Shire CEO Flem­ming Orn­skov is hatch­ing a few new stretch goals for R&D in 2018 — and be­yond

SAN FRAN­CIS­CO — Shire stock $SH­PG took a hit ear­li­er this week as in­vestors fret­ted over CEO Flem­ming Orn­skov’s con­ces­sion that the com­pa­ny isn’t go­ing to meet a longterm rev­enue goal he set for him­self, like­ly falling two or three bil­lion dol­lars shy of the $20 bil­lion en­vi­sioned for 2020.

That can hap­pen, Orn­skov tells me, when you set some stretch goals for your­self fol­low­ing a deal like the Bax­al­ta buy­out. You can push your­self and the com­pa­ny, push hard, but still get dinged by things like an un­ex­pect­ed gener­ic at­tack or an up­set win by a ri­val like Roche. Things hap­pen in this busi­ness you can’t al­ways pre­dict.

But Orn­skov — an in­tense­ly com­pet­i­tive ex­ec still work­ing on a makeover of Shire in­to the in­dus­try’s pre­mier rare dis­ease play­er — wasn’t in an apolo­getic frame of mind as he toured the scene in San Fran­cis­co. And he is still think­ing about the near-term and long-term ob­jec­tives — his new stretch goals, if you will — that will have to be met to live up to the mark he’s set for him­self and the com­pa­ny.

An­dreas Busch

He’s still plan­ning to beat con­sen­sus in 2020, he tells an­a­lysts. He’s clear­ly pumped that An­dreas Busch, a close col­league from Orn­skov’s days at Bay­er, has just stepped in to man­age the pipeline as Shire ex­e­cutes a new step in the on­go­ing re­or­ga­ni­za­tion of R&D with a big move in­to Cam­bridge, MA. And the CEO has a mul­ti-point im­prove­ment plan for the R&D side of the com­pa­ny as he di­vides the op­er­a­tion in­to two di­vi­sions — one for rare dis­eases and the oth­er for neu­ro­sciences.

The way Orn­skov sees it, the rare dis­eases pipeline looks great. “That part of the busi­ness has one of the most at­trac­tive pipelines we have ever had.”

It may be a lit­tle light on trans­la­tion­al and ear­ly-stage pro­grams, he re­flects, and that’s where the com­pa­ny could be ex­pect­ed to fo­cus with some new deals as they look to field new drugs. He’s not ab­solute­ly rul­ing out a Phase III rare dis­ease deal if some­thing comes along he can’t over­look, but it clear­ly isn’t like­ly.

But where Shire’s ex­ec­u­tive team can have the most im­me­di­ate im­pact is on the neu­ro­sciences side of the com­pa­ny, dom­i­nat­ed by some ag­ing AD­HD fran­chise drugs he’s sug­gest­ed may be spun out at some point. This week, in­stead of a spin­out, Orn­skov promised to do more deals — li­cens­ing, part­ner­ing and bolt-ons — that would make the group at­trac­tive wher­ev­er it was, in­side or out­side Shire.

Says Orn­skv: “I think for me it’s the same ul­ti­mate goal to build a busi­ness that is so strong that peo­ple will be beg­ging me to spin it out be­cause it would be so strong as a stand­alone.”

Or maybe they’ll be beg­ging him to keep it.

JP­Mor­gan is all about the year ahead. But I asked Orn­skov, who’s been work­ing on a makeover at Shire for the past 5 years, where the biggest op­por­tu­ni­ties are go­ing to emerge a few years down the road.

If there’s one key R&D are­na that Orn­skov wants to con­cen­trate more on over the long run, it’s gene ther­a­py. Af­ter that, he’s pay­ing very close at­ten­tion to gene edit­ing and how the com­pa­ny should po­si­tion it­self as new tech­nolo­gies like CRISPR be­gin to play out in hu­man stud­ies.

“We have to think what our busi­ness will be in 10 years,” he says. And that has him think­ing about he­mo­phil­ia A and B as well as gene ther­a­pies for the eye — a fa­vorite re­search field of his.

Shire has a gene ther­a­py pro­gram en­ter­ing the clin­ic on he­mo­phil­ia — ‘654’ — which he sees as a com­peti­tor to lead­ing ri­vals from Bio­Marin and Roche. So there is a gene ther­a­py team there they can build around, as the com­pa­ny is play­ing catchup on a crit­i­cal front.

“We need to be in it so we can al­so fig­ure out where the in­no­va­tion is go­ing,” he says.

Orn­skov can get a bit testy when he’s called on some­thing like falling short of a goal. And don’t ever ex­pect him to apol­o­gize about his man­age­ment style.

“Every­body has a dif­fer­ent man­age­r­i­al style,” says the CEO. His in­volves set­ting stretch goals — “but I know if I have a stretch I will achieve more. Suc­cess­ful peo­ple set stretch goals.”

And they’re still com­ing.

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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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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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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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.

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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Martin Shkreli (Photo by Drew Angerer/Getty Images)

Mar­tin Shkre­li re­ceives life­time in­dus­try ban, forced to re­turn al­most $65M in prof­its af­ter an­ti­com­pet­i­tive scheme

Martin Shkreli will have to find a new nickname.

A federal judge banned the former biotech CEO and “Pharma Bro” from the drug industry on Friday, ordering him to pay nearly $65 million in illicit profits. Shkreli was convicted of securities fraud in 2017 and is currently serving a seven-year prison sentence, though he originally gained notoriety for raising the price of the antiparasitic drug Daraprim from $13.50 to $750 in 2015.