FDA ex­perts shrug off retinopa­thy con­cerns, of­fer (near) unan­i­mous thumbs up for No­vo Nordisk's semaglu­tide

An FDA pan­el of out­side ex­perts to­day vot­ed al­most unan­i­mous­ly to en­dorse No­vo Nordisk’s next big di­a­betes/obe­si­ty drug semaglu­tide, like­ly set­ting up a near-term ap­proval for one of this year’s top block­busters in the pipeline. There was one ab­sten­tion and ze­ro op­po­si­tion to the key ques­tion.

No­vo Nordisk is a ma­jor play­er in di­a­betes and pre­dictably ran a thor­ough late-stage ef­fort to nail down their drug’s abil­i­ty to low­er HbA1c — a crit­i­cal mea­sure for the con­trol of the dis­ease. In the process, though, they al­so care­ful­ly tracked in­ci­dents of di­a­bet­ic retinopa­thy — dam­age to the blood ves­sels in the eye, which can lead to blind­ness — spot­light­ing what would be­come a big is­sue for its sci­en­tists on Wednes­day.

Ac­cord­ing to No­vo Nordisk, no one else has done sim­i­lar eye ex­ams as re­searchers de­cid­ed to do in their big SUS­TAIN 6 out­comes study. In their case, the sci­en­tists main­tained that the drug wasn’t di­rect­ly re­spon­si­ble for in­ci­dents of retinopa­thy, but rather pinned the blame for the cas­es on the sud­den drop in HbA1c which the drug was re­spon­si­ble for. And that risk could be man­aged, the com­pa­ny says, through the right warn­ing la­bel to alert pa­tients who are at risk.

The FDA, while of­fer­ing a clear nod for the drug’s ef­fi­ca­cy pro­file, made these retinopa­thy cas­es the cen­tral fea­ture of the pan­el dis­cus­sion, ask­ing the ex­perts to ei­ther sign off on the ad­verse safe­ty is­sue as some­thing that could be man­aged or stop the drug now un­til more da­ta could be gath­ered.

Eli Lil­ly has a lot rid­ing on the fi­nal out­come for No­vo Nordisk. In its last big Phase III tri­al, SUS­TAIN 7, re­searchers pit­ted semaglu­tide against Lil­ly’s up-and-com­ing GLP-1 drug Trulic­i­ty, and beat it. An ag­gres­sive mar­keter, No­vo will go toe-to-toe with Lil­ly in the glob­al di­a­betes mar­ket if it gets the chance.

No­vo is al­so de­vel­op­ing semaglu­tide specif­i­cal­ly for obe­si­ty, a mar­ket that has de­fied sev­er­al re­cent at­tempts by small biotechs with on­ly rel­a­tive­ly mod­est amounts of weight loss to boast about. And there is al­so a pro­gram un­der­way in NASH.

Pegged as a po­ten­tial $2.2 bil­lion drug in 5 years in one re­cent as­sess­ment, No­vo ex­ecs dur­ing their Q2 call re­viewed the Phase II obe­si­ty da­ta they had nailed down in June, out­lin­ing plans for a loom­ing Phase III that they be­lieve can show their GLP-1 drug can knock off up to around 15% of an obese per­son’s weight rel­a­tive to place­bo.

If semaglu­tide gets a clean bill of health from reg­u­la­tors, as ap­pears like­ly, No­vo can start test­ing those fore­casts.

It’s al­so worth not­ing that an ap­proval here would shove up the FDA’s record for 2017 to 35 or more, putting it on track to re­turn to the strong ap­proval rate for new drugs reg­u­la­tors scored in 2015, be­fore see­ing a big dropoff in 2016. The FDA has al­ready reg­is­tered a record num­ber of OKs on the gener­ics front, as new com­mis­sion­er Scott Got­tlieb fol­lows through on his list of goals at the FDA.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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