Otonomy, Inc.

Financing led by BOLD Capital Partners, alongside early investments from Horizons Ventures and participation from a syndicate of leading angel investors in the life sciences Capital to support clinical development of first-in-class, worm-derived medicines for allergies and autoimmune disorders and advancement of discovery engine Peter Barton Hutt, former Chief Counsel for the Food and Drug Administration, joins Holoclara’s Board of Directors PASADENA, Calif.--(BUSINESS WIRE)-- Holoclara, a biotechnology company pioneering the development of worm-derived therapies for allergies and autoimmune disorders, today announced the completion of an oversubscribed $16 million Series A financing led by BOLD Capital Partners. Horizons Ventures, Tarrasque, Endurance28, Freeflow Ventures, and a syndicate of leading angel investors also participated in the round. In connection with the financing, Peter Barton Hutt, former Chief Counsel for the Food and Drug Administration (FDA) and currently senior counsel at Covington & Burling LLP, has been appointed to the company’s Board of Directors. The capital will be used to progress development of Holoclara’s orally available therapeutics into the clinic this year. The company additionally continues to advance its broader discovery engine to isolate and identify from worms particular molecules with therapeutic potential. “Holoclara was born out of an unmet clinical need. During my physician training, I met countless patients with life-altering allergies and autoimmune disorders that were often undiagnosed and untreated. Medicine lacked the tools to address these debilitating diseases — despite compelling evidence I and other worm scientists unearthed pointing to worms as a promising, if unexpected, therapeutic approach,” said Andrea Choe, M.D., Ph.D., co-founder and Chief Executive Officer of Holoclara. “With our world-class team and leading investors, we are taking matters into our own hands to usher in a new era of medicines inspired by nature that can transform the lives of millions of people.” The company’s discovery engine builds on Dr. Choe’s research uncovering a unique pheromone language shared by roundworms, which may have evolved over hundreds of millions of years. She further discovered molecules derived from these worms that blocked formation of disease in animal models of allergic and autoimmune disease. These findings, published in the Proceedings of the National Academy of Sciences (PNAS) journal, became the foundation of Holoclara’s technology, enabling the company to replicate therapeutic molecules using synthetic chemistry for the development of safe and orally available medicines with the potential to treat, and even cure, prevalent diseases. "Holoclara’s ‘against the grain’ approach of seeking inspiration from worms intrigued the BOLD Capital team from the very start,” said Neal Bhadkamkar, Ph.D., General Partner of BOLD Capital Partners and member of Holoclara’s Board of Directors. “By focusing on a particular worm species that had co-evolved with humans over hundreds of thousands of years as a gut-resident parasite, Holoclara discovered powerful immune modulatory compounds in their secretions. The company is mining this previously unexplored vein for the potential to create therapies for chronic inflammatory conditions that work with the immune system rather than against it.” “I’m pleased to advise Holoclara as the company moves its groundbreaking science harnessing worm biology closer to the clinic and into the hands of countless patients suffering from allergic and autoimmune diseases today,” said Peter Barton Hutt. “Holoclara has assembled a strong and experienced scientific and medical team to pursue this new generation of medicine.” Dr. Choe founded Holoclara with Paul W. Sternberg, Ph.D., professor at California Institute of Technology (Caltech), and Frank C. Schroeder, Ph.D., professor at Cornell University. Additional leading scientists and entrepreneurs sit on the company’s Board of Directors, including Dr. Bhadkamkar, Mr. Hutt, and Seemay Chou, Ph.D., CEO of Arcadia Science. Its advisors include Rick Maizels, Ph.D., professor at University of Glasgow; Brian Stoltz, Ph.D., professor at Caltech; Stan Lapidus, serial entrepreneur and former CEO of Cytyc Corporation and EXACT Sciences; Colleen Cutcliffe, Ph.D., CEO of Pendulum Therapeutics; Joe Betts-LaCroix, CEO of Retro Biosciences; and Carl LeBel, Ph.D., biotech executive formerly of Amgen, Otonomy, and Frequency Therapeutics. About Holoclara Holoclara is the first biotechnology company to create safe, orally available worm-derived therapies designed to provide relief to millions of people living with allergic and autoimmune disease. Founded and led by physician-scientist Andrea Choe, Holoclara mines potent immunomodulatory molecules from gut roundworms, building upon decades of preclinical data proving their therapeutic efficacy across a wide range of diseases. With a team of world-renowned experts in worm biology and biotechnology and the backing of leading life sciences and technology investors, Holoclara is uniquely positioned to harness the therapeutic potential of worms to address historically untreatable diseases. To learn more, visit .

