Dealing With False Positives During Drug Screening Process


Image Credit: Alex Green (Pexels)


Discovering New Drugs is a Long and Expensive Process – Chemical Compounds that Dupe Screening Tools Make it Even Harder

Modern drug discovery is an expensive and complicated process. Hundreds of scientists and at least a decade are often required to produce a single medicine. One of the most critical steps in this process is the first one – identifying new chemical compounds that could be developed into new medicines.

Researchers rely heavily on bioassays to identify potential drug candidates. These tests measure a compound’s ability to act on a biological target of interest. Candidates that show up as a “hit” by interacting with a target of interest (such as fitting into a binding site on the target) move on to further study and development. Advances in technology called high-throughput screening have allowed researchers to run thousands of compounds through bioassays in a short time, significantly streamlining the process.

But some of these “hits” don’t actually interact with the target as intended. And for the unwary researcher, this can lead down a rabbit hole of lost time and money.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It was written by and represents the research-based opinions of Martin Clasby, Research Assistant Professor of Medicinal Chemistry, University of Michigan.

I am a medicinal chemist who has been working in the drug discovery field for over 26 years, and one of the greatest challenges I have faced in my research is selecting good candidates from drug screening tests. One particular category of compounds, known as pan-assay interference compounds, or PAINS, is a common pitfall.

Bioassays involve placing a chemical compound together with the target of interest and measuring the strength of their interaction. Researchers assess interaction strength using a number of methods depending on how the bioassay is designed. A common assay design emits light when there is an interaction, where the intensity of the light depends upon the strength of interaction.

PAINS refer to compounds that often come up as false positives during the screening process. Because of certain characteristics of these molecules, they can interact with a target in nonspecific or unexpected ways. Some can even react chemically with the target. So while PAINS may come up as a hit in a screen, it doesn’t necessarily mean they actually do what researchers hoped they’d do. Common worst offenders include compounds like quinones, catechols and rhodanines.


Unlike desired drug compounds that interact specifically with a target of interest, PAINS react nonspecifically with a wide variety of targets. Image credit: Bcary (Wikipedia Commons)

There are a number of ways that PAINS dupe bioassays.

Some PAINS have properties that cause them to emit light (or fluoresce) under certain conditions. Since many bioassays detect light as a signal for a hit, this can confuse the assay readout and result in a false positive.

Other PAINS can act as redox cyclers in bioassays – producing hydrogen peroxide that can block the target and be misread as a hit.

Similarly, some PAINS form colloidal aggregates – clumps of molecules that interfere with the target of interest by absorbing it or modifying the molecular structure. In rare cases, these clumps can even elicit a desired interaction with the target of interest because of their large size.

Trace impurities left over from manufacturing can also elicit a PAINS response.

To make things even more complicated, because PAINS react with targets much more strongly than most compounds that are true drug candidates, PAINS often appear as the most promising hits in screening.

Curcumin, the bright yellow chemical commonly found in the turmeric in curry, is one notorious example of a pan-assay interference compound.

 

What Can be Done About PAINS?

An estimated 5% to 12% of compounds in the screening libraries academic institutions use for drug discovery consist of PAINS. Scientists misled by a false positive can waste considerable time if they try to develop these compounds into usable drugs.

Since researchers became aware of the existence of PAINS, medicinal chemists have identified frequent offenders and actively remove these compounds from screening libraries. However, some compounds will always fall through the cracks. It is ultimately up to the researcher to identify and discard these PAINS when they show up as false positives.

There are a few things researchers can do to filter out PAINS. In some cases, visually inspecting compounds for structural similarities with other known PAINS can be enough. For other cases, additional experiments are necessary to eliminate false positives.

Testing for the presence of hydrogen peroxide, for example, can help identify redox cyclers. Likewise, adding detergents can help break up colloidal aggregates. And bioassays that do not use light detection to register hits can circumvent PAINS that emit light.

Even the most experienced medicinal chemist needs to be cognizant of the dangers of these false positives. Taking steps to ensure that these types of compounds don’t make it to the next stage of drug discovery can avoid wasted time and effort and ultimately lead to a more efficient and cost-effective drug discovery process.

