Dutch Amarna Therapeutics Announces the Appointment of Steen Klysner as Chief Executive Officer

On September 3, 2020 Amarna Therapeutics, a privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform named SVec that promises to transform gene-replacement and immunotherapy across many disease areas, reported the appointment of Steen Klysner, Ph.D. as its new Chief Executive Officer (CEO) as per September 1 (Press release, amarna therapeutics, SEP 3, 2020, View Source [SID1234564445]).. Founder and CEO Ben van Leent will become a member of Amarna’s Supervisory Board

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"I am very pleased to welcome Steen Klysner as our new CEO. Steen brings an extensive background as a biotech CEO to Swedish ExpreS2ion Biotech Holding AB & the Danish ExpreS2ion Biotechnologies ApS, preceded by an impressive track record in execution and value creation within the biotech industry. His leadership experience makes Steen an ideal candidate to lead Amarna into its next stage of growth and development," said Thomas Eldered, Chairman of Amarna’s Supervisory Board. "We are extremely grateful for Ben van Leent’s leadership and contributions to Amarna as both founder and CEO, and we are excited about the opportunity to focus his outstanding expertise as member of our Board."

"I am incredibly honored to have been given the opportunity to lead Amarna," said Dr. Klysner. "SVec has the potential to enable major medical breakthroughs, so that patients can be actually cured of life-threatening diseases for which, to date, effective treatment have not become available. Together with Amarna’s highly qualified and experienced team, I’m fully committed to advance the company’s groundbreaking technology into the next important clinical development stages."

Steen Klysner comes to Amarna with over 30 years of experience in the life sciences industry. Prior to joining Amarna, Dr. Klysner served as CEO of the Swedish ExpreS2ion Biotech Holding AB in parallel with the Danish ExpreS2ion Biotechnologies ApS. Earlier, he was Senior Vice President (SVP) of preclinical R&D and SVP of Quality of Allergopharma, the Allergy Business Unit of Merck KGaA. He also served as CEO of Nordic Vaccine in Copenhagen, focusing on the development of non-invasive vaccination based on an integrated nanoparticle adjuvant and delivery platform. Prior to that he has also held positions at Pharmexa, Novo Nordisk and ALK.

Dr. Klysner holds a Ph.D. from Technical University of Denmark combined with an Industrial Scientist Research Degree from the Danish "Academy of Technical Sciences", a M.Sc. degree in Biochemistry from the University of Copenhagen and a B.Sc. in sports from the University of Copenhagen.

Finally, Dr. Klysner is author/co-author of numerous patents and scientific publications in (inter)-national peer-reviewed medical journals.

"I am deeply grateful to have had the opportunity to build Amarna to where it is today. In my new role as member of the Supervisory Board, my efforts will be towards helping raise the awareness of Amarna and its SVec gene delivery vector platform and using it to help build a robust pipeline," said Ben van Leent. "I have full confidence that Steen, a very passionate and talented leader, will provide the leadership and expertise needed to guide Amarna through the next phases of growth. I look forward to working with Steen to drive forward Amarna’s product candidates."

Lodo Therapeutics Acquires Hibiskus BioPharma And Licenses Rights To Novel Proteasome Inhibitors

On September 3, 2020 Lodo Therapeutics Corp. (Lodo), a biotechnology company reinventing natural product drug discovery by applying its informatics-enabled technology platform to previously undruggable disease targets, reported that it has completed the acquisition of Hibiskus BioPharma, Inc. (Hibiskus) (Press release, Lodo Therapeutics, SEP 3, 2020, View Source [SID1234564444]). In a related transaction, Lodo has acquired exclusive worldwide rights from the University of California, Riverside (UCR) and Michigan State University (MSU) to preclinical proteasome and immunoproteasome inhibitors developed by the co-founders of Hibiskus. Together, these two transactions enable Lodo to consolidate the proteasome inhibitor portfolio and related intellectual property, know-how and early stage research.

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These selective proteasome and immunoproteasome inhibitors were discovered by Hibiskus co-founder and cancer researcher André Bachmann, PhD, Professor of Pediatrics and Associate Chair for Research at Michigan State University, College of Human Medicine, and co-founder Michael Pirrung, PhD, a Distinguished Professor and drug researcher at UCR. Dr. Pirrung is a member of Lodo’s Scientific Advisory Board (SAB).

Professor Bachmann noted, "Despite recent advances, far too many cancers remain poorly treated. Preclinical studies conducted at the National Cancer Institute (NCI) and elsewhere suggest these novel proteasome inhibitors could potentially play a role in the treatment of a number of solid tumor cancers, and we are delighted that the experienced and committed team at Lodo will now be overseeing their preclinical and clinical development."

