Herantis Pharma Advances HER-096 for Parkinson's Disease: Final Cohort of Phase 1b Trial Now Underway
• Herantis Pharma has dosed the first patients in the final cohort of its Phase 1b trial for HER-096, a first-in-class peptidomimetic with disease-modifying potential for Parkinson's disease.
• HER-096 represents a potential breakthrough as it crosses the blood-brain barrier, allows for convenient subcutaneous administration, and targets the underlying disease mechanisms rather than just symptoms.
• The trial, funded by the Michael J. Fox Foundation and Parkinson's UK, has successfully completed its first patient cohort with topline data expected in September 2025.
Hillhurst Bio Secures $6.3 Million in Grants to Advance Novel Parkinson's Disease Treatment
• Hillhurst Biopharmaceuticals has received $6.3 million in grants, including $2 million from The Michael J. Fox Foundation and $4.3 million from the Farmer Family Foundation in collaboration with Massachusetts General Hospital.
• The funding will support a Phase 2a clinical trial evaluating Hillhurst's innovative liquid drug therapy designed to protect against Parkinson's disease progression through cytoprotective pathways.
• The 36-participant study, scheduled to begin in early 2025, will assess safety, pharmacokinetics, and biomarkers associated with a 14-day dosing regimen of the novel therapeutic approach.
FDA Grants IDE Approval for Newronika's Novel Adaptive Deep Brain Stimulation System for Parkinson's Disease
• Newronika has received FDA Investigational Device Exemption (IDE) approval to commence a pivotal trial evaluating their adaptive deep brain stimulation system for movement disorders, particularly Parkinson's disease.
• The company's innovative AlphaDBS system utilizes real-time brain signal feedback to deliver personalized stimulation therapy, potentially offering improved outcomes over conventional DBS treatments.
• The upcoming multinational pivotal trial will compare the adaptive system's performance against traditional DBS in advanced Parkinson's disease patients, marking a crucial step toward U.S. regulatory approval.
Kazia Therapeutics Launches Phase 1b Trial of Paxalisib with Immunotherapy for Advanced Breast Cancer
• Kazia Therapeutics has initiated the ABC-Pax trial to evaluate paxalisib combined with immunotherapy in advanced breast cancer patients.
• The Phase 1b study will assess the safety and efficacy of paxalisib with pembrolizumab or olaparib in triple-negative breast cancer.
• The trial utilizes a novel liquid biopsy platform for real-time monitoring of cancer and immune cell behavior during treatment.
• This combination aims to reprogram dormant cancer cells, making them susceptible to immune attack, potentially improving patient outcomes.
Grifols Receives $21 Million Grant to Identify Parkinson's Disease Biomarkers
• Grifols has been awarded a $21 million grant from the Michael J. Fox Foundation to discover plasma-based biomarkers for Parkinson's disease.
• The Chronos PD study will analyze over 100 million biospecimens to identify early risk indicators and potential therapeutic targets.
• The initiative aims to develop new diagnostic tools and disease-modifying therapies by tracking plasma protein evolution over a decade.
• Grifols' subsidiary, Alkahest, will lead the AI-driven analysis of multiomics data to accelerate biomarker discovery.
Alector Announces Strategic Priorities for 2025, Highlights Neurodegenerative Disease Programs
• Alector anticipates topline data from the Phase 3 INFRONT-3 trial of latozinemab for frontotemporal dementia with a granulin gene mutation (FTD-GRN) by Q4 2025.
• Enrollment in the Phase 2 PROGRESS-AD trial of AL101/GSK4527226 for early Alzheimer's disease is expected to complete in mid-2025, with 75% of participants enrolled.
• Alector is advancing its preclinical pipeline, utilizing the Alector Brain Carrier (ABC) technology to enhance therapeutic delivery for neurodegenerative diseases.
• The company's cash reserves of $457.2 million as of September 30, 2024, are projected to sustain operations through 2026, supporting ongoing clinical and research programs.
Blarcamesine Shows Sustained Cognitive Benefits in Early Alzheimer's Patients
• Anavex Life Sciences' blarcamesine demonstrates significant reduction in clinical decline over three years for early Alzheimer's patients.
