University of Kansas

Presentations will highlight findings from studies evaluating investigational ND0612 in Parkinson's disease and RADICAVA® (edaravone) formulations in ALS JERSEY CITY, N.J., April 3, 2025 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) announced 11 presentations will be shared at the American Academy of Neurology (AAN) 2025 Annual Meeting, taking place April 5-9 in San Diego, CA, and online. The presentations will discuss two posters in Parkinson's disease (PD) and nine in amyotrophic lateral sclerosis (ALS). "We are honored to join the scientific community at this year's AAN meeting, where we will discuss key insights from our clinical and real-world studies across PD and ALS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. "Our presence at the meeting, and the data presented, underscore our dedication to advancing impactful research and deepening our understanding of these progressive neurodegenerative diseases." PD data to be presented at the meeting include: Investigational ND0612: Presentations will highlight findings from the pivotal Phase 3 BouNDless trial (NCT04006210), including an oral presentation detailing the impact of 24-hour treatment with ND0612 on PD motor state transitions throughout the day, as well as an analysis of quality of life outcomes in people with PD experiencing motor fluctuations. Impact of 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612) on Motor Function: Efficacy Outcomes from the Phase 3 BouNDless Study (Rajesh Pahwa, M.D.; University of Kansas Medical Center) Scientific Platform Session S37 (#001): 1:00 p.m. – 1:12 p.m. PDT, April 9 Quality of Life With 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612): QoL Results from a Phase 3, Randomized, Active-Controlled Study (Harini Sarva, M.D., FAAN; New York Presbyterian Hospital) Poster Session P11 (#5-007): 8:00 a.m. – 9:00 a.m. PDT, April 9 ALS data to be presented at the meeting include: RADICAVA ORS Studies: A presentation will share final results from the MT-1186-A03 extension study, which evaluated the safety and tolerability of RADICAVA ORS® (edaravone) over 96 weeks in ALS patients who had previously participated in MT-1186-A01. Additionally, findings from the MT-1186-A04 extension study will be presented, assessing the continued efficacy and safety of RADICAVA ORS over an additional 48 weeks, comparing investigational daily dosing with the U.S. Food and Drug Administration (FDA)-approved on/off regimen. Lastly, an analysis will examine the impact of RADICAVA ORS on survival and function in ALS patients, leveraging data from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database and propensity score matching to compare RADICAVA ORS-treated patients with participants from studies MT-1186-A01 through A04. Phase 3, Open-Label, Safety Extension Study of Oral Edaravone (Radicava ORS®) Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Scientific Platform Session S27 (#007): 2:12 p.m. – 2:24 p.m. PDT, April 8 Extension Study MT-1186-A04 Evaluating Oral Edaravone (Radicava ORS®) Continued Efficacy and Safety up to an Additional 48 Weeks in Patients With ALS (Stephen Apple, M.D.; MTPA) Poster Session P9 (#2-007): 11:45 a.m. – 12:45 p.m. PDT, April 8 Long-term Function and Survival of Radicava ORS® (Oral Edaravone)-Treated Patients With ALS vs Propensity Score–Matched PRO-ACT Historical Controls (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Poster Session P8 (#2-003): 8:00 a.m. – 9:00 a.m. PDT, April 8 Real-World Data: Multiple posters highlighting real-world data will be presented, including two studies evaluating at-home telespirometry in ALS. One examines changes in erect and supine slow vital capacity based on non-invasive ventilation (NIV) status, while the other assesses point-of-care quality classifications for slow and forced vital capacity measurements. Additionally, an analysis of real-world data will examine trends in ALS treatment utilization in the U.S. from 2016 to 2022, highlighting shifts in prescribing patterns, adoption of FDA-approved therapies and the decreasing proportion of untreated patients. Other presentations include an analysis of referral patterns and healthcare utilization among people with ALS enrolled in the OPTUM claims database, a secondary analysis describing demographics and healthcare resource utilization across specialty vs. non-specialty treatment centers, and the application of a clinical algorithm to electronic medical records to reduce the delay in time to diagnosis and treatment for patients with ALS. Smartphone─Mediated At-Home Telespirometry Erect and Supine Slow Vital Capacity Decline Differences Based On NIV Status in Subjects With ALS (Stephen Apple, M.D.; MTPA) Poster Session P8 (#2-004): 8:00 a.m. – 9:00 a.m. PDT, April 8 Smartphone─Mediated, At-Home Telespirometry Vital Capacity (VC) Measurements in ALS: Point-of-Care Quality Assessment for Slow VC and Forced VC (Stephen Apple, M.D.; MTPA) Poster Session P8 (#2-006): 8:00 a.m. – 9:00 a.m. PDT, April 8 Evolution of the Treatment Landscape for Patients Diagnosed with Amyotrophic Lateral Sclerosis in the United States (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P9 (#2-006): 11:45 a.m. – 12:45 a.m. PDT, April 8 