WASHINGTON--(BUSINESS WIRE)--KeifeRx, a privately held biotechnology company developing disease-modifying therapies for neurodegenerative disorders, today announced that it has entered into a research collaboration and option agreement with Amneal Pharmaceuticals to support the pre-IND development of KFRX06, KeifeRx's preclinical candidate designed to inhibit leucine-rich repeat kinase 2 (LRRK2), a gene strongly implicated in Parkinson's disease biology. Under the agreement, KeifeRx will conduc. ...

PresentationAntti VuolantoChief Executive Officer Hello, and welcome to Herantis Pharma's Phase Ib Biomarker Data webinar. I am CEO, Antti Vuolanto, joined today by CSO, Henri Huttunen; and CFO, Tone Kvale. We are really excited to discuss yesterday evening's announced biomarker data for HER-096, our lead asset currently in development for the treatment of Parkinson's disease. These data present a significant milestone for the company, providing evidence of biological response to HER-096 dosing in humans wi ...

Core Insights - AC Immune SA has released interim results from the Phase 2 VacSYn trial for its anti-alpha-synuclein immunotherapy ACI-7104.056, indicating potential disease modification in early Parkinson's disease [1][2] - The results show stabilization of disease-related biomarkers, suggesting a slowing of neuronal damage and progression of Parkinson's disease [2][3] Biomarker Results - Elevated levels of neurofilament light (NfL) are associated with neuronal damage; stabilization of NfL levels indicates a potential slowing of neurodegeneration [2] - Plasma glial fibrillary acidic protein (GFAP) and dopamine transporter (DaT) SPECT imaging trends also suggest disease modification [3] Antibody Response - ACI-7104.056 demonstrated a 100% responder rate in generating antibodies against the a-syn target antigen, with serum antibody titers over 500-fold higher than the placebo group at week 76 [3][4] - Antibody responses were boosted with each immunization, and average IgG antibody levels in cerebrospinal fluid (CSF) were also over 500-fold higher than in the placebo group [4] Clinical Measures - Clinical measures of motor symptoms indicate a trend toward stabilization in the active treatment group, with no meaningful progression in the MDS-UPDRS Part III score compared to an expected increase in the placebo group [5][6] - The difference in change from baseline scores between the active treatment and placebo groups was enhanced when stratified by levodopa (L-DOPA) ON/OFF state [6] Safety and Tolerability - Interim results from weeks 50 and 76 show that ACI-7104.056 is generally safe and well-tolerated [6] - Final data from Part 1 of the VacSYn trial are anticipated in mid-2026 [6] Market Reaction - Following the announcement, AC Immune's stock price increased by 12.43%, reaching $3.14 [6]

Core Insights - Amneal Pharmaceuticals announced positive interim results from its Phase 4 ELEVATE-PD study for CREXONT, a new treatment for Parkinson's disease, showcasing significant clinical benefits for patients [1][2]. Group 1: Study Results - The first 55 patients in the study showed substantial improvements after six weeks of treatment with CREXONT, including an increase in daily "Good On" time by 1.86 hours, a reduction in "Off" time by 0.77 hours, and an increase in "Good On" time per dose by 0.79 hours [3][4]. - The MDS-UPDRS total score improved by an average of -14.2 points, indicating enhanced motor function [4]. - CREXONT demonstrated a longer duration of benefit with each dose compared to other oral carbidopa/levodopa therapies, highlighting its differentiated clinical performance [4][5]. Group 2: Safety and Efficacy - Treatment-emergent adverse events (TEAEs) were generally mild to moderate, with common side effects including nausea (5.5%), falls (3.6%), dizziness (3.6%), and urinary tract infection (3.6%) [6]. - The ongoing ELEVATE-PD study aims to provide longer-term data and patient-reported outcomes in 2026, further supporting CREXONT's impact on motor symptom control and functional independence [6][8]. Group 3: Product Information - CREXONT is an innovative formulation that combines immediate-release granules and extended-release pellets, designed to optimize levodopa delivery and absorption, providing the longest-lasting plasma levels of any oral CD/LD therapy available [2][7]. - The ELEVATE-PD study is an open-label, multi-center clinical trial designed to evaluate the real-world efficacy and safety of switching to CREXONT in adults with moderately severe Parkinson's disease [8].

