ZYNE Zynerba Pharmaceuticals Inc.

4.5
+0.04  (+1%)
Previous Close 4.46
Open 4.49
52 Week Low 2.55
52 Week High 9
Market Cap $132,329,092
Shares 29,439,175
Float 26,107,811
Enterprise Value $83,353,134
Volume 1,568,803
Av. Daily Volume 5,977,689
Stock charts supplied by TradingView

Drug Pipeline

Drug Stage Notes
Zygel ZYN002
Fragile X syndrome
Phase 2/3
Phase 2/3
Phase 2/3 trial did not meet primary endpoint - June 30, 2020. Further trial to be initiated in 2021.
Zygel ZYN002 - BRIGHT
Autism Spectrum Disorder (ASD)
Phase 2
Phase 2
Phase 2 data released May 27, 2020. Trial achieved statistically significant and improvements from in all subscales of the Aberrant Behavior Checklist. Intends to meet FDA 1H 2021.
ZYN002 BELIEVE 1
Developmental and Epileptic Encephalopathies (DEE)
Phase 2
Phase 2
Phase 2 top-line data released September 18, 2019. 10/46 patients reported a serious adverse event (SAE). Will pursue individual syndromes rather than considering DEE as a single disorder.
Zygel ZYN002
22q11.2 Deletion Syndrome
Phase 2
Phase 2
Phase 2 trial delayed due to COVID-19.
ZYN002
Adult Epilepsy Patients with Refractory Focal Seizures
Phase 2
Phase 2
Phase 2b trial initiation postponed until after the completion of childhood neuropsychiatric studies.
ZYN002 - STOP
Osteoarthritis
Phase 2
Phase 2
Phase 2 data released August 14, 2017 - primary endpoint not met. Noted January 2018 that development will be discontinued.
ZYN001
Fibromyalgia
Phase 2
Phase 2
Development to be discontinued - noted January 2018.
ZYN001
Peripheral Neuropathic Pain (PNP)
Phase 2
Phase 2
Development to be discontinued - noted January 2018.

Latest News

  1. DEVON, Pa., Jan. 04, 2021 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Armando Anido, Chief Executive Officer of Zynerba, will participate in two upcoming virtual investor conferences. Mr. Anido will participate in a fireside chat during the H.C. Wainwright BioConnect Conference which will be available on demand January 11-14, 2021. He will also present a company overview during the ICR Conference 2021 on Thursday, January 14, 2021 at 10:00 a.m. ET.

    Investors interested in arranging a virtual meeting with the Company's management during the ICR Conference 2021 should…

    DEVON, Pa., Jan. 04, 2021 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Armando Anido, Chief Executive Officer of Zynerba, will participate in two upcoming virtual investor conferences. Mr. Anido will participate in a fireside chat during the H.C. Wainwright BioConnect Conference which will be available on demand January 11-14, 2021. He will also present a company overview during the ICR Conference 2021 on Thursday, January 14, 2021 at 10:00 a.m. ET.

    Investors interested in arranging a virtual meeting with the Company's management during the ICR Conference 2021 should contact the conference coordinator or . Webcasts of both conferences will be accessible under the Events & Webcasts tab of the Investor section of Zynerba's website at www.zynerba.com.

    About Zynerba Pharmaceuticals, Inc.

    Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.  

    Cautionary Note on Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. These and other risks are described in the Company's periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

    Investor Contact

    Peter Vozzo

    Westwicke, an ICR Company

    Office: 443-213-0505

    Cell: 443-377-4767



    Primary Logo

    View Full Article Hide Full Article
  2. - Single Trial to be Conducted in Patients with Fragile X Syndrome to Confirm Positive Results Seen in the Population of Responders in the CONNECT-FX Trial -

    - Conference Call and Webcast Today, December 17, 2020 at 8:30 am ET -

    DEVON, Pa., Dec. 17, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided an update on its meeting with the U.S. Food and Drug Administration (FDA) regarding its Fragile X syndrome (FXS) program. The Company plans to conduct a double-blind, placebo-controlled pivotal trial in patients with FXS who have a highly methylated FMR1 gene to confirm the…

    - Single Trial to be Conducted in Patients with Fragile X Syndrome to Confirm Positive Results Seen in the Population of Responders in the CONNECT-FX Trial -

