Genetic Medicines with Curative Intent
The definition of “advocate” is “to support a cause or proposal.” We believe that truly advocating for patients and families requires much more than “support.” It requires commitment, dedication, compassion and listening to ensure we are continually doing what is right for patients, their families and the greater community. We seek to inspire and lift up the advocates who work tirelessly to shine a light on debilitating diseases and conditions.
At 4DMT, our priority is to weave the patient perspective into the fabric of our work and daily activities. We believe in the importance of collaborating closely with patients, their families, patient advocacy organizations, physicians, researchers and regulatory authorities to educate on the work we are doing. We seek to advance our overall understanding of the diseases we treat, including the unmet patient needs that exist for each disease.
Neovascular age-related macular degeneration (wet AMD) is a type of age-related macular degeneration where abnormal blood vessels (choroidal neovascularization or CNV) grow into the macula, the central area of the retina. As a result, CNV causes swelling of and fluid retention in the retina, bleeding and scarring, and causes blurry vision and reduced ability to see small details. The ongoing leaking of the abnormal blood vessels is stimulated by vascular endothelial growth factor otherwise known as VEGF. This process distorts and can potentially destroy central vision and may progress to blindness without treatment. Wet AMD is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks.
People who have type 1 or type 2 diabetes may develop a complication called diabetic retinopathy or damage to the small blood vessels of the eye’s retina that can lead to vision loss. Diabetic macular edema (DME), which is associated with diabetic retinopathy and progresses faster, occurs when fluid from these damaged blood vessels leaks into the macula — an area in the center of the retina that helps us see objects directly ahead of us — which causes swelling of the retina and is also associated with vision loss. DME is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks.
About 4D-150 for the treatment of wet AMD & DME
4D-150 is being evaluated in the currently-enrolling PRISM clinical trial, a Phase 1/2 dose-escalation and randomized, controlled, masked expansion study of intravitreal 4D-150 in adults with wet AMD. The primary endpoints of the study are safety and tolerability. Secondary endpoints include the number of supplemental aflibercept injections over 52 weeks, change from baseline in best corrected visual acuity (BCVA) over time and central subfield thickness (CST) measured by SD-OCT.
4D-150 will be evaluated in the SPECTRA clinical trial, a Phase 2 randomized, active-controlled, double-masked study of intravitreal 4D-150 genetic medicine in adults with DME. The primary endpoint of this study is the annualized number of aflibercept injections in the study eye. Secondary endpoints include change from baseline in BCVA, CST and percentage of subjects with improvement in diabetic retinopathy severity scale. Enrollment is anticipated to begin in Q3 2023.
At 4DMT, we are committed to developing safe, effective and transformative therapies for patients with rare diseases and limited or no treatment options. We understand the importance of bringing these therapies to the broadest group of patients as quickly as possible. We believe that the best path to potential regulatory approval, and subsequent access for the greatest number of patients, is by executing our clinical studies (or trials) as efficiently as possible. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory review and potential approval.
After careful deliberation, we have determined that at this point in time, we must focus our efforts on conducting our clinical trials as efficiently as possible.
Therefore, participation in 4DMT clinical trials is the only way for patients to gain access to 4DMT’s investigational therapies early in development. We will continually evaluate the possibility of compassionate use, “Right to Try” and other expanded access programs.
Investigational therapies have not yet been approved by FDA or other regulatory agencies. Furthermore, investigational therapies may or may not be effective in the treatment of a condition, and use of the product may cause unexpected serious side effects.