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Release – Tonix Pharmaceuticals to Present at the 2022 Q2 Investor Summit



Tonix Pharmaceuticals to Present at the 2022 Q2 Investor Summit

Research, News, and Market Data on Tonix Pharmaceuticals

 

CHATHAM, N.J., April 28, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, announced today that Jessica Morris, Chief Operating Officer of Tonix Pharmaceuticals, will present at the 2022 Q2 Investor Summit on Wednesday, May 4, 2022, at 11:00 a.m. ET at the Westin New York Grand Central, New York, NY.

Investors interested in arranging a meeting with the Company’s management during the conference should contact the Investor Summit conference coordinator. A webcast of the presentation will be available under the IR Events tab of the Tonix website at www.tonixpharma.com.

About Tonix Pharmaceuticals Holding Corp.
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.

1TNX-1500 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
2TNX-2900 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
3TNX-801 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
4TNX-1840 and TNX-1850 are investigational new biologics at the pre-IND stage of development and have not been approved for any indication. 
5TNX-3500 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
6TNX-102 SL is an investigational new drug and has not been approved for any indication.
7TNX-1300 is an investigational new biologic and has not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 799-8599

Olipriya Das, Ph.D. (media)
Russo Partners
Olipriya.Das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Release – ProMIS Neurosciences Reports New Milestones in Potential Therapeutic Approaches for Amyotrophic Lateral Sclerosis ALS



ProMIS Neurosciences Reports New Milestones in Potential Therapeutic Approaches for Amyotrophic Lateral Sclerosis (ALS)

News and Market Data on ProMIS Neurosciences

 

TORONTO, Ontario and CAMBRIDGE, MA, April 28, 2022 (GLOBE NEWSWIRE) — ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of potential therapeutics targeting misfolded proteins such as toxic oligomers implicated in the development of neurodegenerative diseases, announced today new milestones in potential therapeutic approaches for ALS.

Almost all cases of ALS, and about half of cases of the related disease frontotemporal degeneration (FTD), feature intracellular aggregates of the protein TDP-43 in the brain and spinal cord. Although normal TDP-43 protein is critical for the survival of neurons, misfolded aggregates of TDP-43 possess many neurotoxic activities and are believed to be a driver of disease. Using its discovery platform, ProMIS generated high-affinity monoclonal antibodies that are selective for the misfolded, toxic form of TDP-43 and has nominated monoclonal antibody PMN267 as the lead candidate based on its binding profile and activity in cell systems. Recent data generated by two independent sources have now provided additional support for the therapeutic potential of PMN267.

Dr. Gene Yeo’s laboratory at the University of California San Diego has shown that an “intrabody” version of PMN267 delivered inside cells via a gene therapy vector significantly reduced the amount of misfolded TDP-43 aggregates in human motor neurons derived from ALS patients, the cell type predominantly affected in ALS.

In an aggressive mouse model of ALS/FTD conducted at a contract research organization, testing of PMN267 as an injectable antibody treatment also produced evidence for protection against disability. These results are in line with reports indicating that antibodies with effector function can be taken up inside neurons and trigger degradation of their target, in this case toxic TDP-43 aggregates.

ProMIS CSO Dr. Neil Cashman was pleased by the results, saying “these are encouraging findings that support the activity of PMN267 as a conventional antibody and as an intrabody constructed from PMN267”. Dr. Larry Altstiel, ProMIS’ CMO, said “this is promising work to be moved forward as rapidly as possible to address the tragic human disease ALS.”

ProMIS also notes the progress made in targeting another ALS/FTD target called RACK1 (receptor for activated C kinase 1). ProMIS’ encouraging preclinical data implicating RACK1 in ALS were presented recently as a poster at the American Academy of Neurology in Seattle, entitled “RACK1 Knockdown Alleviates TDP-43-Associated Global Translational Suppression in vitro and Neurodegeneration in vivo.”

