CRISPR up­start ticks off an­oth­er box on the IPO prep list as RA, SVB Leerink step in on a mega-raise

Bioreg­num Opin­ion Col­umn by John Car­roll

With 2020 set to bring the cur­tain down on a biotech IPO boom un­like any­thing we’ve seen be­fore, you have to won­der which com­pa­nies are set­ting the stage to try and fol­low up with their own S-1 in 2021. And Lo­can­abio may be in the mix af­ter to­day.

The San Diego-based biotech just brought in a mega-round, al­ways help­ful ahead of any IPO. In this case, that’s $100 mil­lion from a slate of in­vestors that’s adding groups with a well-known taste for IPO strate­gies: Vi­da Ven­tures, RA Cap­i­tal, In­vus, Acu­ta Cap­i­tal and an in­vest­ment arm of mar­ket mak­er SVB Leerink. Ra­jul Jain, an MD who had been on the ex­ec­u­tive team at Kite be­fore join­ing Vi­da with Arie Bellde­grun, is mov­ing to the Lo­can­abio board.

They’re all jump­ing on a plat­form play set up by a fa­mil­iar line­up of A list back­ers: ARCH, Temasek, Light­stone Ven­tures, UCB Ven­tures and GV (the VC for­mer­ly known as Google Ven­tures). They were in­spired to in­vest in a com­pa­ny that us­es CRISPR Cas9 to tar­get faulty RNA, a con­cept that was hatched by UC San Diego sci­en­tists David Nelles and Gene Yeo.

Ra­jul Jain

The biotech brought in Jim Burns as CEO about a year ago — an old hand at the drug de­vel­op­ment game who jumped from a lengthy stint at Sanofi-Gen­zyme to Case­bia and then on to Lo­can­abio. Burns made the jump right af­ter CRISPR Ther­a­peu­tics took over full con­trol of Case­bia and its gene edit­ing ef­forts. And there’s been some busy re­cruit­ing go­ing on at Lo­cana to fill out the ex­ec­u­tive team.

The biotech is al­so work­ing di­rect­ly in a hot field, us­ing gene ther­a­py tech to de­liv­er a pro­tein-based gene edit­ing ap­proach to cor­rect­ing ge­net­ic dis­eases with a once-and-done ap­proach. But they don’t have any hu­man da­ta — though that’s not a dis­qual­i­fi­er in this fetid en­vi­ron­ment on Wall Street.

They do, though, have a mouse study they are proud of. The team tout­ed a new pa­per — “The sus­tained ex­pres­sion of Cas9 tar­get­ing tox­ic RNAs re­vers­es dis­ease phe­no­types in mouse mod­els of my­oton­ic dy­s­tro­phy” — back in Sep­tem­ber.

Jim Burns

Lo­can­abio’s lead pro­gram is guid­ed to my­oton­ic dy­s­tro­phy type 1, with pre­clin­i­cal fol­lowups in Hunt­ing­ton’s dis­ease, spin­ocere­bel­lar atax­ia 1, ALS and fron­totem­per­al de­men­tia, where they see spe­cif­ic ge­net­ic trig­gers that can be fixed.

That’s most­ly the kind of sto­ry that sells well on the Street, though there’s no telling what kind of year 2021 will be as new vac­cines promise to bring the pan­dem­ic to a close — in the US and Eu­rope, in any case. Lo­can­abio is be­ing point­ed at Wall Street with the right pro­file, we just need to see now how long the jour­ney takes.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Yanay Ofran (L) and Anat Binur (Ukko)

Leaps by Bay­er backs a pro­tein en­gi­neer­ing start­up tak­ing on Aim­mune — and Nestlé — in peanut al­ler­gy

Little capsules of peanut powder drove Nestlé’s $2.6 billion buyout of Aimmune. Now, with $40 million in new funding, a fledgling biotech is promising to bring a more sophisticated version of that protein therapy that can go much, much further.

Ukko’s goal is two-pronged — with the initial products spanning therapeutic and food — but it’s grounded in the same protein engineering platform, co-founder and CEO Anat Binur told Endpoints News.

As tar­get­ed ther­a­pies get ever more pre­cise, Deer­field un­veils $50M bet on an Har­vard pro­fes­sor's chem­istry in­sights

Behind the seemingly simple concept of targeted cancer therapies is the drug developer’s headache that the target is always changing. Each generation of kinase inhibitors may be ostensibly hitting the same oncogene, but in addition to blocking the wildtype oncogene, they must now also address the mutations that have developed along the way, spurring resistance to current drugs.

The more those target kinases evolve, too, the more they could resemble off-target kinases you don’t want to bind. So each iteration requires more selectivity — sometimes down to differences of a few atoms.

Vincent Sandanayaka (file photo)

UP­DAT­ED: Ex-MD An­der­son chief De­Pin­ho is help­ing launch an­oth­er biotech — and he's stick­ing with fa­mil­iar ground

Years after co-founding SINE-focused Karyopharm and stirring up controversy at MD Anderson, Ronald DePinho is helping uncloak a new biotech targeting solute carrier transporter proteins — and Karyopharm’s former head of chemistry is leading the charge.

Nirogy Therapeutics emerged from stealth mode on Tuesday with a $16.5 million Series A round and plans to hit the clinic by 2022. The financing should be enough to carry the startup’s lead program, a small molecule lactate transport inhibitor, through Phase I, CEO Vincent Sandanayaka said.

Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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