Eton Pharmaceuticals is a corporation that specializes in the creation, procurement, and distribution of prescription medications. Their product line includes Biorphen, Alaway Preservative Free, zonisamide oral suspension, topiramate oral suspension, lamotrigine for oral suspension, cysteine injection, and ephedrine ready-to-use injection. The company was established in April 2017 and is based in Deer Park, Illinois.

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Other Diseases

Endocrinology and Metabolic Disease

Digestive System Disorders

Small molecule drug

Growth factors

Chemical drugs

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Endocrinology and Metabolic Disease	7
Nervous System Diseases	6
Neoplasms	5
Infectious Diseases	2
Immune System Diseases	2
Hemic and Lymphatic Diseases	2

Top 5 Drug Type	Count

Small molecule drug	9
Unknown	1
Growth factors	1
Chemical drugs	1

Top 5 Target	Count

TPH1 x TPH2	1
Top I(DNA topoisomerase I)	1
GR(Glucocorticoid receptor)	1
IGF-1R(Insulin-like growth factor I receptor)	1
AXL x RET x ROS1 x TYRO3 x Tie-2 x TrkB x VEGFR1 x VEGFR2 x VEGFR3 x c-Kit x c-Met	1

Drugs associated with Eton Pharmaceuticals, Inc.

Elafibranor

Target

PPARα x PPARδ

Mechanism

PPARα agonists [+1]

Active Org.

Ipsen SA [+4]

Originator Org.

Ipsen Biopharm Ltd.

Active Indication

Primary Biliary Cholangitis [+1]

Inactive Indication

Cardiovascular Diseases [+11]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date10 Jun 2024

Palovarotene

Target

RARγ

Mechanism

RARγ agonists

Active Org.

Ipsen SA [+2]

Originator Org.

Clementia Pharmaceuticals, Inc.

Active Indication

Cardiovascular Diseases [+2]

Inactive Indication

Dry Eye Syndromes [+4]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date21 Jan 2022

Odevixibat

Target

ISBT

Mechanism

ISBT inhibitors

Active Org.

Medison Pharma Ltd. [+9]

Originator Org.

Albireo Pharma, Inc.

Active Indication

Cholestatic pruritus [+5]

Inactive Indication

Cholestasis, Progressive Familial Intrahepatic 1 [+2]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

EU [+3]

First Approval Date16 Jul 2021

Clinical Trials associated with Eton Pharmaceuticals, Inc.

NCT04753216

/ CompletedPhase 2IIT

A Phase II Trial of Irinotecan Liposome and Bevacizumab in Women With Platinum Resistant Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

This phase II trial investigates the effect of irinotecan liposome and bevacizumab in treating patients with ovarian, fallopian tube, or primary peritoneal cancer that shows less response to platinum therapy (platinum resistant), has come back (recurrent), or does not respond to treatment (refractory). Irinotecan liposome may help block the formation of growths that may become cancer. Bevacizumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Giving irinotecan liposome and bevacizumab may kill more cancer cells.

Start Date16 Mar 2021

Sponsor / Collaborator

Northwestern University

[+2]

NCT00764699

/ TerminatedPhase 2/3IIT

Effect of Increlex® on Children With Crohn Disease

Patients with Crohn disease often have poor weight gain and short stature, yet the etiology of the poor growth is not well defined. Studies in chronically ill patients who do not have Crohn disease have suggested that inflammation causes IGF-1 deficiency due to inadequate IGF-1 generation. Previous studies of GH use in Crohn patients have demonstrated improvement in linear growth, weight and bone mineralization. However, GH can cause glucose intolerance in chronically ill children, particularly those who require treatment with corticosteroids. Recently the FDA has approved recombinant IGF-1 (rhIGF) for treatment of IGF-1 deficient short stature. This medication has not been studied in Crohn disease. The purpose of this study is to test the hypothesis that poor growth in Crohn disease is associated abnormal IGF-1 generation which leads to poor linear growth, decreased weight and osteoporosis and that replacement of IGF-1 with rhIGF will correct growth and improve bone density. To test our hypothesis we will recruit 20 patients with Crohn disease from our pediatric gastroenterology practice. Each will have been previously diagnosed with Crohn disease for a minimum of one year and will be studied at baseline and six month intervals for one year while on treatment with Increlex.

Start Date01 Oct 2008

Sponsor / Collaborator

Nationwide Children's Hospital

[+1]

NCT00684957

/ TerminatedPhase 1/2IIT

Differential Effects of rhGH vs. rhIGF-1 on Cardiovascular Risk Factors in Adult Patients With Growth Hormone Deficiency

The purpose of this study is to see if giving growth hormone or insulin-like growth factor-1 (IGF-1) to subjects with growth hormone deficiency effects cardiovascular risk factors differently.

Start Date01 Jan 2008

Sponsor / Collaborator

Columbia University

[+1]

100 Clinical Results associated with Eton Pharmaceuticals, Inc.

0 Patents (Medical) associated with Eton Pharmaceuticals, Inc.

Literatures (Medical) associated with Eton Pharmaceuticals, Inc.

05 Nov 2024·Blood

Changes in Treatment Patterns over Time Among Real-World Patients with Follicular Lymphoma at Predominantly Community Facilities in the US

Author: Cockrum, Paul ; Dennen, Syvart ; Dillon, Allison ; Kambhampati, Swetha

BACKGROUND: Follicular lymphoma (FL) is characterized by a relapsing and remitting course that can lead to multiple lines of therapy (LOTs). While frontline treatments are well-established, optimal treatment in third line (3L) or later is not well-defined and the introduction of novel therapies in recent years has increased treatment options. This study examines changes in treatment patterns over the time periods of 2015-2018 and 2019-2023 for first (1L) through fifth line (5L) among US real-world FL patients (pts).METHODS: This retrospective, observational study used Optum Market Clarity claims and electronic health record (EHR) FL data, which has 93% encounters from confirmed community facilities in the US. Inclusion criteria were: ≥1 inpatient or ≥2 outpatient claims or ≥1 EHR record with diagnosis of FL from 2008-2023 (index date); ≥12 months pre-index enrollment or EHR clinical activity with no evidence of cancer; ≥1 LOT initiated in 2015 or later.LOTs consist of all therapies administered in the first 28 days of treatment. Adding new therapies after this period initiated a new LOT. Maintenance lines were excluded from analysis. This study is descriptive only and no statistical testing was performed.RESULTS: In 1L, there were 2906 pts from 2015-2018 and 1878 from 2019-2023. Most treatments were rituximab (R)-based and changed little over time. For 2015-2018 vs 2019-2023, >80% of treatments were either R + chemo (consisting of R + bendamustine, R-CHOP, R-EPOCH, R-ICE, and similar therapies) (61% vs 58%) or R monotherapy (mono) (28% vs 25%). Chemo (mono or combination treatment with platinum-based therapies, cytarabine, vincristine, and similar), the third largest category, was unchanged (5% vs 5%). Small increases were seen in ofatumumab (OF) or obinutuzumab (OB) + bendamustine (2% vs 5%) and R + lenalidomide (R2) (1% vs 2%). Utilization of other therapies was uncommon.In 2L there were 1300 pts from 2015-2018 and 956 from 2019-2023. R-based regimens were the most common, but were less utilized and had larger decreases between time periods than in 1L. For 2015-2018 vs 2019-2023, decreases were seen in R mono (41% vs 34%) and R + chemo (35% vs 31%). Increases were seen in R2 (2% vs 6%), OF and OB mono or with bendamustine (3% vs 7%), and lenalidomide or venetoclax mono (2% vs 5%).In 3L, there were 614 pts from 2015-2018 and 497 from 2019-2023. R mono was the most common therapy but dropped between periods (41% vs 28%). R + chemo decreased slightly (23% vs 21%) as did PI3Ki-containing (5% vs 2%). Similar over time were chemo (10% vs 10%) and BTKi-containing (7% vs 8%). Increases were seen in OF and OB monotherapy (1% vs 4%), R2 (5% vs 7%), lenalidomide or venetoclax mono (3% vs 9%), CAR-T (0% vs 2%), and tazemetostat (taz)-containing (0% vs 2%).In 4L, there were 344 pts from 2015-2018 and 288 from 2019-2023. As in 3L, R mono was the most common therapy but dropped between periods (46% vs 28%), while R + chemo showed a small decrease (20% vs 17%). Similar over time were PI3Ki-containing (5% vs 5%), and R2 (3% vs 4%). Chemo (11% vs 13%) increased slightly, and increases were also seen in BTKi-containing regimens (7% vs 11%), OF or OB mono (0% vs 4%), lenalidomide or venetoclax mono (3% vs 8%), CAR-T (0% vs 2%), and taz-containing (0% vs 2%).In 5L, there were 180 pts from 2015-2018 and 149 from 2019-2023. R mono was the most common therapy, with utilization dropping between periods (56% vs 32%). Similar across time were R + chemo (12% vs 13%), BTKi-containing (9% vs 10%), and PI3Ki-containing (4% vs 3%). Increases were seen in lenalidomide or venetoclax mono (4% vs 13%), chemo (6% vs 9%), R2 (3% vs 7%), CAR-T (0% vs 3%), and taz-containing (0% vs 3%).While mosunetuzumab, a bispecific monoclonal antibody (bsAb), was approved in late 2022, there were not sufficient pts to assess utilization.CONCLUSIONS: R-based regimens, particularly R mono, continue to be the mainstay of FL treatment in US community facilities. While 1L treatments changed little over time, in later lines, R mono use decreased, replaced by more novel therapies such as R2, BTKis, OF, OB, lenalidomide, venetoclax, taz, and CAR-T. Utilization of newer therapies in later lines in the community remains low, with the standard treatments of R mono and R + chemo making up 44% to 65% of treatments in 2L-5L during 2019-2023. Future research should evaluate use of other novel therapies such as bsAbs and the impact of later line sequencing on clinical outcomes.

