Edaravone

SHANGHAI, Oct. 31, 2025 /PRNewswire/ -- Shanghai Zhimeng Biopharma Inc. ( "Zhimeng") announced that its self-developed new-generation potassium channel opener CB03-154 for the treatment of amyotrophic lateral sclerosis (ALS) has officially initiated the phase 2/3 clinical trial and completed the first patient first dose at the 2nd Affiliated Hospital of Hebei Medical University. Previously (in July 2025), CB03-154 received approval from the China Center for Drug Evaluation (CDE) to conduct the ALS phase 2/3 adaptive clinical trial. The CB03-154 has also been granted orphan drug status in the United States in October 2023 for the treatment of ALS. This phase 2/3 clinical trial is a multicenter, randomized, double-blind, placebo-controlled study with open-label extension. This study is led by Professor Fan Dongsheng from the 3rd Affiliated Hospital of Peking University as the leading investigator nationwide. This study will enroll approximately 240 patients diagnosed with ALS in about 15 centers in China. The disease duration of to be enrolled patients will be within 24 months from the first symptom onset to the screening visit. The primary efficacy endpoint of the study is the change from baseline at week 39 of the revised ALS Functional Rating Scale (ALSFRS-R) score. Amyotrophic Lateral Sclerosis (ALS) is a progressive and fatal central nervous system disease with unknown etiology. It mainly affects the motor neurons in the cerebral cortex, brainstem, and spinal cord. The pathological features of ALS include the loss of upper and lower motor neuron cell bodies, as well as the degeneration of the corticospinal tract/corticobulbar tract and axons of lower motor neurons, and the occurrence of denervation changes in muscles. Clinically, it mainly presents as progressive skeletal muscle weakness, atrophy, fasciculation, bulbar palsy, and pyramidal tract signs. The median survival duration is generally 2 to 3 years after the onset of symptoms. The main cause of death is neuromuscular respiratory failure. The annual incidence of ALS in Europe and the United States is 2/100,000 to 3/100,000, and the prevalence rate is 3-5/100,000. The peak age of onset is 50 to 75 years. Approximately 10% of ALS patients are familial, while the remaining 90% are sporadic. Currently, there are no treatments that can stop or reverse the progression of the disease. Therefore, clinical management focuses on early diagnosis and early treatment to maximize the survival time. Currently, only three drugs have been approved worldwide for ALS, namely Riluzole, Edaravone, and Tofersen (trade name Qalsody). These approved drugs can only slightly improve the patient's function or slightly prolong the survival time. Therefore, ALS still has a huge unmet medical need. Although the pathogenesis of ALS has not been fully deciphered, studies have found that it is closely related to changes in ion channels, synaptic transmission and connections, as well as the imbalance of excitation and inhibition of neurons. Among them, the potassium ion (K+) channel is the most widely distributed and has the largest variety, mainly participating in regulating the excitability of neurons and the frequency and amplitude of action potential generation. Currently, there are no marketed KCNQ2/3 potassium ion channel openers available for ALS patients. The only KCNQ2/3 potassium ion channel opener that was approved by the FDA and EMA, ritigabine (Potiga, Ezogabine/Retigabine), was once studied in ALS patients and showed a favorable signal. However, due to the risks of pigmentation-induced vision impairment and other risk factors, it was withdrawn from the market in 2017. CB03-154 is a new generation selective voltage-gated potassium channel (KCNQ2/3) opener, independently developed by Zhimeng. Compared with the first-generation potassium channel opener ritigabine, CB03-154 has superior selectivity and stability. In vitro studies have confirmed that CB03-154 can significantly reduce the excessive excitation of iPSC motor neurons from ALS patients. Moreover, in the SOD-1 ALS transgenic mouse model, daily oral administration of CB03-154 (10 mg/kg) can effectively prolong the survival time, delay the onset, improve motor ability, alleviate neuronal damage, and slow down the rate of muscle atrophy. The initiation of this Phase 2/3 clinical trial is a significant step for CB03-154 in the field of rare diseases, and it brought hopes to provide an important new treatment option for ALS patients. About Zhimeng Founded in 2018, Shanghai Zhimeng Biopharma, Inc. is a biopharmaceutical company dedicated to develop small-molecule drug discovery with focused on the functional cure of chronic hepatitis B (CHB), as well as novel therapies for CNS disorders, including epilepsy, neuropathic pain (e.g. cancer-related pain), amyotrophic lateral sclerosis (ALS), major depressive disorder (MDD), and bipolar disorder. Disclaimer This