IGF-1

AUSTIN, Texas, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Lumos Pharma (NASDAQ:LUMO), a clinical stage biopharmaceutical company focused on therapeutics for rare diseases, announced today that updated analyses of data from the Company’s Phase 2 OraGrowtH210 and OraGrowtH212 clinical trials have been accepted for oral presentation at the 62nd Annual European Society for Paediatric Endocrinology Meeting, or ESPE 2024, to be held November 16-18, 2024 in Liverpool, UK. Two abstracts on data from the OraGrowtH Trials evaluating oral LUM-201 for Pediatric Growth Hormone Deficiency (PGHD) will be presented on Sunday, November 17th, by Elżbieta Petriczko, MD, PhD, Pomeranian Medical University, Szczecin, Poland, and on Monday, November 18th, by Peter E. Clayton, MD, University of Manchester, Manchester, UK, respectively. LUM-201 Oral Abstract Presentations: Sunday, November 17th – Session: Free Communications 7: GH and IGFs – 10:50AM Local Time FC7.6 Abstract entitled, Growth, IGF-1 and IGFBP-3 Responses to Oral LUM-201 in OraGrowtH210 and OraGrowtH212 Trials in Pediatric Growth Hormone Deficiency (PGHD) Over 12 to 24 Months on Treatment (Elżbieta Petriczko, MD, PhD, et al) Monday, November 18th – Session: Free Communications 15: Late Breaking – 9:40AM Local Time FC15.2 Abstract entitled, Amount and Pattern of Pulsatile GH Secretion Induced by the Oral Growth Hormone Secretagogue LUM-201 Is Related to Growth and IGF-1 Responses in Moderate Pediatric Growth Hormone Deficiency (PGHD) (Peter E. Clayton, MD, et al) About Lumos Pharma Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The Company was founded and is led by a management team with longstanding experience in rare disease drug development. Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel, oral growth hormone (GH) secretagogue, seeking to transform the ~$4.7B global GH market from injectable to oral therapy. LUM-201 is currently being evaluated in multiple Phase 2 clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU. For more information, please visit https://lumos-pharma.com/. Investor & Media Contact: Lisa MillerLumos Pharma Investor Relations512-792-5454ir@lumos-pharma.com Source: Lumos Pharma, Inc.

iStock, hapabapa The regulator cited deficiencies at a third-party manufacturing facility. Camurus is seeking approval for its extended-release subcutaneous formulation of octreotide, which would allow more convenient once-monthly dosing for patients with acromegaly. The FDA has rejected Camurus’ investigational subcutaneous formulation of octreotide, dubbed CAM2029, for the treatment of the rare hormonal disorder acromegaly, the Swedish drugmaker announced on Tuesday. In a Complete Response Letter (CRL), the regulator cited “facility-related deficiencies” discovered last month during an inspection of a third-party manufacturer as the reason for the rejection and did not point to other issues, particularly as regards CAM2029’s clinical efficacy or safety data. The FDA needs to receive “satisfactory” responses from Camurus’ third-party provider before it can resolve the CRL, according to the company. CEO Fredrik Tiberg in a statement called the CRL “disappointing” but noted that the company remains “confident in the data supporting our [New Drug Application] and the potential of CAM2029 to address unmet medical needs of patients with acromegaly.” CRLs from the FDA rejecting drug candidates due to manufacturing issues are on the rise . Out of a total of 238 CRLs issued between 2017 and 2023, 30% involved quality/manufacturing issues —particularly problems with manufacturing processes, facility inspections, or chemical, manufacturing and controls. Camurus will work with its external manufacturer and with the FDA “to bring CAM2029 to patients living with acromegaly as soon as possible,” Tiberg said. To this end, the company said that it has already held labelling discussions with the regulator and that CAM2029’s prescribing information is “well advanced.” Afflicting around sixty patients per one million people, acromegaly is a rare and slowly progressive disease characterized by the abnormal growth of bones and enlarged hands, feet and facial features. Acromegaly also affects internal organs and can typically include symptoms such as headaches, joint pain and fatigue. Acromegaly is often caused by a tumor in the pituitary gland, which leads to the excess production of growth hormone and insulin growth factor-1. When uncontrolled, acromegaly worsens patients’ quality of life or can heighten their risk of death. Octreotide, the active ingredient of CAM2029, is a peptide drug that is well-known as an acromegaly therapy. It works by mimicking the function of somatostatin, a naturally occurring hormone, and activating its corresponding receptor. This mechanism of action helps suppress growth hormone, addressing the underlying cause of acromegaly. Octreotide also lowers the levels and activity of insulin and glucagon. The pharma industry has long taken advantage of octreotide’s therapeutic effects for acromegaly. Sun Pharmaceuticals won FDA approval for Bynfezia Pen (octreotide acetate) in 1988, which is indicated for the three-times-daily subcutaneous treatment of acromegaly. The drug is also approved for carcinoid tumors and vasoactive intestinal peptide tumors. Camurus’ investigational formulation is an extended-release version of octreotide that would allow for once-monthly dosing, giving patients a more convenient treatment option. In the Phase III ACROINNOVA 2 study, CAM2029 led to better biochemical control of acromegaly versus the current standard of care. Camurus’ injection also improved quality of life and symptom burden.

