At Prothena, every step we take to heal, cure and prevent disease, we take in collaboration with the patients and their families who generously participate in clinical studies.
Our current clinical programs include birtamimab for the potential treatment of AL amyloidosis, prasinezumab for the potential treatment of Parkinson’s disease, NNC6019/PRX004 for the potential treatment of ATTR amyloidosis, and PRX005 and PRX012 for the potential treatment of Alzheimer’s disease.
Birtamimab is a humanized monoclonal antibody being developed for the potential treatment of patients with Mayo Stage IV AL amyloidosis. The confirmatory Phase 3 registration enabling AFFIRM-AL study (NCT04973137) evaluating the efficacy and safety of birtamimab in patients with Mayo Stage IV AL amyloidosis is ongoing under a Special Protocol Assessment (SPA) agreement with the FDA.
Prasinezumab is a humanized monoclonal antibody being developed for the potential treatment of Parkinson’s disease, and is the focus of a worldwide collaboration between Prothena and Roche. The Phase 2b PADOVA study (NCT04777331) evaluating the efficacy and safety of prasinezumab in patients with early Parkinson’s disease is ongoing. Also, the open label extension portion of the Phase 2 PASADENA study (NCT03100149) evaluating prasinezumab in participants with early Parkinson’s disease is ongoing. Both studies are being conducted by our partners at Roche.
NNC6019/PRX004 is a humanized monoclonal antibody being developed for the potential treatment of ATTR amyloidosis. The Phase 1 study (NCT03336580) is complete. Novo Nordisk has acquired Prothena’s NNC6019/PRX004 and is conducting the ongoing Phase 2 study (NCT05442047) in patients with ATTR cardiomyopathy. More information on the acquisition can be found here.
PRX005 is a humanized monoclonal antibody being developed for the potential treatment of Alzheimer’s disease and is the first target of three in a collaboration with Bristol Myers Squibb. The Phase 1 single ascending dose (SAD) portion of the study in healthy volunteers demonstrated dose-proportional PRX005 concentrations in plasma with robust central nervous system (CNS) penetration of this potentially best-in-class investigational anti-MTBR-tau antibody. Single doses of PRX005 across three dose cohorts were generally safe and well tolerated, meeting the primary objective of the study. The Phase 1 multiple ascending dose (MAD) portion of the study is ongoing in healthy volunteers and patients with Alzheimer’s disease.
PRX012 is a next-generation monoclonal antibody being developed for the potential treatment of Alzheimer’s disease. The Phase 1 study with PRX012 is ongoing.
Prothena is committed to developing safe and effective therapies for patients through thoughtful and efficient drug development. Consistent with this commitment, Prothena conducts clinical trials to generate safety and efficacy data required for regulatory approval to make our medicines available broadly to patients as quickly as possible. Prothena believes that enrollment in these clinical trials should be the first and most preferable route for patients to access investigational treatments.
Patients facing serious or life-threatening illnesses who are ineligible or unable to participate in a clinical trial and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors, may consider expanded access programs for unapproved therapies. We support the need for expanded access programs and our goal is to provide access to study drugs at the appropriate time and in the correct context for patients with life-threatening diseases.
We consider a number of factors when determining whether to provide expanded access for a given investigational drug outside of a clinical trial:
Prothena has determined, after assessing these considerations, that expanded access cannot currently be offered for any of our investigational drugs. We periodically re-evaluate this policy and its application to our study drugs.
If and when we provide expanded access for a particular investigational drug, we will review each individual request on a case-by-case basis in a fair and equitable manner and respond to the physician who makes the request. During this process, we will need to ensure that:
The above are general requirements, and specific requirements will be applied for a given investigational drug or clinical program if and when Prothena initiates an expanded access program.
Note that investigational drugs have not yet been approved or cleared by regulatory authorities and regulatory authorities have not found these products to be safe and effective for their specific use. Furthermore, investigational drugs may, or may not, be effective in the treatment of the applicable condition, and use of such investigational drugs may cause unexpected serious side effects.
– Patients should consult with their treating physician about their eligibility to enroll in any of Prothena’s clinical trials. To get more details about Prothena’s clinical trials, please visit the “Clinical Trials” page on Prothena’s website or on www.ClinicalTrials.gov. If you are a physician who has questions about Prothena’s investigational therapies, please contact us at email@example.com.
– The publication of this policy by Prothena is not a guarantee of access to any specific investigational drug by any individual patient. Prothena may revise this expanded access policy at any time.