Nitisinone

BOSTON--(BUSINESS WIRE)-- Cycle Pharmaceuticals Limited (Cycle) is pleased to announce the launch of tiopronin delayed-release tablets as a treatment option for patients with cystinuria.1 Tiopronin delayed-release tablets are available in 100 mg and 300 mg doses, and are approved by the US Food and Drug Administration (FDA).1 Generic tiopronin delayed-release tablets must be taken without food.1 Cystinuria is the most common inheritable cause of kidney stone disease. Incidence of the condition is estimated to be 1 in 7,000 worldwide.2 The launch of tiopronin delayed-release tablets is a partnership between Cycle and Torrent Pharma Inc. (Torrent) utilizing Anovo Specialty Pharmacy (Anovo) in which all three companies are committed to providing patients with rare diseases the medication and patient support they need. Tiopronin delayed-release tablets are a treatment option for patients with cystinuria who want to benefit from the support services provided by Cycle Vita™, a dedicated support platform which provides individualized product support* for patients, and ongoing dispensing and support services provided by Anovo, the exclusive specialty pharmacy for this product. “Cystinuria is a chronic lifelong condition in which stones commonly reoccur.3 In addition, close control of a patient’s diet, specifically a reduction in salt and methionine intake, can have an impact on the likelihood of stones forming.3 We are fully aware of the unique personal challenges these patients face in effectively managing their cystinuria. That’s why we’re providing individualized support including a Bridge Program* and Co-pay Assistance Program*. In addition, a Patient Nutrition Program* will be available to reinforce the clinic’s nutrition plan,” says Chikai Lai, SVP & Chief Commercial Officer, Cycle. Tiopronin delayed-release tablets are Cycle’s fifth commercial product to be launched in the US, building on the launch of: NITYR® (nitisinone) Tablets in 2017 SAJAZIR™ (icatibant) Injection in 2021 JAVYGTOR™ (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022; and TASCENSO ODT® (fingolimod) in 2023 Tiopronin delayed-release tablets are indicated in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 9 years of age and older with severe homozygous cystinuria, who are not responsive to these measures alone. Tiopronin delayed-release tablets must be taken without food.1 To find out more about Cycle Vita, please visit or call +1 (888) 360-8482 and find the Full Prescribing Information available at . Important Safety Information Important Safety Information is below. Full Prescribing Information is available at . INDICATIONS Tiopronin delayed-release tablets are a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for prevention of cystine stone formation in adult and pediatric patients 9 years of age and older with severe homozygous cystinuria who are not responsive to these measures alone. WARNINGS AND PRECAUTIONS Proteinuria, including nephrotic syndrome, and membranous nephropathy, have been reported with tiopronin use. Pediatric patients receiving greater than 50 mg/kg of tiopronin per day may be at increased risk for proteinuria. Hypersensitivity reactions (drug fever, rash, fever, arthralgia and lymphadenopathy) have been reported. MOST COMMON ADVERSE REACTIONS Most common adverse reactions (≥10%) are nausea, diarrhea or soft stools, oral ulcers, rash, fatigue, fever, arthralgia, proteinuria, and emesis. DRUG INTERACTIONS Alcohol: Tiopronin is released faster from tiopronin delayed-release tablets in the presence of alcohol and the risk for adverse events associated with tiopronin delayed-release tablets when taken with alcohol is unknown. Avoid alcohol consumption 2 hours before and 3 hours after taking tiopronin delayed-release tablets. USE IN SPECIFIC POPULATIONS: Pregnancy: Available published case report data with tiopronin have not identified a drug-associated risk for major birth defects, miscarriage, or adverse maternal or fetal outcomes. Renal stones in pregnancy may result in adverse pregnancy outcomes. Tell your healthcare provider immediately if you become pregnant or planning to become pregnant. Lactation: There are no data on the presence of tiopronin in either human or animal milk or on the effects of the breastfed child. A published study suggests that tiopronin may suppress milk production. Because of the potential for serious adverse reactions, including nephrotic syndrome, advise patients that breastfeeding is not