Pfiz­er un­cou­ples from Gly­coMimet­ics and its failed sick­le cell dis­ease drug

Once large­ly over­looked, sick­le cell dis­ease (SCD) has re­cent­ly seen a flur­ry of ap­provals. For Pfiz­er — an SCD drug that has fal­tered in the piv­otal stage of de­vel­op­ment — cer­tain­ly doesn’t pass muster, not in this mar­ket. On Mon­day, the 170-year-old com­pa­ny sev­ered ties with Mary­land-based Gly­coMimet­ics, walk­ing away from the al­liance the two en­tered in­to near­ly a decade ago.

Pfiz­er tied up with the Rockville biotech to work on an ex­per­i­men­tal drug — riv­ipansel — for va­so-oc­clu­sive crises that oc­cur as a re­sult of SCD in a deal worth up to $340 mil­lion ($22.5 mil­lion up­front, with $115 mil­lion — which was lat­er ad­just­ed to $80 mil­lion — in de­vel­op­ment mile­stones) in 2011. At the time, the ther­a­py had been grant­ed or­phan drug and fast track sta­tus and was in a Phase II tri­al.

Last year, the drug failed the main and sec­ondary goals in a piv­otal late-stage study. On Mon­day, Gly­coMimet­ics — which has now re­brand­ed in­to an on­col­o­gy-fo­cused biotech with piv­otal acute myeloid leukemia tri­als on­go­ing — dis­closed that Pfiz­er had elect­ed to ter­mi­nate the agree­ment.

Sick­le cell dis­ease, which en­com­pass­es a group of in­her­it­ed red blood cell dis­or­ders that typ­i­cal­ly af­flict those of African an­ces­try, im­pacts he­mo­glo­bin — and is char­ac­ter­ized by episodes of sear­ing pain as well as or­gan dam­age.

In re­cent years — three ther­a­pies have won FDA ap­proval. The first — ap­proved in 2017 to re­duce the se­vere com­pli­ca­tions that come with SCD — is Em­maus’ En­dari, which is a pu­ri­fied (phar­ma­ceu­ti­cal grade) ver­sion of the amino acid L-glu­t­a­mine. Late last year two oth­er ther­a­pies re­ceived FDA ap­provals in quick suc­ces­sion. No­var­tis’ crizan­l­izum­ab, brand­ed Adakveo, was giv­en the FDA nod for its abil­i­ty to pre­vent va­so-oc­clu­sive crises (VOCs) —pe­ri­od­ic episodes of de­bil­i­tat­ing pain that oc­cur when sick­le-shaped red blood cells get stuck in­side blood ves­sels and de­prive the body of oxy­gen-rich blood. Soon af­ter, Glob­al Blood Ther­a­peu­tics’ vox­elo­tor, chris­tened Oxbry­ta, was ap­proved on the ba­sis of da­ta that showed it in­creased he­mo­glo­bin lev­els, al­though its use was not as­so­ci­at­ed with few­er pain crises.

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.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

UP­DAT­ED: 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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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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President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

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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#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.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.