NeuBase Therapeutics Announces Issuance of Foundational Patent Covering the Key Building Blocks of PATrOL™ Enabled Therapies

Source:  NeuBase Therapeutics, Inc. 10/17/19

NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, today announced that the U.S. Patent and Trademark Office has issued U.S. Patent No. 10,370,415 covering the Company’s proprietary DNA and RNA binding technology. This foundational patent enables PATrOL™-based therapies to target the secondary structures of DNA and RNA.

“The technology in this patent covers bi-specific nucleobases, which uniquely enables NeuBase to bind and manipulate the hard-to-reach double helix of the human genome and secondary structures of RNA,” said Dietrich Stephan, Ph.D., chief executive officer of NeuBase Therapeutics. “The broad protection provided by the patent asserts our position as a leader in this field. This proprietary and modular technology will be used in conjunction with our peptide backbone technology to address a wide range of rare genetic diseases.”

For more information about the NeuBase PATrOL platform and technology visit neubasetherapeutics.com.

About NeuBase Therapeutics
NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

NeuBase Therapeutics Announces Appointment of Cancer Biologist and RNA Therapeutics Specialist Steven Dowdy, Ph.D., to Scientific Advisory Board

Source:  NeuBase Therapeutics, Inc. 10/11/19

NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, today announced the addition of Steven Dowdy, Ph.D., to its scientific advisory board (SAB). Dr. Dowdy joins preeminent scientists George Church, Ph.D., and Samuel Broder, M.D., on the NeuBase SAB.

“Dr. Dowdy’s considerable expertise in RNA therapeutics perfectly complements the expertise of our current SAB as well as the direction of our company, and we are fortunate to have him as an advisor as we advance our PATrOL™-enabled therapies,” said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. “Attracting these renowned scientists to our team, all leading experts in their respective fields, demonstrates the immense promise and breadth of the PATrOL platform. We are hard at work turning that promise into reality for patients who are currently suffering from devastating genetic diseases.”

Dr. Dowdy added, “NeuBase’s PATrOL™ platform is a powerful tool for addressing diseases characterized by genetic mutations, including cancer. PATrOL™-enabled drugs can access targets that no other antisense therapies are able to access, such as double-stranded genomic DNA and miRNA, as well as mRNA secondary structures. In addition, the inventors of the PATrOL™ platform have published on the ability to have single-base discriminatory power which opens up a world of potential selectivity for disease-causing mutations. I look forward to helping advance a new generation of therapeutics and bringing the promise of the PATrOL platform into reality.”

Dr. Dowdy is a cancer biologist, specializing in the development of RNA therapeutics and in understanding cell cycle controls. He is a professor of cellular and molecular medicine at the University of California, San Diego, School of Medicine, where his research focuses on the delivery of therapeutics into cells, including the development of targeting and endosomal escape technologies. Dr. Dowdy is also a member of the Board of Directors of the Oligonucleotide Therapeutics Society.

About NeuBase Therapeutics, Inc.
NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL™ technology, NeuBase aims to first tackle rare, genetic neurological disorders.

NeuBase Therapeutics Reports Financial Results for the Fiscal Third Quarter of 2019

Source:  NeuBase Therapeutics, Inc. 8/14/19

Completed two financings in July 2019 totaling approximately $14 million in gross proceeds with participation from institutional investors including Greenlight Capital   

NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, today reported financial results for the three and nine month periods ended June 30, 2019, which only include the financial results for Ohr Pharmaceutical prior to the merger and associated financings.

“Over the past several months, we have positioned the Company with the capital and industry expertise required to advance our strategy to develop antisense oligonucleotide therapeutics by leveraging our PATrOL™ platform,” said Dietrich Stephan, Ph.D., chairman and chief executive officer of NeuBase. “Looking ahead, we are developing next-generation antisense oligonucleotide therapeutics for rare genetic diseases with a goal of transforming patients’ lives. We are currently advancing several preclinical programs, including NT0100 to target Huntington’s Disease and NT0200 to target myotonic dystrophy.”

Fiscal Third Quarter of 2019 and Recent Operating Highlights

• Shares of the Company’s common stock commenced trading on the Nasdaq Capital Market under the ticker symbol “NBSE” as of market open on July 15, 2019.
• Completed two financings raising an aggregate of approximately $14 million in gross proceeds, which occurred alongside the Nasdaq listing and included a $5 million investment from Greenlight Capital.
• Appointed four experienced executives from the biotechnology industry to the board of directors: Dr. Dov Goldstein, Dr. Diego Miralles, Dr. Franklyn Prendergast, and Eric Richman.
• Appointed Dr. Danith Ly, the primary inventor of NeuBase’s peptide nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform technology, as the Company’s chief scientific officer.
• Expanded the scientific advisory board with the appointments of Dr. Samuel Broder (former National Cancer Institute Director) and Dr. George Church (Harvard Medical School professor).

Financial Results for the Three Months Ended June 30, 2019:

• For the three months ended June 30, 2019, the Company reported a net loss of approximately $1.1 million, or ($0.40) per share, compared to a net loss of approximately $0.5 million, or ($0.19) per share, in the three months ended June 30, 2018. Loss per share amounts have been retroactively adjusted for the reverse stock split effected on February 4, 2019.
• For the three months ended June 30, 2019, total operating expenses were approximately $1.1 million, consisting of approximately $0.9 million in general and administrative expenses, $0.1 million of research and development expenses, and $0.2 million in depreciation and amortization.  This compares to total operating expenses of $1.2 million in the three months ended June 30, 2018, comprised of approximately $0.8 million in general and administrative expenses, $0.1 million in research and development expenses, and $0.3 million in depreciation and amortization.
• At June 30, 2019, the Company had cash and cash equivalents of approximately $1.4 million, compared to cash and equivalents of approximately $3.8 million at September 30, 2018. Subsequent to the end of the quarter, NeuBase completed two financings alongside the closing of the merger in July 2019, raising gross proceeds of approximately $14 million. The Company believes that its current cash balance will provide sufficient capital to fund operations through the end of fiscal 2020.