recommended during treatment with tiopronin delayed-release tablets. Tell your healthcare provider immediately if you plan on breast feeding. Pediatric Use: Tiopronin delayed-release tablets are indicated in pediatric patients 9 years of age and older with severe homozygous cystinuria, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation who are not responsive to these measures alone. Tiopronin delayed-release tablets are not approved for use in pediatric patients weighing less than 20 kg or in pediatric patients unable to swallow tablets. Geriatric Use: This drug is known to be substantially excreted by the kidney, and the risk of adverse reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, care should be taken in dose selection, and it may be useful to monitor renal function. For more detailed information, please refer to the full Prescribing Information at . If you get any side effects, talk to your healthcare provider. This includes possible side effects not listed in this material. You may also report side effects directly by calling Torrent Pharma Inc. at: 1-800-912-9561 or FDA at: 1-800-FDA-1088 or . This material is for education purposes only. It is not intended to be, and should not be used as, a substitute for medical advice. US-TIO-2400004 (February 2024) References Tiopronin Delayed-Release Tablets. Prescribing Information. Torrent Pharma Inc. Leslie, S.W., Sajjad, H. and Nazzal, L. (2023). Cystinuria. [online] Nih.gov. Available at: [Accessed Feb 9 2024]. Cystinuria UK - Diet - what should I eat (2023). Cystinuria UK - Diet - what should I eat? [online] Cystinuriauk.co.uk. Available at: [Accessed Feb 9 2024]. * Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at +1 (888) 360-8482. The US Prescribing Information provides the indications for which the product is approved in the US. Please consult the US Prescribing Information and product labeling for information about the specific uses for which the product is indicated in the US. –ENDS– Cycle Vita™, SAJAZIR™ and JAVYGTOR™ are trademarks of Cycle Pharmaceuticals Limited in the United States. NITYR® is a registered trademark of Cycle Pharmaceuticals Limited in the United States. TASCENSO ODT® is a registered trademark of Handa Neuroscience, LLC. ©2024 Cycle Pharmaceuticals Limited. All rights reserved. About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease patient community. Cycle focuses on rare metabolic, immunological, and neurological genetic conditions. Within neurological conditions, we focus on multiple sclerosis. Cycle is headquartered in Cambridge, UK and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook. About Torrent Pharma Inc. Torrent Pharma is one of the leading Pharma companies in India. Torrent is the pioneer in initiating the concept of niche marketing in India and today is ranked amongst the leaders in therapeutic segment of cardiovascular (CV), central nervous system (CNS), gastro-intestinal (GI) and women healthcare (WHC). Torrent also has significant presence in diabetology, pain management, gynecology, oncology and anti-infective segments. Torrent Pharma is present in more than 40 countries. It is ranked first amongst Indian Companies for having largest market share in Brazil and Germany. About Anovo Anovo was founded in 2012 as an exclusive provider of comprehensive end-to-end services for manufacturers of rare disease drugs and patients who need the drugs, including specialty wholesale/3PL distribution services, specialty pharmacy services, and patient support hub programs. Anovo benefits all parties involved by navigating all the complexities of managing rare disease medications and providing the related support needed by patients, caregivers and prescribers. View source version on businesswire.com: Contacts Marketing@cyclepharma.com Cycle Pharmaceuticals Tel: +44 1223 354 118 Source: Cycle Pharmaceuticals Limited View this news release online at:

