Latest news with #AnimalRule
Business Wire
7 hours ago
- Business
- Business Wire
Tivic Receives Two Investigational New Drug Applications for Entolimod™ for the Treatment of Acute Radiation Syndrome (ARS) and Advanced Cancers
FREMONT, Calif.--(BUSINESS WIRE)-- Tivic Health® Systems, Inc. (Nasdaq: TIVC), a diversified immunotherapeutics company, today announced it has received, from Statera Biopharma, two investigational new drug applications (INDs) for its lead candidate, Entolimod™. The two INDs cover the use of Entolimod to treat acute radiation syndrome, including both hematopoietic (ARS-H) and gastrointestinal (ARS-GI) sub-syndromes, and the use of Entolimod to treat advanced cancers, including as an anti-tumor agent and for conditions resulting from cancer treatments. 'Transfer of these INDs to Tivic allows us to formally engage the FDA regarding Entolimod's regulatory pathway and enables clinical trials in neutropenia, lymphocyte exhaustion, and other cancer-related conditions,' stated Tivic CEO, Jennifer Ernst. 'While the IND for advanced cancers allows exploration of Entolimod's anti-tumor activity, building on prior developments, we remain focused in the near-term on Entolimod's first indication--acute radiation syndrome.' About Entolimod Entolimod is a novel TLR5 agonist that triggers NF-kB signaling, activating antiapoptotic and cell protective mechanisms. Under the FDA's Animal Rule, Entolimod has been the subject of extensive trials for the treatment of acute radiation syndrome (ARS), and has demonstrated robust survival, enhanced GI tract recovery and improved hematopoiesis in animal models. Entolimod has been granted Fast Track Designation for ARS. Tivic also holds the exclusive rights for Entolimod for the treatment of neutropenia and has the option to license additional indications, including lymphocyte exhaustion, immunosenescence, and chronic radiation syndrome. About Tivic Tivic's dual platform strategy utilizes the body's biopharmaceutical and bioelectronic systems to treat unmet medical needs through targeting the immune system. Tivic's biologics compounds activate an innate immune pathway to prevent cell death in the bone marrow and epithelial tissues across systems impacted by radiation and age. The company's lead drug candidate, Entolimod™ for acute radiation syndrome, is a novel TLR5 agonist that has been granted Fast Track designation and is in late-stage development. Tivic's bioelectronic program is developing a novel, non-invasive medical device designed to target the neural pathways implicated in many prevalent and debilitating diseases. Early trials show promising signals that Tivic's approach may regulate specific biologic responses, and the company believes its early-stage vagus nerve stimulation device has the potential to deliver clinical outcomes similar to or better than those of surgically implanted devices. To learn more about Tivic, visit: Forward-Looking Statements This press release may contain 'forward-looking statements' that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as 'anticipate,' 'believe,' 'contemplate,' 'could,' 'estimate,' 'expect,' 'intend,' 'seek,' 'may,' 'might,' 'plan,' 'potential,' 'predict,' 'project,' 'target,' 'aim,' 'should,' 'will' 'would,' or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on Tivic Health Systems Inc.'s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate, including as a result of interactions with and guidance from the FDA and other regulatory authorities; changes to the company's relationship with the its partners; timing and success of clinical trials and study results; the failure to obtain FDA or similar clearances or approvals and noncompliance with FDA or similar regulations; the company's future development of its ncVNS treatment, Entolimod and Entolasta; changes to the company's business strategy; regulatory requirements and pathways for approval; the company's need for, and ability to secure when needed, additional working capital; the company's ability to maintain its Nasdaq listing; and changes in tariffs, inflation, legal, regulatory, political and economic risks. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors. Accordingly, you are cautioned not to place undue reliance on such forward-looking statements. For a discussion of risks and uncertainties relevant to the company, and other important factors, see Tivic Health's filings with the SEC, including, its Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 21, 2025, under the heading 'Risk Factors,' as well as the company's subsequent filings with the SEC. Forward-looking statements contained in this press release are made as of this date, and Tivic Health Systems, Inc. undertakes no duty to update such information except as required by applicable law.
News.com.au
29-07-2025
- Health
- News.com.au
Biocurious: It's a case of animal magnetism as Island eyes the FDA's new rule on non-human testing
Having acquired a second anti-viral program, Island Pharmaceuticals hopes to avail of an FDA approval shortcut for one of the world's nastiest diseases Island was so impressed with Galidesivir that it fast-tracked the acquisition process The company hopes both Galidesivir and its legacy program ISLA-101 will become eligible for potentially lucrative FDA priority review vouchers The term 'Animal Rule' is redolent of George Orwell's seven rules of Animal Farm. Who could forget the perverse 'all animals are equal'? Or how about the 'kill the pig' chant in Lord of the Flies as the stranded lads descend into animalistic savagery? In drug development, Animal Rule now means something less foreboding – but just as far reaching. Three years ago, the US Food & Drug Administration (FDA) devised the Animal Rule to enable approvals without the rigmarole of a human trial. Conventional wisdom goes that drug makers must test out their candidates on human flesh and blood. Trialling drugs on our nearest relative – primates – can only go so far. But what if the disease is so awful and fatal that human testing would not be ethical or feasible? In these cases, the FDA might approve treatments based on 'adequate and well-controlled' animal efficacy studies. These remedies must prevent 'serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic chemical, biological, radiological, or nuclear' agents. Tackling some of the world's worst viruses The Animal Rule concession is likely to be crucial for Island Pharmaceuticals (ASX:ILA), which this month acquired the rights to an antiviral compound, Galidesivir. The vendor was the Nasdaq-listed Biocryst Pharma. A broad spectrum anti-viral, Galidesivir is claimed to be effective against more than 20 viruses, including the hideous hemorrhagic Ebola and Marburg. Biocryst tested healthy volunteers in phase 1 trials for some of the 'lesser' diseases, establishing safety. Subsequent primate and hamster testing for yellow fever, Zika, Marburg and Ebola produced what Island CEO Dr David Foster dubs 'phenomenal results'. These include 100% survival, whereas the control animals were dead within days. In particular, the Marburg primate results could provide the foundation for availing of the Animal Rule. Foster says the program supplements Island's existing program ISLA-101, to treat and prevent the mosquito-borne dengue fever. The deets of the deal The deal sees Island acquiring Galidesivir from the Nasdaq-listed Biocryst Pharma for US$550,000. The initial agreement was by way of a US$50,000 option fee. 'After considerable diligence we became very excited about the asset and elected not to (avail of option) and go straight to an acquisition,' Foster says. Island also pays $US500,000 to Biocryst on phase II trial completion and a further US$500,000 on FDA approval. Thus far, Biocryst has completed one acceptable animal study. 'We may be one successful animal study away from submitting a New Drug Application ,' Foster says. 'This can happen very quickly and we are laying the groundwork for primate study. We hope to have it completed 12 months from closing the acquisition.' The obvious question is: if Galidesivir is so promising, why was Biocryst happy to dispose of it for not much more than a song? Foster notes the program was funded almost entirely by US grants, which eventually dried up (see below). Biocryst also shifted strategy in favour of becoming a rare diseases company. With cash of $7.25 million as of June 30, Island is well funded for a second simian study. America's quiet war on (bio) terror US authorities have been keenly interested in Galidesivir's development, because of the potential for Ebola and Marburg to be 'weaponised'. Uncle Sam has poured at least US$70 million into developing the compound, as a counter bioterrorism strategy against such horrible hemorrhagic viruses. Via the disaster preparation agency BARDA, the US has a long history of dealing in germ warfare – and it's not monkeying around. BARDA, for instance, has spent hundreds of millions of dollars on anthrax and smallpox vaccines. Foster notes government contracts – such as those offered by BARDA – don't entail hiring an army of salespeople. 'It's a great commercial opportunity,' he says. 'We could select any number of viruses. Zika stands out, but we're laser focused on Marburg.' Let's not put the moz on ISLA-101 Foster adds that Island has not lost is passion for its original program ISLA-101, targeting the mosquito-borne dengue fever. Usually not fatal but most unpleasant, dengue fever is spreading, to the extent that half of the world's populace is vulnerable. Currently about 400 million people are infected annually. Island has carried out a randomised, placebo-controlled, double-blinded phase 2a/b study, dubbed Protect. The trial covers both treatment and prophylactic cohorts. In the June quarter, Island released top-line results. These showed a 'considerable reduction' in viral load in the preventative cohort. The study also showed a 'clear drug signal' in the treatment group. The latter received a weakened form of the virus (or a placebo). As a repurposed drug with a long-established safety profile, ISLA-101 should benefit from a truncated approval pathway. Similarly, Galidesivir initially was intended to be used for hepatitis C. This means the drugs could be commercialised much faster than traditional therapies, which might take more than a decade (and billions of dollars) to get to market. Island saddles up for multiple PRVs The potential Animal Rule shortcut aside, Island could be in line for a coveted FDA Priority Review Voucher (PRV), for both the Galidesivir and ISLA-101 programs. Irreverent folk dub PRVs as 'Willy Wonka' vouchers, given their potential to create untold riches (without the choccie overload tummy aches). The FDA might bestow a PRV on the maker of an approved therapy, the idea being that a second drug is subject to fast-track assent. Most likely, the recipient sells the fungible paperwork to another party. As a result, PRVs have changed hands for US$100 million to US$150 million. In the case of blockbuster drugs, getting to market three months earlier can mean billions more in sales. In the case of Galidesivir, Biocryst would take a 25% stake in any PRV spoils. Island chairman Jason Carroll is unaware of any other ASX biotech with the ability to secure two PRVs. Come to think of it, so are we ... Off to the races Island is assembling a dossier to present to the FDA, in view of getting feedback on using the Animal Rule. To date, no Australian company has availed of the concession … but there's always a first. "What people are calling a pony is going to win the Melbourne Cup,' Carroll says. Island may be off to the races. But it still needs some luck on its side because infectious disease drug developers only get one-third of their therapies to market. That's a much better strike rate relative to the wider drug pantheon. 'We are in a fortunate position to have two phenomenal assets,' Foster says. 'We want to set up the next ISLA-101 trial, but Galidesivir will lead the way because it is such a short path to approval.' Ticking the boxes A recent Island board appointee, Carroll heads psychedelic drug developer Tryptamine Therapeutics (ASX:TYP). 'When you look at a pharma company value there are a few things to ask,' Carroll says. 'Does it work? Is it safe and effective and how do you protect its value? Can you make it? How many trials and how does pay for commercialisation?' Evidently, both Galidesivir and ISLA-101 pass muster on these counts. 'This is a much bigger opportunity than what most people think,' Carroll says. '… this is one of the most significant products any Australian biotech can get hold of.'
Yahoo
27-05-2025
- Business
- Yahoo
Traws Pharma Announces Receipt of FDA Guidance on Tivoxavir Marboxil and Reports Plans for Pursuing Stockpiling for Pandemic Preparedness
NEWTOWN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (NASDAQ: TRAW) ('Traws Pharma', 'Traws' or 'the Company'), a clinical-stage biopharmaceutical company developing novel therapies to target critical threats to human health from respiratory viral diseases, today announced receipt of written responses to questions submitted for a Type B pre-Investigational New Drug Application meeting with the U.S. Food and Drug Administration (FDA, the Agency). The FDA provided feedback on development paths for potential approval of tivoxavir marboxil (TXM) for bird flu and seasonal flu, including on the use of the Animal Rule. The Animal Rule is intended to provide a path to approval in situations when human clinical studies would be unethical or impractical. 'The FDA has provided detailed feedback on the development paths for approval, including a combined clinical study enrolling seasonal flu and bird flu-infected subjects, as such infections occur. We are requesting a Type D meeting with the FDA to specify next steps and look forward to working closely with the Agency to develop TXM for bird flu and seasonal flu,' said C. David Pauza, PhD, Chief Science Officer for Traws Pharma. 'The consistent finding of therapeutic effects of TXM in three animal models of bird flu increases our confidence that this compound should be rapidly progressed for stockpiling and pandemic readiness by government agencies,' said Robert R. Redfield, MD, Chief Medical Officer for Traws Pharma and former Director of the U.S. Centers for Disease Control and Prevention (CDC). 'To that end, we have initiated a dialog with the Biomedical Advanced Research and Development Authority (BARDA), the responsible agency for stockpiling for pandemic preparedness in the United States, and we plan to engage with respective international regulatory agencies for the same purpose. With the known history that previous H5N1 virus outbreaks in human populations are associated with death rates of up to 50%1, we believe that these actions should be pursued with great urgency.' 'This initial interaction with the FDA has helped guide the development of TXM as a broad therapeutic against pandemic and seasonal influenza,' commented Iain Dukes, MA DPhil, Interim CEO of Traws Pharma. 'We are committed to progressing the process for stockpiling TXM to prevent any pandemic outbreaks associated with bird flu.' About Tivoxavir Marboxil Tivoxavir marboxil (TXM) is an investigational oral, small molecule CAP-dependent endonuclease inhibitor designed to be administered as a single-dose for the treatment of bird flu and seasonal influenza. It has potent in vitro activity against a range of influenza strains in preclinical studies, including a human isolate of the highly pathogenic avian flu H5N1 (bird flu). Consistent, positive preclinical data from three animal species indicate that a single dose of TXM demonstrated a therapeutic effect against H5N1 bird flu. Seasonal influenza represents an estimated multi-billion dollar antiviral market opportunity, largely driven by global health organizations, practice guidelines and government tenders2,3, with upside potential from potential pandemic flu outbreaks including H5N1 bird flu. We believe that these data support further development of tivoxavir marboxil as a treatment for bird flu and seasonal flu. Source information: Per link Per link TRAW data on file About Traws Pharma, Inc. Traws Pharma is a clinical stage biopharmaceutical company dedicated to developing novel therapies to target critical threats to human health in respiratory viral diseases. Traws integrates antiviral drug development, medical intelligence and regulatory strategy to meet real world challenges in the treatment of viral diseases. We are advancing novel investigational oral small molecule antiviral agents that have potent activity against difficult to treat or resistant virus strains that threaten human health: bird flu and seasonal influenza, and COVID-19/Long COVID. Tivoxavir marboxil is in development as a single dose treatment for bird flu and seasonal influenza, targeting the influenza cap-dependent endonuclease (CEN). Ratutrelvir is in development as a ritonavir-independent COVID treatment, targeting the Main protease (Mpro or 3CL protease). For more information, please visit and follow us on LinkedIn. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding the Company, its business and product candidates, including the potential opportunity, benefits and the regulatory plans for tivoxavir marboxil. The Company has attempted to identify forward-looking statements by terminology including 'believes', 'estimates', 'anticipates', 'expects', 'plans', 'intends', 'may', 'could', 'might', 'will', 'should', 'preliminary', 'encouraging', 'approximately' or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws' clinical trials, our ability to take advantage of expedited regulatory pathways for tivoxavir marboxil, our ability to obtain regulatory approval of tivoxavir marboxil, the expectations of our interactions with regulatory authorities, including both the FDA and BARDA and international regulatory agencies, market conditions, regulatory requirements, changes in government regulation, the extent of the spread and threat of the bird flu, and those discussed under the heading 'Risk Factors' in Traws' filings with the U.S. Securities and Exchange Commission (SEC). Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events, except to the extent required by law. Traws Pharma Contact:Nora BrennanTraws Pharma, Investor Contact:Bruce Mackle LifeSci Advisors, LLC646-889-1200bmackle@
