Mark Midei Death, Obituary – Mark Midei Of Maryland, Vice-President for Medical Affairs at BioJiva has reportedly died by suicide. BioJiva is a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases. The company recently announced the results of its completed multicenter, randomized, double-blind, placebo-controlled pilot Phase 2 clinical trial evaluating RT001.
The company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease). The change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) was the prespecified primary outcome for this clinical research. The medication was administered for a total of 24 weeks.
At 24 weeks, the data demonstrated that patients treated with RT001 experienced less worsening in ALSFRS-R score (a 3.3-point reduction from baseline) in comparison to the larger worsening that was experienced by the placebo group (a 4.6-point drop from baseline). In a similar vein, individuals treated with RT001 saw less worsening in their score on the 40-item ALS assessment questionnaire (ALSAQ40), which was measured as a 13.3-point increase from baseline.
This was in comparison to patients in the placebo group. (a 17.2-point increase from baseline). Despite the fact that these findings point to a possible indication of directional improvement with RT001 treatment, the trial was too small to attain statistical significance due to its limitations.
This randomized Phase 2 pilot research (NCT04762589) was carried out at four ALS Centers of Excellence in Europe over the course of six months. Following this, there was an additional six months of open-label extension in which all patients received RT001 treatment. The participants all had a symptom duration of fewer than three years, which was a requirement for inclusion in the study. Patients who participated in the study had baseline ALSFRS-R scores that ranged from 25 to 44, with a mean of 38.2 (plus or minus 5.0).
39 out of the 43 participants who were enrolled in the study finished the original randomized phase that lasted for 24 weeks, and of those 39, 32 also finished the open-label extension that lasted for an entire year of treatment. At Week 24, patients who were getting RT001 showed a worsening from baseline in ALSFRS-R score of 3.3 points (from 38.2 to 34.9).
Whereas individuals who were receiving placebo showed a worsening from baseline of 4.6 points. (from 38.2 to 33.6). The difference in disease progression was more significant with RT001 treatment for patients who were enrolled with a more severe disease (ALSFRS-R 41 and ALSAQ40 20; n = 33). (3.4-point reduction for RT001 vs. 6.3-point reduction for placebo).
A signal of clinical benefit associated with therapy with RT001 was also seen to be present after 12 months, according to the findings of the open-label extension component of the study that lasted for 24 weeks. After 24 weeks, there was no longer a concurrent placebo that was delivered; instead, comparisons at 12 months were done using data from the PRO ACT Database1, which is a verified and predicted historical control database of the course of ALS.
Patients who were treated with RT001 showed a reduction in their ALSFRS-R score of 7.2 +/- 6.7 points after 12 months, whereas the PRO ACT Database shows an expected worsening on the ALSFRS score after 12 months of 10.1 points. RT001 treatment resulted in a drop of 5.6 +/- 9.2 points on the ALSFRS-R score after 12 months for the most severely affected individuals in the trial (ALSFRS-R score of 41 or higher and ALSAQ40 score of 20 or lower; total number of patients: 14). This indicated an even larger slowing in the progression of disease.