Shares of Athersys (ATHX) are skyrocketing today, up 19% in pre-market trading, after the clinical-stage biotech announced encouraging summary results from its exploratory clinical study of the intravenous administration of MultiStem® cell therapy to treat patients who are suffering from acute respiratory distress syndrome (ARDS).
The study results provide further confirmation of tolerability and a favorable safety profile associated with MultiStem treatment. Importantly, MultiStem treatment was associated with lower mortality and a greater number of ventilator-free and intensive care unit (ICU) free days in the first month following diagnosis relative to patients receiving placebo. Furthermore, analysis of initial biomarker data reflects lower levels of inflammatory markers/cytokines following MultiStem treatment, an expected mechanism of action in this patient population.
Patients in the exploratory study were evaluated through 28 days for the primary clinical assessment and will be further assessed through a one-year follow-up period. Data highlights from the initial evaluation include the following results from the double-blind, randomized, placebo-controlled portion of the study:
- Lower mortality of 25% in the MultiStem treatment group vs. 40% in the placebo group;
- 40.2% higher ventilator-free (VF) days, (12.9 VF days in the MultiStem treatment group vs. 9.2 VF days for the placebo group);
- 27.2% higher ICU-free days, (10.3 days in MultiStem subjects vs. 8.1 days for subjects receiving placebo);
- In more severe ARDS patients (as evident in a prospectively defined analysis), the difference between MultiStem treatment and placebo was greater – 25% mortality in MultiStem group vs. 50% in placebo group, 14.6 VF days in MultiStem group vs. 8.0 VF days in placebo group, and 11.4 ICU-free days in MultiStem group versus 5.9 ICU-free days in placebo group; and
- MultiStem treatment was well tolerated in this very sick ARDS patient population, with no serious adverse events related to administration.
The study was designed to evaluate the impact of MultiStem treatment in subjects with acute onset of moderate to severe ARDS and was conducted at sites in the United States and United Kingdom. The study included two parts – a small initial dose confirmation phase, followed by the larger double-blinded, placebo-controlled and randomized phase (Phase 2a portion). Treatment was required to begin within four days of ARDS diagnosis with an average treatment time of approximately two days from the diagnosis. Six subjects were treated with MultiStem in the initial portion of the study, and in the Phase 2a portion of the study, 20 subjects were treated with an intravenous (IV) administration of 900 million MultiStem cells and 10 subjects received IV placebo. As disclosed previously, the study was not powered for the efficacy outcomes.
Athersys will continue to evaluate the data as the one-year follow-up period is completed for all patients in the trial. Athersys and the study investigators plan to present more detailed and comprehensive results at a medical science conference after additional analyses.
“We believe this exploratory study met all of its key objectives and view it as an important indication of the potential relevance of MultiStem in another critical care area where there is substantial unmet medical need,” commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. “The consistent pattern of results suggesting a beneficial effect on mortality and key clinical parameters provides us with confidence that this is another condition where administration of MultiStem can have a meaningful therapeutic impact.
“We also believe these results provide strong support for the ARDS trial that our partner, HEALIOS K.K. (Healios), is authorized to conduct in Japan under the recently implemented regenerative medicine regulatory framework designed to expedite development. Healios is actively working towards initiation of this study, and we continue to support their efforts, as well as advance our other key programs,” concluded Dr. Van Bokkelen.
The Street Verdict
Overall, Athersys has had 3 bullish analysts in its corner over the last 12 months. Importantly, the 12-month average price target of $7.66 showcases 359% in upside potential for the stock. (See ATHX’s price targets and analyst ratings on TipRanks)