Fusariosis

A total of 19 patients with serious/life-threatening invasive fungal infections have been enrolled, including aspergillosis, mucormycosis, fusarium, candidiasis, cryptococcosis, and endemic mycoses such as coccidioidomycosis and histoplasmosis All 5 patients who completed the desired course of treatment had complete clinical resolution of their infection; patients with ongoing treatment continue to experience significant clinical improvement All patients who transitioned to MAT2203 after developing renal toxicity following treatment with AmBisome® (liposomal amphotericin B) saw a reversal of renal impairment with a return to baseline renal function with no subsequent renal issues BEDMINSTER, N.J., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Matinas BioPharma (NYSE American: MTNB), a clinical-stage biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform delivery technology, today provided an update from its ongoing Compassionate/Expanded Use Access Program (the “Program”) with MAT2203, the Company’s proprietary, LNC-delivered oral formulation of the broad-spectrum antifungal drug amphotericin B. To date, 19 patients have been enrolled in the Program at prestigious healthcare institutions, including the University of Michigan, Johns Hopkins, Nationwide Children’s Hospital, City of Hope, Vanderbilt University Medical Center, the National Institutes of Health, Children’s Hospital of Philadelphia, Memorial Sloan Kettering Cancer Center, and the University of California, San Diego School of Medicine. The majority of enrolled patients are post-transplant or are undergoing treatment for underlying malignancies. The infections being treated with MAT2203 include a variety of micro-organisms (including Aspergillus, Mucorales species, Candidiasis, Fusarium and suspected Coccidioides) occurring at multiple sites of infection, including brain, bladder/colon, bone, lung, sinus, and skin. Most patients were receiving AmBisome prior to enrollment but developed treatment-limiting nephrotoxicity and most also required treatment for either azole-resistant organisms or had clinically failed azole therapy and had no other treatment options. “We are encouraged by the positive clinical impact of MAT2203 in each of our patients who faced few or no treatment options,” said Dr. Marisa Miceli, an internationally recognized infectious disease physician specializing in the treatment of invasive fungal infections and Professor of Medicine in the Division of Infectious Diseases at the University of Michigan. “Of the seven patients we have treated to date under the Program, all were either unable to receive azole therapy due to drug-drug interactions, intolerance or resistance and some of them experienced serious treatment-limiting toxicities while attempting therapy with IV-administered amphotericin B. Treatment with MAT2203 was well tolerated and led to favorable clinical and radiological response and we did not observe any renal toxicity. While additional study is warranted, MAT2203 may be particularly promising for the treatment of invasive fungal infections in patients with limited options and therefore satisfy a currently significant unmet medical need.” Of the 19 patients enrolled in the Program, 15 have available follow-up and 4 recently initiated or will soon commence treatment with MAT2203. 12 of the 15 patients had either complete clinical resolution or objective improvement in clinical markers of infection (including radiologic and mycologic).Of the 5 patients who completed their full individually specified course of treatment (ranging from 2 weeks to 1 year, depending on the infection) all have had complete clinical resolution with no relapses or recurrence of their infection.5 additional patients have shown objective improvement in clinical markers and are continuing treatment with MAT2203 as planned.5 of the 15 patients were unable to complete their individually specified course of treatment, although 2 saw significant clinical improvement of their fungal infection and are included in the 12 overall successful cases. 2 patients transitioned to palliative care shortly after starting therapy with MAT2203 because of unanticipated progression of their malignant disease.1 patient discontinued therapy after two days due to underlying GI issues (i.e., Crohn’s disease).1 patient passed away due to progression of their underlying disease approximately 8 weeks into therapy (but previously experienced significant clinical improvement of their fungal infection).1 patient discontinued MAT2203 treatment following 10 weeks of therapy due to underlying GI issues (long-standing nausea/vomiting), but with improvement in their fungal infection. Importantly, all patients who experienced renal toxicity following treatment with AmBisome saw their renal function return to baseline after transitioning to MAT2203 therapy and suffered no further renal side effects over the course of extended treatment with MAT2203. “We continue to be excited about the ongoing, consistent positive clinical impact of MAT2203, seen in these extremely ill patients,” said Theresa Matkovits, PhD, Chief Development Officer at Matinas. “We look forward to advancing MAT2203 into Phase 3 development in the ORALTO registration trial and to validating these results in a well-controlled clinical trial in which we believe the clinical probability of success is quite high. If approved, oral, effective, and safe MAT2203 could represent a dramatic improvement to current clinical standard of care and become the treatment of choice for patients and physicians battling invasive fungal infections. We are grateful to the participants in the Program and to their physicians for recognizing the clinical potential of MAT2203 in treating a broad spectrum of deadly invasive fungal infections.” MAT2203 is not yet licensed or approved anywhere globally. About MAT2203Matinas BioPharma is developing MAT2203 as a potential oral broad-spectrum treatment for invasive deadly fungal infections. Although amphotericin B is a fungicidal agent, it is currently only available through an intravenous route of administration, which is known to be associated with several significant safety issues such as renal toxicity and anemia due to very high circulating levels of amphotericin B. MAT2203 has the potential to overcome the significant limitations of the currently available amphotericin B products due to its targeted oral delivery. Combining comparable fungicidal activity with targeted delivery results in a lower risk of toxicity and potentially creates the ideal antifungal agent for the treatment of invasive fungal infections. MAT2203 was successfully evaluated in the completed Phase 2 EnACT study in HIV patients suffering from cryptococcal meningitis, meeting its primary endpoint, and achieving robust survival. MAT2203 will be further evaluated in a single Phase 3 registration trial (the “ORALTO” trial) as an oral step-down monotherapy following treatment with AmBisome (liposomal amphotericin B) compared with the standard of care in patients with invasive aspergillosis who have limited treatment options. About Matinas BioPharmaMatinas BioPharma is a biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform delivery technology. In addition to MAT2203, preclinical and clinical data have demonstrated that this novel technology can potentially provide solutions to many challenges of achieving safe and effective intracellular delivery of both small molecules and larger, more complex molecular cargos including small oligonucleotides such as ASOs and siRNA. The combination of its unique mechanism of action and flexibility with routes of administration (including oral) positions Matinas’ LNC technology to potentially become a preferred next-generation orally available intracellular drug delivery platform. For more information, please visit www.matinasbiopharma.com. Forward-looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including those relating to our business activities, our strategy and plans, the future development of its product candidates, including MAT2203, the Company’s ability to identify and pursue development, licensing and partnership opportunities for its products, including MAT2203, or platform delivery technology on favorable terms, if at all, and the ability to obtain required regulatory approval and other statements that are predictive in nature, that depend upon or refer to future events or conditions. All statements other than statements of historical fact are statements that could be forward-looking statements. Forward-looking statements include words such as "expects," "anticipates," "intends," "plans," "could," "believes," "estimates" and similar expressions. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results to be materially different from any future results expressed or implied by the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to continue as a going concern, our ability to obtain additional capital to meet our liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials of our product candidates; our ability to successfully complete research and further development and commercialization of our product candidates; the uncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals; our ability to protect the Company’s intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company’s products; and the other factors listed under "Risk Factors" in our filings with the SEC, including Forms 10-K, 10-Q and 8-K. Investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this release. Except as may be required by law, the Company does not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. Matinas BioPharma’s product candidates are all in a development stage and are not available for sale or use. Investor Contact LHA Investor RelationsJody Cain Jcain@lhai.com310-691-7100

