IND

Inventiva reports preliminary 2023 fiscal year financial Information¹ and provides an update on its clinical trial NATiV3

Retrieved on: 
Thursday, February 15, 2024

Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, stated: “2023 has been an eventful year for the company.

Key Points: 
  • Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, stated: “2023 has been an eventful year for the company.
  • This allowed us to draw down the second tranche of €25 million of the €50 million EIB loan in January 2024.
  • We have advanced our pivotal NATiV3 Phase III clinical trial with lanifibranor in NASH after the implementation of the revised study design in early 2023, with 913 patients randomized to date.
  • An adverse event of elevated aminotransferases has been reported in a patient enrolled in the trial following a scheduled visit.

Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights

Retrieved on: 
Thursday, February 15, 2024

In addition, the Company provided 2024 financial guidance and business highlights.

Key Points: 
  • In addition, the Company provided 2024 financial guidance and business highlights.
  • “2023 was a year of strong progress for Prothena as we advanced our protein dysregulation portfolio and moved closer to becoming a fully integrated commercial company.
  • Total revenue for the fourth quarter and full year of 2023 included BMS collaboration revenue of $0.3 million and $91.3 million, respectively.
  • As of December 31, 2023, Prothena had $621.0 million in cash, cash equivalents and restricted cash, and no debt.

Prelude Therapeutics Reports Full Year 2023 Financial Results and Outlines Key Objectives for 2024

Retrieved on: 
Thursday, February 15, 2024

WILMINGTON, Del., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Prelude Therapeutics Incorporated (Nasdaq: PRLD), a clinical-stage precision oncology company, today reported its financial results for the fiscal year ended December 31, 2023, and outlined key objectives for 2024.   

Key Points: 
  • “Our partnership with AbCellera represents a strategic step to expand our pipeline, based on our core competencies in medicinal chemistry, cancer biology and clinical development.
  • A second cohort of patients with AML is expected to initiate in the first half of 2024.
  • Under the terms of the agreement, Prelude and AbCellera will jointly discover, develop, and commercialize products emerging from the collaboration.
  • The decrease in general and administrative expenses was primarily due to our continued management of general and administrative expenses.

Nurix Therapeutics Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides a Corporate Update

Retrieved on: 
Thursday, February 15, 2024

NX-2127 exhibited dose-dependent PK, leading to robust and sustained degradation of BTK and biologically relevant degradation of IKZF1 (Ikaros).

Key Points: 
  • NX-2127 exhibited dose-dependent PK, leading to robust and sustained degradation of BTK and biologically relevant degradation of IKZF1 (Ikaros).
  • NX-2127 had a manageable safety profile that was consistent with previous reports for BTK-targeted and immunomodulatory therapies.
  • During the year ended November 30, 2023, Nurix achieved research milestones under its collaborations with Gilead and Sanofi totaling $12.5 million and $7.0 million, respectively.
  • Cash, cash equivalents and marketable securities was $295.3 million as of November 30, 2023, compared to $268.7 million as of August 31, 2023.

Taysha Gene Therapies Provides Update on Deprioritized Pipeline Programs

Retrieved on: 
Thursday, February 15, 2024

DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.

Key Points: 
  • DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.
  • Taysha has been working to find ways to advance its deprioritized programs.
  • On November 13, 2023, Taysha terminated its existing loan and security agreement and entered into a new loan and security agreement that provides consent to allow the Company to transfer intellectual property (IP) for several deprioritized programs to third parties in a more efficient manner.
  • “Today’s announcement demonstrates meaningful progress to advance important development work for several deprioritized programs.

Kurome Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for KME-0584 for the Treatment of Acute Myeloid Lymphoma (AML) and High-Risk Myelodysplastic Syndromes (MDS)

Retrieved on: 
Thursday, February 15, 2024

Kurome Therapeutics Inc. is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared the IND for KME-0584, allowing the company to proceed with a Ph1 clinical trial in relapsed/refractory (R/R) AML and high-risk (HR) MDS patients.

