PRINCETON - Bristol Myers Squibb (NYSE: BMY) today announced that Pomalyst (pomalidomide) was approved by the U.S. Food and Drug Administration (FDA) for patients with AIDS-related Kaposi sarcoma whose disease has become resistant to highly active antiretroviral therapy (HAART), or in patients with Kaposi sarcoma who are HIV-negative.3 Patients with AIDS-related Kaposi sarcoma should continue HAART for their HIV as recommended by their physician.

Pomalyst was granted accelerated approval, Breakthrough Therapy designation and Orphan Drug designation in these indications based on overall response rates observed in a Phase 1/2 open label, single-arm clinical trial (12-C-0047). Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Kaposi sarcoma is a rare form of cancer that usually presents as skin lesions, but can also develop in several other areas of the body including the lungs, lymph nodes and digestive system. The disease occurs at a rate of about 6 cases per million people each year in the United States, and mostly affects people who are immunocompromised.1,4,5 This oral therapy is the first new treatment option available for those with Kaposi sarcoma in more than 20 years.1,2

'Patients with Kaposi sarcoma have had few options to manage their disease for two decades,' said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. 'We're excited that the additional research into Pomalyst in this rare disease area has resulted in our ability to provide a much-needed oral treatment option for patients.'

As described in the Boxed Warnings of the prescribing information, Pomalyst can cause fetal harm and is contraindicated in females who are pregnant. Pomalyst is only available through a restricted distribution program, Pomalyst REMS. Deep vein thrombosis, pulmonary embolism, myocardial infarction and stroke can occur in patients treated with Pomalyst and thromboprophylaxis is recommended.

About 12-C-0047

The approval of Pomalyst was based on the findings of a Phase 1/2 open-label, single-arm study conducted evaluating the safety, pharmacokinetics and efficacy of Pomalyst in patients with HIV-positive and HIV-negative symptomatic Kaposi sarcoma, the majority of whom had advanced disease.3 The study was performed under a Cooperative Research and Development Agreement (CRADA) by a team led by Dr. Robert Yarchoan of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institute (NCI).6

A total of 28 patients (18 HIV-positive, 10 HIV-negative) received 5 mg of Pomalyst, once daily for 21 of 28-day cycles, until disease progression or unacceptable toxicity. All HIV-positive patients continued concomitant highly active antiretroviral therapy (HAART). The trial excluded patients with symptomatic pulmonary or visceral Kaposi sarcoma, history of venous or arterial thromboembolism, or procoagulant disorders. Patients received thromboprophylaxis with aspirin 81 mg once daily throughout therapy. The median time to first response was 1.8 months (0.9 to 7.6).3

The primary endpoint of the study was overall response rate (ORR), which included complete response (CR), clinical complete response (cCR) and partial response (PR), as assessed by investigators according to the AIDS Clinical Trial Group (ACTG) Oncology Committee response criteria for Kaposi sarcoma. For all patients, the ORR was 71% (95% CI: 51, 87) with 14% (4/28) of patients achieving CR and 57% (16/28) of patients achieving a PR, respectively. The median duration of response for all patients was 12.1 months (95% CI: 7.6, 16.8). Additionally, half (50%) of patients who responded maintained a response at more than 12 months with Pomalyst.3

The most common adverse reactions including laboratory abnormalities (30%) are decreased absolute neutrophil count or white blood cells, elevated creatinine or glucose, rash, constipation, fatigue, decreased hemoglobin, platelets, phosphate, albumin, or calcium, increased ALT, nausea and diarrhea.

Adverse reactions were evaluated in 28 patients who received treatment with Pomalyst. Adverse reactions (20%) included maculopapular rash (71%), constipation (71%), fatigue (68%), nausea (36%), diarrhea (32%), cough (29%), dyspnea (29%), peripheral edema (29%), upper respiratory tract infection (29%), muscle spasms (25%), hypothyroidism (21%), dry skin (21%) and chills (21%). Grade 3 or 4 adverse reactions included maculopapular rash (3.6%), diarrhea (3.6%) and peripheral edema (3.6%). Grade 3 or 4 laboratory abnormalities (5%) worsening from baseline included decreased absolute neutrophil count (50%), decreased phosphate (25%), elevated glucose (7%) and elevated creatine kinase (7%).

Permanent discontinuation due to an adverse reaction occurred in 11% (3/28) of patients who received Pomalyst. No new safety signals were identified, and the safety of Pomalyst in Kaposi sarcoma was consistent with the known safety profile of Pomalyst in approved indications.3

About Kaposi sarcoma

Kaposi sarcoma is a rare form of cancer that usually presents as skin lesions, but can also develop in several other areas of the body including the lungs, lymph nodes and digestive system. Kaposi sarcoma is caused by Kaposi sarcoma-associated herpesvirus, also called human herpesvirus-8, and most commonly arises in persons infected with HIV who are immunocompromised. Although the use of combination anti-retroviral treatments (cART or HAART) has reduced the incidence of Kaposi sarcoma in the United States, it still occurs at a rate of approximately 6 cases per million people each year.5 The disease is more prevalent in areas of the world where HIV treatments are less available, and where more persons are infected with Kaposi sarcoma-associated herpesvirus, such as sub-Saharan Africa, and in some countries is the most common tumor in men overall.5

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

Bristol Myers Squibb Cautionary Statement Regarding Forward-Looking Statements

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