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.”

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; 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.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Leen Kawas, Athira CEO

Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Christian Hogg, Hutchmed CEO

Hutchmed files for $600M+ IPO in Hong Kong as lead on­col­o­gy drug su­r­u­fa­tinib awaits FDA's good graces

In oncology, a flush of Chinese-developed drugs has the biopharma industry rethinking the poles of power in R&D as the blossoming nation continues to make a name for itself and pick up bundles of cash in the process. Now, as its lead drug faces a pivotal FDA review, the company formerly known as Chi-Med is planting its flag on home soil with a massive public offering.

Hutchmed — recently renamed from Chi-Med, or Hutchison China MediTech — will look to raise $603 million as part of a Hong Kong IPO that serves as a homecoming of sorts for the Chinese-based oncology player, which has listed on Nasdaq since 2016.

Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Eef Schimmelpennink, Lenz CEO (Business Wire)

A re­place­ment for read­ing glass­es? An RA, Ver­sant-backed start­up thinks its eye­drops could solve far­sight­ed­ness

The brain trusts at RA Capital and Versant Ventures have developed an eye for winners in the red-hot biotech space, but every once in a while a candidate comes along with so much potential it makes for an obvious investment. That’s what the partners think they’ve found in San Diego biotech looking to challenge reading glasses for farsightedness.

Lenz Therapeutics launched its rebrand from Presbyopia Therapies with a $47 million Series A and backing from RA and Versant to advance its late-stage-ready small molecule for farsightedness, a market where the biotech thinks it could have a shot at 120 million US patients and 2 billion around the world, the company said.