Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

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)

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi calls it quits on mR­NA Covid-19 shots, scrap­ping vac­cine from $3.2B Trans­late Bio buy­out

Sanofi is throwing in the towel on mRNA-based Covid-19 vaccines.

The French drugmaker will halt development on its unmodified mRNA Covid-19 shot despite what it said were positive Phase I/II results, a spokesperson told Endpoints News on Tuesday morning. Sanofi said the reason it’s stopping the Covid-19 mRNA program, developed in partnership with its new $3.2 billion acquisition Translate Bio, is because the market is too crowded.

Brian Hubbard, Anji Pharmacetuticals CEO

Look­ing to rewrite the rules of drug li­cens­ing, start­up An­ji is on the hunt for 'dy­nam­ic eq­ui­ty' joint ven­tures

Licensing is one of the most common ways big drugmakers leverage biotech innovation to drive gains across their pipelines — and the structure of those deals is pretty well established. But one biotech with home bases in China and the US thinks it may have a better way.

On Tuesday, Cambridge-based biotech Anji Pharma closed a $70 million Series B with two late-stage molecules in the fold and a mission to rewrite the rules of drug licensing through what it calls “dynamic equity” deals and a joint venture-heavy game plan. The round was funded in whole by Chinese hedge fund CR Capital.

Con­tract re­search is hav­ing a mo­ment right now. Will M&A splash­es dri­ve the in­dus­try to even greater heights?

Contract research organizations are a fairly mysterious bunch. They’re typically considered the skilled laborers behind big drug development — the stage crews who run the trials behind some of the most (and least) successful data reveals in biopharma history.

But all that is changing.

This year, a couple of huge, out-of-the-blue M&A deals sounded the alarm on just how much money is flying around in this corner of the industry.

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Biohaven CEO Vlad Coric

Bio­haven turns out a dud in fa­tal neu­rode­gen­er­a­tive dis­ease, cast­ing doubt on drug's chances in ALS

With one migraine drug on the market, Biohaven has gone big with its next phase of growth, targeting major neurodegenerative diseases in Alzheimer’s and ALS. The former effort has already gone up in smoke, and a new failure elsewhere in the pipeline could now bode poorly for the latter.

Biohaven’s verdiperstat failed to outperform placebo in reducing the symptoms of patients with multiple system atrophy (MSA), a rare and fatal neurodegenerative disorder, according to a “focused analysis” of the drug’s Phase III trial in that disease toplined Monday.

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