Category Archives: Induced Pluripotent Stem Cells

Ncardia inks $60M funding deal to expand discovery and commercial production services for stem cells – FierceBiotech

Ncardia is the result of the 2017 merger of Pluriomics and Axiogenesis. (Getty/designer491)

Ncardia, a human-induced pluripotent stem cell developer,picked up $60 million via a strategic partnership with Kiniciti.

The Belgian iPSC contract research, development and manufacturing company is partnering with the U.S. investment firm to boost discovery, clinical and commercial production capabilities, the companiessaid Tuesday.

Ncardia manufactures iPSC derived cells and provides assay development, disease modeling and cell-based screening. The company is the result of a 2017 combination ofPluriomics and Axiogenesis. Within months of the merger,Ncardiasigned disease modeling licensing pacts with Roche and Evotec.

Now, through its Kiniciti deal, Ncardia can expand globally and invest in good manufacturing practice capabilities for cell therapies, the company said. Ncardia's discovery services will benefit from the financing, said Kiniciti CEO Geoff Glass in a statement.

RELATED:On back of Roche pact, newly formed Ncardia wins iPSC deal with Evotec

That includes building more human cellular models that can predict whether drugs are safe and effective earlier in the development stage, said Stefan Braam, Ph.D., CEO and co-founder of Ncardia, in a statement. The manufacturing technology will go toward iPSC-based allogenic platforms for immuno-oncology.

Kiniciti is a platform company from private equity firm Welsh, Carson, Anderson & Stowe that launched in February with$250 million to invest in non-therapeutic cell and gene therapy startups.Biospring Partners joinedas an investor earlier this month for an undisclosed amount.

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Ncardia inks $60M funding deal to expand discovery and commercial production services for stem cells - FierceBiotech

ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics – Yahoo Finance

- Life Edit's genome editing capabilities to be fully integrated with ElevateBio's other cell and gene enabling technologies, including induced pluripotent stem cells (iPSCs), viral vector, and cellular engineering

- Brings a broad array of editing modalities, including deletion, insertion, base editing, and CRISPRa/CRISPRi

CAMBRIDGE, Mass., October 27, 2021--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that it has acquired all of AgBiome Delta, LLCs (AgBiome) shares of Life Edit Therapeutics, Inc. (Life Edit). Life Edit offers a powerful suite of gene editing technologies that have the potential for any genomic sequence of interest to be removed, added, or altered. Life Edit holds one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors that offer greater specificity and broad genome access. These nucleases were derived from AgBiome's proprietary non-pathogenic microbe collection, which could potentially reduce immunogenicity risks.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211027005153/en/

"ElevateBios powerful suite of enabling technologies, which now includes Life Edits genome editing capabilities as well as our existing iPSC, viral vector, and cell engineering platforms, is designed to disrupt the rapidly advancing fields of cell and gene therapy," said David Hallal, Chairman and Chief Executive Officer of ElevateBio. "Our vision is to build a world-class center of excellence in genome engineering to push the boundaries of therapeutic development and drive innovation for our own therapeutic pipeline as well as provide access to these critical technologies to our growing number of industry partners."

"Genome editing is a central component of all cell and gene therapy development, and access to novel RNA-guided nucleases and base editors that offer specificity and broad genome coverage will be critical. We believe Life Edit's technology is one of the most versatile in the field, opening up enormous potential," Mitchell Finer, Chief Executive Officer of Life Edit and President, R&D of ElevateBio. "This integration will also enable Life Edit to have greater access to ElevateBios drug development and manufacturing capabilities as we build and advance the pipeline, which will initially focus on developing in vivo gene therapies to address neurologic conditions with high unmet need. In addition, by combining Life Edit's gene engineering capabilities with ElevateBios iPSC technology, our goal is to expand the number of therapeutic uses, including potentially making universal or hypoimmune cells that go undetected by the immune system."

