Zynerba:+2,000% (Yes, 2,000%!) Increase In Stock Forecast In 2023 – Here’s Why
From my analysis of Zynerba Pharmaceuticals Inc. (ZYNE) and the extremely optimistic analyst stock price projections stated above, I think Zynerba would…
Each of the 3 analyst following Zynerba Pharmaceuticals, Inc. (ZYNE) forecast the stock jumping by 2,000% (yes, 2,000%!) by the end of 2023.
This article looks at what the company is focused on, its clinical trial progress to date, its latest quarterly financials and the investment risks associated with such an investment.
An original article by Lorimer Wilson, Managing Editor of munKNEE.com – Your KEY To Making Money!
About Zynerba Pharmaceuticals, Inc.
ZYNE reports – in paraphrased excerpts from its site – that:
it is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare (less than 200K patients in the U.S.) and near-rare (around 1M patients in the U.S.) neuropsychiatric disorders which “modulate pathways in the central nervous system (CNS) that, when disrupted, cause neurological diseases such as Fragile X syndrome (FSX), 22q11.2 deletion syndrome (q22) and autism spectrum disorder (ASD)”;
it has put together an experienced and dedicated team (see page 32 here) with proven development and commercialization track records and extensive experience in transdermal delivery, orphan diseases, neurology, and psychiatry led by Armando Anido, Chairman and Chief Executive Officer since October 2014, who has more than 30 years of executive, operational and commercial leadership experience in the biopharmaceutical industry, particularly in leading CNS transdermal patch and gel products through the entire product life cycle.
AboutFragile X Syndrome (FSX)
FXS, as described by Elizabeth Berry-Kravis, et al, in the Journal of Neurodevelopmental Disorders (see here), and paraphrased here:
is a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder;
affects 1 in 3,600 – 4,000 males and 1 in 4,000 – 6,000 females and is the most commonly inherited intellectual disability in males and a significant cause of intellectual disability in females, and the leading genetic cause of ASD;
negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as anxiety, aggression toward others, irritability, temper tantrums, shyness, and a preference for solitary activities;
affects approximately 78,000 people in the U.S. and approximately 121,000 in the EU and U.K.; and
has yet to receive any regulatory approvals for treatment despite decades of preclinical research and interventional clinical trials.
About 22q11.2 Deletion Syndrome (22q)
DiGeorge syndrome is more accurately known by the broader term “22q11.2 deletion syndrome” and
is the second most common chromosomal disorder after Down syndrome. It is caused by a small missing piece of the 22nd chromosome near the middle of the chromosome at a location designated q11.2,
is linked to poorer adaptive behaviors for children such as self-care and communication skills that affect daily life such as:
major anxiety which is the most common neuropsychiatric symptom,
withdrawn behavior,
ADHD,
cognitive impairment,
ASD,
are at an increased risk of developing mental illnesses such as schizophrenia
is estimated to occur in every 3,000 to 6,000 live births, suggesting that there are approximately 83,000 people with 22q in the U.S.
Given its prevalence and severity, however, the disease has not received any regulatory approvals for treatment despite decades of preclinical research and interventional clinical trials.
About Zygel
Zynerba is leading the way in the clinical development of Zygel, also known as ZYN002, a pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel designed to provide controlled delivery into the bloodstream through the skin for the potential treatment of behavioral symptoms associated with FXS, 22q and ASD.
“recent studies suggest that cannabidiol may modulate the endocannabinoid system (ECS) which regulates neuronal development and cognitive function” such as:
sleep, mood, stress, appetite and digestion, learning and memory, reproduction and fertility, chronic pain, inflammation and other immune system responses, skin and nerve function, bone remodeling and growth, motor control, cardiovascular system function, muscle formation and liver function, and is the pathogenesis of FXS.
In addition, the company reports that:
been issued a patent for the “Treatment of 22q11.2 Deletion Syndrome With Cannabidiol,” by the U.S. Patent and Trademark Office. The patent expires in 2040 and is one of seven corresponding U.S. patents and one corresponding EU patent related to methods of treating FXS, 22q and ASD,
has received orphan drug designation for cannabidiol, the active ingredient in Zygel, from the FDA and the European Commission for the treatment of FXS and by the FDA for the treatment of 22q and
has had Zygel designated a Fast Track development program for treatment of behavioral symptoms of FXS.
Clinical Trials
Zynerba conducted a Phase 2 clinical study on the efficacy, safety and impact of Zygel by administering the transdermal gel to children and adolescents with FSX and 22q and found that:
75% of patients showed clinically meaningful improvements at week 14 compared to the baseline,
Zygel was well tolerated,
and the safety profile was consistent with previously released data from other Zygel clinical trials.
In the near term, while the data from the Company’s ASD clinical development program to date had been progressing well, the Company has decided:
to defer the start of the Phase 3 development program in ASD until the first half of 2024 given:
the difficult financing market,
and the higher than expected cancellations of initial screening visits at the investigational centers as a result of unusually high rates of RSV (respiratory syncytial virus), influenza, and the continued impact of COVID-19 among children and adolescents, resulting in slower than anticipated enrollment and
to prioritize its resources on FXS and 22q.
For more detailed information please refer to the Company’s latest corporate presentation here.
to have concluded its confirmatory Pivotal Phase 3 trial results in the first half of 2024 for FXS and
to finalize regulatory pathway with the FDA in 2023 and then initiate Phase 3 soon after receipt of the RECONNECT topline results with a successful completion expected to satisfy requirements for an
NDA submission in the U.S. and a marketing authorization application in the EU.
By 2026, Zynerba intends:
to launch Zygel for FXS into a $1.9B+ U.S. market opportunity,
to establish a fully integrated organization with a U.S. commercial presence,
to prepare for EU approval in FXS and
to advance the 22q Ph3 program towards completion.
Beyond 2026, Zynerba plans:
to launch Zygel in FXS via strategic partners in EU elsewhere,
to launch Zygel into the additional multi-billion dollar market of 22q,
to optimize Zygel growth with additional synergistic indication and
to accelerate further growth through complimentary asset licensing and acquisition.
3Q 2022 Financial Results
Zynerba Pharmaceuticals, Inc., with a market capitalization of only $28M, is still in the product research and development of novel compounds and, as such, has no products and no revenue and requires a great deal of money to fund their R&D expenses. That being said, it reported in its last quarter financial report (seehere) ending September 30, 2022, that it had $55.9 million in cash and cash equivalents which the company deemed sufficient to see the company through mid-2024. For a detailed look at all the financial metrics related to Zynerba in the last quarter and for every year going back to 2018 go here.
Stock Price History and Forecast
Zynerba’s stock price declined 80% in 2022 which was only slightly more than the 70% decline experienced by the 6 major clinical-stage psychedelic stocks I follow in the munKNEE Psychedelic Drug Stocks Index (see details here). Based on 3 analyst ratings (see here), however, the stock is forecast to jump by 2015% to $11.00 by the end of 2023 from its $0.52 close on February 17th, 2023.
Investment Risks
The company reports that their cash position of US$55 million is sufficient to advance the company through its key value-creating clinical milestones without the need to raise additional financing over the next 1.5 years. That being said, however,
were Zynerba to have repeated set-backs in its various research studies current investors and potential new investors alike could lose confidence in the eventual success of the company’s efforts and such negative sentiment could negatively impact the stock price,
were another company successful in developing a treatment for FXS, 22q or ASD before Zynerba did, that would most likely negatively impact the stock price, as well and, even more so, if such a competitor were to take the treatment for one of those diseases to market and, finally,
were senior management to depart, especially the CEO, that might cause the company to lose focus which would slow down its progress in its research and development.
Conclusions
From my analysis of Zynerba Pharmaceuticals, in and of itself, and the extremely optimistic analyst stock price projections stated above, I think Zynerba would make a great buy and hold investment. In addition to that, were the Company to bring any one of its products directly to market itself, or be acquired by a major pharmaceutical company, its stock price should go up dramatically.
Zelensky Departs Washington Mostly Empty-Handed Amid Mood Shift In West
By all accounts, Zelensky came away from his Washington visit with nothing new. Biden did announce a fresh $325 million aid package for Ukraine from already committed funds, but the hoped-for long range missile approval never came (however, more cluster bombs are being sent). And as we detailed Thursday, House Republican leadership once again failed to move forward on a mere procedural vote for the Pentagon funding bill, due in large part to GOP members rejecting Biden's proposed $24 billion more in Ukraine aid.
Thursday's package announced by Biden, as Zelensky visited the White House and Capitol Hill, was run-of-the-mill and entirely to be expected. "Today I approved the next tranche of U.S. security assistance to Ukraine including more artillery, more ammunition, more anti-tank weapons and next week, the first U.S. Abrams tanks will be delivered to Ukraine," Biden said.
As for the earlier in the day (Thurs.) meeting with Congressional leaders, House Speaker Kevin McCarthy explained when asked why the Ukrainian leader's request to address Congress was denied, "Zelensky asked for a joint session, we just didn't have time. He's already given a joint session."
Instead in a closed-door meeting, Zelensky later acknowledged he discussed with lawmakers "the battlefield situation and priority defense needs."
But if there is any level of consolation for Kiev, it's seen in the Pentagon announcement which came late in the day Thursday. Facing potential USgovernment shutdown on Oct.1st, given at this point Congress is not expected to pass the 12 appropriations bills needed to fund government operations before next fiscal year, the Pentagon has said it will exempt its operations supporting Ukraine from a shutdown.
The military typically suspends any activities not deemed vital to national security during government shutdowns, thus the DoD is in effect saying Ukraine aid remains "vital to national security".
"Operation Atlantic Resolve is an excepted activity under a government lapse in appropriations," Pentagon spokesman Chris Sherwood told Politico, in reference to the operational name still used for actions supporting Kiev.
But Politico points out a potential shutdown would still negatively impact US support to Ukraine:
Sherwood noted that while DOD’s activities related to Ukraine will continue, furloughs and other activities halted under the shutdown could still have a negative impact.
"Training would happen, but depending on whether or not there were certain personnel that were not able to report for duty, for example, that could have an impact," said Pentagon spokesperson Brig. Gen. Patrick Ryder on Thursday.
This Pentagon exemption to keep Ukraine-related support active during a government shutdown seems to be the only significant thing Zelensky came away with.
Zelensky visited the US in person, made a speech at the UN, and came home with an amount of ammo so small the Pentagon won't give numbers and a handful of the worst air defense systems currently in use by a major power.
It appears to have been the main object of discussion when Zelensky met with Secretary of Defense Lloyd Austin in Washington during the trip. The Pentagon said this was "to reaffirm the steadfast US support for Ukraine."
Meanwhile, Bloomberg takes note of Zelensky "showing the strain" amid increasing divisions among allies:
The Ukrainian president allowed a dispute with one of his biggest allies to spin out of control at the United Nations General Assembly this week, and that’s just a hint of the tensions building behind the scenes.
Zelenskiy has been leading his country through Russia’s brutal assault for 19 months, all the time fighting on another front to wring the weapons and finance he needs from his US and European supporters. Now he suspects that President Joe Biden’s commitment is wavering and other leaders may be taking their cue from the US, according to a person who met with him recently.
He grew very emotional at times during that discussion, the person said, and was scathing in his criticism of nations that he said weren’t delivering weapons quickly enough.
Washington's lackluster greeting of Zelensky this week (compared to how he was received in December 2022) came simultaneous to Poland declaring it will no longer arm Ukraine, amid a fierce diplomatic spat over blockage of Ukraine grain imports by Warsaw, to protect Polish farmers.
The Economist is also taking note of the significant mood shift among Western allies...
A "long war" indeed... given a G7 leader from a European country has told reporters this week that the West is prepared for a years-long war, something likely to last some six or seven years, according to the quote.
"A senior official from one European G-7 country said the war may last as much as six or seven more years and that allies need to plan financially to continue support for Kyiv for such a long conflict," Bloomberg wrote.
Years ago, a rich person or company would indicate wealth by donating libraries and orphanages or traverse the world in bespoke yachts or aircraft.
You may commission a boat to extract an enemy submarine off the depths of the ocean floor. Perhaps you'd get an ivy league school of public policy and government named in your honor. If you were really industrious, you might even gather a bunch of your high-powered banker friends in a locked room on a distant island off the coast of Georgia to establish what we now call the Federal Reserve.
Nowadays, however, it's quite in vogue to gather large stockpiles of your cash and put it to work not for your people or your country. Billionaires think much bigger than that. If you're really rich, you put your money to work for the good of planet Earth.
Take, for example, Microsoft (MSFT) - Get Free Report and Amazon (AMZN) - Get Free Report, who both recently pledged hundreds of millions of dollars between the two to capture carbon out of the atmosphere and dispose of it where it may no longer be accessed.
Businessman and former New York City Mayor Michael Bloomberg is the latest billionaire to take a stab at the seemingly unsolvable climate crisis – and he's putting a vast sum of his money up to the challenge.
Aerial view of Jon Amos Power plant shows smoke stacks and cooling, Coal, Poca, West Virginia. (Photo by: Visions of America/Joseph Sohm/Universal Images Group via Getty Images)
Bloomberg's charitable organization, Bloomberg Philanthropies, announced on Wednesday that it would front $500 million to shut down all existing coal plants in the U.S. and prevent future natural gas plants from being constructed.
The pledge may sound anti-industrialist, but Bloomberg's "Beyond Carbon" initiative claims it will "turbocharge," environmental progress.
The initiative includes the following bullet points:
Finish the job on coal. With 372 of 530 coal plants announced to retire or closed to date – more than 70 percent of the country’s coal fleet – this next phase will shut down every last U.S. coal plant.
Slash gas plant capacity in half, and block all new gas plants.
Increase U.S. clean energy four-fold. Accelerate the clean energy transition to reach the goal of 80 percent of total electricity generation.
"Bloomberg Philanthropies has helped retire more than 70 percent of all U.S. coal plants, which accounts for more than 80 percent of all emissions reductions in the United States since 2010," Bloomberg, who's a special envoy on climate ambition and solutions to the U.N. proudly announced on Wednesday.
Beyond Carbon will also fund the following organizations as a part of its mission toward progress:
Earthjustice
Hip Hop Caucus
Sierra Club
RMI
League of Conservation Voters
Advanced Energy United
Coalfield Development
No stranger to controversy, Bloomberg has supported a number of divisive policies during his tenure in the public eye, including calls for banning polystyrene foam that is commercially known as Styrofoam, smoking, trans fats, and restricting sugary drinks in some public businesses.
Now, it seems the billionaire is more concerned about climate change as a threat to public health than fat or soda.
"This work has helped achieve more than 80 percent of all U.S. emissions reductions since 2010 and plant closures from the campaigns are estimated to have saved 49,900 lives, prevented nearly 77,500 heart attacks, and saved billions in healthcare costs," Bloomberg Philanthropies writes.
"But the U.S. is now at a pivotal moment — without rapid progress on clean energy, we will fail to meet our U.S. climate targets, and public health risks will skyrocket. Beyond Carbon’s next phase will ensure the U.S. delivers on our global climate commitments by retiring the last remaining coal plants, stopping the expansion of natural gas, and quadrupling clean energy capacity while continuing to prioritize environmental justice and workforce transition."
A receding hairline, a total loss of hair from the crown, and ultimately, the classical horseshoe-shaped pattern of baldness: Previous research into male…
A receding hairline, a total loss of hair from the crown, and ultimately, the classical horseshoe-shaped pattern of baldness: Previous research into male pattern hair loss, also termed androgenetic alopecia, has implicated multiple common genetic variants. Human geneticists from the University Hospital of Bonn (UKB) and by the Transdisciplinary Research Unit “Life & Health” of the University of Bonn have now performed a systematic investigation of the extent to which rare genetic variants may also contribute to this disorder. For this purpose, they analyzed the genetic sequences of 72,469 male participants from the UK Biobank project. The analyses identified five significantly associated genes, and further corroborated genes implicated in previous research. The results have now been published in the prestigious scientific journal Nature Communications.
Credit: University Hospital Bonn / Katharina Wislsperger
A receding hairline, a total loss of hair from the crown, and ultimately, the classical horseshoe-shaped pattern of baldness: Previous research into male pattern hair loss, also termed androgenetic alopecia, has implicated multiple common genetic variants. Human geneticists from the University Hospital of Bonn (UKB) and by the Transdisciplinary Research Unit “Life & Health” of the University of Bonn have now performed a systematic investigation of the extent to which rare genetic variants may also contribute to this disorder. For this purpose, they analyzed the genetic sequences of 72,469 male participants from the UK Biobank project. The analyses identified five significantly associated genes, and further corroborated genes implicated in previous research. The results have now been published in the prestigious scientific journal Nature Communications.
Male-pattern hair loss is the most common form of hair loss in men, and is largely attributable to hereditary factors. Current treatment options and risk prediction are suboptimal, thus necessitating research into the genetic underpinnings of the condition. To date, studies worldwide have focused primarily on common genetic variants, and have implicated more than 350 genetic loci, in particular the androgen receptor gene, which is located on the maternally inherited X chromosome. In contrast, the contribution to this common condition of rare genetic variants has traditionally been assumed to be low. However, systematic analyses of rare variants have been lacking. “Such analyses are more challenging as they require large cohorts, and the genetic sequences must be captured base by base, e.g., through genome or exome sequencing of affected individuals,” explained first author Sabrina Henne, who is a doctoral student at the Institute of Human Genetics at the UKB and the University of Bonn. The statistical challenge lies in the fact that these rare genetic variants may be carried by very few, or even single, individuals. “That is why we apply gene-based analyses that first collapse variants on the basis of the genes in which they are located,” explained corresponding author PD Dr. Stefanie Heilmann-Heimbach, who is a research group leader at the Institute of Human Genetics at the UKB at the University of Bonn. Among other methods, the Bonn researchers used a type of sequence kernel association test (SKAT), which is a popular method for detecting associations with rare variants, as well as GenRisk, which is a method developed at the Institute of Genomic Statistics and Bioinformatics (IGSB) at the UKB and the University of Bonn.
Possible relevance of rare variants in male-pattern hair loss
The research involved the analysis of genetic sequences from 72,469 male UK Biobank participants. Within this extensive data set, Bonn geneticists, together with researchers from the IGSB and the Center for Human Genetics at the University Hospital Marburg, examined rare gene variants that occur in less than one percent of the population. Using modern bioinformatic and statistical methods, they found associations between male-pattern hair loss and rare genetic variants in the following five genes: EDA2R, WNT10A, HEPH, CEPT1, and EIF3F.
Prior to the analyses, EDA2R and WNT10A were already considered candidate genes, as based on previous analyses of common variants. “Our study provides further evidence that these two genes play a role, and that this occurs through both common and rare variants,” explained Dr. Stefanie Heilmann-Heimbach. Similarly, HEPH is located in a genetic region that has already been implicated by common variants, namely the EDA2R/Androgen receptor, which is a region that has consistently shown the strongest association with male-pattern hair loss in past association studies. “However, HEPH itself has never been considered as a candidate gene. Our study suggests that it may also play a role,” explained Sabrina Henne. “The genes CEPT1 and EIF3F are located in genetic regions that have not yet been associated with male-pattern hair loss. They are thus entirely new candidate genes, and we hypothesize that rare variants within these genes contribute to the genetic predisposition. HEPH, CEPT1, and EIF3F represent highly plausible new candidate genes, given their previously described role in hair development and growth.” Furthermore, the results of the study suggest that genes that are known to cause rare inherited diseases affecting both skin and hair (such as the ectodermal dysplasias) may also play a role in the development of male-pattern hair loss. The researchers hope that the puzzle pieces they have discovered will improve understanding of the causes of hair loss, and thus facilitate reliable risk prediction and improved treatment strategies.
The research was supported by funding from the Medical Faculty of the University of Bonn. Prof. Dr. Markus Nöthen, Director of the Institute of Human Genetics at UKB and co-author of the study, is a member of the Transdisciplinary Research Area (TRA) “Life and Health” at the University of Bonn. The publication costs in open access format were funded by the DEAL project of the University of Bonn.
Journal
Nature Communications
DOI
10.1038/s41467-023-41186-w
Article Title
Analysis of 72,469 UK Biobank exomes links rare variants to male-pattern hair loss
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