Rid­ing on rosy es­ti­mates of Zol­gens­ma sales, Re­genxBio sells part of its roy­al­ty for $200M

Re­genxBio’s jour­ney as a drug de­vel­op­er may have been less than rosy, but there’s been lit­tle doubt about the val­ue of the gene ther­a­py tech­nol­o­gy it ob­tained from Jim Wil­son’s labs.

Health­care Roy­al­ty Man­age­ment has made that clear once again, strik­ing a deal to buy a por­tion of its roy­al­ties on Zol­gens­ma for $200 mil­lion.

Ac­cord­ing to the lat­est quar­ter­ly fil­ing from Re­genxBio, it’s re­ceived $40.7 mil­lion in roy­al­ties in the first nine months of 2020, com­pared to just over $10 mil­lion in the same pe­ri­od in 2019.

Ken Mills

“This agree­ment with HCR pro­vides us with sig­nif­i­cant ad­di­tion­al non-di­lu­tive fund­ing to con­tin­ue our mo­men­tum in the clin­ic fo­cused on RGX-314 and our rare neu­rode­gen­er­a­tive dis­ease plat­form, in­clud­ing RGX-121, as well as the op­por­tu­ni­ty to de­vel­op new in­no­va­tions for pa­tients in oth­er dis­ease ar­eas,” said Ken­neth Mills, Re­genxBio pres­i­dent and CEO, in a state­ment.

It’s a crit­i­cal mo­ment for the com­pa­ny as its pipeline re­bounds from a cou­ple of reg­u­la­to­ry and clin­i­cal set­backs in­to late-stage de­vel­op­ment. There are al­so plans to build new in-house man­u­fac­tur­ing fa­cil­i­ties with a 2,000-liter scale to se­cure a sus­pen­sion cell cul­ture process ready for com­mer­cial roll­out.

No­tably, there is a chance that Re­genxBio can get the roy­al­ty rights back if one of two caps the par­ties agreed up­on is met: Ei­ther HCR gets from No­var­tis $260 mil­lion by No­vem­ber 7, 2024, or $320 mil­lion there­after.

A sup­posed once-and-done treat­ment for spinal mus­cu­lar at­ro­phy, Zol­gens­ma has a list price of $2.1 mil­lion. While that has earned it the du­bi­ous ti­tle of the most ex­pen­sive drug in the world, No­var­tis has em­pha­sized that pay­ment is spread out over five years — mean­ing $425,000 a year — and that it’s a life­sav­ing, life-chang­ing ther­a­py for pa­tients who faced a de­press­ing prog­no­sis.

AveX­is, which No­var­tis snapped up for $8.7 bil­lion, de­vel­oped the pro­gram on Re­genxbio’s NAV tech plat­form and AAV9 vec­tor.

The 2018 ac­qui­si­tion im­me­di­ate­ly added $140 mil­lion to Re­genxbio’s vault. Short­ly af­ter­ward, the phar­ma gi­ant forked over an­oth­er $60 mil­lion in an­nu­al fees and $40 mil­lion for a com­mer­cial mile­stone — plus $80 mil­lion more that was paid ear­li­er this year.

Zol­gens­ma is cur­rent­ly ap­proved in the US, Japan, Eu­rope, Brazil and Cana­da, al­beit on­ly for younger chil­dren (in­fants be­low the age of 2 in the US). De­spite some hic­cups, No­var­tis is al­so con­duct­ing tri­als for old­er SMA pa­tients.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Jim Tananbaum, Foresite

Fore­site re­turns to the SPAC well, as in­vestors won­der how long the run can last

Six months after launching his first biotech SPAC, Foresite’s Jim Tananbaum has started a second. On Tuesday, the longtime life science investor filed to raise $100 million by selling 10 million shares of the blank check company FS development II.

It’s a quick return to Wall Street for Foresite, although other firms have moved quicker. Perceptive Advisors raised a $130 million SPAC in June and were back before the end of July to raise another $125 million. By that point, the firm was evidently nearing a deal for the June SPAC, which would announce a half-billion-dollar merger with Cerevel Therapeutics on July 30.

Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

As new Covid-19 task force gets un­der­way, threat looms of vac­cine, mon­o­clon­al an­ti­body-re­sis­tant vari­ants

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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