Aegle Therapeutics Receives Rare Pediatric Disease Designation from the FDA for AGLE-102 for Patients with Dystrophic Epidermolysis Bullosa

Aegle Therapeutics | October 06, 2020

Aegle Therapeutics Corporation, a first-in-class biotechnology company committed to delivering cell-free therapy to patients affected by severe dermatological conditions, today announced that the FDA has granted Rare Pediatric Disease (RPD) Designation to AGLE-102™ for the treatment of dystrophic epidermolysis bullosa (DEB), a skin blistering disorder.  AGLE-102 is a composite of mesenchymal stem cell-derived extracellular vesicles that deliver proteins, genetic material and regenerative healing factors to diseased and damaged tissue. "The FDA's grant of Rare Pediatric Disease designation following its earlier grant of Fast Track Designation to AGLE-102 for DEB underscores the significant unmet medical need of children and adults living with this debilitating disease. AGLE-102 has the potential to be the first multifaceted approach to treat this rare patient population," said Shelley Hartman, CEO of Aegle Therapeutics. The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the U.S. If Aegle's new drug application ("NDA") for DEB is approved, Aegle may receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold to another company for their programs.

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Spotlight

The Government is committed to sustainable development. Renewable materials, produced by agriculture as feedstocks for industry and energy, will play a vital part. Plants sustain life and have amazing diversity of form and function. Agriculture, manufacturing industry, end-user businesses and the science base can work together in using this diversity to deliver benefits for the economy, the environment and society.

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CELL AND GENE THERAPY

Aeglea BioTherapeutics Announces Leadership Transition and Corporate Restructuring

Aeglea BioTherapeutics, Inc. | August 25, 2022

Aeglea BioTherapeutics, Inc. a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced a leadership transition and corporate restructuring to focus resources on AGLE-177 in development for patients living with Homocystinuria. The restructuring has further reduced the headcount of the Company resulting in an approximately 25% reduction year-to-date. The Company also plans to realize additional cost savings by transitioning patients out of the ongoing extension studies of pegzilarginase in Arginase 1 Deficiency while it engages with the FDA on a regulatory path forward. The implementation of the restructuring plan will prioritize development of AGLE-177 and result in extending the Company's cash runway into the fourth quarter of 2023. "The restructuring announced today increases the operational focus and capital deployment towards success of the AGLE-177 program in Homocystinuria. We believe AGLE-177 has the potential to become the best-in-class treatment option for these patients and we believe focused execution of the current Phase 1/2 trial and its transition to late-stage development are key strategic priorities for Aeglea." Russell J. Cox, chair of the board of directors of Aeglea The Company announced that Anthony Quinn, M.B., Ch.B., Ph. D., president and chief executive officer, and member of Aeglea's board of directors, has stepped down from his current positions and transitioned to an advisory role effective immediately. The board of directors has appointed Jim Kastenmayer, Ph.D., J.D., the Company's current general counsel, as interim president and chief executive officer as the Company initiates a search for a permanent replacement. Additionally, the Company's chief commercial officer, Michael Hanley, has been appointed chief business officer with expanded responsibilities in internal business operations. Mr. Cox continued, "On behalf of the board of directors, I want to thank Anthony for his leadership and unwavering commitment to patients during his tenure. Under his leadership, Aeglea has made substantial progress over the last five years with programs for ARG1-D and homocystinuria in clinical development and a research pipeline of other human enzyme-based therapeutics. He has built a strong, capable team with the experience to advance AGLE-177 through clinical and commercial development. Aeglea will continue to benefit from Anthony's scientific and clinical expertise, and I look forward to working with Anthony in his new role as an advisor to the Company." "It has been an enormous privilege to have led the evolution of Aeglea from a research organization to a company with development, manufacturing and commercial capabilities," said Dr. Quinn. "I am honored to have worked with the incredible team at Aeglea and am very excited about Aeglea's upcoming milestones. I look forward to continuing my engagement with the company in an advisory role." "I and the rest of the organization are excited to continue our work and focus on our mission to change lives by bringing innovative therapies to underserved rare disease communities. Delivering data from our Phase 1/2 trial for AGLE-177 later this year is our near-term focus in advancing this mission," said Mr. Kastenmayer. About AGLE-177 in Homocystinuria AGLE-177 is a novel recombinant human enzyme, which is engineered to degrade the amino acid homocysteine and its dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease 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. 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. Aeglea is investigating AGLE-177 in an ongoing Phase 1/2 clinical trial for the treatment of Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease Designation. Aeglea's other clinical program, 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 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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INDUSTRIAL IMPACT

Thermo Fisher Scientific’s Largest Single-Use Technology Manufacturing Site Opens in Greater Nashville