Financial Results for the Nine Months Ended June 30, 2019:

• For the nine months ended June 30, 2019, the Company reported a net loss of approximately $3.2 million, or ($1.12) per share, compared to a net loss of approximately $6.9 million, or ($3.36) per share, in the same period of 2018. Loss per share amounts have been retroactively adjusted for the reverse stock split effected on February 4, 2019.
• For the nine months ended June 30, 2019, total operating expenses were approximately $3.2 million, consisting of approximately $2.5 million in general and administrative expenses, $0.2 million of research and development expenses, and $0.5 million in depreciation and amortization.  This compares to total operating expenses of approximately $7.5 million in the same period of 2018, consisting of approximately $2.9 million in general and administrative expenses, $4.2 million of research and development expenses, $0.8 million in depreciation and amortization, $0.7 million in impairment of goodwill, and $1.2 million in gain on settlement of accounts payable and long term liabilities.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of genomic loci or secondary and tertiary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

NeuBase Therapeutics Announces Addition of Industry Pioneer Dr. Samuel Broder and World-Renowned Geneticist Dr. George Church to Scientific Advisory Board

Source:  NeuBase Therapeutics, Inc. 7/30/19

Experts from Harvard and National Cancer Institute to aid in development of novel antisense oligonucleotide therapies for genome-wide transcriptional regulation

NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, today announced the addition of Samuel Broder, M.D, Ph.D. and George Church, Ph.D. to its scientific advisory board (SAB), bringing additional expertise in genetics, genomics, and oncology to a team already recognized for leadership in neurology, genomics and therapeutic development. The SAB will work closely with the NeuBase management team as the Company advances its next-generation gene regulating therapies through development, first in genetic diseases such as the repeat expansion disorders Huntington’s disease and myotonic dystrophy, as well as oncology, and then more broadly.

“We are privileged to have such illustrious colleagues join us as founding members of our scientific advisory board,” said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. “Drs. Broder and Church are each renowned and respected experts in their fields and recognize the immense potential of the PATrOL™ platform to transform millions of lives and fulfill the promise of the Human Genome Project. We are looking forward to utilizing their expertise as we advance the first PATrOL™-enabled therapies for patients suffering from Huntington’s disease and myotonic dystrophy, as well as ultimately helping those suffering from other diseases such as cancer.”

Dr. Danith Ly, chief scientific officer of NeuBase, added, “I am thrilled to welcome Drs. Broder and Church to our SAB. We believe that antisense approaches are the answer to treating a range of genetic diseases, and NeuBase’s PATrOL™ platform represents the future of antisense therapies. I am confident that the expertise Dr. Broder and Dr. Church bring to the table will perfectly complement our team, which is recognized for its leadership in neurology, genomics and therapeutic development, and will help NeuBase bring our PATrOL™-enabled therapies to patients who so desperately need them.”

Dr. Broder is the former Director of the National Cancer Institute (NCI). He was appointed to the position by President Ronald Reagan in 1989 and served through 1995. While at the NCI, he oversaw the development of numerous anti-cancer therapeutic agents, helped launch a number of large-scale clinical trials related to the prevention, diagnosis and treatment of cancer and inaugurated the highly successful Specialized Programs of Research Excellence (SPORE) program. His laboratory played a major role in developing the first three agents approved by FDA specifically to treat AIDS: Retrovir^® (AZT), Videx^® (ddI) and HIVID^® (ddC) and laid the foundation for many other therapies. As the executive vice president for medical affairs and chief medical officer at Celera Corporation, he helped advance Celera’s Human Genome Project, culminating in generating the blueprint of the human genome which has enabled many of the advances in the current life sciences industry. Most recently, he served as senior vice president and health sector head at Intrexon Corporation with a focus on novel gene therapies. He was elected to the National Academy of Medicine of the National Academies of Sciences, Engineering, and Medicine in 1993.

Dr. Church is a pioneer of genome engineering, DNA sequencing and synthetic biology. He brings significant expertise both in genetics and the biotechnology industry to the NeuBase SAB. He has cofounded 24 biotechnology companies, authored over 500 papers and 140 patent publications. Dr. Church is professor of genetics at Harvard Medical School, and professor of health sciences and technology at Massachusetts Institute of Technology and Harvard Medical School. He is the director of the Center for Genomically Engineered Organs (CGEO), the Harvard DOE Technology Center, the Lipper Center for Computational Genetics and is a founding core member of the Wyss Institute for Biologically Inspired Engineering. Dr. Church is a member of the National Academy of Sciences (2011) and the National Academy of Engineering (2012) and has received the Franklin Institute’s Bower Award for Achievement in Science (2011). He holds a Ph.D. in biochemistry and molecular biology from Harvard University.

About NeuBase Therapeutics, Inc.
NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL™ technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Humanigen Announces Exploration of Potential Rights Offering

Source:  Humanigen, Inc.

Humanigen, Inc., (HGEN) (“Humanigen”), a clinical stage biopharmaceutical company focused on the development of next generation CAR-T and other cell therapies, announced today that it is contemplating conducting a broadly syndicated rights offering involving issuing a dividend of rights and over-subscription rights to purchase shares of its capital stock (“Shares”) to its stockholders as of a future record date pursuant to an offering statement on Form 1-A (the “Offering Statement”) which would be filed with the Securities and Exchange Commission (the “SEC”).

A broadly syndicated rights offering is a democratization of the public offering process allowing all stockholders of record to participate in the process.  In this scenario, stockholders as of the record date would receive a right but not the obligation to purchase Shares during a to-be-determined exercise period. Stockholders as of the record date would also receive over-subscription rights to purchase additional shares beyond their pro rata percentage ownership.  Humanigen has engaged RHK Capital to gauge feedback and interest for this potential rights offering.

"We are evaluating conducting a rights offering primarily to raise additional capital to pursue our GM-CSF neutralization strategy in a clinical collaboration with Kite, a Gilead company.  If we proceed with this offering to our stockholders, our stockholders will have the first opportunity to purchase additional securities to maintain or increase their current percentage ownership in our company and provide capital to us at what we believe are attractive price levels," stated Cameron Durant, Humanigen’s chief executive officer.

Please send all inquiries to croberts@rhk.capital if you are interested in speaking with an advisor in regards to the Shares and the potential terms for the rights offering. The company welcomes your feedback as it tests the waters for this potential rights offering to its stockholders.

About Humanigen  
Humanigen, Inc. is developing its portfolio of next-generation cell and gene therapies for the treatment of cancers via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms.  There is a direct correlation between the efficacy of CAR-T therapy and the incidence of life-threatening toxicities (referred to as the efficacy/toxicity linkage).  We believe that our GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with serious and potentially life-threatening CAR-T therapy-related side effects while preserving and potentially improving the efficacy of the CAR-T therapy itself, thereby breaking the efficacy/toxicity linkage.  The company’s immediate focus is combining FDA-approved and development stage CAR-T therapies with lenzilumab, the company’s proprietary Humaneered^® anti-human-GM-CSF immunotherapy, which is its lead product candidate.  A clinical collaboration with Kite, a Gilead Company, was recently announced to evaluate the sequential use of lenzilumab with Yescarta^®, axicabtagene ciloleucel, in a multicenter clinical trial in adults with relapsed or refractory large B-cell lymphoma.  The company is also focused on creating next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, the company is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders.  The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T cell engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT).  The company has established several partnerships with leading institutions to advance its innovative cell and gene therapy pipeline.  For more information, visit www.humanigen.com

About RHK Capital
Advisory Group Equity Services, Ltd. (dba RHK Capital) was founded in 1984. RHK Capital is a boutique investment banking firm specializing in small to medium-sized transactions. RHK is led by a management team with extensive financial industry experience and a desire to provide companies and individuals with the tools and expertise to accomplish their financial goals. In addition to investment banking, RHK has grown to include businesses in general securities, emerging market securities, distressed and high yield debt securities, investment management, mortgages, and business lending. As a division of Advisory Group Equity Services (AGES), all securities are offered through Advisory Group Equity Services Ltd., a registered broker-dealer, member of the Financial Industry Regulatory Authority and the Securities Investor Protection Corporation.

