Zynerba Archives - Green Market Report

StaffOctober 6, 2022
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3min6591

Zynerba Pharmaceuticals (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today announced that the U.S. Patent and Trademark Office has issued Patent No. 11,458,110, titled “Treatment of Fragile X Syndrome With Cannabidiol,” which includes claims directed to methods of treating Fragile X syndrome with cannabidiol.

This new patent, which expires in 2038, is part of an expanding international intellectual property portfolio covering Zynerba’s transdermal cannabidiol product candidate, Zygel. There are currently four corresponding U.S. patents and one corresponding EU patent related to methods of treating Fragile X syndrome. Last year Green Market Report reported that Zynerba
published the results from its open-label Phase 2 BELIEVE (Open-Label Study to Assess the Safety and Efficacy of Zygel (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) study in the Journal of the American Medical Association (JAMA) Network Open.

The study demonstrated a 58% median monthly reduction in seizures at month 5, and a 43.5% reduction over the 6.5 month study period (the primary efficacy endpoint). In addition, the parents/caregivers noted improvements in social or interpersonal engagement and irritability (77%); alertness, energy, and sleep (53%); and cognition or concentration (47%).

48 patients with a mean age of 10.5 years were enrolled in BELIEVE and included in the safety analysis. 60% had at least one treatment-related adverse event (AE) over the 6.5 month trial period and 96% of these AE’s were mild or moderate. During the treatment period, 10 patients (21%) reported serious adverse events (SAEs). Two SAEs were considered to be possible treatment-related: nonconvulsive status epilepticus and lower respiratory tract infection, in separate patients. All SAEs were resolved, and none resulted in an alteration of the study medication. The study warned that there was no control group and so it is impossible to know how much was due to the placebo effect and the desire for cannabis products to work.

 


Adam JacksonAugust 10, 2022
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7min810

Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) posted positive results as the company continues to further its research and development goals — with signs of a FDA approval looming in the distance. The cannabis biotech company reported financial results for the second quarter ending June 30, 2021,

Research and development expenses were $5.4 million for the second quarter of 2022, the company said, including stock-based compensation of $0.5 million. General and administrative expenses were $3.7 million in the second quarter this year, including stock-based compensation expense of $0.6 million. As an emerging biotech company, Zynerba does not have revenue at this time as it develops drugs.

The company said that it has continued to focus resources toward developing treatment for two orphan neuropsychiatric disorders, Fragile X syndrome and 22q Deletion Syndrome.

“During the second quarter, we were pleased to announce positive top-line results from our Phase 2 trial of Zygel in patients with 22q,” said CEO Armando Anido. “In addition to further progressing 22q, we are focused on completing the Phase 3 RECONNECT trial for children and adolescents with Fragile X syndrome, with top-line results expected in the second half of 2023.”

With a cash runway extending past expected availability of top-line results from our RECONNECT trial, we remain well-positioned on achieving our goal of bringing the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome to market.”

This echoes Zynerba’s belief that the results from RECONNECT, if positive, will be sufficient to support the submission of a New Drug Application (NDA) for Zygel in patients with Fragile X syndrome.

The company also said it plans to meet with the U.S. Food and Drug Administration (FDA) to discuss the data and the regulatory path forward for its open label Phase 2 INSPIRE trial of Zygel in children and adolescents with 22q Deletion Syndrome — also called DiGeorge syndrome, a chromosomal disorder with no known cure that results in poor development of several body systems.

The company said it plans to move forward in 22q as an orphan indication and has previously received orphan drug designation from the FDA for cannabidiol, the active ingredient in Zygel, for the treatment of 22q.

While data from the company’s ASD clinical development program to date are compelling, given the difficult financing market, the company has deferred the start of the Phase 3 development program in ASD as it has prioritized its resources on FXS and 22q in the near term.

Net loss for the second quarter of 2022 was $9.9 million, with basic and diluted loss per share of $0.24 cents. Cash and cash equivalents were $62.5 million, versus $67.8 million as of December 31, 2021.

