gene ther­a­pies to a mass au­di­ence of pet own­ers — but not at a mil­lion dol­lars or more per shot.

Their mar­ket re­search high­lights that half of all pet own­ers would pay $1,000 to treat their an­i­mals. Close to one in 4 would go as high as $5,000. Com­bine the greater ef­fi­cien­cies be­ing made in man­u­fac­tur­ing gene ther­a­pies with a po­ten­tial mass mar­ket of 10s of mil­lions, and Scout be­lieves it can treat those crit­ters and make mon­ey at it. Be­sides, they add, an­i­mal drug de­vel­op­ment isn’t near­ly as ex­pen­sive as R&D for hu­mans.

Per­haps they are on to some­thing that would help the rest of the field when it comes to gene ther­a­py costs.

Their lead pro­gram is ane­mia in cats with CKD, and Scout says they’ve reg­is­tered hikes in red blood cells in their test an­i­mals.

Scout has a top syn­di­cate back­ing their play. Dig­i­tal­is Ven­tures’ Com­pan­ion Fund and River­Vest Ven­ture Part­ners led the round, joined by new in­vestor Green­Spring As­so­ci­ates as well as ex­ist­ing in­vestors Fra­zier Health­care Part­ners, Adage Cap­i­tal Man­age­ment and Cor­re­la­tion Ven­tures.


Im­age: Shut­ter­stock

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.