An 11-year-old boy who was born deaf can hear after receiving an experimental gene therapy, developer Eli Lilly reported on Tuesday.The boy, identified as Aissam Dam by The New York Times, was the first participant treated in a small study Lilly is running to test the therapy, which is designed to correct a particular type of inherited hearing loss.While only from one person, the result is a striking example of gene therapys potential. According to Lilly, Dam experienced restored hearing across sound frequencies within a month of treatment. In some frequencies, Dams hearing was within the normal range at day 30, Lilly said.Lilly plans to present full data from Dam, as well as a second participant in its study, at a medical conference in early February.“Gene therapy for hearing loss is something physicians and scientists around the world have been working toward for over 20 years,“ said John Germiller, a director of clinical research at the Children's Hospital of Philadelphia who administered Lillys gene therapy to Dam.“These initial results show that it may restore hearing better than many thought possible, he added in a statement provided by the company.Lillys success spotlights advancing research around a gene called otoferlin. Mutations in this gene disrupt production of functional otoferlin protein, which is needed for so-called hair cells in the inner ear to transmit sound signals to the brain. Lillys therapy delivers, via an engineered virus, copies of the otoferlin gene into the ear, resulting in the needed protein.Otoferlin-mediated hearing loss is estimated to affect around 200,000 people worldwide, according to Lilly.Other groups are working on a similar approach. In China, several deaf children have been successfully treated by gene therapies developed there, as MIT Technology Review has documented. Last year, Regeneron Pharmaceuticals acquired Decibel Therapeutics for its otoferlin-directed gene therapy, and a study of that treatment is currently recruiting participants, according to a federal database.In France, meanwhile, a company called Sensorion recently received a green light from regulators to proceed with an otoferlin gene therapy its developing.Lillys treatment, dubbed AK-OTOF-101, came via a $610 million acquisition of Akuous in late 2022.The expanding otoferlin treatment pipeline comes as the broader gene therapy field sees setbacks as well as successes.Many young biotech companies working on genetic medicines have struggled to secure sufficient funding, leading to layoffs, while clinical trial delays have stalled others' progress.But the field has delivered approved medicines that can deliver dramatic results for diseases like spinal muscular atrophy, sickle cell disease and beta thalassemia. The first gene therapy approved in the U.S., Luxturna, can help correct an inherited cause of blindness.Alongside progress for otoferlin-directed treatments, companies working on other types of hearing loss drugs have struggled. Otonomy shut down in December 2022 after years of setbacks, while Frequency Therapeutics merged with Korro Bio after a clinical trial failure in February last year. '

FRAMINGHAM, Mass.--( BUSINESS WIRE )-- Alzheon, Inc. , a clinical-stage biopharmaceutical company developing a broad portfolio of product candidates and diagnostic assays for patients suffering from Alzheimer’s disease (AD) and other neurodegenerative disorders, today announced that it has strengthened its commercial organization with the promotion of Glenn E. Pauly to Chief Commercial Officer and the appointments of Dina Lynch to Vice President of Market Access, and Nate Greene to Vice President of Brand Marketing. These appointments will support preparations for the commercial launch of investigational agent ALZ-801, which is currently being evaluated in the APOLLOE4 Phase 3 trial in Early Alzheimer’s disease patients with two copies of the APOE4 gene. “Our well-differentiated efficacy results combined with a favorable safety profile, showing no increase in vasogenic brain edema in more than 2,800 patients, position ALZ-801 to potentially become the first oral agent that can slow or even stop and prevent Alzheimer’s pathology in patients and healthy individuals at risk for the disease,” said Martin Tolar, MD, PhD, Founder, President, and CEO of Alzheon. “Alzheon’s novel therapeutic approach has an opportunity to transform the standard of care and improve access to treatment for all Alzheimer’s patients. APOLLOE4 Phase 3 trial evaluating oral ALZ-801 in APOE4/4 patients with Early Alzheimer’s disease is expected to read out in the third quarter of 2024, and we are now building the team to prepare the NDA filing and commercial launch of the product in the U.S. in 2025.” Mr. Pauly joined Alzheon in July of 2022 as the Head of Commercial, where he began the process of internal planning and building of organizational infrastructure to support preparations for a commercial launch of ALZ-801. He has more than 20 years of commercial launch, sales, market access, and leadership experience in the biopharmaceutical industry. Prior to joining Alzheon, Mr. Pauly was the West Division General Manager and Vice President at Biogen with responsibility for the launch of the anti-amyloid antibody Aduhelm®. Previously, he held roles at Johnson & Johnson, Genentech, and AstraZeneca, where he supported launches and commercialization for multiple products including Remicade®, Actemra®, Xolair®, and Fasenra®. Ms. Lynch brings over 25 years of biopharma industry experience to Alzheon, with extensive expertise in building and managing market access functions. Ms. Lynch joins Alzheon from Nabriva Therapeutics, where she held dual roles as Vice President, Sales and Managed Care. Previously, she served as Vice President, Market Access at both Otonomy and Optimer. Mr. Greene brings 25 years of experience in the biopharma industry in central nervous system (CNS) diseases. He has broad experience in marketing, sales, and access leadership as well as commercial strategy, operations, business development and alliance management. Previously, Mr. Greene held commercial and executive roles at Janssen, Otsuka, and Biogen, with over a dozen launches in CNS disorders and Alzheimer’s. “We are committed to delivering a disruptive option to the Alzheimer’s disease market as quickly as possible and I am excited to have Dina and Nate join the commercial team,” said Mr. Pauly. “Their depth of experience and understanding of access pathways and marketing strategies are what we need during this crucial period. Having these strong leaders further enhances our ability to establish critical partnerships and patient advocacy programs to support a successful commercial launch in 2025.” About ALZ-801 ALZ-801/valiltramiprosate is an investigational oral agent in Phase 3 development as a potentially disease modifying treatment for AD. 1,3 ALZ‑801 is designed to block the formation of neurotoxic soluble beta amyloid oligomers causing cognitive decline in Alzheimer’s patients. In mechanism of action studies, ALZ-801 has fully inhibited the formation of neurotoxic soluble beta amyloid oligomers at the Phase 3 clinical dose. 5,6 ALZ‑801 acts through a novel enveloping molecular mechanism of action to fully block formation of neurotoxic soluble amyloid oligomers in the human brain 7 associated with the onset and progression of cognitive decline in AD patients. 1–4 ALZ-801 received Fast Track designation from the U.S. Food and Drug Administration in 2017 for Alzheimer’s disease. In clinical trials, ALZ-801 has shown favorable safety results. 5–7,9 The initial Phase 3 program for ALZ-801 is focusing on Early AD patients with the APOE4/4 genotype, with potential future program expansion to AD treatment and prevention in patients carrying one copy of the APOE4 gene and noncarriers. 