Zikani Therapeutics, Inc.

WATERTOWN, Mass., Jan. 24, 2024 /PRNewswire/ -- ZWI Therapeutics, Inc. (ZWI), a biotechnology company focused on the development of protein therapeutics using proprietary new polymers, announces a Series A financing, the appointment of pharmaceutical industry veterans Drs. Larry Miller and Ron Cohen as Independent Directors to the company's Board of Directors, and Dr. Ed Mascioli as Chief Executive Officer and Board Director. "After closing our Series A $10 million financing from Co-win Ventures and Sherpa Healthcare Partners, engaging such high caliber executives in our company is a testimony to the confidence successful entrepreneurs see in the ZWI technology. Ed Mascioli's significant experience leading multiple biotech companies is important in our Company's growth" said Dr. Shaoyi Jiang, the Robert Langer '70 Family and Friends Professor at Cornell University, ZWI Board member, and co-founder. "As board members, Ron and Larry collectively bring decades of successful pharmaceutical industry experience to ZWI. We will utilize their extensive expertise as ZWI advances its pipeline of wholly-owned and differentiated candidates that aim to tackle immunogenicity of protein therapeutics - a major challenge in biologics development" said Dr. Mascioli. ZWI created new polymers, based on polycarboxybetaine, with several advantages over polyethylene glycol (PEG), including reduced immunogenicity and preserved pharmacokinetics, and can be used on many protein therapeutics, lipid nanoparticles, and hydrogels. These novel polymers are supported by dozens of issued and pending patents and over 300 publications. ZWI's lead program, ZWI-001 (uricase-polycarboxybetaine conjugate), has demonstrated preclinical proof of concept and is currently in development to treat hyperuricemia in severe gout. ZWI has gained meaningful guidance on CMC, non-clinical, and clinical plans from the FDA via an INTERACT meeting and is progressing toward filing an IND. About ZWI Therapeutics, Inc. ZWI, founded in 2020 by Drs. Bob Langer and Shaoyi Jiang, is a biotechnology company using a novel, patented polymer, polycarboxybetaine, to develop protein-polymer conjugates with potentially superior safety and efficacy over currently marketed therapeutics conjugated with polyethylene glycol (PEG). I n preclinical animal models, polycarboxybetaine conjugates have demonstrated consistently low or no immunogenicity and excellent pharmacokinetics compared to PEG conjugates. These attributes are expected to translate to enhanced clinical durability and safety compared to pegylated drugs. The ZWI platform polymer technology is also broadly useful in the development of nanoparticles and hydrogels, the latter especially for cellular therapies. ZWI will out-license its technology to pharmaceutical companies for specific development programs. ZWI is headquartered in Waltham, MA, USA. For more information about ZWI platforms and pipeline, please visit , follow up on LinkedIn or contact us at info@zwitx.com. About Gout Gout is diagnosed in 3.9% of the U.S. population- up to 13 million patients - and is the most common form of inflammatory arthritis. Gout is a serious, lifelong disease that needs ongoing treatment and requires changes in lifestyle habits to achieve control. If poorly treated, advanced gout can lead to the same level of work loss, physical disability, and diminished quality of life as is seen in advanced rheumatoid arthritis. Gouty arthritis is caused by an accumulation of uric acid crystals in joints that causes severe pain, disfigurement and mobility restrictions. Uric acid crystals can also deposit in other tissues throughout the body, such as the kidney, which can lead to kidney stones, the skin (deposits called tophi) and even in vasculature walls. Beyond pain and disability, deposition of uric acid crystals triggers the inflammatory cascade and contributes to well-known comorbidities, including thromboses, stroke, and major adverse cardiovascular events (MACE). Control of uric acid levels is the key to controlling gout and the inflammatory sequelae. Approximately 5-10% of patients, estimated at 250,000-400,000 people in the US, treated with oral urate lowering therapies do not respond adequately, and are termed refractory gout. Only one product is currently approved to treat refractory gout: KRYSTEXXA® (pegloticase, pegylated uricase). Refractory gout is an area of significant unmet medical need in a large market. Ron Cohen, M.D. is President, CEO, and founder of Acorda Therapeutics, Inc., a public biotechnology company developing therapies for disorders of the nervous system. Dr. Cohen previously was an officer of Advanced Tissue Sciences, Inc., a biotechnology company engaged in the growth of human organ tissues for transplantation uses. Dr. Cohen