Podcasts about investigational new drug ind

Tonix Pharmaceuticals Holdings has announced a groundbreaking collaborative research agreement with Makana Therapeutics, aiming to explore the potential of TNX-1500, Tonix's anti-CD40L monoclonal antibody candidate, in advancing the field of xenotransplantation—the transplantation of animal-derived organs into human patients. In an interview with Steve Darling from Proactive, Tonix CEO Dr. Seth Lederman shared details of the preclinical partnership, which will evaluate TNX-1500 in combination with Makana's proprietary human-compatible organs and cells, derived from genetically engineered pigs. The collaboration will initially focus on kidney, heart, and islet cell transplant models, with the broader objective of developing new treatment options for patients suffering from organ failure. The preclinical studies are designed to generate data to support a future Investigational New Drug (IND) application to the U.S. Food and Drug Administration, with the goal of enabling compassionate use of xenotransplantation therapies in patients who have no alternative treatment options. Makana's approach centers around genetically engineered pigs that have been modified to lack swine leukocyte antigen (SLA), a key driver of immune rejection. These animals demonstrate enhanced compatibility with human biology and offer several potential advantages over competing technologies, including high fertility rates, increased litter sizes, and robust health profiles—key factors that support scalability and commercial viability in global markets. TNX-1500 is a next-generation anti-CD40L monoclonal antibody designed to modulate immune response while minimizing the risk of thrombosis—a common side effect associated with earlier anti-CD40L agents. The drug candidate holds promise in preventing organ transplant rejection, making it an ideal component of xenotransplantation protocols. #proactiveinvestors #tonixpharmaceuticalsholdingcorp #nasdaq #tnxp #Biotech #MPOXVaccine #Smallpox #TNX801 #VaccineDevelopment #ClinicalTrials #PharmaceuticalNews #MedicalResearch #WHO #GlobalHealth #InfectiousDiseases #Biopharma #ProactiveInvestors

In this episode, Dr. Nick Meyers and Dr. Eric Hardter, discuss effective strategies for de-risking Investigational New Drug (IND) applications. Discover key insights on how to navigate the complexities of IND submissions, including the importance of pre-IND meetings, optimizing your non-clinical and CMC packages, and understanding FDA expectations. Gain valuable tips on engaging with regulatory agencies, addressing potential issues, and ensuring your IND is well-prepared to avoid clinical holds. Whether you're a seasoned professional or new to the field, this episode offers practical advice to help streamline your drug development process.

Silo Pharma CEO Eric Weisblum joined Steve Darling from Proactive to discuss the company's positive findings from a preclinical study on its novel dual-target formulation designed to address major depressive disorder and severe stress-related conditions. Weisblum highlighted the promising potential of the treatment to deliver enhanced outcomes by combining two therapeutic approaches. The study revealed that this formulation, which simultaneously targets two key pathways linked to mood and stress regulation, demonstrated superior effectiveness in reducing stress-induced behaviors compared to treatments focusing on a single pathway. Animal models showed notable improvements in behavioral outcomes, suggesting the approach could be particularly effective in managing severe psychiatric challenges. Weisblum shared that the formulation is being explored as a potential intranasal preventative treatment for post-traumatic stress disorder (PTSD). The company is now preparing for an Investigational New Drug (IND) submission to progress to human clinical trials. “This study validates our innovative dual-target approach,” Weisblum stated. “By addressing two critical mechanisms simultaneously, we aim to set a new benchmark in care for patients struggling with debilitating stress-related conditions.” Silo Pharma's advancements reflect its dedication to developing groundbreaking solutions for complex psychiatric disorders. The company is optimistic about transitioning its research into clinical development to meet the urgent need for improved mental health treatments. #proactiveinvestors #silopharmainc #nasdaq #silo #BiotechNews #SPC15 #PTSDTreatment #ChronicPain #Fibromyalgia #FDAApproval #EricWeisblum #PharmaUpdates #ProactiveInvestors#invest #investing #investment #investor #stockmarket #stocks #stock #stockmarketnews

Tonix Pharmaceuticals CEO Dr. Seth Lederman joined Steve Darling from Proactive to share exciting news about a significant achievement for the company. Tonix Pharmaceuticals has been awarded a potential contract worth up to $34 million over five years by the Defense Threat Reduction Agency (DTRA), an agency within the U.S. Department of Defense. This contract aims to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections, thereby enhancing the medical readiness of military personnel in biological threat environments. Dr. Lederman explained that Tonix's program will concentrate on the optimization and development of its TNX-4200 program. The goal is to develop an orally available CD45 antagonist with broad-spectrum efficacy against various viral families through extensive preclinical evaluation. The program is expected to establish essential physicochemical properties, pharmacokinetics, and safety attributes to support an Investigational New Drug (IND) submission, ultimately funding a first-in-human Phase 1 clinical study. The agreement with DTRA is a strategic move to address the DoD's objective of protecting U.S. Joint Forces from potential biological weapon threats. The DoD announced in December 2022 that it aims to move beyond the traditional ‘one bug, one drug' approach and is seeking broad-spectrum drugs, as predicting which viruses or how many may be deployed in a biological threat scenario is challenging. Dr. Lederman emphasized that the collaboration with DTRA underscores Tonix Pharmaceuticals' commitment to advancing medical readiness and protection for military personnel. By developing a broad-spectrum antiviral agent, Tonix aims to provide a versatile and robust solution to potential viral threats, enhancing the preparedness and resilience of the U.S. military in diverse and unpredictable biological threat environments. The TNX-4200 program's focus on creating an effective, orally available antiviral agent highlights Tonix's innovative approach to addressing complex medical challenges. The successful development of this broad-spectrum antiviral could significantly impact the way viral infections are managed in military and potentially civilian populations, offering a proactive measure against a wide array of viral threats. Tonix Pharmaceuticals continues to be at the forefront of medical innovation, dedicated to developing cutting-edge solutions that meet the evolving needs of the healthcare and defense sectors. The support from the DoD through this substantial contract is a testament to the potential and importance of Tonix's TNX-4200 program in safeguarding the health and readiness of military personnel against biological threats. #proactiveinvestors #tonixpharmaceuticalsholdingcorp #nasdaq #tnxp #BroadSpectrumAntiviral, #USDepartmentOfDefense, #BiologicalWarfare, #DrugDevelopment, #ImmuneSystem, #SyntheticBiology, #VirusResearch, #InfectiousDisease, #R&D, #FrederickMaryland, #PharmaceuticalInnovation, #HealthcareTechnology, #Biotech, #MedicalResearch, #DefenseContract, #AntiviralAgent, #Healthcare, #PandemicPreparedness, #ScientificResearch#invest #investing #investment #investor #stockmarket #stocks #stock #stockmarketnews

Hemogenyx Pharmaceuticals PLC CEO Dr Vladislav Sandler tells Proactive's Stephen Gunnion that 2023 was a significant year for the company, marked by notable progress with its HEMO-CAR-T product candidate. The key achievement for the year was the submission of a new Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to commence a phase one clinical trial for the treatment of relapsed refractory acute myeloid leukemia. However, the process faced a setback due to issues with lentivirus purity, resulting in a clinical hold. After re-manufacturing the lentivirus, Hemogenyx successfully addressed the FDA's concerns and received approval to proceed with the clinical trials. Furthermore, Sandler reported a successful fundraising which will support the upcoming clinical studies. The company also entered a service agreement with Prevail Infoworks for the clinical trials. In addition to its advancements in HEMO-CAR-T, Hemogenyx continued developing other programs, such as its proprietary Chimeric Bait Receptor (CBR) platform, showing promise in treating non-Hodgkin lymphoma and exploring applications in solid rare tumors. For 2024, the primary focus remains on advancing these clinical trials, particularly the phase one studies, with an immediate goal to initiate patient treatment. #Hemogenyx, #Pharmaceuticals, #CarTTherapy, #ClinicalTrials, #FDA, #CancerResearch, #AcuteMyeloidLeukemia, #GeneTherapy, #Biotech, #Medicine, #Healthcare, #Innovation, #DrugDevelopment, #Biopharma, #MedicalResearch, #InvestigationalDrug, #Lentivirus, #Fundraising, #ChimericReceptor, #ViralInfections #ProactiveInvestors #invest #investing #investment #investor #stockmarket #stocks #stock #stockmarketnews

Tonix Pharmaceuticals CEO Dr. Seth Lederman recently joined Steve Darling from Proactive to delving into the significant development involving the U.S. Food and Drug Administration's (FDA) clearance of the Investigational New Drug (IND) application for TNX-2900. This innovative drug represents a proprietary magnesium-enhanced formulation of intranasal oxytocin, designed to address Prader-Willi syndrome (PWS) in children and adolescents. Dr. Lederman was quick to highlight the importance of this milestone, noting that TNX-2900 had already been granted Orphan Drug designation by the FDA back in 2022. The IND clearance paves the way for a crucial Phase 2 study, aimed at determining the optimal dosage for TNX-2900 in treating PWS. This study will involve approximately 36 PWS patients, thoughtfully divided into four distinct groups, each comprising around nine patients. Within these groups, one will receive a placebo, while the remaining three will be administered varying dosage regimens of TNX-2900. Tonix Pharmaceuticals' strategic plan includes the intention to collaborate with a partner to further advance TNX-2900 through its clinical development for PWS. This collaborative effort underscores the commitment to addressing a pressing medical need, as PWS stands out as the most common genetic cause of life-threatening childhood obesity. Importantly, PWS affects both males and females equally, spanning all races and ethnicities. This complex syndrome arises due to the absence of expression of specific genes located on the paternally acquired chromosome 15. In essence, this groundbreaking development in the treatment of Prader-Willi syndrome represents a significant step forward in the pursuit of better outcomes for children and adolescents grappling with this challenging condition, demonstrating Tonix Pharmaceuticals' dedication to advancing medical science and improving the lives of those affected by rare diseases. #proactiveinvestors #tonixpharmaceuticalsholdingcorp #nasdaq #tnxp #PraderWilliSyndrome #MedicalInnovation #HealthcareNews #ClinicalTrials #DrugDevelopment #OxytocinResearch #MagnesiumFormulation #RareDiseaseAwareness #OrphanCondition #HealthTech #PediatricHealth #GeneticDisorders #SethLetterman #Phase2Trial #InnovativeMedicine #MedicalBreakthrough #PatientCare #ScientificAdvancements #HealthScience #TreatmentOptions #WellnessJourney #ResearchUpdate #MedicalDiscovery #FutureOfMedicine #invest #investing #investment #investor #stockmarket #stocks #stock #stockmarketnews

MGC Pharmaceuticals Ltd (LSE:MXC, OTC:MGCLF, ASX:MXC) CEO and Managing Director Roby Zomer speaks to Thomas Warner from Proactive London following the completion of a share consolidation and capital raise. With these restructuring initiatives completed, Zomer shares his enthusiasm for the future of the plant-based pharmaceutical company and outlines the strategic roadmap for 2024. Zomer emphasises the importance of the recent share consolidation, which involved restructuring the company's capital and introducing new investors aligned with MGC Pharma's long-term vision as a pharmaceutical entity. The CEO highlighted that this move would facilitate the execution of the company's 2024 work plan, presented over the past six months during various roadshows. Acknowledging the impact of the restructuring on existing shareholders, Zomer expresses optimism about the real opportunity it now presents. He explained that the capital raised in the past 18 months, coupled with new long-term investors, positions MGC Pharma to build a sustainable future, providing returns to shareholders who faced dilution. Looking ahead to 2024, Zoma outlined key initiatives in the company's work plan. The focus will be on completing studies on CimetrA, a drug with potential applications, to enable submission to the FDA for an Investigational New Drug (IND) designation. Simultaneously, ongoing clinical trials will pave the way for seeking early access schemes in the United States and eventually obtaining marketing authorization globally. The second drug, CannEpil; designed for drug-resistant epilepsy, will also undergo enrolment and IND submission, with efforts split evenly between both drugs. Zoma highlights the commercial focus on CannEpil in the UK and Ireland, but says CimetrA takes precedence in terms of IND submission, with both drugs considered essential for the company's progress. As the pharmaceutical landscape evolves, Zomer anticipates increased sales of CimetrA through existing schemes, additional purchase orders, and revenue growth. By mid-2024, Zomer envisions CimetrA entering the IND process, with results from dose-finding studies and the initiation of the IND submission for CanAppeal.

In this episode, Vera interviews Dr. Kristin Yarema, President of Cell Therapy at Poseida Therapeutics, about Poseida's cell therapy approach and technologies.In April 2023, Dr. Yarema became the President of Cell Therapy at Poseida. She brings a wealth of knowledge from the biopharmaceutical realm, with expertise in oncology and allogeneic T-cell immunotherapy.Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. With their extensive genetic engineering capabilities, they are paving the way for potentially better and safer treatments for conditions including blood cancers such as multiple myeloma, solid tumors like breast and ovarian cancer, and rare genetic liver diseases.In this episode, Dr. Yarema talks about the advantages of “off the shelf” or allogeneic CAR-T cell therapy vs. autologous cell therapy — and what makes Poseida's approach unique.Dr. Yarema discusses Poseida's two Phase I programs in collaboration with Roche, highlighting the strategic partnership's significance for Poseida.She shares what Poseida is looking forward to with the Investigational New Drug (IND) clearance for the first dual CAR program targeting CD19 and CD20 for the treatment of B-cell malignancies. Dr. Yarema also discusses how the recent $50 million strategic investment from Astellas Pharma in Poseida signifies a notable endorsement and potential growth trajectory for the company.The episode concludes with Dr. Yarema sharing her thoughts about the promise of allogeneic CAR-T cell therapy for patients.For more life science and medical device content, visit the Xtalks Vitals homepage.Follow Us on Social MediaTwitter: @Xtalks Instagram: @Xtalks Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Successfully navigate the complexities of the Chemical, Manufacturing, and Controls (CMC) portions of Investigational New Drug (IND) applications. Listen in as experts provide their insider perspectives on common CMC- and IND- related questions.

SmallCapVoice.com Inc. (“SCV”) announces the availability of a new interview with Dr. Adi Zuloff-Shani, CEO of Clearmind Medicine Inc. (NASDAQ: CMND) (CSE: CMND) (FWB: CWY), to discuss the biotech company's use of non-hallucinogenic psychedelic compounds to treat physical and mental health, including addiction. Clearmind's intellectual portfolio consists of seven patent families. Its lead candidate, CMND-100, is derived from 5-methoxy-2-aminoindane (MEAI), a psychoactive molecule that exerts a euphoric alcohol-like experience and a reduced desire to consume alcohol. CMND-100 is headed for clinical trials in early 2023 as a potential treatment for alcohol-use disorder (AUD), and Clearmind is also exploring the use of its MEAI-based compound to treat cocaine addiction and depression. Earlier this month, Clearmind was granted a patent from the United States Patent and Trademark Office (USPTO) for the use of its proprietary MEAI as an alcoholic beverage substitute. Speaking with SCV's Stuart Smith, Dr. Zuloff-Shani explains the global need for such treatment. “The number of alcoholics is striking,” she says. “We're talking about millions around the world. Alcohol consumption contributes to 3 million deaths each year globally. It is the third leading, preventable cause of death in the U.S. and the yearly cost is enormous – around $200-$250 billion and that's in 2010.” Despite the statistics, Dr. Zuloff-Shani says that existing treatment options in this largely underserved market are largely ineffective. “You would think that a huge problem probably has a lot of treatments to offer,” she explains. “But it's just the opposite. Since 1950, only three treatments were approved by the FDA. I cannot say that any of them are very effective or have no adverse events associated with it.” In pre-clinical studies, however, MEAI has demonstrated promising efficacy in individuals who struggle with alcohol, helping to break the addiction cycle. Clearmind's interest in the component came from the testimonials of people who purchased it to better manage their alcohol consumption. As Dr. Zuloff-Shani explains in the interview, Clearmind paid attention to those experiences and the company selling the MEAI component online. Convinced of MEIA's potential as a safe treatment option, it then acquired certain worldwide patents of MEAI used that information as an entry point for its own compound. Now, Clearmind is working on an Investigational New Drug (IND) application with the FDA as it advances toward clinical studies of MEAI next year. “Usually when you develop a compound, you start from the other direction. You have an idea. You develop a compound and then you go to petri dishes and to animals, and finally you walk that you will have the same signal as, as you had in animals and in petri dishes in humans,” explains Dr. Zuloff-Shani. “Here, we are just doing the reverse process. We understand [MEAI] has the potential to be effective and safe … we decided that we want to do it in a safe place and take the FDA route.” Smith then shifts the conversation toward Clearmind's most recent developments and the strength of its management team, staff and scientific advisory board. Dr. Zuloff-Shani describes the structure of Clearmind's operations and how it benefits corporate objectives and overhead. “We have a very impressive staff in the company,” says Dr. Zuloff-Shani, who is well-versed in navigating regulatory pathways through her more than 20 years of experience in the biotech and pharmaceutical industries. “We don't have a lot of people, and this is by choice. We prefer to work in outsourcing because that enables us to manage our expenses better. On the other hand, it allows us to really collaborate in every field that we are dealing with.” The interview concludes with what Clearmind expects to achieve in the New Year and how it plans to overcome the challenges associated with advancing toward clinical trials.

Top 5 Most Read RNS's on Vox Markets for Wednesday 4th January 2023 5. Union Jack Oil #UJO - US$12m Net Revenues Landmark Reached at Wressle Union Jack Oil announces that material landmark net revenues of US$12,000,000 have been achieved from the Wressle hydrocarbon development, located within licences PEDL180 and PEDL182 in North Lincolnshire on the western margin of the Humber Basin. Union Jack holds a 40% economic interest in this development. 4. Vast Resources #VAST - Q4 2022 Baita Plai Production Report Vast Resources announce that in Q4 2022 it achieved a 47% overall increase in copper concentrate production, a 248% increase in copper concentrate sold and a 35% increase in copper concentrate grade from its Baita Plai Polymetallic Mine in Romania compared with Q3 2022. 3. Hemogenyx Pharma #HEMO - HEMO-CAR-T Process Qualification Run Completed Hemogenyx Pharmaceuticals announce the successful completion of its second Process Qualification ("PQ") run of the end-to-end process for the manufacture of HEMO-CAR-T cells. This PQ run is the second of minimum three identical manufacturing runs required for the submission of an Investigational New Drug ("IND") application to the US Food and Drug Administration ("FDA"). 2. Gfinity #GFIN - Athlos Platform integration into Playgendary Gfinity, a world leading esports and gaming solutions provider today announced that Athlos Game Technologies has entered a one year rolling commercial agreement with leading global mobile game operator, Playgendary a global top ten mobile game developer with three billion downloads 1. hVIVO PLC #HVO - £5.2m RSV contract and Notice of Trading update hVIVO has signed a £5.2m contract with a global biotechnology company headquartered in Asia Pacific to test their respiratory syncytial virus vaccine candidate, using hVIVO's established RSV Human Challenge Study Model. The Company also announces that it will provide a trading update for the year ended 31 December 2022 on 25 January 2023.

In this latest episode of the In Conversation With series, DDW's Megan Thomas speaks with Dr Mark Varney, the Chief Scientific Officer at PsychoGenics, who discusses both his and the company's work on CNS drug discovery in more detail.  PsychoGenics has taken a novel approach to CNS drug discovery, as its phenotypic approach can deliver treatments that work via novel, unknown or multiple mechanisms. Phenotypic screening has accurately predicted better therapeutic activity with fewer side effects for CNS disorders, leading to the identification of some of the most efficacious drug candidates. PsychoGenics' phenotypic drug discovery significantly reduces the time and cost to reaching approved Investigational New Drug (IND) status, resulting in the identification of a viable drug candidate from a few hundred analogs tested in lead optimisation in just over a year.  

Listen as members of Veristat's regulatory team outline the fundamentals— and the pitfalls— as you determine your readiness to file an Investigational New Drug (IND) from a manufacturing perspective.

Tonix Pharmaceuticals (NASDAQ:TNXP) CEO Seth Lederman joins Proactive's Stephen Gunnion with the news that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to support a Phase 2 clinical trial with TNX-601 ER. Lederman explains that TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily formulation of tianeptine as a potential treatment for major depressive disorder (MDD). He said the IND application is an important milestone as the company advances TNX-601 ER into clinical development, with the phase Phase 2 trial in MDD expected to start in the first quarter of 2023. #ProactiveInvestors #TonixPharmaceuticals #MajorDepressiveDisorder #TNX601 #Nasdaq

Welcome all to IS PHARMACOLOGY DIFFICULT Podcast! I am Dr Radhika Vijay.In today's episode, We will gain Pre knowledge before we actually enter the main field of "Clinical trials". What are the intermediate factors or events in between Pre clinical tests and clinical trials in the humans is the whole concept of today's conversation.After knowing the important various factors affecting Clinical Trials, we will have an overview and details of terms like "Informed Consent", "Investigational New Drug" (IND)  Application,IEC, ICH, various drug controlling institutions like FDA, DCGI, etc.All in all it would be a very informative episode with a melodious ending with few verses of a truly inspiring composition and song by Michael Bolton.For all the updates and latest episodes of my podcast, please visit www.ispharmacologydifficult.com where you can also sign up for a free monthly newsletter of mine. It actually contains lot of updates about the medical sciences, drug information and my podcast updates also.You can follow me on different social media handles like twitter, insta, facebook and linkedin. They all are with same name "IS PHARMACOLOGY DIFFICULT". If you are listening for the first time, do follow me here, whatever platform you are consuming this episode, stay tuned, do rate and review on ITunes, Apple podcasts, stay safe, stay happy, stay enlightened, Thank you!!You can access various links via- https://linktr.ee/ispharmacologydifficult

TRACON Pharmaceuticals Inc (NASDAQ:TCON) CEO Charles Theuer joined Proactive's Stephen Gunnion to share news that the US Food and Drug Administration has approved its Investigational New Drug (IND) application for the initiation of a phase one-two clinical trial of YH001 in combination with envafolimab and doxorubicin for the treatment of sarcoma, including in patients who have not received prior therapy. Theuer telling Proactive that the trial will assess the safety and efficacy of YH001 and envafolimab in patients with the rare sarcoma subtypes of alveolar soft part sarcoma and chondrosarcoma.

Sky Bioscience Inc CEO Punit Dhillon joined Proactive with updates on the company's Phase 1 and 2 trials for its SBI-100 Ophthalmic Emulsion. Dhillon told Proactive that Skye plans to file its Investigational New Drug (IND) for a US phase 2 clinical study in the fourth quarter of this year. Meanwhile, the completion of the Phase 1 drug has been updated to early September 2022. The company also recently received regulatory approval for its first in-human Phase 1 clinical study of SBI-100. "Phase 1 ... we are going to continue to generate the data and push towards getting safety and tolerability data in that healthy volunteer study," Dhillon said. "What's really exciting for us is moving our Phase 2 program aggressively forward, which is where we're, again, measuring safety but also looking at efficacy in glaucoma patients and patients that have ocular hypertension."

FSD Pharma Inc CEO Anthony Durkacz told Proactive that the company is ready to proceed with its planned Phase 2 clinical trial of FSD-201 as soon as it gets the go-ahead from the US Food and Drug Administration and Health Canada. That's after submitting Investigational New Drug (IND) applications to the two bodies this week. Armed with cash of about $50 mullion following the $16.4 million disposal of its former cannabis production facility in Cobourg, Ontario, Durkacz said FSD is also moving full-steam ahead on its two other drug candidates and may look for other opportunities. A special dividend may also be on the cards, he added.

"Wall Street doesn't believe in this target," by Dario Altieri. A scientist shares his 12-year journey that led to the discovery of a drug now in clinic.   TRANSCRIPT Narrator: Wall Street Doesn't Believe in This Target by Dario C. Altieri, MD (10.1200/JCO.22.00180) March 2, 2009. Just published in the Journal of Clinical Investigation.1 And we even got the cover. Twists and turns of heat shock protein-90 (Hsp90), the chaperone, the evolutionary capacitor. Great name and important cancer target. People smiled when I talked about this at the Hsp90 conference. No, no, really there is a lot of it in mitochondria, and only in mitochondria of tumor cells. And, I don't know why, but Hsp90 drugs don't touch it: somehow, they don't get to mitochondria. So, I made my own. Took an old Hsp90 inhibitor, 17-allylamino-17-demethoxygeldanamycin and attached it to triphenylphosphonium, a carrier that basically gets anything into the mitochondria. No, of course, I didn't do the synthesis in my laboratory. What do I know about medicinal chemistry? Outsourced it. Like sneakers and sweaters. And after three months, there it was: an Hsp90 inhibitor that only goes to the mitochondria, causes aggregation of a lot of proteins, and kills tumor cells in minutes. Makes sense, right? Mitochondria must control protein folding, especially in cancer, and they do it with chaperones. Inhibit the process and tumor cells can't cope. Normal cells don't seem to mind. So, strong preclinical activity, and against a lot of different tumor types. Better than any other Hsp90 inhibitor. Good safety. Totally different mechanism of action. And even a cool name, Gamitrinib. Tired of curing mice. What if this were to work in people? Ideas are made in academia; drugs are made in companies. Okay, fine, then I'll launch a startup, spinout, whatever they are called. The (former) doctor-turned scientist now turns entrepreneur, and then captain of industry. Problem is, I am not like that. More like an (aged) boy scout. The inner soapbox says: it belongs to the American taxpayers; they funded it; it's theirs. Excellent start. What else? If doctors and scientists become businessmen (or businesswomen), who will take care of humanity and discover new things? Perfect for a campaign ad. Sold. Bring it to the patients solely from academia: no pharma, no biotech, no investors, no nothing. Soapbox meme for the day: Yes, we can. It's going to cost. So? I'll write a grant, that's what I do for a living anyway. And the fact that I know zero about drug discovery? Or drug development? Laboratory-Clinical Transition Award from the Department of Defense. Great title. Three years of funding. Perfect for me. Pass-through money, nothing for the laboratory, but it pays the bills of outsourcing. First things first. Synthesize Good Laboratory Practice (GLP) Gamitrinib. Already getting a pretty good hang on the acronyms. Hey, we made this drug lots of times before and has never taken this long. It's almost a year and counting. The Department of Defense (DoD) is on my case because I am already behind. What's going on? Yes, I understand that we don't make anything in this country anymore. New import permits that need to clear the Indian government? The what? This is just a chemical, not an international incident. Yes, I get it, nothing I can do about it. My new job is mailman. And telephone operator. Finally shipped the GLP drug for the toxicology. Two animal species, says the US Food and Drug Administration (FDA). Rats and dogs sound good to me. Should I feel sorry for the dogs? Rats not so much. But what if Gamitrinib poisons the mitochondria in the brains? Or hearts? Wait, you said it's just perfect? Animals are doing great, all of them. And no toxicity at all, like giving them…water. Wow, that's some news. Feeling quite pleased with myself. See? I said it all along: mitochondria are wired differently in cancer. That's why the drug is safe for normal tissues. Maybe I should write a review article about that. Serious boost of the citation index. I am sorry, what? Yes, of course I know that the drug is purple. Okay, you filtered it before giving it to the animals and instead of purple it turned white? And you did that to all animals. For the entire time. Oh, what do I think it means? I think it means that you filtered out the drug and we have been giving animals…water. Yes, I get it. I need a new formulation. And start over. Note to self: find a new Contract Research Organization (CRO) that doesn't give water to the animals. Formulation experts. Big pharma ex-pats who now have their own CRO. Everybody is an entrepreneur here. Is this drug oral? Nope. Is it soluble in water? Not at all. So, it's an intravenous (IV) infusion? Yes, that's what it is. Sorry, then it's not a drug. It's not? And what about things like, you know, paclitaxel or doxorubicin? Aren't they also insoluble and given IV? Last time I checked, we used them for half a century and saved millions. Oh, now we think differently? I see, Fail Fast: that's how we think now. And mine, whatever it is if it is not a drug, has already failed. Nobody likes to take risks. Soapbox meme for the day: If nobody takes risks, how do we make progress, exactly? So, maybe I am in good company: paclitaxel and doxorubicin would also Fail Fast today. It's doable. Nobody likes it but it's doable. Sterile-filter the emulsion components and then bring the particle size below 200 nm. Nice. How do I do that? With a microfluidizer. And why nobody likes it? Oh, because the microfluidizer is a dirty machine and where you make Good Manufacturing Practice is called a clean room. Impeccable logic. But a place in California may do it. For a fee, of course. Oh, and you have to buy the machine. Buy what? Or lease it, whatever. People may not like it, but the whole thing works like a charm. Except, of course, when the microfluidizer stops for no reason in the middle of the run. Media fill looks good. Drug is stable for months in the new formulation. Release testing coming together nicely. I am running out of money. Burned through not just one but two DoD grants and all my research kitty. Nothing saved for the swim back: talk about risk-taking. At least the repeat toxicology is paid for and looks good. The drug, the real thing this time, is safe. They even did ECGs on the dogs. Thank goodness I didn't have to read those, but they are normal: no QTc liability. Can't drop the ball now, but I really need money. Here is how you do it: silence the inner soapbox and enchant the big pharma suits that are coming over. Use the right words. It's not early stage anymore. Asset totally derisked. Sure it's ready for prime time. It works. I am a natural. Maybe I should have done this before. A lot of nodding around the table. The suits must be in awe with the great pharmacokinetics, long half-life, and fabulous safety. A hand goes up. I am sorry? Sales data? Sales of what? What is the unit price? No, no, no, we are not there yet. I haven't even filed an Investigational New Drug (IND) application. Something different now. Analysts who advise big-time investors. They don't wear suits. Sweaters for sure. Maybe black tees a la Steve Jobs. They like new things and totally live by risks. Sounds like my crowd. And don't forget, they can get tons of money from people who already have tons of money and want to make even more money. My crowd? Voices out of a polyphone. Yes, it is Hsp90. Yeah, the chaperone. Sure, I know, it has been around for a long time. But this is a completely new story: nobody ever tested a cancer drug that goes to a subcellular organelle: that's really where the action is. Yes, Hsp90. And mitochondria, they used to be bacteria two and a half billion years ago, but they turned out to be important in cancer. I know that too, Hsp90 drugs didn't fare well in the clinic. Lot of toxicity, basically no efficacy. Yes, very unfortunate. But this one has a completely different mechan…Sure, I would like to hear that perspective. I am sorry, did you say, Wall Street doesn't believe in this target? Triaged the first time but funded on the resubmission. Could have been worse. This one is a grant from the National Cancer Institute. And a nice award from the Gateway Foundation is coming too. Enough to pay for the clinical trial. Single site, standard phase I. Accelerated dose escalation. Up to 35 patients with advanced cancer. All comers. Drug vials ready to go. And a fantastic clinical investigator to run the trial. You really don't want me in the clinic. The only thing missing is IND approval. Right, there is that. No, not a commercial IND, investigator-initiated IND, thank you very much. The FDA people are the nicest in the world. Super-helpful, don't believe otherwise. Or maybe they just feel sorry for the clueless applicant. Thirty days to respond to the questions. Totally getting a promotion to a higher rank of telephone operator. And publisher of FDA modules. And certifier of United States Pharmacopeia (USP) . recommendations. And fixer of Chemistry, Manufacturing, and Controls deficiencies. Oh, and let's not forget the specs for polytetrafluoroethylene filters. Then the examiner mutters two words at the end of a phone call. Good luck. Then, nothing. No more questions, e-mails, or phone calls. Right on the thirty-day mark. Were you expecting this? It's a letter; it says study may proceed. What would the day look like? The first patient to be dosed. Maybe I should go to the clinic: it's in town, not far from where I am. I don't think I can pass muster as one of those confidence-inspiring docs in pharma ads. But I do well as chief executive officer. The cufflinks look good, and so do the shoes. I can impress the family. My Italian accent can pass as straight from South Philly, so I have that also going for me. And I can more than hold my own if I need to talk about Philadelphia Eagles football and worries with Jalen Hurts' arm for next season. I used to be good with my patients. Or at least I convinced myself of that. Yes, this is an experimental drug straight out of our backyard, right here in Philadelphia. No, I don't know if it will work, but I sure hope it will. And thank you, thank you so much for being part of the trial. What if I make these people even sicker than they are? I took an oath a long time ago. Anyway, I know the literature on phase I studies, chances are it just won't do anything, so nobody gets hurt and I am finally done with it. I never thought this moment would arrive. There is none of that. January 10, 2022. It's just a late-night e-mail on the anniversary of my mom passing from lung cancer. Hey, the first patient did great at the starting dose of Gamitrinib. No problem whatsoever. The next patient will now get twice the dose. I hope we get that started this month. Happy new year. And that was that. Twelve years, 10 months, and nine days from that Journal of Clinical Investigation paper.1 Affiliation: 1The Wistar Institute, Philadelphia, PA Dr. Lidia Schapira: Welcome to JCO's Cancer Stories: The Art of Oncology. I'm Lidia Schapira, Associate Editor for Art of Oncology, and Professor of Medicine at Stanford University. And I'll be the host of this show. Cancer Stories is brought to you by the ASCO Podcast Network, a collection of nine programs covering a range of educational and scientific content and offering enriching insight into the world of cancer care. You can find all of the shows including this one at podcast.asco.org. With me today is Dr. Dario Altieri, president, and CEO at the Wister Institute. We'll be discussing his Art of Oncology article: Wall Street Doesn't Believe in This Target. Our guest is a named inventor for patent number 2,699,794. Titled: Mitochondria Targeted Anti-Tumor Agents. Dario, welcome to our podcast. Dr. Dario Altieri: Thank you so much for having me, Lidia. It's a great privilege. Dr. Lidia Schapira: My first question to you and to our authors is this, people who enjoy writing are usually also readers, what are you reading now? Dr. Dario Altieri: Well, absolutely it has been a passion of mine since the floods. I am an absolute avid reader of novels, and history, in particular, contemporary history and modern history. Those are my favorite topics. Dr. Lidia Schapira: Do you read in English, Italian, or other languages? Dr. Dario Altieri: I typically read in English, even though some of the Italian literature is best read in the native tongue. And so, I am still attached to that. Dr. Lidia Schapira: You're clearly a very accomplished scientist. But tell me a little bit about your writing in this particular area in what I'll call creative nonfiction. How has this writing helped you perhaps process experiences or communicate with others? Dr. Dario Atieri: It has been, it's been a passion of mine for a very long time, I think. In finishing up college, of course, my major was contemporary literature and philosophy. The question was whether to continue on in a classic literature career or go to medical school, probably the wrong choice was made. But it has remained with me for a very long time, and it's a form of expression that I truly enjoy. In writing, this particular contribution was a bit transformative for me. It doesn't happen every time that you write a scientific article to express a little bit about yourself and your passions and dreams. Dr. Lidia Schapira: Let's talk a little bit about your passions and dreams in this article. You described an intensely personal journey of 12 years that led to the discovery or the availability of this drug now in the clinic. When did you think that you wanted to share this story with your colleagues? And tell me a little bit about the process of writing this article? Dr. Dario Altieri: It has certainly been a roller coaster experience. I would like to describe it as life-defining and life-changing. I've learned so much and so many things, not just about the process, but also a little bit about myself. I recognize reaching the clinic, especially in a phase one trial, is really just the beginning. But for me, as a basic scientist, somebody who has seen his last patient in the 13th century. As a basic scientist, that was a little bit of a milestone, and I wanted to share what it took, the experiences that I lived through, especially with our youngest colleagues, scientists, and doctors, starting their own careers in oncology, whether it's basic research, clinical research, translational research, I really don't think it matters. And so, issues of resilience, staying the course, passion, and not really giving up are the parameters that I had hoped to convey with this contribution. Dr. Lidia Schapira: In your article, I was so impressed by how you used humor, often self-deprecating humor, and the particular narrative style and writing style that you chose and defended as you were revising it. You know, this choppy phrasing, a staccato, and you said, this is what it feels like, how can I pack it into a small number of words and describe it all? Tell us a little bit about how you allowed your imagination to take over and how you found the proper voice and style for this particular narrative. Dr. Dario Altieri: Again, it's been a thrilling experience and it's been a thrilling experience to answer to the editors and the reviewers of the JCO, who provided incredible insightful comments. The challenge was, how do I tell a story without sounding obvious, fright, or expected, and more importantly, without sounding boring? And I think to paraphrase one of our reviewers about this journey. What the reviewer said, the author, that would be me, has encountered many of the absurdities of the path in drug development, something that we don't talk about too much because it's been the realm of a drug company for the longest time. And so, I wanted to try to capture that absurdity in a positive way. Things that the reviewer indicated, may be second nature to the pharmaceutical industry, but for academic investigators, that's been publicly funded for 30 years, is not second nature and is unusual, and is a world all in itself. And so, that was the impetus of trying to use literature advice on short sentences that are really intended to convey the impression of the moment that was what I tried to accomplish. Dr. Lidia Schapira: Well, you certainly picked a catchy title, and we have not published this sort of article in Art of Oncology before. For our listeners, tell us a little bit about why Wall Street doesn't care about your discovery? Dr. Dario Altieri: Unfortunately, I think, I mean, I don't know for sure. But I think that dealing with this particular molecule, heat shock protein 90 in the clinic has been difficult. Hsp90 has long been recognized as an important cancer target. There have been several generations of small molecule inhibitors that have been tested in the clinic. And unfortunately, I hope I'm not offending anybody, but unfortunately, the clinical results of those studies, and some of them moved all the way to really large phase two trials have been disappointing. And so, that is the idea that perhaps this was a dead target. And therefore, trying to leverage industry or biotechnology interest around it was quite a remarkable challenge. Dr. Lidia Schapira: What message do you want the young investigators to take away from your story in terms of the collaboration between academia where thoughts start, as you say, in your article, and all of the rest of the partners that you actually need it to bring this discovery and this idea to fruition? Dr. Dario Altieri: Lydia, this may sound trite, I really hope to convey one simple notion. It's not even a message, it's a very personal account. And that is don't give up. If you have run the controls. If you have done your experiments enough time. If you're convinced of the results, if you explore alternative explanations, and you keep coming back to the same conclusions, go for it. That has been a little bit of my own personal experience and if there are things that you don't know about, that's perfectly fine. Actually, that is the fun of the process, and the things that I didn't know about drug development, I can fill in the encyclopedia. I've learned some of them through people who have been doing this for a living, for a very long time. And that has been truly inspiring for me, a life lesson and professional lesson about how we can think of a drug target that has been discounted and remain true to the core value of strong basic research and try to advance that to the clinic, whether this will ever become something useful for our patients? I don't have the faintest idea. I certainly hope so. But that would be the experiment that is being done right now in the clinic. Dr. Lidia Schapira: In your article towards the end, you just give us two little glimpses into something that is personal and meaningful to you by telling us that there's an anniversary of a loss, the passing of your mother from cancer. Can you tell us a little bit more about that, and why you chose to put that sentence just where you did? Dr. Dario Altieri: I didn't know if anybody would have noticed, frankly, so I appreciate you bringing it up, Lidia. It's been a very personal journey for me as well. Both my parents died of lung cancer. They were a different generation. Both were heavy smokers. I remember those dates very well and I remember the void that they're passing is created. And so, I thought it was an interesting circumstance, that in fact, the first patient was enrolled in a clinical trial, the notion about that and of course, I am technically conflicted. So, I am not supposed to know anything about what is happening in the clinic. But it was interesting that the first notion about the first dosing came on that day, on January 10. Dr. Lidia Schapira: Well, I'm sure other readers will notice that too, the timing of that in the article and the fact that there was some emotion implied, I think, in how you chose to end your story by saying that this had happened in the clinic, but somehow, you were not there, that you had to be removed. Tell us a little bit more about that, about why you needed to be removed from the clinical site and why do you talk about yourself as a former doctor? In my mind, once you are you always are, but somehow you feel that you need to make the distinction. What does it all mean to you? Dr. Dario Altieri: Well, Lidia, let me just say you don't want me in the clinic right now. At 64years of age, like I said that the last patient was a very long time ago. I have to say, sometimes I miss those days, just as a personal account. I need to be removed because I'm technically conflicted on the trial, I was the IND holder, and then the FDA asked me to transfer the IND to the clinical investigator as proper because I'm not involved in patient care or research, in this particular case. And technically, because I am the inventor on a patent, I could potentially stand to benefit financially from the results of the trial, something that is certainly not on my mind, but that I have been reminded of. And so, I try to stay away as much as I can. Obviously, I think about this every day. But whatever information I can gain, that I can gather from my colleagues across town will be wonderful, but I'm not the one initiating those calls. Dr. Lidia Schapira: So back to the humorous side of your essay, you say that you've learned to be a telephone operator and a mailman, and a whole bunch of other things. Have those lessons been useful to the other aspects of your life? Or do you see that as a total waste of your time? Dr. Dario Altieri: Not at all. Not at all. I have been an incredible component and I think I was trying to be humorous and to take myself seriously, but not too seriously. But in fact, maintaining that level of interaction, particularly with aspects of the work that I've never encountered, for instance, regulatory aspects of an early-stage clinical trial with the Food and Drug Administration, that has been part of the life journey and I only have very good things to say about my experience. You know, it's been interesting, Lidia, being part of the experience of being a telephone operator and a mailman. I had this sense, and I could be completely wrong, but I had this sense that people out there want to see us taking small risks. They want to see testing new drugs, they want to see new targets being somehow examined, developed, if at all possible. I had the sense that there was support, you know, for the idea, and this was an entirely publicly funded program. I funded both the preclinical and now the clinical trial of Gamitrinib out of the American taxpayer's commitment and in many different study sections, in dealing with the FDA, in dealing with other regulatory consultants, I always get the sense people who wanted to help, then had perhaps the mindset, okay, we don't know whether this is going to work or not but let's give it a try. Let's give it a shot. It was wonderful, that was an absolutely awe-inspiring experience. Dr. Lidia Schapira: I'm glad they did and I'm glad you shared your experience with all of us. Is there something else that you'd like our listeners or your readers to know about you or this story? Dr. Dario Altieri: I just would like to say that I would do it again, 12 years, I would do every step of the way but I think I'm done. If I were to start over, I'll do it again, but I don't think I'm ready to do it again with another target. Dr. Lidia Schapira: And with that, I want to thank you and I want to thank our listeners. Until next time, thank you for listening to this JCO's Cancer Stories: The Art of Oncology podcast. If you enjoyed what you heard today, don't forget to give us a rating or review on Apple podcasts or wherever you listen. While you're there, be sure to subscribe so you never miss an episode of JCO's Cancer Stories: The Art of Oncology podcast. This is just one of many of ASCO's podcasts. You can find all of the shows at the podcast.asco.org. The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product or service organization activity or therapy should not be construed as an ASCO endorsement.

BioVaxys Technology Corp. founder and chief medical officer Dr David Berd joined Proactive's Stephen Gunnion with news that the company has started its planed in vivo animal toxicology study of CoviDTH. Berd telling Proactive that the study will run parallel to the company's preparation of an Investigational New Drug (IND) submission to the US Food and Drug Administration.

The Coronavirus pandemic has put a spotlight on drug supply and the approval process of new medications. In this episode, we will discuss what frontline pharmacists need to know to about the drug approval process.    Guest speaker: Amanda Melton, PharmD, BCPS Senior Clinical Manager Center for Pharmacy Practice Excellence Vizient   Moderator: Gretchen Brummel, PharmD, BCPS Pharmacy Executive Director Vizient Center for Pharmacy Practice Excellence   Show Notes: [01:20] Patients accessing drugs through clinical trials [02:29] Pharmacists should familiarize themselves with the inclusion and exclusion criteria of clinical trials in order to help clinicians identify good patients candidates [03:00] ClinicalTrials.gov [3:17] Patients that don't qualify for a clinical trial can apply for access through an Expanded Access Program (EAP)   [3:35] What Pharmacists need to know about Expanded Access Programs (EAPs) [4:26] Goal of the EAP is to get the drug to the patient [5:03] Step by step process for accessing a drug under the Expanded Access Program (EAP) [5:42] Investigational New Drug (IND) application [6:30] Four stakeholders: manufacturer, FDA, patient and Institutional Revenue Board (IRB) [6:50] Getting access to the FDA by using Form 3926 [7:54] Timing for submitting an EAP [9:00] How Emergency Use Authorizations (EUAs) differ from Expanded Access Programs (EAPs)  [10:00] How long an EUA lasts [10:41] When an Emergency Use Authorization (EUA) is issued, it does not mean the drug is approved [11:15] Why apply for an EUA, instead of a biologics license [12:07] Issuance of an EUA for a product doesn't preclude the manufacturer from seeing approval for the product   Links | Resources: FDA: Emergency Use Authorization Click here FDA: Expanded Access Click here NIH U.S. Library of Medicine: ClinicalTrials.gov Click here Reagan-Udall Foundation for the FDA: COVID-19 Hub Click here   Subscribe Today! Apple Podcasts Google Podcasts Spotify Stitcher Android RSS Feed  

