Hypoparathyroidism

Ascendis to Spotlight Latest Updates for TransCon™ Rare Endocrinology Disease Portfolio at ENDO 2024

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목요일, 5월 30, 2024
ENDO, Achondroplasia, Endocrine Society, Drug, Health, GHD, Saint Andrew's Society, Hypoparathyroidism, Turner syndrome

“In addition to physician and caregiver presentations in pediatric GHD, we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine Medical Sciences.

Key Points: 
  • “In addition to physician and caregiver presentations in pediatric GHD, we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine Medical Sciences.
  • “And with World Hypoparathyroidism Day coinciding with the first day of ENDO, we are pleased to host an informational booth focused on deepening physician awareness of the significant health and quality-of-life impacts of hypoparathyroidism.”
    Ascendis presentations during ENDO 2024 include:
    June 3, 2:45 – 3:00 p.m ET.
  • Baseline Demographics and Design of the SkybriGHt Registry Study.
  • A US Multi-Center, Prospective, Non-Interventional, Long-Term Effectiveness and Safety Study of Children and Adolescents with Growth Hormone Deficiency Treated with Lonapegsomatropin

Ascendis Pharma Announces Extension of U.S. Food and Drug Administration Review Period for TransCon™ PTH for Adults with Hypoparathyroidism

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화요일, 5월 14, 2024
New Drug Application, Patient, Microsoft Access, EAP, Food, NDA, FDA, PDUFA, Hypoparathyroidism, Pharmaceutical industry

COPENHAGEN, Denmark, May 14, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food and Drug Administration (FDA) notified the Company that information submitted in response to the FDA’s ongoing review of the New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism constituted a major amendment to the NDA. Accordingly, the FDA has extended the PDUFA target action date by three months, to August 14, 2024, to provide time for a full review of the submission.

Key Points: 
  • Accordingly, the FDA has extended the PDUFA target action date by three months, to August 14, 2024, to provide time for a full review of the submission.
  • “We have responded to all requests received to date from FDA and will work with the agency as they continue their review of our NDA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.
  • “Adults with hypoparathyroidism in the United States, who are receiving TransCon PTH in our clinical trials and our Expanded Access Program (EAP) will continue to receive their medication, and the EAP remains open for enrollment for eligible patients.
  • We remain committed to bringing TransCon PTH to adults with hypoparathyroidism in the United States, who face an urgent need for new treatments.”

Septerna to Present Preclinical Data from GPCR Drug Discovery Engine at ENDO 2024

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월요일, 5월 20, 2024
Health, Other Science, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, ENDO, Molecule, Patient, PTH1R, G protein-coupled receptor, TSHR, GPCR, Endocrine system, Hypoparathyroidism, Concept, Injection (medicine), Phosphate, Pharmaceutical industry

Septerna, a biotechnology company discovering and advancing novel, oral small molecule medicines targeting G protein-coupled receptors (GPCRs), today announced that the company will present the first preclinical data on the company’s parathyroid hormone 1 receptor (PTH1R) and thyroid stimulating hormone receptor (TSHR) programs at the Endocrine Society’s Annual Meeting (ENDO 2024) held June 1-4 in Boston.

Key Points: 
  • Septerna, a biotechnology company discovering and advancing novel, oral small molecule medicines targeting G protein-coupled receptors (GPCRs), today announced that the company will present the first preclinical data on the company’s parathyroid hormone 1 receptor (PTH1R) and thyroid stimulating hormone receptor (TSHR) programs at the Endocrine Society’s Annual Meeting (ENDO 2024) held June 1-4 in Boston.
  • “We are excited to present for the first time new preclinical findings from our breakthrough GPCR Native Complex Platform™ at ENDO 2024, marking a significant moment in our evolution to a clinical-stage company,” said Jeffrey Finer, M.D., Ph.D., Chief Executive Officer and Co-founder of Septerna.
  • “Led by our PTH1R program, which has the potential to be the first oral replacement therapy for hypoparathyroidism, these promising preclinical data validate our approach to discover novel GPCR-targeted medicines and reaffirm our dedication to deliver transformative therapies to patients in need of better treatment options.”
    Title: Characterization of a Novel Oral Small Molecule PTH1R Agonist: Proof of Concept for an Alternative to Injectable Peptide-based Therapy for Hypoparathyroidism
    Poster Session (P085): Bone and Mineral Metabolism: Clinical Osteoporosis
    Oral Abstract and Rapid-Fire Session (OR14): Bone and Mineral Metabolism: PTH, Calcium, and Phosphate Metabolism
    Presenter: Jun Zhang, Ph.D., Senior Director of Disease Biology, Septerna
    Presenter: Amer Mirza, Ph.D., Vice President, Disease Biology and Translational Sciences, Septerna

Amolyt Pharma Announces Abstracts Accepted for Presentation at the GRS Congress, ECE and ENDO

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화요일, 5월 7, 2024
ENDO, Cortex, Congress, European Society of Endocrinology, Patient, Hypoparathyroidism, Abstract, Rat, Novel, Acromegaly, Pharmacokinetics, Cohort, Combined, GRS, ECE, Trial of the century, Female, Pharmacodynamics, Fragility, Pharmaceutical industry

LYON, France and CAMBRIDGE, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced that the company has had multiple abstracts accepted for presentation at the 10th International Congress of the Growth Hormone Research Society (GRS), which is being held May 10-11 in Stockholm, Sweden, the 26th European Congress of Endocrinology (ECE), which is being held May 11-14 in Stockholm, Sweden, and at the 2024 Endocrine Society Meeting (ENDO), which is being held June 1-4 in Boston, Massachusetts.

