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

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA.