Wellcome Trust Clinical Research Facility

BV100 Phase 2 preliminary results suggest BV100 is generally safe and well tolerated and showed strong initial signs of efficacy in VABP patientsStrategic investment from GIBF and launch of BV100 clinical program in ChinaSecond lead asset, alpibectir Phase 2a trial completed in April showing human proof of concept in tuberculosis BASEL, Switzerland, Oct. 16, 2024 (GLOBE NEWSWIRE) -- BioVersys AG, a multi-asset, clinical stage biopharmaceutical company focusing on research and development of novel antibacterial products for serious life-threatening infections caused by multi-drug resistant ("MDR") bacteria, announced today the completion of the last patient's last visit in the Phase 2 clinical trial with its lead asset BV100, the company's novel formulation of rifabutin suitable for intravenous administration. The Phase 2 multicenter, open label, randomized, active controlled study evaluated the pharmacokinetics, efficacy and safety of BV100 in adult patients with ventilator associated bacterial pneumonia (VABP) suspected or confirmed to be due to Carbapenem Resistant Acinetobacter baumannii (CRAB). Preliminary analysis of the data shows that the primary endpoint was met. BV100 is generally safe and well tolerated, met the expected pharmacokinetic targets and showed strong signs of efficacy in VABP patients with confirmed CRAB infection. In Part A of the study, the 14- and 28-day all-cause mortality (ACM) for BV100 was 12.5% and 25% respectively compared to the best available therapy control arm with 40% and 60% ACM respectively in patients with carbapenem resistant Acinetobacter infections. Similar trends in the test of cure and microbiological endpoints were seen. Full top line results from the trial are expected in early 2025 once analysis of the data has been finalized. BV100 is based on the newly identified mode of action for the active uptake of rifabutin into Acinetobacter baumannii-calcoaceticus complex and is being developed for hospital infections caused by Acinetobacter baumannii, including CRAB strains. There is a serious lack of effective and safe treatment options for CRAB infections and mortality rates in hospitals can be as high as 50%. CRAB has been designated a priority pathogen by the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC). Due to the high unmet need and based on the current carbapenem resistance rates in Acinetobacter baumannii, we estimate in excess of 1 million hospital treated patients across the United States, Europe, the Middle East and China that could benefit from BV100. BioVersys is committed to advancing the clinical development of BV100 in a global Phase 3 registration trial, which it expects to initiate in the second half of 2025. To date, BV100 has been administered to over 230 subjects, at doses of up to 900 mg and over periods of up to 14 days in Phase 1 and 2 clinical trials and was considered generally safe and well tolerated. The strong clinical data package comprising the first in human studies, the Phase 2 in VABP patients, two drug-drug interaction studies, studies in renally and hepatic impaired patients and a bronchoalveolar study provides the foundation for progressing BV100 into the final clinical Phase. Dr. Glenn E. Dale, Chief Development Officer of BioVersys: "We are making great progress with our clinical evaluation of BV100, and we are very pleased to have met this latest milestone with the final visit of our last patient in the Phase 2 trial, with BV100 demonstrating strong signs of efficacy. We are now looking forward to finalizing the data ahead of our planned readout early next year, which we expect will build on the promising safety profile that BV100 demonstrated in Phase 1 and provide a further basis for our planned Phase 3 study." Prof. David Paterson: "BV100 could bring a much-needed solution for patients with an unmet need due to carbapenem resistant infections caused by Acinetobacter. Its safety profile and ease of use in an ICU setting, together with the strong signs of efficacy seen in the Phase 2 trial, make BV100 a compelling treatment option to be tested in a pivotal Phase 3 trial of VABP patients with confirmed CRAB infection.” In addition to the progress of BV100, the company's second clinical asset, alpibectir a first in class molecule for tuberculosis which is developed in partnership with GSK, completed a Phase 2a clinical trial earlier this year. Alpibectir delivered a proof of concept in patients and was also shown to be generally safe and well tolerated. Alpibectir will continue to be developed in close partnership with GSK. In May 2024, BioVersys announced the expansion of the strategic collaboration with GSK and the extension of the Series C round by CHF 14.7 million. This was complemented by the strategic investment of the investment firm Guangzhou Sino-Israel Bio-Industry Investment Fund 2 LLP (GIBF) of US$ 6 mio (approximately CHF 5.17 mio) announced in September 2024. This investment provides a strategic partnership to extend our development of BV100 to China as well as adding additional flexibility to our cash position. BV100 Phase 1 study in China is expected to start in 1H 2025 as part of our preparation for a global Phase 3 registration study. Dr. Marc Gitzinger, Chief Executive Officer and founder of BioVersys: "BioVersys has made significant progress during 2024 on multiple