The lead preclinical molecule, TIR-199, now known as LODO-141, is an irreversible and potent hybrid cyclic peptide proteasome inhibitor from the syrbactin natural product family. The foundational work of Dr. Bachmann and his colleagues identifying the proteasome inhibitor properties of the syrbactin family was published in Nature. LODO-141 is structurally distinct from marketed proteasome inhibitors, retains activity against chemoresistance to these inhibitors, and is highly selective with a well-characterized mechanism of action.

Studies conducted in chemoresistant cell lines published earlier this year in Leukemia Research showed that LODO-141 is more effective than three marketed proteasome inhibitors in resistant multiple myeloma and mantle cell lymphoma. Preclinical studies conducted by the NCI and other researchers and reported in the Journal of Biological Chemistry produced promising early data. LODO-141 demonstrated antiproliferative activity in multiple human solid tumor cell lines including renal, colon, NSCLC, CNS, melanoma, ovarian, prostate and breast cancer. In vivo efficacy has been demonstrated in a multiple myeloma xenograft model, and most notably, in hollow fiber models of solid tumor carcinomas. These early data support Lodo’s plans to develop LODO-141 as a treatment for solid tumors and possibly other cancers.

Dale Pfost, PhD, Chairman and CEO of Lodo, commented, "This is our second targeted transaction at Lodo and reflects our disciplined acquisition strategy based on the extensive experience of our senior team in executing value-creating transactions. We believe these novel proteasome inhibitors are an excellent fit for Lodo. They are derived from natural products and have the potential for improved efficacy and therapeutic index compared to existing proteasome inhibitors, a mainstay of multiple myeloma treatment. Early studies conducted at the NCI and elsewhere suggest that LODO-141 may have utility in treating solid tumors. We intend to assess its potential as a single agent and also in combination with cancer immunotherapy."

Dr. Pfost added, "Importantly, these compounds are compatible with our next-generation drug discovery technology, and we anticipate using our P4 Platform to optimize manufacturing and identify additional analogs with potential improvements in anti-cancer activity and safety profile. Lodo is uniquely positioned to expand the chemical space around these molecules with clearly differentiated profiles that we intend to develop specifically for the treatment of solid tumors."

Dr. Pirrung noted, "I am pleased that Lodo will be developing our novel proteasome inhibitors and exploring ways to expand their utility. As a member of Lodo’s SAB with long-standing interests in natural product derived drugs, I appreciate the potential of Lodo’s platform to transform natural product drug discovery by making it rational, efficient and highly productive. My past work with Lodo CEO Dale Pfost and CFO Don Marvin, along with the talented team of scientists they have assembled, bode well for their success in reinventing this rich source of innovative drugs."

The addition of the proteasome and immunoproteasome inhibitor portfolio via the license from UCR, along with the insights and know-how Lodo is acquiring from Hibiskus, expands the company’s pipeline of novel cancer compounds. Lodo anticipates having candidates from two initiatives—its Wnt pathway inhibitor program and selective proteasome inhibitor program—positioned to begin clinical testing starting in 2023.

Hibiskus BioPharma was established with support from Spartan Innovations and Red Cedar Ventures, both wholly owned subsidies of the Michigan State University Foundation. Lodo is acquiring all the operating assets of Hibiskus and consolidating them at its facilities in New York City. Further financial details of the transactions were not disclosed.

Late-breaking Libtayo® (cemiplimab) pivotal data in advanced non-small cell lung cancer and basal cell carcinoma to be presented at ESMO

On September 3, 2020 Sanofi and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that will present new, positive data for PD-1 inhibitor Libtayo (cemiplimab) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 from September 19-21 (Press release, Sanofi, SEP 3, 2020, View Source [SID1234564443]). Among the accepted abstracts are two late-breaking oral presentations on the investigational use of Libtayo monotherapy in first-line advanced non-small cell lung cancer (NSCLC) and locally advanced basal cell carcinoma (BCC) previously treated with a hedgehog inhibitor.

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Additional presentations will include patient-reported quality-of-life and real-world patient data for Libtayo in advanced cutaneous squamous cell carcinoma (CSCC).

"The Libtayo clinical development program, as monotherapy or in combination with either conventional or novel therapies, focuses on the real-world challenges of patients confronting difficult-to-treat or rare cancers," said Peter Adamson, M.D., Global Development Head, Oncology and Pediatric Innovation at Sanofi. "This development approach was evidenced by the initial approval of Libtayo for patients with advanced cutaneous squamous cell carcinoma, and it continues to guide our Libtayo clinical development program for non-small cell lung cancer and basal cell carcinoma."