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Life Edit was spun out of AgBiome in October 2020 and AgBiome continues to retain rights for gene editing outside of human therapeutics.

About Life Edit Therapeutics:Life Edit has one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors active in mammalian cells. They were developed from a proprietary collection of non-pathogenic organisms and offer gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit has a large and diverse library of RNA-guided nucleases, including Type II and Type V systems that encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with one of our proprietary deaminases. The company's nuclease collection has a broad range of Protospacer Adjacent Motifs (PAMs). These short sequences must accompany the DNA sequence for the enzyme to edit a gene, which offers unprecedented access to genomes. Life Edit has identified several classes of DNA modifying enzymes, including novel deaminases that can edit cytidine (C) or adenine (A). Many of the company's RGNs are smaller than widely used CRISPR-Cas systems, offering ease and flexibility for in vivo delivery and manufacturing.

Life Edit is headquartered in Morrisville, NC. Visit us at http://www.lifeeditinc.com, or follow Life Edit on LinkedIn and Twitter.

About ElevateBio:ElevateBio is a cell and gene therapy technology company built to power the development and manufacturing of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering, that can be leveraged across the entire portfolio and by strategic partners. At the center of the business model is ElevateBio BaseCamp, a centralized research and development (R&D) and manufacturing company that offers R&D, process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also developing its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is located in Waltham, MA. Visit us at http://www.elevate.bio, or follow ElevateBio on LinkedIn, Twitter, or Instagram.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211027005153/en/

Contacts

Investor: Catherine Hu chu@elevate.bio 646-535-8276

Media: Courtney Heath ScientPR Courtney@scientpr.com 617-872-2462

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ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics - Yahoo Finance

ReNeuron enters cancer therapy collaboration with UCL – ShareCast

Cell-based therapeutic specialist ReNeuron has entered a collaboration agreement with University College London (UCL), it announced on Tuesday, to conduct research into the generation of immune cells from induced pluripotent stem cells (iPSCs), for anti-cancer cell therapies.

The AIM-traded firm said it would be working alongside Dr Claire Roddie, associate professor at the UCL Cancer Institute, and the team at the UCL CAR-T cell cancer therapy programme.

It said it would provide UCL with iPSCs from its CTX immortalised neural progenitor cell line to be assessed for their ability to differentiate into functional T cells and natural killer (NK) cells.

If this first objective was met, the CTX-iPSC cell line would be further used to generate chimeric antigen receptors (CAR) T cells, or CAR-NK cells.

ReNeuron explained that CARs allow T and NK cells to target receptors present on the surface of cancer cells, allowing them to recognise and specifically kill tumour cells.

Dr Roddie and her team had extensive expertise in generating CAR cells, the company said, adding that both groups would work collaboratively to generate haematopoietic stem cells, lymphoid progenitors and cytotoxic T cells from the CTX-iPSCs.

We are very much looking forward to working with Dr Roddie and her team at UCL in this exciting and rapidly growing research space, said chief scientific officer Dr Stefano Pluchino.

This collaboration is another strong development for ReNeuron which demonstrates the uniqueness and strong translational potential of our proprietary iPSC technology platform and will allow us to further expand into the oncology space.

If this initial research is successful, significantly large numbers of cancer patients can be treated with next generation alternate cancer therapies.

At 1243 BST, shares in ReNeuron Group were up 4.91% at 119.6p.

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ReNeuron enters cancer therapy collaboration with UCL - ShareCast

Shah on the Potential Utility of NK Cells in Multiple Myeloma – OncLive

Nina Shah, MD, discusses the potential utility of natural killer cells in multiple myeloma.

Nina Shah, MD, hematologist and oncologist, associate professor of medicine, Department of Medicine, University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center, discussesthepotential utility of natural killer (NK) cells in multiple myeloma.

