Ayala Pharmaceuticals Announces Completion of Enrollment in Part A of RINGSIDE, a Pivotal Phase 2/3 Study of AL102 in Desmoid Tumors

Research, News, and Market Data on Ayala Pharmaceuticals

 

Interim Results expected mid-2022

REHOVOT, Israel and WILMINGTON, Del., Feb. 23, 2022 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers, today announced that it has completed patient enrollment in Part A of the Phase 2/3 RINGSIDE clinical trial evaluating AL102 in desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor (GSI). The Company expects to report interim results from Part A by mid-2022.

“Completion of enrollment in Part A of the RINGSIDE trial is an important milestone in our AL102 clinical program for desmoid tumors,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Part A will help us determine the optimal dose for the randomized portion of the study, we will be looking for safety and signs of anti-tumor activity based on MRI. We are extremely grateful to the patients who are participating and we look forward to sharing top line data around mid-year.”

A total of 36 patients have been enrolled in Part A of the RINGSIDE study which is evaluating the safety, tolerability of AL102, as well as tumor volume by MRI at 16 weeks. Three dosing regimens of AL102 are being tested to determine the optimal dose regimen to advance forward.

In Part A, the effect of food on AL102 absorption was tested in a sub-study and PK results indicate that no food restrictions are required.

Part B of the study will be double-blind, placebo-controlled, and will start immediately after dose selection from Part A, enrolling up to 156 patients age 12 and up with progressive disease, randomized between AL102 or placebo. The study’s primary endpoint will be progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR,) and patient-reported Quality of Life (QOL) measures. Patients who participated in Part A are eligible to enroll into an open-label extension study at the selected dose, and long-term efficacy and safety will be monitored.

For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282.

About AL102

AL102 is a potent, selective, oral gamma secretase inhibitor (GSI). AL102 is currently being developed for the treatment of desmoid tumors, as well as in combination with Novartis’ B-cell maturation antigen (BCMA)-targeting agents for the treatment of multiple myeloma (MM).

About Desmoid Tumors

Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

About Ayala Pharmaceuticals
Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations and in a Phase 2 clinical trial for patients with TNBC (TENACITY) bearing Notch activating mutations and other gene rearrangements. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE) and is being evaluated in a Phase 1 clinical trial in combination with Novartis’ BMCA targeting agent, WVT078, in Patients with relapsed/refractory Multiple Myeloma. For more information, visit www.ayalapharma.com.

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
jallaire@lifesciadvisors.com

Ayala Pharmaceuticals:
+1-857-444-0553
info@ayalapharma.com 

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL101 and AL102, the promise and potential impact of our preclinical or clinical trial data, the timing of and plans to initiate additional clinical trials of AL101 and AL102, the timing and results of any clinical trials or readouts, the sufficiency of cash to fund operations, and the anticipated impact of COVID-19, on our business. These forward-looking statements are based on management’s current expectations. The words ”may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have incurred significant losses since inception and anticipate that we will continue to incur losses for the foreseeable future. We are not currently profitable, and we may never achieve or sustain profitability; we will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of AL101 and AL102; we have a limited operating history and no history of commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability; we are heavily dependent on the success of AL101 and AL102, our most advanced product candidates, which are still under clinical development, and if either AL101 or AL102 does not receive regulatory approval or is not successfully commercialized, our business may be harmed; due to our limited resources and access to capital, we must prioritize development of certain programs and product candidates; these decisions may prove to be wrong and may adversely affect our business; the outbreak of COVID-19, may adversely affect our business, including our clinical trials; our ability to use our net operating loss carry forwards to offset future taxable income may be subject to certain limitations; our product candidates are designed for patients with genetically defined cancers, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to marketable products; we were not involved in the early development of our lead product candidates; therefore, we are dependent on third parties having accurately generated, collected and interpreted data from certain preclinical studies and clinical trials for our product candidates; enrollment and retention of patients in clinical trials is an expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control; if we do not achieve our projected development and commercialization goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and our business will be harmed; our product candidates may cause serious adverse events or undesirable side effects, which may delay or prevent marketing approval, or, if approved, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales; the market opportunities for AL101 and AL102, if approved, may be smaller than we anticipate; we may not be successful in developing, or collaborating with others to develop, diagnostic tests to identify patients with Notch-activating mutations; we have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for any of our product candidates; even if we obtain FDA approval for our product candidates in the United States, we may never obtain approval for or commercialize them in any other jurisdiction, which would limit our ability to realize their full market potential; we have been granted Orphan Drug Designation for AL101 for the treatment of ACC and may seek Orphan Drug Designation for other indications or product candidates, and we may be unable to maintain the benefits associated with Orphan Drug Designation, including the potential for market exclusivity, and may not receive Orphan Drug Designation for other indications or for our other product candidates; although we have received Fast Track designation for AL101, and may seek Fast Track designation for our other product candidates, such designations may not actually lead to a faster development timeline, regulatory review or approval process; we face significant competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively; we are dependent on a small number of suppliers for some of the materials used to manufacture our product candidates, and on one company for the manufacture of the active pharmaceutical ingredient for each of our product candidates; our existing collaboration with Novartis is, and any future collaborations will be, important to our business. If we are unable to maintain our existing collaboration or enter into new collaborations, or if these collaborations are not successful, our business could be adversely affected; enacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates, if approved, and may affect the prices we may set; if we are unable to obtain, maintain, protect and enforce patent and other intellectual property protection for our technology and products or if the scope of the patent or other intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and we may not be able to compete effectively in our markets; we may engage in acquisitions or in-licensing transactions that could disrupt our business, cause dilution to our stockholders or reduce our financial resources; and risks related to our operations in Israel could materially adversely impact our business, financial condition and results of operations.

These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission (SEC) on March 24, 2021 and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Avivagen Announces Approval in Vietnam

Research, News, and Market Data on Avivagen

 

Ottawa, ON /Business Wire/ February 22, 2022 /– Avivagen Inc.  (TSXV:VIV, OTCQB:VIVXF) (“Avivagen”), a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that safely enhances feed intake and supports immune function, thereby supporting general health and performance, is pleased to announce that Avivagen’s oxidized carotenoid-based feed additive product has received approval for use in Vietnam.

“We are very excited to secure approval for our product in Vietnam, a critical feed market in Southeast Asia with strong growth potential over the coming years,” says Kym Anthony, Chief Executive Officer, Avivagen.  “We continue to make strong in-roads with feed producers across Asia and see considerable opportunity to further grow our presence in the swine and poultry feed markets in Vietnam and across the region.”

