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.”

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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