Em­bold­ened by AS­CO da­ta, FDA etch­es speedy ap­proval path for Io­vance's cer­vi­cal can­cer ther­a­py

Io­vance’s promis­ing da­ta snap­shot de­tail­ing the po­ten­tial of its cer­vi­cal can­cer drug at AS­CO has con­vinced the FDA to ac­cel­er­ate its path to ap­proval.

On Tues­day, the drug de­vel­op­er said the US reg­u­la­tor has rec­om­mend­ed that an on­go­ing mid-stage study of its ex­per­i­men­tal ther­a­py may be suf­fi­cient to sup­port an ap­pli­ca­tion to mar­ket LN-145 in pa­tients with ad­vanced cer­vi­cal can­cer.

Maria Fardis LinkedIn

“This feed­back is en­cour­ag­ing. The abil­i­ty to use the cur­rent study, as well as the Break­through Ther­a­py des­ig­na­tion re­cent­ly grant­ed to LN-145, al­lows us to plan on a path to BLA sub­mis­sion in the sec­ond half of 2020,” Io­vance chief Maria Fardis said in a state­ment. The com­pa­ny’s shares climbed about 2.6% to $25.26 be­fore the bell.

In or­der to sup­port the ap­pli­ca­tion to mar­ket the bi­o­log­ic, Io­vance will need to en­roll be­tween 75 to 100 pa­tients in the on­go­ing tri­al, the Cal­i­for­nia-based com­pa­ny said. The es­ti­mat­ed en­roll­ment of the study, called in­no­vaTIL-04, pri­or to the FDA meet­ing was 59 pa­tients, ac­cord­ing to the clin­i­cal­tri­als.gov data­base.

Jef­feries an­a­lysts pre­dict­ed the com­pa­ny will need about 6 months of fol­low-up af­ter en­rolling the last pa­tient and sug­gest­ed that Io­vance could file with 75 pa­tients in the mid­dle of 2020 and with 100 pa­tients by the fourth quar­ter. “We be­lieve IO­VA could file based on 75 pa­tients giv­en Keytru­da re­ceived ac­cel­er­at­ed ap­proval in ad­vanced cer­vi­cal can­cer based on a PII tri­al en­rolling 77 pa­tients.”

In May, Io­vance gar­nered en­thu­si­asm af­ter it re­port­ed da­ta from 27 evalu­able cas­es of ad­vanced cer­vi­cal can­cer. The biotech­nol­o­gy com­pa­ny’s tu­mor in­fil­trat­ing lym­pho­cyte (TIL) ther­a­py in­duced a 44% ob­jec­tive re­sponse rate (ORR) — in­clud­ing one com­plete re­sponse, 9 par­tial re­sponse and two un­con­firmed par­tial re­spons­es. Mer­ck’s Keytru­da — the cur­rent stan­dard of care in this pa­tient pop­u­la­tion — was ap­proved as a sec­ond-line cer­vi­cal can­cer ther­a­py with a 14% ORR.

Jef­feries’ Biren Amin es­ti­mat­ed peak sales of $382 mil­lion for LN-145 by 2030 based on about 4600 sec­ond-line ad­vanced cer­vi­cal can­cer pa­tients and 50% mar­ket pen­e­tra­tion, us­ing a con­ser­v­a­tive per year price of ~$250,000 per pa­tient (and no an­nu­al price in­creas­es).

Boris Peak­er Cowen

LN-145 is al­so be­ing test­ed for use in head and neck can­cer.

In the ear­ly stages of can­cer, the im­mune sys­tem at­tacks its preda­tor by mo­bi­liz­ing lym­pho­cytes to at­tack and in­fil­trate the tu­mor. How­ev­er, the de­fen­sive abil­i­ty of these tu­mor-in­fil­trat­ing lym­pho­cytes (TILs) is typ­i­cal­ly brief as can­cer cells quick­ly mu­tate, evad­ing de­tec­tion. Io­vance’s tech­nol­o­gy is en­gi­neered to un­sheath the cloak of in­vis­i­bil­i­ty worn by can­cer cells to su­per­charge TIL ef­fi­ca­cy. The com­pa­ny first ex­tracts TILs from the pa­tient and ex­pands them in a lab to the tune of bil­lions. The am­pli­fi­ca­tion process elim­i­nates the im­mune-sup­pres­sive en­vi­ron­ment cre­at­ed by the tu­mor and en­hances the repli­ca­tion and ac­ti­va­tion of ag­gres­sive an­ti-tu­mor TIL. Once a sur­feit of TILs has been cul­tured, the cells are re-in­fused in­to the pa­tient.

Da­ta pre­sent­ed at AS­CO from an­oth­er study showed Io­vance’s lead ex­per­i­men­tal ther­a­py, li­fileu­cel, con­ferred an ORR of 38% (2 com­plete re­spons­es and 23 par­tial re­spons­es) in 66 con­sec­u­tive­ly dosed post-PD-1 pa­tients with late-stage un­re­sectable melanoma.

Io­vance’s an­nounce­ment on Tues­day is pos­i­tive, but “large­ly in­line with in­vestor ex­pec­ta­tions post AS­CO. This al­so ex­pe­dites that cer­vi­cal can­cer time­lines in­line with the melanoma study, both of which are now ex­pect­ed to re­port fi­nal re­sults and BLA fil­ing in 2H20,” Cowen’s Boris Peak­er wrote in a note.

So­cial im­age: An­drew Harnik, AP Im­ages

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

FDA plans new stud­ies on ac­cel­er­at­ed ap­proval dis­clo­sures in bio­phar­ma ads

When people read biopharma companies’ websites about new drugs approved via the FDA’s accelerated pathway, like Biogen’s new Alzheimer’s drug, do they understand that these drugs may only be reasonably likely to predict clinical benefit and still require confirmatory studies?

That’s what the FDA’s Office of Prescription Drug Promotion wants to firm up as an agency analysis of direct-to-consumer websites for accelerated approval drugs previously found that only 21% of the disclosures used language directly from the label.

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An ex­pen­sive watch, shell com­pa­nies and fake in­voic­es: How two Is­raeli traders tapped in­to a $100M glob­al biotech in­sid­er trad­ing ring

It appears that we have reached the end of the saga about the global insider trading ring that collectively reaped $100 million from placing “timely, profitable” trades in biotech stocks like Ariad, Pharmacyclics and Receptos.

Tomer Feingold and Dov Malnik — Israeli traders living in Switzerland — were the last out of eight to be charged as the SEC unraveled the scheme, which ran from 2013 through 2017. Together, according to a statement in March, the pair had pocketed more than $4 million.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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