Update for June 9, 2020

Regulatory Update
 
The FDA placed a hold on the AAVance trial, due to abnormalities in MRI imaging. Since the trial is fully enrolled and LYS-SAF302 is a single treatment, gene therapy for Sanfilippo syndrome, Lysogene stated the forecast completion in January 2022 is not expected to be delayed.
 
The FDA designated MacroGenics’ margetuximab an Orphan Drug for the treatment of gastric and gastroesophageal junction cancer. 
 
Announced Research Updates
 
Tenax announced that in the 6-week, 37 patient, Phase II, HELP trial, levosimendan did not reduce pulmonary capillary wedge pressure (PCWP) during exercise compared to placebo, but did reduce PCWP at rest in patients with pulmonary hypertension and heart failure with preserved ejection fraction.
 
Gilead and Galapagos announced that in the 52-week, 1,759 patient, Phase III, FINCH 1 trial, the percentage of patients that achieved ACR20/50/70 with filgotinib 200 mg was 78%/62%/44%, filgotinib 100 mg was 76%/59%/38%, compared to adalimumab with 74%/59%/39% in patients with moderate-to-severe rheumatoid arthritis with an inadequate response to MTX. Gilead and Galapagos announced that in the 52-week, 975 patient, Phase III, FINCH 3 trial, the percentage of patients that achieved ACR20/50/70 with filgotinib 200 mg/MTX was 75%/62.3%/47.8%, filgotinib 100 mg/MTX was 73.4%/59.4%/40.1%, compared to filgotinib 200 mg monotherapy with 74.8%/61.4%/45.2%, and methotrexate monotherapy was 61.8%/48.3%/29.8% in patients with moderate-to-severe rheumatoid arthritis naive to methotrexate.
 
Alnylam announced that in the 6-month, 39 patient, Phase III, ILLUMINATE-A trial, treatment with lumasiran decreased 24-hour urinary oxalate excretion by 53.5% compared to placebo in patients with primary hyperoxaluria type 1.
 
Sarepta announced 1-year interim results from 3 patients enrolled in a Phase I/IIa, open-label trial, treatment with a low dose of SRP-9003 improved the North Star Assessment for Dysferlinopathy (NSAD), time to rise, four-stair climb, 100-m walk test and 10-meter walk test in patients with limb-girdle muscular dystrophy. In the same trial, 3 patients that received a high dose of SRP-9003 demonstrated a higher mean expression of transduced beta-SG in the muscle sarcolemma at 60-days compared to that seen with the lower dose.
 
Published Research Updates
 
In a 12-week, 391 patient, Phase III, JADE MONO-2 trial, an Investigator Global Assessment (IGA) response of clear [0] or almost clear [1], with improvement of 2 or more grades, was achieved by 38.1% of patients treated with abrocitinib 200 mg and 28.4% of those treated with 100 mg compared to 9.1% with placebo in patients with moderate to severe atopic dermatitis. A 75% improvement in the Eczema Area and Severity Index score (EASI-75) was achieved in 61% of patients treated with abrocitinib 200 mg and 44.5% of those treated with 100 mg compared to 10.4% with placebo.
 
In a 24-week, 303 patient, Phase III trial, roxadustat, given orally three times per week, was non-inferior to darbepoetin, injected once per week, in maintaining hemoglobin between 10 to 12 g/dL (95.2% vs 91.3%) in Japanese hemodialysis patients.
 
In a 27 patient, Phase II, open-label trial, treatment with camrelizumab added to gemcitabine, vinorelbine and pegylated liposomal doxorubicin followed by camrelizumab maintenance resulted in an overall response in 74% of patients with relapsed or refractory primary mediastinal B-cell lymphoma.