    - Conference Call and Webcast Today, December 17, 2020 at 8:30 am ET -

    DEVON, Pa., Dec. 17, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided an update on its meeting with the U.S. Food and Drug Administration (FDA) regarding its Fragile X syndrome (FXS) program. The Company plans to conduct a double-blind, placebo-controlled pivotal trial in patients with FXS who have a highly methylated FMR1 gene to confirm the positive results observed in this population of responders in the CONNECT-FX trial. In the first half of 2021, Zynerba will review the trial design and protocol for the new trial through a Type C meeting with the FDA and expects to initiate the pivotal trial before the end of 2021. Zynerba believes that positive results from this confirmatory trial would be sufficient to support the submission of a New Drug Application (NDA) for Zygel™ in FXS.

    "We believe that Zygel has the potential to meaningfully relieve the behavioral symptoms of the most impacted individuals with Fragile X syndrome. We are committed to bringing this important therapy to patients and their families within the Fragile X community," said Armando Anido, Chairman and Chief Executive Officer of Zynerba. "We are thankful for our ongoing constructive dialogue with the FDA on our path forward to NDA submission. Completing the development of Zygel in FXS and preparing for a successful launch will be the primary focus of the Company."

    "The results reported in the analysis of children with a highly methylated FMR1 gene are a source of considerable hope for the patients and their families who are impacted by Fragile X syndrome," said Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF). "Zynerba has been an important partner to the families of children with Fragile X. We thank them for their continued dedication and commitment to bringing an FDA-approved treatment to our families and look forward to helping with the recruitment of patients for this study."

    The Company's development plan for Zygel in other indications includes the following:

    • Developmental and epileptic encephalopathies (DEE): Evaluation of potential target indications is ongoing, and Zynerba now expects to finalize target syndrome selection in 2021 in one or more DEE syndromes.
    • 22q11.2 deletion syndrome (22q): Zynerba expects to resume recruitment for the 14-week open label Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q once COVID-19-related restrictions in Australia are eased. After recruitment has resumed, the Company will be able to provide a timeframe for completion of this trial.
    • Autism spectrum disorder (ASD): In the first half of 2021, Zynerba intends to discuss data supporting the potential efficacy of Zygel in ASD, including the results of the Phase 2 BRIGHT trial in children and adolescents with moderate-to-severe ASD with the FDA to determine the regulatory path forward.

    Financial Impact on Cash

    The Company expects that its cash runway will extend into the beginning of 2023 as a result of these changes to its development plans.

    Conference call information

    Zynerba management will host a live conference call and webcast today at 8:30 am Eastern Time to discuss the recent FDA meeting and the regulatory pathway for Zygel in FXS. The call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345 (international) and referencing conference ID 4088027. To access the live webcast or the replay, visit the investor page of the Company's website at http://ir.zynerba.com/. The webcast will be recorded and available on the Company's website for 30 days.

    About Zynerba Pharmaceuticals, Inc.

    Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.  

    Cautionary Note on Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the Company's cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company's expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; the Company's ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company's clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company's product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the U.S. Food and Drug Administration and foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company's planned clinical trial evaluating Zygel™ in patients with a highly methylated FMR1 gene may not confirm the results of the CONNECT-FX trial or may not be determined to be sufficient to support an NDA submission; the Company's ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company's reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company's product candidates the Company's ability to commercialize its product candidates; the size and growth potential of the markets for the Company's product candidates, and the Company's ability to service those markets; the Company's ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company's product candidates; the Company's expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. This list is not exhaustive and these and other risks are described in the Company's periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

    Investor Contact

    William Roberts, Vice President, Investor Relations and Corporate Communications

    Zynerba Pharmaceuticals

    484.581.7489



    Primary Logo

    View Full Article Hide Full Article
  3. President & CEO Purnanand Sarma Announced as the Recipient of TiE Boston's 2020 Lifetime Achievement Award

    Immunome, Inc. (NASDAQ:IMNM), a biopharmaceutical company utilizing a proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics, with a focus on oncology and infectious diseases including COVID-19, today announced the recent appointments of Richard Baron to Immunome's Board of Directors and as Audit Committee Chair, and Sandra Stoneman, Esq. as Chief Legal Officer and Corporate Secretary. The company also today announced that its President and Chief Executive Officer, Purnanand Sarma, Ph.D. is the recipient of TiE Boston's 2020 Lifetime Achievement Award. The award recognizes individuals who…

