De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When Re­genxBio dis­closed that the FDA had placed a par­tial clin­i­cal hold on one of its lead gene ther­a­pies, ex­ecs out­lined sev­er­al cus­tom­ary next steps: con­tin­u­ing as­sess­ment and mon­i­tor­ing, de­lay­ing a re­lat­ed IND fil­ing, and work­ing with the FDA to ad­dress the mat­ter.

As it turned out, they were plan­ning some­thing much less mun­dane. Two days af­ter an­nounc­ing the hold in its Q3 up­date, Re­genxBio filed a law­suit seek­ing to set it aside, the FDA Law Blog not­ed.

The law­suit shed light on the in­ter­ac­tions be­tween the biotech and reg­u­la­tors, re­veal­ing that there was ac­tu­al­ly a full clin­i­cal hold on the di­a­bet­ic retinopa­thy tri­al in ad­di­tion to the par­tial hold on wet age-re­lat­ed mac­u­lar de­gen­er­a­tion and Re­genxBio with­drew the IND.

Ac­cord­ing to the com­plaint, the com­pa­ny had dis­cussed re­sults from its Phase I/IIa tri­al with the FDA and was on track to be­gin the next phase be­fore the end of 2019.

But on Oc­to­ber 18, 2019, with­out no­tice or ex­pla­na­tion, FDA placed RGX-314 on a clin­i­cal hold, ef­fec­tive­ly halt­ing RE­GENXBIO’s de­vel­op­ment of this po­ten­tial­ly life-al­ter­ing treat­ment for reti­nal dis­eases that are lead­ing caus­es of adult blind­ness. Since is­su­ing the clin­i­cal hold or­der, FDA has re­buffed RE­GENXBIO’s re­peat­ed at­tempts to ob­tain an ex­pla­na­tion of the ba­sis for the clin­i­cal hold.

Fail­ing to pro­vide ad­vance warn­ing or ex­pla­na­tion for the hold, Re­genxBio claims, is in vi­o­la­tion of the FDA’s own reg­u­la­tions. And that led to an “ar­bi­trary and capri­cious” fi­nal de­ci­sion, they wrote.

But that’s not it.

Since they didn’t get a chance to re­view or re­but the rea­sons for the hold, which harmed their rep­u­ta­tion and prop­er­ty in­ter­est in RGX-314, Re­genxBio be­lieves the FDA vi­o­lat­ed the Fifth Amend­ment’s Due Process Clause. Fur­ther­more, they charged a par­tic­u­lar sec­tion of the Food, Drug, and Cos­met­ic Act rep­re­sent­ed an un­con­sti­tu­tion­al vest­ing of leg­isla­tive pow­er in the Sec­re­tary of Health and Hu­man Ser­vices.

Along with the agency, the fed­er­al gov­ern­ment, HHS Sec­re­tary Alex Azar, act­ing FDA com­mis­sion­er Brett Giroir (who has tak­en over from Ned Sharp­less while Stephen Hahn goes through the con­fir­ma­tion process), and FDA reg­u­la­to­ry project man­ag­er Ed­ward Thomp­son were al­so named as de­fen­dants. Thomp­son al­leged­ly first no­ti­fied Re­genxBio of the holds cit­ing “is­sues as­so­ci­at­ed with [RGX-314’s] de­liv­ery sys­tems.”

Fol­low­ing mul­ti­ple ex­changes, the FDA ap­par­ent­ly told Re­genxBio that it would pro­vide a writ­ten ex­pla­na­tion of the ba­sis for the hold by this Fri­day, No­vem­ber 15.

Why risk the ire of reg­u­la­tors when an up­date is due so soon? As a pro­ce­dur­al mat­ter to pre­serve their rights, the com­pa­ny said.

“This ac­tion was tak­en on the rec­om­men­da­tion from coun­sel as we con­tin­ue to work with the FDA to ad­dress this mat­ter, and we hope this step will help en­sure the FDA will pro­vide Re­genxBio with their spe­cif­ic con­cerns about the un­spec­i­fied de­vice,” it wrote in an email to End­points News.

At­tor­ney Deb­o­rah Livor­nese of­fered this take on the FDA Law Blog:

While it seems un­like­ly that the mer­its of the clin­i­cal hold will be re­solved through the ju­di­cia­ry process in a help­ful time­frame, the com­plaint has like­ly in­creased the chances that FDA will re­spond with a thor­ough ex­pla­na­tion of its rea­son for the hold when it does pro­vide the writ­ten ba­sis.

The biotech added that the plan is still to start the wet AMD tri­al and file an IND for di­a­bet­ic retinopa­thy in Q2 2020, “as we be­lieve that there are read­i­ly avail­able and suit­able al­ter­na­tives for all of the de­vices used in our stud­ies.”

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Robert Davis, Merck CEO

Mer­ck shares slide as re­sponse to its oral Covid an­tivi­ral plunges on lat­est up­date

After a second look, Merck says that its big breakthrough on the Covid front doesn’t look so good.

The pharma giant updated its data on its oral antiviral molnupiravir on Friday, and said that its drug reduced the rate of hospitalization and death by 30% — a sharp plunge from the 50% rate reported at the beginning of October during their interim readout.

And the news sparked a sharp retreat on Merck’s shares, which fell 4% ahead of the bell on Friday.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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