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.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Radek Spisek, Sotio CEO (Cellestia)

A qui­et Czech biotech bags $315M to dri­ve its blos­som­ing can­cer pipeline through the clin­ic

In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.

After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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