Now a two-time los­er, As­traZeneca’s hopes for selume­tinib dwin­dle af­ter an­oth­er PhI­II flop

As­traZeneca’s one-time block­buster hope­ful selume­tinib is now a two-time los­er in late-stage de­vel­op­ment. The phar­ma gi­ant was forced to con­cede Tues­day morn­ing that the drug flopped in KRAS-pos­i­tive non-small cell lung can­cer. The drug whiffed on pro­gres­sion-free sur­vival and failed to score on over­all sur­vival as well.

As­traZeneca had to walk away from the first Phase III for selume­tinib last year af­ter the drug — in-li­censed from Ar­ray — failed for an eye can­cer called uveal melanoma.

The back-to-back fail­ures leave shrink­ing hope that the drug can ever live up to the block­buster fu­ture that CEO Pas­cal So­ri­ot had fore­cast for it sev­er­al years ago. The drug was a sig­nif­i­cant piece of As­traZeneca’s promise to build rev­enues to $45 bil­lion by 2023. Now it’s an­oth­er ex­am­ple of the hit-and-miss for­tunes of the phar­ma gi­ant since So­ri­ot took the helm.

As­traZeneca’s plans for the drug have dwin­dled down to thy­roid can­cer, where it won’t be giv­en good odds of suc­cess. As­traZeneca is known for stay­ing rigid­ly fo­cused on adding im­por­tant drugs to its port­fo­lio, a strat­e­gy that has led to the dis­count sales of trou­bled drugs like bro­dalum­ab and lesin­u­rad.

Shares of As­traZeneca slipped a lit­tle Tues­day morn­ing, while shares of Ar­ray $AR­RY cratered, drop­ping 18% in pre-mar­ket trad­ing.

As selume­tinib and oth­er drugs fad­ed, As­traZeneca’s suc­cess with Tagris­so loomed large as an ex­am­ple of what the can­cer drug side of the com­pa­ny could ac­com­plish. Those hopes ex­tend to the PD-L1 drug dur­val­um­ab, now in a big Phase III pro­gram as Bris­tol-My­ers, Mer­ck and Roche all divvy up the mar­ket with ap­proved drugs. But As­traZeneca has had oth­er set­backs on the can­cer front as well. Last spring, just weeks af­ter As­traZeneca ($AZN) re­port­ed a suc­cess for a com­bi­na­tion study of dur­val­um­ab and treme­li­mum­ab, the phar­ma gi­ant was forced to con­cede that the CT­LA-4 drug treme­li­mum­ab flunked a so­lo chal­lenge for mesothe­lioma.

Sean Bo­hen, As­traZeneca CMO

As­traZeneca CMO Sean Bo­hen was left to read the last rites over the lung can­cer study. And he didn’t sound hope­ful that this drug had much of a fu­ture in a pre­pared state­ment:

A ran­dom­ized Phase II tri­al showed promis­ing ac­tiv­i­ty of selume­tinib in com­bi­na­tion with do­c­etax­el in pa­tients with KRASmu­ta­tion-pos­i­tive lung can­cer. It is dis­ap­point­ing for pa­tients that these re­sults have not been con­firmed in Phase III. We ex­pect to present da­ta at a forth­com­ing med­ical meet­ing. We re­main com­mit­ted to fur­ther de­vel­op­ing treat­ments in the lung can­cer set­ting, such as our im­munother­a­py com­bi­na­tions and tar­get­ed EGFR treat­ments.

 

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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FDA ap­proves the third NSOMD drug in 18 months as Roche/Genen­tech beefs up its port­fo­lio of drugs for neu­ro­log­i­cal dis­or­ders

There were no FDA approved treatments for neuromyelitis optica spectrum disorder at the start of 2019. Now, as of Friday, there are three.

The latest entrant to the market is the Roche/Genentech drug satralizumab after US regulators gave it the thumbs up late Friday. An IL-6 inhibitor, the drug joins Alexion’s Soliris and AstraZeneca spinout Viela Bio’s Uplizna. The annual cost of satralizumab — which will hit the market as Enspryng — will be $190,000 for 13 doses, a Genentech spokesperson said, though the first year of treatment requires 15 doses and cost about $220,000.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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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.

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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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

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 14. 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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