Aligns with Eton’s Mission to Develop and Distribute Medicines that Have a Life Changing Impact for Patients with Ultra-rare Conditions Acquisition to Bolster Eton’s Commercial Pediatric Endocrinology Portfolio DEER PARK, IL, USA I October 03, 2024 I 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 entered into an asset purchase agreement to acquire Increlex® (mecasermin injection) from Ipsen S.A. (“Ipsen”). The acquisition is expected to close near year-end 2024. “Eton’s mission is to develop and distribute medicines that make a life changing impact for patients with the rarest of conditions. Increlex, a critical medication for patients with the ultra-rare condition of severe IGF-1 deficiency, aligns perfectly with our mission and our significant expertise in serving extremely rare patient populations,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leveraging our strong presence in the pediatric endocrinology community, we aim to raise awareness of this underdiagnosed and undertreated condition. We look forward to collaborating with Ipsen to ensure continuity of care and long-term supply for patients globally.” Increlex is a biologic product used to treat children and adolescents from 2- to 18-years-old 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 40 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. Post closing, Eton will immediately commercialize the product in the U.S. without disruption to patient supply. Outside the U.S., Ipsen will continue distributing the product during a six-month transition period to ensure no disruption to patient supply, after which the commercialization will be continued by Eton. The transaction will be financed by Eton’s cash on hand and an expansion of the Company’s existing credit facility with SWK Holdings. Ipsen reported global sales for Increlex of €17.3 million in 2023. Important Safety Information Contraindications Warnings and Precautions Adverse Reactions Common adverse reactions include hypoglycemia, local and systemic hypersensitivity, and tonsillar hypertrophy. U.S. Indication INCRELEX ® (mecasermin) is indicated for the treatment of growth failure in pediatric patients aged 2 years and older with severe primary IGF-1 deficiency* (IGFD), or with hormone (GH) gene deletion who have developed neutralizing antibodies to GH. Limitations of use: INCRELEX ® is not a substitute to GH for approved GH indications. INCRELEX ® is not indicated for use in patients with secondary forms of IGFD, such as GH deficiency, malnutrition, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids. *Severe primary IGF-1 deficiency (IGFD) is defined by height standard deviation score ≤ -3.0 and basal IGF-1 standard deviation score ≤ -3.0 and normal or elevated GH. Full U.S. Prescribing Information for Increlex ® is available at: http://increlex.com/pdf/hcp-full-prescribing-information.pdf You are encouraged to report negative effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch , or call 1-800-FDA-1088. EU Indication In the European Union, INCRELEX is indicated for the long-term treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor 1 deficiency (Primary IGFD). Severe Primary IGFD is defined by: height standard deviation score <–3.0 and basal IGF-1 levels below the 2.5th percentile for age and gender and GH sufficiency. Exclusion of secondary forms of IGF 1 deficiency, such as malnutrition, hypopituitarism, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids. Severe Primary IGFD includes patients with mutations in the GH receptor (GHR), post-GHR signaling pathway, and IGF 1 gene defects; they are not GH deficient, and therefore, they cannot be expected to respond adequately to exogenous GH treatment. In some cases, when deemed necessary, the physician may decide to assist in the diagnosis by performing an IGF-I generation test. Detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu About Eton Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com . SOURCE : Eton