Researchers develop new compound that offers superior therapeutic approach to treat multiple sclerosis

Medical Xpress | February 21, 2019

Multiple sclerosis is an autoimmune disease that affects more than 2.3 million people worldwide. This debilitating condition periodically shutters communication between the brain and other parts of the body, resulting in symptoms that range from numbness and tingling in the arms and legs to blindness and paralysis. While treatments are available to alleviate inflammation, no therapies exist to protect neurons or repair the degraded myelin sheath that normally surrounds nerves. Previous studies have shown that estrogens and estrogen-like compounds reduce multiple sclerosis-like inflammation and disability in mice. At first glance, these treatments appear promising, but they carry a host of negative side effects, from feminizing male mice to increase the risk of cancer, heart disease, and stroke. An interdisciplinary team of scientists has piggy-backed on this approach with a new concept that sidesteps the negative side effects while alleviating symptoms.

Spotlight

The international regenerative medicines market – which includes the likes of cell therapies, gene/gene-modified cell therapy and tissue engineering – is set to climb to around $67 billion by 2020.  Alongside, discovering therapies that will treat cancer and orphan diseases, many in the industry are hungry to land grab market share and produce the next blockbuster product.

Spotlight

The international regenerative medicines market – which includes the likes of cell therapies, gene/gene-modified cell therapy and tissue engineering – is set to climb to around $67 billion by 2020.  Alongside, discovering therapies that will treat cancer and orphan diseases, many in the industry are hungry to land grab market share and produce the next blockbuster product.

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MEDICAL

Palatin Announces Initiation of Pivotal Phase 3 PL9643 MELODY-1 Clinical Trial in Patients with Dry Eye Disease

Palatin Technologies, Inc | December 29, 2021

Palatin Technologies, Inc. a specialized biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin peptide receptor system, announced the initiation of the pivotal Phase 3 MELODY-1 clinical study of PL9643 in patients with dry eye disease. The study is designed to enroll up to 400 patients at multiple U.S. sites, and top-line results are currently expected second half calendar 2022. "We are excited to initiate the MELODY-1 study of PL9643 in patients suffering from dry eye disease. This is the second melanocortin peptide Palatin has advanced into Phase 3, demonstrating Palatin's expertise and efficiency in melanocortin peptide development for indications with unmet medical need." Carl Spana, Ph.D., CEO and President of Palatin The pivotal Phase 3 MELODY-1 clinical study is a multi-center, randomized, double–masked and vehicle–controlled study evaluating the safety and efficacy of the melanocortin agonist, PL9643 ophthalmic solution, compared to vehicle in subjects with dry eye disease . The study design is based on positive Phase 2 results of PL9643 for the treatment of dry eye disease, and an end-of-phase 2 meeting with the with the U.S. Food and Drug Administration, reaching agreement on all key elements of a pivotal Phase 3 clinical program, including study design, endpoints, interim assessment, and patient population. In addition, to support a New Drug Application filing, a second phase 3 study and long-term safety study will be required. If the program progresses as planned, top-line results from MELODY-1 would be available second half calendar 2022, MELODY-2 data read out second half calendar 2023, with a potential NDA submission first half calendar 2024. "Progressing PL9643 into Phase 3 is a significant milestone for the Company and patients with DED," said Michael Raizman, MD, Chief Medical Officer of Palatin. "PL9643, with its differentiating factors, could provide a more tolerable and effective treatment option for patients suffering from dry eye disease." About Dry Eye Disease Dry eye disease is a common inflammatory disease that, left untreated, can become extremely painful and lead to permanent damage to the cornea and vision. Dry eye disease affects the cornea and conjunctiva of the eye resulting in irritation, redness, pain, and blurred vision. It is estimated to affect over 20 million people in the United States. The disease is characterized by insufficient moisture and lubrication in the anterior surface of the eye, leading to dryness, inflammation, pain, discomfort, irritation, diminished quality of life, and in severe cases, permanent vision impairment. Existing therapy for dry eye disease is generally regarded as inadequate by many physicians and patients, and often requires week or months to demonstrate activity. About Melanocortin Receptor Agonists and Inflammation The melanocortin receptor system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors, MC1r through MC5r. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects. Many tissues and immune cells located in the eye express melanocortin receptors, empowering our opportunity to directly activate natural pathways to resolve disease inflammation. About Palatin Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential.

