Reg­u­lus Ther­a­peu­tics just fin­ished dos­ing in IND-en­abling tox­i­c­i­ty stud­ies for its sec­ond-gen­er­a­tion au­to­so­mal dom­i­nant poly­cys­tic kid­ney dis­ease (ADP­KD) can­di­date RGLS8429.

Based on FDA dis­cus­sions and da­ta from a Phase Ib tri­al on orig­i­nal ADP­KD can­di­date RGLS4326, Reg­u­lus is shift­ing away from the first-gen­er­a­tion can­di­date and pri­or­i­tiz­ing the new­er can­di­date, ac­cord­ing to a state­ment.

“We have de­ter­mined that ad­vanc­ing our next-gen­er­a­tion com­pound RGLS8429 is more com­pelling than fur­ther de­vel­op­ment of RGLS4326,” said Reg­u­lus CEO Jay Ha­gan. “The ex­ten­sive work and in­vest­ment we have made in RGLS4326 will di­rect­ly in­form the ad­vance­ment of RGLS8429, and we be­lieve will make this tran­si­tion both ex­pe­di­tious and pro­duc­tive.”

The Cal­i­for­nia biotech is ex­pect­ing to meet with the FDA pre-IND by the end of the year —  and pend­ing an FDA de­ci­sion on their IND, they an­tic­i­pate start­ing a Phase I study on their can­di­date in Q2 next year.  By ear­ly 2023, Reg­u­lus ex­pects to be able to re­port top-line da­ta in the first co­hort of RGLS8429-treat­ed pa­tients.

Sutro and BioNo­va launch op­tion agree­ment for an­ti-CD74 ADC worth $200M

The San Fran­cis­co biotech Sutro Bio­phar­ma and Shang­hai-based BioNo­va joint­ly an­nounced an op­tion agree­ment for BioNo­va to de­vel­op and com­mer­cial­ize an­ti-CD74 ADC STRO-001 for pa­tients with hema­to­log­ic can­cers. The com­mer­cial­iza­tion deal is for cer­tain Asian mar­kets, in­clud­ing Chi­na, Hong Kong, Macau and Tai­wan.

As part of the agree­ment, BioNo­va has the op­tion to re­ceive ex­clu­sive rights to de­vel­op and com­mer­cial­ize the ADC in those spe­cif­ic Asian mar­kets. In the mean­time, BioNo­va will pur­sue clin­i­cal de­vel­op­ment, reg­u­la­to­ry ap­proval, and com­mer­cial­iza­tion of STRO-001 in mul­ti­ple in­di­ca­tions, in­clud­ing non-Hodgkin’s lym­phoma, mul­ti­ple myelo­ma, and leukemia.

Sutro will re­tain both de­vel­op­ment and com­mer­cial rights of STRO-001 glob­al­ly out­side of those Asian mar­kets, in­clud­ing the US.

BioNo­va will pay Sutro an ini­tial li­cens­ing op­tion pay­ment of $4 mil­lion, with po­ten­tial mile­stone pay­ments to­tal­ing up to $200 mil­lion. Sutro will al­so re­ceive tiered and dou­ble-dig­it roy­al­ties based on an­nu­al net sales in those Asian mar­kets.

Sutro will pro­vide STRO-001 to BioNo­va un­der ap­pro­pri­ate clin­i­cal and com­mer­cial sup­ply ser­vice agree­ments.

“This strate­gic part­ner­ship of STRO-001 at this stage of its clin­i­cal de­vel­op­ment demon­strates the shared vi­sion of Sutro and BioNo­va of the po­ten­tial for this promis­ing ADC,” said Sutro CEO Bill Newell in a state­ment.

Bris­tol My­ers Squibb shows in­ter­im re­sults from five-year ex­ten­sion tri­al on Zeposia

Bris­tol My­ers Squibb un­veiled in­ter­im re­sults to­day from the Phase III open-la­bel ex­ten­sion tri­al Day­break, show­ing five years of da­ta on the ap­proved MS drug ozan­i­mod in pa­tients with re­laps­ing mul­ti­ple scle­ro­sis.

In the ex­ten­sion study, safe­ty was con­sis­tent with pri­or find­ings. Treat­ment with ozan­i­mod, al­so known as Zeposia, lead to a low re­lapse rate — 0.103. Ac­cord­ing to a Bris­tol My­ers state­ment, 75% and 71% of pa­tients were re­lapse-free when test­ed at the 3- and 4-year mark, re­spec­tive­ly.

“Ear­ly and ef­fec­tive in­ter­ven­tion can sig­nif­i­cant­ly im­pact phys­i­cal and cog­ni­tive re­sults over time, with low re­lapse rates an im­por­tant in­di­ca­tor of pa­tient out­comes,” said study in­ves­ti­ga­tor and clin­i­cal re­search di­rec­tor for the UCSF MS Cen­ter in San Fran­cis­co Bruce Cree. “These da­ta from the DAY­BREAK tri­al pro­vide a clear pic­ture of the long-term safe­ty and ef­fi­ca­cy pro­file of Zeposia, and re­in­force its po­ten­tial when used ear­ly in the treat­ment process for peo­ple liv­ing with re­laps­ing forms of MS.”

This sto­ry has been cor­rect­ed to clar­i­fy that RGLS8429 has not yet reached clin­i­cal test­ing.

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.