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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche touted positive durability and safety data for its spinal muscular atrophy drug Evrysdi, Novartis also made a splash with its multi-million dollar gene therapy for the disease.

Novartis rolled out interim data from two long-term follow-up studies Monday at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. In the first study, LT-001, all children in the trial that were treated after showing symptoms of SMA “maintained all previously achieved motor milestones” up to 7.5 years after being dosed. The average time since Zolgensma was given was 6.86 years.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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