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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

John Oyler, BeiGene CEO (Paul Yeung/Bloomberg via Getty Images)

Bris­tol My­ers wants to pull out of its Abrax­ane deal in Chi­na. BeiGene says no way

A year and a half after Chinese officials ordered BeiGene to stop selling Bristol Myers Squibb’s Abraxane in the wake of an alarming inspection of a US facility, the manufacturing issues at the root of the import suspension still appear unresolved.

And Bristol Myers wants to axe the Abraxane supply deal altogether.

But BeiGene, which is currently in arbitration proceedings against its Big Pharma partner, won’t let it off the hook so easily.

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Madhu Natarajan, Takeda rare disease development head

Drawn to the idea of turn­ing B cells in­to 'pro­tein fac­to­ries,' Take­da jumps in­to a mile­stone-heavy, $900M pact

Madhu Natarajan can trace his fascination with the idea of taking B cells and turning them into protein factories back 20 years, when he had his own lab at UT Southwestern. So when Natarajan, now the rare disease development head for Takeda, sat down for a meet-up with execs from Seattle-based Immusoft at the last in-person JP Morgan conference, they went straight into a brainstorming session.

“That B cells can take up residence and do what they do for a long time,” says Natarajan, pumping out proteins and “leveraging it into a therapeutic context,” hits his sweet spot for discovery deals. And he was deeply impressed by what he heard.

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FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.