Referral Patterns for Patients With Amyotrophic Lateral Sclerosis Enrolled in a US-Based Administrative Claims Database (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P8 (#2-009): 8:00 a.m. – 9:00 a.m. PDT, April 8 Treatment of Amyotrophic Lateral Sclerosis in General and Specialist US Healthcare Settings: Results From a Real-World Survey (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P8 (#2-012): 8:00 a.m. – 9:00 a.m. PDT, April 8 Application of a Clinical Algorithm on Real-World Electronic Medical Record Data to Assist With Earlier Detection of Amyotrophic Lateral Sclerosis (Amanda E. Fiander, M.D., FRCPC, CSCN (EMG); Maritime Neurology) Poster Session P8 (#2-010): 8:00 a.m. – 9:00 a.m. PDT, April 8 About RADICAVA ORS® (edaravone) The U.S. Food and Drug Administration (FDA) approved RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA granted RADICAVA ORS Orphan Drug Exclusivity based on its major contribution to patient care by providing an oral suspension route of administration that avoids the burdens of IV administration. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. Each 105 mg (5mL) dose of RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.1 Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021) and Brazil (February 2024). Marketing authorization for RADICAVA® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA ORS, along with the previously available IV RADICAVA® (edaravone), have been used to treat over 16,000 people with ALS, with over 2.0-million days of therapy, and have been prescribed by over 2,400 HCPs.2-4 INDICATION RADICAVA ORS® (edaravone) is indicated for the treatment of amyotrophic lateral sclerosis (ALS). IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions RADICAVA ORS® (edaravone) is contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred. Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA ORS, treat per standard of care, and monitor until the condition resolves. Sulfite Allergic Reactions RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people. Adverse Reactions The most common adverse reactions (≥10%) reported in RADICAVA® (edaravone)-treated patients and at least 2% more frequently than placebo were contusion (15% vs 9%), gait disturbance (13% vs 9%), and headache (10% vs 6%), respectively. In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. Pregnancy Based on animal data, RADICAVA ORS may cause fetal harm. To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or . Please see the full Prescribing Information, also available at . About ND0612 ND0612 is an investigational drug-device combination therapy – a 24-hours/day, continuous subcutaneous (SC) infusion of liquid levodopa/carbidopa (LD/CD) for the treatment of motor fluctuations in people with Parkinson's disease (PD). Development of investigational ND0612 is being led by NeuroDerm, Ltd., a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on X (formerly Twitter), Facebook and LinkedIn. About Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma Corporation (MTPC) is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety. In addition, MTPC is working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to . About NeuroDerm, Ltd. NeuroDerm, Ltd. is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), based in Israel, inspired to reduce disease burden and improve the quality of life of patients and their families through innovative drug-device combination therapies and technologies. NeuroDerm is an integrated pharmaceutical and medical technology company developing central nervous system (CNS) product candidates. For additional information, please visit NeuroDerm's website at  or follow the Company on LinkedIn.  Media inquiries: [email protected] 1 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022 2 Data on file. Mitsubishi Tanabe Pharma America, Inc. 3 Data on file. Mitsubishi Tanabe Pharma America, Inc. 4 Data on file. Mitsubishi Tanabe Pharma America, Inc. 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– FDA aligned on plan to initiate parallel Phase 2 trials of rademikibart in patients with moderate-to-severe asthma or COPD experiencing an acute exacerbation – – Expect to initiate both trials in Q2 2025 – SAN DIEGO, April 01, 2025 (GLOBE NEWSWIRE) -- Connect Biopharma Holdings Limited (Nasdaq: CNTB) (Connect or Connect Biopharma), a clinical-stage biopharmaceutical company focused on transforming acute and chronic care of asthma and chronic obstructive pulmonary disease (COPD), today announced positive feedback from its Type C meeting with the U.S. Food and Drug Administration (FDA), Division of Pulmonology, Allergy, and Critical Care, in the Office of Immunology and Inflammation. “We are pleased to have the FDA’s alignment on our two parallel Phase 2 trials evaluating rademikibart in patients experiencing an acute exacerbation of asthma or COPD, an area where no biologic therapies have been approved or systematically studied,” said Barry Quart, Pharm.D., CEO and Director of Connect Biopharma. “During our Type C meeting, the FDA acknowledged the unmet need for reducing recurrent exacerbations during the vulnerable 28-day period following an acute exacerbation of asthma or COPD. In our previously