Vietnam represents the largest feed market in Southeast Asia according to a report by the US Grains Council, which indicates total annual feed consumption of over 32 MMT in 2020. Additionally, The Grains Council report states that Vietnam is a strong growth market which is expected to surpass 35 MMT of feed within the next few years.[i] Production within the country is dominated by swine, with feed for swine accounting for approximately 38% of total feed, followed by poultry accounting for 21.6% of total feed. (2020)[ii]. In addition to swine and poultry Vietnam has a surprisingly large and rapidly growing dairy industry which represents an important third target market segment for use of Avivagen’s product.

“Demand for effective alternatives to antibiotics is growing across all of Southeast Asia,” says Lesley Nernberg, Technical Sales and Marketing Consultant, Asia, Avivagen Inc. “Approval in Vietnam will allow Avivagen to capitalize on a marketplace ready for these types of innovations.”

The use of Avivagen’s product in the Vietnam feed industry is supported by the positive results of two research trials conducted specifically under Vietnamese commercial production conditions. The trials were run in collaboration with the Institute for Animal Sciences for South Vietnam and were conducted with pigs. “The results of these two trials make clear the benefits of Avivagen’s product on improving the health and productivity of piglets. We’re confident that the use of our product in this segment, along with poultry and dairy, will have a significantly positive impact for producers throughout the country,” says Jamie Nickerson, President, Avivagen Inc. “Comparable trials are underway around the world, and we expect many to result in additional regulatory approvals and customer wins over the coming months.”

About Avivagen

Avivagen is a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that, by safely supporting immune function, promote general health and performance.  It is a public corporation traded on the TSX Venture Exchange under the symbol VIV and is headquartered in Ottawa, Canada, based in partnership facilities of the National Research Council of Canada. For more information, visit www.avivagen.com. The contents of the website are expressly not incorporated by reference in this press release.

About OxC-beta™ Technology and OxC-beta™ Livestock

Avivagen’s OxC-beta™ technology is derived from Avivagen discoveries about ?-carotene and other carotenoids, compounds that give certain fruits and vegetables their bright colours. Through support of immune function the technology provides a non-antibiotic means of promoting health and growth. OxC-beta™ Livestock is a proprietary product shown to be an effective and economic alternative to the antibiotics commonly added to livestock feeds. The product is currently available for sale in the United States, Philippines, Mexico, Taiwan, New Zealand, Thailand, Brazil, Australia, and Malaysia.

Avivagen’s OxC-beta™ Livestock product is safe, effective and could fulfill the global mandate to remove all in-feed antibiotics as growth promoters. Numerous international livestock trials with poultry and swine using OxC-beta™ Livestock have proven that the product performs as well as, and, sometimes, in some aspects, better than in-feed antibiotics.

Forward Looking Statements
This news release includes certain forward-looking statements that are based upon the current expectations of management. Forward-looking statements involve risks and uncertainties associated with the business of Avivagen Inc. and the environment in which the business operates. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking, including those identified by the expressions “aim”, “anticipate”, “appear”, “believe”, “consider”, “could”, “estimate”, “expect”, “if”, “intend”, “goal”, “hope”, “likely”, “may”, “plan”, “possibly”, “potentially”, “pursue”, “seem”, “should”, “whether”, “will”, “would” and similar expressions.

Statements set out in this news release relating to the future growth and prospects for Avivagen, benefits to Avivagen resulting from receipt of regulatory approval, the benefits to customers from the use of Avivagen’s products, Avivagen’s expectations as to the results of ongoing trials, the potential for additional regulatory approvals and the possibility for OxC-beta™ Livestock to replace antibiotics in livestock feeds as growth promoters are forward-looking statements. These forward-looking statements are subject to a number of risks and uncertainties that could cause actual results or events to differ materially from current expectations. For instance, Avivagen’s products may not gain market acceptance or regulatory approval in new jurisdictions or for new applications and may not be widely accepted as a replacement for antibiotics as growth promoters in livestock feeds due to many factors, many of which are outside of Avivagen’s control.  Readers are referred to the risk factors associated with the business of Avivagen set out in Avivagen’s most recent management’s discussion and analysis of financial condition available at www.SEDAR.com. Except as required by law, Avivagen assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those reflected in the forward-looking statements.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

For more information:
Avivagen Inc.
Drew Basek
Director of Investor Relations
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Phone: 416-540-0733
E-mail: d.basek@avivagen.com
Kym Anthony
Chief Executive Officer
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Head Office Phone: 613-949-8164
Website: www.avivagen.com

---

[i] https://grains.org/markets-tools-data/markets/market-profiles/vietnam/

[ii] https://public.tableau.com/app/profile/alltech.digital/viz/AlltechGlobalFeedSurvey2021/AlltechGlobalFeedSurvey2021

Avivagen Announces Approval in Vietnam

Research, News, and Market Data on Avivagen

 

Ottawa, ON /Business Wire/ February 22, 2022 /– Avivagen Inc.  (TSXV:VIV, OTCQB:VIVXF) (“Avivagen”), a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that safely enhances feed intake and supports immune function, thereby supporting general health and performance, is pleased to announce that Avivagen’s oxidized carotenoid-based feed additive product has received approval for use in Vietnam.

“We are very excited to secure approval for our product in Vietnam, a critical feed market in Southeast Asia with strong growth potential over the coming years,” says Kym Anthony, Chief Executive Officer, Avivagen.  “We continue to make strong in-roads with feed producers across Asia and see considerable opportunity to further grow our presence in the swine and poultry feed markets in Vietnam and across the region.”

Vietnam represents the largest feed market in Southeast Asia according to a report by the US Grains Council, which indicates total annual feed consumption of over 32 MMT in 2020. Additionally, The Grains Council report states that Vietnam is a strong growth market which is expected to surpass 35 MMT of feed within the next few years.[i] Production within the country is dominated by swine, with feed for swine accounting for approximately 38% of total feed, followed by poultry accounting for 21.6% of total feed. (2020)[ii]. In addition to swine and poultry Vietnam has a surprisingly large and rapidly growing dairy industry which represents an important third target market segment for use of Avivagen’s product.

“Demand for effective alternatives to antibiotics is growing across all of Southeast Asia,” says Lesley Nernberg, Technical Sales and Marketing Consultant, Asia, Avivagen Inc. “Approval in Vietnam will allow Avivagen to capitalize on a marketplace ready for these types of innovations.”

The use of Avivagen’s product in the Vietnam feed industry is supported by the positive results of two research trials conducted specifically under Vietnamese commercial production conditions. The trials were run in collaboration with the Institute for Animal Sciences for South Vietnam and were conducted with pigs. “The results of these two trials make clear the benefits of Avivagen’s product on improving the health and productivity of piglets. We’re confident that the use of our product in this segment, along with poultry and dairy, will have a significantly positive impact for producers throughout the country,” says Jamie Nickerson, President, Avivagen Inc. “Comparable trials are underway around the world, and we expect many to result in additional regulatory approvals and customer wins over the coming months.”

About Avivagen

Avivagen is a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that, by safely supporting immune function, promote general health and performance.  It is a public corporation traded on the TSX Venture Exchange under the symbol VIV and is headquartered in Ottawa, Canada, based in partnership facilities of the National Research Council of Canada. For more information, visit www.avivagen.com. The contents of the website are expressly not incorporated by reference in this press release.

About OxC-beta™ Technology and OxC-beta™ Livestock

Avivagen’s OxC-beta™ technology is derived from Avivagen discoveries about ?-carotene and other carotenoids, compounds that give certain fruits and vegetables their bright colours. Through support of immune function the technology provides a non-antibiotic means of promoting health and growth. OxC-beta™ Livestock is a proprietary product shown to be an effective and economic alternative to the antibiotics commonly added to livestock feeds. The product is currently available for sale in the United States, Philippines, Mexico, Taiwan, New Zealand, Thailand, Brazil, Australia, and Malaysia.

Avivagen’s OxC-beta™ Livestock product is safe, effective and could fulfill the global mandate to remove all in-feed antibiotics as growth promoters. Numerous international livestock trials with poultry and swine using OxC-beta™ Livestock have proven that the product performs as well as, and, sometimes, in some aspects, better than in-feed antibiotics.

Forward Looking Statements
This news release includes certain forward-looking statements that are based upon the current expectations of management. Forward-looking statements involve risks and uncertainties associated with the business of Avivagen Inc. and the environment in which the business operates. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking, including those identified by the expressions “aim”, “anticipate”, “appear”, “believe”, “consider”, “could”, “estimate”, “expect”, “if”, “intend”, “goal”, “hope”, “likely”, “may”, “plan”, “possibly”, “potentially”, “pursue”, “seem”, “should”, “whether”, “will”, “would” and similar expressions.

Statements set out in this news release relating to the future growth and prospects for Avivagen, benefits to Avivagen resulting from receipt of regulatory approval, the benefits to customers from the use of Avivagen’s products, Avivagen’s expectations as to the results of ongoing trials, the potential for additional regulatory approvals and the possibility for OxC-beta™ Livestock to replace antibiotics in livestock feeds as growth promoters are forward-looking statements. These forward-looking statements are subject to a number of risks and uncertainties that could cause actual results or events to differ materially from current expectations. For instance, Avivagen’s products may not gain market acceptance or regulatory approval in new jurisdictions or for new applications and may not be widely accepted as a replacement for antibiotics as growth promoters in livestock feeds due to many factors, many of which are outside of Avivagen’s control.  Readers are referred to the risk factors associated with the business of Avivagen set out in Avivagen’s most recent management’s discussion and analysis of financial condition available at www.SEDAR.com. Except as required by law, Avivagen assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those reflected in the forward-looking statements.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

For more information:
Avivagen Inc.
Drew Basek
Director of Investor Relations
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Phone: 416-540-0733
E-mail: d.basek@avivagen.com
Kym Anthony
Chief Executive Officer
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Head Office Phone: 613-949-8164
Website: www.avivagen.com

---

[i] https://grains.org/markets-tools-data/markets/market-profiles/vietnam/

[ii] https://public.tableau.com/app/profile/alltech.digital/viz/AlltechGlobalFeedSurvey2021/AlltechGlobalFeedSurvey2021

  Image Credit: Janine (Wikimedia)

Prominent Study Suggests “Magic Mushroom” Ingredient on Depression has Long-Term Benefit

 

What happens months after research is completed to determine the effectiveness of an experimental substance such as psilocybin, the chemical found in “psychedelic” mushrooms?  Are the subjects whose results were recorded earlier revisited to determine long-term impact or effectiveness?

In a follow-up to a year-old study, researchers at Johns Hopkins measured the impact of treatment for major depressive disorder of a psychedelic treatment with psilocybin for periods up to a year. The initial data demonstrated that those treated were still benefitting one month after treatment. It has now been 12 months, the participants were again measured, and the conclusion of the research shows substantial antidepressant effects of psilocybin-assisted therapy, given with supportive psychotherapy, may last at least a year for some patients.

“Our findings add to evidence that, under carefully controlled conditions, this is a promising therapeutic approach that can lead to significant and durable improvements in depression,” said Natalie Gukasyan, M.D., assistant professor of psychiatry and behavioral sciences at the Johns Hopkins University School of Medicine. Professor Gukasyan, added the caveat, “the results we see are in a research setting and require quite a lot of preparation and structured support from trained clinicians and therapists, and people should not attempt to try it on their own.”

About the Study

The researchers recruited 27 participants with an approximately two-year history of depression. Men totaled 8, women made up the remaining 19. Eighty-eight percent of the participants had previously been treated with common antidepressant medications, and 58% reported using antidepressants in their current depressive episodes. The average age of participants was 40.  

Participants were randomized into one of two groups in which they received the intervention either immediately, or after an eight-week waiting period. At the time of treatment, all participants were provided with preparatory meetings with the treatment facilitators. Then participants received two doses of psilocybin, given approximately two weeks apart between August 2017 and April 2019. The subjects returned for follow-up after one day and one week after each session, and then at one, three, six, and 12 months following the second session.  24 participants completed both psilocybin sessions and all follow-up assessment visits.

The researcher’s initial report stated that psilocybin treatment in both groups produced large decreases in depression and that depression severity remained low one, three, six, and 12 months after treatment. Depressive symptoms were measured before and after treatment using the GRID-Hamilton Depression Rating Scale. This is a standard depression assessment tool, in which a score of 24 or more indicates severe depression, 17–23 moderate depression, 8–16 mild depression, and 7 or less no depression. For most participants, scores for the overall treatment decreased from 22.8 at pretreatment to 8.7 at one week, 8.9 at four weeks, 9.3 at three months, 7 at six months, and 7.7 at 12 months after treatment. Participants had stable rates of response to the treatment and remission of symptoms throughout the follow-up period, with 75% response and 58% remission at 12 months.

Take-Away

There continues to be a growth in clinical level medical research and analysis of once-taboo psychedelics, particularly psilocybin. Treatment with psilocybin has shown promise in research settings for treating a range of mental health disorders and addictions. In this most recent release of study results at Johns Hopkins Medical. The chemical found in some mushrooms “not only produces significant and immediate effects, it also has a long duration, which suggests that it may be a uniquely useful new treatment for depression.” The significance is, standard antidepressants, must be taken over long periods of time. Based on this small study, psilocybin has the potential to relieve symptoms for extended periods measured in months, with only one or two treatments.

Paul Hoffman

Managing Editor, Channelchek

Suggested Reading

Will Investors Experience a Quicker High in Psychedelics as Cannabis Creates the “Model”?

A Quick Look at 8 Small Caps in the Growing Psychedelics Space

Cannabis Bank Sowing New Seeds as it Announces SPAC Merger

Marijuana is Winning the Sports Battle

 

Sources

https://jamanetwork.com/journals/jamapsychiatry/fullarticle/2772630?resultClick=1

https://tripsitter.com/magic-mushrooms/legal/

https://journals.sagepub.com/doi/10.1177/02698811211073759

 

Stay up to date. Follow us:

Study Links Gene to Cognitive Resilience in the Elderly

 

Anne Trafton | MIT News Office

 

Many people develop Alzheimer’s or other forms of dementia as they get older. However, others remain sharp well into old age, even if their brains show underlying signs of neurodegeneration.

Among these cognitively resilient people, researchers have identified education level and amount of time spent on intellectually stimulating activities as factors that help prevent dementia. A new study by MIT researchers shows that this kind of enrichment appears to activate a gene family called MEF2, which controls a genetic program in the brain that promotes resistance to cognitive decline.

The researchers observed this link between MEF2 and cognitive resilience in both humans and mice. The findings suggest that enhancing the activity of MEF2 or its targets might protect against age-related dementia.

“It’s increasingly understood that there are resilience factors that can protect the function of the brain,” says Li-Huei Tsai, director of MIT’s Picower Institute for Learning and Memory. “Understanding this resilience mechanism could be helpful when we think about therapeutic interventions or prevention of cognitive decline and neurodegeneration-associated dementia.” Tsai is the senior author of the study, which appeared in Science Translational Medicine. The lead authors are recent MIT Ph.D. recipient Scarlett Barker and MIT postdoctoral fellow and Boston Children’s Hospital physician Ravikiran (Ravi) Raju.

 

Protective Effects

A large body of research suggests that environmental stimulation offers some protection against the effects of neurodegeneration. Studies have linked education level, type of job, number of languages spoken, and amount of time spent on activities such as reading and doing crossword puzzles to higher degrees of cognitive resilience.

The MIT team set out to try to figure how these environmental factors affect the brain at the neuronal level. They looked at human datasets and mouse models in parallel, and both tracks converged on MEF2 as a critical player.

MEF2 is a transcription factor that was originally identified as a factor important for cardiac muscle development but later was discovered to play a role in neuron function and neurodevelopment. In two human datasets comprising slightly more than 1,000 people altogether, the MIT team found that cognitive resilience was highly correlated with expression of MEF2 and many of the genes that it regulates.

Many of those genes encode ion channels, which control a neuron’s excitability, or how easily it fires an electrical impulse. The researchers also found, from a single-cell RNA-sequencing study of human brain cells, that MEF2 appears to be most active in a subpopulation of excitatory neurons in the prefrontal cortex of resilient individuals.

To study cognitive resilience in mice, the researchers compared mice who were raised in cages with no toys, and mice placed in a more stimulating environment with a running wheel and toys that were swapped out every few days. As they found in the human study, MEF2 was more active in the brains of the mice exposed to the enriched environment. These mice also performed better in learning and memory tasks.

When the researchers knocked out the gene for MEF2 in the frontal cortex, this blocked the mice’s ability to benefit from being raised in the enriched environment, and their neurons became abnormally excitable.

“This was particularly exciting as it suggested that MEF2 plays a role in determining overall cognitive potential in response to variables in the environment,” Raju says.

The researchers then explored whether MEF2 could reverse some of the symptoms of cognitive impairment in a mouse model that expresses a version of the tau protein that can form tangles in the brain and is linked with dementia. If these mice were engineered to overexpress MEF2 at a young age, they did not show the usual cognitive impairments produced by the tau protein later in life. In these mice, neurons overexpressing MEF2 were less excitable.

“A lot of human studies and mouse model studies of neurodegeneration have shown that the neurons become hyperexcitable in early stages of disease progression,” Raju says. “When we overexpressed MEF2 in a mouse model of neurodegeneration, we saw that it was able to prevent this hyperexcitability, which might explain why they performed cognitively better than control mice.”

 

Enhancing Resilience

 The findings suggest that enhancing MEF2 activity could help to protect against dementia; however, because MEF2 also affects other types of cells and cellular processes, more study is needed to make sure that activating it wouldn’t have adverse side effects, the researchers say.

The MIT team now hopes to further investigate how MEF2 becomes activated by exposure to an enriching environment. They also plan to examine some of the effects of the other genes that MEF2 controls, beyond the ion channels they explored in this study. Such studies could help to reveal additional targets for drug treatments.

“You could potentially imagine a more targeted therapy by identifying a subset or a class of effectors that is critically important for inducing resilience and neuroprotection,” Raju says.

 

Suggested Reading

Advancing Research into Alzheimer’s Disease with Stem Cells

Understanding Alzheimer’s, Dementia, ALS, and Neurodegenerative Diseases

The Anti-Aging and Rejuvenating Properties of Stem Cells

FDA Oncology Director Expresses Concerns Over Applicability of Chinese Data

 

 

Stay up to date. Follow us:

Study Links Gene to Cognitive Resilience in the Elderly

 

Anne Trafton | MIT News Office

 

Many people develop Alzheimer’s or other forms of dementia as they get older. However, others remain sharp well into old age, even if their brains show underlying signs of neurodegeneration.

Among these cognitively resilient people, researchers have identified education level and amount of time spent on intellectually stimulating activities as factors that help prevent dementia. A new study by MIT researchers shows that this kind of enrichment appears to activate a gene family called MEF2, which controls a genetic program in the brain that promotes resistance to cognitive decline.

The researchers observed this link between MEF2 and cognitive resilience in both humans and mice. The findings suggest that enhancing the activity of MEF2 or its targets might protect against age-related dementia.

