Af­ter an R&D odyssey filled with set­backs, Clo­vis wins an FDA OK for ru­ca­parib

Richard Padzur, On­col­o­gy Cen­ter of Ex­cel­lence, FDA

Clo­vis On­col­o­gy $CLVS won some re­demp­tion to­day, gain­ing an ac­cel­er­at­ed ap­proval for its first drug and set­ting it on a short path to com­mer­cial­iza­tion.

The FDA has ap­proved its PARP in­hibitor ru­ca­parib, to be sold as Rubra­ca, to treat ovar­i­an can­cer cas­es with BR­CA mu­ta­tions. The drug is en­ter­ing a mar­ket that will be sliced and diced by a va­ri­ety of com­peti­tors. The Foun­da­tion­Fo­cus CDxBR­CA com­pan­ion di­ag­nos­tic was al­so OK’d for use with Rubra­ca.

Just last Oc­to­ber the Boul­der, CO-based biotech’s stock tanked af­ter an­a­lysts were unim­pressed by the lat­est da­ta cut, with me­di­an pro­gres­sion-free sur­vival time in a sin­gle-arm study at ten months. That’s fine for an FDA ap­proval in can­cer, though, and Rubra­ca will now weigh in against As­traZeneca’s pi­o­neer­ing Lyn­parza, which may prove the fa­vorite of the two.  There’s al­so an up­com­ing de­ci­sion on Tesaro’s ri­val ni­ra­parib, though that is for a dif­fer­ent ini­tial pa­tient pop­u­la­tion. Pfiz­er is com­ing up from be­hind with a PARP, ta­la­zoparib, that it ac­quired in the $14 bil­lion Medi­va­tion buy­out.

Leerink’s Sea­mus Fer­nan­dez not­ed:

While this cur­rent ap­proval is un­like­ly to im­pact the ini­tial com­pet­i­tive dy­nam­ics for TSRO’s (OP) ni­ra­parib or AZN’s (OP) Lyn­parza (ola­parib) in 2L main­te­nance, da­ta from Rubra­ca’s Phase 3 ARIEL3 tri­al in this set­ting are now ex­pect­ed in mid-2017 (vs. 2H:17 pre­vi­ous­ly). De­spite this ear­li­er read­out, we con­tin­ue to see ni­ra­parib as the ul­ti­mate win­ner in 2L main­te­nance (ap­proval like­ly in 1H:17), with its Phase 3 NO­VA tri­al hav­ing al­ready es­tab­lished a strong pro­gres­sion-free sur­vival (PFS) ben­e­fit in a broad pop­u­la­tion.

Tesaro re­port­ed ear­li­er that the ni­ra­parib arm hit the pri­ma­ry end­point of their study with a me­di­an PFS of 12.9 months com­pared to 3.8 months for the con­trol arm. Among germline BR­CA mu­ta­tion pa­tients, the me­di­an PFS for pa­tients treat­ed with ni­ra­parib was 21.0 months, com­pared to 5.5 months for con­trol.

It’s all good for Clo­vis to­day, though, which saw its shares surge an ini­tial 15% af­ter gain­ing an ap­proval that fol­lowed mul­ti­ple set­backs along the way. The biotech was forced to re­state its da­ta sub­mit­ted for an ap­proval of rocile­tinib, prompt­ing an em­bar­rass­ing and dev­as­tat­ing drop in the num­ber of re­spons­es that the biotech had claimed for their drug. An FDA pan­el sub­se­quent­ly re­ject­ed the drug, prompt­ing Clo­vis to re­struc­ture and lay off staffers, while bury­ing ro­ci as a los­er.

Clo­vis did not cut its com­mer­cial group, though, con­fi­dent that it could fi­nal­ly score a win here.

“To­day’s ap­proval is an­oth­er ex­am­ple of the trend we are see­ing in de­vel­op­ing tar­get­ed agents to treat can­cers caused by spe­cif­ic mu­ta­tions in a pa­tient’s genes,” said Richard Paz­dur, MD, di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search and act­ing di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “Women with these gene ab­nor­mal­i­ties who have tried at least two chemother­a­py treat­ments for their ovar­i­an can­cer now have an ad­di­tion­al treat­ment op­tion.”

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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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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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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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

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.