Zynerba Archives - Green Market Report

StaffStaffMay 12, 2021
lab2.jpg?fit=960%2C640&ssl=1

3min3010

Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) reported financial results for the first quarter ended March 31, 2021. As an emerging biotech company, Zynerba does not have revenue at this time as it develops drugs. Research and development expenses were $4.6 million for the first quarter of 2021, including stock-based compensation of $0.6 million. General and administrative expenses were $3.3 million in the first quarter of 2021, including stock-based compensation expense of $0.6 million.

The net loss for the first quarter of 2021 was $8.0 million with a basic and diluted loss per share of $(0.20). Zynerba beat the Estimize EPS Consensus by $0.01 by reporting a net loss per share of ($0.20).

“We are committed to delivering on our important milestones in 2021 as we develop Zygel in multiple neuropsychiatric indications, including initiating a confirmatory pivotal Phase 3 trial, RECONNECT, in the third quarter of 2021, after productive dialogue and alignment with the FDA,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “With a cash runway that takes us well into the first half of 2024, we believe that we are ideally positioned to continue our efforts to develop the first FDA-approved treatment for patients with Fragile X syndrome.”

Fragile X Update

Wither regards to Fragile X, Zynerba said it expects to initiate RECONNECT (A Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome), a pivotal, multi-national confirmatory Phase 3 trial of Zygel in children and adolescents with FXS, in the third quarter of 2021. The trial is designed to confirm the positive results observed in a population of responders in the company’s previously conducted CONNECT-FX trial. The RECONNECT trial will be an 18-week trial which will enroll approximately 200 children and adolescents of which approximately 160 patients will have complete (100%) methylation of their FMR1 gene and approximately 40 patients will have partial methylation of their FMR1 gene.

As of March 31, 2021, cash and cash equivalents were $93.1 million, compared to $59.2 million as of December 31, 2020. Zynerba said it believes that the company’s cash and cash equivalents as of March 31, 2021, are sufficient to fund operations and capital requirements well into the first half of 2024.


Debra BorchardtDebra BorchardtMarch 10, 2021
lab.jpg?fit=960%2C640&ssl=1

15min3310

Biotech company  Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) reported its fourth-quarter and full-year results for the period ending in December. The company has no revenues to report at this time. The research and development expenses were $5.6 million for the fourth quarter of 2020, including stock-based compensation of $0.6 million. General and administrative expenses were $4.6 million in the fourth quarter of 2020, including stock-based compensation expense of $0.6 million. The net loss for the fourth quarter of 2020 was $9.6 million with a basic and diluted loss per share of $(0.33). Zynerba beat estimates which were ($0.38).

For 2020, research and development expenses were $35.7 million, including stock-based compensation of $2.2 million. General and administrative expenses were $16.4 million for full-year 2020, including stock-based compensation expense of $3.0 million. The net loss for the full year 2020 was $51.3 million with basic and diluted net loss per share of $(1.90).

“We expect to make significant progress in 2021 on all four indications for which we are developing Zygel, including initiating a pivotal trial in patients with Fragile X syndrome who have a highly methylated FMR1 gene to confirm the positive results in this population of responders in the CONNECT-FX trial,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “Screening in the INSPIRE trial of patients with 22q11.2 deletion syndrome has resumed now that COVID-19 restrictions in Australia have begun to ease. Once enrollment is complete we will update our expectation on when we will see topline results for this trial.”


StaffStaffNovember 9, 2020
Daily-Hit-Header.png?fit=960%2C400&ssl=1

9min3790

It’s time for your Daily Hit of cannabis news for November 9, 2020

On the Site

Canopy Growth

Canopy Growth Corporation (NYSE: CGC) reported net revenue of $135.3 million for the second quarter fiscal 2021 ended September 30, 2020, causing the stock to jump in early trading. This was a 77% increase over last year’s fiscal second-quarter revenue of $76.6 million. Still, Canopy delivered a net loss of $96.6 million versus last year’s net income of $242 million for the same time period.

The company also reported a loss per share of ($0.09) which beat the MarketWatch estimate for a loss of ($0.28). The stock was lately trading at $26, an increase of 13%. The company attributed the revenue growth to an increase in Canadian recreational revenue, continued strength in Storz & Bickel vaporizer sales and ThisWorks, and contribution from BioSteel, which was acquired in October 2019 . The net loss was driven by lower other income. Canopy also said that the increase versus the prior year period also benefited from favorable comparison, as Q2 2020 results included a $32.7 million charge for returns, return provisions, and pricing allowances primarily related to restructuring the company’s recreational softgel & oil portfolio.

Tilray

Tilray, Inc. (Nasdaq: TLRY) reported that its total revenue for the third quarter was flat at $51.4 million and up 2.0% sequentially. Net losses fell to  $(2.3) million versus last year’s net loss of $(36.4) million and fell sequentially from $(81.7) million in the second quarter. The most significant driver of the change in net loss during the period was the revaluation of the outstanding warrants associated with the equity offering completed in March. Tilray stock was slightly higher in after-hours trading.

The company attributed the disappointing results on the discontinuation of bulk sales and a slight decrease in Canada medical sales which caused cannabis segment revenue to fall by 11% to $31.4 million. Total cannabis kilogram equivalents sold decreased 53% to 5,107 kilograms from 10,848 kilograms in the prior year’s third quarter. Adult-Use and International Medical sales grew 26% and 42%, respectively. Excluding the year-over-year impact related to bulk sales, total cannabis revenue increased by 24%. Hemp segment revenue increased 28% to $20.0 million (C$26.5 million).

Aurora Cannabis

Canadian-based cannabis company, Aurora Cannabis (NYSE: ACB) reported their Q1 earnings this morning. The results were mixed at best, with shares rising 21% on the potential for cannabis legalization under a Biden administration. Unless otherwise stated, these figures are in Canadian dollars. 

The company’s adjusted gross margin before fair value adjustments on total cannabis net revenue didn’t waver much quarter to quarter, with Aurora Cannabis reporting a 48% adjusted gross margin compared to 50% in Q4 2020. Before fair value adjustments, the company’s adjusted gross margin on cannabis net revenue was 52%. 

Canopy Rivers

Canopy Rivers Inc. (OTC: CNPOF) today released its unaudited condensed interim consolidated financial statements in Canadian dollars and acknowledged taking a $112 million hit for its PharmHouse investment.  The total comprehensive loss for the quarter was $87.0 million. On a positive note, its investment into TerrAscend has appreciated implying an investment value of $214 million.

The company reported that its royalty, interest, and lease income (before provisions for credit losses) was $4.1 million for the quarter. It included income from its various royalty, convertible debenture, and loan agreements, among other items. Other comprehensive income was $23.4 million, net of tax, for the quarter, which included a net increase in the fair value of financial assets of $27.4 million attributed to the positive change in the fair value of the investment in TerrAscend. TerrAscend’s share value increase from $2.87 on June 30, 2020, to $9.75 as of the close of markets on November 6, 2020.

Zynerba

Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) reported a net loss of $9 million for the third quarter ending September 30, 2020, with a basic and diluted net loss per share of $(0.31). This beat the Yahoo Finance average analyst estimate for a loss of ($0.43). Six analysts have a Hold rating on the company, while two give it a Buy rating. The company still does not have a revenue-producing drug, but Zynerba said it has enough money until that time comes.

Planet 13

Planet 13 (CSE:PLTH) (OTCQX:PLNHF) is potentially one of the most well-known dispensaries around. They are incredibly innovative, massive in size, and just all around at the top of the dispensary game. Planet 13 is even more special because currently, they’re only located in Las Vegas, Nevada. For having operations in only one state, they sure do gain a ton of attention!

The cannabis megastore just announced their third consecutive month of generating over $7.5 million in revenue. The company’s October revenue clocked out at $7.6 million, with gross margins above 50%. Planet 13’s figures include sales for the SuperStore, as well as Nevada wholesale. 

In Other News

Schultze Special Purpose Acquisition Corp. (NASDAQ: SAMA, SAMAW, and SAMAU) and Clever Leaves International Inc. announced today that they have amended their definitive agreement, which was entered into on July 25, 2020 and is anticipated to become a NASDAQ-listed public company trading under the ticker symbol “CLVR”.

