A fast-mov­ing en­try in the gene edit­ing race is speed­ing to the clin­ic, fu­eled by an $83.5M round

The last time I talked to Arthur Tzian­a­bos a lit­tle more than a year ago, the CEO at Ho­mol­o­gy Med­i­cines had a team of about 10 and a new ap­proach to in vi­vo gene trans­fer and gene edit­ing that was still in pre­clin­i­cal liftoff mode. To­day, Tzian­a­bos is whip­ping the cov­er off a whop­ping $83.5 mil­lion ven­ture round with some A-lis­ter sup­port so his team of 40-plus can take their work in­to hu­mans for the first time.

“We re­al­ly have made a tremen­dous amount of progress,” Tzian­a­bos tells me by way of catch­ing up.

There’s a lead in­di­ca­tion now — an in­born er­ror of me­tab­o­lism in­volv­ing the liv­er is as spe­cif­ic as he wants to get right now — with a long list of pre­clin­i­cal projects in CD34 dis­eases, cys­tic fi­bro­sis, CNS dis­eases, Duchenne mus­cu­lar dy­s­tro­phy and so on. When you’re a pre­clin­i­cal biotech work­ing on the foun­da­tion for a plat­form tech that seeks to ed­it and cor­rect genes us­ing your own ade­no-as­so­ci­at­ed virus vec­tors, you can think about what you want to keep and what you want to part­ner.

Ho­mol­o­gy is one of the up­starts trav­el­ing one step be­hind the pi­o­neer­ing crews that boot­ed up gene edit­ing in a big way. That group in­cludes Ed­i­tas, CRISPR Ther­a­peu­tics and In­tel­lia — which all made vir­tu­al­ly overnight de­buts on­to the pub­lic mar­kets with a set of IPOs that at­tract­ed a tremen­dous amount of in­vestor at­ten­tion — and which sagged as the long clin­i­cal road to an ac­tu­al ther­a­py came in­to view.

These days, the pi­o­neers al­so are spend­ing a sig­nif­i­cant amount of time wran­gling over patents as they work to get in­to the clin­ic. It’s no easy task birthing a new tech­nol­o­gy, as Ed­i­tas found out when it had to de­lay its time­line re­cent­ly in or­der to work through a de­lay on the man­u­fac­tur­ing side.

Tzian­a­bos and his team are learn­ing from the lead­ers, and may al­so be mov­ing faster to­ward the clin­ic than the out-front ri­vals know.

“We want to be there in a short pe­ri­od of time,” says the CEO about hu­man tri­als. (No, he isn’t be­ing more spe­cif­ic than that.) He has plen­ty of cash now to get through a proof-of-con­cept tri­al, and a chance to sign up a part­ner or two to help ex­pand the pipeline work more quick­ly.

Tzian­a­bos gives his Bed­ford, MA-based biotech high scores for their pre­clin­i­cal ef­fort on fine tun­ing gene trans­fer and edit­ing, start­ing with a page out of the book of na­ture on the way DNA is re­paired to point to an in vi­vo ap­proach that can be a sim­pler and more di­rect means of po­ten­tial­ly cur­ing ge­net­ic dis­eases.

But he’s al­so start­ing to look at what needs to hap­pen on man­u­fac­tur­ing to pre­vent fu­ture foul-ups.

“I think peo­ple got a lit­tle over their skis on that point,” he says about the field. And he has built up in­ter­nal and ex­ter­nal man­u­fac­tur­ing ops.

“CMC, bi­o­log­ics man­u­fac­tur­ing is com­pli­cat­ed any­way,” says Tzian­a­bos, point­ing to the cri­sis that Gen­zyme went through be­fore Sanofi ac­quired the com­pa­ny. The new gene edit­ing com­pa­nies are com­ing straight out of acad­e­mia, he adds, and that kind of tech al­ways takes time to in­dus­tri­al­ize.

At Ho­mol­o­gy, says the CEO, “that was one of our main fo­cus­es right out of the gate.”

Add it all up, he says, and “I would put us not too far be­hind where these guys (the pi­o­neers) are. That’s what at­tract­ed this team to the tech­nol­o­gy.”

It is al­so what at­tract­ed Deer­field, which led the round. New in­vestors in­clude Fi­deli­ty Man­age­ment and Re­search Com­pa­ny, HBM Health­care In­vest­ments, Mav­er­ick Ven­tures, No­var­tis, Rock Springs Cap­i­tal, Vi­da Ven­tures, Vi­vo Cap­i­tal and Alexan­dria Ven­ture In­vest­ments. Ex­ist­ing in­vestors 5AM Ven­tures, ARCH Ven­ture Part­ners and Temasek al­so jumped back in.

As of now, Ho­mol­o­gy has raised a to­tal of $127 mil­lion in a lit­tle more than a year, of­fer­ing an­oth­er ex­am­ple of the kind of funds that are be­ing spent to pur­sue a rad­i­cal new tech­nol­o­gy. It’s still ear­ly stages, with years of clin­i­cal work ahead, but Ho­mol­o­gy is mov­ing fast.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Evotec CEO Werner Lanthaler, File Photo

Ox­ford, Evotec ramp up LAB10x with AI ex­perts at Sen­syne — fo­cused on biotech spin­outs

Ox­ford is al­ly­ing it­self with Evotec and ar­ti­fi­cial in­tel­li­gence out­fit Sen­syne Health to ramp up some new biotech spin­outs while look­ing to “ac­cel­er­ate da­ta-dri­ven drug dis­cov­ery and de­vel­op­ment.”

The big idea here is that Ox­ford sci­en­tists — some of the best drug hunters in the world — can uti­lize Sen­syne’s AI plat­form for their work, re­ly­ing on the chemists and hands-on de­vel­op­ers at Evotec to push ahead to a crit­i­cal proof of con­cept mo­ment. And they’ll do it through a project leader called LAB10x, which gets £5 mil­lion over the next three years to fund the work.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Suf­fer­ing No­var­tis part­ner Cona­tus is pack­ing it in on NASH af­ter a se­ries of un­for­tu­nate tri­al events

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Dean Hum. Nasdaq via YouTube

Gen­fit goes to Chi­na with a deal worth up to $228M for NASH drug

Fresh off the high of its Nas­daq IPO de­but, and the low of com­par­isons to Cymabay — whose NASH drug re­cent­ly stum­bled — Gen­fit on Mon­day un­veiled an up to $228 mil­lion deal with transpa­cif­ic biotech Terns Phar­ma­ceu­ti­cals to de­vel­op its flag­ship ex­per­i­men­tal liv­er drug — elafi­bra­nor — in Greater Chi­na.

The deal comes more than a week af­ter Gen­fit $GN­FT is­sued a fiery de­fense of its dual PPAR ag­o­nist elafi­bra­nor, when com­peti­tor Cymabay’s PPARδ ag­o­nist, se­ladel­par, fiz­zled in a snap­shot of da­ta from an on­go­ing mid-stage tri­al. The main goal at the end of 12 weeks was for se­ladel­par to in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er fat con­tent, but da­ta showed that pa­tients on the place­bo ac­tu­al­ly per­formed bet­ter.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.