Latest news with #SeanBrynjelsen
Yahoo
29-05-2025
- Business
- Yahoo
FDA approves Eton's application for hydrocortisone oral solution
Eton Pharmaceuticals' new drug application (NDA) for Khindivi, an oral solution of hydrocortisone, has received US Food and Drug Administration (FDA) approval as a replacement therapy for children aged five years and above with adrenocortical insufficiency. This rare condition is characterised by the adrenal glands' failure to produce adequate levels of cortisol. Khindivi is the sole FDA-approved hydrocortisone oral solution and is available in a 1mg/ml concentration. This formulation is particularly beneficial as it removes the necessity of splitting or crushing tablets, providing precise dosing in a ready-to-use liquid solution that does not require mixing, shaking or refrigeration. The product is designed to be suitable for those who struggle with swallowing pills or have specific administration needs, such as a gastric tube. The company's current team of paediatric endocrinology rare disease specialists will promote the oral solution. Khindivi will soon be available in the country via speciality pharmacy Anovo that caters to patients with rare and chronic conditions. Khindivi is not approved for increased dosing at times of stress or acute events. Eton also markets Alkindi Sprinkle, another FDA-approved hydrocortisone treatment for paediatric adrenocortical insufficiency. Eton Pharmaceuticals CEO Sean Brynjelsen stated: 'The FDA approval of Khindivi is a tremendous achievement for Eton and more importantly, a pivotal step forward for paediatric patients with adrenal insufficiency. As a home-grown programme, our team expertly navigated the development, clinical and regulatory pathway. 'Being in a position to commercialise Khindivi within days of this approval is a further testament to the executional excellence from our entire company.' In February 2025, the company secured a patent from the US Patent and Trademark Office for the desmopressin oral solution formulation of its ET-600 product candidate for the treatment of diabetes insipidus. "FDA approves Eton's application for hydrocortisone oral solution" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Upturn
28-05-2025
- Business
- Business Upturn
Eton Pharmaceuticals Announces U.S. FDA Approval for KHINDIVI™ (hydrocortisone) Oral Solution
• KHINDIVI is the first and only FDA-approved hydrocortisone oral solution • Commercial launch expected the week of June 2nd • Eton expects combined peak sales of KHINDIVI and ALKINDI SPRINKLE® (hydrocortisone) oral granules to exceed $50 million DEER PARK, Ill., May 28, 2025 (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 announced the U.S. Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for KHINDIVI™ (hydrocortisone) Oral Solution as a replacement therapy in pediatric patients five years of age and older with adrenocortical insufficiency. KHINDIVI is the only FDA-approved oral solution formulation of hydrocortisone. It comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, and to offer simple and accurate dosing specifically tailored to each patient's needs. It does not require refrigeration, mixing, or shaking – it is a ready-to-use oral liquid solution. KHINDIVI is designed to offer administration simplicity and dosing accuracy, and to provide a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube. 'The FDA approval of KHINDIVI is a tremendous achievement for Eton and more importantly, a pivotal step forward for pediatric patients with adrenal insufficiency. As a home-grown program, our team expertly navigated the development, clinical and regulatory pathway. In addition, being in a position to commercialize KHINDIVI within days of this approval is a further testament to the executional excellence from our entire company,' said Sean Brynjelsen, CEO of Eton Pharmaceuticals. 'For decades, patients have been seeking an FDA-approved hydrocortisone liquid that allows incremental, accurate dosing in the preferred dosage form for children. We are excited to now make it available to patients. Our commercial team is fully mobilized and ready to hit the ground running within the first week of approval. We're committed to ensuring that pediatric endocrinologists across the country are aware of this important new treatment option,' continued Brynjelsen. 'Managing adrenal insufficiency in pediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments that address the individualized pharmacokinetic needs of each child,' said Dr. Kyriakie Sarafoglou, Professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota. 'The availability of an FDA-approved oral hydrocortisone liquid solution offers physicians a new tool to dose patients accurately, which is important to clinical outcomes during this dynamic period of growth and development.' 'For families facing the daily challenges of pediatric congenital adrenal hyperplasia (CAH), timely access to the right treatments is critical,' said Dina Matos, Executive Director of the CARES Foundation—the only U.S. organization solely focused on the CAH community. 'The introduction of KHINDIVI is a significant advancement, particularly because accurately splitting pills to achieve proper dosing for children has long been a struggle. The ability to dose patients more accurately is critical for treatment outcomes. We commend Eton for working to make this therapy accessible through specialty channels. This marks meaningful progress for our community and a vital step toward easing the daily burden on parents and caregivers.' KHINDIVI will be promoted by Eton's existing team of pediatric endocrinology rare disease specialists. Eton currently commercializes ALKINDI SPRINKLE (hydrocortisone) Oral Granules, which is FDA-approved for pediatric patients with adrenocortical insufficiency. The addition of KHINDIVI will provide adrenal insufficiency patients and caregivers with an additional option when choosing the treatment that best meets their individual needs. Adrenocortical insufficiency is a rare, but serious condition in which the adrenal glands do not produce sufficient cortisol. Eton estimates that there are more than 5,000 adrenal insufficiency patients in the U.S. between the ages of 5 and 17, and expects peak sales of KHINDIVI, combined with ALKINDI SPRINKLE, will exceed $50 million per year. KHINDIVI will be available in the coming days in the United States exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pays for qualifying patients. Clinicians seeking to prescribe KHINDIVI can e-prescribe by selecting Anovo #5 or fax in a patient referral form to 855-813-2039. Additional product details can be found on the product website, INDICATION KHINDIVI is a corticosteroid indicated as replacement therapy in pediatric patients 5 years of age and older with adrenocortical insufficiency. Limitation of Use KHINDIVI is not approved for increased dosing during periods of stress or acute events. Use a different hydrocortisone-containing drug product for stress dosing. IMPORTANT SAFETY INFORMATION Contraindication Hypersensitivity to hydrocortisone or any of the other ingredients in KHINDIVI oral solution. Warnings and Precautions Adrenal Crisis: Undertreatment or sudden discontinuation of therapy with KHINDIVI may lead to symptoms of adrenal insufficiency, adrenal crisis, and death. Adrenal crisis may also be induced by stress events, such as infections or surgery when patients require higher doses of corticosteroids. During periods of stress (e.g., infections, surgery), switch to another oral hydrocortisone product and increase the dose if oral medications are tolerated. Monitor patients when switching to KHINDIVI to ensure KHINDIVI is providing the same level of hydrocortisone exposure as the previously used oral hydrocortisone formulation. If symptoms of adrenal insufficiency occur, increase the total daily dosage of KHINDIVI. Systemic Adverse Reactions Due to Inactive Ingredients: Hyperosmolarity KHINDIVI is not approved in pediatric patients less than 5 years of age. The inactive ingredients polyethylene glycol 400, propylene glycol, and glycerin undergo substantial systemic absorption, individually or in combination, resulting in increased plasma osmolarity in all pediatric patients, especially in pediatric patients less than 5 years of age. Monitor pediatric patients using KHINDIVI for signs and symptoms consistent with hyperosmolarity. Metabolic Acidosis and Other Adverse Reactions The inactive ingredient polyethylene glycol 400 and propylene glycol that may result in metabolic acidosis, hypoglycemia, hepato-renal injury, and central nervous system toxicity (e.g., seizure and coma), may increase the risk of adrenal crisis. Monitor laboratory values and for physical signs and symptoms of these adverse reactions. Laxative Effects Due to Inactive Ingredients The inactive ingredients polyethylene glycol 400 and glycerin, whether alone or in combination, may cause gastrointestinal irritation resulting in vomiting and/or diarrhea. These gastrointestinal reactions may increase the risk of adrenal crisis. Monitor for signs or symptoms of gastrointestinal irritation and associated fluid and electrolyte abnormalities. Immunosuppression and Increased Risk of infection with Use of a Dosage Greater Than Replacement: The use of a greater