 Under the amended terms, the initial expected enterprise value has been reduced to $206 million from $255 million and the minimum cash condition for SAMA has been reduced to $26 million from $60 million. Additionally, the cash consideration payable to certain Clever Leaves’ shareholders at closing has been amended, thereby increasing the equity rollover consideration of the transaction to approximately 97% while Schultze Special Purpose Acquisition Sponsor, LLC agreed to restructure its’ equity ownership to better align with the capital retained at closing. In connection with these revised terms, institutional investors have committed over $10 million through a private placement to be funded at closing of the Business Combination. Additionally, select SAMA stockholders have agreed not to redeem their shares held thereby providing a path to over $16 million of additional committed capital and thus having adequate capital to consummate the transaction. When including SAMA’s cash in trust, the parties expect to have over $80 million of cash on its balance sheet following closing.

 


StaffStaffNovember 9, 2020
lab2-2-scaled.jpg?fit=960%2C641&ssl=1

5min5350

Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) reported a net loss of $9 million for the third quarter ending September 30, 2020, with a basic and diluted net loss per share of $(0.31). This beat the Yahoo Finance average analyst estimate for a loss of ($0.43). Six analysts have a Hold rating on the company, while two give it a Buy rating. The company still does not have a revenue-producing drug, but Zynerba said it has enough money until that time comes.

Research and development expenses for the third quarter of 2020 were $5.8 million, including stock-based compensation of $0.5 million. General and administrative expenses for the third quarter of 2020 were $3.4 million, including stock-based compensation expense of $0.7 million.   As of September 30, 2020, cash and cash equivalents were $64.3 million and the company said it believes that the current cash and cash equivalents are sufficient to fund operations and capital requirements until late in the fourth quarter of 2021.

“We made good clinical, operational and regulatory progress during the third quarter of 2020 including presenting new data from the pivotal CONNECT-FX and Phase 2 BRIGHT trials, and completing our discussions with the FDA to clarify our clinical path forward to late stage clinical trials in patients with certain developmental and epileptic encephalopathies,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “The fourth quarter of this year is another important period for Zynerba. In particular, we look forward to announcing the results of our fourth quarter meeting with the FDA to discuss our pivotal CONNECT-FX results in patients with a fully methylated FMR1 gene and to understand the regulatory path forward.”

Fragile X

Zynerba said it expects to announce the outcome of its fourth-quarter meeting with the U.S. Food and Drug Administration (FDA) to discuss the CONNECT-FX Trial and the Regulatory path forward for Zygel in pediatric patients in the fourth quarter of 2020. This past summer the company disclosed that its latest top-line results from the 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial failed to produce the necessary threshold for positive results. However, the company went back to the drawing board and in October said, “Although the CONNECT-FX full analysis set did not achieve statistical significance in its endpoints, building on current scientific evidence, a pre-planned ad hoc analysis of patients having at least 90% methylation (“full methylation” or FMet) of the impacted FMR1 gene, representing 80% of the overall study population, was performed. The results, including the achievement of statistical significance (p=0.020) in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo, suggesting that Zygel may have benefit in patients with full methylation of the FMR1 gene.”

Autism

The company also reviewed its autism study saying that patients receiving Zygel in this study achieved statistically significant caregiver-reported improvements compared to baseline across all subscales of the Autism Impact Measure, which was designed to measure change in frequency and impact of core ASD symptoms: Atypical behavior (p<0.001), Communication (p<0.001), Peer Interaction (p<0.001), Repetitive Behavior (p<0.001), and Social Reciprocity (p=0.0053). In addition, statistically significant improvements compared to baseline were observed at week 14 of treatment with Zygel in the Autism Parenting Stress Index (p<0.0001). Zynerba also measured notable improvements in behaviors utilizing the Qualitative Caregiver Behavioral Problems Survey after 14 weeks of study drug. Clinically meaningful improvements were observed by a majority of surveyed caregivers in behavioral, social, and emotional behavioral problems.


Debra BorchardtDebra BorchardtJune 30, 2020
zynerba-homepage-scaled.jpg?fit=1200%2C544&ssl=1

5min12700

Some drugs work and at other times they don’t, which is the whole point of testing and the importance of trying to find solutions to patient conditions. Unfortunately for Zynerba Pharmaceuticals, Inc. (ZYNE) its latest top-line results from the 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial failed to produce the necessary threshold for positive results. The stock was selling off as a result of the news.

