UP­DAT­ED: FDA warns small biotech Ocu­gen of po­ten­tial $10K fines for fail­ing to post tri­al re­sults

The FDA is back to warn­ing com­pa­nies of $10,000 fines for fail­ing to re­port the re­sults of clin­i­cal tri­als, this time go­ing af­ter clin­i­cal stage biotech com­pa­ny Ocu­gen, known most re­cent­ly for try­ing to bring a Covid-19 vac­cine from In­dia to the US and oth­er mar­kets.

The tri­al in ques­tion with FDA, com­plet­ed in May 2019, was a Phase III study of bri­moni­dine tar­trate na­noemul­sion eye drop to treat dry eye dis­ease. The com­pa­ny said in an SEC fil­ing in Nov. 2019 that the Phase III tri­al has com­plet­ed “and al­though the study showed that OCU310 is well tol­er­at­ed, as demon­strat­ed by no ad­verse events re­gard­ing as ‘se­vere,’ it did not meet its co-pri­ma­ry end­points for symp­toms and signs.”

The FDA is now giv­ing Ocu­gen un­til May 15 to post the re­sults from the tri­al. Al­though as not­ed by the non­prof­it Transparimed, which has ded­i­cat­ed re­sources to help this kind of re­port­ing, it’s un­clear why the FDA is tak­ing so long to tar­get com­pa­nies af­ter their tri­als fin­ish, and to fol­low up af­ter the com­pa­ny doesn’t re­spond to an ini­tial let­ter. The agency pre­vi­ous­ly sent more than 40 “Pre-No­tices of Non­com­pli­ance” to en­cour­age com­pli­ance with the Clin­i­cal­Tri­als.gov re­quire­ments.

But it’s been al­most a year since the FDA warned the first com­pa­ny for this type of in­frac­tion, as it warned Ac­celeron for its fail­ure to post re­sults from a can­cer tri­al, and sim­i­lar­ly said the com­pa­ny has 30 days to cor­rect this fail­ure or it may have to pay a $10,000 penal­ty or face crim­i­nal pros­e­cu­tion. It’s un­clear if any of the fines have ac­tu­al­ly been levied.

The agency al­so has tar­get­ed in­di­vid­u­als run­ning tri­als, in­clud­ing Los An­ge­les-based urog­y­ne­col­o­gist An­drey Petrikovets, who failed to re­lease re­sults from his tri­al, known as,”ICE-T Post­op­er­a­tive Mul­ti­modal Pain Reg­i­men Com­pared to the Stan­dard Reg­i­men in Same Day Vagi­nal Pelvic Re­con­struc­tive Surgery: A Ran­dom­ized Con­trolled Tri­al.”

Mean­while, the pres­sure is on FDA to do more in this area. A group known as the Uni­ver­si­ties Al­lied for Es­sen­tial Med­i­cines is call­ing on FDA com­mis­sion­er Rob Califf to help the agency do its job of en­forc­ing these tri­al pub­lish­ing re­quire­ments.

“Al­though vi­o­la­tions of the law have been so fre­quent and so fla­grant that they could have gen­er­at­ed over $30 bil­lion in fines to date, the FDA has his­tor­i­cal­ly ne­glect­ed its du­ty to en­force FDAAA,” the UAEM wrote in a let­ter to Califf last week. “The FDA should there­fore es­tab­lish an ac­tion­able pri­or­i­ti­za­tion frame­work for en­force­ment of FDAAA, and demon­strate an in­tent to be­gin more rig­or­ous en­force­ment.”

Ocu­gen told End­points News in an emailed state­ment:

Ocu­gen is com­mit­ted to com­pli­ance and takes trans­paren­cy re­gard­ing clin­i­cal da­ta and tri­al re­port­ing se­ri­ous­ly. We sub­mit­ted in­for­ma­tion to up­date the site af­ter tri­al was com­plet­ed. In No­vem­ber 2021, we up­dat­ed the site with ad­di­tion­al in­for­ma­tion, which is not re­flect­ed. We’ve been aware of the lat­est U.S. Food & Drug Ad­min­is­tra­tion’s re­quest to up­date clin­i­cal­tri­als.gov with re­sults of our dis­con­tin­ued, OCU310 pro­gram.  We’ve been in di­a­log with the agency ever since re­ceipt of their let­ter and we will sub­mit in­for­ma­tion as re­quest­ed ahead of their dead­line.

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with com­ment from Ocu­gen above.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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John Evans, Beam Therapeutics CEO

Beam's base-edit­ed al­lo­gene­ic CAR-T gets FDA go-ahead af­ter four-month wait

The FDA wanted more information on four key areas before it would let Beam Therapeutics proceed with human testing for a cell therapy in a certain type of leukemia. It appears the biotech has answered the agency’s queries.

The US regulator cleared the base-edited, off-the-shelf CAR-T, Beam said Friday morning, lifting a hold from this summer. More details on specific next steps for the Phase I will come out next year, the Boston-area biotech said.

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Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Lex­i­con slams FDA over hear­ing de­nial fol­low­ing a CRL for its SGLT2 in­hibitor can­di­date

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.