Pursuing first-in-class therapies targeting the PAM (PI3K/AKT/mTOR) pathway

The PI3K/AKT/mTOR pathway has been implicated in breast, prostate, endometrial, ovarian, and hematological cancers, among others. We are currently developing our lead drug candidate, gedatolisib, a PAM pathway inhibitor, to treat advanced breast cancer and metastatic castration resistant prostate cancer.

Gedatolisib pipeline

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The safety and efficacy of investigational agents and/or investigational use of approved products have not been established. Ibrance® is a registered trademark of Pfizer, Inc. Nubeqa® is a registered trademark of Bayer AG. ER, estrogen receptor; HER2, human epidermal growth factor receptor 2; PIK3CA, phosphatidylinositol-4,5-biphosphate 3-kinase catalytic subunit alpha; MT, mutated; HNSCC, head and neck squamous cell carcinoma; SCLC, squamous cell lung cancer; mCRPC, metastatic castration resistant prostate cancer.

Our lead drug candidate, gedatolisib, offers a new approach to inhibiting the PAM (PI3K/AKT/mTOR) pathway

To overcome the challenges of inhibiting the PAM pathway, we are developing a potential first-in-class multi-target PAM inhibitor that:

  • Targets all Class I PI3K isoforms and mTORC1 and mTORC2
  • Offers a favorable pharmacokinetic profile designed to limit the toxicities typically associated with single-target PI3K, AKT, or mTOR inhibitors

Gedatolisib is an investigational pan-class I isoform PI3K and mTOR inhibitor with low nanomolar potency for the p110α, p110β, p110γ, and p110δ PI3K isoforms, as well as mTORC1 and mTORC2, that induces comprehensive blockade of the PAM pathway. Its mechanism of action and pharmacokinetic properties are highly differentiated from other currently approved and investigational therapies that target PI3Kα, AKT, or mTORC1 alone. We believe this will enable gedatolisib to treat a broader patient population than these single-target inhibitors

Learn how and why we target the PAM pathway

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Ex vivo studies found that gedatolisib inhibited higher levels of PI3K/AKT/mTOR involved signaling activity than single target PI3K, AKT, or mTOR therapies, regardless of PIK3CA mutational status. Superior drug synergy when combined with other targeted therapies has also been demonstrated.

Initial clinical development is focused on breast and prostate cancers

Gedatolisib’s initial clinical development program is focused on the treatment of patients with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 -negative (HER2-), advanced or metastatic breast cancer and patients with metastatic castration resistant prostate cancer. Unlike PI3K or AKT therapies that are only approved to treat patients with PIK3CA or PTEN mutations, gedatolisib is under development for patients with and without PIK3CA mutations.

Learn about VIKTORIA-1, evaluating 2nd line treatment in advanced breast cancer

Discover VIKTORIA-2, evaluating 1st line treatment in advanced breast cancer

Explore our prostate cancer trial

Expanded Access Program

Celcuity believes the best way for patients to access medicines prior to approval is through participation in clinical trials. To bring our new treatments to patients, we conduct clinical trials to assess the safety and efficacy of our investigational medicines. This information is submitted to regulatory agencies, such as the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Health Canada, to enable approval for patient use. We are currently conducting clinical trials to assess the safety and efficacy of an investigational drug to be used as treatment for metastatic breast cancer, as well as for advanced prostate cancer.

Making our investigational therapy available through an Expanded Access Program is a complex matter that we have considered carefully. Providing early access to an investigational therapy can involve unknown risks, as well as an unfounded assumption of therapeutic benefit. Furthermore, an Expanded Access Program may make it harder to complete the studies necessary for the regulatory review and approval process that is ultimately the fastest path to making new therapies available to as many patients as possible.

Based on these considerations, Celcuity’s Expanded Access Program is only being offered to patients who initially received our investigational medicine as a participant in a clinical trial that is no longer open. Participation in our Expanded Access Program will end once the investigational medicine is approved and becomes commercially available in the patient’s country.

We encourage patients to speak with their physicians about the possibility of enrolling in clinical trials to gain access to investigational therapies.