Much crit­i­cized Mallinck­rodt bows out of PhRMA ahead of some tight­ened re­stric­tions on mem­ber­ship

Mark Tyn­dall, Mallinck­rodt

Just ahead of some new, more mus­cu­lar rules gov­ern­ing who can be a part of the in­dus­try lob­by­ing or­ga­ni­za­tion PhRMA, one of its most heav­i­ly crit­i­cized mem­bers is pulling out.

PhRMA con­firmed to me Wednes­day af­ter­noon that Mallinck­rodt — at the cen­ter of a lin­ger­ing drug pric­ing scan­dal — has re­signed from the trade group.

Just a few months ago, Mallinck­rodt agreed to pay a $100 mil­lion fine to re­solve a probe of the long, rather sor­did his­to­ry be­hind Ac­thar, an in­fan­tile spasm drug which cost $28,000 a vial when Mallinck­rodt picked it up in the $5.6 bil­lion ac­qui­si­tion of Quest­cor. Quest­cor had been jack­ing up the price on Ac­thar when it paid No­var­tis $135 mil­lion to gain US rights to a ther­a­py that posed a di­rect threat to its drug fran­chise. And Mallinck­rodt was forced to pay the fine for il­le­gal­ly main­tain­ing a drug mo­nop­oly — not the kind of sanc­tion PhRMA likes to see for mem­bers.

To­day, a lit­tle more than two years lat­er, the FTC says Ac­thar costs $34,000 a vial. Over 15 years the price on Ac­thar has gone up 85,000%. Mallinck­rodt earned $1.16 bil­lion on Ac­thar last year, when it spent a to­tal of $262 mil­lion on R&D, 7.7% of its net rev­enue of $3.4 bil­lion.

This was all play­ing out when Marathon Phar­ma­ceu­ti­cals grabbed an FDA ap­proval for an old steroid, de­flaza­cort, specif­i­cal­ly for Duchenne mus­cu­lar dy­s­tro­phy and priced the ther­a­py — avail­able from UK sources at around $1,000 a year — at $89,000. Marathon CEO Jeff Aronin, who still sits on PhRMA’s board, ducked low when a con­tro­ver­sy burst out over the price as well as ques­tion­able claims about what it spent on re­search­ing the steroid and lat­er sold de­flaza­cort to PTC Ther­a­peu­tics for $140 mil­lion, plus a roy­al­ty stream and a po­ten­tial $50 mil­lion mile­stone.

Stephen Ubl, PhRMA CEO

PhRMA, mean­while, was rolling out a mul­ti­mil­lion dol­lar ad cam­paign aimed at im­prov­ing pub­lic opin­ion about drug­mak­ers. The pub­lic has reg­is­tered on­ly no­to­ri­ous­ly low sup­port for phar­ma com­pa­nies over­all. The group launched a re­view of its mem­ber­ship rules, sug­gest­ing that Marathon and oth­ers might not stay mem­bers once they tight­ened the cri­te­ria for join­ing, clear­ly pre­fer­ring to stick with big play­ers who tra­di­tion­al­ly in­vest heav­i­ly in R&D.

For Mallinck­rodt’s part, the de­par­ture was rou­tine and am­i­ca­ble. A spokesper­son tells me:

Mallinck­rodt rou­tine­ly eval­u­ates its en­gage­ment in trade as­so­ci­a­tions and pol­i­cy or­ga­ni­za­tions and has con­clud­ed that the sig­nif­i­cant fi­nan­cial and time com­mit­ment re­quired as a full PhRMA mem­ber out­weighs its di­rect pol­i­cy val­ue to us at this time, giv­en our present size and staff foot­print. We con­tin­ue to sub­scribe to the PhRMA Code of Con­duct, sup­port many of the group’s po­si­tions and ini­tia­tives and look for­ward to con­tin­u­ing our pos­i­tive work­ing re­la­tion­ship with PhRMA and its mem­bers.

Mallinck­rodt was singing a dif­fer­ent tune when they joined in ear­ly 2015.

“We are proud to be sit­ting at the same ta­ble as most of the top in­no­v­a­tive phar­ma­ceu­ti­cal com­pa­nies in the world, which puts us in a po­si­tion to help shape our in­dus­try in a way that sup­ports pa­tients and providers for the com­ing decades,” said Mark Tyn­dall, Vice Pres­i­dent of Gov­ern­ment Af­fairs, Pol­i­cy and Ad­vo­ca­cy, at the time.

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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