    President & CEO Purnanand Sarma Announced as the Recipient of TiE Boston's 2020 Lifetime Achievement Award

    Immunome, Inc. (NASDAQ:IMNM), a biopharmaceutical company utilizing a proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics, with a focus on oncology and infectious diseases including COVID-19, today announced the recent appointments of Richard Baron to Immunome's Board of Directors and as Audit Committee Chair, and Sandra Stoneman, Esq. as Chief Legal Officer and Corporate Secretary. The company also today announced that its President and Chief Executive Officer, Purnanand Sarma, Ph.D. is the recipient of TiE Boston's 2020 Lifetime Achievement Award. The award recognizes individuals who have made invaluable contributions in the fields of science, business and entrepreneurship.

    "I am thrilled to welcome Richard and Sandra to the Immunome team as we accelerate our efforts in discovering novel antibodies and advancing our lead programs into the clinic," said President and CEO, Purnanand Sarma, Ph.D. "The combination of their expertise and experience will be invaluable in supporting Immunome's growth." He further stated, "I am also honored to receive TiE Boston's 2020 Lifetime Achievement Award and very much appreciate the opportunity to continue to work with TiE in driving innovation and fostering next generation entrepreneurs."

    Richard Baron has over 30 years of experience as an advisor to companies in the pharmaceutical, biotechnology and medical device industries and as a Chief Financial Officer. He was previously the Chief Financial Officer of Zynerba Pharmaceuticals (NASDAQ:ZYNE), Globus Medical (NYSE:GMED), Avid Radiopharmaceuticals, Animas Corporation (NASDAQ:PUMP) and others. Baron was a practicing certified public accountant, beginning his career in audit and tax services at Coopers & Lybrand and Arthur Young. He received his B.S. in economics at The Wharton School of the University of Pennsylvania. Baron's background and experience advising life sciences companies and in finance and accounting added significantly to the Board's skills in these areas.

    Sandra Stoneman, Esq. is a seasoned attorney, with more than 20 years of experience advising high-growth companies in the life sciences and technology sectors. Prior to joining Immunome, Stoneman was an attorney at Duane Morris, leading the Firm's life sciences practice group. She has received numerous accolades for her transactional work in the life sciences field, including being continuously listed in U.S. News/Best Lawyers, Chambers USA and LMG Life Sciences. Stoneman received her J.D. from Temple University School of Law and her B.A. from the State University of New York at Binghamton.

    About Immunome, Inc.

    Immunome is a biopharmaceutical company utilizing our proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics designed to change the way diseases are currently being treated with an initial focus on oncology and infectious diseases, including COVID-19. Immunome's proprietary discovery platform identifies novel therapeutic antibodies and their targets by leveraging highly educated components of the immune system, memory B cells, from patients whose bodies have learned to fight off their disease.

    Forward-Looking Statements

    This press release includes certain disclosures that contain "forward-looking statements" intended to qualify for the "safe harbor" from liability established by the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Immunome's beliefs and expectations regarding the advancement of its oncology and COVID-19 therapeutic antibody programs, execution of its clinical and strategic plans, anticipated upcoming milestones for IMM-BCP-01 and IMM‐ONC‐01, including expectations regarding therapeutic potential and benefits thereof, and IND filings. Forward-looking statements may be identified by the words "anticipate," believe," "estimate," "expect," "intend," "plan," "project," "may," "will," "could," "should," "seek" and similar expressions. Forward-looking statements are based on Immunome's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, those risks and uncertainties associated with: the impact of the COVID-19 pandemic on Immunome's business, operations, strategy, goals and anticipated milestones, Immunome's ability to execute on its strategy including with respect to the timing of IND filings, Immunome's ability to fund operations, as well as those risks and uncertainties set forth more fully under the caption "Risk Factors" in the final prospectus dated October 1, 2020 and filed pursuant to Rule 424(b) under the Securities Act of 1933, as amended, with the United States Securities and Exchange Commission (SEC) and elsewhere in Immunome's filings and reports with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Immunome undertakes no duty to publicly update or revise any forward looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable law.

    View Full Article Hide Full Article
  4. The Law Offices of Frank R. Cruz is investigating potential claims against the board of directors of Zynerba Pharmaceuticals ("Zynerba" or the "Company") (NASDAQ:ZYNE) concerning whether the board breached its fiduciary duties to shareholders.

    If you are a shareholder, click here to participate.

    On September 18, 2019, Zynerba announced results from its BELIEVE 1 Trial evaluating topical gel Zygel in children and adolescents with Developmental and Epileptic Encephalopathy. While Zynerba asserted that Zygel was well-tolerated, the Company disclosed a 96% rate of treatment emergent adverse events, 60% rate of treatment related adverse events. Zynerba also revealed that, among ten patients who reported serious adverse events, two "(lower respiratory…

    The Law Offices of Frank R. Cruz is investigating potential claims against the board of directors of Zynerba Pharmaceuticals ("Zynerba" or the "Company") (NASDAQ:ZYNE) concerning whether the board breached its fiduciary duties to shareholders.

    If you are a shareholder, click here to participate.

    On September 18, 2019, Zynerba announced results from its BELIEVE 1 Trial evaluating topical gel Zygel in children and adolescents with Developmental and Epileptic Encephalopathy. While Zynerba asserted that Zygel was well-tolerated, the Company disclosed a 96% rate of treatment emergent adverse events, 60% rate of treatment related adverse events. Zynerba also revealed that, among ten patients who reported serious adverse events, two "(lower respiratory tract infection and status epilepticus) were determined to be possibly related to treatment."

    On this news, Zynerba's stock price fell $2.46 per share, or 21.77%, to close at $8.84 per share on September 18, 2019.

    Our investigation concerns whether the Company's board of directors breached its fiduciary duties to shareholders and/or grossly mismanaged the Company in connection with the above alleged misconduct.

    Follow us for updates on Twitter: twitter.com/FRC_LAW.

    If you still hold Zynerba shares purchased before August, 2019 and wish to discuss this matter with us, or have any questions concerning your rights and interests with regards to this matter, please contact Frank R. Cruz, of The Law Offices of Frank R. Cruz, 1999 Avenue of the Stars, Suite 1100, Los Angeles, California 90067 at 310-914-5007, by email to , or visit our website at www.frankcruzlaw.com.  If you inquire by email please include your mailing address, telephone number and number of shares purchased.

    This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and ethical rules.

    View Full Article Hide Full Article
  5. - New Efficacy Data Describe Strong Evidence of Seizure Reduction over 12 Months of Treatment, Including a 73% Median Reduction from Baseline in Monthly Seizure Frequency at Month 12 -

    - Zygel™ Improves the Profound Sleep Disturbance Often Experienced by Developmental and Epileptic Encephalopathy (DEE) Patients Enrolled in the BELIEVE Trial -

    DEVON, Pa., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting two posters describing the efficacy, safety and quality of life results of the Phase 2 BELIEVE (Open Label Study to Assess the Safety and Efficacy of Zygel™…

    - New Efficacy Data Describe Strong Evidence of Seizure Reduction over 12 Months of Treatment, Including a 73% Median Reduction from Baseline in Monthly Seizure Frequency at Month 12 -

    - Zygel™ Improves the Profound Sleep Disturbance Often Experienced by Developmental and Epileptic Encephalopathy (DEE) Patients Enrolled in the BELIEVE Trial -

    DEVON, Pa., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting two posters describing the efficacy, safety and quality of life results of the 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) clinical trial at the 2020 virtual annual meeting of the American Epilepsy Society (AES). Both posters are available on the Zynerba corporate website at http://zynerba.com/publications/.

    "These newly reported data are exciting as they describe the compelling anti-seizure activity of Zygel in children and adolescents suffering from developmental and epileptic encephalopathies, or DEEs, through twelve months of treatment," said Zynerba's Chief Medical Officer, Joseph M. Palumbo, MD, FAPA, MACPsych. "This is particularly exciting, as this trial was conducted in a refractory seizure population in which patients failed to show adequate response despite taking as many as four anti-seizure drugs. Additionally, we observed an equally compelling seizure reduction in children with a coexisting diagnosis of autism spectrum disorder."

