Clementia Initiates Pivotal Phase 3 MOVE Trial for Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva
Patient Dosing Has Commenced; Registration Study to Enroll 80 Patients Worldwide
Company to Host Conference Call at
“FOP is a rare, devastating, and debilitating disease for patients. It causes great hardship to families and caregivers. We are very excited to be part of this first-ever multicenter Phase 3 clinical trial for patients with FOP, for which there are no approved treatments,” said
UCSF is the first site to enroll a patient in the MOVE Trial, which will be conducted at approximately 20 centers in 16 countries around the world (
“The initiation of this study is a landmark event for Clementia, fully transitioning us into a late-stage clinical organization,” commented
“Palovarotene is rapidly advancing through clinical development and could be the first treatment to be approved for patients with FOP,” said
About the Phase 3 MOVE Trial
All patients in the MOVE Trial receive a single daily dose of palovarotene, with increased dosing at the time of a flare-up. Data from the FOP patients in Clementia’s Natural History Study (NHS) will serve as the control. Patients will be treated with palovarotene for 24 months, with three planned interim analyses. The first interim analysis will occur when the first 35 patients have completed their one-year CT scans, with the next two interim analyses coming 6 and 12 months afterwards. Clementia expects top-line results from the study in late 2020, with the first interim readout expected in early 2019.
The primary efficacy endpoint of the MOVE Trial is the annualized change in new HO volume as assessed by low-dose WBCT scan (excluding head) compared to untreated patients from the NHS. Secondary endpoints include the proportion of patients with any new HO, the change in the number of body regions with new HO, the proportion of patients reporting flare-ups and the rate of flare-up occurrence. Exploratory endpoints include functional assessments of joint function and changes in patient-reported physical function. Safety evaluations include adverse events, assessments of growth in children, clinical laboratory tests and vital signs. Full details of the study can be found at www.clinicaltrials.gov, NCT03312634.
Palovarotene is a retinoic acid receptor gamma agonist (RARγ) being investigated as a treatment for patients with ultra-rare and debilitating bone diseases, including fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), and other diseases with high medical need. Preliminary Phase 2 data in patients with FOP mirror the decrease in heterotopic ossification (HO) volume observed in mouse models of FOP and support the initiation of the confirmatory Phase 3 MOVE Trial. Palovarotene also inhibits the formation of osteochondromas (OCs) in mouse models of multiple exostoses, supporting development in the MO indication. Palovarotene has received Orphan Drug designation for FOP and MO from the
About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare, severely disabling disorder characterized by heterotopic ossification (HO), or bone that forms outside the normal skeleton in muscles, tendons or soft tissue. In FOP, HO progressively restricts movement by locking joints leading to a cumulative loss of function, progressive disability, and increased risk of early death. FOP is caused by a mutation in the ACVR1 gene, resulting in excess signaling in the bone morphogenetic pathway, a key pathway controlling bone growth and development, by way of both ligand-dependent and independent mechanisms. There are currently no approved treatments for FOP.
Conference Call Information
To participate in the conference call, please dial (866) 916-2014 (domestic) or (636) 812-6655 (international) and refer to conference ID 5689444. The webcast can be accessed in the Investor Relations section of the company's website at www.clementiapharma.com. The replay of the webcast will be available in the investor section of the company’s website at www.clementiapharma.com for 60 days following the call.
Clementia is a clinical-stage biopharmaceutical company innovating new treatments for people with ultra-rare bone disorders and other diseases with high medical need. The company’s lead candidate palovarotene, a novel RARγ agonist, is currently being evaluated in the Phase 3 MOVE Trial to treat fibrodysplasia ossificans progressiva (FOP), with additional clinical studies planned in multiple osteochondromas (MO, also known as hereditary multiple exostoses) and other diseases. For more information, please visit www.clementiapharma.com.
The information stated above was prepared by
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This press release may include “forward-looking statements” within the meaning of the applicable securities laws. Each forward-looking statement contained in this press release is subject to known and unknown risks and uncertainties and other unknown factors that could cause actual results to differ materially from historical results and those expressed or implied by such statement. In addition to statements which explicitly describe such risks and uncertainties, readers are urged to consider statements labeled with the terms “believes,” “belief,” “expects,” “intends,” “anticipates,” “will,” or “plans” to be uncertain and forward-looking. Applicable risks and uncertainties include, among others, our ability to generate revenue and become profitable; the risks related to our heavy reliance on palovarotene, our only current product candidate; the risks associated with the development of palovarotene and any future product candidate, including the demonstration of efficacy and safety; our heavy dependence on licensed intellectual property, including our ability to source and maintain licenses from third-party owners; as well as the risks identified under the heading “Risk Factors” in our Prospectus on Form 424(b) filed with the
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Source: Clementia Pharmaceuticals Inc.