Weekly Update for the Week Ending November 2, 2019

Regulatory Update
 
The FDA approved diroximel fumarate (Vumerity, Biogen/Alkermes) on 10/30/2019 for the treatment of relapsing forms of multiple sclerosis. Diroximel fumarate is an oral prodrug of monomethyl fumarate, which allows the 505(b)(2) pathway based on Biogen’s dimethyl fumarate (Tecfidera) to be used. Biogen licensing diroximel fumarate from Alkermes.
 
Chugai announced that in October 2019 the FDA accepted the BLA and the EMA accepted the MAA of satralizumab for the treatment of neuromyelitis optica spectrum disorder.
 
Announced Research Updates
 
Actinium announced interim data from 75 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.
 
Athenex announced that in two 57-day, Phase III trials, treatment of actinic keratosis with tirbanibulin resulted in complete clearance in 44% and 54% of the patients compared to 5% and 13% for vehicle treated groups. One-year follow-up found recurrence rates of 74% and 72% with tirbanibulin. Athenex plans to submit an NDA for tirbanibulin in 1Q21 and an MAA in 2Q21.
 
AZ announced preliminary results from the 1,000 patient, Phase III, open-label, POSEIDON trial of patients with metastatic non-small cell lung cancer with PD-L1 expression levels, but not mutation in the epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) gene. Progression free survival was improved with both durvalumab, tremelimumab and platinum-based chemotherapy and durvalumab and platinum-based chemotherapy compared to platinum-based chemotherapy alone as first line treatment
 
AZ announced that in a 26 patient, Phase II, open-label trial, treatment with selumetinib resulted in a partial response in 71% of patients with patients with neurofibromatosis type 1.
 
TG Therapeutics announced that in the 112 patient, open-label, Phase IIb, UNITY-NHL trial, treatment with umbralisib achieved the predetermined target overall response rate of 40% to 50% in patients with follicular lymphoma who have received at least 2 prior lines of therapy including an anti-CD20 monoclonal antibody and an alkylating agent.
 
Cyclerion announce that in a 12-week, 156 patient, Phase II trial, treatment with praliciguat did not improve the urine albumin to creatinine ratio compared to placebo in patients with type 2 diabetes and diabetic nephropathy. Cyclerion also announce that in the 12-week, 196 patient, Phase II, CAPACITY trial, treatment with praliciguat did not improve cardiopulmonary exercise testing compared to placebo in heart failure patients with preserved ejection fraction. Cyclerion is discontinuing development of praliciguat for the treatment of heart failure with preserved ejection fraction and plans to license the drug to another company for development as a treatment for diabetic nephropathy.
 
Published Research Updates
 
In a 3 year, 3,289 patient, Phase IIb trial, immunization with M72/AS01E resulted in 0.3 active pulmonary tuberculosis cases per 100 person-years compared to 0.6 cases per 100 person-years with placebo in African patients with withlatent Mycobacterium tuberculosis infection. M72/AS01E's efficacy was 49.7%. 70% of the patient population had already received the BCG vaccine.
 
The 26-week interim data from a 52-week, 3 patient, Phase IIb trial, found that 26-weeks after a single infusion of etranacogene dezaparvovec resulted in a mean increase in Factor IX (FIX) activity of 47% of normal range (individual results: 33%, 51%, 57%) and no patient required a Factor IX infusion, experienced a bleeding event or required immunosuppression.