As­traZeneca gains a speedy Imfinzi re­view in race to beat Roche on front­line SCLC

Well, Roche’s Tecen­triq had a good few-month run as the lone front­line im­munother­a­py on the small-cell lung can­cer mar­ket. That reign may soon end.

Over the hol­i­day week­end, As­traZeneca an­nounced its im­muno-on­col­o­gy drug Imfinzi has been grant­ed an FDA ‘pri­or­i­ty re­view’ for small-cell lung can­cer, or SCLC. The des­ig­na­tion comes on the heels of a 537-per­son tri­al show­ing the treat­ment helped pa­tients live a lit­tle un­der 3 months longer than un­der the stan­dard-of-care. A PDU­FA date was set for the first quar­ter of 2020.

Al­though the most com­mon type of lung can­cer, non-small cell lung can­cer or NSCLC, has been one of the most com­mon tar­gets for im­munother­a­pies, SCLC has proven more elu­sive. In March, Roche’s Tecen­triq be­came the first such ther­a­py to get the FDA’s bless­ing as a front­line treat­ment (Op­di­vo and Keytru­da have each been ap­proved as a sec­ond-line ther­a­py). Imfinzi, like Tecen­triq, is a PD-L1 in­hibitor.

The two drugs per­formed sim­i­lar­ly on over­all sur­vival, the pri­ma­ry end­point, in their re­spec­tive Phase III tri­als. Tecen­triq showed an OS of 12.3 months against a 10.3-month place­bo arm, com­pared with Imfinzi’s 13 months against a 10.3-month place­bo arm. As­traZeneca has al­so high­light­ed their drug’s dura­bil­i­ty of re­sponse. In both cas­es, pa­tients al­so re­ceived stan­dard chemother­a­py.

For As­traZeneca, the new des­ig­na­tion rep­re­sents an­oth­er step in a 2019 pipeline resur­gence and a ma­jor win on a drug that flopped big last year.

That No­vem­ber, the British drug­mak­er said that af­ter 5 years, its MYS­TIC tri­al com­bin­ing Imfinzi and a CT­LA-4 in­hibitor had failed.  This Au­gust, they an­nounced the com­bo had failed again.  Nev­er­the­less, new on­col­o­gy chief José Basel­ga has seen ap­provals this year for the BTK in­hibitor Calquence and pos­i­tive read­outs from tri­als test­ing an Imfinzi triple-mix in lung can­cer and test­ing their PARP in­hibitor Lyn­parza in ovar­i­an and prostate can­cers.

SCLC cas­es rep­re­sent 10-15% of lung can­cer cas­es, with NSCLC pa­tients cov­er­ing the rest. As­traZeneca has not an­nounced the ex­act PDU­FA date, but giv­en the FDA’s re­cent break­neck pace in ap­provals, it may not mat­ter.

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.