Alex­ion's cash cow re­ceives la­bel ex­pan­sion; Matthew Dal­las tapped to CFO post at Zealand Phar­ma

Back in June, Alex­ion‘s cash cow Soliris scored an ad­di­tion­al FDA OK for the treat­ment of neu­romyelitis op­ti­ca spec­trum dis­or­der (NMOSD) — and to­day the drug was waved for a la­bel ex­pan­sion by the Eu­ro­pean Com­mis­sion (EC).

The EC based its ap­proval on re­sults from the Phase III ran­dom­ized, dou­ble-blind, place­bo-con­trolled PRE­VENT study and long-term ex­ten­sion study (ECU-NMO-302) — with the lat­ter still un­der­way. “The study met its pri­ma­ry end­point of pro­long­ing the time to first ad­ju­di­cat­ed re­lapse and re­duc­ing the risk of re­lapse. Per the da­ta, 98% of adult AQP4 an­ti­body-pos­i­tive pa­tients treat­ed with Soliris were re­lapse-free com­pared to 63% re­ceiv­ing place­bo at 48 weeks,” read a press re­lease.

A sup­ple­men­tal NDA for the drug is be­ing re­viewed in Japan and the drug en­joys or­phan drug des­ig­na­tion for the treat­ment of NMOSD in the US, the EU and Japan.

Last week, ru­mors cropped up that Alex­ion faced a pos­si­ble buy­out from Am­gen, but those were squashed when Am­gen swooped in with a $13.4 bil­lion deal to pluck Cel­gene‘s block­buster Ote­zla on Mon­day.

→ Af­ter woo­ing Al­ny­lam ex­ec Em­manuel Du­lac to the post of CEO in April, Zealand Phar­ma has fi­nal­ly found a suc­ces­sor in Matthew Dal­las for their CFO Mats Blom — who an­nounced his de­par­ture in Jan­u­ary. Dal­las has served in the same role at Aveo Phar­ma­ceu­ti­cals and CoLu­cid Phar­ma­ceu­ti­cals (ac­quired by Eli Lil­ly). Dal­las has al­so held stints at Gen­zyme, NEN Life Sci­ence Prod­ucts and Kim­ber­ly Clark. Ivan Møller, who has been serv­ing as in­ter­im CFO, will re­sume his role as SVP of tech­ni­cal de­vel­op­ment & op­er­a­tions.

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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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.

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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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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TG Ther­a­peu­tics scores pri­or­i­ty re­view for PI3Kδ in­hibitor; Gala­pa­gos search­es for IBD tar­gets in new pact

TG Therapeutics’ pitch for umbralisib as a safer PI3Kδ inhibitor has convinced the FDA to put it on the fast lane.

Regulators have accepted the NDA for the once-daily drug, a dual inhibitor of PI3K-delta and CK1-epsilon first aimed at two indications: marginal zone lymphoma patients who have received at least one anti-CD20-based regimen, and follicular lymphoma patients who have gone through at least two lines of systemic therapies.