IMproving Symptomatic Treatment With Pyridostigmine and Amifampridine: a Randomized Double-blinded, Placebo Controlled Crossover Trial in Patients With Myasthenia Gravis (IMPACT-MG)

A randomized, double-blind, placebo controlled, crossover intervention study evaluating the effect of pyridostigmine (part 1) and amifampridine (part 2) in Myasthenia Gravis (MG).

Start Date22 Mar 2023

Sponsor / Collaborator

Academisch Ziekenhuis Leiden

[+1]

NCT05123053 / RecruitingPhase 2IIT

Amifampridine for the Treatment of Transient Vocal Weakness After OnabotulinumtoxinA Injection for Spasmodic Dysphonia

Botox injections into the thyroarytenoid muscle are a predictable and effective treatment for SD, but typically result in transient symptoms of voice weakness and breathiness during the first 2-3 weeks after injection. Investigators hypothesize that voice weakness and breathiness after Botox treatment can be alleviated using amifampridine.

Start Date28 Oct 2021

Sponsor / Collaborator

Augusta University Research Institute

EUCTR2018-002405-64-IT / Not yet recruitingPhase 3

Long term safety study of amifampridine phosphate in patients with Congenital Myasthenic Syndromes (CMS) - CMS-002

Start Date06 May 2019

Sponsor / Collaborator

Catalyst Pharmaceuticals, Inc.

100 Clinical Results associated with Amifampridine phosphate

100 Translational Medicine associated with Amifampridine phosphate

100 Patents (Medical) associated with Amifampridine phosphate

696

Literatures (Medical) associated with Amifampridine phosphate

02 Sep 2024·OPHTHALMIC GENETICS

Congenital Myasthenic Syndrome associated with acetylcholine receptor deficiency: case report and review of the literature

Review

Author: Bayer-Vile, Julie ; Batheja, Aashish ; Silverstein, Evan ; Couser, Natario

INTRODUCTION:

Congenital Myasthenic Syndromes are a diverse group of conditions with a broad array of genetic underpinnings and phenotypic presentations. Acetylcholine receptor deficiency is one form that usually involves pathogenic variants in the Cholinergic Receptor Nicotinic Epsilon Subunit (CHRNE) gene encoding the ɛ-subunit of the acetylcholine receptor.

METHODS:

We report a case of a 4-year-old male with suspected Congenital Myasthenic Syndrome with Acetylcholine Receptor Deficiency who presented with ocular symptoms and generalized muscle weakness. We additionally summarize published findings regarding the genetic, phenotypic, and clinical considerations of Congenital Myasthenic Syndrome with Acetylcholine Receptor Deficiency.

RESULTS:

Exome sequencing revealed biallelic variants in CHRNE gene with a pathogenic frameshift variant and a variant of uncertain significance. After suboptimal response to pyridostigmine and albuterol, the patient experienced benefit with 3,4-DAP. The most commonly reported clinical characteristics in the literature are ptosis, muscle fatigability or weakness, and ophthalmoplegia.

CONCLUSION:

We present the case of a patient with biallelic variants in CHRNE gene including a variant of uncertain significance. Evaluation of variants of this gene, including the variant of uncertain significance identified in this case report, through further cases and studies may improve our understanding of Congenital Myasthenic Syndrome with Acetylcholine Receptor deficiency.

01 Aug 2024·PEDIATRIC NEUROLOGY

Advancing the Understanding of Vesicle-Associated Membrane Protein 1-Related Congenital Myasthenic Syndrome: Phenotypic Insights, Favorable Response to 3,4-Diaminopyridine, and Clinical Characterization of Five New Cases

Article

Author: Lochmüller, Hanns ; Martorell, Loreto ; Afonso Ribeiro, Joana ; Estévez-Arias, Berta ; Todorova, Albena ; Pugliese, Alessia ; Shen, Xinming ; Ortez, Carlos ; Matalonga, Leslie ; Fernández-Mayoralas, Daniel M ; Tournev, Ivailo ; Polavarapu, Kiran ; Lau, Jarred ; Natera-de Benito, Daniel ; Guergueltcheva, Velina ; Thompson, Rachel ; Engel, Andrew G ; Laurie, Steven ; Jou, Cristina ; Selcen, Duygu ; Nascimento, Andres

BACKGROUND:

Congenital myasthenic syndromes (CMS) are a group of inherited neuromuscular junction (NMJ) disorders arising from gene variants encoding diverse NMJ proteins. Recently, the VAMP1 gene, responsible for encoding the vesicle-associated membrane protein 1 (VAMP1), has been associated with CMS.

METHODS:

This study presents a characterization of five new individuals with VAMP1-related CMS, providing insights into the phenotype.

RESULTS:

The individuals with VAMP1-related CMS exhibited early disease onset, presenting symptoms prenatally or during the neonatal period, alongside severe respiratory involvement and feeding difficulties. Generalized weakness at birth was a common feature, and none of the individuals achieved independent walking ability. Notably, all cases exhibited scoliosis. The clinical course remained stable, without typical exacerbations seen in other CMS types. The response to anticholinesterase inhibitors and salbutamol was only partial, but the addition of 3,4-diaminopyridine (3,4-DAP) led to significant and substantial improvements, suggesting therapeutic benefits of 3,4-DAP for managing VAMP1-related CMS symptoms. Noteworthy is the identification of the VAMP1 (NM_014231.5): c.340delA; p.Ile114SerfsTer72 as a founder variant in the Iberian Peninsula and Latin America.

CONCLUSIONS:

This study contributes valuable insights into VAMP1-related CMS, emphasizing their early onset, arthrogryposis, facial and generalized weakness, respiratory involvement, and feeding difficulties. Furthermore, the potential efficacy of 3,4-DAP as a useful therapeutic option warrants further exploration. The findings have implications for clinical management and genetic counseling in affected individuals. Additional research is necessary to elucidate the long-term outcomes of VAMP1-related CMS.

24 Jul 2024·ACS Applied Materials & Interfaces

Triphasic Oxygen Storage in Wet Nanoparticulate Polymer of Intrinsic Microporosity (PIM-1) on Platinum: An Electrochemical Investigation

Article

Author: Marken, Frank ; Li, Zhongkai ; Azevedo Beluomini, Maisa ; Boldrin Zanoni, Maria Valnice ; McKeown, Neil B. ; Ramos Stradiotto, Nelson ; Sain, Sunanda ; Carta, Mariolino ; Fletcher, Philip J.

The triphasic interaction of gases with electrode surfaces immersed in aqueous electrolyte is crucial in electrochemical technologies (fuel cells, batteries, sensors). Some microporous materials modify this interaction locally via triphasic storage capacity for gases in aqueous environments linked to changes in apparent oxygen concentration and diffusivity (as well as activity and reactivity). Here, a nanoparticulate polymer of intrinsic microporosity (PIM-1) in aqueous electrolyte is shown to store oxygen gas and thereby enhance electrochemical signals for oxygen reduction in aqueous media. Oxygen reduction current transient data at platinum disk electrodes suggest that the reactivity of ambient oxygen in aqueous electrolyte (typically D_oxygen = 2.8 × 10^-9 m² s^-1; c_oxygen = 0.3 mM) is substantially modified (to approximately D_app,oxygen = 1.6 (±0.3) × 10^-12 m² s^-1; c_app,oxygen = 50 (±5) mM) with important implications for triphasic electrode processes. The considerable apparent concentration of oxygen even for ambient oxygen levels is important. Potential applications in oxygen sensing, oxygen storage, oxygen catalysis, or applications associated with other types of gases are discussed.

119

News (Medical) associated with Amifampridine phosphate

12 Nov 2024

·www.globenewswire.com

Zenas BioPharma Reports Third Quarter 2024 Financial Results and Provides Corporate Updates

-Completed initial public offering, raising $258.7 million in gross proceeds, to fund the expanded clinical development of lead product candidate, obexelimab, and support company growth strategy- -Concluded targeted enrollment of Phase 3 INDIGO trial evaluating obexelimab for the treatment of Immunoglobulin G4-Related Disease (IgG4-RD)- -Advancing multiple obexelimab Phase 2 and Phase 3 clinical trials with results expected throughout 2025- WALTHAM, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zenas BioPharma, Inc. (“Zenas” or the “Company”) (Nasdaq: ZBIO), a clinical-stage global biopharmaceutical company committed to being a leader in the development and commercialization of transformative immunology-based therapies, today reported financial results for the third quarter ended September 30, 2024, and provided recent corporate updates. “In the third quarter, we successfully completed our initial public offering, providing capital to fund our growth strategy for the Company and expand the clinical development of our lead product candidate, obexelimab, for the treatment of autoimmune diseases,” said Lonnie Moulder, Founder and Chief Executive Officer of Zenas. “With the achievement of targeted enrollment for INDIGO, the largest IgG4-RD clinical trial ever conducted, we expect to report topline results for this registration-directed study by the end of 2025.” Recent corporate highlights Obexelimab Obexelimab is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. This unique mechanism of action and self-administered, subcutaneous injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. Completed targeted enrollment of the Phase 3 INDIGO trial, a global Phase 3 registration-directed, randomized, double-blind placebo-controlled trial of obexelimab in patients with IgG4-RD. With the completion of targeted enrollment for the largest clinical trial ever conducted in this patient population, the Company expects to report topline results for INDIGO by the end of 2025.Initiated the Phase 2 MoonStone trial, a Phase 2, multicenter, randomized, double-blind, placebo-controlled trial, to evaluate the efficacy and safety of obexelimab in patients with Relapsing Multiple Sclerosis (RMS). The Company expects to report the 12-week primary endpoint results for MoonStone by the third quarter of 2025.Initiated the Phase 2 SunStone trial, a Phase 2, multicenter, randomized, double-blind, placebo-controlled trial, to evaluate the efficacy and safety of obexelimab to reduce disease activity in patients with Systemic Lupus Erythematosus (SLE). SunStone remains on track to complete enrollment in 2025 and the Company expects to report topline results for SunStone in the first half of 2026.Continued enrollment in the Phase 2 SApHiAre trial, a global, multicenter, open-label safety and dose confirmation run-in period (SRP) to evaluate the safety and activity of obexelimab in patients with warm Autoimmune Hemolytic Anemia (wAIHA). The Company expects to provide initial data for SApHiAre later this year. Other corporate highlights Beyond progress with obexelimab, during the third quarter and more recently, the Company: Completed its upsized initial public offering, raising approximately $258.7 million in gross proceeds, including full exercise of the underwriters’ option to purchase additional shares, to fund its planned activities for obexelimab and the Company’s growth strategy.Bolstered its leadership team with the appointments of Chief Commercial Officer, Orlando Oliveira, and Chief Legal Officer, Jeff Held. With a career spanning nearly 25 years, Mr. Oliveira brings a wealth of experience building high-performing global commercial teams, driving revenue growth and fostering strategic partnerships within the biopharmaceutical industry, having served as head of international and commercial roles at Mirati Therapeutics (acquired by Bristol-Myers Squibb), Agios Pharmaceuticals (oncology business acquired by Servier) and at TESARO (acquired by GlaxoSmithKline). Mr. Held leads the Legal and Compliance function at Zenas and has over 30 years of legal experience at several publicly traded life sciences and private technology companies, including most recently as General Counsel at Deciphera Pharmaceuticals, where he built the legal and compliance function as Deciphera progressed from research through clinical development and commercial launches, along with executing on multiple equity financings and the sale of the company to ONO Pharmaceutical in June 2024.Out-licensed ZB005, a human IgG4 monoclonal antibody designed to bind only to the active form of C1s. Zenas held the development and commercialization rights for ZB005 in China, Hong Kong, Macau and Taiwan through an exclusive license with Dianthus. In October 2024, Zenas BioPharma (HK) Limited, a wholly owned subsidiary of Zenas established in Hong Kong, entered into an agreement with a private, China-based biotechnology company, under which Zenas HK transferred its rights and obligations to ZB005 under the Company’s agreement with Dianthus to such party for a non-refundable upfront payment in addition to potential regulatory and commercial milestone payments. Third quarter 2024 financial results for the quarter ended September 30, 2024 Research and development (R&D) expenses were $33.5 million for the quarter ended September 30, 2024, compared to $9.4 million for the quarter ended September 30, 2023. The increase in R&D expenses primarily relates to increased costs for the clinical development and manufacturing of obexelimab.General and administrative (G&A) expenses totaled $7.5 million for the quarter ended September 30, 2024, compared to $5.0 million for the quarter ended September 30, 2023. The increase in G&A cost was primarily due to increases in personnel and operations costs, as well as costs incurred to satisfy the requirements of becoming and operating as a public company.Net loss was $38.6 million for the quarter ended September 30, 2024, compared to net income of $35.6 million for the quarter ended September 30, 2023. The net income for the quarter ended September 30, 2023, is the result of collaboration revenue received from an upfront payment from Bristol-Myers Squibb Company when Zenas granted BMS an exclusive license to develop, manufacture, and commercialize obexelimab in Japan, South Korea, Taiwan, Singapore, Hong Kong and Australia.As of September 30, 2024, the Company’s cash balance, including cash, cash equivalents and short-term investments was $386.8 million, which includes the net proceeds of $234.4 million from the Company’s IPO, after deducting underwriting discounts and estimated offering expenses payable by the Company. The Company expects that its cash, cash equivalents, and short-term investments, as of September 30, 2024, will fund its operating expenses and capital expenditure requirements into the fourth quarter of 2026. About Zenas BioPharma, Inc. Zenas is a clinical-stage global biopharmaceutical company committed to becoming a leader in the development and commercialization of transformative immunology-based therapies for patients in need. Our core business strategy combines our experienced leadership team with a disciplined product candidate acquisition approach to identify, acquire and develop product candidates globally that we believe can provide superior clinical benefits to patients living with autoimmune diseases. Zenas’ lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. We believe that obexelimab’s unique mechanism of action and self-administered, subcutaneous injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. For more information about Zenas BioPharma, please visit www.zenasbio.com and follow us on LinkedIn. Forward looking statements This press release contains “forward-looking statements” which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning Zenas’s plans, objectives, expectations and intentions; the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting INDIGO trial topline results, the 12-week primary endpoint data for the MoonStone trial, completing enrollment and reporting topline results for the SunStone trial and reporting initial SApHiAre trial data; its growth strategy; and cash balance guidance. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies, many of which already have approved therapies in the Company’s current indications; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers, many of which are located outside of the United States, including the Company’s current sole contract manufacturing organization for drug substance and drug product, WuXi Biologics (Hong Kong) Limited, which is located in China; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other information we file with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. The Zenas BioPharma word mark and logos are trademarks of Zenas BioPharma, Inc. or its affiliated companies. Zenas BioPharma, Inc. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands except share and per share amounts) Unaudited Three Months Ended September 30, 2024 2023 Revenue: Collaboration revenue $— $50,000 Total revenue — 50,000 Operating expenses: Research and development 33,530 9,352 General and administrative 7,454 5,024 Total operating expenses 40,984 14,376 (Loss) income from operations (40,984) 35,624 Other income (expense), net: Other income (expense), net 2,378 (16) Total other income (expense), net 2,378 (16) Net (loss) income $(38,606) $35,608 Net (loss) income per share attributable to common stockholders - basic $(5.02) $2.42 Net (loss) income per share attributable to common stockholders - diluted $(5.02) $1.96 Weighted-average common stock outstanding - basic 7,697,695 1,537,918 Weighted-average common stock outstanding - diluted 7,697,695 1,898,391 Zenas BioPharma, Inc.SELECTED CONSOLIDATED BALANCE SHEET DATA(in thousands)Unaudited September 30, 2024Cash, cash equivalents and short-term investments $386,799 Working capital 349,781 Total assets 403,432 Accumulated deficit (334,786)Total stockholders’ equity 359,435 Investor Contact: Matthew OsborneInvestor Relations and Corporate CommunicationsMatt.osborne@zenasbio.com Media Contact: Argot PartnersZenas@argotpartners.com

Phase 2Phase 3Executive ChangeLicense out/inFinancial Statement

12 Nov 2024

·www.globenewswire.com

Zenas BioPharma Completes Targeted Enrollment of the Phase 3 INDIGO Trial of Obexelimab in Immunoglobulin G4-Related Disease (IgG4-RD)

-INDIGO is the largest clinical trial conducted in patients living with IgG4-RD- -Topline INDIGO results expected by the end of 2025- WALTHAM, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zenas BioPharma, Inc. (“Zenas” or the “Company”) (Nasdaq: ZBIO), a clinical-stage global biopharmaceutical company committed to being a leader in the development and commercialization of transformative immunology-based therapies, today announced the completion of its targeted enrollment for the Phase 3 INDIGO trial of its lead product candidate, obexelimab, for the treatment of patients with IgG4-RD. “With the completion of targeted enrollment in our INDIGO trial, the largest IgG4-RD clinical trial ever conducted, Zenas has demonstrated its capabilities to execute on our expansive clinical development plans for obexelimab,” said Lonnie Moulder, Founder and Chief Executive Officer of Zenas. “Importantly, we expect to report topline results by the end of next year and advance the Company toward our goal of becoming a leader in the development and commercialization of transformative immunology-based therapies for patients in need.” About the Phase 3 INDIGO Trial in IgG4-RD The Phase 3 INDIGO trial is a global, registration-directed, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of obexelimab in approximately 190 patients with active IgG4-RD and is being conducted at approximately 100 sites in 20 countries. Following an initial screening period, patients were randomized 1:1 to 250 mg of obexelimab or placebo administered as a subcutaneous injection every seven days for 52 weeks, followed by an opportunity for eligible patients to continue in an open-label extension period where all patients will receive treatment with obexelimab. The primary efficacy endpoint of INDIGO is the time to first IgG4-RD flare, as determined per protocol by the investigator and the adjudication committee. Secondary endpoints include annualized flare rate, the proportion of patients achieving complete remission, and use and quantity of rescue medication. More information on the INDIGO trial (NCT05662241) is available at clinicaltrials.gov About Obexelimab Obexelimab is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. This unique mechanism of action and self-administered, subcutaneous injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. Obexelimab has been evaluated in five completed clinical trials in a total of 198 patients who received obexelimab either as an intravenous infusion or as a subcutaneous injection. Obexelimab was well tolerated and demonstrated clinical activity across these five clinical trials, providing the Company an initial clinical proof of concept for obexelimab as a B cell inhibitor for the treatment of patients living with certain autoimmune diseases. Currently, Zenas is conducting multiple Phase 2 and Phase 3 trials of obexelimab in several autoimmune diseases including IgG4-RD, Multiple Sclerosis, Systemic Lupus Erythematosus, and warm Autoimmune Hemolytic Anemia. About IgG4-RD IgG4-RD is a chronic fibro-inflammatory disease that can affect virtually all organ systems, including the pancreas, biliary tract, salivary and lacrimal glands, lungs, and kidneys. Patients with IgG4-RD may present with a single organ involved but more frequently present with multiple organ involvement. As the disease progresses and patients experience new or worsening symptoms (i.e., flares), lesions develop in additional organs and the cellular inflammation characterizing early disease moves toward a more fibrotic stage, which can lead to major irreversible tissue damage and ultimately organ failure. We estimate that the currently diagnosed population of IgG4-RD patients in the U.S. is approximately 20,000, with comparable prevalence rates globally. Despite the growing recognition of IgG4-RD and advances in the understanding of its pathophysiology, there are no approved therapies for the treatment of this disease and there remains high unmet medical need. The current standard of care is treatment with glucocorticoids (GCs). Although GCs are initially effective, treatment with GCs can often result in various complications and co-morbidities. Most patients can relapse within 12 months of discontinuing GC treatment, and maintenance therapy with GCs has not been shown to prevent recurrence of disease. The pathogenesis of IgG4-RD suggests that B cell-targeted therapies may provide therapeutic benefit. Although not approved by any regulatory authorities to treat IgG4-RD, certain B cell depleting agents (e.g. rituximab) are occasionally administered to patients with IgG4-RD. However, B cell depleting agents are often associated with infections, including serious opportunistic infections, and can compromise a patient’s ability to mount a response to vaccinations. About Zenas BioPharma, Inc. Zenas is a clinical-stage global biopharmaceutical company committed to becoming a leader in the development and commercialization of transformative immunology-based therapies for patients in need. Our core business strategy combines our experienced leadership team with a disciplined product candidate acquisition approach to identify, acquire and develop product candidates globally that we believe can provide superior clinical benefits to patients living with autoimmune diseases. Zenas’ lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. We believe that obexelimab’s unique mechanism of action and self-administered, subcutaneous injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. For more information about Zenas BioPharma, please visit www.zenasbio.com and follow us on LinkedIn. Forward looking statements This press release contains “forward-looking statements” which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning Zenas’s plans, objectives, expectations and intentions; and the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting INDIGO trial topline results. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies, many of which already have approved therapies in the Company’s current indications; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers, many of which are located outside of the United States, including the Company’s current sole contract manufacturing organization for drug substance and drug product, WuXi Biologics (Hong Kong) Limited, which is located in China; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other information we file with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. The Zenas BioPharma word mark and logos are trademarks of Zenas BioPharma, Inc. or its affiliated companies. Investor Contact: Matthew Osborne Investor Relations and Corporate CommunicationsMatt.osborne@zenasbio.com Media Contact: Argot PartnersZenas@argotpartners.com

Phase 3ImmunotherapyClinical ResultPhase 2

07 Nov 2024

·www.globenewswire.com

Zenas BioPharma to Participate in Upcoming Healthcare Investor Conferences

WALTHAM, Mass, Nov. 07, 2024 (GLOBE NEWSWIRE) -- Zenas BioPharma, Inc. (“Zenas” or the “Company”) (Nasdaq: ZBIO), a clinical-stage global biopharmaceutical company committed to being a leader in the development and commercialization of transformative immunology-based therapies, today announced the Company’s participation at the following healthcare investor conferences: Guggenheim’s Inaugural Healthcare Innovation Conference on November 12, 2024, in Boston, MAJefferies London Healthcare Conference on November 19, 2024 presentation at 4:00 p.m. to 4:25 p.m. GDT, in LondonCiti’s 2024 Global Healthcare Conference on December 3, 2024 presentation at 9:30 a.m. to 10:10 a.m. ET, in Miami, FLEvercore ISI HealthCONx Conference on December 4, 2024 presentation at 1:20 p.m. to 1:40 p.m. ET, in Coral Gables, FL Live webcasts and archived replays of the Company’s presentations at the Jefferies, Citi and Evercore conferences can be accessed under “Events and Presentations” in the Investors and Media section of the Zenas BioPharma website. About Zenas BioPharma, Inc. Zenas is a clinical-stage global biopharmaceutical company committed to becoming a leader in the development and commercialization of transformative immunology-based therapies for patients in need. Our core business strategy combines our experienced leadership team with a disciplined product candidate acquisition approach to identify, acquire and develop product candidates globally that we believe can provide superior clinical benefits to patients living with autoimmune diseases. Zenas’ lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. We believe that obexelimab’s mechanism of action and chronic dosing regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. For more information about Zenas BioPharma, please visit www.zenasbio.com and follow us on X at @ZenasBioPharma and LinkedIn. The Zenas BioPharma word mark and logos are trademarks of Zenas BioPharma, Inc. or its affiliated companies. Investor Contact:Matthew OsborneInvestor Relations and Corporate CommunicationsMatt.osborne@zenasbio.com Media Contact:Argot PartnersZenas@argotpartners.com

Acquisition

100 Deals associated with Amifampridine phosphate

External Link

KEGG	Wiki	ATC	Drug Bank
D10689	Amifampridine phosphate	N07XX05	DB11640

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Lambert-Eaton Myasthenic Syndrome	EU	SERB SA	23 Dec 2009
Lambert-Eaton Myasthenic Syndrome	IS	SERB SA	23 Dec 2009
Lambert-Eaton Myasthenic Syndrome	LI	SERB SA	23 Dec 2009
Lambert-Eaton Myasthenic Syndrome	NO	SERB SA	23 Dec 2009

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Myasthenia Gravis	Phase 3	US	Catalyst Pharmaceuticals, Inc.	18 Apr 2018
Myasthenic Syndromes, Congenital	Phase 3	US	Catalyst Pharmaceuticals, Inc.	01 Jan 2016
Juvenile Spinal Muscular Atrophy	Phase 2	IT	Catalyst Pharmaceuticals, Inc.	21 Jan 2019
Bulbo-Spinal Atrophy, X-Linked	Preclinical	US	Catalyst Pharmaceuticals, Inc.	-
Multiple Sclerosis	Discovery	US	BioMarin Pharmaceutical, Inc.	-

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03819660 (SMA3, CTgov)	Phase 2	Juvenile Spinal Muscular Atrophy	13	Amifampridine Phosphate 10 MG Oral Tablet	iyrlxpocdp(zjtsoykvhe) = gezlgagwnq mhitmryeiu (ecsctaooyo, zhipifopwj - yaeckhowvx) View more	-	30 Nov 2023
NCT03304054 (CTgov)	Phase 3	Myasthenia Gravis	93	Amifampridine Phosphate (Amifampridine Phosphate - MuSK)	facwellohf(jlscrdpfvx) = jtibwuyfwm invuobsblp (xrctptqzuu, zgqprnigsx - bqeztjugxl) View more	-	13 Aug 2021
				Placebo+amifampridine phosphate (Placebo - MuSK)	facwellohf(jlscrdpfvx) = bmhgfylctn invuobsblp (xrctptqzuu, bypsofwxua - jyuhjvjvba) View more
NCT02090725 (3-4DAP, CTgov)	Phase 2	Lambert-Eaton Myasthenic Syndrome \| Muscle Weakness	4	3-4 Diaminopyridine	zarbmehvph(jgdsrmpmot) = xnaaqpnwit ynoqzkaszd (trxtpsiqhx, vszrotbbni - xrrzewqfgw) View more	-	21 May 2019
NCT02970162 (Pubmed) Manual	Phase 3	Lambert-Eaton Myasthenic Syndrome	26	Amifampridine	uhsyiqhdip(uhclrssnki) = Other measures of efficacy, including Clinical Global Impression-Improvement, 3TUG, and QMG limb domain score also improved. edvkujlmlf (cydbbrtdnx ) View more	Positive	01 Mar 2019
				Placebo
NCT02970162 (CTgov)	Phase 3	Lambert-Eaton Myasthenic Syndrome	26	Amifampridine Phosphate (Amifampridine Phosphate)	rlibwmnfdd(cvsnziilla) = vlmunostvj llfhnzxdro (cbapcfhqfd, phcfwtfclf - wzotcpzwzg) View more	-	24 Dec 2018
				Placebo Oral Tablet (Placebo (for Amifampridine Phosphate))	rlibwmnfdd(cvsnziilla) = qolghziyfh llfhnzxdro (cbapcfhqfd, zzlnmlxyvy - ehjbxiulvz) View more
P6.452 (AAN2018)	Not Applicable	Myasthenic Syndromes, Congenital	2	3,4 DAP	lanpvdyjkv(uvtuqlgkga) = xoqijzvryp vsrcaumipj (vjwgjnjiak )	Positive	10 Apr 2018
NCT01377922 (CTgov)	Phase 3	Lambert-Eaton Myasthenic Syndrome	38	Placebo (Placebo)	xdwiggykyb(lnnbookrjv) = sfgytmoyaa vinbxbqgjx (qwokivakxa, iurufdpswe - ciustxrqrq) View more	-	04 Jan 2018
				Amifampridine Phosphate (Amifampridine Phosphate)	xdwiggykyb(lnnbookrjv) = shupppylcx vinbxbqgjx (qwokivakxa, fypvxewnov - ftxfgqzpej) View more

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