Laminopathies

MINNEAPOLIS, Feb. 20, 2024 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced the appointment of Eldon Mayer as Chief Commercial Officer. Mr. Mayer joins Celcuity with over 30 years of biopharmaceutical commercial experience in companies ranging from early-stage biotechs to full scale pharmaceutical companies across many therapeutic areas, including oncology. “Eldon is an exceptional leader with a proven track record of building commercial organizations from the ground up to support the launch of a biotech company’s first drug. His significant commercial experience at multiple biopharma companies, large and small, launching a variety of different drugs in different markets and scaling them, make him the ideal candidate to lead the potential commercial launch of Celcuity’s inaugural product, gedatolisib,” said Brian Sullivan, Celcuity’s Chief Executive Officer and co-founder. “We believe gedatolisib has a unique opportunity to become a transformational therapy in multiple tumor types, and Eldon has the right background to help gedatolisib realize this potential.” Prior to joining Celcuity, Mr. Mayer served as Executive Vice President and Chief Commercial Officer at Eiger Biopharmaceuticals where he led the commercial planning and execution of several pipeline assets including international partnerships as well as the global launch of Zokinvy® for progeria and progeroid laminopathies. Prior to Eiger, he was Executive Vice President and Chief Commercial Officer of Rigel Pharmaceuticals where he built and led the commercial organization and launched Tavalisse® (fostamatinib disodium hexahydrate) for chronic immune thrombocytopenia. Mr. Mayer has also served as Senior Vice President of Commercial Operations at Questcor Pharmaceuticals where he built a large commercial team that generated over $1 billion in annual sales of Acthar® Gel (repository corticotropin injection). Questcor was acquired by Mallinckrodt for $5.6 billion in 2014. Earlier in his career, he held positions of increasing responsibilities at companies including Schering-Plough, ALZA, Chiron, and Connetics, in functional areas including strategic planning, marketing, sales, market research and analytics, operations and finance. “I am excited to join Celcuity and work alongside driven, collaborative colleagues during this pivotal time for the company,” said Eldon Mayer. “With significant milestones for gedatolisib approaching, I look forward to leading the commercial and marketing efforts as we aim to bring this novel therapeutic option to the clinic.” About Celcuity Celcuity is a clinical-stage biotechnology company focused on development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is gedatolisib, a potent, pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are highly differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients. More detailed information about the VIKTORIA-1 study can be found at ClinicalTrials.gov. A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is expected to be initiated in the first quarter of 2024. The company's CELsignia companion diagnostic platform is uniquely able to analyze live patient tumor cells to identify new groups of cancer patients likely to benefit from already approved targeted therapies. Further information about Celcuity can be found at Celcuity.com. Follow us on LinkedIn and Twitter. Forward-Looking Statements This press release contains statements that constitute "forward-looking statements" including, but not limited to, our beliefs and expectations about the commercial potential of gedatolisib, the timeframe for clinical trials, achieving milestones to support commercialization, and Mr. Mayer’s ability to successfully lead Celcuity’s commercialization and marketing activities. Forward-looking statements are subject to numerous risks, uncertainties, and conditions, many of which are beyond our control, such as unexpected delays in clinical trials or unfavorable results; competition, market and regulatory issues that may affect our ability to commercialize gedatolisib; and those risks set forth in the Risk Factors section in Celcuity's Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 23, 2023. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Celcuity undertakes no obligation to update these statements for revisions or changes after the date of this press release, except as required by law. Contacts: Celcuity Inc. Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 ICR Westwicke Maria Yonkoski, maria.yonkoski@westwicke.com (203) 682-7167

The Progeria Research Foundation Marks Significant Achievement as Zokinvy™ (lonafarnib) Receives Japanese Approval for the Treatment of Progeria and Processing-Deficient Progeroid Laminopathies PEABODY, Mass., Jan. 20, 2024 /PRNewswire/ -- The Progeria Research Foundation (PRF) today announced a new milestone with the approval of Zokinvy™ (lonafarnib) by the Japanese Ministry of Health, Labor and Welfare (MHLW), for the treatment of Progeria and processing-deficient Progeroid Laminopathies (PL). Progeria is an ultra-rare, fatal pediatric "rapid-aging" disease. PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007. Eiger BioPharmaceuticals (Eiger) and PRF began a partnership in 2018 to bring this drug to market. Approval from the U.S. Food and Drug Administration (FDA) for Zokinvy was granted in November, 2020 and the European Medicines Administration (EMA) in July, 2022. Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by PRF and Boston Children's Hospital (BCH) demonstrated that in patients with Progeria, Zokinvy reduced the incidence of mortality by 72% and increased average survival time by an average of 30% (4.3 years). Without Zokinvy treatment, children with Progeria die of heart disease at an average age of 14.5 years. Prevalence data from PRF's International Patient Registry indicate an expected 6 children with Progeria are living in Japan today. "We're thrilled by this wonderful news," said Audrey Gordon, President and Executive Director of The Progeria Research Foundation. "On the heels of the 2020 FDA approval and the 2022 EMA approval for Zokinvy, now children and young adults with Progeria and PL in Japan will be able to access this life-extending, heart-strengthening treatment through the convenience of a prescription." "Since co-founding The Progeria Research Foundation in 1999, we have poured our hearts and souls into the most promising research toward treatments and the cure for every child with Progeria," said Leslie Gordon, MD, PhD, PRF Medical Director and Zokinvy Clinical Trial Investigator. "After more than a decade of Zokinvy research funded by PRF and co-conducted by PRF and BCH, Zokinvy's authorization in Japan is a win for the worldwide Progeria community. Thank you to the courageous children, their families, and our many expert Progeria research teams and pharmaceutical partners who made this key milestone possible." Over the course of its 24-year existence, PRF has made tremendous strides toward its mission to discover the cause, treatments and cure for Progeria. In partnership with the National Institutes of Health (NIH), PRF was the driving force behind the 2003 Progeria gene discovery. PRF holds bi-annual scientific workshops, funds research grants breaking new ground in areas such as RNA therapeutics and Genetic Base Editing, and funds clinical drug trials. PRF's core patient-focused programs include the Progeria International Patient Registry, Medical & Research Database, Cell & Tissue Bank, Diagnostics Testing Program, and a Clinical Care Handbook for families and physicians. "After conducting 16 years of Progeria clinical trials at Boston Children's Hospital, this milestone demonstrates the positive impact of a dedicated research team, rigorous testing, and the steadfast bravery of the wonderful children and their families," said Dr. Monica Kleinman, Principal Investigator for the Progeria clinical trials at Boston Children's Hospital. "Being a part of this achievement for these children is not only professionally rewarding but also a testament to the transformative potential of scientific advancement in Progeria research." About Progeria Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and PL are ultra-rare, multisystemic, premature aging diseases that accelerate mortality in young patients due to accumulation of cellular progerin in HGPS or an abnormal lamin A protein in PL. Progeria is caused by a genetic mutation in the LMNA ("lamin A") gene, and results in a disease-causing abnormal protein called progerin. There are approximately 400-450 children worldwide with Progeria. Thanks to PRF-funded research, we now know that progerin is produced in all of us as we age, but at a much lower rate than in children with Progeria. Due to this discovery of the biological connection between Progeria, heart disease and aging, finding the cure for one of the rarest diseases on earth could provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process, as well as help the entire aging population. About The Progeria Research Foundation The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria. Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, Acting Science Advisor to the President of the United States and former Director of the National Institutes of Health (NIH). PRF has funded and co-coordinated all Zokinvy-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children's Hospital, and supports scientists who conduct Progeria research worldwide. PRF's International Patient Registry includes over 370 children with Progeria in 70 countries. PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria and its aging-related conditions, including heart disease. The organization fills a void, putting these children and Progeria at the forefront of scientific efforts. For more information and to support PRF's mission, please visit . SOURCE Progeria Research Foundation

[18-January-2024] Clinical trial data demonstrated Zokinvy treatment extended life by an average of 4.3 years in children and young adults with Hutchinson-Gilford progeria Eiger to receive $500,000 approval milestone payment from AnGes Zokinvy approved in the U.S. (2020), 30 European countries (2022), and now Japan (2024) PALO ALTO, Calif., Jan. 18, 2024 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc, (Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, today announced that it and its partner AnGes, Inc. received marketing approval from the Ministry of Health, Labour and Welfare for Zokinvy (lonafarnib), a treatment for Hutchinson-Gilford progeria syndrome (HGPS) and processing deficient progeroid laminopathy (PDPL). "We and our partner, AnGes, are pleased that Zokinvy is now approved in Japan for patients living with progeria, an ultra-rare and fatal pediatric disease that can result in premature death," said David Apelian, MD, PhD, MBA, CEO of Eiger. "We would like to thank the Progeria Research Foundation for their continued support of the regulatory submission as well as the patients and their families." Collectively known as progeria, HGPS and PL are devastating ultra-rare and fatal pediatric diseases that cause dramatically accelerated aging and premature death. The main cause of death is heart attack or stroke due to severe hardening of the arteries.3,4 The approval was based on the positive results of two pivotal clinical trials demonstrating that Zokinvy, an oral disease-modifying agent which targets the cause of progeria, lowered the risk of death in children by 72% and extended life by an average of 4.3 years (p<0.0001) in children and young adults with HGPS.2 About Progeria Collectively known as progeria, Hutchinson-Gilford progeria syndrome and progeroid laminopathies are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. HGPS is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. Progeroid laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the LMNA and/or ZMPSTE24 genes yielding farnesylated proteins that are distinct from progerin.4,5 Without Zokinvy therapy, children with HGPS commonly die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), by an average age of 14.5 years. Disease manifestations include severe failure to thrive, scleroderma–like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes.3 About Zokinvy® (lonafarnib) Zokinvy is a first-in-class disease-modifying agent that blocks the accumulation of defective progerin and progerin-like proteins which leads to cellular instability and premature aging in children and young adults with progeria. Zokinvy has demonstrated a statistically significant survival benefit in children and young adults with HGPS.1,4 The most commonly reported adverse reactions were gastrointestinal (vomiting, diarrhea, nausea), and most were mild or moderate (Grade 1 or 2) in severity. Many progeria patients have received continuous Zokinvy therapy for more than 10 years.1,2 Zokinvy is FDA approved for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. For Important Safety Information and prescribing information for Zokinvy in the U.S., please visit Eiger and AnGes entered into an exclusive distribution agreement for the treatment of HGPS and PDPL indications, Zokinvy (Lonafarnib), in Japan on May 10, 2022. In March 2023, the Ministry of Health, Labour and Welfare designated Zokinvy as an orphan drug. About Eiger Eiger is a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases. Eiger's lead product candidate, avexitide, is a well characterized, first-in-class GLP-1 antagonist for the treatment of post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). Avexitide is the only drug in development for PBH with Breakthrough Therapy designation from the FDA. For additional information about Eiger and its clinical programs, please visit . Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts, including statements regarding our future financial condition, timing for and outcomes of clinical results, prospective products, preclinical and clinical pipelines, regulatory objectives, business strategy and plans and objectives for future operations, are forward-looking statements. Forward-looking statements are our current statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, the timing of our ongoing and planned clinical development; our capability to provide sufficient quantities of any of our products or product candidates for studies or to meet anticipated full-scale commercial demands; our ability to identify, pursue and enter into partnering opportunities for our virology assets; the sufficiency of our cash, cash equivalents and investments to fund our operations into the fourth quarter of 2024, including the scope and impact of any savings from our workforce reduction and cash conservation efforts; the revenue potential of avexitide in post-bariatric hypoglycemia and congenital hyperinsulinism; our ability to finance, independently or through collaborations, the continued advancement of our development pipeline; and the potential for success of any of our products or product candidates. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Eiger makes, including additional applicable risks and uncertainties described in the "Risk Factors" section in Eiger's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023 and Eiger's subsequent filings with the SEC. The forward-looking statements contained in this press release are based on information currently available to Eiger and speak only as of the date on which they are made. Eiger does not undertake and specifically disclaims any obligation to update any forward-looking statements, whether as a result of any new information, future events, changed circumstances or otherwise. Investors: Sylvia Wheeler Wheelhouse Life Science Advisors swheeler@wheelhouselsa.com Media: Aljanae Reynolds Wheelhouse Life Science Advisors areynolds@wheelhouselsa.com References: 1. Data on file, Eiger BioPharmaceuticals. 2. Summary of Product Characteristics, July 2022. 3. Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford Progeria Syndrome. 2003 Dec 12 [Updated 2019 Jan 17]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2020. 4. Gordon LB, Shappell H, Massaro J, et al. Association of lonafarnib treatment vs no treatment with mortality rate in patients with Hutchinson-Gilford progeria syndrome. JAMA. 2018;319(16):1687-1695. doi:10.1001/jama.2018.3264. 5. Marcelot A, Worman HJ, and Zinn-Justin S. Protein structural and mechanistic basis of progeroid laminopathies. FEBS Journal. 2021:288:2757-2772. Doi:10.111/febs.15526. Company Codes: NASDAQ-NMS:EIGR