Bristol-Myers Squibb Co (NYSE:BMY) announced the U.S. Food and Drug Administration (FDA) has approved Opdivo injection, for intravenous use for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. The recommended dose for mUC is 240 mg administered as an intravenous infusion over 60 minutes every two weeks until disease progression or unacceptable toxicity. In the CheckMate -275 trial, 19.6% (95% CI: 15.1-24.9; 53/270) of patients responded to treatment with Opdivo. The percentage of patients with a complete response was 2.6% (7/270) and the percentage of patients with a partial response was 17% (46/270). Among responders, the median duration of response was 10.3 months (range: 1.9+-12.0+ months). The median time to response was 1.9 months (range: 1.6-7.2).

Opdivo is associated with the following Warnings and Precautions including immune-mediated: pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, skin adverse reactions, encephalitis, other adverse reactions; infusion reactions; and embryo-fetal toxicity.

“Our goal to help more patients is further realized in today’s approval for Opdivo in this population and we are excited that our Immuno-Oncology therapy is now an option and potential hope for these patients,” said Chris Boerner, president of U.S. Commercial, Bristol-Myers Squibb. “This is evidence of our commitment to Immuno-Oncology and to bringing therapies, like Opdivo, to more and more patients in need of additional choices.”

The FDA granted the application priority review and previously granted Breakthrough Therapy Designation to Opdivo for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

“Most people don’t know how common bladder cancer is and that it is the fifth most diagnosed cancer. That’s why we are dedicated to raising awareness and supporting research efforts that may offer more treatment options to patients who need them,” said Stephanie Chisolm, director of Education and Research at Bladder Cancer Advocacy Network. “This approval is another exciting step forward for the bladder cancer community and provides needed hope to patients and their families.”

Approval Based on Notable Objective Response Rate

CheckMate -275 is a Phase 2, open-label, single-arm, multicenter study evaluating Opdivo in patients with locally advanced or mUC who have disease progression during or following treatment with a platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.2 In this study, 270 patients received Opdivo 3 mg/kg administered intravenously every two weeks until disease progression or unacceptable toxicity. The recommended dose is 240 mg administered as an intravenous infusion over 60 minutes every two weeks until disease progression or unacceptable toxicity. The primary endpoint was confirmed objective response rate (ORR) as defined by an independent radiographic review committee (IRRC). The median age of patients participating in the study was 66 years (range: 38-90), and 29% of patients had received ≥2 prior systemic regimens in the metastatic setting prior to enrolling in the study. Patients were included in the trial regardless of their PD-L1 status.

In the trial, efficacy was evaluated in 270 patients with 6 months follow-up by confirmed ORR as determined by an IRRC, Opdivo demonstrated an ORR of 19.6% (95% CI: 15.1-24.9). Additional efficacy breakdown by PD-L1 expression were as follows:

                   
Outcome, % (n)    

All Patients
(n=270)

   

PD-L1 <1%
(n=146)

    PD-L1 ≥1%
(n=124)
 

Confirmed ORR by IRRC
(95% CI)

    19.6% (53)
(15.1-24.9)
    15.1% (22)
(9.7-21.9)
    25.0% (31)
(17.7-33.6)
 
Complete Response Rate     2.6% (7)     0.7% (1)     4.8% (6)  
Partial Response Rate     17.0% (46)     14.4% (21)     20.2% (25)  
                     

“As an oncologist, a nearly twenty-percent response rate in advanced and metastatic bladder cancer is extremely encouraging and clinically meaningful in this patient population,” said Dr. Jonathan E. Rosenberg, MD, Memorial Sloan Kettering Cancer Center.

Selected Safety Profile

The safety of Opdivo has been studied in 270 patients in the CheckMate -275 study. Patients were treated with Opdivo for a median of 3.3 months (range: 0-13.4+). In this study, serious adverse events occurred in 54% of patients. The most frequent serious adverse events reported in at least 2% of patients were urinary tract infection, sepsis, diarrhea, small intestine obstruction, and general physical health deterioration. The most common adverse reactions (≥20%) were fatigue (46%), musculokeletal pain (30%), nausea (22%), and decreased appetite (22%). Opdivo was discontinued due to adverse reactions in 17% of patients, and 46% of patients had a dose delay for an adverse reaction. Treatment-related death occurred in four patients due to pneumonitis or cardiovascular failure.

Shares of Bristol Myers Squibb closed today at $50.50, up $1.21 or 2.45%. BMY has a 1-year high of $77.12 and a 1-year low of $46.01. The stock’s 50-day moving average is $55.81 and its 200-day moving average is $57.53.

On the ratings front, BMY has been the subject of a number of recent research reports. In a report issued on January 30, Credit Suisse analyst Vamil Divan reiterated a Hold rating on BMY, with a price target of $52, which represents a slight upside potential from current levels. On January 27, Jefferies’ Jeffrey Holford reiterated a Buy rating on the stock and has a price target of $60.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Vamil Divan and Jeffrey Holford have a yearly average return of 8.4% and 5.7% respectively. Divan has a success rate of 51% and is ranked #587 out of 4373 analysts, while Holford has a success rate of 59% and is ranked #668.

Overall, 6 research analysts have assigned a Hold rating and 3 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $56.67 which is 12.2% above where the stock opened today.

Bristol-Myers Squibb Co. engages in the discovery, development, licensing, manufacturing, marketing, distribution, and sale of biopharmaceutical products.