Zynerba Pharmaceuticals (NASDAQ: ZYNE)

Technical Analysis
Positive Trend
Short-term resistance: ~$15.70
Current Support: Exponential Moving Average (EMA)

Yahoo! Finance (2019). Stock price graph for Zynerba Pharmaceuticals 4/25/19 to 5/25/19. Retrieved from https://finance.yahoo.com/quote/ZYNE?p=ZYNE

Zynerba Pharmaceuticals, is a company I see value in. From a technical analysis standpoint, the positive uptrend is an attractive characteristic. Also, with the recent pullback, if ZYNE continues to be supported by the exponential moving average (EMA), it is currently at a “good buy” area. I have not taken a position; however, this company is currently on my watch list. The company does not have any approved products and does not expect to submit a new drug application (NDA) until Q1 or Q2 of 2020.

Their pipeline product ZygelTM (ZYN002 CBD Gel) is being developed for patients suffering from Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD) in pediatric patients, 22q Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE).3 It has been granted Fast Track Designation for treatment of behavioral symptoms associated with Fragile X Syndrome (FXS) by the U.S. Food and Drug Administration (FDA).4

ZygelTM (ZYN002 CBD Gel)
Zygel, a transdermal non-euphoric cannabinoid, is expected to be seeking an initial indication for the treatment of behavioral symptoms associated with Fragile X Syndrome (FXS).

Fragile X syndrome (FXS) is caused by a genetic mutation on the FMR1 gene and a subsequent lack of Fragile X mental retardation protein (FMRP), which accounts for many neuropsychiatric symptoms of Fragile X. FMRP deficiency may result in a range of intellectual disabilities, social deficits, psychiatric problems, and dysmorphic physical features. Premutation of FMR1 is associated with fragile X-associated tremor ataxia syndrome, fragile X-associated primary ovarian insufficiency, psychiatric problems, hypertension, migraines, and autoimmune problems.1,2

Efficacy Results
Recent studies suggest that FXS and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system, as well as effects on GABA and 5-HT1A receptors, and improve certain core social and behavioral symptoms. Zygel is a pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). 1,4

As of May 25, 2019, the most recent data available on Zygel is a Phase 2, 12-week open-label study that evaluated the safety, tolerability and efficacy of Zygel. The study design is summarized within Figure 1.1

Figure 1. Study Design

The primary efficacy endpoint was the Anxiety, Depression, and Mood Scale (ADAMS) Total Score. The secondary endpoints included the ADAMS subscale scores, which includes social avoidance, manic/hyperactive behavior, depressed mood, general anxiety, and compulsive behavior; and the Aberrant Behavior Checklist (FXS Factor Structure; ABC-CFXS) subscale scores, which includes social avoidance, irritability, socially unresponsive/lethargic, hyperactivity, stereotypy, and inappropriate speech. Efficacy results are summarized in Table 1.1

Table 1. Efficacy at Week 121

Scale: ADAMSBaseline
(n=20)
Week 12
(n=18)
Week 12 Δ
(% Improvement Group Mean)
P-Valuea
Total Score33.418.1-14.1 (45.8)<0.0001
Social Avoidanceb10.24.8-5.1 (52.9)0.0002
Manic/Hyperactive Behavior9.46.1-2.7 (35.1)0.0003
Depressed Mood2.82.0-0.9 (28.6)0.1417
General Anxiety10.04.6-4.8 (54.0)<0.0001
Compulsive Behavior2.81.4-1.2 (50)0.0262
Scale: ABC-CFXSBaseline
(n=20)
Week 12
(n=18)
Week 12 Δ
(% Improvement Group Mean)
P-valuea
Social Avoidanceb5.12.3-2.8 (54.9)0.0005
Irritability18.210.6-7.1 (41.8)0.0096
Social Unresponsive/Lethargicc8.74.1-5.1 (52.9)0.0034
Hyperactivity14.59.8-3.9 (32.4)0.0237
Stereotypy7.93.2-4.9 (59.5)0.0006
Inappropriate Speech6.13.5-2.4 (42.6)0.0018

aCompared with baseline
bPrefers isolation from others, prefers solitary activities, avoids new social activities
cLack of attention/interaction, inactive/lack of movement, can resist physical contact

SAFETY:
Through Month 12, patients reported 43 treatment-emergent adverse events (TEAEs). The most common TEAEs were gastroenteritis (14%) and upper respiratory tract infection (12%). One patient developed skin rash and one patient developed dry skin; both resolved and the patients remained in the study. No serious AEs were reported. In Period 1, there were two discontinuations, one patient for worsening eczema (not treatment-related) and 1 patient for administrative reasons; in Period 2, there was one discontinuation for administrative reasons. There have been no clinically meaningful trends in vital signs, ECGs, or clinical safety labs, including liver function tests. No THC has been detected in plasma. Overall, Zygel was well tolerated.1

CONCLUSION:
Zygel induced a statistically significant impact on emotional and behavioral symptoms experienced by children and adolescents with FXS while being well tolerated. The Phase 3, multi-national, randomized, double blind placebo controlled clinical trial is necessary to support and confirm the positive results described within this Phase 2 trial. 1

Enrollment is ongoing in the multi-national, randomized, double blind placebo controlled Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX), a pivotal clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/).4

REFERENCES:

  1. HS Heussler et al. Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X Syndrome (FXS). 57th Annual Meeting of the American College of Neuropsychopharmacology (ACNP). Hollywood, FL. December 9-12, 2018.
  2. Rajaratnam A et al. Fragile X syndrome and fragile X-associated disorders. F1000Res. 2017 Dec 8;6:2112.
  3. ZygelTM (ZYN002 CBD Gel).; (2019) Available at https://zynerba.com/in-development/zygel/
  4. Zynerba Pharmaceuticals Receives Fast Track Designation for Zygel™ for the Treatment of Behavioral Symptoms Associated with Fragile X Syndrome (FXS).; (2019). Available at https://zynerba.com/zynerba-pharmaceuticals-receives-fast-track-designation-for-zygel-for-the-treatment-of-behavioral-symptoms-associated-with-fragile-x-syndrome-fxs/

Disclosure: I have no positions in the stock mentioned. I wrote this article myself, and it expresses my own opinions and not of those of my employer. I am not receiving compensation for it. I have no business relationship with the company whose stock is mentioned in this article.

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