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

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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How the bio­phar­ma in­dus­try is help­ing to pay for the bi­par­ti­san in­fra­struc­ture bill

Senators on Sunday finalized the text of a massive, bipartisan infrastructure bill that contains little that might impact the biopharma industry other than two ways the legislators are planning to pay for the $1.2 trillion deal.

On the one hand, senators are seeking to further delay a Trump-era Medicare Part D rule related to drug rebates, this time until 2026. Senators claim the rule could end up saving about $49 billion, but the PBM industry has attacked it as it would remove rebates from a safe harbor that provides protection from federal anti-kickback laws. The pharmaceutical industry, however, is in favor of the rule and opposes this latest delay as it continues to point its finger at the PBM industry for the rising cost of out-of-pocket expenses.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; 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.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.