    The first poster entitled, "ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents With Developmental and Epileptic Encephalopathies: An Open-Label Clinical Trial [BELIEVE (ZYN2-CL-025)]" describes the evidence of tolerability and efficacy of Zygel observed in children and adolescents with developmental and epileptic encephalopathies (DEE) who participated in the Phase 2 BELIEVE trial treated for up to 12 months.

    As shown in Figure 1, new results presented today include 12-month treatment data showing improvements in the median percentage reduction from baseline in monthly frequency of consciousness-impairing seizures (focal impaired awareness seizures, or FIAS, and tonic-clonic seizures, or TCS, including generalized tonic-clonic seizures [GTCS] and focal to bilateral tonic-clonic seizures [FBTCS]), ranged from 44% at month three to 73% at month 12.

    Figure 1: Median Percentage Reduction From Baseline in 28-Day Frequency of FIAS and TCS by Time Point, Patients With FIAS and/or TCS at Baseline

    https://www.globenewswire.com/NewsRoom/AttachmentNg/774e0c2b-497d-4d92-87a2-c1341361cc52

    When analyzed by seizure type, median reductions from baseline at month six for FIAS, GTCS, and FBTCS were 45%, 60%, and 59%, respectively. At month 12, the median reductions for FIAS, GTCS and FBTCS were 100%, 83% and 59% respectively.

    Additionally, as shown in Figure 2, a substantial percentage of patients achieved ≥35% and ≥50% reduction in FIAS and TCS by month three (58% and 46%, respectively), and these reductions in seizures continued through month 12 (89% and 83%, respectively).

    Figure 2: Percentage of Patients With 35% and 50% Reduction in FIAS and TCS by Time Point, Patients With FIAS and/or TCS at Baseline

    https://www.globenewswire.com/NewsRoom/AttachmentNg/51acd725-7207-4e96-9f95-76e8649a27e8

    Epileptic encephalopathies represent a particularly severe form of epilepsy, associated with cognitive and behavioral deficits, including impaired social-communication and restricted, repetitive behaviors that are the hallmarks of autism spectrum disorder (ASD)1. Zynerba conducted an exploratory analysis to evaluate the efficacy of Zygel in reducing seizures in DEE patients with coexisting ASD enrolled in BELIEVE.

    As seen in Figure 3, over the 12-month treatment period, the median percentage reduction from baseline in monthly frequency of consciousness-impairing seizures (FIAS or TCS) in patients with DEE and ASD improved over time, and these reductions in seizures ranged from 45% at month three to 74% at month 12.

    Figure 3. Median Percentage Reduction From Baseline in 28-Day Frequency of FIAS and TCS by Time Point, Patients With Coexisting ASD at Baseline

    https://www.globenewswire.com/NewsRoom/AttachmentNg/d2655600-bf08-4e50-b2ac-185fd1c77f16

    As seen in Figure 4, a substantial percentage of patients achieved ≥35% and ≥50% reduction in FIAS and TCS by month three (60% and 40%, respectively), and these reductions continued through month 12 (75%).

    Figure 4: Percentage of Patients With 35% and 50% Reduction in FIAS and TCS by Time Point, Patients With Coexisting ASD at Baseline

    https://www.globenewswire.com/NewsRoom/AttachmentNg/8d70258a-c987-48e9-a4bb-f645bc473a54

    Zygel was well tolerated in BELIEVE. Most treatment-emergent adverse events (any event, whether unrelated or related to study drug) were mild or moderate. There were no clinically significant changes in vital signs, ECGs, or laboratory findings except for one patient with a transient, benign, isolated elevation of alkaline phosphatase at week 26 that was not considered related to study medication.

    The authors of the poster concluded that:

    • These data suggest meaningful reductions in FIAS and TCS with Zygel treatment which is maintained through to 12 months of treatment with Zygel;
    • In the subgroup of patients with ASD, Zygel demonstrated meaningful reductions in FIAS and TCS seizures, with most children reaching either the 35% or 50% responder threshold by month three and month six respectively;
    • Zygel was well tolerated over 18 months of treatment in a medically fragile patient population of children and adolescents with DEEs; and
    • The positive benefit/risk profile of Zygel in this trial supports further study in patients with DEEs and FIAS and TCS.

