Update for March 15, 2023

Regulatory Update
 
The FDA approved zavegepant nasal spray (Zavzpret, Pfizer), on 3/10/2023, for the acute treatment of migraine with or without aura.

• Zavegepant was originally called vazegepant. The chemical properties of zavegepant allow it to be delivered by nasal inhalation and oral administration. 
• Pfizer gained zavegepant when it bought Biohaven Pharmaceuticals in May 2022. As part of the deal, Pfizer also owns rimegepant (Nurtec ODT), an approved oral CGRP receptor antagonist used for the same indication as zavegepant.​
  

The FDA approved trofinetide (Daynue, Acadia Pharmaceuticals), on 3/10/2023, for the treatment of Rett syndrome.
 
The FDA extended the PDUFA date for the valoctocogene roxaparvovec in order to review newly submitted three-year data from the Phase 3 GENEr8-1 study. The new PDUFA date is 6/30/2023.
 
The FDA accepted the resubmitted BLA for remestemcel-L to treat steroid-refractory acute graft versus host disease and set a PDUFA date for 8/2/2023.
 
The FDA accepted the NDA for eplontersen, for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy and set a PDUFA date for 12/22/2023.
 
Announced Research Updates
 
Cytokinetics announced 10-week data from the 41 patient, cohort IV of the REDWOOD-HCM trial, where NT-proBNP was decreased by 66% with aficamten added to background therapy and 54% of patients saw at least a one-step improvement in their NYHA Functional Class in patients with non-obstructive HCM. Cytokinetics announced that in the 48-week, 19 patient,Phase II, FOREST-HCM trial, treatment with aficamten resulted in a 32 mmHg reduction in resting LVOT-G and a 47 mmHg reduction in Valsalva LVOT-G.
 
Aclaris announced that in a 12-week, 95 patient, Phase IIa trial, treatment with zunsemetinib did not decrease inflammatory nodule/abscess count compared to placebo in patients with moderate to severe hidradenitis suppurativa.
 
Incyte discontinued the Phase 3, LIMBER-304 trial when an interim analysis found that adding parsaclisib to ruxolitinib did not reduce spleen volume compared to ruxolitinib alone in patients with myelofibrosis who had an inadequate response to treatment with ruxolitinib.
 
Redx announced that in the 16 patient Module 2 of the Phase II PORCUPINE2 trial, treatment with RXC004 did not result in appropriate efficacy to justify development of the drug used alone to treat biliary tract cancer.
 
Lilly announced 10-year follow-up data from the 4.5-year, 1,100 patient, Phase III, A4 trial, where almost six years after the end of the trial, solanezumab had not slowed the decline in cognition due to Alzheimer's disease (AD) or decreased amyloid plaque in patients with amyloid plaque but no AD symptoms. Solanezumab binds only to soluble amyloid-beta protein and was not expected to significantly remove deposited amyloid plaques.
 
Adicet Bio announced interim data from 16 patients enrolled in the 78 patient, Phase I, GLEAN-1 trial, where treatment with ADI-001 resulted in overall response rate (ORR) of 75% and a complete response (CR) rate of 69% in patients with relapsed/refractory B-cell non-Hodgkin lymphoma.
 
Published Research Updates
 
In the 52-week, 433 patient, Phase III, ELEVATE UC 52 trial, 27% of patients treated with etrasimod achieved remission after the 12-week induction period compared to 7% with placebo in patients with moderately-to-severely active ulcerative colitis with an inadequate or loss of response or intolerance to at least one approved ulcerative colitis therapy. After 52 weeks 32% of etrasimod patients maintained remission compared to 7% with placebo. In the 12-week, 354 patient, Phase III, ELEVATE UC 12 trial, 25% of patients treated with etrasimod achieved remission compared to 15% with placebo in patients with ulcerative colitis who had an inadequate or loss of response or intolerance to at least one approved ulcerative colitis therapy.
 
In the 24-week, 323 patient, Phase III, STELLAR trial, patients treated with sotatercept improved their six-minute walk distance by 34.4 meters compared to 1 meter with placebo in patients with pulmonary arterial hypertension.
 
In a 12-week, 415 patient, Phase IIIb trial, patients treated with gefapixant achieved a Leicester Cough Questionnaire (LCQ) Total Score of 4.34 compared to 3.59 with placebo (0.75 point improvement) in patients with recent-onset refractory chronic cough or unexplained chronic cough.
 
In the 142 patient, Phase III, DeFi trial, treatment with nirogacestat resulted in an 88% lower risk of disease progression compared to placebo in patients with progressive desmoid tumors.
 
In a 30-day, 900 patient trial, the immune response for administering a yellow-fever vaccine (YF-17D) and dengue vaccine (TAK-003) sequentially or concomitantly was non-inferior in healthy patients. The immune repose reached for DENV-1 did reach non-inferiority, but titers were similar to those observed in other TAK-003 trials.
 
In a 95 patient, Phase Ib trial, treatment with magrolimab plus azacitidine resulted in a complete remission rate of 33% and an overall response rate of 75% in patients with untreated higher-risk myelodysplastic syndromes.