Merdad Parsey, Gilead CMO

Crown jew­el in big Gilead buy­out lands break­through des­ig­na­tion for MDS

Months af­ter Gilead snagged ma­grolimab in the $4.9 bil­lion buy­out of Cal­i­for­nia biotech Forty Sev­en — the com­pa­ny’s largest ac­qui­si­tion in three years at the time — the an­ti-CD47 mon­o­clon­al an­ti­body is now on an ex­pe­dit­ed track to ap­proval.

On Tues­day morn­ing, Gilead an­nounced ma­grolimab’s FDA break­through ther­a­py des­ig­na­tion for pa­tients with new­ly-di­ag­nosed myelodys­plas­tic syn­drome (MDS). No new MDS treat­ments have been ap­proved in 14 years, ac­cord­ing to the com­pa­ny.

Forty Sev­en was was found­ed in 2014 to de­vel­op drugs tar­get­ing CD47, the “don’t eat me sig­nal” used by can­cer cells to avoid the im­mune sys­tem. The biotech brought its first cut of pos­i­tive da­ta for ma­grolimab to ASH last fall, which sent its share price soar­ing as Gilead neared a buy­out.

Af­ter a few failed of­fers, Gilead end­ed up pay­ing $95 a share — a long way from Forty Sev­en’s dip to $6 a share in Oc­to­ber.

The drug’s break­through des­ig­na­tion was based on a Phase Ib study which showed a 91% ob­jec­tive re­sponse rate in in­ter­me­di­ate-, high- and very high-risk MDS pa­tients when ad­min­is­tered in com­bi­na­tion with azac­i­ti­dine. Forty-two per­cent of pa­tients ex­pe­ri­enced com­plete re­mis­sion, ac­cord­ing to the com­pa­ny.

“The Break­through Ther­a­py des­ig­na­tion rec­og­nizes the po­ten­tial for ma­grolimab to help ad­dress a sig­nif­i­cant un­met med­ical need for peo­ple with MDS and un­der­scores the trans­for­ma­tive po­ten­tial of Gilead’s im­muno-on­col­o­gy ther­a­pies in de­vel­op­ment,” Gilead CMO Mer­dad Parsey said.

A Phase III EN­HANCE tri­al is un­der­way in pre­vi­ous­ly un­treat­ed high­er risk MDS pa­tients. The drug was al­so grant­ed fast track des­ig­na­tion for the po­ten­tial treat­ment of MDS, acute myeloid leukemia (AML), dif­fuse large B-cell lym­phoma (DL­B­CL) and fol­lic­u­lar lym­phoma. It has or­phan drug des­ig­na­tion by the FDA for MDS and AML, and by the EMA for AML.

Mark Chao

“We haven’t pro­vid­ed time­lines yet in terms of when those might read out,” Mark Chao, Gilead’s VP of on­col­o­gy clin­i­cal re­search and clin­i­cal lead for the ma­grolimab pro­gram, said over the phone. Chao co-found­ed Forty Sev­en, then made the jump to Gilead.

At the time, the Forty Sev­en buy­out was the largest ac­qui­si­tion since the $11.9 bil­lion Kite Phar­ma pur­chase in 2017. The Forty Sev­en team won big in the deal — co-founders Irv Weiss­man and Ravin­dra Ma­jeti took home $194 mil­lion and $123 mil­lion re­spec­tive­ly, and CEO Mark Mc­Camish earned a $105 mil­lion pay­out.

“You know, one of the rea­sons that we’re ex­cit­ed about this is … we’ve seen strong ac­tiv­i­ty,” Chao said, cit­ing the Phase Ib ef­fi­ca­cy da­ta. “We think that that’s ac­tu­al­ly pret­ty ben­e­fi­cial in the sense that azac­i­ti­dine alone gen­er­al­ly has much low­er com­plete re­mis­sion rates and re­sponse rates.”

The drug was well-tol­er­at­ed, and just 1% of pa­tients dis­con­tin­ued treat­ment due to an ad­verse event that was linked to the stan­dard drug, ac­cord­ing to Chao.

On Sun­day, Gilead an­nounced it struck a deal to buy out Im­munoMedics for $21 bil­lion, which will give it Trodelvy, new­ly ap­proved for metasta­t­ic triple neg­a­tive breast can­cer.

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.

Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

#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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#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.

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.