We now know the full, ear­ly-stage sto­ry be­hind the drug that in­spired Gilead CEO Dan O’Day’s re­cent $5 bil­lion ac­qui­si­tion of Forty Sev­en.

Fol­low­ing up on their AS­CO ab­stract from a cou­ple of weeks ago, the team at Forty Sev­en is mak­ing their re­turn ap­pear­ance this week hold­ing clear­ly promis­ing ear­ly-stage da­ta on their lead drug ma­grolimab as they pon­der whether they should roll on a quest to ob­tain an ac­cel­er­at­ed ap­proval.

Mark Chao

“I think the ef­fi­ca­cy is there. This Phase Ib could sup­port sin­gle-arm ac­cel­er­at­ed ap­proval,” says Mark Chao, a Forty Sev­en founder and clin­i­cal de­vel­op­ment chief at the biotech sub. “Our drug ac­tu­al­ly has ac­tiv­i­ty in very hard to treat pa­tients.”

Irv Weiss­man

That makes this drug a stand­out among the CD47 “don’t eat me” path­way crowd, which has not mea­sured up in many oth­er cas­es. This drug is the brain­child of Stan­ford’s leg­endary Irv Weiss­man, and it may be his lega­cy.

At ASH last fall, Forty Sev­en made a huge splash with its first cut of the pos­i­tive da­ta, spurring a soar­ing share price as Gilead closed in for the buy­out.

The new da­ta al­so un­der­score the promise this drug has in oth­er can­cers, Chao adds, as Gilead and the new crew at Forty Sev­en look to ex­pand their hori­zons.

Re­searchers have da­ta on 68 pa­tients who have been treat­ed with ma­grolimab plus azac­i­ti­dine in a sin­gle arm study in­volv­ing cas­es of un­treat­ed high­er-risk myelodys­plas­tic syn­drome (MDS) and acute myeloid leukemia.

Of the 33 MDS pa­tients, 30 (91%) scored an ob­jec­tive re­sponse, with 14 (42%) demon­strat­ing a com­plete re­sponse. And there was ev­i­dence of a deep­en­ing re­sponse, with 56% of MDS pa­tients achiev­ing a CR af­ter a min­i­mum of 6 months of fol­lowup.

On the AML side, 64% (n=16/25) of pa­tients evalu­able for ef­fi­ca­cy achieved an ob­jec­tive re­sponse, in­clud­ing 56% (n=14/25) with a CR or a CR with in­com­plete blood count re­cov­ery. And there was a sub­set of high-risk pa­tients who did par­tic­u­lar­ly well: TP53-mu­tant AML (n=12), with 75% achiev­ing a CR or CRi. Me­di­an du­ra­tion of re­sponse and me­di­an over­all sur­vival have not yet been reached in MDS, AML or TP53- mu­tant AML.

“The sig­nal is re­al and con­tin­ues to be en­cour­ag­ing,” adds Chao, “en­cour­ag­ing on where else we could go.”

One of the next stops is at the FDA, where they’ll be fol­low­ing up with reg­u­la­tors who have been ea­ger to try out new can­cer drugs where physi­cians have few choic­es.

So­cial: Dan O’Day, Gilead CEO (Key­stone via AP)

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.