MedTech

Aeglea BioTherapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Pegzilarginase

Aeglea BioTherapeutics
Aeglea BioTherapeutics, Inc. a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced that a Marketing Authorization Application for pegzilarginase for the treatment of Arginase 1 Deficiency has been submitted to and successfully validated by the European Medicines Agency. The MAA was submitted by Immedica Pharma AB, Aeglea's commercialization partner in Europe and the Middle East.

Pegzilarginase is a novel, recombinant human arginase 1 enzyme that in clinical trials has been shown to normalize the elevated levels of the amino acid arginine in patients with ARG1-D, a rare, progressive disease characterized by high levels of arginine. People living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.

"For progressive diseases like ARG1-D, every day that passes without an approved therapy increases the disease burden and worsens the outcome for patients and their families. If approved, we believe pegzilarginase has the potential to benefit these families. We are thrilled with the progress being made with the validation of the MAA and look forward to continuing to work with and support our partner Immedica throughout the EMA review process."

Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea

Dr. Quinn continued, "I would like to thank all the patients, families, investigators, staff and advocates who participated in our clinical trials and helped in the advancement of pegzilarginase, potentially the first health authority-approved treatment to address the underlying driver of ARG1-D, elevated arginine levels."

Immedica's MAA submission includes data from multiple clinical studies in ARG1-D, including the double-blind, placebo-controlled PEACE Phase 3 study and its ongoing long-term extension, a Phase 1/2 clinical trial and an open-label extension study. Results from these trials demonstrate that pegzilarginase is able to rapidly and sustainably lower arginine levels and showed improvements in measures of mobility. In the PEACE study, most treatment-emergent adverse events were mild or moderate in severity and there were no discontinuations due to treatment-emergent adverse events. The EMA has granted pegzilarginase Orphan Drug Designation.

About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality.

The PEACE Phase 3 clinical trial met its primary endpoint with a 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. Aeglea's Phase 1/2 and Phase 2 Open-Label Extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.

About Aeglea BioTherapeutics
Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. Pegzilarginase achieved the primary endpoint of arginine reduction in the PEACE Phase 3 clinical trial and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. Aeglea also has an ongoing Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families.

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Nimbus Therapeutics | January 08, 2024

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Virica Expands Bioprocessing Capabilities with Carleton University Partnership

Virica Biotech | January 17, 2024

Virica Biotech Inc. (“Virica”), a leading developer of cell enhancers for scaling of viral vector as well as cell and gene therapy manufacturing will expand its bioprocessing services through a partnership with Carleton University. Slated to open this spring, Virica’s new facility at the university multiplies the Company’s capacity to provide high throughput virology services for customers looking to optimize production of their cell and gene therapies. “Carleton’s new Health Sciences Building provides us with greater access to modern infrastructure and analytical expertise,” said Jean Simon Diallo, Ph.D., CEO of Virica Biotech. “Their state-of-the-art analytical research facilities and world-class researchers across multiple disciplines open the door to exciting opportunities for partnering with Carleton University as we continue to invest to meet customer demand.” “We are very pleased to welcome Virica Biotech to the Carleton campus,” said Rafik Goubran, Ph.D., Vice-President (Research and International) and Chancellor’s Professor, Carleton University. “This multi-year, multi-million-dollar research and infrastructure partnership will help drive innovation and talent development in the Ottawa region for the creation and manufacturing of advanced therapies.” This partnership expands on Carleton’s history of supporting Ottawa biotechnology companies. The new open concept facility will include high throughput equipment to accelerate Virica’s bioprocess development and optimizations. In addition to expanded modern infrastructure, this new location enables the development and recruitment of world-class talent with experiential learning and training opportunities for Carleton students. Through this partnership, Carleton will coordinate the establishment of a scholarship fund designed to empower graduate students from traditionally underrepresented groups in science. About Virica Biotech Virica develops cell enhancers for viral vectors that improve the yield and quality of vaccines and cell and gene therapies, allowing developers to economically deploy their products at scale. Virica’s Viral Sensitizer (VSE™) platform reduces production inefficiencies caused by anti-viral defenses in manufacturing cells. Purpose formulated VSE combinations substantially increase manufacturing yields and reduce the cost of goods for a range of life-changing products, including vaccines, gene therapies, and cell therapies. About Carleton University Carleton University is a dynamic, research-intensive institution that engages in partnerships to address the world’s most pressing challenges. The university’s corporate collaborations bring together world-class companies, researchers and a new generation of talent with over 30,000 students to deliver innovations and results that are driving a more prosperous, sustainable future.

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Heron Therapeutics Announces FDA Approval of ZYNRELEF® Indication Expansion to Include Additional Orthopedic and Soft Tissue Procedures

Heron Therapeutics, Inc. | January 29, 2024

Heron Therapeutics, Inc. a commercial-stage biotechnology company announced that the U.S. Food and Drug Administration (the "FDA") has approved its supplemental New Drug Application ("NDA") for ZYNRELEF® extended-release solution to expand the indication for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. ZYNRELEF was previously approved for foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures in adults. This expanded indication for ZYNRELEF will now cover an estimated 13 million procedures annually, an estimated increase of 86% over prior indicated procedures. To obtain this labeling expansion, Heron successfully conducted studies for cesarean section, spinal surgery, augmentation mammoplasty, and total shoulder arthroplasty. 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Cell and Gene Therapy

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Solvias | January 23, 2024

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