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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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David Meline, incoming Moderna CFO

Am­gen vet David Meline finds a new CFO roost at Mod­er­na, tak­ing a ride on the Covid-19 tiger as de­part­ing ex­ec cash­es out with $12M

We found out a few weeks ago that Moderna CFO Lorence Kim isn’t waiting around to see how the biotech wunderkind makes out in its frantic race to field a messenger RNA vaccine that can quell Covid-19. And now we know who’s stepping on board to take his place in the latest move in the executive suite.

David Meline, who forged his rep during a 6-year run at Amgen, slipped out the exit right after his Q2 “retirement” party in California — presumably virtual — and started the next chapter of his career at a biotech company betting big on revolutionizing the vaccine R&D space.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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