FDA re­buffs lit­tle As­ser­tio Ther­a­peu­tic­s' long-act­ing ACTH for­mu­la­tion, shares sink

Tiny As­ser­tio Ther­a­peu­tics’ shares plunged pre-mar­ket on Tues­day, af­ter the FDA has spurned its man-made ver­sion of the hor­mone ACTH, which was be­ing re­viewed as a di­ag­nos­tic for pa­tients pre­sumed to have adreno­cor­ti­cal in­suf­fi­cien­cy.

The Lake For­est, Illi­nois-based drug­mak­er said its de­vel­op­ment part­ner West Ther­a­peu­tic De­vel­op­ment had re­ceived a com­plete re­sponse let­ter from the US reg­u­la­tor, which in­di­cat­ed that that cer­tain “phar­ma­co­dy­nam­ic pa­ra­me­ters were not ad­e­quate­ly achieved” for the prod­uct.

As­ser­tio’s shares $AS­RT were down more than 28% at 84 cents pre-mar­ket on Tues­day.

The prod­uct is an in­jectable for­mu­la­tion of long-act­ing cosyn­tropin (syn­thet­ic adreno­cor­ti­cotrop­ic hor­mone, or ACTH). The ACTH stim­u­la­tion test is the most spe­cif­ic test for di­ag­nos­ing adren­al in­suf­fi­cien­cy. Blood cor­ti­sol lev­els are mea­sured be­fore and af­ter a syn­thet­ic form of ACTH is giv­en by in­jec­tion. The nor­mal re­sponse af­ter the in­jec­tion is a rise in blood cor­ti­sol lev­els. Pa­tients with adren­al in­suf­fi­cien­cy see lit­tle or no in­crease in cor­ti­sol lev­els.

Adren­al in­suf­fi­cien­cy, in­clud­ing Ad­di­son’s dis­ease, is a dis­or­der that oc­curs when the adren­al glands don’t pro­duce ad­e­quate lev­els of cer­tain hor­mones, in­clud­ing cor­ti­sol.

As­ser­tio, which re­cent­ly wooed for­mer As­traZeneca ex­ec­u­tive David Whead­on to its board, al­so has a painkiller un­der FDA re­view and an acute mi­graine ther­a­py in de­vel­op­ment.

The com­pa­ny does not de­vel­op new chem­i­cal en­ti­ties, but rather its tech­nol­o­gy is de­signed to al­low oral drugs to be re­tained in the stom­ach for a pe­ri­od that eclipses the im­me­di­ate-re­lease and some ex­tend­ed-re­lease for­mu­la­tions that cur­rent­ly ex­ist — there­by the­o­ret­i­cal­ly al­low­ing for greater ef­fi­ca­cy, in­creased tol­er­a­bil­i­ty, and the con­ve­nience of once- or twice-dai­ly dos­ing. It has three FDA ap­proved prod­ucts for neu­ro­log­i­cal con­di­tions.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Israel Lowy (Regeneron)

#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Regeneron entered the PD-(L)1 game late, so they devised a two-pronged strategy to catch up with Big Pharma rivals: They would push it into cancers where PD-1s had yet been tested, and they would prove that it’s as powerful in the big indications as any other on the market.

They cleared a hurdle on the first goal Friday, showing a 31% response in patients with the rare skin cancer basal cell carcinoma. And with the data they’re rolling out Monday, Regeneron cancer chief Israel Lowy is ready to declare success on the second.

Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two American drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend. The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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