• Delayed-start analysis reveals early treatment with blarcamesine leads to greater cognitive stability, highlighting the importance of early intervention.
• The ATTENTION-AD trial confirms blarcamesine's favorable safety profile, with mostly mild to moderate adverse events and no treatment-related deaths.
• Peer-reviewed data from a Phase IIb/III study published in JPAD supports blarcamesine's potential as a novel oral treatment for early Alzheimer's.
Neumirna Therapeutics Secures €20M to Advance Epilepsy Drug NMT.001 into Clinical Trials
• Neumirna Therapeutics raised €20M in Series A funding to advance its RNA-based therapies for neurological conditions, including drug-resistant epilepsy.
• The funding will support clinical trials for NMT.001, a therapeutic candidate showing unprecedented efficacy in preclinical studies for drug-resistant epilepsy.
• FutureNeuro, an Irish research center, played a key role in the preclinical development of NMT.001, highlighting the importance of industry-academia collaboration.
• Neumirna's RNA-based platform targets underlying causes of diseases like epilepsy and Parkinson's, offering potential transformative solutions for patients.
Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024
• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach.
• Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein.
• A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease.
• The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.
Anavex Life Sciences Announces EMA Acceptance of Blarcamesine MAA for Alzheimer's Treatment
• The European Medicines Agency (EMA) has accepted Anavex's Marketing Authorization Application (MAA) for blarcamesine to treat Alzheimer's disease.
• Anavex presented Phase IIb/III ATTENTION-AD Open-Label-Extension (OLE) trial data at the J.P. Morgan 2025 Healthcare Conference.
• Preliminary Phase 2 clinical study results of ANAVEX3-71 for schizophrenia showed a dose-dependent effect on key EEG biomarkers.
• Anavex reported a strong cash position of $132.2 million, anticipating a cash runway of approximately four years.
Gain Therapeutics' GT-02287 Enters Phase 1b Trial for Parkinson's Disease
• Gain Therapeutics has initiated a Phase 1b clinical trial in Australia for GT-02287, targeting both idiopathic and GBA1-linked Parkinson's disease.
• The trial aims to evaluate the safety, tolerability, and biomarker changes over three months of GT-02287 administration in Parkinson's patients.
• GT-02287 previously demonstrated a favorable safety profile and significant GCase enzyme engagement in a Phase 1 study with healthy volunteers.
• Interim results from the Phase 1b trial are expected by mid-2025, potentially paving the way for Phase 2 trials in the U.S. and EU.
HanAll, Daewoong, and NurrOn Announce Positive Phase 1 Results for Parkinson's Disease Therapy HL192 (ATH-399A)
• HanAll Biopharma, Daewoong Pharmaceutical, and NurrOn Pharmaceuticals completed a Phase 1 study of HL192 (ATH-399A) for Parkinson's disease, meeting primary endpoints for safety and tolerability.
• The Phase 1 trial included single ascending dose (SAD), multiple ascending dose (MAD), and food effect components in 76 healthy participants, including older adults.
• HL192 (ATH-399A) demonstrated a favorable safety profile with no serious treatment-related adverse events and supported once-daily dosing, paving the way for further clinical trials.
• The companies plan to initiate the next clinical trial in people with Parkinson's disease to further evaluate the safety and efficacy of HL192 (ATH-399A).
Anavex's Blarcamesine MAA Accepted by EMA for Alzheimer's Treatment
• Anavex Life Sciences' blarcamesine MAA has been accepted by the EMA for review as a potential Alzheimer's treatment, marking a regulatory milestone.
• Clinical data from Phase IIb/III trials showed blarcamesine significantly improved cognition and slowed neurodegeneration in early Alzheimer's patients.
• Blarcamesine offers a convenient once-daily oral administration and a favorable safety profile without requiring routine MRI monitoring.
• If approved, blarcamesine could provide a novel treatment option for the 7 million Europeans affected by Alzheimer's disease.
