Pablo Legorreta, Royalty Pharma CEO (Patrick T. Fallon/Bloomberg via Getty Images)

Glax­o­SmithK­line of­floads roy­al­ty rights for 2 can­cer drugs in $342M wind­fall it will use to fi­nance con­sumer spin­off

As Glax­o­SmithK­line con­tin­ues plans to spin off its Pfiz­er-part­nered con­sumer health di­vi­sion, the British phar­ma pulled in a new heap­ing of cash to ac­com­plish that goal.

GSK sold its roy­al­ty rights for Ex­elix­is drugs Cabome­tyx and Cometriq to Roy­al­ty Phar­ma on Thurs­day morn­ing, net­ting a $342 up­front pay­ment and a promise for up to $50 mil­lion more in mile­stones de­pend­ing on reg­u­la­to­ry ap­provals in new in­di­ca­tions. The pro­ceeds will go to­ward the spin­out ef­forts, a GSK spokesper­son con­firmed to End­points News, as the drug­mak­er looks to raise more than $2.2 bil­lion in cash to fi­nance the split.

“In Feb­ru­ary 2020, we an­nounced our pro­gramme to pre­pare GSK for a sep­a­ra­tion in­to two great new com­pa­nies … which would large­ly be fund­ed by dis­pos­als,” the spokesper­son told End­points. “This is a smart deal from our per­spec­tive, which de­liv­ers a sig­nif­i­cant amount of cash for an as­set in which we no longer have any com­mer­cial­i­sa­tion or de­vel­op­ment in­volve­ment.”

The GSK split re­mains on sched­ule for 2022, the spokesper­son added.

GSK had orig­i­nal­ly ob­tained 3% roy­al­ties as part of a 2002 col­lab­o­ra­tion with Ex­elix­is for cabozan­ti­nib prod­ucts — the gener­ic name for both Cabome­tyx and Cometriq. Un­der Thurs­day’s deal, Roy­al­ty Phar­ma gains roy­al­ties on net cabozan­ti­nib sales out­side the US through the full term of the roy­al­ty, as well as US roy­al­ty sales through Sep­tem­ber 2026.

The move con­tin­ues Roy­al­ty’s large­ly suc­cess­ful strat­e­gy of buy­ing up roy­al­ties on drugs, di­ag­nos­tics and med­ical prod­ucts. CEO Pablo Legor­re­ta rode the busi­ness mod­el to Nas­daq last June in a mas­sive IPO that raised $2.2 bil­lion and a de­but val­u­a­tion of near­ly $17 bil­lion.

Legor­re­ta has en­gi­neered roy­al­ty deals for sev­er­al promi­nent drugs over the years, in­clud­ing Hu­mi­ra, Tru­va­da, Im­bru­vi­ca and Trodelvy. Last year alone, Roy­al­ty ac­quired roy­al­ty sales in Roche and PTC Ther­a­peu­tics’ ris­diplam for $650 mil­lion and resid­ual roy­al­ties in Ver­tex’s cys­tic fi­bro­sis fran­chise from the CF Foun­da­tion for $575 mil­lion up­front.

Pre­vi­ous­ly, Roy­al­ty had been ob­lig­at­ed to pay 50% of those roy­al­ties to the CF Foun­da­tion. But the new deal, agreed to last No­vem­ber, elim­i­nat­ed that com­mit­ment, re­sult­ing in what Roy­al­ty says is a po­ten­tial $5.8 bil­lion an­nu­al wind­fall.

In Cabome­tyx, Roy­al­ty gets a drug that’s on track to be an up and com­ing block­buster, hav­ing won ap­provals as a monother­a­py and in com­bi­na­tion with Op­di­vo as a first-line treat­ment for re­nal cell car­ci­no­ma. The Op­di­vo cock­tail green­light came in Jan­u­ary as pa­tients treat­ed with Op­di­vo and Cabome­tyx in a Phase III study lived twice as long with­out their tu­mors pro­gress­ing as those treat­ed with Pfiz­er’s Su­tent.

Last year, sales of Cabome­tyx and Cometriq to­taled $742 mil­lion and €289 mil­lion, re­spec­tive­ly.

Both Cabome­tyx and Cometriq are ty­ro­sine ki­nase in­hibitors, though they come in dif­fer­ent for­mu­la­tions. Where­as Cabome­tyx comes in the form of a tablet to treat kid­ney can­cer, Cometriq is an oral cap­sule used for thy­roid can­cer.

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.

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.

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.

Fifth Cir­cuit sides with FTC in ma­jor pay-for-de­lay set­tle­ment case

The US Court of Appeals for the Fifth Circuit on Tuesday upheld the Federal Trade Commission’s ruling that generic drugmaker Impax Laboratories should be charged with antitrust violations for accepting payments worth about $100 million to delay the entry of a generic opioid for more than two years.

The court’s opinion upheld the FTC’s anticompetitive findings on the deal between Impax (now owned by Amneal Pharmaceuticals) and Endo Pharmaceuticals, under which Endo committed to not market an authorized generic, which increased Impax’s projected profits by $24.5 million. Endo agreed to pay Impax credits for the shrunken market that Impax would inherit if, as expected, Endo made a successful hop to a reformulated Opana ER.

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.