Alex Zhavoronkov (Insilico)

In­sil­i­co preps first can­di­date for IND stud­ies, hop­ing to launch tri­al in IPF by end of 2021

Over the last sev­er­al years, Alex Zha­voronkov has turned In­sil­i­co in­to one of the most well-con­nect­ed AI biotechs out there. Now, the Hong Kong-based com­pa­ny is one step clos­er to reach­ing the clin­ic.

In­sil­i­co is bring­ing its first can­di­date in­to IND-en­abling stud­ies, Zha­voronkov an­nounced Wednes­day, with the goal of launch­ing an in-hu­man tri­al at some point lat­er this year. The move marks what he says is a first in AI drug dis­cov­ery, as both the tar­get and small-mol­e­cule in­hibitor in­volved in the pro­gram are com­plete­ly new and AI-gen­er­at­ed.

Zha­voronkov, who told End­points News he builds self-dri­ving ro­bots in his spare time, isn’t ready just yet to say what the tar­get or ex­per­i­men­tal drug are, but he not­ed that the fo­cus will be id­io­path­ic pul­monary fi­bro­sis. The tar­get it­self plays a key role in fi­brot­ic and in­flam­ma­to­ry path­ways, sit­ting on the in­ter­sec­tion of profi­brot­ic path­ways, he said.

More im­por­tant to In­sil­i­co is the process by which they dis­cov­ered the com­pound — de­spite us­ing more than 60 dif­fer­ent ap­proach­es, the main method was through its pro­pri­etary AI plat­form’s neur­al net­works. In­sil­i­co re­searchers start­ed by look­ing at how dif­fer­ent tis­sues sus­cep­ti­ble to fi­bro­sis can change over the course of pa­tients’ lives, but that op­er­a­tion is too dif­fi­cult for hu­mans to do on their own, as the req­ui­site datasets are mas­sive.

“We train on age, af­ter that we re-train on fi­bro­sis, get the first list of pri­or­i­tized like­ly tar­gets, and then we put them in­to bi­o­log­i­cal path­ways,” Zha­voronkov told End­points.

Af­ter that, In­sil­i­co ap­plied a sep­a­rate neur­al net­work to comb through data­bas­es over where gov­ern­ments were is­su­ing grant re­search and com­pa­nies were tak­ing clin­i­cal tri­als. That helped Zha­voronkov de­ter­mine whether the tar­get is ac­tu­al­ly new or if oth­er com­pa­nies had al­ready tried to make some­thing work with it.

Last­ly, Zha­voronkov want­ed to be able to trust the an­swers his neur­al net­works were spit­ting out. So he es­sen­tial­ly asked his AI to find po­ten­tial hid­den links be­tween the da­ta to in­crease their con­fi­dence that a drug would work, while al­so look­ing at how cer­tain key opin­ion lead­ers viewed the bi­ol­o­gy they were try­ing to crack.

In­sil­i­co de­vel­oped its plat­form in col­lab­o­ra­tion with Big Phar­mas to in­cor­po­rate how the “best of the best” hu­man re­searchers think about tar­get se­lec­tion, Zha­voronkov said, adding that in lots of cas­es, how­ev­er, tar­gets are cho­sen for busi­ness rea­sons rather than sci­ence. He wants to try to change that with his plat­form and Wednes­day’s an­nounce­ment.

“We need to find this ul­ti­mate bal­ance be­cause you re­al­ize that more than one tar­get can be im­pli­cat­ed in this dis­ease,” Zha­voronkov said. “So it is pos­si­ble there is not just one mag­ic bul­let, there could be sev­er­al bul­lets.”

When this new pro­gram ul­ti­mate­ly reach­es the clin­ic, by the end of this year at the ear­li­est, it will be a typ­i­cal Phase I study. But Zha­voronkov said he has the “lux­u­ry” of ex­per­i­ment­ing with every step of the tri­al giv­en oth­er phar­ma com­pa­nies haven’t yet en­tered this space in this fash­ion.

The im­me­di­ate next steps are to con­tin­ue push­ing out the plat­form to oth­er play­ers. In­sil­i­co has made the core of its AI open source in the hopes of bring­ing down drug dis­cov­ery costs across the in­dus­try. Zha­voronkov said val­i­dat­ing this tar­get in IPF cost In­sil­i­co on­ly $1.8 mil­lion.

“If I do not in­vent, I do not live,” Zha­voronkov said. “We will nev­er stop and we al­so have a huge pipeline of projects in AI.”

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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