John SchroyerDecember 5, 2022
Epidiolex remains the only cannabis-derived medicine approved by the U.S. FDA.
Epidiolex Archives - Green Market Report
John SchroyerDecember 5, 2022
StaffMay 5, 2022Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced financial results for the first quarter of 2022, raised 2022 financial guidance, and provided business updates.
“We’re pleased to raise our top- and bottom-line guidance, driven by our continued execution and significant progress across commercial and R&D in the first quarter, which positions us well for the rest of the year and to achieve Vision 2025,” said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals. “Our recent launches of Xywav, in both narcolepsy and idiopathic hypersomnia (IH), and Rylaze in acute lymphoblastic leukemia (ALL), continue to generate increased prescriber and patient adoption, and demonstrate our ability to deliver innovative new medicines to improve the lives of patients and their families. On the corporate development front, our three recent transactions are aligned with our broader strategy, allowing us to focus on our highest priorities, enhance our pipeline in areas of key interest in neuroscience and oncology and drive long-term shareholder value.”
GAAP net income in 1Q22 was $1.6 million, or $0.03 per diluted share, compared to $121.8 million, or $2.09 per diluted share, for 1Q22. Non-GAAP adjusted net income in 1Q22 was $261.9 million, or $3.73 per diluted share, compared to $228.8 million, or $3.92 per diluted share, for 1Q22.
Epidiolex Results
Jazz reported that its Epidiolex/Epidyolex net product sales increased 6% to $157.9 million in the first quarter of 2022 compared to the same period in 2021, on a proforma basis. Epidiolex/Epidyolex net product sales in the fourth quarter were favorably impacted by approximately $18 million, due to a temporary increase in specialty pharmacy inventory levels at the end of 2021. The majority of this increase reversed in 1Q22, reducing 1Q22 revenues.
“Excluding this impact, we saw double-digit percentage revenue growth in 1Q22 compared to 1Q21, and sequential growth in underlying demand, despite challenges posed by the Omicron variant.”
Jazz said that Epidyolex is now commercially available and fully reimbursed in four of the five key European markets: United Kingdom, Germany, Italy and Spain, with an anticipated launch in France in 2022. The company said it has made significant progress on its European rollout with launches in Spain, Italy and Switzerland in 3Q21 and Ireland and Norway in 1Q22.
Jazz said it expects to initiate a Phase 3 pivotal trial of Epidiolex for Epilepsy with Myoclonic-Atonic Seizures (EMAS), the fourth target indication for Epidiolex, in 1H22. The company continues to strengthen the durability of Epidiolex. Patent US 11,207,292 is Orange Book listed and extends through 2039. This patent covers the composition of the botanically derived cannabidiol (CBD) preparation used in Epidiolex and the treatment of indicated disorders using that CBD preparation.
StaffNovember 10, 2021
After the market closed on Tuesday, Jazz Pharmaceuticals plc (Nasdaq: JAZZ) released its financial results for the third quarter of 2021 and updated financial guidance for 2021. Total revenues for Jazz increased 39% to $838.1 million versus last year’s third quarter. This beat the Yahoo Finance average analyst estimate for revenue of $834 million. The GAAP net loss for the quarter was ($52.8 million), or ($0.86) per diluted share, compared to $148.2 million, or $2.64 per diluted share, for the same time period in 2020.
Non-GAAP adjusted net income for 3Q21 was $261.4 million, or $4.20 per diluted share, compared to $242.1 million, or $4.31 per diluted share, for 3Q20. This also beat the estimate for earnings of $3.37. The stock was moving higher by almost 2% to lately sell at $134. The average price target is $205.
Outlook
Jazz is also raising its full-year earnings guidance, resulting in a reduced GAAP net loss and increased non-GAAP adjusted net income (ANI) on an absolute and per share basis. The revenue guidance given in August was for a range of total revenues between $3,020 to $3,180 and that was changed to a range of $3,020 to $3,100.
The updated non-GAAP ANI range exceeds the upper end of the prior range. The company is also reducing both SG&A and R&D expense guidance on a GAAP and non-GAAP adjusted basis, reflecting progress within its transformation initiatives, improved financial discipline, and strategic capital allocation. The company is narrowing its net sales guidance range for neuroscience and oncology, with a reduced mid-point for oncology net sales guidance which reflects the ongoing impacts of COVID-19 on our legacy products and the Rylaze competitive landscape at launch in 3Q21, resulting in a reduced mid-point for total revenues guidance.