New ‘Blood Advances®’ Publication Supports Humanigen’s GM-CSF Neutralization Strategy with Lenzilumab in GvHD

Source:  Humanigen, Inc. 10/8/19

• GM-CSF from the donor T cells initiates a cascade that drives lethal GvHD
• Without GM-CSF signaling, there was a significant reduction in acute GvHD
• Provide a logical rationale to administer lenzilumab (to neutralize GM-CSF) early after allogeneic transplant to prevent or treat high risk acute GvHD
• Humanigen, in conjunction with the Impact Partnership, planning a multi-center, randomized, phase II trial with lenzilumab in high risk GvHD patients

Humanigen, Inc. (HGEN) (“Humanigen”), a clinical stage biopharmaceutical company focused on the development of next generation cell and gene therapies using its proprietary granulocyte-macrophage colony stimulating factor (GM-CSF) neutralization and gene knockout platforms to simultaneously improve the efficacy and safety of chimeric antigen receptor T (CAR-T) and other cell therapies, today commented on an article and associated visual abstract published October 8, 2019 in ‘blood advances’, the official journal of the American Society of Hematology, entitled “Donor T-cell derived GM-CSF drives alloantigen presentation by dendritic cells in the gastrointestinal tract”. A link to the article and associated visual abstract can be found at: http://www.bloodadvances.org/content/3/19/2859

The study utilized a fate-reporting system to track the role and lineage of GM-CSF secreting donor T cells during development of graft versus host disease (GvHD) in a mouse model. The study demonstrated that GM-CSF signaling was required to initiate the cascade that drives lethal GvHD. In the absence of GM-CSF signaling, there was a significant reduction in acute GvHD. This work builds on earlier work published in Science Translational Medicine entitled, “Graft-versus-host disease, but not graft-versus-leukemia immunity, is mediated by GM-CSF-licensed myeloid cells¹” and helps validate the key role of GM-CSF in the pathophysiology of GvHD. Collectively, the data provide a logical rationale to administer lenzilumab, a proprietary Humaneered® anti-GM-CSF immunotherapy, to prevent or treat acute GvHD.

On July 22, 2019 Humanigen secured an exclusive worldwide license from the University of Zurich for technologies to prevent and treat GvHD through GM-CSF neutralization. Humanigen is currently planning a multi-center, randomized, double blind, phase II trial in conjunction with the Impact Partnership in the UK in patients at high risk of acute GvHD.

About Humanigen, Inc. 
Humanigen, Inc. is developing its portfolio of next-generation cell and gene therapies for the treatment of cancers via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. The company is focused on breaking the efficacy/toxicity linkage with T-cell therapies, making T-cell therapies potentially safer, more effective and more efficiently administered for patients and providers thereby improving healthcare resource utilization for payers. The company’s immediate focus is combining FDA-approved and development stage CAR-T therapies with lenzilumab, the company’s proprietary Humaneered® anti-human-GM-CSF immunotherapy, which is its lead product candidate. A clinical collaboration with Kite, a Gilead Company, was recently announced to evaluate the sequential use of lenzilumab with Yescarta®, axicabtagene ciloleucel, in a multicenter clinical trial in adults with relapsed or refractory large B-cell lymphoma. The company is also focused on creating next-generation combinatory gene-edited CAR-T therapies using GM-CSF gene knockout technologies and developing its own portfolio of proprietary first-in-class CAR-Ts leveraging ifabotuzumab (anti-EphA3 immunotherapy) for various hematologic and solid cancers and HGEN005 (anti-EMR1 immunotherapy) for various eosinophilic disorders. The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T and immunotherapy treatments to break the efficacy/toxicity linkage and prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplant (HSCT). The company has established several partnerships with leading institutions to advance its innovative cell and gene therapy pipeline. For more information, visit www.humanigen.com

Humanigen Secures Exclusive Worldwide License for the Prevention of GvHD through GM-CSF Neutralization from the University of Zurich

Source:  Humanigen, Inc. 7/22/19

• Expands Humanigen’s extensive intellectual property portfolio to include prevention of Graft-versus-Host Disease (GvHD)
• Strengthens Humanigen’s leadership position and platform in granulocyte macrophage-colony stimulating factor (GM-CSF) neutralization to include allogeneic hematopoietic stem cell therapy (HSCT)
• GM-CSF neutralization has the potential to break the efficacy/toxicity linkage associated with allogeneic HSCT

Humanigen, Inc., (HGEN) (“Humanigen”) announced that it has secured an exclusive worldwide license agreement from the University of Zurich (UZH) for technology used to prevent GvHD through GM-CSF neutralization.  The technology was recently featured in a publication in Science Translational Medicine entitled “Graft-versus-host disease, but not graft-versus-leukemia immunity, is mediated by GM-CSF-licensed myeloid cells”¹. The Humanigen license covers various patent applications filed by UZH which complement and broaden Humanigen’s leadership position in the application of GM-CSF and expand Humanigen’s development platform to include improving allogeneic HSCT.

Allogeneic HSCT is a potentially curative therapy for patients with hematological cancers.  However, between 40-60% of patients experience acute or chronic GvHD, carrying a 50% mortality rate.  Donor-derived T cells are responsible for mediating the beneficial graft-versus-leukemia (GvL) effect but have also been linked to destruction of healthy tissue such as skin, gut, and liver resulting in GvHD.  Depleting donor grafts of T cells can prevent or reduce the risk of GvHD. However, this results in a reduced GvL effect and increased relapse rates.  There is a significant unmet medical need for an agent that can uncouple the beneficial GvL effect from harmful GvHD and there are currently no approved agents for the prevention of GvHD.

In the Science Translational Medicine article, the authors demonstrated in a murine model of GvHD, that donor T cell-derived GM-CSF drives GvHD through activation, expansion, and trafficking of myeloid cells but has no effect on the GvL response.  Neutralization of GM-CSF (either using a neutralizing antibody or through GM-CSF gene knock-out) was able to uncouple the myeloid-mediated immunopathology resulting in GvHD from the T cell-mediated control of leukemic cells (GvL).   This discovery provides a clear mechanistic proof-of-concept for neutralizing GM-CSF to prevent GvHD without compromising, and potentially improving, the GvL effect in patients undergoing allogeneic HSCT.

The strong link between T cell-mediated efficacy and myeloid cell mediated toxicity mirrors the findings that have been reported with CAR-T cell therapies where T cell-produced GM-CSF has emerged as a key driver of the myeloid inflammatory cascade resulting in neurotoxicity and cytokine release syndrome and potentially impairing improved CAR-T efficacy through effects on myeloid-derived suppressor cells.  GM-CSF neutralization has the potential to eliminate or reduce the off-target inflammatory cascade while preserving the on-target efficacy of T cell therapies, thereby breaking the efficacy/toxicity linkage.

“Humanigen has pioneered the strategy of neutralizing GM-CSF to improve the safety and efficacy of T cell therapies,” stated Dr. Cameron Durrant, CEO of Humanigen.  “This license agreement builds on our leadership position aimed at breaking the efficacy/toxicity barrier that currently exists for T cell therapies, including allogeneic HSCT. The agreement broadens our already extensive GM-CSF neutralization intellectual property portfolio to include prevention of GvHD in allogeneic HSCT.”

1. Tugues et al., Sci.Transl.Med. 28 November 2018: Vol. 10, Issue 469, eaat8410

About Humanigen, Inc. 
Humanigen, Inc. is developing its portfolio of Humaneered® monoclonal antibodies to address cutting-edge CAR-T optimization and the need for new oncology drugs that provide safer, better, and more effective cancer therapies.  Derived from the company’s Humaneered platform, lenzilumab, ifabotuzumab, and HGEN005 are monoclonal antibodies with first-in-class mechanisms.  Lenzilumab, which neutralizes human GM-CSF, is in development as a potential biologic therapy to make CAR-T and allogeneic HSCT safer and more effective, as well as a potential treatment for hematologic cancers.  Ifabotuzumab, which targets the Eph type-A receptor 3 (EphA3), is being investigated as a potential treatment for a range of solid tumors, both as an optimized naked antibody and as part of an antibody-drug conjugate, as well as a backbone for a novel CAR-T construct, and a bispecific antibody platform. HGEN005 which selectively targets the eosinophil receptor EMR1 is being explored as a potential treatment for a range of eosinophilic diseases including eosinophilic leukemia both as an optimized naked antibody and as the backbone for a novel CAR-T construct.  For more information, visit www.humanigen.com

Humanigen Secures Exclusive Worldwide License to Gene-Editing Technology from Mayo Clinic to Improve CAR-T

Source:  Humanigen, Inc. 7/2/19

• Expands Humanigen’s intellectual property portfolio to include gene-edited CAR-T cells
• Builds and further strengthens Humanigen’s leadership position in GM-CSF neutralization
• GM-CSF neutralization has the potential to break the efficacy/toxicity linkage of CAR-T

Humanigen, Inc., (HGEN) (“Humanigen”) announced today that it has entered into an exclusive worldwide license with the Mayo Clinic to certain technologies used to create CAR-T cells lacking GM-CSF expression through various gene-editing tools including CRISPR-Cas9 (GM-CSF knock-out).  The license covers various patent applications and know-how developed by Mayo in collaboration with Humanigen.  These licensed technologies complement and broaden Humanigen’s leadership position in the GM-CSF neutralization space and expand Humanigen’s discovery platform aimed at improving CAR-T to include gene-edited CAR-T cells.

Acute toxicities such as neurotoxicity (NT) and cytokine release syndrome (CRS) remain a critical unmet need in CAR-T therapy.  These toxicities necessitate inpatient therapy, drive unfavorable inpatient reimbursement, are associated with an overall 15% non-disease relapse mortality rate¹, and increase the overall costs of CAR-T therapy.  Strategies to improve the safety profile of CAR-T without negatively impacting efficacy are needed to improve its benefit-risk profile, cost-effectiveness and to enable CAR-T to move beyond use solely in relapsed/refractory patients to earlier lines of therapy.

A strong link exists between efficacy and toxicity of current and next generation CAR-T therapies, including allogeneic CAR-T, suggesting that NT and CRS might be on-target effects.  However, clinical correlative analysis and pre-clinical modeling evidence points to GM-CSF and myeloid cells as key to the inflammatory cascade.  Emerging evidence suggests that GM-CSF is the communication conduit between the on-target CAR-T/tumor engagement and the non-specific and off-target inflammatory cascade caused by myeloid cells resulting in NT and CRS.  GM-CSF neutralization is emerging as a potential key switch to shutting down the off-target inflammatory cascade while preserving the on-target efficacy of the CAR-T therapy, thereby breaking the efficacy/toxicity linkage.  With this license agreement, Humanigen significantly expands its intellectual property portfolio to include gene-edited CAR-T cells which can be engineered to lack the ability of producing GM-CSF which may improve the efficacy and safety profile of CAR-T.

“Humanigen has pioneered the strategy of neutralizing GM-CSF to improve the safety and efficacy of CAR-T,” stated Dr. Cameron Durrant, CEO of Humanigen.  “This license agreement builds on our leadership position and gives us more tools to construct our portfolio aimed at breaking the efficacy toxicity barrier that currently exists for CAR-T therapies”.

1. Anand K, Burns E, et al. J Clin Oncol 37, 2019 (suppl; abstract 2540)

About Humanigen, Inc. 
Humanigen, Inc. is developing its portfolio of Humaneered® monoclonal antibodies to address cutting-edge CAR-T optimization and the need for new oncology drugs that provide safer, better, and more effective cancer therapies.  Derived from the company’s Humaneered platform, lenzilumab, ifabotuzumab, and HGEN005 are monoclonal antibodies with first-in-class mechanisms.  Lenzilumab, which neutralizes human GM-CSF, is in development as a potential biologic therapy to make CAR-T safer and more effective, as well as a potential treatment for hematologic cancers.  Ifabotuzumab, which targets the Eph type-A receptor 3 (EphA3), is being investigated as a potential treatment for a range of solid tumors, both as an optimized naked antibody and as part of an antibody-drug conjugate, as well as a backbone for a novel CAR-T construct, and a bispecific antibody platform. HGEN005 which selectively targets the eosinophil receptor EMR1 is being explored as a potential treatment for a range of eosinophilic diseases including eosinophilic leukemia both as an optizimized naked antibody and as the backbone for a novel CAR-T construct.  For more information, visit www.humanigen.com.

Citius Reaches First Interim Analysis Milestone for Mino-Lok® Pivotal Trial

Source:  Citius Pharmaceuticals, Inc. 10/7/19

- Study Reaches 40% Completion Triggering Data Cutoff for Futility Analysis

Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, today reported that the data cutoff for the most recent patient treated and completing 8 weeks of observation has been reached. The Company is preparing the submission to the independent Drug Monitoring Committee ("DMC") for review and evaluation.

The objective of the Phase III pivotal trial for Mino-Lok is to evaluate the efficacy and safety of Mino-Lok in salvaging colonized central venous catheters in subjects with catheter-related or central line-associated bloodstream infection (CRBSI/CLABSI) compared to the standard of care using antibiotic locks.

The primary endpoint for the trial is the time to a catheter failure event between randomization and the test of cure (TOC).

The Mino-Lok protocol is based on reaching 92 catheter failure events for the trial, which corresponds to approximately 144 patients treated in both arms combined.
There are 2 interim analyses planned:

• Analysis of futility will be assessed upon reaching 37 catheter failure events (40% of the total number of anticipated events at approximately 58 patients); and
• Analysis for superiority will be performed when 69 catheter failure events (75% of the total number of anticipated events at approximately 108 patients).

"We are extremely pleased to have reached this important milestone in the pivotal trial. With the great work performed at such institutions such as MD Anderson Cancer Center, MedStar Georgetown University Hospital, Henry Ford Hospital, Cleveland Clinic, Carolinas Medical Center, and Massachusetts General Hospital, among others, we have been able to progress to our first analysis of the data. There is a huge medical need for a proven antibiotic lock. We are working hard to provide an alternative to submitting a very sick patient to two surgical procedures to remove and possibly replace a tunneled or implanted venous port. We believe that this pivotal trial will be the largest and best controlled clinical trial evaluating catheter salvage in diagnosed CLABSI patients to date. We are doing everything possible to keep the momentum going," said Myron Holubiak, Chief Executive Officer of Citius Pharmaceuticals, Inc.

About Citius Pharmaceuticals, Inc.Citius is a specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives, cancer care and unique prescription products that use innovative, patented or proprietary formulations of previously-approved active pharmaceutical ingredients. We seek to achieve leading market positions by providing therapeutic products that address unmet medical needs; by using previously approved drugs with substantial safety and efficacy data, we seek to reduce the risks associated with pharmaceutical product development and regulatory requirements. Citius develops products that have intellectual property protection and competitive advantages to existing therapeutic approaches. For more information, please visit www.citiuspharma.com.

About Mino-Lok^®Mino-Lok^® is an antibiotic lock solution used to treat patients with CLABSIs/CRBSIs. CLABSIs/CRBSIs are very serious, especially in cancer patients receiving therapy through central venous catheters (CVCs), and in hemodialysis patients where venous access presents a challenge. There are currently no approved therapies to salvage infected central venous catheters (CVCs).
Mino-Lok^® is under investigation and not approved for commercial use.

Citius Announces Closing of $7.0 Million Underwritten Offering Priced At-the-Market

Source:  Citius Pharmaceuticals, Inc. 9/27/19

Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, today announced the closing of the previously announced underwritten at-the-market offering of 7,821,230 shares of its common stock (Common Stock) (or Common Stock equivalent) and common warrants to purchase up to an aggregate of 7,821,230 shares of Common Stock (the Offering). Each share of Common Stock (or Common Stock equivalent) was sold together with a common warrant to purchase one share of Common Stock at a combined effective price of $0.8951 per share and accompanying common warrant. The Company has granted the underwriter a 30-day option to purchase up to an additional 1,173,184 shares of Common Stock and/or common warrants to purchase up to 1,173,184 shares of Common Stock.

H.C. Wainwright & Co. acted as the sole book-running manager for the Offering.

The gross proceeds of the Offering were approximately $7.0 million and the net proceeds from the Offering were approximately $6.1 million, after deducting underwriting discounts and commissions and other offering expenses. Citius intends to use the net proceeds from the Offering for general corporate purposes, including its Phase 3 clinical Mino-Lok trial for the treatment of catheter related bloodstream infections, the investigational new drug (IND) regulatory pathway for Mino-Wrap and its Phase 2b clinical trial of Halo-Lido cream for the treatment of hemorrhoids, and working capital and capital expenditures.

The common warrants issued in the Offering are exercisable immediately at an exercise price of $0.77 per share and will expire five years from the date of issuance.

A registration statement on Form S-1 (File No. 333-233759) relating to the securities was declared effective by the U.S. Securities and Exchange Commission (SEC) on September 24, 2019. The Offering was made only by means of a prospectus forming part of the effective registration statement. A final prospectus relating to and describing the terms of the Offering has been filed with the SEC. Electronic copies of the final prospectus relating to the Offering may be obtained for free by visiting the SEC's website at www.sec.gov or from H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, New York 10022, by email at placements@hcwco.com or by telephone at 646-975-6996.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall 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 the registration or qualification under the securities laws of any such state or other jurisdiction.

About Citius Pharmaceuticals, Inc.
Citius is a specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives, cancer care and unique prescription products that use innovative, patented or proprietary formulations of previously-approved active pharmaceutical ingredients. We seek to achieve leading market positions by providing therapeutic products that address unmet medical needs; by using previously approved drugs with substantial safety and efficacy data, we seek to reduce the risks associated with pharmaceutical product development and regulatory requirements. Citius develops products that have intellectual property protection and competitive advantages to existing therapeutic approaches. For more information, please visit www.citiuspharma.com.

Citius Announces Change To Primary Endpoint In Mino-Lok® Phase 3 Study

Source:  Citius Pharmaceiticals, Inc. 9/4/19

NEW ENDPOINT IMPROVES ABILITY TO CONDUCT STUDY MORE EXPEDITIOUSLY WITH SIGNIFICANTLY FEWER PATIENTS

Clinical Trial Cost Savings Estimated to Approach $10 million

Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, today reported that the FDA responded to the Company's proposal to refine the endpoints in the current Phase 3 pivotal trial for Mino-Lok.

The current Phase 3 trial being conducted compares Mino-Lok therapy (MLT) to antibiotic lock therapy (ALT) to not only disinfect colonized catheters causing bacteremias, but also to keep the treated catheters functioning and infection free for 8 weeks post therapy.

