IPO bound? CRISPR Ther­a­peu­tics ups B round to a cool $140M

Gene edit­ing play­er CRISPR Ther­a­peu­tics just added a large­ly un­need­ed $38 mil­lion to its B round with a group of crossover in­vestors in tow — prob­a­bly sig­nal­ing that their S-1 is ei­ther ready to roll or in late-stage edit­ing.

Ver­tex Phar­ma­ceu­ti­cals and Bay­er Glob­al In­vest­ments – a col­lab­o­ra­tor – led the round, which brings the to­tal in the B round to a whop­ping $140 mil­lion.

Franklin Tem­ple­ton In­vest­ments, New Leaf Ven­ture Part­ners, funds ad­vised by Clough Cap­i­tal Part­ners L.P. and Welling­ton Cap­i­tal Man­age­ment L.L.P., and “oth­er undis­closed funds spe­cial­iz­ing in life sci­ences” all par­tic­i­pat­ed.

Savvy in­vestors will note the crossovers in the mix, as well as the fact that CRISPR Ther­a­peu­tics is now the last of the big three gene edit­ing play­ers yet to go pub­lic. Ed­i­tas ($ED­IT), which got off to an ear­ly lead on CRISPR/Cas9 tech, start­ed things off with a suc­cess­ful IPO in Feb­ru­ary, fol­lowed quick­ly by No­var­tis part­ner In­tel­lia ($NT­LA).

Rodger No­vak, CRISPR Ther­a­peu­tics CEO

CRISPR CEO Rodger No­vak told me last spring that his com­pa­ny had enough mon­ey to pay for a two-and-a-half-year run­way on R&D. The added mon­ey will help make a case to gen­er­al in­vestors that the biotech – with an R&D base in Cam­bridge, MA – de­serves the same warm wel­come that was giv­en its two ri­vals.

And just to com­plete the pre-IPO pic­ture, the en­thu­si­as­tic No­vak is stay­ing mum this morn­ing, de­clin­ing any in­ter­views.

It won’t hurt that Sean Park­er’s de­ci­sion to fund the first hu­man study in­volv­ing the gene edit­ing tech just grabbed head­lines around the world. Gene edit­ing may be some years away from de­liv­er­ing its first new ther­a­py to pa­tients, but it’s one of the hottest things in biotech right now.

Biotech IPOs in gen­er­al, mean­while, have not been do­ing as well as pre­vi­ous years. The bloom came off the biotech bub­ble at the end of 2015, forc­ing down the val­ue of shares and mak­ing it hard­er – but not im­pos­si­ble – to make the pub­lic leap.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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