Hypophosphatasia

AM-Pharma Initiates Phase 2 Trial of Ilofotase Alfa for Cardiac Surgery-Associated Renal Damage

Retrieved on: 
Tuesday, January 16, 2024

CSA-RD is defined as the long-term loss of renal function in cardiac surgery patients and arises from acute kidney injury (AKI) immediately following the surgery.

Key Points: 
  • CSA-RD is defined as the long-term loss of renal function in cardiac surgery patients and arises from acute kidney injury (AKI) immediately following the surgery.
  • Ilofotase alfa has a proven safety and tolerability profile and a strong record of renal protective effects as observed in previous clinical studies.
  • Notably, prior Phase 2 and Phase 3 trials with ilofotase alfa demonstrated significant improvements in Major Adverse Kidney Events by day 90 (MAKE90) in sepsis-associated AKI.
  • Ilofotase alfa was recently evaluated in a Phase 1b pilot study as enzyme replacement therapy in hypophosphatasia (HPP) patients.

Rallybio Highlights Portfolio Advances and Outlines Expected Milestones in 2024

Retrieved on: 
Thursday, January 4, 2024

Rallybio expects to initiate its Phase 2 dose confirmation study for RLYB212 in the second half of 2024.

Key Points: 
  • Rallybio expects to initiate its Phase 2 dose confirmation study for RLYB212 in the second half of 2024.
  • Rallybio expects to complete this manufacturing work and provide an update on the development plan for RLYB116 in the second half of 2024.
  • Rallybio and EyePoint expect to provide an update on this collaboration in the first half of 2024.
  • Rallybio and Exscientia plan to provide an update on the progress of the program in the second half of 2024.

Rampart Bioscience Announces $85M Series A Financing to Develop Novel DNA-Based Medicines with Veteran Team

Retrieved on: 
Tuesday, October 24, 2023

Rampart Bioscience, Inc., a biotechnology company developing next-generation biologics, today announced an $85 million Series A financing to create medicines that aim to unburden patients from disease and their treatments.

Key Points: 
  • Rampart Bioscience, Inc., a biotechnology company developing next-generation biologics, today announced an $85 million Series A financing to create medicines that aim to unburden patients from disease and their treatments.
  • The new financing builds on an initial $40 million seed investment from OrbiMed.
  • The Rampart team has tremendous heart and a fierce commitment to reshaping the treatment landscape for those in dire need of better options,” said Louis Breton, Rampart Co-founder, Chief Executive Officer, and Board Director.
  • “Rampart’s DNA medicines contain novel structural elements that drive nuclear trafficking and retention while avoiding immune responses,” said Jeffrey Bartlett, Ph.D., Rampart Co-founder and Chief Innovation and Technical Officer.

AM-Pharma Announces Positive Clinical Data From Phase 1b Study Evaluating Ilofotase Alfa in Hypophosphatasia Patients

Retrieved on: 
Wednesday, October 11, 2023

AM-Pharma B.V. today announced positive clinical results from the Phase 1b study evaluating the company’s proprietary recombinant alkaline phosphatase, ilofotase alfa, as potential enzyme replacement therapy in adult hypophosphatasia (HPP) patients.

Key Points: 
  • AM-Pharma B.V. today announced positive clinical results from the Phase 1b study evaluating the company’s proprietary recombinant alkaline phosphatase, ilofotase alfa, as potential enzyme replacement therapy in adult hypophosphatasia (HPP) patients.
  • Patients were followed for 10 days after dosing to evaluate changes in the biochemical fingerprint of HPP, safety and tolerability.
  • In addition, ilofotase alfa was well-tolerated with pharmacokinetics consistent with observations from previous clinical studies.
  • "The encouraging early results confirming dose-dependent reductions of PLP and PPi levels upon treatment with ilofotase alfa underline the compound's therapeutic potential in that indication and I'm looking forward to evaluating ilofotase alfa in next stage clinical studies."

AM-Pharma Announces Updated Clinical Development Strategy

Retrieved on: 
Tuesday, September 26, 2023

AM-Pharma B.V. today announced an updated clinical development strategy to evaluate ilofotase alfa in two indications.