Key Points: 
  • LYON, France and CAMBRIDGE, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced that the company has had multiple abstracts accepted for presentation at the 10th International Congress of the Growth Hormone Research Society (GRS), which is being held May 10-11 in Stockholm, Sweden, the 26th European Congress of Endocrinology (ECE), which is being held May 11-14 in Stockholm, Sweden, and at the 2024 Endocrine Society Meeting (ENDO), which is being held June 1-4 in Boston, Massachusetts.
  • These presentations cover both of Amolyt’s development programs, eneboparatide in Phase 3 for the treatment of hypoparathyroidism and AZP-3813 in Phase 1 for the treatment of acromegaly.
  • Details of the presentations are as follows:
    10th International Congress of the Growth Hormone Research Society (GRS) – May 10-11, Stockholm, Sweden
    Title: Additive Effect of Combined Treatment with the Small Peptide GH Receptor Antagonist, AZP-3813, and the Somatostatin Analog, Octreotide, in Decreasing IGF1 Levels in the Rat
    26th European Congress of Endocrinology (ECE) – May 11-14, Stockholm, Sweden
    Title: Increased Urinary Excretion of Calcium and Nephrolithiasis: Real-Life Data from the Epi-Hypo Cohort of Hypoparathyroidism Patients
    Title: A Phase 1 Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZP-3813, a Novel, Small Peptide Growth Hormone Receptor Antagonist, in Healthy Subjects
    Poster Display: 9:40am CEST on Sunday, May 12 to 3:30pm CEST on Tuesday, May 14, 2024
    Title: Additive Effect of Combined Treatment with the Small Peptide GH Receptor Antagonist, AZP-3813, and the Somatostatin Analog, Octreotide, in Decreasing IGF1 Levels in the Rat
    Title: Increased Bone Fragility Over Time in Women with Chronic Hypoparathyroidism: Real-world Data from the HypoparaNet Italian Cohort
    Poster Display: 9:40am CEST on Sunday, May 12 to 3:30pm CEST on Tuesday, May 14, 2024
    2024 Endocrine Society Meeting (ENDO) – June 1-4, Boston, Mass.
  • Title: A Phase 1 Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZP-3813, a Novel Small Peptide Growth Hormone Receptor Antagonist, in Healthy Subjects
    Title: Increased Fragility of Bone Over Time in Female Patients with Chronic Hypoparathyroidism: Real-world Data from the Italian Cohort
    Title: Enhanced Ability of the Small Peptide GH Receptor Antagonist, AZP-3813, to Decrease IGF1 When Combined with the Somatostatin Analog, Octreotide
    Title: Eneboparatide, a Potent Stimulator of Urinary Calcium Reabsorption, Induces Prolonged Renal Cortex Retention and PTH1 Receptor Signalling

Ascendis Pharma Reports First Quarter 2024 Financial Results

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목요일, 5월 2, 2024
FDA, Endocrine gland, Hypoparathyroidism, Element, GHD, Achondroplasia, Commercial, New Drug Application, Biologics license application, Development, Prescription, ASCO, Turner syndrome, PDUFA, Patient, Society, Melanoma, Vision, Inborn errors of metabolism, Pharmaceutical industry

If approved, U.S. launch planned in Q3

Key Points: 
  • If approved, U.S. launch planned in Q3
    — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million
    COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.

United Kingdom’s MHRA Approves YORVIPATH® (palopegteriparatide) in Great Britain for the Treatment of Adults with Chronic Hypoparathyroidism

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수요일, 4월 24, 2024
Hypoparathyroidism, United, Parathyroid hormone, European Commission, Marketing, PTH, Health care, European, Commission, MHRA, European Medicines Agency, Pharmaceutical industry

YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily.

Key Points: 
  • YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily.
  • “With today’s MHRA approval of YORVIPATH, we are expanding our global geographic reach to meet the needs of adults with chronic hypoparathyroidism in Great Britain,” said Camilla Harder Harvig, Executive Vice President and Chief Commercial Officer at Ascendis Pharma.
  • Orphan status provides 10 years of market exclusivity in Great Britain with respect to similar medicinal products in the approved orphan indication of chronic adult hypoparathyroidism.
  • TransCon PTH is also in development for the treatment of adults with chronic hypoparathyroidism in the United States, Japan, and other countries.

