Update for May 17, 2022

Regulatory Update
 
The FDA approved tirzepatide (Mounjaro, Lilly), on 5/13/2022, as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Tirzepatide was approved with a black box warning regarding an increased risk for thyroid c-cell tumors and is contraindicated in patients with medullary thyroid carcinoma Multiple Endocrine Neoplasia syndrome type 2.
 
The FDA rejected bimekizumab for the treatment of moderate to severe plaque psoriasis due to inspection observations that need to be corrected.
 
Biogen and Eisai completed a rolling submission for lecanemab for the treatment of early Alzheimer's disease.
 
The FDA delayed the PDUFA dates for Amicus’ combination of cipaglucosidase alfa and miglustat by three months to allow more time to review new information. The new dates are 8/29/2022 for the miglustat NDA and 10/29/2022 for the cipaglucosidase alfa BLA.
 
Green Val­ley discontinued a Phase III Alzheimer’s trial in May 2022 evaluating oligomannate due to insufficient funding to support the trial. The trial had enrolled 257 Alzheimer’s patients in North Amer­i­ca, 118 in Eu­rope and 64 in Chi­na.
 
KemPharm bought Orphazyme in May 2022 and took over development of arimoclomol. KemPharm plans to refile an NDA for arimoclomol in 1Q23.
 
Announced Research Updates
 
Roche announced interim results from the 534 patient, Phase III, SKYSCRAPER-01 trial, where adding tiragolumab to atezolizumab did not improve PFS compared to atezolizumab alone in patients with PD-L1-high locally advanced or metastatic NSCLC.
 
J&J announced that in the 295 patient, Phase Ib, TRIMM-2 trial, treatment with teclistamab plus daratumumab improved the overall response rate (ORR) compared to daratumumab alone in heavily pretreated patients with multiple myeloma.
 
Gamida announced that in a long-term extension of a 125 patient, Phase III trial, patients who received an umbilical cord blood transplant (UCBT) and were treated with omidubicel did not achieve a statistically significant improvement in non-relapse mortality or survival one year after transplant compared to UCBT alone.
 
BMS announced that in the 1,221 patient, open-label, Phase III, POETYK PSO-LTE trial, a two-year extension of the Phase III, POETYK PSO-1 and POETYK PSO-2 trial, efficacy at 60-weeks with deucravacitinib was maintained, with 77.7% of patients maintaining PASI-75 and 58.7% maintaining a Physicians Global Assessment score of 0 or 1 (clear/almost clear skin).
 
Published Research Updates
 
In the 36 patient cohort 1 of the 15-day, 72 patient, Phase II/III, CAPELLA trial, 88% (21/24) of patients that had oral lenacapavir added to a failing HIV-treatment regimen achieved at least a 0.5 log10 copies/mL reduction in viral load compared to 17% (2/12) that received the failing regimen only in patients with multidrug resistant HIV-1 infection.
 
In the 178 patient, open-label, Phase III, ATTAIN trial, treatment with etirinotecan pegol did not improve overall survival compared to physician’s choice of eribulin, ixabepilone, vinorelbine, gemcitabine, paclitaxel, docetaxel, or nab-paclitaxel in patients with breast cancer and brain metastases.
 
In a 12-week, 147 patient, Phase II trial, treatment with BI 1015550 resulted in a 5.7 ml increase in Forced Vital Capacity (FVC) compared to a 81.7 ml decrease with placebo in patients with idiopathic pulmonary fibrosis (IPF) who were not receiving an antifibrotic drug. Among IPF patients receiving an antifibrotic drug, there was a 2.7 ml increase in Forced Vital Capacity (FVC) compared to a 59.2 ml decrease with placebo.
 
In a 52-week, 24 patient, Phase III trial, treatment with Oxalobacter formigenes did not decrease plasma oxalate levels compared to placebo in patients with PH.
 
In a 24-week, 463 patient, Phase III SEED trial, treatment with dorzagliatin reduced HbA1c 1.07% compared to a 0.50% decrease with placebo, in Chinese patients with type 2 diabetes. In a 24-week, 767 patient, Phase III trial, adding dorzagliatin to metformin lowered HbA1c 1.02% compared to a 0.36% decrease with metformin monotherapy in Chinese patients with type 2 diabetes.
 
In the 4-week, 115 patient, Phase III, URIROX-1 trial, treatment with reloxaliase reduced the 24-hour urinary oxalate excretion by 22.6% compared to a 9.7% decrease with placebo in patients with enteric hyperoxaluria.