NK cells are a potential type of allogeneic cellular therapy for patients with multiple myeloma, explains Shah. However, a challenge with NK cells is that there are not as many NK cells as other cells; therefore, NK cells need to be expanded and often engineered, Shah explains. One example of engineeringinducibleNK cells is being evaluated by Fate Therapeutics. The NK cells are engineered from a human induced pluripotent stem cellbank to then be cultured, Shah adds.

Moreover, the NK cells strongly express CD16 and Fc receptor on the cell surface. Additionally, CD38 is engineered so utilizing daratumumab (Darzalex) is an option with both engineering tactics, Shah continues. Additionally, the cells are engineered in the context of CAR so targeting BCMA is an option to kill cells. An IL-15 receptor fusion protein was also engineered to allow the NK cells to persist. Preclinical data have shown that the cells are active and persistent, Shah explains.

Overall, more data are anticipated, which will include patients with relapsed/refractory multiple myeloma who received NK cells with a monoclonal antibody, such as daratumumab, Shah explains. Moreover, this approach would be given off-the-shelf, which could open an accessible option to patients, Shah concludes.

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Shah on the Potential Utility of NK Cells in Multiple Myeloma - OncLive

Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of…

Cambridge, MA, - Exacis Biotherapeutics, Inc., a development-stage immuno-oncology company working to harness the immune system to cure cancer, today announced initiation of a strategic partnership with Toronto-based Centre for Commercialization of Regenerative Medicine (CCRM) for specialty manufacturing services related to the development of Exacis innovative, iPSC-derived mRNA-engineered NK cell products to treat cancer. The partnership also includes a cash investment into Exacis by CCRM Enterprises Holdings Ltd., the for-profit venture investment arm of CCRM, which will be used to fund operations.

Exacis CEO Gregory Fiore, MD, commented, We welcome CCRM as a key partner to allow us to rapidly advance our virus-free manufacturing processes to make novel NK cell products that are engineered for performance and to avoid rejection. CCRM is a recognized leader in iPSC-derived cell therapy development and manufacturing and we are thrilled to have them as a partner. Their confidence in Exacis is evidenced by the accompanying investment, by CCRM Enterprises Holdings Ltd., underscoring the unique value proposition offered by Exacis differentiated platform and approach to cell therapies. We look forward to partnering with CCRMs CDMO experts to apply our mRNA based technologies to develop best-in-class products to treat challenging hematologic and solid tumors.

Cynthia Lavoie, PhD, President and CIO of CCRM Enterprises Inc. added, We are pleased to support Exacis by way of an investment, and with our sector expertise and specialized infrastructure. This is a successful model that we have employed in the past to support promising technologies and together we will develop leading cell therapy products that utilize the substantial potential of the Exacis platform as it advances its iPSC-derived cell programs.

About Exacis Biotherapeutics

Exacis is a development stage immuno-oncology company focused on harnessing the human immune system to cure cancer by engineering next generation off-the-shelf NK and T cell therapies aimed at hematologic malignancies and solid tumors. Exacis was founded in 2020 with an exclusive global license to a broad suite of patents covering the use of mRNA based cell reprogramming and gene editing technologies for use in generating engineered NK and T cells for oncology applications. These patents were developed and are owned by Factor Bioscience.

Exacis differentiated cell therapy platform avoids the use of DNA and viruses and uses instead a proprietary mRNA based technology. Exacis uses the technology to generate iPSCs and to edit their genomes to create stealthed, potent allogeneic cell products, termed ExaNK, ExaCAR-T or ExaCAR-NK cells.

About CCRM

CCRM is a global, public-private partnership headquartered in Toronto, Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About CCRMEnterprises Inc.

CCRMEnterprises Inc. is the for-profit venture investment arm of the Centre for Commercialization of Regenerative Medicine (CCRM).CCRM Enterprises invests in early stage ventures and projects developing therapeutics and enabling technologies. Through an extensive network of investors, it can bring together risk capital to support these early-stage ventures as they scale up along the development pathway.By tapping into CCRMs manufacturing infrastructure and expertise, CCRM Enterprises provides capital-efficient support to accelerate and de-risk these high potential, early-stage ventures, further enabling the development of an advanced therapies ecosystem. CCRM Enterprises Holdings Ltd. is the associated entity that holds shares in CCRM portfolio companies. Learn more about our investing strategy here.