Anavex's Blarcamesine Shows Efficacy in Phase IIb/III Alzheimer's Trial
• Anavex Life Sciences' blarcamesine (ANAVEX®2-73) demonstrated pre-specified clinical efficacy in a Phase IIb/III study for early Alzheimer's disease.
• The trial data confirmed blarcamesine's mechanism of action through SIGMAR1 gene activation, showing a significant reduction in clinical decline.
• Blarcamesine significantly slowed clinical progression by 36.3% in the primary endpoint ADAS-Cog13 (p=0.008) over 48 weeks in the ITT analysis.
• The company is preparing for regulatory submission in Europe (EMA) in the current quarter of 2024, with blarcamesine showing a good safety profile.
Anavex's ANAVEX3-71 Shows Promise in Schizophrenia Phase 2 Trial with EEG Biomarker Improvements
• Anavex Life Sciences reports positive preliminary results from its Phase 2 trial of ANAVEX3-71 in schizophrenia, showing dose-dependent effects on EEG biomarkers.
• The trial demonstrated that ANAVEX3-71 improved 40 Hz Auditory Steady-State Response (ASSR) Inter Trial Coherence (ITC) and Resting State Alpha Power compared to placebo.
• ANAVEX3-71 was well-tolerated, with no serious adverse events reported, suggesting potential for a novel treatment approach for schizophrenia.
• Part B of the Phase 2 study is ongoing with more participants and longer treatment duration, with data expected in the first half of 2025.
FDA Approves AbbVie's Vyalev for Advanced Parkinson's Disease
• The FDA has approved AbbVie's Vyalev (foscarbidopa and foslevodopa) as a 24-hour subcutaneous infusion for motor fluctuations in advanced Parkinson's disease.
• Vyalev is the first and only levodopa-based therapy delivered as a continuous 24-hour infusion, offering a non-surgical treatment option for patients.
• Clinical trials showed Vyalev significantly increased "on" time without troublesome dyskinesia and decreased "off" time compared to oral carbidopa/levodopa.
• The most common adverse reactions were infusion site events, hallucinations, and dyskinesia, with Medicare coverage expected in the second half of 2025.
FDA Backs Alpha-Synuclein Assay for Parkinson's Trials, Aiding Drug Development
• The FDA supports using the α-synuclein seed amplification assay (αSyn-SAA) biomarker in clinical trials for Parkinson's disease and related conditions, potentially accelerating drug development.
• The decision follows collaboration between The Michael J. Fox Foundation and the Critical Path Institute, leveraging data from the Parkinson's Progression Markers Initiative.
• αSyn-SAA demonstrates high accuracy in detecting Parkinson's early, even before symptoms, enabling better-targeted therapies and efficient clinical trials.
• Amprion's SAAmplify-αSYN Biomarker Test is currently the only validated synSAA assay available in the U.S. for improving the diagnosis of Parkinson’s and other synucleinopathies.
Blarcamesine Shows Promise in Slowing Alzheimer's Decline in Phase IIb/III Trial
• Blarcamesine significantly slowed clinical decline in early Alzheimer's patients by 38.5% (50mg) and 34.6% (30mg) compared to placebo on the ADAS-Cog13 cognitive endpoint.
• The trial demonstrated blarcamesine's benefits on key biomarkers, including amyloid-beta and brain volume, indicating a potential impact on underlying Alzheimer's pathology.
• Anavex Life Sciences expects to submit blarcamesine for regulatory approval in Europe (EMA) in Q4 2024, marking a significant step toward potential availability.
• Blarcamesine's oral administration and safety profile, without neuroimaging adverse events, could offer a convenient and accessible treatment option for early Alzheimer's disease.
Anavex's Phase 2b/3 Trial of Blarcamesine Shows Promising Results in Alzheimer's Disease Treatment
Anavex Life Sciences Corp. announced significant findings from its Phase 2b/3 trial of blarcamesine (ANAVEX®2-73) for early Alzheimer's disease, showing a notable slowing in cognitive decline and reduction in amyloid beta biomarkers, alongside a decrease in brain atrophy rate.
© Copyright 2025. All Rights Reserved by MedPath