fronts. Our lead asset BV100 holds a lot of promise towards treating patients with very severe, drug-resistant bacterial infections, with limited or no treatment options. We are now looking forward to engaging with regulatory authorities to expedite the Phase 3 development of our lead asset in order to make this drug available as soon as possible to patients in need. Besides our clinical assets, BV100 and alpibectir, we are also very happy with the progress our pre-clinical pipeline in the form of BV200 and BV500, which is also moving forward. On the business side, we have extended our collaboration with GSK and we recently announced the investment from GIBF that will enable us to extend our clinical development to China. We remain committed to bringing new treatment options for drug resistant infections to patients in dire need and take great encouragement from the headway we are making towards that goal." About BV100BV100 is a novel formulation of rifabutin suitable for intravenous administration, with a recently discovered novel mode of action showing an active uptake of rifabutin into the Gram-negative bacterial species, Acinetobacter baumannii. For the first time, the lead candidate allows for the targeting of the RNA-polymerase enzyme in Gram-negative bacteria with a human-suitable dose. BV100 is being developed for the treatment of infections caused by Acinetobacter baumannii calcoaceticus complex (ABC), including Carbapenem-Resistant ABC (CRAB) in critically important indications of ventilator associated bacterial pneumonia (VABP), hospital-acquired bacterial pneumonia (HABP) and bloodstream infections (BSI). BV100 was granted QIDP Designation by the U.S. FDA in May 2019 for use in the treatment of VABP, HABP and BSI, making BV100 eligible for priority FDA review, Fast Track designation, and a five-year extension of market exclusivity upon approval of the first QIDP indication. About Acinetobacter baumanniiAcinetobacter baumannii calcoaceticus complex (ABC) are Gram-negative bacteria found in the environment (e.g., in soil and water) and an opportunistic pathogen in humans, typically infecting critically ill and immunocompromised patients, that can result in severe pneumonia and bloodstream infections in addition to affecting other parts of the body. ABC is considered a significant worldwide threat in the healthcare setting given its ability to survive for prolonged periods on surfaces, combined with its ability to develop or acquire resistance to standard of care antibiotics, e.g. carbapenems. Carbapenem-resistance as well as multidrug-resistance (MDR) rates for ABC are among the highest recorded for any bacteria in current times (The Lancet 2022; 399: 629–55). Incidence and resistance rates for ABC are trending upwards and COVID-19 has exacerbated this significantly. BioVersys forecasts the annual number of carbapenem-resistant A. baumannii infections in hospitals to have surpassed one million globally and due to the limited treatment options, such infections come with high (up to 50%) mortality rates. About alpibectirAlpibectir is a first in class small molecule acting on a transcriptional regulator protein in Mycobacterium tuberculosis (TB). Alpibectir rejuvenates the existing drug ethionamide (Eto), by both potentiating the activity of Eto and restoring the antibacterial activity of Eto towards Eto-resistant TB. The resulting fast bactericidal effect of alpibectir/Eto at low and safe doses offers the potential to replace Isoniazid (INH) in future TB drug regimens for MDR/XDR-TB but also in TB-meningitis patients or INH-mono-resistant patients. Alpibectir is being co-developed with our long-term partner GSK and the molecule originates form a very successful public-private partnership with the Pasteur Institute Lille and the University of Lille. The program was supported by several grants from the Wellcome Trust, EU IMI joint undertaking and EDCTP. Alpibectir in combination with Eto has the orphan drug designation (ODD) and QIDP designation from the US FDA. About tuberculosis (TB) Tuberculosis (TB) is one of the leading causes of death worldwide. Its causative agent is the bacterial pathogen Mycobacterium tuberculosis (Mtb). Worldwide, an estimated 10.6 million people developed TB in 2022 and an estimated 1.30 million died from TB. The WHO estimates that there were 410’000 new cases with resistance to rifampicin – the most effective first-line drug – most of them were multi-drug resistant (MDR). MDR-TB remains a public health crisis and a health security threat. Worldwide, only 63% of MDR-TB patients are currently successfully treated.1 In the modern world of global travel, and ease with which infections spread, it is very worrying to note that two-thirds of the global total of TB cases was in eight countries: India (27%), Indonesia (10%), China (7.1%), the Philippines (7.0%), Pakistan (5.7%), Nigeria (4.5%), Bangladesh (3.6%) and the Democratic Republic of the Congo (3.0%). Furthermore, 3.3% of all new and 17% of reoccurring TB cases were MDR/RR-TB. About BioVersysBioVersys AG is a multi-asset, clinical stage biopharmaceutical company focused on identifying, developing and commercializing novel antibacterial products for serious life-threatening infections caused by multi-drug resistant (“MDR”) bacteria. Derived from the company’s two internal technology platforms (TRIC and Ansamycin Chemistry), candidates