Future steps, including plans for global studies, are currently being evaluated by Biogen, with the final results from the phase 2a study expected to be released in an upcoming scientific forum.
Following positive data from a phase 2a study, Biogen has announced that it will exercise its option to acquire TMS-007, an investigational drug for acute ischemic stroke, from TMS Co. Biogen also announced it will evaluate the next steps for the clinical development of the treatment, including plans for global studies.
Results from the phase 2a study showed that the treatment met its primary safety objective with no incidence of symptomatic intracranial hemorrhage (sICH), as well as demonstrated positive impacts on both blood vessel reopening in the brain and patient functional recovery. Final data results from the study are expected to be presented at a future scientific forum.
As part of the acquisition, Biogen will make a 1-time, $18-million payment, with TMS eligible to receive up to an additional $335 million in potential post-acquisition development and commercial payments should TMS-007 achieve certain developmental and sales thresholds. Biogen noted that it will be solely responsible for the costs and expenses related to the development, manufacturing, and commercialization of TMS-007 following the acquisition.
"We are encouraged by these results and made the decision to acquire TMS-007 based on the totality of the safety, imaging, and clinical outcome data from the phase 2a study,” Alfred Sandrock Jr, MD, PhD, head of research and development, Biogen, said in a statement. “It has been almost 25 years since the last thrombolytic agent was approved for acute ischemic stroke and we believe this novel investigational drug may expand the number of eligible patients who could potentially receive thrombolytic therapy and thus have a higher chance of functional independence after stroke.”
TMS-007 is a small molecule plasminogen activator with a proposed novel mechanism of action associated with breaking down blood clots and potentially inhibiting local inflammation at the site of thrombosis.
Additional data from the multicenter, singe-administration, double-blind, randomized, placebo-controlled, ascending dose trial showed that treatment with TMS-007 had a significant improvement on the secondary end point of functional independence at 90 days. In total, 40% of patients who received the therapy achieved scores of 0 or 1 on the modified Rankin Scale (mRS), compared to 18% of patients who received placebo (P = .05).
These improvements were also supported by objective angiographic evidence of recanalization in the subset of patients with a visible occlusion receiving TMS-007. Using magnetic resonance angiography, researchers concluded that the recanalization rate was 58.3% (14 of 24) for patients who received the investigational treatment compared to 26.7% (4 of 15) for placebo-treated patients (odds ratio, 4.23 [95% CI, 0.99-18.07]).
The study featured 90 participants in Japan (TMS-007: n = 52; placebo: n = 38) who were dosed up to 12 hours after the onset of stroke symptoms. The average time to treatment was 9.5 and 9.3 hours for patients who received TMS-007 and placebo, respectively. All patients who received TMS-007 were dosed beyond the time window of approved thrombolytic agents.