Af­ter abrupt­ly pulling its mar­ket­ing ap­pli­ca­tion, DBV may be left with peanuts in race against Aim­mune

They say slow and steady wins the race. In the sprint to mar­ket the first peanut al­ler­gy treat­ment, hare DBV Tech­nolo­gies $DB­VT has just fum­bled, an­nounc­ing on Wednes­day that it had re­scind­ed the mar­ket­ing ap­pli­ca­tion for its peanut al­ler­gy patch, fol­low­ing dis­cus­sions with FDA reg­u­la­tors who are seem­ing­ly un­hap­py with the state of man­u­fac­tur­ing and qual­i­ty con­trol da­ta sub­mit­ted. Mean­while, tor­toise Aim­mune $AIMT is now in pole po­si­tion to leapfrog its ri­val to se­cure first-mover ad­van­tage in the so-far un­der­served mar­ket.

To be sure, both com­pa­nies have had their share of ups and downs. In Oc­to­ber 2017, DBV re­port­ed its treat­ment — dubbed Vi­askin Peanut — was un­able to mark a sta­tis­ti­cal­ly sig­nif­i­cant sep­a­ra­tion from a place­bo in a Phase III tri­al, oblit­er­at­ing its shares, al­though the Parisian drug de­vel­op­er said it would march ahead with a mar­ket­ing ap­pli­ca­tion. Four months lat­er in 2018, arch ri­val Aim­mune came out with piv­otal da­ta on its drug, AR101, which were de­cid­ed­ly pos­i­tive, but in­vestors took is­sue with the mag­ni­tude of pa­tients that dropped out of the drug arm. In re­sponse, Nestlé Health Sci­ence-backed Aim­mune al­so so­lid­i­fied its plans to sub­mit its ap­pli­ca­tion, which is ex­pect­ed by the end of 2018.

Daniel Tassé

“Al­though the agency did not ref­er­ence any med­ical or clin­i­cal ques­tions with the sub­mis­sion of Vi­askin Peanut, the FDA did com­mu­ni­cate that the lev­el of de­tail with re­gards to da­ta on man­u­fac­tur­ing and qual­i­ty con­trols was in­suf­fi­cient in the BLA,” said DBV chief Daniel Tassé said in a state­ment on Wednes­day.

JMP an­a­lyst Li­isa Bayko was not as con­vinced.  “Man­age­ment as­sumes that there are no con­cerns around Vi­askin Peanut’s clin­i­cal mod­ule be­cause there were no ques­tions from the FDA re­gard­ing that sec­tion of the BLA. How­ev­er, the com­pa­ny nev­er di­rect­ly asked if the rest of the BLA was ac­cept­able for re­view,” she wrote in a note.

Un­sur­pris­ing­ly the two com­pa­nies’ shares were trad­ing in op­po­site di­rec­tions: DBV’s stock crashed near­ly 49% pre-mar­ket, while Aim­mune’s shares climbed about 6% be­fore the bell.

An­a­lysts keen­ly watch­ing the tri­als and tribu­la­tions of the pair of drug de­vel­op­ers in­di­cat­ed that Aim­mune is now well-poised to be­come the first FDA-ap­proved treat­ment. They al­so ex­pressed out­rage that DBV pro­vid­ed no clear time­line on when and how the ap­pli­ca­tion’s is­sues would be re­solved.

Derek Archi­la

“We are sur­prised this type of in­for­ma­tion would not have been dis­cussed with the agency pri­or to the BLA sub­mis­sion (com­pa­ny has fast track and break­through sta­tus al­low­ing them in­creased agency in­ter­ac­tion) to en­sure the re­quired da­ta was part of the pack­age…this event will on­ly in­crease in­vestors’ skep­ti­cism on man­age­ment’s abil­i­ty to ex­e­cute on its reg­u­la­to­ry ini­tia­tives, which were al­ready con­tro­ver­sial,” Stifel’s Derek Archi­la wrote.

“While DB­VT’s new CEO in­di­cat­ed he an­tic­i­pates be­ing able to pro­vide the ad­di­tion­al in­for­ma­tion need­ed to sup­port the re-fil­ing of the BLA, he of­fered no clear time­lines for a BLA re-sub­mis­sion nor tim­ing on when he ex­pects to up­date in­vestors. The CEO on­ly in­di­cat­ed on the call it would not be “un­rea­son­able” to ex­pect an up­date from the com­pa­ny dur­ing 1Q19, but ul­ti­mate­ly we don’t know when it will oc­cur.”

Bri­an Sko­r­ney

Baird’s Bri­an Sko­r­ney was even more skep­ti­cal of DBV’s for­tunes, sug­gest­ing the de­lay could spell fur­ther dis­as­ter:

(T)his dy­nam­ic like­ly low­ers the prob­a­bil­i­ty that FDA would be will­ing to over­look the Phase 3 fail­ure. Un­der a 1H19 re­view, an Ad­Com would like­ly be set up with the com­pa­ny and pa­tient ad­vo­cates mak­ing the case that “there is noth­ing else avail­able”. Un­der any like­ly re­sub­mis­sion time­line, AR101 is like­ly to have been ap­proved by a Vi­askin Ad­Com and the FDA may be less flex­i­ble when a stan­dard of care ex­ists.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Sanofi, Re­gen­eron etch out an­oth­er PhI­II vic­to­ry for Dupix­ent, eas­ing se­vere itch and clear­ing le­sions

Sanofi and Regeneron can boast of another inflammatory disease where Dupixent has proven effective.

The best-selling drug, which targets both IL-4 and IL-13, has delivered a clean sweep in a Phase III trial for prurigo nodularis, a chronic disease characterized by itch so intense that it can affect patients’ sleep and psychology. Thick skin lesions can cover most of the body.

On the primary endpoint, 37% of patients taking Dupixent saw a clinically meaningful reduction in itch compared to 22% of those on placebo (p=0.0216) at week 12. All secondary endpoints were also met, including clearance of skin lesions and improvement in quality of life.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Oc­u­lar Ther­a­peu­tix ham­mered by a PhII fail­ure in dry eye dis­ease — shares tank

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.