Neil Thompson. Healx

As­tex vet Neil Thomp­son looks to build dis­cov­ery from scratch at rare dis­ease AI up­start out of Cam­bridge, UK

David Brown Healx

As a co-in­ven­tor of silde­nafil — a pill orig­i­nal­ly de­signed to treat coro­nary hy­per­ten­sion — David Brown knows how big of an im­pact drug hunters can make when they take a ther­a­py be­ing used to treat one dis­ease and di­rect it to an­oth­er. Af­ter 40 years in the in­dus­try, re­pur­pos­ing is at the cen­ter of his lat­est rare dis­ease ven­ture at Healx.

And now, step­ping the gas ped­al on new dis­cov­ery projects, Brown has re­cruit­ed Neil Thomp­son as the Cam­bridge, UK-based start­up’s new CSO.

Thomp­son, a 15-year vet­er­an of As­tex who’s been work­ing as an in­de­pen­dent con­sul­tant since its sale to Ot­su­ka two years ago, joins an am­bi­tious team of 35 that has its sight set on bring­ing for­ward 100 rare dis­ease treat­ments by 2025.

“Healx has prob­a­bly the most com­pre­hen­sive AI plat­form bring­ing to­geth­er in­for­ma­tion in rare dis­eases,” Thomp­son ex­plains. “So it in­te­grates the in­for­ma­tion around the dis­ease, the pub­lic lit­er­a­ture, patents, drugs, all that in­for­ma­tion and the al­go­rithms tell us which drugs are most like­ly to work in which dis­eases. So it’s a great plat­form for a bi­ol­o­gist like me to read the out­put and add that ex­tra phar­ma­col­o­gy, drug dis­cov­ery ex­per­tise to in­ter­pret and fo­cus the best com­pounds com­ing out of that pre­dic­tion.”

It’s a twist on the AI-dri­ven ap­proach to drug dis­cov­ery mak­ing the rounds, and its on­ly fi­nanc­ing round so far — a Se­ries A to­tal­ly $10 mil­lion — looks tiny com­pared to the re­cent megaround com­mand­ed by fel­low British out­fit Benev­o­len­tAI.

Tim Guil­liams Healx

But Thomp­son says that’s more than enough at this stage for him to build out the team, with the key fo­cus be­ing hir­ing drug dis­cov­ery and phar­ma­col­o­gy ex­perts to start as­sem­bling a port­fo­lio. Un­der its busi­ness mod­el, Healx will then col­lab­o­rate with pa­tient ad­vo­ca­cy groups and bio­phar­ma com­pa­nies to ini­ti­ate clin­i­cal tri­als and ul­ti­mate­ly com­mer­cial­ize the treat­ments.

So far the com­pa­ny, which is led by Brown as well as co-founder and CEO Tim Guil­liams, boasts of one col­lab­o­ra­tion on frag­ile X syn­drome, in which it de­liv­ered a clin­ic-ready as­set with­in 15 months.

Mov­ing for­ward, one key chal­lenge in mar­ry­ing AI with drug dis­cov­ery will be fos­ter­ing smooth team­work among spe­cial­ists in com­pu­ta­tion, bioin­for­mat­ics bi­ol­o­gy and chem­istry who can work to­geth­er smooth­ly, Thomp­son ac­knowl­edges.

“I think the se­cret there is re­cruit­ment of the right in­di­vid­u­als,” he said, peo­ple who are ex­perts in their own area but ready to “peek out of the box,” com­mu­ni­cate to oth­ers out­side their field and be flex­i­ble. “It’s all about get­ting the right team to­geth­er in an en­vi­ron­ment that is mo­ti­va­tion­al but with clear ob­jec­tives.”

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.

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.”

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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.

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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.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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