Update for October 20, 2020

Regulatory Update
 
The FDA approved a combination of three Ebola monoclonal antibodies, atoltivimab, maftivimab, and odesivimab-ebgn (Inmazeb, Regeneron) for the treatment of Zaire ebolavirus (Ebola virus) infection in adult and pediatric patients.
 
The FDA designated Inventiva’s lanifibranor a Breakthrough Therapy for the treatment of non-alcoholic steatohepatitis (NASH).
 
Scynexis submitted an NDA for ibrexafungerp for the treatment of vulvovaginal candidiasis.
 
The EMA has granted PRIME status to Akero’s efruxifermin for the treatment of NASH.
 
CHMP recommended approval to EMA of Alnylam’s lumasiran for the treatment of hyperoxaluria type 1.
 
CHMP recommended approval to EMA of the long-acting injectable combination of ViiV’s cabotegravir (Vocabria) and Janssen’s rilpivirine (Rekambys) for the treatment of Human Immunodeficiency Virus type 1.
 
Announced Research Updates
 
Gilead and Galapagos announced results from the 58-week, 1,348 patient, Phase IIb/III, SELECTION trial that evaluated filgotinib as a treatment of moderately to severely active ulcerative colitis. 

• During the first 10 weeks of the trial patients received filgotinib 100 mg, 200 mg or placebo. Treatment with filgotinib 100 mg did not differ from placebo in the percentage of patients achieving remission. However, compared to placebo, remission was achieved in more biologic-naive patients treated with filgotinib 200 mg (26.1% vs. 15.3%) and in biologic-experienced patients (11.5% vs. 4.2%). 
• The 558 patients that achieved remission were re-randomized to filgotinib or placebo for an additional 48 weeks. In the maintenance part of the trial, 37.2% of patients treated with filgotinib 200 mg achieved clinical remission compared to 11.2% with placebo

After failure in the Phase III, LAUREL trial, Roche discontinued development of etrolizumab as a treatment for ulcerative colitis.
 
Lysogene is investigating the death of one of 18 patients that received LYS-SAF302 in the Phase II/III AAVance trial. LYS-SAF302 is a gene therapy that provides the SGSH gene, to produce an enzyme that is absent in children with Sanfilippo syndrome.
 
Astex and Otsuka announced that guadecitabine did not improve overall survival in 302 acute myeloid leukemia patients in the Phase III ASTRAL-2 trial or in 417 myelodysplastic syndrome/chronic myelomonocytic leukemia patients in the Phase III ASTRAL-3 trial.
 
Revance announced that in the 36-week, 301 patient, Phase III, ASPEN-1 trial, treatment with a single dose of daxibotulinumtoxinA resulted in a decrease in the average Toronto Western Spasmodic Torticollis Rating Scale scores at weeks four and six of 12.7 points with 125 units and 10.9 points with 250 units compared to 4.3 points with placebo in patients with cervical dystonia. Researchers estimated the median duration of effect as 24.0 weeks with 125 units and 20.3 weeks with 250 units, which corresponded to a loss of 80% of the peak treatment effect.
 
Published Research Updates
 
A 9,302 patient, Phase III trial was stopped early after an interim analysis failed to find a benefit with Sanofi Pasteur’s Clostridium difficile vaccine compared to placebo in preventing symptomatic C. difficile infection (0.29 infections per 100 person-years vs 0.28 infections per 100 person-years) in patients 50 years or older who were at high risk of C difficile infection.
 
In a 35-week, 135 patient, open label extension of Amylyx Pharmaceuticals’ CENTAUR trial, all patients received AMX0035 during the extension, where, overall survival was 25 months in patients that originally received AMX0035 for the first 24-weeks and 18.5 months for those who originally received placebo.