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U.S. Food and Drug Administration Approves Bristol Myers Squibb’s Pomalyst (pomalidomide) for AIDS-Related and HIV-Negative Kaposi Sarcoma

PRINCETON, N.J.--(BUSINESS WIRE)--May 15, 2020 -- 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. See additional Important Safety Information below.

“Pomalyst has shown positive results in Kaposi sarcoma patients, regardless of their HIV status,” said Robert Yarchoan, M.D., Chief of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institute (NCI). “Also, it provides a therapy that is taken orally and works by a different mechanism of action than the cytotoxic chemotherapy drugs generally used to treat Kaposi sarcoma.”

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 Pomalyst (pomalidomide)

Indications

Pomalyst ® is a thalidomide analogue indicated for the treatment of adult patients:

• in combination with dexamethasone, for patients with multiple myeloma (MM) who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of the last therapy.

• with AIDS-related Kaposi sarcoma (KS) after failure of highly active antiretroviral therapy (HAART) or in patients with KS who are HIV-negative. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase quality, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

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. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

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

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol-Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2019, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol-Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

1 Schneider JW, Dittmer DP. Diagnosis and treatment of Kaposi sarcoma. Am J Clin Dermatol. 2017;18(4):529-539.

2 Polizzotto MN, Uldrick TS, Wyvill KM, et al. Pomalidomide for symptomatic Kaposi's sarcoma in people with and without HIV infection: a Phase I/II study. J Clin Oncol. 2016;34(34):4125-4131.

3 POMALYST (pomalidomide). Bristol Myers Squibb Pharmaceutical Corporation. Full prescribing information. 5/2020.

4 American Cancer Society. What is Kaposi Sarcoma? Available at: https://www.cancer.org/cancer/kaposisarcoma/about/what-is-kaposi-sarcoma.html. Accessed April 2020.

5 American Cancer Society. Key Statistics About Kaposi Sarcoma. Available at: https://www.cancer.org/cancer/kaposi-sarcoma/about/what-is-key-statistics.html. Accessed April 2020.

6 ClincalTrials.gov. Pomalidomide for Kaposi Sarcoma in People With or Without HIV. Available at: https://clinicaltrials.gov/ct2/show/NCT01495598. Accessed April 2020.

Source: Bristol Myers Squibb

Posted: May 2020

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