Update for February 16, 2021

Regulatory Update
 
The FDA approved trilaciclib (Cosela, G1 Therapeutics), on 2/12/2021, to reduce the frequency of chemotherapy-induced bone marrow suppression in extensive-stage small cell lung cancer patients being treated with a platinum/etoposide containing regimen or a topotecan containing regimen.
 
The FDA granted Breakthrough Therapy Designation to asciminib for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia.
 
Announced Research Updates
 
Galapagos and Gilead announced that an analysis of interim data from patients enrolled in the 52-week, 1,500 patient, Phase III, ISABELA 1 & 2, IPF trials, by the Independent Data Monitoring Committee, resulted in a recommendation to stop the trials, because the ziritaxestat benefit-risk profile did not support continuation. 
 
Sumitomo Dainippon announced that in the 1,250 patient, open-label, Phase III, CanStem303C trial, adding napabucasin to FOLFIRI with or without bevacizumab did not improve overall survival (OS) compared to the combination without napabucasin in patients with metastatic colorectal cancer. There also was not an improvement in OS in a subset of patients with phosphorylated signal transducer and activator of transcription 3 (pSTAT3) biomarker mutations.
 
Mesoblast announced that in a 24-month, 404 patient, Phase III trial, the reduction in pain using a Visual Analog Score (VAS) at 12-months and 24-months was greater with  rexlemestrocel-L alone and rexlemestrocel-L plus hyaluronic acid than placebo in patients with chronic low back (LBP) pain due to degenerative disc disease refractory to conventional treatments. The decrease was about half with rexlemestrocel-L alone compared to the combination. In a subgroup of patients receiving opioids for LBP, rexlemestrocel-L plus hyaluronic acid reduced opioid use, while patients in the placebo group increased opioid use.
 
Takeda announced that in the eight-week, 352 patient, Phase III, SOLSTICE trial, 55.7% of patients treated with maribavir achieved confirmed CMV viremia clearance compared to 23.9% with investigator-assigned treatment (ganciclovir, valganciclovir, foscarnet, or cidofovir) in transplant patients with refractory/resistant CMV infection.
 
Amicus announced that in the 52-week, 123 patient Phase III, PROPEL trial, patients treated with cipaglucosidase alfa/miglustat had a non-significant improvement in their six-minute walk-test distance (6MWD) by 21 meters compared to a 7 meter improvement with alglucosidase alfa (p=0.072) in patients with late-onset Pompe disease. Patients treated with cipaglucosidase alfa/miglustat had a significant slowed rate of FVC decline compared to alglucosidase alfa (0.9% decline vs 4% decline).
 
Merck announced interim results from 41 patients enrolled in a 118 patient, Phase II, open-label trial, treatment with belzutifan plus cabozantinib resulted in a 22% ORR in patients with RCC.
 
Published Research Updates
 
In a 52-week, 217 patient, Phase III trial, oral daprodustat was non-inferior to darbepoetin in maintaining hemoglobin levels (12 g/dl vs 11.9 g/dl) during weeks 40 to 52 in non-dialysis dependent Japanese patients with anemia associated with chronic kidney disease. 
 
In a 128 patient, Phase II trial, treatment with spartalizumab resulted in a 7.4% overall response rate in patients with neuroendocrine tumors and 4.8% in patients with gastroenteropancreatic neuroendocrine carcinomas.
 
In the 267 patient, open-label, Phase III, PHOEBE trial, treatment with pyrotinib plus capecitabine resulted in progression-free survival of 12.5 months compared to 6.8 months with pyrotinib plus lapatinib in Chinese patients with HER2-positive relapsed or metastatic breast cancer previously treated with taxanes and trastuzumab.
 
In a 52-week, 2,781 patient, Phase III trial, treatment with roxadustat improved hemoglobin 1.75 g/dl compared to a 0.4 g/dl increase with placebo in non-dialysis-dependent CKD patients with anemia.
 
In a 432 patient, Phase III trial, adding andecaliximab to mFOLFOX6 did not improve overall survival compared to mFOLFOX6 alone (12.5 vs 11.8 months) in patients with untreated human epidermal growth factor receptor 2-negative gastric or gastroesophageal junction adenocarcinoma.
 
In a 608 patient Phase III, open label trial, where treatment with enfortumab vedotin resulted in overall survival of 12.88 months compared to 8.97 months with investigator-chosen chemotherapy (standard docetaxel, paclitaxel, or vinflunine), in patients with locally advanced or metastatic urothelial cancer who progressed after receiving platinum-containing chemotherapy and a PD-1 or PD-L1 inhibitor. 
 
In a 24-week, 213 patient, Phase III, TIMES 1 trial, imeglimin lowered HbA1c 0.87% more than placebo in Japanese patients with type 2 diabetes.
 
In a 147 patient, open-label, Phase II trial, treatment with sunitinib, crizotinib, or savolitinib did not improve progression-free survival compared to cabozantinib in patients with metastatic papillary renal cell carcinoma.
 
In a 200 patient, Phase II trial, 66% of patients treated with Lu-PSMA-617 achieved a 50% or greater reduction in their PSA response rate (PSA50-RR) compared to 37% treated with cabazitaxel in patients with metastatic castration-resistant prostate cancer (mCRPC), eligible for cabazitaxel treatment.