Acacia gets 2nd FDA rejection as manufacturing fault drags on

fiercebiotech | April 03, 2019

Acacia Pharma has suffered its second FDA rejection in seven months. The British drug developer moved quickly to bounce back from the complete response letter (CRL) it received in October, only to be hit with a second rejection after its contract manufacturer failed to resolve the problems.
After disclosing the first CRL for post-operative nausea and vomiting drug Barhemsys last year, Acacia continued to prepare to introduce the drug in the first half of 2019 in the belief the problems behind the rejection would be quick to resolve. Acacia told investors the issue was limited to one regulatory compliance deficiency at the third party that produces the active ingredient in Barhemsys, and that the FDA had no qualms with the quality of the drug or the R&D data included in the submission. Within one month of the CRL, the contract manufacturer submitted a plan to fix the problem and Acacia refiled for FDA approval of Barhemsys. Shares in Acacia rose sharply in March and April in anticipation of the May 5 PDUFA decision.

Spotlight

Microbial fermentations are used for the production of a wide variety of products including biopharmaceuticals, enzymes, amino acids and antibiotics. Contaminations caused by bacteria or phage can have a significant financial impact upon manufacturers as fermentation raw materials must be replaced, additional downtime for root cause analysis incurred and delays to the production schedule diminish facility productivity.

Spotlight

Microbial fermentations are used for the production of a wide variety of products including biopharmaceuticals, enzymes, amino acids and antibiotics. Contaminations caused by bacteria or phage can have a significant financial impact upon manufacturers as fermentation raw materials must be replaced, additional downtime for root cause analysis incurred and delays to the production schedule diminish facility productivity.

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INDUSTRIAL IMPACT

BioConsortia Showcases Breakthrough Microbial Products and Pipeline of Innovation

BioConsortia, Inc. | September 05, 2022

BioConsortia’s innovative microbial products and technical capabilities were on full display August 30th at its Davis, CA headquarters in an investor-focused Technology Showcase event. BioConsortia demonstrated rapid advancements in microbial product and pipeline developments for reducing nitrogen fertilizers, controlling soil pests and plant diseases for larger and better-quality crop yields, as well as protecting crops post-harvest to avoid food waste, while minimizing environmental impact and protecting our planet. The Showcase event reintroduced CRIMSON™ fungicide and biostimulant ZAFFRE™, two products currently awaiting final registrations, and highlighted BioConsortia’s new fungicides, nematicides and nitrogen fixing products that are progressing through registration and commercialization and moving into evaluation with potential partners, as follows: NO-NEM™ bionematicide advances: BioConsortia highlighted progress in bringing novel, safe and effective nematocidal microbes to market. Experts estimate global crop losses of $125 billion or 14% of crop value each year. Currently available chemical controls like methyl bromide, organophosphates and carbamates have an unacceptable level of toxicity to non-target organisms and are being withdrawn from use. NO-NEM is BioConsortia’s first nematicide product heading into full development phase. It has shown excellent performance against economically important pests in row crops, as well as fruits and vegetables, such as soybean cyst nematodes and root knot nematodes. “Today we highlight the breadth of innovations developed to replace chemical crop inputs with superior microbe-based solutions for nitrogen fixing, disease control, nematode control, crop yields and post-harvest food safety. The recent progress of our pipeline – including some exciting technology breakthroughs - means we are ready to advance multiple major products into the commercialization phase with marketing partners.” Marcus Meadows-Smith, CEO of BioConsortia R&D Pipeline Expanded through Technical Innovation Additions to BioConsortia’s R&D pipeline aim to leapfrog currently available microbial products, providing improved efficacy, increased shelf-stability, and reduced field use rates, as follows Leveraging GenePro™ microbe editing platform for nitrogen-fixing breakthroughs: BioConsortia’s proprietary genomics and gene-editing platform, GenePro™, facilitates the discovery and engineering of a wide diversity of microbes faster and more efficiently. BioConsortia’s scientists affirmed GenePro has resulted in the discovery of novel genetic components driving nitrogen-fixation. BioConsortia’s proprietary bacterial strains fix nitrogen under conditions other N-fixing strains will not. This discovery validates the company’s position as a leader in microbe-derived alternatives for agriculture, has resulted in proprietary intellectual property, and will continue to drive our research focus. Expanding library through AMS: BioConsortia’s patent-protected “AMS” - Advanced Microbial Selection™ platform uses evolutionary biology practices to find beneficial microbes that the plant has selected for specific gains. AMS underpins the breadth, depth, and success of BioConsortia’s product pipeline. The company announced the addition of more than 2000 rice diazotrophic microbial strains to its library, following a research program using AMS. This large addition to the library has greatly accelerated BioConsortia’s nitrogen-fixation project for rice, a crop with a paddy production model that represents a unique opportunity to impact the sustainability practices of both large and small growers of one of the world’s most fertilized crops. Novel Bactericide Project: BioConsortia also introduced a new project focused on optimizing microbes for the production of metabolites active against bacterial disease. Project leaders highlighted early technological success within this project – again reliant on the GenePro platform, which focuses on utilizing novel tools to address evolving bacterial disease resistance and increasing bacterial pest pressure resulting from climatic challenges and global trade flows. Novel Multi-spectrum Insecticide: Additionally, the company updated progress on a novel, developmental microbial active ingredient designed to deliver broad-spectrum control of both nematodes and critically important coleopteran and lepidopteran pests. The project aims to deliver a novel mode of action to support and extend crop trait lifecycles in the face of emerging insect resistance. Nematicide leads were originally characterized as strong root colonizers employing the company’s RhizoViz™ system, which uses proteins to visualize colonization robustness in different soils and environmental conditions. BioConsortia’s nematicide pipeline is now among the most expansive in agricultural research. “BioConsortia’s pipeline progress highlights the strengths of the company’s R&D platform – unique in the industry in terms of discovery of plant-affiliated microbes and their optimization as solutions for agriculture,” said Dr. Hong Zhu, Senior VP of Research and Development at BioConsortia. About BioConsortia BioConsortia, Inc. develops superior microbial products that protect plants, enhance fertility, and increase yields while improving the sustainability of agriculture for our environment. Pioneering the use of directed selection within microbial communities, our patented Advanced Microbial Selection process and cutting-edge GenePro genomics and gene-engineering platform enable us to predict, design, and unleash the natural power of microbes.

