American Society for Bone and Mineral Research

Rani Therapeutics Reports Third Quarter 2023 Financial Results; Provides Corporate Update

Retrieved on: 
Wednesday, November 8, 2023

In October 2023, Rani announced preclinical data from a 60-day, repeat oral-administration GLP safety study of the RaniPill capsule in healthy animals.

Key Points: 
  • In October 2023, Rani announced preclinical data from a 60-day, repeat oral-administration GLP safety study of the RaniPill capsule in healthy animals.
  • Presented an Abstract on RT-102 at the 2023 Annual Meeting of the American Society for Bone and Mineral Research.
  • Rani has completed two preclinical studies of the RaniPill HC with antibodies, adalimumab and an undisclosed interleukin (“Interleukin”).
  • Initiation of Phase 2 clinical trial of RT-102, a RaniPill GO containing teriparatide for osteoporosis, expected by year-end 2023.

Inozyme Pharma Reports Third Quarter 2023 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, November 7, 2023

BOSTON, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today reported financial results for the third quarter ended September 30, 2023 and provided business highlights.

Key Points: 
  • Patient recruitment is underway, and the Company remains on track to report topline data in mid-2025.
  • Exploratory efficacy data reported suggested clinical benefit for ENPP1 Deficiency, including improvement in key biomarkers, patient-reported outcomes (PROs), and functional outcomes.
  • R&D Expenses were $13.3 million for the quarter ended September 30, 2023, compared to $12.2 million for the prior-year period.
  • Net loss was $16.6 million, or $0.29 loss per share, for the quarter ended September 30, 2023, compared to $16.4 million, or $0.38 loss per share, for the prior-year period.

Ultragenyx Reports Third Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, November 2, 2023

NOVATO, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended September 30, 2023 and provided its financial guidance for the year.

Key Points: 
  • For the full year 2023, the company expects:
    Crysvita revenue in the range of $325 million to $340 million.
  • This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
  • In May 2023, Ultragenyx announced the last patient had been dosed in the Phase 3 study.
  • ET to discuss the third quarter 2023 financial results and provide a corporate update.

NextCure Provides Business Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023

Financial Results for Quarter Ended September 30, 2023

Key Points: 
  • Financial Results for Quarter Ended September 30, 2023
    Cash, cash equivalents, and marketable securities as of September 30, 2023, were $118.2 million as compared to $159.9 million as of December 31, 2022.
  • Research and development expenses were $11.0 million for the quarter ended September 30, 2023, as compared to $13.5 million for the quarter ended September 30, 2022.
  • General and administrative expenses were $4.6 million for the quarter ended September 30, 2023, as compared to $5.7 million for the quarter ended September 30, 2022.
  • Net loss was $14.3 million for the quarter ended September 30, 2023, as compared with a net loss of $18.9 million for the quarter ended September 30, 2022.

Preclinical Data Demonstrate Anti-Siglec-15 Treatment Reduced Bone Loss and Enhanced Bone Quality in Mice with Moderate-to-Severe Osteogenesis Imperfecta (OI)

Retrieved on: 
Tuesday, October 17, 2023

These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.

Key Points: 
  • These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.
  • Osteogenesis imperfecta is a rare disorder that results in high bone turnover, abnormal bone formation, bone fragility and recurrent fractures.
  • There is no cure for OI and current anti-resorptive treatments increase bone mineral density (BMD) primarily by inhibiting bone loss; however, these agents also inhibit bone formation.
  • Unlike anti-resorptive therapies, NC605 enhances osteoblast recruitment, resulting in overall enhanced bone quality.

Ascendis Pharma Presents TransCon™ PTH (palopegteriparatide) Phase 3 52-Week Skeletal Dynamics Data at ASBMR 2023

Retrieved on: 
Monday, October 16, 2023

COPENHAGEN, Denmark, Oct. 16, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) shared 52-week data from the open-label extension period of its ongoing Phase 3 PaTHway Trial of TransCon PTH (palopegteriparatide) showing that adults with chronic hypoparathyroidism, whose bones tend to be over-mineralized due to insufficient parathyroid hormone (PTH) exposure, trended toward a new skeletal steady state closer to age-appropriate norms with continued use of TransCon PTH. The results were consistent regardless of sex, menopausal status, or duration of disease and were consistent with results previously reported through Week 110 in the Company’s Phase 2 PaTH Forward Trial.

Key Points: 
  • The results were consistent regardless of sex, menopausal status, or duration of disease and were consistent with results previously reported through Week 110 in the Company’s Phase 2 PaTH Forward Trial.
  • On September 14, 2023, TransCon PTH received a positive CHMP opinion recommending approval in the European Union for the treatment of adults with chronic hypoparathyroidism.
  • TransCon PTH is also in development in the United States and Japan.
  • Registered attendees of ASMBR 2023 conference can access the abstract, poster, and presentation (#1114) through the conference organizer’s website .

