Update for August 11, 2022

Regulatory Update
 
The FDA accepted the NDA for donanemab for the treatment of Alzheimer’s disease and set a PDUFA date for 1Q2023
 
The FDA informed Travere that interim data from the 404 patient, Phase III DUPLEX trial was not enough to support approval of sparsentan. Final data from the DUPLEX trial will be available in the first half of 2023 with a projected NDA submission by the end of the year if the data is positive.
 
The FDA extended PDUFA date for omaveloxolone by three months to allow additional time to review newly submitted data. The new PDUFA date is 2/28/2023.
 
The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) will meet on 9/22/2022, to review the BLA for RBX2660 to reduce recurrent C. difficile infection after antibiotic treatment.
 
Announced Research Updates
 
Ambit Biosciences announced that in a 10 patient, Phase I/II trial, 90% of patients treated with quizartinib plus azacitidine achieved a complete response in patients with myelodysplastic syndrome and myelodysplastic syndrome/myeloproliferative neoplasms whose tumors harbored FLT3 or CBL mutations. After a median of 13.2 months, only one patient remained on quizartinib and azacitidine.
 
Karuna announced that in the five-week, 252 patient, Phase III, EMERGENT-2 trial, treatment with xanomeline and trospium decreased the total PANSS score by 9.6 points compared to placebo in patients with schizophrenia who were experiencing acute psychosis.
 
AstraZeneca announced that in the 193 patient, Phase II, SAVANNAH trial, treatment with osimertinib plus savolitinib resulted in a 32% ORR in patients with mutated EGFR NSCLC and MET over expression. The ORR was 49% in 108 NSCLC patients with high levels of MET.
 
Hutchmed announced that in the 691 patient, Phase III, FRESCO-2 trial, treatment with fruquintinib improved OS compared to placebo in patients with advanced, refractory metastatic colorectal cancer.
 
CStone announced interim data from the Phase III, GEMSTONE-301 trial, where patients treated with sugemalimab achieved progression-free survival of 10.5 months compared to 6.2 months with placebo in patients with locally advanced/unresectable Stage III squamous non-small cell lung cancer (NSCLC) without disease progression after concurrent or sequential chemoradiotherapy.
 
BeiGene announced that in the 674 patient, Phase III, RATIONALE 301 trial, overall survival with tislelizumab was non-inferior to sorafenib in patients with unresectable hepatocellular carcinoma.
 
Verona announced that in the 12-week, 789 patient, Phase III, ENHANCE-2 trial, treatment with ensifentrineincreased FEV1 by 94 mL compared to placebo in patients with chronic obstructive pulmonary disease.
 
Daiichi Sankyo and AstraZeneca announced interim data from 75 patients with previously untreated or pretreated advanced or metastatic non-small cell lung cancer (NSCLC) without actionable genomic alterations enrolled in the open-label, Phase Ib, TROPION-Lung02 trial. The ORR for 38 patients treated with datopotamab deruxtecan in combination with pembrolizumab was 37% and when platinum chemotherapy was added to the two drugs the ORR was 41% in 37 patients.
 
Kazia announced interim results from nine patients in the 12-patient, initial exploratory stage of a Phase I trial, where adding paxalisib to radiotherapy resulted in a 100% overall response rate (ORR) compared to a historical ORR for radiotherapy alone of 20% to 45% in patients with brain metastases.
 
Cornerstone announced that in a 20 patient, Phase Ib trial, treatment with devimistat plus gemcitabine and cisplatin resulted in an overall response rate of 45% in patients with locally advanced unresectable or metastatic biliary tract cancer.
 
Published Research Updates
 
In a revised report, ICER found evidence to support efficacy for AMX0035 in the treatment of amyotrophic lateral sclerosis (ALS) to be comparable or better compared to standard of care alone. If AMX0035 is priced similar to intravenous edaravone at $171,000 a year, the drug would exceed cost effectiveness thresholds. ICER calculated an annual health-benefit price benchmark (HBPB) range for AMX0035 of $9,100 – $30,600.
 
In a 12-week, 52 patient, Phase II trial, treatment with cilofexor resulted in a 21% decrease in serum alkaline phosphatase (ALP), 30% decrease in gamma-glutamyl transferase (GGT), 49% decrease in alanine aminotransferase (ALT), and 42% decrease in aspartate aminotransferase (AST) compared to placebo in patients with primary sclerosing cholangitis. In a 96-week, 47 patient, open-label extension of the Phase II trial, liver biochemistry and biomarkers increased during a four-week period with no drug.  The values then decreased when back on cilofexor for the 96-week extension by 8.3% for ALP, 29.8% for GGT, 29.8% for ALT, and 16.7% for AST.
 
In a 12-week, 57 patient, Phase II trial, treatment with durvalumab and tremelimumab, followed by durvalumab alone resulted in progression-free survival of 49% in patient with advanced or metastatic sarcoma.
 
In the six-month, 400 patient, Phase III, ST PETER HIV trial, adding varenicline or cytisine to nicotine replacement therapy (NRT) did not decrease risky drinking and smoking compared to NRT alone in HIV patients who engage in risky drinking and smoking.