Lipodystrophy

Despite the sudden supply hurdle, Theratechnologies is confident that it can “avoid any impact on patients in 2025,” the company’s CEO, Paul Lévesque, said in a statement. Theratechnologies' reliance on a small pool of contract manufacturers has come back to bite the Quebecois biopharma, which announced Tuesday that supplies of its HIV med Egrifta SV could soon run short.Egrifta SV—approved to reduce excess abdominal fat in HIV patients with lipodystrophy—is at risk of facing a “temporary supply disruption” in early 2025, Theratechnologies warned in a release.The reason behind the supply squeeze? An “unexpected voluntary shutdown” of one of Theratechnologies’ contract manufacturer’s facilities following a disappointing FDA inspection, the company said.Despite the sudden supply hurdle, Theratechnologies is confident that it can “avoid any impact on patients in 2025,” the company’s CEO, Paul Lévesque, said in a statement. Theratechnologies will work closely with its manufacturer and the FDA to resume production of its HIV drug, the CEO added.In the meantime, Theratechnologies is rolling out measures to manage inventory of Egrifta SV to help meet patient demand until early January. The company estimates that its efforts will siphon some $1.6 million from the company’s expected revenue in the 2024 fiscal year. Taking a closer look at the situation, Theratechnologies’ Egrifta SV manufacturer recently undertook a three-month voluntary shutdown of its facility to address observations made during a prior FDA inspection. The issues at hand relate to the manufacturing environment at the contractor’s plant, rather than production of Egrifta SV itself, Theratechnologies stressed.The manufacturer is finalizing remediation measures and informed Theratechnologies that it plans to restart production by mid-October, according to Theratechnologies' release. Under the current plan, a batch of the drug is set to be manufactured on Oct. 21, Theratechnologies added.The company did not name the contract manufacturer associated with the supply disruption.As it stands, Theratechnologies depends exclusively on Bachem and Jubilant for production and supply of all raw materials, drug substance and drug product related to Egrifta SV, the company stated in its most recent annual report.In the report, Theratechnologies noted that there are a “limited number” of qualified manufacturers with the necessary know-how and capacity to manufacture the company’s drug substance and drug product. Replacing third-party manufacturers is a pricey and time-consuming endeavor, the company added, noting the process could take 2 or 3 years. Theratechnologies’ supply hiccup is the second caused by third-party manufacturing problems in less than a week.On Friday, Coherus revealed that its Neulasta biosimilar Udenyca would suffer a temporary supply disruption, with stocks of the drug likely to be “substantially depleted" by the middle of October.Coherus blamed the issue on “over-commitments and capacity constraints” facing the U.S.-based contract manufacturer in charge of the cancer biosimilar’s final packaging. 

Another pharma company is facing supply constraints because of third-party contract manufacturing issues. On Tuesday, Theratechnologies warned there is a risk its lipodystrophy drug for HIV, Egrifta SV, will be in short supply early next year due to an “unexpected” site shutdown at its third-party manufacturer. Earlier this week, Endpoints News reported that Coherus is also warning of supply constraints for its cancer drug due to issues at its contract manufacturer. Following an FDA inspection, Theratechnologies’ unnamed contract manufacturer voluntarily shut down its facility for three months after the agency found issues at the facility. No issues were found with the direct manufacturing of Egrifta SV, Theratechnologies said. The contractor expects to begin production activities again by mid-October, with a batch of Egrifta SV scheduled to be made on Oct. 21. In the meantime, Theratechnologies is managing the inventory of the HIV drug to supply patients for the rest of the year and is “confident” no patients will be impacted in 2025, CEO Paul Lévesque said in a statement announcing the problem . Theratechnologies said the FDA is also reviewing the timeline to start distributing Egrifta SV again. The agency requested the pharma company to submit a Prior Approval Supplement, detailing the changes the third-party manufacturer will make at the site. Theratechnologies expects to lose $1.6 million in revenue in 2024 because of the shortfall. It pulled in $53.7 million in Egrifta SV sales last year. Theratechnologies faced other regulatory hurdles with the FDA earlier this year, with another more concentrated formulation of Egrifta SV being rejected over manufacturing issues .

EDMONTON, Alberta--( BUSINESS WIRE )-- Entos ® Pharmaceuticals (Entos), a clinical-stage genetic medicines company committed to using its Fusogenix™ PLV™ technology to develop cures and improve the lives of patients and their families, today announced the publication of a research article in Cell. The article, titled “ Safe and Effective Delivery of DNA and RNA Using Proteolipid Vehicles ,” details the development of a novel delivery platform for nucleic acid medicines that overcomes the limitations of current delivery systems. Incorporating fusion associated small transmembrane (FAST) proteins into a proteo-lipid vehicle (PLV), or FAST-PLV, this new platform can effectively deliver mRNA or plasmid DNA into a wide variety of cells and tissues with low immunogenicity and broad distribution throughout the body. Entos led research that involved collaborators from the University of Alberta , Dalhousie University , Oisín Biotechnologies and Aegis Life, Inc. , to develop the platform and conduct studies demonstrating its advantages over lipid nanoparticle (LNP) delivery systems. The research findings were published by Cell online this morning and will appear in the journal’s September 19 th print issue. “Currently AAV and LNPs are the main delivery platforms for genetic medicine. Both platforms have significantly advanced the field with approved therapies and vaccines for a variety of serious and often life-threatening diseases,” said John Lewis, Ph.D., senior author of the study and Bird Dogs Chair of Translational Oncology at the University of Alberta and CEO of Entos Pharmaceuticals. “Despite these advancements, gaps remain in the ability of current technologies to fully realize the potential of genetic medicines. We designed the FAST-PLV platform to bridge these gaps and deliver on the promise of genetic medicines that patients urgently need.” The FAST-PLV platform is a fusion-powered delivery system for genetic medicines that combines aspects of viral and non-viral approaches to leverage a completely new mechanism for intracellular delivery of RNA and DNA. While AAV and LNP rely on endocytosis, the FAST protein enables direct fusion of the PLV to the plasma membrane of target cells. This substantially improves the potency and tolerability of genetic medicines, while enabling the development of formulations that are inherently non-liver tropic, enabling the delivery of therapeutic doses of nucleic acid therapies to cells and tissues throughout the body. In this work, the platform was evaluated for safety, efficacy, and biodistribution in various animal models, as well as demonstrating that the FAST-PLV platform is non-immunogenic, which allows for repeat dosing of genetic medicines. “I am incredibly proud of the strides we've made in genetic medicine with our FAST-PLV platform. This breakthrough not only underscores the potential of our technology to overcome current limitations in gene delivery but also reinforces our commitment to developing safer, more effective, and re-dosable therapies for patients. The publication of our work in Cell is a significant milestone, and we believe it marks the beginning of a new era in the field of genetic medicine,” said Arun Raturi PhD., senior author and CSO of Entos Pharmaceuticals. FAST-PLVs form the basis of Entos’ Fusogenix™ PLV™ delivery platform, which is being utilized by Entos Pharmaceuticals to develop novel therapies for congenital generalized lipodystrophy, obesity, Stargardt disease and cystic fibrosis in addition to its clinical VAX-002 vaccine program. Entos and its valued partners are developing applications in the central and peripheral nervous systems, oncology, longevity and more. About Entos Pharmaceuticals Inc. A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and re-dosable nucleic acid delivery technologies. Since its inception in 2016, Entos® has been dedicated to advancing next-generation genetic medicines using our proprietary Fusogenix TM PLV TM drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California and London, United Kingdom, respectively. For more information, visit www.entospharma.com , or follow Entos on LinkedIn . Forward Looking Statements This press release contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements contained in this press release include statements regarding Entos and its belief as to the mode of action and potential and benefits of the Fusogenix proteolipid vehicle platform and the mode of action and potential and benefits of Covigenix VAX-002 for the treatment of COVID-19; and other statements related to anticipated developments in the Company’s business and technologies. In any forward-looking statement in which Entos expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation, or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause the Company’s actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of Fusogenix as a nucleic acid delivery vehicle, the efficacy of Covigenix VAX-002, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results, and financial condition. Investors should consult with the U.S. Securities Commission for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements except as required by applicable laws. Entos ® word mark and design logo, Fusogenix TM and PLV TM are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.