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.

 

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

Hal Barron and Emma Walmsley, GSK

GSK’s ‘break­through’ BC­MA can­cer drug gets a pri­or­i­ty re­view — and a big win for the on­col­o­gy R&D team

After largely whiffing the past 2 years on the pharma R&D front, GlaxoSmithKline research chief Hal Barron has seized boasting rights to a key win that puts them back in the cancer drug development game.

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Who are the young bio­phar­ma lead­ers shap­ing the in­dus­try? Nom­i­nate them for End­points' spe­cial re­port

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.

John Oyler, Endpoints

BeiGene lines up its first shot at crack­ing the megablock­buster PD-1 mar­ket for lung can­cer. But can they over­come un­der­dog sta­tus?

BeiGene took another big step towards challenging Merck, Bristol-Myers Squibb, AstraZeneca and some other Big Pharma heavyweights for a share of the lucrative lung cancer market for the PD-(L)1s racking up billions in annual revenue.

The China-based biotech $BGNE run by CEO John Oyler posted positive top-line progression-free survival results for their pivotal Chinese study on their PD-1 antibody tislelizumab combined with chemo for squamous non-small cell lung cancer in frontline cases. Squamous NSCLC accounts for about 30% of the overall lung cancer market.

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Brex­it fears, Wood­ford woes over­shad­owed UK biotech and cut 2019 fi­nanc­ing by al­most half

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

Blue­print Med­i­cines po­ten­tial­ly de­lays Ay­vak­it de­ci­sion; Con­trol beats treat­ment in mesothe­lioma tri­al

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III VOYAGER before they give a judgment.