Shares of small-cap biopharma Acceleron Pharma Inc (NASDAQ: XLRN) are advancing to an all-time high Tuesday following a data readout.
Primary, Secondary Endpoints Met
Acceleron said a Phase 2 study dubbed PULSAR that evaluated its sotatercept in patients with pulmonary arterial hypertension, or PAH, met its primary and key secondary endpoints.
The experimental drug showed a statistically significant reduction in pulmonary vascular resistance – the primary endpoint - at week 24 versus placebo.
The study also achieved the key secondary endpoint of significant improvements in six-minute walk distance, and other secondary endpoints, including amino-terminal brain natriuretic propeptide, and WHO functional class. It was also found to be generally well tolerated.
The PULSAR study is a double-blind, placebo-controlled study in which 106 patients were randomized to receive placebo 0.3 mg/kg of sotatercept, or 0.7 mg/kg of sotatercept subcutaneously, every 21 days in combination with stable background PAH-specific therapies over a 24-week treatment period.
High Unmet Need
PAH is a chronic, rapidly progressing disorder which leads to elevated blood pressure in pulmonary circulation. This in turn limits physical activity and results in heart failure and reduced life expectancy, with the 5-year survival rate at 57%.
"PAH is a debilitating disease of high unmet medical need, so we're encouraged by these data that signal that sotatercept could deliver added benefit to patients," said CEO Habib Dable.
Positive development of Sotatercept provides Acceleron with two source of value-expanding the Acceleron story beyond the prevailing consensus of being "just a luspatercept royalty stream" and increasing its M&A attractiveness, Credit Suisse analyst Martin Auster said in a note.
The analyst shoots for an approval timeframe of 2024 and projects peak sales of $800 million, with an opportunity to achieve $1 billion+ at peak as longer-term data mature and the profile becomes fully elucidated.
Acceleron said it looks forward to upcoming interactions with authorities.
The company also noted that 97 of the 106 patients enrolled in the PULSAR trial are participating in the 18-month extension period of the trial.
Acceleron's stock was soaring 51% to $80 per share at time of publication.