FDA feedback on MAT2203 Phase 3 program supports a patient population in invasive aspergillosis with limited treatment options, acknowledges potential for LPAD pathway; composite superiority endpoint under evaluation to strengthen commercial opportunity without increasing study size Patient with Candida krusei infection achieved complete clinical resolution with MAT2203; additional patient enrolled in the Compassionate/Expanded Use Access Program Oral LNC formulation of docetaxel showed reduction in tumor size comparable to IV-docetaxel with no systemic toxicity in preclinical melanoma model Conference call begins at 4:30 p.m. Eastern time today BEDMINSTER, N.J., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Matinas BioPharma (NYSE American: MTNB), a clinical-stage biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform delivery technology, reports financial results for the three and nine months ended September 30, 2023 and provides a business update. “We are very excited by the consistent, positive clinical data generated with MAT2203 in our Compassionate/Expanded Use Access Program. The patients treated under this program suffer from invasive fungal infections with few treatment options. We plan to treat a similar patient population in our Phase 3 study – those with invasive aspergillosis and limited treatment options,” said Jerome D. Jabbour, Chief Executive Officer of Matinas. “Our recent meeting with FDA supports our Phase 3 strategy, including the potential for LPAD registration. We’re highly encouraged by FDA’s openness to a superiority composite primary endpoint, which could be significant in positioning MAT2203 for commercial success. We look forward to finalizing the Phase 3 study design as soon as possible and advancing our ongoing partnership discussions for this important product.” Key Program Updates MAT2203 (Oral Amphotericin B) Program FDA Meeting Feedback A meeting was held with the U.S. Food and Drug Administration (FDA) in mid-October with both the Director and Deputy Director of the Office of Infectious Diseases in attendance. The FDA agreed with the Phase 3 study design for the treatment of invasive aspergillosis in patients with limited treatment options and confirmed that MAT2203 may be a candidate for the Limited Population Pathway for Antifungal and Antibacterial Drugs (LPAD).During discussions regarding the statistical assumptions for a noninferiority Phase 3 trial design, FDA expressed openness to a proposed alternative superiority composite endpoint, which we believe would best position MAT2203 for commercial success and differentiation upon approval. The revised superiority composite endpoint is being finalized and will be submitted to FDA for alignment in the next few weeks.The Company believes that a superiority study incorporating a composite endpoint would not change the projected study size (<200 patients) or enrollment timeline (22-24 mos.). The FDA has communicated its commitment to work with the Company on an off-cycle basis to finalize the Phase 3 protocol as soon as possible. General MAT2203 Updates A total of 12 patients have been enrolled in the Company’s Compassionate/Expanded Use Access Program. In October, the Company announced that a 61-year-old male with a challenging medical history achieved complete clinical resolution of a Candida krusei infection following only two weeks of treatment with MAT2203. Treatment with IV-amphotericin B was discontinued due to renal toxicity and the patient was transitioned to MAT2203, which was well-tolerated with no adverse effects. Treatment with MAT2203 led to complete resolution of his symptoms and improvement of his kidney function to baseline.Earlier this month, an additional patient was enrolled in this Program at Vanderbilt University Medical Center. This patient is suffering from a CNS-based fusarium infection and required transition from IV-amphotericin due to significant electrolyte abnormalities. The patient may also be able to be discharged from the hospital in order to receive treatment at home.University of Minnesota Medical School researchers published results from the Phase 2 EnACT trial evaluating MAT2203 for the treatment of cryptococcal meningitis as a Major Article and Editor’s Choice in Clinical Infectious Diseases, an official publication of the Infectious Diseases Society of America (IDSA).The published results of the EnACT Phase 2 trial, the ongoing positive clinical outcomes in the Compassionate/Expanded Use Access Program, and the recent additional clarity and support from the FDA for the MAT2203 Phase 3 program have all resulted in significant new interest from potential partners for licensing, acquiring or otherwise collaborating on MAT2203.During a meeting held in the third quarter, the Biomedical Advanced Research and Development Authority (BARDA) invited the Company to submit a White Paper for Phase 3 funding consideration. The Company intends to submit the White Paper following finalization of the Phase 3 study design. LNC Platform Updates Oral LNC-Docetaxel The Company announced positive results from an in vivo animal study of an oral LNC formulation of docetaxel, a well-known chemotherapeutic agent used in the management of multiple metastatic and unresectable tumors. Anti-tumor effects of daily oral LNC-docetaxel were comparable to IV-docetaxel with statistically significant reductions in tumor volume compared with untreated