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CELL AND GENE THERAPY

Kriya Acquires Redpin Therapeutics, Adding Neurology Pipeline to Gene Therapy Portfolio

Kriya Therapeutics | November 17, 2022

Kriya Therapeutics, Inc., a fully integrated gene therapy company advancing a broad portfolio of innovative therapeutics announced the acquisition of Redpin Therapeutics, Inc., a privately held biotechnology company developing regulatable gene therapies for intractable diseases of the nervous system. The acquisition serves as the foundation for Kriya’s neurology therapeutic area portfolio, with two lead gene therapy programs focused on epilepsy and trigeminal neuralgia. Redpin’s proprietary chemogenetics platform can selectively activate or silence disease-causing neurons, while leaving normal functioning cells unaffected. Currently available treatments for neurological disorders typically rely on systemically administered drugs or surgical interventions to address local neuron dysfunction. However, these approaches can have limited efficacy and off-target side effects. In contrast, chemogenetics leverages gene products that are selectively responsive to a chosen small molecule. Redpin’s technology utilizes gene therapy to express engineered ion channels that are responsive to modulation by the FDA-approved anti-smoking drug varenicline to either inhibit specific overactive neurons or stimulate underactive ones. “Redpin’s innovative chemogenetics platform has the potential to transform the lives of patients suffering from intractable neurological conditions. We are looking forward to integrating Redpin’s platform and pipeline into Kriya’s gene therapy engine as we advance our mission to develop life-changing gene therapies that can address diseases affecting millions of patients around the world.” Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya “Redpin has a built a breakthrough, validated chemogenetics approach that could have significant potential in the targeted treatment of neurological disorders,” said Dr. Elma Hawkins, Redpin Therapeutics’ President, Chief Executive Officer and Co-founder. “Kriya Therapeutics is the perfect company to take this leading ion channel-based platform forward and deliver an effective treatment option for patients. We look forward to working with the Kriya team to progress these unique targeted therapies to patients in need as rapidly as possible.” Redpin’s investors include 4BIO Capital, Arkin Bio Ventures, Takeda Ventures Inc, New York Ventures and Alexandria Venture Investments. About Epilepsy Epilepsy is a brain disorder that causes recurrent spontaneous seizures. Approximately 3.4 million people in the U.S. suffer from active epilepsy, according to the U.S. Centers for Disease Control and Prevention. Current treatments for epilepsy include medication, neurostimulation and surgery to remove sections of the affected part of the brain. However between 20% to 40% of patients with newly diagnosed epilepsy will become refractory to treatment, and will continue to suffer seizures. Uncontrolled epilepsy can lead to sudden unexpected death in epilepsy (SUDEP). About Trigeminal Neuralgia Trigeminal neuralgia also known as Tic Douloureux, is a severe and chronic disorder that affects the trigeminal, or fifth cranial, nerve. TN patients experience progressive intense stabbing facial pain attacks that can become more severe and last longer over time. Approximately 10,000 to 15,000 new cases of TN occur each year in the U.S., according to the National Organization for Rare Disorders. While currently available treatments include anti-seizure medications and surgical procedures, a significant proportion of patients are refractory to these approaches. About Kriya Therapeutics Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market and delivering significant reductions in cost. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in metabolic disorders, neurology and ophthalmology. Kriya is backed by leading life sciences and technology investors, and has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. About Redpin Based in New York City, Redpin Therapeutics is a privately held, preclinical stage gene therapy company developing a proprietary chemogenetics platform for targeted cell therapies to address currently intractable diseases of the central nervous system. Using a powerful and innovative technology that inverts traditional drug development by using gene therapy to target an engineered receptor to any cell type responsible for disease and modulating its function with an already-approved drug. This has the potential to deliver effective treatments for a wide range of currently intractable neurological and psychiatric diseases and disorders. Redpin has a worldwide exclusive license from the Howard Hughes Medical Institute for the therapeutic use of this technology, and the company is working with urgency to apply it to advance important medicines for patients in desperate need of effective therapies.

