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

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Kenneth Galbraith, incoming Zymeworks CEO

Zymeworks re­places half its C-suite, aims to lay off 25% of to­tal work­force as new CEO takes over

New Zymeworks CEO Kenneth Galbraith is aiming to hit the ground running when his tenure officially begins next month, but he’ll be doing so with a much different looking team.

In a lengthy press release outlining the biotech’s 2022 goals, Galbraith said Zymeworks will be laying off at least 25% of its staff over the course of the year. Half of its C-suite will also be replaced immediately as Galbraith looks to remake the company in his image after Ali Tehrani, Zymeworks’ founder and CEO since 2003, stepped down two weeks ago.

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Troy Wilson, Kura CEO

FDA lifts par­tial hold on Ku­ra's Phase Ib AML pro­gram as biotech re­dou­bles mit­i­ga­tion ef­forts

Kura Oncology is clear to resume studies for its early-stage leukemia program after the FDA lifted a clinical hold Thursday afternoon.

Regulators had placed the hold on a Phase Ib study of KO-539, an experimental oral treatment for some genetic subsets of acute myeloid leukemia last November after a patient died while taking the drug. Kura expects to begin enrolling patients again imminently, CEO Troy Wilson told Endpoints News.