Bris­tol My­ers snares an­oth­er ap­proval from the old Cel­gene pipeline, nab­bing an over­looked OK

Bris­tol My­ers Squibb has got­ten an­oth­er ap­proval from the old Cel­gene pipeline yes­ter­day, this time for a once low-pro­file com­pound.

The FDA green-light­ed Onureg, an oral drug pre­vi­ous­ly known as CC-486. It’s chem­i­cal­ly known as azac­i­ti­dine — the same com­pound that in its IV form, brand­ed as Vi­daza, has long been used to treat acute myeloid leukemia and myelodys­plas­tic syn­dromes. For Bris­tol My­ers, it marks the sec­ond ma­jor ap­proval since the Cel­gene merg­er was com­plet­ed, af­ter the mul­ti­ple scle­ro­sis drug Zeposia was OK’d ear­li­er this year.

With three CVR drugs — ide-cel CAR-T,  liso-cell CAR-T and Zeposia — grab­bing the spot­light as the two sides ham­mered out merg­er talks last year, CC-486 re­ceived lit­tle press un­til the fall. But at ASH last De­cem­ber, the com­pa­nies showed off over­whelm­ing­ly pos­i­tive da­ta vs. place­bo from a Phase III tri­al in AML pa­tients. In the 472-per­son study, me­di­an over­all sur­vival was 24.7 months for those in the treat­ment arm, 14.8 months for those on place­bo.

The FDA grant­ed pri­or­i­ty re­view on May 1 off that da­ta, and ap­proved it just three months lat­er.

The drug al­ready has some proven mar­ket po­ten­tial; at its peak, Vi­daza brought in $600 mil­lion per year for Cel­gene, which ac­quired the com­pound in a $2.9 bil­lion buy­out of Pharmion in 2007. And in an ed­i­to­r­i­al in Blood last March, An­drew Wei, an on­col­o­gist at Aus­tralia’s Monash Uni­ver­si­ty, said that if the CC-486 Phase III re­sults were pos­i­tive, main­te­nance ther­a­py would “like­ly … be ac­cept­ed as a new stan­dard of care.”

On the oth­er hand, pa­tients now have more op­tions than they did in 2007. For one, there’s Id­hi­fa, the drug Agios and Cel­gene got ap­proved three years ago and that Bris­tol My­ers al­so picked up in the merg­er. Roche won an OK for Ven­clex­ta the fol­low­ing year, and they have an MDM2 in­hibitor called idasanut­lin that is now in Phase II stud­ies.

Bris­tol My­ers is al­so look­ing to ex­pand the po­ten­tial in­di­ca­tion for the drug. Phase II­Is are on­go­ing test­ing the drug in lym­phoma and MDS.

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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Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

Norbert Bischofberger, Kronos CEO

Three more biotechs look to jump on­to Nas­daq amid IPO boom, in­clud­ing Nor­bert Bischof­berg­er's Kro­nos

Three drug developers announced plans to go public on Friday, a sign that the IPO window for biopharma is wide open.

First up is Daly City, CA-based Spruce Biosciences. They filed for an $86 million IPO to develop their pipeline for classic congenital adrenal hyperplasia (CAH). Currently, only steroids are available to treat the condition, which affects the adrenal glands above the kidneys. Spruce’s tildacerfont, a non-steroidal option, is in a Phase IIb trial in adults with classic CAH and poor disease control. The company expects a topline readout here in the next 12 to 15 months. The small molecule is also in a Phase IIb study in adults with classic CAH and good disease control. Spruce expects topline data here in the first half of 2022.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.