05 Nov 2024·Blood

Hematologic Toxicity Among Real-World US Patients with Follicular Lymphoma Treated with Tazemetostat Compared to Other Standard-of-Care Therapies

Author: Cockrum, Paul ; Dennen, Syvart ; Dillon, Allison ; Kambhampati, Swetha

BACKGROUND: While follicular lymphoma (FL) is generally an indolent disease, its relapsing and remitting course can require multiple lines of therapy (LOTs). Over the last decade, a better understanding of the molecular pathogenesis of FL has led to the emergence of novel therapies, particularly in later lines. Tazemetostat (taz) is an oral medication approved as monotherapy (mono) for patients (pts) with R/R FL with EZH2 gain-of-function mutations who received at least 2 prior systemic therapies or R/R FL pts who have no satisfactory alternative treatment options.The range of treatments for R/R FL each come with their own profile of toxic effects. Treatments with fewer adverse events (AEs) reduce healthcare resource utilization (HCRU) and have the potential to improve pt quality of life (QoL). We describe rates of hematologic AEs and granulocyte colony-stimulating factor (G-CSF) use using real-world (RW) data from the US, focusing on third-line (3L) and fourth-line (4L) therapies. In addition, we report results for taz mono pts, describing a pt group of unknown line to allow sufficient sample size.METHODS: This retrospective, observational study used Optum Market Clarity claims and electronic health record (EHR) data, which contains 93% encounters at confirmed community facilities in the US. Non-taz pts were included if they had ≥1 inpatient or ≥2 outpatient claims or ≥1 EHR record with a diagnosis of FL from January 1, 2008 to December 31, 2023 (index date); ≥12 months pre-index continuous enrollment or EHR clinical activity with no evidence of cancer; 3L or 4L initiated after January 1, 2015; ≥1 EHR record on or after 3L/4L treatment initiation.For taz mono pts, modified inclusion criteria were employed to allow sufficient sample for analysis: ≥1 diagnosis of FL in claims or EHR data; ≥1 taz fill in claims or EHR; and ≥1 EHR record on or after first taz fill. Due to the limited criteria, taz mono pts may have additional cancers, could not be assigned a LOT, and may have been observed during a LOT in progress. Based on indication, taz mono pts are expected to be predominantly in 3L and 4L.Hematologic AEs (anemia, neutropenia, and thrombocytopenia) were identified using lab values in EHR or claims data and graded according to the Common Terminology Criteria for AEs v5.0. G-CSF administrations were measured using both claims and EHR data. Results of ≤4 pts are not reportable per Optum rules; these are reported as ≤[percentage]. Due to this rule, all grades of AEs are combined to increase reportable figures.Regimens were categorized based on clinical input. Rituximab (R) + chemo consists of R-CHOP, R-CVP, R-ICE, or similar, excluding R + bendamustine (B). Chemo includes mono or combination treatment with platinum-based therapies, cytarabine, vincristine, and similar. Results are reported for categories with ≥40 pts. This analysis was descriptive only; no adjustments for pt characteristics and no statistical testing was performed.RESULTS: There were 50 taz mono pts of unknown LOT, while there were 993 pts in 3L treatment categories with ≥40 pts. We present the sample size for each category followed by the percentage with any grade anemia, neutropenia, thrombocytopenia, and ≥1 G-CSF use during the line: taz mono (n=50, 28%, 10%, ≤8%, ≤8%), R mono (n=507, 16%, 6%, 3%, 2%), R + chemo (n=162, 56%, 33%, 30%, 67%), chemo (n=109, 41%, 18%, 18%, 32%), R + B (n=101, 32%, 17%, 15%, 51%), BTKi-containing (n=64, 47%, 20%, 22%, 8%), and R2 (n=50, 50%, 30%, 18%, 16%).In the 4L of our analysis, there were 517 pts in treatment categories of ≥40 pts. The 4L categories and results, with taz mono repeated, are: taz mono (n=50, 28%, 10%, ≤8%, ≤8%), R mono (n=288, 15%, 6%, 2%, ≤1%), chemo (n=69, 48%, 23%, 25%, 28%), R + chemo (n=63, 68%, 32%, 40%, 68%), R + B (n=55, 31%, 16%, 13%, 38%), and BTKi-containing (n=42, 43%, 29%, 29%, 17%).CONCLUSIONS: Taz mono was most numerically comparable to R mono in rates of hematologic AEs and G-CSF use and had numerically lower rates of each than all other regimens. In most cases, the differences were clinically meaningful. The reduced hematologic toxicity observed with taz mono combined with its oral formulation may be helpful to consider when selecting treatment for FL pts, depending on their personal circumstances. Further research should evaluate rates of AEs adjusted for pt characteristics, the RW effectiveness of taz, and its impact on pt QoL compared to other standard-of-care therapies (SOC) in a real-world setting.

05 Nov 2024·Blood

Healthcare Resource Utilization and Costs to Patients and Payers Among Real-World Patients with Follicular Lymphoma in the US Treated with Tazemetostat Compared to Other Standard-of-Care Therapies