Late-breaking oral presentations include:

EMPOWER-Lung 1: Phase 3 first-line cemiplimab monotherapy vs platinum-doublet chemotherapy in advanced NSCLC with programmed cell death ligand-1 (PD-L1) ≥50% (Abstract 1158, LBA52; Ahmet Sezer, M.D.; Proffered Paper Presentation)
Primary analysis of Phase 2 results for cemiplimab in patients with locally advanced BCC who progress on or are intolerant to hedgehog inhibitors (HHIs) (Abstract 3933, LBA47; Alexander Stratigos, M.D.; Mini-oral Presentation)
"Libtayo monotherapy continues to demonstrate significant results in pivotal trials across diverse cancers, and we are excited to share these data at ESMO (Free ESMO Whitepaper)," said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology, at Regeneron. "These trial outcomes highlight the potential of Libtayo to advance treatment of non-small cell lung cancer and basal cell carcinoma, and will form the basis for regulatory submissions in the U.S. and European Union."

Additional Libtayo presentations include:

Time to clinically meaningful changes in pain in patients with advanced CSCC treated with cemiplimab in a Phase 2 clinical trial (Abstract 3955; Poster 1087P; Michael Migden, M.D.; Poster Presentation)
Demographics, prior therapies and reasons for cemiplimab treatment: prospective CemiplimAb-rwlc Survivorship and Epidemiology (C.A.S.E.) study in patients with advanced CSCC (Abstract 1996; Poster 1094P; Guilherme Rabinowits, M.D.; Poster Presentation)
EMPOWER-Lung 4: Phase 2, randomized, open-label high dose or standard dose cemiplimab alone/plus ipilimumab in the second-line treatment of advanced NSCLC (Abstract 4033; Poster 1269P; Byoung Yong Shim, M.D.; Poster Presentation)
Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.

About Libtayo

Libtayo is a fully-human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.

Libtayo is the first immunotherapy approved in the U.S., EU, and other countries for adults with metastatic CSCC or locally advanced CSCC who are not candidates for curative surgery or curative radiation. In the U.S., the generic name for Libtayo in its approved indication is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.

The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. In skin cancer, this includes trials in adjuvant and neoadjuvant CSCC in addition to the pivotal trial in advanced BCC. Libtayo is also being investigated in pivotal trials in NSCLC and cervical cancer, as well as in trials combining Libtayo with either conventional or novel therapeutic approaches for both solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS

What is Libtayo?

Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.

It is not known if Libtayo is safe and effective in children.

What is the most important information I should know about Libtayo?
Libtayo is a medicine that may treat a type of skin cancer by working with your immune system. Libtayo can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one problem at the same time. These problems may happen anytime during treatment or even after your treatment has ended.

Call or see your healthcare provider right away if you develop any symptoms of the following problems or these symptoms get worse:

Lung problems (pneumonitis). Signs and symptoms of pneumonitis may include new or worsening cough, shortness of breath, and chest pain.
Intestinal problems (colitis) that can lead to tears or holes in your intestine. Signs and symptoms of colitis may include diarrhea (loose stools) or more frequent bowel movements than usual; stools that are black, tarry, sticky or that have blood or mucus; and severe stomach-area (abdomen) pain or tenderness.
Liver problems (hepatitis). Signs and symptoms of hepatitis may include yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach area (abdomen), drowsiness, dark urine (tea colored), bleeding or bruising more easily than normal, and feeling less hungry than usual.
Hormone gland problems (especially the adrenal glands, pituitary, thyroid and pancreas). Signs and symptoms that your hormone glands are not working properly may include headaches that will not go away or unusual headaches, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, dizziness or fainting, feeling more hungry or thirsty than usual, hair loss, feeling cold, constipation, deeper voice, very low blood pressure, urinating more often than usual, nausea or vomiting, stomach-area (abdomen) pain, and changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness.
Kidney problems, including nephritis and kidney failure. Signs of these problems may include decrease in your amount of urine, blood in your urine, swelling in your ankles, and loss of appetite.
Skin problems. Signs of these problems may include rash, itching, skin blistering, and painful sores or ulcers in the mouth, nose, throat, or genital area.
Problems in other organs. Signs of these problems may include headache, tiredness or weakness, sleepiness, changes in heartbeat (such as beating fast, seeming to skip a beat, or a pounding sensation), confusion, fever, muscle weakness, balance problems, nausea, vomiting, stiff neck, memory problems, seizures (encephalitis), swollen lymph nodes, rash or tender lumps on skin, cough, shortness of breath, vision changes, or eye pain (sarcoidosis), seeing or hearing things that are not there (hallucinations), severe or persistent muscle pain, severe muscle weakness, low red blood cells (anemia), bruises on the skin or bleeding, and changes in eyesight.
Rejection of a transplanted organ. Your doctor should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had.
Infusion (IV) reactions that can sometimes be severe and life-threatening. Signs of these problems may include chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, fever, feeling of passing out, back or neck pain, and facial swelling.
Getting medical treatment right away may help keep these problems from becoming more serious.

