Biologics license application

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

Retrieved on: 
Tuesday, January 16, 2024

The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.

Key Points: 
  • The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.
  • RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age.
  • “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics.
  • ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment.

Atara Biotherapeutics to Present Recent Progress and Key Upcoming Milestones at the 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced Pascal Touchon, President and Chief Executive Officer of Atara, will present the Company’s 2023 accomplishments across strategic priorities and key upcoming milestones at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11 at 9:45 a.m. PST / 12:45 p.m. EST.

Key Points: 
  • Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced Pascal Touchon, President and Chief Executive Officer of Atara, will present the Company’s 2023 accomplishments across strategic priorities and key upcoming milestones at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11 at 9:45 a.m. PST / 12:45 p.m. EST.
  • “Our off-the-shelf, allogeneic CAR EBV T cell pipeline now spans both oncology and autoimmune indications and is designed to overcome current limitations of autologous CAR T and other allogeneic cell therapy approaches.
  • An archived replay of the webcast will be available on the Company's website for 30 days following the live presentation.
  • A new corporate presentation will be available on Monday, January 8 at 8:00 a.m. EST / 5:00 a.m. PST.

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

Axogen Announces Plan for Leadership Transition with CEO Karen Zaderej to Retire from Company by January 2025

Retrieved on: 
Thursday, January 4, 2024

While the transition is planned to be no later than Jan. 5, 2025, it could occur sooner depending on the timing of a new CEO appointment.

Key Points: 
  • While the transition is planned to be no later than Jan. 5, 2025, it could occur sooner depending on the timing of a new CEO appointment.
  • Ms. Zaderej has led the transformation of Axogen from a small start-up company to a category leader in the growing market of nerve repair.
  • Under her leadership, Axogen has built awareness of the patient quality of life impact related to nerve damage and has created the market for nerve repair.
  • I am proud of the accomplishments of our team and know that Axogen is positioned well for continued growth.

Vaxcyte Provides Clinical and Regulatory Progress Update on Potential Best-in-Class Pneumococcal Conjugate Vaccine (PCV) Franchise

Retrieved on: 
Thursday, January 4, 2024

-- Enrollment in Ongoing VAX-24 Infant Phase 2 Study Continues to Progress; Topline Data from Primary Immunization Series Expected by End of First Quarter of 2025, Followed by Topline Data from Booster Dose by End of 2025 --

Key Points: 
  • Vaxcyte’s carrier-sparing PCV franchise candidates, including VAX-24, a 24-valent PCV proceeding to Phase 3, and VAX-31, the Company’s next-generation 31-valent PCV, are being studied for the prevention of invasive pneumococcal disease (IPD).
  • The VAX-24 Phase 2 infant study continues to enroll participants in the second and final stage of the study.
  • This is a randomized, observer-blind, dose-finding two-stage clinical study evaluating the safety, tolerability and immunogenicity of VAX-24 in healthy infants.
  • Topline safety, tolerability and immunogenicity data from the ongoing adult Phase 1/2 study in the third quarter of 2024.

Medytox, Inc. Announces the Formation of Luvantas Subsidiary and Appointment of Key Executives

Retrieved on: 
Wednesday, January 3, 2024

IRVINE, Calif., Jan. 3, 2024 /PRNewswire/ -- Medytox, Inc. is pleased to announce the formation of Luvantas, Inc. ( www.luvantas.com ) as its wholly-owned North American subsidiary.

Key Points: 
  • IRVINE, Calif., Jan. 3, 2024 /PRNewswire/ -- Medytox, Inc. is pleased to announce the formation of Luvantas, Inc. ( www.luvantas.com ) as its wholly-owned North American subsidiary.
  • More information on this state-of-the-art formulation of Botulinum toxin will be provided once regulatory approvals are obtained.
  • In connection with the formation of Luvantas, Thomas A. Albright has been appointed its President and CEO and Stephanie A. Yee has been appointed its CFO.
  • As CEO, Tom brings 40 years of healthcare experience to Luvantas with medical aesthetic leadership roles since 2000.

INOVIO Plans to Submit a BLA for INO-3107 as a Potential Treatment for RRP in Second Half of 2024 Under Accelerated Approval Program

Retrieved on: 
Wednesday, January 3, 2024

This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.

Key Points: 
  • This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.
  • "Based on productive discussions with the FDA, we believe we now have established a path to submitting a BLA for INO-3107 under the accelerated approval program," said Dr. Jacqueline Shea, INOVIO's President & Chief Executive Officer.
  • "Our plan is to complete the submission of our BLA in the second half of 2024 and request a Priority Review.
  • Concurrently, we will continue advancing our commercial plans, with the goal of being ready to launch INO-3107 in 2025."

ImmunityBio Announces $320 Million Investment by Oberland Capital, with $210 Million Funded at Closing, Bringing Total Financing in 2023 to $850 Million

Retrieved on: 
Tuesday, January 2, 2024

ImmunityBio, Inc. ( NASDAQ: IBRX ), a clinical-stage immunotherapy company (“ImmunityBio” or the “Company”), today announced an up to $320 million royalty financing and equity investment in the Company by Oberland Capital, with $210 million of gross proceeds received at closing on December 29, 2023.

Key Points: 
  • ImmunityBio, Inc. ( NASDAQ: IBRX ), a clinical-stage immunotherapy company (“ImmunityBio” or the “Company”), today announced an up to $320 million royalty financing and equity investment in the Company by Oberland Capital, with $210 million of gross proceeds received at closing on December 29, 2023.
  • The proceeds will also be used to fund ongoing business operations and clinical trials expanding N-803 (Anktiva®) indications into multiple solid tumors.
  • Oberland Capital has also an option to purchase an additional $10 million of common stock at a future date.
  • Jefferies LLC acted as exclusive financial advisor to the Company on the transaction.

EQS-News: Formycon and Fresenius Kabi announce File Acceptance for FYB202, a biosimilar candidate to Stelara® (ustekinumab), by the U.S. Food and Drug Administration

Retrieved on: 
Saturday, December 30, 2023

Formycon and Fresenius Kabi announce File Acceptance for FYB202, a biosimilar candidate to Stelara® (ustekinumab), by the U.S. Food and Drug Administration

Key Points: 
  • Formycon and Fresenius Kabi announce File Acceptance for FYB202, a biosimilar candidate to Stelara® (ustekinumab), by the U.S. Food and Drug Administration
    The issuer is solely responsible for the content of this announcement.
  • Press Release // November 30, 2023
    Formycon and Fresenius Kabi announce File Acceptance for FYB202, a biosimilar candidate to Stelara® (ustekinumab), by the U.S. Food and Drug Administration
    Munich – Formycon AG (ISIN: DE000A1EWVY8/ WKN: A1EWVY) and its commercialization partner Fresenius Kabi today announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Biologics License Application (“BLA”) for FYB202, a proposed biosimilar candidate to Stelara®1.
  • FYB202 is a human monoclonal antibody that targets interleukin 12 and interleukin 23 for the treatment of immune-mediated disorders.
  • “The FDA file acceptance for our Stelara® biosimilar candidate FYB202 underlines the great expertise and capabilities of #TeamFormycon.