CELL AND GENE THERAPY
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.
Orion Biotechnology | July 15, 2022
Orion Biotechnology Canada Ltd, a drug discovery company targeting previously undruggable G Protein-Coupled Receptors and Peptilogics, a biotech company engineering peptide therapeutics by combining computation and biology to improve the treatment landscape for patients with life-threatening diseases, announced today that they have entered a research and development collaboration to leverage AI for drug discovery against an undrugged GPCR target.
The collaboration will combine the capabilities of Peptilogics’ AI platform (NautilusTM) with Orion Biotechnology’s proprietary drug discovery platform. The companies will leverage expertise in peptide design and engineering to drive drug discovery against an undrugged peptidergic GPCR linked to highly prevalent, life-threatening diseases.
Peptilogics’ Nautilus™ platform enables in silico predictive peptide design across diverse targets to efficiently access new functional chemical space and custom-design therapeutics. Additionally, Peptilogics’ supercomputer accelerates model development, prediction and evaluation of peptide sequences to produce higher quality hits with greater chance of success. Synergistically, Orion Biotechnology’s platform explores receptor-ligand shape space using proprietary multiplex synthesis technology to generate precision-engineered peptides with enhanced potency and tailored signaling activity. Orion’s platform generates data matrices for AI-assisted lead optimization, offering one of the fastest drug discovery and development solutions to target peptidergic GPCRs.
On the announcement of this collaboration, Dr. Oliver Hartley, VP Drug Discovery at Orion Biotechnology, commented, “We are pleased to be partnering with Peptilogics to push the boundaries of receptor pharmacology and AI computing to accelerate innovation for improved patient outcomes. An AI-driven paradigm shift is happening in drug discovery, and Orion is pleased to be at the forefront of applying AI to the highly valuable space around GPCRs. The ideal fit between Orion’s platform and unique knowledge of receptor-ligand interactions with Peptilogics’ machine learning models will position us to make AI-driven GPCR drug discovery a reality.”
“Using Peptilogics’ Nautilus™ platform, we combine proprietary deep generative models, predictive models and biophysical simulation to design multiparameter-optimized peptides that have the potential to address historically challenging and novel drug targets. Our AI algorithms enable efficient navigation of the vast peptide design space, allowing us to uncover molecules that may not be discovered through traditional screening. We are excited to partner with Orion Biotechnology for their deep GPCR expertise. By combining the strengths of Peptilogics’ Nautilus™ AI-driven peptide design platform and Orion’s GPCR platform, we aim to reduce the risk, time and cost for drug design.”
Nicholas Nystrom, Ph.D., Chief Technology Officer of Peptilogics
About Orion Biotechnology
Orion Biotechnology Canada Ltd. is unlocking the therapeutic potential of previously undruggable G Protein-Coupled Receptors (GPCRs). Orion’s world-renowned experts in GPCR pharmacology and peptide/protein engineering leverage Orion’s proprietary drug discovery platform to rapidly and efficiently advance a diversified portfolio of precision engineered GPCR-targeted therapeutic candidates for the treatment of cancer and other serious diseases.
Peptilogics engineers peptide therapeutic candidates to radically improve the treatment landscape for patients with life-threatening diseases. Through biological and pharmaceutical expertise, novel artificial intelligence algorithms, and purpose-built super-computing, Peptilogics is advancing an extensive therapeutic pipeline and accelerating discovery efforts at a pace and scale that was previously impossible. Peptilogics is backed by visionary investors in life science and technology including Peter Thiel, Presight Capital, Founders Fund, CARB-X, and Cystic Fibrosis Foundation.
Arbor Biotechnologies Inc | August 29, 2022
Arbor Biotechnologies, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that it has entered into an agreement with Acuitas Therapeutics, a leader in the development of lipid nanoparticles.
As part of the agreement, the companies will combine the optimized delivery of Acuitas' highly validated LNP technology with Arbor’s differentiated, proprietary CRISPR gene editing technology designed for use in vivo in patients with rare liver diseases.
“We are building a robust, proprietary portfolio of genomic medicines, beginning with severe liver diseases, for which LNPs are known to provide an optimal delivery approach with their ability to efficiently target hepatocytes, limit off target toxicity and have minimal immunogenicity. We are looking forward to working with Acuitas, a leading global developer of clinically-validated LNP technology. Importantly, we believe this partnership accelerates our path to the clinic, with an ability to leverage established and scalable manufacturing.”
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
Commented Dr. Thomas Madden, President & CEO of Acuitas Therapeutics: “We are excited to collaborate with Arbor on the development of novel genomic medicines for patients who currently have few, if any, therapeutic options. Arbor’s commitment to addressing this unmet clinical need resonates with Acuitas. We look forward to supporting their advance into the clinic.”
About Arbor Biotechnologies
Arbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, our proprietary discovery engine is focused on discovering genetic editing capabilities spanning knockdowns to whole gene insertions, which has enabled us to generate the most extensive toolbox of proprietary genomic editors in the industry to date. Leveraging our wholly-owned nucleases as the chassis for genetic modification, we can work backward from disease pathology to choose the optimal editing approach that specifically addresses the underlying cause of disease, resulting in a potentially curative medicine for a wider range of genetic disorders. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology.
About Acuitas Therapeutics
Founded in February 2009, Vancouver-based Acuitas Therapeutics is a private biotechnology company that specializes in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles. The company partners with pharmaceutical and biotechnology companies, as well as non-governmental organizations and academic institutes to advance nucleic acid therapeutics into clinical trials and to the marketplace. The team works with partners to develop new therapies to address unmet clinical needs based on its internationally recognized capabilities in delivery technology. Acuitas Therapeutics has agreements in place with several partners to use its proprietary lipid nanotechnology in the development of COVID-19 vaccines. These include Pfizer/BioNTech for COMIRNATY®, which has received full approval in the U.S. and Canada and is authorized for Emergency Use in Europe, the UK and many other countries. The Acuitas team is currently working on therapeutics focused on addressing cancer, HIV/AIDS, tuberculosis, malaria, rabies, and other serious diseases.