Hemolytic-Uremic Syndrome

Presented positive interim data from randomized Dose Expansion cohort of the Phase 2 PRISM study evaluating 4D-150 in wet AMD patients with severe disease activity and high treatment burden, enabling advancement into Phase 3 pivotal development, with initiation expected in Q1 2025Positive interim clinical data for 4D-710 in cystic fibrosis and 4D-310 in Fabry disease continue to demonstrate the potential of these product candidates and the efficient delivery, transgene expression and clinical activity of 4DMT’s three clinical-stage vectorsStrategic agreements with Astellas Pharma in rare monogenic retinal diseases and Arbor Biotechnologies in CNS gene editing continue to validate the versatility of 4DMT’s Therapeutic Vector Evolution platform Completed upsized public follow-on offering with total gross proceeds of $300 million supplements our cash position of $299 million (as of December 31, 2023) and extends our operational runway into the first half of 2027 EMERYVILLE, Calif., Feb. 29, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today reported full year 2023 financial results and provided operational highlights. “2023 was a transformative year for 4DMT with incredible progress across our product pipeline and platform in multiple therapeutic areas, especially for our lead program 4D-150 in wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME),” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “Robust enrollment ahead of schedule across multiple cohorts in the PRISM clinical trial of patients with wet AMD and the SPECTRA clinical trial of patients with DME have set the stage for multiple important data readouts in 2024, starting with our recent positive interim data at Angiogenesis, Exudation, and Degeneration 2024 Conference in the hardest-to-treat wet AMD patients. As of the most recent data update, 110 patients have been dosed with 4D-150 with no clinically significant inflammation, which continues to build on the emerging profile of 4D-150 as a potentially best-in-class long-acting genetic medicine with the opportunity for broad market adoption. We look forward to further updates in the broad wet AMD population and DME patients in the second half of 2024, setting the stage for our first Phase 3 clinical trial in wet AMD currently expected to initiate in the first quarter of 2025. We also look forward to providing updates on other key programs beyond 4D-150 in 2024, including 4D-175 for geographic atrophy, 4D-710 for cystic fibrosis and 4D-310 for Fabry disease cardiomyopathy. Our recent financing and resulting cash position allows us to execute on our vision of becoming a fully integrated, next-generation genetic medicines company.” Recent Highlights in Large Market Ophthalmology Portfolio 4D-150 for Wet Age-Related Macular Degeneration: Rapidly advanced intravitreal 4D-150 for wet AMD in the Phase 1/2 PRISM clinical trial Randomized Dose Expansion cohort (N=51) in patients with severe disease activity and high treatment burden presented at Angiogenesis, Exudation, and Degeneration 2024 Conference (data cutoff January 19, 2024): Favorable safety profile demonstrated, with no significant or recurrent intraocular inflammation, and no 4D-150–related serious adverse events (SAEs) or study eye SAEs. All patients were off steroidsAt 24 weeks, 4D-150 resulted in robust reductions in overall treatment burden, % of patients with 0-1 injections, % of patients injection-free, plus improved retinal anatomical control vs. the aflibercept control Durable responses demonstrated beyond 1 year in Phase 1 3E10 vg/eye dose cohort, with 3 patients injection-free through 80–104 weeks (up to 2 years) of follow-upEnrollment completed ahead of schedule in Phase 2 PRISM Population Extension cohort (N=32) evaluating 4D-150 in patients with broad disease activity and treatment burden versus the Dose Expansion cohort Received RMAT and PRIME designations, enabling increased collaboration between the FDA and EMA on regulatory planningAnnounced preliminary pivotal Phase 3 clinical trial design and plans 4D-150 for Diabetic Macular Edema: Enrollment and dosing completed ahead of schedule in Dose Confirmation cohort (N=22) of the Phase 2 SPECTRA clinical trial in DME Recent Highlights in Pulmonary Portfolio 4D-710 for Cystic Fibrosis Lung Disease: Presented positive interim data from the Phase 1/2 AEROW clinical trial at the North American Cystic Fibrosis ConferenceDose ranging continues (5E14 – 2E15 vg) with lung biopsy CFTR expression significantly above normal controls, demonstrating the feasibility of efficient delivery and CFTR transgene expression at lower doses; first participant dosed in lower dose Cohort 3 (5E14 vg)Received Rare Pediatric Disease Designation in January 2024 and Orphan Drug Designation from FDA in February 2024 Recent Highlights in Cardiology Portfolio 4D-310 for Fabry Disease