Vamorolone

Reports Q3 2024 Total Revenues of $128.7 Million, Representing a 25.3% YoY Increase Record Revenues Driven by Continued Organic Growth in its Rare Disease Franchise Robust Financial Performance Highlights Operational Excellence and Strong Commercial Execution FIRDAPSE® Q3 2024 Net Product Revenues of $79.3 Million, Representing a 19.7% YoY Increase AGAMREE® Q3 2024 Net Product Revenues of $15.0 Million, Demonstrating Early U.S. Launch Success FYCOMPA® Q3 2024 Net Product Revenues of $32.1 Million Q3 2024 GAAP Net Income of $43.9 Million, $0.35 Per Share Diluted Q3 2024 Non-GAAP Net Income of $71.1 Million, $0.57 Per Share Diluted Updating 2024 Full Year Total Revenue Guidance to Between $475 Million and $485 Million Conference Call and Webcast to be Held on November 7, 2024, at 8:30 AM ET CORAL GABLES, Fla., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) (Nasdaq: CPRX) today reported financial results for the third quarter of 2024 and provided a corporate update. "Our strong financial performance reflects our unwavering commitment to operational and commercial excellence, driving sustained momentum and long-term growth. The early success of our newest product, AGAMREE®, along with the continued robust organic growth of our flagship product, FIRDAPSE®, highlights the strength of our U.S. commercial strategy, with revenues exceeding expectations and significantly contributing to our overall success," stated Richard J. Daly, President and CEO of Catalyst. "Through our strategic partnerships, we are advancing initiatives to expand the geographic footprint of our rare disease portfolio in key territories outside the U.S. As we pursue opportunities aligned with our long-term growth objectives and forge new partnerships to broaden our global presence, our achievements to date underscore the strength of our strategic vision to address critical unmet medical needs, driven by the outstanding execution of our teams. With a resolute focus on sustained growth and value creation, we are optimistic about the path ahead and remain confident in meeting our full-year 2024 revenue guidance, laying a strong foundation for continued success." Financial Highlights For the Three Months Ended September 30, 2024 2023 % Change(In thousands, except per share data) Product Revenue, Net$126,424$102,617 23.2% FIRDAPSE Product Revenue, Net$79,303$66,224 19.7% FYCOMPA Product Revenue, Net$32,075$36,393 (11.9%) AGAMREE Product Revenue, Net$15,046 N/A N/A GAAP Net Income (Loss)$43,884($30,764) 242.6% Non-GAAP Net Income (Loss)**$71,080($25,643) 377.2% GAAP Net Income (Loss) Per Share – Basic$0.37($0.29) 227.6% Non-GAAP Net Income (Loss) Per Share – Basic**$0.60($0.24) 350.0% GAAP Net Income (Loss) Per Share – Diluted$0.35($0.29) 220.7% Non-GAAP Net Income (Loss) Per Share – Diluted**$0.57($0.24) 337.5% As of September 30, 2024, and December 31, 2023(In thousands)Cash and Cash Equivalents$442,331$137,636 221.4% **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. Beginning with the third quarter of 2024, Catalyst will no longer include acquisition in-process research and development ("IPR&D") in its non-GAAP financial measures. Previously reported non-GAAP net income for the third quarter of 2023 included $81.5 million in acquisition IPR&D relating to Catalyst's acquisition, during that period, of the North American license for AGAMREE, which had not yet been approved by the U.S. Food and Drug Administration (“FDA”) at the time of the acquisition. The commercialization was subsequently approved by the FDA during the fourth quarter of 2023. Prior period amounts have been revised to conform to the current period presentation. Catalyst is making this change to its presentation following discussions with the Staff of the Division of Corporation Finance of the U.S. Securities and Exchange Commission. Recent Business Highlights Achieved total revenues of $128.7 million, including net product revenues of $126.4 million for the third quarter (“Q3”) of 2024, reflecting a 23.2% increase over net product revenues for Q3 of 2023. Reported record quarterly FIRDAPSE net product revenues of $79.3 million for Q3 2024, reflecting a 19.7% increase over Q3 2023, driven by sustained organic growth. Achieved robust AGAMREE net product revenues of $15.0 million for Q3 2024, demonstrating continued momentum in its second full quarter of U.S. commercial availability. Reported FYCOMPA® net product revenues of $32.1 million for Q3 2024, an 11.9% decrease compared to Q3 2023 revenues of $36.4 million. Announced that DyDo Pharma, Inc. (Catalyst’s sub-licensee in Japan), received regulatory approval in Japan to commercialize FIRDAPSE 10 mg tablets for the treatment of Lambert-Eaton myasthenic syndrome in Japan, marking a key milestone in expanding the product’s commercial footprint. DyDo has advised Catalyst that it expects to launch in Japan by the end of Q4 2024. Announced an exclusive agreement with KYE Pharmaceuticals, Inc. for the Canadian commercial rights to AGAMREE for Duchenne muscular dystrophy and other potential indications, further expanding Catalyst’s North American footprint. This marks the licensing of Catalyst’s second rare disease product in Canada, and KYE has advised us that it expects to file a Health Canada application for AGAMREE by early 2025. Expect total revenues for the full year 2024 to be between $475 million and $485 million, which includes the $2.1 million milestone payment received from DyDo that is included in License and other revenue. Revising full-year 2024 net product revenue guidance for FIRDAPSE to be between $300 million and $310 million; reaffirming FYCOMPA net product revenue guidance to be between $130 million and $135 million; and increasing AGAMREE net product revenue guidance to be between $40 million and $45 million, respectively. Revising full year research and development (“R&D”) expense guidance to less than $15 million due to delays in the timing of certain R&D related initiatives. Third Quarter 2024 Financial Results Total revenues: Total revenues during the third quarter of 2024 were $128.7 million, compared to $102.7 million for the third quarter of 2023, representing an increase of approximately 25.3%. Product revenue, net: Product revenue, net for the third quarter of 2024 was $126.4 million, compared to $102.6 million for the third quarter of 2023, representing an increase of approximately 23.2%. License and other revenue: License and other revenue includes $2.1 million from the arrangement with DyDo for both the three and nine months ended September 30, 2024, which consisted of a milestone payment earned upon DyDo receiving regulatory approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan. Research and development expenses: Research and development expenses for the third quarter of 2024 were $3.3 million, compared to $83.7 million for the third quarter of 2023, representing a decrease of approximately 96.1%. Research and development expenses for the third quarter of 2023 included acquisition IPR&D of $81.5 million. Selling, general and administrative expenses: Selling, general and administrative expenses for the third quarter of 2024 were $45.9 million, compared to $33.6 million for the third quarter of 2023, representing an increase of approximately 36.7%. Amortization of intangible assets: Amortization of intangible assets was $9.3 million in the third quarter of 2024, compared to $8.5 million in the third quarter of 2023, representing an increase of approximately 10.1%. Operating income (loss): Operating income (loss) for the third quarter of 2024 was $50.9 million, compared to ($37.2) million for the third quarter of 2023, representing an increase of approximately 236.9%. GAAP net income (loss): GAAP net income (loss) for the third quarter of 2024 was $43.9 million ($0.37 per basic and $0.35 per diluted share), compared to GAAP net income (loss) of ($30.8) million (($0.29) per basic and diluted share) for the third quarter of 2023. Non-GAAP net income (loss): Non-GAAP net income (loss) for the third quarter of 2024 was $71.1 million ($0.60 per basic and $0.57 per diluted share), compared to non-GAAP net income (loss) of ($25.6) million (($0.24) per basic and diluted share) for the third quarter of 2023. Cash and cash equivalents: Cash and cash equivalents were $442.3 million as of September 30, 2024. Catalyst’s Form 10-Q for the third quarter of 2024, filed with the U.S. Securities and Exchange Commission on November 6, 2024, provides more detailed financial information and analysis of Catalyst’s financial condition and results of operations. Conference Call & Webcast DetailsThe Company will host a conference call and webcast on Thursday, November 7, 2024, at 8:30 AM ET to discuss the financial results and provide a business update. US/Canada Dial-in Number:800-715-9871International Dial-in Number:646-307-1963 A webcast will be accessible under the investor section on the Company's website at www.catalystpharma.com. A webcast replay will be available on the Catalyst website for 30 days after 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, Catalyst’s focus is on in-licensing, commercializing, and developing innovative therapies. Guided by its deep commitment to patient care, Catalyst prioritizes 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 partnerships. Catalyst is headquartered in Coral Gables, FL., and was recognized on the Forbes 2024 list as one of America's most successful small-cap companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking StatementsThis press release contains forward-looking statements. 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 the launch of AGAMREE® will continue to surpass initial expectations and exceed 2024 full-year revenue forecasts for sales of the product that have been published by the Company, (ii) whether AGAMREE's commercialization will ultimately be profitable, cash flow positive, and accretive to Catalyst, (iii) Catalyst's ability to continue to successfully sell its current products and continue to be profitable and cash flow positive, (iv) whether Catalyst's total revenue forecast for 2024 that is included in this press release will prove to be accurate, (v) whether Catalyst will complete additional acquisitions of products, and the timing of any such acquisitions, (vi) the impact of pending Paragraph IV litigation relating to FIRDAPSE® if the results of these litigation matters are adverse, (vii) whether AGAMREE will be approved for commercialization in Canada, (viii) whether DyDo can successfully commercialize FIRDAPSE in Japan, and (ix) those factors described in Catalyst's Annual Report on Form 10-K for the 2023 fiscal year, Catalyst's Quarterly Report on Form 10-Q for the third quarter of 2024, and Catalyst's other filings with the SEC, could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC and may be found on Catalyst's website or obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. CATALYST PHARMACEUTICALS, INC.CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited)(in thousands, except share and per share data) For the Three MonthsEnded September 30, For the Nine MonthsEnded September 30, 2024 2023 2024 2023Product revenue, net$126,424 $102,617 $347,518 $287,398License and other revenue 2,271 71 2,396 238Total revenues 128,695 102,688 349,914 287,636 Operating costs and expenses: Cost of sales (a) 19,277 14,167 47,202 36,158Research and development 3,284 83,662 8,850 91,178Selling, general and administrative (a) 45,880 33,560 133,548 91,674Amortization of intangible assets 9,345 8,487 28,033 23,506Total operating costs and expenses 77,786 139,876 217,633 242,516Operating income (loss) 50,909 (37,188) 132,281 45,120Other income (expense), net 6,296 (833) 9,801 2,684Net income (loss) before income taxes 57,205 (38,021) 142,082 47,804Income tax provision (benefit) 13,321 (7,257) 34,129 11,238Net income (loss)$43,884 $(30,764) $107,953 $36,566 Net income (loss) per share: Basic$0.37 $(0.29) $0.92 $0.34Diluted$0.35 $(0.29) $0.87 $0.32 Weighted average shares outstanding: Basic 118,931,153 106,568,137 117,976,056 106,133,077Diluted 125,407,279 106,568,137 124,519,838 113,751,370 (a) exclusive of amortization of intangible assets CATALYST PHARMACEUTICALS, INC.RECONCILIATION OF NON-GAAP METRICS (unaudited)(in thousands, except share and per share data) For the Three MonthsEnded September 30, For the Nine MonthsEnded September 30, 2024 2023 2024 2023GAAP net income (loss)$43,884 $(30,764) $107,953 $36,566Non-GAAP adjustments: Stock-based compensation expense 4,424 3,810 17,080 10,000Depreciation 106 81 283 232Amortization of intangible assets 9,345 8,487 28,033 23,506Income tax provision (benefit) 13,321 (7,257) 34,129 11,238Non-GAAP net income (loss)$71,080 $(25,643) $187,478 $81,542 Non-GAAP net income (loss) per share: Basic$0.60 $(0.24) $1.59 $0.77 Diluted$0.57 $(0.24) $1.51 $0.72 Weighted average shares outstanding: Basic 118,931,153 106,568,137 117,976,056 106,133,077 Diluted 125,407,279 106,568,137 124,519,838 113,751,370 CATALYST PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(in thousands) September 30,2024(unaudited) December 31,2023Assets Current Assets: Cash and cash equivalents$442,331 $137,636Accounts receivable, net 58,266 53,514Inventory 20,017 15,644Prepaid expenses and other current assets 18,384 12,535Total current assets 538,998 219,329Operating lease right-of-use asset, net 2,301 2,508Property and equipment, net 1,380 1,195License and acquired intangibles, net 166,016 194,049Deferred tax assets, net 48,473 36,544Investment in equity securities 14,842 16,489Total assets$772,010 $470,114 Liabilities and Stockholders’ Equity Current Liabilities: Accounts payable$11,563 $14,795Accrued expenses and other liabilities 93,832 61,268Total current liabilities 105,395 76,063Operating lease liability, net of current portion 2,889 3,188Other non-current liabilities 2,785 2,982Total liabilities 111,069 82,233Total stockholders’ equity 660,941 387,881Total liabilities and stockholders’ equity$772,010 $470,114 Source: Catalyst Pharmaceuticals, Inc. Contact information: Investor ContactMary ColemanCatalyst Pharmaceuticals, Inc.(305) 420-3200mcoleman@catalystpharma.com Media ContactDavid SchullRusso Partners(858) 717-2310david.schull@russopartnersllc.com

October 1, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the positive outcome of the LIONHEART study, confirming vamorolone’s distinctive action also as a mineralocorticoid receptor antagonist, setting it apart from other corticosteroids. The LIONHEART study, an open-label randomized, placebo- and eplerenone-controlled study involving 30 healthy adult male subjects, met its primary endpoint. It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. This increased ratio in urine provides clinical evidence for vamorolone’s unique mode of action also as a mineralocorticoid receptor antagonist (MRA) in humans. Combined with its known properties as a dissociative glucocorticoid receptor agonist, these findings further differentiate vamorolone’s pharmacological profile, distinguishing it from other corticosteroids. Cardiac complications such as cardiomyopathy are a leading cause of morbidity and mortality in boys with Duchenne muscular dystrophy (DMD). While treatment with corticosteroids (and ACE inhibitors) has demonstrated a delay in the onset of cardiomyopathy, the addition of MRAs including eplerenone to standard of care has also shown an improvement in left ventricular systolic dysfunction, the benefit of which increases with earlier initiation [1-3]. “Mineralocorticoid receptor antagonists are strongly recommended but late when cardiac function is already reduced and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi, PhD, MD, Cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid” “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone,” stated Shabir Hasham, MD, Chief Medical Officer of Santhera. “We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterize long-term outcomes including any beneficial impact on cardiac complications in DMD.” AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [4-7]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [4-7]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has been shown to be a dual mineralocorticoid antagonist and a dissociative glucocorticoid agonist based on animal experiments [7]. It has no restriction of growth [8] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [9]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom for the treatment of DMD. The LIONHEART study (SNT-I-VAM-026) is an open label randomized, 3-arm, parallel-group, placebo and eplerenone controlled study to evaluate the mineralocorticoid receptor antagonism (MRA) effect of vamorolone in 30 healthy adult male subjects after challenge with fludrocortisone, a known mineralocorticoid receptor agonist. The primary endpoint was defined as the ratio of sodium to potassium (Na/K) and the corresponding logarithm of the ratio (log10(10*Na/K) in urine at different time-points. Further analysis of the data is ongoing and will be presented at medical conferences. References: [1] Birnkrant et al. (2018) Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. [2] Raman et a. (2015) Lancet Neurol 14: 153–161 doi:10.1016/s1474-4422(14)70318-7 [3] Raman et al. (2017) Orphanet J Rare Dis 12: 39. doi:10.1186/s13023-017-0590-8 [4] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [5] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [6] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [7] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [8] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [9] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.