As­traZeneca’s shares spike on a sur­prise coup in PhI­II lung can­cer da­ta

Sean Bo­hen, As­traZeneca

As­traZeneca $AZN shares popped more than 7% this morn­ing on pos­i­tive Phase III pro­gres­sion-free sur­vival da­ta for Imfinzi (dur­val­um­ab) in treat­ing ad­vanced, in­op­er­a­ble and chemo-re­sis­tant non-small cell lung can­cer.

An­a­lysts did a quick dou­ble take and im­me­di­ate­ly cheered it as a re­mark­able – and com­plete­ly un­ex­pect­ed – hit.

That’s the right tar­get for As­traZeneca, which re­cent­ly won its first ap­proval for the world’s fifth check­point in­hibitor. On Wednes­day Mer­ck moved to the front of that field with an ap­proval to use a com­bi­na­tion of Keytru­da with chemo as a first-line ther­a­py on NSCLC, putting it on top of the heap of ri­vals look­ing to carve out a big piece of the block­buster mar­ket.

This As­traZeneca tri­al wasn’t the big study an­a­lysts have been wait­ing for.

As­traZeneca has been bet­ting heav­i­ly on MYS­TIC, us­ing a com­bi­na­tion of dur­val­um­ab and the CT­LA-4 drug treme­li­mum­ab for NSCLC. That would cre­ate some ma­jor mar­ket op­por­tu­ni­ties for the phar­ma gi­ant, which has been mak­ing a rep for it­self in the on­col­o­gy field in re­cent years — even as it’s plagued by prat­falls and dis­ap­point­ments in oth­er ar­eas of R&D.

As­traZeneca bad­ly needs every shred of good news that it can find, and there were some high fives in the or­ga­ni­za­tion on this pos­i­tive read­out, which helps the com­pa­ny dis­tin­guish it­self in the most in­tense­ly com­pet­i­tive field in bio­phar­ma.

Sea­mus Fer­nan­dez, Leerink

Sea­mus Fer­nan­dez at Leerink gave it a big thumbs up, not­ing:

This news comes as an ear­ly up­side sur­prise, as we do not cur­rent­ly have fore­casts for this in­di­ca­tion in our mod­el nor do we be­lieve this op­por­tu­ni­ty is wide­ly ap­pre­ci­at­ed by in­vestors. Im­por­tant­ly, this puts AZN square­ly in the IO fight in with a unique NSCLC in­di­ca­tion for monother­a­py PD1/PDL1 agents not cur­rent­ly shared by oth­er mem­bers of the class. AZN es­ti­mates that it is at least 2-3 years ahead of oth­er com­peti­tors, and our search­es on­ly un­cov­ered rel­a­tive­ly small sin­gle-arm Ph 2 stud­ies un­der­way for po­ten­tial com­peti­tors.

Oth­er an­a­lysts at Deutsche Bank were al­so im­pressed, of­fer­ing some block­buster num­bers for As­traZeneca on this one in­di­ca­tion.

Look for the da­ta to make the short trip to reg­u­la­tors as As­traZeneca seeks to cap­i­tal­ize on fore­casts that this drug could be worth more than $2 bil­lion a year for the com­pa­ny.

Said R&D chief Sean Bo­hen:

We look for­ward to work­ing with reg­u­la­to­ry au­thor­i­ties around the world to bring Imfinzi to lung can­cer pa­tients as soon as pos­si­ble. Along­side this, we con­tin­ue to ex­plore Imfinzi’s full po­ten­tial as monother­a­py as well as in com­bi­na­tion with treme­li­mum­ab and oth­er med­i­cines in ar­eas of con­tin­ued un­met need across mul­ti­ple types of can­cer.

At a time when the check­point can­cer mar­ket would seem to be ripe for ma­tur­ing, with 5 ap­proved, there’s in­stead been a se­ries of star­tling re­ver­sals and ad­vances. Bris­tol-My­ers Squibb made the move to the lead, then blew it with a bad­ly de­signed Phase III on front­line use. Mer­ck leapfrogged its ri­val and then, just days ago, Roche’s Tecen­triq failed a con­fir­ma­to­ry Phase III in blad­der can­cer, af­ter it had been giv­en an ac­cel­er­at­ed ap­proval.

Big sur­pris­es are be­com­ing the or­der of the day in check­point in­hi­bi­tion, a field worth tens of bil­lions of dol­lars.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

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