Core Insights - Umecrine Cognition has published data indicating that early treatment with golexanolone may delay the progression of Parkinson's disease symptoms and postpone the need for L-DOPA treatment [1][4] Company Overview - Karolinska Development AB holds a 60 percent ownership stake in Umecrine Cognition, which is focused on developing a new class of drugs to alleviate cognitive symptoms associated with Parkinson's disease [4] - The company is a Nordic life sciences investment firm that identifies and invests in breakthrough medical innovations in the Nordic region [5][6] Research Findings - A preclinical study shows that early administration of golexanolone leads to stronger and longer-lasting relief from symptoms compared to later treatment [3] - The study demonstrated significant reductions in severe fatigue and lack of movement, along with enhanced motor coordination and improved gross motor function [3][4] - Golexanolone appears to rebalance key neurotransmitter systems in the brain, suggesting its potential as a disease-modifying treatment rather than merely providing symptomatic relief [4]

Core Insights - Prothena Corporation (PRTA) reported a third-quarter 2025 adjusted loss per share of 67 cents, which was wider than the Zacks Consensus Estimate of a loss of 60 cents, compared to a loss of $1.10 per share in the same quarter last year [1][7] - Revenues for the quarter totaled $2.4 million, significantly missing the Zacks Consensus Estimate of $25 million, and up from $0.1 million in the year-ago quarter [1][7] Financial Performance - Research and development (R&D) expenses decreased by 42.9% year over year to $28.9 million, attributed to lower clinical trial, manufacturing, personnel, and consulting expenses [4] - General and administrative expenses were reported at $13.2 million, down 21% year over year [4] - As of September 30, 2025, Prothena had $331.7 million in cash, cash equivalents, and restricted cash, with no debt [4][7] Pipeline Developments - Prothena is collaborating with Roche to evaluate prasinezumab for the treatment of Parkinson's disease, with Roche set to initiate the late-stage PARAISO study by the end of 2025, expecting peak sales potential of over $3.5 billion [5] - Novo Nordisk has acquired Prothena's clinical-stage antibody, Coramitug, for treating ATTR amyloidosis with cardiomyopathy, and has initiated the late-stage CLEOPATTRA study [6] - Prothena is advancing an early-stage pipeline for neurological indications in collaboration with Bristol Myers Squibb (BMY), including BMS-986446, which has received Fast Track designation from the FDA for Alzheimer's disease [8][9] Future Expectations - Prothena anticipates earning a clinical milestone when enrollment criteria are met in the ongoing phase III study by Novo Nordisk [8] - The company expects a net cash burn from operating and investing activities in 2025 to be between $170 million and $178 million, with a projected year-end cash balance of approximately $298 million [12] - The projected net loss for 2025 is estimated to be in the range of $240 million to $248 million [12]