Author: Cockrum, Paul ; Dennen, Syvart ; Dillon, Allison ; Kambhampati, Swetha

BACKGROUND: Follicular lymphoma (FL) tends to become relapsed or refractory (R/R) to treatment, requiring multiple lines of therapy (LOTs). While frontline therapy is well-established, optimal treatment at third line (3L) and beyond is unclear. Recent approvals of novel therapies have expanded treatment options. Tazemetostat (taz) is an oral medication approved as monotherapy (mono) for patients (pts) with R/R FL with EZH2 gain-of-function mutations who received at least 2 prior systemic therapies or R/R pts who have no satisfactory alternative treatment options.Previous research has examined costs and healthcare resource utilization (HCRU) in frontline treatment. For later lines, further evidence is needed on the real-world (RW) financial impact to pts and payers and the associated HCRU. This study examines average all-cause per-pt-per-month (PPPM) cost to payers, PPPM out-of-pocket (OOP) costs to pts, and PPPM HCRU (days with inpatient [IP] admissions, ED use, and outpatient [OP] visits) for RW pts treated in the 3L and fourth line (4L) in the US. We also report outcomes for taz mono pts of unknown line to allow sufficient sample size.METHODS: This retrospective, observational study used Optum Market Clarity claims and electronic health record (EHR) data, representing encounters at 93% confirmed community facilities in the US. Non-taz pts were included if they had ≥1 IP or ≥2 OP claims or ≥1 EHR record with a diagnosis of FL between 2008-2023 (index); ≥12 months pre-index claims enrollment or EHR activity with no evidence of cancer; enrollment ≥30 days before and after line start; 3L or 4L therapy initiated 2015-2023. Due to small sample size, 3L and 4L were combined.Non-taz pts were assigned treatment categories with clinical input. Rituximab (R) + chemotherapy (chemo) consists of R + bendamustine, R-CHOP, and similar. Chemo includes mono or combination treatment with platinum-based therapies, cytarabine, vincristine, and similar.For taz mono pts, modified inclusion criteria allowed sufficient sample for analysis: ≥1 diagnosis of FL in claims or EHR data; ≥1 taz fill in claims; ≥30 days enrollment after first taz fill. Therefore, taz mono pts may have additional cancers and cannot be assigned a LOT. Taz mono pts are expected to be in predominantly 3L and 4L.All-cause HCRU and costs (2023 USD) PPPM were calculated from LOT start to end, with end defined as either initiation of subsequent therapy, treatment gap, death, or censoring.This study is descriptive only. No adjustments were made for pt characteristics and no statistical testing was performed.RESULTS: There were 49 taz mono pts of unknown LOT and 408 pts in 3L/4L (treatment categories <10 pts excluded). R mono and R + chemo made up >60% of pts. Sample size, average PPPM costs to payers, and PPPM OOP costs to pts were, respectively: taz mono (n=49, $15,921, $437); R2 (n=21, $25,967, $461); BTKi-containing (n=15, $20,123, $638); R + chemo (n=81, $16,396, $320); PI3Ki-containing (n=21, $13,840, $415); R mono (n=211, $13,514, $578); and chemo (n=43, $12,281, $254).PPPM IP admissions, days with ED visits, and days with OP visits were, respectively: taz mono (0.121, 0.099, 2.5); R2 (0.312, 0.534, 4.2); chemo (0.239, 0.179, 3.4); PI3Ki-containing (0.210, 0.230, 2.7), R + chemo (0.124, 0.131, 4.2), BTKi-containing (0.082, 0.248, 3.3); and R mono (0.031, 0.063, 3.2).CONCLUSIONS: While statistical testing was not performed due to small sample size, we descriptively assess outcomes. Taz mono was less costly PPPM to US payers than other novel therapies, such as R2 and BTKi-containing regimens. It was similar to R + chemo, PI3Ki-containing, and R mono. The cost of regimens with infusions/injections was driven by OP and physician-administered drug costs. Costs of regimens with only oral drugs (taz mono, BTKi-containing) were driven by OP pharmacy and had low OP facility costs.Taz mono had lower OOP costs than BTKi-containing regimens, another novel oral therapy, and R mono. It was similar to R2, PI3Ki-containing, and R + chemo. OOP costs can vary markedly by payer type, and region.Taz mono had fewer OP days than all other regimens. IP use was lower for taz mono than all other therapies except BTKi-containing and R mono, and ED use was lower than all other therapies except R mono.Taz's oral formulation and safety profile may have led to the observed lower HCRU and costs. The impact of cost and HCRU on both the health system and pts should be considered when selecting treatment.

102

News (Medical) associated with Eton Pharmaceuticals, Inc.

