Periodic Reporting for period 1 - OPTM.RX (Novel drug for dry age related macular degeneration)
Reporting period: 2023-07-01 to 2024-08-31
OphtiMed Rx is dedicated to developing innovative treatments for age-related macular degeneration (AMD), a leading cause of vision loss worldwide. The focus of the company’s project, supported by the Women Tech EU grant, is to advance the development of OM-301, a novel anti-senescence drug targeting dry AMD. The project aims to address the significant unmet medical need for effective treatments for dry AMD, a condition for which no approved therapies currently exist.
The company’s core innovation lies in its drug candidate’s ability to target cellular senescence, a key driver of age-related diseases. Cellular senescence occurs when cells lose their ability to divide and function normally, contributing to tissue damage and inflammation. By inhibiting the effects of senescence, OM-301 aims to protect retinal cells from degeneration, thereby preventing vision loss. This project builds on years of research and scientific advancements in the field of ocular therapeutics.
Context and Objectives
Dry AMD is a chronic and progressive disease that affects millions of people, leading to irreversible vision loss. The lack of approved treatments has created a significant clinical and societal burden. OphtiMed Rx is determined to address this gap with OM-301, which has demonstrated promising results in preclinical studies. The project’s overall objective is to complete the necessary nonclinical studies to demonstrate the safety, efficacy, and pharmacokinetics of OM-301 and prepare for first-in-human (FIH) clinical trials.
Specifically, the project involves:
• Developing an ocular formulation suitable for chronic treatment.
• Conducting nonclinical efficacy, safety, and ADME/PK studies in animal models.
• Preparing for regulatory interactions with the FDA and EMA to initiate clinical trials.
The expected impact of this project extends beyond the development of a single drug. Success in these early stages could pave the way for a new class of treatments targeting age-related diseases through anti-senescence mechanisms. This aligns with global healthcare priorities of addressing the growing challenges of aging populations and the increasing prevalence of age-related conditions.
The company’s core innovation lies in its drug candidate’s ability to target cellular senescence, a key driver of age-related diseases. Cellular senescence occurs when cells lose their ability to divide and function normally, contributing to tissue damage and inflammation. By inhibiting the effects of senescence, OM-301 aims to protect retinal cells from degeneration, thereby preventing vision loss. This project builds on years of research and scientific advancements in the field of ocular therapeutics.
Context and Objectives
Dry AMD is a chronic and progressive disease that affects millions of people, leading to irreversible vision loss. The lack of approved treatments has created a significant clinical and societal burden. OphtiMed Rx is determined to address this gap with OM-301, which has demonstrated promising results in preclinical studies. The project’s overall objective is to complete the necessary nonclinical studies to demonstrate the safety, efficacy, and pharmacokinetics of OM-301 and prepare for first-in-human (FIH) clinical trials.
Specifically, the project involves:
• Developing an ocular formulation suitable for chronic treatment.
• Conducting nonclinical efficacy, safety, and ADME/PK studies in animal models.
• Preparing for regulatory interactions with the FDA and EMA to initiate clinical trials.
The expected impact of this project extends beyond the development of a single drug. Success in these early stages could pave the way for a new class of treatments targeting age-related diseases through anti-senescence mechanisms. This aligns with global healthcare priorities of addressing the growing challenges of aging populations and the increasing prevalence of age-related conditions.
OphtiMed Rx has successfully completed the development of a stable, formulation of OM-301 intended for clinical ocular administration, along with placebo prototype batches. These formulations have been used in ongoing nonclinical studies to evaluate the drug’s efficacy and safety profile in relevant preclinical models. The results from these studies will inform the design of future clinical trials and support the company’s regulatory strategy.
The company has also completed a pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA). This crucial milestone has provided valuable feedback on the nonclinical and clinical development plans for OM-301, including guidance on the design of Good Laboratory Practice (GLP) toxicology studies. These studies are essential to ensuring the safety of the drug before it can be tested in humans.
In parallel, OphtiMed Rx is exploring opportunities for regulatory engagement with the European Medicines Agency (EMA) to seek scientific advice on the development program. The company plans to follow a parallel development path for both U.S. and European markets, ensuring that the drug meets regulatory requirements in both regions.
The company has also completed a pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA). This crucial milestone has provided valuable feedback on the nonclinical and clinical development plans for OM-301, including guidance on the design of Good Laboratory Practice (GLP) toxicology studies. These studies are essential to ensuring the safety of the drug before it can be tested in humans.
In parallel, OphtiMed Rx is exploring opportunities for regulatory engagement with the European Medicines Agency (EMA) to seek scientific advice on the development program. The company plans to follow a parallel development path for both U.S. and European markets, ensuring that the drug meets regulatory requirements in both regions.
Results Beyond the State of the Art: Pre-IND Completion
A key milestone achieved during this project was the successful completion of the pre-IND meeting with the FDA. This meeting provided critical regulatory guidance and confirmed that OM-301 is on the right track for development. The feedback from the FDA has helped refine OphtiMed Rx’s clinical and nonclinical plans, ensuring that future studies will meet regulatory requirements for first-in-human (FIH) clinical trials. This progress positions OM-301 beyond the current state of the art, particularly in the area of treatments for dry AMD, where no approved therapies exist.
Potential Impact and Next Steps
Completing the pre-IND is a significant step toward ensuring that OM-301 can move into clinical development. The FDA's feedback has helped shape the design of upcoming nonclinical studies, including Good Laboratory Practice (GLP) toxicology studies, which are essential to determining safety profiles and appropriate dosing for human trials.
For further uptake and success, key needs include:
1. Completion of GLP toxicology studies: These studies are critical to proceed to FIH trials.
2. Regulatory and market access: Continued engagement with regulatory bodies such as the FDA and EMA will be crucial for fast-tracking OM-301's development through pathways like Fast Track designation in the U.S. and conditional approval in the EU.
3. Funding and commercialization: Securing additional financing to fund ongoing preclinical studies, manufacturing, and phase I trials is vital. OphtiMed Rx is actively pursuing seed round funding for these activities.
4. IPR support: Protecting the intellectual property of OM-301 will be important as it moves closer to commercialization, ensuring long-term competitiveness in global markets.
Overview of Results
At the end of this project, OphtiMed Rx has:
• Developing an ocular formulation suitable for chronic treatment.
• Conducting nonclinical efficacy, safety, and ADME/PK studies in preclinical models.
• Successfully completed the pre-IND meeting with the FDA, receiving key guidance for the continued development of OM-301.
• Finalized the design of critical preclinical studies to ensure regulatory compliance.
• Advanced the regulatory strategy for fast-track options in both U.S. and EU markets.
With these achievements, OphtiMed Rx is well-positioned to bring OM-301 closer to clinical use, offering hope for millions affected by dry AMD.
A key milestone achieved during this project was the successful completion of the pre-IND meeting with the FDA. This meeting provided critical regulatory guidance and confirmed that OM-301 is on the right track for development. The feedback from the FDA has helped refine OphtiMed Rx’s clinical and nonclinical plans, ensuring that future studies will meet regulatory requirements for first-in-human (FIH) clinical trials. This progress positions OM-301 beyond the current state of the art, particularly in the area of treatments for dry AMD, where no approved therapies exist.
Potential Impact and Next Steps
Completing the pre-IND is a significant step toward ensuring that OM-301 can move into clinical development. The FDA's feedback has helped shape the design of upcoming nonclinical studies, including Good Laboratory Practice (GLP) toxicology studies, which are essential to determining safety profiles and appropriate dosing for human trials.
For further uptake and success, key needs include:
1. Completion of GLP toxicology studies: These studies are critical to proceed to FIH trials.
2. Regulatory and market access: Continued engagement with regulatory bodies such as the FDA and EMA will be crucial for fast-tracking OM-301's development through pathways like Fast Track designation in the U.S. and conditional approval in the EU.
3. Funding and commercialization: Securing additional financing to fund ongoing preclinical studies, manufacturing, and phase I trials is vital. OphtiMed Rx is actively pursuing seed round funding for these activities.
4. IPR support: Protecting the intellectual property of OM-301 will be important as it moves closer to commercialization, ensuring long-term competitiveness in global markets.
Overview of Results
At the end of this project, OphtiMed Rx has:
• Developing an ocular formulation suitable for chronic treatment.
• Conducting nonclinical efficacy, safety, and ADME/PK studies in preclinical models.
• Successfully completed the pre-IND meeting with the FDA, receiving key guidance for the continued development of OM-301.
• Finalized the design of critical preclinical studies to ensure regulatory compliance.
• Advanced the regulatory strategy for fast-track options in both U.S. and EU markets.
With these achievements, OphtiMed Rx is well-positioned to bring OM-301 closer to clinical use, offering hope for millions affected by dry AMD.