than replacement dosage can suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Monitor for the development of infection and consider dosage reduction as needed. Growth Retardation: Long-term use in excessive doses may cause growth retardation. Use the minimum dosage of KHINDIVI to achieve desired clinical response and monitor the patient's growth. Cushing's Syndrome Due to Use of Excessive Doses of Corticosteroids: Prolonged use with supraphysiologic doses may cause Cushing's syndrome. Monitor patients for signs and symptoms of Cushing's syndrome every 6 months. Decrease in Bone Mineral Density: Corticosteroids decrease bone formation and increase bone resorption which may lead to the development of osteoporosis. Use the minimum dosage of KHINDIVI to achieve desired clinical response. Psychiatric Adverse Reactions: Use may be associated with severe psychiatric adverse reactions, such as euphoria, mania, psychosis with hallucinations and delirium, or depression. Symptoms typically emerge within a few days or weeks of starting the treatment. Most reactions resolve after either dose reduction or withdrawal, although specific treatment may be necessary. Monitor patients for behavioral and mood disturbances during treatment. Instruct caregivers and/or patients to seek medical advice if psychiatric symptoms develop. Ophthalmic Adverse Reactions: Cataracts, glaucoma, and central serous chorioretinopathy have been reported with prolonged use of high doses. Monitor patients for blurred vision or other visual disturbances, and if they occur, refer them to an ophthalmologist. Gastrointestinal Adverse Reactions: There is an increased risk of gastrointestinal perforation in patients with certain gastrointestinal disorders. Signs of gastrointestinal perforation, such as peritoneal irritation, may be masked in patients receiving corticosteroids. Corticosteroids should be used with caution if there is a probability of impending perforation, abscess, or other pyogenic infections; diverticulitis, fresh intestinal anastomoses, and active or latent peptic ulcer. Concurrent administration of corticosteroids with nonsteroidal anti-inflammatory drugs (NSAIDs) may increase the risk of gastrointestinal adverse reactions. Monitor patients receiving corticosteroids and concomitant NSAIDs for gastrointestinal adverse reactions. Risk of Kaposi's Sarcoma with Use of a Dosage Greater Than Replacement: Kaposi's sarcoma has been reported to occur in patients receiving corticosteroid therapy, most often for chronic conditions at a dosage greater than replacement (supraphysiologic dosage). If patients take a supraphysiologic chronic dosage of KHINDIVI, they are at increased risk of developing Kaposi's sarcoma. Vaccination: Administration of live vaccines may be acceptable in KHINDIVI-treated pediatric patients with adrenocortical insufficiency who receive replacement corticosteroids. Adverse Reactions The serious adverse reactions associated with KHINDIVI are adrenal crisis, systemic adverse reactions due to inactive ingredients, immunosuppression and increased risk of infection with dosage greater than replacement, Cushing's Syndrome, growth retardation, Kaposi's Sarcoma risk, psychiatric, ophthalmic and gastrointestinal adverse reactions. To report a suspected adverse event related to KHINDIVI, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or the U.S. Food and Drug Administration (FDA) at or call 1-800-FDA-1088. Please see full Prescribing Information for more information. INDICATION AND IMPORTANT SAFETY INFORMATION Contraindication Hypersensitivity to hydrocortisone or any of the ingredients in ALKINDI SPRINKLE. Warnings and Precautions Adrenal Crisis: Undertreatment or sudden discontinuation of therapy may lead to symptoms of adrenal insufficiency, adrenal crisis, and death. Adrenal crisis may also be induced by stressor events, such as infections or surgery. Monitor patients closely when switching from other forms of hydrocortisone to ALKINDI SPRINKLE. Instruct patients and/or caregivers to contact their healthcare provider if the full dose of ALKINDI SPRINKLE is not administered, as a repeat dose may be required. Increase the dose during periods of stress. Switch patients who are vomiting, severely ill, or unable to take oral medications to parenteral corticosteroid formulations. Immunosuppression and Increased Risk of Infection with Use of a Dosage Greater Than Replacement: Use of a greater than replacement dosage can suppress the immune system and increase the risks of new infections or exacerbation of latent infections with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic infections. Monitor patients for signs and symptoms of infections. Growth Retardation: Long-term use in excessive doses may cause growth retardation. Use the minimum dosage of ALKINDI SPRINKLE to achieve desired clinical response and monitor the patient's growth. Cushing's Syndrome Due to Use of Excessive Doses of Corticosteroids: Prolonged use with supraphysiologic doses may cause Cushing's syndrome. Monitor patients for signs and symptoms of Cushing's syndrome every 6 months; pediatric patients under one year of age may require more frequent monitoring. Decrease in Bone Mineral Density: Corticosteroids decrease bone formation and increase bone resorption, which may lead to inhibition of bone growth and development of osteoporosis. Use the minimum dosage of ALKINDI SPRINKLE to achieve desired clinical response. Psychiatric Adverse Reactions: Use may be associated with severe psychiatric adverse reactions, such as euphoria, mania, psychosis with hallucinations and delirium, or depression. Symptoms typically emerge within a few days or weeks of starting the treatment. Most reactions resolve after either dose reduction or withdrawal, although specific treatment may be necessary. Monitor patients for behavioral and mood disturbances during treatment. Instruct caregivers and/or patients to seek medical advice if psychiatric symptoms develop. Ophthalmic Adverse Reactions: Cataracts, glaucoma, and central serous chorioretinopathy have been reported with prolonged use of high doses. Monitor patients for blurred vision or other visual disturbances, and if they occur, refer them to an ophthalmologist. Gastrointestinal Adverse Reactions: There is an increased risk of gastrointestinal perforation in patients with certain gastrointestinal disorders. Signs of gastrointestinal perforation, such as peritoneal irritation, may be masked in patients receiving corticosteroids. Corticosteroids should be used with caution if there is a probability of impending perforation, abscess, or other pyogenic infections; diverticulitis; fresh intestinal anastomoses; and active or latent peptic ulcer. Concurrent administration of corticosteroids with nonsteroidal anti-inflammatory drugs (NSAIDs) may increase the risk of gastrointestinal adverse reactions. Monitor patients receiving corticosteroids and concomitant NSAIDs for gastrointestinal adverse reactions. Risk of Kaposi's Sarcoma with Use of a Dosage Greater Than Replacement: Kaposi's sarcoma has been reported to occur in patients receiving corticosteroid therapy, most often for chronic conditions at a dosage greater than replacement (supraphysiologic dosage). If patients take a supraphysiologic chronic dosage of ALKINDI SPRINKLE, they are at increased risk of developing Kaposi's sarcoma. Vaccination: Administration of live vaccines may be acceptable in ALKINDI SPRINKLE-treated pediatric patients with adrenocortical insufficiency who receive replacement corticosteroids. Adverse Reactions Common adverse reactions for corticosteroids include fluid retention, alteration in glucose tolerance, elevation in blood pressure, behavioral and mood changes, increased appetite, and weight gain. To report a suspected adverse event related to ALKINDI SPRINKLE, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or the U.S. Food and Drug Administration (FDA) at or call 1-800-FDA-1088. INDICATION ALKINDI SPRINKLE is a corticosteroid indicated for replacement therapy in pediatric patients with adrenocortical insufficiency. Please see full Prescribing Information for more information. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eight commercial rare disease products: KHINDIVI®, INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at 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: InvestorsLisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793 E: [email protected]
Yahoo
28-05-2025
- Business
- Yahoo
Eton Pharmaceuticals Announces U.S. FDA Approval for KHINDIVI™ (hydrocortisone) Oral Solution