controls at Day 14 (high dose oral LNC -63%; low dose oral LNC -57%; IV docetaxel -68%), and similar reductions in tumor weight at Day 14. No systemic toxicities were noted. Body weight was stable over treatment duration and hematologic parameters were similar to untreated controls.Potential next steps include evaluating the efficacy of the current LNC-docetaxel formulation in other tumor models and evaluating longer-term treatment regimens to confirm lack of toxicity. Additionally, the Company plans to evaluate the potential anti-tumor activity of LNC formulations of small oligonucleotides. Internal Oral LNC Small Oligonucleotide Program The Company is investigating a variety of LNC formulations of two small oligonucleotides designed to target inflammatory cytokines IL-17A and TNFa, and has conducted a series of in vitro and in vivo studies to evaluate the biological activity associated with oral delivery as well as the corresponding associated clinical benefit of IL-17A knockdown in an imiquimod (IMQ) induced murine psoriasis disease model.These preliminary studies have documented biological activity in the form of cytokine knockdown and have also provided some evidence of associated tangible clinical benefit with improvements in skin lesion appearance (redness, scaling) in this qualitative psoriasis model. These data remain under evaluation and the analyses are focused on (a) clarifying the strength and time course of cytokine inhibition, and (b) evaluating cytokine mRNA levels and specific tissue responses in these models to better understand and interpret these data.Additional in vivo studies of LNC-formulated small oligonucleotides in other disease models are ongoing with additional data expected in the fourth quarter of this year. Third Quarter Financial Results The Company reported no revenue for the third quarter of 2023 compared with $1.1 million of revenue for the third quarter of 2022, which was generated from the Company’s research collaborations with BioNTech SE. Total costs and expenses for the third quarter of 2023 were $6.1 million compared with $6.5 million for the third quarter of 2022. The decrease was primarily attributable to decreases in costs for the manufacturing of clinical trial materials, and lower clinical trial consulting and headcount costs. The net loss for the third quarter of 2023 was $6.1 million, or $0.03 per share, compared with a net loss for the third quarter of 2022 of $5.5 million, or $0.03 per share. Nine Month Financial Results Revenue for the first nine months of 2023 was $1.1 million compared with $2.1 million for the first nine months of 2022. Total costs and expenses for the first nine months of 2023 were $19.0 million compared with $21.2 million for the first nine months of 2022. The net loss for the first nine months of 2023 was $17.6 million, or $0.08 per share, compared with a net loss for the first nine months of 2022 of $17.4 million, or $0.08 per share. Cash, cash equivalents and marketable securities as of September 30, 2023, were $18.2 million compared with $28.8 million as of December 31, 2022. Based on current projections, the Company believes its cash position is sufficient to fund planned operations into the third quarter of 2024. The Company is seeking to extend its cash runway by securing non-dilutive funding from potential third-party development partners and government grant programs through agencies such as BARDA, as well as from potential public or private equity offerings. Conference Call and Webcast Matinas will host a conference call and webcast today beginning at 4:30 p.m. Eastern time. To participate in the call, please dial 800-267-6316 or 203-518-9783. The live webcast will be accessible on the Investors section of the company’s website and archived for 90 days. About Matinas BioPharma Matinas BioPharma is a biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform delivery technology. Matinas’ lead LNC-based therapy is MAT2203, an oral formulation of the broad-spectrum antifungal drug amphotericin B, which although highly potent, can be associated with significant toxicity. Matinas’ LNC platform provides oral delivery of amphotericin B without the significant nephrotoxicity otherwise associated with IV-delivered formulations. MAT2203 also allows for safe, longer-term use outside of a hospital setting, which could have substantial favorable pharmacoeconomic impact. MAT2203 was successfully evaluated in the completed Phase 2 EnACT study in cryptococcal meningitis, meeting its primary endpoint and achieving robust survival. MAT2203 will be further evaluated as an oral step-down monotherapy treatment following IV amphotericin B in a single pivotal Phase 3 study in the treatment of aspergillosis in persons with limited treatment options who are unable to be treated with azoles for reasons related to drug-drug interactions, resistance or for whom these antifungal agents are unable to be used for other clinical reasons. In addition to MAT2203, preclinical and clinical data have demonstrated that this novel technology can potentially provide solutions to