Ultragenyx Pharmaceutical Inc Announces U.S. FDA Approval of Dojolvi™ For The Treatment of Long-Chain Fatty Acid Oxidation Disorders

Ultragenyx | July 03, 2020

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.

“With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We are grateful to the patients, caregivers, families, dietitians and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it.”

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MediPharm Labs Enters into $6.2M Purchase Agreement for the Sale of Australian Facility

MediPharm Labs Corp. | July 12, 2022

MediPharm Labs Corp. a pharmaceutical company specialized in precision-based cannabinoids, has entered into a Share Purchase Agreement with OneLife Botanicals PTY for the sale of MediPharm Labs Australia Pty Ltd a wholly-owned subsidiary of the Company, for a minimum value of $6.9M AUD. The Agreement is subject to routine conditions and is anticipated to close within 90 days. The Agreement includes the assets of MPLA, specialized licensing, operational knowledge, and Australian and New Zealand customers currently served from that facility. All international contracts outside of Australia and New Zealand will remain with MediPharm Labs and be serviced from our Barrie GMP facility. In July 2021, MediPharm Labs was the first purpose-built cannabis facility to receive a pharmaceutical Drug Establishment License. Awarded by Health Canada, this license is recognized in over 50 countries as part of the Pharmaceutical Inspection Co-operation Scheme. At this time, the Company has completed a review of its international manufacturing platform and identified significant potential savings in moving all domestic and international manufacturing to the Canadian facility. After an extensive sale process, the Agreement with OneLife Botanicals was identified as a strategic fit for both parties. OneLife Botanicals is an emerging leader in Australia region for cannabis-based wellness and medical products, with plans to launch OTC CBD products across Australia. Purchasing the MPLA facility expedites OneLife Botanicals' timing to commercialization. The Company and OneLife Botanicals will also enter into a transition services agreement to allow for the two companies to smoothly transition products and services produced in the facility, and to work together on future commercial opportunities. David Pidduck, CEO of MediPharm Labs commented, "This sale represents a major milestone for MediPharm Labs as it continues to focus on rightsizing the business to achieve profitability. As we look to maintain our strong cash balance, this transaction paired with corporate restructuring completed in June 2022 should allow us to reduce our quarterly burn rate as we improve sales. I want to personally thank our Australian team. This group of professionals are trailblazers in the Australian and international medical cannabis market, achieving major milestones such as multiple customer product launches and our first deliveries to Germany. OneLife Botanicals is gaining one of the most capable medical cannabis manufacturing teams in Australia." "This purchase aligns and expedites the strategic aim of our organization to produce high quality medicinal cannabis products by integrating manufacturing into the end-to-end supply chain. The integration of a fully operational facility brings forward our objectives to take our products and brand to the market. OneLife Botanicals CEO Andrew Grant Our company undertook extensive research in considering this important acquisition and is highly confident about the capabilities and capacity of the operation to manufacture products in accordance with all compliance and quality standards. The integration of this business with our existing cultivation facility facilitates savings in capital investment, access to operational efficiencies and establishes us as a significant supplier in the Australian medicinal cannabis industry. We intend to build on the existing customer base and develop additional product distribution opportunities to become a formidable participant in the market." About MediPharm Labs Founded in 2015, MediPharm Labs specializes in the development and manufacture of purified, pharmaceutical-quality cannabis concentrates, active pharmaceutical ingredients (API) and advanced derivative products utilizing a Good Manufacturing Practices certified facility with ISO standard-built clean rooms. MediPharm Labs has invested in an expert, research driven team, state-of-the-art technology, downstream purification methodologies and purpose-built facilities with five primary extraction lines for delivery of pure, trusted and precision-dosed cannabis products for its customers. Through its wholesale and white label platforms, MediPharm Labs formulates, develops processes, packages and distributes cannabis extracts and advanced cannabinoid-based products to domestic and international markets. In 2021, MediPharm Labs received a Pharmaceutical Drug Establishment Licence from Health Canada, becoming the only company in North America to hold a domestic Good Manufacturing Licence for the extraction of natural cannabinoids. The Company carries out its operations in compliance with all applicable laws in the countries in which it operates. About OneLife Botanicals Onelife Botanicals is a niche producer of medicinal cannabis in Melbourne, Australia. The company has a passion for developing and producing products that provide a range of health benefits and assist in treating a range of specific health conditions. The company operates an existing cultivation site with extensive Infrastructure, including fit for purpose glasshouses for growing Medicinal Cannabis in a controlled environment. The glasshouses facilitate the optimum growing environment through temperature and climate control, allowing natural sunlight to be maximized in a protected and secure environment. The Directors and stakeholders are focused on identifying and exploring market opportunities, along with embracing innovation in the production processes. The success of the business is able to be achieved with an extensive Research and Development program, aimed at staying ahead of changes in growing methods, technological solutions and product applications, along with advanced automation ensuring that we minimize operating expenditure and maximise quality control.

