No­var­tis finds its next mass-mar­ket oph­thal­mol­o­gy drug can­di­date at a small vir­tu­al biotech with a glob­al view

Like me, you may nev­er have heard about Lu­bris Bio­Phar­ma be­fore to­day. But No­var­tis has. And that is by far more im­por­tant.

To­day the phar­ma gi­ant reached out and ex­er­cised an op­tion on a re­com­bi­nant lu­bri­cant Lu­bris drew from na­ture’s med­i­cine chest and ap­plied for dry eye dis­ease. There are no terms, no big biobucks on the record (though some­one told the Boston Busi­ness Jour­nal that the pack­age was worth up to $1 bil­lion). But the deal marks a def­i­nite tran­si­tion phase for lit­tle Lu­bris as it shifts its fo­cus on the same ther­a­py to a dif­fer­ent set of ail­ments in mind, while No­var­tis eyes an ad­di­tion to its late-stage pipeline.

Lu­bris’ big idea was to take a nat­ur­al lu­bri­cant — lu­bricin — that is found just about wher­ev­er two tis­sues in the bod­ies meet and in­dus­tri­al­ize it, turn­ing it in­to a re­place­ment pro­tein ther­a­py dubbed ECF843. Last year they test­ed it in a small Phase II dry eye study with 40 mod­er­ate to se­vere pa­tients, putting it up against sodi­um hyaluronate (HA), which fig­ures promi­nent­ly in a slew of over-the-counter ar­ti­fi­cial tear prod­ucts like Blink.

Here’s what they found in hu­mans:

Lu­bricin demon­strat­ed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments against HA in the fol­low­ing ob­jec­tive signs of dry eye: corneal flu­o­res­cein stain­ing (OD/OS: 43.8%, 50.0%, vs. 26.5%, 23.3%, p<.0398, p<.0232), TF­BUT (p<.010), eye­lid ery­the­ma (p<0.004), con­junc­ti­val ery­the­ma (p<.0013).

Symp­toms of the dis­ease dropped 70% from base­line and CEO Ed Tru­itt tells me that when the study tran­si­tioned to self-ap­pli­ca­tion Lu­bricin pa­tients used much less of it to get bet­ter re­sults.

Ed Tru­itt

Hav­ing that hu­man da­ta, as op­posed to pre­clin­i­cal an­i­mal re­sults, is what re­al­ly cap­tured No­var­tis’ at­ten­tion, says Tru­itt. They struck the op­tion deal, qui­et­ly, last year. And when the Big Phar­ma play­er ex­er­cised the op­tion to­day, No­var­tis was care­ful to high­light how this drug fit in­to its pipeline for front-of-the-eye ther­a­pies — a mass mar­ket with an eye on mil­lions of pa­tients — as well as its deal­mak­ing strat­e­gy, which in­clud­ed buy­ing En­core Med­ical for the treat­ment of pres­by­opia.

Tru­itt him­self is based in Seat­tle, one of four full-time staffers. There’s an of­fice in Fram­ing­ham, MA. Sci­en­tif­ic founders are Dr. Greg Jay of Brown, Tan­nin Schmidt of the Uni­ver­si­ty of Cal­gary and David Sul­li­van of Mass Eye and Ear and Ben Sul­li­van for the work he did while at UCSD. Their fund­ing so far has most­ly come from an­gels and high net worth in­di­vid­u­als, which is why you haven’t been hear­ing about any ven­ture rounds. But it’s been enough.

The big ad­van­tage of any vir­tu­al com­pa­ny is keep­ing costs low. You can stretch a buck fur­ther, turn­ing to con­tract staffs in man­u­fac­tur­ing and re­search.

The down side?

If you don’t have your own lab, where the team is com­mit­ted and push­ing hard, burn­ing the mid­night oil, you have con­tract groups do­ing specif­i­cal­ly what they have been told to do, says Tru­itt. In­stead of man­ag­ing the staff, you wind up man­ag­ing dozens of work­ers at the ven­dors.

“Things can go a lit­tle slow­er,” says Tru­itt. “They don’t think out­side the box.”

On the oth­er hand, now that No­var­tis has put mon­ey in­to the com­pa­ny, fol­low­ing a Eu­ro­pean deal with Mi­lan-based Dom­pé Group, Tru­itt has a “sub­stan­tial run­way” to go af­ter the next set of non-eye tar­gets, says the CEO. That will start with dry mouth, maybe a side ef­fect of head and neck can­cer ther­a­py that de­stroys the sali­vary glands. In­ter­sti­tial cys­ti­tis, re­coat­ing the dam­aged blad­der lin­ing, is on the short list.

But Tru­itt is al­so hap­py to note that his pro­tein ther­a­py could be used in place of HA for a long list of prod­ucts. And maybe more part­ners will line up for these oth­er in­di­ca­tions as they con­tin­ue to work on new hu­man stud­ies.

It’s time to push on to the next big thing at lit­tle Lu­bris.


Im­age: Shut­ter­stock

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.

Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

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 weeks 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.