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Zynerba Pharmaceuticals

Zynerba’s 4Q shows momentum as it heads into key clinical trial

CEO Armando Anido says the company’s strong cash position is expected to take it through its NDA submission and potential approval for Zygel in Fragile X syndrome

A doctor's stethoscope and a marijuana plant
Zynerba Pharmaceutical’s principal line of business are cannabinoid treatments delivered via the skin to treat neurological and psychiatric disorders

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) said Monday that it saw “clinical momentum” in the fourth quarter as it posted financial results that showed it has $59.8 million to fund clinical trials for its cannabis-derived treatments.

Devon, Pennsylvania-based Zynerba is on track to report top-line results from tests of its Zygel, a CBD gel treatment for children and adolescents with Fragile X syndrome, the most common form of inherited learning disability and attention deficit disorder.

Zygel was previously referred to as ZYN002, a synthetic cannabidiol, which is a non-psychoactive cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. The company has now selected Zygel as the new brand name for ZYN002.

READ: Zynerba Pharmaceuticals’ quest for CBD gel to treat Fragile X syndrome ventures where big pharma fears to tread

“The fourth quarter of 2018 was a period of strong clinical and corporate momentum, as we achieved our remaining 2018 milestones and positioned ourselves well for a watershed year in 2019,” Zynerba Pharmaceuticals CEO Armando Anido said in a statement.

The company said enrollment is speeding up in the CONNECT-FX clinical trial, which is a pivotal, multinational, placebo-controlled study evaluating the potential and safety of Zygel in Fragile X syndrome.

Clinical investigative sites are enrolling patients in the United States, Australia, and New Zealand. Patients who have completed the double-blind phase are now enrolling into the 12-month open-label phase.

Top-line results are expected in the second half of 2019 from its CONNECT-FX clinical trial, which will evaluate the potency and safety of ZYN002, a CBD gel, in children aged three to 17 with Fragile X syndrome.

Fragile X is also the most commonly known single gene cause of autism spectrum disorder.

“We continued enrollment into CONNECT-FX, our pivotal trial of Zygel in Fragile X Syndrome, and completed enrollment in BELIEVE 1, our Phase 2 trial in developmental and Epileptic encephalopathies,” said Anido.

In addition to Fragile X syndrome and ultra-rare epilepsies known as developmental and epileptic encephalopathies, Zygel is also in Phase 2 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 of chromosome 22 can affect every system in the body.

Financial outlook

The company’s cash and cash equivalent position as of December 31, 2018 was $59.8 million. Research and development expenses for the fourth quarter were $4.9 million. The net loss for the pre-revenue company was $7.8 million, or $0.44 per share.

In addition to the $59.8 million cash position the company had at the end of the year, the company received $18.1 million in net proceeds from the shares sold in the first quarter of 2019.

“We have a cash position that we expect to take us through our expected NDA submission and potential approval for Zygel in Fragile X Syndrome,” said Anido.

Management said it believes the cash position including the $59.8 million and the $18.1 million in net proceeds from the shares sold in the first quarter of 2019, is “sufficient to fund operations” beyond the expected NDA submission and potential approval in Fragile X syndrome and “into the first quarter of 2021.”

Contact Uttara Choudhury at [email protected]

Follow her on Twitter@UttaraProactive 

 

Quick facts: Zynerba Pharmaceuticals

Price: $11.60

Market: NASDAQ
Market Cap: $269.56 m
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