Argenx SE’s surprise phase III blowup with subcutaneous Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in the platelet disorder primary immune thrombocytopenia (ITP) – blamed on a higher-than-expected placebo response – had investors speculating about possible read-throughs to other indications.
Xenon Pharmaceuticals Inc.’s Kv7 potassium channel opener XEN-1101 remains very much alive despite the primary endpoint miss in top-line data from the phase II proof-of-concept X-Nova trial in moderate to severe major depressive disorder.
Index Pharmaceuticals Holding AB CEO Jenny Sundqvist said liquidation of the company in the wake of phase III data with cobitolimod in moderate to severe ulcerative colitis (UC) is “one of the options that will be on the table.” Shares of the Stockholm-based firm (STO:INDEX) closed Nov. 22 at SEK0.24 (US2 cents), down SEK0.41, or 63%, on word that an independent data monitoring committee (DMC) has completed the planned dose-selection analysis, including safety review and assessment for futility, of induction Study 1 of the phase III program called Conclude, testing the Toll-like receptor 9 agonist cobitolimod. The DMC concluded that Index’s lead compound is unlikely to meet the primary endpoint, and the company said development will be stopped.
Merck & Co. Inc. continued to broaden its reach in neurodegenerative diseases by paying, through a subsidiary, as much as $610 million to take over preclinical-stage Caraway Therapeutics Inc. The deal involves an undisclosed up-front payment along with contingent milestone rewards.
Low expectations on Wall Street for the Pulmonary-Allergy Drugs Advisory Committee (PADAC) sit-down on Merck & Co. Inc.’s P2X3 receptor antagonist gefapixant for chronic cough (CC) translated to negative balloting by panelists. PADAC members were polled on a single question: Does the evidence demonstrate that [Merck’s] gefapixant provides a clinically meaningful benefit to adults with refractory or unexplained CC? The results came out 12 no, 1 yes.
With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
Reneo Pharmaceuticals Inc.’s last patient visit Oct. 9 in the pivotal study with mavodelpar called Stride – due to yield top-line data in December 2023 – kept interest strong in primary mitochondrial myopathy (PMM), where parties are pursuing development bids by way of assorted strategies.
Eyebiotech Ltd. doubled the size of its series A round, raising $130 million to advance a pair of assets, with a phase Ib/IIa trial dubbed Amarone underway testing the drug called Restoret in patients with diabetic macular edema and neovascular, or wet, age-related macular degeneration.
The blowup in phase II with Aclaris Pharmaceuticals Inc.’s oral MK2 inhibitor zunsemetinib (ATI-450) for moderate to severe rheumatoid arthritis (RA) sent shares (NASDAQ:ACRS) into a tailspin and sparked Wall Street speculation about the firm’s other mid-stage prospect. Wayne, Pa.-based Aclaris’ stock ended Nov. 13 at 64 cents, down $4.11, or 86%, after investors learned that ATI-450 missed the study’s primary and secondary endpoints. Development of the candidate, which was also under phase IIa investigation in psoriatic arthritis, will be stopped.
With Valneva SE’s accelerated approval from the U.S. FDA for chikungunya vaccine Ixchiq, attention turned to the February 2024 meeting of the Advisory Committee on Immunization Practices (ACIP), which will vote on the product. A single-dose, live-attenuated vaccine, Ixchiq is designed to prevent disease caused by the virus in people 18 and older who are at increased risk of exposure to the bug.
