Breaking News
February 20, 2018 - Lab-grown cerebellar cells may help explain how ASD develops at molecular level
February 20, 2018 - New Treatment Apalutamide (Erleada) Approved for Prostate Cancer That Resists Hormone Therapy
February 20, 2018 - Do You Really Need My Signature on That?
February 20, 2018 - HIV-1 genetic diversity is higher in vaginal tract than in blood during early infection
February 20, 2018 - Researchers aim to find out how PTSD affects decisions of police
February 20, 2018 - UH Cleveland Medical Center explores novel treatments for uterine fibroids
February 20, 2018 - Flu Vax Efficacy 25% Against Predominant H3N2 Strain So Far
February 20, 2018 - HIV screening most optimal at 25 years of age if no risk factors
February 20, 2018 - Loyola Medicine primary care physician offers advice to minimize risk of flu
February 20, 2018 - Safe sleep recommendations for parents that may help reduce child’s risk of SUID
February 20, 2018 - Why Do So Few Docs Have Buprenorphine Waivers?
February 20, 2018 - Low levels of alcohol good for the brain
February 20, 2018 - Experimental treatment improves invisible symptoms of a man with spinal cord injury
February 20, 2018 - Myriad’s EndoPredict offers better prediction of breast cancer recurrence, analysis shows
February 20, 2018 - Researchers identify fifteen genes that determine our facial features
February 20, 2018 - Morning Break: New Health IT Player; Luxturna No Bargain; Nuclear Freakout
February 20, 2018 - How does it compare? Hospice care at home, at assisted living facility, at nursing home
February 19, 2018 - Scientists develop water-soluble warped nanographene for bioimaging
February 19, 2018 - It’s Not Your Imagination: You’re Hungrier After Losing Weight
February 19, 2018 - Antihypertensive Use At Delivery Rising in Preeclampsia
February 19, 2018 - A centuries-old math equation used to solve a modern-day genetics challenge
February 19, 2018 - Liquid biopsies could be used as new predictive marker for metastatic TNBC
February 19, 2018 - Russian researchers develop new multi-layered biodegradable scaffolds
February 19, 2018 - Are ‘Vaccine Skeptics’ Responsible for Flu Deaths?
February 19, 2018 - Hidden genetic effects behind immune diseases may be missed, study suggests
February 19, 2018 - Emergency nurses experience regular verbal and physical abuse
February 19, 2018 - Study sheds light on biology that guides behavior across different stages of life
February 19, 2018 - Morning Break: Transgender Breast Feeding; Brazilian ‘Pro-Vaxxers’; Post-Stroke Exercise
February 19, 2018 - Meningitis vaccination strategy in Africa found to be effective, economical
February 19, 2018 - Researchers uncover how excess calcium may influence development of Parkinson’s disease
February 19, 2018 - Psoriasis drug also effective at reducing aortic inflammation
February 19, 2018 - Excess emissions can make serious contributions to air pollution, study shows
February 19, 2018 - Researchers reveal potential biological roots behind individuality
February 19, 2018 - Diabetes Drugs Differ on HF; School-Based Obesity Program Flop; Plaque Type in ACS
February 19, 2018 - Surgical infections linked to drug-resistant bugs, study suggests
February 19, 2018 - Poor awareness may hinder a child’s early dental care
February 19, 2018 - Research finds rising trend in incidence of merkel cell carcinoma
February 19, 2018 - Researchers uncover Ras protein’s role in uncontrolled cancer growth
February 19, 2018 - FDA Approves Apalutamide (Erleada) to Help Curb a Tough-to-Treat Prostate Cancer
February 19, 2018 - Educational Tool Boosts Cervical Length Screening
February 19, 2018 - Spider’s web inspires removable implant that may control type 1 diabetes
February 19, 2018 - Scientists develop fluorescent probe to identify cancer stem cells
February 19, 2018 - University Hospital of Santiago de Compostela participates in large pancreatic cancer study
February 19, 2018 - New blood test shows promise to revolutionize diagnosis of tick-borne diseases
February 19, 2018 - Report: Use, Not Price, Drives State Health Costs
February 19, 2018 - Emergency services crews often unprepared for diabetic crises
February 19, 2018 - Scientists in Sweden create DNA nanowires that offer hope for treatment of diseases
February 19, 2018 - ID Break: Clean Hands, Fewer Abx; $11 Million HIV Cure?; MenB Vax for Kids
February 19, 2018 - Patient exposure to X-rays depends on how dentists are paid
February 19, 2018 - Study reveals parents’ views toward children’s tanning bed use
February 19, 2018 - Shot may help reduce risk of shingles
February 19, 2018 - FDA approves first treatment to reduce risk of NSCLC progression
February 19, 2018 - FDA Expands Approval of Imfinzi (durvalumab) to Reduce the Risk of Non-Small Cell Lung Cancer Progressing
February 19, 2018 - D.C. Week: Congress Passes Spending Bill
February 19, 2018 - Heart-muscle patches made with human cells improve heart attack recovery
February 19, 2018 - FDA Approves First Blood Test to Detect Concussions
February 19, 2018 - Survival Bump in Bladder Cancer with Keytruda
February 18, 2018 - Scientists describe the mechanism of heart regeneration in the zebrafish
February 18, 2018 - Scientists uncover the structure of microtubule motor proteins
February 18, 2018 - Light-activated cancer drugs without toxic side effects are closer to becoming reality
February 18, 2018 - Pioneering research could provide novel insight into how genomic information is read
February 18, 2018 - Pearls From: David Putrino, PhD
February 18, 2018 - Researchers uncover how cancer stem cells drive triple-negative breast cancer
February 18, 2018 - Morning Break: Anti-Anti-Vaxxers; Private Piercings Prohibited; A Case for Pelvic Massage
February 18, 2018 - Lower-dose radiation effective, safe for HPV+ head and neck cancer after induction chemo
February 18, 2018 - Specialist residential service for adults with autism opens in Swansea
February 18, 2018 - FDA Moves to Limit Loperamide Doses per Package
February 18, 2018 - Alcohol use disorder – Genetics Home Reference
February 18, 2018 - Autism might be better detected using new two-minute questionnaire
February 18, 2018 - Hand hygiene-intervention practices may reduce risk of infection among nursing home patients
February 18, 2018 - Researchers develop most sophisticated mini-livers to date
February 18, 2018 - Obamacare Helped More Young Women Get Prenatal Care: Study
February 18, 2018 - School-Based Program Fails to Dent Kids’ Obesity
February 18, 2018 - Research compares neural activity in children with and without autism spectrum disorder
February 18, 2018 - Poor fitness levels increase the risk dementia, concludes study
February 18, 2018 - Risk Score May Reveal if Kids are Victims of Ill-Treatment
February 18, 2018 - Adding Folic Acid to Corn Masa Flour May Prevent Birth Defects
February 18, 2018 - Acute treatment suppresses posttraumatic arthritis in ankle injury
February 18, 2018 - A Role for Budesonide in Autoimmune Hepatitis?
February 18, 2018 - Lupus patients exhibit altered cell proteins, a discovery with potential implications for diagnostics
GW Pharmaceuticals and its U.S. Subsidiary Greenwich Biosciences Announce Publication of Landmark Epidiolex (cannabidiol) Study in The Lancet

