RegulationOrphan Drug (United States), Priority Review (China), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Molecular FormulaC5H6N2

InChIKeyNUKYPUAOHBNCPY-UHFFFAOYSA-N

CAS Registry504-24-5

110

Clinical Trials associated with Dalfampridine

NCT04516603

/ WithdrawnPhase 2IIT

Randomized Placebo-controlled Phase II Cross-over Study on the Influence of Fampridine on Working Memory in Healthy Subjects

Proof-of-concept study on the effects of 10 mg fampridine (oral administration) on working memory in healthy participants.
The hypotheses is that fampridine improves working memory performance.

Start Date01 Jan 2040

Sponsor / Collaborator

University of Basel

[+1]

NCT07832331

/ Not yet recruitingPhase 1IIT

Single Dose 4-Aminopyridine for Diagnosing Recurrent Laryngeal Nerve Paralysis Continuity After Trauma

This study aims to evaluate the role of single-dose 4-aminopyridine on the diagnosis of recurrent laryngeal nerve (RLN) paralysis. In addition, the investigational treatment will be used to test the hypothesis that 4-aminopyridine can speed the determination of nerve continuity after RLN injuries.

Start Date01 Dec 2026

Sponsor / Collaborator

Hershey Medical Center

NCT06294821

/ Not yet recruitingPhase 2IIT

4-Aminopyridine to Delay Carpal Tunnel Release or Resolve Carpal Tunnel Syndrome

The investigators have found recent promising data supporting the use of a currently FDA approved drug, 4-aminopyridine, in the treatment of nerve injury including compression neuropathy. The purpose of this research is determine whether 4-aminopyridine can treat carpal tunnel syndrome or delay the need for formal surgical release in patients with known carpal tunnel syndrome who would otherwise undergo surgery.

Start Date01 Nov 2026

Sponsor / Collaborator

University of Arizona

100 Clinical Results associated with Dalfampridine

100 Translational Medicine associated with Dalfampridine

100 Patents (Medical) associated with Dalfampridine

8,506

Literatures (Medical) associated with Dalfampridine

01 Jan 2027·EXPERIMENTAL NEUROLOGY

The glioma metabolite, D-2-hydroxyglutarate (D-2-HG), reduces synaptic transmission and epileptiform bursts in neocortical slices

Article

Author: Shao, Li-Rong ; Stafstrom, Carl E ; Christiansen, McKenzie

80% of patients with low-grade gliomas containing mutations of the metabolic enzyme isocitrate dehydrogenase (IDH) experience seizures, of which nearly half are drug-refractory. Studies in isolated neuronal cultures have led to the hypothesis that the metabolite of the mutant IDH, D-2-hydroxyglutarate (D-2-HG), is epileptogenic by mimicking glutamate and activating NMDA receptors. To test this hypothesis, we used whole-cell patch-clamp techniques to determine the direct effects of D-2-HG on neuronal and synaptic functions and epileptiform activity in neocortical slices that preserve natural and balanced excitatory-inhibitory neural networks. Acute bath application of D-2-HG (10 mM) for 10-30 min did not cause significant neuronal depolarization, induce epileptiform activity, or alter the input-output relationship in layer V pyramidal neurons. D-2-HG slightly lowered action potential (AP) threshold (∼2-3 mV) and significantly reduced AP amplitude. In neocortical slices with ongoing epileptiform bursts (induced by 0 Mg²⁺ plus 4-aminopyridine), addition of D-2-HG reduced the frequency of the bursts (prolonged inter-burst intervals) and seizure burden. Moreover, D-2-HG diminished electrically evoked mono- and poly-synaptic excitatory postsynaptic currents. Together, the results demonstrate that short-term exposure to D-2-HG inhibits seizure-like activity in neocortical slices possibly by suppressing excitatory synaptic transmission. Thus, these data do not support the initial hypothesis that D-2-HG may excite neurons and promote seizures by mimicking glutamate actions.

01 Jan 2027·NEUROPHARMACOLOGY

4-methyl-3-aminopyridine: A novel active blocker of voltage-gated potassium ion channels in the central nervous system

Article

Author: Brugarolas, Pedro ; El-Saadi, Saadi ; Rodríguez-Rangel, Sofia ; Sánchez-Rodríguez, Jorge E ; Coronado, Oscar Gutiérrez ; Ortega, Brenda Mata ; Sun, Yang

Aminopyridines, including 4-aminopyridine (4AP), 3,4-diaminopyridine, and [¹⁸F]3-fluoro-4-aminopyridine, are voltage-gated potassium (K_V) channel blockers used clinically to enhance conduction in neurological disorders and to image demyelination by positron emission tomography (PET). Developing new aminopyridines may yield improved therapeutics or imaging agents. Here, we characterized the physicochemical properties (pK_a, logD), K_V channel-blocking activity, toxicity (LD₅₀), and pharmacokinetics of a novel compound, 4-methyl-3-aminopyridine (4Me3AP). 4Me3AP was less basic and more lipophilic than 4AP and showed greater blocking potency across multiple K_V channels expressed in Xenopus oocytes. In mice, 4Me3AP exhibited lower acute toxicity (LD₅₀ = 29.3 mg/kg) than 4AP (LD₅₀ = 12.7 mg/kg) and a longer plasma half-life. These findings indicate that 4Me3AP is a potent K_V channel blocker with favorable pharmacological properties, supporting its potential for symptomatic treatment of demyelinating diseases.

01 Dec 2026·JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Fampridine for Symptomatic Treatment in Chronic Inflammatory Demyelinating Polyneuropathy: A Randomized, Double‐Blinded, Placebo‐Controlled Crossover Study

Article

Author: Hansen, Peter Nørregaard ; Andersen, Henning ; Markvardsen, Lars ; Krøigård, Thomas ; Tankisi, Hatice ; Sindrup, Søren

ABSTRACT:

Background and Aims:

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an immune‐mediated neuropathy that may cause persistent disability despite immunoglobulin treatment. Ion channel dysfunction has been demonstrated in CIDP, but no therapies specifically target this mechanism. We investigated whether fampridine improves clinical function and electrophysiological measures in CIDP patients with residual symptoms.

Methods:

In this two‐center, randomized, double‐blind, placebo‐controlled crossover trial, patients with CIDP receiving stable subcutaneous or intravenous immunoglobulin were assigned to 4 weeks of fampridine (10 mg twice daily) or placebo, separated by a washout period. Co‐primary outcomes were changes in Six‐Spot‐Step Test (SSST) and Nine‐Hole Peg Test (9‐HPT). Secondary outcomes included clinical scores, patient‐reported outcomes, nerve conduction studies, and nerve excitability testing.

Results:

Twenty‐eight patients were included in the study. No significant differences were observed between fampridine and placebo for SSST ( p = 0.154) or 9‐HPT ( p = 0.857). Grip strength and R‐ODS worsened during fampridine compared with placebo (Bonferroni‐adjusted p = 0.007 for both), while other clinical outcomes showed no differences. Median nerve CMAP amplitude with proximal stimulation increased significantly during fampridine treatment (adjusted p = 0.040). No significant changes were observed in other electrophysiological variables, including nerve excitability testing. No serious adverse events occurred and adverse effects were predominantly mild.

Interpretation:

Our study did not provide evidence that fampridine improves clinical function in CIDP patients with residual disability during stable immunoglobulin therapy. Targeting ion channel dysfunction in CIDP remains of interest, but alternative strategies may be required.

News (Medical) associated with Dalfampridine

23 Jul 2026

·www.einpresswire.com

Thousands with MS to get “life-changing” drug on NHS to help them walk more freely

Thousands of people with multiple sclerosis (MS) in England are to be offered a new ‘signal booster’ therapy on the NHS to help them walk more easily and live more independently. Fampridine is the first medicine designed to improve walking in adults with MS and will now be routinely available on the NHS from today, NHS England has announced. Taken as a pill twice a day, fampridine acts like a ‘signal booster’ for nerves damaged by MS, helping electrical messages travel more effectively to the muscles. For some patients, an improvement in walking could make everyday tasks easier, from moving around the house and getting out independently to spending more time with family and friends. MS is a lifelong neurological condition caused by damage to nerves in the brain and spinal cord. Symptoms vary but can include weakness, fatigue, loss of sensation, problems with balance and coordination, and difficulty walking. Until now, support for MS-related walking difficulties has largely involved physiotherapy and exercises, walking aids, devices to support the feet and treatments to manage muscle stiffness. Clinical trials found fampridine (Fampyra) improved walking speed in 43% of patients and also helped people walk for longer. Patients will initially try the medicine for 2 to 4 weeks, with their walking assessed before and after treatment – with those who experience a clear benefit continuing to take it for as long as it keeps working, with regular reviews taking place. It is estimated that around 5,000 people living with MS will be eligible in England in the first year. Professor Frankie Swords, NHS National Medical Director, said: “Walking difficulties can have a huge impact on the freedom and independence of people with MS, so this signal-boosting pill could be life-changing for thousands of patients. “Fampridine helps electrical messages travel along nerves damaged by MS, giving people the chance to walk more easily and helping them be more mobile to do more of the everyday things that matter to them. “For some, even a modest improvement could mean getting around the house more easily, going out with greater confidence and living more independently.” Professor James Palmer, National Medical Director for Specialised Services at NHS England, said: “For people with MS who have spent years relying on physiotherapy, walking aids or help from others, having access to a one-of-a-kind treatment specifically for walking difficulties offers real hope of greater independence. “Thousands of patients will now be able to try fampridine on the NHS, with treatment continuing where it makes a clear difference to their mobility and daily lives.” Aysen Slack , 65, from Eastbourne lives with MS. Over the past few years it has been progressing more quickly, affecting her mobility. When she found out about fampridine a few years ago, she started paying for it privately. Unfortunately, the cost wasn’t feasible in the long-term, so she stopped taking the drug. Aysen said: “It’s such great news that fampridine will be available for people on the NHS in England. Fampridine seemed to be working well for me, but paying for the medication was a significant expense and I could not keep doing that forever. So I had to make the difficult decision to stop taking it. “I would definitely like to try fampridine again on the NHS. My mobility has decreased a lot and even in my flat I have to use sticks now. It would make a huge difference to my life if I were able to improve my walking.” The drug will be available for adults with any type of MS whose walking difficulties are assessed as having an Expanded Disability Status Scale score of between 4 (able to walk without aid or rest for around 500 metres) and 7 (unable to walk further than 5 metres and largely restricted to a wheelchair). The rollout follows a rigorous assessment by NHS England’s Clinical Priorities Advisory Group – which reviews specialist medicines, devices and treatments according to their benefit for patients and value for money – and brings access in England in line with other UK nations. Ceri Smith, Head of Policy and Evidence at the MS Society, said: “We’re delighted that fampridine has now been approved for use on the NHS. Over 120,000 people live with MS in England and Fampridine is the only licensed treatment that helps improve walking ability and speed for some of these people. “For many of them, it’s life-changing, allowing them to live more independently or stay in employment. It’s vital that MS services now have the support they need to make fampridine available to all people with MS who could benefit from it, regardless of where in England they live.” NHS England’s Clinical Priorities Advisory Group looks at drugs and treatments that are not being assessed by the National Institute for Health and Care Excellence; for example, where they are not licensed for the clinical indication in question. Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

07 Dec 2025

·www.einpresswire.com

New Investigator Sponsored Data Suggest Trilaciclib May Mitigate Chemotherapy-Induced TP53-Mutant Clonal Hematopoiesis

MORRISTOWN, NJ, UNITED STATES, December 7, 2025 / EINPresswire.com / -- Pharmacosmos Therapeutics Inc. today announced the presentation of new investigator-sponsored research at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition. The abstract, titled “CDK4/6 inhibition mitigates chemotherapy-induced expansion of TP53-mutant clonal hematopoiesis” (Abstract #8419), was featured during a Plenary Scientific Session on Sunday, December 7 from 2:00–4:00 p.m. ET. The study, led by investigators from Washington University in St. Louis and Memorial Sloan Kettering Cancer Center, evaluated whether the intravenous CDK4/6 inhibitor trilaciclib may reduce the expansion of chemotherapy-associated TP53-mutant clonal hematopoiesis (CH), a known risk factor for therapy-related myeloid neoplasms (tMN). Pharmacosmos Therapeutics provided trilaciclib and blood samples from clinical trials with trilaciclib for use in this investigator-sponsored study but did not sponsor or conduct the research. The research showed that across three placebo-controlled trials involving patients with small cell lung cancer (SCLC), metastatic colorectal cancer (mCRC), and metastatic triple-negative breast cancer (mTNBC), administration of trilaciclib prior to chemotherapy was associated with a significantly lower rate of TP53-mutant CH clone expansion compared to placebo. In preclinical murine models, concomitant treatment with trilaciclib and carboplatin prevented expansion of p53-mutant hematopoietic stem cells, while preserving normal progenitor cells under cytotoxic stress. Principal investigator and senior author Kelly Bolton, MD, PhD from Washington University in St. Louis, described this trial as “the first time that an approach has been identified to reducing expansion of TP53-mutant CH during therapy. The data generated regarding trilaciclib’s role are emerging across multiple clinical contexts, consistently demonstrating a potential pattern of reduced TP53-mutant CH growth.” Additionally, co-corresponding author Omar Abdel-Wahab, MD from Memorial Sloan Kettering Cancer Center added that “therapy related myeloid neoplasms are a very troubling complication of cancer therapy. There are currently very limited effective treatments when therapy-related myeloid neoplasms occur. We are very excited about this study, as the results provide the first clue about a strategy to prevent the development of therapy-related myeloid neoplasms.” These data represent the first demonstration in patients of a pharmacologic strategy that may help limit chemotherapy-induced expansion of pre-leukemic TP53-mutant clones. Further studies are needed to confirm clinical significance. About Clonal Hematopoiesis and Therapy-Related Myeloid Neoplasms Clonal hematopoiesis (CH) refers to the presence of somatic mutations in hematopoietic stem or progenitor cells that confer a selective growth advantage, leading to clonal expansion with age or under genotoxic stress. Expansion of TP53-mutant clones has been linked to an increased risk of developing therapy-related myeloid neoplasms (tMN), a rare but serious late complication of prior cytotoxic therapy. Currently, there are no approved interventions to prevent CH expansion or reduce the risk of tMN. About COSELA ® (trilaciclib) COSELA ® (trilaciclib) is an intravenously administered cyclin-dependent kinase (CDK) 4/6 inhibitor approved by the U.S. Food and Drug Administration to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC). COSELA is not approved for reducing clonal hematopoiesis or preventing therapy-related myeloid neoplasms. The recommended dose of COSELA is 240 mg/m², administered as a 30-minute intravenous infusion completed within four hours prior to the start of chemotherapy. Important Safety Information CONTRAINDICATION COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib. WARNINGS AND PRECAUTIONS Injection-Site Reactions, Including Phlebitis And Thrombophlebitis COSELA administration can cause injection-site reactions, including phlebitis and thrombophlebitis, which occurred in 56 (21%) of 272 patients receiving COSELA in clinical trials, including Grade 2 (10%) and Grade 3 (0.4%) adverse reactions. Monitor patients for signs and symptoms of injection-site reactions, including infusion-site pain and erythema during infusion. For mild (Grade 1) to moderate (Grade 2) injection-site reactions, flush line/cannula with at least 20 mL of sterile 0.9% Sodium Chloride Injection, USP or 5% Dextrose Injection, USP after end of infusion. For severe (Grade 3) or life-threatening (Grade 4) injection-site reactions, stop infusion and permanently discontinue COSELA. Injection-site reactions led to discontinuation of treatment in 3 (1%) of the 272 patients. Acute Drug Hypersensitivity Reactions COSELA administration can cause acute drug hypersensitivity reactions, which occurred in 16 (6%) of 272 patients receiving COSELA in clinical trials, including Grade 2 reactions (2%). Monitor patients for signs and symptoms of acute drug hypersensitivity reactions. For moderate (Grade 2) acute drug hypersensitivity reactions, stop infusion and hold COSELA until the adverse reaction recovers to Grade ≤1. For severe (Grade 3) or life-threatening (Grade 4) acute drug hypersensitivity reactions, stop infusion and permanently discontinue COSELA. Interstitial Lung Disease/Pneumonitis Severe, life-threatening, or fatal interstitial lung disease (ILD) and/or pneumonitis can occur in patients treated with cyclin-dependent kinases (CDK)4/6 inhibitors, including COSELA, with which it occurred in 1 (0.4%) of 272 patients receiving COSELA in clinical trials. Monitor patients for pulmonary symptoms of ILD/pneumonitis. For recurrent moderate (Grade 2) ILD/pneumonitis, and severe (Grade 3) or life-threatening (Grade 4) ILD/pneumonitis, permanently discontinue COSELA. Embryo-Fetal Toxicity Based on its mechanism of action, COSELA can cause fetal harm when administered to a pregnant woman. Females of reproductive potential should use an effective method of contraception during treatment with COSELA and for at least 3 weeks after the final dose. ADVERSE REACTIONS Serious adverse reactions occurred in 30% of patients receiving COSELA. Serious adverse reactions reported in >3% of patients who received COSELA included respiratory failure, hemorrhage, and thrombosis. Fatal adverse reactions were observed in 5% of patients receiving COSELA. Fatal adverse reactions for patients receiving COSELA included pneumonia (2%), respiratory failure (2%), acute respiratory failure (<1%), hemoptysis (<1%), and cerebrovascular accident (<1%). Permanent discontinuation due to an adverse reaction occurred in 9% of patients who received COSELA. Adverse reactions leading to permanent discontinuation of any study treatment for patients receiving COSELA included pneumonia (2%), asthenia (2%), injection-site reaction, thrombocytopenia, cerebrovascular accident, ischemic stroke, infusion-related reaction, respiratory failure, and myositis (<1% each). Infusion interruptions due to an adverse reaction occurred in 4.1% of patients who received COSELA. The most common adverse reactions (≥10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia. DRUG INTERACTIONS COSELA is an inhibitor of OCT2, MATE1, and MATE-2K. Co-administration of COSELA may increase the concentration or net accumulation of OCT2, MATE1, and MATE-2K substrates in the kidney (e.g., dofetilide, dalfampridine, and cisplatin). To report suspected adverse reactions, contact Pharmacosmos Therapeutics at 1-800-790-4189 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . This information is not comprehensive. Please see the full Prescribing Information. About Pharmacosmos Therapeutics We are a family-owned company innovating in iron metabolism, blood disorders, and cell cycle biology. With 700+ employees globally, we prioritize quality, collaboration, and patient care to drive life-changing treatments. Forward Looking Statement This press release includes forward-looking statements, including statements regarding the potential mechanism and investigational use of trilaciclib. Such statements are based on current expectations and assumptions and involve risks and uncertainties that may cause actual results to differ materially. COSELA ® (trilaciclib) is approved only to decrease the incidence of chemotherapy-induced myelosuppression in adults receiving certain regimens for extensive-stage small cell lung cancer. The effects of trilaciclib on clonal hematopoiesis or therapy-related myeloid neoplasms have not been established, and COSELA is not approved for these indications. Cory Steinberg Pharmacosmos Therapeutic, Inc. cos@pharmacosmos.us Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Drug ApprovalASHClinical ResultClinical Study

12 Feb 2025

·www.prnewswire.com

Alkermes plc Reports Financial Results for the Fourth Quarter and Year Ended Dec. 31, 2024 and Provides Financial Expectations for 2025

— Total Revenues of $1.56 Billion in 2024; Net Sales of Proprietary Products Increased Approximately 18% Year-Over-Year — — GAAP Net Income from Continuing Operations of $372 Million and Diluted GAAP Earnings per Share from Continuing Operations of $2.20 for 2024 — — ALKS 2680 Phase 2 Studies in Narcolepsy Type 1 and Type 2 Ongoing With Data Expected in H2 2025 — DUBLIN, Feb. 12, 2025 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today reported financial results for the quarter and year ended Dec. 31, 2024 and provided financial expectations for 2025. "2024 marked the completion of a multi-year effort to transition the business into a highly profitable, pure-play neuroscience company. We enter 2025 with a diversified portfolio of proprietary commercial products generating substantial profitability and an advancing development pipeline that represents a significant value creation opportunity in one of the most exciting potential new therapeutic categories in neuroscience," said Richard Pops, Chief Executive Officer of Alkermes. "Looking ahead, we are well positioned to deliver on our financial goals and advance the development programs for our portfolio of orexin 2 receptor agonists. This year, we have clear objectives for our pipeline as we complete the phase 2 studies for ALKS 2680 in narcolepsy, with data expected in the second half of the year, and prepare to initiate the ALKS 2680 phase 2 study in idiopathic hypersomnia and advance ALKS 4510 and ALKS 7290 into planned phase 1 studies in disease areas beyond central disorders of hypersomnolence. Each of these initiatives is an important element of our strategy to unlock what we believe is a multi-billion-dollar market opportunity for this category." "2024 was Alkermes' strongest year of financial and operational performance to date. Financially, we generated more than $1 billion in revenue from our proprietary commercial product portfolio, delivered EBITDA from continuing operations of approximately $452 million, repurchased $200 million of the company's ordinary shares, retired approximately $290 million of debt and ended the year debt-free with approximately $825 million of cash and investments on the balance sheet. Operationally, we completed the sale of our manufacturing business in Ireland and made significant progress advancing our neuroscience development pipeline," said Blair Jackson, Chief Operating Officer of Alkermes. "We will continue to manage the business with a sharp focus on efficiency and profitability as we invest in the programs that we believe will drive the company's next phase of growth." Key Financial Highlights *As a result of the successful resolution of the arbitration with Janssen Pharmaceutica N.V., the twelve months ended December 31, 2023 included approximately $195.4 million of back royalties (and related interest) related to U.S. net sales of long-acting INVEGA® products that would ordinarily have been recognized in prior periods. Revenue Highlights LYBALVI Revenues for the fourth quarter were $77.0 million. Fourth quarter revenues and total prescriptions grew 37% and 30%, respectively, compared to the fourth quarter of 2023. During the quarter, the company recorded LYBALVI® revenue of approximately $4 million related to year-end inventory fluctuations. ARISTADAi Revenues for the fourth quarter were $96.6 million. Fourth quarter revenues grew 16% compared to the fourth quarter of 2023. During the quarter, the company recorded ARISTADA® revenue of approximately $9 million related to year-end inventory fluctuations and gross-to-net favorability, primarily driven by Medicaid utilization adjustments. VIVITROL Revenues for the fourth quarter were $134.1 million. Fourth quarter revenues grew 31% compared to the fourth quarter of 2023. During the quarter, the company recorded VIVITROL® revenue of approximately $23 million related to year-end inventory fluctuations and gross-to-net favorability, primarily driven by Medicaid utilization adjustments. Manufacturing & Royalty Revenues Royalty revenues from XEPLION®, INVEGA TRINZA®/TREVICTA® and INVEGA HAFYERA®/BYANNLI® for the fourth quarter were $36.5 million. VUMERITY® manufacturing and royalty revenues for the fourth quarter were $35.0 million. FAMPYRA® manufacturing and royalty revenues for the fourth quarter were $22.9 million. The company does not expect to record any FAMPYRA revenue going forward. RISPERDAL CONSTA® manufacturing revenues for the fourth quarter were $14.7 million. Key Operating Expenses Please see Note 1 below for details regarding discontinued operations. Balance Sheet At Dec. 31, 2024, the company recorded cash, cash equivalents and total investments of $824.8 million, compared to $813.4 million at Dec. 31, 2023. In December 2024, the company prepaid and retired in full all of its outstanding long-term debt in the amount of approximately $290 million. Financial Expectations for 2025 All line items are according to GAAP, except as otherwise noted. Notes and Explanations 1. The company determined that upon the separation of its former oncology business, completed on Nov. 15, 2023, the oncology business met the criteria for discontinued operations in accordance with Financial Accounting Standards Board Accounting Standards Codification 205, Discontinued Operations. Accordingly, the accompanying selected financial information has been updated to present the results of the oncology business as discontinued operations for the three and twelve months ended Dec. 31, 2023. Conference Call Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. EST (1:00 p.m. GMT) on Wednesday, Feb. 12, 2025, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes' website at . The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes' website. About Alkermes plc Alkermes plc is a global biopharmaceutical company that seeks to develop innovative medicines in the field of neuroscience. The company has a portfolio of proprietary commercial products for the treatment of alcohol dependence, opioid dependence, schizophrenia and bipolar I disorder, and a pipeline of clinical and preclinical candidates in development for neurological disorders, including narcolepsy and idiopathic hypersomnia. Headquartered in Ireland, Alkermes also has a corporate office and research and development center in Massachusetts and a manufacturing facility in Ohio. For more information, please visit Alkermes' website at . Non-GAAP Financial Measures This press release includes information about certain financial measures that are not prepared in accordance with generally accepted accounting principles in the U.S. (GAAP), including non-GAAP net income, EBITDA and Adjusted EBITDA. These non-GAAP measures are not based on any standardized methodology prescribed by GAAP and are not necessarily comparable to similar measures presented by other companies. Non-GAAP net income adjusts for certain one-time and non-cash charges by excluding from GAAP results: share-based compensation expense; amortization; depreciation; non-cash net interest expense; change in the fair value of contingent consideration; certain other one-time or non-cash items; and the income tax effect of these reconciling items. EBITDA represents earnings before interest, tax, depreciation and amortization. Adjusted EBITDA excludes share-based compensation expense in addition to the components of EBITDA from earnings. The company's management and board of directors utilize these non-GAAP financial measures to evaluate the company's performance. The company provides these non-GAAP financial measures of the company's performance to investors because management believes that these non-GAAP financial measures, when viewed with the company's results under GAAP and the accompanying reconciliations, are useful in identifying underlying trends in ongoing operations. However, non-GAAP net income, EBITDA and Adjusted EBITDA are not measures of financial performance under GAAP and, accordingly, should not be considered as alternatives to GAAP measures as indicators of operating performance. Further, non-GAAP net income, EBITDA and Adjusted EBITDA should not be considered measures of the company's liquidity. A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. Note Regarding Forward-Looking Statements Certain statements set forth in this press release constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the company's expectations concerning its future financial and operating performance, business plans or prospects, including expected drivers of growth, value creation and profitability; and the company's expectations regarding development plans, activities and timelines for, and the potential therapeutic and commercial value of, ALKS 2680 and the company's other orexin portfolio candidates. The company cautions that forward-looking statements are inherently uncertain. The forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those expressed or implied in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: whether the company is able to achieve its financial expectations, including those related to profitability; clinical development activities may not be completed on time or at all; the results of the company's development activities may not be positive, or predictive of final results from such activities, results of future development activities or real-world results; the unfavorable outcome of arbitration, litigation, or other proceedings or disputes related to the company's products or products using the company's proprietary technologies; the U.S. Food and Drug Administration (FDA) or regulatory authorities outside the U.S. may make adverse decisions regarding the company's products; the company and its licensees may not be able to continue to successfully commercialize their products or support revenue growth from such products; there may be a reduction in payment rate or reimbursement for the company's products or an increase in the company's financial obligations to government payers; the company's products may prove difficult to manufacture, be precluded from commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; and those risks and uncertainties described under the heading "Risk Factors" in the company's most recent Annual Report on Form 10-K and in subsequent filings made by the company with the U.S. Securities and Exchange Commission (SEC), which are available on the SEC's website at . Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by law, the company disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release. VIVITROL® is a registered trademark of Alkermes, Inc.; ARISTADA®, ARISTADA INITIO® and LYBALVI® are registered trademarks of Alkermes Pharma Ireland Limited, used by Alkermes, Inc. under license; BYANNLI®, INVEGA®, INVEGA HAFYERA®, INVEGA TRINZA®, RISPERDAL CONSTA®, TREVICTA® and XEPLION® are registered trademarks of Johnson & Johnson or its affiliated companies; FAMPYRATM is a trademark of Merz Pharmaceuticals, LLC; and VUMERITY® is a registered trademark of Biogen MA Inc., used by Alkermes under license. Alkermes Contacts: For Investors: Sandy Coombs +1 781 609 6377 For Media: Katie Joyce +1 781 249 8927 SOURCE Alkermes plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financial StatementPhase 1Phase 2

100 Deals associated with Dalfampridine

External Link

KEGG	Wiki	ATC	Drug Bank
D04127	Dalfampridine	N07XX07	DB06637

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Multiple Sclerosis	United States	Merz Pharmaceuticals LLC	22 Jan 2010

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Ischemic stroke	Phase 3	United States	Acorda Therapeutics, Inc.	01 Dec 2014
Ischemic stroke	Phase 3	Canada	Acorda Therapeutics, Inc.	01 Dec 2014
Multiple Sclerosis, Chronic Progressive	Phase 3	Japan	Biogen, Inc.	01 Aug 2013
Multiple Sclerosis, Primary Progressive	Phase 3	Japan	Biogen, Inc.	01 Aug 2013
Multiple Sclerosis, Relapsing-Remitting	Phase 3	Japan	Biogen, Inc.	01 Aug 2013
Multiple Sclerosis, Secondary Progressive	Phase 3	Japan	Biogen, Inc.	01 Aug 2013
Episodic Ataxia	Phase 3	Germany	Biogen International GmbH	22 May 2013
Episodic Ataxia, Type 2	Phase 3	Germany	Biogen International GmbH	22 May 2013
Job Syndrome	Phase 3	Germany	Biogen International GmbH	22 May 2013
Syncope, Vasovagal	Phase 3	Germany	Biogen International GmbH	22 May 2013

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ECTRIMS2025	Not Applicable	Multiple Sclerosis, Secondary Progressive	335	Fampridine alone	tbxqyojred(mmqwfsksuy) = hgvfmxpshl hqlaefrsvh (aancqwmqae )	Positive	24 Sep 2025
				Siponimod alone	tbxqyojred(mmqwfsksuy) = eonzvwnmko hqlaefrsvh (aancqwmqae )
ECTRIMS2024	Not Applicable	Multiple Sclerosis	197	Fampridine	thnwpyhlks(hegcdvexvb) = Mt2 -11.6, DE= 2.4; Mt3 -11.3, DE=2.3; Mt4 -11.3, DE=2.3; Mt5 -11.4, DE=2.3, (p<0.001) aukfwqvbpi (rxbgwrttxp ) View more	Positive	18 Sep 2024
NCT01645787 (CTgov)	Phase 2/3	Spinal Muscular Atrophy \| Juvenile Spinal Muscular Atrophy	11	4-aminopyridine (Ampyra) (Short Term 4-aminopyridine (Ampyra))	rcqiwnmljb(zjrmlzfjtt) = jhpmrofgig lhskpzjmqi (bsjcphihei, aqtcsjsfdg - hnveuzhahr) View more	-	03 Sep 2024
				placebo+4-aminopyridine (Short Term Placebo)	rcqiwnmljb(zjrmlzfjtt) = uhptpgbaua lhskpzjmqi (bsjcphihei, qnbqpdxfdh - xfdulisdbr) View more
NCT05613114 (Pubmed \| J Clin Neurosci)	Not Applicable	Spastic Paraplegia, Hereditary	-	Dalfampridine 10 mg twice daily	husfnsdqpw(jgzuqsighw) = exuzqrbqgk ycelpvneff (siljkwtepw )	Positive	01 Nov 2023
				Placebo	husfnsdqpw(jgzuqsighw) = zspohjiiee ycelpvneff (siljkwtepw )
NCT03847545 (MUST, Pubmed \| Clin Biomech (Bristol))	Not Applicable	Multiple Sclerosis	14	Fampridine treatment	odkdmsntrj(uhvxnvynef) = qqgaqqubwf gbtmuosmrm (tosigauvke ) View more	-	17 Nov 2022
NCT04026568 (CTgov)	Phase 2/3	Nerve injury	1	4-Aminopyridine (Single Dose 4AP)	lasaadrtcf(xmobcgpyij) = lquujwioqs rmfzneajhw (mpnsihrebo, szaubfgadm - jurxekqrch) View more	-	14 Sep 2022
				Placebo oral tablet (Placebo)	vgphjfknqf = xdsbtmirbc mryokezsuj (qhrdwkwddd, ywaevcmhes - pujjgtblns) View more
NCT01480063 (LIBERATE, Pubmed \| CNS Drugs)	Not Applicable	Multiple Sclerosis	4,646	Prolonged-release fampridine (PR-FAM) 10-mg tablet	nxawqcmguy(juumpbceaa) = Seventeen (< 1%) patients experienced actual events of seizure zjenuhjoze (eknqsoafgb )	-	28 Jul 2021
NCT02208050 (CTgov)	Phase 4	Multiple Sclerosis, Primary Progressive \| Multiple Sclerosis, Secondary Progressive \| Multiple Sclerosis, Chronic Progressive	64	PR-Fampridine (PR-Fampridine)	errixacnst = vwgismfwkv raduyzkniy (jyoxucfvcp, fbpxmvmeio - cclmhkskru) View more	-	09 Jul 2021
				Placebo (Placebo)	errixacnst = rdprkzlkxn gnnuprzgun (yunctyjbkd, yykkqxjkxh - ovqtotcxad) View more
NCT00056810 (Pubmed) Manual	Phase 2	Guillain-Barre Syndrome	12	4-aminopyridine	wkpygbpakk(qqkwdyszux) = superior to placebo fgyfbfbcqh (voeytovmcw )	Positive	08 Apr 2021
NCT02271217 (Pubmed \| Restor Neurol Neurosci)	Phase 3	Ischemic stroke	377	Dalfampridine extended release 7.5mg	sfwzcmgpzi(bpfndpgogj) = secltnavjc qcznnfbgxn (xwowexfrlj )	Negative	24 Sep 2020
				Dalfampridine extended release 10mg	sfwzcmgpzi(bpfndpgogj) = dzxcfwlccp qcznnfbgxn (xwowexfrlj )

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