Eloxx Pharmaceuticals, Inc.

First two subjects dosed in Phase 1 clinical trial of ZKN-013; ZKN-013 is being developed for the potential treatment of rare dermatological and other diseases associated with nonsense mutations Positive written FDA feedback and guidance from a pre-Investigational New Drug Application (PIND) meeting provides pathway to IND application submission to initiate a Phase 2 clinical trial in the US with ELX-02 in patients with nonsense mutation alport syndrome (NMAS) Secured binding commitment for additional $3.2 million financing to advance our clinical programs July 11, 2024 -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases with nonsense mutations, today provided significant pipeline and financing updates. “Dosing of the first healthy volunteers in the Phase 1 trial for ZKN-013, our lead TURBO-ZM™ is a meaningful milestone for Eloxx, as it further demonstrates our ability to advance novel candidates targeting rare diseases into clinical trials,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “We were also very pleased with the outcome of our pre-IND meeting with the FDA, which has increased our confidence in the potential of ELX-02 to have a meaningful impact on the treatment of Alport syndrome.” The first two subjects have been dosed in the ongoing Phase 1 healthy volunteer single-ascending dose study of ZKN-013. ZKN-013 is designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP). This Phase 1 study evaluates the safety and pharmacokinetics in healthy volunteers. ZKN-013 has been exclusively licensed to Almirall, S.A. (BME:ALM), who has global rights to develop and commercialize ZKN-013. Under the terms of the license agreement, Eloxx is eligible for additional development, regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales. Eloxx has received FDA’s final minutes from the hybrid in-person and video conference PIND meeting the company held with the Office of Cardiology, Hematology, Endocrinology, and Nephrology - Division of Cardiology and Nephrology. We believe that the FDA’s positive feedback and guidance provides a pathway to an IND application submission to initiate a validating Phase 2 clinical trial in the US with ELX-02 in NMAS patients. As part of the meeting, the FDA reviewed the clinical data from the three patients from the Phase 2 trial conducted in the UK and provided feedback on the proposed clinical trial design. “We are grateful to the FDA for their engagement and support for the clinical program to find an appropriate development path for ELX-02 to treat NMAS patients,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “Based on the FDA feedback and guidance, Eloxx plans to engage with the FDA for a potential IND application submission to the FDA to initiate a larger clinical trial in the US to validate the results of its previously reported Phase 2 study. In April 2024, the FDA Office of Orphan Products Development (OOPD) granted Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome. Eloxx recently secured financing with the lenders under its Loan and Security Agreement, dated as of September 30, 2021 (as amended, amended and restated, supplemented, or otherwise modified from time to time) for an aggregate of $3.175 million financing of which $500,000 has been funded in advance. This financing is in addition to the $288,00 financing closed in May and both financings have identical terms and conditions. This additional financing is intended to support the advancement of our clinical programs and provide funding for general corporate purposes. We expect to receive the remainder $2.675 million of bridge loan financing on or about July 12, 2024. Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com. The content above comes from the network. if any infringement, please contact us to modify.

ELX-02 Granted Orphan Drug Designation (ODD) from U.S. Food and Drug Administration (FDA) for Treatment of Alport Syndrome Pre-Investigational New Drug (IND) Meeting Requested with U.S. FDA for ELX-02 to Discuss Planned next Study In Nonsense Mutation Alport Syndrome Signed Global Licensing Partnership for ZKN-013 with Almirall Pharmaceuticals with $3M upfront and up to $470M in milestones and Tiered Royalties on Global Sales New Paper Published on Autosomal Dominant Polycystic Kidney Disease (ADPKD) shows that ELX-02 treatment prevents cyst formation in diseased PKD organoids with nonsense mutations and a single healthy gene copy WATERTOWN, Mass., April 16, 2024 (GLOBE NEWSWIRE) --Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today provided program updates for ELX-02 and ZKN-013, including Orphan Drug Designation (ODD) for ELX-02. Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS). Additionally, with the global licensing partnership with Almirall announced in March, the company has begun the development of ZKN-013 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDDEB) and Familial Adenomatous Polyposis (FAP) patients with nonsense mutations. The publication of a recent paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD), confirms the potential of ELX-02 for the treatment of all rare genetic kidney diseases with nonsense mutations in the disease causing genes. “The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “The designation further strengthens our belief that ELX-02 has the potential to be transformational in supporting the ultra-rare subset of Alport Syndrome patient population with nonsense mutations. We look forward to engaging the FDA on initiating the larger clinical trial to support the potential for regulatory marketing approval of ELX-02 in this patient population.” ELX-02: Regulatory and Clinical Updates