Update for June 8, 2022

Regulatory Update
 
The FDA withdrew approval of umbralisib for the treatment of marginal zone lymphoma and follicular lymphoma due to increased mortality seen in the UNITY-CLL trial.
 
The FDA extended the PDFA date for AMX0035 by three months. The new date is 9/29/2022.
 
Gamida completed a rolling BLA for omidubicel.
 
The FDA designated efanesoctocog alfa a Breakthrough Therapy for the treatment of hemophilia A.
 
The EMA designated gavorestat an Orphan Drug for the treatment of galactosemia.
 
Announced Research Updates
 
Sage announced that in the 15-day, 195 patient, Phase III, SKYLARK trial, patients treated with zuranolone had a 15.6 point reduction in their HAMD-17 total score compared to an 11.6 point decrease with placebo in patients with severe postpartum depression. 
 
MaaT Pharma announced that in the 10-week, 21 patient, open-label, dose-ranging, Phase Ib, CIMON trial, treatment with MaaT033 resulted in rapid and persistent engraftment of MaaT033 in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome who have received intensive chemotherapy.
 
Immunic announced that in the Phase II, CALDOSE-1 trial, treatment with vidofludimus did not improve achievement of remission compared to placebo in patients with moderate-to-severe ulcerative colitis
 
Mirati announced that in a 19 patient cohort of the 740 patient, Phase II, KRYSTAL-1 trial, treatment with adagrasib resulted in an ORR of 32% in patients with advanced NSCLC and CNS metastases harboring the KRAS G12C mutation.
 
Cullinan reported interim results from 73 patients enrolled in a Phase I/IIa trial where treatment with CLN-081 resulted in a partial response in 38% of patients with non-small cell lung cancer (NSCLC) harboring epidermal growth factor(EGFR) exon 20 insertion mutations resistant to platinum-based therapy.
 
Biogen announced 12-month data from the VALOR trial, where patients who originally received tofersen had an improvement in clinical function, respiratory function and muscle strength compared to patients who originally received placebo, then switched to tofersen in the open label extension (six-months later).
 
Immutep announced that in the 114 patient, Phase II, open-label TACTI-002 trial, treatment with eftilagimod alpha plus pembrolizumab resulted in an overall response rate (ORR) of 38.6% in patients with non-small cell lung cancer (NSCLC). 
 
Seres announced that in the 24-week, 263 patient, open-label, Phase III, ECOSPOR IV trial, 8.4% of patients treated with SER-109 experienced a recurrence of C. difficile infection at eight weeks in patients with recurrent C. difficile infection. After 24 weeks 86% of patients treated maintained a sustained clinical response.
 
Lilly announced one-year results from ten patients enrolled in the long term extension ADjoin trial, where continued treatment with lebrikizumab resulted in clear skin in 8/10 patients who had achieved EASI-75 while participating in the Phase III, ADvocate 1 or 2 trials.
 
Published Research Updates
 
In the 88 patient, Phase II, open-label, MERECA trial, treatment with ilixadencel plus sunitinib did not improve overall survival compared to sunitinib alone in patients with metastatic renal cell carcinoma.
 
In the 37 patient advanced/metastatic urothelial carcinoma cohort of the open-label, Phase II, PIVOT-2 trial, treatment with bempegaldesleukin plus nivolumab resulted in an ORR of 35%.
 
In the 165 patient, Phase II portion of the MajesTEC-1 trial, treatment with teclistamab resulted in an ORR of 63% in patients with relapsed or refractory multiple myeloma.