Zynerba Pharma (ZYNE) : positive results of cannabidiol (CBD) gel in pediatric and adolescent patients with Fragile X syndrome

• Sector(s): Healthcare
• Industry: Drug Manufacturers—Specialty & Generic
• Full Time Employees: 28
• Founded in 2007 
• Headquartered in Devon, Pennsylvania
• http://zynerba.com

Zynerba Pharma announces 'positive' top line results from an open label exploratory Phase 2 FAB-C clinical trial evaluating ZYN002 cannabidiol (CBD) gel in pediatric and adolescent patients with Fragile X syndrome

ZYN002 successfully met the primary endpoint and showed clinically 'meaningful improvements'

• ZYN002 also achieved clinically meaningful improvements in all measures of the Aberrant Behavior Checklist for Fragile X (ABC-FXS), which address the key symptoms of FXS including social avoidance, temper tantrums, repetitive movements, and hyperactivity.
• "The data from the FAB-C trial are very exciting and demonstrate that ZYN002 may have a profound effect on improving many of the most disabling symptoms of Fragile X, such as anxiety and difficult behaviors."
• With these data, Zynerba anticipates that it will meet with the FDA in the first half of 2018 with the goal of moving quickly into a pivotal Phase 2/3 program in pediatric and adolescent patients with FXS in 2018.

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=Zynerba (ZYNE) : cannabis-based epilepsy gel fails study

Aug 7 (Reuters) - Zynerba Pharmaceuticals Inc's stock lost more than half its value on Monday after the U.S. drug developer said its synthetic cannabis-based gel for epilepsy failed a mid-stage study.

Zynerba's gel contains a synthetically processed formulation of cannabidiol (CBD), a non-psychoactive component of the cannabis plant.

Although many U.S. states have sanctioned the medical and/or recreational use of cannabis, drugs derived from the plant could take longer than others to hit the market.

Under U.S. federal law, marijuana is considered a dangerous substance with no medicinal value, making additional approvals for marijuana-derived treatments necessary prior to launch.

Two doses of Zynerba's ZYN002 gel were tested against a placebo in the study.

Neither dose induced a statistically significant improvement in seizure frequency versus the placebo in patients who were already on up to three anti-epileptic medications.

The 188-patient study comprised adults suffering from epilepsy with partial seizures, which occurs when epileptic activity takes part in a localized part of the brain.

Even though the availability of the drug in blood plasma increased with the higher dose, it did not translate into a clinically meaningful benefit.

Based on this data, Cantor Fitzgerald analysts said they were uncertain whether there was a suitable path forward for development of ZYN002 in adult focal seizures.

Data from studies evaluating the gel's use in osteoarthritis patients and in children with Fragile X Syndrome is expected in the coming months, the company said.

Zynerba is still evaluating data to determine next steps, but said it was not giving up on epilepsy as a target, on a conference call with analysts.

Britain's GW Pharmaceuticals, considered a pioneer in cannabis-drug development, is expected to file a U.S. marketing application for its CBD-based epilepsy drug, Epidiolex, this year.

A New England Journal of Medicine editorial earlier this year noted that Epidiolex data represented "the beginning of solid evidence for the use of cannibinoids in epilepsy," after an era of anecdote and emotion-based debates.

The intrinsically low bioavailability of CBD in Zynerba's gel seemed like a potentially significant hurdle for a systemically acting topical medicine, Leerink's Paul Matteis said in a client note titled "ZYNE Topical CBD Failure Removes an Overhang for GW".

Bioavailability denotes the degree by which a drug's active ingredients are absorbed by the blood.

Zynerba Pharma (ZYNE) reported earnings on Tue 1 Aug 2017 (b/o)

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Zynerba Pharma misses by $0.05; Top-line Phase 2 results for STAR 1 trial in epilepsy and STOP trial in osteoarthritis remain on track for reporting in August 2017:

• Reports Q2 (Jun) loss of $0.64 per share, $0.05 worse than the Capital IQ Consensus of ($0.59). 
• "We expect to announce top-line results from the STAR 1 trial soon, followed by top-line data from the STOP trial later this month; and we remain on track to report top-line results from the FAB-C Fragile X study in September," said Armando Anido, Chairman and Chief Executive Officer. "We also met a significant milestone during the quarter in initiating the Phase 1 program for ZYN001, a pro-drug of THC delivered via patch, and expect to initiate our Phase 2 program by the end of this year. With two clinical stage assets, Zynerba is well-positioned to address a number of serious unmet medical needs."
• The Company believes that the current cash and cash equivalent position of $70.2 million is sufficient to develop five Phase 3-ready programs and, assuming support from the FDA to move forward, initiate at least one Phase 3 program and fund operations and capital requirements into 2019.

=Zynerba Pharma (ZYNE) reported earnings on Mon 27 March 2017 (b/o)

Zynerba Pharma misses by $0.05 :

• Reports Q4 (Dec) loss of $0.71 per share, $0.05 worse than the Capital IQ Consensus of ($0.66).
• As of December 31, 2016, cash and cash equivalents totaled $31.0 million, compared to $41.5 million as of December 31, 2015.
• Cash outlook: In the first quarter of 2017, the Company completed a follow-on public offering, selling 3,220,000 shares of our common stock at an offering price of $18.00 per share, resulting in gross proceeds of $58.0 million. Net proceeds received after deducting underwriting and commissions and offering expenses were $54.3 million. Based on Zynerba's cash position of $31.0 million in cash and cash equivalents at year-end 2016, and including proceeds from the Company's follow-on public offering in the first quarter of 2017, the Company estimates that this balance is sufficient to develop five Phase 3 ready programs and, assuming feedback from the FDA supports a decision to move forward, initiate at least one Phase 3 program and fund operations and capital requirements into 2019.
• Anticipated 2017 Milestones
  • ZYN002, a patent-protected, synthetic CBD formulated as a permeation-enhanced gel for transdermal delivery
    • ZYN002 is currently being evaluated in three Phase 2 clinical trials in epilepsy patients with focal seizures, in osteoarthritis and in pediatric patients with FXS which has been designated as an Orphan drug by the US FDA. The Company expects to report top-line data for all three trials in 2017.
    • Top-line results from the Phase 2 STAR1 clinical trial in adult epilepsy patients with focal seizures and from the Phase 2 STOP clinical trials in patients with knee pain due to osteoarthritis are anticipated in July/August 2017;
    • Top-line results from the FAB-C exploratory Phase 2 clinical trial in pediatric patients with Fragile X syndrome are expected in the third quarter of 2017;
  • ZYN001, a patent-protected, pro-drug of THC that enables transdermal delivery via a patch
    • In the first half of 2017, Zynerba expects to initiate Phase 1 studies to evaluate the safety and pharmacokinetic (PK) profile and tolerability of ZYN001 in healthy volunteers
    • Zynerba expects to begin two Phase 2 clinical trials for ZYN001 in patients with fibromyalgia and peripheral neuropathic pain in the second half of the year.