About T and Natural Killer (NK) Cell Therapies

T and NK cells are types of human immune cells that are able to recognize and destroy cancer cells and can be modified through genetic engineering to target specific tumors.

Contact Exacis Biotherapeutics for further information and media:

Exacis Biotherapeutics info@exacis.com

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Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of...

ASU professor researches origins of Alzheimers to find a cure – Eight, Arizona PBS

Tuesday, Oct. 12, 2021

ASU Associate Professor Dr. David Brafman is using personalized medicine to study the origins of Alzheimers. Dr. Brafman is tracing the origins of Alzheimers as a way to reprogram stem cells and potentially find a cure for the disease. We recently spoke to Dr. Brafman about his research.

What we use to model this disease is a special type of Stem cell called induced pluripotent stem cells and these cells are derived from patients and reprogram cells to essentially take on the characteristics of early development, Brafman said.

Brafman says this type of research can help his lab find the origins of the disease. He also says this research can help identify why some people are more predisposed to develop this disease, as well as commonalities between those who develop it.

A difficult aspect of treating Alzheimers is that by the time many develop noticeable symptoms, treatment becomes very difficult.

What were trying to identify is genetic diagnostic markers that might have profiles that predispose them towards Alzheimers disease so we could maybe introduce therapeutic interventions earlier, Brafman said

Another therapeutic strategy thought about is potentially swapping the harmful genes that lead to Alzheimers for less harmful ones. Brafman said his lab is looking at the potential for genome editing and genetic risk factors that may lead to the disease.

Brafman said this research could be used to trace the origins of other diseases down the road as well, and not just Alzheimers.

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ASU professor researches origins of Alzheimers to find a cure - Eight, Arizona PBS

CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform – WWNY

Published: Oct. 15, 2021 at 8:50 AM EDT|Updated: 17 hours ago

HANGZHOU, China, Oct. 15, 2021 /PRNewswire/ -- On Oct. 11th, 2021, CellOrigin Inc. released data about its second generation of iPSC-CAR-Macrophage which has a genetically integrated secondary signal to confer controlled CAR-iMac polarization, in the 5th International Conference of IGC China, 2021, Beijing.

Recently, CellOrigin Biotech, a company committed to iPSC-derived innate immune cell therapeutics, has announced a new round of investment by Kunlun Capital. The investment will be used for the CMC development for its current pipeline of iPSC-derived innate immune cells such as iPSC-CAR-Macrophage and rationlly designed iPSC-NK cells. Before, CellOrigin have also acquired investment from Shulan Health and Nest. Bio Ventures.

CellOrigin Biotech has a long term focus on iPSC-derived innate immune cells and its applications in new cancer immune cells. Dr. Jin Zhang, the scientific co-founder of CellOrigin used to be trained as a research fellow at the Boston Children's Hospital and Harvard Medical School. Now, his team worked closely with clinicians at the First Affiliated Hospital of Zhejiang University, and for the first time his team reported the induced pluripotent stem cell or iPSC-derived CAR-macrophages (CAR-iMac), and its applications in cancer immunotherapies.

CellOrigin Biotech holds the domestic and global patents for iPSC-derived CAR-Macrophage, and the engineering for polarization. With this proprietary platform, they are collaborating with research groups in genome engineering and synthetic biology worldwide to fully unleash the potential of iPSC-derived immune cells, which are highly editable, expandable and clonal. Eventually, they would like to achieve a goal of bring more effective, universal and safe immune cell products to cancer patients, especially for those with solid tumors. The investigator initiated trials has been initiated at the First Hospital of Zhejiang University. The core proprietary technology platform and the core patents including the engineered macrophages from pluripotent stem cells has been authorized and is in the process of entering different countries worldwide.