Entera Bio Reports Rapid Pharmacodynamic (PD) Response and Consistent Pharmacokinetic (PK) Data for its First-in-Class Oral PTH(1-34) Mini Tablets at the ASBMR 2023 Annual Meeting

Retrieved on: 
Monday, October 16, 2023

A Phase 1 study comparing oral EB613, subcutaneous (SC) hPTH(1-34) 20 g (Forteo®) and a new generation of Entera’s oral peptide delivery platform is ongoing.

Key Points: 
  • A Phase 1 study comparing oral EB613, subcutaneous (SC) hPTH(1-34) 20 g (Forteo®) and a new generation of Entera’s oral peptide delivery platform is ongoing.
  • One of the first objectives of this study is to rapidly evaluate the pharmacodynamic (PD) effects of Entera’s oral PTH(1-34) tablets.
  • Abstract Title: Pharmacokinetic (PK) Profile of EBP05/EB613 Oral Teriparatide Tablets in Women of Post Menopausal Age Versus Young Adult Men.
  • “The data showed a consistent PK profile following administration of oral EBP05 tablets in both young men and women of menopausal age.

Amolyt Pharma Announces Data on Prevalence of Bone Disease in Patients with Hypoparathyroidism and Favorable Bone Effects of Eneboparatide at the American Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting

Retrieved on: 
Monday, October 16, 2023

LYON, France, and CAMBRDIGE, Mass., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced data from two posters that were presented at the American Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting, which is being held October 13-16, 2023, in Vancouver, BC, Canada. The data include a prospective analysis of bone disease in patients with hypoparathyroidism from the Canadian National Hypoparathyroidism Registry with evidence of significant bone disease burden in postmenopausal females (PMF). In addition, data from both pre-clinical and clinical studies of eneboparatide continue to demonstrate its ability to restore balanced bone turnover, preserving bone integrity, while maintaining calcium homeostasis and normalizing urinary calcium.

Key Points: 
  • The data include a prospective analysis of bone disease in patients with hypoparathyroidism from the Canadian National Hypoparathyroidism Registry with evidence of significant bone disease burden in postmenopausal females (PMF).
  • In addition, data from both pre-clinical and clinical studies of eneboparatide continue to demonstrate its ability to restore balanced bone turnover, preserving bone integrity, while maintaining calcium homeostasis and normalizing urinary calcium.
  • “We’re very pleased to have a strong presence at this year’s ASBMR Annual Meeting.
  • These data increase our understanding of the relationship between hypoparathyroidism and bone strength.

MBX Biosciences Presents Positive Results from Phase 1 Study of MBX 2109 in Late-Breaking Oral Presentation at the American Society for Bone and Mineral Research Annual Meeting

Retrieved on: 
Monday, October 16, 2023

CARMEL, Ind., Oct. 16, 2023 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc., a clinical stage biopharmaceutical company developing Precision Endocrine Peptide™ (PEP™) therapeutics to treat an array of endocrine disorders, today announced the presentation of positive results from its Phase 1 single and multiple ascending dose trial of MBX 2109 in healthy adults, as a late-breaking oral presentation at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, held in Vancouver, Canada from October 13-16, 2023. MBX 2109 is the company’s long-acting parathyroid hormone (PTH) peptide prodrug in development for the treatment of hypoparathyroidism (HP).

Key Points: 
  • MBX 2109 is the company’s long-acting parathyroid hormone (PTH) peptide prodrug in development for the treatment of hypoparathyroidism (HP).
  • Single and repeat doses of MBX 2109 were generally well-tolerated, and the majority of treatment emergent adverse events were mild in severity.
  • The observed PK parameters following single and repeat doses of MBX 2109 reflected the intended prodrug design and support once-weekly dosing.
  • MBX Biosciences is preparing for an End-of-Phase 1 meeting with the U.S. Food and Drug Administration this quarter, and the company anticipates initiating its Phase 2 trial of MBX 2109 in patients with HP thereafter.

Samsung Bioepis Presents Phase 1 and 3 Clinical Results for SB16, a Proposed Biosimilar to Proliaⁱ (denosumab), at ASBMR 2023

Retrieved on: 
Monday, October 16, 2023

The primary PK endpoints were area under the concentration-time curve (AUC) from time zero to infinity, AUC from time zero to the last quantifiable concentration, and maximum serum concentration.

Key Points: 
  • The primary PK endpoints were area under the concentration-time curve (AUC) from time zero to infinity, AUC from time zero to the last quantifiable concentration, and maximum serum concentration.
  • “We are pleased to present clinical data for SB16 for the first time at the ASBMR Annual Meeting.
  • Through Phase 1 and 3 clinical studies, SB16 demonstrated biosimilarity to reference denosumab, meeting all the primary endpoints in both studies,” said Ilsun Hong, Vice President and Product Evaluation Team Leader at Samsung Bioepis.
  • The details of the SB16 abstracts presented at ASBMR 2023 Annual Meeting are as below: