News brief­ing: Lo­cus Bio­sciences bags up to $12.1M for CRISPR an­tibac­te­r­i­al; Old Ver­tex drug heads to PhI­II for de­men­tia

In its lat­est ef­fort to spur the de­vel­op­ment of new an­tibac­te­r­i­al drugs, the non-prof­it CARB-X is award­ing Lo­cus Bio­sciences up to $12.1 mil­lion for its CRISPR Cas-3-en­hanced bac­te­rio­phage for se­ri­ous re­cur­ring uri­nary tract in­fec­tions (rUTIs).

The Mor­risville, NC-based biotech will re­ceive up to $2.05 mil­lion ini­tial­ly, fol­lowed by up to $10 mil­lion more if it meets cer­tain mile­stones. Its can­di­date, LBP-KP01, tar­gets Kleb­siel­la pneu­mo­ni­ae, a com­mon cause of an­tibi­ot­ic-re­sis­tant in­fec­tions like rUTIs. The CARB-X funds will sup­port pre­clin­i­cal de­vel­op­ment, po­ten­tial­ly lead­ing up to Phase I.

Paul Garo­fo­lo

“This fund­ing is an­oth­er im­por­tant val­i­da­tion of Lo­cus’ rapid de­vel­op­ment of in­no­v­a­tive CRISPR-Cas3 en­hanced bac­te­rio­phage prod­ucts, en­abling Lo­cus to ad­vance a ro­bust pipeline that in­cludes prod­ucts tar­get­ing four of the most com­mon bac­te­r­i­al pathogens as well as prod­ucts tar­get­ing mi­cro­bio­me-re­lat­ed dis­or­ders such as in­flam­ma­to­ry bow­el dis­ease, phar­ma­ceu­ti­cal re­sponse to im­mune-on­col­o­gy ther­a­pies, in­fec­tions as­so­ci­at­ed with im­mune check­point in­hibitors and col­orec­tal can­cer,” Lo­cus CEO Paul Garo­fo­lo said in a state­ment.

Erin Duffy

LBP-KP01 is a bac­te­rio­phage cock­tail, en­gi­neered with a CRISPR-Cas3 con­struct. The ap­proach “has the po­ten­tial to kill with laser-sharp pre­ci­sion the bac­te­ria caus­ing an in­fec­tion with­out caus­ing dam­age to oth­er cells,” CARB-X R&D chief Erin Duffy said in a state­ment.

Back in Sep­tem­ber, BAR­DA promised Lo­cus up to $77 mil­lion for its LBP-EC01 pro­gram, in­tend­ed to treat re­cur­rent UTIs caused by E. coli. The award start­ed with $11 mil­lion and ex­tends 5 years. If ap­proved, the E. coli and K. pneu­mo­ni­ae cock­tails could treat more than 90% of UTIs, ac­cord­ing to CARB-X.

CARB-X is a glob­al non-prof­it part­ner­ship led by Boston Uni­ver­si­ty to sup­port the de­vel­op­ment of prod­ucts that tar­get an­tibi­ot­ic-re­sis­tant bac­te­r­i­al in­fec­tions. Since 2016, it’s giv­en out 72 awards to­tal­ing $257 mil­lion, with the po­ten­tial for more funds if mile­stones are met. It plans on pour­ing up to $480 mil­lion to­tal in non-di­lu­tive fund­ing in­to an­tibac­te­r­i­al de­vel­op­ment by 2022. — Nicole Feud­is

An old Ver­tex drug heads to Phase III for de­men­tia

EIP Phar­ma has re­port­ed new pos­i­tive da­ta for an old Ver­tex drug and hopes to send it in­to Phase III next year.

The com­pa­ny was eval­u­at­ing ne­flamapi­mod, a p38 ki­nase in­hibitor in-li­censed from Ver­tex in 2014, in a Phase II study for Lewy body de­men­tia. The study met its pri­ma­ry end­point, with pa­tients on the drug show­ing a greater im­prove­ment in cog­ni­tion than pa­tients on place­bo, per an at­ten­tion and ex­ec­u­tive func­tion as­sess­ment.

EIP said the treat­ment ef­fect emerged by the fourth week and main­tained lev­els through the 16-week study pe­ri­od. The p-val­ue on the pri­ma­ry end­point was 0.015.

There were no se­ri­ous drug-re­lat­ed side ef­fects. The study it­self en­rolled 91 pa­tients and ran­dom­ized them even­ly in­to the drug arm and place­bo arm. – Max Gel­man

Ver­ri­ca her­alds suc­cess in gen­i­tal warts

The FDA just re­buffed Ver­ri­ca’s bid for their first ap­proved drug, hand­ing them a CRL for a drug/de­vice top­i­cal treat­ment known as VP-102 in mol­lus­cum con­ta­gio­sum. But the com­pa­ny is try­ing to ex­pand the drug’s po­ten­tial for when­ev­er they can get it across the fin­ish line.

On Tues­day, they an­nounced the drug had cleared an 87-per­son Phase II for ex­ter­nal gen­i­tal warts. In the study, 35% of pa­tients on the drug saw their warts clear, com­pared with 2% of pa­tients who re­ceived the place­bo (tech­ni­cal­ly known as a “ve­hi­cle” in der­ma­to­log­i­cal stud­ies).

With a Phase III in com­mon warts al­ready sched­uled, that sets up Ver­ri­ca to have VP-102 in late stage for three in­di­ca­tions. Still, they will have to deal with the is­sues they say the FDA flagged in the CRL, in­clud­ing hu­man fac­tors study and CMC is­sues. — Ja­son Mast

Is­raeli life sci­ences fund rais­es new $300 mil­lion round

Pere­grine Ven­tures, an Is­raeli life sci­ences fund, an­nounced to­day that it had se­cured a new $300 mil­lion round, more than triple the size of its pre­vi­ous pot.

The firm fo­cus­es on $20-$30 mil­lion in com­pa­nies ei­ther near­ing an M&A or an IPO. Much of their fo­cus so far has been on med tech and health tech, but they’ve had sev­er­al biotech place­ments and plan to con­tin­ue those with the newest fund. That in­cludes in­vest­ments in Ot­ic Phar­ma (now known as Novus) and Ra­ziel Ther­a­peu­tics.

“With the re­newed fo­cus on biotech, health, and life sci­ence in the wake of the Covid-19 cri­sis, the po­ten­tial of Pere­grine Growth is great,” co-founder and man­ag­ing part­ner Eyal Lif­schitz said in a state­ment. – Ja­son Mast

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.