Cardiomyopathy: Interim clinical and biopsy data from Phase 1/2 INGLAXA clinical trials presented in the late-breaking session at WORLDSymposium™ 2024: 4D-310 demonstrated clinically meaningful improvements on multiple cardiac endpoints through 12-24 months in five evaluable patientsCardiac biopsies from one patient at week 6 and 24 showed robust and durable delivery, transgene expression and clearance of Gb3 substrate in cardiomyocytesSafety and tolerability profile maintained; previously reported cases of atypical hemolytic uremic syndrome (aHUS) (n=3) have fully resolved Reached agreement with FDA on a proposed plan to address clinical hold. Single non-human primate safety study evaluating intravenous 4D-310 with the rituximab/sirolimus immunosuppressive regimen underway Strategic Agreements & Partnerships In July 2023, entered license agreement with Astellas Pharma for their use of R100 vector for genetic targets implicated in rare monogenic ophthalmic diseases and received $20 million upfront with potential future milestones of up to $942.5 million, including potential near-term development milestones of $15 million for the initial targetIn December 2023, entered strategic partnership with Arbor Biotechnologies to engineer, co-develop and co-commercialize AAV-delivered CRISPR/Cas-based therapeutics for up to six product candidates in the CNS therapeutic area Expected Upcoming Milestones 4D-150 for Wet AMD: Phase 2 PRISM Population Extension cohort (N=32) in patients with broad disease activity and treatment burden: Initial interim 24-week landmark analysis expected in Q3 2024 Phase 3 Planning: Additional FDA and EMA regulatory interactions under RMAT and PRIME planned in Q2 2024; update expected in Q3 2024Phase 3 clinical trial initiation expected in Q1 2025 4D-150 for DME: Phase 2 SPECTRA clinical trial Dose Confirmation cohort (N=22): Initial interim 24-week landmark data expected in H2 2024 4D-175 for Geographic Atrophy: IND filing expected in Q2 2024Phase 1 initiation expected in H2 2024 4D-710 for CF Lung Disease: Update on FDA feedback on development plan for monotherapy and approved CF modulator combination regimens expected in Q1 2024Interim update from Phase 1/2 AEROW clinical trial expected in mid-2024 4D-725 for A1AT Deficiency Lung Disease: Program update and initiation of IND-enabling studies expected in 2024 4D-310 for Fabry Disease Cardiomyopathy: FDA submission of preclinical NHP safety data in combination with rituximab/sirolimus immunosuppression regimen expected in Q2 2024 4D-110 for Choroideremia and 4D-125 for X-Linked Retinitis Pigmentosa: Program updates expected in 2024 Full Year 2023 Financial Results Cash and Cash Equivalents and Marketable Securities: Cash and cash equivalents and marketable securities were $299 million as of December 31, 2023, as compared to $218 million as of December 31, 2022. The net increase in cash was primarily a result of cash inflows from $129 million of net proceeds from our public offering of common stock completed in May, $19 million of net proceeds under our Open Market Sales Agreement, and the $20 million upfront payment in connection with the Astellas License Agreement and was partially offset by cash used in operations. In addition, in February 2024 we completed a public offering of common stock and prefunded warrants that resulted in us receiving net proceeds of approximately $281 million. We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027. R&D Expenses: Research and development expenses were $97.1 million for 2023, as compared to $80.3 million for 2022. This increase was driven by the progression of our existing clinical trials, primarily 4D-150 in wet AMD and DME, along with increased payroll and stock-based compensation expense due to higher headcount. G&A Expenses: General and administrative expenses were $36.5 million for 2023, as compared to $32.9 million for 2022. Net Loss: Net loss was $100.8 million for 2023, as compared to net loss of $107.5 million for 2022. About 4DMT 4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing five clinical-stage and two preclinical product candidates, each tailored to address rare and large market diseases in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at www.4DMT.com and follow us on LinkedIn. Forward Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits of 4DMT’s product candidates, as well as the plans, announcements and related timing for the clinical development of our clinical and preclinical product candidates, and statements regarding our financial performance, results of operations and anticipated cash runway. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward looking statements. 4D Molecular Therapeutics, Inc.Statements of Operations (Unaudited)(in thousands, except share and per share amounts) Year Ended December 31, 2023 2022 Collaboration and license revenue $20,723 $3,129 Operating expenses: Research and development 97,096 80,253 General and administrative 36,494 32,908 Total operating expenses 133,590 113,161 Loss from operations (112,867) (110,032)Other income (expense), net: 12,030 2,538 Net loss $(100,837) $(107,494)Net loss per share, basic and diluted $(2.58) $(3.32)Weighted-average shares outstanding used in computing net loss per share, basic and diluted 39,130,067 32,351,221 4D Molecular Therapeutics, Inc.Balance Sheet Data (Unaudited)(in thousands) As of December 31, 2023 2022 Cash and cash equivalents and marketable securities $299,186 $218,462 Working capital 277,637 204,780 Total assets 339,891 261,846 Total liabilities 32,062 30,509 Accumulated deficit (415,327) (314,490)Total stockholders’ equity 307,829 231,337 Contacts: Media: Katherine SmithInizio Evoke CommsKatherine.Smith@inizioevoke.com Investors: Julian PeiHead of Investor Relations and Corporate CommunicationsInvestor.Relations@4DMT.com

4D-310 demonstrated clinically meaningful cardiac endpoint improvements through 12-24 months in contractility (echocardiography), peak VO2 (cardiopulmonary exercise testing) and/or cardiac quality of life (KCCQ) in all five evaluable patients Cardiac biopsies were obtained from a patient at week 6 and 24 and showed robust and durable delivery, transgene expression and clearance of Gb3 substrate in cardiomyocytes No new treatment-related adverse events > Grade 1 since last interim update and through 12-33 months; previously reported cases of atypical hemolytic uremic syndrome (aHUS) (n=3) fully resolved FDA submission of data from non-human primate (NHP) study evaluating 4D-310 with rituximab/sirolimus (R/S) immunosuppressive regimen in Q2 2024 Efficient delivery, transgene expression and clinical activity now demonstrated in the clinic with three proprietary vectors generated from 4DMT’s Therapeutic Vector Evolution platform EMERYVILLE, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced updated interim safety and efficacy data on six adults with Fabry disease cardiomyopathy treated with a single intravenous (IV) infusion of 4D-310 (1E13 vg/kg) with follow-up of 12-33 months overall, including 12-24 month follow-up data on cardiac contractility, exercise capacity, quality of life and cardiac biopsy data. The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024. “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important cardiac endpoints including cardiac function, exercise capacity and quality of life,” said Robert Kim, M.D., Chief Medical Officer of 4DMT. “Current therapies do not adequately address Fabry-related cardiovascular manifestations, and cardiovascular disease is the most common cause of death in these patients. 4D-310 continues to be well tolerated, with the previously reported cases of aHUS having fully resolved and no new drug-related adverse events over Grade 1 reported.” “These promising clinical results continue to validate our Therapeutic Vector Evolution platform, as well as the potential of the C102 vector for targeted IV delivery to cardiomyocytes. Cardiac biopsies also showed robust and durable delivery and transgene expression from 4D-310, and remarkably, a reduction in Gb3 substrate in cardiomyocytes,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “No approved therapy has been shown to definitively clear accumulated Gb3 from cardiomyocytes in patients with Fabry disease, which speaks to 4D-310’s highly differentiated profile. We remain on-track to submit results from the non-clinical study to evaluate 4D-310 in NHPs with the R/S immunosuppressive regimen to the FDA in Q2 2024 to address the clinical hold.” INGLAXA Phase 1/2 Clinical Trial Design & Enrollment Dose escalation and dose expansion trial assessing a single IV infusion of 4D-310 in a broad and diverse Fabry disease patient population Enrolled six patients, each treated with 1E13 vg/kg of 4D-310 and a prophylactic corticosteroid immunosuppressive regimen Protocol amended to change immunosuppressive regimen to rituximab and sirolimus (enrollment on this amendment pending) Cardiac biopsies allowed at week 6 and 26 in the INGLAXA-2 trial (to date, one patient had biopsies performed) INGLAXA Phase 1/2 Interim Data Summary (Data Cutoff: December 5, 2023) Safety and Tolerability Previously reported cases of aHUS (n=3) have fully resolved No clinically significant cardiac or liver toxicities No new 4D-310–related adverse events > Grade 1 since the last interim update in February 2023 Echocardiography Contractility Assessments (Global Longitudinal Strain, GLS) Improved GLS in all five evaluable patients by month 12 Two patients who reached 24 months of follow-up both showed clinically meaningful improvement with change from baseline exceeding the minimal detectable difference of -1.5% (-2.8 and -2.9%, respectively) Cardiopulmonary Exercise Testing (CPET) Peak VO2 Assessments 3 of 4 evaluable patients demonstrated clinically meaningful improvement by CPET peak VO2 with change from baseline exceeding the minimal clinically important difference (MCID) of +1.5 at month 12 (+1.8, +2.0 & +7.0) One patient who reached 24 months of follow up continued to show meaningful improvement with change from baseline exceeding the MCID of +1.5 (+7.8) Cardiac Quality of Life Assessments (Kansas City Cardiomyopathy Questionnaire) Two patients had low baseline scores <90 (scale 0-100); both patients had clinically meaningful improvements in QOL (exceeding the MCID of +5) One patient had 12 months follow-up: Clinical & Overall Summary Scores improved by +5.7 and +11.7, respectively One patient had 24 months follow-up: Clinical & Overall Summary Scores improved by +12.5 and +8.3, respectively Three patients had baseline values >90 (92.7, 100 & 100); all remained stable through 12-24 months (+1.1 to -1.6). Cardiac Biopsy Assessments of Transgene Expression and Substrate Clearance Cardiac biopsies at week 6 and 26 from one patient showed robust & durable 4D-310–mediated transgene expression in cardiomyocytes All samples positive for transgene RNA (ISH) & AGA protein (IHC) At week 26, mean Gb3 inclusion body volume per cardiomyocyte was reduced 15% from week 6 and 61% vs. historical sample collected approximately 7 years prior to enrollment and analyzed independently by investigator Detailed results can be found in the Scientific Posters and Publications section of our website here. Next Steps for Development of 4D-310 FDA submission of data from the NHP study evaluating the safety and biodistribution of IV 4D-310 with the R/S immunosuppressive regimen compared to the prior prednisone regimen expected in Q2 2024 About 4D-310 and Fabry Disease Cardiomyopathy 4D-310 utilizes the cardiac targeted and evolved C102 vector to efficiently deliver a functional copy of the GLA gene (encodes for AGA enzyme) to the heart after a single low dose IV administration. The product candidate is designed to generate high local levels of AGA directly within heart tissue, as well as other affected organs, with the goal of reversing the cardiomyopathy in Fabry patients. Cardiomyopathy is the leading cause of death in the Fabry disease population. Affecting more than 50,000 people in the United States and European Union, Fabry disease is a genetic disorder of the GLA gene that results in the body’s inability to produce AGA, causing accumulation of the substrate globotriaosylceramide (Gb3) in critical organs, including the heart, kidney, and blood vessels. Cardiomyopathy is the leading cause of death in the Fabry disease patient population. Such substrate accumulation can lead to life-threatening hypertrophic cardiomyopathy, heart failure, arrhythmias, various degrees of kidney dysfunction and cerebrovascular stroke. Significant unmet medical needs remain for these patients despite enzyme replacement therapy (ERT), the current standard of care. ERT requires biweekly intravenous dosing which markedly decreases patients’ quality of life. In addition, while benefit has been demonstrated in the kidney, ERT has not been shown to clearly benefit the heart. About 4DMT 4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing five clinical-stage and two preclinical product candidates, each tailored to address rare and large market diseases in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at and follow us on LinkedIn. Forward Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits of 4DMT’s product candidates, as well as the plans, announcements and related timing for the clinical development of our clinical and preclinical product candidates, and related timing for the clinical development of 4D-310. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward looking statements. Contacts: Media: Katherine Smith Inizio Evoke Comms Katherine.Smith@inizioevoke.com Investors: Julian Pei Head of Investor Relations and Corporate Communications Investor.Relations@4DMT.com 267-644-5097

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH. Ruxoprubart is an Anti-Bb antibody that selectively blocks the alternative pathway while maintaining the intactness of the classical pathway required for clearing infection. The drug has completed phase I trial in 40 healthy volunteers with a clean safety pro was well-tolerated across all doses. Total alternative pathway inhibition was achieved, with the duration of AP inhibition showing a dose-dependent response. Ruxoprubart is currently undergoing a Phase II trial in treatment naïve PNH patients, showing promising results. Ruxoprubart is potentially useful in other clinical indications, where the complement alternative pathway is dysregulated. The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV). The ODD designation provides Ruxoprubart with a range of benefits, including financial incentives, regulatory support, and extended market exclusivity, ensuring that this groundbreaking treatment can reach those who need it. CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a Phase II trial in treatment-naïve PNH patients, which aims to evaluate the safety and efficacy of the drug and bringing us one step closer to providing an effective and innovative therapy for PNH patients in USA and around the world. "We are delighted by the FDA's decision to grant orphan drug designation to Ruxoprubart, underscoring the pressing need for innovative therapeutic solutions for patients with PNH," says Robert Bard, VP Regulatory Affairs. This ODD approval marks a significant advancement in the therapeutic landscape with Ruxoprubart 's unique ability to selectively block the alternative pathway (AP) while preserving the classical pathway required for clearing infections in PNH patients. Orphan Drug Designation (ODD) The ODD from the US FDA is a special designation granted to facilitate the development and assessment of prospective new medications. The FDA's Office of Orphan Products Development bestows orphan status upon drugs in development for the treatment, diagnosis, or prevention of rare diseases or conditions impacting fewer than 200,000 people in the US. This Designation aims to offer various advantages to drug developers in order to bolster the creation of innovative medications. These advantages include the potential for market exclusivity lasting seven years upon FDA approval, eligibility for tax credits related to qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance, and the potential qualification for expedited development programs. Ruxoprubart (NM8074) Distinguishing itself from Iptacopan (Fabhalta) by exhibiting no affinity for Factor B, our lead candidate Ruxoprubart selectively binds to protein Bb of the alternative pathway. This humanized anti-Bb monoclonal antibody serves as a potent blocker of the Alternative Pathway. Having successfully completed the phase I trial in healthy volunteers with expected outcomes, Ruxoprubart has progressed to a phase II trial, focusing on treatment-naïve PNH patients in a multi-dose regimen. Notably, the Anti-Bb molecule marks a pioneering biologic undergoing testing in treatment-naïve PNH patients. FDA has also approved the initiation of Phase II trials for multiple indications, including C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and ANCA vasculitis (AAV) with trials to be conducted in the United States. Overseas regulatory approvals cover trials for PNH for the adult population and aHUS, extending to adult and pediatric population. Paroxysmal Nocturnal Hemoglobinuria (PNH) The dysregulation of the Alternative Pathway (AP) is widely recognized as a pivotal factor influencing the course of PNH disease. Classified as an orphan disease, current PNH treatment options remain limited and imperfect. Current sanctioned treatments either hinder infection clearance or induce hyperlipidemia. PNH is characterized by low PNH-RBC clone size, hemolysis, hemoglobinuria, low hemoglobin levels, elevated LDH, and numerous other symptoms that can lead to chronic issues, including premature death if left untreated. Chronic symptoms and multiple organ damage are also prevalent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs exist for PNH treatment with potential symptom management, they are not without significant side effects. The imperative for additional treatment options for PNH, both effective and well-tolerated, remains recognized, with the potential to improve the disease outcome. About NovelMed NovelMed is dedicated to pioneering novel biologics for diverse complement-mediated diseases. As the first to conceptualize and validate an anti-Bb antibody for chronic complement-mediated and complement-associated disorders, NovelMed advances its leading product candidate through clinical trials, primarily focusing on hemolytic and renal disorders. "Rooted in a commitment to creativity and compassion, NovelMed aims to deliver transformative therapies reshaping the treatment landscape for rare disease patients," says Dr. Rekha Bansal, Chief Executive Officer. Positioned as a leader in developing Alternative Pathway (AP)-targeted therapies, NovelMed is dedicated to tackling debilitating diseases in hematology, ophthalmology, nephrology, dermatology, and neurology. Seeking Partnership to Advance the Development of Ruxoprubart for Rare Diseases PNH, a hemolytic disorder resulting in a low PNH-RBC clone size, increased RBC transfusions, elevated LDH values, and decreased hemoglobin values, remains an orphan disease with limited treatment options. NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel Ruxoprubart through further development and approval across multiple rare disease indications in various countries. For additional information, please visit . Contact: Ya Gao BD@novelmed.com (216) 440-2696