for Alport Syndrome Nonsense Mutation Program ELX-02 Granted ODD from U.S. FDA: In April, the FDA Office of Orphan Products Development (OOPD) granted ODD for ELX-02 for the treatment of Alport Syndrome. The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023. FDA ODD is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States with a reasonable clinical or preclinical hypothesis for efficacy in the target population. Requested a Pre-Investigational New Drug (IND) meeting with U.S. FDA: The request for a pre-IND meeting formally initiates communications with the FDA regarding development of ELX-02 for the treatment of NMAS. The purpose of the pre-IND meeting will be to request feedback on the Eloxx’s planned clinical trial in nonsense mutation Alport syndrome patients. Presented Clinical Data Updates at Alport Workshop Meeting in March: Prof. Daniel Gale presented updated results from the Proof-of Concept Study in 3 NMAS patients treated with ELX-02 Meaningful reduction in Podocyte Foot Process Effacement after treatment measured as an average 60% increase in Filtration Slit Density in post treatment kidney biopsies in all 3 patients. Results confirm the expression of functional collagen 4 protein in-line with the mechanism of protein induction of ELX-02. Changes in UPCR noted to be consistent with biopsy results with reduction or stabilization of proteinuria during or up to 2 months post completion of dosing. ZKN-013: Update on Global Licensing Partnership with Almirall In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations. Received $3 million in upfront payment Additional payments of ELX-02 of up to $470 million and tiered royalties on global sales: Eloxx shall be eligible for additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales Initiated Phase 1 Single Ascending Dose (SAD) study: Initiated Phase 1 SAD study in Australia with initial results expected in 2nd half of 2024 Recently Published Paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD) may Have Other Rare Kidney Disease Applications A paper was published in April 2024, on ADPKD titled Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease, authored by Courtney E. Vishy, Chardai Thomas, Thomas Vincent, Daniel K. Crawford, Matthew M. Goddeeris, and Benjamin S. Freedman. Freedman and colleagues engineered base-edited human iPS cells to resolve the functional genetics of human PKD. They discovered that cyst formation in diseased organoids could be prevented with a single healthy gene copy. This insight enables the demonstration of both genetic and pharmacological therapeutic approaches for clinically relevant nonsense mutations and may have the potential to support other rare kidney diseases. About Nonsense Mutation Alport Syndrome Nonsense Mutation Alport syndrome (NMAS) is an ultra-rare Type IV Collagenopathy characterized by nonsense mutations in the genes (COL4A3, COL4A4, and COL4A5) that result in a less than full length (truncated) Type 4 Collagen. This disorder mostly affects children with a median age at diagnosis of 9 to 20 years. It is characterized by rapid and progressive damage to the kidneys, ear, and eyes, starting with worsening of kidney morphology to proteinuria and finally kidney failure, hearing loss and eye abnormalities. 90% NMAS patients progress to kidney failure and hearing loss before the age of 30. There are no approved therapies. It is estimated that there are approximately 11,000 NMAS patients in the US and >20,000 patients in US, Europe, Japan and China. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit . Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes 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, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at and the “Financials & Filings” page of our website at 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. Contact Investors John Woolford john.woolford@westwicke.com 443.213.0506 Media Laureen Cassidy laureen@outcomescg.com Source: Eloxx Pharmaceuticals

Group includes leading experts in multiple disciplines including immunology, liver diseases, protein engineering, antibody drug conjugates and platform innovation BRIDGEWATER, NJ / ACCESSWIRE / April 15, 2024 / Tharimmune, Inc. (Nasdaq:THAR) ("Tharimmune" or the "Company"), a clinical-stage biotechnology company developing a portfolio of therapeutic candidates in inflammation & immunology announced today the formation of a new Scientific Advisory Board (SAB) that includes a group of leading experts in fields spanning immunology, liver disease clinical trials, protein engineering, antibody drug conjugates as well as Knob domains, the smallest antibody like fragments. "Tharimmune's mission is to advance immunology and oncology treatments for people suffering from high unmet need conditions and help them look forward to a better tomorrow," said Randy Milby, Chief Executive Officer of Tharimmune. "We are thrilled to have this venerated group of advisors as their valued feedback constructs a technical roadmap to push our programs ultimately to benefit patients." Tharimmune's lead clinical-stage asset, TH104, is designed as an ideal product candidate for multiple liver-related and other pruritogenic inflammatory conditions, by avoiding the first-pass metabolism usually in traditional oral formulations and designed for studying cholestatic conditions. The SAB's collaboration will drive our clinical-stage asset, TH104 in patients with moderate-to-severe chronic pruritis associated with primary biliary cholangitis (PBC) with a planned Phase 2 study in 2024 and help identify future areas of interest and development for the novel platform and includes: Andreas Kremer, MD, PhD