1–4 ALZ-801 Phase 2 Biomarker Trial Biomarker Effects of ALZ-801 in APOE4 Carriers With Early Alzheimer's Disease ( NCT04693520 ): This ongoing trial was designed to evaluate the effects of 265 mg twice daily oral dose of ALZ-801 on biomarkers of AD pathology in subjects with Early AD, who have either the APOE4/4 or APOE3/4 genotype and constitute 65-70% of Alzheimer's patients. The trial also included evaluation of clinical efficacy, safety, tolerability, and pharmacokinetic profile of ALZ-801 over 104 weeks of treatment. An ongoing long-term extension of the trial evaluates ALZ-801 for an additional 52 weeks of treatment for a total of 156 weeks. ALZ-801 APOLLOE4 Phase 3 Trial An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early Alzheimer's Disease Subjects ( NCT04770220 ): This ongoing trial is designed to evaluate the efficacy, safety, biomarker and imaging effects of 265 mg twice daily oral dose of ALZ-801 in Early AD subjects with two copies of the apolipoprotein ε4 allele (APOE4/4 homozygotes), who constitute approximately 15% of Alzheimer's patients. This is a double-blind, randomized trial comparing oral ALZ-801 to placebo treatment over 78 weeks. The APOLLOE4 trial is supported by a $51 million grant from the National Institute on Aging . About Alzheon Alzheon, Inc. is a clinical-stage biopharmaceutical company developing a broad portfolio of product candidates and diagnostic assays for patients suffering from Alzheimer’s disease and other neurodegenerative disorders. We are committed to developing innovative medicines by directly addressing the underlying pathology of neurodegeneration. Our lead Alzheimer’s clinical candidate, ALZ-801/valiltramiprosate , is an oral agent in Phase 3 development as a potentially disease modifying treatment for AD. ALZ-801 is an oral small molecule that has been observed to fully block the formation of neurotoxic soluble amyloid oligomers in preclinical tests. Our clinical expertise and technology platform are focused on developing drug candidates and diagnostic assays using a precision medicine approach based on individual genetic and biomarker information to advance therapies with the greatest impact for patients. Alzheon Scientific Publications 1 Tolar M, et al: Neurotoxic Soluble Amyloid Oligomers Drive Alzheimer’s Pathogenesis and Represent a Clinically Validated Target for Slowing Disease Progression , International Journal of Molecular Sciences , 2021; 22, 6355. 2 Abushakra S, et al: APOE ε4/ε4 Homozygotes with Early Alzheimer’s Disease Show Accelerated Hippocampal Atrophy and Cortical Thinning that Correlates with Cognitive Decline , Alzheimer’s & Dementia , 2020; 6: e12117. 3 Tolar M, et al: Aducanumab, Gantenerumab, BAN2401, and ALZ-801—the First Wave of Amyloid-Targeting Drugs for Alzheimer’s Disease with Potential for Near Term Approval, Alzheimer’s Research & Therapy , 2020; 12: 95. 4 Tolar M, et al: The Path Forward in Alzheimer’s Disease Therapeutics: Reevaluating the Amyloid Cascade Hypothesis , Alzheimer’s & Dementia , 2019; 1-8. 5 Hey JA, et al: Discovery and Identification of an Endogenous Metabolite of Tramiprosate and Its Prodrug ALZ-801 that Inhibits Beta Amyloid Oligomer Formation in the Human Brain , CNS Drugs , 2018; 32(9): 849-861. 6 Hey JA, et al: Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer’s Disease , Clinical Pharmacokinetics , 2018; 57(3): 315–333. 7 Abushakra S, et al: Clinical Effects of Tramiprosate in APOE4/4 Homozygous Patients with Mild Alzheimer’s Disease Suggest Disease Modification Potential , Journal of Prevention of Alzheimer’s Disease , 2017; 4(3): 149-156. 8 Kocis P, et al: Elucidating the A β 42 Anti-Aggregation Mechanism of Action of Tramiprosate in Alzheimer’s Disease: Integrating Molecular Analytical Methods , Pharmacokinetic and Clinical Data , CNS Drugs , 2017; 31(6): 495-509. 9 Abushakra S, et al: Clinical Benefits of Tramiprosate in Alzheimer’s Disease Are Associated with Higher Number of APOE4 Alleles: The “APOE4 Gene-Dose Effect,” Journal of Prevention of Alzheimer’s Disease, 2016; 3(4): 219-228.