“Last year, we set the ambitious corporate objective of completing five key commercial launches through 2020 and 2021. With the launch of Xywav for idiopathic hypersomnia earlier this month, we have now accomplished this goal, demonstrating our significant execution capabilities and commitment to bring important new medicines forward for patients,” said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. “The successful integration of GW underscores our ability to deliver on transformative M&A to grow our business. While there’s more work to be done, I’m confident we have the right strategy, teams, and capabilities in place to realize the blockbuster potential of Epidiolex and to discover, develop and launch additional novel, innovative medicines leveraging cannabinoid science. Our commercial execution, productive R&D engine and culture of commitment to patients and their families provide a strong foundation for significant and sustained growth.”
Epidiolex Update
Jazz reported that Epidiolex/Epidyolex net product sales were $160.4 million in the quarter, an increase of 21% compared to the same period of 2020 on a pro-forma basis, despite short-term COVID-19 pressure. Recent market research indicates approximately 40% of prescribers are moving Epidiolex up in their treatment algorithm. The company has made significant progress on its European rollout with launches in Spain, Italy and Switzerland in 3Q21. Epidyolex is now commercially available and fully reimbursed in four of the five key European markets: United Kingdom, Germany, Italy and Spain, with an anticipated launch in France in 2022.
The company said it expects to initiate a Phase 3 pivotal trial of Epidiolex for Epilepsy with Myoclonic-Atonic Seizures (EMAS), the fourth target indication for Epidiolex, in 1H22. Jazz also said it continues to strengthen the durability of Epidiolex, and expects a composition of matter-like patent, extending through 2039, to be issued later this year.
Renée Galá, executive vice president and chief financial officer, added, “This is an exciting time of transformation for Jazz, underpinned by operational execution, financial discipline and strategic capital allocation across our business. We continue to deliver on our business and financial targets which has enabled us to rapidly reduce our net leverage ratio to 4.4 times in just five months following the close of the GW transaction. We have also delivered on revenue growth and diversification. Recently launched or acquired products now make up over 50% of net product sales, and we remain on track to meet our goal of at least 65% in 2022. In addition, our prior investments in corporate development are translating into near-term catalysts as we advance JZP385, JZP150 and Zepzelca into important new clinical trials. We will continue to prioritize disciplined capital allocation to assets and activities that drive growth and value, while remaining focused on achieving our net leverage ratio target of less than 3.5 times by the end of next year.”
Debra BorchardtAugust 4, 2021
After the market closed on Tuesday, Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced financial results for the second quarter of 2021 and affirmed non-GAAP adjusted financial guidance for 2021. Jazz also said that it plans to expand the use of GW Pharmaceutical’s drug Epidiolex to other forms of epilepsy.
Total revenues increased 34% to $751.8 Million in the second quarter of 2021 versus $562 million in 2020. This beat the Yahoo Finance average analyst estimate for $735 million. The GAAP net income (loss) for the second quarter of 2021 was ($363.3 million), or ($6.11) per diluted share, compared to $114.8 million, or $2.06 per diluted share, for the second quarter of 2020. The non-GAAP adjusted net income for the second quarter of 2021 was $240.6 million, or $3.90 per diluted share, compared to $207.3 million, or $3.71 per diluted share, for the second quarter of 2020. The average analyst estimate was $3.42. The average price target is $207 and the stock was moving higher in early trading to $171.
“As we enter what we expect to be a period of sustained growth, I have never been more excited about the future for Jazz. The recent approval and launches of Xywav and Rylaze exemplify Jazz today. We are rapidly establishing ourselves as an innovative biopharmaceutical company with expanding R&D capabilities and substantial commercial prowess, underscored by our consistent execution across the business. The addition of the GW cannabinoid platform and related pipeline complement and enhance our own growing R&D capabilities, accelerating our ability to improve the lives of patients,” said Bruce Cozadd, chairman, and chief executive officer of Jazz Pharmaceuticals. “We have now executed four of five planned product launches since the beginning of 2020 and look forward to our anticipated launch of Xywav in idiopathic hypersomnia later this year, a critical step forward for these underserved patients. With 41% of our second quarter net product sales from recently launched or acquired products, we are well on track to meet our revenue diversification targets while driving significant shareholder value.”