GW Pharmaceuticals and its U.S. Subsidiary Greenwich Biosciences Announce Publication of Landmark Epidiolex (cannabidiol) Study in The Lancet

image_pdfDownload PDFimage_print

LONDON and CARLSBAD, Calif., Jan. 24, 2018 (GLOBE NEWSWIRE) — GW Pharmaceuticals plc (Nasdaq:GWPH) (“GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, along with its U.S. subsidiary Greenwich Biosciences, announced today that The Lancet has published results from a Phase 3 study of Epidiolex® (cannabidiol) in patients with Lennox-Gastaut syndrome (LGS).1 Epidiolex, GW’s lead product candidate and the potential first in a new category of anti-epileptic drugs (AEDs), is a pharmaceutical formulation of purified cannabidiol (CBD), a cannabinoid lacking euphoric side effects, which is being studied for the treatment of a number of rare, severe pediatric-onset epilepsy disorders. In this study, Epidiolex significantly reduced monthly drop seizure frequency compared to placebo in highly treatment-resistant patients when added to existing treatment. Treatment with Epidiolex was generally well tolerated, with a safety profile consistent with prior reported experience.

A New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for Epidiolex in the treatment of LGS and Dravet syndrome (another rare childhood-onset epilepsy) was accepted in December with an assigned PDUFA goal date of June 27th 2018 and, if approved, the medicine is expected to be available in the U.S. by prescription in the second half of 2018. A Marketing Authorisation Application (MAA) was submitted to the European Medicines Agency (EMA) in December 2017, with an expected decision in early 2019.

“Publication of this landmark study by The Lancet is an exciting achievement and marks the second time that Epidiolex data have been published in a highly prestigious journal, following last year’s publication in The New England Journal of Medicine,” said Justin Gover, GW’s Chief Executive Officer. “These publications highlight the potential of Epidiolex to address the significant unmet need in LGS and Dravet syndrome, two very challenging epilepsy conditions, and we look forward to working with the FDA and EMA as they review our marketing applications for Epidiolex. We are absolutely focused on the goal of making this important new medicine available to appropriate patients and their caregivers as quickly as possible.”

