May CURE Update: Epidiolex, Sabril, and Epilepsy Education Events

I was recently interviewed by Business Insider about the FDA’s review of a potential new anti-epilepsy drug, Epidiolex. In Phase III clinical trials, 44% of patients with Lennox-Gastaut Syndrome (LGS) and 43% of patients with Dravet syndrome who used Epidiolex saw a 50% or greater reduction in their seizures compared to 24% and 27% of participants who took the placebo.

Epidiolex could be the first drug derived from cannabidiol (CBD) to gain FDA approval. Unlike THC, one of the other components in marijuana, cannabidiol is not an intoxicant. So, Epidiolex does not have hallucinogenic effects. The FDA is expected to make its final decision about Epidiolex in June.

In other news: CURE is announcing the expansion of its Day of Science event; CURE is urging insurance companies to maintain reasonable access to Sabril; and you can now support CURE while drinking your morning cup of coffee. Read on….

 

DAY OF SCIENCE IS EXPANDING TO SIX LOCATIONS!

CURE’s 2018 Day of Science events kick-off in June. At these free events, you can learn about cutting-edge epilepsy therapies and treatments, via:

  • Q&A sessions with panels of epilepsy physicians
  • Small-group roundtable discussions led by experts

 

You’ll also get the opportunity to engage with others in the epilepsy community.

Registration is now open for the Days of Science in:

 

Stay tuned for details about CURE’s Days of Science in the following cities:

  • Houston
  • Los Angeles
  • Miami
  • Raleigh-Durham

 

Interested in volunteering? Please contact the Day of Science team at DOS@CUREepilepsy.org or (312) 255-1801.

Zynerba Pharmaceuticals Announces Twelve Month ZYN002 Data from STAR 2 Study in Patients with Focal Seizures at the 2018 Annual Meeting of the American Academy of Neurology (AAN)

Zynerba Pharmaceuticals, Inc., a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, is reporting new longer term open label clinical data today in the Emerging Science session of the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, CA.

In a poster presentation entitled, “Transdermal Cannabidiol (CBD) Gel for the Treatment of Focal Epilepsy in Adults” (poster P4.468), Dr. John Messenheimer presents additional data from ongoing STAR 2 (Synthetic Transdermal CAnnabidiol for the TReatment of Epilepsy) 24-month open label extension study evaluating ZYN002 cannabidiol (CBD) transdermal gel in adult patients with focal seizures. The presentation includes data through twelve months of open label exposure to ZYN002.

The key findings include that responses to ZYN002 in the STAR 2 open label extension, as measured by reductions in focal seizures from the baseline period of STAR 1, are associated with continued treatment with ZYN002. In addition, ZYN002 was shown to be well tolerated through 12 months of treatment in STAR 2.

“These data continue to suggest that focal seizures may be reduced with longer-term exposure to transdermally-delivered CBD,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer. “In this population of patients, the use of ZYN002 for an additional 12 months in STAR 2 was well tolerated and appeared to result in clinically meaningful seizure reductions both across and within the originally randomized STAR 1 groups. These data continue to provide insight into the potential for ZYN002 in certain epilepsies, and we look forward to initiating a Phase 2b study in adult refractory focal seizures in the second half of 2018.”

GW Pharmaceuticals and U.S. Subsidiary Greenwich Biosciences Announces the Unanimous Positive Result of FDA Advisory Committee Meeting for First Plant-Based Pharmaceutical [CBD] Treatment for Seizures in Patients with Two Rare, Severe Forms of Epilepsy

GW Pharmaceuticals, 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, today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) unanimously recommended supporting the approval of the New Drug Application (NDA) for the investigational cannabidiol oral solution (CBD), also known as Epidiolex, for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome in patients two years of age and older. If approved, Epidiolex would be the first pharmaceutical formulation of purified, plant-based CBD, a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs (AEDs). This public meeting was presented live through FDA’s website.

LGS and Dravet syndrome, which develop in childhood, are devastating forms of epilepsy with high morbidity and mortality rates and a significant burden on families and caregivers. More than 90% of patients with LGS or Dravet syndrome have multiple seizures per day, which puts them at constant risk for falls and injury. Physicians who treat LGS and Dravet syndrome patients struggle to reduce the sheer volume of dangerous seizures with currently available therapies. If approved, Epidiolex would be the first-ever FDA-approved medicine for Dravet syndrome patients.

