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

107

Clinical Trials associated with Dalfampridine

NCT04516603

/ WithdrawnPhase 2

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]

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

NCT07171203

/ RecruitingPhase 1IIT

Phase I Study of Neoadjuvant Therapy Imatinib Mesylate and Fampridine in KIT Mutant Gastrointestinal Stromal Tumor (GIST)

The goal of this clinical trial is to learn what dose of the drug fampridine can be given safely together with imatinib (Gleevec) in patients with gastrointestinal stromal tumor (GIST) with a DNA mutation in exon 11 of the KIT gene.
The main questions this study aims to answer are:
* What is the maximum dose of fampridine that can be given safely together with imatinib (Gleevec)?
* Is the combination of the two drugs efficacious against the tumor?
Participants will:
* Take the drugs before tumor surgery (neoadjuvant treatment) for at least 2 months with the option to continue for a longer period of time if treatment seems safe and effective.
* Visit the clinic at the scheduled appointments for checkups and tests.

Start Date01 Jun 2026

Sponsor / Collaborator

University of California San Diego

100 Clinical Results associated with Dalfampridine

100 Translational Medicine associated with Dalfampridine

100 Patents (Medical) associated with Dalfampridine

10,193

Literatures (Medical) associated with Dalfampridine

01 May 2026·TALANTA

Toward greener electrochemical sensors: Comparative study of biochar and carbon black as carbon-based materials in screen-printed electrodes

Article

Author: Purdeková, Mária ; Kováč, Miroslav ; Gemeiner, Pavol ; Švorc, Ľubomír ; Sarakhman, Olha ; Barek, Jiří ; Hatala, Michal

This study presents, for the first time, a comparative investigation of carbon black (CB) and biochar (BIO) derived from digestate composed of wood and corn silage as carbon-based materials for fabricating disposable screen-printed electrochemical sensors (SPEs). Using a bulk mixing strategy, five SPE variants with different BIO:CB weight ratios (0:100, 25:75, 50:50, 75:25, and 100:0) were prepared and characterized by Fourier-transform infrared spectroscopy (FTIR), scanning electron microscopy coupled with energy-dispersive X-ray spectroscopy (SEM/EDX), Brunauer-Emmett-Teller (BET) analysis, and cyclic voltammetry (CV). The analytical performance of the sensors was evaluated for the determination of acetaminophen (AC) and its toxic metabolite 4-aminophenol (4-AP) in 0.1 mol L^-1 phosphate buffer (pH 7.4) using differential pulse voltammetry. The obtained sensitivities for AC were 0.088, 0.075, 0.077, and 0.007 μA/μmol L^-1, and for 4-AP were 0.075, 0.090, 0.060, and 0.047 μA/μmol L^-1, corresponding to the SPEs with the BIO:CB ratios of 0:100, 25:75, 50:50, and 75:25, respectively. The sensor with 100 % CB exhibited the highest sensitivity and the lowest limits of detection for both analytes and was selected for the simultaneous voltammetric determination of AC and 4-AP in pharmaceuticals, achieving recovery values between 90.0 % and 106.4 %. These findings provide a direct comparison between CB and BIO studied as electrode materials, showing that under the tested conditions CB delivers the highest analytical performance. Nevertheless, the limited incorporation of renewable BIO is feasible, allowing the electrode composition to be tailored to achieve the desired balance between analytical efficiency and sustainability, thereby supporting the further development of greener and cost-effective electrochemical sensing platforms.

01 Mar 2026·JOURNAL OF NEURO-OPHTHALMOLOGY

Clinicopathologic Correlates: Progressive Downbeat Nystagmus in Spinocerebellar Ataxia Type 27B

Article

Author: Hale, David E ; Gold, Daniel R ; Mu, Weiyi

Abstract::

A 61-year-old woman presented with episodic dizziness that met diagnostic criteria for vestibular migraine. Vestibular and ocular motor examination was normal, aside from a 1°/second downbeat nystagmus (DBN) that was only seen with removal of fixation (in the dark) using video oculography. Two years later, lithium was initiated for treatment of depression and visual bouncing and jumping developed even when her head was still. On repeat examination, she had clear spontaneous (20°/second) DBN even with fixation in room light. Lithium therapy was discontinued, and for days and weeks, there was subjective improvement in oscillopsia and objective lessening of her DBN. However, DBN remained significant and symptomatic after several months. A broad search for reversible nutritional, metabolic, and immune-mediated causes of DBN and cerebellar dysfunction was completed and was unremarkable. Therapy with 4-aminopyridine was initiated and she had a robust improvement in oscillopsia and DBN. During the following years, she developed mild gait ataxia, and genetic testing revealed GAA repeat expansions in 1 copy of the fibroblast growth factor 14 ( FGF14 ) gene, consistent with a diagnosis of spinocerebellar ataxia type 27B (SCA27B).

