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

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.