completed Phase 2 study for the maintenance treatment of asthma, a 600 mg subcutaneous (SC) loading dose of rademikibart was well-tolerated and delivered robust improvement in pulmonary function in less than 24 hours. This promising data suggests that rademikibart could provide meaningful and rapid benefit to the millions of patients who experience exacerbations of asthma or COPD and are seen in emergency departments or are hospitalized each year.” Based on the advice from the FDA at the meeting, Connect plans to begin enrolling patients in these trials during the second quarter of this year. The two parallel Phase 2 randomized, double-blind, placebo-controlled trials are expected to each enroll approximately 160 patients and include patients with uncontrolled, moderate-to-severe asthma or COPD with eosinophils ≥300 cells/µL who are experiencing an acute exacerbation. The trials are designed to evaluate the benefits of a single 600 mg SC dose of rademikibart in patients over 28 days following an acute exacerbation. Based on the results of a recently published trial of a similar design, approximately 45% of patients receiving the current standard of care experienced treatment failure in the 28 days following an acute exacerbation, demonstrating the continued unmet need for better treatments for these patients1. Connect expects to report data from both rademikibart Phase 2 trials in the first half of 2026 with a cash runway into 2027. “Asthma and COPD patients face a heightened risk of further exacerbations within the first 28 days following an initial episode. While biologics have demonstrated significant success in maintenance therapy, none have been approved to address airway symptoms immediately after an acute exacerbation,” said Mario Castro, MD, MPH, Professor and Chief of Pulmonary, Critical Care and Sleep Medicine at the University of Kansas School of Medicine. “The pulmonology community is encouraged by the plan to evaluate the potential benefits of a single 600 mg SC dose of rademikibart following an acute exacerbation in these Phase 2 trials in patients with asthma and COPD.” About Connect Biopharma and Rademikibart Connect Biopharma is a clinical-stage biopharmaceutical company dedicated to transforming care for asthma and COPD. Headquartered in San Diego, California, the company is advancing rademikibart, a next-generation, potentially best-in-class anti-interleukin-4-receptor alpha (IL-4Rα) antibody. With an initial focus on acute exacerbations—an area with significant unmet need—rademikibart has the potential to also drive chronic utilization in asthma and COPD amongst the approximately 1 million asthma patients and 1.3 million COPD patients in the U.S. who experience acute exacerbations annually. In a Phase 2 trial for asthma, rademikibart demonstrated strong efficacy and safety data, with clinically meaningful reductions in exacerbations and rapid, statistically significant improvements in FEV1, observed within one week—and in most cases, within 24 hours via home spirometry. For more information visit www.connectbiopharm.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended (the “Act”). Forward-looking statements are statements that are not of historical fact and include, without limitation, statements regarding future events, our future financial condition, results of operations, business strategy and plans, prospective products (as well as their potential to achieve a differentiated, competitive, or favorable benefit or profile or trend, including on safety, tolerability, improvement, maintenance, clinical response, dosing, efficacy and/or convenience), planned or expected product approval applications or approvals, anticipated milestones, expected data readouts and enrollments, research and development plans and costs, potential future partnerships, expectations about existing partnerships, timing and likelihood of success, objectives of management for future operations, future results of anticipated product development efforts, and adequacy of existing cash and potential partnership funding to fund operations and capital expenditure requirements, as well as statements regarding industry trends. These statements are based on management’s current expectations of future events only as of the date of this press release and are inherently subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond our control, including, among other things: the ability of our clinical trials to demonstrate safety and efficacy of our product candidates and other positive results; whether we will need expanded or additional trials in order to obtain regulatory approval for our product candidates; our ability to obtain and maintain regulatory approval of our product candidates; existing regulations and regulatory developments in the U.S., the PRC, Europe and other jurisdictions; the ability of our current cash and investments position to support planned operations; our plans and ability to obtain, maintain, protect and enforce our intellectual property rights and our proprietary technologies, including extensions of existing patent terms where available; our continued reliance on third parties to conduct additional clinical trials of our product candidates, and