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Kazia Therapeutics study of paxalisib in primary CNS Lymphoma at Dana-Farber enrols first patient

The DFCI study (NCT04906096) is an open-label phase II clinical trial of paxalisib in PCNSL and is expected to recruit up to 25 patients and up to two years to complete.

Kazia Therapeutics Ltd -
Kazia will provide support for the phase-II study, including study drug and a financial grant.

Kazia Therapeutics Ltd’s (ASX:KZA) (FRA:NV9M) (NASDAQ:KZIA) phase-II study of paxalisib in Primary CNS Lymphoma has been initiated at Dana-Farber Cancer Institute in Boston, Massachusetts, with the first patient enrolled to the study.

Lymphoma is a cancer of white blood cells that occurs in the lymphatic system and can spread almost anywhere in the body while primary CNS lymphoma (PCNSL) occurs exclusively in the brain and central nervous system.

The PI3K inhibitor class, to which paxalisib belongs, is well validated in lymphoma outside the brain; three of the four FDA-approved PI3K inhibitors are treatments for forms of lymphoma, but they are assumed ineffective for PCNSL since they cannot cross the blood-brain barrier.

Leading cancer centre

Dana-Farber Cancer Institute (DFCI) is a world-leading cancer treatment and research centre with its principal teaching affiliate being Harvard National Medical School.

DFCI participates in as many as 600 clinical trials at any given time and has been an important contributor to the development of many important new cancer therapies.

"Very sound rationale"

Chief executive officer Dr James Garner said: “We are delighted to see this study underway and congratulate Dr Nayak and her team on getting it launched.

“With paxalisib now in an international pivotal study for glioblastoma, we are increasingly focused on identifying other groups of patients who may benefit from the drug.

“There is a very sound rationale to explore PCNSL in this disease and we are looking forward to seeing the study progress.”

Strong research focus

The DFCI study (NCT04906096) is an open-label phase II clinical trial of paxalisib in PCNSL, and is expected to recruit up to 25 patients, taking up to two years to complete.

Kazia will provide support including study drug and a financial grant.

Principal investigator Dr Lakshmi Nayak said: “Our center has a strong research focus in primary CNS lymphoma.

“We are very interested to explore the potential for a brain penetrant PI3K inhibitor in this disease.

“Patient outcomes in PCNSL remain far from optimal, and there is a pressing need for new treatment options.”

Primary CNS Lymphoma

Lymphoma is a haematological malignancy (blood cancer) that originates from lymphocytes, a type of white blood cell involved in the immune system.

PCNSL is a specific form of the disease that originates in the brain and central nervous system.

Three of the four PI3K inhibitors approved by the US Food and Drug Administration (FDA) are treatments for various forms of lymphoma, provide a strong validation for PI3K as a target in this disease.

Paxalisib is the only PI3K inhibitor in mainstream development with the ability to penetrate the blood-brain barrier, and as such has a unique rationale for development in PCNSL.

The incidence of PCNSL has been increasing with time. Patients are typically in their 60s or older and the disease is slightly more common in men.

Mainstays of treatment comprise chemotherapy and radiotherapy, but recurrence is common and only approximately 30% of patients remain alive five years after diagnosis.

Many of the drugs used to treat lymphoma elsewhere in the body are ineffective in PCNSL due to their inability to cross the blood-brain barrier.

DFCI Clinical Trial

The CNS Lymphoma Center (CNSLC) at Dana-Farber/Brigham and Women's Cancer Center is the first centre of its kind in the world dedicated to providing comprehensive care and research for patients with primary or secondary CNS lymphoma.

With the most advanced treatment options available, including surgery, chemotherapy, stem cell transplant, radiation therapy, immunotherapy and targeted therapies, and with extensive clinical trial options, CNSLC is uniquely qualified to treat patients with CNS lymphoma and advance the outcome of patients with these tumours.

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