Independent Researchers Find Genprex’s TUSC2 May Be a Novel Target and Biomarker for Thyroid Cancer Therapy

Source:  Genprex, Inc. 1/29/2020

• Study results show TUSC2 overexpression decreased thyroid cancer tumor metastasis and increased sensitivity to apoptosis by increasing SMAC/DIABLO and Cytochrome C protein levels
• Study highlights the possibility that TUSC2 may also be effective in thyroid cancer therapy and adds to the growing body of research on TUSC2 beyond the non-small cell lung cancer indication the Company is initially pursuing
• TUSC2 is the active agent in the Company’s Oncoprex™ immunogene therapy

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, announced that independent researchers reported in a recent study that TUSC2, a tumor suppressor gene and the active agent in Genprex’s Oncoprex™ immunogene therapy, is a potential target and biomarker for thyroid carcinoma. Genprex has no affiliation with these researchers.

Published in the International Journal of Molecular Sciences, the study reports that TUSC2 overexpression decreased thyroid cancer proliferation, migration and invasion. Cell proliferation, migration and invasion ability are essential steps in tumor metastasis. TUSC2 forced expression reduced thyroid cancer cell proliferation and could represent an important tool to arrest cancer cell proliferation, while TUSC2 restoration decreased the migration and invasion of thyroid cancer cell lines. 

The study also found that TUSC2 increased sensitivity to apoptosis by increasing the SMAC/DIABLO and Cytochrome C proteins, which play major roles in apoptosis. TUSC2 forced expression increased these protein levels, and, inversely, the silencing of TUSC2 induced resistance to apoptosis.

Based on the results of the study, researchers concluded that TUSC2 is negatively associated with thyroid cancer aggressiveness and, thus could be a novel target and biomarker for thyroid cancer therapy.

“We continue to be encouraged by data resulting from studies conducted at multiple research institutions suggesting that TUSC2 may be an effective treatment for many types of cancer, now including thyroid cancer,” said Rodney Varner, Genprex’s Chairman and Chief Executive Officer.

The authors further state that thyroid carcinoma is the most common endocrine cancer and includes many different forms. Anaplastic thyroid carcinoma (ATC) is the rarest but most lethal subtype. ATC patients usually present a rapidly enlarging neck mass, a high rate of distant metastases and approximately 95 percent mortality at six months. Conversely, papillary thyroid carcinoma (PTC), the most common type of thyroid cancer, is generally characterized by good outcomes, as it is highly curable by surgery and radioiodine therapy. However, some PTC patients have an aggressive disease and can develop distant metastasis.

The same researchers have previously reported that TUSC2 is downregulated in almost all ATC samples and in the vast majority of PTC samples, suggesting TUSC2’s important role in thyroid cancer progression. In 2019, an estimated 50,000 patients in the U.S. were diagnosed with thyroid cancer.

Genprex is conducting clinical and pre-clinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing pre-clinical data also suggest that TUSC2 may be effective against breast cancer, glioblastoma, head and neck cancer, kidney cancer, and soft tissue sarcomas. This new independent study raises the possibility that TUSC2 may also be used to treat thyroid cancer.

About Genprex, Inc.
Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients based upon a unique proprietary technology platform. Genprex’s platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The company’s lead product candidate, Oncoprex™ immunogene therapy for non-small cell lung cancer (NSCLC), has a multimodal mechanism of action whereby it has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells, re-establish pathways for apoptosis, or programmed cell death, in cancer cells, and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the FDA granted Fast Track Designation for Oncoprex™ imunogene therapy for NSCLC in combination therapy with osimertinib (AstraZeneca’s Tagrisso®). For more information, please visit the company’s web site at www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Genprex, Inc. Announces Closing of $8 Million At-The-Market Common Stock Offering

Source:  Genprex, Inc. 1/28/2020

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, announced today that it closed its previously announced common stock offering priced at-the-market under Nasdaq rules. The Company sold an aggregate of 7,620,000 shares of its common stock at a price of $1.05 per share for gross proceeds to the Company of $8 million, before deducting commissions and estimated offering expenses. There were no warrants issued in the offering.

A.G.P./Alliance Global Partners acted as the lead placement agent for the offering, and Joseph Gunnar & Co., LLC acted as co-placement agent for the offering.

The Company intends to use the net proceeds from the offering to advance its lead clinical programs in non-small cell lung cancer (NSCLC) and for working capital and general corporate purposes.

“The successful closing of this transaction is further evidence that our corporate vision and proprietary technology in the gene therapy cancer treatment market are gaining traction with institutional investors,” commented Rodney Varner, Chief Executive Officer at Genprex. “This offering significantly improves our balance sheet and allows us the financial flexibility to further develop our pipeline and advance our clinical trials in NSCLC for our lead drug candidate, Oncoprex, in combination with already approved lung cancer therapies. We are excited to generate additional clinical data in NSCLC from Oncoprex’s novel mechanisms of action.”

The securities were offered pursuant to an effective shelf registration statement on Form S-3 (File No. 333-233774) previously filed and declared effective by the U.S. Securities and Exchange Commission (the “SEC”) on October 28, 2019.

A prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available for free on the SEC's website at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained from either A.G.P./Alliance Global Partners, 590 Madison Avenue, 36th Floor, New York, New York 10022 or by email at prospectus@allianceg.com or Joseph Gunnar & Co. LLC, 30 Broad Street, 11th Floor, New York, New York 10004 or by email at investmentbanking@jgunnar.com.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities described herein, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Genprex, Inc.
Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients based upon a unique proprietary technology platform. Genprex’s platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The company’s lead product candidate, Oncoprex™ immunogene therapy for non-small cell lung cancer (NSCLC), has a multimodal mechanism of action whereby it has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells, re-establish pathways for apoptosis, or programmed cell death, in cancer cells, and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the FDA granted Fast Track Designation for Oncoprex™ imunogene therapy for NSCLC in combination therapy with AstraZeneca’s Tagrisso®. For more information, please visit the company’s web site at www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Genprex Receives U.S. FDA Fast Track Designation for Gene Therapy that Targets Lung Cancer

Source:  Genprex, Inc. 1/21/2020

Lung cancer is the world’s leading cause of cancer deaths

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, today announced that the U.S Food and Drug Administration (FDA) has granted Fast Track Designation for Genprex’s Oncoprex™ immunogene therapy in combination with EGFR inhibitor osimertinib (AstraZeneca’s Tagrisso®, which had worldwide sales in 2018 of $1.86 billion, $2.31 billion in the first 9 months of 2019 and is currently AstraZeneca’s highest grossing product) for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone. Oncoprex is comprised of the TUSC2 (Tumor Suppressor Candidate 2) gene complexed with a lipid nanoparticle. TUSC2 is the active agent in Oncoprex.

Genprex has treated more than 50 lung cancer patients with Oncoprex in Phase I and II clinical trials. The company believes the data from these trials are encouraging as to both safety and efficacy. 

“Genprex is excited to receive this important FDA designation,” said Rodney Varner, Chairman and Chief Executive Officer of Genprex. “In addition to potentially facilitating and expediting our pathway to approval, we believe that this FDA designation validates our plan to commercialize Oncoprex immunogene therapy in combination with EGFR inhibitors for the treatment of lung cancer. We hope that Fast Track Designation helps us bring our gene therapy to patients more rapidly and that our unique gene therapy platform is more widely recognized for its potential in cancer treatment.”

FDA may award Fast Track Designation if it determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This provision is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. 

Fast Track drug candidates must show advantages over available therapies, such as superior effectiveness, avoiding serious side effects, improving diagnosis and outcome, decreasing significant toxicity, and the ability to address public health needs.

Fast Track Designation recipients may also be eligible for accelerated approval or rolling review of the recipient’s Biologics License Application (BLA). In addition, Fast Track product candidates could be eligible for priority review if supported by clinical data at the time of BLA submission.

The initial disease indication for Oncoprex is NSCLC. Lung cancer is the world’s leading cause of cancer death, taking more lives each year than colon, breast and prostate cancers combined. Each year, there are more than 2 million new lung cancer cases and 1.7 million deaths from lung cancer worldwide. In the United States, there are more than 228,000 new cases of lung cancer and more than 142,000 deaths from lung cancer each year. NSCLC represents 84 percent of all lung cancers, and the five-year relative survival rate for metastatic lung cancer is less than 5 percent.

Genprex is preparing to initiate a Phase I/II clinical trial evaluating Oncoprex in combination with osimertinib, as well as a new Phase I clinical trial evaluating Oncoprex in combination with a checkpoint inhibitor.

For more information on the U.S. FDA’s Fast Track Designation, please visit the FDA’s Fast Track webpage.

About Genprex, Inc.
Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients based upon a unique proprietary technology platform. Genprex’s platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The company’s lead product candidate, Oncoprex™ immunogene therapy for non-small cell lung cancer (NSCLC), has a multimodal mechanism of action whereby it has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells, re-establish pathways for apoptosis, or programmed cell death, in cancer cells, and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. For more information, please visit the company’s web site at www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Genprex Announces $1.26 Million Registered Direct Offering

Source:  Genprex, Inc. 11/20/19

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology platform, announced today a registered direct offering of 3,167,986 shares of its common stock at a price to the public of $0.40 per share, for gross proceeds of approximately $1.26 million prior to deduction of commissions and offering expenses payable by Genprex. In a concurrent private placement, the Company agreed to issue to the investors in the registered direct offering unregistered warrants to purchase up to 3,167,986 shares of the Company’s common stock.

Joseph Gunnar & Co. is acting as the exclusive placement agent.

The warrants will be exercisable 6 months from the issuance date, have an exercise price of $0.46 per share, and will expire 5 years from such date. The warrants will be exercisable for 100% of shares of common stock purchased by each investor in the registered direct offering. In addition, the Company has agreed to reduce the exercise price of an aggregate of 2,283,740 warrants held by the purchasers in the registered direct offering to $0.46, which warrants will not be exercisable for six months from the closing of the registered directed offering and the expiration date of the warrants will be extended by six months to January 27, 2024.

The Company intends to use the net proceeds of the offering for working capital and general corporate purposes. The closing of the offering is expected to take place on or about November 25, 2019, subject to the satisfaction or waiver of customary closing conditions.

