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.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.