Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the nat­ur­al process by which cells clean out tox­ic pro­teins be har­nessed to cre­ate po­ten­tial treat­ments for neu­rode­gen­er­a­tive dis­or­ders?

That’s the ques­tion Li­bra Ther­a­peu­tics will be try­ing to an­swer, as the new biotech of­fi­cial­ly launched Wednes­day morn­ing with $29 mil­lion in Se­ries A fi­nanc­ing. The com­pa­ny has three pre­clin­i­cal pro­grams at the ready, with its lead can­di­date tar­get­ing ALS and fron­totem­po­ral de­men­tia. But CEO Isaac Vein­bergs said he hopes to de­vel­op ther­a­pies for a wide range of dis­eases, in­clud­ing Parkin­son’s, Alzheimer’s and Hunt­ing­ton’s.

“All these dis­eases have the com­mon­al­i­ty of the ac­cu­mu­la­tion of tox­ic pro­teins that Li­bra in­tends to ad­dress,” Vein­bergs told End­points News. “On one side pre­vent­ing the pro­duc­tion of these pro­teins, and on the oth­er side in­creas­ing the clear­ance of these pro­teins, there­by en­hanc­ing the home­osta­sis of neu­rons and pro­long­ing their vi­a­bil­i­ty and func­tion.”

Wednes­day’s round was co-led by the Boehringer In­gel­heim Ven­ture Fund (BIVF), Ep­i­darex Cap­i­tal and San­té. Oth­er firms chip­ping in in­clud­ed Yon­jin Ven­ture, Dol­by Fam­i­ly Ven­tures, and Six­ty De­gree Cap­i­tal.

Li­bra’s sci­ence comes from Axxam S.p.A, an Ital­ian re­search or­ga­ni­za­tion pro­vid­ing the new biotech with pro­pri­etary chem­i­cal mat­ter. The cen­tral idea in­volves reg­u­lat­ing cel­lu­lar au­tophagy — that nat­ur­al clean­ing process — to com­bat the buildup of tox­ic pro­teins typ­i­cal­ly seen in neu­rode­gen­er­a­tive dis­eases.

In the lead pro­gram, Li­bra is fo­cus­ing on the loss of func­tion in the C9orf72 gene, which is the most fre­quent ge­net­ic cause of ALS and FTD. Though the en­tire mech­a­nism of the gene is not ful­ly un­der­stood, stud­ies have sug­gest­ed it is in­volved in in­tra­cel­lu­lar traf­fick­ing and au­tophagy in neu­ronal cells, Vein­bergs said. By boost­ing au­tophagy ca­pa­bil­i­ties, Li­bra aims to coun­ter­act the lost ac­tiv­i­ty and ex­punge the ac­cu­mu­lat­ed pro­teins.

“It’s tak­ing out the trash from in­side the cells in or­der to main­tain a bal­anced and clean in­side-the-cell sta­tus,” Vein­bergs said. “There­by the cells can func­tion cor­rect­ly, they don’t have a back­up of these pro­teins that then are detri­men­tal to the func­tion of these neu­rons.”

Most cas­es of ALS are not hered­i­tary, Vein­bergs added, with about 10 to 20 per­cent be­ing fa­mil­ial. About a third of such cas­es are as­so­ci­at­ed with the ex­pan­sion of C9orf72. De­spite com­pris­ing a rel­a­tive­ly small por­tion of all ALS cas­es, it’s the largest risk fac­tor in fa­mil­ial ALS, and since so much about the dis­ease is still un­known, Vein­bergs be­lieves it’s a good start­ing point.

Ul­ti­mate­ly, Vein­bergs wants this pro­gram to im­prove the qual­i­ty of life for ALS pa­tients rather than fo­cus sim­ply on de­lay­ing dis­ease pro­gres­sion. Where Li­bra hopes to dif­fer­en­ti­ate it­self from oth­er ALS treat­ments is in this re­gard, with Vein­bergs say­ing cur­rent­ly ap­proved ther­a­pies on­ly in­ter­vene to­ward the end of the pro­gres­sion.

“What we would love to be able to ac­com­plish is to try to slow down the pro­gres­sion of the dis­ease to have more im­pact in the long run,” Vein­bergs said. “Whether that trans­lates in­to less neu­ronal loss in ear­li­er or mid-to-late dis­ease pro­gres­sion, that leads to, say, pa­tients not go­ing in­to a wheel­chair, not need­ing a res­pi­ra­tor, hav­ing bet­ter mo­toric con­trols in the ear­li­er stages, that to me would be in­creas­ing in the qual­i­ty of life.”

Li­bra’s two oth­er pre­clin­i­cal pro­grams, fur­ther along the pipeline, are di­rect­ed less at the au­tophagy of cells and more at the pro­duc­tion of the neu­ro­tox­ic pro­teins them­selves. The com­pa­ny is still build­ing out its chem­i­cal mat­ter there, though, and still work­ing in an­i­mal mod­els.

It’s al­so too ear­ly to give a time­line for when the lead pro­gram could hit the clin­ic, Vein­bergs said. With the new funds, Li­bra is seek­ing to get there with the lead pro­grams and get close with an­oth­er. But every­thing will be de­pen­dent on the progress Li­bra makes with­in the next six to 12 months.

“We re­al­ize that this is an ex­treme­ly chal­leng­ing area, neu­rode­gen­er­a­tion as a whole,” Vein­bergs said. “Hav­ing very good start­ing points from the chem­istry per­spec­tive, from the ca­pa­bil­i­ties per­spec­tive, I think gives us an edge on try­ing to progress these pro­grams to where peo­ple have not had a lot of suc­cess.”

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, 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.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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An Ar­ray co-founder re-emerges as CEO of a small aca­d­e­m­ic spin­out, look­ing to re­make an old class of can­cer drugs

Tony Piscopio hadn’t worked as a bench scientist in years when, around 2011, he got put in touch with a team at the University of Colorado trying to revitalize an old approach to treating cancer.

Piscopio, who had co-founded Array Biopharma before heading to South Korea to launch a new company, was back in the states, unattached and intrigued. He founded a three-person company with two professors, Xuedong Liu and Gail Eckhardt, and while they worked on the biology side, he returned to his old chemist chair and began drawing up potential compounds on a computer, along with manufacturing processes to make them. Outsourcing companies synthesized or analyzed the results.

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Af­ter three years of courtship (and turn­downs), Mer­ck pounced on the first glance of clin­i­cal da­ta in $1.85B Pan­dion takeover

It’s almost become cliché for biotech executives to talk about the importance of keeping your options open and being prepared to go all the way. But when it comes to negotiating with a giant like Merck, a little patience can indeed go a long way.

Just ask Pandion Therapeutics.

Days ago we already learned that Merck is shelling out $1.85 billion to pick up the biotech and its slate of autoimmune hopefuls. What we didn’t know until the SEC disclosure dropped Thursday is that the deal comes after Pandion turned down two other proposals from Merck over the past three years and held out until the last minute for a sweetened deal.

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