The new proposed primary endpoint is planned to demonstrate a significant difference in the time to catheter failure when comparing MLT to ALT. This is clinically important because eliminating the source of infection enables antibiotic treatment of the bacteremia to work more effectively and expeditiously. Additionally, if a catheter can be maintained for the time that it is needed, the patient does not need to be subjected to the procedures for removing and replacing the catheter that are associated with some serious adverse events.

The FDA noted that time to catheter failure is an acceptable primary efficacy endpoint. They also instructed the Company to identify and consider the various types of reasons for catheter failure, and to show the clinical significance of this endpoint.

"Citius is very pleased about the change in efficacy endpoints," said Mr. Myron Holubiak, President and CEO of Citius Pharmaceuticals. "We believe this focus will allow us to demonstrate the real benefit of Mino-Lok therapy (MLT) which is that MLT works more efficiently and expeditiously to break up biofilm and eliminate the bacteria causing the bacteremia than current antibiotic lock therapies (ALTs). We now have a clearer way forward to prove the superior efficacy of Mino-Lok to ALTs, and we believe we can do this faster with fewer patients than originally planned. At this time Citius believes that the change to the primary endpoint will result in fewer than 150 total subjects in Phase 3 trial.  These changes would enable Citius to realize clinical trial cost savings approaching $10 million."

The Company has recently submitted a response to the FDA with the new sample size estimate which the agency is reviewing.

About Citius Pharmaceuticals, Inc.Citius is a specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives, cancer care and unique prescription products that use innovative, patented or proprietary formulations of previously-approved active pharmaceutical ingredients. We seek to achieve leading market positions by providing therapeutic products that address unmet medical needs; by using previously approved drugs with substantial safety and efficacy data, we seek to reduce the risks associated with pharmaceutical product development and regulatory requirements. Citius develops products that have intellectual property protection and competitive advantages to existing therapeutic approaches. For more information, please visit www.citiuspharma.com.

About Mino-Lok^®
Mino-Lok^® is an antibiotic lock solution used to treat patients with catheter-related blood stream infections (CRBSIs). CRBSIs are very serious life-threatening infections, especially in cancer patients receiving therapy through central venous catheters (CVCs), and in hemodialysis patients where venous access presents a challenge. The current Phase 3 trial is being conducted in 36 sites. There are currently no approved therapies to salvage infected central venous catheters (CVCs).

Cyclo Therapeutics, Inc. – The New Name for CTD Holdings.

Source:  Cyclo Therapeutics, Inc. 10/4/19

Cyclo Therapeutics, Inc. (OTCQB: CTDH) announced today that it has completed its name change from CTD Holdings, Inc. following appropriate regulatory filings with the State of Florida and FINRA. The new name better reflects the company’s business as a biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, most notably Trappsol^® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease. The company will continue to trade on the OTCQB Exchange under the symbol CTDH. The new name was overwhelmingly approved by shareholders at their meeting on August 27^th.

“As we continue to develop our clinical programs in disease areas that can be potentially treated with Trappsol^® Cyclo™, the Cyclo Therapeutics, Inc. name better reflects the Company’s main business going forward. We look forward to continued progress and higher recognition of our important biopharmaceutical products,” said Company Chairman & 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

CTD Holdings Announces New Collaboration with the Chattanooga Center for Neurologic Research to Advance Alzheimer’s Disease Study

Source:  CTD Holdings, Inc. 9/27/19

CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced a new collaboration with the Chattanooga Center for Neurologic Research (CCNR) based in Chattanooga, TN. CCNR is a biomedical research company led by globally recognized vascular neurologist Thomas G. Devlin, MD, PhD. In close partnership with Erlanger Health System and the University of Tennessee College of Medicine, Dr. Devlin has spearheaded the launch of various biotechnology companies whose commercialized products are having widespread impact on neurologic disease.

The collaboration is expected to lead to selection of CCNR and the Erlanger Health System as a site in CTD’s clinical trial in Alzheimer’s Disease, now in the design stage. As a first step in the collaboration, CCNR and CTD representatives will exchange confidential information on priority scientific topics and clinical capacity.

CTD Chairman and CEO N. Scott Fine said, “Given the significant overlap between stroke, dementia, and Alzheimer’s disease, we are delighted to undertake this collaboration with Dr. Devlin and his team as we build our respective Alzheimer’s programs. Dr. Devlin’s expertise as a world-class clinical researcher in neurovascular disease and stroke is directly applicable as we build out the CTD Alzheimer’s platform.”

Alzheimer’s Disease affects 5.7 million Americans. The disease takes an enormous toll on affected individuals, families and communities as loss of memory and ability to manage daily functions worsens. There are no approved disease modifying treatments. Physicians prescribe treatments for symptoms associated with the disease: these are not effective in all individuals.

Dr. Devlin said, “Tennessee is part of what’s known as the 'stroke belt' in the United States, with a stroke risk over 30% higher than other areas. We have turned this huge medical liability for our patients into a tremendous opportunity to partner with the best biomedical companies worldwide to spearhead new treatments. With Tennessee having the second highest death rate from Alzheimer’s disease of any state in the United States, we are now totally committed to continuing to identify visionary and innovative industry partners to align with as we launch our next major research initiative – Alzheimer’s disease. CTD Holdings is the perfect partner and I anticipate years of close and productive collaboration in our quest to cure Alzheimer’s.”

CTD Chief Scientific Officer Sharon Hrynkow, PhD stated, “Our proprietary cyclodextrin product, Trappsol^® Cyclo™, which has an affinity for cholesterol, has shown encouraging findings in a single late-onset Alzheimer’s patient who has used it through an FDA-approved expanded access program for more than one year (see ClinicalTrials.gov NCT03624842). We are excited about the collaboration with Dr. Devlin and his CCNR team as we work to develop new knowledge about our drug’s potential benefit to Alzheimer’s patients.”

For more information on Dr. Devlin’s current work using artificial intelligence applications to improve stroke care, see HERE and HERE.

About CTD Holdings:
CTD Holdings, 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

CTD Holdings Announces FDA Approval of US Expanded Access Treatment Program in Niemann-Pick Disease Type C

Source:  CTD Holdings, Inc. 9/13/19

CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that the company will provide Trappsol^® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C. The company received notice today of the FDA approval of the individual IND application from the treating physician, Caroline Hastings, MD, pediatric hematologist/oncologist at the UCSF Benioff Children’s Hospital in Oakland, CA.

CTD’s Chairman and CEO, N. Scott Fine, said, “CTD was the first to provide cyclodextrins for use in NPC patients on an expanded access basis in the United States, this was in 2009. We are pleased to once again offer our product for intravenous administration to this pediatric patient on an expanded access basis, even as we advance our formal clinical trials for registration of the drug for the NPC indication.”

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. Because of the NPC defect in this disease, 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.

