Bio­Marin taps AI up­start Deep Ge­nomics for dis­cov­ery pact; Prax­is says it has a path to lift­ing clin­i­cal hold

Years af­ter dou­ble FDA/EMA re­jec­tions left Bio­Marin’s oligonu­cleotide for Duchenne mus­cu­lar dy­s­tro­phy lan­guish­ing on the shelf, the com­pa­ny is go­ing back to the draw­ing board for new oligonu­cleotide drug can­di­dates — with a healthy dose of ar­ti­fi­cial in­tel­li­gence.

Its part­ner of choice is Deep Ge­nomics, a Toron­to-based shop led by star re­searcher Bren­dan Frey. Paid an undis­closed up­front, their man­date is to iden­ti­fy and val­i­date new tar­get mech­a­nisms in rare dis­eases as well as lead can­di­dates. Bio­Marin will then take over the pre­clin­i­cal work.

Found­ed in 2014 ahead of a new crop of star­tups that promise to boost the prob­a­bil­i­ty of suc­cess in phar­ma, Deep Ge­nomics’ claim to fame is go­ing from tar­get iden­ti­fi­ca­tion to de­clar­ing a can­di­date in 18 months with a plat­form that “does the whole thing.

“I ex­pect that we’ll iden­ti­fy lead can­di­dates for over half of the pro­grams with­in 12 months,” Frey told End­points News. “That’s fast and it means that we can see paths to suc­cess and growth in a mat­ter of a year or two, in­stead of five years like you see with oth­er deals.”

As Deep Ge­nomics po­si­tions it­self as the go-to AI ther­a­peu­tics com­pa­ny for nov­el tar­gets and oligonu­cleotides, Frey added that he and chief busi­ness of­fi­cer Aman­da Kay are look­ing to do an­oth­er deal in the first quar­ter of 2021.

With the dis­cov­ery deal, Bio­Marin is tak­ing a crack at an area where it was se­vere­ly bruised.

The rare dis­ease drug de­vel­op­er once bet $680 mil­lion on Pros­en­sa and dris­apersen, its ex­on-skip­ping an­ti­sense oligonu­cleotide, which ul­ti­mate­ly flopped piv­otal stud­ies and failed to gain ap­provals. In re­cent years it’s turned its fo­cus to gene ther­a­py, with the he­mo­phil­ia A pro­gram Roc­ta­vian lead­ing a pack that al­so fea­tures po­ten­tial once-and-done treat­ments for phenylke­tonuria and hered­i­tary an­gioede­ma. — Am­ber Tong

Prax­is says it has a path to lift­ing clin­i­cal hold

One week af­ter the FDA slapped Prax­is with a stock-dent­ing full clin­i­cal hold, the neu­ro­science up­start says it has a path to get its tri­al up and run­ning.

Prax­is said Tues­day that the agency had asked the com­pa­ny to con­duct more an­i­mal tox­i­col­o­gy stud­ies, par­tic­u­lar­ly for how the drug af­fects fer­til­i­ty, re­pro­duc­tion and em­bryo de­vel­op­ment. Prax­is said they be­lieve their on­go­ing fer­til­i­ty and re­pro­duc­tive tests will an­swer the agency’s ques­tions.

Those stud­ies are set to be com­plet­ed in the first quar­ter of 2021. The FDA al­so asked for changes to the in­ves­ti­ga­tor’s brochure, which sum­ma­rizes all da­ta on a drug for the agency, and to the con­tra­cep­tion re­quire­ments in their study.

If they can man­age to con­vince the FDA, they plan to start the Phase II/III study in ma­jor de­pres­sive dis­or­der in the first half of 2021. In­vestors had pre­vi­ous­ly ex­pressed high hopes for the drug, PRAX-114, in the hard-to-treat con­di­tion, seed­ing the com­pa­ny with a $100 mil­lion launch round and a $190 mil­lion IPO.

For now they’ll wait and see. The stock $PRAX is down 9% pre-mar­ket, from $31.48 to $28.75. — Ja­son Mast

Sketch­ing plans for ac­cel­er­at­ed OK in Fab­ry, Avro­bio touts promis­ing first looks for its oth­er gene ther­a­pies 

Avro­bio’s sec­ond — and third — gene ther­a­py pro­grams have shined in their clin­i­cal de­buts, set­ting a rosy back­drop for the ex­pan­sion of the pipeline.

In ad­di­tion to da­ta on the first pa­tients to be treat­ed with their lentivi­ral ex vi­vo gene ther­a­py for Gauch­er dis­ease and cysti­nosis, the biotech said that the Fab­ry dis­ease da­ta con­tin­ue to “re­flect sus­tained and durable re­sults” 3.5 years af­ter ini­tial dos­ing. That paves the way for po­ten­tial ac­cel­er­at­ed ap­proval.

Ge­off MacK­ay

“Three months post-gene ther­a­py in­fu­sion, the first Gauch­er dis­ease pa­tient’s lev­els of the tox­ic metabo­lite plas­ma lyso-Gb1, as well as plas­ma chi­totriosi­dase, were low­er than the base­line lev­els when the pa­tient was still on en­zyme re­place­ment ther­a­py (ERT),” CEO Ge­off MacK­ay sum­ma­rized in a state­ment. “Ad­di­tion­al­ly, the first pa­tient in the in­ves­ti­ga­tor-spon­sored tri­al for cysti­nosis, now out one year, re­mains off both oral and eye drop cys­teamine and we are thrilled to an­nounce that a third pa­tient has been dosed.”

With Gauch­er dis­ease type 1, the lyso-Gb1 re­duc­tion reg­is­tered at 22% and plas­ma chi­totriosi­dase, a bio­mark­er of the “Gauch­er cells” that lead to in­flam­ma­tion and se­vere or­gan dam­age, dropped by 17% com­pared to the pa­tient’s ERT base­line. She re­mains off ERT, hav­ing dis­con­tin­ued one month be­fore the gene ther­a­py in­fu­sion.

As for cysti­nosis — a con­di­tion char­ac­ter­ized by buildup of the amino acid cys­tine — a 56% de­crease in the num­ber of crys­tals in the pa­tient’s skin sug­gests Avro­bio’s drug helped him pro­duce his own func­tion­al cysti­nosin pro­tein, in turn pre­vent­ing the tox­ic ac­cu­mu­la­tion.

Hav­ing added a new pro­gram for Gauch­er type 3, Avro­bio now has six pipeline as­sets that they say “share tremen­dous syn­er­gies in clin­i­cal de­vel­op­ment, man­u­fac­tur­ing, reg­u­la­to­ry process­es and com­mer­cial­iza­tion.” — Am­ber Tong

Evox part­ners with its old par­ent

Evox, the Take­da and Eli Lil­ly-part­nered ex­o­some start­up, is get­ting a new col­lab­o­ra­tor: its old par­ent.

The Ox­ford spin­out an­nounced Tues­day that it signed a strate­gic col­lab­o­ra­tion with Ox­ford Uni­ver­si­ty. The biotech will team with the Ox­ford-Har­ring­ton Rare Dis­ease Cen­tre to de­vise ways of ap­ply­ing ex­o­somes in the treat­ments of rare dis­ease. The part­ner­ship will last three years.

An in­creas­ing­ly hot space for drug de­vel­op­ment, ex­o­some ther­a­peu­tics in­volve mim­ic­k­ing the cel­lu­lar postal ser­vices tis­sues use to send mes­sages through­out the body. By copy­ing these dis­crete en­velopes, com­pa­nies hope to hit new tar­gets and shut­tle drugs in­to spaces they wouldn’t or­di­nar­i­ly reach. — 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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Pascal Soriot (AP Images)

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.