To support the CMC of its pipeline products, on Oct 1st, CellOriginhas announced the launch of its 3000 square feet GMP facility at Hangzhou, China.

About Kunlun Capital

Founded in 2015, Kunlun capital is committed to long-term value investment, establishing long-term partnership with entrepreneurs, and focusing on investing in enterprises with high technical barriers, excellent founding team and explosive growth potential. In recent years, Kunlun capital has successively invested in KEYA Medical, EdiGene, Cytek (NASDAQ:CTKB), Hui-Gene Therapeutics, OBiO, Okeanos, Ucell Biotech, CellOrigin, Soonsolid, Inke (HK:03700), Dada (NASDAQ:DADA), Dreame, Bamboocloud, Pony.ai, PingCAP, Leyan Technologies.

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CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform - WWNY

Personalized Cell Therapy Market Size Worth US$ 53.8 Billion With a CAGR of 23.5% By 2028 Otterbein 360 – Otterbein 360

Coherent Market Insights released a new market study on 2021-2028Personalized Cell Therapy Market with 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. At present, the market is developing its presence. The Research report presents a complete assessment of the Market and contains a future trend, current growth factors, attentive opinions, facts, and industry validated market data. Report offering you more creative solutions that combine our deep geographic experience, intimate sector knowledge and clear insights into how to create value in your business. The research study provides estimates for 2021-2028 Personalized Cell Therapy Market Forecast till 2028*.

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Manufacturers are focusing on strategies such as expansion of their manufacturing facilities to produce large volume of cell therapy candidates. This will aid manufacturers in penetrating the untapped markets in emerging economies and increase the number of personalized cell therapy manufacturing procedures, worldwide. For instance, in April 2019, Kite, a subsidiary of Gilead Company, announced that it will build a new production facility in Frederick County, Maryland, U.S. for the production of innovative personalized cell therapies useful for cancer treatment.

Browse 23 Market Data Tables and 19 Figures spread through 157 Pages and in-depth TOC on Personalized Cell Therapy Market, by Cell Type (Lymphocytes, Mesenchymal Stem Cell, Hematopoietic Stem Cell, and Others), by Therapeutic Area (Cardiovascular Diseases, Neurological Disorders, Inflammatory Diseases, Diabetes, and Cancer), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa), Global Forecast to 2027

The rising regulatory approval of investigational personalized cell therapy candidates is expected to significantly drive growth of the personalized cell therapy market over the forecast period. For instance, in July 2019, Personalized Stem Cells Inc. received approval from the U.S. FDA for its investigational new drug application that involves usage of adipose-derived stem cells for the treatment of osteoarthritis. Personalized Stem Cells Inc. initiated the first clinical trial for this new therapy candidate in August 2019. The company used stem cells for the treatment of knee osteoarthritis in this clinical trial. The adoption of inorganic strategies such as partnerships and joint ventures by major players is increasing, which is expected to fuel growth of the personalized cell therapy market. For instance, in December 2016, Bayer AG and Versant Ventures launched its joint venture BlueRock Therapeutics, a next-generation regenerative medicine company, for the development of induced pluripotent stem cell (iPSC) therapies to cure various diseases. Moreover, an investment of US$ 225 million was made by Bayer and Versant to strengthen BlueRock Therapeutics manufacturing platform and product pipeline.

The major focus of startup biotech companies are on the development of personalized cell therapies, which is fueling growth of the global personalized cell therapy market. For instance, in October 2019, ElevateBio, a U.S.-based cell and gene therapy developer, initiated a new startup, HighPassBio, for developing novel T cell immunotherapies. In 2019, Phase 1 clinical trial was initiated on an engineered T cell receptor (TCR) T cell therapy for HA-1 expressing tumors targeting relapse of leukemia following hematopoietic stem cell transplant (HSCT).

