Ea­ger to tout piv­otal win, Omeros how­ev­er keeps key pa­ra­me­ters shroud­ed

Omeros has brought some stel­lar piv­otal da­ta to the FDA to build a case for its blood clot drug, nar­so­plimab. They just can’t say ex­act­ly how they got to the con­clu­sion yet.

Gre­go­ry De­mop­u­los

Re­port­ing pre­lim­i­nary da­ta from its sin­gle-arm, open-la­bel tri­al, the Seat­tle-based biotech said 56% of the pa­tients — who were ex­pe­ri­enc­ing throm­bot­ic mi­croan­giopa­thy fol­low­ing hematopoi­et­ic stem cell trans­plants — re­ceiv­ing at least one dose of nar­so­plimab achieved com­plete re­spon­der sta­tus. Among the sub­set who re­ceived at least four weeks of dos­ing, 68% met the cri­te­ria.

So what sep­a­rates a re­spon­der from a non-re­spon­der?

That will have to “re­main con­fi­den­tial for com­pet­i­tive busi­ness rea­sons,” the com­pa­ny said in a state­ment, stress­ing that FDA reg­u­la­tors agreed to the “high­ly rig­or­ous” set of re­sponse cri­te­ria.”

Omeros shares $OMER trad­ed up 7.65% to $15.47.

While Omeros had orig­i­nal­ly des­ig­nat­ed “clin­i­cal ac­tiv­i­ty as as­sessed by platelet count” as the pri­ma­ry end­point, the com­pa­ny changed it at the re­quest of the FDA, a spokesper­son told End­points News. The sec­ondary end­points were al­so mod­i­fied.

Rafael Duarte

They in­clude: 100-day sur­vival rates of 65% among those re­ceiv­ing at least one dose, 81% among those who re­ceived at least four weeks of treat­ment, and 93% among the com­plete re­spon­der group; “sub­stan­tial and sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in platelet count, LDH and hap­to­glo­bin”; and in­creased he­mo­glo­bin across all groups.

“The re­sponse rate in this high-risk pop­u­la­tion would be ex­pect­ed to be 10 to 15 per­cent with a 100-day sur­vival rate of less than 20 per­cent,” said Rafael Duarte, who heads the hematopoi­et­ic pro­gram at Uni­ver­si­ty Hos­pi­tal Puer­ta de Hi­er­ro Ma­jada­hon­da in Madrid, in a glow­ing state­ment. “The re­sponse rate and 100-day sur­vival achieved with nar­so­plimab in this tri­al demon­strate an un­prece­dent­ed ef­fect in this con­di­tion.”

Omeros dis­closed that 21% of pa­tients died dur­ing the tri­al “due to caus­es com­mon in stem cell trans­plant” but no oth­ers dis­con­tin­ued for ad­verse events, which ranged from nau­sea, vom­it­ing and di­ar­rhea to hy­pokalemia, neu­trope­nia and fever.

The tri­al was de­signed to en­roll 89 pa­tients, al­though the com­pa­ny didn’t spec­i­fy the fi­nal num­ber in­clud­ed for the analy­sis.

In a pre­vi­ous da­ta cut in­volv­ing 19 pa­tients, Omeros high­light­ed that the drug helped ex­tend me­di­an over­all sur­vival to 347 days, a big im­prove­ment com­pared to the 21 days record­ed in lit­er­a­ture.

The rolling BLA that they ini­ti­at­ed in Oc­to­ber is ex­pect­ed to be com­plet­ed by the first half of next year, ac­cord­ing to CEO Gre­go­ry De­mop­u­los: “the non­clin­i­cal sec­tions have been sub­mit­ted and the da­ta from this tri­al form the ef­fi­ca­cy ba­sis of the ap­pli­ca­tion.”

Pre­vi­ous­ly known as OMS721, nar­so­plimab is dec­o­rat­ed with or­phan drug and break­through ther­a­py des­ig­na­tions from the FDA. If ap­proved, the MASP-2 in­hibitor would be Omeros’ sec­ond drug on the mar­ket.

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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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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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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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.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.