The Zygel CBD gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 212 patients did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS). Zynerba also said that Zygel also did not demonstrate statistical significance versus placebo in the three key secondary endpoints, which were the change from baseline to the end of the treatment period in the Irritability subscale score of the ABC-CFXS, the Socially Unresponsive/Lethargic subscale score of the ABC-CFXS and Improvement in Clinical Global Impression (CGI-I).

“This study identified a key population of patients who appear to benefit from treatment of their behavioral symptoms of FXS with Zygel,” said Randi J. Hagerman, MD, an investigator in the clinical trial and Medical Director and Endowed Chair in Fragile X Research at UC Davis MIND Institute and Distinguished Professor at the Department of Pediatrics at UC Davis School of Medicine. “Zygel has the potential to be an important therapeutic option for the most severely impacted patients with Fragile X.”

Study Results

While the drug wasn’t a statistical success, the company felt that enough patients responded positively that it wants to meet with the FDA to discuss a future path for the drug.

“The results from CONNECT-FX identified a significant patient population who responded well to Zygel and may provide us with a pathway towards licensure,” said Armando Anido, Zynerba’s Chairman and Chief Executive Officer. “We intend to discuss the results of the study with the FDA as soon as possible. On behalf of the entire Zynerba team, I want to sincerely thank the patients, families and investigators who participated in this study as well as the National Fragile X Foundation, the FRAXA Research Foundation, and the Fragile X Association of Australia for their assistance in this study.”

The company posted the following study details: Two hundred and forty-five (245) patients with Fragile X syndrome, confirmed with the full mutation of the FMR1 gene, were enrolled at 21 clinical sites in the United States, Australia, and New Zealand. Unknown to the patients and their caregivers, all patients were given placebo during the first two weeks (called a “placebo run-in” which is often used in neuropsychiatric clinical trials), and as a result 33 patients were not randomized. The remaining 212 patients were included in the Intent-to-Treat (ITT) population (Zygel: n=110; placebo: n=102) and were randomized to receive either trial drug or placebo for an additional 12 weeks. One patient did not receive study medication so 211 patients are included in the safety analysis (Zygel: n=109; placebo: n=102.) One patient did not have a post-baseline efficacy measure, resulting in 210 patients in the full analysis set (Zygel: n=109; placebo: n=101).

A pre-planned ad hoc analysis of the most severely impacted patients in the trial, as defined by patients having at least 90% methylation (“full methylation”) of the impacted FMR1 gene, demonstrated that patients receiving Zygel achieved statistical significance in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo (p=0.020). This group comprised 80% of the patients enrolled in the CONNECT-FX study. The Company believes that full methylation occurs in approximately 60% of the overall FXS patient population. Based on this analysis, Zynerba intends to meet with the FDA regarding a regulatory path forward for Zygel.

 

 

 


StaffStaffMay 27, 2020
lab2-2-scaled.jpg?fit=960%2C641&ssl=1

4min10920

Zynerba Pharmaceuticals, Inc. (ZYNE) announced positive top-line results from the exploratory, open-label Phase 2 BRIGHT study (An Open-Label Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Autism Spectrum Disorder) trial. Autism Spectrum Disorder is a developmental disorder that affects communication and behavior in approximately one million pediatric and adolescent patients between the ages of five and 17 in the U.S.

The stock was moving higher by over 8% at one point in early trading to lately sell at $6.69. Most of the analysts covering the stock have rated it a Hold according to Yahoo Finance. The average price target is $15.33.

The trial was designed to assess the safety, tolerability, and efficacy of the Zynerba drug called Zygel in pediatric and adolescent patients with autism spectrum disorder (ASD). Zygel was administered to patients with moderate-to-severe symptoms of ASD as add-on therapy to their standard of care utilizing a variety of efficacy assessments.

“We are very encouraged by the compelling top-line results of the BRIGHT trial and we expect to meet with the FDA to discuss the clinical pathway for developing Zygel for the treatment of behavioral symptoms of ASD in the second half of this year,” said Armando Anido, Zynerba’s Chairman and Chief Executive Officer. “Our goal is to develop Zygel for patients suffering from debilitating neuropsychiatric disorders including ASD, Fragile X syndrome, 22q and DEE. I want to thank the patients, families, physicians, clinical staff, and the Zynerba team for their support of this key study in ASD.”

Key findings from the trial disclosed today include:

  • All five subscales of the Aberrant Behavior Checklist – Community (ABC-C) as well as the Parent Rated Anxiety Scale – Autism Spectrum Disorder (PRAS-ASD) showed both statistically significant and clinically meaningful improvements at 14 weeks of treatment from baseline;
  • The results observed in other efficacy outcome measures, including Clinical Global Impressions – Improvement scale (CGI-I), support the subscale results observed in the ABC-C;
  • Zygel was well tolerated in this trial with no serious or severe adverse events reported.

“I am very impressed with the improvements my patients made over the 14-week treatment period while receiving Zygel; the reduction in irritability, communication deficits, and repetitive movements were especially noteworthy since some of these are core autistic behaviors,” said Helen Heussler, FRACP, Associate Professor at Children’s Health Queensland, Medical Director Child Development and principal investigator in the BRIGHT trial. “The magnitude of effect on autistic behaviors in this trial is significant, including hyperactivity and stereotypy, which are among the most difficult behaviors to improve with therapeutic intervention. The results of this study strongly suggest the potential of this drug as an important treatment for ASD and I look forward to participating in future clinical studies with Zygel.”


Debra BorchardtDebra BorchardtMay 11, 2020
lab.jpg?fit=960%2C640&ssl=1

4min9320

Zynerba Pharmaceuticals, Inc. (ZYNE) reported its earnings for the first quarter ending March 31, 2020, with no revenue and a net loss of $12.3 million and a net loss per share of $(0.53). Despite COVID-19 disruptions, the company said that it expects timelines for delivery of top-line results from all of its ongoing trials will not be impacted.

“The past few months have been historic due to the outbreak of COVID-19, and we applaud everyone on the front line of this global pandemic, including healthcare workers, first responders, humanitarians, and our industry colleagues who are dedicated to the rapid development of treatments and vaccines,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “In response to the pandemic, Zynerba implemented important initiatives that we believe protect the safety of patients, clinical investigators and their staff, and our Zynerba employees and should allow us to conclude all three of our ongoing clinical trials and report top-line results within our stated timelines.”

Second Quarter Events

While the first quarter kept chugging along, Zynerba’s second quarter looks to be shaping up as a big one for news. If Zynerba gets positive results from its Fragile X study, the company said it intends to request a meeting with the FDA to determine the acceptability of the data as a basis for a New Drug Application (NDA) and to seek advice on the preparation of the marketing authorization. Zynerba also said it expects to submit its NDA for Zygel in FXS to the FDA in the second half of 2020, with potential approval by mid-year 2021.

Zynerba is also expecting top-line results from its trail for Zygel in treating children with autism in the second quarter. The U.S. Patent and Trademark Office has issued U.S. Patent No. 10,568,848, titled “Treatment of Autism with Cannabidiol” which includes claims directed to methods of treating ASD by transdermally administering, via a gel or cream, a therapeutically effective amount of purified CBD. The patent expires in 2038.

Zynerba said that it anticipates that it will pursue an indication that includes the syndromes and encephalopathies in the DEE category that presents with focal impaired-awareness seizures and expects to disclose the outcome of the interactions with the FDA in the second quarter of 2020.

Enough Cash Cushion

The company believes that its cash runway is sufficient to fund operations and capital requirements beyond the expected NDA submission and potential approval of Zygel in FXS and into the second half of 2021. The company reported that it has cash and cash equivalents of $60.6 million, compared to $70.1 million as of December 31, 2019. Research and development expenses for the first quarter of 2020 were $6.9 million, including stock-based compensation of $0.5 million. General and administrative expenses for the first quarter of 2020 were $3.9 million, including stock-based compensation expense of $0.8 million.


Debra BorchardtDebra BorchardtMarch 10, 2020
lab.jpg?fit=960%2C640&ssl=1

4min10770

Cannabis pharmaceutical company  Zynerba Pharmaceuticals, Inc. (ZYNE) stock was moving higher in premarket trading after the company reported financial results for the fourth quarter and full-year ending December 31, 2019.

The company had no revenue for 2019 as it is currently developing drugs. The net loss for the fourth quarter of 2019 was $10.7 million with a basic and diluted net loss per share of $(0.46). The net loss for the full year of 2019 was $32.9 million with a basic and diluted net loss per share of $(1.50).

The company said it has $70.1 million in cash. Zynerba believes that the cash runway is sufficient to fund operations and capital requirements beyond the expected NDA submission and potential approval of Zygel in FXS and into the second half of 2021.

“The fourth quarter of 2019 capped off a year of strong execution by Zynerba,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “With a number of shots-on-goal in our clinical pipeline, each with near term milestones, our outlook is promising for the remainder of 2020 and beyond. We are positioned for major news events throughout this year and next, including the topline results from our pivotal CONNECT-FX trial of Zygel™ in patients with Fragile X syndrome which are expected late next quarter.”

Zygel

Enrollment is complete with 212 patients randomized into CONNECT-FX, a pivotal, multinational, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Zygel in treating common behavioral symptoms of FXS.

Zynerba said it expects to report topline results late in the second quarter of 2020. If the results are positive, Zynerba intends to request a meeting with the FDA to determine the acceptability of the data as a basis for a New Drug Application (NDA) and to seek advice on the preparation of the marketing authorization. The company expects to submit its NDA for Zygel in FXS to the U.S. Food and Drug Administration (FDA) in the second half of 2020, with potential approval by mid-year 2021.

Autism Trial

Enrollment is complete in Phase 2 BRIGHT trial assessing the safety, tolerability and efficacy of Zygel for the treatment of pediatric and adolescent patients with ASD. The 14-week trial is evaluating the efficacy and safety of Zygel in 37 children and adolescents (ages four through 17) with moderate-to-severe ASD. The mean age of the patients is 9.2 years old and  92% of the enrolled patients are male. Zynerba expects to report topline results from this study in the second quarter of 2020.


Debra BorchardtDebra BorchardtFebruary 3, 2020
shutterstock_757996678.jpg?fit=960%2C641&ssl=1

9min20700

GW Pharmaceuticals

GW Pharmaceuticals (NASDAQ:GWPH) has filed a supplemental marketing application in the U.S. seeking approval to use Epidiolex (cannabidiol) oral solution to treat seizures associated with tuberous sclerosis complex, a rare inherited disorder characterized by the growth on non-cancerous tumors in many parts of the body. It affects approximately 40-80 thousand individuals in the U.S. and nearly one million people worldwide.

“The submission of this sNDA for Epidiolex is an important step towards the prospect of offering a new treatment option for those patients with TSC who battle difficult-to-treat seizures,” said GW Pharmaceuticals CEO, Justin Gover. “Having already obtained approval for Epdiolex in the treatment of seizures associated with Lennox-Gastaut Syndrome and Dravet Syndrome, this submission is based on positive Phase 3 data showing that Epidiolex reduced TSC-associated seizures, which include both focal and generalized seizures types. We look forward to working with the FDA toward an expected approval later this year.”

The statement said that TSC is a condition that causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys, and lungs and is a leading cause of genetic epilepsy. TSC is typically diagnosed in childhood. More than 60% of individuals with TSC do not achieve seizure control with standard treatments.

Study Data

The sNDA is supported by data from a Phase 3 safety and efficacy study, results of which were recently presented at the American Epilepsy Society 2019 annual meeting. The study met its primary endpoint with patients treated with Epidiolex 25 mg/kg/day experiencing a significantly greater reduction from baseline in TSC-associated seizures compared to placebo (49% vs 27%; p=0.0009). Results for the 50 mg/kg/day dose group were similar, with seizure reductions of 48% from baseline vs 26.5% for placebo (p=0.0018).

Zynerba

Zynerba Pharmaceuticals (NASDAQ:ZYNE)reported that it has successfully achieved patient screening target in its 14-week CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial assessing the efficacy and safety of Zygel CBD Gel in children and adolescents ages three to 17 with full mutation Fragile X syndrome.

“This is an important milestone for patients with Fragile X syndrome and their caregivers as we move toward completion of enrollment in this pivotal trial,” commented Armando Anido, Chief Executive Officer of Zynerba. “I would like to thank all of our clinical investigators and their staff for their assistance thus far. Through strict entry criteria and overall trial design, we expect to enhance the study’s ability to demonstrate a strong signal of activity and minimize response variability. Having now achieved our screening target, we remain confident that we will announce topline results late in the second quarter of this year.”

