Tagged News
Foresee Pharmaceuticals Launches Phase 2 Trial of Mirivadelgat for Pulmonary Hypertension Associated with Interstitial Lung Disease
- Foresee Pharmaceuticals has initiated a multinational Phase 2 trial evaluating mirivadelgat, an aldehyde dehydrogenase 2 activator, in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD).
- The double-blind, three-arm study will enroll approximately 126 subjects across multiple sites in Taiwan, measuring pulmonary vascular resistance as the primary endpoint over a 12-week treatment period.
- The trial represents a potential new therapeutic approach for PH-ILD patients, a condition with limited treatment options, by targeting the aldehyde dehydrogenase 2 pathway rather than conventional pulmonary vasodilator mechanisms.
Highlighted Clinical Trials:
Foresee Pharmaceuticals Co., Ltd.
Posted 3/1/2025
Apertura Gene Therapy Partners with Rett Syndrome Research Trust to Advance Blood-Brain Barrier-Crossing Gene Therapy Platform
- Apertura Gene Therapy and Rett Syndrome Research Trust announced a collaboration to license Apertura's TfR1-targeted AAV capsid technology for developing genetic medicines to treat Rett syndrome.
- The TfR1 CapX platform enables intravenous delivery of genetic medicines across the blood-brain barrier, achieving over 50% neuronal and 90% astrocyte transduction in preclinical studies.
- The partnership will integrate the capsid technology into RSRT's MECP2 Editing Consortium, a $40 million initiative aimed at bringing three genetic medicines to clinical trials by 2028.
- Rett syndrome affects primarily girls due to MECP2 gene mutations, causing severe developmental regression and requiring round-the-clock care throughout life.
FDA Budget Cuts Linked to Drug Approval Delays as Small Biotech Faces PDUFA Postponement
- Recent government efficiency cuts at the FDA appear to be causing delays in drug approval timelines, with 66% of healthcare professionals surveyed expressing concern about potential PDUFA date postponements.
- Stealth BioTherapeutics' treatment for rare Barth syndrome, elamipretide, has experienced an unexplained decision delay, with no typical safety or manufacturing concerns cited as reasons for the postponement.
- Industry experts warn that while widespread disruption isn't yet evident, continued FDA resource constraints could significantly impact clinical trial oversight and create approval backlogs lasting years.
ImmVira Reports Favorable Safety Profile for Oncolytic Virus MVR-C5252 in Phase I Glioma Trial
- ImmVira presented Phase I clinical results for MVR-C5252, an oncolytic herpes simplex virus therapy targeting malignant glioma, at the 2025 AACR annual meeting.
- The study demonstrated a favorable safety profile with no serious adverse events or dose-limiting toxicities in three patients who received treatment via convection-enhanced delivery.
- MVR-C5252 is engineered with PD-1 antibody and IL-12 to combine oncolytic effects with immune activation, addressing a cancer with less than 5% five-year survival rate.
- The therapy has received Investigational New Drug approval in both the U.S. and China, plus FDA Orphan Drug Designation for this rare indication.
BPGbio's BPM31510 Demonstrates Breakthrough Blood-Brain Barrier Penetration for Mitochondrial Disease Treatment
- BPGbio's novel CoQ10 formulation BPM31510 successfully crosses the blood-brain barrier and delivers CoQ10 to the cerebellum, potentially addressing ataxic symptoms in primary CoQ10 deficiency.
- Advanced spatial quinomics studies in Coq4 KI/KI mice demonstrated significant CoQ10 level increases across multiple critical tissues including brain, kidney, muscle, and heart.
- The compound has received FDA Rare Pediatric Disease Designation for primary CoQ10 deficiency and shows superior bioavailability compared to standard oral CoQ10 supplements.
- Preclinical studies revealed BPM31510 preserved cell viability in severely metabolically impaired human cells, while cells treated with CoQ10 alone died within a week.
Mallinckrodt to Present Five Clinical Studies on TERLIVAZ for Hepatorenal Syndrome at DDW 2025
- Mallinckrodt will present five clinical analyses of TERLIVAZ (terlipressin) for hepatorenal syndrome with rapid reduction in kidney function (HRS-AKI) at Digestive Disease Week 2025 in San Diego.
- TERLIVAZ is the first and only FDA-approved treatment for improving kidney function in adults with HRS-AKI, a rare and life-threatening condition affecting approximately 42,000 Americans annually.
- The presentations will include real-world evidence on treatment outcomes in various patient populations, including those on transplant waitlists and with alcohol-associated liver disease.
Ipsen Secures Dual CHMP Approvals for Rare Liver Disease Treatments Following Regulatory Strategy Shift
- Ipsen received CHMP approval for odevixibat under the new brand name Kayfanda for Alagille syndrome, marking the second approval for the same drug after rebranding due to orphan status complications.
- The company simultaneously secured CHMP recommendation for Iqirvo (elafibranor) as a treatment for primary biliary cholangitis, representing a rare dual approval achievement.
- Odevixibat demonstrated statistically significant improvements in scratching severity in the ASSERT trial, the world's first phase 3 study completed in Alagille syndrome patients.
- Both approvals address significant unmet medical needs in rare cholestatic liver diseases, with analysts estimating the PBC market alone could exceed $1.5 billion annually.
Chugai's Vabysmo Becomes First Approved Treatment for Angioid Streaks in Japan
- Chugai Pharmaceutical has received Japanese regulatory approval for Vabysmo as the first-ever treatment for choroidal neovascularization associated with angioid streaks, a rare eye disease that can lead to vision loss.
- The approval follows positive Phase III NIHONBASHI study results showing statistically significant visual acuity improvement of +5.8 letters at week 12 and a reduction in central retinal thickness of -106.4 μm.
- Vabysmo, a bispecific antibody targeting both VEGF-A and Ang-2 pathways, was generally well-tolerated with no new safety concerns identified during the clinical trial.
Study Reveals Significant Diagnostic Delays for Minority Patients with Generalized Myasthenia Gravis
- Project ASPIRE research reveals racial and ethnic minority patients with generalized myasthenia gravis (gMG) face nearly four-month longer diagnostic delays compared to white patients, despite seeking care at similar timeframes.
- Key barriers to timely gMG diagnosis include poor symptom recognition by both patients and physicians, limited access to specialists, and challenges with medical record transfers between healthcare systems.
- Minority patients reported significantly higher stress levels during their diagnostic journey, with nearly double the percentage rating their experience as "very stressful" compared to white patients.
Ascelia Pharma Advances Orviglance NDA Following Positive FDA Meeting, Submission Planned for Mid-2025
- Ascelia Pharma has received positive guidance from the FDA regarding its New Drug Application for Orviglance, a novel contrast agent for MRI liver imaging in patients with impaired kidney function.
- The company's Phase 3 SPARKLE study met its primary endpoint with statistical significance (p<0.001), demonstrating Orviglance's ability to improve visualization of focal liver lesions in the target patient population.
- Following successful completion of nine clinical studies involving 286 participants, Ascelia plans to submit the Orviglance NDA by mid-2025, likely in early August, with a cash runway extending through at least the end of 2025.