Your healthcare provider will check you for these problems during your treatment with Libtayo. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may delay or completely stop treatment if you have severe side effects.

Before you receive Libtayo, tell your healthcare provider about all your medical conditions, including if you:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus;
have had an organ transplant;
have lung or breathing problems;
have liver or kidney problems;
have diabetes;
are pregnant or plan to become pregnant; Libtayo can harm your unborn baby
Females who are able to become pregnant:

Your healthcare provider will give you a pregnancy test before you start treatment.
You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of Libtayo. Talk with your healthcare provider about birth control methods that you can use during this time.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Libtayo.
are breastfeeding or plan to breastfeed. It is not known if Libtayo passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of Libtayo.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Libtayo include tiredness, rash, diarrhea, muscle or bone pain, and nausea. These are not all the possible side effects of Libtayo. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals and Sanofi at 1-877-542-8296.

Phosplatin Therapeutics Announces Oral Presentation of Phase 1b Combination Study of PT-112 Plus Avelumab at ESMO Virtual Congress 2020

On September 3, 2020 Phosplatin Therapeutics, a clinical stage pharmaceutical company focused on oncology therapeutics, reported that new clinical data from a dose escalation study (NCT 03409458) of lead candidate PT-112, an immunogenic cell death inducer, used in combination with PD-L1 checkpoint inhibitor avelumab, will be presented at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 taking place September 19-21 (Press release, Phosplatin, SEP 3, 2020, View Source [SID1234564442]). Avelumab is co-developed and co-commercialized by Merck KGaA, Darmstadt, Germany and Pfizer Inc. This abstract is one of only seven abstracts that have been selected for the Mini Oral presentation format in the Investigational Immunotherapy category.

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Title: "Phase 1b dose escalation study of novel immunogenic cell death (ICD) inducer PT-112 plus PD-L1 inhibitor avelumab in solid tumors"

Presenter: Daniel D. Karp, MD, Professor, Division of Cancer Medicine, Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center

Session Date / Time: Released 9am CET September 18, 2020

Availability: On demand streaming for the duration of the conference

Abstract Number: 1026MO

Abstracts selected for the on-demand Mini Oral format will be made publicly available at 12:05am CET on Friday September 18, 2020. The Mini Oral presentation will be available on the ESMO (Free ESMO Whitepaper) website (registration required) from 9am CET on September 18, 2020.

"The data to be presented at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020 further validate our development hypothesis with PT-112, and the demonstration of its feasibility in combination with immune checkpoint inhibition," said Robert Fallon, co-founder and chief executive officer, Phosplatin Therapeutics. "There is a large, unmet need for patients with advanced cancer not responding to immunotherapy, who essentially have no standard of care. The combination of PT-112 with avelumab has a strong underlying combination rationale based upon PT-112’s immunogenic cell death properties, and offers potential for advancing the treatment landscape."

The study was conducted as part of a collaboration agreement between Phosplatin Therapeutics, Pfizer, Inc. and Merck KGaA, Darmstadt, Germany (EMD Serono in the US and Canada). Under the terms of the collaboration, Phosplatin Therapeutics is the Sponsor of Phase 1b/2a clinical trials in several indications. Pfizer and Merck KGaA supply avelumab for the trials.

About PT-112

PT-112 is the first small molecule conjugate of pyrophosphate developed in oncology therapeutics. PT-112 promotes immunogenic cell death (ICD), or the release of damage associated molecular patterns (DAMPs), that lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a potential best-in-class small molecule inducer of this immunological form of cancer cell death and is under Phase II development. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress within the Developmental Therapeutics category. The novelty of its pyrophosphate moiety also results in osteotropism, or the propensity of the drug to reach the mineralized bone. This property is of interest in cancer types that originate in bone or frequently lead to metastatic bone involvement, such as metastatic castrate-resistant prostate cancer (mCRPC). The first human clinical results in mCRPC were presented at the 2020 Genitourinary Cancers Symposium.

