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)

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

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.

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.