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.

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.

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.

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.