EQS-News: Amolyt Pharma, an Andera Partners’ portfolio company, announces its sale to AstraZeneca

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수요일, 4월 10, 2024
PTHR1, AstraZeneca, Acromegaly, Patient, Sale, Acquisition, Endocrine disease, Cooley LLP, Centerview Partners, Alexion, Pharma, Hypoparathyroidism, Fine chemical

Amolyt Pharma, an Andera Partners’ portfolio company, announces its sale to AstraZeneca

Key Points: 
  • Amolyt Pharma, an Andera Partners’ portfolio company, announces its sale to AstraZeneca
    The issuer is solely responsible for the content of this announcement.
  • Alexion, AstraZeneca Rare Disease is looking forward to welcoming talent from Amolyt Pharma.
  • The company led by Thierry Abribat and an exceptional management team has developed innovative therapies for rare endocrine diseases.
  • Centerview Partners LLC and Goldman Sachs Bank Europe SE acted as financial advisors to Amolyt, and Cooley LLP and Jones Day acted as legal counsels for Amolyt and its shareholders.

Amolyt Pharma Enters into Definitive Agreement to be Acquired by AstraZeneca

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목요일, 3월 14, 2024
PTHR1, AstraZeneca, Acromegaly, Patient, Acquisition, Endocrine disease, Cooley LLP, Centerview Partners, Alexion, Hypoparathyroidism, Pharmaceutical industry

LYON, France and CAMBRIDGE, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- Amolyt Pharma, a global, clinical-stage biopharmaceutical company specializing in developing therapeutic peptides for rare endocrine and related diseases, today announced it has entered into a definitive agreement for AstraZeneca to acquire Amolyt Pharma at a purchase price of $800 million upfront and a potential milestone payment of $250 million. Alexion, AstraZeneca Rare Disease is looking forward to welcoming talent from Amolyt Pharma.

Key Points: 
  • Alexion, AstraZeneca Rare Disease is looking forward to welcoming talent from Amolyt Pharma.
  • We are grateful to the investigators, patients, and patient associations who have supported us to reach these goals,” said Thierry Abribat, Ph.D., Founder and Chief Executive Officer of Amolyt Pharma.
  • Under the terms of the agreement, AstraZeneca will acquire all of Amolyt Pharma’s outstanding shares for a total consideration of up to $1.05B, on a cash and debt free basis.
  • Centerview Partners LLC and Goldman Sachs Bank Europe SE acted as financial advisors to Amolyt, and Cooley LLP and Jones Day acted as legal counsels for Amolyt and its shareholders.

Sofinnova Partners Portfolio Company Amolyt Pharma to be Acquired by AstraZeneca

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목요일, 3월 14, 2024
Professional Services, Health, Other Health, Finance, Clinical Trials, Pharmaceutical, Biotechnology, Partner, PTHR1, AstraZeneca, Disease, Acromegaly, Patient, Lead, Acquisition, Endocrine disease, Cooley LLP, Investment, Series, Centerview Partners, Alexion, Endocrinology, Hypoparathyroidism

We are thankful to Sofinnova Partners and all the investors for their continued confidence in our team and for their support of our strategy,” said Thierry Abribat, Ph.D., Founder and Chief Executive Officer of Amolyt Pharma.

Key Points: 
  • We are thankful to Sofinnova Partners and all the investors for their continued confidence in our team and for their support of our strategy,” said Thierry Abribat, Ph.D., Founder and Chief Executive Officer of Amolyt Pharma.
  • “Congratulations to the Amolyt team on its proposed acquisition by AstraZeneca.
  • Thierry, is a visionary leader and embodies the serial biotech entrepreneurs we partner with,” added Antoine Papiernik, Managing Partner & Chairman of Sofinnova Partners.
  • Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage.

Entera Bio Announces Full Year 2023 Financial Results and Provides Business Updates

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금요일, 3월 8, 2024
OXM, Woman, Injection, Research, Bone fracture, Clinical research associate, Type D, FDA, BMD, PTH, Food, Hypoparathyroidism, Obesity, Coeliac disease, Weight loss, Oxyntomodulin, Cmax, Elective surgery, Sport, Short bowel syndrome, OPKO Health, DSM-IV codes, Ecosystem, SABRE, Patient, D&O, Intellectual property, Fracture, Athlete, Sports medicine, Tablet, Osteoporosis, Insurance

JERUSALEM, March 08, 2024 (GLOBE NEWSWIRE) -- Entera Bio Ltd. (NASDAQ: ENTX), (“Entera” or the “Company”) a leader in the development of orally delivered peptides, today reported financial results and key business updates for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • JERUSALEM, March 08, 2024 (GLOBE NEWSWIRE) -- Entera Bio Ltd. (NASDAQ: ENTX), (“Entera” or the “Company”) a leader in the development of orally delivered peptides, today reported financial results and key business updates for the fourth quarter and year ended December 31, 2023.
  • Following our December 2023 financing, we expect to have sufficient cash to fund operations through the first half of 2025,” said Miranda Toledano, CEO of Entera.
  • Financial Results for the year Ended December 31, 2023
    As of December 31, 2023, Entera had cash and cash equivalents of $11.0 million.
  • Operating expenses for year ended December 31, 2023 were $8.9 million, as compared to $13.0 million for the year ended December 31, 2022.