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

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

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This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

Brex­it fears, Wood­ford woes over­shad­owed UK biotech and cut 2019 fi­nanc­ing by al­most half

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

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→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III Voyage trial before they give a judgment.

Io­n­is, Akcea boost­ed by a pos­i­tive PhII for their No­var­tis castoff car­dio drug — and they plan to push ahead in­to piv­otals

Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.

Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.

Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.

Hal Barron and Emma Walmsley, GSK

GSK’s ‘break­through’ BC­MA can­cer drug gets a pri­or­i­ty re­view — and a big win for the on­col­o­gy R&D team

After largely whiffing the past 2 years on the pharma R&D front, GlaxoSmithKline research chief Hal Barron has seized boasting rights to a key win that puts them back in the cancer drug development game.

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Who are the young bio­phar­ma lead­ers shap­ing the in­dus­try? Nom­i­nate them for End­points' spe­cial re­port

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.