GSK li­cens­es Ion­is' ex­per­i­men­tal hep B treat­ments in up to $262M deal

GSK $GSK may have walked away from two of Io­n­is’ $IONS an­ti­sense drugs (in­clud­ing the now-ap­proved Tegse­di) in 2017 — but the British drug­mak­er held on as a part­ner to the biotech­nol­o­gy com­pa­ny’s ex­per­i­men­tal he­pati­tis B treat­ments.

On Tues­day, GSK ex­er­cised its op­tion to li­cense Io­n­is’ drugs — IO­N­IS-HBVRx and IO­N­IS-HBV-LRx — fol­low­ing pos­i­tive mid-stage da­ta. In re­turn, Io­n­is re­ceives up to $262 mil­lion in mile­stone pay­ments, in­clud­ing a $25 mil­lion li­cense fee. In ad­di­tion, Io­n­is is al­so el­i­gi­ble to re­ceive tiered roy­al­ties on net sales. Now, GSK is re­spon­si­ble for all de­vel­op­ment, reg­u­la­to­ry and com­mer­cial­iza­tion ac­tiv­i­ties and costs for the two drugs.

Hep B is trans­mit­ted through con­tact with blood and bod­i­ly flu­ids of an in­fect­ed per­son and is a high­ly in­fec­tious virus, 50 to 100 times more in­fec­tious than HIV — even slight laps­es in in­fec­tion con­trol can re­sult in pa­tient-to-pa­tient trans­mis­sion. The chron­ic in­fec­tion, which af­fects more than 200 mil­lion glob­al­ly, can lead to life-threat­en­ing health con­di­tions, in­clud­ing cir­rho­sis, liv­er fail­ure and liv­er can­cer. Cur­rent treat­ments that are ef­fec­tive in re­duc­ing cir­cu­lat­ing virus in the blood, do not ef­fi­cient­ly in­hib­it anti­gen pro­duc­tion and se­cre­tion, which are as­so­ci­at­ed with poor prog­no­sis.

The an­ti­sense drugs are en­gi­neered to ad­dress the root cause of the dis­ease — by re­duc­ing the pro­duc­tion of vi­ral pro­teins as­so­ci­at­ed with hep B in­fec­tion and repli­ca­tion, in­clud­ing hep B sur­face anti­gen, which is found in both acute and chron­ic in­fec­tions. Mean­while, GSK al­ready sells a hep B vac­cine called En­ger­ix-B.

Io­n­is has a myr­i­ad of big phar­ma part­ner­ships un­der its belt, in­clud­ing Roche, Bio­gen, Bay­er and As­traZeneca. Un­der chief Stan­ley Crooke, who found­ed the com­pa­ny about thir­ty years ago, Io­n­is has honed a mod­el in which it de­vel­ops an­ti­sense prod­ucts for a pletho­ra of in­di­ca­tions, de-risks them in the clin­ic and then se­cures a phar­ma part­ner to con­duct piv­otal tri­als and com­mer­cial­ize. In re­turn, the com­pa­ny prof­its from a steady stream of up­front and mile­stone pay­ments, as well as roy­al­ties. Last De­cem­ber, Crooke dis­closed plans to tran­si­tion to the role of ex­ec­u­tive chair­man in 2020.

Hal Bar­ron

Un­der re­search chief Hal Bar­ron, GSK has been work­ing on con­jur­ing up some ex­cite­ment for its pipeline by large­ly fo­cus­ing on on­col­o­gy, al­though oth­er ar­eas that are un­der­served are al­so of in­ter­est. In an in­ter­view with the Fi­nan­cial Times in Ju­ly, he said that tak­ing “smart risks” in R&D is how he sees the once glacial gi­ant trans­form­ing its re­search work. “(O)nce you start fear­ing fail­ure, you be­come so con­ser­v­a­tive that you end up ba­si­cal­ly mov­ing to­wards things that are ob­vi­ous­ly go­ing to work and there­fore like­ly to be not very in­no­v­a­tive . . . And, ul­ti­mate­ly, it’s a death spi­ral.”

So­cial im­age: GSK, AP Im­ages

 

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.

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.

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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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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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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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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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.

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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