Core Viewpoint - AbbVie has submitted a New Drug Application (NDA) to the FDA for tavapadon, a novel treatment for Parkinson's disease, based on positive results from the TEMPO clinical development program [1][2][6]. Clinical Development Program - The submission is supported by results from the TEMPO clinical development program, which includes two Phase 3 trials (TEMPO-1 and TEMPO-2) for early Parkinson's disease and one Phase 3 trial (TEMPO-3) for patients on levodopa experiencing motor fluctuations [2][3]. - TEMPO-1 and TEMPO-2 showed statistically significant improvement in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III combined score at week 26 [2][4]. - TEMPO-3 demonstrated increased "on" time without dyskinesia for patients on levodopa [2][6]. Trial Details - TEMPO-1 enrolled 529 adults aged 40-80 with early Parkinson's disease, evaluating two fixed doses of tavapadon over 27 weeks [4]. - TEMPO-2 involved 304 adults with flexible doses of tavapadon, also over 27 weeks [5]. - TEMPO-3 included 507 adults on stable doses of levodopa, assessing tavapadon as adjunctive therapy [7]. Safety and Efficacy - The majority of adverse events reported in the trials were non-serious and mild to moderate in severity, with low incidence of serious adverse events (SAEs) [8]. - Common adverse reactions included nausea, headache, and dizziness for patients without levodopa, and nausea and dyskinesia for those on adjunctive therapy [8]. Market Potential - If approved, tavapadon will enhance AbbVie's leadership in Parkinson's disease treatment by providing a once-daily oral option [6]. - More than 11 million people globally are living with Parkinson's disease, indicating a significant market opportunity for effective treatments [9]. About Tavapadon - Tavapadon is an investigational selective D1/D5 receptor partial agonist studied for use both with and without levodopa [10]. - It is not yet approved by any health regulatory authority [10]. AbbVie's Commitment - AbbVie has over three decades of experience in neuroscience and is dedicated to advancing treatment options for neurological disorders [11]. - The company aims to address serious health issues and improve the lives of patients through innovative medicines [12].

Financial Data and Key Metrics Changes - The company continues to be profitable even through the transition from legacy products to new growth products [4] - KELBRE has shown a 25% growth in prescriptions year-over-year and a 23% growth in the first half of the current year [16] - GOCOVRI has seen a significant increase in patient retention due to Medicare redesign, with 97% of patients having less than $25 co-pay by June [24] Business Line Data and Key Metrics Changes - KELBRE has grown to 35% of the business, with an 80% satisfaction rate among adult users, significantly higher than Strattera's 53% [13] - ZERZUVEY, the first oral treatment for postpartum depression, is expected to set the standard of care, with 80% of women receiving it as a first-line treatment [7] - Anapco, the new infusion device for Parkinson's, is performing well, with demand matching that of AbbVie's product [30] Market Data and Key Metrics Changes - The back-to-school season has shown a 26% growth in the last four-week rolling average for KELBRE, indicating strong momentum [17] - The company has seen a 36% growth in unit shipments of ZERZUVEY quarter-over-quarter, reflecting a healthy brand performance [36] Company Strategy and Development Direction - The acquisition of Sage allows the company to enter women's health while maintaining its CNS focus, indicating a strategic expansion [5] - The company is prioritizing M&A to fill its portfolio with post-Phase II assets, aiming for launches between 2026 and 2030 [58][59] - The company is taking a pause to reevaluate the Sage pipeline while integrating it with its own discovery programs [40] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of ZERZUVEY to become the standard of care for postpartum depression [8] - The company anticipates a strong second half of the year, driven by back-to-school momentum and continued growth in its product portfolio [19] - Management is confident that the increased demand from Medicare redesign will offset the mandatory rebates on manufacturers [26] Other Important Information - The company is exploring the potential of its pipeline, particularly focusing on SPN-817 for seizures, which has shown promising results [41] - The company is also considering the future of XERZUVEY and its potential new indications in collaboration with Biogen [39] Q&A Session Summary Question: Can you discuss the acquisition rationale for Sage? - The acquisition allows entry into women's health while maintaining CNS focus, addressing a significant unmet need in postpartum depression [5] Question: What are the expectations for the collaboration with Biogen? - The collaboration is expected to be mutually beneficial, with both companies learning from each other across various functions [10] Question: How is KELBRE performing in the adult ADHD market? - KELBRE has achieved 35% of the business in adults, with an 80% satisfaction rate, indicating strong market acceptance [13] Question: What is the outlook for GOCOVRI following Medicare redesign? - The redesign has improved patient retention, with 97% of patients having low co-pays, which is expected to continue [24] Question: How is Anapco performing compared to AbbVie's product? - Anapco's performance is in line with AbbVie's product, with both addressing a significant need in the market for advanced Parkinson's treatment [30] Question: What is the company's approach to M&A? - The company remains focused on acquiring post-Phase II assets to enhance its portfolio and ensure future growth [58][59]

