CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Axcella | February 16, 2023
On February 15, 2023, Axcella Therapeutics, a clinical-stage biotechnology firm that develops innovative approaches to complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a phase 2b/3 trial in the United States for AXA1125 for the treatment of Long COVID Fatigue.
The company stated that it has obtained FDA regulatory guidance in favor of a trial that would serve as the registration trial for patients with Long COVID Fatigue. The trial now has acceptance from both the U.K. and U.S. regulatory authorities.
Last month, the company also announced a regulatory path to registration for the treatment of Long COVID Fatigue with AXA1125, having received regulatory guidance from the U.K.'s regulatory agency, The Medicines and Healthcare products Regulatory Agency (MHRA), supporting a single trial that can serve as the registration trial for patients suffering from Long COVID Fatigue.
The guidance follows the company's submission of data to both regulatory agencies, including results from the Phase 2a randomized, double-blind, placebo-controlled study, which showed that patients who received AXA1125 experienced statistically and clinically significant improvements in both physical (p=0.0097) and mental fatigue (p=0.0097) scores compared to those on the placebo.
Consistent with the Ph2a trial, the trial for phase 2b/3 will register patients with fatigue lasting at least 12 weeks post-COVID-19 infection. The primary endpoint will use the same patient reported outcome tool, the Chalder Fatigue Questionnaire (CFQ-11), to measure fatigue improvements alongside physical function, quality of life, and ability to return to work as additional endpoints. The trial participants will receive AXA1125 or a placebo for three months.
Long COVID is a persistent long-term effect of the pandemic, which affects a large number of patients worldwide, estimated to be around one hundred million. Fatigue is the most frequently reported symptom, with recent estimates indicating that 15-20% of Americans with COVID suffer from ongoing health problems, and up to four million Americans are unable to work because of Long COVID symptoms. In addition, Long COVID has caused approximately $1 trillion in lost earnings and $544 billion in increased medical expenses.
Founded in 2010, Axcella is a clinical-stage biotechnology firm dedicated to introducing a novel approach to treating complex diseases using endogenous metabolic modulator (EMM) compositions. Its product candidates contain EMMs and derivatives that are engineered in distinct ratios and combinations to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. The company's pipeline includes lead therapeutic candidates undergoing Phase 2 development for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence, the treatment of Long COVID, and the treatment of non-alcoholic steatohepatitis (NASH).
INDUSTRIAL IMPACT, MEDICAL
Accesswire | March 24, 2023
Moderna, Inc. a biotechnology company pioneering messenger RNA therapeutics and vaccines, and Generation Bio Co. a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that the two companies have entered into a strategic collaboration to combine Moderna's biological and technical expertise with core technologies of Generation Bio's non-viral genetic medicine platform. The collaboration aims to expand the application of each company's platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines.
"Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Ph.D., Moderna's Senior Vice President for Research and Early Development. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients."
"Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, Ph.D., Chief Strategy Officer of Generation Bio. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of novel targeting for our stealth ctLNPs to reach immune cells."
About the Collaboration
Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed ctLNP to deliver ceDNA. In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver.
Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement. The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio.
Further, Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration.
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Sana Biotechnology | January 30, 2023
Sana Biotechnology, Inc., a firm focused on producing and delivering engineered cells as medicines, recently announced that the FDA has approved the company's Investigational New Drug (IND) application to begin a first-in-human trial of SC291 in patients with different types of B-cell malignancies.
SC291 is an allogeneic CAR T cell therapy that targets CD19. It is developed by Sana using its hypoimmune platform. The hypoimmune platform's objective is to overcome the allogeneic cells' immunologic rejection, which, if valid for SC291, could lead to prolonged CAR T cell persistence and increased rates of durable complete responses in patients with B-cell lymphomas or leukemias.
The hypoimmune platform includes disruption of the major histocompatibility (MHC) class I and class II expression to conceal cells from the adaptive immune system, which consists of antibody and T cell responses and overexpression of CD47 to inhibit activation of the innate immune cell system, especially macrophages and natural killer (NK) cells. The company has provided evidence from numerous preclinical models demonstrating the potential of this platform to cloak cells from immune detection and the therapeutic potential of SC291 for patients with B-cell malignancies.
About Sana Biotechnology
Founded in 2018, Sana Biotechnology is a firm focused on utilizing engineered cells as medicines for patients. It is constructing differentiated capabilities across the spectrum of cell and gene therapy. The company is motivated by three goals as it seeks solutions for people with poor outcomes or diseases that are currently incurable. The first is the ability to repair and control the genes in any cell in the body. It is developing innovative delivery technologies in order to deliver any payload to any cell in a precise, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next comes the ability to develop pluripotent stem cells ex vivo into immune-cloaked functional cells, to replace any missing or damaged cells in the body. Finally, it believes that it can also enable broader access to its therapies by focusing on scalable manufacturing solutions, the manufacturing costs, and aligning with key stakeholders.