Clinical Trial News

Ropeginterferon alfa-2b, approved for polycythemia vera (PV) treatment, shows safety and efficacy in clinical trials. Emerging studies suggest higher initial doses and rapid titration may enhance tolerability and efficacy. Ongoing research explores its application in myeloproliferative neoplasms, aiming to optimize dosing for improved patient outcomes.

LOY-001, a drug aimed at extending the lifespan of large dogs, has cleared key FDA hurdles, potentially available by 2026. It targets the IGF-1 hormone, higher in large breeds, to match levels in smaller breeds. Pending further FDA approval, it could significantly impact service dogs' longevity and quality of life.

Ensysce Biosciences, Inc. reflects on its 2022 milestones, including advancements in its opioid abuse deterrent and overdose protection programs, and outlines future goals in a letter to shareholders from CEO Dr. Lynn Kirkpatrick.

In April 2022, FDA approved axi-cel for adults with refractory or early relapsed LBCL, based on ZUMA-7 trial showing longer event-free survival vs standard therapy. Axi-cel had higher rates of cytokine release syndrome and neurologic toxicity but marks a paradigm shift in LBCL treatment.

A phase II study investigated the safety and efficacy of durvalumab in treating BCG-unresponsive non-muscle-invasive bladder cancer, showing a 12% complete response rate at month 6 with manageable side effects.

STK-001, Stoke Therapeutics' treatment for Dravet syndrome, shows promise in reducing seizures in children and adolescents, with Phase 1/2a trials indicating safety and efficacy. Interim results suggest a shift towards syndrome management, with further data expected to inform Phase 3 trials. STK-001 aims to restore NaV1.1 levels, potentially improving quality of life.

Evusheld, the only authorized COVID-19 preexposure prophylaxis, may not neutralize subvariant XBB.1.5, responsible for nearly 30% of cases, as per FDA. Efficacy against several dominant omicron subvariants remains undetermined. FDA advises healthcare providers to inform patients of increased risk with variants not neutralized by Evusheld.

A study involving over 2,400 patients with type 2 diabetes and overweight or obesity found that initiating a GLP-1 agonist without a structured behavioral intervention led to a 2% weight loss at 72 weeks. One-third of the patients achieved clinically significant weight loss, defined as at least 5% change from baseline. The study highlights the potential of GLP-1 agonists for weight loss in real-world settings, despite the absence of structured weight loss interventions.

Syros Pharmaceuticals reported a Q1 2023 EPS of $-0.85, beating expectations of $-1.09. The company highlighted progress in clinical trials for MDS, AML, and APL, including the SELECT-MDS-1 trial's amendment to include overall survival as a key secondary endpoint. Syros anticipates pivotal data from SELECT-MDS-1 in Q3 2024 and remains on track for milestones across its programs. Financials show a net loss of $23.8M, with $166M in cash, expected to fund operations into 2025.

Editas Medicine reported Q4 2022 earnings, highlighting clinical progress with EDIT-301 for sickle cell disease and beta thalassemia, strategic shifts towards in-vivo gene editing, and financial stability extending into 2025. The company aims to advance its gene editing technologies and clinical trials, focusing on delivering transformative medicines.