CTD is currently developing Trappsol^® Cyclo™ as a treatment for NPC in 2 main clinical trials, one based in the United States (a Phase I study) and one based in Europe and Israel (a Phase I/II study). Both trials are nearing completion of enrollment, and design of the pivotal trial is underway. Dr. Hastings serves as the Co-Principal Investigator for the Phase I study in the US in addition to her role as Senior Clinical Advisor to the Phase I/II study. She is also the first physician in the US to administer cyclodextrins (CTD’s product) to NPC patients on an expanded access basis.

“For patients who are not eligible for ongoing clinical trials, expanded access programs such as this one are a critical means for them to receive experimental therapies,” said Dr. Hastings.

CTD’s Chief Scientific Officer and Senior Vice President for Medical Affairs Sharon Hrynkow PhD added, “We have learned a great deal about Trappsol^® Cyclo™ in NPC through expanded access programs over the years: the positive safety and tolerability profile when the drug is administered intravenously coupled with the neurologic and systemic benefits as observed in multiple patients and by multiple physicians show the value of these kinds of programs for patients and for CTD.”

Dr. Hrynkow and Dr. Hastings have presented at several scientific and medical conferences on the outcomes of CTD’s longstanding expanded access programs globally using Trappsol^® Cyclo™ by intravenous administration in NPC patients. Data from the company’s expanded access programs formed the basis of the clinical trial applications for the current trials underway in the US, Sweden, Israel and the United Kingdom. See ClinicalTrials.gov for more information on CTD’s ongoing trials NCT02939547, NCT02912793 and NCT03893071, and CTD’s website for company presentations www.ctd-holdings.com.

About CTD Holdings:
CTD Holdings, 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 used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.ctd-holdings.com

CTD Holdings Holds Shareholders’ Meeting: All Proposals are Approved

Source:  CTD Holdings, Inc. 8/27/19

CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biopharmaceutical company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that it held a meeting of shareholders in New York City for the purpose of approving three proposals, all of which were approved by a wide margin.

The three proposals approved were:

1. Changing the company’s name to Cyclo Therapeutics, Inc.,
2. Adoption of the Company’s 2019 Omnibus Equity Incentive Plan, and
3. Granting the Board of Directors authority to effect a reverse split of shares. 

“We deeply appreciate our shareholders’ confidence in management as evidenced by the approval of these proposals,” said Company Chair and CEO, N. Scott Fine. “These changes position the Company for continued progress in our biopharmaceutical development programs.”

The name change to Cyclo Therapeutics, Inc. will be implemented in the next few weeks following FINRA approval. The Omnibus Equity Incentive Plan becomes effective immediately. Any reverse split of shares will be effected at a strategically favorable time in the future determined by our Board of Directors prior to the Company’s next shareholders meeting.

About CTD Holdings:
CTD Holdings, 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 used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.ctd-holdings.com.

CTD Holdings Announces Filing of Data From Expanded Access Program in Late Onset Alzheimer’s Disease in Support of Its Patent Application Filed In 2018

Source:  CTD Holdings, Inc. 8/13/19

CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biopharmaceutical company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that it has filed supporting data for its patent application titled “Methods for Treating Alzheimer’s Disease.” The original patent application was filed October 29, 2018 for the use of hydroxypropyl beta cyclodextrins in the treatment of Alzheimer’s Disease. According to the US Centers for Disease Control and Prevention, there were 5.7 million Americans living with Alzheimer’s Disease in 2018.

“The data filed with the US Patent and Trademark Office are from a single patient suffering from Alzheimer’s Disease who has been intravenously using Trappsol^® Cyclo™, our proprietary hydroxypropyl beta cyclodextrin, for one year,” said N. Scott Fine, CTD’s Chairman and CEO. The patient is using the drug on an expanded access basis (see ClinicalTrials.gov NCT03624842). “As reported to the FDA in a required annual report, the data suggest a positive safety profile in this patient, overall stabilization of disease, and improvement in certain behavioral aspects of the disease.” 

The original patent application covers dosing regimens and routes of administration. The patent application lists co-inventors Sharon H. Hrynkow, PhD; Jeffrey L. Tate, PhD; and N. Scott Fine of CTD Holdings.

Hydroxypropyl beta cyclodextrins have been shown to decrease amyloid beta, one of the hallmarks of Alzheimer’s disease, in animal and cell culture studies, and to increase the memory span in a mouse model for Alzheimer’s disease (see J. Yao et al., J. Exp. Med. 2012 vol 209(13):2501-13).

“There is currently no cure for Alzheimer’s Disease and only few treatment options to address symptoms,” said Dr. Hrynkow, CTD’s Chief Scientific Officer and Senior Vice President for Medical Affairs. “Filing data in support of our patent application is an important step forward as we begin development of this drug for an Alzheimer’s indication.”

About CTD Holdings:
CTD Holdings, 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 used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.ctd-holdings.com

CTD Holdings Scientific Advisory Board Endorses Alzheimer’s Disease as Next Indication for Company’s Trappsol® Cyclo™ Drug Development Program

Source:  CTD Holdings, Inc. 7/18/19

CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that the Company’s Scientific Advisory Board endorsed Alzheimer’s Disease as the company’s next drug development indication. The Scientific Advisory Board met via videoconference on July 17, 2019. The endorsement was made based on the company’s data from an ongoing expanded access program in late-onset Alzheimer’s Disease with Trappsol^® Cyclo™ and on data from two ongoing trials using Trappsol^® Cyclo™ in Niemann-Pick Disease Type C, a rare genetic disease which may also lead to cognitive decline. The expanded access program in late-onset Alzheimer’s Disease is now in its second year. CTD’s Niemann-Pick Disease Type C trials and the expanded access program in Alzheimer’s are based on intravenous administration of the drug.

CTD Chairman and CEO N. Scott Fine said, “CTD is grateful to the Scientific Advisory Board for its insights and its work. We will move forward to build and launch a clinical trial in Alzheimer’s Disease.”

The Scientific Advisory Board is led by two Co-Chairs: Rita Colwell, PhD, Distinguished University Professor both at the University of Maryland at College Park and at Johns Hopkins University Bloomberg School of Public Health, and former Director of the National Science Foundation, and Sharon Hrynkow, PhD, CTD’s Chief Scientific Officer and Senior Vice President for Medical Affairs.

Scientific Advisory Board members are:
M. Flint Beal, MD, University Professor of Neurology and Neuroscience in the Department of Neurology and Neuroscience at the Weill Medical College of Cornell University - New York Presbyterian Hospital;

Caroline Hastings, MD, Pediatric Hematologist/Oncologist and senior clinician-scientist and educator at the UCSF Benioff Children’s Hospital, Oakland CA; and

Benny Liu, MD, Associate Division Chief of Gastroenterology at Highland Hospital in Oakland, CA and an Assistant Clinical Professor at the University of California, San Francisco (UCSF).
The Scientific Advisory Board plans to meet in late 2019 to consider additional future drug development indications.

