Corneal Ulcer

ROCKVILLIE, Md. and SUZHOU, China, March 6, 2024 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of cancer, metabolic, autoimmune, ophthalmology and other major diseases, today announced that the results of two clinical studies of IBI311 (anti-IGF-1R monoclonal antibody),a Phase I trial in healthy subjects and a Phase II trial in patients with thyroid eye disease (TED), in the form of oral presentation at the 39th Asia Pacific Academy of Ophthalmology (APAO) Congress and the 21st International Congress of Endocrinology (ICE), respectively. A Phase I Study of IBI311 (Anti-IGF-1R Monoclonal Antibody) in Chinese Healthy Volunteers Abstract No.: 200683 The oral presentation at the 39th APAO annual meeting was based on a single dose escalation Phase 1 clinical study (NCT05480597) to evaluate the safety and tolerability of a single intravenous infusion of IBI311 in Chinese healthy volunteers. The primary endpoint of the study was safety variables, and secondary endpoints included pharmacokinetic (PK) parameters and immunogenicity results. The results showed that IBI311 was safe and well tolerated, with the most common treatment-emergent AEs being Grade 1 or 2, transient, and most resolving without treatment. All IBI311 dose groups were tested negative for anti-drug antibodies during the study. These indicated that a single intravenous administration of IBI311 at various dose levels in healthy subjects is safe and well tolerated, and the PK profile supports dose selection for subsequent clinical development. A Phase II Study of IBI311 for the Treatment of Thyroid Eye Disease Abstract No.: # 792 The oral presentation at the 21st International Congress of Endocrinology was based on a multicenter, randomized, double-masked, placebo-controlled Phase II study (NCT05795621) to evaluate the efficacy and safety of IBI311 in subjects with moderately to severely active TED. A total of 33 eligible subjects were randomized in a 2: 1 ratio to receive either IBI311 or placebo. The primary endpoint was the proptosis response rate at Week 12 of the study eye (defined as the percentage of subjects with a reduction in proptosis of ≥2 mm from baseline in the study eye without deterioration ≥ 2 mm increase of proptosis in the fellow eye). After 12 weeks, all subjects received IBI311 until Week 21. The results of the study showed: The primary endpoint was achieved: at Week 12, the proptosis responder rate in the study eye was significantly higher in the IBI311 group than in the placebo group (59.1% vs 18.2%, OR=11. 55, P=0. 0309). With continued treatment and follow-up to week 24, the IBI311 treatment group achieved up to 72.7% proptosis responder rate, 3.37 mm reduction in proptosis from baseline, 88.2% diplopia responder rate (defined as diplopia improvement ≥ grade 1). Safety: The overall safety profile of IBI311 was favorable, with the majority of AE being mild or moderate in severity, and no treatment-emergent AE leading to drug discontinuation/interruption in the IBI311 group. These results suggested that IBI311 significantly improves the proptosis in patients with TED in 12 weeks of treatment, and can lead to clinical benefits such as further improvement of proptosis and improvement of diplopia after continuation of treatment, with a favorable safety profile. Professor Xianqun Fan, the leading principal investigator of the study, the Academician of the Chinese Academy of Engineering and professor of ophthalmology at the Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, stated, " As an organ-specific autoimmune disease closely related to thyroid disease, TED is one of the most common orbital diseases in adults, and seriously affects the visual function and appearance of patients. At present, there is no targeted therapy approved for the treatment of thyroid ophthalmopathy in China, and the options for therapeutic interventions are limited. IBI311 is currently the most advanced candidate in the clinical development in the TED therapeutic area. As the Principal Investigator of the IBI311 series of studies, I am very pleased to see the progress of IBI311 in TED presented in an oral presentation at the ophthalmology and endocrinology Congress. This is an academic recognition of Chinese studies. I also wish that IBI311 will be approved and available in the clinic in the near future to alleviate the disease burden of Chinese TED patients. " Dr. Lei Qian, Vice President of Clinical Development of Innovent, stated, "We are pleased to present the clinical development progress of IBI311 in thyroid eye disease at APAO Congress and ICE. This is another achievement of in-depth collaboration between Innovent and academia. It is also a prestigious recognition by the international ophthalmology and endocrinology academic community to Innovent IBI311's progress in the treatment of thyroid eye disease. We look forward to the approval of IBI311in the near future, providing treatments to TED patients. We will continue to make efforts to facilitate people's pursuit of a happy life and serve more patients." About Thyroid Eye Disease (TED) TED, the most common orbit-related disease in adults, is an autoimmune disease involving ocular tissues and usually associated