With the mid-way point of the first quarter of 2021 upon us, we look at our refreshed list of key clinical trials still expected to read out data by the end of the quarter. We focus this week on Phase 3 readouts, while looking at Phase 1 and 2 trial readouts next week.
First, let’s review the week that was with notable price-moving clinical trial updates.
Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) shares closed Thursday up 13% to $4.47 following the presentation of data from its Phase 2 MARIO-trial evaluating eganelisib in combination with nivolumab (Opdivo) in platinum-refractory, I/O naïve patients with advanced urothelial cancer. In the overall population, the combination arm demonstrated an overall response rate (ORR) of 30% compared with 25% in the nivolumab monotherapy arm. When broken down into population groups, patients in the PD-L1 negative patient population saw the greatest benefit with an ORR of 26% for the combo compared with 14% for nivolumab monotherapy.
Amicus Therapeutics (Nasdaq: FOLD) shares closed Friday down 33% to $12.57 following the release of top-line data from its Phase 3 Propel trial of AT-GAA for the treatment of late-onset Pompe disease (LOPD). The primary endpoint of the change in 6-minute walk distance as compared with baseline measurements, was not met. While numerically greater, with patients walking on average 21 meters farther compared to 7 meters with those treated with current standard of care alglucosidase alfa, statistical significance for superiority was not achieved (p=0.072). The company still intends to complete its Biologics License Application (BLA) for approval with the FDA, which it commenced late-2020. Completion of the BLA submission is expected next quarter.
Galapagos NV (NASDAQ:GLPG) shares closed the week down 19% to $70.68 following news their Phase 3 ISABELA trial with Gilead Sciences (NASDAQ:GILD) of ziritaxestat in patients with idiopathic pulmonary fibrosis has been discontinued due to insufficient efficacy following a review by the Independent Data Monitoring Committee (IDMC).
KalVista Pharmaceuticals, Inc. (NASDAQ: KALV) shares surged to close the week up 180% to $42.57 following its announcement of positive top-line data from its Phase 2 trial of KVD900 as an oral on-demand treatment for hereditary angioedema (HAE) attacks. Attacks treated with KVD900 significantly reduced use of rescue treatment (p=0.001), with 15% of KVD900 treated attacks rescued compared to 30% on placebo at 12 hours. This efficacy benefit of KVD900 was maintained at 24 hours (p=0.0005).
Erytech Pharma S.A. (NASDAQ: ERYP) shares closed the week down 22% to 9.05 following news that its Phase 3 TRYbeCA-1 trial in patients with second-line pancreatic cancer, will continue through to final analysis following interim analysis by an Independent Data Monitoring Committee (IDMC). There was potential for the trial to be stopped early if there were sufficient efficacy, which investors had been looking for.
-
Phase 3 data readouts to watch for the remainder of 1Q 2021: