AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinsons disease – The…

Berlin, Germany, and Research Triangle Park, N.C., USA, July 11, 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 United States (U.S.) Food and Drug Administration (FDA) has granted Fast Track Designation for AB-1005, which is being developed for moderate Parkinsons disease. AB-1005 has also been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation, for the treatment of Parkinsons disease.

AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy being studied for the treatment of moderate Parkinsons disease. Earlier this year, AskBio presented the 18-month Phase Ib clinical trial results for AB-1005, which met its primary objective of evaluating the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.

These designations clearly underscore the importance of developing innovative therapies for those living with Parkinsons disease, where a significant unmet need still exists, said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinsons and MSA, AskBio. They further highlight the willingness of key regulatory bodies to support the accelerated development of AB-1005 with a focus on the potential benefit to patients.

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.1 The purpose of the Program is to get important new therapeutics to patients earlier.1 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

The UK MHRA Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market, facilitating patient access. This designation providesInnovation Passport holders with the opportunity to work with the UK MHRA and partners to create product-specific Target Development Profiles (TDP) for new therapies. The TDP will define key regulatory and development features, identify potential pitfalls, offer access to specialist toolkits, and create a roadmap for delivering early patient access.2,3

The FDA Fast Track and the UK MHRA Innovation Passport designations represent important accomplishments for the clinical development of AB-1005 and receiving these highlights our goal of bringing a safe neurorestorative treatment to patients with moderate Parkinsons disease, said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. We look forward to advancing our Phase II REGENERATE-PD clinical trial, which is currently enrolling patients in the U.S. with sites in the European Union and UK planned to open later this year.

We are excited about the opportunity to potentially accelerate the development of AB-1005, leveraging the frequent interaction with relevant regulatory bodies, said Christian Rommel, PhD, Global Head of Research & Development at Bayers Pharmaceuticals Division. The granted designations for AB-1005 highlight the demand to advance novel therapeutic modalities, like gene therapy, for people living with the debilitating effects of Parkinsons disease.

AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

AskBio is also exploring GDNF therapy beyond Parkinsons disease and is currently enrolling patients in the U.S. with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I trial to assess the preliminary safety, tolerability, and efficacy of GDNF therapy for this rapidly progressing condition.4

About Parkinsons disease Parkinsons disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels.5At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons.6 The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement.7Even with medication, the symptoms of Parkinsons disease can fluctuate during the course of the day. According to the Parkinsons Foundation, more than 10 million people worldwide suffer from Parkinsons disease, with approximately one million living in the U.S.8 There is no cure, and the effectiveness of current treatments frequently decreases over time.9

About REGENERATE-PD REGENERATE-PD is a Phase II, randomized, double-blind, surgery-controlled trial of the efficacy and safety of intraputaminal AB-1005 in the treatment of adults (45-75 years) with moderate stage Parkinson's disease. Subjects will receive either bilateral image-guided infusion of AB-1005 into the putamen, single dose (active treatment arm), or bilateral partial burr/twist holes (control arm). The trial will include an estimated 87 subjects with trial sites located in the U.S., European Union, and the UK. For more information about the REGENERATE-PD clinical trial, visit clinicaltrials.gov (NCT06285643), or visit askbio.com.

About AB-1005 AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-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.10,11 GDNF is a homodimer that is a distantly related member of the transforming growth factor- 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. GDNF has long been evaluated as a potential disease modifying neurorestorative treatment for diseases, such as Parkinsons, marked by progressive degeneration of midbrain dopaminergic neurons.12

About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntingtons disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinsons disease, and Pompe disease. AskBios gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at http://www.askbio.com or follow us on LinkedIn.

About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, Health for all, Hunger for none, the companys products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to http://www.bayer.com.

Find more information about AskBio at http://www.askbio.com Find more information about Bayer at https://pharma.bayer.com/ Follow Bayer on Facebook: http://www.facebook.com/bayer

Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayers public reports which are available on the Bayer website at http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

1 US FDA Fast Track. Available at: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track Accessed: July 2024. 2 UK Government. Innovative Licensing and Access Pathway. Available at: https://www.gov.uk/guidance/innovative-licensing-and-access-pathway Accessed: July 2024 3 UK Government. First Innovation Passport awarded to help support development and access to cutting-edge medicines. Available at: https://www.gov.uk/government/news/first-innovation-passport-awarded-to-help-support-development-and-access-to-cutting-edge-medicines Last accessed: July 2024 4 ClinicalTrials.gov. GDNF Gene Therapy for Multiple System Atrophy. Available at: Study Details | GDNF Gene Therapy for Multiple System Atrophy | ClinicalTrials.gov. Accessed:July 2024. 5 Michael J. Fox Foundation. Parkinsons 101 What is Parkinsons disease? Available at:https://www.michaeljfox.org/parkinsons-101. Accessed: July 2024. 6 DeMaagd G, Philip A. Parkinsons Disease and Its Management: Part 1: Disease Entity, Risk Factors, Pathophysiology, Clinical Presentation, and Diagnosis. P T. 2015 Aug;40(8):504-32. PMID: 26236139; PMCID: PMC4517533. 7 Parkinsons Foundation. Motor-fluctuations. Available at:https://www.parkinson.org/library/fact-sheets/motor-fluctuations. Accessed: July 2024. 8 Parkinsons Foundation. Who has Parkinsons? Available at:https://www.parkinson.org/understanding-parkinsons/statistics. Accessed: July 2024 9 Mayo Clinic. Parkinsons disease. Available at:https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062. Accessed: July 2024 10 Heiss JD, Lungu C, Hammoud DA, Herscovitch P, Ehrlich DJ, Argersinger DP, Sinharay S, Scott G, Wu T, Federoff HJ, Zaghloul KA, Hallett M, Lonser RR, Bankiewicz KS. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinsons disease. Mov Disord. 2019 Jul;34(7):1073-1078. 11 Kells AP, Eberling J, Su X, Pivirotto P, Bringas J, Hadaczek P, Narrow WC, Bowers WJ, Federoff HJ, Forsayeth J, Bankiewicz KS. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul 14;30(28):9567-77. 12 Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 1993;260(5111):1130-1132

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AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinsons disease - The...