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.

Paul Hudson, Getty Images

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

Parkin­son's trans­plants emerge as stem cell pi­o­neer Jeanne Lor­ing joins race

Jeanne Loring hadn’t studied Parkinson’s in 22 years when she got an email from a local neurologist.

The neurologist, Melissa Houser, didn’t know Loring had ever published on the disease. She was just looking for a stem cell researcher who might hear her out. 

“I think I was just picked out a hat,” Loring told Endpoints News. 

At a meeting in Loring’s Scripps Research office, Houser and a Parkinson’s nurse practitioner, Sherrie Gould, asked her why there was so much research done in stem cell transplants for other neurodegenerative diseases but not Parkinson’s. They wanted to know if she would work on one. 

What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.

Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

And it’s impressive.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,500+ biopharma pros reading Endpoints daily — and it's free.