Gene edit­ing star CRISPR Ther­a­peu­tics files for $90M IPO

Em­manuelle Char­p­en­tier. Cred­it: Bian­ca Fioret­ti, Hall­bauer & Fioret­ti

In one of the most high­ly an­tic­i­pat­ed biotech IPOs of the year, CRISPR Ther­a­peu­tics has filed its S-1 out­lin­ing its plans to raise $90 mil­lion from an ini­tial pub­lic of­fer­ing.

CRISPR Ther­a­peu­tics is one of a hand­ful of the most promi­nent gene edit­ing com­pa­nies in the world, found­ed by Em­manuelle Char­p­en­tier, who’s cred­it­ed as one of the pi­o­neer­ing sci­en­tists be­hind CRISPR/Cas9 tech, which promis­es to open up a new chap­ter in drug de­vel­op­ment.

Just days ago CRISPR Ther­a­peu­tics be­gan ramp­ing up Case­bia, a gene edit­ing joint ven­ture with Bay­er which start­ed out with $335 mil­lion in fund­ing to back the next five years of R&D work. Bay­er has agreed to buy $35 mil­lion of shares in the IPO, ac­cord­ing to the S-1. Ver­tex, mean­while, paid $105 mil­lion in cash to start its col­lab­o­ra­tion. And while CRISPR Ther­a­peu­tics is based in Switzer­land, its re­search head­quar­ters is in Cam­bridge, MA, just like Case­bia’s.

Top pro­grams at CRISPR Ther­a­peu­tics in­clude new ther­a­pies for be­ta-tha­lassemia and sick­le cell dis­ease.

The com­pa­ny has been one of the best fund­ed in the in­dus­try. Ac­cord­ing to the S-1, CRISPR Ther­a­peu­tics end­ed June with $246.8 mil­lion in cash. And with the IPO, it ex­pects to have enough cash to fund op­er­a­tions over the next 24 months.

CRISPR Ther­a­peu­tics CEO Rodger No­vak

But that $90 mil­lion cit­ed in the S-1 may just be a place­hold­er for a much larg­er fig­ure. Both Ed­i­tas $ED­IT and In­tel­lia $NT­LA en­joyed suc­cess­ful IPOs this year, star­ing down a tough mar­ket and win­ning over in­vestors at­tract­ed to gene edit­ing’s hot rep.

Much of that new mon­ey will con­tin­ue the biotech’s pre­clin­i­cal work. The biotech ex­pects to launch its first clin­i­cal ef­fort for he­mo­glo­binopa­thy pro­grams in late 2017.

The biggest share­hold­ers are Ver­sant, with 21% of the stock, Cel­gene Alpine In­vest­ment with 12% and NEA and SR One (GSK) with about 10% each.

CRISPR Ther­a­peu­tics, head­ed by CEO Rodger No­vak, has been play­ing its cards close to its vest in re­cent months,  but it sent a clear sig­nal about the up­com­ing IPO in June, when it added a com­plete­ly un­need­ed $38 mil­lion to its last ven­ture round, tak­ing it to a whop­ping $140 mil­lion. Biotechs that go pub­lic these days need plen­ty of cash in the bank and in­sid­er in­vestors lin­ing up to buy shares.

CRISPR Ther­a­peu­tics would seem to be go­ing in­to this IPO with all the right box­es checked.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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