are designed and developed to overcome resistance mechanisms, block virulence production and directly affect the pathogenesis of harmful bacteria towards the identification of new treatment options in the antimicrobial and microbiome fields. This enables BioVersys to address the high unmet medical need for new treatments against life-threatening resistant bacterial infections and bacteria-exacerbated chronic inflammatory microbiome disorders. The company’s most advanced research and development programs address nosocomial infections of Acinetobacter baumannii (BV100, Phase 2), and tuberculosis (alpibectir, Phase 2a, in collaboration with GlaxoSmithKline (GSK) and a consortium of the University of Lille, France). BioVersys is located in the biotech hub of Basel, Switzerland. BioVersys contact Sylvia Mundt, Tel. +41 61 633 22 50; Mail: IR@bioversys.com www.bioversys.com https://twitter.com/Bioversyshttps://www.linkedin.com/company/bioversys-ag 1 Global Tuberculosis Report 2023 WHO

TUESDAY, Oct. 1, 2024 -- A new test called CheekAge, based on a quick swab of cells in the mouth, might someday be used to predict how long a person has to live, developers report. The test tracks what are known as epigenetics: The way in which a person's environment or lifestyle affects how their genes function throughout the life span. A key marker of epigenetics is DNA methylation, molecular changes in DNA segments that can alter a gene's activity without changing its essential composition. The new CheekAge test is a kind of "epigenetic clock" that analyzes specific epigenetic DNA methylation markers in cells swabbed from the inside of the cheek. Certain patterns of methylation appear to be associated with expected life span, according to researchers at Tally Health, the New York company that is developing the test. "Specific methylation sites are especially important for this correlation, revealing potential links between specific genes and processes and human mortality captured by our clock,” explained study first author Dr. Maxim Shokhirev , head of computational biology and data science at Tally Health. His team published its findings Oct. 1 in the journal Frontiers in Aging . The study was funded by Tally Health and the Wellcome Trust. The new findings were based on data from the Lothian Birth Cohorts (LBC) program of the University of Edinburgh. It's been tracking the lifestyles, genetics and health of over 1,500 Scottish people born between 1921 and 1936. The participants have undergone DNA methylation testing using blood cells once every three years. Over 450,000 separate methylation sites on their genomes were tested. Those blood cells' epigenetics results were compared to results from CheekAge tests. “[Our results show that] CheekAge is significantly associated with mortality in a longitudinal dataset and outcompetes first-generation clocks trained in datasets containing blood data,” concluded the authors. More specifically, "for every increase by a single standard deviation in CheekAge," the odds for death from any cause rose by 21%, the researchers said in a journal news release. Right now, blood-based epigenetic testing is the gold standard, but of course a simple cheek swab would be easier on patients than a blood draw. Based on the new study, results from CheekAge appear to mirror those of blood tests, Shokhirev said. “The fact that our epigenetic clock trained on cheek cells predicts mortality when measuring the methylome in blood cells suggests there are common mortality signals across tissues,” he said. "This implies that a simple, noninvasive cheek swab can be a valuable alternative for studying and tracking the biology of aging," Shokhirev said. His team also zeroed on sites for methylation that seemed especially key to when a person might die. One such gene site is PDZRN4, possibly involved in suppressing cancer, while another is ALPK2, which appears tied to both cancer and heart health. Other methylation sites had links to osteoporosis , inflammation and metabolic syndrome, the researchers said. “It would be intriguing to determine if genes like ALPK2 impact lifespan or health in animal models,” said senior study author Dr. Adiv Johnson , head of scientific affairs and education at Tally Health. “Future studies are also needed to identify what other associations besides all-cause mortality can be captured with CheekAge," Johnson said. "For example, other possible associations might include the incidence of various age-related diseases or the duration of ‘healthspan,’ the period of healthy life free of age-related chronic disease and disability.” Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

