Io­n­is finds new part­ner in a young biotech and its hope­ful founder

Om­ri Gottes­man came to the US from the UK 10 years ago, wide-eyed like many oth­ers.

The hu­man genome project was long-com­plete and, with great fan­fare, re­searchers were be­gin­ning to try and lever­age those in­sights in­to treat­ments. Mount Sinai School of Med­i­cine of­fered him a fel­low­ship fo­cused on just that: ge­nom­ic med­i­cine.

“At the time, there was a lot of hope and hype that we had solved health, and would be able to dis­cov­er and pre­vent every­thing and treat every­one,” Gottes­man told End­points News. It was the rea­son I came to Mount Sinai.”

Om­ri Gottes­man

Over the en­su­ing decade, hu­man bi­ol­o­gy proved much more elu­sive than they imag­ined. Ge­net­ics was more com­pli­cat­ed than A-T and C-G. There were no easy hacks. Still, the field ad­vanced and af­ter 4 years at Mount Sinai and a stint at Re­gen­eron, Gottes­man de­cid­ed he want­ed to ad­vance it on his own and build a new plat­form around the orig­i­nal mis­sion: An­a­lyze ge­net­ic in­for­ma­tion to home in on ide­al drug tar­gets. He called the new com­pa­ny Em­piri­co and soon raised $30 mil­lion.

It’s not a unique con­cept, but it’s one Gottes­man pulled off well enough to to­day score a three-year part­ner­ship with Io­n­is Phar­ma­ceu­ti­cals, one of the old­est and more suc­cess­ful ge­net­ics-based drug de­vel­op­ers.

Io­n­is will make a $10 mil­lion eq­ui­ty in­vest­ment in­to Em­piri­co and of­fer $30 mil­lion in near-term op­er­a­tional and pre­clin­i­cal mile­stones, with an­oth­er $620 mil­lion promised for clin­i­cal, reg­u­la­to­ry and sales goals. In ex­change, Em­piri­co will make up-to 10 plat­form-dis­cov­ered drug tar­gets avail­able to Io­n­is.

“We spent about a year build­ing the dis­cov­er-first plat­form,” Gottes­man said. “Part of the mo­ti­va­tion for the Io­n­is col­lab­o­ra­tion – and ex­plor­ing col­lab­o­ra­tions more broad­ly – is that we now have more po­ten­tial tar­gets than our ca­pac­i­ty” to de­vel­op ther­a­pies for them.

On the Io­n­is side, this is the lat­est col­lab­o­ra­tion for a biotech that has sought many since its found­ing and is try­ing to keep ear­ly-stage strong while some late-stage pro­grams come to fruition. The deal al­so in­cludes an op­tion for Em­piri­co to li­cense a drug back from Io­n­is as it strives to be­come a tar­get-to-mar­ket com­pa­ny.

You’ve heard of plat­forms like Em­piri­co be­fore; it’s the mixed ma­chine learn­ing and wet lab ap­proach tak­en by a grow­ing num­ber of biotechs. Gottes­man and his team use their com­put­er plat­form to in­ter­ro­gate ge­net­ic da­ta for the roots of dis­eases. Should the com­put­er find a mis­pelling or oth­er vari­ant in ge­net­ic code that ap­pears to cause the mal­func­tion, they then pro­duce cell lines with that mis­pelling in the lab. Should those cell lines show the same mal­func­tion as the dis­ease, they then use their plat­form to iden­ti­fy the best form for a ther­a­py.

They’ve used that method to build 5 pre­clin­i­cal tar­gets, lead­ing to a small mol­e­cule pro­gram for an up­per air­way dis­ease and an siR­NA can­di­date for glau­co­ma.

The tar­gets for the Io­n­is col­lab­o­ra­tion will be those Em­piri­co finds can be best-treat­ed with their part­ner’s sig­na­ture an­ti­sense tech­nol­o­gy. The biotech has al­ready iden­ti­fied a cou­ple, al­though they didn’t dis­closed them.

A 40-year-old con­cept, an­ti­sense ther­a­pies have re­cent­ly brought some of the ge­net­ics-based health gains Gottes­man and oth­ers sought. Along with a hand­ful of ap­proved drugs, last year a neu­rol­o­gist in Boston cre­at­ed a per­son­al­ized ther­a­py for a young girl with a nev­er-be­fore-seen ge­net­ic er­ror. And yes­ter­day, Io­n­is founder Stan­ley and Rosanne Crooke launched a char­i­ty to make those kinds of ge­net­ics-based ul­tra-per­son­al­ized med­i­cines broad­ly ac­ces­si­ble.

Those be­spoke ther­a­pies are ar­guably the apex of the vi­sion Gottes­man and oth­ers had 10 years ago. It’s a vi­sion he thinks has made more progress than some think, even if it hasn’t brought any utopi­an dream.

“Over the last decade, we’ve re­al­ized it’s re­al­ly hard­er than it seems,” he said. “But ac­tu­al­ly, if you work at it, there’s re­al val­ue.”

So­cial im­age cred­it: Io­n­is via YouTube

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

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

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.

Bull­ish biotech mar­ket pro­pels Pli­ant to $144M IPO — as No­var­tis pro­vides a $10M boost

After pharma partner Novartis boosted its IPO with a $10 million private placement, Pliant Therapeutics has wrapped its journey to the Nasdaq on a high note.

Pliant had penciled in a $86 million raise back in May. But as has become the norm in recent months, that initial number has turned out to be a mere placeholder, making way for the final haul of $144 million.

The South San Francisco biotech did so by pricing at $16, the high end of the range, while bringing the number of shares offered up to 9 million.

Credit: AP Images

Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

Cameron Durrant, Humanigen CEO (Columbia University Technology Ventures via YouTube)

Cameron Dur­rant hus­tled his way from the OTC side­lines right in­to the Covid-19 drug race. Death or glo­ry lies straight ahead

Over the past few months, Covid-19 has gone from being a monolithic threat to one of the biggest overnight boons the biopharma industry has ever seen. And amid all the furor over Moderna’s swelling stock price, plenty of chatter over what new drugs and vaccines will cost and investors’ uninhibited zeal for all things related to pandemic products, it’s been one little biotech’s golden ticket back from the land of the living dead.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.