In a blow to Eli Lil­ly, No­vo Nordisk lands FDA OK for their next big en­try in the di­a­betes mar­ket: Ozem­pic

An en­er­gized FDA has come through with an ap­proval of No­vo Nordisk’s once-week­ly GLP-1 di­a­betes drug semaglu­tide, open­ing the door to a mar­ket many an­a­lysts are con­vinced is worth more than $2 bil­lion a year in peak sales. And it could spell big trou­ble for ri­val Eli Lil­ly.

Mads Krogs­gaard Thom­sen

Type 2 di­a­betes is a mas­sive and fast-grow­ing dis­ease, with a glob­al mar­ket that has re­ward­ed a hand­ful of key play­ers like No­vo Nordisk a multi­bil­lion-dol­lar mar­ket. And No­vo ran a typ­i­cal­ly thor­ough Phase III pro­gram.

In do­ing so, No­vo al­so came up with su­pe­ri­or da­ta to Eli Lil­ly’s Trulic­i­ty, set­ting up a head-to-head show­down that could cost Lil­ly dear­ly.

Re­searchers al­so came up with some trou­bling in­stances of di­a­bet­ic retinopa­thy, which can lead to blind­ness. But a near unan­i­mous vote by a group of out­side ex­perts at the FDA felt that was an is­sue that could be mon­i­tored, in light of the ben­e­fits in glycemic con­trol the drug of­fered.

No­vo al­so isn’t done with the de­vel­op­ment of this drug, now Ozem­pic, for the big US mar­ket. Re­searchers are busi­ly putting it through an­oth­er big test as an obe­si­ty ther­a­py af­ter trig­ger­ing some im­pres­sive weight loss among obese pa­tients. And they would be squar­ing off against some unim­pres­sive ri­vals now on the mar­ket.

In an R&D up­date dur­ing its Cap­i­tal Mar­kets Day at its HQ near Copen­hagen just days ago, in­ves­ti­ga­tors for the com­pa­ny said they will re­cruit 4,500 pa­tients for a Phase II­Ia obe­si­ty tri­al for semaglu­tide. And they plan to launch the 68-week study next year, along­side a “land­mark” car­dio­vas­cu­lar out­comes study with 12,500 peo­ple en­rolled.

“We are very ex­cit­ed about the first ap­proval of Ozem­pic and look for­ward to mak­ing this im­por­tant in­no­va­tion avail­able to peo­ple in the US with type 2 di­a­betes in the be­gin­ning of 2018,” said Mads Krogs­gaard Thom­sen, ex­ec­u­tive vice pres­i­dent and chief sci­ence of­fi­cer, in a state­ment. “Type 2 di­a­betes is a com­plex dis­ease, but with the unique clin­i­cal pro­file of Ozem­pic, we be­lieve it has the po­ten­tial to set a new stan­dard for the treat­ment of the dis­ease.”

The FDA is now just a few ap­provals from break­ing the 45 NDAs of­fered in 2015.

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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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.

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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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

An un­ortho­dox pro­pos­al for Bio­gen's Medicare-man­dat­ed Aduhelm tri­al

Biogen has gone full blitz since Medicare announced it would only cover its new Alzheimer’s drug when used in clinical trials, accusing the agency of discriminating against Alzheimer’s patients and trying to get physicians to change regulators’ minds.  Critics, meanwhile, cheered what they see as a necessary wall protecting payers and patients from an unproven and unsafe drug.

Far less attention, though, has gone to what a Medicare-funded clinical trial would actually look like. Biogen has operated as if it would be a standard late-stage Alzheimer’s trial, enrolling a couple thousand patients and giving half placebo.

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