Thermo Fisher Scientific Inc. | August 23, 2022

Thermo Fisher Scientific Inc., the world leader in serving science, opened its largest single-use technology manufacturing site in Greater Nashville. The $105 million, 400,000-square-foot facility enables the company to help meet rapidly growing demand for the bioprocessing materials needed to produce vaccines and breakthrough therapies for cancer and other diseases. This new site is part of Thermo Fisher’s $650 million multi-year investment to expand its bioprocessing production capabilities. “Customers depend on our best-in-class technologies, services and expertise. This continued investment in bioprocessing manufacturing allows us to better serve them in delivering greater supply through scalable solutions. As the largest single-use manufacturing site in Thermo Fisher’s network and one of the largest in the world, the Lebanon facility near Nashville enables customers to bring medicines to patients faster than ever before.” Daniella Cramp, senior vice president and president, bioproduction, Thermo Fisher Scientific The Lebanon facility will manufacture customizable, single-use BioProcess Containers and fluid transfer assembly systems that are in high demand by biopharma companies. The completed site will include 92,000 square-feet of clean room and will also include a dedicated line for the recently released Thermo Scientific DynaDrive™ Bioreactor. The site location is bringing critical materials closer to biopharma customers in the region, with Nashville among the top ten fastest-growing biotech hubs in the U.S.1 The Lebanon site currently employs approximately 300 people, and the completed facility will create 1,400 new jobs in roles across engineering, procurement, quality, warehousing, site leadership and more. This site is part of Thermo Fisher’s global bioprocessing supply network that expands across 100 countries to help ensure critical medicines reach patients. The Thermo Fisher network of sites have enabled more than 12 billion COVID-19 vaccine doses and partnered with customers to supply its technology and materials for many currently approved COVID vaccines and therapeutics. “Tennessee’s strong business climate and thriving healthcare sector make our state the ideal location for a company like Thermo Fisher Scientific to thrive,” said Governor Bill Lee. “I thank Thermo Fisher for its decision to invest in Wilson County and create new jobs across the region so that Tennessee continues to lead the nation in innovation and opportunity.” “Tennessee is emerging as a global leader in biotechnology, and new investment from Thermo Fisher Scientific will bring even more innovation to the region,” said Senator Marsha Blackburn. “This new facility will provide high-paying jobs to Tennesseans and create cutting-edge technology to keep our research and development communities thriving.” About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

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MEDTECH

BiOWiSH Technologies and SABIC Agri-Nutrients Company Announce Bio-Enhanced Mineral Fertilizer Collaboration

BiOWiSH Technologies, Inc. | July 22, 2022

BiOWiSH Technologies, Inc. and SABIC Agri-Nutrients Company announced the successful completion of a year-long technical evaluation of BiOWiSH® Crop Liquid technology coated on SABIC’s urea in nine countries across five continents and nine different crop types. BiOWiSH® Crop Liquid is a blend of proprietary microbial cultures that can be coated onto dry fertilizer or mixed with liquid fertilizers to create an enhanced efficiency fertilizer specifically designed to optimize yield potential and improve soil productivity. Featuring the company’s proprietary HoloGene 3™ technology, BiOWiSH® Crop Liquid creates a high-performance partnership between the plant and BiOWiSH microorganisms. The new alliance has a combined genetic profilethat enhances native microbial activity in the soil, promotes soil quality, and supports the plant’s natural ability to resist environmental stress. This optimizes crop yield potential by increasing nutrient availability and improving soil productivity and plant vigor. This unique mode of action, combined with the product’s industry-leading shelf life, is proven to achieve consistent desired results across a broad range of operating conditions, climates and environments – all at a low cost to farmers. “We are thrilled to confirm that SABIC AN Bio-Enhanced Urea powered by BiOWiSH® has performed extremely well in the field,” Munif Al-Munif, General Manager Agri-Nutrients Technology at SABIC AN “Our uniform, high-crushing strength urea combined with the superior technology of BiOWiSH® Crop Liquid delivers extended stability and consistent performance at a price per acre farmers can afford. In an industry challenged with high regulatory pressures and global food demand, we are excited to offer a sustainable product that safely increases crop production and generates higher profits for growers,” he added. The market launch of SABIC AN Enhanced Urea will initially be fulfilled through SABIC AN partners in select target countries while the teams work together to expand in other regions around the world. The launch follows extensive successful testing of the product across multiple international markets, including the United States. The Bio-Enhanced Urea, powered by BiOWiSH®, will be available at the same price as standard urea through the Archer-Daniels-Midland Company (ADM) and American Plant Food (APF) for a limited time in select areas. Customers can contact their ADM or APF representative for more details. In addition, SABIC AN and BiOWiSH have constructed a joint development platform designed to advance further customizations across a range of SABIC AN’s high-efficiency fertilizers. This partnership is the foundation for future products and other agronomic innovations. “We appreciate the close collaboration with SABIC AN’s team,” said BiOWiSH CEO Ian Edwards. “It is very exciting that a market-leading fertilizer company such as SABIC AN recognizes the importance of biologicals and is placing such high focus and energy on bringing Bio-Enhanced Mineral Fertilizer to customers across the globe. Together, we will work toward our shared goal of developing agricultural solutions that help farmers maximize productivity and increase income – while at the same time, improving environmental outcomes.” SABIC AN Bio-Enhanced Urea will be available to farmers in additional locations in the future, enabling growers to experience the benefits of BiOWiSH®-powered fertilizer. About BiOWiSH Technologies Headquartered in Cincinnati, Ohio, BiOWiSH Technologies, Inc. is a global provider of biotechnology solutions for the agriculture, aquaculture, and environmental management industries. As a leader in the agricultural market, we help farmers increase crop production sustainably, safely and cost effectively. Our revolutionary BiOWiSH® Crop Liquid can be coated onto dry fertilizer or mixed with liquid fertilizer to create an enhanced efficiency fertilizer that optimizes yield potential, expresses plant vigor and improves soil productivity across a broad range of operating conditions, climates and environments. By unifying nature and science, BiOWiSH reinvents the way food is grown. About SABIC Agri-Nutrients Company SABIC AN is a public joint stock company listed on the Saudi Stock Exchange where SABIC owns 50.1% with the remaining 49.9% being held by the private sector and the public. It is one of the leading global fertilizer producers. SABIC AN portfolio includes urea, ammonia, phosphate and other specialized products.

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