Category: News

Research Triangle Park, N.C.– MAY 2, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver nine presentations offering insights into its adeno-associated virus (AAV) research and development, a key area of gene therapy focus for the company, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which takes place May 7–11, 2024, in Baltimore, Maryland, USA. Presentations will focus on AAV immune-mediated responses as well as the results from the ongoing Phase 1 clinical trial of AB-1002 gene therapy in patients with advanced heart failure. Luke Roberts, MBBS, PhD, Medical Director for Clinical Development at AskBio, will deliver an oral presentation sharing new clinical data from the company’s ongoing Phase 1 trial of AB-1002 in patients with advanced heart failure. This follows AskBio’s recent news that AB-1002 was granted FDA Fast Track Designation for the treatment of congestive heart failure (CHF). AB-1002 (also known as NAN-101) is an investigational gene therapy that has not yet received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio previously communicated that the delivery of AB-1002 was well tolerated and resulted in positive preliminary efficacy outcomes in some patients with non-ischemic CHF and may validate that the AAV2i8 vector capsid used is highly cardiotropic when administered as a single intracoronary infusion at relatively low doses. Preliminary data from the Phase 1 trial of AB-1002 were presented at the 2023 American Heart Association Scientific Sessions in November, and AskBio began enrolling patients in its Phase 2 GenePHIT study of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms in January 2024. AskBio’s ASGCT presence will also include key presentations showcasing the company’s continued commitment to optimizing AAV as a gene therapy, with a focus on preventing or reducing AAV immune response-related adverse events, which remains a vital area of investigation across the gene therapy treatment landscape. Attendees can look forward to an ASGCT spotlight speaker presentation on current immune modulation strategies given by Shari Gordon, PhD, Senior Director of Immunology at AskBio, on Day 3. On Day 4, Shari Gordon will deliver on behalf of Audry Fernandez, PhD, Principal Scientist & Group Lead, Immunology R&D at AskBio, an oral presentation on pre-clinical research into AAV-specific immune responses, and Liujiang Song, PhD, Principal Scientist for R&D Capsid and Biology at AskBio, will offer insights into AAV biology and vector intracellular fate during an oral presentation on AAV episome configuration using third generation long-read sequencing technologies and advanced bioinformatics. “Our presence at ASGCT this year highlights our continued commitment to sharing AAV developments with the gene therapy community. Covering clinical and pre-clinical research, our presentations show our robust progress and ongoing ambition to bring to patients transformative therapies that were once unthinkable,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “With our clinical and early-stage programs advancing, these are exciting times at AskBio.” With an ambitious

Berlin, Germany, and Research Triangle Park, NC, USA, April 18, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.[1],[2] “The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of potentially bringing effective treatments to patients with advanced congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We look forward to completing our Phase II GenePHIT clinical trial, which is currently enrolling patients with severe heart failure, and are committed to exploring the full potential of AB-1002 for the treatment of this devastating disease.” The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.[3] The purpose of the Program is to get important new therapeutics to patients earlier.3 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. “The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division. “This designation underpins the potential of AB-1002 to address currently high unmet medical need, and we are excited about the opportunity to accelerate its development.” AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio is currently enrolling patients in the Phase II GenePHIT (Gene PHosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.[4]  About GenePHIT GenePHIT is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.4 Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo. Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test (6MWT).4 For more information, please visit clinicaltrials.gov

Berlin, Germany, and Research Triangle Park, NC, USA, April 16, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA. The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based on timing from PD clinical diagnosis and the severity of PD symptoms at trial screening.1 As of November 3, 2023, 57 nonserious adverse events (AEs) and 6 serious adverse events (SAEs) were reported. Most AEs were transient and were expected perioperative events (<1 month from treatment). These included headache, tremor, dyskinesia, arthralgia, musculoskeletal chest pain, fatigue, COVID-19, and magnetic resonance imaging (MRI) abnormalities. The 6 SAEs reported in 3 patients (n = 1 in the Mild Cohort and n = 2 in the Moderate Cohort) were all assessed as unrelated to the treatment by the Investigator and the Sponsor. Bilateral infusions of AB-1005 within the putamen (up to 1.8 mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent. Neurosurgical delivery of AB-1005 resulted in putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. Scheduled 6-month postoperative MRIs revealed findings of asymptomatic unilateral T1 hypointensity adjacent to 3 of the putaminal infusion trajectories. Clinical follow-up for up to 5 years post administration is ongoing.2 “These early findings are encouraging and show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Further, they highlight areas of potential future exploration in our upcoming Phase II REGENERATE PD trial, which will look more closely at the potential efficacy of AB-1005 in the treatment of Parkinson’s disease.” Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD.1 Mild Cohort The Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) is an internationally recognized tool used to assess the severity of PD symptoms, including motor symptoms. The Mild Cohort (n = 6) demonstrated relative stability from baseline to 18 months for both MDS-UPDRS Part II patient-reported Activities of Daily Living scores and Part III clinician-rated Motor Examination scores in “ON” and “OFF” medication states. Patient-reported PD Motor Diaries provide a tool for assessing patient motor state over an extended 3-day period and then normalized to a 16-hour waking day. The Mild Cohort (n = 5) showed a -1.3 hour reduction in “Good ON” time, a 0.2 hour increase in “ON” time with troublesome

Not intended for UK Media Berlin, Germany and Research Triangle Park, NC, USA, February 13, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been randomized in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks. This milestone in the development of AB-1002 for the treatment of CHF potentially brings this investigational therapy one step closer to treating patients with high unmet medical need.[2] GenePHIT will include between 90 and 150 adults with left ventricular ejection fraction between 15 and 35 percent, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.1 “The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community,” said Timothy D. Henry, MD, MSCAI, Principal Investigator and Steering Committee Member. “GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy overall for the treatment of congestive heart failure. The initiation of this trial brings us a step closer to potentially changing the course of this deadly and devastating disease.” “Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone,” said Roger J. Hajjar, MD, Scientific Chair CHF, AskBio. “The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure. Although there is still much to learn about this early-stage investigational gene therapy, we hope today’s announcement, which highlights AskBio’s ability to advance AB-1002 gene therapy for the treatment of congestive heart failure, is encouraging news for everyone hoping to see new treatment options.” “Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients.” AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AB-1002 is manufactured by Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of

Berlin, Germany/Research Triangle Park, North Carolina, USA, January 4, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2 The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based upon the timing from a PD diagnosis and the severity of their PD symptoms at screening.1 Neurosurgical delivery of AB-1005 was well tolerated by all patients with target putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. No serious adverse events have been attributed to AB-1005, with continued clinical follow-up for up to 5 years post administration ongoing.2 “We are encouraged by these early data, which show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Although there is still much to learn about this early-stage investigational gene therapy, these first findings will inform our work in this space and have the potential to contribute to the clinical advancement of AB-1005 for the treatment of Parkinson’s disease.” Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD. Additionally, brain imaging was performed to longitudinally assess safety and potential changes in dopamine handling or abnormal metabolic patterns associated with PD.1 AskBio is planning to present the 18-month study data, including secondary endpoints, at a scientific meeting in Q2 2024. Planning is underway for a Phase II trial that is expected to begin screening patients in the first half of 2024. The trial design has been harmonized with feedback from U.S. and European health authorities. “People living with Parkinson’s disease deserve options to address their unmet medical need,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The positive outcome of the AB-1005 Phase Ib clinical trial is an important step forward in our goal to deliver much-needed treatments in areas where innovation has the potential to make a tremendous impact.” About AB-1005 AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery.3,4 GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake.

Research Triangle Park, N.C. – November 17, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been randomized at the Ohio State University Wexner Medical Center in the Phase 1 REGENERATE MSA-101 clinical trial of AB-1005, a gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).[1] This marks a significant milestone in the development of AB-1005 gene therapy, an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) that is delivered to the putamen, and brings this therapeutic one step closer to potentially reaching patients. AB-1005 is also currently being investigated for the treatment of mild to moderate Parkinson’s disease with the enrollment of the Phase 1b study having now been completed.[2]   “It means a lot to the MSA community to know that the first patient has been enrolled in the Phase 1 REGENERATE MSA-101 trial,” said Philip M. Fortier, MA, President and Executive Director, Defeat MSA Alliance. “There is no cure for MSA, and there are currently no treatments to stop or slow the progression of the disease. This makes it especially hard for patients, given the rapid decline many will experience. Today’s milestone hopefully brings us one step closer to potentially changing the outcome for MSA patients.” MSA-P, which can initially be difficult to distinguish from Parkinson’s disease, is marked by slow movement, lack of coordination, imbalance, and dizziness, among other symptoms, as individuals experience increasing difficulty with movement.[3] This is the result of a progressive loss of nerve cells in the brain and spinal cord. Affecting an estimated 100,000–500,000 worldwide, MSA is a rare disease that in most cases seems to occur at random.3,[4],[5] Symptoms tend to appear in people during their 50s, followed by a rapid progression within 5–10 years.3 “In those with MSA-P, the loss of dopamine producing neurons leads to markedly disabling symptoms, such as profound motor impairment throughout the hands, legs, and trunk. Previous experience with a similar approach in a population of Parkinson’s disease patients has been very encouraging and supported the consideration of this approach in MSA-P. The intent is for GDNF levels in the brain to help preserve dopamine neurotransmission, which is noticeably reduced in MSA-P,” said Nicolas M. Phielipp, MD, University of California Irvine, REGENERATE MSA-101 Principal Investigator. “We’re including a genetic sequence in the AAV2 vector that codes for the GDNF protein and are delivering this to the putamen. In this way, we’re targeting local brain cells and adjacent brain tissue that can benefit from the protein’s growth properties. This trial marks the first step toward understanding the potential that GDNF gene therapy might have for patients with MSA-P.” “Enrolling the first patient in our REGENERATE MSA-101 trial is an important step in AskBio’s ongoing work to advance GDNF gene therapy” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “The clinical advancement of AB-1005 for the treatment of MSA-P