Fi­nal­ly sat­is­fied with Oc­u­lar's man­u­fac­tur­ing set­up, FDA ap­proves eye drug/de­vice Dex­ten­za

Af­ter two FDA re­jec­tions that cul­mi­nat­ed in a man­age­ment shake-up and staff cuts, Oc­u­lar Ther­a­peu­tix’s $OCUL eye drug/de­vice Dex­ten­za has fi­nal­ly got the reg­u­la­to­ry nod, about a month ahead of the FDA’s ex­pect­ed de­ci­sion date.

Fol­low­ing oph­thalmic surgery, pa­tients are typ­i­cal­ly pre­scribed steroids via up to 70 top­i­cal eye drops to treat oc­u­lar pain. In lieu of this com­plex reg­i­men, Dex­ten­za of­fers a full course of post-sur­gi­cal steroid treat­ment with a one-time place­ment of an oph­thalmic in­sert that re­leas­es the cor­ti­cos­teroid dex­am­etha­sone for up to a month fol­low­ing in­ser­tion.

Last year, the Bed­ford, MA-based com­pa­ny’s shares took a big hit when the FDA slapped Oc­u­lar with a sec­ond com­plete re­sponse let­ter, cit­ing man­u­fac­tur­ing de­fi­cien­cies in a pre-NDA in­spec­tion the agency un­der­took months pri­or. The reg­u­la­tor’s first re­jec­tion in 2016 was al­so re­lat­ed to man­u­fac­tur­ing is­sues.

Oc­u­lar, which inked a $300 mil­lion-plus part­ner­ship with Re­gen­eron $REGN in 2016 to de­vel­op a more con­ve­nient for­mu­la­tion for the lat­ter’s flag­ship in­jectable eye drug Eylea among oth­er an­ti-VEGF drugs, has a hit-and-miss record with Dex­ten­za in the clin­ic. The prod­uct has yield­ed pos­i­tive re­sults in di­min­ish­ing post-sur­gi­cal pain, but has came up short in a study for oc­u­lar itch­ing as­so­ci­at­ed with al­ler­gic con­junc­tivi­tis.

The biotech has one prod­uct on the mar­ket ap­proved to close corneal in­ci­sions af­ter cataract surgery, ReSure Sealant, which won the reg­u­la­to­ry nod back in 2014. Al­though Oc­u­lar’s hy­dro­gel-based drug de­liv­ery plat­form is de­signed to pro­vide a com­pet­i­tive ad­van­tage over the cur­rent treat­ment par­a­digm in post-sur­gi­cal oc­u­lar pain and in­flam­ma­tion, adop­tion and/or ac­cess to Dex­ten­za will ul­ti­mate­ly de­pend on pric­ing and re­im­burse­ment.

Raghu­ram Sel­vara­ju

Last month, Eye­Point Phar­ma $EYPT an­nounced that the CMS had hand­ed Dexy­cu, its in­jectable steroid ad­min­is­tered af­ter oc­u­lar surgery to con­trol in­flam­ma­tion, per­ma­nent re­im­burse­ment in the form of a J-code, which is typ­i­cal­ly is­sued to drugs used in of­fice-based pro­ce­dures and not to ther­a­pies as­so­ci­at­ed with cataract surgery, which are re­im­bursed with­in a pack­age price, not­ed WC Wain­wright’s Raghu­ram Sel­vara­ju.

“There has been doubt among in­vestors on how DEX­TEN­ZA can be re­im­bursed af­ter a C-code ex­pires in three years. With a J-code is­sued to DEXY­CU, which is clear­ly a prod­uct that can­not be dis­so­ci­at­ed from cataract surgery, we be­lieve it is high­ly like­ly that DEX­TEN­ZA could al­so se­cure a J-code af­ter reg­u­la­to­ry ap­proval,” Sel­vara­ju wrote in a note.

On Mon­day, the com­pa­ny said it planned to sub­mit an ap­pli­ca­tion for a J-code ahead of the Jan­u­ary 2019 dead­line.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”