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Zynerba in exciting run-up to clinical readouts on CBD skin gel Zygel to treat Fragile X syndrome

Snapshot

With topline data from the CONNECT-FX trial on the way, investors are eyeing opportunities defined by marijuana-adjacent companies

Zynerba Pharmaceuticals -

Quick facts: Zynerba Pharmaceuticals

Price: 3.41 USD

NASDAQ:ZYNE
Market: NASDAQ
Market Cap: $85.08 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
  • On track to report topline results from tests of its CBD gel treatment for children with Fragile X syndrome in 2Q 2020

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 May, 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.

Top executives from Zynerba intend to meet with the Food and Drug Administration (FDA) in the second half of 2020 to discuss 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 positive, Zynerba ended its first quarter with ample cash reserves of $60.6 million as it advances the flagship Zygel CBD gel. For the first quarter ended March 31, 2020, the company reported research expenses of $6.9 million as it heads into key trials.

Management believes the cash runway is sufficient to fund operations and capital requirements beyond the expected New Drug Application (NDA) submission and potential approval of Zygel for Fragile X syndrome and into the second half of 2021.

Crucially, Zynerba has completed enrolment 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 Fragile X syndrome, with 212 patients randomized. The company expects to report topline results late in the second quarter of 2020 and weigh the acceptability of the data as a basis for the NDA.  

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. 

As of May 8, 2020, 96% of the 188 patients who completed the 14-week blinded portion of the CONNECT-FX trial have enrolled in the open-label extension trial.

Zynbera is also conducting the 14-week Phase 2 INSPIRE trial to evaluate the safety, tolerability and efficacy of Zygel in about 20 children and adolescents (ages six through 17) with genetically confirmed Deletion Syndrome (22q). Zynerba expects to report topline results from this study in the third quarter of 2020.

The company said that based on positive topline results from BELIEVE 1, an open-label multidose Phase 2 clinical trial it also expects to meet with the FDA to discuss a pathway for Zygel in Developmental and Epileptic Encephalopathies (DEE) in the first half of 2020.

The BELIEVE 1 trial enrolled 48 patients between the ages of three and 16 who received weight-based initial doses of 250 milligrams (mg), or 500 mg daily of Zygel. In a positive sign, Zygel achieved a 44% median seizure reduction in focal impaired awareness and convulsive seizures in DEE patients by month two, and reductions were sustained through month six of treatment.

In response to the coronavirus pandemic, Zynerba has implemented initiatives to ensure the safety of patients, clinical investigators and their staff. Zynerba believes this should allow it to conclude all its ongoing clinical trials and report topline results within its stated timelines.

In a boost for the company, the US patent office recently 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.  

Clearly, the company has set the stage for the next 12 months to be potentially transformational as it reports on its trials.

Inflection Points

  • Expects to report topline results late in the second quarter of 2020 from its pivotal Phase 2/3 clinical trial CONNECT-FX
  • 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
  • What GW Pharmaceuticals achieved with its cannabis-derived Epidiolex treatment for seizures underlines the latent potential of Zynerba
  • If GW Pharma’s Epidiolex is aimed at 35,000 to 40,000 patients suffering rare epilepsies, Zygel is looking at double the market for just Fragile X syndrome and almost a million patients are diagnosed with autism 
  • 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.

The analyst also observed that the next major catalyst for the company is pivotal data for Zygel for the treatment of Fragile X Syndrome “expected in late June 2020.”

“We are encouraged that Zygel demonstrated symptomatic relief for ASD (which has some of the same symptoms as FXS) although the Fragile X trial will be placebo-controlled,” Henry 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.

“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.”

Contact the author Uttara Choudhury at [email protected]

Follow her on Twitter: @UttaraProactive

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