Rac­ing No­var­tis on Eylea ri­val, the FDA trips up Re­gen­eron at the fin­ish line

An ag­gres­sive Re­gen­eron $REGN has run in­to a road­block in its quest to get out ahead of a po­ten­tial Eylea ri­val from No­var­tis.

The big biotech, not known for ca­su­al­ly ced­ing mar­ket ter­ri­to­ry to any com­peti­tor, says its sup­ple­men­tal BLA for its once-every-12 week dose of Eylea for wet, age-re­lat­ed mac­u­lar de­gen­er­a­tion has been re­ject­ed by the FDA.

Fac­ing an Au­gust 11 PDU­FA dead­line, says Re­gen­eron, the FDA kicked out the ap­pli­ca­tion “due to on­go­ing la­bel­ing dis­cus­sions” — and that could in­di­cate a big ad­van­tage for its ri­vals at No­var­tis.

Re­gen­eron isn’t say­ing what the stick­ing points are, but with No­var­tis’ RTH258 $NVS breath­ing down its neck, a prob­lem with the la­bel could play a ma­jor role in the an­tic­i­pat­ed show­down be­tween the two com­pa­nies.

Rather than start over with a new ap­pli­ca­tion, Re­gen­eron says it ex­pects to get through la­bel ne­go­ti­a­tions and get a de­ci­sion from reg­u­la­tors in a cou­ple of months. A spokesper­son for Re­gen­eron ex­plains to me that that is be­cause they are mak­ing a Class 1 re­sub­mis­sion, which starts the count­down clock over at 2 months once the added in­for­ma­tion ar­rives.

An­a­lysts aren’t go­ing to be hap­py about even a short in­ter­rup­tion here, or an in­di­ca­tion that Re­gen­eron may be left in an un­com­pet­i­tive sit­u­a­tion. 

Jef­feries’ an­a­lyst Biren Amin called the set­back a sur­prise, and not­ed that the de­lay could be ex­tend­ed if Re­gen­eron feels a need to go be­yond the Phase IV da­ta it had sup­plied for the sBLA. “We think REGN may bring up the AL­TAIR da­ta how­ev­er that would be con­sid­ered a ma­jor da­ta amend­ment and re­quire a more sign­fi­ciant FDA re­view,” Amin not­ed.

Eylea’s $5 bil­lion in rev­enue makes it the pro­duc­tive cash cow that sup­ports the com­pa­ny’s R&D work and co-com­mer­cial­iza­tion pacts with Sanofi on a slate of new, up-and-com­ing drugs. But the ag­ing fran­chise has been peak­ing out, leav­ing an­a­lysts a lit­tle dis­sat­is­fied with its big earn­er. New drug sales, though, are on the rise. And Re­gen­eron and Sanofi are al­so ex­pect­ed to pick up a near-term ap­proval for cemi­plimab, the world’s sixth PD-1/L1 drug.

Dirk Sauer

No­var­tis, mean­while, has been ac­cel­er­at­ing its work on the com­pet­ing drug RTH258, lean­ing heav­i­ly on its 12-week dos­ing sched­ule to of­fer an ear­li­er treat­ment reg­i­men for pa­tients who un­der­stand­ably may pre­fer few­er eye in­jec­tions. 

No­var­tis knows it will have a fight on its hands, but be­lieves they’re po­si­tioned to over­come Re­gen­eron on its home turf.

“I can’t spec­u­late on the FDA la­bel for Eylea or for ours, but the da­ta are not the same,” Dirk Sauer, head of No­var­tis’ oph­thal­mol­o­gy unit, told us a few months ago. “We did a prospec­tive analy­sis, while they went back and did a ret­ro­spec­tive analy­sis. Ret­ro­spec­tive analy­ses, from an an­a­lyt­i­cal and sta­tis­ti­cal point of view, are weak­er.”

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

UP­DAT­ED: In­di­v­ior's Shaun Thax­ter heads to prison, join­ing In­sys' John Kapoor among jailed opi­oid ex­ecs

Update: An earlier version of this article misidentified the jailed Insys CEO. Former CEO John Kapoor was sentenced to 5.5 years in prison in January. Endpoints News regrets the error.

The Justice Department’s years-long battle with Indivior has arrived at a rare place: the jailing of a pharmaceutical executive.

A US district court sentenced long-running Indivior CEO Shaun Thaxter to 6 months in federal prison for his role in company efforts to mislead a major healthcare provider about the safety and abusability of their opioid addiction drug Suboxone, which generated billions in revenue over the last decade. Thaxter joins former Insys CEO John Kapoor as one of the only two executives to face prison time for their roles in the opioid epidemic.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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