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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Tillman Gerngross (Adagio)

Till­man Gern­gross' Covid-19 an­ti­body moon­shot scores $336M with the help of new ace CFO. Is an IPO next?

Less than a year into its existence, serial biotech entrepreneur Tillman Gerngross’ antibody play Adagio has raced ahead into a pivotal trial for its lead drug for Covid-19 on the back of some very promising preclinical data. Now, crossover investors led by Peter Kolchinsky at RA are rolling up the Brinks truck — and that could spell an IPO in the offing for Adagio.

Adagio has bagged $336 million as part of a Series C round led by RA Capital to advance lead single-shot antibody ADG20 through a pivotal Phase I/II/III trial for the treatment of mild to moderate Covid-19 patients at high risk of infection, the biotech said Monday.

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UP­DAT­ED: New Kaiser analy­sis shows how lim­it­ing price ne­go­ti­a­tions to tar­get­ed drugs may bet­ter fo­cus up­com­ing leg­is­la­tion

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

Biotech's IPO raise ap­proach­es $5.5B as Nas­daq con­tin­ues to prove fruit­ful with 2 de­buts and three new fil­ings

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

It was another busy week in the biotech IPO market as the second quarter continues to churn out significant investment into the sector.

Recursion led the way with a $436 million raise on Friday, pricing its IPO at $18, the high end of its range. Our own John Carroll went in depth on that raise over the weekend. Also on Friday, preclinical cancer biotech Biomea Fusion debuted with a $153 million raise priced at its own high end of $17 per share. The two companies helped push the combined IPO raise for 2021 to nearly $5.5 billion.

When is a drug re­al­ly a de­vice? Court knocks down FDA ap­peal in try­ing to sort that grey area

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

Q1: A flood of in­vestor cash drove biotech's num­bers to new record highs, and the tor­rent of cash is mov­ing up­stream fast

If you thought biotech was booming last year, wait until you get a load of the numbers from Q1 2021.

On virtually every level, with one exception, the money engine was working around the clock in the first 3 months of this year. Venture capital has reached such a fever peak that the average B round now weighs in at an average mega-weight value of $100 million. The money flow is also finding its way to the mouth of the R&D river, where discovery work now merits the big bucks instead of cautionary seed funds.

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Jami Rubin (EQRx)

Ja­mi Ru­bin, once fa­bled for grilling bio­phar­ma ex­ecs, de­camps to head fi­nance at drug pric­ing dis­rupter

As Goldman Sachs’ top pharmaceutical analyst, Jami Rubin was known for asking the tough questions. Now, as she takes the lead on EQRx’s mission to rewrite the rules of drug pricing, we’ll see how good her answers are.

Rubin made the jump to biotech on April 5, becoming EQRx’s new CFO, the company said Monday. She’s coming from PJT Partners, where she’s been a partner providing strategic guidance for biotech and pharmaceutical companies for the last couple years. With EQRx’s recent $500 million Series B round in the books, it wouldn’t be a surprise if she was already lining up a public debut.

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Seagen gets Au­gust PDU­FA for Pad­cev ex­pan­sions; Adap­tate pulls in new cash for gam­ma delta T cell an­ti­bod­ies

Seagen is riding the wave of two new priority reviews straight to the FDA.

The Bothell, WA-based biotech and their partners at Astellas announced Monday that two supplemental BLAs for Padcev had been accepted by US regulators. FDA has set Aug. 17 as the PDUFA date for the reviews.

“With our recent regulatory submissions, we intend to provide the highest level of clinical evidence supporting Padcev use — overall survival data from a randomized Phase III trial — and expand availability in multiple countries where there is unmet medical need,” said Astellas oncology chief Andrew Krivoshik.