Key Points: 
  • Kurome Therapeutics Inc. is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared the IND for KME-0584, allowing the company to proceed with a Ph1 clinical trial in relapsed/refractory (R/R) AML and high-risk (HR) MDS patients.
  • Kurome plans to initiate the clinical trial in the latter half of 2024.
  • “This is a major milestone for the Kurome team and our collaborators and validates our unique approach to AML and MDS as well as the suitability of KME-0584 for initial clinical testing,” said Jan Rosenbaum, Ph.D., CEO and CSO at Kurome.
  • “We look forward to getting our clinical trial underway and testing our approach of targeting dysregulated immune signalling in the setting of AML and HR-MDS by targeting both IRAK1 and IRAK4 together to improve efficacy in this difficult to treat R/R patient population.”

OnKure Announces the Appointment of Samuel Agresta, M.D., as Chief Medical Officer

Retrieved on: 
Thursday, February 15, 2024

BOULDER, Colo., Feb. 15, 2024 (GLOBE NEWSWIRE) -- OnKure, Inc. today announced the appointment of Samuel Agresta, M.D., M.P.H.

Key Points: 
  • BOULDER, Colo., Feb. 15, 2024 (GLOBE NEWSWIRE) -- OnKure, Inc. today announced the appointment of Samuel Agresta, M.D., M.P.H.
  • as Chief Medical Officer.
  • “Sam’s leadership experience and expertise in oncology drug development will be invaluable as we advance our portfolio,” said Nick Saccomano, Ph.D., Chief Executive Officer of OnKure.
  • Dr. Agresta also served as a Director and Chief Medical Officer at Infinity Pharmaceuticals, Inc. where he was responsible for the Phase 1 and 2 developments of the PI3Ky inhibitor, IPI-549.

Navigating the Investigational New Drug (IND) Application Process, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Thursday, February 15, 2024

TORONTO, Feb. 15, 2024 /PRNewswire-PRWeb/ -- Attend an informative webinar delving into the investigational new drug (IND) application process, a pivotal milestone in drug development. The IND application is an important milestone in drug development. An IND aims to demonstrate that every aspect of the drug is safe for human exposure. Sponsors need to evaluate IND readiness to ensure they have enough data to support the initiation of clinical trials. Submission of an incomplete IND lacking important studies or data can lead to a clinical hold, thus delaying clinical trials and increasing costs for the sponsor.

Key Points: 
  • In this free webinar, learn practical tips and recommendations for successfully navigating the investigational new drug (IND) application process, including actionable insights to enhance the chances of regulatory success.
  • Attendees will get an overview of the required data to support the IND opening and the initiation of clinical trials.
  • TORONTO, Feb. 15, 2024 /PRNewswire-PRWeb/ -- Attend an informative webinar delving into the investigational new drug (IND) application process, a pivotal milestone in drug development.
  • The IND application is an important milestone in drug development.

Agios Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, February 15, 2024

CAMBRIDGE, Mass., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments.
  • A total of 109 patients are on PYRUKYND® therapy, a 9 percent increase from the third quarter of 2023.
  • Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.
  • ET to discuss fourth quarter and full year 2023 financial results and recent business highlights.

Ampio Provides Update on Results from Pre-IND Enabling Studies

Retrieved on: 
Wednesday, February 14, 2024

ENGLEWOOD, Colo., Feb. 14, 2024 /PRNewswire/ -- Ampio Pharmaceuticals, Inc. (NYSE American: AMPE) (the "Company" or "Ampio"), a pre-revenue stage biopharmaceutical company focused on development of a potential treatment for osteoarthritis as part of its OA-201 program, today announced the efficacy results from recently completed nonclinical pre-IND enabling studies with OA-201, Ampio's only product development opportunity.

Key Points: 
  • ENGLEWOOD, Colo., Feb. 14, 2024 /PRNewswire/ -- Ampio Pharmaceuticals, Inc. (NYSE American: AMPE) (the "Company" or "Ampio"), a pre-revenue stage biopharmaceutical company focused on development of a potential treatment for osteoarthritis as part of its OA-201 program, today announced the efficacy results from recently completed nonclinical pre-IND enabling studies with OA-201, Ampio's only product development opportunity.
  • Ampio reported that the efficacy results of these studies do not support an Investigational New Drug ("IND") submission which was anticipated in early 2025.
  • The Company's management and the Board are currently assessing both internal and external options.
  • The Company will be taking immediate actions to preserve its cash in order to be able to adequately fund any option identified by the Board.