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

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, 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.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

Seagen warns in­vestors against TRC Cap­i­tal’s lat­est 'mi­ni-ten­der of­fer'; BeiGene goes af­ter a new in­di­ca­tion for top PD-1 play­er

TRC Capital, which has selected various biotechs like Vertex and Biogen for the “mini-tender” treatment, jumped back into the game last month with an offer to buy shares in Seagen for $151. The problem, says Seagen, is that price was 4.28% lower than what the stock was selling for at the time they made the offer on Feb. 20, giving TRC a shot at an instant windfall.

So why sell for less than what it’s worth? Seagen notes warnings from regulatory authorities that these offers essentially try to trick investors into believing that they’re being offered a premium for the stock.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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Af­ter three years of courtship (and turn­downs), Mer­ck pounced on the first glance of clin­i­cal da­ta in $1.85B Pan­dion takeover

It’s almost become cliché for biotech executives to talk about the importance of keeping your options open and being prepared to go all the way. But when it comes to negotiating with a giant like Merck, a little patience can indeed go a long way.

Just ask Pandion Therapeutics.

Days ago we already learned that Merck is shelling out $1.85 billion to pick up the biotech and its slate of autoimmune hopefuls. What we didn’t know until the SEC disclosure dropped Thursday is that the deal comes after Pandion turned down two other proposals from Merck over the past three years and held out until the last minute for a sweetened deal.

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