“It’s increasingly understood that there are resilience factors that can protect the function of the brain,” says Li-Huei Tsai, director of MIT’s Picower Institute for Learning and Memory. “Understanding this resilience mechanism could be helpful when we think about therapeutic interventions or prevention of cognitive decline and neurodegeneration-associated dementia.” Tsai is the senior author of the study, which appeared in Science Translational Medicine. The lead authors are recent MIT Ph.D. recipient Scarlett Barker and MIT postdoctoral fellow and Boston Children’s Hospital physician Ravikiran (Ravi) Raju.

 

Protective Effects

A large body of research suggests that environmental stimulation offers some protection against the effects of neurodegeneration. Studies have linked education level, type of job, number of languages spoken, and amount of time spent on activities such as reading and doing crossword puzzles to higher degrees of cognitive resilience.

The MIT team set out to try to figure how these environmental factors affect the brain at the neuronal level. They looked at human datasets and mouse models in parallel, and both tracks converged on MEF2 as a critical player.

MEF2 is a transcription factor that was originally identified as a factor important for cardiac muscle development but later was discovered to play a role in neuron function and neurodevelopment. In two human datasets comprising slightly more than 1,000 people altogether, the MIT team found that cognitive resilience was highly correlated with expression of MEF2 and many of the genes that it regulates.

Many of those genes encode ion channels, which control a neuron’s excitability, or how easily it fires an electrical impulse. The researchers also found, from a single-cell RNA-sequencing study of human brain cells, that MEF2 appears to be most active in a subpopulation of excitatory neurons in the prefrontal cortex of resilient individuals.

To study cognitive resilience in mice, the researchers compared mice who were raised in cages with no toys, and mice placed in a more stimulating environment with a running wheel and toys that were swapped out every few days. As they found in the human study, MEF2 was more active in the brains of the mice exposed to the enriched environment. These mice also performed better in learning and memory tasks.

When the researchers knocked out the gene for MEF2 in the frontal cortex, this blocked the mice’s ability to benefit from being raised in the enriched environment, and their neurons became abnormally excitable.

“This was particularly exciting as it suggested that MEF2 plays a role in determining overall cognitive potential in response to variables in the environment,” Raju says.

The researchers then explored whether MEF2 could reverse some of the symptoms of cognitive impairment in a mouse model that expresses a version of the tau protein that can form tangles in the brain and is linked with dementia. If these mice were engineered to overexpress MEF2 at a young age, they did not show the usual cognitive impairments produced by the tau protein later in life. In these mice, neurons overexpressing MEF2 were less excitable.

“A lot of human studies and mouse model studies of neurodegeneration have shown that the neurons become hyperexcitable in early stages of disease progression,” Raju says. “When we overexpressed MEF2 in a mouse model of neurodegeneration, we saw that it was able to prevent this hyperexcitability, which might explain why they performed cognitively better than control mice.”

 

Enhancing Resilience

 The findings suggest that enhancing MEF2 activity could help to protect against dementia; however, because MEF2 also affects other types of cells and cellular processes, more study is needed to make sure that activating it wouldn’t have adverse side effects, the researchers say.

The MIT team now hopes to further investigate how MEF2 becomes activated by exposure to an enriching environment. They also plan to examine some of the effects of the other genes that MEF2 controls, beyond the ion channels they explored in this study. Such studies could help to reveal additional targets for drug treatments.

“You could potentially imagine a more targeted therapy by identifying a subset or a class of effectors that is critically important for inducing resilience and neuroprotection,” Raju says.

 

Suggested Reading

Advancing Research into Alzheimer’s Disease with Stem Cells

Understanding Alzheimer’s, Dementia, ALS, and Neurodegenerative Diseases

The Anti-Aging and Rejuvenating Properties of Stem Cells

FDA Oncology Director Expresses Concerns Over Applicability of Chinese Data

 

 

Stay up to date. Follow us:

Wednesday, February 16, 2022

Item 9 Labs (INLB)
1Q22 Results In-line with Expectations

Item 9 Labs Corp. (OTCQX: INLB) is a vertically integrated cannabis operator and dispensary franchisor delivering premium products from its large-scale cultivation and production facilities in the United States. The award-winning Item 9 Labs brand specializes in best-in-class products and user experience across several cannabis categories. The company also offers a unique dispensary franchise model through the national Unity Rd. retail brand. Easing barriers to entry, the franchise provides an opportunity for both new and existing dispensary owners to leverage the knowledge, resources, and ongoing support needed to thrive in their state compliantly and successfully. Item 9 Labs brings the best industry practices to markets nationwide through distinctive retail experience, cultivation capabilities, and product innovation. The veteran management team combines a diverse skill set with deep experience in the cannabis sector, franchising, and the capital markets to lead a new generation of public cannabis companies that provide transparency, consistency, and well-being. Headquartered in Arizona, the company is currently expanding its operations space by 650,000+ square feet on its 50-acre site, one of the largest properties in Arizona zoned to grow and cultivate flower. For additional information, visit item9labscorp.com.

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

1Q22 Results. For the quarter ended December 31st, Item 9 reported revenue of $6.2 million, an increase of $3.1 million, or 103.5%, compared with $3.0 million in 1Q21. Net loss was $3.3 million, or $0.04 per share. We had projected revenue of $6.2 million and a net loss of $3.8 million, or $0.04 per share. Adjusted EBITDA for the quarter was a loss of $1.2 million.


Moving Forward.  As the Company released FY21 results less than a month ago, there isn’t a lot of new activity to report. The Nevada facility is 95% complete and should receive its Temporary Certificate of Occupancy in 2Q22. The Arizona expansion should be up and running during the summer. Recall, this will increase plant material output by 250-300%, reducing the need for outsourced product by …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

Wednesday, February 16, 2022

Baudax Bio (BXRX)
Adjusting price target for stock split and updated funding expectations

Baudax Bio is a biopharmaceutical company focused on developing therapies for post-operative pain, peri-operative pain, and anesthesia. The company currently has one approved therapy in ANJESO for post-operative pain. Proprietary ANJESO (meloxicam) injection is the first and only once-daily IV analgesic. The company also has a pipeline of early-stage candidates with two novel neuromuscular blocking agents (NMBAs), a proprietary related reversal agent to their NMBAs, and Dex-IN, an intranasal formulation of dexmedetomidine (Dex) that has sedative, analgesic, and anti-anxiety properties.

Gregory Aurand, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Board approved a 1:35 reverse split. A shareholder meeting held February 9th approved an amendment granting the Board of Directors authority to effect a reverse split. Last night the Board approved and filed a 1 for 35 reverse split, effective February 16, 2022. This split will help BXRX regain Nasdaq compliance.


New CDC proposal encourages non-opioid therapies.  Last updated in 2016, the CDC has opened a public comment 60-day period for updated guidelines. The updated guidelines remove hard limitations on opioid dosage but, given opioid side effects and addiction problems, continue to recommend non-opioid therapies, like ANJESO, whenever possible to treat chronic and acute pain …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

PDS Biotech Announces Clinical Trial with Mayo Clinic to Study PDS0101 in Early Stage Pre-Metastatic- HPV-Associated Head and Neck Cancer

Research, News, and Market Data on PDS Biotech

 

The study evaluates PDS0101 with and without KEYTRUDA^® prior to surgery for HPV-associated oropharyngeal cancer

FLORHAM PARK, N.J., Feb. 15, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing novel cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technologies, today announced the initiation of an Investigator-Initiated Trial (ITT), MC200710, for PDS0101 alone or in combination with the checkpoint inhibitor, KEYTRUDA^®, in patients with HPV-associated oropharyngeal cancer (HPV(+)OPSCC) at high risk of recurrence. The trial is being led by Drs. David Routman, Katharine Price, Kathryn Van Abel, and Ashish Chintakuntlawar of Mayo Clinic, a nationally and internationally recognized center of excellence for the treatment of head and neck cancers.

HPV(+)OPSCC is now the most common type of head and neck cancer and has been increasing significantly in recent years.  Oropharyngeal cancer is a disease in which cancer cells form in the tonsil tissues of the back (base of tongue) and side (palatine tonsils) of the throat. According to the National Cancer Institute, smoking or being infected with the human papillomavirus (HPV), especially HPV16, can increase the risk of oropharyngeal cancer. The American Cancer Society estimates there are over 54,000 new cases of oral and oropharyngeal cancer and over 11,000 related deaths in the United States, annually.

“We are excited to have a team of doctors at Mayo Clinic conducting a trial to study our novel Versamune^®-based T cell immunotherapy platform and lead asset, PDS0101 in earlier-stage disease,” commented Dr. Lauren Wood, Chief Medical Officer of PDS Biotech. “This upcoming trial not only broadens our addressable patient population of those affected by the increasing incidence of HPV(+)OPSCC, but also allows us to better understand the activity of PDS0101 alone or in combination with KEYTRUDA^® in earlier stages of disease.”

The study treatment will be administered before patients proceed to transoral robotic surgery (TORS) with curative intent. Treatment in this setting is referred to as neoadjuvant treatment. PDS0101 has been shown to induce killer T-cells that target and kill HPV-positive cancers, either alone or in combination with checkpoint inhibitors in preclinical studies, and in combination in clinical studies of patients with advanced recurrent/metastatic HPV-associated cancers.  This study will explore whether PDS0101 with or without checkpoint inhibition may increase HPV-specific anti-tumor responses, potentially resulting in tumor shrinkage, pathologic regression, and decreases in circulating tumor DNA (ctDNA).

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technology platforms.

Our Versamune^®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them.  The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate, and ovarian cancers.  

Our Infectimune™-based vaccines have demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T-cell responses including long-lasting memory T-cell responses. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® based products; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^® based products and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the acceptance by the market of the Company’s product candidates, if approved; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, the Company’s product candidates; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: rich@cg.capital

PDS Biotech Announces Clinical Trial with Mayo Clinic to Study PDS0101 in Early Stage Pre-Metastatic- HPV-Associated Head and Neck Cancer

Research, News, and Market Data on PDS Biotech

 

The study evaluates PDS0101 with and without KEYTRUDA^® prior to surgery for HPV-associated oropharyngeal cancer

FLORHAM PARK, N.J., Feb. 15, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing novel cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technologies, today announced the initiation of an Investigator-Initiated Trial (ITT), MC200710, for PDS0101 alone or in combination with the checkpoint inhibitor, KEYTRUDA^®, in patients with HPV-associated oropharyngeal cancer (HPV(+)OPSCC) at high risk of recurrence. The trial is being led by Drs. David Routman, Katharine Price, Kathryn Van Abel, and Ashish Chintakuntlawar of Mayo Clinic, a nationally and internationally recognized center of excellence for the treatment of head and neck cancers.

HPV(+)OPSCC is now the most common type of head and neck cancer and has been increasing significantly in recent years.  Oropharyngeal cancer is a disease in which cancer cells form in the tonsil tissues of the back (base of tongue) and side (palatine tonsils) of the throat. According to the National Cancer Institute, smoking or being infected with the human papillomavirus (HPV), especially HPV16, can increase the risk of oropharyngeal cancer. The American Cancer Society estimates there are over 54,000 new cases of oral and oropharyngeal cancer and over 11,000 related deaths in the United States, annually.

“We are excited to have a team of doctors at Mayo Clinic conducting a trial to study our novel Versamune^®-based T cell immunotherapy platform and lead asset, PDS0101 in earlier-stage disease,” commented Dr. Lauren Wood, Chief Medical Officer of PDS Biotech. “This upcoming trial not only broadens our addressable patient population of those affected by the increasing incidence of HPV(+)OPSCC, but also allows us to better understand the activity of PDS0101 alone or in combination with KEYTRUDA^® in earlier stages of disease.”

The study treatment will be administered before patients proceed to transoral robotic surgery (TORS) with curative intent. Treatment in this setting is referred to as neoadjuvant treatment. PDS0101 has been shown to induce killer T-cells that target and kill HPV-positive cancers, either alone or in combination with checkpoint inhibitors in preclinical studies, and in combination in clinical studies of patients with advanced recurrent/metastatic HPV-associated cancers.  This study will explore whether PDS0101 with or without checkpoint inhibition may increase HPV-specific anti-tumor responses, potentially resulting in tumor shrinkage, pathologic regression, and decreases in circulating tumor DNA (ctDNA).

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technology platforms.

Our Versamune^®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them.  The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate, and ovarian cancers.  

Our Infectimune™-based vaccines have demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T-cell responses including long-lasting memory T-cell responses. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® based products; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^® based products and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the acceptance by the market of the Company’s product candidates, if approved; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, the Company’s product candidates; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: rich@cg.capital

Tuesday, February 15, 2022

Axcella Therapeutics (AXLA)
AXA1125 Receives Fast Track Designation For NASH Indication

Axcella Health Inc. is a clinical-stage biotechnology company focused on treating complex diseases and improvinge health using endogenous metabolic modulator, or EMM, compositions. Its product candidates are comprised of multiple EMMs that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. The company’s pipeline includes two lead therapeutic candidates:, AXA1665 for the reduction in risk of recurrent overt hepatic encephalopathy (OHE) , and AXA1125 for the treatment of non-alcoholic steatohepatitis (NASH).

