Breaking News
December 18, 2018 - California Farm Implicated in Outbreak of E. coli Tied to Romaine Lettuce
December 18, 2018 - Mobile health has power to transform HIV/AIDS nursing
December 18, 2018 - Celiac Vaccine in Clinical Trials at Columbia
December 18, 2018 - Research into mental health first aid prompts practical guidance and resources for workplace
December 18, 2018 - Researcher conducts study to investigate peripheral blood markers of Alzheimer’s disease
December 18, 2018 - Researchers identify link between mucus in the small airways and pulmonary fibrosis
December 18, 2018 - EU Commission’s Health Policy Platform to host EKHA program on transplantation
December 18, 2018 - Survivors of childhood Hodgkin lymphoma have high risk of developing solid tumors
December 18, 2018 - Small changes to cafeteria design can get kids to eat healthier, new assessment tool finds
December 18, 2018 - From Machines to Cyclic Compounds
December 18, 2018 - New study reveals best assessment tools to establish delirium severity
December 18, 2018 - Rice University scientists develop synthetic protein switches to control electron flow
December 18, 2018 - Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy
December 18, 2018 - Researchers identify potential target for new breast cancer treatments
December 18, 2018 - National Biofilms Innovation Centre award grant to Neem Biotech for novel anti-biofilm drug development
December 18, 2018 - Artificial intelligence and the future of medicine
December 18, 2018 - Montana State doctoral student receives grant for her work to improve neuroscience tool
December 18, 2018 - Early postpartum initiation of opioids associated with persistent use
December 18, 2018 - Russian scientists identify molecular ‘switch’ that could be target for treatment of allergic asthma
December 18, 2018 - Surgeons make more mistakes in the operating room during stressful moments, shows study
December 18, 2018 - Immune cells explode themselves to inform about the danger of invading bacteria
December 18, 2018 - Malnutrition in children with Crohn’s disease linked with increased risk of surgical complications
December 18, 2018 - FDA Approves Motegrity (prucalopride) for Adults with Chronic Idiopathic Constipation (CIC)
December 18, 2018 - The long and short of CDK12
December 18, 2018 - Hologic’s Cynosure division introduces TempSure Surgical RF technology in North America
December 18, 2018 - CMR Surgical partners with Nicholson Center to launch U.S.-based training program for Versius
December 18, 2018 - Findings reinforce guidelines for cautious use of antipsychotics in younger populations
December 18, 2018 - Study finds new strains of hepatitis C virus in sub-Saharan Africa
December 18, 2018 - New battery-free, implantable device aids weight loss
December 18, 2018 - Parental alcohol use disorder associated with offspring marital outcomes
December 18, 2018 - Novel Breast Imaging Technique Might Cut Unnecessary Biopsies
December 18, 2018 - What can a snowflake teach us about how cancer spreads in the body?
December 18, 2018 - Management of nausea and vomiting in pregnancy costs the NHS more than previously thought
December 18, 2018 - Green leafy vegetables may reduce risk of developing liver steatosis
December 18, 2018 - Veganism linked to nutrient deficiencies and malnutrition if not planned correctly
December 18, 2018 - Coming Soon: A Tiny Robot You Swallow to Help You Stay Healthy
December 18, 2018 - Modified malaria drug proven effective at inhibiting Ebola
December 18, 2018 - Study finds epigenetic differences in the brains of individuals with schizophrenia
December 18, 2018 - Fitness instructors’ motivational comments influence women’s body satisfaction
December 18, 2018 - Study focuses on modification of lipid nanoparticles for successful brain cell targeting
December 18, 2018 - New gut bacteria may be effective against obesity, metabolic and mental disorders
December 18, 2018 - New two-in-one powder aerosol to upgrade fight against deadly superbugs in lungs
December 18, 2018 - Biofilms feed with swirling flows
December 17, 2018 - Study identifies specific neurological changes related to traumatic brain injury
December 17, 2018 - New study confirms geographic bias in lung allocation for transplant
December 17, 2018 - Research focuses on optimization of solid lipid nanoparticle that encapsulates Vinorelbine bitartrate
December 17, 2018 - Carpal tunnel syndrome – Genetics Home Reference
December 17, 2018 - A novel insulin accelerant
December 17, 2018 - Tips for caring for patients with disabilities, from a mother and physician
December 17, 2018 - Menopause-related sexual, urinary problems tied to worse quality of life
December 17, 2018 - In-school nutrition programs among students limit increases in BMI, finds study
December 17, 2018 - Risk for Hospitalization for Heart Failure Greater With Diabetes
December 17, 2018 - Food assistance may help older adults adhere to diabetes meds
December 17, 2018 - Supporting a family’s goals during a difficult pregnancy
December 17, 2018 - Neurons with Good Housekeeping Are Protected from Alzheimer’s
December 17, 2018 - New approach to tumor analysis could improve prognosis for bowel cancer patients
December 17, 2018 - New ‘epigenetics-based’ cervical cancer test outperforms Pap smear and HPV tests
December 17, 2018 - Ten year follow-up after negative colonoscopy related to reduced risk of colorectal cancer
December 17, 2018 - CTF along with NTAP and Sage announce first-ever open data portal for neurofibromatosis
December 17, 2018 - Intimacy: The Elusive Fountain of Youth?
December 17, 2018 - Will saliva translate to a real diagnostic tool?
December 17, 2018 - DFG establishes nine new Research Units and one new Clinical Research Unit
December 17, 2018 - Assisted living’s breakneck growth leaves patient safety behind
December 17, 2018 - America’s teens report dramatic increase in their use of vaping devices in just one year
December 17, 2018 - Enlarged heart linked to a higher risk of dementia
December 17, 2018 - Prostate cancer detection using MRI now first-line investigation tool
December 17, 2018 - Loughborough academics part of new project investigating effectiveness of personalized breast cancer screening
December 17, 2018 - Adolescents who use cognitive reappraisal had better metabolic measures, shows study
December 17, 2018 - Probiotics may offer therapeutic benefits for biopolar patients
December 17, 2018 - Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide for the Treatment of Dry Age-Related Macular Degeneration with Geographic Atrophy
December 17, 2018 - Studies reveal role of red meat in gut bacteria, heart disease development
December 17, 2018 - Eisai enters into agreement with Eurofarma for its anti-obesity agent lorcaserin
December 17, 2018 - Researchers use brain connectome to reassess neuroimaging findings of Alzheimer’s disease
December 17, 2018 - “Miracle” baby survives Ebola in Congo and rapid a new Ebola detection device
December 17, 2018 - Mechanisms behind neonatal diabetes uncovered
December 17, 2018 - AHF urges the WHO to expedite approval process for vaccine effective against Ebola
December 17, 2018 - Study finds misuse of benzodiazepines to be highest among young adults
December 17, 2018 - TGen receives PayPal grant to underwrite costs of genetic tests for children with rare disorders
December 17, 2018 - New research highlights why HIV-infected patients suffer higher rates of cancer
December 17, 2018 - Antibiotic-resistant bacteria could soon be targeted with Alzheimer’s drug
Clementia Initiates Pivotal Phase 3 MOVE Trial for Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva

Clementia Initiates Pivotal Phase 3 MOVE Trial for Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva

image_pdfDownload PDFimage_print

MONTREAL, Dec. 12, 2017 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (NASDAQ:CMTA), a clinical-stage biopharmaceutical company innovating new treatments for people with ultra-rare bone disorders and other diseases, today announced the start of the MOVE Trial, its Phase 3 registration study evaluating the safety and efficacy of palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva (FOP). Palovarotene, Clementia’s lead product candidate, is a retinoic acid receptor gamma agonist (RARγ) being investigated as a treatment for patients with ultra-rare and debilitating bone diseases, including FOP and multiple osteochondromas (MO), as well as other diseases.

“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 Edward Hsiao, M.D., Ph.D., associate professor in endocrinology and the Institute for Human Genetics at the University of California San Francisco (UCSF) School of Medicine, and one of the principal investigators of the MOVE Trial. “Palovarotene has shown promise in its Phase 2 program. Our team looks forward to working with patients and our fellow investigators from around the world to complete this important study.”

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 (Argentina, Australia, Brazil, Canada, France, Germany, Italy, Japan, Netherlands, Russia, South Africa, South Korea, Spain, Sweden, United Kingdom and United States). Additional sites are expected to begin enrolling patients in the coming months, with completion of enrollment expected by the end of 2018.

“The initiation of this study is a landmark event for Clementia, fully transitioning us into a late-stage clinical organization,” commented Donna Grogan, M.D., chief medical officer of Clementia. “To date we have treated over fifty FOP patients in our Phase 2 clinical program, and we are encouraged by palovarotene’s safety profile and the efficacy data showing meaningful reductions in new heterotopic ossification (HO) volume following a flare-up.” HO is a hallmark sign of FOP, in which bone grows where it should not, outside the skeleton and in abnormal places, such as muscles, tendons and soft tissue in patients with FOP.

“Palovarotene is rapidly advancing through clinical development and could be the first treatment to be approved for patients with FOP,” said Clarissa Desjardins, Ph.D., chief executive officer of Clementia. “Our team is motivated by the medical need for patients with FOP and wishes to thank the patients, their families and caregivers, and the investigators around the world who have been working hand-in-hand with us over the past three years to advance palovarotene to this important milestone.”

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.

About Palovarotene

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 U.S. Food and Drug Administration (FDA). In addition, palovarotene has been granted Fast Track and Breakthrough Therapy designations for FOP from the FDA, and was granted orphan status for the treatment of FOP in the EU.

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.

About Clementia Pharmaceuticals Inc.

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.

UC Disclaimer

The information stated above was prepared by Clementia Pharmaceuticals Inc. to report on research conducted on its product and reflects solely the opinion of Clementia. Nothing in this statement shall be construed to imply any support or endorsement of Clementia, or any of its products, by The Regents of the University of California, its officers, agents and employees.

Cautionary Note Regarding Forward-Looking Statements

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 Securities and Exchange Commission (“SEC”), as well as the other information we file with the SEC or on SEDAR. We caution investors not to rely on the forward-looking statements contained in this press release when making an investment decision in our securities. You are encouraged to read our filings with the SEC or on SEDAR, available at www.sec.gov or www.sedar.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements, whether as a result of new information, future events or otherwise, except as required by law.

Source: Clementia Pharmaceuticals Inc.

Posted: December 2017

Tagged with:

About author

Related Articles