Biodexa’s eRapa Shows Promise in FAP Treatment Trial, Phase 2
Biodexa scientists continue working on Phase 2 eRapa FAP Treatments: Image by Tung Lam.
- Phase 2 FAP treatment sees around 75% non-progression of polyps after 12 months.
- Overall size of polyps goes down by an average of 17% on after one year.
- Cohort 2: 89% of polyps didn’t get worse and shrank overall by around 29%.
- Findings from this trial will help determine dosages for the next, big phase study.
Promising Drug, Phase 2 Trial, New Hope for FAP
Biodexa Pharmaceuticals PLC is a clinical-stage biologic (biopharmaceutical) drug company. It’s known for developing the kind of medicines that help people suffering from otherwise hard-to-treat conditions. The article looks at a Phase 2 trial using eRapa to treat Familial Adenomatous Polyposis (FAP). This genetic condition causes precancerous (abnormal) polyps to grow in the colon and rectum. eRapa™ is a rapamycin (Rapa) formulation trialed in patients with FAP. This study aims to reduce polyp progression, but has it succeeded?
The results of Biodexa’s Phase 2 clinical trial of eRapa for FAP are promising. Dr. Carol Burke, a specialist gastroenterologist, has been the study’s principal investigator. Her research teams conducted experiments across seven U.S. centers. So far, the tests have demonstrated significant reductions in polyp burden (size and number). They also saw high non-progression rates over 12 months. Partial funding for this phase 2 trial has come from a Texas cancer research institute.
FAP Treatment: A Study Overview
The method used in the Phase 2 study was basic. There were 30 adult participants (median age 43) split into three different dosing groups. That means each group received varying doses of eRapa over the 12-month trial.
The controlled dosages looked like this:
- Group 1: 0.5 mg every alternate day
- Group 2: 0.5 mg daily every other week
- Group 3: 0.5 mg every day
The trial monitored safety, how well participants tolerated Rapa treatment, and how well it reduced polyps. That leads us to the trial’s key findings.
FAP Treatment Breakthroughs
The Phase 2 study of eRapa in Familial Adenomatous Polyposis (FAP) demonstrated several notable breakthroughs, as shown in this table.
| Cohort | FAP Treatment Breakthroughs | Details |
|---|---|---|
| Overall | Patients without disease progression | 75% at 12 months |
| Overall | Average reduction in polyp size | 17% at 12 months |
| Group 2 | Percentage of group 2 that didn’t get worse | 89% at 12 months |
| Group 2 | Group 2 polyp size reduction | 29% at 12 months |
| All | Safety and tolerability | Appeared safe and well-tolerated |
| All | Participant adherence rate | 95% at 12 months |
| All | Serious adverse events (SAEs) | 4 x grade 3+ SAEs, 1 x SAE (unspecified) |
These findings offer much hope for eRapa’s future advancement and possible application in the management of FAP.
About Serious Adverse Events (SAEs)
SAEs are basically unwanted effects, ranging in seriousness on a scale of 1-5. That can be severe or even life-threatening. Depending on the severity, SAEs can lead to hospitalization, require medical intervention, or, in a worst-case scenario, cause death.
The five serious adverse events are, in order of seriousness, as follows:
- Grade 1: Mild; does not interfere with daily activities
- Grade 2: Moderate; may interfere with daily activities
- Grade 3: Severe; may require hospitalization or medication
- Grade 4: Life-threatening; requires urgent medical intervention
- Grade 5: Death related to the intervention
Implications and Next Steps
The results of this FAP Treatment Phase 2 trial are better than expected. It seems that eRapa might delay or even end the need for surgical interventions in some patients. It’s a simple remedy that could improve the quality of life for many FAP sufferers. Thanks to these outcomes, an upcoming Phase 3 study will take the Phase 2 findings up a level. The new study is also supported by a Texas cancer research institute to the tune of $17M.
What to Expect from FAP Treatment Phase 3
The Phase 3 study will be a double-blind, placebo-controlled trial. That means a trial that fairly tests a new medicine by removing any bias and comparing it to a “dummy” treatment. Phase 3 plans to include around 140 high-risk patients diagnosed with FAP. The main goal will be to see how long FAP patients can go without their condition getting worse.
This includes several factors, but especially the five listed below:
- Whether a person requires major surgery
- The removal of large polyps
- The advancement of the disease stage
- The diagnosis of severe cell changes or cancer
- Any deaths
Limitations and Future Research
LIMITATIONS
The Phase 2 FAP Treatment trial was good but not perfect. Researchers and participants knew who got the medicine (eRapa) and who had the placebo. This matters because it can influence the results. Another downside was that Phase 2 only involved 30 people, which is quite small in terms of research. The small size may also exclude the conditions of some FAP patients. Lastly, a year-long study doesn’t tell everything about the longer-term effect
FUTURE RESEARCH
Scientists agree that future research needs larger-scale trials. That’s why Phase 3 includes around 140 people. Also, neither the researchers nor the participants will know who’s getting what. That makes it a double-blind trial. This approach gives scientists more solid data on how well eRapa works and if it’s safe. They will also want to follow the test subjects (participants) for longer to see how eRapa affects them in the longer term. Such a study has lots of room for researchers to experiment. They could vary eRapa dosages further or combine other treatments to see what works best.
Closing Comments
That ends this summary on Biodexa’s Phase 2 trial of eRapa for treating Familial Adenomatous Polyposis (FAP). There is no doubt the results are encouraging for FAP sufferers. Across all three trial groups, 75% did not experience a worsening of their condition. In the second FAP treatment group, that number rose to 89%, and 29% saw a reduction in polyp size. The average reeducation in polyp size across all groups was 17%.
These data suggest eRapa™ might delay or even prevent the need for surgery in FAP patients. Thanks to these outcomes, the research is ongoing (Phase 3 trial). The upcoming study aims to further validate and build upon these encouraging results.
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Resource links
Phase 2 Clinical Trial Results of eRapa™ at 12 months in Familial Adenomatous Polyposis (FAP)
