Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Us­ing a lap­top bor­rowed from his for­mer em­ploy­er, South Lon­don’s St George’s Hos­pi­tal, Sir An­drew Dil­lon set about es­tab­lish­ing NICE — launched by the then health sec­re­tary Frank Dob­son — in 1999.  On Thurs­day, the UK cost-ef­fec­tive­ness watch­dog said its first and on­ly chief ex­ec­u­tive — Dil­lon — is step­ping down in March 2020.

Back in the day, de­ci­sions about which drugs and in­ter­ven­tions were fund­ed by the Na­tion­al Health Ser­vice (NHS) were made at the lo­cal lev­el, but this ‘post­code pre­scrib­ing’ sys­tem was fraught with skewed health­care de­ploy­ment mak­ing the struc­ture un­sus­tain­able. A na­tion­al sys­tem was deemed nec­es­sary — and NICE was formed to bridge that gap.

NICE got off to a con­tro­ver­sial start. In its sem­i­nal months, the agency de­ter­mined that the flu treat­ment Re­len­za was not cost-ef­fec­tive, cit­ing in­suf­fi­cient ev­i­dence that the prod­uct re­duced the sever­i­ty of the ill­ness for the im­muno­com­pro­mised: the el­der­ly and asth­ma suf­fer­ers. The mak­er of Re­len­za, Glaxo Well­come (erst­while GSK $GSK), threat­ened to leave the UK if the de­ci­sion was not re­versed — but NICE stuck to its guns. GSK nev­er left — in fact, the drug­mak­er’s glob­al head­quar­ters in Brent­ford, Mid­dle­sex was opened by then Prime Min­is­ter Tony Blair in 2002.

David Haslam NICE

Un­der Dil­lon, NICE’s rep­u­ta­tion as a stern but typ­i­cal­ly fair ar­biter of what qual­i­fies as NHS wor­thy has grown over time, as has the re­mit of its au­thor­i­ty. The agency has made a tan­gi­ble im­pact on pa­tients — from im­prov­ing out­comes and sav­ing costs by en­sur­ing sus­pect­ed can­cer cas­es are seen by spe­cial­ists ur­gent­ly to re­duc­ing the lev­el of in­ap­pro­pri­ate an­tibi­ot­ic pre­scrib­ing at the pri­ma­ry care lev­el to tack­le an­timi­cro­bial re­sis­tance. Re­cent­ly, NICE has come un­der pres­sure for spurn­ing Ver­tex’s $VRTX cys­tic fi­bro­sis med­i­cine and GW Phar­ma’s $GW­PH cannabis-de­rived drug for rare forms of pe­di­atric epilep­sy.

“The role of Chief Ex­ec­u­tive of NICE must be one of the most chal­leng­ing and po­ten­tial­ly con­tro­ver­sial in British pub­lic life. Sir An­drew has car­ried out this role for 20 enor­mous­ly suc­cess­ful years, and every­one who knows him – whether in gov­ern­ment, the life sci­ences in­dus­try, or in health and so­cial care – is full of ad­mi­ra­tion for his calm and skill­ful lead­er­ship,” NICE’s Chair, Sir David Haslam, said in a state­ment. “He was there right at the birth of NICE, and will leave it as an in­ter­na­tion­al­ly re­spect­ed, world lead­ing, and huge­ly in­flu­en­tial or­gan­i­sa­tion. That’s quite a lega­cy…”

Mike Thomp­son ABPI

The As­so­ci­a­tion of British Phar­ma­ceu­ti­cal In­dus­try (ABPI) — which is rec­og­nized by the gov­ern­ment as the in­dus­try body ne­go­ti­at­ing on be­half of the brand­ed phar­ma­ceu­ti­cal in­dus­try for a range of things, in­clud­ing the pric­ing scheme for med­i­cines in the UK — re­spond­ed to the news of Dil­lon’s de­par­ture with a glow­ing re­view.

“Sir An­drew has led NICE with a laser fo­cus on es­tab­lish­ing how the lat­est med­i­cines can ben­e­fit pa­tients and the NHS. He has played a cen­tral role in build­ing the cred­i­bil­i­ty of the or­gan­i­sa­tion, in­sist­ing on trans­par­ent process­es, and a con­tin­u­ous di­a­logue with all stake­hold­ers, recog­nis­ing that as the sci­ence evolves, NICE needs to evolve too,” ABPI chief Mike Thomp­son said in a state­ment.

NICE’s board plans to ad­ver­tise the post dur­ing the au­tumn.

The an­nounce­ment comes days af­ter it was re­vealed that Ian Hud­son, chief ex­ec­u­tive of the UK’s Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA), is step­ping down. Hud­son — who spent 18 years with the agency, in­clud­ing six years as chief ex­ec­u­tive — will make his ex­it in Oc­to­ber along with John Wilkin­son, the di­rec­tor of de­vices at MHRA.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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FDA's ODAC shrugs off ob­jec­tions to Mesoblast's GVHD drug for chil­dren, vot­ing 8-2 in fa­vor and im­prov­ing the odds of an ap­proval

The FDA’s Oncologic Drugs Advisory Committee once again waved through an investigational drug, clearing the potential final hurdle before the agency’s decision.

Thursday’s winner was Mesoblast $MESO, an Australian stem-cell company that submitted its Ryoncil drug for the treatment of steroid refractory acute graft-versus-host disease in children younger than 12. ODAC gave Ryoncil the thumbs up by an 8-2 vote, shrugging off concerns about trial design and pushing aside the need for an additional study.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.