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INDUSTRIAL IMPACT

Antheia and Ginkgo Bioworks Announce Partnership to Accelerate Production of Essential Medicines using Synthetic Biology

Antheia | August 13, 2021

Antheia, a synthetic biology company enabling next-generation plant-inspired medicines, and Ginkgo Bioworks, which is building the leading horizontal platform for cell programming, today announced a partnership to accelerate the development and production of essential medicines. Ginkgo, which recently announced a business combination with Soaring Eagle Acquisition Corp. (NASDAQ: SRNG), serves customers across industries seeking to develop new and better products. Antheia plans to leverage Ginkgo's high throughput enzyme design and screening capabilities to broaden its pipeline of critical active pharmaceutical ingredients (APIs) and key starting materials (KSMs). Nearly half of all medicines today are sourced from nature, and many of the most widely used essential medicines are sourced directly from medicinal plants. The World Health Organization classifies "essential medicines" as medicines "that satisfy the priority health care needs of the population." The supply chains for most critical plant-based medicines are fragile, and depend on a years-long process of growing, harvesting, transporting, and processing specialty plants. During times of extreme demand or constrained supply, many plant-based medicines, including widely used analgesics and sedatives, can be in shortage, as was recently the case during the COVID-19 pandemic. "Antheia is committed to using synthetic biology to enable more equitable access to essential medicines," said Kristy Hawkins, CSO and co-founder at Antheia. "By partnering with Ginkgo Bioworks, a global leader in organism engineering, we are greatly increasing our ability to develop essential medicines at the speed and scale necessary to drive change in global pharma supply chains." Synthetic biology platforms, such as those created by Antheia and Ginkgo Bioworks, make it possible for critical medicines to be produced on-demand in a much more efficient and environmentally friendly process compared to today's fragmented production systems. Additionally, when it comes to plant-based pharmaceuticals, biomanufacturing has significant advantages in supply chain resiliency and agility, cost, quality control, sustainability and efficiency compared to the conventional production methods, which are based on crop farming. "We're proud to partner with Antheia, a leader in the application of synthetic biology, as they leverage our platform to produce essential medicines at scale," said Tom Knight, co-founder at Ginkgo Bioworks. "Antheia and Ginkgo are both committed to using biology to build a better future, and we look forward to a long lasting partnership that will drive substantial impact." Antheia is focused on plant-inspired pharmaceuticals that are too complex to be produced through scalable synthetic chemistry processes. Antheia has managed to efficiently produce these highly-complex pharmaceuticals by pioneering whole-cell engineering, a technique that reconstructs multi-step biosynthetic pathways of unprecedented complexity in yeast cells. As Antheia brings its engineered microbes to commercial scale, it continually invests in strain optimization to ensure highly efficient production of the pharmaceutical compound of interest. Through this partnership, Antheia plans to leverage Ginkgo's extensive and rapid cell programming platform and biological codebase to greatly expand and accelerate its strain and enzyme engineering work. "Antheia is at the cutting edge of synthetic biology innovation, and its whole-cell engineering platform is capable of producing entire classes of medicines that were previously inaccessible," said Barry Canton, co-founder and Chief Technology Officer at Ginkgo Bioworks. "We are thrilled that Ginkgo's platform can support innovators like Antheia as they create next generation manufacturing technologies for essential medicines." About Antheia Antheia is unlocking the medicinal power of nature with synthetic biology. Through a novel whole-cell engineering approach to reconstruct complex molecules in yeast, Antheia's platform enables the discovery and manufacturing of plant-inspired drugs of unprecedented complexity and diversity. Antheia's team of scientists and technologists is headquartered in Menlo Park, California.

