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AXS-02 (oral zoledronate)

Axsome Therapeutics, Inc. · Phase 3 active Small molecule

AXS-02 (oral zoledronate) is a Bisphosphonate Small molecule drug developed by Axsome Therapeutics, Inc.. It is currently in Phase 3 development for Osteoporosis, Bone metastases and hypercalcemia of malignancy (investigational oral formulation).

AXS-02 is an oral formulation of zoledronate, a bisphosphonate that inhibits osteoclast-mediated bone resorption by blocking farnesyl pyrophosphate synthase in the mevalonate pathway.

AXS-02 is an oral formulation of zoledronate, a bisphosphonate that inhibits osteoclast-mediated bone resorption by blocking farnesyl pyrophosphate synthase in the mevalonate pathway. Used for Osteoporosis, Bone metastases and hypercalcemia of malignancy (investigational oral formulation).

At a glance

Generic nameAXS-02 (oral zoledronate)
SponsorAxsome Therapeutics, Inc.
Drug classBisphosphonate
TargetFarnesyl pyrophosphate synthase
ModalitySmall molecule
Therapeutic areaBone metabolism / Oncology
PhasePhase 3

Mechanism of action

Zoledronate is a potent bisphosphonate that binds to hydroxyapatite in bone and is taken up by osteoclasts, where it inhibits farnesyl pyrophosphate synthase, disrupting the mevalonate pathway and leading to osteoclast apoptosis. This reduces bone resorption and is used to treat conditions characterized by excessive bone loss or abnormal bone metabolism. The oral formulation (AXS-02) aims to improve patient convenience and compliance compared to intravenous administration.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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Frequently asked questions about AXS-02 (oral zoledronate)

What is AXS-02 (oral zoledronate)?

AXS-02 (oral zoledronate) is a Bisphosphonate drug developed by Axsome Therapeutics, Inc., indicated for Osteoporosis, Bone metastases and hypercalcemia of malignancy (investigational oral formulation).

How does AXS-02 (oral zoledronate) work?

AXS-02 is an oral formulation of zoledronate, a bisphosphonate that inhibits osteoclast-mediated bone resorption by blocking farnesyl pyrophosphate synthase in the mevalonate pathway.

What is AXS-02 (oral zoledronate) used for?

AXS-02 (oral zoledronate) is indicated for Osteoporosis, Bone metastases and hypercalcemia of malignancy (investigational oral formulation).

Who makes AXS-02 (oral zoledronate)?

AXS-02 (oral zoledronate) is developed by Axsome Therapeutics, Inc. (see full Axsome Therapeutics, Inc. pipeline at /company/axsome-therapeutics-inc).

What drug class is AXS-02 (oral zoledronate) in?

AXS-02 (oral zoledronate) belongs to the Bisphosphonate class. See all Bisphosphonate drugs at /class/bisphosphonate.

What development phase is AXS-02 (oral zoledronate) in?

AXS-02 (oral zoledronate) is in Phase 3.

What are the side effects of AXS-02 (oral zoledronate)?

Common side effects of AXS-02 (oral zoledronate) include Osteonecrosis of the jaw, Atypical femoral fractures, Renal impairment, Hypocalcemia, Gastrointestinal upset.

What does AXS-02 (oral zoledronate) target?

AXS-02 (oral zoledronate) targets Farnesyl pyrophosphate synthase and is a Bisphosphonate.

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