press release contains forward-looking statements. While Zhimeng considers the projections to be based on reasonable assumptions, these forward-looking statements may be called into question by a number of hazards and uncertainties, so that actual results may differ materially from those anticipated in such forward-looking statements. For more information, please visit: SOURCE Shanghai Zhimeng Biopharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Prescription medications were found to be a key driver of Medicare expenditures and patient liability ARLINGTON, Va., Oct. 23, 2025 /PRNewswire/ -- For individuals enrolled in Medicare, medical expenses in the first year following an ALS diagnosis top $47,000—more than three times higher than the average Medicare beneficiary, according to a new study led by the ALS Association. The findings, which were published in the October issue of The American Journal of Managed Care, also show that while only about one third of Medicare beneficiaries with ALS take an FDA-approved medication, the cost of these drugs is a key contributor to the disease's overall financial burden. "ALS is a devastating disease—physically, emotionally, and financially. Our goal was to quantify the medical costs accumulated throughout the first year following an ALS diagnosis and get a better understanding of the types of care that drive up these costs," said Sarah Parvanta, Ph.D., MPH, the Association's senior director of mission informatics and co-author of the study. "Understanding the magnitude of the financial burden faced by people living with ALS and the health care system is essential if we are going to reduce the harms of ALS and make it a livable disease." Approximately two out of three Americans living with ALS are covered by Medicare, based on data collected by the National ALS Registry and ALS Focus™ Survey Program. People diagnosed with ALS qualify for Medicare when they begin receiving disability benefits from the Social Security Administration, regardless of their age. For this cohort study, researchers from the ALS Association in partnership with Avalere Health analyzed 100% Medicare fee-for-service claims from 2017–2021, including Part A and Part B medical claims and Part D prescription drug event data. They found that people diagnosed with ALS during that timeframe had an average annual Medicare expenditure of $47,450 versus $13,889 for beneficiaries without ALS. The portion of these costs not covered by Medicare (patient liability) was also more than three times higher for people with ALS ($6,802 versus $2,050). The biggest expense for all Medicare beneficiaries was short-term acute hospital care ($11,237 for people with ALS versus $3,249 for those without ALS). Other notable cost drivers for people living with ALS included durable medical equipment ($5,583) and prescription drugs ($6,309). Surprisingly, only about 5% of Medicare beneficiaries with ALS had a claim for edaravone during the first 12 months following their diagnosis and a little more than 31% had a claim for riluzole. This low level of drug utilization was unexpected given clinical practice guidelines. "We know the financial burden of ALS is underestimated by this study because the Medicare dataset did not include any non-medical costs, like a wheelchair-accessible vehicle, home modifications, or lost income," Dr. Parvanta said. "These expenses can be overwhelming and cause stress, added work burden, and debt for people living with ALS and their families." An ALS Focus survey of 440 people living with ALS and caregivers conducted in 2020 found that one in four participants (25%) had to borrow money or went into debt because of their ALS treatment or the need to provide caregiving. Survey participants also said that covering the cost of medical treatments (41%) or medical services (39%), understanding their health insurance coverage (41%), and managing medical billing paperwork (38%) were highly stressful. "Making ALS livable will require new, innovative, and most-likely costly treatments that can slow down or stop further progression of the disease as well as restore lost function," said senior author Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. "This study shows that current ALS treatment and care already presents affordability challenges, so policymakers must consider how Medicare will address this significant cost burden. People living with ALS should not have to forfeit their financial health to improve their physical well-being." About the ALS Association The ALS Association is the largest ALS organization in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The mission of the ALS Association is to make ALS livable and cure it. For more information about the ALS Association, visit our website at . About ALS Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression. SOURCE The ALS Association WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED