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Zynerba's CBD skin gel Zygel offers a goldmine of therapies for Fragile X syndrome, and other conditions

Snapshot

All eyes are on Zynerba's meeting with the FDA in the fourth quarter of 2020 to discuss pivotal CONNECT-FX data and the regulatory path for potential approval

Zynerba Pharmaceuticals - Zynerba's CBD skin gel Zygel offers a goldmine of therapies for Fragile X syndrome, and other conditions

Quick facts: Zynerba Pharmaceuticals

Price: 3.65 USD

NASDAQ:ZYNE
Market: NASDAQ
Market Cap: $106.78 m
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  • Developing novel transdermal CBD treatments to treat patients suffering rare epilepsies and neurological disorders
  • Lead development candidate Zygel offers a potential goldmine of therapies for multiple conditions
  • Zygel is being developed for patients suffering from Fragile X syndrome, a genetic condition that causes learning disabilities 

What Zynerba does:

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) is doing trailblazing work pushing clinical trials for cannabis-derived treatments. The specialty pharma company is developing novel transdermal CBD treatments for patients suffering rare epilepsies and neurological disorders.

The Devon, Pennsylvania-based company’s main product candidate is Zygel, a patent-protected CBD skin gel, which was earlier known as ZYNOO2. 

Recent studies have suggested that neuropsychiatric conditions like Fragile X Syndrome — the most common form of inherited learning disability and attention deficit disorder — are associated with a disruption in the endocannabinoid system, and CBD may improve certain core social and behavioral symptoms by modulating the biological system.

Zygel also offers a potential goldmine of therapies for other conditions. In addition to Fragile X Syndrome, the gel is in clinical development in patients with refractory epilepsy, Autism Spectrum Disorder and 22q11.2 Deletion Syndrome, a disorder caused by a small missing piece of the 22nd chromosome. This tiny missing portion can affect every system in the body. 

Zygel is also being tested for a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE).

How is it doing: 

In September, the Food and Drug Administration (FDA) granted orphan drug designation to Zynerba's CBD use in treating 22q11.2 deletion syndrome (22q). This is a shot in the arm for Zynerba which is conducting a 14-week Phase 2 INSPIRE trial to weigh the safety and potential of its CBD skin gel Zygel in 20 children with 22 q. It expects to report topline results from the study in the third quarter of 2020. 

The FDA also notified Zynerba in Septmber that it will hold a teleconference with the company in the fourth quarter of 2020, to discuss its pivotal CONNECT-FX data and the regulatory path forward in patients with Fragile X syndrome and a fully methylated FMR1 gene (FMet). The company said it expects to disclose the outcome of the meeting in the fourth quarter.

An ad hoc analysis of its recent CONNECT-FX trial showed that Zygel achieved statistical significance in improving behavioral metrics among the most severely impacted patients — 80% of the total participants — after 12 weeks. The median improvement measured by the Social Avoidance subscale of the Fragile X Aberrant Behavior Checklist was 40% for patients on Zygel and 21% for patients on placebo.

Fragile X is a genetic condition caused by a mutation in the FMR1 gene. The mutation causes the X chromosome to appear constricted, or fragile, under a microscope, hence its name. Patients with Fragile X often have learning disabilities and other cognitive impairments.

BELIEVE trial data released last year has built on the strong safety and tolerability profile of Zygel, and provided evidence of its anti-seizure activity in children and adolescents suffering from developmental and epileptic encephalopathies, or DEEs. Zynerba said in September that it has concluded its iterative meetings with the FDA utilizing their ‘Written Response Only’ format to discuss the clinical pathway for Zygel in DEE. Zynerba said the FDA supports a development program which would “treat focal-impaired awareness and convulsive seizures.”

However, Zynerba explained that due to the “heterogeneity of patients who fall under the DEE umbrella," the FDA suggested that Zynerba pursue "individual syndromes" rather than considering DEE patients "as a single disorder or condition." The company is in the process of finalizing its evaluation of which epileptic syndromes it may pursue with Zygel. The company said it would complete its target assessments and communicate the company’s path forward around the end of the year.

Zygel also showed promise as a treatment for patients with Autism Spectrum Disorder. Zynerba reported positive results from its 14-week Phase 2 BRIGHT trial, where Zygel was given to 37 patients aged between 3 and 17 with moderate-to-severe autism spectrum disorder (ASD) as an add-on therapy. The data was robust across multiple subscales, including the Aberrant Behavior Checklist-Community (ABC-C) irritability subscale and Parent Rated Anxiety scale, showing both “statistically significant” and “clinically meaningful (>25%) improvements” at 14 weeks of treatment from baseline.

The company said it will also discuss the results from the trial with the FDA to clarify the clinical pathway for developing Zygel for the treatment of behavioral symptoms of ASD, which is characterized by challenges with social skills, speech and repetitive movements.

In another boost for the company, the US patent office granted Zynerba a patent for treating ASD, with CBD. This is in addition to the patent it received in December 2019 for Zygel to treat Fragile X syndrome. The patent, which expires in 2038, covers the company's method of transdermally administering 250 mg to 500 mg of CBD daily in the form of a gel or cream. 

On the financial front, Zynerba ended the second quarter with ample cash reserves of $77 million as of June 30, 2020, up from $70.1 million at the end of 2019. During the quarter, the company had offerings for more than 5.6 million shares, netting $27.2 million in proceeds. 

Management believes the cash runway is sufficient to fund capital requirements beyond the expected New Drug Application submission and potential approval of Zygel for Fragile X syndrome and into the fourth quarter of 2021.

Inflection Points

  • Will discuss the pivotal CONNECT-FX data and the regulatory path for potential approval of Zygel with the FDA in the fourth quarter
  • Expects to submit its NDA for Zygel in Fragile X syndrome to the FDA in the second half of 2020, with potential approval by mid-year 2021
  • If successful, Zygel has the potential to become the first product for the treatment of behavioral symptoms of Fragile X syndrome
  • A positive data readout for Zynerba in any one of its many clinical trials can create serious momentum

What the broker says:

Roth Capital recently repeated a 'Buy' rating on Zynerba and boosted its price target to $11.00 from $10.50 citing higher odds of Zygel being approved to treat autism spectrum disorder. The current share price is around $5.76.

“The company intends to meet with the FDA in 2H20, and we anticipate two positive phase 3 trials to be required prior to FDA approval,” Roth analyst Scott Henry said in a note to investors.

"Increasing our probability of approval for the ASD indication to 35% from 25% increases our price target to $11/share from $10.50/share," he added.

What the boss says:

“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,” Zynerba Pharmaceuticals CEO Armando Anido has said.

“The meeting with the FDA will be an important milestone for patients and their families who live with the debilitating behavioral impact of Fragile X. Our ongoing evaluation of the pivotal CONNECT-FX data continues to clarify the impact that Zygel achieved in the most severely impacted children and adolescents with FXS, as well as the excellent tolerability profile." 

Contact the author Uttara Choudhury at [email protected]

Follow her on Twitter: @UttaraProactive

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