Shire Plc

Denali plans to submit the drug, tividenofusp alfa, for accelerated approval early this year in the hopes that the treatment will become available to patients by the end of 2025 or early 2026.\n After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is climbing closer to its goal of accelerated approval by unveiling data showing the drug met its primary safety endpoints and reduced key biomarkers of the disease.Treated patients also experienced long-term improvements in their hearing and cognition, the company said in a Feb. 6 release. The phase 1/2 open-label trial enrolled 47 boys around the age of 5. Patients received weekly intravenous infusions of the drug, tividenofusp alfa (tivi), in either fixed or escalating doses. The primary analysis looked at data after 24 weeks of treatment, with the long-term follow-up planned for about five years. So far, some patients have more than three years of follow-up.Denali will present the phase 1/2 results at the 21st Annual WORLDSymposium conference in San Diego, California.The trial\'s primary endpoints are the rates of adverse events and infusion-related reactions, as well as other safety indicators, according to the presentation.Side effects were common, with 72% of patients experiencing mild to moderate treatment-emergent adverse events, according to the presentation. In the 24-week treatment period, the most common adverse events were infusion-related reactions, anemia, vomiting, fever, upper respiratory infection and rash.Though three patients experienced serious but manageable adverse events—and one patient discontinued treatment due in part to a reaction to the drug infusion—the trial met its safety expectations, the company said in the release.The company plans to submit the drug for accelerated approval early this year in the hopes that the treatment will become available to patients by the end of 2025 or early 2026, Denali said in its Feb. 6 press release.“Safety and tolerability endpoints are met and we consider tividenofusp alfa to be ‘well tolerated,’” Denali said in an emailed comment to Fierce Biotech. Hunter syndrome is rare and the trial is small, so there are no pre-defined standards for the safety endpoints to be compared against, Denali said.  \"Today’s update is in line with prior updates and reinforces tivi’s potential as a best-in-class treatment option for Hunter syndrome, including normalization of multiple central and peripheral markers,\" analysts from William Blair wrote on Feb. 6. \"While early and in a limited number of patients, these appear to be translating to clinical benefit.\"Hunter syndrome, also called mucopolysaccharidosis type II (MPS II), is characterized by patients\' inability to break down certain sugars, which leads to tissue and organ damage that ultimately causes stiff joints, delayed growth and an enlarged spleen and liver, among other symptoms. The disease can also lead to cognitive and behavioral problems, and in severe cases, patients may not survive beyond their teenage years.There is one approved enzyme replacement therapy for Hunter syndrome in Takeda’s Elaprase. The Japanese drugmaker acquired the medicine when it bought Shire Pharmaceuticals in 2019.In the latest presentation, Denali said that patients saw a sharp decline of the biomarker heparan sulfate in both their cerebrospinal fluid and urine after 24 weeks, with levels dropping by an average of about 90% from baseline. These lower levels persisted for as long as 153 and 177 weeks, respectively, according to the biotech. Heparan sulfate is a liver sugar that patients with Hunter syndrome have difficulty breaking down.Tivi also restored patients’ liver volumes to normal and reduced levels of neurofilament, a sign of neuron damage, in the blood, according to the presentation. Neurofilament levels increased slightly at 24 weeks, a data point that drew scrutiny from analysts when first presented as an exploratory endpoint in 2021. By 49 weeks, neurofilament dropped by about 20%, with the decline then escalating to more than 70% in subsequent measurements, according to Denali. The investigators also tested patients’ hearing using pure tone average, a measurement of hearing sensitivity at sound frequencies important for speech. At 24 weeks, the volume a sound needed to be played in order for patients to respond dropped by an average of nearly nine decibels. In the eight patients tested at 153 weeks, the threshold dropped by more than 12 decibels.Patients also saw statistically significant improvements in two tests of cognition and behavior, the Bayley Scales of Infant and Toddler Development and the Vineland Adaptive Behavior Scales, Denali said.“Our primary analysis in 47 participants with MPS II and the additional long-term data in up to more than four years support the potential of tividenofusp alfa to address neurocognitive, behavioral and physical effects for all individuals living with MPS II,” Denali chief medical officer Carole Ho, M.D., said in the release. “We expect the progress achieved in our Hunter syndrome program to inform and accelerate additional therapeutics programs in our lysosomal storage disease portfolio, including Sanfilippo syndrome Type A.”While Denali plans to submit tivi for accelerated approval early this year, the company is also continuing its investigation of the drug in an ongoing phase 2/3 double-blind, randomized trial in about 54 pediatric and young adult patients with Hunter syndrome.Tivi is a version of the iduronate 2-sulfatase enzyme that is engineered to penetrate the blood-brain barrier by binding to transferrin receptor 1, which normally shuttles iron into the brain. Editor\'s note: This story was updated at 2:15 p.m. ET on Feb. 6 to include an analyst note.