26 Dec 2024

·www.globenewswire.com

Eton Pharmaceuticals Closes Acquisition of Increlex® (mecasermin injection)

Acquisition bolsters Eton’s commercial pediatric endocrinology portfolio Product is now available through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions Dec. 20, 2024 -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that it has completed its previously announced asset purchase of Increlex® (mecasermin injection) from Ipsen S.A. (“Ipsen”). Increlex® is a biologic product used to treat pediatric patients 2 years of age and older who suffer from severe primary insulin-like growth factor 1 deficiency (SPIGFD). “We are excited to close this transformational acquisition and add another important treatment to our commercial portfolio. Increlex® is perfectly aligned with our expertise and strong relationships in pediatric endocrinology and we’re well-positioned to leverage our existing sales team to increase awareness of SPIGFD, an underdiagnosed and undertreated condition,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “In the U.S., Increlex® is now available through a specialty pharmacy dedicated to rare and chronic conditions and we are proud to be able to continue supplying this crucial product worldwide without disruption.” Increlex® is a biologic product used to treat pediatric patients 2 years of age and older who suffer from severe primary insulin-like growth factor 1 deficiency (SPIGFD) because their bodies do not make enough insulin-like growth factor 1 (IGF-1). The medicine is approved in 41 territories, including the United States (U.S.) and the European Union (EU). It is estimated that approximately 200 patients in the United States and 900-1,000 patients in Europe live with SPIGFD. Increlex® is the only treatment approved by the U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA) for SPIGFD. Increlex® is now available in the United States exclusively through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. AnovoRx 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. Outside the U.S., Ipsen will continue distributing Increlex® during a six-month transition period, after which Eton will take over commercialization. The transaction was financed by Eton’s cash on hand and an expansion of the Company’s existing credit facility with SWK Holdings. Clinicians seeking to prescribe Increlex® can e-prescribe by selecting AnovoRx #5 or fax in a patient referral form to 855-831-2039. Additional product details can be found on the product website, https://www.increlex.com/en-us. Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has six commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. The content above comes from the network. if any infringement, please contact us to modify.

