The RNAi therapeutic zilebesiran, which was the focus of a $2.8 billion deal last year between Alnylam Pharmaceuticals Inc. and Roche Holding AG, demonstrated significant systolic blood pressure reductions in patients with mild to moderate hypertension who were part of the Kardia-2 phase II study. A single dose of zilebesiran, a subcutaneous therapy that targets liver angiotensinogen, met the primary endpoint showing clinically and statistically significant placebo-adjusted reductions in 24-hour mean systolic blood pressure at month three as measured by ambulatory blood pressure monitoring in each of three cohorts, and it demonstrated encouraging safety and tolerability.
Lumicell touts benefits of Lum System in breast cancer
The U.S. FDA’s Imaging Drugs Advisory Committee will vote later today on what could be the nation’s first approved intraoperative technology for use in breast cancer that directly examines the lumpectomy cavity for residual cancer. The purpose of Lumicell Inc.’s Lum System, which combines imaging drug Lumisight (pegulicianine) with the Newton, Mass., company’s Direct Visualization System, is to eliminate the need for a second surgery to address positive margins in some patients. In avoiding a second surgery, patients could begin chemotherapy earlier, and more eligible patients might opt for a lumpectomy rather than a more invasive mastectomy, Kelly Hunt, chair of the Department of Breast Surgical Oncology at the MD Anderson Cancer Center and president of the Society of Surgical Oncologists, told the committee members.
Phase I atopic dermatitis data boost Apogee stock
Apogee Therapeutics Inc. shares (NASDAQ:APGE) were trading at $58.41, up $16.67, or almost 40%, after the company disclosed positive interim phase I data with APG-777 in healthy volunteers. The IL-13-targeting antibody given subcutaneously for atopic dermatitis (AD) and other inflammatory diseases beat its study objectives ahead of schedule with a half-life of about 75 days. The outcome could mean maintenance dosing every three or every six months – two to four shots per year, as compared to the current AD paradigm of as many as 26.
FDA clears Formosa’s eye drop for postsurgical pain, inflammation
Taiwan’s Formosa Pharmaceuticals Inc. and Aimmax Therapeutics, Inc. said the U.S. FDA approved APP-13007 (clobetasol propionate ophthalmic suspension 0.05%), a twice-daily corticosteroid eye drop for postoperative eye pain and inflammation. The approval makes APP-13007 the first novel postoperative steroid approved in more than 15 years in the U.S. ophthalmic market, coming on the PDUFA date of March 4, 2024. Developed with Formosa’s APNT nanoparticle formulation platform, APP-13007 is now indicated to treat postoperative inflammation and pain after ocular surgery in the U.S., providing “critical foundation and momentum” for entry into the global marketplace, according to Formosa President and CEO Erick Co.
Telix to buy Artms for $82M to shore up radioisotope supply
In a move to build up its dominance in the radiopharma market, Telix Pharmaceuticals Ltd. said it plans to acquire radioisotope production technology firm Artms Inc. and its advanced cyclotron-based isotope production platform, manufacturing plant and stockpile of ultra-pure rare metals. Telix has been steadily acquiring isotope suppliers as the long-term supply of medical isotopes is becoming an urgent issue. Although demand for the isotopes is growing, the facilities that can make these products are aging and beginning to come offline, as earlier reported by BioWorld.
US agencies propose to tighten focus on PBMs and GPOs
Three U.S. federal government agencies have announced that their focus on consolidation in health care markets may soon ramp up, given concerns that consolidation may be having an impact on the cost and/or quality of patient care. Among the business types that are included in the request for information are pharmacy benefits managers (PBMs) and group purchasing organizations (GPOs), two business types of keen interest for the medical device and pharmaceutical industries.
CROI 2024: HIV is active in the CNS despite ART
On March 4, 2024, several groups of scientists discussed the challenges of investigating the effects of HIV in the central nervous system (CNS) at the oral abstract session on neuropathogenesis of HIV held during the 31st Conference on Retroviruses and Opportunistic Infections (CROI), in Denver. A cure for HIV will require eliminating the virus in all its reservoirs, those tissues where HIV remains latent but retains the capacity for reactivation and replication. However, despite antiretroviral therapy (ART), the virus could continue to replicate continuously at a low level in some reservoirs, including the CNS.
Exonate’s topical retinal disease drug to advance on early data
Exonate Ltd. is preparing to advance its eye drop candidate to treat retinal vascular diseases, EXN-407, into a phase IIb study following promising tolerability and early signs of efficacy in a phase Ib/IIa study. “The planning process is underway, and it is our intention to enroll the first patient in Q4,” a spokesperson for the Cambridgeshire and Nottingham, U.K.-based company told BioWorld.
3D organoids arising from amniotic fluid cells may be a potent prenatal tool
Organoids are 3D models created from human stem cells and resemble fetal tissues. In an article published in Nature Medicine on March 4, 2024, researchers from University College London provided details on the possibility of generating organoids from epithelial cells collected from amniotic fluid without terminating the pregnancy.
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