• KHINDIVI is the first and only FDA-approved hydrocortisone oral solution• Commercial launch expected the week of June 2nd• Eton expects combined peak sales of KHINDIVI and ALKINDI SPRINKLE® (hydrocortisone) oral granules to exceed $50 million DEER PARK, Ill., May 28, 2025 (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 announced the U.S. Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for KHINDIVI™ (hydrocortisone) Oral Solution as a replacement therapy in pediatric patients five years of age and older with adrenocortical insufficiency. KHINDIVI is the only FDA-approved oral solution formulation of hydrocortisone. It comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, and to offer simple and accurate dosing specifically tailored to each patient's needs. It does not require refrigeration, mixing, or shaking – it is a ready-to-use oral liquid solution. KHINDIVI is designed to offer administration simplicity and dosing accuracy, and to provide a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube. 'The FDA approval of KHINDIVI is a tremendous achievement for Eton and more importantly, a pivotal step forward for pediatric patients with adrenal insufficiency. As a home-grown program, our team expertly navigated the development, clinical and regulatory pathway. In addition, being in a position to commercialize KHINDIVI within days of this approval is a further testament to the executional excellence from our entire company,' said Sean Brynjelsen, CEO of Eton Pharmaceuticals. 'For decades, patients have been seeking an FDA-approved hydrocortisone liquid that allows incremental, accurate dosing in the preferred dosage form for children. We are excited to now make it available to patients. Our commercial team is fully mobilized and ready to hit the ground running within the first week of approval. We're committed to ensuring that pediatric endocrinologists across the country are aware of this important new treatment option,' continued Brynjelsen. 'Managing adrenal insufficiency in pediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments that address the individualized pharmacokinetic needs of each child,' said Dr. Kyriakie Sarafoglou, Professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota. 'The availability of an FDA-approved oral hydrocortisone liquid solution offers physicians a new tool to dose patients accurately, which is important to clinical outcomes during this dynamic period of growth and development.' 'For families facing the daily challenges of pediatric congenital adrenal hyperplasia (CAH), timely access to the right treatments is critical,' said Dina Matos, Executive Director of the CARES Foundation—the only U.S. organization solely focused on the CAH community. 'The introduction of KHINDIVI is a significant advancement, particularly because accurately splitting pills to achieve proper dosing for children has long been a struggle. The ability to dose patients more accurately is critical for treatment outcomes. We commend Eton for working to make this therapy accessible through specialty channels. This marks meaningful progress for our community and a vital step toward easing the daily burden on parents and caregivers.' KHINDIVI will be promoted by Eton's existing team of pediatric endocrinology rare disease specialists. Eton currently commercializes ALKINDI SPRINKLE (hydrocortisone) Oral Granules, which is FDA-approved for pediatric patients with adrenocortical insufficiency. The addition of KHINDIVI will provide adrenal insufficiency patients and caregivers with an additional option when choosing the treatment that best meets their individual needs. Adrenocortical insufficiency is a rare, but serious condition in which the adrenal glands do not produce sufficient cortisol. Eton estimates that there are more than 5,000 adrenal insufficiency patients in the U.S. between the ages of 5 and 17, and expects peak sales of KHINDIVI, combined with ALKINDI SPRINKLE, will exceed $50 million per year. KHINDIVI will be available in the coming days in the United States exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pays for qualifying patients. Clinicians seeking to prescribe KHINDIVI can e-prescribe by selecting Anovo #5 or fax in a patient referral form to 855-813-2039. Additional product details can be found on the product website, INDICATION KHINDIVI is a corticosteroid indicated as replacement therapy in pediatric patients 5 years of age and older with adrenocortical insufficiency. Limitation of Use KHINDIVI is not approved for increased dosing during periods of stress or acute events. Use a different hydrocortisone-containing drug product for stress dosing. IMPORTANT SAFETY INFORMATION Contraindication Hypersensitivity to hydrocortisone or any of the other ingredients in KHINDIVI oral solution. Warnings and Precautions Adrenal Crisis: Undertreatment or sudden discontinuation of therapy with KHINDIVI may lead to symptoms of adrenal insufficiency, adrenal crisis, and death. Adrenal crisis may also be induced by stress events, such as infections or surgery when patients require higher doses of corticosteroids. During periods of stress (e.g., infections, surgery), switch to another oral hydrocortisone product and increase the dose if oral medications are tolerated. Monitor patients when switching to KHINDIVI to ensure KHINDIVI is providing the same level of hydrocortisone exposure as the previously used oral hydrocortisone formulation. If symptoms of adrenal insufficiency occur, increase the total daily dosage of KHINDIVI. Systemic Adverse Reactions Due to Inactive Ingredients: Hyperosmolarity KHINDIVI is not approved in pediatric patients less than 5 years of age. The inactive ingredients polyethylene glycol 400, propylene glycol, and glycerin undergo substantial systemic absorption, individually or in combination, resulting in increased plasma osmolarity in all pediatric patients, especially in pediatric patients less than 5 years of age. Monitor pediatric patients using KHINDIVI for signs and symptoms consistent with hyperosmolarity. Metabolic Acidosis and Other Adverse Reactions The inactive ingredient polyethylene glycol 400 and propylene glycol that may result in metabolic acidosis, hypoglycemia, hepato-renal injury, and central nervous system toxicity (e.g., seizure and coma), may increase the risk of adrenal crisis. Monitor laboratory values and for physical signs and symptoms of these adverse reactions. Laxative Effects Due to Inactive Ingredients The inactive ingredients polyethylene glycol 400 and glycerin, whether alone or in combination, may cause gastrointestinal irritation resulting in vomiting and/or diarrhea. These gastrointestinal reactions may increase the risk of adrenal crisis. Monitor for signs or symptoms of gastrointestinal irritation and associated fluid and electrolyte abnormalities. Immunosuppression and Increased Risk of infection with Use of a Dosage Greater Than Replacement: The use of a greater than replacement dosage can suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Monitor for the development of infection and consider dosage reduction as needed. Growth Retardation: Long-term use in excessive doses may cause growth retardation. Use the minimum dosage of KHINDIVI to achieve desired clinical response and monitor the patient's growth. Cushing's Syndrome Due to Use of Excessive Doses of Corticosteroids: Prolonged use with supraphysiologic doses may cause Cushing's syndrome. Monitor patients for signs and symptoms of Cushing's syndrome every 6 months. Decrease in Bone Mineral Density: Corticosteroids decrease bone formation and increase bone resorption which may lead to the development of osteoporosis. Use the minimum dosage of KHINDIVI to achieve desired clinical response. Psychiatric Adverse Reactions: Use may be associated with severe psychiatric adverse reactions, such as euphoria, mania, psychosis with hallucinations and delirium, or depression. Symptoms typically emerge within a few days or weeks of starting the treatment. Most reactions resolve after either dose reduction or withdrawal, although specific treatment may be necessary. Monitor patients for behavioral and mood disturbances during treatment. Instruct caregivers and/or patients to seek medical advice if psychiatric symptoms develop. Ophthalmic Adverse Reactions: Cataracts, glaucoma, and central serous chorioretinopathy have been reported with prolonged use of high doses. Monitor patients for blurred vision or other visual disturbances, and if they occur, refer them to an ophthalmologist. Gastrointestinal Adverse Reactions: There is an increased risk of gastrointestinal perforation in patients with certain gastrointestinal disorders. Signs of gastrointestinal perforation, such as peritoneal irritation, may be masked in patients receiving corticosteroids. Corticosteroids should be used with caution if there is a probability of impending perforation, abscess, or other pyogenic infections; diverticulitis, fresh intestinal anastomoses, and active or latent peptic ulcer. Concurrent administration of corticosteroids with nonsteroidal anti-inflammatory drugs (NSAIDs) may increase the risk of gastrointestinal adverse reactions. Monitor patients receiving corticosteroids and concomitant NSAIDs for gastrointestinal adverse reactions. Risk of Kaposi's Sarcoma with Use of a Dosage Greater Than Replacement: Kaposi's sarcoma has been reported to occur in patients receiving corticosteroid therapy, most often for chronic conditions at a dosage greater than replacement (supraphysiologic dosage). If patients take a supraphysiologic chronic dosage of KHINDIVI, they are at increased risk of developing Kaposi's sarcoma. Vaccination: Administration of live vaccines may be acceptable in KHINDIVI-treated pediatric patients with adrenocortical insufficiency who receive replacement corticosteroids. Adverse Reactions The serious adverse reactions associated with KHINDIVI are adrenal crisis, systemic adverse reactions due to inactive ingredients, immunosuppression and increased risk of infection with dosage greater than replacement, Cushing's Syndrome, growth retardation, Kaposi's Sarcoma risk, psychiatric, ophthalmic and gastrointestinal adverse reactions. To report a suspected adverse event related to KHINDIVI, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or the U.S. Food and Drug Administration (FDA) at or call 1-800-FDA-1088. Please see full Prescribing Information for more information. INDICATION AND IMPORTANT SAFETY INFORMATION Contraindication Hypersensitivity to hydrocortisone or any of the ingredients in ALKINDI SPRINKLE. Warnings and Precautions Adrenal Crisis: Undertreatment or sudden discontinuation of therapy may lead to symptoms of adrenal insufficiency, adrenal crisis, and death. Adrenal crisis may also be induced by stressor events, such as infections or surgery. Monitor patients closely when switching from other forms of hydrocortisone to ALKINDI SPRINKLE. Instruct patients and/or caregivers to contact their healthcare provider if the full dose of ALKINDI SPRINKLE is not administered, as a repeat dose may be required. Increase the dose during periods of stress. Switch patients who are vomiting, severely ill, or unable to take oral medications to parenteral corticosteroid formulations. Immunosuppression and Increased Risk of Infection with Use of a Dosage Greater Than Replacement: Use of a greater than replacement dosage can suppress the immune system and increase the risks of new infections or exacerbation of latent infections with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic infections. Monitor patients for signs and symptoms of infections. Growth Retardation: Long-term use in excessive doses may cause growth retardation. Use the minimum dosage of ALKINDI SPRINKLE to achieve desired clinical response and monitor the patient's growth. Cushing's Syndrome Due to Use of Excessive Doses of Corticosteroids: Prolonged use with supraphysiologic doses may cause Cushing's syndrome. Monitor patients for signs and symptoms of Cushing's syndrome every 6 months; pediatric patients under one year of age may require more frequent monitoring. Decrease in Bone Mineral Density: Corticosteroids decrease bone formation and increase bone resorption, which may lead to inhibition of bone growth and development of osteoporosis. Use the minimum dosage of ALKINDI SPRINKLE to achieve desired clinical response. Psychiatric Adverse Reactions: Use may be associated with severe psychiatric adverse reactions, such as euphoria, mania, psychosis with hallucinations and delirium, or depression. Symptoms typically emerge within a few days or weeks of starting the treatment. Most reactions resolve after either dose reduction or withdrawal, although specific treatment may be necessary. Monitor patients for behavioral and mood disturbances during treatment. Instruct caregivers and/or patients to seek medical advice if psychiatric symptoms develop. Ophthalmic Adverse Reactions: Cataracts, glaucoma, and central serous chorioretinopathy have been reported with prolonged use of high doses. Monitor patients for blurred vision or other visual disturbances, and if they occur, refer them to an ophthalmologist. Gastrointestinal Adverse Reactions: There is an increased risk of gastrointestinal perforation in patients with certain gastrointestinal disorders. Signs of gastrointestinal perforation, such as peritoneal irritation, may be masked in patients receiving corticosteroids. Corticosteroids should be used with caution if there is a probability of impending perforation, abscess, or other pyogenic infections; diverticulitis; fresh intestinal anastomoses; and active or latent peptic ulcer. Concurrent administration of corticosteroids with nonsteroidal anti-inflammatory drugs (NSAIDs) may increase the risk of gastrointestinal adverse reactions. Monitor patients receiving corticosteroids and concomitant NSAIDs for gastrointestinal adverse reactions. Risk of Kaposi's Sarcoma with Use of a Dosage Greater Than Replacement: Kaposi's sarcoma has been reported to occur in patients receiving corticosteroid therapy, most often for chronic conditions at a dosage greater than replacement (supraphysiologic dosage). If patients take a supraphysiologic chronic dosage of ALKINDI SPRINKLE, they are at increased risk of developing Kaposi's sarcoma. Vaccination: Administration of live vaccines may be acceptable in ALKINDI SPRINKLE-treated pediatric patients with adrenocortical insufficiency who receive replacement corticosteroids. Adverse Reactions Common adverse reactions for corticosteroids include fluid retention, alteration in glucose tolerance, elevation in blood pressure, behavioral and mood changes, increased appetite, and weight gain. To report a suspected adverse event related to ALKINDI SPRINKLE, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or the U.S. Food and Drug Administration (FDA) at or call 1-800-FDA-1088. INDICATION ALKINDI SPRINKLE is a corticosteroid indicated for replacement therapy in pediatric patients with adrenocortical insufficiency. Please see full Prescribing Information for more information. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eight commercial rare disease products: KHINDIVI®, INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at 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:InvestorsLisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@ MediaEliza Schleifstein, ES MediaT: 917-763-8106E: eliza@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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21-05-2025
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individual investors who own 43% along with institutions invested in Eton Pharmaceuticals, Inc. (NASDAQ:ETON) saw increase in their holdings value last week
Significant control over Eton Pharmaceuticals by individual investors implies that the general public has more power to influence management and governance-related decisions A total of 25 investors have a majority stake in the company with 50% ownership Institutions own 34% of Eton Pharmaceuticals We check all companies for important risks. See what we found for Eton Pharmaceuticals in our free report. A look at the shareholders of Eton Pharmaceuticals, Inc. (NASDAQ:ETON) can tell us which group is most powerful. We can see that individual investors own the lion's share in the company with 43% ownership. Put another way, the group faces the maximum upside potential (or downside risk). While individual investors were the group that reaped the most benefits after last week's 15% price gain, institutions also received a 34% cut. Let's take a closer look to see what the different types of shareholders can tell us about Eton Pharmaceuticals. See our latest analysis for Eton Pharmaceuticals Many institutions measure their performance against an index that approximates the local market. So they usually pay more attention to companies that are included in major indices. As you can see, institutional investors have a fair amount of stake in Eton Pharmaceuticals. This implies the analysts working for those institutions have looked at the stock and they like it. But just like anyone else, they could be wrong. When multiple institutions own a stock, there's always a risk that they are in a 'crowded trade'. When such a trade goes wrong, multiple parties may compete to sell stock fast. This risk is higher in a company without a history of growth. You can see Eton Pharmaceuticals' historic earnings and revenue below, but keep in mind there's always more to the story. Our data indicates that hedge funds own 18% of Eton Pharmaceuticals. That worth noting, since hedge funds are often quite active investors, who may try to influence management. Many want to see value creation (and a higher share price) in the short term or medium term. Looking at our data, we can see that the largest shareholder is Opaleye Management Inc. with 11% of shares outstanding. EcoR1 Capital, LLC is the second largest shareholder owning 7.2% of common stock, and Nantahala Capital Management, LLC holds about 4.4% of the company stock. In addition, we found that Sean Brynjelsen, the CEO has 4.0% of the shares allocated to their name. A deeper look at our ownership data shows that the top 25 shareholders collectively hold less than half of the register, suggesting a large group of small holders where no single shareholder has a majority. Researching institutional ownership is a good way to gauge and filter a stock's expected performance. The same can be achieved by studying analyst sentiments. There are plenty of analysts covering the stock, so it might be worth seeing what they are forecasting, too. While the precise definition of an insider can be subjective, almost everyone considers board members to be insiders. The company management answer to the board and the latter should represent the interests of shareholders. Notably, sometimes top-level managers are on the board themselves. I generally consider insider ownership to be a good thing. However, on some occasions it makes it more difficult for other shareholders to hold the board accountable for decisions. We can report that insiders do own shares in Eton Pharmaceuticals, Inc.. It has a market capitalization of just US$525m, and insiders have US$27m worth of shares, in their own names. Some would say this shows alignment of interests between shareholders and the board. But it might be worth checking if those insiders have been selling. The general public-- including retail investors -- own 43% stake in the company, and hence can't easily be ignored. While this size of ownership may not be enough to sway a policy decision in their favour, they can still make a collective impact on company policies. While it is well worth considering the different groups that own a company, there are other factors that are even more important. I like to dive deeper into how a company has performed in the past. You can access this interactive graph of past earnings, revenue and cash flow, for free. If you are like me, you may want to think about whether this company will grow or shrink. Luckily, you can check this free report showing analyst forecasts for its future. NB: Figures in this article are calculated using data from the last twelve months, which refer to the 12-month period ending on the last date of the month the financial statement is dated. This may not be consistent with full year annual report figures. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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14-05-2025
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Q1 2025 Eton Pharmaceuticals Inc Earnings Call
David Krempa; Chief Business Officer; Eton Pharmaceuticals Inc Sean Brynjelsen; President, Chief Executive Officer, Director; Eton Pharmaceuticals Inc James Gruber; Chief Financial Officer, Treasurer, Company Secretary; Eton Pharmaceuticals Inc Chase Knickerbocker; Analyst; Craig-Hallum Madison El-Saadi; Analyst; B. Riley Swayampakula Ramakanth; Analyst; H.C. Wainwright Operator Good afternoon, and welcome to the Eton Pharmaceuticals first quarter 2025 financial results conference call. (Operator Instructions) Please be advised that this call is being recorded at the company's request. At this time, I'd like to turn it over to David Krempa, Chief Business Officer at Eton Pharmaceuticals. Please proceed. David Krempa Thank you, operator. Good afternoon, everyone, and welcome to Eton's first quarter 2025 conference call. This afternoon, we issued a press release that outlines the topics we plan to discuss on today's call. The release is available on our website, Joining me on our call today, we have Sean Brynjelsen, our CEO; James Gruber, our CFO; and Ipek Erdogan-Trinkaus, our Chief Commercial Officer. In addition to taking live questions on today's call, we will be answering questions that are e-mailed to us. Investors can send their questions to investorrelations@ Before we begin, I would like to remind everyone that remarks made during this call may contain forward-looking statements and involve risks and uncertainties that could cause actual results to differ materially from those contained in these forward-looking statements. Please see the forward-looking statements disclaimer in our earnings release and the risk factors in the company's filings with the SEC. Now I will turn the call over to our CEO, Sean Brynjelsen. Sean Brynjelsen Thank you, David. Good afternoon, everyone, and thank you for joining us today. If you were aware or with us for our Investor Day in March, you heard about Eton's transformational 2024, and I'm proud to say the momentum has continued into 2025. Our existing products are generating strong growth. We recently added 2 high-value commercial assets to our portfolio with the acquisition and relaunch of Increlex and Galzin, and we've advanced our pipeline candidates, setting the stage for 2 potential approvals in the next 9 months. We've seen now sequential growth in product revenue for 17 straight quarters since the launch of Alkindi Sprinkle. Though we are very proud of this track record of commercial execution, we are just getting started. With attractive growth prospects for our existing products and a strong late-stage pipeline, we expect this streak to continue well into the future, and we're very excited about what lies ahead. I'd like to begin with one of our important new products, Increlex. Increlex is a complex biologic product used in treating patients 2 years of age and older who suffer from severe primary insulin-like growth factor 1deficiency. SPIGFD affects an estimated 200 children in the United States. When we signed the transaction in the fourth quarter, we were very excited about the deal given the strong strategic fit within pediatric endocrinology and what we saw is a very attractive growth opportunity. We are now nearly 5 months into the transaction, and I am pleased to say that it is exceeding our expectations. Eton saw a tremendous opportunity to leverage our existing pediatric endocrinology sales force and commercial infrastructure as well as make new investments into community initiatives to raise awareness of this ultra-rare condition, which unfortunately had seen an increased number of children going undiagnosed in recent years. At its peak, more than a year or a decade ago, Increlex had 185 patients in the United States, but that number has been declining for years and was at only 67 patients when we completed our acquisition in late December. With the significant investments we have made plus the hard work of our commercial team over the last 5 months, I am pleased to say that the trend appears to have reversed. We've now reached over 90 active patients and remain confident that we can reach our goal of 100 patients by the end of this year and even higher levels in the years to come. Missed diagnoses have been a long-standing problem with this condition. When physicians are presented with short of stature patients and IGF-1 deficiency is not necessarily top of mind. And there's an ingrained habit of automatically prescribing growth hormones, SPIGFD patients generally have normal growth hormone levels, so this treatment is ineffective and delays a proper diagnosis, causing SPIGFD patients to miss the Increlex treatment window. We feel that it's important for all shorter stature patients to be screened for severe primary IGF-1 deficiency and are using our deep relationships in the pediatric endocrinology community to drive greater awareness of SPIGFD and push for the screening. Eton is also working to expand access for US patients by seeking to harmonize the US and EU labels. The definition of what constitutes severe primary IGF-1 deficiency differs between the regions. While the height criteria is consistent, the IGF level is not. In the United States, a patient's IGF level must be at least 3 standard deviations below the median. While in the EU, patients must be in the bottom 2.5 percentile for their age and gender, which translates to approximately 2 standard deviations. We have completed statistical analysis of the patient registry that has tracked hundreds of Increlex patients over the last decade, and we believe it shows that the product is safe and effective in the slightly broader EU label population. We expect to submit a supplemental filing to the FDA in the second half of 2025. By harmonizing the 2 definitions, we estimate that up to 1,000 US patients could clinically benefit, significantly expanding treatment opportunities beyond the current 200 patients. We are proud of the team's hard work to close, integrate and relaunch Increlex that the growth achieved in such a short period of time is impressive, and it is clear that the long-term growth opportunity remains with or without the label harmonization. During the quarter, we announced the out-licensing of Increlex' international rights to Esteve Pharmaceuticals for an upfront payment of $4.3 million. Increlex's international opportunity is relatively small, highly fragmented across more than 30 different countries and has slim margins. In addition, the out-licensing eliminated the need (inaudible) million G&A investment to support global infrastructure, maintain foreign regulatory approvals and facilitate commercial and distribution activities. We believe the transaction will be far more additive to Eton's profitability than if we had commercialized the product internationally ourselves. The transaction also provided us with a $4.3 million upfront payment, recouping a meaningful portion of our initial purchase price and providing excess capital that can be reinvested into our attractive pipeline opportunities. And finally, it eliminated the distraction and resource burden that would have come with managing more than 30 territories and several other team members to fully dedicate their time and attention to our 3 high-value US product launches in 2025. Esteve shares a similar philosophy of putting patients first. So we are confident that they will be a good partner for the product and ensure that the international patients have access to this meaningful therapy. Turning now to Alkindi. This has been a strong growth driver for us for 17 straight quarters with no signs of slowing down. In fact, the pace of referrals has actually increased in 2025. As you may remember, beginning in January, we made our existing sales force 100% focused on pediatric endocrinology. We believe this has driven increased efficiency, which is apparent not only in the strong launch of Increlex, but also in an increased rate of new Alkindi Sprinkle prescriptions. Through April, the number of new patient referrals received this year exceeds the first 4 months of any other year since launch. Despite the relatively high discontinuation rate, we have still added a significant number of new patients this year and are quickly closing in on our 500 active patients. Eton has been encouraged by Alkindi's strong start to the year and are expecting the rate of new patient adds to our adrenal insufficiency franchise to accelerate even further in the second half of 2025 with the anticipated launch of ET-400. As we have discussed at length, we continue to see a large portion of the market using unapproved compounded hydrocortisone due to the preference for a liquid dosage form. We believe approximately 50% of young children are using a non-FDA liquid hydrocortisone today. With an estimated 5,000 adrenal insufficiency patients under 9 years of age, we continue to see a very compelling market opportunity for ET-400. ET-400's PDUFA date is just 2 weeks away, and we are prepared to launch quickly upon potential approval. We've manufactured our launch inventory in our specialty sales force and promotional campaigns are ready to go live. We have been engaged in communications with the agency throughout the review and we're optimistic that they will meet their PDUFA goal date of May 28. It's a very exciting time at Eton. After many years of hard work, our team is very excited to be on the cusp of making this important medication available to the patients in need. Transitioning now to another significant opportunity. The treatment of Wilson's disease or Wilson disease, more correctly, a rare genetic disorder that causes excessive copper accumulation in the body. Patients suffering from this condition do not metabolize copper normally with their bodies absorbing the copper instead, prevents it from leak in the body and Galzin is an FDA-approved treatment for patients with Wilson disease who have been initially treated with a chelating agent. It is the only FDA-approved zinc therapy for Wilson disease today. As with Increlex, we (inaudible) Galzin as an acquisition because we saw significant opportunities for Eton to add value, grow the product and improve the outcomes for patients. And similar to severe primary IGF-1 deficiency, Wilson disease is a severely underdiagnosed condition with a lack of product investment leading to inadequate awareness and education. Wilson disease is estimated to impact approximately 10,000 people in the United States, but we estimate that only 2,000 of those patients are diagnosed and actively on a therapy today. Unfortunately, most patients are not diagnosed until they are in their 20s or 30s when symptoms begin to present after years of excessive copper buildup. This delayed diagnosis leads to worse outcomes, including neurological damage and liver failure. The increased frequency of genetic testing in recent years has led to earlier diagnosis, allowing patients to proactively start zinc therapy before liver or other damage occurs, but an unmet need still remains. Of the roughly 2,000 patients that have been diagnosed and are on treatment, we estimate approximately 800 use zinc therapy, while the remainder are on chelating agents. However, due to historical challenges with access, affordability and awareness, most patients on zinc therapy appear to be using over-the-counter supplements rather than the FDA-approved prescription product. The nutritional supplements are a different form of zinc, which have been shown to be less effective than Galzin. We acquired Galzin because we feel that we are the right company to address this dynamic. We relaunched the product in March with robust patient services, including a $0 co-pay. For the first time ever, every Wilson disease patient who wants Galzin can access it regardless of insurance status. We believe Eton Cares is one of the most generous high-touch patient assistant programs in the industry and one of the many things that sets us apart from other rare disease companies. Our relaunch has received a warm reception from the community, including patient advocates and leading Wilson disease physician. We are currently migrating patients to Eton's commercial infrastructure and Eton Cares program. This migration kicked off in March and will be a multi-month process as the previous pharmacies work through inventories that remained in the channel. We expect the conversion to be largely complete by the end of the quarter -- or the third quarter, at which time Galzin should begin producing meaningful revenue. I'm pleased to be able to solve the access and affordability issues that have impacted Galzin users for more than a decade. However, we believe that there is more that can be done to improve the lives and outcomes of Wilson disease patients. After access and affordability, the 2 most common complaints about zinc therapy are the burdensome, dosing requirements and unpleasant GI side effects. We've set out to tackle these challenges with the development of ET-700, which we disclosed for the first time in March. ET-700 is an extended-release version of Galzin, which we believe will eliminate the need for 3 times per day dosing as well as potentially reduce the GI side effects that are reported by some patients on zinc therapy today. We initiated ET-700 development last year prior to the acquisition of Galzin and have now filed a patent on our proprietary formulation. We're now advancing this program at full speed and working with the top Wilson disease thought leaders to prepare a clinical study protocol. We are preparing for the manufacturing registration batches later this year and have a meeting with the FDA in the second quarter to discuss our proposed clinical program. If everything goes as planned, we expect to file an NDA in 2027. We believe this product candidate has the potential to generate more than $100 million of peak revenue. Our metabolic portfolio, which includes Carglumic Acid, Betaine, Nitisinone and GoLike continues to provide steady revenue and cash flow. The group of products produced a solid year-over-year growth in the first quarter, but will have reduced significance to Eton going forward due to the rapidly increasing revenue contributions from our high-margin pediatric endocrinology products. Turning now to our development pipeline. During the quarter, we were pleased to report that ET-600 passed its pivotal bioequivalency study, which allowed us to submit an NDA for the product in late April. ET-600 is Eton's proprietary patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. Leading pediatric endocrinologists have long expressed the need for this product because it allows for the small precise and titratable doses required to treat pediatric patients. ET-600 is the same pediatric endocrinology prescriber base as Alkindi Sprinkle, ET-400 and Increlex. This should provide an important head start once commercialization activities begin. We expect our application to be assigned a 10-month review, which would allow for an approval and launch potentially as early as the first quarter of 2026. Pre-launch commercial activities are already underway, and we are excited about the prospects for this important product. Eton is also continuing to advance Amglidia, which we acquired late last year. Amglidia is designed for the treatment of the ultra-rare condition of neonatal diabetes mellitus, which impacts an estimated 300 children in the United States and is within our pediatric endocrinology call plan. Although the product has been approved in the EU since 2018, there are currently no FDA-approved oral treatments for the condition, and therefore, it is not possible for infants in the US to receive the correct dose in an FDA-approved manner. Today, caregivers must either obtain a suspension from a compounding pharmacy or crush adult tablets to create a suspension at home. Similar to ET-400 and ET-600, Amglidia gives an opportunity to bring a liquid formulation to the market to provide precise and accurate pediatric dosing. Our acquisition terms allow us to have an FDA meeting to receive confirmation of the clinical pathway before any payment occurred. This meeting occurred in April, and we were pleased with how it went. The FDA was receptive to what we believe is a feasible clinical pathway to bring this critical treatment to patients in the US In the first quarter, we also unveiled another new internal development program, ET-800. Our French development partner, CROSSJECT, will continue to work on advancing the Zeneo hydrocortisone autoinjector and separately Eton will manage the development of this injectable vial product, which we are calling ET-800. In addition to the large retail opportunity for hydrocortisone injection, which we have discussed extensively, there was an even greater use in the hospital setting. A total of more than 5 million vials per year and approximately $100 million in sales today. Today, hospitals use a lyophilized for freeze dried powder vial, which must be manually reconstituted prior to administration. We have developed and filed a patent on a proprietary ready-to-use liquid formulation that we believe will save time, reduce risk of medical errors and is an important factor as hydrocortisone is often used in the emergency room and operating rooms. If development activities progress as planned, we expect to make registration batches in the coming months and submit an NDA in early 2027. On the business development front, we expect acquisition and licensing transactions to remain a central part of our long-term story, and we continue to evaluate new opportunities. We've demonstrated that we can successfully execute value-creating transactions, and we expect to continue to do so. However, our strong position allows us the luxury to remain disciplined and focus solely on products that are aligned with our ultra-rare disease strategy and can meet our high threshold for financial returns and value creation opportunities. As you have heard today, it's a very busy time at Eton, we have made great strides in our mission to bring as many new rare disease treatments to patients as possible. With 2 major product launches, relaunches already underway this year and the largest launch in our history, potentially a matter of days away, there's never been a more exciting time for the organization. Following the expected launch of ET-400, we see a clear path to reaching $100 million of revenue in the near term and much higher levels as our pipeline products come to market. Through years of hard work, our team has built an extraordinary organization, and we continue to make progress every day toward our goal of becoming one of the leading ultra-rare disease companies in the world. With that, I'll turn it over to James, our Chief Financial Officer, to discuss the financials and to discuss the tariff questions. James? James Gruber Thank you, Sean. I'll start by addressing the tariff situation in the US since that seems to be at the top of everyone's list of questions right now. We believe Eton would see minimal impact from any of the tariff proposals discussed to date. The majority of our products are produced in the US, Eton does not hold any IP in any foreign countries, and we do not have any intercompany transfer pricing arrangements. Our primary exposure will be with Increlex and Alkindi, which are both manufactured in Europe. However, the anticipated cost of products purchased from Europe represents less than 5% of our forecasted revenue. So a 20% tariff on European purchases would impact total company gross margin by less than 100 basis points, and the potential impact would likely be even smaller in future years since our late-stage pipeline products, ET-400 and ET-600, will also be manufactured within the US In short, we are not concerned about the impact of tariffs, but we will continue to monitor the situation closely. Turning to our financial results. Our first quarter revenue was $17.3 million compared to $8.0 million in the first quarter of 2024, an increase of 117%. Net sales during the quarter included $3.3 million of licensing revenue, of which $1.8 million was from the licensing of Increlex rights outside of the US Although Eton is receiving $4.3 million upfront, accounting guidance results in recording $1.8 million immediately with the remaining $2.5 million recognized over the licensing term. We also recorded $1.5 million of licensing revenue from a regulatory milestone event associated with our previous divestiture of DS-200. There was no licensing revenue recognized in the prior year quarter. Product sales were $14.0 million for the first quarter of 2025 compared with $8.0 million in the first quarter of 2024, an increase of 76%. This growth was driven primarily by increased sales of Alkindi Sprinkle and the addition of Increlex, which was acquired in late December. We expect product sales to continue growing quarter-over-quarter throughout the rest of 2025 and beyond, and we continue to expect to exit 2025 at an approximately $80 million annual revenue run rate. Gross profit for the quarter was $9.9 million compared with $5.0 million in the prior year period, primarily due to increased product sales. Adjusted gross profit, which excludes the impact of acquired inventory step-up adjustments and intangible amortization, was $12.0 million or 69.5% of total revenue versus $5.2 million of adjusted gross profit or 65.6% of total revenue in the prior year period. This increase was driven by continued growth of higher-margin Alkindi Sprinkle and the recognition of higher-margin licensing revenue in the first quarter of 2025. We expect to report full year 2025 adjusted gross margin of approximately 70% and long-term adjusted gross margin to exceed 75% by 2028. R&D expenses for the quarter were $1.2 million compared with $0.7 million in the prior year period, primarily due to increased expenses associated with our ET-700 and ET-800 project development activities. It's worth noting that in April, we paid a $2.2 million NDA application fee related to our ET-600 submission. That cost will be fully recorded as R&D expense in the second quarter, and we also expect to record a $500,000 expense for an Amglidia licensing payment in the second quarter of 2025. Besides these 2 one-time items, we expect R&D spending to remain largely in line with historical levels for the remainder of 2025. General and administrative expenses for the quarter were $9.2 million compared with $5.2 million in the prior year period. As mentioned in our fourth quarter call, increased SG&A expenses in 2025 were planned and necessary to build out the infrastructure needed to support the significant growth in our product portfolio and revenue base. These incremental investments include our new dedicated 5-person metabolic sales team, which launched on January 2, commercial investments made in the product relaunches of Increlex and Galzin, investment in our ET-400 launch readiness activities and additional corporate staff to support the growing portfolio in the areas of quality, regulatory and finance. On an adjusted basis, which removes the impact of share-based compensation, transaction-related costs and other one-time expenses, G&A expense was $7.3 million compared to $4.4 million in the prior year period. In addition to the planned increases in our infrastructure, SG&A expenses during the quarter were also affected by relaunch and prelaunch commercial activities in the period. We are not planning to make further significant investments in SG&A this year and anticipate that adjusted G&A spending will remain flat or slightly decline for the remainder of 2025. We believe that the investments we've made in G&A during the quarter will support revenue levels much higher than where we are today. And as a result, we expect to return to minimal G&A growth in 2026 and beyond. Adjusted EBITDA for the first quarter of 2025 was $3.7 million compared to $0.5 million in the first quarter of 2024. Total company net loss was $1.6 million for the quarter compared to a net loss of $0.8 million in the prior year period. Net loss per basic and diluted share was $0.06 during the quarter compared to net loss per basic and diluted share of $0.03 in the prior year period. On a non-GAAP basis, we reported net income of $2.4 million for the first quarter of 2025 compared to $0.2 million in the prior year period and diluted earnings per share of $0.07 for the first quarter of 2025 compared to $0.00 in the prior year period. Eton finished the first quarter with $17.4 million of cash-on-hand, and we generated $2.1 million of operating cash flow during the quarter. This concludes our remarks on first quarter results. And with that, we'll turn it over to the operator for Q&A. Operator (Operator Instructions) Chase Knickerbocker, Craig-Hallum. Chase Knickerbocker Congrats on the results here. Just first on Increlex. Sean, have you had your meeting with FDA to discuss that label expansion at this point? Sean Brynjelsen The meeting request has gone in. So we'll be looking forward to having that, I would say, in the coming weeks, possibly most likely July, in my opinion, and then it would follow ideally with the submission in the third quarter of the actual update. We believe the data we have is compelling. It's based on patient registry data out of Europe and should support that label change. Chase Knickerbocker Great. And maybe staying on the FDA front. I appreciate the commentary on ET-400. Any additional color on any recent interaction with FDA that you can give us? I mean, are things progressing as you would expect with the review at this latest stage as in things like manufacturing inspections and final label discussions, et cetera? Sean Brynjelsen Yes. The -- typically, the last step in a review process of an NDA drug application would be the labeling discussions. And so the FDA provides commentary on your label and what I mean by that is the package insert. We've received that commentary. We have already had the submission sent back to the agency. And so we believe that should be the final step. I mean we could hear of something else, but it seems, to me, that we are on track for approval here in 2 weeks or less. Chase Knickerbocker Great. And on the Amglidia front, it sounds like you liked what you heard there. Does that mean you expect to just need a fairly simple kind of PK study to support an NDA filing there? Or just kind of any additional color? Sean Brynjelsen Yes. The -- so we like the feedback from the agency because you can tell during the tone, they would like to see us develop and get this product on market. They provided a pathway. It