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.”

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.