“We are pleased by the Advisory Committee’s unanimous recommendation to approve Epidiolex, which would provide an important treatment option for patients with LGS and Dravet syndrome, two of the most severe and treatment-resistant forms of epilepsy,” said Justin Gover, GW’s Chief Executive Officer. This favorable outcome marks an important milestone in our company’s unwavering commitment to address the significant unmet need for patients with LGS and Dravet syndrome and our resolve to study Epidiolex under the highest research and manufacturing standards. We look forward to our ongoing discussions with the FDA as it continues to review the Epidiolex NDA.

A Powerful Drug Derived from Marijuana Just Got a Major Green Light on its Way to FDA Approval

Business Insider – An experimental drug derived from cannabis to treat epilepsy is on the brink of becoming the first of its kind to win US government approval.

On Thursday, a panel of outside experts convened by the Food and Drug Administration voted unanimously in favor of the drug’s safety and effectiveness. Their recommendation will play a key role in the FDA’s approval decision for the drug, which is made by GW Pharmaceuticals.

If the FDA gives final approval — a decision is expected in June — the new drug would be sold under the name Epidiolex as a syrup. It would be the first drug whose active ingredient is cannabidiol, the compound in marijuana thought to be responsible for many of its therapeutic effects.

Cannabidiol, or CBD, doesn’t contain THC, marijuana’s main psychoactive ingredient, and is not linked with euphoria or the drug’s characteristic high. CBD appears to help reduce seizures, at least in two of the hardest-to-treat forms of epilepsy, known as Lennox-Gastaut syndrome and Dravet syndrome. That’s according to two large clinical trials the FDA considered Tuesday ahead of the vote.

GW Pharma’s long road to FDA approval

In the absence of a research-backed drug, some desperate parents of children with epilepsy have turned to CBD oils and other CBD-based products at dispensaries — but most of those are not heavily regulated.

Laura Lubbers, the chief scientific officer of a nonprofit called Cure that funds epilepsy research, told Business Insider her group saw GW’s drug as a “long-awaited” treatment. That’s especially true for patients who haven’t responded to other drugs.

“What’s different with this drug is that this is a well-studied and well-controlled product,” Lubbers said.

Because GW Pharmaceuticals was able to show that its product addresses a critical need, it was able to apply for a designation to fast-track the Food and Drug Administration’s often protracted approval process.

One clinical trial of the drug looked at its effects in 225 young people with Lennox-Gastaut syndrome. The researchers split the study participants into groups and gave them either a high dose of the drug, a low dose, or a placebo for 14 weeks. The results were presented at an American Academy of Neurology meeting last year, and they showed that participants in the high-dose group saw their seizure occurrence drop by 42%. Those given the low dose saw a decrease of roughly 37%. By comparison, those given the placebo saw only a 17% reduction in seizure occurrence.

The second trial, the results of which were published in May 2017 in the New England Journal of Medicine, looked at 120 children with Dravet syndrome. Half were given the drug, and half received a placebo. Forty-three percent of the participants given the drug saw their seizures reduced by half, and 5% stopped having seizures entirely. The group given the placebo saw barely any improvement.

The FDA vote and the future of cannabis-derived drugs

While Epidiolex would be the first cannabidiol-based drug to land FDA approval, the agency has already given the green light to other drugs that contain a lab-made version of THC. Sold under the brand names Marinol and Syndros, the drugs are designed to treat some negative side effects of chemotherapy and AIDS, such as nausea, loss of appetite, and weight loss.

In its public meeting on Thursday, a panel of outside scientists convened by the FDA decided that Epidiolex was safe and effective. Their unanimous vote serves as a recommendation that will be considered when the final decision on whether to approve the drug is made.

“This is clearly a breakthrough drug for an awful disease,” John Mendelson, a panel member and senior scientist at the Friends Research Institute, said after the vote.

Epidiolex would be designed to treat only two types of epilepsy, so FDA approval would mean the drug would be prescribed for a small group of patients. But medical professionals could technically prescribe it “off-label” for other conditions as well. (The anesthetic ketamine, for example, may be prescribed this way for some hard-to-treat forms of depression.)

“We would expect that once this is approved as a drug it’s quite likely this will be tried in other populations off-label so it has a big opportunity to affect others,” Lubbers said.

According to the Centers for Disease Control and Prevention, epilepsy affects more than 3.4 million Americans. Though GW Pharma’s current drug focuses only on two rare types of the condition, the company has said it is exploring treatments for various other forms of epilepsy, too.

If this initial drug gets the green light, that approval will likely galvanize new research into other marijuana-based drugs as well.

