Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
As Novartis AG’s approved prostate cancer therapy, Pluvicto (177Lu-PSMA-617), continues on a growth trajectory, the firm signed a licensing deal with Arvinas Inc. potentially valued at north of $1 billion for global development and commercialization of ARV-766, the latter’s second-generation proteolysis targeting chimera (PROTAC) androgen receptor degrader targeting the same disease.
Stable disease in about half the patients tested wasn’t enough for Wall Street, and shares of Vincerx Pharma Inc. (NASDAQ:VINC) nosedived by $3.72, or 78%, to close April 9 at $1.06 on the disclosure of preliminary phase I data with small-molecule drug conjugate VIP-236 in metastatic solid tumors.
Mixed opinions from the U.S. FDA’s Oncology Drugs Advisory Committee last month didn’t stop the agency from green-lighting an expanded label for Abecma (idecabtagene vicleucel) to include adults with relapsed or refractory multiple myeloma (r/r MM) after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody.
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.
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.
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.
An increasingly popular target across varied cancer types is the immune system regulator V-domain Ig suppressor of T-cell activation (VISTA), where a number of developers have taken early stage aim – among them Sensei Biotherapeutics Inc., with SNS-101, which Wainwright analyst Edward White believes could be the first anti-VISTA monoclonal antibody approved as a therapeutic agent. But there’s plenty of work ahead.
The fast-shifting obesity space gained more clinical results as Viking Therapeutics Inc. shared data from its phase I, multiple ascending-dose trial with oral VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
