Al­ny­lam makes RNAi his­to­ry again as patisir­an gets ap­proved in Eu­rope; Ver­tex buys in­to UK-based ma­chine learn­ing group

→ Just days af­ter Al­ny­lam land­ed a his­toric FDA OK on patisir­an — and quick­ly launched it as On­pat­tro — the Eu­ro­pean Com­mis­sion has come through with its own mar­ket­ing au­tho­riza­tion. The drug, which treats hered­i­tary AT­TR amy­loi­do­sis by si­lenc­ing mes­sen­ger RNA and block­ing the pro­duc­tion of TTR pro­tein, is the first RNAi ther­a­py to be ap­proved in Eu­rope. It’s al­so a like­ly block­buster-to-be, with Clar­i­vate peg­ging 2022 pro­ject­ed sales at $1.21 bil­lion even though the dis­ease af­flicts on­ly about 30,000 peo­ple world­wide. In­ot­ersen, a ri­val prod­uct from Akcea and Io­n­is, was ap­proved in Eu­rope in Ju­ly.

Ver­tex is part­ner­ing with UK-based Ge­nomics plc on their plat­form for ge­net­ics and ma­chine learn­ing. Ver­tex $VRTX made a £10.5 mil­lion eq­ui­ty in­vest­ment in Ge­nomics plc as part of its £25 mil­lion Se­ries B fund­ing round — al­so an­nounced to­day. “Hu­man bi­ol­o­gy and ge­net­ics are a foun­da­tion of drug dis­cov­ery at Ver­tex and the team at Ge­nomics brings to­geth­er a unique com­bi­na­tion of so­phis­ti­cat­ed da­ta sci­ence and hu­man ge­net­ics,” said Ver­tex CSO David Alt­shuler. “This part­ner­ship will pi­o­neer new us­es of ge­nom­ic tools and tech­nolo­gies to ad­vance Ver­tex’s on­go­ing work and in­vest­ment at our Ox­ford Re­search Site and glob­al­ly to bring new med­i­cines to the pa­tients that need them.”

→ We fi­nal­ly know what to call Chi­na’s re­vamped FDA. The drug reg­u­la­tor will now go by the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion, or NM­PA, to re­flect its new man­date af­ter a gov­ern­ment re­or­ga­ni­za­tion sep­a­rat­ed med­i­cine reg­u­la­tion from oth­er con­sumer goods — in­clud­ing every­thing food-re­lat­ed. NM­PA re­ports to the new­ly cre­at­ed State Ad­min­is­tra­tion for Mar­ket Reg­u­la­tion.

Evotec and the CH­DI Foun­da­tion have ex­tend­ed their neu­ro­science col­lab­o­ra­tion for a few more years. In the pact, CH­DI will help fund the work of up to 75 sci­en­tists fo­cused on Hunt­ing­ton’s dis­ease.

Tem­pus, a pre­ci­sion med­i­cine com­pa­ny pro­vid­ing da­ta an­a­lyt­ic ser­vices to physi­cians and re­searchers, has raised $110 mil­lion in Se­ries E fi­nanc­ing. Er­ic Lefkof­sky — best known for co-found­ing Groupon — start­ed the com­pa­ny to as­sist re­al-time de­ci­sion-mak­ing for pa­tients, first in can­cer care. Har­ness­ing their pool of pa­tient da­ta, the com­pa­ny al­so op­er­ates an organoid de­vel­op­ment and test­ing fa­cil­i­ty to build bi­o­log­i­cal mod­els that they be­lieve can in­form clin­i­cal tri­als.

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.

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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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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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.

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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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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