Robert LeBoyer, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Fast Track Designation Is A Strong Positive.  Axcella announced that the FDA has granted Fast Track Designation for AXA1125 in its non-alcoholic steatohepatitis (NASH) with fibrosis indication. AXA1125 is a formulation of amino acids in development to resolve NASH by controlling metabolic, inflammatory, and fibrotic pathways. AXA 1125 is currently in the Phase 2b EMMPACT clinical trial for the NASH indication.


Fast Track Designation Signifies Recognition Of Potential Impact.  AXA1125 was granted Fast Track Designation after the FDA reviewed clinical data from the previous studies. This designation is intended to assist development and shorten time to market by allowing the company to have more frequent communications about clinical plans and requirements. It can also allow for rolling submission and …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

Data Presented at 2022 International Stroke Congress Suggests Role for Non-invasive Vagus Nerve Stimulation (nVNS) for Treatment of Acute Stroke

News and Market Data on electroCore

 

Clinical Trial (n=69) suggests safety and feasibility of nVNS for the acute treatment of stroke.

Relative Ischemic Lesion Growth Decreased by 65.9% with nVNS vs. Sham Treatment.

ROCKAWAY, N.J., 
Feb. 09, 2022 (GLOBE NEWSWIRE) — 
electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, today announced top line data from three abstracts being presented at the American Heart Associations’ 2022 
International Stroke Conference (
February 9-11, 2022, 
New Orleans) on the possible role of nVNS in the acute treatment of stroke.

The nVNS research presented at the 
International Stroke Conference 2022 includes:

Non-invasive Vagus Nerve Stimulation for The Acute Treatment of Ischemic or Hemorrhagic Stroke (TR-VENUS)

In this randomized, sham controlled, multi-center study conducted at nine academic centers in 
Turkey, the intent-to-treat (ITT) population included 69 subjects (61 ischemic stroke and eight intracerebral hemorrhage) distributed into three treatment arms: sham (n=25), standard-dose nVNS (7 stimulations over one hour; n=19), and high-dose nVNS (7 stimulations in hour 1 and hour 5; n=25). Every subject in the ITT population received all prespecified treatment stimulations per protocol and 97% of all randomized subjects in the study received their first stimulation less than 6 hours from stroke onset. The study met its primary objective with the composite primary safety endpoint being consistent across the three treatment arms indicating that nVNS was able to be administered safely in this acute stroke population. The study also met all secondary safety and feasibility endpoints. Relative infarct growth, measured by diffusion weighted imaging, in the high dose nVNS group (63.3%) was lower than in the sham group (185.8%; p=0.05) against baseline. Dr. Murat Arasava, one of the primary investigators of the TR-VENUS study and Professor of Neurology at the 
Hacettepe University in 
Ankara, Turkey commented, “We are pleased to have successfully completed this first trial of nVNS as a possible treatment for acute stroke and believe our data suggests that nVNS may be safe and feasible in both ischemic and hemorrhagic stroke. The efficacy signal suggested by the reduction in relative infarct growth rate in the ischemic population, which needs to be confirmed in larger studies, clearly provides the basis for additional research to further define the role for nVNS for the acute treatment of stroke.”

Non-invasive Vagus Nerve Stimulation in Acute Ischemic Stroke (NOVIS)

NOVIS is a prospective randomized clinical trial with blinded outcome assessment being conducted at the 
Leiden University Medical Center (Leiden, 
The Netherlands). 150 patients with ischemic stroke are being randomly allocated (1:1) to nVNS for five days in addition to standard treatment versus standard treatment alone. The primary endpoint is the final infarct volume on day five assessed with MRI. This study features a greater number of stimulations over five days than TR-VENUS, and advanced imaging endpoints including measurement of the penumbra, that should provide additional insight and clarity surrounding the role of nVNS for the acute treatment of ischemic stroke. Dr.  Anne van der Meij, from the 
Leiden University Medical Center, is presenting the poster at the 
International Stroke Conference and commented, “Our study is now more than 50% enrolled and our efficacy endpoints may provide greater definition, and possibly confirmation, of the efficacy of nVNS for the acute treatment of ischemic stroke.”

Effect Of Non-invasive Vagus Nerve Stimulation in Hemorrhagic Brain Injury and Permanent Ischemic Stroke in Rats

Dr.  Ilknur Ay of the 
Massachusetts General Hospital and 
Harvard Medical School presented the results of a pre-clinical study conducted in her lab at the 
Athinoula A. Martinos Center for Biomedical Imaging that supports prior evidence that nVNS may have therapeutic potential in ischemic stroke. The lack of adverse events in two different validated models of Intracerebral Hemorrhage in rats shown in this study suggests that nVNS could be safely administered as early as an ambulatory setting before the stroke etiology (ischemic vs. hemorrhagic) has been determined.

Stroke is ranked as the second leading cause of death worldwide with an annual mortality rate of about 5.5 million. The burden of stroke lies not only in its high mortality rate, but in the high morbidity rate as well with up to 50% of stroke survivors being chronically disabled. Stroke is a disease of immense public health importance with serious economic and social consequences. The public health burden of stroke is set to rise over future decades because of demographic transitions of populations across the world. According to the 
American Heart Association by 2030, 3.88% of the US population >18 years of age is projected to have had a stroke. Between 2012 and 2030, real (2010$) total direct annual stroke-related medical costs are expected to increase from 
$71.55 billion to 
$184.13 billion. Real indirect annual costs (attributable to lost productivity) are projected to rise from 
$33.65 billion to 
$56.54 billion over the same period. Overall, total annual costs of stroke are projected to increase to 
$240.67 billion by 2030, an increase of 129%.

Eric Liebler, Senior Vice President of Neurology for electroCore, commented, “We congratulate all of the investigators, patients and sites that have first in human study suggesting a role for nVNS in treatment of acute stroke. If effective, nVNS would represent a breakthrough treatment that could be safely deployed much earlier, more safely and more easily than current stroke treatments. As time to treatment is considered one of the most critical factors in the acute treatment of stroke, the ability to treat as early as in an ambulatory setting, before the type of stroke (ischemic or hemorrhagic) is confirmed by imaging, would represent a significant advance in the treatment of stroke. We look forward to the anticipated full publication of the TR-VENUS study in a peer reviewed journal later this year and the completion of the NOVIS study in 2023.”