    The second poster entitled, "Quality of Life and Sleep Assessments in Children with Developmental and Epileptic Encephalopathies Treated With ZYN002 (CBD) Transdermal Gel: BELIEVE (ZYN2-CL-025)" describes the impact of Zygel on profound sleep disturbances experienced by DEE patients enrolled in the BELIEVE trial.

    Dr. Palumbo continued, "These new data highlight the impact Zygel has on the profound sleep disturbance often experienced by DEE patients in this trial. Importantly, disrupted sleep in children with epilepsy has previously been reported to be associated with negative outcomes in overall family functioning. The Sleep Disturbance Scale for Children (SDSC) utilized in the BELIEVE trial shows that half of the enrolled children exhibited clinically significant sleep problems at study baseline, which improved while receiving Zygel."

    The Sleep Disturbance Scale for Children (SDSC) is a 26-item scale completed by caregivers that assesses six different sleep categories. The authors of the poster concluded that treatment with Zygel may be associated with improvements in disorders of initiating and maintaining sleep, disorders of arousal/nightmares, sleep wake transitions, and overall sleep, as well as clinically meaningful improvements observed in vitality, cognition/concentration, socially avoidant behaviors, seizure severity, behavior, and mood.

    As shown in Figure 5 and Table 1 below, statistically significant improvements from baseline in sleep scores were observed in the Total Sleep Score (p=0.012), Disorders of Initiating or Maintaining Sleep (DIMS; p=0.006), Disorder of Arousal/Nightmares (DA; p=0.031), and Sleep Wake Transition Disorder (SWTD; p=0.030).

    Figure 5: The Sleep Disturbance Scale for Children (SDSC) – Percentage of Patients Above Threshold for Clinically Significant Sleep Problems at Baseline and Week 26

    https://www.globenewswire.com/NewsRoom/AttachmentNg/f1e79e50-2018-4a4a-a9c1-b27719635f5c



    Table 1. Change From Baseline in the SDSC

    SDSC Factorst-Score

    Mean (SD)
    Change

    (negative number is

    improvement)
    P value
    Total Score    
    Baseline (n=46)71.6 (12.68)  
    Week 26 (n=37)63.9 (13.40)−5.10.012*
    Disorders of Initiating and Maintaining Sleep (DIMS)
    Baseline (n=46)69.6 (14.67)  
    Week 26 (n=38)63.2 (15.76)−5.10.006*
    Sleep Breathing Disorders (SBD)
    Baseline (n=46)60.6 (15.46)  
    Week 26 (n=40)58.9 (15.08)0.400.797
    Disorders of Arousal/Nightmares (DA)
    Baseline (n=46)51.5 (9.91)  
    Week 26 (n=39)49.0 (5.07)−1.70.031*
    Sleep Wake Transition Disorder (SWTD)
    Baseline (n=46)65.0 (13.09)  
    Week 26 (n=39)60.2 (13.78)−4.60.030*
    Disorders of Excessive Somnolence (DOES)
    Baseline (n=46)68.5 (16.76)  
    Week 26 (n=40)63.1 (13.82)−3.60.100
    Sleep Hyperhidrosis (SHY)
    Baseline (n=46)52.7 (12.28)  
    Week 26 (n=40)50.6 (9.95)−2.80.154

    *P<0.05 for change from baseline to week 26; statistically significant

    About Zynerba Pharmaceuticals, Inc.

    Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.  

    Cautionary Note on Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the Company's cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company's ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company's clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company's product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the U.S. Food and Drug Administration and foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company's ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company's reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company's product candidates the Company's ability to commercialize its product candidates; the size and growth potential of the markets for the Company's product candidates, and the Company's ability to service those markets; the Company's ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company's product candidates; the Company's expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. This list is not exhaustive and these and other risks are described in the Company's periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

    Zynerba Contact

    Will Roberts, VP Investor Relations and Corporate Communications

    484.581.7489

    Media contact

    Molly Devlin

    Evoke KYNE

    215.928.2199




    1 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481888/

     



    Primary Logo

    View Full Article Hide Full Article
View All Zynerba Pharmaceuticals Inc. News