The shares of common stock described above (but not the warrants or the shares of common stock underlying the warrants) are being offered pursuant to a "shelf" registration statement on Form S-3 (File No. 333-233774) that was filed by the Company with the Securities and Exchange Commission (SEC) and was declared effective on October 28, 2019. The Company will file a prospectus supplement with the SEC relating to such shares of common stock. Copies of the prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering may be obtained, when available, from Joseph Gunnar & Co., 30 Broad Street, 11th Floor, New York, NY 10004, or by email at investmentbanking@jgunnar.com. In connection with the private placement, the Company has agreed to a file a registration statement registering for resale the shares of common stock issuable upon exercise of the warrants issued in the private placement within 45 days of the closing of the private placement.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Genprex, Inc.
Genprex, Inc. is a clinical stage gene therapy company developing potentially life-changing technologies for cancer patients based upon a unique proprietary technology platform. Genprex’s platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The company’s lead product candidate, Oncoprex™ immunogene therapy for non-small cell lung cancer (NSCLC), has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. For more information, please visit the company’s web site at www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Genprex Reports Positive Preclinical Data for the Treatment of Some of the Most Resistant Metastatic Lung Cancers

Source:  Genprex, Inc. 11/19/19

• Company’s TUSC2 gene therapy found to increase effectiveness of anti-PD1 immunotherapy and anti-PD1 immunotherapy combined with platinum chemotherapy in humanized mouse model
• TUSC2 in combination with anti-PD1 and chemotherapy resulted in complete eradication of anti-PD1 resistant lung metastases
• Company’s Oncoprex™ immunogene therapy may improve on first-line standard of care for lung cancer 

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company, reported that its collaborators from The University of Texas MD Anderson Cancer Center (“MD Anderson”) presented positive preclinical data for the combination of TUSC2 immunogene therapy with an anti-PD1 antibody, pembrolizumab, and for the combination of TUSC2 immunogene therapy, pembrolizumab, and chemotherapy for the treatment of some of the most resistant metastatic lung cancers, including the KRAS and LKB1 mutations, at the American Association of Cancer Research Tumor Immunology and Immunotherapy Meeting 2019. The TUSC2 gene is a tumor suppressor gene and is the active agent in Genprex’s Oncoprex™ immunogene therapy. 

The poster, entitled “Efficacy of Novel Immunogene-Combinations for KRAS and LKB1 mutant NSCLC in a Humanized Mouse Model” shows that TUSC2 confers sensitivity to checkpoint blockade for some of the most resistant metastatic human cancers, including the KRAS and LKB1 mutations, in mice with human immune cells (humanized mice) with lung metastases. When TUSC2 was combined with anti-PD1 therapy, pembrolizumab, in humanized mice with KRAS and LKB1 lung metastases, there was significantly increased antitumor activity than when compared to either agent alone. This combination and model also demonstrated TUSC2-related NK (Natural Killer) cell activation. A significantly higher percentage of CD56⁺ NK and CD56⁺CD59⁺ active NK cells, which are immune cells that have been activated to kill cancer cells, were found in the mice that received TUSC2 alone and in those that received the combination of TUSC2 and pembrolizumab than in those that received pembrolizumab alone.  

The poster also shows that TUSC2 increases the effectiveness of anti-PD1 checkpoint blockade combined with platinum chemotherapy in humanized mice with lung metastases with KRAS and LKB1 mutations, thus demonstrating that TUSC2 may improve on first-line standard of care for lung cancer. The combination of TUSC2 with pembrolizumab and carboplatin, a platinum chemotherapy, in humanized mice with KRAS and LKB1 lung metastases resulted in metastasis regression significantly greater than either TUSC2 alone or pembrolizumab combined with carboplatin treatments. This model showed significantly fewer or no visible tumor nodules after treatment with the TUSC2 combination as compared with other groups, and it showed strong antitumor efficacy. The combination of TUSC2 with pembrolizumab and carboplatin resulted in complete eradication of anti-PD1 resistant lung metastases in the humanized mouse model.

“These data not only further support existing preclinical data showing that Oncoprex immunogene therapy is synergistic with anti-PD1 therapy, but they also offer new data demonstrating that Oncoprex improves on the combination of anti-PD1 therapy and chemotherapy, today’s first line standard of care for lung cancer,” said Julien L. Pham, MD, MPH, President and Chief Operating Officer of Genprex. “In a sophisticated humanized mouse model, the combination of TUSC2 with pembrolizumab and carboplatin resulted in complete eradication of anti-PD1 resistant lung metastases in some of the most resistant cancer mutations. This is highly encouraging and provides us with a strong indication that the combination could lead to similar results in the clinic.”  

About Genprex, Inc.
Genprex, Inc. is a clinical stage gene therapy company developing potentially life-changing technologies for cancer patients based upon a unique proprietary technology platform. Genprex’s platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The company’s lead product candidate, Oncoprex™ immunogene therapy for non-small cell lung cancer (NSCLC), has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. For more information, please visit the company’s web site at www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Cyclo Therapeutics Signs Master Services Agreement with Worldwide Clinical Trials

Source:  Cyclo Therapeutics, Inc. 12/27/19

Worldwide will serve as CRO for the Company’s Clinical Programs Evaluating Trappsol^® Cyclo™ for the Treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease

Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that it has signed a Master Services Agreement with Worldwide Clinical Trials (Worldwide), a leading Contract Research Organization (CRO), to serve as CRO for the Company’s clinical programs evaluating Trappsol^® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin formulation, for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease.

The Company currently supports a Phase I clinical trial for NPC in the United States, which recently completed enrollment (ClinicalTrials.gov NCT02939547); an Extension Protocol for the US study, which includes home-based infusions (NCT03893071); and a Phase I/II trial in Europe and Israel for NPC which is nearing completion of enrollment (NCT02912793). The Company will share its design of a pivotal trial in scientific advice meetings with regulators in the US and Europe in first and second quarters of 2020, respectively.

Cyclo Therapeutics is also developing a clinical program to address Alzheimer’s Disease, building on its Expanded Access program for a late onset patient (NCT03624842).

“We are very excited to take this next step in working with Worldwide, an industry leader in the rare disease and neurological disease space,” said N. Scott Fine, Company Chairman and CEO. “Worldwide will play a vital role as we build and execute our clinical programs in NPC and Alzheimer’s Disease.”

Worldwide Clinical Trials’ President and Chief Operating Officer, Peter Benton, said, “The scientific, medical and operational experts at Worldwide are privileged to be associated with Cyclo Therapeutics’ innovative clinical development program seeking to address the significant unmet clinical needs for NPC and Alzheimer’s patients.”

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071), and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com
 
About Worldwide Clinical Trials:
Worldwide Clinical Trials employs more than 1,700 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia, and Asia. Founded by physicians committed to advancing medical science, Worldwide is out to change how the world experiences CROs—in the best possible way. From early phase and bioanalytical sciences through late phase, post-approval and real-world evidence, we provide world-class, full-service drug development services.

With infrastructure and talent spanning 60 countries, we execute predictable, successful studies with operational excellence across a range of therapeutic areas, including central nervous system, cardiovascular, metabolic, general medicine, oncology and rare diseases. We never compromise on science or safety. We’re never satisfied with the status quo. We’re the Cure for the Common CRO.
For more information, visit http://www.worldwide.com.

Cyclo Therapeutics, Inc. to Present Clinical Trial Data on Niemann-Pick type C Disease at the 16th Annual WORLDSymposium

Source:  Cyclo Therapeutics, Inc. 12/16/19

Data show that Trappsol^® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin drug, reduces levels of trapped cholesterol in liver tissue of Niemann-Pick Disease Type C Patients

Cyclo Therapeutics, Inc. (OTCQB:CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced its presentation at the WORLDSymposium (“We’re Organizing Research on Lysosomal Diseases”) to be held in Orlando, Florida, between February 10-13, 2020. The presentation is entitled, “Trappsol^® Cyclo™ hydroxypropyl beta cyclodextrin administered intravenously in patients with Niemann-Pick Disease Type C reduces cholesterol in liver tissue.” The presentation includes data from the Company’s ongoing Phase I clinical trial in the United States to evaluate Trappsol^® Cyclo™ administered intravenously in NPC in subjects 18 years and older (see ClinicalTrials.gov NCT02939547 for study parameters). 

Niemann-Pick Disease Type C is a rare and often fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell. The defect leads to cholesterol accumulation in every cell in the body, causing symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States, and only one, Miglustat/Zavesca, in Europe.

Co-authors of the presentation are the two Co-Principal Investigators of the Phase I trial, Caroline Hastings, MD, UCSF Benioff Children’s Hospital Oakland, and Benny Liu, MD, also affiliated with UCSF Benioff Children’s Hospital Oakland for this trial and the Alameda Health System also in Oakland, joined by Bryan Hurst, MPhil of Boyd Consultants, UK, and Cyclo Therapeutics’ Chief Scientific Officer and Senior Vice President for Medical Affairs Sharon Hrynkow PhD.

The presentation includes liver biopsy data from the first 8 subjects participating in the Phase I trial. Specifically, liver biopsy tissue was stained with a fluorescent marker, filipin, which binds unesterified cholesterol, in liver cells of study subjects both at baseline and after 7 doses of Trappsol^® Cyclo™. Dosing is every two weeks with an approximate 8 hour infusion of the study drug. All subjects receive the study drug at either 1500 mg/kg body weight or 2500 mg/kg body weight. Results show that filipin staining in liver tissue in 5 of 8 subjects had marked reductions in filipin staining after 7 doses of Trappsol^® Cyclo™, 2 had moderate reductions, and 1 had a mild reduction as compared to baseline. These are the first results showing that Trappsol^® Cyclo™, or any hydroxypropyl beta cyclodextrin product, reduces unesterified cholesterol accumulation in liver tissue of NPC patients. These results are consistent with the Company’s previously presented biochemical data showing that Trappsol^® Cyclo™ decreases cholesterol synthesis and increases cholesterol metabolism (WORLDSymposium presentation, 2019). A more complete interpretation of the present data will be possible at study unblinding in early 2020. The presentation abstract is available on the Cyclo Therapeutics’ website at: www.cyclotherapeutics.com
 
The details for Cyclo Therapeutics’ presentation are as follows:
Date: Wednesday, February 12, 2020
Time: 4:30 pm to 6:30 pm/Latebreaking Poster Session; Poster LB-18
Place: Hyatt Regency, Orlando, Florida

In addition to the Phase I trial, Cyclo Therapeutics’ supports a Phase I/II trial in NPC also using Trappsol^® Cyclo™ administered intravenously. The Phase I/II trial is nearing completion of enrollment. Cyclo Therapeutics has submitted a Type C Meeting Request with the FDA and expects to meet with FDA in early 2020 to discuss the proposed development plan for a Phase III pivotal trial using Trappsol^® Cyclo™ intravenously in NPC patients.

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071), and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com

Cyclo Therapeutics Signs Agreement with Worldwide Clinical Trials to Conduct Pivotal Trial in Niemann-Pick Disease Type C

Source:  Cyclo Therapeutics, Inc. 11/8/19

Pivotal trial will study the intravenous administration of the Company’s Trappsol® Cyclo™ drug and will focus on systemic and neurologic outcomes

Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that it has signed an agreement with Worldwide Clinical Trials (Worldwide), a leading Contract Research Organization (CRO), to conduct a pivotal trial that will evaluate the Company’s proprietary hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, administered by intravenous infusion to treat NPC. The Company currently supports a Phase I trial in the United States, which recently completed enrollment (ClinicalTrials.gov NCT02939547); an Extension Protocol for the US study, which includes home-based infusions (NCT03893071); and a Phase I/II trial in Europe and Israel, which is nearing completion of enrollment (NCT02912793). The Company plans to share its design of the pivotal trial in scientific advice meetings with regulators in the US and Europe in first and second quarters of 2020, respectively. Enrollment of the trial is expected to commence shortly thereafter. 

Niemann-Pick Disease Type C is a rare and fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell. Because of the NPC protein defect, cholesterol accumulates abnormally in every cell in the body, causing symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States, and only one, Miglustat/Zavesca, in Europe.

“We are very excited to work with Worldwide, an industry leader in the rare disease space, on this critical clinical program,” said N. Scott Fine, Company Chairman and CEO. “Our early phase trials are showing promising results, as we have reported publicly during the past year. Because of this, we are working quickly to present our pivotal trial design to regulators and hope to commence our pivotal trial in the early part of 2020. Worldwide will play a vital role as we build and execute the pivotal study.”

Worldwide Clinical Trials’ Chief Medical and Scientific Officer, Michael F. Murphy, M.D., Ph.D., said, “The scientific, medical and operational experts at Worldwide are privileged to be associated with Cyclo Therapeutics’ innovative clinical development program seeking to address the significant unmet clinical need represented by NPC. The acumen demonstrated by Cyclo Therapeutics during the process of program and protocol design has been exceptional – greatly facilitating the collaborative research and development effort demanded by orphan diseases with complex and variable phenotypes. Orphan disease research requires a tenacious and uncompromising commitment by all contributors, and commitment and follow through have long been woven into the business fabric at Worldwide.”

A manuscript, published on October 21, 2019 in the Orphanet Journal of Rare Diseases, a scientific, peer-reviewed publication (see https://www.ncbi.nlm.nih.gov/pubmed/31639011), documented the most extensive set of case studies to-date on expanded access use of hydroxypropyl beta cyclodextrin to treat patients with NPC. Eighty percent of the patient use data presented in the manuscript derive from Cyclo Therapeutics’ Trappsol® Cyclo™ product.

Cyclo Therapeutics’ Chief Scientific Officer and Senior Vice President for Medical Affairs, Sharon Hrynkow PhD, the senior author on the manuscript said, “In our review of the data on expanded access use in 12 NPC patients, we found that hydroxypropyl beta cyclodextrin was not only safe when administered intravenously, but also that individual patients showed improvements in disease symptoms, including reduction in the size of the liver, clearance of interstitial lung disease, and neurologic improvements in terms of gait, balance, and ability to focus on tasks. This manuscript’s findings, coupled with initial data from our ongoing NPC clinical trials, are promising indications that Trappsol® Cyclo™ when administered intravenously has benefit as a treatment in NPC.”

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071), and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com.

About Worldwide Clinical Trials:
Worldwide Clinical Trials employs more than 1,700 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia, and Asia. Founded by physicians committed to advancing medical science, Worldwide is out to change how the world experiences CROs—in the best possible way. From early phase and bioanalytical sciences through late phase, post-approval and real-world evidence, we provide world-class, full-service drug development services.

With infrastructure and talent spanning 60 countries, we execute predictable, successful studies with operational excellence across a range of therapeutic areas, including central nervous system, cardiovascular, metabolic, general medicine, oncology and rare diseases. We never compromise on science or safety. We’re never satisfied with the status quo. We’re the Cure for the Common CRO.
For more information, visit http://www.worldwide.com.

Cyclo Therapeutics Announces Its Publication of the Most Extensive Set of Case Study Reports To-Date on Expanded Access Use of Hydroxypropyl Beta Cyclodextrin in Niemann-Pick Disease Type C

Source:  Cyclo Therapeutics, Inc. 10/31/19

Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug

Cyclo Therapeutics, Inc, (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease, today announced its publication of the most extensive set of case studies to-date on expanded access use of hydroxypropyl beta cyclodextrin to treat patients with Niemann-Pick Disease Type C (NPC). The manuscript was published on October 21, 2019 in the Orphanet Journal of Rare Diseases, a scientific, peer-reviewed publication (see https://www.ncbi.nlm.nih.gov/pubmed/31639011). Eighty percent of the patient use data presented in the manuscript derive from Cyclo Therapeutics’ Trappsol^® Cyclo™ product. 

“This manuscript represents years of work and the efforts of many. We are proud that our company’s Founder, C.E. Rick Strattan, worked closely with the Hempel family, Caroline Hastings, MD, and other experts, to develop the expanded access protocol authorized by the FDA in 2009, allowing cyclodextrin use in NPC patients,” said N. Scott Fine, Company Chairman and CEO. “We were equally proud to have supplied our cyclodextrin product to US and Brazilian NPC families as other physicians adapted the expanded access Hempel-Hastings protocol for their own patients. Looking back, we are indebted to these pioneers and to the teams of scientists, especially Dr. Benny Liu, whose seminal work in animal models of NPC formed the foundation for expanded use programs with cyclodextrins in NPC patients. This manuscript is a milestone in NPC.”

