Io­vance won’t be able to file for its first-ever ap­proval by the end of this year af­ter all.

At is­sue is the po­ten­cy as­says Io­vance is us­ing to de­fine what would be the first-ever tu­mor-in­fil­trat­ing lym­pho­cyte (TIL) ther­a­py. Reg­u­la­tors want to have more da­ta on the cur­rent as­says, or po­ten­tial­ly see dif­fer­ent as­says in the BLA, the biotech sug­gest­ed.

Maria Fardis

All of that work would push the BLA sub­mis­sion in metasta­t­ic melanoma to 2021, when Io­vance has com­plet­ed the re­quired clin­i­cal fol­low-up and fin­ished re­fin­ing its as­says as well as de­vel­op­ing al­ter­na­tive ones. That means a launch won’t hap­pen un­til 2022 at the ear­li­est, with cer­vi­cal can­cer to fol­low in the same year.

Chem­istry, man­u­fac­tur­ing and con­trol is­sues have loomed large among late-stage de­vel­op­ers of cell ther­a­py since the FDA slammed Bris­tol My­ers and blue­bird with a sur­prise refuse-to-file let­ter cit­ing a faulty sec­tion on CMC. The com­pa­nies still man­aged to sort it out and se­cure pri­or­i­ty re­view for the BC­MA CAR-T, ide-cel, but not af­ter five months of Cel­gene in­vestors fret­ting about the fate of their $7 bil­lion CVR pay­out.

An­a­lysts sus­pect­ed it was al­so a key rea­son why J&J-part­nered Leg­end Biotech pushed back their EMA fil­ing — to pre­pare a strong CMC pack­age and stay on the safe side.

For Io­vance the de­lay trig­gered a steep drop in stock price, as in­vestors trad­ed shares $IO­VA down 20.46% to $25.50.

Most an­a­lysts, though, ap­pear to echo Chardan an­a­lyst Geu­lah Livshits’ as­sess­ment that “this is a solv­able (but not triv­ial) is­sue.”

“In our view, the key word is ‘re­fine,’ which does not sug­gest the re­quire­ment for ad­di­tion­al clin­i­cal stud­ies or sub­stan­tive work,” Mizuho an­a­lyst Mara Gold­stein wrote in a note. “How­ev­er, this does raise the un­cer­tain­ty pro­file as the ex­act point of con­cern is un­known to us.”

Un­like CAR-T, TILs are non-en­gi­neered and more het­ero­ge­neous; mea­sur­ing the po­ten­cy is thus more com­plex. Io­vance is al­so go­ing af­ter sol­id tu­mors with li­fileu­cel, CEO Maria Fardis not­ed in a state­ment.

Biren Amin of Jef­feries ex­pects a de­lay be­tween four and six months. While he pre­vi­ous­ly pre­dict­ed 2021 and 2022 sales of $61 mil­lion and $458 mil­lion, re­spec­tive­ly, he’s now di­al­ing down those es­ti­mates to $211 mil­lion for 2022.

So­cial: Shut­ter­stock

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.

The biotech industry has seen no shortage of innovation in recent years, but in one area — drug pricing — the field has been anything but innovative. Now, two brash startups taking different roads to upset the drug pricing model will partner up to create a sort of “super-disruptor.”

EQRx and UK-based AI specialist Exscientia will team up on a discovery-through-commercialization collaboration the partners hope will work better than the sum of its parts to bring cheaper medicines to patients faster, the companies said Thursday.