Open-Label Phase 2 Trial of ZYN002 in Developmental and Epileptic Encephalopathies

Zynerba Pharmaceuticals announced that it has initiated the Phase 2 BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy [DEE]) clinical trial.

As quoted in the press release: “DEE is a heterogeneous group of epilepsy syndromes that involve significant developmental impairment or regression of developmental progress, and are highly resistant to treatment,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer. “Little attention is paid to DEE outside of Lennox-Gastaut and Dravet syndromes despite the high unmet need. The BELIEVE 1 study will assess children and adolescents with a variety of epilepsy syndromes that are characterized as DEE. Our goal is to deliver an effective, well tolerated, and easy to administer therapeutic option for individuals living with DEE and their families.”

Cannabidiol helps reduce seizures in people with severe form of epilepsy, study suggests

A key cannabis compound helped to reduce the number of drop seizures in patients with a severe form of epilepsy, newly published results of a clinical trial suggest.

The study, published by The Lancet, suggests that cannabidiol, alongside other anti-epilepsy treatments, reduced drop seizures in those who suffer from Lennox-Gastaut syndrome.

Drop seizures involve sudden falls due to loss of muscle tone. Lennox-Gastaut is a severe, lifelong form of epilepsy involving frequent seizures and cognitive impairment. The syndrome causes about one to four per cent of childhood epilepsy cases, researchers say.

In the 14-week clinical trial, the frequency of seizures was reduced in those who took a pharmaceutical formulation of cannabidiol. But researchers say the long-term efficacy and safety of the cannabis compound, as well its interaction with other epilepsy drugs, still need to be studied and confirmed.

The study’s lead author, Dr. Elizabeth Thiele, a neurologist who specializes in pediatric epilepsy at the Massachusetts General Hospital, said she and her colleagues are “pleased that our study has potentially found an additional option to add to patients’ existing treatment to reduce drop seizures.” In a news release, she noted that “it’s important to highlight that the drug used in this trial is a pharmaceutical formulation, and not medical marijuana.”

CURE Responds to DEA’s Decision on Marijuana Scheduling

Chicago, IL – This month the Drug Enforcement Administration (DEA) announced their decision to maintain marijuana as a Schedule I drug, despite petitions from Citizens United for Research in Epilepsy (CURE) and many other research and patient-focused organizations concerned with the federal barriers prohibiting researchers from fully understanding the potential of medical cannabis. Despite this setback, CURE is pleased with the DEA’s decision to expand the number of DEA-registered marijuana manufacturers for research, of which there is currently only one, so more researchers will be able to conduct much-needed research on cannabidiol (CBD) – the major non-psychoactive ingredient in marijuana, as well as the many other cannabinoids found in marijuana.

An estimated 3 million Americans currently live with epilepsy and for two-thirds of them, the cause is unknown, making treatment difficult if not impossible to pinpoint. The positive results that some people with epilepsy have been seeing from CBD-rich marijuana extracts are giving so many parents what they have been lacking for so long – hope. CBD may not be the answer for all of these families, but when available medications and surgeries do not stop the uncontrollable seizures, every avenue must be explored.

As the leading nongovernmental agency fully committed to funding research in epilepsy, we believe researchers should be encouraged and supported to test not only pure CBD, but also high CBD/low-THC cannabis, pure-THC and other types of medical marijuana to determine the efficacy of these and other combinations on seizure control and the genesis of epilepsy. We are hoping for more changes to marijuana access that makes it easier for researchers to continue their vital work.

The Fight to Improve Research Access to Cannabis Continues

WASHINGTON, D.C.— Due to its incredible medicinal potential, we continue to believe that more research must be done on marijuana rich in cannabidiol (CBD). In late April, CURE signed a group letter addressed to Chuck Rosenberg, the head of the Drug Enforcement Administration (DEA), urging him to remove cannabis from Schedule I in the Controlled Substances Act.

This would eradicate federal barriers to research, paving the way for more progressive research and new treatments. CURE has joined with other organizations in support of the Compassionate Access, Research Expansion, and Respect States Act (CARERS, S. 683, H.R. 1538) which would facilitate safe and legal access to medical cannabis for patients and physicians acting in accordance with state law and lift federal barriers to research. The CARERS Act would also remove low-THC cannabis from the CSA allowing individuals in states that have created protections for low-THC therapies to more easily access this potential treatment option.

Current regulatory hurdles make it difficult for researchers to gain access to marijuana rich in CBD. There is no debate that the hoops through which researchers must jump to acquire marijuana, or any chemical found in it, are hindering scientific advancement—and CURE is committed to helping researchers overcome these obstacles to advance research in this important area.