Update for November 11, 2020

Thank-you to active and veteran armed forces service men and women for your sacrifice.

Regulatory Update
 
The FDA rejected SPN-812 (Viloxazine, Supernus Pharmaceuticals) for the treatment of ADHD, in November 2020, due to questions regarding analytical testing.
 
The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted 0 yes, 10 no and 1 uncertain on whether there was enough evidence to recommend approval of Biogen/Eisai’s aducanumab for the treatment of Alzheimer’s disease.
 
The FDA accepted the NDA for BioMarin’s vosoritide for the treatment of achondroplasia and set a PDUFA date for 8/20/2021. The FDA will not have an advisory committee review the drug, but reminded BioMarin that it requested a two-year study in 2018. BioMarin feels that due to the positive outcome in a 121 patient, Phase III study and five-year data from Phase II trials, it has submitted enough data to warrant approval.
 
The FDA requested additional information on Bluebird Bio’s beta beglogene darolentivec manufacturing, to prove analytical comparability between clinical-grade and commercial-grade manufacturing, therefore Bluebird has delayed submission of a BLA to late 2022.
 
In September 2020, the FDA requested that Sarepta use a different potency assay when manufacturing its microdystrophin gene therapy, before using the drug in a Phase III trial. In November 2020, Sarepta announced they would delay the Phase III trial and instead run a 10 patient, open-label trial to evaluate the safety and validate the commercial manufacturing process. The FDA has not indicated if the open-label trial would satisfy the requirements for approval or if the Phase III trial will be needed.
 
Orphazyme submitted an MAA to the EMA for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).
 
Announced Research Updates
 
Aurinia announced that in the 12-week, 508 patient, Phase II/III, AUDREY trial, patients treated with voclosporin ophthalmic drops did not achieve an improvement in the Schirmer Tear Test at 4 weeks compared to placebo in patients with dry eye syndrome. Aurinia ceased development of voclosporin to treat dry eye syndrome after failure of the AUDREY trial.
 
BMS announced that in the 16-week, 666 patient, Phase III, POETYK PSO-1 trial, more patients treated with deucravacitinib achieved PASI 75 and a Physician's Global Assessment (sPGA) score of 0 to 1 compared to apremilast in patients with moderate to severe psoriasis.
 
BMS announced that in the 16-week, 180 patient, Phase II trial, 52.9% of patients treated with deucravacitinib 6 mg and 62.7% of patients treated with 12 mg achieved ACR-20 compared to 31.8% who received placebo in patients with psoriatic arthritis.
 
Sanofi paused dosing in clinical trials for fitusiran, for treating Hemophilia and Hemophilia B, to investigate new adverse effects.
 
Arena announced that in the 12-week, 140 patient, Phase IIb, ADVISE trial, treatment with etrasimod did not improve the Eczema Area and Severity Index (EASI) compared to placebo in patients with moderate-to-severe atopic dermatitis.
 
ViiV announced the early discontinuation of the 3,223 patient, Phase III, HPTN 084 study, when an interim analysis demonstrated that treatment with cabotegravir injected every two months reduced the incidence of acquiring HIV compared to daily oral emtricitabine/tenofovir disoproxil fumarate (0.21% incidence compared to 1.79%) in patients at increased risk of HIV acquisition.
 
Published Research Updates
 
In an 8-week, 27 patient, Phase II trial, two day-long psilocybin sessions plus 11 hours of psychotherapy reduced the GRID-HAMD score from 22.8 to 8 (from moderate to mild depression) by week four, which was maintained through week eight, in patients with moderate-to-severe major depressive disorder.
 
In a 2.5-year, 5,113 patient, open-label, long-term cardiovascular safety trial, treatment with LG Life Sciences/Sanofi’s gemigliptin resulted in a low incidence of cardiovascular ADR. The estimated incidence from Cox proportional hazard model was 1.35%.