The company said in a statement that it expects to disclose topline results of this study late in the second quarter of 2020. If the results are positive, the Company expects to meet with the U.S. Food and Drug Administration to determine the acceptability of the data as a basis to submit its New Drug Application for Zygel in FXS in the second half of 2020, with potential approval by mid-year 2021.


Debra BorchardtDebra BorchardtNovember 6, 2019
stocks.jpg?fit=960%2C640&ssl=1

5min9810

Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE) saw its stock lift ever so slightly on news that the company has positive results from its drug trials. Shares were up by two cents in pre-market trading to $7.15.

Study Results

The company said that the topline results of this six-month Phase 2 evaluation of Zygel in 48 children and adolescents with various DEEs showed meaningful reductions in seizures and excellent tolerability. Patients experienced 44% to 58% monthly median reductions in focal impaired-awareness seizures (FIAS; previously known as complex partial seizures) and/or convulsive seizures (CS; focal to bilateral tonic-clonic seizures and generalized tonic-clonic seizures), the most common and debilitating seizure types, starting at month two and continuing through month six.

Zynerba also noted that enrollment was progressing in CONNECT-FX, a pivotal, multinational, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Zygel in treating common behavioral symptoms of FXS in three through 17-year old patients with Fragile-X Syndrome. The company expects to report top-line results in the first half of 2020.

In addition to those results, Zynerba said it was conducting the Phase 2 BRIGHT trial to assess the safety, tolerability, and efficacy of Zygel for the treatment of child and adolescent patients with Autism Spectrum Disorder (ASD). The 14-week trial is designed to evaluate the efficacy and safety of Zygel in approximately 36 children and adolescents (ages four through 17) with ASD as confirmed by DSM-5 diagnostic criteria for ASD. The efficacy assessments include the Aberrant Behavior Checklist, Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism Impact Measure, and Clinical Global Impression – Severity and Improvement. Zynerba expects to report topline results from this study in the first half of 2020.

“The third quarter of 2019 was a remarkable period of progress and execution for Zynerba,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We announced compelling topline safety and efficacy results from our six month BELIEVE Phase 2 trial of Zygel™ in childhood epilepsies. In this study, patients experienced median reductions in their most common and debilitating seizures of 44% or more starting at month two and continuing through month six. In this medically fragile patient population, and consistent with our prior trials, Zygel was very well tolerated. Caregivers also reported important improvements in seizure intensity and duration in their children, and in socio-behavioral and cognitive impairments that are common in this population. Finally, we continued to progress towards full enrollment in our pivotal CONNECT-FX trial in children and adolescents with Fragile X syndrome, and expect to announce topline results in the first half of next year.”

Financial Results

For the quarter ending September 30, 2019, Zynerba reported an $8.3 million credit to research and development expenses for amounts expected to be received through the AOF for the period from January 1, 2018, through September 30, 2019. “Excluding the $8.3 million reduction in research and development expenses for amounts expected to be received through the AOF for the period from January 1, 2018, through September 30, 2019, research and development expenses increased by $1.8 million to $6.7 million for the three months ended September 30, 2019 from $4.9 million for the three months ended September 30, 2018. The increase was primarily related to an increase in clinical trial and manufacturing costs related to our Zygel program. Stock-based compensation included in the R&D costs were $0.6 million.”

General and administrative expenses for the third quarter of 2019 were $3.5 million, including stock-based compensation expense of $0.8 million. The net loss for the third quarter of 2019 was $1.9 million with a basic and diluted net loss per share of $(0.08).


Don't Miss This Week's Exclusive News

Our team will cultivate the cannabis industry’s critical news and deliver it directly to your inbox every Friday. Don't miss it. Subscribe Now.

We respect your privacy. See our privacy policy.


About Us

The Green Market Report focuses on the financial news of the rapidly growing cannabis industry. Our target approach filters out the daily noise and does a deep dive into the financial, business and economic side of the cannabis industry. Our team is cultivating the industry’s critical news into one source and providing open source insights and data analysis


READ MORE


Back to Top