Breaking News
November 22, 2018 - Response to daily stressors plays important role in cognitive health of older adults
November 22, 2018 - Efficient method for producing induced pluripotent stem cells
November 22, 2018 - Report outlines inequalities in access to healthcare
November 22, 2018 - New research center will pave way to better diagnosis and treatment of skin diseases
November 22, 2018 - Mylan Initiates Voluntary Nationwide Recall of 15 Lots of Valsartan Tablets, USP, Amlodipine and Valsartan Tablets, USP, and Valsartan and Hydrochlorothiazide Tablets, USP, Due to the Detection of Trace Amounts of NDEA (N-Nitrosodiethylamine) Impurity Found in the Active Pharmaceutical Ingredient
November 22, 2018 - Lung Cancer Tumor Markers: MedlinePlus Lab Test Information
November 22, 2018 - Patchy distribution of joint inflammation resolved
November 22, 2018 - Researchers find crucial inhibitory role for signal peptide in GluK1 trafficking
November 22, 2018 - nutritional supplement, could slow some cancers
November 22, 2018 - Researchers awarded $7 million funding in Pancreatic Cancer Collective’s ‘New Therapies Challenge’
November 22, 2018 - UMN researchers focus on improving dermatologic care for sexual and gender minority patients
November 22, 2018 - Researchers harness visible light to develop safer building block for drug discovery
November 22, 2018 - Scientists identify key protein involved in Hantavirus Pulmonary Syndrome
November 22, 2018 - ACAAI: Oral Immunotherapy Is Protective in Peanut Allergy
November 22, 2018 - Arthritis and depression often occur together in older adults
November 22, 2018 - Scientists uncover unexpected ‘foreign’ genes in tiny itch-inducing chigger mite
November 22, 2018 - Negative social cues on tobacco packaging could help reduce smoking intentions
November 22, 2018 - Study finds no differences in quit rates for ‘dual users’ of both traditional and electronic cigarettes
November 22, 2018 - Immunity connects gut microbiota and age-related pathologies
November 22, 2018 - New diagnostics based on nanopore analytics and AI can identify single influenza virions
November 22, 2018 - Blocking activity of plasma protein can be possible way to protect against radiation-induced injury
November 22, 2018 - Secondhand Pot Smoke Found in Kids’ Lungs
November 22, 2018 - Air pollution may be linked to heightened dementia risk
November 22, 2018 - Brain implant lets people with limb paralysis compose and send emails, select videos and even play music, just by thinking
November 22, 2018 - Should health-care workers press charges against violent patients?
November 22, 2018 - Obesity linked to COPD in never-smokers
November 22, 2018 - Improving dementia treatment and staff training in care homes saves thousands of pounds per year
November 22, 2018 - Researchers identify genetic changes that could increase risk for death by suicide
November 22, 2018 - John Snow Labs launches new Data Market to help healthcare and life science innovators
November 22, 2018 - New inhibitor could hold key to treating Acute Respiratory Distress Syndrome
November 22, 2018 - Misconceptions about opioid use undermine pain control among Asian cancer patients
November 22, 2018 - Owlstone Medical named Medtech Company of the Year
November 22, 2018 - Anabolic steroids may increase risk of early death in men
November 22, 2018 - Researchers develop model that predicts transmissibility of viral zoonoses
November 22, 2018 - Evidence mounts that an eye scan may detect early Alzheimer’s disease
November 22, 2018 - Sensors could provide dexterity to robots, with potential surgical applications
November 22, 2018 - Why Are We Still So Fat?
November 22, 2018 - MRI scans may help predict dementia risk
November 22, 2018 - Groundbreaking research projects show how AI can be used to predict under diagnosed chronic diseases
