Biotech Beat: Arena Pharmaceuticals, Inc. (ARNA) and Exelixis, Inc. (EXEL)


Arena Pharmaceuticals, Inc. (NASDAQ:ARNA) investors have a smile on their faces Monday evening, after the drug maker announced positive Phase 2 results for ralinepag, an investigational, long-acting, orally administered prostacyclin receptor agonist under development for the treatment of pulmonary arterial hypertension (PAH). In this 61-patient study, the primary efficacy analysis demonstrated a statistically significant absolute change from baseline in pulmonary vascular resistance (PVR) compared to placebo. Ralinepag also demonstrated numerical improvement in 6-minute walk distance (6MWD).

Arena Pharmaceuticals shares reacted to the news, jumping nearly 40% to $25.65 in after-hours trading.

Ralinepag improved median PVR by 163.9 dyn.s.cm-5 from baseline compared to a 0.7 dyn.s.cm-5 worsening from baseline in the placebo arm (P=0.02).  Patients treated with ralinepag had a 29.8% improvement in PVR compared to the placebo arm (P=0.03) and a 20.1% improvement in PVR compared to baseline. Additionally, adverse events observed in the study were consistent with other prostacyclin treatments for the management of PAH, with headache, nausea, diarrhea, jaw pain and flushing being the most commonly reported adverse events. The company plans to present full study results at future medical congresses.

“The positive outcome of this Phase 2 trial in a contemporary PAH patient population is an important milestone in the development of ralinepag for the treatment of patients suffering from this grievous illness,” stated Preston Klassen, M.D., MHS, Executive Vice President, Research and Development and Chief Medical Officer of Arena.  “It is exciting to see the positive nonclinical pharmacological profile translating into potentially the first oral prostacyclin therapy that may approach consistent therapeutic levels without the complexity of parenteral (IV) therapy.  These data give us confidence to move expeditiously toward a Phase 3 clinical program.”

Vallerie McLaughlin, M.D., Kim A. Eagle MD Endowed Professor of Cardiovascular Medicine at the University of Michigan and Director of the Pulmonary Hypertension Program, added, “PAH is a complex and serious disease, often with a poor prognosis despite the use of currently available treatments. New therapeutic options to manage patients with PAH are needed. The results of this Phase 2 study of ralinepag, in patients already receiving, in most cases, multiple background therapies, showed a clinically meaningful improvement in PVR, a well-established indicator of treatment benefit, believed to be correlated with long-term clinical outcomes in patients with PAH.”

On the ratings front, ARNA has been the subject of a number of recent research reports. In a report issued on June 29, Cantor analyst William Tanner reiterated a Buy rating on the stock, with a price target of $37.00, which represents a potential upside of 90% from where the stock is currently trading. On May 19, Leerink Swann’s Joseph Schwartz initiated coverage with a Buy rating on the stock and has a price target of $5.00.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, William Tanner and Joseph Schwartz have a yearly average return of 12.5% and 15.0% respectively. Tanner has a success rate of 57% and is ranked #580 out of 4594 analysts, while Schwartz has a success rate of 53% and is ranked #237.

Exelixis, Inc. (NASDAQ:EXEL) and Bristol-Myers Squibb Co (NYSE:BMY) announced today the initiation of a phase III study, CheckMate 9ER, of Cabometyx in combination with Opdivo (nivolumab) in patients with previously untreated, advanced or metastatic renal cell carcinoma (RCC). The primary endpoint for the trial is progression-free survival (PFS). Exelixis is conducting this study based on scientific evidence that Cabometyx results in an active immune tumor environment where a checkpoint inhibitor could play a synergistic role.

“There is strong scientific evidence showing that CABOMETYX results in a more immune permissive tumor environment, and we are eager to determine if combining these active agents with complementary and potentially cooperative mechanisms of action has the potential to further improve patient outcomes,” said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. “We are excited to initiate this first clinical trial from our broad development program with Bristol-Myers Squibb looking at the potential of Opdivo in combination with CABOMETYX, with or without Yervoy, in a variety of tumor types.”

“While existing therapies have improved outcomes for some patients with advanced or metastatic kidney cancer, high rates of relapse and disease progression demonstrate a need for additional therapeutic options, especially among poor and intermediate risk patients,” said Fouad Namouni, M.D., Head of Development, Oncology, Bristol-Myers Squibb. “Combination therapy with agents that target different and complementary pathways—in this case, the combination of immune checkpoint inhibitors and tyrosine kinase inhibitors—may be a potential new approach for these patients.”