Strug­gling Adap­ti­m­mune gets a boost from the FDA as T cell can­cer drug wins an in­side track at the agency

Adap­ti­m­mune has been strug­gling as of late, los­ing its R&D chief dur­ing a CEO tran­si­tion as its share price $ADAP steadi­ly drift­ed down to alarm­ing lows. But the strug­gling biotech got a much-need­ed boost to­day from the FDA, which put one of its top can­cer drugs on the in­side track re­served for drugs that qual­i­fy as a Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py.

Adri­an Raw­cliffe

RMAT — which ex­tends to cell ther­a­pies — hasn’t re­ceived near­ly as much at­ten­tion as the FDA’s much cool­er sound­ing break­through ther­a­py des­ig­na­tion, but it comes with much the same open-door in­vi­ta­tion from the agency, an op­por­tu­ni­ty to dis­cuss fast-track­ing sur­ro­gate end­points, and a chance to shoot ahead in gain­ing a mar­ket­ing de­ci­sion.

And Adap­ti­m­mune — now helmed by Adri­an Raw­cliffe, who stepped up to re­place found­ing CEO James No­ble — needs to prove it­self. Shares are down 91% from last fall’s spike.

The RMAT des­ig­na­tion goes to ADP-A2M4, one of the biotech’s top in-house TCR pro­grams af­ter they hand­ed the lead — NY-ESO — to their part­ners at Glax­o­SmithK­line. They won the des­ig­na­tion off of some very ear­ly-stage da­ta in syn­ovial sar­co­ma, where ex­ecs be­lieve they can show clear head­way in de­vel­op­ing a T cell ther­a­py for sol­id tu­mors. In this case, the drug tar­gets MAGE-A4, a com­mon anti­gen.

The biotech has on­ly put out da­ta on 14 pa­tients, but there’s some promise there, with a 50% par­tial tu­mor re­sponse (con­firmed as well as un­con­firmed, it should be not­ed) in 7 of the pa­tients. It’s now in Phase II, with a PD-1 com­bo ap­proach be­ing as­sem­bled on a par­al­lel track, and Adap­ti­m­mune is promis­ing to have this on the mar­ket as a ground­break­ing new ther­a­peu­tic ap­proach in 2022.

Adap­ti­m­mune has won some cau­tious sup­port from an­a­lysts on the da­ta up­dates, with some con­cerns about this new drug’s abil­i­ty to tran­scend var­i­ous tu­mors apart from sar­co­ma. And the biotech has quite a moun­tain to climb. Its stock bumped up slight­ly Tues­day morn­ing, with a 5% jump.

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.

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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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