November 21, 2018 - Stand Up to Cancer supports potential approach to more efficiently target pancreatic cancer
November 21, 2018 - Using smartphone confocal microscopes to stop cancer
November 21, 2018 - Self-care program for COPD patients reduces emergency room visits and burdensome symptoms
November 21, 2018 - Blueprint Medicines Announces Updated NAVIGATOR Trial Results in Patients with Advanced Gastrointestinal Stromal Tumors Supporting Development of Avapritinib Across All Lines of Therapy
November 21, 2018 - Cocaine adulterant may cause brain damage
November 21, 2018 - Amid the devastation, a Stanford doctor stitches up George, a search dog
November 21, 2018 - America’s Health-Care System Is Making the Opioid Crisis Worse
November 21, 2018 - Colorectal cancer screening reduces need for intense treatments among male patients
November 21, 2018 - Scientists find evidence of prions in the eyes of CJD patients
November 21, 2018 - Study explores individual and organizational risk factors for one-year mortality in ICU survivors
November 21, 2018 - Pulmonologists want more information on inhalation devices for COPD
November 21, 2018 - Cessation fatigue predicts relapse rate after attempts to quit smoking
November 21, 2018 - Special care should be taken with drugs that inhibit epigenetic factors, study suggests
November 21, 2018 - More than one in ten heavy cannabis users experience withdrawal after quitting cannabis
November 21, 2018 - Reflections on the California fires
November 21, 2018 - Donna Lynne Appointed to Key Leadership Role at CUIMC
November 21, 2018 - Smoke-free laws associated with reduced systolic blood pressure
November 21, 2018 - Achieving new guideline blood pressure goals may prevent 3 million cardiovascular events
November 21, 2018 - LDR brachytherapy for treating early-stage prostate cancer lacks conclusive data
November 21, 2018 - Older adults with CVD more likely to experience rapid functional decline
November 21, 2018 - Purified omega-3 and aspirin reduce pre-cancerous bowel polyps, shows study
November 21, 2018 - Study warns that potential epigenetic therapy may boost lung cancer stem cells
November 21, 2018 - Noise pollution in hospital impact quality and safety of healthcare
November 21, 2018 - Interventions to improve sleep may reduce risk of falls
November 21, 2018 - Outdoor air pollution linked to intellectual disabilities in children
November 21, 2018 - Study highlights need for better screening tools to detect maternal sepsis
November 21, 2018 - Higher Risk for Amputation, DKA With SGLT2 Inhibitors for T2DM
November 21, 2018 - Researchers stop ‘sneaky’ cancer cells in their tracks
November 21, 2018 - People who are afraid to draw their blood over-estimate the risk of fainting
November 21, 2018 - Personalized physical exercise reverses functional, cognitive deterioration in the elderly
November 21, 2018 - COPD linked to obesity in older women who have never smoked
November 21, 2018 - AHA: Cold-Weather Drinks Are Here, But Watch Out for the Calories
November 21, 2018 - Crowds line up at 1st East Coast pot shops
November 21, 2018 - Merck declares 2018 Life Science Award winners
November 21, 2018 - Many people underestimate the impact of sprains, say foot scientists
November 21, 2018 - Lower levels of protein make squamous carcinoma cells more invasive
November 21, 2018 - Study highlights a new predictor of type 2 diabetes
November 21, 2018 - NTU and TTSH join forces to improve doctor-patient communication
November 21, 2018 - New low-cost injectable hydrogel could help wounds heal faster
November 21, 2018 - Merck Announces Winners of 2018 Life Science Awards
November 21, 2018 - Check your medical records for dangerous errors
Clementia Announces Plan to Submit a New Drug Application for Palovarotene for the Treatment of FOP Based on Positive Phase 2 Results

Clementia Announces Plan to Submit a New Drug Application for Palovarotene for the Treatment of FOP Based on Positive Phase 2 Results

image_pdfDownload PDFimage_print

MONTREAL, Oct. 23, 2018 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage biopharmaceutical company innovating treatments for people with ultra-rare bone disorders and other diseases, today announced that it plans to submit a New Drug Application (NDA) for palovarotene to the U.S. Food and Drug Administration (FDA) in the second half of 2019. Based on recent interactions between Clementia and the FDA, including a Type B meeting held earlier today as part of palovarotene’s Breakthrough Therapy Designation, the FDA has agreed that available data would support filing of an NDA for palovarotene for the prevention of heterotopic ossification (HO) associated with flare up symptoms in patients with fibrodysplasia ossificans progressiva (FOP). The FDA based its assessment on the efficacy and safety data available from the completed Phase 2 clinical program, which showed a statistically significant reduction in mean new HO volume, or bone volume, associated with flare-ups of FOP at 12 weeks as compared to placebo or untreated flare-ups.

The data provided to the FDA included the results from Clementia’s Phase 2 studies, which were recently presented at ASBMR 2018. In these studies, 92 palovarotene-treated flare-ups in 62 patients across three different dosing regimens were evaluated compared to 46 placebo or untreated flare-ups in 41 patients from the Company’s natural history study. Patients treated with palovarotene at the time of a flare-up demonstrated a greater than 70 percent reduction in mean new HO volume at 12 weeks compared to the untreated group. Further, the reduction in mean new HO volume among those treated with the episodic 20/10 mg dosing regimen (20 mg for 4 weeks starting at the time of a flare-up followed by 10 mg for 8 weeks) was statistically significant (p=0.02). Palovarotene was generally well tolerated across all dosing regimens of the Phase 2 clinical program. There were dose-related increases in retinoid-associated adverse events (AEs) with most being mild or moderate in severity, and only one patient discontinued participation in the study because of an AE.

Following the completion of standard non-clinical, clinical pharmacology and CMC studies to be agreed upon with the FDA, Clementia plans to submit its NDA in the second half of 2019 to seek approval of the palovarotene 20/10 mg episodic dosing regimen.

“The identification of a path to an NDA submission in the second half of 2019 is a significant milestone for Clementia and for patients with this ultra-rare and devastating genetic bone disease,” said Clarissa Desjardins, Ph.D., founder and chief executive officer of Clementia. “We are thankful for the collaboration with the FDA’s Division of Bone, Reproductive and Urologic Products, potentially bringing the first approved treatment option to individuals affected by FOP. We are also grateful to the patients and their families, as well as the investigators and clinical sites, without whom none of this work would have been possible.”

Clementia’s ongoing Phase 3 MOVE Trial will continue as planned, evaluating a dosing regimen of palovarotene which includes a chronic 5 mg daily dose in addition to the episodic 20/10 mg dosing regimen at the time of a flare-up. The Company believes that, if successful, the data from the MOVE Trial may provide the basis for a supplemental NDA for an additional treatment regimen option for patients with FOP.

Clementia also plans to engage international regulatory authorities in 2019 to discuss the registration strategy for palovarotene and anticipates providing an update at a later stage.

About Palovarotene

Palovarotene is an RARγ agonist being developed as a treatment for patients with ultra-rare and debilitating bone diseases, including fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), as well as other diseases. Palovarotene was in-licensed from Roche Pharmaceuticals, where it was previously evaluated in more than 800 subjects, including 450 patients treated for up to two years. Palovarotene has received Orphan Drug status for FOP and MO from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In addition, palovarotene has been granted Fast Track and Breakthrough Therapy designations for FOP from the FDA.

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 company innovating treatments for people with ultra-rare bone disorders and other diseases with high medical need. The Company is preparing for a 2019 NDA submission to the FDA to seek approval of its lead product candidate, palovarotene, a novel RARγ agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP). The ongoing Phase 3 MOVE Trial is evaluating an additional dosing regimen of palovarotene which includes a chronic 5 mg daily dose in addition to the episodic 20/10 mg dosing regimen at the time of a flare-up. Palovarotene is also in a Phase 2 trial, the MO-Ped Trial, for the treatment of multiple osteochondromas (MO, also known as multiple hereditary exostoses, or MHE). In addition, Clementia has commenced a Phase 1 trial for an eye drop formulation of palovarotene for the potential treatment of dry eye disease and is also investigating other conditions that may benefit from RARγ therapy. For more information, please visit www.clementiapharma.com and connect with us on Twitter @ClementiaPharma.

Cautionary Note Regarding Forward-Looking Statements

This press release may include “forward-looking statements” within the meaning of the applicable securities laws, including with respect to the proposed timing of submission of the NDA for palovarotene. 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, the Company’s ability to successfully complete in a timely manner the studies required to be completed in order to submit the NDA, the Company’s ability to generate revenue and become profitable; the risks related to its heavy reliance on palovarotene, its only current product candidate; the risks associated with the development of palovarotene and any future product candidate, including the demonstration of efficacy and safety; its dependence on licensed intellectual property, including the ability to source and maintain licenses from third-party owners; as well as the risks identified under the heading “Risk Factors” in the Company’s Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”), as well as the other information its file with the SEC or on SEDAR. Clementia cautions investors not to rely on the forward-looking statements contained in this press release when making an investment decision in its securities. Investors are encouraged to read the Company’s 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 the Company undertakes 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: October 2018

Tagged with:

About author

Related Articles