Niemann-Pick Disease Type C is a rare and fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell. Because of the NPC protein defect, cholesterol accumulates abnormally in every cell in the body, causing symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States, and only one, Miglustat/Zavesca in Europe.

“In our extensive review of the data on expanded access use in 12 NPC patients, we found that hydroxypropyl beta cyclodextrin was not only safe when administered intravenously, but also that individual patients showed improvements in disease symptoms,” said Dr. Caroline Hastings, lead author on the manuscript. “This included reduction in the size of the liver, clearance of interstitial lung disease, and neurologic improvements in terms of gait, balance, and ability to focus on tasks. As well, we did not see that adding another route of administration by lumbar puncture, or “intrathecal”, added clinical benefit following intravenous administration alone.”

Other highlights of the manuscript are that physicians noted that their patients receiving intravenous cyclodextrin showed increased alertness, improved ability to communicate, and enhanced overall well-being.

Dr. Hastings, a pediatric hematologist oncologist at UCSF Benioff Children’s Hospital in Oakland, CA, is also Co-Principal Investigator of Cyclo Therapeutics’ clinical trial site using Trappsol^® Cyclo™ via intravenous administration for NPC. Co-authors on the manuscript, entitled “Expanded Access with Intravenous hydroxypropyl-beta-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: A case report analysis” are Camilo Vieira, MD (Brazil); Benny Liu, MD (US); Cyrus Bascon, MD (US); Claire Gao, BA (US); Raymond Wang, MD (US); Alicia Casey, MD (US); and Sharon Hrynkow, PhD, Cyclo Therapeutics’ Chief Scientific Officer and Senior Vice President for Medical Affairs.

Dr. Vieira was the first physician in Brazil to use cyclodextrins (also Cyclo Therapeutics’ Trappsol^® Cyclo™ product) in NPC patients, via an expanded access program. Dr. Liu made the seminal discovery in the mouse model of NPC that cyclodextrins could clear cholesterol from the liver and brain, delay onset of symptoms, and prolong life. Dr. Liu is Co-Principal Investigator along with Dr. Hastings at the UCSF Benioff clinical site of Cyclo Therapeutics.

Cyclo Therapeutics’ Dr. Sharon Hrynkow said, “The work described in this manuscript formed the basis of Cyclo Therapeutics’ initial discussions with regulators in the United States, Europe and Israel on the design of our formal clinical trials, now underway. We are deeply grateful to Dr. Hastings and all of the physicians as well as the patients and families who shared their data as we developed these case studies. We look forward to continuing to work in collaboration with families and physicians as Cyclo Therapeutics advances Trappsol^® Cyclo™ on the pathway to market approval for the benefit of NPC patients globally.”
 
About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071) and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com.

Cyclo Therapeutics Signs Agreement with Worldwide Clinical Trials to Conduct Alzheimer’s Disease Clinical Trial

Source:  Cyclo Therapeutics, Inc. 10/30/19

Trial Will Evaluate Safety and Efficacy of the Company’s Trappsol^® Cyclo™ Drug in Alzheimer’s Disease

Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease, today announced that it has signed an agreement with Worldwide Clinical Trials (Worldwide), a leading Contract Research Organization (CRO), to conduct a clinical trial to evaluate safety and efficacy in Alzheimer’s Disease. The trial will evaluate Trappsol^® Cyclo™ given by intravenous infusion. The Company has previously reported encouraging data on safety and efficacy of the product based on an FDA-approved expanded access program in a single patient with late-onset Alzheimer’s Disease. 

Alzheimer’s Disease affects 5.7 million Americans. While a few symptomatic treatments are available, there are no approved disease modifying treatments.

“We are very excited to work with Worldwide, an industry leader, on this critical clinical program,” said N. Scott Fine, Company Chairman and CEO. “Our work to advance an innovative approach to treat this devastating disease may lead to improvements in the lives of Alzheimer’s patients and their families. We are pleased to have Worldwide’s team of experts working closely with us as we begin our development pathway.”

“The scientific, medical and operational experts at Worldwide offer nearly 40 years of hard-won insight gleaned from our experience on the front lines of Alzheimer’s Disease research. The journey toward halting progression of this devastating neurodegenerative disorder takes tenacity, and we’re not about to give up now,” said Henry Riordan, executive vice president, Scientific Solutions, for Worldwide. “We are thrilled to be selected by the innovators at Cyclo Therapeutics as their partner in the development of this exciting new treatment approach.”

Cyclo Therapeutics and Worldwide are designing the trial protocol and expect to schedule a scientific advice meeting with the U.S. FDA in early 2020. Patient enrollment is expected to begin shortly thereafter.

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071) and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com
 
About Worldwide Clinical Trials:
Worldwide Clinical Trials employs more than 1,700 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia, and Asia. Founded by physicians committed to advancing medical science, Worldwide is out to change how the world experiences CROs – in the best possible way. From early phase and bioanalytical sciences through late phase, post-approval and real-world evidence, we provide world-class, full-service drug development services.

With infrastructure and talent spanning 60 countries, we execute predictable, successful studies with operational excellence across a range of therapeutic areas, including central nervous system, cardiovascular, metabolic, general medicine, oncology and rare diseases. We never compromise on science or safety. We’re never satisfied with the status quo. We’re the Cure for the Common CRO.
For more information, visit http://www.worldwide.com.

