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

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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