About ProMIS Neurosciences
ProMIS Neurosciences, Inc. is a development stage biotechnology company focused on discovering and developing therapeutics selectively targeting toxic misfolded oligomers implicated in the development and progression of neurodegenerative diseases, in particular Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD). The Company’s proprietary target discovery engine is based on the use of two complementary computational modeling techniques. The Company applies its molecular dynamics, computational discovery platform -ProMIS™ and Collective Coordinates – to predict novel targets known as Disease Specific Epitopes on the molecular surface of misfolded proteins. ProMIS is headquartered in Toronto, Ontario, with offices in Cambridge, Massachusetts. ProMIS is listed on the Toronto Stock Exchange under the symbol PMN, and on the OTCQB Venture Market under the symbol ARFXF

To learn more, visit us at www.promisneurosciences.com, follow us on Twitter and LinkedIn

For Investor Relations please contact:
Alpine Equity Advisors
Nicholas Rigopulos, President
nick@alpineequityadv.com
Tel. 617 901-0785

The TSX has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This information release contains certain forward-looking information. Such information involves known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements to be materially different from those implied by statements herein, and therefore these statements should not be read as guarantees of future performance or results. All forward-looking statements are based on the Company’s current beliefs as well as assumptions made by and information currently available to it as well as other factors. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Due to risks and uncertainties, including the risks and uncertainties identified by the Company in its public securities filings, actual events may differ materially from current expectations. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Source: ProMIS Neurosciences Inc.

Release – electroCore to Announce First Quarter 2022 Financial Results on May 5



electroCore to Announce First Quarter 2022 Financial Results on May 5

News and Market Data on electroCore

 

ROCKAWAY, N.J.
April 28, 2022 (GLOBE NEWSWIRE) — 
electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, announced today that it will report financial results for the first quarter ended 
March 31, 2022, after the close of the market on 
Thursday, May 5, 2022. Management will host a conference call and webcast at 
4:30 PM EDT to discuss the financial results and answer questions.

Thursday, May 5, 2022, 4:30 PM EDT
Domestic: 877-269-7756
International: 201-689-7817
Conference ID: 13728177
Webcast: electroCore Earnings Webcast

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.

Contact:
Rich Cockrell

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

Release – PDS Biotechnology Announces Conference Call and Webcast for First Quarter 2022 Financial Results



PDS Biotechnology Announces Conference Call and Webcast for First Quarter 2022 Financial Results

Research, News, and Market Data on PDS Biotech

 

FLORHAM PARK, N.J., April 28, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing molecularly targeted cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technologies, today announced that the Company will release financial results for the first quarter of 2022 on Wednesday, May 11, 2022, before the market opens. Following the release, management will host a conference call to review the financial results and provide a business update.

Wednesday, May 11, 2022, 8:00 AM EDT
Domestic: 877-407-3088
International: 201-389-0927
Conference ID: 13728184
Webcast: PDS Biotech Earnings Webcast

After the live webcast, the event will be archived on PDS Biotech’s website for six months.

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

Our Versamune®-based molecularly targeted 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.

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

Baudax Bio (BXRX) – NobleCon 18 Presentation

Wednesday, April 27, 2022

Baudax Bio (BXRX)
NobleCon 18 Presentation

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, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

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

    NobleCon 18. Gerri Henwood, President and Chief Executive Officer, presented at the conference. The presentation highlighted continued ANJESO growth, reduced cash burn and pipeline progress of the Company’s Neuromuscular Blockers (NMBs) and NMB Reversal Agents. A replay of the presentation can be found here.

    ANJESO is growing.  Growth has been strong as Baudax Bio continues to win formularies, not only in regional hospitals and ambulatory surgery centers (ASCs), but also integrated delivery networks (IDNs) and national ASC groups. Highly positive clinical experience has helped account penetration …


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. 

 

BioSig Technologies (BSGM) – NobleCon 18 Presentation

Wednesday, April 27, 2022

BioSig Technologies (BSGM)
NobleCon 18 Presentation

BioSig Technologies, Inc. is a medical technology company commercializing a proprietary biomedical signal processing platform designed to improve the electrophysiology (EP) marketplace. PURE EP is a computerized system designed to reveal the full range of cardiac signals and to provide physicians with signal clarity during procedures performed to address cardiac arrhythmias. The PURE EP System has received FDA 510(k) clearance and installed its first commercial sale in February 2021. The company looks to apply their unique bioelectronic technology across additional disease conditions, including nervous system disorders, auto-immune diseases, hypertension, and pain.

Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

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

    NobleCon 18. Kenneth Londoner, Chairman and CEO, presented at the conference. The presentation highlighted the PURE EP market opportunity, growth in procedure usage, and commercialization strategy. A presentation replay can be found here.

    Expanding usage.  PURE EP has now been used in over 2200 procedures at 17 sites, showing sequential quarterly growth every quarter. This is a strong indication of demand and need for the technology, despite some pandemic headwinds. AFib is the fastest growing sector in cardiology, with one in four over the age of 60 having AFib, so need should only increase …


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. 

 

Release – Ayala Pharmaceuticals Announces Poster Presentation on AL101



Ayala Pharmaceuticals Announces Poster Presentation on AL101 at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting

Research, News, and Market Data on Ayala Pharmaceuticals

 

REHOVOT, Israel and WILMINGTON, Del., April 27, 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 and aggressive cancers, primarily in genetically defined patient populations, today announced that it will present a poster featuring data on AL101 in adenoid cystic carcinoma (ACC) at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, to take place June 3-7, 2022 in Chicago, Illinois.

Details of the poster presentation are as follows:
 
Abstract Title: Results of ACCURACY: A phase 2 trial of AL101, a selective gamma
secretase inhibitor, in subjects with recurrent/metastatic (R/M) adenoid
cystic carcinoma (ACC) harboring Notch activating mutations (Notchmut)
Abstract Number: 6046
Session Title: Head and Neck Cancer
Session Date and Time: Monday, June 6, 2022, 1:15 PM-4:15 PM CDT

The ongoing Phase 2 ACCURACY clinical trial is an open-label, single-arm, multi-center study to assess the clinical activity of AL101 using radiographic assessments of patients with R/M ACC demonstrating disease progression within 6 months prior to dosing. The company will present safety, efficacy, PK and PD data from the 4mg and 6mg AL101 cohorts in the trial.

About Adenoid Cystic Carcinoma (ACC)

ACC is a rare malignancy of the secretory glands including salivary glands, accounting for about 10% of all salivary gland tumors with an annual incidence of 3,400 in the U.S. There is currently no approved standard of care for patients with recurrent/metastatic ACC. Patients with locoregional disease undergo surgery and radiation therapy, with recurring disease treated by chemotherapy. ACC is an immunologically “cold” tumor that is refractory to chemotherapy, with a recurrence rate of about 60% after initial surgery. The Notch pathway has been determined to be an oncogenic driver of ACC and its dysregulation plays a key role in tumorigenesis and correlates with a distinct pattern of metastasis and a poor prognosis.

About AL101

AL101 is an investigational small molecule Gamma Secretase Inhibitor (GSI) that is designed to potently and selectively inhibit Notch 1, 2, 3 and 4, and is currently being evaluated in the Phase 2 ACCURACY clinical studies in patients with adenoid cystic carcinoma (ACC). AL101 is designed to inhibit the expression of Notch gene targets by blocking the final cleavage step by the gamma secretase required for Notch activation. Ayala obtained an exclusive, worldwide license to develop and commercialize AL101 from Bristol-Myers Squibb Company in November 2017. AL101 was granted U.S. FDA Fast Track Designation and Orphan Drug Designation for the treatment of ACC.

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 and aggressive cancers, primarily in genetically defined patient populations. 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. 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.

Release – Onconova Therapeutics Announces Acceptance Of Abstract For Publication At The ASCO Annual Meeting



Onconova Therapeutics Announces Acceptance Of Abstract For Publication At The ASCO Annual Meeting

News and Market Data on Onconova Therapeutics

 

NEWTOWN, Pa., April 27, 2022 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced the acceptance of an abstract for publication at the American Society of Clinical Oncology (ASCO) Annual Meeting.

Details on the abstract are provided below.

Abstract Title: Narazaciclib’s kinase inhibitory activity is differentiated from approved CDK4/6 inhibitors in preclinical models

Abstract Number: e15096

Publication Date and Time: May 26, 2022, at 5:00 p.m. ET

About Onconova Therapeutics

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China.

Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.

For more information, please visit www.onconova.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the timing of Onconova’s and investigator-initiated clinical development and data presentation plans, and the mechanisms and indications for Onconova’s product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680
ir@onconova.us
https://www.onconova.com/contact

Investor Contact:
Bruce Mackle
LifeSci Advisors
929-469-3859
bmackle@lifesciadvisors.com

Lineage Cell Therapeutics (LCTX) – New Program Added To The Development Pipeline

Tuesday, April 26, 2022

Lineage Cell Therapeutics (LCTX)
New Program Added To The Development Pipeline

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences, Noble Capital Markets, Inc.

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

    Photoreceptor Development Program Announced.  Lineage Cell announced a new program to develop photoreceptor cells for transplantation. This is an additional application of Lineage’s proprietary cell-based technology for growing pluripotent cells into differentiated cells that can be transplanted to repair areas where cells have been lost to disease. The new program will develop cell transplants for the rod and cone photoreceptor cells in the eye.

    Photoreceptor Program Fits With OpRegen.  Lineage Cell’s most advanced product is OpRegen, a retinal pigmented epithelial (RPE) cell transplant therapy for age-related macular degeneration (dry AMD). The new photoreceptor program will develop cells to repair a different type of cell in the retina. The photoreceptor cell transplants have potential to address conditions such as retinitis pigmentosa …


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. 

 

Release – Lineage to Present at B. Riley Securities 2022 Neuro & Ophthalmology Virtual Investor Conference on April 27 2022

 



Lineage to Present at B. Riley Securities 2022 Neuro & Ophthalmology Virtual Investor Conference on April 27, 2022

Research, News, and Market Data on Lineage Cell Therapeutics

 

Fireside Chat with B. Riley Research Analyst Scheduled for 3:30pm Eastern Time

Topics Will Include Two New Cell Therapy Programs Launched Following Roche and Genentech Partnership for RG6501 (OpRegen®) Program

CARLSBAD, Calif.–(BUSINESS WIRE)–Apr. 26, 2022– 

Lineage Cell Therapeutics, Inc.
 (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced today  Brian M. Culley, Lineage’s Chief Executive Officer, will present at the B. Riley Securities 2022 Neuro & Ophthalmology Virtual Investor Conference, in a fireside chat hosted by  Mayank Mamtani, Managing Director, Senior Biotech Research Analyst and Group Head of Healthcare Research at 
B. Riley Securities, on 
Wednesday April 27th, 2022 at 
3:30pm EST.

Interested parties can register to view both the live event and replay on the Events and Presentations section of Lineage’s website. Additional videos are available on the Media page of the Lineage website.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, which is now being developed under a worldwide collaboration with Roche and
Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Mike Biega
(Mbiega@soleburytrout.com)
(617) 221-9660

|Russo Partners – Media Relations
Nic Johnson or  David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Source: 
Lineage Cell Therapeutics, Inc.

Release – electroCore Completes Sale of New Jersey Tax Benefits



electroCore Completes Sale of New Jersey Tax Benefits

News and Market Data on electroCore

 

ROCKAWAY, N.J.
April 26, 2022 (GLOBE NEWSWIRE) — 
electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, today announced that it has completed the sale of its available tax benefits through the New Jersey Economic Development Authority’s Technology Business Tax Certificate Transfer program for fiscal year 2021. As a result, the Company has received approximately 
$445,000 in non-dilutive cash from the sale of these net operating loss (NOL) tax benefits.

“We are pleased to have received 
$445,000 from the New Jersey NOL program,” commented  Brian Posner, Chief Financial Officer of electroCore. “This is the third consecutive year that we have benefited from this program which provides us non-dilutive funding that will be beneficial to us as we continue to invest in our sales channels and marketing initiatives to expand consumer awareness of gammaCore.”

The New Jersey Technology Business Tax Certificate Transfer program enables qualified, unprofitable NJ-based technology or biotechnology companies with fewer than 225 US employees (including parent company and all subsidiaries) to sell a percentage of their NOL and research and development tax credits to unrelated profitable corporations. NOLs may be sold for at least 80 percent of their value, up to a maximum lifetime benefit of 
$20 million per business. This allows qualifying technology and biotechnology companies with NOLs to turn tax losses and credits into cash proceeds to fund their growth and operations, including research and development or other allowable expenditures.

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 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 is 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 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 regarding electroCore’s business prospects, its sales and marketing and product development plans, future cash flow projections, anticipated costs, its product portfolio or potential markets for its technologies, the availability and impact of payor coverage, the potential of nVNS generally and gammaCore in particular to treat COVID-19, and other statements that are not historical in nature, particularly those using terminology such as “anticipates,” “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 obtain additional financing necessary to continue electroCore’s business, sales and marketing and product development plans, the uncertainties inherent in the development of new products or technologies, the ability to successfully commercialize gammaCore™, competition in the industry in which electroCore operates and general market conditions. All forward-looking statements are made as of the date of this press release, and electroCore undertakes no obligation to update 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 refer to all information set forth in this document and should also refer to the disclosure of risk factors set forth in the reports and other documents electroCore files with the
SEC, available at www.sec.gov.

Contact:
Rich Cockrell

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

PTSD MDMA and Psilocybin


Image Credit: Peter Murphy (Flickr)


Latest Trials Confirm the Benefits of MDMA – the Drug in Ecstasy – for Treating PTSD

 

For people with post-traumatic stress disorder, recalling memories of physical or sexual assault, combat or disaster-related events can induce intense anxiety or panic attacks as well as debilitating flashbacks.

In the U.S., about 7% of people suffer from PTSD and lose an average of about four working days each month as a result. Trauma-specific psychotherapy, like cognitive processing or “talk” therapy, is the cornerstone of treatment for PTSD. But for approximately half of people, these traditional approaches are ineffective at fully addressing PTSD symptoms over the long term. Antidepressant drugs are frequently used if psychotherapy fails, or in combination with it, but the effects are usually modest.

MDMA (3,4-methylenedioxymethamphetamine) is an active ingredient in the illicit street drug known as ecstasy or molly. People in dance clubs and raves use illicit MDMA because it elevates mood and energy levels, induces a feeling of bonding with others and produces a surreal psychedelic effect. These same effects have been hypothesized to support people with PTSD during psychotherapy sessions, since they can make people more willing and able to share and explore their traumatic experiences. Our new meta-analysis of clinical trials confirms the benefits of MDMA-assisted psychotherapy in the treatment of PTSD.

 

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It was written by and represents the research-based opinions of C. Michael White, Distinguished Professor and Head of the Department of Pharmacy Practice, University of Connecticut. Adrian V. Hernandez, Associate Professor of Comparative Effectiveness and Outcomes Research, University of Connecticut.

 

We are a pharmacist and physician team who investigate the benefits and harms associated with substances of abuse like bath salts, phenibut, cannabis and synthetic marijuana. Through this work we have become intrigued about the therapeutic potential for some psychedelic drugs in the treatment of myriad psychiatric disorders, from PTSD to major depression, especially MDMA and psilocybin (hallucinogenic mushrooms).

It is important to state that using ecstasy or molly products from the street would not help PTSD symptoms because the MDMA needs be used along with carefully crafted psychotherapy in a safe, controlled environment. Ecstasy or molly products purchased illicitly never specify the exact amount of MDMA they contain, so it is impossible to dose it properly for PTSD. Taking too much MDMA or exercising while taking MDMA can cause heart attacks, strokes, seizures and arrhythmias and can damage muscles and kidneys.

 

What is MDMA-Assisted Psychotherapy?

In an MDMA-assisted psychotherapy session, patients take MDMA as a pill upon entering a psychiatrist’s office and then work with a team of therapists who help them divulge traumatic events or discuss aspects of those events over the course of several hours. They usually have non-MDMA sessions before the first MDMA session so they know what to expect. And they have at least one non-MDMA session after each MDMA one to work through the traumatic memories that were revealed and to learn coping strategies. A standard treatment course includes two or three multi-hour MDMA-assisted psychotherapy sessions and several non-MDMA sessions.

The MDMA products used in these sessions are pharmaceutical grade. This means that, unlike illicitly obtained street products, they do not contain other substances of abuse, such as methamphetamine, or contaminants like heavy metals, bacteria or mold. People with hypertension or those at high risk of heart attacks, strokes or arrhythmias should not participate, because they can have unsafe elevations in blood pressure and heart rate. In addition, patients are not allowed to leave for eight hours, until the effects of MDMA have fully worn off.


Assessing the Effectiveness of MDMA-Assisted Psychotherapy

In 2014, we reviewed the available animal data and the few preliminary human studies of MDMA-assisted psychotherapy, but at the time, higher-quality clinical trials had not yet been completed. But in the past few years, larger and higher-quality trials have been published, warranting an in-depth assessment.

So we recently reviewed the data comparing antidepressant use to placebos for patients with PTSD and performed a meta-analysis study of the six different clinical trials that assessed the usefulness of MDMA-assisted psychotherapy versus psychotherapy alone. All of the trials we analyzed included both men and women who had experienced a multitude of traumatic events that led to PTSD. The studies used the same points scale to determine the effectiveness of therapy, making it easier to compare data across studies. Scores above approximately 50 points mean a patient has severe PTSD, and scores reduced by more than 10 points from baseline are clinically meaningful.

We found that daily antidepressant therapy reduced PTSD by 6 to 14 points compared with the placebo, but a range of 27% to 47% of patients across the studies withdrew before the end of the trials. In contrast, MDMA-assisted psychotherapy reduced the scores by 22 points compared with those receiving psychotherapy with placebo, and patients were twice as likely to no longer meet the criteria for PTSD diagnosis by the end of the trials. In addition, only 8% of patients withdrew from MDMA-assisted psychotherapy trials. The main adverse effects included teeth grinding, jitteriness, headache and nausea. One of these MDMA trials found that participants’ blood pressure and heart rate were elevated in the course of MDMA therapy, but not to a concerning extent.

For several of the trials in our meta-analysis, investigators sent a questionnaire to participants 12 months after their last MDMA session to assess the long-term impact. Overall, 86% of participants said they received substantial benefits from the combined MDMA-assisted psychotherapy. Eighty-four percent of participants reported having improved feelings of well-being, 71% had fewer nightmares, 69% had less anxiety and 66% had improved sleep. The results from across all of the studies suggested that MDMA-assisted therapy was helping to alleviate the PTSD itself, not simply suppressing symptoms.

 

Looking Ahead

The U.S. Drug Enforcement Administration identifies MDMA and psilocybin as Schedule I controlled substances. According to the DEA, these substances have no currently accepted medical use in the U.S. and come with high abuse potential.

However, it’s worth noting an important exception. Cannabadiol, or CBD, a chemical that comes from the plant Cannabis sativa, is classified as a Schedule I drug. But the Food and Drug Administration approved its use in 2018 for the treatment of two rare and severe childhood seizure disorders. That doesn’t mean that the CBD in your lotion or seltzer has proof of benefit for most of the ills people are using it for, but its full therapeutic potential is still being explored. Given the strong consistent beneficial effects and manageable adverse events in the newer trials designed with FDA input, we suspect that MDMA-assisted psychotherapy will become an FDA-approved option for PTSD by the end of 2023. Psilocybin – commonly known as hallucinogenic mushrooms – also shows promise for treating major depression, but further research is needed.

The DEA’s stringent policies made it exceptionally hard for scientists to conduct research on Schedule I drugs for decades by criminalizing the possession of the products, even in research settings. But in 2018, the agency streamlined the application process for securing a waiver for research purposes. This made it easier for researchers to conduct trials into the pharmaceutical value of psychedelic drugs. Within the next decade, this shift will almost certainly accelerate the discovery of new treatments for patients suffering from mental illness.

 

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