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

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK; First plas­ma tri­al sug­gests mor­tal­i­ty re­duc­tion

Count NIH director Francis Collins out for any wager that the FDA would authorize a Covid-19 vaccine in October.

The discussion came up during a call with reporters because some states and local governments have been told by the CDC to have vaccination plans ready to go by Oct. 1. Pharma execs, most notably from Pfizer and BioNTech, have raised hopes about a licensure during that month; President Donald Trump last week sounded an optimistic note about having a vaccine on the market “right around” Election Day on Nov. 3 — or possibly before.

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