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

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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