with Graves' disease (GD) and is the most common orbit-related disease in adults. TED occurs in approximately 25 to 50% of GD patients and can also be seen in other thyroid diseases, even in euthyroidism[1]. The annual incidence of TED is estimated to be 16/100,000 in women and 2.9/100,000 in men[2]，and the estimated prevalence of clinically relevant TED ranges from 0.1% to 0.3%[3]. According to disease severity, it can be classified into mild, moderate and severe. Although TED appears to affect women more often, severe cases occur more frequently in men. Patients aged 30 to 50 years are most commonly affected, and severe cases occur more frequently in patients over 50 years[4]. At present, the pathogenesis of TED is not fully understood, but several studies have shown that OFs present in muscle fibers, orbital fibrous connective tissue space are key factors leading to orbital soft tissue enlargement in TED[5]. The natural history of TED is classified into active and inactive phases[6]. The most common symptoms are dry eye, ocular gritty, photophobia, lacrimation, diplopia, and pressure behind the eye, while typical signs include upper eyelid retraction, eyelid edema, periorbital and conjunctival edema, and proptosis. TED is usually mild to moderate, and about 3–5% of patients with TED are severe, manifesting as severe pain, vision-threatening corneal ulcers, or compressive optic neuropathy[7]. In addition to potentially affecting vision, TED can have an extremely severe impact on the patient's appearance and social functioning as well as quality of life. Currently, the first-line treatment for moderately severe active TED is intravenous glucocorticoid therapy, which suffers from unsatisfactory improvement of proptosis and systemic side effects. The second-line treatment includes other immunomodulators, which also have risks related to unclear improvement of proptosis and treatment. Teprotumumab, Tocilizumab and Rituximab are recommended by the Chinese Clinical Diagnosis and Treatment Guidelines for Thyroid Eye Disease (2022)[8], the European Group on Graves' orbitopathy (EUGOGO)[9] and the consensus on thyroid eye disease of the American Thyroid Society and the European Thyroid Society[10] as the second-line treatment options for moderate to severe active TED. Teprotumumab targeting IGF-1R is recommended as first-line therapy for patients with clinically significant proptosis. About IBI311 IBI311 is a recombinant anti-IGF-1R antibody developed by Innovent Biopharmaceuticals for the treatment of TED. IGF-1R, a transmembrane tyrosine kinase receptor that plays a role in development, metabolism, and immune regulation, is overexpressed in OFs, B, and T cells of TED patients[11]. IBI311 can bind IGF-1R, block IGF-1R signaling pathway activation mediated by IGF-1 and other related ligands or agonistic antibodies, thus reducing the expression of downstream inflammatory factors, inhibiting the synthesis of hyaluronic acid and other glycosaminoglycan caused by OFs activation, as well as related inflammatory reactions including tissue congestion and edema. IBI311 can inhibit adipocyte cellularization of OFs, thereby reducing the disease activity of patients with TED and improving proptosis, diplopia, ocular congestion and edema and other symptoms and signs. About Innovent Innovent is a leading biopharmaceutical company founded in 2011 with the mission to provide high-quality biologics that are affordable to all. The company discovers, develops, manufactures and commercializes innovative medicines that treat some of the most intractable illnesses. Its pioneering therapies to treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has 10 products in the market, 3 new drug applications under the NMPA review, 4 assets in Phase III or pivotal clinical trials and 19 more molecules in early clinical stage. Innovent partners with over 30 global healthcare leaders, including Eli Lilly, Roche, Sanofi, Adimab, Incyte and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit , or follow Innovent on Facebook and LinkedIn. Statement: Innovent does not recommend the use of any unapproved drug (s)/indication (s). Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent Biologics ("Innovent"), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or is otherwise inaccurate. References: SOURCE Innovent Biologics

ROCKVILLIE, MD. and SUZHOU, China, Feb. 19, 2024 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, today announced that the primary endpoint has been achieved in the Phase 3 registrational study (RESTORE-1) of IBI311, a recombinant anti-insulin-like growth factor 1 receptor (IGF-1R) antibody in Chinese subjects with Thyroid Eye Disease (TED). Innovent plans to submit the new drug application (NDA) for IBI311 in the treatment of TED to the Center for Drug Evaluation (CDE) of the National Drug Administration (NMPA). RESTORE-1 (CTR20223393) is a multicenter, randomized, double-masked, placebo-controlled Phase 2/3 clinical trial to evaluate the efficacy and safety of IBI311 in subjects with TED. The Phase 3 results of RESTORE-1 showed that the primary endpoint was successfully met: at Week 24, the proptosis responder rate in the study eye (the percentage of