DURHAM, N.C.--( BUSINESS WIRE )--Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS ® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, gene insertion, and gene excision, today announced changes within the Company’s clinical leadership team strengthening its infectious disease and hepatitis capabilities as it prepares to initiate development of its first in vivo gene editing candidate for hepatitis. Murray Abramson, MD, MPH has been appointed Senior Vice President, Head of Clinical Development and will oversee all clinical, safety, and medical affairs activities including clinical and operational strategy for our lead PBGENE-HBV program, as well as for the Company’s PBGENE-PMM program as it approaches Investigational New Drug (IND) and/or Clinical Trial Application (CTA) filings. Dr. Abramson is an infectious diseases and drug development expert who was most recently the Senior Vice President, clinical innovation at Tempus A.I. following his nine years as Vice President of Global Clinical Operations at Biogen. Dr. Abramson also spent over twelve years at Merck in medical and medical research operations roles. Prior to joining Merck, Dr. Abramson was a full-time faculty member at Duke University School of Medicine and Director, Infectious Diseases Clinical Research at Duke Clinical Research Institute. Dr. Abramson received his Medical Degree at Duke University Medical Center and his Masters of Public Health degree in Epidemiology from the University of North Carolina at Chapel Hill. In addition, John Fry has been appointed as a Strategic Clinical Advisor, specializing in hepatitis, and is supporting the clinical development strategy for PBGENE-HBV leveraging his multi-decade experience in early phase clinical research in the antiviral field, including multiple first-in-human studies evaluating novel therapies for chronic hepatitis B. Most recently, John was the Executive Vice President of Aligos Therapeutics following senior roles at Alios BioPharma, part of Janssen Pharmaceuticals, including serving as the Head of Early Development for Infectious Diseases. During his career, John also served in clinical research roles at PDL BioPharma, Gilead, Abbott Laboratories and Wellcome. “I’m delighted to welcome Murray Abramson to the Precision team, strengthening our core clinical capabilities and deepening our expertise in virology , ” said Michael Amoroso, Chief Executive Officer of Precision Biosciences. “His extensive knowledge and experience combined with our recently announced hepatitis scientific advisory board, will play an essential role in setting us up for global clinical success as we prepare to advance our first wholly owned program, PBGENE-HBV, in hepatitis B through clinic with anticipated Phase 1 data milestones in 2025.” “We are also fortunate that John Fry is providing instrumental advice and counsel to the PBGENE-HBV program built on many years of successfully executing clinical trials, including early-stage hepatitis B clinical trials,” added Mr. Amoroso. “As a near clinical stage company with the mission of delivering lasting cures in high impact diseases where no adequate treatments exist, we look forward to leveraging Murray’s and John’s passion and experience developing novel therapies for patients with infectious diseases.” In addition to these changes, Alan List, MD, Chief Medical Officer communicated his intention to retire from Precision effective September 13, 2024, following the one-year anniversary of the divestment of azer-cel for oncology along with the Company’s allogeneic CAR T infrastructure. Dr. List will assume a strategic advisory role as a clinical consultant to the company and directly support the CEO. In this capacity, Dr. List will provide ongoing platform advice and support to Precision BioSciences, while also providing continuity to ongoing clinical strategy and operations through Precision’s scientific advisory board. “I would like to thank Dr. List for his contributions to Precision BioSciences. He was instrumental in leading the ex vivo cell therapy hematology and oncology programs and building our clinical capabilities to execute the PBGENE-HBV clinical study which is planned for later this year,” added Mr. Amoroso. “Alan is a world class hematologist oncologist and alongside his many clinical accomplishments, his work at Precision BioSciences resulted in multiple CAR T partnerships which may potentially benefit patients with cancer and autoimmune disease as these programs advance through clinical studies. Alan has been a trusted thought partner, and I will miss his steady presence on the leadership team. We look forward to continuing our work with Alan as a strategic advisor as we continue to advance our programs forward as an in vivo gene editing company.” About Precision BioSciences, Inc. Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS ® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com . The ARCUS ® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including editing efficiency; the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the suitability of ARCUS nucleases for gene elimination, insertion and excision and differentiation from other gene editing approaches due to its small size, simplicity and distinctive cut; the expected timing of regulatory processes (including filings such as IND’s and CTA’s and studies for PBGENE-HBV); expectations about operational initiatives, strategies, and further development of our programs; expectations about achievement of key milestones; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions. Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the US and foreign regulatory landscape applicable to our and our collaborators’ or other licensees’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ and other licensees’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees’ ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ or other licensees’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2024, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com . All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.