Update for May 24, 2022

Regulatory Update
 
The FDA accepted the NDA for sparsentan for the treatment of IgA nephropathy and set a PDUFA date for 11/7/2022
 
The FDA placed a hold on HIV trials using injectable lenacapavir, in December 2021, due to concerns regarding the compatibility of vials made of borosilicate glass with lenacapavir solution, which could potentially lead to the formation of sub-visible glass particles. The FDA allowed trials for injectable lenacapavir to resume in May 2022, after Gilead changed to an aluminosilicate glass vial that did not have compatibility problems with the drug.
 
KalVista announced that generic name for KVD900 is sebetralstat
 
Announced Research Updates
 
BeiGene announced interim results from 32 patient enrolled in the 63 patient, open-label, Phase II, TRUCE-02 trial, where treatment with tislelizumab plus nab-paclitaxel resulted in a complete response rate of 62.5% in Chinese patients with high-risk non–muscle invasive bladder cancer (HR-NMIBC).
 
Enanta announced that in a 14-day, 81 patient, Phase IIb trial, treatment with EDP-938 did not reduce the total symptom score compared to placebo in patients with mild, community-acquired RSV.
 
Tricida announced it had stoped the 1,600 patient, Phase III, VALOR-CKD trial after 1,480 patients were enrolled and 237 had reached the primary endpoint of renal death, end-stage renal disease (ESRD), or a 40% or greater reduction in the estimated glomerular filtration rate (eGFR). No interim results for veverimer were announced for VALOR-CKD, but Tricida anticipates that final results will be available in 3Q22. VALOR-CKD is being stopped early because Tricida does not have enough funding to complete the trial as originally designed with completion forecast for 2024. 
 
Chinook Therapeutics announced interim results from a cohort of 20 patients with IgA nephropathy (IgAN) enrolled in the 12-week, 80 patient, Phase II AFFINITY trial, where treatment with atrasentan lowered the 24-hour urine protein creatinine ratio (UPCR) in 20 patients at six weeks by 38%. At 12 weeks, UPCR was reduced 49.9% in 18 patients and at 24 weeks, UPCR was lowered by 58.5% in 11 patients.
 
Vera Therapeutics announced that in the 72-week, 16 patient, Phase IIa, JANUS trial, treatment with atacicept decreased serum anti-Gd-IgA1 levels by 28% with 25 mg and 39% with 75 mg in patients with IgA nephropathy (IgAN).
 
Biohaven Pharmaceutical announced that in a 48-week, 213 patient, Phase III trial, treatment with troriluzole was no different that placebo in improving the modified functional Scale for the Assessment and Rating of Ataxia in patients with spinocerebellar ataxia. 
 
Idorsia announced that the Phase III, PRECISION trial, adding aprocitentan 12.5 mg and 25 mg to a fixed dose combination of amlodipine, valsartan and hydrochlorothiazide lowered blood pressure compared to adding placebo to the same combination in the 730 patient, 4-week, Part 1 of the study of patients with resistant hypertension. In the open-label, 32-week, Part 2 of PRECISION, blood pressure reduction was maintained with aprocitentan, amlodipine, valsartan and hydrochlorothiazide.  All patients received the same combination of drugs and patients who switched from placebo achieved the same blood pressure reduction. In the 12-week, 614 patient, Part 3 of the trial aprocitentan was withdrawn for four weeks then patients were re-randomized to aprocitentan or placebo for 12 weeks. Patients who received aprocitentan had a reduction in blood pressure, while those on placebo had an increase.
 
Published Research Updates
 
In the 135 patient, Phase II, CITYSCAPE trial, treatment with tiragolumab plus atezolizumab improved the objective response rate (ORR) (31.3% vs 16.2%) and progression-free survival (PFS) (5.4 vs 3.6 months) compared to atezolizumab monotherapy in patients with PD-L1-positive metastatic non-small cell lung cancer. 
 
In the 12-month 477 patient, open-label, Phase III, EMERALD trial, treatment with elacestrant resulted in progression-free survival (PFS) at 12 months of 22.3% compared to 9.4% with investigator’s choice of fulvestrant, anastrozole, letrozole, or exemestane in patients with ER+/HER2- advanced breast cancer. In patients with an ESR1 mutation 12-month PFS was 26.8% with elacestrant compared to 8.2% with physician’s choice.
 
In the 24-week, 171 patient, Phase IIb, ALPINE II trial, patients treated with aldafermin did not demonstrate an improvement in fibrosis by at least one stage with no worsening of steatohepatitis compared to placebo in patients with biopsy-confirmed NASH with F2-F3 liver fibrosis.