serves on the Boards of ZWI Therapeutics and of the Biotechnology Innovation Organization (BIO). He is past Chair of the Board of BIO's Emerging Companies Section, and of the New York Biotechnology Association (NYBA). He is a recipient of the NY CEO Lifetime Achievement Award and the Ernst & Young Entrepreneur of the Year Award for the New York Metropolitan Region. He was named Neuroinvestment's CEO of the Year and is an inductee of the National Spinal Cord Injury Association's "Spinal Cord Injury Hall of Fame". Dr. Cohen has been recognized by the NYBA as the "The Cures Start Here" Business Leader of the Year, by the Westchester County Association as its Leadership Award recipient, by Life Science Leader Magazine as True Life Science Leader of the Year, by PR Week/MM&M as one of the Health Influencer 50, by Scientific American as one of the 100 Most Influential People in Biotech, and by PharmaVOICE Magazine as one of the 100 Most Inspirational People in the Biopharmaceutical Industry. Dr. Cohen received his B.A. degree from Princeton University, and his M.D. from the Columbia College of Physicians & Surgeons. He completed a residency in Internal Medicine at the University of Virginia Medical Center, and is Board Certified in Internal Medicine. Larry Miller, M.D. currently serves as the Chief Executive Officer and Board Chair of Apnimed, a private biotechnology company which he co-founded in 2017 to develop therapeutics to treat obstructive sleep apnea. Since 1991, Dr. Miller has founded or co-founded 10 life sciences/healthcare companies including Macrolide Pharmaceuticals, HPR, Avicenna, PharMetrics, ImpactRx, Serenex, Optio Research, Tetraphase, and Activate, and has served as CEO or Board member or both in each case. Two of these companies completed public offerings and seven were acquired. Dr. Miller also co-founded the Mediphase funds, where he led investments in both private and public life sciences/healthcare companies, having served previously in a similar role at Hambrecht & Quist Capital Management. Dr. Miller received an A.B. from Harvard College, an M.D. from Harvard Medical School, and completed clinical training in internal medicine and pulmonary disease at the Massachusetts General Hospital. He is Board certified in Internal Medicine, Pulmonary Disease, and Clinical Pharmacology. Ed Mascioli, M.D. has been CEO of multiple private biotech companies, successfully selling two. Prior to his role as CEO, Dr Mascioli was a Venture Partner at SV Life Sciences. Previously, Dr. Mascioli was at Pfizer where he established a new unit in rare, orphan, and genetic diseases with assets aggregated from both external to and within Pfizer in hemophilia, muscular dystrophy, hereditary emphysema, cystic fibrosis, and mucopolysaccharidoses. Dr. Mascioli was also at MPM Capital, a leading healthcare venture capital firm with over $2.5B assets under management, where he led 10 financings totaling $299M, including new equity investments, bridge loans, and equity re-financings. Dr. Mascioli participated in the raising of two venture funds totaling $1.4B. As the investor representative, Dr. Mascioli has served as a Director on the Boards of Arriva Pharmaceuticals, Elixir Pharmaceuticals, Mindset BioPharmaceuticals, and Oxagen, Ltd., and as an Observer on the Boards of CGI Pharmaceuticals, Radius Health, and Peptimmune. Dr. Mascioli started his career on the faculty of Harvard Medical School as an Assistant Professor of Medicine and on the staff of the Beth Israel Deaconess Hospital in Boston. Dr. Mascioli holds an M.S. from MIT, an M.D. from the University of Massachusetts Medical School, and a B.A., cum laude, from Brandeis University. Co-win Ventures was founded in 2009 and is a top-tier China headquartered venture capital firm with a global vision to invest in high-tech and healthcare industries. With over USD 950 million under management to date, it has invested in more than 130 private companies with about half of them already exited through IPOs and M&A. Co-win has won multiple national and world-renowned venture capital awards. With professional teams based in China and the US, Co-Win becomes seeks to become a long-term partner for portfolio start-ups, assisting in building their companies, integrating industrial resources, and navigating with strategic insights. Sherpa Healthcare Partners was founded in 2018 by financiers who previously worked together in one of China's leading venture capital firms where they built and led its professional medical investment team, including setting up and leading investments in a dedicated healthcare fund. The founders have been teamed together in close co-operation, built on shared values, mutual trust, and complementary capabilities. Sherpa has now become an influential healthcare VC firm in the industry.

LEXINGTON, Mass.--(BUSINESS WIRE)-- KSQ Therapeutics, a clinical-stage biotechnology company developing therapies to treat cancer and autoimmune diseases using its proprietary, integrated discovery CRISPRomics® platform, today announced the appointment of Mahesh Karande, President and CEO of Omega Therapeutics, to the company's board of directors. "Mahesh’s deep experience in company leading, product commercialization, fundraising, and deal-making are great additions to the skillset we've assembled on our board," said Qasim Rizvi, Chief Executive Officer of KSQ Therapeutics. "His insights and guidance will be invaluable as we continue to advance KSQ-4279 through clinical studies and work towards bringing our engineered TIL (eTIL™) cell therapy programs into the clinic." "KSQ's platform and science are impressive and have yielded multiple exciting and novel targets that provide significant opportunities to help patients with various cancers and solid tumors," said Karande. "I look forward to working closely with the company to maximize the potential of its programs, including KSQ-4279 and the eTIL cell therapy franchise, and I'm excited to see where the CRISPRomics platform can take us as we move into new and exciting areas, including NK cells, Tregs and myeloid cells." Mahesh is the President and CEO of Omega Therapeutics (Nasdaq: OMGA), a clinical-stage biotechnology company pioneering Omega Epigenomic Controllers, a new class of programmable mRNA medicines. At Omega, he has led the company’s development of their Epigenomic Programming platform and the translation of novel science into a broad pipeline in oncology, regenerative medicine, immunology, inflammatory conditions, and important monogenic conditions. Before Omega, he was CEO of Macrolide Pharma, where he re-assessed the company's core strategy and optimized a path forward for the highest shareholder return. Mahesh previously held numerous leadership roles at Novartis Pharmaceuticals, most recently as a corporate-level Vice President and US franchise Head leading the oncology solid tumors business. He also served as President of Africa, President of Egypt and Regional Vice President of strategy and business development for emerging markets, where he oversaw the execution of more than 50 partnership deals. He has led more than 10 product launches in the U.S. and across global markets. Prior to entering the pharmaceutical industry, Mahesh worked for the global management consulting firm McKinsey. Mahesh has an M.B.A. from the Wharton School, University of Pennsylvania. He is also a graduate of the Georgia Institute of Technology, where he completed his M.S. in engineering, and the University of Bombay, where he completed undergraduate studies in engineering. About KSQ Therapeutics KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer and autoimmune disease across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ's proprietary CRISPRomics® discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. For more information, please visit the company's website at and follow @ksq_tx on Twitter.

