NATIONAL INSTITUTES OF HEALTH

South Rampart Pharma Secures New Investment for Novel Non-Opioid Pain Treatment

16 hours ago

• South Rampart Pharma has secured investment from Gulf South Angels to advance SRP-001, a non-opioid pain medication that targets the central nervous system, into FDA fast-tracked Phase Two clinical trials. • The New Orleans-based company, spun out of LSU Health School of Medicine in 2016, has raised $9 million to date and is seeking an additional $8.5 million in Series A funding for crucial efficacy testing. • SRP-001 differentiates itself from recently approved non-opioid alternatives by targeting the central nervous system rather than peripheral neurons, potentially addressing the critical need for non-addictive pain management solutions.

NIH-Funded Teams Develop Precision Gene Delivery Systems for Neural Cells

19 hours ago

• Research teams funded by the NIH have created a versatile toolkit of gene delivery systems that can target specific neural cell types in the human brain and spinal cord with unprecedented precision. • The new delivery platform uses modified adeno-associated viruses (AAVs) to transport genetic material into targeted cells, potentially transforming how scientists study neural circuits without requiring genetically modified animals. • This breakthrough could accelerate the development of precise gene therapies for neurological disorders like ALS, Alzheimer's, and Parkinson's disease by targeting only affected cells rather than merely treating symptoms.

First Patient Dosed in Phase 2 OASIS Trial of TNX-102 SL for Acute Stress Reaction Following Trauma

a day ago

• Tonix Pharmaceuticals has initiated dosing in the Phase 2 OASIS trial evaluating TNX-102 SL for reducing acute stress reaction severity and acute stress disorder frequency following traumatic events. • The investigator-initiated study, sponsored by UNC and supported by a $3 million DoD grant, will enroll approximately 180 motor vehicle collision survivors through emergency departments across the U.S. • TNX-102 SL, a sublingual cyclobenzaprine formulation that has shown promise in improving sleep quality in PTSD patients, addresses a significant unmet need as no medications currently exist for immediate post-trauma treatment.

FDA Grants IND Clearance for Immunoglobulin Eye Drops to Treat Dry Eye Disease

a day ago

• The FDA has granted Investigational New Drug clearance for immunoglobulin eye drops (SLG-100/GRF312), developed by Selagine and Grifols, to treat dry eye disease in upcoming Phase II clinical trials. • The novel treatment offers broad-spectrum anti-inflammatory and immunomodulatory properties, potentially providing mechanistic superiority over current narrow-spectrum therapies that primarily target T-cell inflammation. • A first-in-human pilot trial demonstrated significant reduction in dry eye symptoms with no difference in tolerability or adverse events compared to vehicle, showing promise for the over 100 million people affected globally.

FDA Overhauls COVID-19 Vaccine Approval Process, Limiting Access for Healthy Adults and Children

2 days ago

• The FDA has announced major changes to COVID-19 vaccine approvals, requiring placebo-controlled clinical trials for shots intended for healthy adults and children, effectively ending routine annual approvals for these groups. • Under the new framework, COVID-19 vaccines will remain readily available through streamlined approvals only for adults 65 and older and those with health conditions that increase risk of severe COVID-19. • The policy shift, spearheaded by FDA Commissioner Marty Makary and vaccine chief Vinay Prasad, represents a significant departure from the previous "one-size-fits-all" approach to COVID-19 vaccination recommendations.

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

7 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval

a month ago

• The FDA has asked Novavax to conduct an additional randomized, controlled study of its COVID-19 vaccine NVX-CoV2601 before considering full approval, despite the vaccine having emergency use authorization since 2022. • Interim results from a phase 2/3 study showed Novavax's updated XBB.1.5 variant vaccine generated 5.8 times higher neutralizing antibody levels compared to the original formulation, with a favorable safety profile. • The FDA's request represents a significant setback for Novavax, which had expected approval based on prior conversations with regulators and had already passed its April 1 PDUFA date.

Spear Bio and Beckman Coulter Receive FDA Breakthrough Device Designations for Alzheimer's Blood Tests

4 months ago

• Spear Bio's pTau 217 blood test received FDA Breakthrough Device Designation, offering a less invasive method for early Alzheimer's diagnosis. • Beckman Coulter's Access p-Tau217/β-Amyloid 1-42 plasma ratio test also gained FDA Breakthrough Device Designation, aiding in identifying amyloid pathology. • Both tests address the critical need for accessible, early Alzheimer's diagnosis, potentially improving patient outcomes and treatment access. • These designations expedite the development and review process, bringing innovative diagnostic tools to market faster for Alzheimer's disease.

mRNA Cancer Vaccines Show Promise in Clinical Trials, Aiming for Personalized Immunotherapy

5 months ago

• Over 60 mRNA cancer vaccine candidates are in clinical trials, signaling a transformative shift in cancer treatment. • BioNTech's BNT111, targeting melanoma-associated antigens, shows positive Phase 2 data when combined with cemiplimab. • Personalized mRNA vaccines, like Moderna's mRNA-4157, are being explored in combination with PD-1 therapies for various cancers. • The first commercial mRNA cancer vaccine is expected by 2029, driven by technological advancements and increased investment.

TIL Therapy and Novel Combinations Advance Melanoma Treatment

5 months ago

• Lifileucel (Amtagvi), a tumor-derived autologous T-cell immunotherapy, received FDA accelerated approval for unresectable or metastatic melanoma after PD-1 inhibitor treatment. • Studies show lifileucel, alone or with pembrolizumab, yields objective response rates of 31.5% to 65.2% in advanced melanoma, even after multiple prior therapies. • IBI363, a PD-1, IL-2α bispecific antibody, demonstrates promising disease control and objective response rates in melanoma patients who have progressed on PD-1 inhibitors. • Personalized cancer vaccine mRNA-4157 (V940) combined with pembrolizumab significantly reduces the risk of disease recurrence in resected stage IIIB to IV melanoma.