• Q1 2024 revenue of $8.0 million, representing 50% growth over Q1 2023 and the 13th straight quarter of sequential product sales growth• Acquired PKU GOLIKE®, launched Nitisinone, and submitted a new drug application (NDA) for product candidate ET-400• Management to hold conference call today at 4:30pm ET DEER PARK, Ill., May 09, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended March 31, 2024. “We have had a very productive start to 2024. In addition to reporting record product sales and our 13th straight quarter of sequential product sales growth, we acquired PKU GOLIKE which adds a compelling new commercial growth asset to our metabolic portfolio. We also launched Nitisinone and submitted an NDA for our highly anticipated ET-400 product candidate,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “It’s an exciting time for Eton as we continue to execute on our vision. With strong growth expected from our existing products, plus the additions of PKU GOLIKE and Nitisinone, we are positioned for a very strong 2024 as we gear up for the potential launch of ET-400 in early 2025,” concluded Brynjelsen. First quarter and Recent Business Highlights 13th straight quarter of sequential growth in product sales and royalty revenue. Eton reported first quarter 2024 product sales of $8.0 million, representing 50% growth over the prior year, driven primarily by the ongoing momentum of ALKINDI SPRINKLE® and Carglumic Acid. The Company expects sequential quarter-over-quarter growth of product sales to continue throughout 2024 and beyond. Acquired U.S. rights to PKU GOLIKE, a medical formula for patients with phenylketonuria (PKU). An estimated 8,000 PKU patients in the U.S. rely on medical formulas such as PKU GOLIKE. The condition is managed by metabolic geneticists and their support staff, the same healthcare professionals that Eton is actively engaged with on its other metabolic products. The U.S. market for PKU medical formulas is estimated to be approximately $100 million annually. With GOLIKE’s attractive product benefits and Eton’s commercial infrastructure, the Company’s goal is to capture at least 10% market share, or $10 million annually, in the coming years. Submitted NDA to the U.S. Food and Drug Administration (FDA) for ET-400. In April, Eton submitted an NDA for ET-400, its proprietary patented formulation of hydrocortisone oral solution. The anticipated 10-month review would allow for potential approval in the first quarter of 2025. The Company continues to see strong interest in the product candidate and believes that ET-400 and ALKINDI SPRINKLE can combine to achieve peak sales of over $50 million annually. Continued growth in ALKINDI SPRINKLE and Carglumic Acid. In the first quarter, the Company launched an ALKINDI SPRINKLE sampling program, which allows physicians to hold product and immediately start a patient on treatment. The Company estimates that Carglumic Acid has now captured over 50% of the patient population and continues to see new patient additions. The launches of Betaine and Nitisinone have further strengthened Eton’s relationships with the metabolic community and have helped increase the frequency of interactions with Carglumic Acid prescribers. Launched rare disease product Nitisinone. The product was launched in February 2024 with full patient and provider support services. The Nitisinone market is estimated to be $50 million annually, split across multiple participants. Eton expects to capture a share of the existing market and the Nitisinone launch should provide the Company with additional opportunities to cross-sell Carglumic Acid and PKU GOLIKE. First Quarter Financial Results Net Revenue: Net revenues for the first quarter of 2024 were $8.0 million compared with $5.3 million in the prior year period, an increase of 50%. The increase was driven primarily by growth in ALKINDI SPRINKLE and Carglumic Acid. Gross Profit: Gross profit for the first quarter of 2024 was $5.0 million compared with $3.3 million in the prior year period, with the increase also driven primarily by growth in ALKINDI SPRINKLE and Carglumic Acid. Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2024 were $0.7 million compared to $0.5 million in the prior year period due to increased development costs associated with product candidate ET-400 and other products in the Company’s R&D pipeline. While the Company expects an increase in second quarter R&D expenses with a $2.0 million ET-400 filing fee, R&D spend for 2024 is forecasted to be approximately $4.0 million excluding the one-time filing fee. General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2024 were $5.2 million compared to $5.3 million in the prior year period, with the decrease due to a slight reduction in employee related expenses and logistics costs. Net Income: Net loss for the first quarter of 2024 was $0.8 million or $0.03 per basic and diluted share compared to a net loss of $2.7 million or $0.10 per diluted share in the prior year period. Cash Position: As of March 31, 2024, the Company had cash and cash equivalents of $16.7 million and expects continued sales growth to result in positive operating cash flow throughout the remainder of 2024. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host a its first quarter 2024 conference call as follows: DateMay 9, 2024 Time4:30 p.m. ET (3:30 p.m. CT) Register* (Audio Only)Click here In addition to taking live questions from participants on the conference call, management will be answering emailed questions from investors. Investors can email questions to: investorrelations@etonpharma.com. The live webcast can be accessed on the Investors section of Eton’s website at https://ir.etonpharma.com/. An archived webcast will be available on Eton’s website approximately two hours after the completion of the event and for 30 days thereafter. * Conference call participants should register to obtain their dial-in and passcode details. Please be sure to register using a valid email address. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Eton Pharmaceuticals, Inc.Statements of Operations(In thousands, except per share amounts)(Unaudited) For the three months ended March 31, March 31, 2024 2023 Revenues: Licensing revenue $— $— Product sales and royalties 7,966 5,304 Total net revenues 7,966 5,304 Cost of sales: Licensing revenue — — Product sales and royalties 2,959 1,958 Total cost of sales 2,959 1,958 Gross profit 5,007 3,346 Operating expenses: Research and development 651 535 General and administrative 5,156 5,345 Total operating expenses 5,807 5,880 Loss from operations (800) (2,534) Other income (expense): Other income — — Interest expense, net (11) (126)Total other income (expense) (11) (126) Loss before income tax expense (811) (2,660) Income tax expense — — Net loss $(811) $(2,660)Net loss per share, basic $(0.03) $(0.10)Weighted average number of common shares outstanding, basic 25,763 25,525 Net loss per share, diluted $(0.03) $(0.10)Weighted average number of common shares outstanding, diluted 25,763 25,525 Eton Pharmaceuticals, Inc.Balance Sheets(In thousands, except share and per share amounts) March 31, 2024 December 31, 2023 (Unaudited) Assets Current assets: Cash and cash equivalents $16,655 $21,388 Accounts receivable, net 4,240 3,411 Inventories 2,318 911 Prepaid expenses and other current assets 1,050 1,129 Total current assets 24,263 26,839 Property and equipment, net 57 58 Intangible assets, net 6,388 4,739 Operating lease right-of-use assets, net 74 92 Other long-term assets, net 12 12 Total assets $30,794 $31,740 Liabilities and stockholders’ equity Current liabilities: Accounts payable $2,263 $1,848 Current portion of debt, net of discount 5,020 5,380 Accrued liabilities 8,017 9,013 Total current liabilities 15,300 16,241 Operating lease liabilities, net of current portion — 22 Total liabilities 15,300 16,263 Commitments and contingencies (Note 11) Stockholders’ equity Common stock, $0.001 par value; 50,000,000 shares authorized; 25,690,562 and 25,688,062 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively 26 26 Additional paid-in capital 120,349 119,521 Accumulated deficit (104,881) (104,070)Total stockholders’ equity 15,494 15,477 Total liabilities and stockholders’ equity $30,794 $31,740 Eton Pharmaceuticals, Inc.Statements of Cash Flows(In thousands)(Unaudited) Three months ended Three months ended March 31, 2024 March 31, 2023 Cash flows from operating activities Net loss $(811) $(2,660) Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 821 872 Depreciation and amortization 252 213 Debt discount amortization 25 29 Changes in operating assets and liabilities: Accounts receivable (829) (1,022)Inventories (1,407) 120 Prepaid expenses and other assets 79 191 Accounts payable 414 (530)Accrued liabilities (1,017) 1,239 Net cash used in operating activities (2,473) (1,548) Cash flows from investing activities Purchases of product license rights (1,868) — Purchases of property and equipment (14) — Net cash used in investing activities (1,882) — Cash flows from financing activities Repayment of long-term debt (385) — Proceeds from stock option exercises 7 132 Payment of tax withholding related to net share settlement of stock option exercises — (181)Net cash used in financing activities (378) (49) Change in cash and cash equivalents (4,733) (1,597)Cash and cash equivalents at beginning of period 21,388 16,305 Cash and cash equivalents at end of period $16,655 $14,708 Supplemental disclosures of cash flow information Cash paid for interest $190 $216 Cash paid for income taxes $— $—