subjects with a reduction in proptosis of ≥2 mm from baseline in the study eye without deterioration ≥ 2 mm increase of proptosis in the fellow eye) was significantly higher in subjects treated with IBI311 than in subjects treated with placebo: 85.8% vs. 3.8%, with a difference of 81.9% (95% CI: 69.8% to 93.9%, P < 0.0001). In addition, the key secondary endpoints of the study such as overall response rate(the percentage of subjects with a reduction in proptosis of ≥2 mm from baseline and improvement in clinical activity score ≥2 in the study eye), percentage of subjects with a clinical activity score (CAS) of 0 or 1, and mean change in proptosis from baseline in the study eye were successfully met: IBI311 significantly improved all the above parameters as compared to the placebo. The overall safety profile of IBI311 was favorable throughout the study with no serious adverse events occurred. The efficacy and safety profiles in the Phase 3 part of the RESTORE-1 study were consistent with its Phase 2 results. Detailed results from the study will be released in medical conferences or journals in the future. As an autoimmune disease involving ocular tissues, the annual incidence of TED is estimated to be 16/100,000 in women and 2.9/100,000 in men1，and the estimated prevalence of clinically relevant TED ranges from 0.1% to 0.3%2. At present, there is no targeted drug approved for the treatment of TED in China, while multiple clinical guidelines recommend the use of IGF-IR targeted antibody in the treatment of TED3,4,5. Particularly, IGF-IR targeted antibody is recommended as first-line therapy for patients with clinically significant proptosis. Professor Xianqun Fan, the leading principal investigator of the study, the Academician of the Chinese Academy of Engineering and professor of ophthalmology at the Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, stated, " As an organ-specific autoimmune disease closely related to thyroid disease, TED is one of the most common orbital diseases in adults, and seriously affects the visual function and appearance of patients. At present, there is no targeted drug approved for the treatment of TED in China, and the therapeutic approaches are limited. As the principal investigator of the RESTORE-1 study, I am very pleased to see that IBI311 achieved the primary endpoint in the Phase 3 study and showed a favorable safety profile, which again demonstrates the excellent efficacy of IBI311. I look forward to sharing the detailed results of this high-quality study in the near future, and wish its successful NDA submission so as to bring benefits to Chinese TED patients as soon as possible." Dr. Lei Qian, Vice President of Clinical Development of Innovent, stated, "There are no targeted drugs approved in the field of TED in China so far, while the treatment costs of overseas targeted drugs are beyond many patients' reach. Driven by the focus of addressing patients' unmet medical needs, we advanced the development of IBI311 rapidly. IBI311 has demonstrated significant efficacy and favorable safety in the treatment of TED in the RESTORE-1 study. We plan to submit its NDA as soon as possible, and bring high-quality, effective and safe biological drugs to Chinese patients with TED. At the same time, with the approval of SINTBILO ® (anti-PCSK9 monoclonal antibody), the NDA acceptance of Mazdutide (GLP-1R/GCGR dual agonist), and the endpoints met in phase 3 study of IBI311, Innovent is strategically establishing its innovative commercial and late-stage portfolio in the fields of cardiovascular and metabolic diseases (CVM), endocrinology and ophthalmology. We will continue to work hard to help people pursue a healthy life." About Thyroid Eye Disease (TED) TED is an autoimmune disease involving ocular tissues and is usually associated with Graves' disease (GD) and is the most common orbit-related disease in adults. TED occurs in approximately 25 to 50% of GD patients and can also be seen in other thyroid diseases, even in euthyroidism6. The annual incidence of TED is estimated to be 16/100,000 in women and 2.9/100,000 in men1，and the estimated prevalence of clinically relevant TED ranges from 0.1% to 0.3%2. According to disease severity, it can be divided into mild, moderate and severe. Although TED appears to affect women more often, severe cases occur more frequently in men. Patients aged 30 to 50 years are most commonly affected, and severe cases occur more frequently in patients over 50 years7. At present, the pathogenesis of TED is not fully understood, but several studies have shown that orbital fibrous present in muscle fibers, orbital fibrous connective tissue space are key factors leading to orbital soft tissue enlargement in TED8. The natural history of TED is divided into active and inactive phases9. The most common symptoms are dry eye, ocular gritty, photophobia, lacrimation, diplopia, and pressure behind the eye, while typical signs include upper eyelid retraction, eyelid edema, periorbital and conjunctival edema, and proptosis. TED is usually mild to moderate, and about 3–5% of patients with TED are severe, manifesting as severe pain, vision-threatening corneal ulcers, or compressive optic neuropathy10. In