Key Takeaways of the Global Personalized Cell Therapy Market:

The global personalized cell therapy market is expected to exhibit a CAGR of 23.5% during the forecast period (2019 2027). This is attributed to rising number of personalized cell therapy candidates in investigational phase.

The rising number of acquisitions by major players in the market who are involved in the development of personalized cell therapies is expected to fuel growth of the personalized cell therapy market. For instance, in July 2018, Novartis acquired CellforCure from LFB group, a company that specializes in innovative personalized cell therapy. Through this acquisition, Novartis acquired the cell and gene manufacturing facility of CellforCure for contract manufacturing of Novartis leading CAR-T cell therapy Kymriah (tisagenlecleucel) located in Les Ulis, France.

Personalized Cell Therapy Market Competitive Landscape

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Personalized Cell Therapy Market Size Worth US$ 53.8 Billion With a CAGR of 23.5% By 2028 Otterbein 360 - Otterbein 360

Citius Pharmaceuticals to Host Investor Webcast to Discuss the Acquisition of Late Phase 3 Cancer Immunotherapy I/ONTAK (E7777) – Yahoo Finance

Investor Webcast will be held on October 20, 2021 at 8:30 a.m. ET

CRANFORD, N.J., Oct. 13, 2021 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR) today announced that it will host a webcast on October 20, 2021 at 8:30 a.m. ET to discuss the Company's previously announced acquisition of I/ONTAK (E7777), an improved formulation of immunotoxin ONTAK, which was previously approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma (CTCL).

Myron Holubiak, Chief Executive Officer of Citius Pharmaceuticals, will be joined by Leonard Mazur, Executive Chairman and Dr. Myron Czuczman, Chief Medical Officer to discuss the Company's strategy and the I/ONTAK opportunity. Jaime Bartushak, Chief Financial Officer of Citius Pharmaceuticals will join management for a question-and-answer session following the presentations.

Pre-registration for the webcast is required. Questions related to the I/ONTAK acquisition may be submitted in advance or during the live call via the webcast portal.

Date

Thursday, October 20, 2021

Time

8:30 a.m. ET

Registration link

To join the webcast, please register prior to the event date using this link

US (Toll free)

877-407-6176

US (Toll) / International

201-689-8451

Webcast (live and archive)

Available at http://www.citiuspharma.com in the "Events" section

Q&A

Questions may be submitted in advance to ir@citiuspharma.com

About I/ONTAK (E7777)I/ONTAK (E7777) is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. I/ONTAK, a purified version of denileukin diftitox, is a reformulation of previously FDA-approved oncology treatment ONTAK. ONTAK was marketed in the U.S. from 2008 to 2014, when it was voluntarily withdrawn from the market to enable manufacturing improvements. These improvements resulted in I/ONTAK, which maintains the same amino acid sequence but features improved purity and bioactivity. I/ONTAK has received regulatory approval in Japan for the treatment of cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). In 2011 and 2013, the FDA granted orphan drug designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively, making it eligible for seven years of market exclusivity post-approval.

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A global, multicenter, open-label, single-arm Pivotal study of I/ONTAK in participants with recurrent or persistent CTCL (NCT01871727) is underway. The last patient has been recruited; top line results are anticipated in the first half of 2022. A BLA for I/ONTAK is expected to be filed with the FDA by the end of 2022.

About Citius Pharmaceuticals, Inc.Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. The Company has two late-stage product candidates, Mino-Lok, an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections (CRBSIs), which is currently enrolling patients in a Phase 3 Pivotal superiority trial, and I/ONTAK (E7777), a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma (CTCL), which has completed enrollment in its Pivotal Phase 3 trial. Mino-Lok was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). I/ONTAK has received orphan drug designation by the FDA for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). Through its subsidiary, NoveCite, Inc., Citius is developing a novel proprietary mesenchymal stem cell treatment derived from induced pluripotent stem cells (iPSCs) for acute respiratory conditions, with a near-term focus on acute respiratory distress syndrome (ARDS) associated with COVID-19. For more information, please visit http://www.citiuspharma.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "plan," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: our ability to successfully integrate I/ONTAK (E7777) into our operations; the risks of relying on a third party to complete the development of I/ONTAK (E7777); risks relating to the results of research and development activities, including those from existing and new pipeline assets, including I/ONTAK (E7777); our ability to successfully undertake and complete clinical trials and the results from those trials for our product candidates; uncertainties relating to preclinical and clinical testing; our need for substantial additional funds; the early stage of products under development; our dependence on third-party suppliers; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our ability to commercialize our products if approved by the FDA; market and other conditions; risks related to our growth strategy, including our ability to successfully integrate and develop I/ONTAK (E7777); patent and intellectual property matters; our ability to attract, integrate, and retain key personnel; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis, including I/ONTAK (E7777); our ability to procure cGMP commercial-scale supply; government regulation; competition; as well as other risks described in our SEC filings. These risks have been and may be further impacted by Covid-19. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings which are available on the SEC's website at http://www.sec.gov, including in our Annual Report on Form 10-K for the year ended September 30, 2020, filed with the SEC on December 16, 2020 and updated by our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof, and we expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Investor Relations for Citius Pharmaceuticals:Ilanit AllenVice President, Investor Relations & Corporate Communications T: 908-967-6677 x113 E: ir@citiuspharma.com

Citius Pharmaceuticals, a late-stage biopharmaceutical company (PRNewsfoto/Citius Pharmaceuticals, Inc.)

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Citius Pharmaceuticals to Host Investor Webcast to Discuss the Acquisition of Late Phase 3 Cancer Immunotherapy I/ONTAK (E7777) - Yahoo Finance

Wolter Earns Young Investigator Award | Newsroom – UNC Health and UNC School of Medicine

The Brain & Behavior Research Foundation named Justin Wolter, PhD, postdoc in the Neuroscience Research Center, as a recipient of the Young Investigator Award.

Justin Wolter, PhD, a postdoctoral researcher in the labs of Jason Stein, PhD, and Mark Zylka, PhD, at the UNC Neuroscience Research Center, the UNC Department of Genetics, and the UNC Department of Cell Biology and Physiology, was named a recipient of the 2021 Young Instigator Award by the Brain & Behavior Research Foundation (BBRF). The award is for $70,000 over two years.

In his research at the UNC School of Medicine, Wolter aims to understand the molecular and cellular mechanisms of neurodevelopmental diseases. With the BBRF award, he will establish a resource to systematically identify genetic interactions between high-risk autism genes and common genetic variation. This project will build upon work in which Wolter established a cell culture-based approach to conduct genome wide association studies in primary human neural progenitor cells.

Wolter will establish a pilot library of genetically diverse induced pluripotent stem cell (iPSC) lines to explore how common and rare genetic variation interact to influence risk and resilience in a genetically defined subtype of autism.

In 2020, Wolter was first author of a Nature paper from the Zylka lab showing how to use the gene-editing technology CRISPR-Cas9 as part of a potential gene therapy approach to treating Angelman syndrome, an autism spectrum disorder.

Initiated in 1987, the BBRF Young Investigator Grant program provides support for the most promising young scientists conducting neurobiological and psychiatric research. This program facilitates innovative research through support of early-career basic, translational and clinical investigators.

This year, the Foundations Scientific Council, led by Herbert Pardes, MD, and comprised of 176 world-renowned scientists with expertise in every area of brain research, reviewed more than 780 applications and selected the 150 meritorious research projects. Many of the Young Investigator grantees are pursuing basic research projects. Others are specifically focusing on new ideas for therapies, diagnostic tools, and technologies. These research projects will provide future insights and advances that will help move the fields of psychiatry and neuroscience forward.

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Wolter Earns Young Investigator Award | Newsroom - UNC Health and UNC School of Medicine