All articles tagged with #phase 1 trial
Enveda reports Phase 1 results for an oral drug candidate that mimics the exercise-induced lactate-phenylalanine pathway (lac-phe) to curb appetite and preserve lean muscle, with the goal of maintaining weight loss without the GI side effects seen with GLP-1 drugs.
A phase 1 trial of the experimental vaccine mKRAS-VAX in 20 high-risk individuals showed robust KRAS-specific immune responses in 90% (about 18-fold on average) and no pancreatic cancer development after 16.5 months; 37.5% had shrinkage or disappearance of at-risk cysts; the vaccine appeared safe with only injection-site reactions and flu-like symptoms. The study, limited by small size and not designed to assess efficacy, suggests potential for cancer interception but requires larger trials.
The first Bundibugyo Ebola vaccine, ChAdOx1 BDBV, is entering a UK phase 1 trial to test safety and immune response in 50 healthy adults (18–55). Recruitment is underway and vaccinations are imminent. Developed by the University of Oxford’s Oxford Vaccine Group with the Serum Institute of India on the same platform as AstraZeneca’s vaccine, the effort is backed by CEPI, which has supplied doses and funded development; if phase 1 succeeds, CEPI plans late-stage trials for potential emergency approval, as part of a broader global Ebola vaccine effort that includes partnerships with Merck, Moderna, and IAVI.
A Life Biosciences Phase 1 trial is testing ER-100, a gene therapy designed to reset the aging clock in eye cells using OSK (OCT4, SOX2, KLF4) to potentially restore vision in open-angle glaucoma. Early preclinical data suggest epigenetic restoration can improve cellular function, but the approach carries safety risks, including cancer, and it remains uncertain whether translating this to humans will succeed or be safe.
An AI-designed universal coronavirus vaccine completed a Phase 1 trial in 39 healthy adults, showing a 100% safety profile and robust immune responses against diverse coronaviruses. Delivered via a needle-free microfluidic DNA platform, the vaccine targets conserved viral core features rather than mutable surface spikes, signaling a potential shift from variant boosters to a single broad defense. If Phase 2 confirms durability and broad protection, the approach could reshape vaccine design and public health logistics, including reduced cold-chain needs and biomedical waste.
IMA401 is a TCR-based bispecific T cell engager that targets an HLA-A*02:01-presented MAGE-A4/8 peptide to recruit T cells and kill tumor cells. In a first-in-human phase 1 basket trial across 61 patients with MAGE-A4/8-positive solid tumors, the maximum tolerated dose was not reached and the RP2D was defined as 1–2 mg biweekly. TRAEs were common but manageable, led by cytokine release syndrome (mostly grade 1–2), lymphopenia and neutropenia; five DLTs occurred (neutropenia), with no DLTs at RP2D when dexamethasone premedication was used. Across efficacy-evaluable patients, confirmed ORR was 14% overall and 20% at RP2D, with higher activity in head and neck cancers (29%). One possibly related death occurred at 2.5 mg without premedication. In pembrolizumab expansion cohorts, tolerability matched monotherapy. PK showed a median half-life of ~17.5 days. These results support continued development of this half-life extended TCR-based TCER platform across diverse solid tumors.
At the AACR meeting, researchers report a phase 1 trial of a patient-specific mRNA vaccine for pancreatic cancer, made from each patient's tumor material after surgery and used with immunotherapy and chemotherapy. Eight patients showed immune responses, and seven of these responders were alive up to six years post-surgery, with about 90% of responders remaining alive at follow-up. While promising, the findings come from a small early trial, and a global phase 2 study is now underway to assess broader efficacy.
In a 14-day phase 1 trial, healthy volunteers taking TLC-2716 showed up to 38.5% lower triglycerides and up to 61% lower remnant cholesterol after meals, by inhibiting LXRα activity in the liver and gut. The oral drug was safe and well tolerated, suggesting potential for treating cardiometabolic disease, but longer studies in overweight/obese individuals with poor lipid profiles are needed to confirm benefits.
OCS-05, a neuroprotective treatment candidate for multiple sclerosis (MS) or other causes of optic nerve inflammation, has shown a favorable safety and pharmacological profile in healthy volunteers, according to Phase 1 trial data. A Phase 2 trial is now evaluating OCS-05’s safety and effectiveness in people with acute optic neuritis due to MS, myelin oligodendrocyte glycoprotein antibody-associated disease, or an unknown cause. The trial will assess changes in eye health, visual acuity, and overall disability.