Update for May 4, 2021

Regulatory Update
 
The FDA accepted the NDA for bardoxolone methyl for the treatment of chronic kidney disease caused by Alport syndrome and set a PDUFA date for 2/25/2022.
 
In April 2021, Lilly discontinued development of mirikizumab for the treatment of psoriasis. Lilly has ongoing trials evaluating mirikizumab in the treatment of ulcerative colitis and Crohn's disease.
 
Beginning in mid-July, Biogen will allow access through a compassionate use program for tofersen to treat rapidly progressing ALS patients with SOD1 mutations. In the fall of 2021, if tofersen is found to be safe and effective in a Phase III trial, an Early Access Program will be initiated for a broader range of ALS patients with SOD1 mutations.
 
Enzyvant resubmitted a BLA for RVT-802 for the treatment of pediatric patients with congenital athymia.
 
The FDA delayed review of pegunigalsidase alfa until a site inspection could be completed at Protalix's manufacturing facility in Carmiel, Israel.
 
The FDA rejected the NDA for tralokinumab and requested additional information on a device used in the delivery of the drug. The EMA’s CHMP recommended approval of the MAA for tralokinumab for the treatment of moderate-to-severe atopic dermatitis.
 
The FDA has extended the PDUFA date for BioMarin's vosoritide by three months to allow additional time to review recently submitted results from a two-year Phase III extension study.
 
Announced Research Updates
 
Aldeyra announced that in the 210 minute 95 patient, Phase III, INVIGORATE trial, treatment with reproxalap 0.25% ophthalmic solution reduced the subject-reported ocular itching score compared to placebo in patients with seasonal allergic conjunctivitis. 
 
Revance announced that in an 84-week, analysis of 568 patients from the Phase III, SAKURA 3 trial, four-weeks after the first injection of daxibotulinumtoxinA, up to 57.9% of patients perceived no static Glabellar lines (GL). After a second injection 68.7% reported no GL and 71.5% after the third treatment in patients with mild and moderate static Glabellar lines. Similar improvements were observed by investigators. Patients with severe static GL also achieved no static GL with a third cycle of daxibotulinumtoxinA.
 
Pharnext announced interim results after 54-months from a long-term extension of the Phase III study PLEO-CMT trial. In the PLEO-CMT-FU extension trial, treatment with PXT3003 continued to demonstrate an improvement in the ONLS compared to placebo in patients with mild to moderate Charcot-Marie-Tooth Type 1A Disease.
 
Brickell announced that 190 patients completed the 52-week, 300 patient, Phase III ARGYLE trial, where treatment with sofpironium bromide improved the proprietary patient-reported Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) scale by one-point in 86.1% of patients that received a 5% gel and 85.8% with the 15% gel in patients with primary axillary hyperhidrosis. A two-point improvement in the HDSM-Ax score was achieved by 69.4% of 5% patients and 61.9% of 15% patients. Mostly mild or moderate adverse events were experienced by 22.5% of patients treated with sofpironium bromide gel 5% and 50.8% with 15%. Most adverse events were transient, so treatment discontinuations decreased over time.
 
AB Science announced that in a 580 patient, Phase IIb/III trial, treatment with masitinib plus docetaxel and prednisone improved progression-free survival compared to docetaxel and prednisone alone in patients with metastatic castration-resistant prostate cancer.
 
Published Research Updates
 
In the 36-week, 1,751 patient, Phase III, PRO2TECT Correction trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin, 1.43 vs 1.38 for a 0.05 g/dL difference, in CKD patients with anemia not on dialysis, who had not received previous ESA treatment. In the 36-week, 1,725 patient, Phase III, PRO2TECT Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin, 0.41 vs 0.42 for a 0.01 g/dL difference, in CKD patients with anemia not on dialysis, treated with an ESA. The incidence of MACE (composite endpoint of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke) with vadadustat compared to darbepoetin in the two PRO2TECT trials involving 3,471 CKD patients with anemia not on dialysis, found that 22% of vadadustat patients experienced a MACE event compared to 19.9% with darbepoetin for a hazard ratio of 1.17.
 
In the 36-week, 369 patient, Phase III, INNO2VATE Correction/Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin, 1.26 vs 1.58 for a 0.31 g/dL difference, in patients receiving chronic dialysis for end-stage renal disease. In the 36-week, 3,554 patient, Phase III, INNO2VATE Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin, 0.19 vs 0.36 for a 0.17 g/dL difference, in patients receiving chronic dialysis for end-stage renal disease. The incidence of MACE (composite endpoint of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke) in the two INNO2VATE trials involving 3,902 patients with anemia and dialysis-dependent chronic kidney disease, was 18.2% with vadadustat, which was non-inferior to 19.3% with darbepoetin for a hazard ratio of 0.96.
 
In the 260 patient, Phase II, INTELLANCE-2, open label trial treatment with depatuxizumab and temozolomide did not improve the overall survival rate compared totemozolomide alone in patients with EGFR-amplified glioblastoma. 
 
In a 4-week, 416 patient, Phase IIb, dose ranging trial, ensifentrine plus tiotropium increased FEV1 compared to tiotropium alone (ensifentrine dose-dependent increase of 77.5 mL to 124.2 mL) in patients with chronic obstructive pulmonary disease.