The higher of two doses of Ionis Pharmaceuticals Inc.’s drug olezarsen for treating adults with familial chylomicronemia syndrome reduced fasting triglyceride levels by 44% from baseline at the six-month mark in a phase III study, but one case of pancreatitis in the trial paints a less attractive picture than top-line data the firm reported in September.
After reviewing data from its phase II trial of farnesoid X receptor agonist ASC-42 in primary biliary cholangitis, Ascletis Pharma Inc. said it was quitting development of the molecule in all indications.
Positive updated phase II data with CAN-2409 in pancreatic cancer led shares of Candel Therapeutics Inc. (NASDAQ:CADL) to close April 4 at $6.40, up $4.72, or 281%, well above the firm’s previous 52-week high. At one point during the day, the stock had climbed to $7.65.
Top-line data from the phase II Kickstart study of Effector Therapeutics Inc.’s tomivosertib as a frontline treatment of non-small-cell lung cancer failed to produce data strong enough to continue development in the indication. The company has decided to halt the study, move ahead with a separate, investigator-sponsored study of tomivosertib in acute myeloid leukemia and focus on another drug in its pipeline for treating breast cancer. Effector’s stock (NASDAQ:EFTR) crumpled on the NCSLC news as shares plunged 82% on April 4 to close at $2.96 each. The closing value was the lowest the company has seen in the past 12 months.
Less than two weeks after going public by way of the merger with Graphite Bio Inc., Lenz Therapeutics Inc. unveiled positive top-line data from its pair of phase III Clarity studies testing two formulations of the muscarinic acetylcholine receptor agonist aceclidine, LNZ-100 and LNZ-101, for presbyopia.
The U.S. FDA accepted for review Daiichi Sankyo Co. Ltd.’s and Astrazeneca plc’s BLA for datopotamab deruxtecan to treat adults with unresectable or metastatic hormone receptor-positive, HER2-negative breast cancer who have received prior systemic therapy for unresectable or metastatic disease.
Trouble continues to dog Verve Therapeutics Inc.’s base editor of the PCSK9 gene, VERVE-101, so the company paused enrollment in a phase Ib study in cholesterol lowering to focus on the similarly designed VERVE-102.
Phase II/III results from Gritstone Bio Inc. with Granite, a personalized neoantigen cancer vaccine for colorectal cancer, turned up the opposite of what some investors expected, and the company’s shares (NASDAQ:GRTS) ended April 2 at $1.20, down $1.15, or 49%.
Disc Medicine Inc. CEO John Quisel said that top-line phase II findings from the study called Aurora with bitopertin in erythropoietic protoporphyria are “hard for us to interpret. This package of data is something that we’re going to have to sort through,” and the Watertown, Mass.-based firm expects to talk with the U.S. FDA about next steps in the second half of this year.
The possibility for expanding Bristol Myers Squibb Co.’s S1P modulator, Zeposia (ozanimod), into Crohn’s disease took a hit on disappointing data from the initial analysis of its Yellowstone study, the first of two phase III trials. Results showed the study failed to meet the primary endpoint, defined as clinical remission as measured by the Crohn’s Disease Activity Index at week 12.