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BUSINESS INSIGHTS

Tris Pharma Announces Development of a Very Low Sodium and Once-Nightly Oxybate Formulation

Tris Pharma, Inc. | July 28, 2022

Tris Pharma, Inc. a fully integrated pharmaceutical company with a robust portfolio of CNS products announced that it has successfully developed an oxybate formulation for the treatment of cataplexy or excessive daytime sleepiness n patients 7 years of age and older with narcolepsy that has the potential to be significantly advantageous to existing approved therapies, such as Jazz Pharmaceuticals' blockbuster XYWAV® and XYREM® franchise. The Tris oxybate formulation is believed to provide an advantage over approved therapies by utilizing Tris's proprietary LiquiXR technology platform and our newly developed RaftWorks design to provide a unique oxybate formulation offering both once-nightly dosing, and very low sodium content. The Tris oxybate formulation is investigational and not yet approved by any regulatory agency. Both XYWAV® and XYREM® require twice a night dosing – once at bedtime and again 2.5 to 4 hours later for the second dose. Further, XYREM contains an express warning based on its high sodium content and associated risk to patients with heart failure, hypertension or impaired renal function. The Tris oxybate formulation seeks to improve on existing therapies by providing convenient once per night dosing, and significantly reduced sodium content such that it will contain less than 100 mg of sodium per 9.0 gm of maximum sodium oxybate equivalent dose. Based on the significant potential advantage the Tris oxybate formulation could provide over existing available therapies, Tris is pursuing an Orphan Drug Designation for its unique oxybate formulation. In a human clinical study, Tris established about 100% bioavailability for one dose of its formulation against two doses of XYREM at 4.5 gm each, equivalent to the 9.0 gm maximum dose of sodium oxybate. If approved, the Tris product will provide significant advantages in that patients will not have to wake up in the middle of the night to take a second dose and will have all the benefits of a very low sodium product. The Tris oxybate product achieved its clinical objectives by combining two distinct proprietary Tris technologies: the LiquiXR platform, and the newly developed RaftWorks design. Working together, these unique formulation platforms provide an interpenetrating network raft in-situ, in which sodium-free drug particles are entrapped in a raft that allows for extended release of the active ingredient. Many drugs, such as oxybate, are poorly absorbed because they have a narrow window of absorption within the human gastro-intestinal tract. The RaftWorks approach permits a slow and extended release of oxybate from a floating raft in-situ, thereby facilitating absorption of the active ingredient within the targeted area in the gastro-intestinal tract. Further, the raft disintegrates at a predetermined time due to the trigger mechanism that is built into the formulation. "This unique formulation design by Tris scientists potentially achieves drug delivery capabilities that have never previously been achieved within our industry. We are looking to build on this approach and develop extended-release formulations for other drugs that are limited by their narrow window of absorption." Ketan Mehta, Founder and Chief Executive Officer of Tris About Narcolepsy Narcolepsy, a chronic and disabling neurological disorder that affects the stability of sleep and wakefulness, is characterized primarily by excessive daytime sleepiness, with or without episodic loss of muscle tone usually triggered by strong emotions during wakefulness (cataplexy) 1-4. Additional symptoms can vary by person and may include disrupted nighttime sleep, and REM-sleep phenomena (sleep paralysis, hallucinations). The exact number of individuals with narcolepsy in the United States is unknown. Several studies have estimated the prevalence of narcolepsy in the United States to be approximately 1 (range: 0.6 to 1.6) in 2,000 people 2,4-6. There is currently no cure for narcolepsy; treatment of symptoms includes medications and lifestyle modifications 1. About Tris Pharma Tris is a fully integrated, innovation-driven CNS company that provides a differentiated approach to target unmet medical needs, including the application of novel technologies designed to enhance patient benefits across therapeutic categories. Tris's CNS portfolio includes treatments for pain; addiction; spasticity in multiple sclerosis, cerebral palsy; narcolepsy; and ADHD.