is Clinician Scientist and Professor of Hepatology in Zurich and lead investigator for multiple clinical trials whose expertise spans autoimmune and cholestatic liver disease, liver fibrosis, hepato- and cholangiocellular carcinoma, the gut-liver-axis and has high expertise in pruritus and fatigue. Alan Bonder, MD, AGAF is Associate Professor of Medicine at Harvard University Medical School and Medical Director of Liver Transplant at Beth Israel Deaconess Medical Center in Boston; his expertise spans chronic liver diseases including cholestatic liver diseases including PBC and non-cholestatic liver diseases with expertise in patient care, clinical trials and pruritis associated with these conditions. The Company is also advancing novel antibodies in bovine animals or cows and derived from ultra-long complementary determining region 3 (CDR3) domains which potentially enable access to epitopes that had previously been hidden or highly inaccessible in traditional antibody development. With a much smaller size compared to traditional antibodies, combined with structural diversity, Knobs can bind to conformational, linear or discontinuous epitopes in "undruggable" areas of validated targets. They are derived from bovines, which unlike other species, express ultralong CDR-H3 regions forming an independently folding mini-domain, which protrudes far out from the surface of the antibody and is diverse in both its sequence and disulfide patterns. These atypical antigen binding sites of bovines potentially provide the ability to interact with different antigenic determinants capable of eliciting an immune response, particularly recessed or concave surfaces, compared to traditional antibodies. This conceivable "multi-specific" capacity of Knobs, are the basis of developing Tharimmune's early-stage pipeline including ADCs and may more efficiently target multiple cell surface portions compared to known or existing biologics. The SAB will collaborate with Tharimmune to develop next generation antibody, Knob domain and ADC platforms and includes: John Lambert, PhD is one of the leading experts in the field of ADC discovery and development. He joined ImmunoGen in 1987 and served as Chief Scientific Officer from 2008 until 2015 and was subsequently Distinguished Research Fellow at ImmunoGen. During his tenure in leadership roles, ImmunoGen invented the antibody-drug conjugate technology that resulted in KADCYLA® (Genentech/Roche) and ELAHERE® (ImmunoGen, now AbbVie), for treatment of HER2+ breast cancer and platinum-resistant ovarian cancers respectively. He is the author/co-author of over 125 peer-reviewed scientific publications. He is a fellow of the American Institute for Medical and Biological Engineering (AIMB) and an Honorary Professor of Queen's University, Belfast, Northern Ireland, UK. Vaughn Smider, MD, PhD is the founder and President of the Applied Biomedical Science Institute (ABS) and is an adjunct Professor at the Scripps Research Institute. Dr. Smider has multiple publications in antibody molecular biology, genetics, and structure, as well as in DNA repair and cancer biology. In these fields he also developed several groundbreaking technologies and was a founder and CSO of Fabrus, Taurus Biosciences (now part of Ligand's spin-out company, OmniAb Inc. (NASDAQ: OABI), and ABS Institute spin-out companies Minotaur Therapeutics and Enkefalos Biosciences and serves as an advisor to several biotechnology companies. The Company previously announced sufficient funding to extend its cash runway into early 2025 for a phase 2 clinical readout of its lead program, TH104. Tharimmune plans to advance both its clinical and non-clinical programs and will announce an R&D update in 2024. About Tharimmune Tharimmune, Inc. is a clinical-stage biotechnology company developing a portfolio of therapeutic candidates for inflammation and immunology. The Company's lead clinical-stage asset, TH104 is known to suppress chronic, debilitating pruritus or "uncontrollable itching" in PBC, a rare and orphan liver disease with no known cure. The Company's early-stage immunology pipeline includes novel multi-specific antibodies targeting unique epitopes with novel mechanisms of action against well-known, validated targets in multiple solid tumors, including PD-1, HER2 and HER3. Tharimmune has a license agreement with OmniAb, Inc. to access the company's antibody discovery technology platform against these and other specified targets. For more information please visit: . Forward Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, contained in this press release, including statements regarding Tharimmune's strategy, future operations, future financial position, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "depends," "estimate," "expect," "intend," "may," "ongoing," "plan," "potential," "predict," "project," "target," "should," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements. Factors that may cause such differences, include, but are not limited to, those discussed under Risk Factors set forth in our Annual Report on Form 10-K/A for the year ended December 31, 2022 and other periodic reports filed by the Company from time to time with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this release. Subsequent events and developments may cause the Company's views to change; however, the Company does not undertake and specifically disclaims any obligation to update or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by applicable law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release. Investor Relations Contact ir@tharimmune.com SOURCE: Tharimmune, Inc. View the original press release on accesswire.com