Avelumab Approved Indications

Avelumab (BAVENCIO) is indicated in the US for the maintenance treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) that has not progressed with first-line platinum-containing chemotherapy. BAVENCIO is also indicated for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Avelumab in combination with axitinib is approved in the US for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

In the US, the FDA granted accelerated approval for BAVENCIO for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

Avelumab Important Safety Information from the US FDA-Approved Label

The warnings and precautions for avelumab (BAVENCIO) include immune-mediated adverse reactions (such as pneumonitis and hepatitis [including fatal cases], colitis, endocrinopathies, nephritis, and other immune-mediated adverse reactions as a single agent or in combination with axitinib [which can be severe and have included fatal cases]), infusion-related reactions, hepatotoxicity in combination with axitinib, major adverse cardiovascular events (MACE) in combination with axitinib [which can be severe and have included fatal cases], and embryo-fetal toxicity.

Common adverse reactions (reported in at least 20% of patients) in patients treated with BAVENCIO monotherapy include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reaction peripheral edema, decreased appetite, urinary tract infection and rash. Common adverse reactions (reported in at least 20% of patients) in patients receiving BAVENCIO in combination with axitinib include diarrhea, fatigue, hypertension, musculoskeletal pain, nausea, mucositis, palmar-plantar erythrodysesthesia, dysphonia, decreased appetite, hypothyroidism, rash, hepatotoxicity, cough, dyspnea, abdominal pain and headache. Grade 3-4 hematology laboratory value abnormalities reported in at least 10% of patients with Merkel cell carcinoma treated with BAVENCIO monotherapy include lymphopenia; in patients receiving BAVENCIO in combination with axitinib, grade 3-4 clinical chemistry abnormalities include blood triglyceride increased and lipase increased.

CNS Pharmaceuticals Announces US Drug Manufacturing Milestones

On September 3, 2020 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the central nervous system, reported an update on progress for the U.S. manufacturing of Berubicin, the Company’s lead drug candidate, in preparation for upcoming clinical trials (Press release, CNS Pharmaceuticals, SEP 3, 2020, View Source [SID1234564441]).

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As previously announced, the Company implemented a dual-track drug product manufacturing strategy and engaged U.S.-based Pharmaceutics International, Inc. ("Pii") and Italy-based BSP Pharmaceuticals S.p.A. ("BSP") for the production of Berubicin. By engaging two separate manufacturers on two separate continents, CNS expects to mitigate COVID-19-related delay risks, diversify its supply chain and provide for localized availability of Berubicin.

Under this dual-track strategy, the Company has achieved several key milestones in its manufacturing efforts and is providing an update on the progress made with its U.S. manufacturer, Pii. First, CNS completed synthesis of Berubicin active pharmaceutical ingredient (API) and shipped API to both manufacturers to prepare an injectable form of Berubicin for clinical use. In preparation for production, CNS and Pii have now agreed on the manufacturing procedure and packaging components for Berubicin and selected a sterile filter manufacturer. The Company has also completed and reviewed a draft of the batch record. Importantly, the Company and Pii completed a successful laboratory simulation of the lyophilization cycle. The Company expects to begin manufacturing of Berubicin at Pii during the third quarter of this year.

"As we prepare to initiate our upcoming Berubicin clinical trials, our execution both on the clinical and manufacturing fronts remain paramount to our success," stated John Climaco, CEO of CNS Pharmaceuticals. "We continue to be encouraged as our partner Pii has now delivered upon many of the critical steps necessary to ensure the quality and availability of Berubicin. We look forward to keeping you updated on our progress as we continue our preparations to submit an IND for Berubicin during the fourth quarter of this year."

CNS’s preparations for filing an IND entail both extensive clinical and manufacturing initiatives. In addition to the progress the Company has made in its manufacturing efforts, it has recently announced critical achievements made on the clinical front. The Company engaged Worldwide Clinical Trials ("Worldwide") as the contract research organization (CRO) for its upcoming Berubicin clinical trials. Worldwide specializes in therapeutic areas with unmet medical needs, including CNS disorders and oncology. Worldwide will work closely with CNS to provide proactive insight and operational support for its upcoming trials. Additionally, the Company engaged Berry Consultants, a leading clinical statistical consulting group, to advise on its Phase 2 trial design for Berubicin. Berry Consultants uses Bayesian statistics to provide innovative clinical trial designs and analysis. The Company has also completed the Phase 1 Clinical Study Report, or CSR, which is now ready for publication.

CNS was recently granted Orphan Drug Designation (ODD) for its lead product, Berubicin, for the treatment of malignant gliomas. The designation provides Berubicin with a special status that can help accelerate its development to treat malignant gliomas by providing CNS with the potential for market exclusivity upon the drug’s approval. The Company plans to file an IND for Berubicin with the FDA during the fourth quarter of 2020.