many of the challenges standing in the way of achieving safe and effective intracellular delivery of both small molecules and larger, more complex molecular cargos such as RNAi, antisense oligonucleotides, and vaccines. The combination of its unique mechanism of action and flexibility with routes of administration (including oral) positions Matinas’ LNC technology to potentially become a preferred next-generation orally available intracellular drug delivery platform. For more information, please visit www.matinasbiopharma.com. Forward-looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including those relating to our business activities, our strategy and plans, our collaboration with National Resilience, Inc., the potential of our LNC platform technology, and the future development of its product candidates, including MAT2203, the Company’s ability to identify and pursue development, licensing and partnership opportunities for its products, including MAT2203, or platform delivery technologies on favorable terms, if at all, and the ability to obtain required regulatory approval and other statements that are predictive in nature, that depend upon or refer to future events or conditions. All statements other than statements of historical fact are statements that could be forward-looking statements. Forward-looking statements include words such as "expects," "anticipates," "intends," "plans," "could," "believes," "estimates" and similar expressions. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results to be materially different from any future results expressed or implied by the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to continue as a going concern, our ability to obtain additional capital to meet our liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials of our product candidates; our ability to successfully complete research and further development and commercialization of our product candidates; the uncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals; our ability to protect the Company’s intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company’s products; and the other factors listed under "Risk Factors" in our filings with the SEC, including Forms 10-K, 10-Q and 8-K. Investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this release. Except as may be required by law, the Company does not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. Matinas BioPharma’s product candidates are all in a development stage and are not available for sale or use. Investor Contact: LHA Investor RelationsJody Cain Jcain@lhai.com310-691-7100 [Financial Tables to Follow] Matinas BioPharma Holdings, Inc.Condensed Consolidated Balance Sheets(in thousands, except for share data) September 30, 2023 December 31, 2022 (Unaudited) (Audited) ASSETS: Current assets: Cash and cash equivalents $6,407 $6,830 Marketable debt securities 11,809 21,933 Restricted cash – security deposit 50 50 Prepaid expenses and other current assets 1,723 5,719 Total current assets 19,989 34,532 Non-current assets: Leasehold improvements and equipment – net 2,021 2,091 Operating lease right-of-use assets – net 3,206 3,613 Finance lease right-of-use assets – net 22 30 In-process research and development 3,017 3,017 Goodwill 1,336 1,336 Restricted cash – security deposit 200 200 Total non-current assets 9,802 10,287 Total assets $29,791 $44,819 LIABILITIES AND STOCKHOLDERS’ EQUITY: Current liabilities: Accounts payable $835 $618 Accrued expenses 1,757 3,099 Operating lease liabilities – current 632 562 Financing lease liabilities – current 5 7 Total current liabilities 3,229 4,286 Non-current liabilities: Deferred tax liability 341 341 Operating lease liabilities – net of current portion 3,052 3,533 Financing lease liabilities – net of current portion 19 22 Total non-current liabilities 3,412 3,896 Total liabilities 6,641 8,182 Stockholders’ equity: Common stock par value $0.0001 per share, 500,000,000 shares authorized at September 30, 2023 and December 31, 2022; 217,264,526 issued and outstanding as of September 30, 2023 and December 31, 2022 22 22 Additional paid-in capital 193,746 190,070 Accumulated deficit (170,259) (152,631)Accumulated other comprehensive loss (359) (824)Total stockholders’ equity 23,150 36,637 Total liabilities and stockholders’ equity $29,791 $44,819 Matinas BioPharma Holdings, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data)Unaudited Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Revenue: Contract Revenue $— $1,063 $1,096 $2,125 Costs and expenses: Research and development 3,295 3,707 10,824 12,811 General and administrative 2,839 2,818 8,151 8,424 Total costs and expenses 6,134 6,525 18,975 21,235 Loss from operations (6,134) (5,462) (17,879) (19,110)Sale of New Jersey net operating losses & tax credits — — — 1,734 Other income, net 79 — 251 13 Net loss $(6,055) $(5,462) $(17,628) $(17,363)Net loss per share – basic and diluted $(0.03) $(0.03) $(0.08) $(0.08) Weighted average common shares outstanding: Basic and diluted 217,264,526 216,864,526 217,264,526 216,792,083 Other comprehensive gain/(loss), net of tax Unrealized gain/(loss) on securities available-for-sale 155 (181) 465 (790)Other comprehensive gain/(loss), net of tax 155 (181) 465 (790)Comprehensive loss $(5,900) $(5,643) $(17,163) $(18,153)

An overall success rate of 28.7% was seen at day 42, the primary endpoint, and 27.2% at day 84, as determined by a data review committee (DRC) using the EORTC-MSG criteriaThe success rate rose to 75.2% at day 42 and 63.4% at day 84 when stable disease was counted as successAll-cause mortality (ACM) at day 42 and day 84 was 11.4% and 15.8%, respectively MANCHESTER, United Kingdom and OSAKA, Japan, Oct. 21, 2023 (GLOBE NEWSWIRE) -- F2G Ltd. and Shionogi & Co., Ltd., today announced full results from the pivotal Phase 2b open-label study (Study 32, NCT03583164) of olorofim in patients with proven invasive fungal infections (IFI) or probable pulmonary invasive aspergillosis and limited or no treatment options. These findings were submitted as a late breaking abstract and exceptionally accepted for an oral presentation during the 11th Congress on Trends in Medical Mycology (TIMM) 2023 held in Athens, Greece, from 20-23 October 2023. Altogether, ten olorofim-related abstracts from F2G and independent experts were accepted for presentation at TIMM, one of the largest global meetings in which scientists and clinicians from all over the world present their latest advances and research findings in medical mycology. “We are excited about this data as it builds on the previous findings we announced at IDWeek 2022 demonstrating that olorofim has the potential to address a high unmet need in treating infections due to moulds including species considered resistant to all approved antifungals,” said John H. Rex, MD, Chief Medical Officer of F2G. Juan Carlos Gomez, Chief Medical Officer of Shionogi & Co., Ltd. added, “In 2022, the World Health Organization published the fungal priority pathogens list, recognizing the burden of invasive fungal infections and highlighting the need for more antifungal agents to overcome increasing antifungal resistance and rare fungal pathogens. We are encouraged by these data because they demonstrate olorofim was generally well tolerated and has the potential to treat patients with life-threatening fungal infections.” Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (Belgium) who presented the data for Study 32, added, “There is a critical need for novel antifungal therapies because lifesaving medical interventions such as organ transplants and cancer therapies, along with an ageing population, have created an increase in immunocompromised people who are at risk of invasive fungal infections from pathogens, and many of these are becoming harder to treat. I am optimistic about olorofim’s potential to address this need.” Key findings: Data came from a Phase 2b open-label study of oral olorofim in 203 patients with limited treatment options for proven IFI or probable pulmonary invasive aspergillosis (IA). 202 patients had IFI meeting the modified ITT (mITT) criteria for analysisCausative organisms included Aspergillus species (101, including 22 cases with azole-resistant strains), Lomentospora prolificans (26), Scedosporium spp. (22), Coccidioides spp. (41), Scopulariopsis spp (6) and other fungi (8).All patients had limited alternative treatment options including failure of available therapy, resistance of infecting isolate to all licensed agents, intolerance to available therapy, inability to manage drug-drug interactions, and/or inability to produce therapeutic drug levels.Treatment was for up to 84 days +/- 7 days with extended therapy permitted when clinically indicated. The median dosing duration in the main phase of the study was 84 days. When including extended therapy, the median treatment duration was 308 days.Olorofim demonstrated an acceptable benefit-risk profile in a well-defined population of patients with limited or no treatment options for infections due to moulds including species considered resistant to all approved antifungals. The overall success rate at day 42 (primary endpoint), as determined by an independent DRC applying the EORTC-MSG criteria1, was 28.7% at day 42 and 27.2% at day 84.The overall success in patients with an IFI other than coccidioidomycosis (n = 161), was 36.0% at day 42 and 34.2% at day 84.For patients with coccidioidomycosis (n = 41, none of whom achieved overall success due to inability to prove fungal eradication in the 84-day study period), benefit (symptoms/signs) was obtained in 31 patients (75.6%, CI 59.7,87.6) achieving complete or partial clinical response at day 42, and 30 patients (73.2%, CI 57.1, 85.8) at day 84.When considering stable disease as a successful clinical response, the rates of successful outcome for the whole population at day 42 and day 84 rose to 75.2% and 63.4%, respectively. ACM at day 42 and day 84 was 11.4% and 15.8%, respectively (IA: 17.8% and 25.7%; non-IA infections 5.0% and 5.9%, respectively).Olorofim was generally well-tolerated, even with dosing >2 year in extension therapy. Changes in liver biochemistry that were