- Arena Pharmaceuticals' hypertension drug has scored in mid-stage testing, further supporting the small drugmaker's move away from its former, disappointing obesity treatment franchise.
- In a Phase 2 trial, ralinepag significantly outperformed placebo at bettering pulmonary vascular resistance (PVR). Of the 61 patients with pulmonary arterial hypertension (PAH) who enrolled in the study, those in the investigational arm had PVR improvements of 20% compared to baseline and 30% compared to the control arm.
- Ralinepag is a prostacyclin therapy, which physicians have used as vasodilators for decades. Preston Klassen, Arena's chief medical officer, said these newest results support pushing ralinepag into Phase 3 testing in a Monday statement.
During a July 10 investor call, Arena executives wouldn't give a precise timeline for when they planned to meet with Food and Drug Administration officials regarding Phase 3 testing of ralinepag. But CEO Amit Munshi said his company is trying to get it done "as soon as we can possibly get there, and then we’ll have some clarity on where to go."
Munshi and other company leadership added there are many parameters to consider for late-stage development. Importantly, they will be analyzing how discrepancies between the patient populations in the experimental and control arms may have affected the Phase 2 outcomes.
For instance, there was about a nine-year average age difference between the groups. Patients receiving ralinepag were also better treated with dual combo background therapies consisting of endothelin receptor antagonists and phopshodiesterase-5 inhibitors, which could have masked the efficacy of Arena's drug, according to the company.
With its sights set on advancing ralinepag, Arena cautioned about the toll such actions may take on developing its other candidates.
"Progressing multiple products into Phase 3 from where we are today might be difficult," Munshi said. "It’s an ongoing discussion, we want to make sure we turn over multiple data cards ..."
Arena's interest in progressing ralinepag makes sense, given that there's a lot of money in PAH drugs.
Just look at Actelion and its suite of products for the rare disease. Last year, sales for Opsumit (macitentan) grew 57% to 831 million Swiss francs ($861 million) while the company raked in 245 million francs ($253 million) from its newly launched treatment Uptravi (selexipag).
Overall, Actelion had revenues of 2.41 billion francs ($2.5 billion) in 2016, making it easier to understand why Johnson & Johnson agreed to shell out $30 billion to acquire the Swiss drugmaker.
Yet for Arena, the topline data represent more than just a potentially lucrative medicine. They also reaffirm the its decision to abandon a highly anticipated — yet ultimately disappointing — obesity drug.
The San Diego-based company's Belviq (lorcaserin) first received the FDA's approval in 2012 as an additive for weight loss regimens of patients with high body mass indexes. Though investors initially saw promise in drug, it was largely a flop. In the first quarter, for example, Arena had just $2.7 million in net product sales.
As such, Arena has worked to diversify its pipeline as a means of bringing on more reliable and profitable products. The drugmaker maintains worldwide rights to the immuno-regulator etrasimod, which is in mid-stage testing for ulcerative colitis, pyoderma gangrenosum and a couple other indications, and APD371, a CB2 receptor agonist for the treatment of Crohn's pain. It also has drug partnerships with Axovant and Boehringer Ingelheim.
More complete data for ralinepag will be presented at an upcoming medical conference, according to the July 10 statement. In the meantime, Arena and its investors seem uplifted by the topline results and the drug's potential against other PAH medications such as Uptravi and Flolan (epoprostenol sodium).
Arena stock rose more than 45% in after hours trading on Monday.