SAN DIEGO, Dec. 16, 2024 /PRNewswire/ -- Arthrosi Therapeutics, Inc., a late-stage biotechnology company developing a potentially best-in-class, highly potent and selective next generation URAT1 inhibitor to reduce serum urate levels, flares and tophi in patients with gout, today announced the appointment of John Leaman, M.D., to its Board as an Independent Board Director. "John brings more than a decade of experience in finance, M&A and corporate strategy, with a strong understanding of gout, and we are excited to welcome him to our Board of Directors," said Litain Yeh, Ph.D., Founder and CEO of Arthrosi Therapeutics. "We believe that his insights and guidance will be invaluable as we continue to advance our pivotal Phase 3 program for AR882 and position the Company as a leader in treating gout, including tophaceous gout patients." John H. Leaman, M.D. is currently the Chief Financial Officer at Cellarity. During his tenure, he has helped oversee the close of a large pharma partnership, as well as leading the Company's Series D crossover financing. Prior to joining Cellarity in 2023, Dr. Leaman served as the Chief Financial Officer and Chief Business Officer at Impel Pharmaceuticals, where helped lead the Company's IPO in 2021. Before that, Dr. Leaman served as the Chief Financial Officer and Head of Corporate Development at Selecta Biosciences and as Head of Corporate Development at InfaCare Pharmaceutical until it was acquired by Mallinckrodt in 2017. Earlier, Dr. Leaman was the Chief Financial Officer of Medgenics and previously held senior roles at Shire plc. and Devon Park Bioventures, a venture capital fund targeting investments in therapeutics companies. He began his career serving a range of life sciences companies as an Associate Principal at McKinsey & Company. Dr. Leaman received an M.D. from the Perelman School of Medicine at the University of Pennsylvania, an M.B.A. from the Wharton School at the University of Pennsylvania, a B.A. in Psychology, Philosophy and Physiology from Oriel College, University of Oxford while completing a Rhodes Scholarship, and a B.S. in Biology from Elizabethtown College. "I am thrilled to join Arthrosi's Board of Directors as the company progresses AR882 through pivotal Phase 3 studies. Gout remains a serious condition for nearly 13 million patients in the U.S. alone and the current standard of care provides only limited benefit, especially in tophaceous gout patients," said Dr. Leaman. "I look forward to working with Arthrosi's Board and leadership team to lay the foundation for our next stage of growth and deliver on the promise of AR882 for patients." About Arthrosi: Arthrosi Therapeutics, Inc., headquartered in San Diego, CA, is focused on developing AR882, a potentially best-in-class, highly potent and selective next generation URAT1 inhibitor to reduce serum urate levels, flares and tophi in patients with gout. Gout remains a large and growing market with ~ 13M patients in the U.S. alone, ~2M of which have tophaceous gout. AR882 has demonstrated encouraging efficacy and safety compared to SOC in Phase 2 studies as well as impressive results in achieving complete resolution of tophi in a Phase 2b study. Arthrosi is currently advancing AR882 in a pivotal Phase 3 program. Media Contact: Shunqi Yan, PhD Founder & Chief Operating Officer [email protected] Investor Contact: Precision AQ Alex Lobo 212-698-8802 [email protected] SOURCE Arthrosi Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Former CEO of NPS Pharma brings strong biotech expertise to further accelerate GEn1E’s development and commercialization of dual signal modulators as novel precision therapies PALO ALTO, Calif., Dec. 11, 2024 (GLOBE NEWSWIRE) -- GEn1E Lifesciences (GEn1E), a clinical-stage, Phase 2 company, focused on accelerating novel therapies for inflammatory and rare diseases using machine learning, is pleased to announce the appointment of François Nader, MD, MBA as chair of its Board of Directors, effective immediately. "We are thrilled to welcome François to GEn1E’s Board of Directors as Chair," said Ritu Lal, PhD, CEO and Co-founder of GEn1E Lifesciences. "François’ deep expertise in biopharmaceutical innovation, strategic leadership, and rare diseases aligns perfectly with our mission to develop novel therapies for patients suffering from inflammatory and rare diseases. His transformational guidance will be invaluable as we continue to accelerate our pipeline to bring precision therapies to patients in need." "We are entering a pivotal time for treating inflammatory and rare diseases, and I'm excited to support GEn1E as it pioneers innovative therapies that could redefine patient care," said Dr. Nader. "I look forward to collaborating with Ritu and the GEn1E team to fully harness the potential of precision signal modulators and to accelerate the company's pipeline in addressing devastating diseases for patients." Dr. Nader currently serves as an Independent Board Director at Moderna and Ring Therapeutics, and Senior Advisor at Blackstone Life Sciences. Dr. Nader is the Chairman of BioLink.org and serves on the Board of Trustees for the Lebanese American University. Dr. Nader brings 30+ years of experience in the life sciences industry, including a highly successful tenure at NPS Pharmaceuticals as President and CEO where he led the company through a period of remarkable growth and achievement, culminating in its acquisition by Shire Pharmaceuticals for $5.2 billion. Formerly, François served as a chair or director of the Board of several public and private companies including chairman for Acceleron Pharma (acquired by Merck for $11.5B), and member of the Board for Alexion Pharmaceuticals (acquired by AstraZeneca for $39B) and Baxalta (acquired by Shire for $32B). Dr. Nader earned his French Doctorate in Medicine from St. Joseph University (Lebanon) and his Physician Executive MBA from the University of Tennessee. About GEn1E Lifesciences: GEn1E Lifesciences is a clinical-stage, Phase 2 company accelerating novel, dual signal modulator precision therapies for rare and inflammatory diseases. GEn1E Lifesciences is based in Palo Alto, California, with a laboratory in Mountain View, California. GEn1E has developed a well-differentiated "Platform-in-a-Mechanism" drug development model that uses AI which spans the entire end-to-end drug development cycle. The company has accelerated several milestones to build a portfolio of 21+ novel Dual Signal Modulators across two targets, which are actively being developed as first-in-class therapies for rare and inflammatory diseases with lead compound in Phase 2 currently enrolling patients in the US. For more information on GEn1E, visit www.gen1e.com and follow GEn1E on LinkedIn. Contacts:PR: info@gen1elifesci.comPartnering: partnering@gen1elifesci.comWeb: http://www.gen1e.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/30ec883f-2e8f-4aae-833c-40edef90287a