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.”

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.