deemed at least possibly due to olorofim were seen in 9.9% of study subjects overall (main phase + extended therapy) and were managed by dose reduction/pause.Changes in the liver biochemistry led to permanent discontinuation in 2.5%.Gastrointestinal intolerance to olorofim, generally self-limiting, was noted in 9.9%. F2G and Shionogi & Co., Ltd. are collaborating to develop and commercialise olorofim, bringing the novel antifungal therapy to patients with invasive fungal infections. F2G has commercial responsibility for olorofim in North America, and Shionogi has commercial responsibility for olorofim in Europe and Asia Pacific. F2G and Shionogi are currently enrolling patients with invasive aspergillosis in a global Phase 3 study (OASIS) to compare treatment with olorofim versus AmBisome® followed by standard of care (NCT05101187). About Study 32 Study 32 was a multicenter, open-label, Phase 2b study to evaluate the safety and efficacy of olorofim (formerly F901318) in patients ≥ 18 years of age with probable pulmonary invasive aspergillosis or proven invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi with limited or no treatment options. It enrolled patients between June 2018 and September 2022. Enrolled patients received an initial loading dose of 150 mg BID (twice a day) of oral olorofim on day one, and subsequent oral doses of 90 mg BID for up to 90 days. Patients were followed for another four weeks after the end of treatment, while some received extended therapy for as long as thought clinically beneficial. For more information on Study 32, visit ClinicalTrials.gov (NCT03583164). About Olorofim Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class. A Phase 2b single-arm open-label study in patients who have limited treatment options for difficult-to-treat invasive fungal infections due to mould such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mould infections has completed enrollment. To expand on the studied patient population, F2G has initiated a global Phase 3 trial (“OASIS”) to compare treatment with olorofim versus AmBisome® followed by standard of care (SOC) in patients with proven or probable invasive fungal infection due to Aspergillus species (NCT05101187). Olorofim has received orphan drug designation from the FDA for the treatment of coccidioidomycosis, scedosporiosis (including lomentosporiosis), invasive Scopulariopsis, and invasive aspergillosis. Olorofim has also received orphan drug designation from the European Medicines Agency (EMA) for the treatment of invasive aspergillosis, invasive scedosporiosis (including lomentosporiosis), and invasive Scopulariopsis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for invasive aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis. Olorofim has received FDA Breakthrough Therapy Designation for both “treatment of invasive fungal infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species” and “treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy.” Olorofim is not approved by the FDA or any other regulatory agency. Invasive fungal infections cause substantial morbidity and mortality, particularly among immunosuppressed patients, and can cause significant morbidity in otherwise healthy individuals when they get into soft tissues, bones and joints, requiring both antifungal therapy and major surgical interventions. Effective antifungal therapies do not currently exist for some of these fungi. Even when therapies exist, some patients with invasive infections may be refractory or unable to tolerate existing antifungal treatments, thus underscoring the need for new and effective treatments. About F2G F2G is a biotech company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides which selectively target a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. For more information, please visit: www.f2g.com About Shionogi Shionogi & Co., Ltd. is a 145-year-old global, research driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain and CNS disorders.Shionogi’s research and development currently target two therapeutic areas: infectious diseases, and pain/CNS disorders. For more information on Shionogi & Co., Ltd., please visit http://www.shionogi.co.jp/en/. Shionogi B.V. is the European headquarters of Shionogi & Co., Ltd. For more information on Shionogi B.V., please visit www.shionogi.eu. Forward-Looking Statements This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also, for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise. Media Contact:Gloria GasaaturaLifeSci Communications+1 646 970 4688ggasaatura@lifescicomms.com SHIONOGI Website Inquiry Form https://www.shionogi.com/global/en/contact.html.EU Media: pressoffice:shionogi.eu _____________________________________1 Segal BH, Herbrecht R, Stevens DA, Ostrosky-Zeichner L, et al. Defining responses to therapy and study outcomes in clinical trials of invasive fungal diseases: Mycoses Study Group and European Organization for Research and Treatment of Cancer consensus criteria. Clin Infect Dis. 2008 Sep 1;47(5):674-83. doi: 10.1086/590566. PMID: 18637757; PMCID: PMC2671230.