Io­vance shares ham­mered on TIL ther­a­py fil­ing de­lay — al­though an­a­lysts aren't as both­ered

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

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.