Update for November 9, 2021

Regulatory Update
 
The FDA extended the PDUFA date for ciltacabtagene autoleucel (cilta-cel) to 2/8/2022 to allow time to review newly submitted information describing an updated analytical method.
 
In a review of carbetocin nasal spray, the FDA did not find evidence to support efficacy in the treatment of hyperphagia, anxiety, and distress behaviors associated with Prader-Willi Syndrome. The FDA’s Psychopharmacologic Drugs Advisory Committee agreed with the FDA review and voted 12 to 1 to reject intranasal carbetocin.
 
The FDA accepted the BLA for toripalimab, in combination with gemcitabine and cisplatin, for the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma and set a PDUFA date for 4/1/2022.
 
Amylyx Pharmaceuticals submitted an NDA for AMX0035 to treat amyotrophic lateral sclerosis.
 
In a revised analysis, ICER found tezepelumab to reduce the annualized asthma exacerbation rate compared to non-biologic standard of care in patients with severe, uncontrolled asthma. In patients with eosinophilic and non-eosinophilic asthma, the benefit was smaller than what is considered clinically important. ICER found tezepelumab to be comparable to inferior to dupilumab in patients with steroid-dependent asthma, but insufficient evidence to compare tezepelumab to omalizumab in patients with eosinophilic asthma. ICER has estimated a health-benefit price benchmark (HBPB) range for tezepelumab of $9,000 to $12,100 per year to achieve a QALY between $100,000 and $150,000 per year.
 
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.
 
In the 52-week, 2,964 patient, open label, Phase III, ASCEND-D trial, patients treated with daprodustat achieved an increase in hemoglobin of 0.28 g/dL compared to a 0.1 g/dL increase with epoetin or daprodustat in CKD patients who were undergoing dialysis. In the 52-week, 3,872 patient, open label, Phase III, ASCEND-ND trial, patients treated with daprodustat achieved an increase in hemoglobin of 0.74 g/dL compared to a 0.66 g/dL increase with epoetin or daprodustat in CKD patients who were undergoing dialysis.
 
DBV Technologies announced that in a 2.5 year extension of the REALISE trial (1,093 total days of exposure to an epicutaneous peanut patch), most children experienced a mild or moderate ADR with the most common being pruritus and application-site erythema.