Update for April 20, 2021

Regulatory Update

The FDA approved estetrol in combination with drospirenone (Nextstellis, Mayne Pharma), on 4/16/2021 for the prevention of pregnancy.
 
The FDA has granted TTP399 Breakthrough Therapy status as an adjunctive therapy to insulin for the treatment of type 1 diabetes
 
The FDA designated CYNK-001 an Orphan Drug for the treatment of malignant gliomas.
 
Amylyx plans to initiate a Phase III, ALS trial for AMX0035 in 3Q21 to satisfy an FDA request for an additional trial before an NDA could be accepted. 
 
The FDA granted FibroGen’s pamrevlumab a Rare Pediatric Disease Designation for the treatment of Duchenne muscular dystrophy (DMD).
 
Ovid discontinued development of gaboxadol (OV101), in April 2021, for the treatment of Angelman and Fragile X syndrome, due to a lack of efficacy in both diseases.
 
Announced Research Updates
 
BeiGene announced interim data from 805 patients enrolled in the Phase III, RATIONALE 303 trial, where overall survival in patients treated with tislelizumab was 17.2 months compared to 11.9 months with docetaxel in patients with locally advanced or metastatic non-small cell lung cancer who had progressed on prior platinum-based chemotherapy. In patients with high PD-L1 expression, overall survival was 19.1 months with tislelizumab compared to 11.9 months with docetaxel.
 
Lilly announced that in 61 MCL patients from the Phase I, BRUIN trial, treatment with pirtobrutinib resulted in an overall response of 52%.
 
Immunic announced that in the 59 patient, cohort 2 of the 24-week, Phase II, EMPHASIS trial, compared to placebo, treatment with vidofludimus 10mg resulted in a 40% decrease in MRI detected lesions in the brain and spinal cord. 
 
TG Therapeutics announced that in the 96-week, 673 patient, Phase III ULTIMATE I trial, treatment with an intravenous infusion of ublituximab every six months resulted in a 60% relative reduction in the annualized relapse rate compared to daily oral teriflunomide in patients with relapsing forms of multiple sclerosis. And in the 96-week, 660 patient, Phase III ULTIMATE II trial, treatment with an intravenous infusion of ublituximab every six months resulted in a 49% relative reduction in the annualized relapse rate compared to daily oral teriflunomide in patients with relapsing forms of multiple sclerosis.
 
Published Research Updates
 
 In the 1-year, 501 patient, Phase III, HELIOS trial, treatment with ASP0113 did not reduce a composite endpoint of all-cause mortality and adjudicated CMV end-organ disease compared to placebo in CMV seropositive subjects who underwent hematopoietic stem cell transplantation. 
 
In a 6-week, 59 patient, Phase II trial, treatment with psilocybin did not improve the 16-item Quick Inventory of Depressive Symptomatology–Self-Report (QIDS-SR-16) compared to escitalopram in patients with long-standing, moderate-to-severe major depressive disorder. While psilocybin demonstrated a benefit in secondary outcomes, the analyses of these endpoints lacked correction for multiple comparisons
 
In the 4-week, 152 patient, open-label Phase II, MIROP trial, switching patients from pregabalin to mirogabalin reduced the visual analog scale by 15.7 mm in Japanese patients with peripheral neuropathic pain.
 
In a 36-week, 31 patient, open-label extension of a Phase II trial, treatment with resmetirom had an 11.1% reduction in MRI-PDFF in NASH patients with persistently mild to markedly elevated liver enzymes at the end of the primary study.
 
An 18-month, 854 patient, Phase II trial, found that at 12 months there was a 64% probability that treatment with lecanemab would result in a 25% or more reduction in the decline of the Alzheimer's Disease Composite Score (ADCOMS) compared to placebo, which missed the 80% threshold for the primary outcome. At 18 months biweekly lecanemab decreased brain amyloid and showed a reduction in the ADCOMS compared to placebo.