About CTD Holdings:
CTD Holdings, 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 used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the company’s website: www.ctd-holdings.com

Exicure Reports Second Quarter 2019 Financial Results and Recent Developments

Source:  Exicure, Inc. 8/8/19

Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, today reported financial results for the second quarter ended June 30, 2019 and provided an update on corporate progress.

“The first half of 2019 continued to bring advancements across our pipeline,” said Dr. David Giljohann, Exicure’s chief executive officer. “We are extremely pleased with the strong progress in both our neurology and oncology franchises, while we simultaneously strengthened our board leadership and balance sheet. Our non-human primate data confirmed our enthusiasm for neurological applications of our SNA platform. Our recent $63.3 million public offering, which closed on August 2, 2019 provides the financial resources to advance these opportunities,” he added. 

Second Quarter Corporate Progress and Recent Developments

• Announced important preclinical data supporting development of SNA technology in the central nervous system (CNS)
  • Presented results from a biodistribution study of SNAs in the CNS of non-human primates.
    • SNAs were observed in all 46 regions of the brain examined.
  • Preclinical research underway in indications including Huntington’s disease, spinocerebellar ataxia type 3 (SCA3), SCA2, SCA1, Friedreich's ataxia and Batten disease.
  • Expect to nominate first candidate for neurological conditions late in 2019.
• Expanded Board with experienced biotechnology leaders
  • Jeffrey L. Cleland, Bosan Hau, Bali Muralidhar and Timothy Walbert.
• AST-008 Phase 1b/2 clinical trial continues to progress—AST-008 is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications.
  • Company expects to complete Phase 1b enrollment of this trial by late 2019.
• Enhanced Financial Resources
  • Closed oversubscribed underwritten public offering of 31,625,000 shares of common stock at $2.00 per share for gross proceeds of $63.3 million with net proceeds of approximately of $58.8 million after deducting underwriters’ discounts and commissions and estimated offering expenses.
  • Insider participation of approximately $2.6 million from David Walt, Jay Ventakensan and Chad Mirkin.
  • Up-listed from the OTC to the Nasdaq Capital Market.

Second Quarter 2019 Financial Results and Financial Guidance
Cash Position: As of June 30, 2019, Exicure had cash and cash equivalents of $17.2 million compared to $26.3 million as of December 31, 2018.

Research and Development (R&D) Expenses: Research and development expenses were $3.4 million for the quarter ended June 30, 2019, compared to $3.8 million for the quarter ended June 30, 2018. The decrease in research and development expenses of $0.4 million was primarily due to lower clinical development programs expense of $0.6 million, partially offset by higher platform and discovery-related expenses of $0.1 million.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.0 million for the quarter ended June 30, 2019, compared to $2.0 million for the quarter ended June 30, 2018. Higher costs for compensation, travel, stock-based compensation and lease expenses were offset by lower legal fees.

Net Loss: Net loss was $5.2 million for the quarter ended June 30, 2019, compared to net loss of $6.8 million for the quarter ended June 30, 2018. The $1.6 million reduction in net loss was due to the addition of $0.4 million of revenue associated with the Dermelix transaction, the $0.4 million reduction in R&D expenses described above and the $0.8 million reduction in loss in Other income attributable to the (non-cash) fair value adjustment of our common stock warrant liability.

Cash Runway Guidance: Exicure believes that, based on its current operating plans and estimates of expenses, as of the date of this press release, its existing cash and cash equivalents will be sufficient to meet its anticipated cash requirements in excess of twelve months.

About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. Exicure believes its proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure's lead program is in a Phase 1b/2 trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL. www.exicuretx.com

Exicure Appoints Dr. Bali Muralidhar of Abingworth LLP and Bosun Hau of Tybourne Capital Management to its Board of Directors

Source:  Exicure, Inc. 8/7/19

Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, today announced the appointment of Dr. Bali Muralidhar, a partner at Abingworth LLP, and Mr. Bosun Hau, a managing director and co-head of private equity at Tybourne Capital Management, to its Board of Directors.

“We are excited to welcome two high caliber leaders like Dr. Muralidhar and Mr. Hau to our board,” said Dr. Chad Mirkin, Ph.D., chairman of the board of Exicure. “Their insight and experience will greatly benefit our strategic priorities and growth as we advance our ongoing clinical programs and expand into new therapeutic areas.”

Bali Muralidhar, M.D., Ph.D., has more than 15 years’ experience in healthcare across a range of functions and currently serves as partner at Abingworth LLP, an international investment group dedicated to life sciences. Prior to joining Abingworth, Dr. Muralidhar was a senior partner at MVM Partners LLP. Prior to MVM, he was a member of Bain Capital’s leverage buyout team, focusing on healthcare. He earned a degree in clinical medicine from the University of Oxford and has a Ph.D. in translational cancer research from the MRC Cancer Cell Unit, University of Cambridge.

"Exicure has developed a new class of genetic medicines with a differentiated platform for oligonucleotide drugs. The SNA technology opens possibilities for DNA and RNA therapies in tissues other than the liver. I am pleased to be joining the board during this important time and to work with such a high-quality team,” said Dr. Muralidhar. “Abingworth is delighted to have led Exicure’s recent oversubscribed financing, which will help enable the company to advance its rich pipeline of development candidates and bring much needed therapies to unserved patients globally.”

Bosun Hau brings more than 15 years of life science experience to Exicure’s board with a focus on emerging growth stage companies. Previously, Mr. Hau served as managing director and partner at Sailing Capital. He also served as a partner at MVM Partners and as a consultant with McKinsey & Company. Prior to McKinsey, he was part of GlaxoSmithKline’s corporate venture group and has also served as an equity research analyst covering the medical device and pharmaceutical industries for JP Morgan and Prudential. Mr. Hau received a B.S. in Molecular and Cellular Biology, a B.S.H.S. in Physiological Sciences and a B.A. in Psychology from the University of Arizona, an M.Sc. in Biotechnology from Johns Hopkins University and an M.B.A in Finance and Health Management from the Wharton School at the University of Pennsylvania.

“I look forward to working with the Exicure board and management team to bring additional strategic perspective to their pipeline and advance innovative drug candidates from discovery through development to benefit patients,” said Mr. Hau.

About Exicure, Inc. Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. Exicure believes that its proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure's lead program is in a Phase 1b/2 trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL and in Cambridge, MA. www.exicuretx.com