Key Points: 
  • AM-Pharma B.V. today announced an updated clinical development strategy to evaluate ilofotase alfa in two indications.
  • The company’s proprietary compound ilofotase alfa is a recombinant alkaline phosphatase with an extensive clinical record and proven safety profile.
  • “Juliane’s expertise in clinical and corporate development, together with her experience as AM-Pharma’s COO over the past four years, will be extremely useful as the company pursues its development strategy.
  • AM-Pharma’s revised clinical strategy is backed by existing investors who have provided additional funding.

Rallybio Reports Fourth Quarter and Full Year 2022 Financial Results

Retrieved on: 
Monday, March 6, 2023

In the fourth quarter of 2022, we initiated a multiple ascending dose Phase 1 study of RLYB116, our inhibitor of complement component 5 (C5), and we look forward to sharing initial data from this study in the fourth quarter of 2023.

Key Points: 
  • In the fourth quarter of 2022, we initiated a multiple ascending dose Phase 1 study of RLYB116, our inhibitor of complement component 5 (C5), and we look forward to sharing initial data from this study in the fourth quarter of 2023.
  • The Company expects results from this cohort of subjects in the fourth quarter of 2023.
  • Rallybio initiated dosing in the first multiple ascending dose cohort of a Phase 1 study of RLYB116 in the fourth quarter of 2022.
  • Fourth Quarter and Full Year 2021 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $10.8 million for the fourth quarter of 2022, compared to $6.1 million for the same period in 2021.

Rallybio Highlights Portfolio Advances and Outlines Expected Milestones in 2023

Retrieved on: 
Thursday, January 5, 2023

We also significantly expanded our earlier-stage pipeline through strategic business development efforts.”

Key Points: 
  • We also significantly expanded our earlier-stage pipeline through strategic business development efforts.”
    Dr. Mackay continued, “We are excited to build on this progress in 2023.
  • Rallybio also continues to advance the development of RLYB114, formulated for intravitreal injection, for the treatment of ophthalmic disorders.
  • In vivo efficacy data are expected in the second half of 2023.
  • In December 2022, Rallybio announced a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases.

Rallybio Reports Third Quarter 2022 Financial Results

Retrieved on: 
Monday, November 7, 2022

Recent Business Highlights and Upcoming Milestones:

Key Points: 
  • Recent Business Highlights and Upcoming Milestones:
    In September 2022, Rallybio announced preliminary platelet elimination results from its ongoing Phase 1b proof-of-concept study for RLYB212.
  • Rallybio also announced in September 2022 that dosing under the amended protocol has commenced, which increases the dose of RLYB212.
  • Rallybio currently expects to initiate the multiple ascending dose Phase 1 study of RLYB116 in the first quarter of 2023.
  • Third Quarter 2022 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $12.1 million for the third quarter of 2022, compared to $5.0 million for the same period in 2021.

Rallybio Reports Second Quarter 2022 Financial Results

Retrieved on: 
Monday, August 8, 2022

Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases, today reported financial results for the second quarter ended June 30, 2022 and provided an update on recent program and corporate developments.

Key Points: 
  • Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases, today reported financial results for the second quarter ended June 30, 2022 and provided an update on recent program and corporate developments.
  • Rallybio expects to discuss the preliminary platelet elimination data by the end of the third quarter of 2022 and to release PoC data in the first quarter of 2023.
  • Rallybio announced in June 2022 that Jeffrey Fryer, CPA, will retire from his position as Chief Financial Officer (CFO).
  • Second Quarter 2022 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $10.1 million for the second quarter of 2022, compared to $6.8 million for the same period in 2021.

Aruvant Announces Oral Presentation at American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Monday, May 16, 2022

"ARU-1801 is the only gene therapy in development that has been shown to achieve durable responses in patients with severe SCD using only reduced intensity conditioninga key differentiator from other investigational gene therapy and gene editing regimens."

Key Points: 
  • "ARU-1801 is the only gene therapy in development that has been shown to achieve durable responses in patients with severe SCD using only reduced intensity conditioninga key differentiator from other investigational gene therapy and gene editing regimens."
  • Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases.
  • The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products.
  • ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD.