Third time the charm? An­a­lyst says a mar­ket­ing de­ci­sion is “im­mi­nent” for Sarep­ta’s Duchenne drug eteplirsen

Every­thing that hap­pens at Sarep­ta – re­al or imag­ined – is grounds for de­bate and ram­pant spec­u­la­tion on Twit­ter. So it’s no sur­prise that when Baird an­a­lyst Bri­an Sko­r­ney is­sued a note late last week say­ing that the FDA’s de­ci­sion on Sarep­ta’s con­tro­ver­sial drug eteplirsen is “im­mi­nent,” the on­line ker­fuf­fle that fol­lowed in­clud­ed plen­ty of taunts, tantrums and tears.

Some nec­es­sary back­ground: Sarep­ta’s NDA for eteplirsen has fa­mous­ly been de­layed twice. The first de­lay came af­ter FDA in­sid­ers round­ly crit­i­cized the drug for fail­ing to show signs of ef­fi­ca­cy. The sec­ond came with their sec­ond sal­vo that took apart Sarep­ta’s coun­ter­at­tack on a point-by-point ba­sis. Both re­views cas­ti­gat­ed the biotech for fail­ing to heed re­peat­ed ad­vice on run­ning a big­ger, bet­ter study. And then there was an ex­pert pan­el vote against an ap­proval, based on the agency’s over­whelm­ing­ly neg­a­tive view.

For any oth­er drug at any oth­er com­pa­ny, that would have been it. The CRO let­ter would have been a fore­gone con­clu­sion.

But that’s not Sarep­ta ($SRPT).

The com­pa­ny – which care­ful­ly in­te­grat­ed the full-throat­ed sup­port of the pa­tient com­mu­ni­ty with some of the most in­flu­en­tial ad­vo­cates in DC — has de­fied re­peat­ed fore­casts of a like­ly re­jec­tion on­ly to be giv­en an­oth­er chance. Their third chance has come in the form of an FDA re­quest for biop­sy in­for­ma­tion on 13 pa­tients. And the gen­er­al be­lief is that if the com­pa­ny can of­fer bio­mark­er ev­i­dence that the drug is pro­duc­ing the dy­s­trophin these boys lack, the agency will go ahead with an ac­cel­er­at­ed ap­proval.

Bri­an Sko­r­ney, Baird an­a­lyst

“We be­lieve that right now, some­one, whether at Sarep­ta or the FDA, is look­ing at base­line and 48-week West­ern blots for 13 PRO­MOVI pa­tients,” Sko­r­ney not­ed. “At this point, we think the de­ci­sion should be pret­ty cut and dry: if the base­line blots show no band, and the vast ma­jor­i­ty of the 48-week blots show a (dy­s­trophin) band, we pre­dict a rapid ac­cel­er­at­ed ap­proval for eteplirsen.”

Es­sen­tial­ly, the FDA has been cre­at­ing a new ac­cel­er­at­ed ap­proval path­way for drugs that stir wide­spread sup­port tied to per­sis­tent lob­by­ing ef­forts. Whether they give Sarep­ta a green light now or not, few biotechs – es­pe­cial­ly in rare, lethal dis­eases – will ig­nore the role of a pas­sion­ate and mo­ti­vat­ed pa­tient pop­u­la­tion in gar­ner­ing spe­cial reg­u­la­to­ry con­sid­er­a­tion for their treat­ment.

Whether that’s a good or a bad thing will be the fo­cus on plen­ty more out­raged Tweets in the days, months and years ahead.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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