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).
Benchling | January 27, 2023
On January 27, 2023, Benchling, the R&D cloud that powers the biotechnology sector, announced that it has passed the System and Organization Controls (SOC) 2 certification. Schellman, an independent Certified Public Accounting (CPA) firm, conducted the Type 2 SOC 2 test according to the AICPA's SOC 2 standards.
Type 2 SOC 2 evaluates the design and operational performance of an organization's security processes and controls to meet its security goals and customer commitments during a predetermined evaluation period. Constant monitoring, evaluation, and modification of the requirements and security measures guarantee a secure environment for doing business.
The CISO at Benchling, Zach Powers, said, "Biotechs are managing an evolving landscape of security and compliance risks, and they require trusted software partners to help them meet these demands." He added, "At Benchling, we make the security, privacy, and compliance of our customers' sensitive data a company-wide, critical investment, not only through Type 2 SOC 2, but also through our best-in-class security team, our trust-but-verify processes, and our adherence to global compliance standards."
(Source – PR Newswire)
Benchling's R&D Cloud is the biotech R&D platform of choice, trusted by over 1,000 organizations worldwide in highly regulated areas such as biopharmaceuticals, agribusiness, industrials, and consumer packaged goods. Benchling is used by customers to securely store, standardize, and analyze crucial data related to the design and development of innovative biotech products. Benchling's objective is to protect client data; hence, the firm utilizes sophisticated data security and privacy protections and complies with tight regulatory regulations such as the NIST Cybersecurity Framework. The organization is also ISO 27001 certified.
The successful completion of Benchling's Type 2 SOC 2 inspection assures that its biotech clients can conduct crucial R&D work in the Benchling R&D Cloud while following the highest security requirements.
Benchling is a San Francisco-based firm that focuses on private and public life science cooperation by developing cloud-based software tools for digital DNA sequence editing, planning and performing experiments, evaluating data, and sharing research. More than 200,000 scientists from over 1,000 firms and 7,500 university and research institutions worldwide have used the Benchling R&D Cloud to create breakthrough discoveries and deliver the next generation of medicines, foods, and materials to market more quickly. In addition, the R&D cloud assists these companies in modernizing their scientific processes and accelerating their cooperation to transform the complexity of biology into world-changing outcomes.
MEDTECH, INDUSTRIAL IMPACT
INOVIO Pharmaceuticals, Inc. | February 01, 2023
On January 31, 2023, INOVIO, a leading biotechnology firm focused on the development and commercialization of DNA medicines to protect and treat people from cancer, HPV-associated diseases, and infectious diseases, announced a strategic reorganization to meet operational efficiency commitments and better position the company to deliver DNA medicines to patients.
Following the previously announced end of clinical development projects in late 2022, the restructuring is estimated to result in yearly savings of roughly $4.3 million and an 11% reduction in full-time personnel. These and other strategic initiatives divert resources needed to progress critical programs, such as INOVIO's product candidate INO-3107 for the treatment of Recurrent Respiratory Papillomatosis (RRP).
INOVIO anticipates a $1.1 million one-time restructuring charge in the first quarter of 2023. Its 2022 fourth-quarter and year-end financial results announcement, scheduled for March 2023, will provide more precise financial guidance.
INOVIO's lead initiatives include therapies for HPV-related disorders, such as INO-3107 for RRP and VGX-3100 for cervical high-grade squamous intraepithelial lesions (HSIL). In the first quarter of 2023, INOVIO is anticipated to release the results of its Phase 3 REVEAL2 study for VGX-3100 and the second cohort of its Phase 1/2 trial for INO-3107.
About INOVIO Pharmaceuticals, Inc.
Headquartered in Plymouth Meeting, PA, INOVIO Pharmaceuticals, Inc. is a biotechnology firm dedicated to bringing precisely designed DNA therapeutics to market in order to treat, cure, and protect individuals from infectious diseases, cancer, and HPV-related diseases. INOVIO's in-development DNA medications are delivered utilizing its investigational proprietary smart device, CELLECTRA®, to elicit immune responses against targeted pathogens and malignancies. Its DNA medicines platform and the proprietary smart device can target almost any DNA sequence. With 15 clinical studies in development, the company has the potential to save and protect lives throughout the world and fuel a new decade of DNA medicines.