So far, we listed 39 abstracts from the European listed biotech companies or their partners for the ASH 2019 Annual meeting, excluding those relating to daratumumab (16 abstracts by Janssen/JNJ). Morphosys will present 7 abstracts and Oncopeptides 6. Cellectis and partners gather 4 presentations. Argenx will have 2 oral presentations, one for efgartigimod in ITP, and one for cusatuzumab in AML.
Morphosys will heavily showcase tafasitamab, with a rolling US submission expected to be completed by year end. Now viewed as a best-in-class anti-CD19 therapy in DLBCL, at least with lenalidomide, the next milestone should include a juicy partnership.
Oncopeptides, on their side, will provide several updates for melflufen, with all the lights on HORIZON and ANCHOR. The CEO of Oncopeptides recently said that from ANCHOR's data, "we will know if Oncopeptides will be a billion+ USD company, or just a few hundred million USD company". The Swedish company plans to file an NDA for melflufen in triple-class-refractory R/R MM in Q1 2020 (as an addon to dexamethasone).
The abstract links can be found on the abstract reference column of the table below. The links to the presentation slides will be added if/when available.
Main highlights/lowlights for the European listed biotech companies:
Oncopeptides presented improved mDOR from HORIZON in the last data cut-off (7.5 months in RR MM 3x-class refractory patients, with ORRs ranging from 24 to 29 depending on the populations considered). ANCHOR mPFS reached an encouraging 14.3 months for the Dara-Meflufen-dex regimen in preliminary Kaplan-Meier estimates, in a less advanced population than HORIZON.
Genmab presented early signs of efficacy for their subcutaneous CD20xCD3 Duobody, with good signs in FL and early signs in DLBCL/High Grade BCL, with a relatively benign safety profile overall. There might be a debate on the steroid pre-dosing though, and the differentiation in terms of safety profile as well, as compared to Roche and Regeneron competitors (other than the mode of administration).
Oryzon Genomics presented an interesting update on the efficacy side in ND AML for iadademstat and azacytidine (ORR 6/8 or 75%), however the safety/tolerability was not as good as previously anticipated (1 death from intracranial hemorrhage). A lower dose will be evaluated to optimize the AEs incidence and the compliance, with a potential re-escalation still possible pending good tolerability. The DOR data were not mature enough to draw any conclusion. The updates, in 2020, should provide more clarity on the potential.
Argenx's cusatuzumab yielded in a 100% ORR during the dose-escalation study in n=12 AML patients (83% CR/CRi or 10/12, of which 4/9 evaluated for MRD obtained a negative status at the 10e-3 sensitivity level), also in combination with azacytidine. The DOR is still under evaluation but 6/12 patients reached or are close (ongoing responses) to reach the 12-month OS bar. 4 relapsed before this bar, including 1 at around 2 months, and 3 between 7 and 12 months. 1 patient underwent a transplant, and one withdrew due to AEs. The only weird thing comes from the cut-off date, from February 2019.
Less encouraging update unfortunately for Celyad, with a null ORR in DEPLETHINK for the NKG2D CAR-T CYAD-01 (2 DLs, 2 preconditioning regimen schedule, mAb process). Better ORR-wise for THINK (5 responses), but no clear signs of improved outcomes with a dose dense schedule. Numerically, that's even the contrary (ORR in 5/10 evaluable AML/MDS patients with the biweekly schedule and 0/7 with the dose dense -weekly- schedule). The company is now betting on improved outcomes with their new OptimAb process (memory phenotype-enriched mix of CAR-T cells, using a PI3K inhibitor and a shorter process).