Update for the Week Ending May 16, 2020

Regulatory Update
 
The FDA approved ripretinib (Qinlock, Deciphera Pharmaceuticals), on 5/18/2020, for the treatment of advanced gastrointestinal stromal tumors (GISTs).
 
The FDA accepted the BLA for Sanofi’s sutimlimab for the treatment of hemolysis in adult patients with cold agglutinin disease (CAD) and set a PDUFA date for 11/13/2020.
 
The FDA accepted the NDA for Rhythm Pharmaceuticals’ setmelanotide for the treatment of pro-opiomelanocortin deficiency obesity and leptin receptor deficiency obesity and set a PDUFA date of 11/27/2020.
 
Sunovion discontinued development of dasotraline and withdrew the NDAs for the treatment of moderate-to-severe binge eating disorder and attention deficit hyperactivity disorder on 5/13/2020. The drug had a PDUFA date of 5/14/2020.
 
Cytodyn completed a rolling BLA for use of leronlimab in combination therapy with HAART for treatment of HIV.
 
Announced Research Updates
 
MyoKardia announced that in the 30-week, 251 patient, Phase III EXPLORER-HCM trial, 37% of patients treated with mavacamten reached the composite endpoint of achievement of a 1.5 mL/kg/min or > improvement in peak VO2 accompanied by an improvement of at least a 1 NYHA functional class or achievement of a 3.0 mL/kg/min or > improvement of peak VO2 with no worsening in NYHA functional class compared to 17% reaching the endpoint with placebo in patients with NYHA Class II or III obstructive hypertrophic cardiomyopathy.
 
Soligenix announced that in cycle 1 of the 8-week, 166 patient, Phase III FLASH trial, 16% of patients treated with 6-weeks of SGX301 had at least a 50% reduction in lesions compared to 4% with placebo in patients with early-stage cutaneous T-cell lymphoma. In the open-label, 12-week, 155 patient, cycle 2 of the FLASH trial, 40% of patients treated with 12-weeks of SGX301 had at least a 50% reductions in lesions.
 
Genfit announced that in the 72-week, 1,070 patient, Phase III, RESOLVE-IT trial, treatment with elafibranor did not result in resolution of non-alcoholic steatohepatitis (NASH) without worsening of fibrosis compared to placebo (19.2% vs 14.7%) in patients with biopsy proven NASH with stage 2 or stage 3 fibrosis.
 
Protalix announced that in the 12-month, Phase III, open-label, BRIDGE trial, 22 patients with Fabry disease stabilized on agalsidase alfa were switched to pegunigalsidase alfa and improved their annualized change in eGFR from ‑5.90 mL/min/1.73m2/year to -1.19 mL/min/1.73m2/year.
 
BMS announced that in the 128 patient, Phase II, dose-ranging, open-label, KarMMa trial, treatment with idecabtagene vicleucel resulted in an overall response rate of 73% in patients with resistant multiple myeloma. The FDA rejected the BLA for ide-cel in May 2020 and requested additional information on the manufacturing processes. BMS hopes to provide the new documentation and refile the BLA before the end of July.
 
Blueprint announced that in the 116 patient, Phase I/II, open label, ARROW trial, treatment with pralsetinib resulted in a 65% ORR in patients with RET fusion-positive non-small cell lung cancer and 61% in 80 patients that were previously treated with platinum-based chemotherapy.
 
J&J announced data after 11.5-months in the 29 patient, Phase Ib/II CARTITUDE-1 trial, where JNJ-4528 demonstrated an overall response rate of 100% and complete response of 86% in patients with relapsed multiple myeloma.
 
Amgen announced data from 42 advanced colorectal cancer patients with a KRAS p.G12C mutation enrolled in the Phase I, open-label, dose ranging, CodeBreak 100 trial, where treatment with AMG 510 led to an overall response rate in 3/42 of all patients and 3/25 that received the highest dose (960 mg). Amgen announced data from 22 patients with 10 cancers other than non-small cell lung cancer or colorectal cancer but with a KRAS p.G12C mutation enrolled in the Phase I, open-label, dose ranging, CodeBreak 100 trial, where treatment with AMG 510 led to a partial response rate in 3/22 patients (10 patients had pancreatic cancer). Amgen plans to file a BLA for AMG 510 by the end of 2020.
 
Helsinn and MEI Pharma announced that in a 64 patient, Phase II, open-label trial, 33% of patients treated with pracinostat 45 mg and azacitidine achieved a complete response rate in patients with high-risk myelodysplastic syndromes.
 
AB Science SA announced that in a 36-week, 355 patient, Phase III trial, treatment with masitinib reduced the severe asthma exacerbation rate by 35% compared to placebo in patients with severe asthma uncontrolled by oral corticosteroids.
 
Urovant announced that in a 40-week, 505 patient, blinded extension of the EMPWUR trial, patients treated with vibegron had a larger decrease in micturitions (-2.2 vs -1.7 per 24 hours), urgency (-3.4 vs -3.2 per 24 hours), and total urinary incontinence episodes (-2.5 vs -1.9 per 24 hours) compared to tolterodine.
 
ImmunoGen announced that in the 60 patient, Phase Ib/II, open-label, FORWARD II trial, treatment with mirvetuximab soravtansine plus bevacizumab resulted in an overall response rate of 43% in patients with folate receptor alpha-positive, resistant ovarian cancer.
 
ReGenTree announced that in the 18 neurotrophic keratopathy patient, Phase III, SEER-1 trial there was no difference between RGN-259 and placebo in corneal wounds healed, but there were more patients with complete corneal healing (6/10 vs 1/8).
 
Published Research Updates
 
In a 22 patient, Phase II trial, treatment with tavokinogene telseplasmid plus pembrolizumab resulted in a 41% objective response rate in patients with advanced melanoma with low frequencies of checkpoint positive cytotoxic lymphocytes.
 
In a 56 patient, Japanese trial, prophylactic landiolol after surgery did not reduce the incidence of postoperative atrial fibrillation compared to placebo in post-esophagectomy patients.
 
In the 448 patient, Phase III, ESCORT trial, overall survival for patients treated with camrelizumab was 8.3 months compared to 6.2 months with docetaxel or irinotecan in patients with metastatic esophageal squamous cell carcinoma.
 
In a 6-month 90 patient, Phase II trial, followed by a 12-month, 77 patient extension, treatment with atabacestat did not improve MMSE scores, and RBANS were worse, compared to placebo in patients with early Alzheimer’s disease.
 
In a 375 patient, Phase IIb/III trial, remimazolam was non-inferior to propofol as a sedative hypnotic for general anesthesia.