Epidiolex Expansion
In the second quarter, Jazz recorded net product sales of $109.5 million for Epidiolex and believes it could become a blockbuster drug. The company said that this represented revenue from the close of the GW transaction on May 5 through the end of the quarter. On an unaudited pro forma basis full second-quarter net product sales were $155.9 million, a 32% increase over the same quarter of 2020. Jazz believes that as COVID restrictions ease in the US, there will be an upturn in patients visiting their physicians, which will drive additional growth of Epidiolex with new patient starts. In addition, Jazz said the European launch is progressing well with favorable pricing and access to date and reimbursement in place in four of the five largest markets. The company also expects to initiate a Phase 3 pivotal trial of Epidiolex for Epilepsy with Myoclonic-Atonic Seizures (EMAS), also known as Doose syndrome, in the first half of 2022. EMAS represents the fourth target indication for Epidiolex.
Robert Iannone — Executive Vice President, Research and Development and Chief Medical Officer said on the earnings call, “Epidiolex is currently approved in three refractory seizure disorders and we expect to initiate a registrational trial in a fourth, epilepsy with myoclonic-atonic seizures or EMAS in the first half of 2022. Patients diagnosed with EMAS, which is also known as Doose syndrome experienced generalized myoclonic-atonic seizures. The onset of EMAS occurs commonly in the first five years of life with the mean age of onset being three years.”
He added, “The trial provides the opportunity to study Epidiolex in a fourth childhood-onset epileptic encephalopathy at a very high unmet need. EMAS is characterized by generalized myoclonic-atonic seizures and this trial will provide the first randomized controlled clinical data with Epidiolex in this seizure-type, which we believe will provide a more fulsome view of the potential effectiveness at Epidiolex in treating a broad range of seizure disorders.”
Outlook
Jazz Pharmaceuticals is reaffirming its previously communicated full year 2021 non-GAAP financial guidance and updating its 2021 GAAP guidance. Revenue is affirmed at $3.02 Billion to $3.18 Billion The non-GAAP adjusted EPS is in the range of $13.40 to $14.70 per share. The company said the guidance includes the addition of GW from the date of close to year-end, approximately eight months. “We have updated our GAAP guidance primarily to reflect the impact of the UK Tax Act, which was enacted in June following our guidance update.”
Debra BorchardtFebruary 26, 2019GW Pharmaceuticals plc (NASDAQ: GWPH) announced its financial results for the quarter ending December 31, 2018 reflecting the company’s first sales of its cannabis drug Epidiolex. The stock jumped almost 8% in after-hours trading as the biotech company beat analyst estimates.
Epidiolex Sales
Epidiolex is the first plant-derived cannabinoid pharmaceutical ever approved by FDA and first ever approved medicine in the U.S. for Dravet syndrome. Net sales began on November 1 and between that day and the end of the year, the company logged sales of $4.7 million. The company said there were approximately 4,500 new patient enrolment forms in the first two month selling period and over 500 physicians had generated dispensed prescriptions in the first two month selling period. Look ahead to 2019, prescriptions in January grew 150% sequentially over December.
Financial Results
GW Pharmaceuticals changed its fiscal year to begin on January 1, 2019. The revenue for the quarter was $6.7 million versus $4.0 million for the same time period in 2017. The average estimate for revenues from Yahoo Finance was $5.35 million. The net loss for the quarter jumped to $71.9 million versus last year’s$61.8 million for the same time period. The company also beat the analyst estimates for earnings by three cents with its loss of $0.20.
“We are pleased by the high level of physician and patient demand for Epidiolex, and by the number of payors that have already made favorable coverage determinations for the product. With US launch taking place part way through the quarter, the two month selling period at the end of 2018 was primarily aimed at setting the commercial wheels in motion for the 2019 launch year. As we move into the New Year, prescription growth trajectory has been highly encouraging and we believe that we are on track to deliver a successful market introduction of this important new treatment,” stated Justin Gover, GW’s Chief Executive Officer. “In addition to the US launch, we look forward to a positive regulatory decision in Europe in the next few months, results of a Phase 3 trial in Tuberous Sclerosis Complex, and a number of advances in the pipeline.”
Debra BorchardtNovember 26, 2018Before Tilray (TLRY) and Canopy Growth (CGC) took over cannabis stock portfolios, GW Pharmaceuticals plc (Nasdaq: GWPH) was the darling of the group. However, the recent positive news from this cannabis biotech company’s epilepsy study serves to remind investors that great good comes from cannabis research.
On Monday, GW Pharma announced positive top-line results of the second randomized, double-blind, placebo-controlled Phase 3 clinical trial of Epidiolex in the treatment of seizures associated with Dravet syndrome, a rare and severe form of childhood-onset epilepsy. The biotech company reported that in this trial, Epidiolex, when added to the patient’s current treatment, achieved the primary endpoint of reduction in convulsive seizures for both dose levels (10 mg/kg per day and 20 mg/kg per day) with high statistical significance compared to placebo. The company said that both doses also demonstrated statistically significant improvements on all key secondary endpoints.
“The positive outcome in this second trial of Epidiolex in patients with Dravet syndrome further reinforces the effectiveness of this newly available medicine in this particularly difficult to treat, childhood-onset epilepsy,” stated Ian Miller, M.D., Director, Epilepsy and Neurophysiology Program at Nicklaus Children’s Hospital in Miami, FL and principal investigator of the trial. “The totality of data from the controlled clinical trials completed for Epidiolex have shown clinically meaningful seizure reductions and a consistent safety and tolerability profile.”
The drug was recently approved by the FDA and given a classification as a schedule five drug in September. Schedule five drugs are considered to have a low level of abuse and include substances like Robitussin cough syrup or Lomotil diarrhea medicine.
“The positive results from this trial follow the recent FDA approval, DEA rescheduling and U.S. launch of EPIDIOLEX for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut Syndrome in patients two years and older. These data show an effective dose range in Dravet syndrome that is consistent with our FDA approved label, and which allows for dosing flexibility to address individual patient needs,” stated Justin Gover, GW’s CEO.
Study Results
The average age of the patients in the study was nine years. The patients were having approximately 12 convulsive seizures a month and approximately 35 total seizures per month. On average, patients were taking three anti-epileptic drugs (AED), having previously tried and discontinued on average, four other AEDs.
The statement said that during the treatment period, patients taking Epidiolex 20 mg/kg/day demonstrated a 46% reduction in convulsive seizures while patients taking Epidiolex 10 mg/kg/day achieved a reduction of 49%, compared to a 27% reduction in patients taking a placebo.
Results from the key secondary efficacy endpoints also showed statistical significance of both dose groups of Epidiolex compared to placebo, although the patients taking the placebo also experienced a drop in seizures. In the Epidiolex 20 mg/kg/day group, 49% of patients achieved a 50% or greater reduction in convulsive seizures from baseline over the treatment period, compared to 44% of patients taking Epidiolex 10 mg/kg/day, and 26 percent of patients taking the placebo.
Stock Performance
The stock jumped over 2% in pre-market trading having experienced a 52-week high of $179 in September, shortly after the change in scheduling. The stock closed on Friday at $124, above its year low of $105.
Debra BorchardtSeptember 27, 2018The Acting Administrator of the Drug Enforcement Administration has placed FDA-approved drugs that contain CBD derived from cannabis and no more than 0.1 percent tetrahydrocannabinols (THC) in schedule V. GW Pharmaceutical’s (GWPH) Epidiolex had been a schedule I controlled substance, with this new directive Epidiolex (and any generic versions of the same formulation that might be approved by the FDA in the future) will be a schedule V controlled substance.
Schedule five drugs are considered to have a low level of abuse and include substances like Robitussin cough syrup or Lomotil diarrhea medicine.
The order made it clear that other cannabis products would remain in the schedule 1 category. The statement said, “As further indicated, any material, compound, mixture, or preparation other than Epidiolex that falls within the CSA definition of marijuana set forth in 21 U.S.C. 802(16), including any non-FDA-approved CBD extract that falls within such definition, remains a schedule I controlled substance under the CSA.” The document is scheduled to be published on September 28, 2018.
The order also said, “Now that Epiodiolex has been approved by the FDA, it has a currently accepted medical use in
treatment in the United States for purposes of the CSA. Accordingly, Epidiolex no longer meets the criteria for placement in schedule I of the CSA.”
The order went on to stress that CBD material, “which includes, among other things, a drug product containing CBD extracted from the cannabis plant, is a Schedule I drug under the Single Convention.”
Dr. George E. Anastassov, Chief Executive Officer of AXIM Biotechnologies remarked, “Today’s news is an important step for the pharmaceutical cannabinoid industry. It signifies an understanding on behalf of the regulatory agencies in the United States, such as the FDA and DEA, that cannabidiol (CBD) has therapeutic potential and a very low potential for abuse and misuse. However, this applies ONLY to Epidiolex, i.e. to the molecule contained within this particular formulation. Otherwise, CBD still remains as a Schedule I controlled substance and the current classification is non-applicable to the rest of the products available on the market.”
Thoma Kikis, Chief Communications Officer of Kannalife Sciences said, “The DEA’s rescheduling of Epidiolex from a Schedule I to a Schedule V controlled substance gives cannabidiol (CBD) more credibility and recognition as a treatment in the U.S. that does, in fact, have medical value. This is a watershed moment for the cannabinoid industry, and very encouraging for Kannalife Sciences and the work we have done to research and develop our own CBD-derived molecule (KLS-13019) for the treatment of diseases with unmet medical needs, such as Chemotherapy-Induced Neuropathic Pain (CINP), Hepatic Encephalopathy (HE), and Chronic Traumatic Encephalopathy (CTE). However, there is still more work to be done.”
GW Pharmaceutical stock jumped over 8% on the news to lately trade at $176.
Debra BorchardtJune 25, 2018The U.S. Food and Drug Administration (FDA) approved GW Pharmaceuticals’ (GWPH) cannabidiol (CBD) drug Epidiolex as an oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome, and Dravet syndrome, in patients two years of age and older. According to the press release, this is the first FDA-approved drug that contains a purified drug substance derived from marijuana and it is also the first FDA approval of a drug for the treatment of patients with the Dravet syndrome.
“Today, the FDA approved a purified form of the drug cannabidiol (CBD). This is one of more than 80 active chemicals in marijuana. The new product was approved to treat seizures associated with two rare, severe forms of epilepsy in patients two years of age and older,” said FDA Commission Scott Gottlieb, M.D.
He went on to add, “But it’s also important to note that this is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use. And it was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition. Moreover, this is a purified form of CBD. It’s being delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits. This is how sound medical science is advanced.”
Gottlieb cautioned though that he remains concerned about the proliferation and illegal marketing of unapproved CBD-containing products with unproven medical claims. The FDA has taken action against companies making unsupported CBD claims in the past couple of years and the agency said it would continue to do so.
GW Says Rescheduling Within 90 Days
Even though the FDA has now approved the use of Epidiolex as a real treatment for these diseases, it doesn’t immediately change the status of cannabis as a controlled substance. The two defining factors of the schedule one designation include not having a medicinal purpose and being a highly addictive drug.
The FDA release stated, “Under the Controlled Substances Act (CSA), CBD is currently a Schedule I substance because it is a chemical component of the cannabis plant. In support of this application, the company conducted nonclinical and clinical studies to assess the abuse potential of CBD. The FDA prepares and transmits, through the U.S. Department of Health and Human Services, a medical and scientific analysis of substances subject to scheduling, like CBD, and provides recommendations to the Drug Enforcement Administration (DEA) regarding controls under the CSA. DEA is required to make a scheduling determination.”
The FDA did not say in its release whether it had delivered a recommendation to the DEA regarding Epidiolex and its schedule status.
However, in GW Pharmaceutical’s statement, the company said it would have to be rescheduled before it could be made available to patients. “Rescheduling is expected to occur within 90 days. Access is expected to be similar to other branded AEDs and EPIDIOLEX is expected to be available to appropriate patients by Fall 2018.”
“While we applaud the FDA for confirming what many of us have long known–that the benefits of cannabis are immense–we do find it a bit comical that the federal government now contradicts itself once again,” said Chris Driessen, President of Organa Brands U.S. “While the FDA touts the benefits of this new drug, the DEA maintains a Schedule One classification for cannabis, stating that it has no medical application and a high potential for abuse. We have to ask–does the left hand know what the right hand is doing?
Management Comments
“Today’s approval of EPIDIOLEX is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” said Justin Gover, GW’s Chief Executive Officer. “This approval is the culmination of GW’s many years of partnership with patients, their families, and physicians in the epilepsy community to develop a much needed, novel medicine. These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care.”
“In my practice, I often see patients with these highly treatment-resistant epilepsies who have tried and failed existing therapies and are asking about CBD,” said Orrin Devinsky, M.D., of NYU Langone Health’s Comprehensive Epilepsy Center and a lead investigator in the EPIDIOLEX clinical program. “I am delighted that my physician colleagues and I will now have the option of a prescription cannabidiol that has undergone the rigor of controlled trials and been approved by the FDA to treat both children and adults.”
“For those living with intractable seizures caused by LGS and Dravet syndrome, EPIDIOLEX represents a true medical advancement,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “Clinical development for these rare and severe conditions is essential, and today’s news brings hope for these patients and their families that a new treatment option may have the potential to help better control their seizures.”
Epidiolex
Epidiolex’s effectiveness was studied in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures when compared with placebo. It uses CBD, which is a chemical component of the Cannabis sativa plant. CBD does not give patients a psychotropic effect like getting “high” that comes from the other part of the cannabis plant – tetrahydrocannabinol (THC).
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