Cyclo Therapeutics, Inc. Chief Scientific Officer, Sharon H. Hrynkow, Ph.D., Appointed to President’s Council on Science and Technology

Source:  Cyclo Therapeutics, Inc. 10/23/19

White House Announcement made on October 22, 2019

Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of diseases with unmet medical need, including Niemann-Pick Disease Type C and Alzheimer’s Disease, today announced that Sharon H. Hrynkow, Ph.D., its Chief Scientific Officer and Senior Vice President for Medical Affairs, has been appointed to the President’s Council of Advisors on Science and Technology (PCAST). On October 22, President Donald J. Trump signed an executive order re-chartering PCAST and announced the first seven members, including Dr. Hrynkow. 

Dr. Hrynkow, a neuroscientist by training, has served in executive leadership roles at the National Institutes of Health, as the inaugural President of the Global Virus Network, a non-profit organization, and as Senior Science Advisor at the Department of State. Dr. Hrynkow leads the Cyclo Therapeutics, Inc. clinical and scientific programs developing the company’s drug, Trappsol^® Cyclo™, for treatment of Niemann-Pick Disease Type C, a rare and fatal disease in children, and for Alzheimer’s Disease. She is Co-Chair of the Company’s Scientific Advisory Board.

A total of 16 members are expected to be appointed to PCAST. Dr. Kelvin Droegemeier, Director of The White House Office of Science and Technology, will chair the Council. See the full announcement HERE.

“Under this Administration, science and technology in America continues to advance by leaps and bounds. PCAST will be critical to our continued efforts, with each member bringing a unique perspective to the table. By convening a diverse group of our Nation’s foremost leaders across a broad range of fields, we can leverage the full innovation ecosystem, solve some of the Nation’s greatest challenges, and ensure America’s science and technology leadership for generations to come,” said Dr. Droegemeier.

“Advances in science and technology are major drivers of US economic growth and they contribute to our nation’s security,” said Dr. Hrynkow. “I am delighted to serve on PCAST to work with colleagues across many disciplines to find solutions to critical science and technology challenges facing our nation.”

“We are all very proud to see Dr. Hrynkow appointed to PCAST, and we know that she will serve with distinction,” said Company Chairman and CEO, N. Scott Fine.

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071) and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com
 
About PCAST:
PCAST was launched by Executive Order in 2001, formalizing a process that had been in place through many previous Administrations to advise the President on matters involving science, technology, education, and innovation policy. The Council provides the President with scientific and technical information that is needed to inform public policy relating to the American economy, the American worker, national and homeland security, and other topics. The Council includes distinguished individuals from sectors outside of the Federal Government and represents diverse perspectives and expertise in science, technology, education and innovation.

Cyclo Therapeutics Announces Completion of Enrollment in its Phase I Trial to Evaluate Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

Source:  Cyclo Therapeutics, Inc. 10/2/19

Top line results are expected to be available in February 2020

Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it has completed patient enrollment in its Phase I trial to evaluate the safety and tolerability of Trappsol^® Cyclo™ administered intravenously to Niemann-Pick Disease Type C (NPC) patients.

“Today’s ‘Last-Patient-In’ announcement is a significant milestone for our company and the NPC community,” said Chairman and CEO, N. Scott Fine. “It completes another important step in our development and registration strategies for Trappsol^® Cyclo™ to treat NPC, a disease which causes much suffering for the patients and their families. We are delighted to share this news with our many supporters and all of our stakeholders.”

Niemann-Pick Disease Type C is a rare and fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to a defect in the NPC protein which is responsible for cholesterol processing in the cell. NPC causes symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.

“We thank all of the patients and their caregivers who have or are currently participating in this trial. We recognize the sacrifice of time and energy on the part of families as they participate in this trial,” said Sharon Hrynkow PhD, Chief Scientific Officer and Senior Vice President for Medical Affairs. “We are also deeply grateful to the study team at UCSF Benioff Children’s Hospital Oakland, especially Co-Principal Investigators Caroline Hastings MD and Benny Liu, MD, for all that they have done and continue to do to support the development of this drug.”

It is anticipated that data from the last patient enrolled in the study will be available in February 2020. Cyclo Therapeutics Inc. will then unblind the study results and analyze the data. Data from the current study combined with those of the companion Phase I/II study, nearing completion of enrollment in EU-Israel (ClinicalTrials.gov NCT02912793), are expected to be used in the company’s market registration applications.

About The Clinical Trial:
Complete enrollment in the study required 12 patients, all of whom were enrolled at one site in the United States, UCSF Benioff Children’s Hospital Oakland, CA with Co-Principal Investigators Caroline Hastings, MD and Benny Liu, MD. The study is a randomized, double-blind study using Trappsol^® Cyclo™ intravenously in NPC patients age 18 years and older. Study subjects receive 7 doses of the drug at either 1500 mg/kg or 2500 mg/kg and are assessed for adverse events, markers for cholesterol metabolism following drug administration, and symptomatic changes using an NPC severity scoring tool, among other tests. (See ClinicalTrials.gov NCT02939547 for additional details.)

The company has reported initial data from this study that suggests a favorable safety profile; a temporal link between administration of the drug and clearance of cholesterol from cells; presence of the drug in the cerebrospinal fluid following IV administration; and reduction in a neuron-specific biomarker, tau, that is associated with neuronal degeneration in NPC patients.