for the manufacture of our product candidates for preclinical studies and clinical trials; and the degree of market acceptance of our product candidates, if approved, by physicians, patients, healthcare payors and others in the medical community. Words such as “aim,” “anticipate,” “believe,” “could,” “expect,” “feel,” “goal,” “intend,” “may,” “optimistic,” “plan,” “potential,” “promising,” “will,” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements necessarily contain these identifying words. The inclusion of forward-looking statements should not be regarded as a representation by Connect Biopharma that any of its expectations, projections or plans will be achieved. Actual results may differ materially due to the risks and uncertainties inherent in our business and other risks described in our filings with the U.S. Securities and Exchange Commission (the “SEC”). Further information regarding these and other risks is included under the heading “Risk Factors” in our periodic reports filed with the SEC, including in our annual report on Form 10-K for the year ended December 31, 2024, and any subsequent filings with the SEC. These forward-looking statements should not be taken as forecasts or promises nor should they be taken as implying any indication, assurance or guarantee that the assumptions on which such forward-looking statements have been made are correct or exhaustive or, in the case of the assumptions, fully stated in this presentation. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You are cautioned not to place undue reliance on the scientific data presented or these forward-looking statements, which speak only as of the date of this presentation. Except as required by law, Connect Biopharma undertakes no obligation to publicly update any forward-looking statements, whether because of new information, future events or otherwise. Connect Biopharma claims the protection of the safe harbor for forward-looking statements contained in the Act for all forward-looking statements. This press release discusses product candidates that are under clinical study, and which have not yet been approved for marketing by the U.S. Food and Drug Administration or by any other regulatory agency. No representation is made as to the safety or effectiveness of these product candidates for the use for which such product candidates are being studied. The trademarks included herein are the property of the owners thereof and are used for reference purposes only. Investor Relations Contact: Alex Lobo Precision AQ Alex.lobo@precisionaq.com(212) 698-8802 Media Contact: Ignacio Guerrero-Ros, Ph.D., or David SchullRusso Partners, LLCIgnacio.guerrero-ros@russopartnersllc.comDavid.schull@russopartnersllc.com(858) 717-2310 or (646) 942-5604 Ramakrishnan S, et.al. Treating eosinophilic exacerbations of asthma and COPD with benralizumab (ABRA): a double-blind, double-dummy, active placebo-controlled randomised trial. Lancet 2025; 13(1): 59-68.

TUESDAY, April 1, 2025 -- For nearly two decades, a stroke had left a woman unable to speak -- until now. Thanks to a new brain implant, her thoughts are being turned into real-time speech, giving her a voice again for the first time in 18 years. The device was tested on a 47-year-old woman with quadriplegia who lost her ability to speak after a stroke . Doctors placed the brain-computer implant during surgery as part of a clinical trial, The Associated Press reported. It “converts her intent to speak into fluent sentences,” said Gopala Anumanchipalli , an assistant professor of electrical engineering and computer sciences at UC Berkeley and a co-author of the study published Monday in the journal Nature Neuroscience . Unlike other brain-computer systems that have a delay, this new technology works in real time. Scientists say that the delay in existing systems makes conversations hard and, sometimes, frustrating. Jonathan Brumberg of the Speech and Applied Neuroscience Lab at the University of Kansas, who reviewed the findings, welcomed the advances. This is “a pretty big advance in our field,” he told The Associated Press . Here’s how it works: The team recorded the woman’s brain activity using electrodes as she silently imagined saying sentences. They also used a synthesizer using her voice before her stroke to re-create the sound she would have made. An AI model was trained to translate her brain signals into sounds. The system sends each small sound unit -- roughly a half-syllable -- into a recorder every 80 milliseconds. That means it works quickly, just like real-time transcription on a phone call. “It’s not waiting for a sentence to finish,” Anumanchipalli said. “It’s processing it on the fly.” Decoding speech that fast could help the device keep up with the speed of natural conversation, Brumberg added. Using a person’s actual voice “would be a significant advance in the naturalness of speech.” Though the research was partly funded by the National Institutes of Health, Anumanchipalli said it was not affected by recent cuts to NIH funding. He added that more research is still needed, but with “sustained investments,” the device could be available to patients within 10 years. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.