Update for September 22, 2020

Regulatory Update
 
The FDA accepted the NDA for Orphazyme’s arimoclomol for the treatment of Niemann-Pick disease Type C and set a PDUFA date for 3/17/2021.
 
The FDA accepted the NDA for ChemoCentryx’s avacopan for the treatment of ANCA-associated vasculitis and set a PDUFA date for 7/7/2021.
 
The FDA granted Gilead’s magrolimab a Breakthrough Therapy designation for the treatment of myelodysplastic syndrome.
 
The FDA designated MedImmune’s mavrilimumab an Orphan Drug for the treatment of giant cell arteritis.
 
Apellis submitted an NDA and MAA for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
 
Agenus began a rolling BLA for balstilimab for the treatment of recurrent/metastatic cervical cancer.
 
Announced Research Updates
 
Myovant announced that in a 28-week, 477 patient, open-label, long-term extension trial for LIBERTY 1 and 2, 87.7% of patients treated with relugolix, estradiol and norethindrone acetate achieved a menstrual blood loss volume of less than 80 mL and an 89.9% reduction in menstrual blood loss.
 
Marinus announced that in the 10-week, 101 patient, Phase III, Marigold trial, treatment with ganaxolone resulted in a 32.2% reduction in 28-day major motor seizure frequency compared to a 4% decrease with placebo in children and young adults with CDKL5 deficiency disorder (CDD). Marinus plans to submit an NDA for ganaxolone for the treatment of CDD in mid-2021 and an MAA by the end of 3Q21.
 
BeiGene announced results from the 334 patient, open-label, Phase III, Chinese RATIONALE 304 trial, where treatment with tislelizumab added to standard chemotherapy (pemetrexed plus carboplatin or cisplatin) resulted in progression-free survival (PFS) of 9.7 months compared to a PFS of 7.6 months in patients treated with standard chemotherapy alone in patients with non-squamous non-small cell lung cancer.
 
Mirati announced interim results from 30 patients enrolled in a 330 patient, Phase II, open-label trial, where treatment with sitravatinib and nivolumab resulted in a 37% objective response rate in patients with advanced or metastatic urothelial carcinoma.
 
Clementia and Ipsen announced interim data after 18-months from 97 patients enrolled in the 48-month, 110 patient, Phase III, MOVE trial, where treatment with palovarotene reduced heterotopic ossification volume by 62% compared to a natural history group of patients with fibrodysplasia ossificans progressiva (FOP).
 
 
Published Research Updates
 
In a 12-week, 483 patient, Phase II, dose-ranging trial, treatment with relugolix reduced pelvic pain 6.2 points with 10 mg, 8.1 points with 20 mg and 10.4 points with 40 mg compared to a 10.6 point decrease with leuprorelin and 3.8 point decrease with placebo in patients with pain associated with endometriosis.
 
In a 12-week, 315 patient Phase II/III trial, adults treated with dasotraline 4-8 mg decreased binge days by one day compared to placebo in adults ages 18–55 years with moderate to severe binge eating disorder.
 
In a 24-week, 242 patient, Phase II trial, patients treated with oligomannate had a decrease in their ADAS-Cog12 Score of 1.39 points with 600 mg and 2.58 points with 900 mg compared to 1.45 points with placebo in Chinese patients with mild to moderate Alzheimer’s disease.
 
In a 66 patient, Phase II, open label, dose ranging trial, treatment with rozanolixizumab improved platelet count to 50x109/L or > in all dosing groups in patients with primary immune thrombocytopenia (ITP).
 
In a 164 patient, Phase I trial, Ad26.ZEBOV and MVA-BN-Filo vaccines were well tolerated and elicited strong, durable humoral and cellular immune responses for the Ebola virus in healthy patients. 
 
The 172 patient, Phase III, open-label, SANET-p trial was stopped early when an interim analysis found that treatment with surufatinib resulted in progression-free survival (PFS) of 10.9 months compared to 3.7 months with placebo in Chinese patients with progressive, advanced pancreatic neuroendocrine tumors. The 198 patient, Phase III, open-label, SANET-ep trial was stopped early when an interim analysis found that treatment with surufatinib resulted in PFS of 9.2 months compared to 3.8 months with placebo in Chinese patients with with unresectable or metastatic, well differentiated, extrapancreatic neuroendocrine tumor.
 
In the 129 patient, Phase I/II, CodeBreak 100 trial, treatment with sotorasib resulted in progression-free survival of 6.3 months in patients with non–small-cell lung cancer and four months in colorectal cancer in patients with advanced solid tumors harboring the KRAS p.G12C mutation.
 
In an extension of a Phase II induction trial, 15% of induction non-responders that received mirikizumab 600 mg and 43.8% of non-responders that received mirikizumab 1000 mg achieved clinical remission after an additional 12-weeks of treatment (24-weeks total). Among initial non-responders 26.3% that achieved clinical remission maintained it at 52-weeks.