About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (ClinicalTrials.gov NCT02939547, NCT02912793 and NCT03893071) and an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com

Cyclo Therapeutics Announces Its Support for The Firefly Fund’s Newborn Screening Program

Source:  Cyclo Therapeutics, Inc. 10/18/19

Announcement Made in Conjunction With October 19^th Global Awareness Day for Niemann-Pick Disease Sponsored by International Niemann Pick Disease Alliance

Cyclo Therapeutics, Inc. (OTCQB:CTDH), formerly CTD Holdings, Inc., a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that the company has provided a grant to The Firefly Fund in support of its newborn screening program for Niemann-Pick Disease Type C. 

“We are proud to support The Firefly Fund and its important work in newborn screening,” said N. Scott Fine, Company Chairman and CEO. “It is our hope that screening for NPC will someday be available globally so that affected families can receive as much help as possible and as soon as possible. We applaud The Firefly Fund for working toward that goal.”

Niemann-Pick Type C Disease (NPC) is a rare and fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell. NPC causes symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.

In June 2017 the Firefly Fund convened a national multi-disciplinary working group that is representative of various community and industry NPC stakeholders who all share a common vision: to add NPC to state and federal newborn screening lists. Pam Crowley Andrews, Co-Founder and Executive Director of The Firefly Fund explained her strategy for seeking a successful nomination to the Federal Recommended Uniform Screening Panel (RUSP), which is a federal list that states use as a guidepost for adding conditions to the various state lists. Ms Andrews explained, “We are building a broad and diverse coalition that is representative of the entire NPC Community. When we began our work, we had one industry partner supporting this initiative and today we have about 10 industry partners, all working on the development of different NPC therapies, that are supporting our newborn screening Initiative. It’s an “All Hands on Deck” approach to this important work that I believe will ultimately be disease–modifying. Knowing earlier about the health status of your child allows parents the option, with guidance from their doctor, to intervene earlier. My husband, Chris Andrews, and I were given the option to intervene early in our younger daughter, Abby, following her diagnosis at 18 months old, before any visible signs or symptoms of the disease. It has now been 3 ½ years since our girls were diagnosed with NPC and our younger daughter is thriving with very few clinical symptoms of NPC. She is an active and neurotypical 5 year old little girl who speaks two languages fluently and enjoys her gymnastics, ballet and knitting classes with her friends. Big sister, Belle (9yo) is so proud of Abby who was just cast in the Nutcracker, which Abby will perform in this December in a theatre in Austin, Texas, with her peers. My husband and I envision a day where all babies born in the United States will be screened for and diagnosed with NPC before any visible signs or symptoms of the disease. We believe that this will be key to rendering Niemann Pick Type C a chronic disease. Newborn Screening is a big and important piece of the NPC puzzle.”
 
Recently Firefly Fund announced the launch of a pilot study of newborn screening for NPC in New York state, referred to as New York Screen Plus, where families will have the option to screen for 13 Lysosomal Storage Diseases (LSDs) in addition to what is already on the state's newborn screening list. This pilot study, under the leadership of Dr. Melissa Wasserstein, will screen approximately 200,000 newborns at 8 different hospitals for 13 LSDs, including NPC, over the course of the next five years. Ms. Andrews said, “When this historic program launches in a few months, it will be the first time any newborn born anywhere in the world will be screened for NPC. Through this study, we plan to collect the evidence necessary to support what NPC expert clinicians already believe — that early diagnosis and intervention for NPC patients improves health outcomes. This study will also give us an opportunity to learn more about how NPC can fit into our existing public health infrastructure.
 
“We are thrilled to have Cyclo Therapeutics, Inc. join our wonderful group of NPC Stakeholders as an Industry Partner supporting Firefly Fund’s NPC Newborn Screening Initiative. It is important that all NPC stakeholders support this project, and not just financially, but in spirit too. It strengthens our message and the quality of our work when we come together as a community and show decision makers that we have consensus among all NPC stakeholders on this important topic of newborn screening,” said Ms. Andrews.

Cyclo Therapeutics’ support for The Firefly Fund is part of its broader effort to support the NPC community while its drug development program for NPC advances. The company currently supports clinical trials for its proprietary hydroxypropyl betacyclodextrin drug, Trappsol® Cyclo™, with sites in the United States, United Kingdom, Sweden and Israel. In addition to its new support for The Firefly Fund, Cyclo Therapeutics' support has included an array of organizations that assist patients and families with NPC, and scientists and physicians working on NPC. These include the International Niemann Pick Disease Alliance (INPDA), the National Niemann Pick Disease Foundation (USA), Niemann Pick UK, and the Australia’s Niemann Pick Disease Association. Cyclo Therapeutics is additionally an industry member of the International Niemann-Pick Disease Registry, a joint initiative between patient organizations and clinicians involved in the care of people with Niemann Pick disease focused on increasing understanding of the disease and speeding potential treatments on their development path.

About Cyclo Therapeutics: Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is in three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071) and in an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.cyclotherapeutics.com
 
About The Firefly Fund: The Firefly Fund, launched on World Rare Disease Day in 2017, is a non-profit organization headquartered in Austin, Texas, with a mission to fund and support the research and education necessary to accelerate a cure for rare genetic neurodegenerative diseases that effect children and have no cure. There are three programs that are the core of what the Firefly Fund is doing for the NPC Community; (1) funding for translational Medical Research; (2) NPC Newborn Screening Initiative; (3) Patient Access Fund. www.firefly.fund
 
About the International Niemann Pick Disease Alliance: The International Niemann Pick Disease Alliance (INPDA) is a global network of non-profit organizations, supporting persons affected by Niemann Pick Diseases (NPD). The alliance was formed in 2009 to provide a forum for patient groups and professionals working in the field of NPD.