Topline data from cystic fibrosis (CF) Phase 2 expansion treatment arms evaluating combination with ivacaftor expected by the end of the third quarter of 2022 On track to initiate a proof-of-concept clinical trial with ELX-02 in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022 On track to submit an Investigational New Drug (IND) application for the inhaled delivery of ELX-02 in the fourth quarter of 2022 On track to submit IND application for ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) patients with nonsense mutations cells in the fourth quarter of 2022 Announces results of ZKN-013 Dose Response Study in APCMin Model that confirmed the results from prior study which concluded in March 2022 and expect to submit an IND application for Familial Adenomatous Polyposis (FAP) in the second half of 2023 Cash and equivalents expected to be sufficient to fund operations into the fourth quarter of 2023 WATERTOWN, Mass., Aug. 15, 2022 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, reported its financial results for the three months ended June 30, 2022 and provided a business update. “We continue to make significant progress advancing our pipeline of novel products for patients with substantial unmet medical needs. With multiple potential value-creating events expected over the next twelve months, including topline data readouts for ELX-02 in both CF and Alport syndrome, we are approaching an exciting and eventful period for the company,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “As further evidence of the potential of our technology platform, today we are pleased to announce results of a repeat study of ZKN-013 in a validated model, which provides further confirmation for the potential of ZKN-013 to treat FAP,” said Sumit Aggarwal, President and CEO of Eloxx Pharmaceuticals. “Based on the continuing positive preclinical results, we now expect to submit an IND to the U.S. Food and Drug Administration in the fourth quarter of 2023 for ZKN-013 for FAP.” Second Quarter 2022 and Subsequent Highlights Class 1 Cystic Fibrosis Phase 2 clinical trials in CF patients, with expansion arms designed to evaluate the safety of ELX-02 and assess short-term biological activity in patients, remains ongoing. Topline results are expected before the end of the third quarter of 2022 once complete patient data sets are received.A head-to-head preclinical study of inhaled (nebulizer-based) delivery of the current subcutaneous formulation of ELX-02 compared to subcutaneous dosing in a CF rodent model has been completed with data analysis ongoing. Human exposure, modeled based on previous animal pharmacokinetic studies, suggests that the lung drug exposure with inhaled delivery of ELX-02 is at least 50-fold greater than with subcutaneous delivery. Higher drug exposure, achievable with inhaled delivery, has been associated with 3 to 6 fold higher activity in CF models. We expect to report topline results before the end of the third quarter of 2022 and remain on track to submit an IND application in the fourth quarter of 2022. Alport Syndrome Eloxx previously announced it has expanded its clinical development pipeline to include the potential treatment with ELX-02 of a subset of Alport Syndrome patients with nonsense mutations in the Collagen Type 4 genes, (COL4A3, COL4A4, and COL4A5). Alport syndrome is a rare genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities.Eloxx remains on track to initiate a proof-of-concept clinical trial in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022. Patients will be dosed for two months with a three month follow-up. Trial primary endpoints include safety while secondary endpoints are reduction in proteinuria and induction of COL4A5 protein expression in the kidney. Initial topline results are expected in the first half of 2023. Recessive Dystrophic Epidermolysis Bullosa and Junctional Epidermolysis Bullosa (JEB) Eloxx continues to expect to file an IND application in the fourth quarter of 2022 and be ready to start a First in Human (FIH) Phase 1 study with ZKN-013 after recently completing 28-day non Good Laboratory Practice (GLP) animal studies. Familial Adenomatous Polyposis Eloxx today announced positive results from a repeat study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP. These results provide further confirmation of ZKN-013’s potential to treat FAP, following the previously reported single-dose data in the APCMin model. The APCMin mouse is a translationally validated model for drug development for FAP, a rare inherited disease, with no approved drug therapies, characterized by proliferation of colon polyps. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients. In the repeat study, APCMin mice received daily oral doses of ZKN-013 at 50, 25, 12.5 mg/kg body weight for 8 weeks.100% survival was achieved at the lowest dose of ZKN-013 tested.ZKN-013 significantly lessened anemic conditions, generally considered to be the major cause of animal death associated with APCMin model. Eloxx intends to file an IND application to the U.S. Food and Drug Administration for ZKN-013 for the treatment of FAP in the second half of 2023. Second Quarter 2022 Financial Results For the three months ended June 30, 2022, we incurred a net loss of $10.6 million, or $0.12 per share, which included $0.7 million in stock-based compensation. For the same period in the prior year, we incurred a net loss of $36.1 million, or $0.54 per share. Since the closing date of Eloxx’s acquisition of Zikani Therapeutics, Inc. in April 2021, the results of Zikani’s operations have been included in the Eloxx’ condensed consolidated financial statements. The second quarter of 2021 included a $22.7 million expense for in process research and development (R&D) related to the Zikani acquisition. Our R&D expenses were $7.7 million for the three months ended June 30, 2022, which includes $0.3 million in stock-based compensation. For the same period in the prior year, R&D expenses were $5.7 million, which included $0.1 million of stock-based compensation. The increase was primarily related to increases in expenses related to preclinical and development activities, the continued development of ELX-02 related primarily to Cystic Fibrosis Foundation funded activities, facility and overhead expenses and salaries and stock-based compensation. Our general and administrative (G&A) expenses were $2.6 million for the three months ended June 30, 2022, which includes $0.4 million in stock-based compensation. For the same period in the prior year, G&A expenses were $7.4 million, which included $3.9 million of stock-based compensation. The decrease was primarily related to decreases in salaries and other personnel related costs, stock-based compensation and facility and overhead expenses. As of June 30, 2022, we had unrestricted cash and cash equivalents of $30.0 million. The Hercules Term Loan Agreement contains customary affirmative and negative covenants, which among others require the Company to maintain at all times a minimum qualified cash balance. As of June 30, 2022, the Company was in compliance with all debt covenants. If our lender does not accelerate our debt upon our failure to comply with the minimum cash covenant, we expect our current cash position will be sufficient to fund our operations into the fourth quarter of 2023. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM 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. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders. For more information, please visit www.eloxxpharma.com. 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 present and historical facts contained in this press release, including without limitation, statements regarding our future financial results, our cash runway, the expected timing of trials and results from clinical studies of our product candidates and the potential of our product candidate to treat nonsense mutations 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 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 preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; 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 financial in the future through product licensing, public or private equity or debt financing or otherwise; 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 quarter ended June 30, 2022, 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 www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financial-information/sec-filings. 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 InvestorsJohn Woolfordjohn.woolford@westwicke.com443.213.0506 MediaLaureen Cassidylaureen@outcomescg.com ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands, except share and per share data) June 30,2022 December 31,2021 ASSETS Current assets: Cash and cash equivalents $30,048 $42,268 Restricted cash 263 299 Prepaid expenses and other current assets 1,281 913 Total current assets 31,592 43,480 Property and equipment, net 190 216 Operating lease right-of-use assets 1,159 1,443 Total assets $ 32,941 $ 45,139 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $1,934 $1,379 Accrued expenses 4,870 4,196 Current portion of long-term debt 1,125 - Advances from collaboration partners 10,723 3,723 Derivative liabilities 166 - Current portion of operating lease liabilities 686 657 Total current liabilities 19,504 9,955 Long-term debt, net of current portion 11,129 11,996 Operating lease liabilities 496 804 Total liabilities 31,129 22,755 Total stockholders’ equity 1,812 22,384 Total liabilities and stockholders' equity $ 32,941 $ 45,139 ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share data) Three Months Ended Six Months Ended June 30,June 30, 2022 2021 2022 2021 Operating expenses: Research and development $7,651 $5,704 $15,550 $9,777 General and administrative 2,645 7,355 5,699 11,696 In process research and development — 22,670 — 22,670 Total operating expenses 10,296 35,729 21,249 44,143 Loss from operations (10,296) (35,729) (21,249) (44,143)Other expense, net 322 329 989 609 Net loss $(10,618) $(36,058) $(22,238) $(44,752) Net loss per share, basic and diluted $(0.12) $(0.54) $(0.26) $(0.84)Weighted average number of shares of common stock used in computing net loss per share, basic and diluted 86,654,120 66,389,865 86,652,587 53,357,401 Source: Eloxx Pharmaceuticals