LGS is a rare, lifelong form of epilepsy that begins in childhood and is associated with a high mortality rate2 and significant developmental delays.3,4 LGS patients suffer from multiple types of seizures, including drop seizures which can result in falls and other injuries. Results from this study represent the only well-controlled clinical evaluation of a cannabinoid medication for this severe, drug-resistant condition.

“The publication of these positive results is an exciting milestone for the LGS community and we are encouraged that a new treatment option could soon be available,” said Christina SanInocencio, executive director of the Lennox-Gastaut Syndrome Foundation. “Additional treatment options are desperately needed for patients who continue to struggle with uncontrolled seizures and these results offer much needed hope to those living with this debilitating condition.”

“LGS is one of the most difficult types of epilepsy to treat and the majority of patients do not have an adequate response to existing therapies,” said Elizabeth Thiele, MD, PhD, director of pediatric epilepsy at Massachusetts General Hospital, professor of Neurology at Harvard Medical School and lead author of the study publication. “These results show that Epidiolex may provide clinically meaningful benefits for patients with LGS.”

The study randomized 171 patients (86 to CBD; 85 to placebo) ages two to 55 years (average age 15), with LGS whose seizures were not controlled by their current AED regimen, to receive either Epidiolex (20mg/kg/day) or placebo in addition to existing treatment. Conducted in 24 study centers in the United States and Europe, on average, patients were taking approximately three AEDs, having previously tried and discontinued an average of six other AEDs. At baseline, patients had a median frequency of 74 drop seizures per month (drop seizures were defined as atonic, tonic or tonic-clonic seizures involving the entire body, trunk or head that led or could have led to a fall, injury, slumping in a chair or hitting the patient’s head on a surface).

Over the 14-week treatment period (two-week dose escalation period followed by 12 weeks of maintenance), patients taking Epidiolex had a significantly greater median reduction in drop seizures compared to placebo (44 percent vs. 22 percent; p=0.0135), the study’s primary endpoint. Sensitivity analyses confirmed that the treatment effect of CBD was established during the first month of treatment and was sustained over the entire treatment period.

Results from key secondary endpoints showed that significantly more patients on Epidiolex experienced a 50 percent or greater reduction in drop seizures compared to placebo (44 percent vs. 24 percent; p=0.0043) and total seizure frequency was significantly reduced with Epidiolex compared to placebo (median percent reduction of 41 percent vs. 14 percent; p=0.0005). CBD patients/caregivers were significantly more likely to report an improvement in overall condition with Epidiolex than placebo (58 percent vs. 34 percent; OR 2.54, 95% CI 1.5-4; p=0.0012) based on the Subject/Caregiver Global Impression of Change (S/CGIC) scale.

Epidiolex was generally well tolerated in the trial. The most common adverse events (AEs) (>10 percent) were diarrhea, somnolence, pyrexia, decreased appetite and vomiting. Overall, 86 percent of patients taking Epidiolex and 69 percent of patients taking placebo experienced an AE, and most were mild or moderate. Of those patients who experienced AEs, the events resolved by the end of the trial for 61 percent of Epidiolex patients and 64 percent of placebo patients.

Twenty patients on Epidiolex experienced serious AEs, including one fatal case of acute respiratory distress syndrome (considered unrelated by the Investigator), compared with four patients with serious AEs on placebo. Twelve patients taking Epidiolex discontinued treatment due to AEs compared with one patient taking placebo.

Across the Epidiolex development program, the most common reported adverse reactions are somnolence, decreased appetite, diarrhea, pyrexia, fatigue, lethargy, rash, nasopharyngitis, and pneumonia; dose-related reversible elevation of liver transaminases without elevation of bilirubin were also observed.

“Uncontrolled seizures significantly impact the lives of patients and their families and there is a tremendous need for new options in difficult-to-treat epilepsies such as LGS,” said Philip Gattone, president and CEO, Epilepsy Foundation. “This randomized, controlled clinical study provides positive evidence of the potential role of cannabidiol in reducing seizures and we are excited about the possibility of a new treatment option for LGS.”

About Lennox-Gastaut Syndrome

The onset of LGS typically occurs between ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections, and genetic neuro-degenerative or metabolic conditions. In up to 30 percent of patients, no cause can be found. Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and non-convulsive seizures. Resistance to anti-epileptic drugs (AEDs) is common in patients with LGS. Most children with LGS experience some degree of intellectual impairment, as well as developmental delays and aberrant behaviors.

About Dravet Syndrome

Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the SCN1A sodium channels. Onset of Dravet syndrome occurs typically during the first year of life in previously healthy and developmentally normal infants. Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with Dravet syndrome often develop multiple types of seizures, including tonic-clonic, myoclonic, and atypical absences and are prone to bouts of prolonged seizures including status epilepticus, which can be life threatening. Risk of premature death including SUDEP (sudden unexpected death in epilepsy) is elevated in patients with Dravet syndrome. Additionally, the majority will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care. There are currently no FDA-approved treatments and nearly all patients continue to experience seizures and other medical needs throughout their lifetime.

About Epidiolex (cannabidiol)

Epidiolex, GW’s lead cannabinoid product candidate is a pharmaceutical formulation of purified cannabidiol (CBD), which is in development for the treatment of several rare childhood-onset epilepsy disorders. GW has submitted a New Drug Application with the FDA for Epidiolex as adjunctive treatment for seizures associated with LGS and Dravet syndrome, which has been assigned a goal date of 27 June 2018 and, if approved, the medicine is expected to be available by prescription in the second half of 2018. GW has also submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in December 2017 with an expected decision date in early 2019. To date, GW has received Orphan Drug Designation from the FDA for Epidiolex for the treatment of Dravet syndrome, LGS, TSC and IS. Additionally, GW has received Fast Track Designation from the FDA for the treatment of Dravet syndrome and conditional grant of rare pediatric disease designation by FDA. The Company has also received Orphan Designation from the European Medicines Agency, or EMA, for Epidiolex for the treatment of LGS, Dravet syndrome, West syndrome and TSC. GW is currently evaluating additional clinical development programs in other orphan seizure disorders including Phase 3 trials in Tuberous Sclerosis Complex and Infantile Spasms.

About GW Pharmaceuticals plc and Greenwich Biosciences

Founded in 1998, GW is a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform in a broad range of disease areas. GW, along with its U.S. subsidiary Greenwich Biosciences, is advancing an orphan drug program in the field of childhood epilepsy with a focus on Epidiolex (cannabidiol), for which GW has submitted an NDA to the FDA for the adjunctive treatment of LGS and Dravet syndrome. The Company continues to evaluate Epidiolex in additional epilepsy conditions and currently has ongoing clinical trials in Tuberous Sclerosis Complex and Infantile Spasms. GW commercialized the world’s first plant-derived cannabinoid prescription drug, Sativex® (nabiximols), which is approved for the treatment of spasticity due to multiple sclerosis in numerous countries outside the United States. The Company has a deep pipeline of additional cannabinoid product candidates which includes compounds in Phase 1 and 2 trials for gliobastoma, schizophrenia and epilepsy. For further information, please visit www.gwpharm.com.

Forward-looking statements

This news release contains forward-looking statements that reflect GW’s current expectations regarding future events, including statements regarding financial performance, the timing of clinical trials, the timing and outcomes of regulatory or intellectual property decisions, the relevance of GW products commercially available and in development, the clinical benefits of Epidiolex® (cannabidiol) and the safety profile and commercial potential of Epidiolex. Forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors, including (inter alia), the success of GW’s research strategies, the applicability of the discoveries made therein, the successful and timely completion and uncertainties related to the regulatory process, and the acceptance of Sativex, Epidiolex and other products by consumer and medical professionals. A further list and description of risks and uncertainties associated with an investment in GW can be found in GW’s filings with the U.S. Securities and Exchange Commission, including the most recent Form 20-F filed on 4 December 2017. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. GW undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

1 Thiele EA, Marsh ED, French JA, et al. Cannabidiol in patients with seizures associated with Lennox-Gastaut syndrome (GWPCARE4): a randomized, double-blind placebo-controlled phase 3 trial. Published online January 24, 2018 http://dx.doi.org/10.1016/S0140-6736(18)30136-3.
2 Autry AR, Trevathan E, Van Naarden Braun K, Yeargin-Allsopp M. Increased risk of death among children with Lennox-Gastaut syndrome and infantile spasms. J Child Neurol. 2010;25(4):441-447.
3 LGS Foundation. About Lennox-Gastaut Syndrome. Available at http://www.lgsfoundation.org/aboutlgs. Accessed October 23, 2017.
4 National Institute of Health. Lennox-Gastaut syndrome. Available at https://ghr.nlm.nih.gov/condition/lennox-gastaut-syndrome#definition. Accessed October 23, 2017.

Source: GW Pharmaceuticals plc

Posted: January 2018

Tagged with:

About author

Related Articles