About electroCore, Inc.
electroCore, Inc. is a commercial stage bioelectronic medicine company dedicated to improving patient outcomes through its non-invasive vagus nerve stimulation therapy platform, initially focused on the treatment of multiple conditions in neurology. The company’s current indications are the preventive treatment of cluster headache and migraine, the acute treatment of migraine and episodic cluster headache, the acute and preventive treatment of migraines in adolescents, and paroxysmal hemicrania and hemicrania continua in adults.

For more information, visit www.electrocore.com.

About gammaCore™
gammaCore™ (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck as an adjunctive therapy to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore can be self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients.

gammaCore (nVNS) is FDA cleared in the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, and the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients, and paroxysmal hemicrania and hemicrania continua in adult patients. gammaCore is CE-marked in the European Union for the acute and/or prophylactic treatment of primary headache (Migraine, Cluster Headache, Trigeminal Autonomic Cephalalgias and Hemicrania Continua) and Medication Overuse Headache in adults.

gammaCore is contraindicated for patients if they:

• Have an active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• Have a metallic device, such as a stent, bone plate, or bone screw, implanted at or near the neck
• Are using another device at the same time (e.g., TENS Unit, muscle stimulator) or any portable electronic device (e.g., mobile phone)
  

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Adolescent patients with congenital cardiac issues
• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (less than 12 years)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia

For more information, please visit gammaCore.com

Forward-Looking Statements
This press release and other written and oral statements made by representatives of electroCore may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s business prospects and clinical and product development plans (including with respect to enrollment in ongoing studies); its pipeline or potential markets for its technologies; the timing, outcome and impact of regulatory, clinical and commercial developments; the issuance of 
U.S. and international patents providing expanded IP coverage; the possibility of future business models and revenue streams from the company’s potential use of nVNS for the acute treatment of stroke and hemorrhagic brain injury, the potential of nVNS generally and gammaCore in particular and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, the potential impact and effects of COVID-19 on the business of electroCore, electroCore’s results of operations and financial performance, and any measures electroCore has and may take in response to COVID-19 and any expectations electroCore may have with respect thereto, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at www.sec.gov.

Investors:
Rich Cockrell

CG Capital
404-736-3838
ecor@cg.capital

or

Media Contact:
Jackie Dorsky
electroCore
908-313-6331
jackie.dorsky@electrocore.com

  Phil & Pam Gladwell (Flickr)

FDA Oncology Director Expresses Concerns Over Applicability of Chinese Data

 

The Food and Drug Administration (FDA) will soon begin reviewing two dozen applications for cancer drugs. The trial data is based, in many cases, entirely on tests from mainland China. The U.S. regulator has begun expressing concerns about the quality of studies conducted in China and whether the results should even apply to patients in the U.S.

 

Background

Eli Lilly (LLY) has applied for a license to roll out a lung-cancer immunotherapy developed in China and sell it at a lower price than similar drugs now in the U.S. The application is for a drug called Sintilab, which is a PD-1 monoclonal antibody checkpoint inhibitor for non-small cell lung cancer. The FDA has concerns over the validity of test results. This could threaten the plan to bring the treatment to market later this year. How the FDA concerns translate into actions will become clearer after February 10^th when they begin reviewing the data from Lilly’s Chinese partner, Innovent Biologics Inc. Lilly’s application is just the first of many applications to be considered by the agency, the decision is expected by late March.

The shift in thinking could stall or threaten applications from many large drugmakers, including Eli Lilly and Novartis, who stand to gain billions of dollars in sales from being the conduit for these medicines. It could also heighten trade tensions between the two countries.

 

“The elephant in the room is obviously, what is the quality of the data that is coming from these foreign countries?” Dr. Richard Pazdur, FDA

 

FDA Considerations

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, has been quoted in academic and medical literature about his new concerns regarding “me-too” drugs coming from China and the fundamental challenges they pose to the agency’s drug approval criteria. This includes whether results, provided primarily from test subjects in China, are applicable to Americans, and the validity of the data. Pazdur has said, “We have nothing against drugs being developed in China. Our issue is, are those results generalizable to the U.S. population?”

Clinical trials, especially extensive, late-stage studies that regulators review to decide whether to approve a new drug, are among the highest research and development expenses. Industry executives are watching to see if the newer mindset leads to delays or outright FDA rejections of increased efforts to bring a pipeline of treatments to American patients from abroad. Perhaps measures and additional assessments of benefits and safety profiles on different populations will be needed in the future. Dr. Pazdur has expressed when drugs are tested only or primarily in one country such as China, it is difficult to assess whether the drug would have the same benefits and safety profile in the U.S. population. Dr. Pazdur also said he was concerned the Chinese studies used outdated study designs, which don’t directly establish whether the China-developed drug works, as well as similar drugs, approved in the U.S.

There are also concerns that go beyond the applicability of the data. Dr. Pazdur has also shown concern about the integrity of data generated by drug studies in China. According to the British Medical Journal, an analysis by Chinese regulators in 2016 found that about 80% of domestic drug applications reviewed at that time, contained fabricated, flawed, or insufficient data in the studies – in some cases, there were discrepancies between original study data and what was submitted to regulators.

 

Take-Away

A growing trend of large drugmakers partnering with testing labs in China as part of an application for U.S. FDA approval may be faced with an FDA, that had once been friendly to the idea, but now has openly changed its tone. The new thinking seems to be, that there may be differences in medical care and populations that affect how a drug performs.

This could impact two dozen applications before the FDA. A final agency decision on whether to clear the Lilly/Innovent treatment is expected by the end of March.

Paul Hoffman

Managing Editor, Channelchek

 

Suggested Reading

Understanding the FDA Medical Device Approval Pathways Helps Investors

Immunotherapy to Treat Cancer May Involve Placing Cancer Cells in the Body

Therapeutic Research Advanced by Stem Cell Science

Imugene (IUGNF) – Three Innovative Platforms That Could Change Cancer Treatment

 

Source

https://www.fda.gov/media/156021/download

https://www.statnews.com/2022/02/04/fda-richard-pazdur-cancer-drug-eli-lilly-china/

https://endpts.com/appearing-to-walk-back-fdas-openness-to-foreign-trials-pazdur-suggests-eli-lillys-pd-1-will-face-tough-road-ahead/

https://cancerletter.com/conversation-with-the-cancer-letter/20220204_1/

https://www.wsj.com/articles/fda-raises-concerns-about-china-developed-drugs-11644408180?mod=Searchresults_pos1&page=1

https://www.nejm.org/doi/full/10.1056/NEJMp2116863

 

Stay up to date. Follow us: