Amifampridine phosphate

- Topline results from pivotal Phase 3 INDIGO trial in Immunoglobulin G4-Related Disease expected around year-end 2025 - - Phase 2 MoonStone trial in Relapsing Multiple Sclerosis enrollment concluding; topline results expected early in the fourth quarter 2025 - - Enrollment of Phase 2 SunStone trial in Systemic Lupus Erythematosus expected to be completed by year-end 2025; topline results expected mid-2026 - - Strengthened leadership team with appointments of Lisa von Moltke, M.D., Head of Research and Development and Chief Medical Officer, and Haley Laken, Ph.D., Chief Scientific Officer - - Cash, cash equivalents and investments of $314.2 million as of March 31, 2025, expected to provide financial runway into the fourth quarter of 2026 - WALTHAM, Mass., May 15, 2025 (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 therapies for autoimmune diseases, today reported financial results for the first quarter ended March 31, 2025, and provided recent corporate updates. “We are pleased with the continued momentum of our obexelimab program across ongoing Phase 2 and Phase 3 clinical trials, for which we expect to report topline results later this year from trials in patients with relapsing multiple sclerosis and IgG4-RD,” said Lonnie Moulder, Founder and Chief Executive Officer of Zenas. “With a strengthened team following the recent additions of Lisa and Haley, we are well positioned to execute on our clinical trials and advance obexelimab as a differentiated B cell inhibitor with a potentially safer, more potent and convenient profile for patients.” Recent corporate highlights Obexelimab, a CD-19 x FcγRIIb inhibitor of B cell function Immunoglobulin G4-Related Disease (IgG4-RD): Advanced the Phase 3 INDIGO trial, a global registration-directed, multicenter, randomized, double-blind, placebo-controlled trial, to evaluate the efficacy and safety of obexelimab in patients with IgG4-RD. INDIGO is the largest clinical trial conducted in patients living with IgG4-RD to date. In the fourth quarter of 2024, Zenas completed the target enrollment of the INDIGO trial and expects to report topline results from the INDIGO trial around year-end 2025.Relapsing Multiple Sclerosis (RMS): Patient screening concluding with the final subject expected to be enrolled in early-June in the Phase 2 MoonStone trial, a multicenter, randomized, double-blind, placebo-controlled trial, to evaluate the efficacy and safety of obexelimab in patients with RMS. The Company expects to report topline results from this trial, including the 12-week primary endpoint results, early in the fourth quarter of 2025.Systemic Lupus Erythematosus (SLE): Continued enrolling in the Phase 2 SunStone trial, a multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of obexelimab in patients with SLE. Zenas expects to complete enrollment in this trial by year-end 2025 and report topline results in mid-2026. Other corporate highlights Beyond progress with obexelimab, during the first quarter and more recently, the Company: Strengthened its leadership team by appointing Lisa von Moltke, M.D., as Head of Research and Development and Chief Medical Officer, and Haley Laken, Ph.D., as Chief Scientific Officer. Dr. von Moltke brings over 30 years of U.S. and international drug development experience spanning multiple therapeutic areas, including autoimmune diseases, early- and late-stage clinical development, and commercialization at large and emerging growth companies. Dr. Laken brings over 25 years of leadership experience in research, development operations, research and development strategy and business development.Out-licensed regional rights to its thyroid eye disease program, including ZB001, an insulin-like growth factor-1 receptor (anti-IGF-1R) monoclonal antibody, to Zai Lab (Zai). Zenas received an upfront payment and is eligible to receive milestone payments and royalties in the future, as consideration for an exclusive sublicense to Zai to develop and commercialize ZB001 and related programs in Greater China. First quarter 2025 financial results As of March 31, 2025, the Company’s cash, cash equivalents and investments was $314.2 million. The Company expects that its cash, cash equivalents and investments, as of March 31, 2025, will fund its operating expenses and capital expenditure requirements into the fourth quarter of 2026.License and collaboration revenue was $10.0 million for the quarter ended March 31, 2025, related to the one-time non-refundable upfront cash payment received in connection with the sublicense agreement with Zai. The Company did not recognize any license and collaboration revenue during the quarter ended March 31, 2024.Research and development (R&D) expenses were $34.9 million for the quarter ended March 31, 2025, compared to $22.6 million for the quarter ended March 31, 2024. The increase of $12.3 million in R&D expenses primarily relates to an increase in costs related to the clinical development and manufacturing of obexelimab.General and administrative (G&A) expenses were $12.4 million for the quarter ended March 31, 2025, compared to $4.9 million for the quarter ended March 31, 2024. The increase of $7.5 million in G&A expenses was due to an increase in personnel costs, including stock-based compensation expenses, pre-commercialization activities including hiring and other expenses including costs associated with operating as a public company.Net loss was $33.6 million for the quarter ended March 31, 2025, compared to net loss of $27.8 million for the quarter ended March 31, 2024. About ObexelimabObexelimab 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 potent 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 Immunoglobulin G4-Related Disease, Relapsing Multiple Sclerosis and Systemic Lupus Erythematosus. About Zenas BioPharma, Inc.Zenas is a clinical-stage global biopharmaceutical company committed to becoming a leader in the development and commercialization of transformative therapies for patients with autoimmune diseases. 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 statementsThis 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, forward-looking statements can be identified 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 the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting INDIGO trial topline results, the last patient to be enrolled in and the 12-week primary endpoint data for the MoonStone trial and the anticipated timing of completing enrollment and reporting topline results for the SunStone trial; its growth strategy; and cash runway 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; significant political, trade, regulatory developments, including changes in relations between the U.S. and China; 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 March 31, 2025, as well as other information we file with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain, speak only as of the date of this press release and may prove incorrect. These statements are based upon information available to the Company as of the date of this press release and while the Company believes such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that the Company has conducted an exhaustive inquiry into, or review of, all potentially available relevant information. 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, these forward-looking statements should not be relied upon as guarantees of future events. 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, logo mark, and the “lightning bolt” design 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 March 31, 2025 2024 Revenue: License and collaboration revenue $10,000 $— Total revenue 10,000 — Operating expenses: Research and development 34,915 22,645 General and administrative 12,415 4,933 Total operating expenses 47,330 27,578 Loss from operations (37,330) (27,578)Other income (expense), net: Other income (expense), net 3,552 (222)Total other income (expense), net 3,552 (222)Income tax benefit 205 — Net loss to common stockholders $(33,573) $(27,800)Net loss per share attributable to common stockholders - basic and diluted $(0.80) $(17.89)Weighted-average common stock outstanding - basic and diluted 41,800,802 1,554,087 Zenas BioPharma, Inc.SELECTED CONSOLIDATED BALANCE SHEET DATA(in thousands)Unaudited March 31, 2025Cash, cash equivalents and investments $314,214 Working capital 268,675 Total assets 333,766 Accumulated deficit (420,964)Total stockholders’ equity 284,317 Investor and Media Contact:Argot PartnersZenas@argotpartners.com

Catalyst Enters 2025 With Strong Momentum; Reiterates Confidence in Full-Year Outlook Delivers Record Q1 2025 Total Revenues of $141.4 Million, a 43.6% YoY Increase FIRDAPSE® and AGAMREE® Drive Sustained Organic Growth, Underscoring Execution Strength Reaffirms Full-Year 2025 Total Revenue Guidance Provided Earlier This Year of Between $545 Million and $565 Million Strengthens Balance Sheet with Cash and Cash Equivalents of $580.7 Million and No Debt as of March 31, 2025 Conference Call and Webcast to be Held on May 8, 2025 , at 8:30 AM ET CORAL GABLES, Fla. , May 07, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals , Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), today reported financial results for the first quarter of 2025 and provided a business update. "Catalyst's exceptional first quarter performance underscores the continued strength of our commercial strategy and the growing demand for our differentiated therapies," said Richard J. Daly , President and CEO of Catalyst. "Our 43.6% year-over-year total revenue growth for the first quarter of 2025 is a testament to our focused execution and the increasing recognition of the value that our therapies deliver. Looking ahead, we see meaningful opportunities to further establish AGAMREE as a cornerstone therapy in the Duchenne muscular dystrophy (DMD) market and expand FIRDAPSE's clinical utility in Lambert-Eaton myasthenic syndrome (LEMS), addressing a significant unmet need for patients in both markets. We continue to evaluate strategically aligned opportunities to strengthen our portfolio and expand our reach to serve more patients. Building on our momentum, we are confident in our ability to execute our 2025 strategic priorities and drive long-term growth." Financial Highlights _________________________________*For Q1 2024, represents product revenue, net from March 13, 2024 (the date of commercial availability) through the end of the quarter. **Statements made in this press release include non-GAAP financial measures. Such information is provided as additional information and not as an alternative to Catalyst's financial statements presented in accordance with U.S. generally accepted accounting principles ("GAAP"). These non-GAAP financial measures are intended to enhance an overall understanding of Catalyst's current financial performance. Catalyst believes that the non-GAAP financial measures presented in this press release provide investors and prospective investors with an alternative method for assessing Catalyst's operating results in a manner that Catalyst believes is focused on the performance of ongoing operations and provides a more consistent basis for comparison between periods. Non-GAAP financial measures should not be considered in isolation or as a substitute for comparable GAAP accounting. Further, non-GAAP measures of net income used by Catalyst may be different from and not directly comparable to similarly titled measures used by other companies. First Quarter 2025 Financial HighlightsThe Company delivered record first quarter 2025 results, reflecting the continued strength of its commercial execution and the durability of its growth strategy. Total Q1 2025 product revenue, net was $141.4 million , a 43.6% year-over-year increase driven by broad-based demand across the commercial portfolio. FIRDAPSE® Q1 2025 product revenue, net was $83.7 million , a 25.3% year-over-year increase, reflecting continued organic growth and a modest benefit from normalized prescription patterns following temporary disruptions related to the Change Healthcare cybersecurity incident in Q1 2024. AGAMREE® Q1 2025 product revenue, net was $22.0 million , compared to product revenue, net of $1.2 million in 2024 for the period that the product was available after its launch on March 13, 2024 . The results underscore continued growth and increasing prescriber engagement. FYCOMPA® Q1 2025 product revenue, net was $35.6 million , a 17.1% year-over-year increase driven by ongoing demand ahead of anticipated generic competition in the second half of 2025. Year-to-date 2025 Select Business HighlightsCatalyst continued to advance its strategic objectives in the first quarter of 2025, achieving key milestones across intellectual property and regulatory progress. On April 8, 2025 , Catalyst announced that Health Canada accepted the New Drug Submission for AGAMREE with Priority Review. The submission, filed by our sub-licensee in Canada , Kye Pharmaceuticals , has the potential to be approved by year-end 2025, which would position AGAMREE as the first authorized treatment option indicated for patients diagnosed with DMD in Canada . During the first quarter of 2025, the SUMMIT Study advanced with the activation of additional sites and continued progress in patient enrollment, with the goal of providing long-term real-world evidence of the benefits of AGAMREE over other DMD treatments. On January 21, 2025 , FIRDAPSE was launched in Japan by sub-licensee DyDo Pharma for the treatment of LEMS, marking a strategic expansion of the product's footprint and enhancing access for underserved LEMS patient populations in this region. On January 8, 2025 , Catalyst announced a settlement agreement with Teva Pharmaceuticals USA, Inc. , and Teva Pharmaceuticals, Inc. (collectively, "Teva"), resolving the patent litigation initiated by Catalyst and SERB in response to Teva's Abbreviated New Drug Application (ANDA) for a generic version of FIRDAPSE. Under the terms of the agreement, Teva may not market its generic product in the U.S. before February 25, 2035 , subject to certain conditions. However, two remaining ANDA litigations regarding Catalyst’s FIRDAPSE patents continue. 2025 Financial Outlook ReaffirmedThe Company is reaffirming its full-year 2025 revenue outlook, reflecting confidence in its continued commercial execution and portfolio performance. Total revenues, net for 2025 is expected to range between $545 million and $565 million . FIRDAPSE product revenue, net for 2025 is expected to range between $355 million and $360 million . AGAMREE product revenue, net for 2025 is expected to range between $100 million and $110 million . FYCOMPA product revenue, net for 2025 is expected to range between $90 million and $95 million , reflecting the anticipated impact of the loss of market exclusivity for the tablets in May 2025 and the oral suspension in December 2025 . First Quarter 2025 Financial Results Cost of sales: Cost of sales for the first quarter of 2025 was $17.9 million , compared to $12.5 million in the first quarter of 2024. As previously disclosed, royalties and milestone obligations related to AGAMREE are expected to increase if full-year product revenue exceeds $100 million . Royalties for FYCOMPA will commence following the loss of market exclusivity in May 2025 and will be subject to tiered rates based on annual net sales and the timing of generic entry. Milestone obligations are recorded when the obligation is incurred and capitalized over the remaining useful life of the asset. Research and development expenses: Research and development expenses for the first quarter of 2025 were $3.9 million, compared to $2.6 million in the first quarter of 2024. The Company anticipates full-year 2025 research and development expenses to range between $15.0 million and $20.0 million , excluding the impact of any additional acquisitions, reflecting investments in the SUMMIT study and the start of initiatives to investigate the potential for AGAMREE’s label to be expanded in the future. Selling, general, and administrative expenses: Selling, general, and administrative (SG&A) expenses for the first quarter of both 2025 and 2024 were $46.9 million. SG&A expenses are expected to increase modestly over the remainder of the year, reflecting personnel additions during 2024 and the strategic realignment of dedicated commercial teams supporting FIRDAPSE and AGAMREE which became effective at the start of the 2025 second quarter. Amortization of intangible assets: Amortization of intangible assets was $9.3 million for the first quarter of both 2025 and 2024. Operating income: Operating income for the first quarter of 2025 was $63.4 million, compared to $27.1 million in the first quarter of 2024, representing an increase of approximately 133.6%. GAAP net income: GAAP net income for the first quarter of 2025 was $56.7 million ( $0 .47 per basic and $0.45 per diluted share), compared to GAAP net income of $23.3 million ( $0.20 per basic and $0.19 per diluted share) in the first quarter of 2024. Non-GAAP net income: Non-GAAP net income for the first quarter of 2025 was $86.6 million ( $0 .71 per basic and $0.68 per diluted share), compared to non-GAAP net income of $46.8 million ( $0.40 per basic and $0.38 per diluted share) in the first quarter of 2024. Non-GAAP net income for all periods excludes from net income stock-based compensation, depreciation, amortization of intangible assets, and the income tax provision. Tax rate: The Company’s effective income tax rate was 20.4% and 20.0% for the three months ended March 31, 2025 and 2024, respectively. Differences in the effective tax and the statutory federal income tax rate of 21% are driven by state income taxes, fluctuations in the value of investments, and anticipated annual permanent differences offset by equity compensation deductions. Cash and cash equivalents: Cash and cash equivalents were $580.7 million as of March 31, 2025. Our Form 10-Q for the first quarter of 2025, filed with the U.S. Securities and Exchange Commission on May 7, 2025, provides more detailed financial information and analysis of our financial condition and results of operations. The webcast will be accessible under the Investors section on the Company's website at www.catalystpharma.com. A webcast replay will be available on Catalyst's website for at least 30 days following the date of the event. About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic agreements. Catalyst, headquartered in Coral Gables, Fla. , was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's Fastest-Growing Companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking StatementsThis press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Catalyst's revenue forecasts for 2025 that are included in this press release will prove to be accurate, (ii) whether Catalyst will continue to be profitable and cash flow positive in 2025 and beyond, (iii) whether Catalyst will complete any acquisitions of additional products, and the timing of any such acquisitions, (iv) the impact of the pending Paragraph IV litigation relating to FIRDAPSE if the results of these litigation matters are adverse, and (v) those factors described in Catalyst's Annual Report on Form 10-K for the 2024 fiscal year, Catalyst's Quarterly Report on Form 10-Q for the first quarter of 2025, and its subsequent filings with the U.S. Securities and Exchange Commission ("SEC"), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC , may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. _________________________________________ (a) exclusive of amortization of intangible assets Source: Catalyst Pharmaceuticals, Inc. Source: Catalyst Pharmaceuticals, Inc.