Update for August 4, 2022

Regulatory Update
 
The FDA accepted the NDA for tofersen to treat superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS) and set a PDUFA date for 1/25/2023.
 
The FDA accepted the NDA for sotagliflozin for the treatment of heart failure and set a PDUFA date for 6/27/2023.
 
The FDA accepted the BLA for the combination of N-803 plus Bacillus Calmette-Guerin (BCG) for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma and set a PDUFA date for 5/23/2023.
 
The FDA accepted the BLA for omidubicel for the treatment hematologic malignancies progressing to allogenic hematopoietic stem cell transplant (HSCT) and set a PDUFA date for 1/30/2023.
 
Cidara submitted an NDA for rezafungin for the treatment of candidemia and invasive candidiasis.
 
Sarepta plans to submit a BLA for delandistrogene moxeparvovec for the treatment of ambulant Duchenne muscular dystrophy in the second half of 2022.
 
MHRA granted marketing authorization for voxelotor for the treatment of hemolytic anemia due to sickle cell disease.
 
ObsEva discontinued development of linzagolix and returned development and marketing rights back to Kissei after the FDA indicated that additional data was required to address NDA deficiencies.
 
CHMP granted an accelerated assessment for leniolisib for the treatment of PI3K delta syndrome.
 
Announced Research Updates
 
J&J announced that in the 20 patient, open-label, Phase 1b/2 CHRYSALIS-2 trial, treatment with lazertinib, amivantamab, carboplatin and pemetrexed resulted in an overall response rate (ORR) of 50% in patients with relapsed/refractory non-small cell lung cancer (NSCLC) and EGFR mutations.
 
AstraZeneca has discontinued development of adavosertib as a treatment for ovarian cancer, solid tumors and uterine serous cancer.
 
Novo announced that in the 26-week, 588 patent, Phase IIIa, ONWARDS 3 trial, once-weekly insulin icodec lowered HbA1c 1.57% compared to a 1.36% decrease with once-daily insulin degludec in patients with type 2 diabetes who were insulin-naive. Novo announced that in the 26-week, 588 patent, Phase IIIa, ONWARDS 4 trial, once-weekly insulin icodec lowered HbA1c 1.16% compared to a 1.18% decrease with once-daily insulin glargine in patients with type 2 diabetes who were also receiving mealtime insulin aspart two to four times daily.
 
Immutep announced that in the 36 patient, Part B of the Phase II, open-label TACTI-002 trial, treatment with eftilagimod alpha plus pembrolizumab resulted in overall survival of 9.7 months in patients with second line checkpoint inhibitor refractory metastatic NSCLC.
 
Concert announced that in a 24-week, 517 patients, Phase III, THRIVE-AA2 trial, treatment with CTP-543 resulted in achievement of a SALT score of 20 or less in 38.3% of patients treated with 12 mg and 33% who received 8 mg compared to 0.8% with placebo in patients with moderate to severe alopecia areata.
 
The results of two failed CNTX-4975 trials have not been announced, presented or published and are only available on clinicaltrials.gov.

• In a 12-week, 302 patient, Phase III trial (NCT03660943), treatment with CNTX-4975 did not reduce the Weekly Pain With Walking score in the index knee compared to placebo in patients with moderate-to-severe osteoarthritis knee pain. 
• In a 12-week, 332 patient, Phase III trial (NCT03429049), treatment with CNTX-4975 did not reduce the WOMAC score in the index knee compared to placebo in patients with moderate-to-severe osteoarthritis of the knee.

 Incyte discontinued the six-month, 157 patient, Phase II/III, GRAVITAS-309 trial, when interim data did not demonstrate efficacy for itacitinib in the treatment of chronic graft-versus-host disease in the 28-day, Part 1 of the trial. Incyte discontinued development of itacitinib after failure in the GRAVITAS-309 trial.
 
Paxalisib was being evaluated as a treatment for glioblastoma as part of the 550 patient, Phase II/III GBM AGILE trial, which is testing multiple treatments for glioblastoma. Paxalisib was dropped from the GBM AGILE trial, when it did not meet efficacy criteria in the Phase II portion of the trial.
 
Isofol announced that in a 490 patient, Phase III, AGENT trial, arfolitixorin did not improve the ORR compared to leucovorin in patients with metastatic colorectal cancer, who were being treated with 5-FU, oxaliplatin, and bevacizumab. Isofol will announce future plans for arfolitixorin in 2023, after a full analysis of the AGENT trial is completed.
 
Published Research Updates
 
In a 48-week, 40 patient, open-label trial, treatment with tofogliflozin and glimepiride each improved liver histological and metabolic parameters in patients with type 2 diabetes and nonalcoholic fatty liver disease.
 
In the 52-week, 407 patient, Phase III, ASCEND-TD trial, switching patients from epoetin to daprodustat was non-inferior to continuing epoetin (10.45 vs 10.51 g/dl) in CKD patients who were undergoing dialysis. 
 
In the 60 patient, open-label Phase II, CONCERTO trial, treatment with olaparib plus cediranib resulted in an objective response rate of 15.3% in PARP inhibitor-naive patients with non-germline-BRCA-mutated platinum-resistant recurrent ovarian cancer.
 
In the 52-week, 316 patient, Phase II, PASADENA trial, treatment with prasinezumab did not improve the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score compared to placebo in patients with early Parkinson's Disease (PD).