Clinical Trial News

Lupin's share price surged over 1% to a 52-week high after USFDA approval for its generic Mirabegron tablets for overactive bladder. Lupin and Zydus are the only companies with approval for both 25 mg and 50 mg strengths, enjoying 6 months exclusivity. Lupin also launched doxorubicin hydrochloride liposome injection in the US.

Dr. Roos Jansåker's clean-bracket study assesses light-activated antibacterial treatment's efficacy in enhancing dental plaque control for adolescents with braces. The Lumoral device, utilizing PBM, aims to reduce plaque and gingival inflammation, potentially benefiting periodontal tissues. Despite PBM's positive effects in healing and reducing pain, its use in dentistry remains limited. The study highlights the challenges of oral hygiene with braces, including increased risk of caries and gingivitis, and the potential of Lumoral to mitigate these risks. The trial results are anticipated in early 2025.

Tisagenlecleucel (Kymriah) is increasingly used in earlier lines of therapy for pediatric and young adult relapsed/refractory B-ALL, reducing HSCT use and high disease burden. Data from the CIBMTR registry show a decrease in late-line tisagenlecleucel use and high disease burden rates, with improved morphological CR rates and favorable long-term safety profiles.

NOX inhibitors, including Calliditas Therapeutics' setanaxib, show potential in treating fibrotic diseases and cancers by reducing ROS levels, thereby allowing CD8+ T-cells to penetrate tumors and enhance immune response. Setanaxib has shown promising results in Phase II trials for squamous cell carcinoma of the head and neck, with plans for further trials in primary biliary cholangitis and Alport Syndrome.

Alzamend Neuro partners with Massachusetts General Hospital for a Phase 2 trial of AL001, an oral therapy for Alzheimer’s-related dementia. The trial aims to determine the minimum effective dose and safety profile of AL001, a novel lithium-delivery system, compared to marketed lithium. Preclinical studies show AL001 prevents cognitive impairment and improves learning and memory in mouse models of Alzheimer’s. A completed Phase 1/2 trial suggests AL001 is well-tolerated and achieves similar blood lithium levels to marketed lithium at lower doses.

ESC Congress 2024 featured 4,400 abstracts, including 112 studies published simultaneously. Deepak Bhatt highlighted FINEARTS-HF, ABYSS, RESHAPE-HF2, and SHAM-PVI. FINEARTS-HF showed finerenone reduced heart failure events by 16%. ABYSS found no benefit in stopping beta-blockers post-MI. RESHAPE-HF2 supported MitraClip use in heart failure with mitral regurgitation. SHAM-PVI found pulmonary vein isolation more effective than a sham procedure for atrial fibrillation.

A Phase II trial showed AstraZeneca's Lynparza (olaparib) effective in treating recurrent prostate cancer without hormone therapy, prompting larger follow-up studies in biomarker-selected subgroups. Lynparza, a PARP inhibitor, demonstrated efficacy in patients with homologous recombination repair gene mutations, particularly BRCA2, with higher response rates than seen with hormonal suppression. The study suggests that not all prostate cancer patients need hormone therapy and highlights the need for biomarker-selected trials to identify responders.

eGenesis secures $191 million to advance genetically edited pig kidneys for human transplantation, addressing the organ shortage crisis. The company's EGEN-2784 candidate, featuring genetic modifications to prevent rejection and viral transmission, achieved the world's first successful transplant of a genetically modified porcine kidney in a human in 2024.

FDA grants full approval to FILSPARI (sparsentan) for IgA nephropathy (IgAN), based on PROTECT Study results showing superior kidney function preservation over irbesartan. FILSPARI is an oral, non-immunosuppressive, once-daily treatment targeting glomerular injury, with a durable benefit on proteinuria and kidney function over two years.

Oppenheimer upgrades Ascendis Pharma to Outperform with a $180 price target, anticipating the U.S. launch of Yorvipath in Q1 2025 and Phase 3 results for TransCon CNP. The firm sees potential for €1.7 billion in global revenue by 2029 and undervaluation in TransCon CNP. Ascendis Pharma faces challenges with Skytrofa sales but aims for approvals of all three product candidates by end-2025. The company ended Q2 2024 with EUR259 million in cash and forecasts EUR220-240 million in Skytrofa revenue for 2024.