didn't seem like there were -- it seemed fairly clear to us and what we need to do. We did have a few clarifying questions. So we are planning a follow-up meeting with the agency to ensure that we're doing what they want and what their expectations are. But I was very encouraged by that, by the comments that we received back and I do expect that would just be a fairly straightforward PK study, as you indicated. Chase Knickerbocker Got it. Pretty impressive continued progress on Increlex, I think you said 90 patients. If I pursue through the commentary on Increlex, on Sprinkle, I mean, it doesn't take much to -- for that $80 million run rate in Q4 to start looking pretty derisked. Is there anything I'm missing there? Or maybe any comments that you would have on that Q4 run rate? Sean Brynjelsen No. I think at the appropriate time, we'll -- we feel very comfortable with the number to first address your question there. The question is, will we be revising that number at some point? I think we're going to leave it where it is right now, and we'll see where the sales go in the second quarter, but we're feeling pretty good about our sales. They've been strong across the board, and we couldn't be happier with the Increlex launch and the quick addition of patients. And we think the coming weeks here will help us to maybe provide some update on the next conference call. Operator Madison El-Saadi, B. Riley. Madison El-Saadi Yes. So could you maybe provide an update on the recent weekly Increlex prescription trends? I mean it really looks like March was a tremendous month. Is that kind of the proxy barometer going forward? And then -- Sean Brynjelsen Yes. Sure. On that point, I would say that we have significantly increased the number of patients. Now we're -- as we increase, we expect that rate would go down. You're getting closer to that whatever that 185 potential patients, let's say, that are in the US So you would -- we would expect that. But as it stands today, we're firing at all cylinders in terms of the launch on that product. We expect to hit our goals -- revenue goals or exceed them -- this year, I think we'll exceed those goals. I'd like to get a few more months under our belt, and then we'll provide an update. And I think we probably -- we did indicate that we're now in the 90s on the patient front. We'll give further updates on that rate and better answer your question in terms of the trajectory of it. Madison El-Saadi Got it. Understood. And then on ET-400, if we assume approval is on time, what's the expected timing to the first commercial revenue that you would book? Do you think this could be an end of 2Q story or maybe early 3Q? Sean Brynjelsen I'm thinking more of a Q3. We will be launching it essentially right around the approval date. So we would expect a June, let's say, first week of June launch on the product. It takes a little bit of time for the patients to get the prescription because it's -- patients have to come back to the office and the doctors have to prescribe it. So I would say there's a bit of a lag between the initial launch and then the actual revenue coming in. So from a revenue standpoint, I would really think more about that sort of Q3, Q4. It will, we believe, have a rapid uptake. We know that it's something that there's a lot of pent-up demand for an oral solution. So let's -- maybe I'll leave it there and it give you an idea. It won't have a massive impact in terms of revenue this year, but we think it will have a much more significant impact in the quarters after that. Madison El-Saadi Got it. And then lastly, I really appreciate the color on the tariff risk exposure, which looks to be really a nonstory. Any commentary to theoretical exposure to the White House Executive Order, the most favored nation policy? Sean Brynjelsen Yes. Thanks for the question. It's probably the same question on others' minds. When I first heard about that, I was -- really didn't know what to make of it. There weren't enough details that were released around that. But now that we got a chance to look and digest that, and I've also been able to do a little bit more background research on it, I do not believe it will have a meaningful impact to us. We only sell in the US So we're not selling products overseas to foreign customers at lower prices than we sell in the US We believe that alone is pretty strong in the sense that we don't have dual pricing as big pharma does. So in my opinion, this is something that would potentially impact big pharma. But we're also less reliant on Medicare, Medicaid than most pharma companies. Really no Medicare business to speak of, it's only Medicaid. And on the Medicaid, we lose money on the Increlex as I think a couple of people are involved. But we make good money on Medicaid through products like (inaudible). There are no plans for us to sell our products overseas. So that reciprocal or most favored nation pricing would not apply, we believe, for our company (inaudible) we are a rare disease company -- an ultra-rare disease company, so I think that it's a unique situation compared to more standard pharmaceuticals. Operator Swayampakula Ramakanth, H.C. Wainwright. Swayampakula Ramakanth This is RK from H.C. Wainwright. So I know you have had more experience with the relaunch of Increlex compared to Galzin. But, in general, what's the feedback from the sales force on Galzin? Anything anecdotally you can tell us about how the relaunch is going and what the expectations are internally for adoption during 2025? Sean Brynjelsen Yes. So Galzin is an important product for the company. It's placing us squarely in the Wilson disease space, and we aspire to become a key player in Wilson disease. Beyond just Galzin, obviously, we have our ET-700 product, which is an extended-release version of Galzin. That product, we think we will have clinical trial results in January. That's a small study we're running, which will show the same efficacy, we believe, as the 3 times a day dosing with hopefully less side effects. So that is really the big opportunity is the extended release version. The Galzin itself is now -- it's been great for patients because Galzin had in the past supply issues, they no longer have that. There's a $0 co-pay. They never had that in the past. They have that today. There's overnight shipping, so we get the product to them very quickly. So we believe that product will add appreciable revenue in the quarters to come. The uptake and the conversion has gone well, and we add patients weekly on Galzin. So we see a steady stream of patients coming off of, let's say, over-the-counter stuff or compounded medicines. And they were forced to go there, frankly, in the past because of the inaccessibility of the medicine and the high co-pays that were charged by the prior company. We have taken away all those blockades. So I think -- and then the last thing I want to say is we're adding a really strong relationship with the Wilson Disease Foundation and with really some of the leading prescribers in the United States that treat Wilson disease we have -- we've been able to get there -- get what their thoughts are, how we can help patients, how we can do the best job to make sure that these patients are served properly. Swayampakula Ramakanth Do you -- by any chance, are you able to give us patient numbers that are on Galzin at this point? Or we have to wait for the next update? Sean Brynjelsen I think for the next update, we'll provide patient numbers because it's still in the transition period, but I can tell you that it is going better than -- according to plan, and I'm very pleased with the launch and the job that our Chief Commercial Officer, Ipek, has done with the organization. We have a dedicated sales team in that space as -- just like we have a dedicated sales team on our other endocrinology products. Swayampakula Ramakanth Perfect. A quick question for Jim. Looking through the cash flow statement, I see an increase in the Medicaid rebates and also on the receivables. So is this -- just trying to understand what's behind those numbers and how sticky are those? James Gruber Sure. Let's start with the receivables. So significant increase there. Part of that was, call it, normal operating activity with the addition of Increlex. We talked about significant impact with Medicaid there. So with that product relaunch, a decent amount of addition -- sorry, with Increlex receivables. With the licensing revenue, even though we only recognized $1.8 million of the Esteve licensing deal revenue in the first quarter, there is a receivable payment of $4.3 million. So we have not received that yet. So a big chunk of the [$5.8] million increase in receivables was licensing revenue and then the remainder was just Increlex sales. On the Medicaid side, the increased liability was almost entirely due to Increlex, the product relaunch. So nothing out of the ordinary with the increased liability other than the fact that we have a traditional patient mix in Increlex and dollars are just higher with that product. Operator And there are no further questions in the queue at this time. Ladies and gentlemen, this concludes today's conference call. Thank you all for your participation, and you may now disconnect. Sign in to access your portfolio Error in retrieving data
