FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Ver­tex has clinched a pri­or­i­ty re­view for the all-im­por­tant cys­tic fi­bro­sis triple that will blaze the trail for treat­ing a large group of pa­tients un­helped by its cur­rent drugs.

Resh­ma Ke­wal­ra­mani

FDA reg­u­la­tors have set a PDU­FA date of March 19, 2020, just a year af­ter the Boston biotech post­ed pos­i­tive Phase III re­sults show­ing that peo­ple with two F508del mu­ta­tions ex­pe­ri­enced sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in lung func­tion af­ter a 4-week reg­i­men of VX-445, teza­caftor and iva­caftor. Af­ter re­view­ing 24-week da­ta among pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion — and thor­ough­ly com­par­ing the VX-445 triple with an­oth­er com­bo fea­tur­ing VX-659 on scores like safe­ty, drug-drug in­ter­ac­tions, and pho­to­sen­si­tiv­i­ty — Ver­tex ul­ti­mate­ly went with VX-445.

Dubbed elex­a­caftor, the new drug is a “cor­rec­tor” de­signed to fix the shape and course of de­fec­tive CFTR pro­tein, bring­ing more of them to the cell sur­face. No­tably, teza­caftor has a sim­i­lar func­tion but does not ad­dress min­i­mal func­tion mu­ta­tions. Mean­while, iva­caftor is a “po­ten­tia­tor” that in­creas­es the func­tion of the pro­tein once it reach­es the cell sur­face.

Iva­caftor is mar­ket­ed on its own as Ka­ly­de­co, while Symdeko con­sists of both iva­caftor and teza­caftor.

Jeff Lei­den

An ap­proval in March would mark a fi­nal vic­to­ry for CEO Jeff Lei­den, who has an­nounced a sur­prise tran­si­tion to the ex­ec­u­tive chair­man role while pass­ing the ba­ton to cur­rent CMO Resh­ma Ke­wal­ra­mani in April 2020. Sub­se­quent dis­cus­sions on pric­ing would al­so draw at­ten­tion to her ne­go­ti­at­ing style, giv­en Ver­tex’s high pro­file con­fronta­tions with Eu­ro­pean sin­gle-pay­er sys­tems.

“If ap­proved, the VX-445 (elex­a­caftor), teza­caftor and iva­caftor triple com­bi­na­tion reg­i­men would be a sig­nif­i­cant ad­vance in CF treat­ment as the first CFTR mod­u­la­tor for those with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, and bring ad­di­tion­al ben­e­fit to pa­tients with two F508del mu­ta­tions,” Ke­wal­ra­mani said in a state­ment.

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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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.

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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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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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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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.

UP­DAT­ED: How much has pre­ci­sion med­i­cine helped? A new NCI study of­fers clues

Does precision medicine work?

The approach, based on finding a drug to target a patient’s specific genes, has undoubtedly saved individual lives, spurring Lazarus-like reversals in health in once-terminally ill patients. But critics have pointed out that its pursuit has meant drug companies spending hundreds of millions of dollars to target mutations that affect narrow slices of the populations, and that many of the gains researchers thought it would bring have eroded as cancers evolve resistance.