01 Mar 2026·Biologia Futura

Central administration of asprosin reduces baseline brain activity but does not attenuate 4-AP-induced epileptiform discharges: an in vivo electrophysiological study

Article

Author: Kalkan, Ömer Faruk ; Özyaşar, Ali Faruk ; Kurt, Abdullah Kaan ; Aktaş, Osman ; Abidin, İsmail ; Küçükosman, Samet Soner ; Aydin Abidin, Selcen ; Şahin, Zafer ; Yildirim, Abdulhamit ; Uzun, İbrahim

In the current study, we investigated the effects of central asprosin administration on baseline brain activity and 4-aminopyridine (4-AP) induced epileptiform activity. Additionally, we examined the effects of asprosin on electrocorticography (ECoG) band powers. Forty-two male Wistar rats were divided into six groups as follows: sham, 4-AP (2.5 mg/kg i.p.), asprosin 20 nmol (i.c.v.), asprosin 50 nmol (i.c.v.), asprosin 50 nmol (i.c.v.) posttreatment, and asprosin 50 nmol (i.c.v.) pre-treatment. Recordings lasting 60-70 min were conducted for all groups under ketamine/xylazine (90/10 mg/kg) anesthesia. In the posttreatment group, asprosin was injected 20 min after the induction of epileptiform activity. In the pre-treatment group, asprosin was injected after baseline recordings, and following a 20-min pre-treatment period, 4-AP was administered. 4-AP alone induced epileptiform activity in all animals, peaking at approximately the 30th minute. Asprosin significantly reduced ECoG power at doses of 20 nmol and 50 nmol. Furthermore, both doses of asprosin reduced alpha, beta, delta, theta, and gamma band activity in ECoG recordings at various time points. However, asprosin pre- and posttreatment had no significant effect on 4-AP-induced epileptiform activity. These findings suggest that asprosin modulates cortical excitability under physiological conditions but is ineffective in attenuating induced epileptiform activity.

News (Medical) associated with Dalfampridine

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

11 Jul 2024

·www.pharmaceutical-technology.com

Merz wins $185m bid for bankrupt Acorda assets and increases US workforce by 50%

Fampyra used to see sales of over $500m in 2017, but sales weakened after generics entered the market. Image credit: Shutterstock / Elena Abrazhevich. Merz Therapeutics has successfully acquired Acorda’s Inbrija (levodopa inhalation powder) and Ampyra (dalfampridine) after the latter went bankrupt earlier this year. The sale was via a “stalking horse” bid, meaning the assets could have gone for more than the $185m opening bid by Merz. These types of bids are used in cases involving bankrupt companies. No competing company joined the auction, however, and Merz closed the transaction at the opening bid value. Merz said the addition of Inbrija and Ampyra, the latter marketed as Fampyra (fampridine) outside of the US, would “immediately add topline revenue” and enhance the biotech’s ability to expedite clinical development of other assets, as per a 10 July press release. Merz’s lead product is Xeomin (incobotulinumtoxinA), used to treat excessive drooling, upper limb spasticity, and cervical dystonia, among others. Inbrija meanwhile is used to treat patients with Parkinson’s disease while Ampyra / Fampyra is approved to help improve walking ability in patients with multiple sclerosis. Merz CEO Stefan König said the deal “strengthens [the company’s] market position in Parkinson’s disease” while “expanding into the multiple sclerosis segment”. See Also: BARDA and Tiba Biotech link to develop ‘flu therapeutics Can a ‘super pill’ help shrink Big Pharma’s climate footprint? Merz also expects to increase its US workforce by more than 50% to support the newly added assets. Inbrija and Fampyra generated global sales of $31m and $73m, respectively, last year. According to GlobalData’s Pharma Intelligence Centre, Fampyra used to see sales of over $500m in 2017. But once it lost exclusivity in 2018, generics flooded the market, and sales weakened. GlobalData is the parent company of Pharmaceutical Technology . Merz forecasts its US business to contribute more than 75% of total global revenues for Inbrija and Fampyra over the next decade. Acorda used to be a big player in the neurodegenerative disorder space up until a few years ago. The formerly listed biotech at one point had $363m to spare when it decided to acquire Finnish biopharma Biotie Therapies , a developer of Parkinson’s disease therapies, in 2016. However, in subsequent years, Acorda struggled financially as sales of its drugs weakened. Biogen , which had a 2009 licence agreement for Fampyra outside the US, returned the rights to Acorda earlier this year. Shares in the company continued to dive and Acorda ultimately filed for bankruptcy, announced via a 1 April press release. In addition, Acorda was delisted from the Nasdaq Stock Market in mid-April, and closed its doors for the final time last month, leaving 97 employees at its New York site out of work.

AcquisitionLicense out/inDrug Approval

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


NCT01645787 (CTgov)	Phase 2/3	Spinal Muscular Atrophy \| Juvenile Spinal Muscular Atrophy	11	4-aminopyridine (Ampyra) (Short Term 4-aminopyridine (Ampyra))	hdbkjoambq(uwtzzwbgrh) = byswzxvkbq nhbwppbiye (ctvvqpftqb, ocmutqvdfw - pbumktidqn) View more	-	03 Sep 2024
				placebo+4-aminopyridine (Short Term Placebo)	hdbkjoambq(uwtzzwbgrh) = iqazajcxkp nhbwppbiye (ctvvqpftqb, qypkerafva - bpcnwsnebk) View more
NCT05613114 (Pubmed \| J Clin Neurosci)	Not Applicable	Spastic Paraplegia, Hereditary	-	Dalfampridine 10 mg twice daily	ysstkfzxol(bsooykylwn) = fsifjrpvot bmfkwziers (ykjivupfww )	Positive	01 Nov 2023
				Placebo	ysstkfzxol(bsooykylwn) = xwdklakxva bmfkwziers (ykjivupfww )
NCT03847545 (MUST, Pubmed \| Clin Biomech (Bristol))	Not Applicable	Multiple Sclerosis	14	Fampridine treatment	khcaudtzmj(qvdzcotvry) = cxjnjwsynz tmnhothfxh (uyzelwpkse ) View more	-	17 Nov 2022
NCT04026568 (CTgov)	Phase 2/3	Nerve injury	1	4-Aminopyridine (Single Dose 4AP)	qbouazcwwx(qyufsakiru) = fcsevnbege tajkhyinze (uyveaqvcck, wchorlihdz - vtavhwqbxq) View more	-	14 Sep 2022
				Placebo oral tablet (Placebo)	sfxaqifdoi = ofhqwoxifm bpmlkcgamd (oswybyedkp, jmcqbcvfto - wcgptqkauu) View more
NCT01480063 (LIBERATE, Pubmed \| CNS Drugs)	Not Applicable	Multiple Sclerosis	4,646	Prolonged-release fampridine (PR-FAM) 10-mg tablet	vtmewyrqod(kwqbvbtdcn) = Seventeen (< 1%) patients experienced actual events of seizure wcexuiukaa (tpskvxdznv )	-	28 Jul 2021
NCT02208050 (CTgov)	Phase 4	Multiple Sclerosis, Primary Progressive \| Multiple Sclerosis, Secondary Progressive \| Multiple Sclerosis, Chronic Progressive	64	PR-Fampridine (PR-Fampridine)	pimoxhzizl = gwkhqcupwp lnktbwfqgx (qgiehpqaad, egwafoplwb - owpjansemj) View more	-	09 Jul 2021
				Placebo (Placebo)	pimoxhzizl = hqosksggzw hwupixifgc (psaymszhah, zmocyxtssn - hpulsybkmd) View more
NCT00056810 (Pubmed) Manual	Phase 2	Guillain-Barre Syndrome	12	4-aminopyridine	vgtarfctyx(bhgxaawyer) = superior to placebo zrpsxmvhol (uqsethfanx )	Positive	08 Apr 2021
NCT02271217 (Pubmed \| Restor Neurol Neurosci)	Phase 3	Ischemic stroke	377	Dalfampridine extended release 7.5mg	gnnqbouowb(outhzrcsgm) = nwtshwhvqs aebmieswzu (asbuwoytqq )	Negative	24 Sep 2020
				Dalfampridine extended release 10mg	gnnqbouowb(outhzrcsgm) = pqcurpzvjp aebmieswzu (asbuwoytqq )
NCT02391961 (CTgov)	Phase 2	Fatigue \| Multiple Sclerosis \| Muscle fatigue ... View more	23	Dalfampridine (Dalfampridine)	oszxbtbvvb(byccvjqpdf) = josjdzgcsd umkjfijqyx (hudlndetwt, 0.454) View more	-	04 Sep 2020
				Placebo (Placebo)	oszxbtbvvb(byccvjqpdf) = ejrmstctnj umkjfijqyx (hudlndetwt, 0.553) View more
NCT01480063 (LIBERATE, EAN2020)	Not Applicable	-	4,646	Prolonged-release fampridine (PR-FAM) 10mg tablet twice-daily	oynivnrlmb(asqrnmrmjh) = emoegkxlgd dxutlhmawg (hrhtjcqznr ) View more	Positive	22 May 2020

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