Adrian Rawcliffe, Adaptimmune CEO

Adap­ti­m­mune says it's (near­ly) ready to head to the FDA with TCR can­cer ther­a­py

Six months af­ter an ini­tial re­veal at AS­CO, Adap­ti­m­mune is back in the lime­light with new da­ta Thurs­day. And the com­pa­ny be­lieves it now has enough to go to reg­u­la­tors for what would be the first-ever TCR ther­a­py.

The biotech’s afa­mi-cel pro­gram put out up­dat­ed fig­ures Thurs­day morn­ing with re­searchers not­ing as of Sept. 1, 16 of 47 pa­tients saw their tu­mors shrink while on the ex­per­i­men­tal treat­ment. It’s good enough for a 34% over­all re­sponse rate and, while low­er than the May up­date of 39%, makes Adap­ti­m­mune con­fi­dent enough to say the study will reach its pri­ma­ry end­point.

Though the fi­nal da­ta cut is ex­pect­ed by the end of the year, “it is math­e­mat­i­cal­ly im­pos­si­ble not to meet the pri­ma­ry end­point,” CEO Adri­an Raw­cliffe told End­points News. Fol­low­ing dis­cus­sions with the FDA, the biotech had set an 18% re­sponse rate as an ac­cept­able thresh­old to meet the pri­ma­ry.

“If you as­sume all fu­ture pa­tients will not re­spond,” he added, “the ones that have yet to be as­sessed, the tri­al will still meet the pri­ma­ry end­point.”

De­spite Raw­cliffe’s rosy pic­ture, in­vestors didn’t ap­pear too con­vinced as Adap­ti­m­mune shares $ADAP closed down about 9% in Thurs­day trad­ing.

As the tri­al ap­proach­es its end, Raw­cliffe not­ed that the way in which re­spons­es are clas­si­fied may have changed for some pa­tients as in­de­pen­dent in­ves­ti­ga­tors chimed in, lead­ing to the low­er nu­mer­i­cal fig­ure. Raw­cliffe said it’s the main rea­son two pa­tients who were pre­vi­ous­ly said to have achieved com­plete re­spons­es are no longer la­beled as such.

Nev­er­the­less, the biotech is pre­pared to sub­mit its BLA next year once the fi­nal da­ta are ready. Raw­cliffe de­scribed Adap­ti­m­mune is “very com­fort­able” with the whole da­ta pack­age as it stands and high­light­ed the study’s dura­bil­i­ty mea­sures. Even though 75% of re­spon­ders are still re­ceiv­ing treat­ment, the tri­al has not reached me­di­an du­ra­tion of re­sponse.

Pa­tients’ re­sponse times stretch from about four weeks to more than 65 weeks.

Adap­ti­m­mune had en­rolled pa­tients with two types of sar­co­mas: ad­vanced syn­ovial sar­co­ma or myx­oid/round cell li­posar­co­ma. By each can­cer, the ORR was 36% in syn­ovial sar­co­ma pa­tients and 25% for those with MR­CLS. Pa­tients with these can­cers gen­er­al­ly don’t have many treat­ment op­tions af­ter chemo, Raw­cliffe said, and the five-year mor­tal­i­ty rate is around 70-80%.

Now look­ing to­ward its reg­u­la­to­ry fu­ture, Adap­ti­m­mune may like­ly face sim­i­lar ques­tions as oth­er com­pa­nies that have brought forth cell ther­a­pies. TCR is sim­i­lar to CAR-Ts, in that a pa­tient’s cells are re­moved, re-en­gi­neered and re­placed to tar­get can­cer cells, but TCR goes af­ter a dif­fer­ent pro­tein.

TCRs have his­tor­i­cal­ly been met with a lot of skep­ti­cism, both from re­searchers and in­vestors, but this is the sec­ond time this week a biotech is tout­ing new da­ta. Im­mat­ics re­vealed an ear­ly-stage study that saw eight of 16 pa­tients reg­is­ter an ob­jec­tive re­sponse against a range of sol­id tu­mor types on Tues­day.

Raw­cliffe him­self ad­mit­ted the com­pa­ny has al­ready been asked about its CMC process­es. It’s been at the top of the to-do list in the six months since AS­CO, how­ev­er, and the CEO is con­fi­dent every­thing will be in or­der when the FDA comes knock­ing.

“There’s been five cell ther­a­pies ap­proved to date, and they all came with chal­lenges of their own,” Raw­cliffe said. “Very few of those chal­lenges were re­lat­ed to the clin­i­cal da­ta, so any­body who tells you this is easy, or that there is ze­ro risk as­so­ci­at­ed with it, is liv­ing in cloud cuck­oo land.”

Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

Steve Worland, eFFECTOR CEO

Sur­prise piv­ot rocks eF­FEC­TOR's I/O plans — al­though ex­ecs promise big­ger slice of the NSCLC mar­ket in the long run

When eFFECTOR Therapeutics went public last summer on the coattails of a reverse merger with Locust Walk’s SPAC, the potential of its lead drug, tomivosertib, as a combo agent with Merck’s flagship PD-1 Keytruda was hailed as the main draw.

But the biotech is now axing those plans and essentially starting over.

In a surprise move, San Diego-based eFFECTOR said it’s halting the development of tomivosertib in non-small cell lung cancer patients who have already progressed on Keytruda monotherapy after running into enrollment challenges in a Phase IIb trial.

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Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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