US gov­ern­ment does not own any remde­sivir patents, GAO re­port finds

The US fed­er­al gov­ern­ment’s con­tri­bu­tions to re­search on Gilead’s Covid-19 an­tivi­ral drug remde­sivir did not re­sult in any patent rights be­cause the re­search did not cre­ate new in­ven­tions, the Gov­ern­ment Ac­count­abil­i­ty Of­fice said in a re­port on Wednes­day af­ter­noon.

The re­port set­tles a long­stand­ing dis­pute over whether the gov­ern­ment might be co-in­ven­tors of remde­sivir, which has lin­gered since be­fore for­mer Cal­i­for­nia at­tor­ney gen­er­al Xavier Be­cer­ra (who’s now Biden’s HHS sec­re­tary) led a group of oth­er AGs last sum­mer in call­ing on the gov­ern­ment to in­ter­vene and low­er the more than $3,000 price tag for remde­sivir be­cause of the gov­ern­ment’s sig­nif­i­cant con­tri­bu­tions to remde­sivir.

The GAO re­port ac­knowl­edges that be­tween 2013 and 2020, the gov­ern­ment pumped $162 mil­lion in­to the de­vel­op­ment of remde­sivir, with the Cen­ters for Dis­ease Con­trol and Pre­ven­tion (CDC), the De­part­ment of De­fense (DOD), and the Na­tion­al In­sti­tutes of Health (NIH) con­duct­ing and fund­ing pre­clin­i­cal re­search. In ad­di­tion, NIH fund­ed three remde­sivir clin­i­cal tri­als, in­clud­ing the one show­ing that the an­tivi­ral short­ened the re­cov­ery time for hos­pi­tal­ized pa­tients with se­vere Covid-19.

Gilead, how­ev­er, al­so con­duct­ed its own re­search that led to the in­ven­tion of remde­sivir and the com­pa­ny said it in­vest­ed $786 mil­lion in remde­sivir R&D from 2000 through De­cem­ber 2020, GAO says. Gilead added in a state­ment that its in­vest­ment in remde­sivir ex­ceed­ed $1 bil­lion in 2020 alone and that cur­rent­ly about half of all hos­pi­tal­ized pa­tients with Covid-19 in the US are treat­ed with the drug.

DOD, NIH and oth­er sci­en­tists at Van­der­bilt and the Uni­ver­si­ty of North Car­oli­na at Chapel Hill ac­knowl­edged to GAO that their sci­en­tists were not co-in­ven­tors of patent­ed remde­sivir dis­cov­er­ies. For in­stance, NIH of­fi­cials told the GAO that Gilead be­gan patent­ing meth­ods of us­ing remde­sivir to treat coro­n­avirus in­fec­tions in 2015, while NIH sci­en­tists be­gan their remde­sivir re­search fo­cus­ing on coro­n­avirus­es in 2016.

“The prin­ci­pal in­ves­ti­ga­tors of NIH-fund­ed coro­n­avirus re­search projects told us that they did not con­sid­er fil­ing in­ven­tion dis­clo­sures be­cause their re­search did not in­volve mak­ing any mod­i­fi­ca­tions to remde­sivir or its par­ent com­pounds. The prin­ci­pal in­ves­ti­ga­tors stat­ed that they viewed such mod­i­fi­ca­tions as the thresh­old for fil­ing such dis­clo­sures,” GAO said.

And even if the gov­ern­ment had filed for a remde­sivir method-of-use patent, DOD and NIH of­fi­cials said that would be of low eco­nom­ic val­ue be­cause the fed­er­al gov­ern­ment did not own the com­pound patents and would have had to li­cense those patents from Gilead if it want­ed to com­mer­cial­ize the method of use or li­cense it to an­oth­er com­pa­ny.

Ac­cord­ing to CDC of­fi­cials, as of last month, they said it was “un­like­ly that CDC would con­duct an in­ven­tor­ship analy­sis or pur­sue in­tel­lec­tu­al prop­er­ty rights giv­en Gilead’s back­ground in­tel­lec­tu­al prop­er­ty and the lim­it­ed po­ten­tial for CDC to li­cense any such rights.”

Chris Morten, a lawyer who cur­rent­ly teach­es at New York Uni­ver­si­ty School of Law, not­ed that the re­port did not clar­i­fy if a CDC sci­en­tist, Michael Lo, co-in­vent­ed Gilead’s remde­sivir com­pound patents, and if so, “CDC would, un­der the de­fault rules of patent law, have a claim to co-own­er­ship. But CDC ap­pears un­in­ter­est­ed in pur­su­ing its le­gal rights.”

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Voting in the 2020 election (AP Images)

The right to vote is fun­da­men­tal — a let­ter from biotech­nol­o­gy in­dus­try lead­ers

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We oppose all attempts to introduce laws that reduce the rights of US citizens to vote or that restrict them from exercising that right. The right to vote is fundamental to democracy. States that have enacted, or are proposing to enact, legislation to restrict voting are undermining our democracy and posing a threat to our nation. As leaders of the life sciences industry, we stand for what we believe is right for our country, our enterprises, our employees and those who benefit from our work. We join the first groups of business leaders who have challenged these laws and will continue to make our collective voices heard on this matter.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.

Kristin Fortney, BioAge Labs CEO

An­ti-ag­ing biotech up­start plucks a drug from Am­gen's dis­card pile, piv­ot­ing from heart fail­ure to mus­cle con­di­tions

Back in April 2019, Amgen quietly shut down a Phase I trial for a drug named AMG 986. There was no safety concern; the molecule just didn’t hit the mark on helping the small band of heart failure patients who received it.

A small biotech, though, believes it would stand a chance in the burgeoning anti-aging field.

BioAge Labs has licensed AMG 986 — now renamed BGE-105 — with plans to parlay the existing IND into a quick Phase I trial teasing out the pharmacodynamic effects and set the stage for mid-stage tests focused on acute muscle indications.