MAYO CLINIC

CG Oncology to host conference call and webcast on December 5, 2024, at 7 am CST to discuss Phase 3 BOND-003 trial results of cretostimogene monotherapy in high-risk BCG-unresponsive NMIBC. Dr. Mark Tyson, II will present the results at the SUO 25th Annual Meeting in Dallas at 11:45 am CST. The webcast will be accessible via the company's Investor Relations website.

A new tool resembling a wiry flower at the end of a long tube is at the center of a multibillion-dollar race among Medtronic, Boston Scientific, and Johnson & Johnson MedTech to dominate the market for minimally invasive devices treating atrial fibrillation using pulsed field ablation (PFA). PFA, which uses electric pulses instead of heat or cooling, is expected to significantly reduce patient time in the cath lab and lower complication risks. Medtech executives view PFA as a transformative technology driving future growth, with Clarivate projecting PFA devices to exceed $1.3 billion in global sales this year.

Ascentage Pharma's olverembatinib, a third-generation BCR-ABL inhibitor, has its new indication for adult patients with chronic-phase chronic myeloid leukemia (CML-CP) resistant and/or intolerant of first-and second-generation tyrosine kinase inhibitors (TKIs) included in the China 2024 National Reimbursement Drug List (NRDL), effective January 1, 2025.

Shaji Kumar discusses 2024-2025 NCCN updates for multiple myeloma, highlighting quadruplet regimens as new standard care, tailored maintenance therapy, and major revisions for relapsed/refractory disease with immunotherapy integration, including CAR T-cell therapies and bispecific antibodies. Diagnostic approaches for smoldering myeloma were also updated.

Widetrial supports an expanded access program (EAP) funded by a $22 million federal grant to Mayo Clinic, providing MN-166 (ibudilast) to ALS patients not eligible for clinical trials. The program aims to enroll 200 patients across Mayo Clinic sites and other U.S. institutions, focusing on measuring treatment effects on disease progression and neurofilament light chain (NfL) levels over six months. Patients can monitor their own NfL levels and contribute to an ALS biobank. MediciNova is also sponsoring a Phase 2b/3 trial, COMBAT-ALS, testing MN-166 against a placebo.

U.S. and Europe differ in labeling cognitively unimpaired people with AD biomarkers: U.S. uses 'preclinical AD' and Europe 'asymptomatic at-risk'. Both agree on not testing asymptomatic people. The debate centers on whether biomarker positivity should define AD, with concerns over distress and resource use. Future preventative treatments may render the distinction moot.

The FDA approved AOP Health’s Rapiblyk (landiolol) for rapid management of atrial fibrillation and atrial flutter in critical care. Based on five studies, Rapiblyk reduced heart rate by 40% to 90% in patients with supraventricular tachycardia, compared to 0% to 11% with placebo. Adverse events occurred in 9.9% of Rapiblyk patients versus 1% with placebo, with hypertension being the most common AE. Contraindications include severe sinus bradycardia, sick sinus syndrome, and heart block greater than first degree.

Vora et al reported in *The Lancet Oncology* that short-course hypofractionated proton-beam therapy guided by F-18–DOPA PET and contrast-enhanced MRI produced good outcomes in patients aged ≥ 65 with newly diagnosed glioblastoma, with 56% alive at 12 months and median overall survival of 13.1 months, compared to historical controls.

Alnylam Pharmaceuticals' vutrisiran sNDA for ATTR-CM accepted by FDA, with an action date of March 23, 2025. Vutrisiran, a generic of AMVUTTRA, treats ATTR by reducing transthyretin. HELIOS-B study showed vutrisiran improved cardiovascular outcomes, survival, and quality of life. Adverse reactions include pain, arthralgia, dyspnea, and vitamin A decrease, requiring supplementation. BridgeBio Pharma's Attruby also approved for ATTR-CM on Nov. 22, 2024.

FDA approves imatinib (Imkeldi), the first oral, liquid tyrosine kinase inhibitor for treating cancers like GIST, MDS, MPD, CML, and Ph+ ALL. Oral imatinib offers precise dosing and convenience, benefiting patients with swallowing difficulties or needing tailored dosing. It can be used from age 1, addressing significant needs in CML, MDS/MPD, GIST, and ALL patients. Imatinib targets the BCR-ABL anomaly, showing efficacy in older patients, especially with chemotherapy. Safety in pediatric Ph+ ALL is yet to be established, and monitoring is crucial due to potential risks like edema and hematologic toxicity.