Cell & Gene Weekly
CGT breakups between big pharma and biotech, first glimpse into earnings season and more...
Happy Friday ! A few pharma-biotech breakups (unfortunately), but apart from that a relatively quiet week … Have a good and restfull weekend ahead ;)
A couple of breakups between big pharma and CGT biotechs
Roche / Genentech terminated their partnership with Adaptimmune tx in the cell therapy space . The collaboration was centered on nucleases from Precision’s ARCUS editing platform: Genentech paid $150 M upfront for the partnership and was eligible to receive $3 bn in biobucks. As a reminder, Roche has a few other shots on target in the cell therapy space, having forged partnerships with Poseida Tx and Arsenal Bio. Perhaps the Adaptimmune breakup also came about due to the “better than expected” BCMA allo-CAR-T data from the Roche-Poseida collab drug in MM, which had an 82% ORR, incl 2/2 ORR in patients that had previously received auto BCMA CAR-Ts. As always, durability is the big unknown with such allo programs, but I’d say this is probably some of the best allo-CAR-T data I had seen - see here for details
Eli Lilly /Prevail terminated their partnership with Precision Bio in gene therapy space. The collaboration was centered around in vivo gene therapies for three gene targets, starting with Duchenne muscular dystrophy (DMD). Lilly paid out $100 M upfront, with a potential for 2.6 bn in biobucks. Perhaps the reason for this breakup is the sheer number of gene therapy (GTx, RNAi/ASO, gene editing tx, etc.) players looking into DMD (incl. Sarepta’s existing footprint in the space, incl. marketed GTx Elevidys)
Source Roche / Adaptimmune: Fiercebiotech; Source Lilly / Precision: Fiercebiotech
CAR-T cell therapy trial tracker - key themes from this week
Since most people in last week’s poll asked for the CAR-T tracker to be on a weekly basis - i will continue with it for the time being: ~8 new CAR-T studies posted on clinical trials.gov in the past week - largely same themes in heme and autoimmune (no solid this week !).
Interesting to see CARTregs appearing on the radar again - as a reminder, they were early runners in the application of cell therapies in autoimmune — back in 2022 I had covered Sangamo’s Treg approach in renal transplantation and Sonoma’s programs in Rheum Arthritis and diabetes - a while has passed since, and the field has since exploded…
3 CAR-T in heme: (1) Ruijin Hospital CD20 CAR-T in r/r B-NHL [source]; (2) Ruijin Hospital CD19 CAR-T in r/r/ NHL [source]; (3) NCI CD19 CAR in CLL [source];
5 CAR-Ts in autoimmune: (1) Sonoma Biotherapeutics CARTreg for hidradenitis suppurativa [source]; (2) PersonGen Bio CD19 gamma-delta CAR-T UTAA09 in autoimmune [source]; (3) Tang-Du Hospital CD19-BCMA CAR-T in Generalized Myasthenia Gravis [source]; (4) EdiGene ATHENA CAR-T in SLE [source]; (5) Shanghai Changzheng Hospital CD19-CD3E-CAR-T in r/r Autoimmune Disease [source]
Status check on commercialization of Cell & Gene therapies
The first from the Cell & Gene bunch to report Q1 2024 earnings: J&J reported $157 M last quarter from Carvykti. Sales are essentially flat vs previous quarter ($159M). That being said, in the coming quarters, we’re likely to see a super ramp-up as Carvykti moves into earlier line following the green light from the FDA 2 weeks ago to move Carvykti in 2L MM (vs. Abecma in 3L+)… as a reminder, J&J have huge ambitions for Carvykti to become a multi-billion drug= ~$5 bn peak sales 😝
Source: J&J website; Peak sales ambition
Quick take news
Bayer and AskBio report 18 month data from Parkinson’s disease therapy trial [source, source]
Top-line findings from the study were announced earlier this year - latest update confirms that the therapy is well tolerated with no attributed serious adverse events in all 11 patients at 18 months, meeting the primary objective. Time to move to the next clinical development stage 😉
AviadoBio treats first subject in trial of FTD-GRN therapy [source]
Astellas writes down another piece of its Audentes gene therapy acquisition [source]
TD Cowen says sickle cell therapies may get higher CMS payments [source]
Voyager Therapeutics Announces Selection of Development Candidate for GBA1 Program in Collaboration with Neurocrine Biosciences [source]
Artiva bio Announces First Patient Dosed in Phase 1 Trial of AlloNK® Cell Therapy Candidate in Lupus Nephritis [source]
What I read this week
For those that dont know it - I would recomment to have a look at the ASCGT Gene, Cell, + RNA Therapy Landscape report on a quarterly basis. It’s a good summary of what happened in the Cell & Gene world over the past ~3 months. Having had a quick skim through it myself, there were a couple of items that I had missed, notably evorcabtagene autoleucel approval in China (CARsgen) and Beqvez approval in Canada (Pfizer). Mea Culpa
Source: ASCGT report
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer
Have I missed anything? Is there something you would like to hear more about? let me know !
Do you think 5 bn annual revenue is likely for Carvykti? Seems ambitious.