Pipeline News and Updates
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We recently added six new drugs to the knowledgebase:
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. Regulatory Update
The FDA accepted the NDA for tofersen to treat superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS) and set a PDUFA date for 1/25/2023. The FDA accepted the NDA for sotagliflozin for the treatment of heart failure and set a PDUFA date for 6/27/2023. The FDA accepted the BLA for the combination of N-803 plus Bacillus Calmette-Guerin (BCG) for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma and set a PDUFA date for 5/23/2023. The FDA accepted the BLA for omidubicel for the treatment hematologic malignancies progressing to allogenic hematopoietic stem cell transplant (HSCT) and set a PDUFA date for 1/30/2023. Cidara submitted an NDA for rezafungin for the treatment of candidemia and invasive candidiasis. Sarepta plans to submit a BLA for delandistrogene moxeparvovec for the treatment of ambulant Duchenne muscular dystrophy in the second half of 2022. MHRA granted marketing authorization for voxelotor for the treatment of hemolytic anemia due to sickle cell disease. ObsEva discontinued development of linzagolix and returned development and marketing rights back to Kissei after the FDA indicated that additional data was required to address NDA deficiencies. CHMP granted an accelerated assessment for leniolisib for the treatment of PI3K delta syndrome. Announced Research Updates J&J announced that in the 20 patient, open-label, Phase 1b/2 CHRYSALIS-2 trial, treatment with lazertinib, amivantamab, carboplatin and pemetrexed resulted in an overall response rate (ORR) of 50% in patients with relapsed/refractory non-small cell lung cancer (NSCLC) and EGFR mutations. AstraZeneca has discontinued development of adavosertib as a treatment for ovarian cancer, solid tumors and uterine serous cancer. Novo announced that in the 26-week, 588 patent, Phase IIIa, ONWARDS 3 trial, once-weekly insulin icodec lowered HbA1c 1.57% compared to a 1.36% decrease with once-daily insulin degludec in patients with type 2 diabetes who were insulin-naive. Novo announced that in the 26-week, 588 patent, Phase IIIa, ONWARDS 4 trial, once-weekly insulin icodec lowered HbA1c 1.16% compared to a 1.18% decrease with once-daily insulin glargine in patients with type 2 diabetes who were also receiving mealtime insulin aspart two to four times daily. Immutep announced that in the 36 patient, Part B of the Phase II, open-label TACTI-002 trial, treatment with eftilagimod alpha plus pembrolizumab resulted in overall survival of 9.7 months in patients with second line checkpoint inhibitor refractory metastatic NSCLC. Concert announced that in a 24-week, 517 patients, Phase III, THRIVE-AA2 trial, treatment with CTP-543 resulted in achievement of a SALT score of 20 or less in 38.3% of patients treated with 12 mg and 33% who received 8 mg compared to 0.8% with placebo in patients with moderate to severe alopecia areata. The results of two failed CNTX-4975 trials have not been announced, presented or published and are only available on clinicaltrials.gov.
Paxalisib was being evaluated as a treatment for glioblastoma as part of the 550 patient, Phase II/III GBM AGILE trial, which is testing multiple treatments for glioblastoma. Paxalisib was dropped from the GBM AGILE trial, when it did not meet efficacy criteria in the Phase II portion of the trial. Isofol announced that in a 490 patient, Phase III, AGENT trial, arfolitixorin did not improve the ORR compared to leucovorin in patients with metastatic colorectal cancer, who were being treated with 5-FU, oxaliplatin, and bevacizumab. Isofol will announce future plans for arfolitixorin in 2023, after a full analysis of the AGENT trial is completed. Published Research Updates In a 48-week, 40 patient, open-label trial, treatment with tofogliflozin and glimepiride each improved liver histological and metabolic parameters in patients with type 2 diabetes and nonalcoholic fatty liver disease. In the 52-week, 407 patient, Phase III, ASCEND-TD trial, switching patients from epoetin to daprodustat was non-inferior to continuing epoetin (10.45 vs 10.51 g/dl) in CKD patients who were undergoing dialysis. In the 60 patient, open-label Phase II, CONCERTO trial, treatment with olaparib plus cediranib resulted in an objective response rate of 15.3% in PARP inhibitor-naive patients with non-germline-BRCA-mutated platinum-resistant recurrent ovarian cancer. In the 52-week, 316 patient, Phase II, PASADENA trial, treatment with prasinezumab did not improve the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score compared to placebo in patients with early Parkinson's Disease (PD). |
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