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CELL AND GENE THERAPY

Spatial Biology CRO Flagship Biosciences Acquires Pharma Services Business from Interpace Biosciences

Flagship Biosciences, Inc. | September 01, 2022

Flagship Biosciences, Inc., a leader in spatial biology and biomarker analytics services, announced the acquisition of Interpace Pharma Solutions® a division of Interpace Biosciences and provider of cytogenetic, molecular pathology, and genomic profiling solutions. This acquisition follows a growth equity investment in Flagship from Ampersand Capital Partners, BroadOak Capital Partners, and Research Corporation Technologies. The strategic combination of Flagship and IPS creates a full repertoire of biomarker lab and analytic services to advance precision therapeutic development and adds a state-of-the-art laboratory in Research Triangle Park, NC. Flagship Biosciences offers cutting-edge spatial biology services, powered by its patented AI image analysis technology that improves the accuracy of tissue-based pathology by delivering thousands of measurements on every cell and enabling the discovery of biomarkers that might be missed using traditional histology methods. Experts in pathology and regulatory strategy, the team at Flagship offers advanced end-to-end biomarker and analytics services to support drug trials, biomarker discovery, and clinical diagnostics. They also offer guidance in the development of companion diagnostics. To better serve their biopharma and diagnostics clients, Flagship Biosciences will integrate the technologies and services of IPS to support all phases of clinical trials and diagnostic development in immuno-oncology, hematology, solid tumors, and various non-oncology sectors. Flagship Biosciences' expanded services menu will now include advanced molecular biomarker solutions, flow cytometry, cytogenetics, genomics, and bioinformatics solutions. New genomic profiling capabilities will enhance biomarker characterization and improve patient stratification for clinical trials and treatment selection. "By combining these organizations, we create a single-source provider offering a larger range of biomarker and analytics services while maintaining and expanding the proven expertise of each laboratory. This will allow our customers to gain deeper insights into their clinical projects through multiple biomarker testing paradigms with cohesive data and analytical capabilities, managed in a simplified project workflow." Trevor Johnson, CEO at Flagship Biosciences About Flagship Biosciences Founded in 2009 and headquartered in Broomfield, Colorado, Flagship Biosciences, Inc. is a technology-driven tissue analysis services company delivering the most accurate and informative data available. We are revolutionizing tissue analysis to improve drug development and diagnostics using the power of AI with a consultative approach. Our services and technology dramatically improve on the data and interpretation from traditional pathology methods, eliminating variability associated with typical tissue assessments, and bringing new insights to tissue analysis results. We provide expert scientific consultation for every client. Our team interprets results, contextualizes tissue biology, and identifies the best course for success. For more information, please visit flagshipbio.com and follow us on Twitter and LinkedIn. About Interpace Pharma Solutions Based in Research Triangle Park, NC, Interpace Pharma Solutions provides complex molecular analysis services supporting clinical trials and diagnostic development. These services include genomic profiling, biomarker solutions, project and data management, bioinformatics analysis, and biorepository services. About Ampersand Capital Partners Founded in 1988, Ampersand is a middle market private equity firm with more than $3 billion of assets under management dedicated to growth-oriented investments in the healthcare sector. With offices in Boston, MA and Amsterdam, Netherlands, Ampersand leverages a unique blend of private equity and operating experience to build value and drive superior long-term performance alongside its portfolio company management teams. Ampersand has helped build numerous market-leading companies across each of the firm's core healthcare sectors. About BroadOak Capital Partners BroadOak Capital Partners, with headquarters in Washington, DC, and San Francisco, is a life-sciences focused boutique financial institution that provides direct investment and investment banking services to companies in research tools and consumables, diagnostics, biopharma services, and adjacent markets. BroadOak has led or participated in investments in more than fifty companies across multiple funds and investment vehicles. About Research Corporation Technologies Research Corporation Technologies is a Tucson, Arizona based technology investment and management company that provides funding and development for promising life science and biomedical companies. RCT has assets of more than $500 million to advance technology and product development through flexible, long-term investment options.

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