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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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FDA+ roundup: Ad­comm date set for Cy­to­ki­net­ics heart drug; New gener­ic drug guid­ance to re­duce fa­cil­i­ty de­lays

The FDA on Wednesday set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, meaning regulators aren’t likely to meet the Nov. 30 PDUFA date that was previously set.

The drug, known as omecamtiv mecarbil, read out its first Phase III in November 2020, hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as the key to breaking into the market, failing to significantly differ in reducing cardiovascular death from placebo.

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Af­ter Covid set­back, Val­ne­va lines up $100M for Pfiz­er-al­lied Ly­me dis­ease PhI­II

Valneva has secured €102.9 million (around $99.9 million USD) in a share offering to push forward its Pfizer-partnered Lyme disease vaccine and a jab for chikungunya that awaits an FDA decision.

The French vaccine maker largely snagged the near $100 million from Deep Track Capital and local state-owned Bpifrance, the company said Tuesday night. The capital injection is nearly equal to the amount Pfizer paid to nab equity in the company earlier this summer as part of the duo’s vaccine tie-up.

Valitor CEO Steven Lo (L) and president and CSO Wesley Jackson

A dozen years in the mak­ing, a UC Berke­ley spin­out nabs funds to take on the eye

Largely funded by government grants for the better part of its first decade, a UC Berkeley spinout has secured a new CEO and the funds to take its research into the clinic in early 2024.

The biotech, named by one of the co-founder’s daughters and originally scrapped together with NIH funds in the aftermath of the 2008-09 financial crisis, is also on a mission to upend the treatment of wet age-related macular degeneration, or AMD, with an injectable drug that it claims could be more durable than the “800-pound gorilla” in the room, Genentech’s Lucentis and Regeneron/Bayer’s Eylea.

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Car­olyn Bertozzi (Illustration: Assistant editor Kathy Wong for Endpoints News)

Car­olyn Bertozzi, re­peat biotech founder and launch­er of a field, shares in chem­istry No­bel win

Carolyn Bertozzi, predicted by some to become a Nobel laureate, clinched one of the world’s top awards in the wee hours of Wednesday, winning the Nobel Prize in Chemistry alongside a repeat winner and a Copenhagen researcher.

The Stanford professor, Morten Meldal of University of Copenhagen and 2001-awardee K. Barry Sharpless of Scripps shared the prize equally. The Nobel is sometimes split in quarters and/or halves.