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MEDICAL

Absci Opens Door to New Biotechnology Campus

Absci | October 23, 2021

Absci Corporation the drug and target discovery company harnessing deep learning AI and synthetic biology to expand the therapeutic potential of proteins, today announced the opening of its new campus located in Vancouver, Washington, a few miles east of the downtown location that housed the company for the last four years. “Absci has been an incredibly positive force in the Vancouver community since establishing its headquarters here in 2016 I have followed Absci’s progress as it expanded within and has now outgrown that initial Vancouver facility. Absci is not only attracting investment and top talent to Washington, it is continuously innovating, creating exciting new possibilities for the future of medicine. I look forward to cutting the ribbon on Absci’s new campus early next month, and to continuing to support the development of our biotechnology ecosystem here in Washington. Washington Governor Jay Inslee The new Absci facility was concepted and constructed in a close collaboration between the company, Portland design firm Bora Architecture & Interiors, and local contractor Bremik Construction. Covering over 85,000 square feet of laboratory, office, and meeting spaces, it features regionally sourced, renewable, and natural building materials, custom finishes, and a vibrant aesthetic. The scalable laboratory space was designed to maximize flow, increase capacity, and readily reconfigure as needed. In addition to tripling capacity for program execution, the incorporation of in-house tissue culture capabilities, high throughput analytical instrumentation, advanced liquid handling and automation, and a dedicated clean air fermentation facility, in conjunction with a state-of-the-art offsite computational cluster, facilitate the expanded scope of Absci’s research and development activities. Additionally, integrated technologies for high volume data capture across Absci’s workflow generate the input for ongoing training of Absci’s Denovium EngineTM deep learning AI models for drug discovery and cell line design. “It’s inspiring to come to work each day in this exceptional building,” said Sean McClain, founder and CEO of Absci. “We designed the light-filled space around collaboration and the pursuit of scientific excellence, and we are thrilled with the pride our Unlimiters feel inhabiting this new campus. We strive every day to bring new insights to challenging problems, and this collaborative setting really encourages all of us to step beyond the impossible to create new possibilities for patients.” “When I visited Absci’s campus, I was impressed by the quality and productivity that imbues the space,” commented Bharat Dixit of ClearB Therapeutics. “The facility is designed with a modern look, yet with practical aspects in mind -- the futuristic gowning rooms, the bright, open lab spaces with lots of natural light and intuitive flow, as well as state-of-the-art robotic equipment, to name a few. And the friendly town hall full of people sharing ideas seems to be a sign of big success ahead.” About Absci Absci is the drug and target discovery company harnessing deep learning AI and synthetic biology to expand the therapeutic potential of proteins. We built our Integrated Drug Creation™ Platform to identify novel drug targets, discover optimal biotherapeutic candidates, and generate the cell lines to manufacture them in a single efficient process. Biotech and pharma innovators partner with us to create the next generation of protein-based drugs, including Bionic Proteins™ containing nonstandard amino acids, and other novel drug designs that may be impossible to make with other technologies. Our goal is to enable the development of better medicines by Translating Ideas into Drugs™. For more information visit www.absci.com. Availability of Other Information about Absci Investors and others should note that we routinely communicate with investors and the public using our website (www.absci.com) and our investor relations website (investors.absci.com), including without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information that we post on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Absci are encouraged to review this information on a regular basis. The contents of our website, or any other website that may be accessed from our website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Absci Forward-Looking Statements Certain statements in this press release that are not historical facts are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements containing the words “will,” “may,” "pursues," “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects,” and “intends,” or similar expressions. We intend these forward-looking statements, including statements regarding drug development efforts, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and we make this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies, and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control, including, without limitation, risks and uncertainties relating to drug development efforts; along with those risks set forth in our Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission on September 7, 2021, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events, or otherwise.

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