addition to potentially affecting vision, TED can have an extremely severe impact on the patient's appearance and social functioning and quality of life. Currently, the first-line treatment option for moderately severe active TED is intravenous glucocorticoid therapy, which suffers from unsatisfactory improvement of proptosis and systemic side effects, and second-line treatment includes other immunomodulators, which also have risks related to unclear improvement of proptosis and treatment. Teprotumumab, Tocilizumab and Rituximab are recommended by the Chinese Clinical Diagnosis and Treatment Guidelines for Thyroid Eye Disease (2022)3, the European Group on Graves' orbitopathy (EUGOGO)4 and the consensus on thyroid eye disease of the American Thyroid Society and the European Thyroid Society5 as the second-line treatment options for moderate to severe active TED. Teprotumumab targeting IGF-1R is recommended as first-line therapy for patients with clinically significant proptosis. About IBI311 IBI311 is a recombinant anti-IGF-1R antibody developed by Innovent for the treatment of TED. IGF-1R is a transmembrane tyrosine kinase receptor that plays a role in the development, metabolism, and immune regulation, and is overexpressed in OFs, B, and T cells of TED patients11. IBI311 can bind IGF-1R, block IGF-1R signaling pathway activation mediated by IGF-1 and other related ligands or agonistic antibodies, reduce the expression of downstream inflammatory factors, thereby inhibiting the synthesis of hyaluronic acid and other glycosaminoglycan caused by OFs activation, as well as related inflammatory reactions including tissue congestion and edema; inhibit adipocyte cellularization of OFs, thereby reducing the disease activity of patients with TED and improving proptosis, diplopia, ocular congestion and edema and other symptoms and signs. About Innovent Innovent is a leading biopharmaceutical company founded in 2011 with the mission to provide high-quality biologics that are affordable to all. The company discovers, develops, manufactures and commercializes innovative medicines that treat some of the most intractable illnesses. Its pioneering therapies to treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has 10 products in the market, 3 new drug applications under the NMPA review, 4 assets in Phase III or pivotal clinical trials and 19 more molecules in early clinical stage. Innovent partners with over 30 global healthcare leaders, including Eli Lilly, Roche, Sanofi, Adimab, Incyte and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit , or follow Innovent on Facebook and LinkedIn. Statement: Innovent does not recommend the use of any unapproved drug (s)/indication (s). Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent Biologics ("Innovent"), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or is otherwise inaccurate. References: 1. Bartley G. The epidemiological characteristics and clinical course of ophthalmology associated with autoimmune thyroid disease in Olmsted Country, Minnesota. Trans Am Ophthalmol Soc 1994; 92: 477-588. 2. Hiromatsu Y, Eguchi H, Tani J, Kasaoka M, Teshima Y. Graves' ophthalmopathy: epidemiology and natural history. Intern Med. 2014;53(5):353-60. 3. Bartalena L, Kahaly GJ, Baldeschi L, et al. The 2021 European Group on Graves' Orbitopathy (EUGOGO) clinical practice guidelines for the medical management of Graves' Orbitopathy. Eur J Endocrinol. 2021; 185 (4): G43-G67. 4. Group of Oculoplastic and Orbital Diseases, Chinese Ophthalmology Branch, Chinese Medical Association; Group of Thyroid Diseases, Chinese Endocrinology Branch, Chinese Medical Association. Chinese Guidelines for the Diagnosis and Treatment of Thyroid Associated Ophthalmopathy (2022). Chinese Journal of Ophthalmology. 2022; 58 (9). 5. Burch HB, et al. Management of Thyroid Eye Disease: a Consensus Statement by the American Thyroid Association and the European Thyroid Association. Eur Thyroid J. 2022; 11 (6): e220189. 6. Li Z, Cestari D M, Fortin E. Thyroid eye disease: what is new to know? Curr Opin Ophthalmol. 2018; 29 (6): 528-534. 7. Edsel I. Thyroid Associated Orbitopathy. Retrieved June 7, 2011, from Medscape Reference: # a1 8. Ali F, Chorsiya A, Anjum V, Ali A. Teprotumumab (TEPEZZA): From the Discovery and Development of Medicines to USFDA Approvals for Active Thyroid Eye Disease (TED) Treatment. Int Ophthalmol. 2021; 41 (4): 1549-1561. 9. Dolman P J. Evaluating Graves' orbitopathy. Best Pract Res Clin Endocrinol Metab. 2012; 26 (3): 229-248. 10. Bahn R S. Graves' ophthalmopathy. N Engl J Med. 2010; 362 (8): 726-738. 11. Douglas RS, Naik V, Hwang CJ, et al. B cells from patients with Graves' disease erase express the IGF-1 receptor: implications for disease pathogenesis. J Immunol 2008; 181: 5768-5774. SOURCE Innovent Biologics

