Update for June 22, 2021

Regulatory Update 
 
The FDA rejected arimoclomol as a treatment for Niemann-Pick disease type C and requested additional safety and efficacy data. The FDA requested additional efficacy evidence beyond the single Phase II/III trial and data to support use of the 5-domain NPC Clinical Severity Scale (NPCCSS) as a primary endpoint.
 
The FDA accepted the BLA for Agenus’ balstilimab for the treatment of recurrent/metastatic cervical cancer and set a PDUFA date for 12/16/2021.
 
The FDA designated Lu-PSMA-617 a Breakthrough Therapy for the treatment of metastatic castration-resistant prostate cancer.
 
Announced Research Updates 
 
Sage and Biogen announced that in the 15-day, 543 patient, Phase III, WATERFALL trial, patients treated with zuranolone for 15 days had a 14.1 point decrease in their HAMD-17 total score compared to a 12.3 point decrease with placebo in patients with major depressive disorder, who had a HAMD-17 score of 24 or greater. At day 42, the difference between zuranolone and placebo were no longer significant.
 
Biogen announced that in the 12-month, Phase III, STAR trial, treatment with timrepigene emparvovec did not improve the number of patients that achieved an improvement of at least 15 letters in best corrected visual acuity (BCVA) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart compared to placebo in patients with choroideremia.
 
Actinium announced interim data from 113 patients in the 150 patient, Phase III, SIERRA trial, where 100% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant compared to 18% that received salvage chemotherapy in patients with active, relapsed or refractory acute myeloid leukemia.
 
Jiangsu HengRui announced that in a 67 patient, Phase II trial, treatment with pyrotinib plus trastuzumab, docetaxel and carboplatin resulted in a complete response rate of 71.4% compared to 36.7% trastuzumab, docetaxel and carboplatin alone in Chinese patients with stage 2-3 HER2-positive breast cancer and invasive carcinoma.
 
Aprea Therapeutics announced that in a 30 patient, Phase I/II trial, treatment with eprenetapopt plus azacitidine and venetoclax resulted in a complete response rate of 37% in patients with TP53 mutant AML.
 
Published Research Updates
 
In the 12-month, 131 patient, Phase III, PolarisDMD trial, treatment with edasalonexent did not improve the North Star Ambulatory Assessment (NSAA) compared to placebo in patients with Duchenne muscular dystrophy (DMD).
 
In the 24-month, 196 patient, Phase II, ADAMANT trial, treatment with AADvac1 did not differ from placebo in cognitive and functional tests in patients with mild AD. In a subgroup of patients with confirmed Alzheimer's disease biomarker profile, AADvac1 slowed the decline in cognitive and functional tests compared to placebo.
 
In the 26-week, 167 patient, Phase III, ADAPT trial, 68% of patients treated with efgartigimod achieved at least a 2-point improvement in their Myasthenia Gravis Activities of Daily Living (MG-ADL) score for four consecutive weeks compared to 30% with placebo in patients with myasthenia gravis on a stable drug regimen.
 
In the 24-week, 107 patient, Phase II, IMbark trial, 10.2% of patients treated with imetelstat achieved a ≥35% reduction in spleen volume and 32.2% achieved a ≥50% reduction in their Total Symptom Score, at in intermediate-2 or high-risk myelofibrosis patients who are relapsed or refractory to a Janus Kinase inhibitor. 
 
After 144 patients were treated for four to seven years in the DIAN-TU trial, treatment with gantenerumab did not reduce the decline in cognitive function, but did demonstrate a reduction in amyloid plaques, cerebrospinal fluid total tau, and phospho-tau181 and attenuated increases of neurofilament light chain. The solanezumab patients had a larger cognitive decline on some measures but did not demonstrate a reduction in biomarkers.