In May, the company entered into a Controlled Equity Offering Sales Agreement — with Cantor Fitzgerald & Co., Canaccord Genuity, LLC, H.C. Wainwright & Co. LLC and Ladenburg Thalmann & Co. Inc. as sales agents — in which the company may sell, from time to time, up to $75 million of its common stock.

Zynerba then sold and issued 488,892 shares of its common stock in the second quarter under the agreement in the open market — resulting in gross proceeds of $0.9 million and net proceeds of $0.8 million, after deducting commissions and offering expenses.

After that, from July 1, 2022 through August 8, 2022, the company sold and issued 1,469,714 shares of its common stock under the agreement in the open market resulting in gross proceeds of $1.8 million, and net proceeds of $1.6 after deducting commissions and offering expenses.

Zynerba also struck an equity purchase deal last month for up to $20 million with Chicago-based firm Lincoln Park Capital Fund (LPC).

Lincoln Park Capital is expected to provide financial flexibility and is aligned with Zynerba’s long-term strategy for value creation, the company said.

Zynerba said it plans to use any net proceeds from the sale of its common stock to LPC for working capital and general corporate purposes, including research and development expenses and capital expenditures.

Management believes that the Company’s cash and cash equivalents are sufficient to fund operations and capital requirements through the end of 2023 or into early 2024, after the expected availability of top-line results from its confirmatory pivotal Phase 3 RECONNECT trial of Zygel in patients with FXS.


Adam JacksonJuly 21, 2022
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4min1370

Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) struck an equity purchase deal on Thursday for up to $20 million with Chicago-based firm Lincoln Park Capital Fund (LPC).

 Lincoln Park Capital is expected to provide financial flexibility and is aligned with Zynerba’s long-term strategy for value creation, the company said

“We are excited to enter into this transaction with Lincoln Park Capital and believe that this agreement provides us with another opportunity to access capital in an efficient manner,” said Zynerba CFO and VP Jim Fickenscher. “The financial flexibility provided by this agreement will further support our clinical development efforts with Zygel in Fragile X syndrome and 22q11.2 deletion syndrome.”

Zynerba said it plans to use any net proceeds from the sale of its common stock to LPC for working capital and general corporate purposes, including research and development expenses and capital expenditures.

 Under the terms of the agreement, Zynerba will have the right in its sole discretion, but not obligation, to sell to LPC up to $20 million worth of shares of its common stock over the 36-month term of the agreement. Additionally, Zynerba controls the timing and amount of any future sales of its shares of common stock, and LPC is obligated to make purchases in accordance with the terms of the purchase agreement, subject to various limitations including those under the Nasdaq listing rules.

Any common stock that is sold by Zynerba will occur at a purchase price that is based on the market prices prevailing at the time of each sale to LPC. There is no upper limit to the price per share that LPC may pay for future stock issuances under the purchase agreement, and LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Zynerba’s common stock. No warrants are being issued in this transaction and the purchase agreement does not contain any rights of first refusal, participation rights, penalties, or liquidated damages provisions in favor of any party. Zynerba may terminate the purchase agreement at any time, at its sole discretion, without any cost or penalty, the release said. 

Outlook

Zynerba said it believes its $69.7 million of cash and cash equivalents as of March 31, 2022, are sufficient to fund planned operations and capital requirements through the end of 2023 or into early 2024, after the expected availability of top-line results from its confirmatory Pivotal Phase 3 RECONNECT trial of Zygel in patients with Fragile X syndrome.


Debra BorchardtSeptember 7, 2021
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4min1070

Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) published the results from its open-label Phase 2 BELIEVE (Open-Label Study to Assess the Safety and Efficacy of Zygel (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) study in the Journal of the American Medical Association (JAMA) Network Open. The gel is seen as an alternative to patients where oral drug delivery is a challenge due to the seizures.

The study demonstrated a 58% median monthly reduction in seizures at month 5, and 43.5% reduction over the 6.5 month study period (the primary efficacy endpoint). In addition, the parents/caregivers noted improvements in social or interpersonal engagement and irritability (77%); alertness, energy, and sleep (53%); and cognition or concentration (47%).