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PHARMA TECH

Porton Advanced and Royaltech Announce Strategic Collaboration to Accelerate the Development of Bacterial Drugs and mRNA Drugs

Porton Advanced Solutions | August 29, 2022

Porton Advanced Solutions.announced a strategic cooperation with Suzhou Royaltech Med Co., Ltd. This strategic collaboration will further integrate the resources and capabilities of both parties, jointly promote the establishment of microbial vectors used for gene therapy and mRNA drug platform technology, and accelerate the R&D process of bioinnovative drugs. Porton Advanced provides an end-to-end gene and cell therapy CDMO service platform covering plasmids, cell therapy, gene therapy, oncolytic virus, nucleic acid therapy and viable bacteria therapy. Suzhou Royaltech focuses on the development of vaccines for tumor immunotherapy with its proprietary technologies, and fast transition from clinical to market . Its R&D pipeline covers the attenuated non-integrated Listeria platform, mRNA platform, and cell therapy platform. All these platforms are in the leading position not only in China but also abroad, and have a complete layout of invention patents. "We are very pleased to enter into this strategic collaboration with Royaltech. The core management team of Royaltech is from the top pharmaceutical companies, with an average of 15 years of R&D and management experience and is advancing an internationally competitive R&D pipeline. Porton Advanced's end-to-end gene and cell therapy CDMO platform with rich project experience and comprehensive quality system will help Royaltech the development of Listeria and mRNA drugs, push its R&D pipeline in a quick, efficient manner, and accelerate the development and implementation of innovative drugs, so that good medicines can benefit the public earlier." Dr. Yangzhou Wang, CEO of Porton Advanced Dr. Chun Xu, Chairman of Royaltech, said: "As a Biotech company, we are grateful to work with Porton Advanced, a very professional CDMO company, to jointly build a unique Listeria and mRNA tumor vaccine platform technology, and constantly develop leading tumor immunotherapeutic biological drugs in China and abroad." About Porton Advanced Solutions Established in Suzhou Industrial Park in December 2018, by its parent company Porton Pharma Solutions Ltd. Porton Advanced has built a CDMO platform integrating plasmid, cell therapy, gene therapy, oncolytic virus, nucleic acid therapy and microbial vectors used for gene therapy providing end-to-end services from cell banking, process development and analytical development, cGMP production to final Fill and Finish , investigator-initiated clinical trials (IIT), investigational new drugs (IND), clinical trials to commercial production. Porton Advanced is dedicated to support sponsors advance their GCT drug development and market launches. Porton Advanced focuses solely on gene and cell therapy services. Built on the professional experience of its cohort of world-class professionals, as well as on the successes of its parent company, Porton Advanced insists on "Customer First" and the tenet of "Compliance, Expertise, Focus, Open Collaboration". With its key focus on protecting IP for its sponsors, through its comprehensive project management and quality systems, Porton Advanced strives to bring gene and cell therapy products to the clinic and the market through its quality CDMO services, and help bring the best medicine to the public sooner. About Royaltech Headquartered in Suzhou Industrial Park, Suzhou Royaltech Med Co., Ltd. was founded in July 2016. In July 2017, a wholly-owned subsidiary, Shanghai Royaltech Co., Ltd. was set up in Shanghai International Medical Zone. As a high-standard innovation center of biological drugs, Shanghai Royaltech focuses on CMC development of innovative tumor vaccine, and fast clinical application of up-to-date cell immunotherapy. Since establishment, Royaltech has submitted several patents for invention to the State Intellectual Property Office. It focuses on the development of innovative tumor vaccine, and fast clinical application of up-to-date cell immunotherapy. Its R&D pipeline covers mRNA, oncolytic virus, bacteria, AAV, cell therapy platform and so on. Its core management team is from top universities in US and top 10 big pharma, with an average of 15 years of R&D and management experience. Our scientists have excellent capability on molecular biological engineering, target discovery and verification, pre-clinical R&D, quality control and assurance, and clinical trials of cell immunotherapy. On the strength of our rich experience on target biomarker, target discovery and verification, and global multicenter clinical trials, we are advancing an internationally competitive R&D pipeline.

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