Drug ApprovalAcquisition

17 Dec 2024

·ir.etonpharma.com

Eton Pharmaceuticals Announces Final Readout of PKU GOLIKE® Clinical Trial

« Back to news DEER PARK, Ill., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases announced the full readout and compelling results from the clinical trial evaluating PKU GOLIKE as a protein substitute for the treatment of phenylketonuria (PKU) in patients during prolonged fasting periods. The study demonstrated that PKU GOLIKE, administered as the last daily dose and compared to standard amino acid protein substitutes, improved metabolic control by reducing harmful phenylalanine (Phe) levels and increasing beneficial tyrosine (Tyr) levels, both essential for brain function and metabolic health. PKU patients often experience significant fluctuations in blood Phe levels during prolonged fasting periods, particularly at night, when protein breakdown causes Phe concentrations to peak in the early morning. These fluctuations are associated with cognitive difficulties and overall health impacts, making nighttime metabolic control an important focus in PKU management. The study was sponsored by Relief Therapeutics Holding SA, and was a randomized, crossover, controlled clinical study conducted by the Inherited Metabolic Disorders Unit at Birmingham Children’s Hospital, UK, on pediatric patients with classical PKU, the condition’s most severe form. The trial compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting, the longest fasting period within 24 hours. At the end of the one-week treatment period, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to those receiving standard amino acid substitutes (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113). Compared to baseline levels measured prior to the start of treatment, the PKU GOLIKE group achieved an average 17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8% increase in blood Tyr levels (P=0.0008) upon awakening after the overnight fasting period. In comparison, when treated with standard amino acid protein substitutes, the same patients experienced an average 27.6% increase in blood Phe levels (P=0.0063) and no significant improvement in blood Tyr levels. Blood sample analysis at three early morning time points across the two groups revealed no significant differences in peak Phe levels upon reawakening in either group. Highlighting the clinical significance of the findings, Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children’s Hospital, stated: “Giving one dose of PKU GOLIKE as the final daily dose of protein substitute resulted in consistently better metabolic control in our cohort of patients with PKU. They all had classical PKU and were a particularly challenging group to control.” These results confirm that PKU GOLIKE’s prolonged-release profile provides clinically and statistically significant improvements in metabolic control during extended fasting periods compared to standard amino acid protein substitutes. Eton expects these findings to support the adoption of PKU GOLIKE among healthcare providers and within the PKU community. The study findings will be presented in a poster titled A Prolonged-Release Formula Has a Positive Impact on Morning Phenylalanine and Tyrosine Fluctuations in Patients with Classical Phenylketonuria at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles, California. Eton promotes PKU GOLIKE with its existing metabolic sales force, which also promotes Eton’s Carglumic Acid, Betaine, and Nitisinone products. PKU patients’ care is typically overseen by metabolic geneticists and their support staff of nurse practitioners and registered dieticians. Medical formulas for PKU are frequently covered by insurance and are regulated by the FDA as medical food products. Patients and healthcare professionals seeking additional information or requesting a product sample can visit pkugolike.com. For more information on this study (NCT05487378), please visit clinicaltrials.gov. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at etonpharma.com. About Phenylketonuria (PKU) Phenylketonuria (PKU) is caused by a defect of the enzyme needed to break down phenylalanine (Phe), leading to a toxic buildup of Phe from the consumption of foods containing protein or aspartame. Untreated PKU can result in global developmental delay or severe irreversible intellectual disability, as well as growth failure, hypopigmentation, motor deficits, ataxia and seizures. Living with PKU requires a limited diet and very careful management. If left unmanaged, PKU can lead to devastating consequences, such as brain damage. People living with PKU do not have the ability to metabolize Phe, which is found in many foods, and they require supplementation of amino acid-based phenylalanine-free medical formulas as part of an effort to prevent protein deficiency and optimize metabolic control. Medical formulas used in PKU are challenged to provide a range of amino acids slowly and without a medicinal aftertaste. About PKU GOLIKE® PKU GOLIKE® products are foods for special medical purposes (FSMPs) for the dietary management of PKU in both children and adults for use under medical supervision. Developed with Relief’s proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE® products are the first prolonged-release amino acid FSMPs, characterized by a special coating that ensures physiological absorption of the amino acids mirroring that of natural proteins. The special coating also masks the unpleasant taste, odor, and aftertaste of the amino acids. PKU GOLIKE PLUS® granules are flavorless and can be mixed with many foods. PKU GOLIKE® products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and PKU GOLIKE PLUS® granules are fortified with 27 essential vitamins and minerals, including ones normally found in protein-rich foods like iron, calcium and vitamin B12. The PKU GOLIKE® line of products are available in convenient packets (PKU GOLIKE PLUS® 3-16 and 16+) and medical formula bars (PKU GOLIKE BAR®). PKU GOLIKE® products have been commercially available in the U.S. since October 2022. For more information, visit pkugolike.com. (Please note this site is intended for U.S. audiences only). 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, Inc. Source: Eton Pharmaceuticals

Clinical Result

16 Dec 2024

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Relief Therapeutics Announces Final Readout of PKU GOLIKE Clinical Trial

