2021-11-23| Trials & Approvals

FDA Approves Aadi Bioscience’s First Product to Treat Rare Cancer

by Rajaneesh K. Gopinath
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Aadi Bioscience is a biopharma focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes. Earlier today, the California-based company received FDA approval for its very first product, Fyarro (sirolimus protein-bound particles for injectable suspension), for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). With this, the drug becomes the first and only FDA-approved treatment for advanced malignant PEComa in adults.

“We are thrilled to have received full FDA approval of Fyarro. The approval of Fyarro is a momentous event not just for Aadi but, importantly, for advanced malignant PEComa patients. We reiterate that all of us at Aadi are incredibly grateful to all of the people with advanced malignant PEComa, their families and caregivers, as well as the healthcare professionals who made the Fyarro clinical studies possible,” said Neil Desai, Founder, CEO, and President of Aadi.

Shares of Aadi were up 49% to $37.98 in premarket trading since the news.

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Ultra-Rare Form of Sarcoma

Malignant PEComa is an ultra-rare and aggressive form of sarcoma with a strong female predominance. It is defined as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,’ are a rare subset of soft-tissue sarcomas, with an undefined cell of origin.

According to estimates that there are approximately 100-300 new patients per year in the U.S. This rare cancer may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract with a female predominance) and can have an aggressive clinical course including distant metastases and ultimately death. Currently, there are no approved drugs for this disease.

Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of the mTOR pathway, making it a rational therapeutic target for this disease. Fyarro is an mTOR inhibitor indicated for the treatment of adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

“The approval of Fyarro, the first approved drug for advanced malignant PEComa, an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease,” added Andrew Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute and the Principal Investigator in the pivotal AMPECT registrational trial.

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The FDA approval is based upon results from the Phase 2 registrational AMPECT trial, which found the overall response rate of the Fyarro arm to be 39% (12/31), with 2 patients achieving a Complete Response after prolonged follow up. Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months, and 58% had a response lasting greater than or equal to 2 years.

“In our AMPECT trial, Fyarro demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, with an acceptable and manageable safety profile. This is a drug that will be welcomed by the physician community as the only approved therapeutic option for patients with advanced malignant PEComa,” Dr. Wagner added.

“Patients living with locally advanced or metastatic PEComa are in urgent need of new treatment options. The approval of Fyarro is a significant advancement for treating patients with this disease. Treating sarcoma patients in my practice, I have seen the need for a therapy that addresses the specific molecular alterations of advanced malignant PEComa. I am encouraged that Fyarro provided a clinically meaningful benefit in overall response rate, with some patients responding for up to several years,” said Robert G. Maki, M.D., Ph.D., Clinical Director of the Sarcoma Program, and Professor of Medicine at the University of Pennsylvania.

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