Summary of Supernus Pharmaceuticals (SUPN) Conference Call Company Overview - Supernus Pharmaceuticals is a CNS-focused biopharmaceutical company with a portfolio of about eight to nine products on the market [2][3] - The company is profitable and has a clean balance sheet, recently completing the acquisition of Sage Therapeutics [2][3] - Supernus has transitioned from the loss of exclusivity on two legacy products and is now in a re-accelerated growth phase driven by key products like Qelbree, Gocovri, Onapgo, and ZURZUVAE [3] Key Products and Market Insights Qelbree (ADHD Treatment) - Qelbree is a novel non-stimulant ADHD medication that works as early as week one, addressing a significant market need for effective non-stimulant options [7][8] - The ADHD market consists of approximately 100 million prescriptions annually, with Supernus currently holding less than 2% market share, indicating substantial growth potential [8][19] - The adult segment represents 67% of the ADHD market, and Supernus has seen growth in this segment, with 35% of their business coming from adults [18][19] - Qelbree has shown a growth rate of 20% plus, with recent data indicating a 25-26% growth during the back-to-school season [18][19] Onapgo (Parkinson's Treatment) - Onapgo is a new continuous infusion device for apomorphine, launched in April, aimed at treating off episodes in Parkinson's patients [23][24] - The device has received positive initial responses, and its market potential is still being evaluated, with estimates ranging from $200 to $300 million [29][30] - The product is designed for advanced patients but may also be used for earlier-stage patients, which could expand its market opportunity [29][30] ZURZUVAE (Postpartum Depression) - ZURZUVAE is the first oral medication approved for postpartum depression (PPD), a condition affecting 1 in 7 women [31] - There is a significant lack of awareness and education regarding PPD, with only about 40% of women diagnosed over the years [31][32] - Supernus is actively working on educating healthcare providers and patients about PPD, emphasizing the importance of screening and diagnosis [32][38] Industry Dynamics and Challenges - The ADHD treatment landscape has historically relied on stimulants, which do not work for 15-30% of patients, creating a demand for effective non-stimulant alternatives [9][10] - In the Parkinson's space, approximately 90-95% of patients experience off episodes after five years of diagnosis, highlighting the need for effective treatments [22][25] - The company is addressing the stigma around ADHD and PPD through influencer partnerships and educational initiatives [21][38] Conclusion - Supernus Pharmaceuticals is positioned for significant growth with its innovative product offerings in ADHD, Parkinson's, and postpartum depression - The company is focused on expanding its market share and improving patient outcomes through education and effective treatment options, indicating a strong potential for future success in the biopharmaceutical industry [3][19][31]

Core Viewpoint - Anavex Life Sciences Corp. is advancing its clinical-stage biopharmaceutical developments, particularly focusing on innovative treatments for Alzheimer's disease and other CNS disorders, with promising financial results and clinical data supporting its lead drug candidate, blarcamesine [1][2]. Recent Highlights - Anavex presented open-label extension data for blarcamesine at the AAIC 2025, showing continued clinically meaningful benefits in early-stage Alzheimer's patients [2][7]. - The company participated in the AAIC 2025, emphasizing the importance of sharing knowledge to advance dementia science [7]. Financial Highlights - Cash and cash equivalents stood at $101.2 million as of June 30, 2025, down from $132.2 million at the end of fiscal 2024, indicating a cash runway of over 3 years at current utilization rates [4][7]. - Research and development expenses for the quarter were $10.0 million, a decrease from $11.8 million in the same quarter of fiscal 2024 [4][7]. - General and administrative expenses increased to $4.5 million from $2.8 million in the comparable quarter of fiscal 2024 [4][7]. - The net loss for the quarter was $13.2 million, or $0.16 per share, compared to a net loss of $12.2 million, or $0.14 per share for the same quarter in fiscal 2024 [4][7]. Clinical Development - Blarcamesine has shown benefits over a period of up to 4 years in treated patients, as measured by clinical endpoints ADAS-Cog13 and ADCS-ADL [7]. - The drug candidate is designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors, with preclinical studies indicating its potential to halt or reverse Alzheimer's disease [9].