Clinical Trial Demonstrates Superior Metabolic Control During Prolonged Fasting in PKU PatientsResults Expected to Promote Awareness and Adoption of PKU GOLIKE ®GENEVA, SWITZERLAND / ACCESSWIRE / December 16, 2024 / RELIEF THERAPEUTICS Holding SA (SIX:RLF )(OTCQB:RLFTF )(OTCQB:RLFTY ) (Relief, or the Company), a biopharmaceutical company committed to delivering innovative treatment options for select specialty, unmet and rare diseases, today announced the full readout and compelling results from the clinical trial evaluating PKU GOLIKE as a protein substitute for the treatment of phenylketonuria (PKU) in patients during prolonged fasting periods. The study demonstrated that PKU GOLIKE, administered as the last daily dose and compared to standard amino acid protein substitutes, improved metabolic control by reducing harmful phenylalanine (Phe) levels and increasing beneficial tyrosine (Tyr) levels, both essential for brain function and metabolic health. PKU patients often experience significant fluctuations in blood Phe levels during prolonged fasting periods, particularly at night, when protein breakdown causes Phe concentrations to peak in the early morning. These fluctuations are associated with cognitive difficulties and overall health impacts, making nighttime metabolic control an important focus in PKU management.The Company-sponsored, randomized, crossover, controlled clinical study was conducted by the Inherited Metabolic Disorders Unit at Birmingham Children's Hospital, UK, on pediatric patients with classical PKU, the condition's most severe form. The trial compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting, the longest fasting period within 24 hours.At the end of the one-week treatment period, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to those receiving standard amino acid substitutes (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113). Compared to baseline levels measured prior to the start of treatment, the PKU GOLIKE group achieved an average 17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8% increase in blood Tyr levels (P=0.0008) upon awakening after the overnight fasting period. In comparison, when treated with standard amino acid protein substitutes, the same patients experienced an average 27.6% increase in blood Phe levels (P=0.0063) and no significant improvement in blood Tyr levels. Blood sample analysis at three early morning time points across the two groups revealed no significant differences in peak Phe levels upon reawakening in either group.Highlighting the clinical significance of the findings, Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children's Hospital, stated: "Giving one dose of PKU GOLIKE as the final daily dose of protein substitute resulted in consistently better metabolic control in our cohort of patients with PKU. They all had classical PKU and were a particularly challenging group to control."These results confirm that PKU GOLIKE's prolonged-release profile provides superior metabolic control during extended fasting periods compared to standard amino acid protein substitutes. The Company expects these findings to support adoption of PKU GOLIKE among healthcare providers and within the PKU community.The study findings will be presented in a poster titled A Prolonged-Release Formula Has a Positive Impact on Morning Phenylalanine and Tyrosine Fluctuations in Patients with Classical Phenylketonuria at the 2025 ACMG Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles.For more information on this study (NCT05487378), please visit clinicaltrials.gov .ABOUT PHENYLKETONURIAPhenylketonuria (PKU) is a genetic disorder caused by a deficiency of the enzyme needed to break down phenylalanine (Phe), leading to a toxic buildup of Phe from the consumption of foods containing protein or aspartame. Individuals with PKU lack the ability to metabolize Phe, which is present in many foods. Without treatment, PKU can cause severe neurological and developmental issues. The standard treatment involves a lifelong phenylalanine-restricted diet supplemented with amino acid-based, phenylalanine-free medical foods to prevent protein deficiency and optimize metabolic control.ABOUT PKU GOLIKE ®PKU GOLIKE products are Foods for Special Medical Purposes (FSMPs) for the dietary management of PKU in children and adults. Developed with Relief's proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE products are the first prolonged-release amino acid FSMPs, characterized by a special coating that ensures physiological absorption of the amino acids mirroring that of natural proteins, while also masking the unpleasant taste and odor typically associated with amino acids. PKU GOLIKE products are marketed in the U.S. by Eton Pharmaceuticals Inc. under an exclusive license and supply agreement with Relief, in key European markets by Relief, and in select countries worldwide through licensing and distribution partners.ABOUT RELIEFRelief is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief's portfolio offers a balanced mix of marketed, revenue-generating products, proprietary, globally patented TEHCLO™ and Physiomimic™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare skin diseases, rare metabolic disorders, and rare respiratory diseases. In addition, Relief is commercializing several legacy products via licensing and distribution partners. Headquartered in Geneva, Relief is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, visit www.relieftherapeutics.com .CONTACT :RELIEF THERAPEUTICS Holding SAJeremy MeinenChief Financial Officer[email protected]DISCLAIMERThis press release contains forward-looking statements, which may be identified by words such as "believe," "assume," "expect," "intend," "may," "could," "will," or similar expressions. These statements are based on current plans and assumptions and are subject to risks and uncertainties that could cause actual results, financial condition, performance, or achievements to differ materially from those expressed or implied. Such factors include, but are not limited to, changes in economic conditions, market developments, regulatory changes, competitive dynamics, and other risks or changes in circumstances. This communication is provided as of the date hereof, and Relief undertakes no obligation to update any forward-looking statements contained herein as a result of new information, future events or otherwise.SOURCE: Relief Therapeutics Holding SA

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