Update for November 2, 2021

Regulatory Update
 
The FDA approved asciminib (Scemblix, Novartis ), on 10/29/2021, to treat resistant Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). Asciminib was also approved for use in patients with Ph+ CML-C with a T315I mutation.
 
Lilly and Pfizer discontinued development of tanezumab in October 2021 after rejections from the FDA and EMA.
 
Lilly initiated a rolling BLA submission for donanemab to slow progression of Alzheimer’s Disease in October 2021. Lilly plans to compare donanemab to aducanumab in brain amyloid plaque clearance in early symptomatic Alzheimer's disease in the Phase III TRAILBLAZER-ALZ 4 trial.
 
Announced Research Updates
 
Cortexyme announced that in the 48-week, 643 patient, Phase II/III, GAIN trial, treatment with atuzaginstat did not slow the decline in cognitive function (ADAS-Cog11 and ADCS-ADL) compared to placebo in patients with mild-to-moderate Alzheimer’s disease. However in 242 patients with P. gingivalis DNA detectable in saliva, there was a 57% slowing of decline in ADAS-Cog11 with 80 mg and a 42% slowing of decline with 40 mg. There was no change in the decline of ADCS-ADL.
 
Rafael announced that in the 528 patient, Phase III AVENGER 500 trial, adding devimistat to modified FOLFIRINOX did not improve overall survival compared to mFOLFIRINOX alone in patients with metastatic adenocarcinoma of the pancreas.
 
Rafael announced the Phase III ARMADA 2000 trial was discontinued when an interim analysis found that adding devimistat to high dose cytarabine and mitoxantrone did not improve OS compared to high dose cytarabine and mitoxantrone alone in patients with relapsed or refractory acute myeloid leukemia. Devimistat also did not improve OS compared to mitoxantrone, etoposide and cytarabine or fludarabine, cytarabine, and filgrastim.
 
Published Research Updates
 
In the 12-week, 23 patient, Phase IIa ROCKET trial, 60% of patients treated with gaboxadol improved their Clinical Global Impressions-Improvement scale score in patients with Fragile X syndrome. 
 
After 16-weeks in the 26-week, 277 patient, Phase III ECZTRA 7 trial, 64.2% of patients treated with tralokinumab plus as needed topical corticosteroids achieved EASI-75 score compared to 50.5% with as needed topical corticosteroids alone in patients with severe atopic dermatitis who were not adequately controlled with or tolerant to cyclosporine.
 
In a three-year long-term extension of the VITAL trial, overall survival was 70% in patients treated with gemogenovatucel-T compared to 40% with placebo. RFS also showed benefit with gemogenovatucel-T.
 
In the 12-month, 97 patient, Phase II, LAICA trial, a 24 hour monthly infusion of levosimendan did not decrease rehospitalization for acute decompensated heart failure compared to placebo in patients with advanced heart failure.