“DEEs are the most severe group of epilepsies and are usually drug-resistant. Antiseizure medications are usually administered orally, which can be extremely challenging in children with behavioral and cognitive problems. Non-oral therapies are needed to provide an alternative route to deliver medications to control seizures and improve developmental outcomes,” said Ingrid E. Scheffer, MBBS, PhD, FRS, Laureate Professor, and chair, Pediatric Neurology Research, University of Melbourne, and the lead investigator in the BELIEVE study. “The data from the BELIEVE study are promising and suggest that Zygel may be a safe and well-tolerated option to improve seizure control, challenging behaviors and other symptoms associated with DEEs.”

Forty-eight (48) patients with a mean age of 10.5 years were enrolled in BELIEVE and included in the safety analysis. 60% had at least one treatment-related adverse event (AE) over the 6.5 month trial period and 96% of these AE’s were mild or moderate. During the treatment period, 10 patients (21%) reported serious adverse events (SAEs). Two SAEs were considered to be possible treatment-related: nonconvulsive status epilepticus and lower respiratory tract infection, in separate patients. All SAEs resolved, and none resulted in alteration of study medication. The study warned that there was no control group and so it is impossible to know how much was due to the placebo effect and the desire for cannabis products to work.

The authors indicated that the incidence of AEs in this study, particularly related to infections, was likely due to the high baseline rate of complex morbidities and seizure severity of these patients at study onset, and to the relatively long, 6.5-month duration of the trial over which events were collected. The study was supported by Zynerba.

 


Debra BorchardtAugust 9, 2021
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4min750

Cannabis biotech company Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE) reported financial results for the second quarter ended June 30, 2021, and provided an overview of recent operational highlights and a pipeline update. Zynerba’s research and development expenses were $5.5 million for the second quarter of 2021, including stock-based compensation of $1.0 million. General and administrative expenses were $4.4 million in the second quarter of 2021, including stock-based compensation expenses of $0.9 million. The net loss for the second quarter of 2021 was $10.0 million, with a basic and diluted loss per share of $(0.25).

This missed analyst estimates from Yahoo Finance, who had expected a loss of $0.24. Seven analysts are covering the company with three buy ratings, three holds, and one strong buy. the average price target is $7.63 and the stock was lately selling at $4.45.

As of June 30, 2021, cash and cash equivalents were $85.8 million, compared to $59.2 million as of December 31, 2020. Management believes that the company’s cash and cash equivalents as of June 30, 2021, are sufficient to fund operations and capital requirements well into the first half of 2024.

“We continue to make progress across our portfolio, particularly in FXS in which our confirmatory pivotal Phase 3 trial, RECONNECT, is expected to start in the third quarter of 2021,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “Following a positive meeting with the FDA on our development program in autism spectrum disorder (ASD), we are evaluating and prioritizing our development options for ASD, 22q and developmental and epileptic encephalopathies and we expect to provide guidance on the path forward in each of these indications by the end of 2021.”

Zynerba said in a statement that 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 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.

In the first half of 2021, Zynerba discussed data supporting the potential efficacy of Zygel for treating autism (ASD), including the results of Phase 2 BRIGHT trial, with the U.S. Food and Drug Administration (FDA) to determine the regulatory path forward. The guidance from the FDA included an agreement on utilizing the irritability subscale of the Aberrant Behavior Checklist – Community (ABC-C) as the primary endpoint to support an indication for the treatment of irritability in ASD. This is the same primary endpoint utilized in the previously completed BRIGHT open-label Phase 2 trial.


StaffMay 12, 2021
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3min690

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 BorchardtMarch 10, 2021
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15min710

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.”


StaffNovember 9, 2020
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9min710

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 stanoprime composition 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.

 


StaffNovember 9, 2020
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5min570

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 BorchardtJune 30, 2020
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5min810

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.

 

 

 


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