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.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.

Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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