-- Eton Cares patient support program offers $0 co-pay to eligible, commercially insured patients* -- -- Current Nitisinone market is estimated to exceed $50 million annually -- -- Product is now available exclusively through Optime Care -- DEER PARK, Ill., Feb. 02, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing, acquiring, and commercializing products to address unmet needs in patients suffering from rare diseases, today announced the commercial availability of Nitisinone Capsules for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Please see important safety information below. Tyrosinemia type 1 is an ultra-rare condition that is estimated to impact fewer than 500 patients in the United States. "Eton is committed to serving patients and families with the rarest of conditions. We are proud to make Nitisinone available to the hundreds of patients in the United States and offer full patient and provider support services. We believe that our existing commercial infrastructure and strong patient support services should help us capture a meaningful percentage of the estimated $50 million annual Nitisinone market,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. Nitisinone Capsules are available exclusively through Optime Care, a specialty pharmacy dedicated to helping patients with rare diseases manage their conditions. Optime Care will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The Eton Cares Program will provide $0 co-pays for all commercially insured patients*, as well as prescription fulfillment, insurance benefits investigation, educational support, and financial assistance for qualifying patients. Clinicians seeking to prescribe Nitisinone Capsules can e-prescribe by selecting Optime Care as the pharmacy or fax in a patient referral form to 866-318-2990. Additional product details can be found on the product website, For questions regarding prescription fulfillment, please contact Optime Care at 1-844-397-0541. *Restrictions, limitations, and/or eligibility requirements may apply USE and IMPORTANT SAFETY INFORMATION What is Nitisinone? Nitisinone is a prescription medicine used to treat adults and children with a hereditary disease called tyrosinemia type 1 (HT-1). Nitisinone should be taken along with a diet limiting tyrosine and phenylalanine. What is the most important information I should know about Nitisinone? Tell your doctor or nurse right away if you have any of these symptoms with Nitisinone: Increased levels of plasma tyrosine, eye symptoms, developmental delay, and skin changes: Inadequate restriction of tyrosine and phenylalanine intake can result in raising plasma tyrosine levels. Plasma tyrosine levels above 500 micromol/L may lead to eye signs and symptoms like corneal ulcers, corneal cloudiness, inflammation of the cornea (keratitis), pink eye (conjunctivitis), eye pain, and sensitivity to light (photophobia), intellectual disability and developmental delay or painful thickening of the skin (hyperkeratotic plaques) on the soles and palms. Your healthcare provider should not adjust Nitisinone dosage in order to lower the levels of tyrosine in the blood. Changes in blood pro > You may develop a reduction in the number of white blood cells, which form part of the immune system (leukopenia) and abnormally low levels of platelets, which help the blood to clot (severe thrombocytopenia). Do not take Nitisinone if: you are allergic to Nitisinone or any other ingredients. Stop using Nitisinone and get medical help right away if you have any symptoms of a serious allergic reaction, including swelling of the face, lips, tongue, or throat; problems breathing or swallowing; severe rash or itching; fainting or feeling dizzy; or very rapid heartbeat. Before taking Nitisinone, tell your doctor if you: are pregnant or plan to become pregnant. Nitisinone may harm your unborn baby. Tell your doctor if you become pregnant or suspect you are pregnant during treatment with Nitisinone. are breastfeeding or plan to breastfeed. It is not known if Nitisinone passes into your breast milk. Talk to your doctor about the best ways to feed your baby during treatment with Nitisinone. are aged 65 and older. Your doctor may need to adjust the dose of Nitisinone based on your requirements. are taking other medicines since Nitisinone can interfere with their effect. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, dietary and herbal supplements. What are the possible side effects of Nitisinone? The most common side effects of Nitisinone (≥1%) include high tyrosine levels, low platelets (thrombocytopenia) or white cells in the blood (leukopenia), and complaints related to the eyes, including pink eye (conjunctivitis), corneal cloudiness, inflammation of the cornea, eye pain and extreme sensitivity to light (photophobia), nosebleed (epistaxis), itching (pruritus), skin inflammation (exfoliative dermatitis), rash (maculopapular rash), dry skin and hair loss (alopecia). For more detailed information, please see full Prescribing Informationfor more information. To report a suspected adverse event related to Nitisinone, contact Eton Pharmaceuticals, Inc. at: 1-855-224-0233 or the US Food and Drug Administration at or call 1-800-FDA-1088. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing, acquiring, and commercializing products to address unmet needs in patients suffering from rare diseases. The Company currently has four FDA-approved rare disease products: ALKINDI SPRINKLE®, 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 . 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: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals.