Update for March 23, 2021

Regulatory Update
 
The FDA approved ponesimod (Ponvory, Johnson & Johnson), on 1/19/2021, for the treatment of relapsing forms of multiple sclerosis.
 
The FDA accepted the NDA for Merck’s belzutifan for the treatment of von Hippel-Lindau disease-associated renal cell carcinoma, not requiring immediate surgery. A PDUFA date was set for 9/15/2021.
 
The FDA accepted the NDA for mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy and set a PDUFA date for 1/28/2022.
 
Announced Research Updates
 
Lilly announced that in the 12-week, 1,160 patient, Phase III, LUCENT-1 trial, more patients treated with mirikizumab 200 mg achieved remission compared to placebo in patients with moderate-to-severe active ulcerative colitis (UC).
 
Solid Biosciences announced preliminary results from six patients at 12-24 months in the Phase I/II, IGNITE-DMD trial, where treatment with SGT-001 microdystrophin gene therapy resulted in an improvement of 1 point in the North Star Ambulatory Assessment (NSAA) score with a low dose of SGT-100 and 0.3 points with a high dose compared to a 4 point decline with placebo in children with Duchenne muscular dystrophy. In the 6-Minute Walk Test (6MWT) treatment with SGT-100 improved the distance walked by 49.7 meters with the high dose and 37 meters with the low dose compared to placebo. Forced vital capacity (% predicted FVC) at one year improved 3.9% and 16.7% with the low-dose and high-dose groups compared to a 10.7% decline with placebo.
 
Sarepta announced two-year interim results from three patients enrolled in a Phase I/IIa, open-label trial, where that treatment with a low dose of SRP-9003 improved the North Star Assessment for Dysferlinopathy (NSAD) by 5.7 points in patients with limb-girdle muscular dystrophy. Patients also saw an improvement in time to rise, four-stair climb, 100-meter walk test and 10-meter walk test. Sarepta also announced one-year results from the same trial, where three patients received a high dose of SRP-9003 and demonstrated a four point improvement in their NSAD, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. Both doses of SRP-9003 increased beta-sarcoglycan protein levels in skeletal and cardiac muscle.
 
Sage Therapeutics announced that in the 12-month 725 patient, open-label, Phase III, SHORELINE trial, a 14-day treatment with zuranolone 30 mg resulted in 489 patients responding to initial treatment. Among responders, 42.9% maintained remission at 1-year, while 25.6% required a second 14-day course of therapy, 11.9% required 3 courses, 10.8% used 4 courses, and 8.8% needed 5 courses. Repeat courses of zuranolone were given 56 days after completion of the 14-day course of treatment.
 
Strongbridge Biopharma announced that in the 8-week, 44 patient, Phase III, LOGICS endogenous Cushing’s syndrome trial, 54.5% of patients that were stabilized on levoketoconazole, but had their therapy withdrawn, had a loss of mean urinary free cortisol response compared with to patients that remained on levoketoconazole.
 
Published Research Updates
 
The incidence of fractures with odanacatib over five years was examined in data from the LOFT trial and its long-term extension. The incidence of subtrochanteric, femoral shaft or hip fractures was 1.93% with odanacatib compared to 3.11% with placebo. The incidence of hip fracture only was 1.53% with odanacatib compared to 3.03% with placebo. Though rare, patients treated with odanacatib had more low-energy fractures and atypical fractures.
 
In the 16-week, 80 patient, open-label, Phase III, PEGASUS trial, patients treated with pegcetacoplan increased their hemoglobin by 3.84 g/dL more than patients treated with eculizumab in patients with paroxysmal nocturnal hemoglobinuria with hemoglobin levels lower than 10.5 g per deciliter despite eculizumab therapy. 
 
In a 50 patient, Phase II trial, inhaled levosimendan was non-inferior to intravenous levosimendan in lowering systolic pulmonary artery pressure (PAP) in children ages one to five scheduled for cardiac surgery.
 
In a 36-week, 818 patient, Phase III trial, patients treated with oligomannate had a decrease in their ADAS-Cog12 Score of 2.15 points less than placebo in Chinese patients with mild to moderate Alzheimer's disease.
 
In the 16-week, 215 patient, Phase II, ECZTRA 5 trial, tralokinumab was non-inferior to placebo for positive anti-tetanus (91.9% vs 96.1%) and anti-meningococcal (86.0% vs 84.2%) responses in patients with moderate-to-severe atopic dermatitis.