With a po­ten­tial cure for lyso­so­mal stor­age dis­or­ders in hand, Avro­bio rais­es $60M to back gene ther­a­py pipeline

In gene ther­a­py, re­spons­es from just one or two pa­tients can have a huge in­flu­ence on an­a­lysts’ opin­ions. Just ask the ex­ec­u­tive team at blue­bird bio, which trum­pets each pa­tient’s progress with de­light — or cal­i­brates a new de­vel­op­ment strat­e­gy when smiles turn to frowns.

So it’s no won­der that Avro­bio is so proud of its very first pa­tient.

Ge­off MacK­ay

At a sci­en­tif­ic con­fer­ence in Japan last fall, re­searchers re­viewed the case of a pa­tient with Fab­ry dis­ease whose blood lev­els of the en­zyme a-Gal A were near ze­ro at the start of treat­ment. With­in 45 days of re­ceiv­ing their gene ther­a­py AVR-RD-01, the pa­tient’s plas­ma a-Gal A ac­tiv­i­ty in­creased in­to the nor­mal range, they said. And 6 months lat­er that hadn’t changed.

In oth­er words, they got the re­sponse they want­ed and Pa­tient 1 cleared the first hur­dle on dura­bil­i­ty. All sys­tems go for a young com­pa­ny that is out to cre­ate a once-and-done fix.

At­las seed­ed the com­pa­ny — Bruce Booth is the proud chair­man — and then launched it in the spring of 2016 with a $25 mil­lion A round that backed the work of 15 staffers. Over the last few months, its staff has dou­bled. And more hires are be­ing planned for lat­er this year.

That up­beat start has helped in­spire a hefty $60 mil­lion round to back the mid-stage work that will soon be­gin for a com­pa­ny that has laid out a broad plan to fol­low some of the lead­ers in lyso­so­mal stor­age dis­or­ders with the promise of a cure that could one day rel­e­gate the cur­rent stan­dard of care for these ail­ments to an his­toric foot­note.

Bruce Booth

The Se­ries B round was co-led by Cor­morant As­set Man­age­ment and Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), and in­clud­ed Ais­ling, Brace Phar­ma Cap­i­tal, Even­tide, Morn­ing­side and Leerink Part­ners, along with ex­ist­ing in­vestors At­las Ven­ture, SV Health In­vestors and Clarus Ven­tures.

Get­ting an in­vest­ment group like Leerink in your round — not an every­day event in biotech — might make you think that there’s an IPO in the off­ing, es­pe­cial­ly with 2018 look­ing like an es­pe­cial­ly good year to take a biotech pub­lic. Gene ther­a­py pi­o­neers have al­so earned a lot of re­spect on Nas­daq, even with some tur­bu­lence for every set­back.

But Avro­bio CEO Ge­off MacK­ay is cir­cum­spect about any near-term of­fer­ings.

The com­pa­ny’s fo­cus this year is on kick­ing off a crit­i­cal Phase II tri­al for Fab­ry dis­ease, he says, to run par­al­lel to the Phase I.

There are no time­lines for the tri­als on pub­lic dis­play, though.

In gene ther­a­py, even af­ter the first US ap­proval, all com­pa­nies in the field tend to get striped with the same brush. MacK­ay’s de­sire now is to make sure that every­one knows that Avro­bio is in the lentivi­ral camp, where you’ll find lead­ers like blue­bird bio.

“AAV is a dif­fer­ent vec­tor,” he says. “It’s not fair to ex­trap­o­late to lentivi­ral.”

And at that point I hadn’t even men­tioned the James Wil­son study sound­ing the alarm around high-dose gene ther­a­pies us­ing AAV tech ear­li­er in the week.

A break­through with a gene ther­a­py would un­doubt­ed­ly have a huge im­pact on the pa­tients who de­pend on steady in­fu­sions of some high-priced ther­a­pies.

“We’re very re­spect­ful of the con­tri­bu­tion Gen­zyme and Shire’s made,” says MacK­ay. “Twen­ty years ago these pa­tients had no op­tions, then the pa­tients had an op­tion. When we look at it with fresh eyes, we see a sig­nif­i­cant op­por­tu­ni­ty to im­prove on the stan­dard of care.”

“Pa­tients now re­ly on bi­week­ly in­fu­sions of en­zymes, with se­vere chal­lenges to cost, com­pli­ance and qual­i­ty of life,” adds the CEO. “When ERT suc­ceeds it slows the pro­gres­sion of dis­ease but doesn’t halt it. The cen­tral premise is to de­liv­er via a one-time in­fu­sion for 24-7 pro­tec­tion, which we hope will halt the pro­gres­sion rather than just slow it down.”

Avro­bio has a long ways to go, but it has big plans for when it ar­rives.

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.

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.

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.