CNS Pharmaceuticals (NASDAQ: CNSP) (“CNS” or the “Company”), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, today announced that the Investigational New Drug (IND) application for its lead product candidate, Berubicin, for the treatment of Glioblastoma Multiforme (GBM) is now approved and in effect as filed with the US Food and Drug Administration (FDA). The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy.  Recent correspondence between the Company and the FDA resulted in modifications to the previously disclosed trial design, including designating overall survival (OS) as the primary endpoint of the study. OS is a rigorous endpoint that the FDA has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm. To view the original press release, visit https://nnw.fm/zWHqI

CNS Pharmaceuticals (NASDAQ: CNSP), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, recently appeared on Gamechangers LIVE, a podcast series that shines a spotlight on gamechangers in various industries, sharing perspective on journeys, mindsets, struggles and successes in an effort to inspire and inform listeners. During the interview, CNSP's chairman and CEO, John Climaco, provided an overview of CNS Pharmaceuticals and its lead drug candidate, Berubicin. Proposed for the treatment of glioblastoma multiforme (“GBM”), an aggressive and incurable form of brain cancer, Berubicin was evaluated in a Phase I clinical trial. As a result of the trial, 44% of patients experienced a clinical benefit of stable disease or better, including one patient who experienced a durable complete response and remains cancer free 14 years after treatment. CNS recently submitted an Investigational New Drug (“IND”) application with the FDA, and, pending approval, it intends to commence a pivotal Phase II clinical trial in the U.S. “I have done all kinds of different things in my career, been in the health care industry for a long time. This is the most exciting thing that I've done,” Climaco said in the interview. “This company and our lead product have the potential to really change the landscape and the game in an area of oncology that hasn't seen a lot of hope for a very long time in glioblastoma.” To view the full press release, visit https://nnw.fm/jfjZB   CNS Pharmaceuticals (NASDAQ: CNSP) is developing novel treatments for primary and metastatic cancers of the brain and central nervous system. Its lead drug candidate, Berubicin, is proposed for the treatment of glioblastoma multiforme (GBM), an aggressive and incurable form of brain cancer. CNS holds a worldwide exclusive license to the Berubicin chemical compound and has acquired all data and know-how from Reata Pharmaceuticals Inc. related to a completed Phase 1 clinical trial with Berubicin in malignant brain tumors, which Reata conducted in 2006. In this trial the overall response rate of stable disease or better was 44%. This 44% disease control rate was based on 11 patients (out of 25 evaluable patients) with stable disease, plus responders. One patient experienced a durable complete response and remains cancer-free as of February 20, 2020. These Phase 1 results represent a limited patient sample size and, while promising, are not a guarantee that similar results will be achieved in subsequent trials. Its second drug candidate, WP1244, is a novel DNA binding agent that has shown in preclinical studies that it is 500 times more potent than the chemotherapeutic agent daunorubicin in inhibiting tumor cell proliferation. 

CNS Pharmaceuticals (NASDAQ: CNSP), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, recently appeared on Gamechangers LIVE, a podcast series that shines a spotlight on gamechangers in various industries, sharing perspective on journeys, mindsets, struggles and successes in an effort to inspire and inform listeners. During the interview, CNSP's chairman and CEO, John Climaco, provided an overview of CNS Pharmaceuticals and its lead drug candidate, Berubicin. Proposed for the treatment of glioblastoma multiforme (“GBM”), an aggressive and incurable form of brain cancer, Berubicin was evaluated in a Phase I clinical trial. As a result of the trial, 44% of patients experienced a clinical benefit of stable disease or better, including one patient who experienced a durable complete response and remains cancer free 14 years after treatment. CNS recently submitted an Investigational New Drug (“IND”) application with the FDA, and, pending approval, it intends to commence a pivotal Phase II clinical trial in the U.S. “I have done all kinds of different things in my career, been in the health care industry for a long time. This is the most exciting thing that I've done,” Climaco said in the interview. “This company and our lead product have the potential to really change the landscape and the game in an area of oncology that hasn't seen a lot of hope for a very long time in glioblastoma.” To view the full press release, visit https://nnw.fm/jfjZB   CNS Pharmaceuticals (NASDAQ: CNSP) is developing novel treatments for primary and metastatic cancers of the brain and central nervous system. Its lead drug candidate, Berubicin, is proposed for the treatment of glioblastoma multiforme (GBM), an aggressive and incurable form of brain cancer. CNS holds a worldwide exclusive license to the Berubicin chemical compound and has acquired all data and know-how from Reata Pharmaceuticals Inc. related to a completed Phase 1 clinical trial with Berubicin in malignant brain tumors, which Reata conducted in 2006. In this trial the overall response rate of stable disease or better was 44%. This 44% disease control rate was based on 11 patients (out of 25 evaluable patients) with stable disease, plus responders. One patient experienced a durable complete response and remains cancer-free as of February 20, 2020. These Phase 1 results represent a limited patient sample size and, while promising, are not a guarantee that similar results will be achieved in subsequent trials. Its second drug candidate, WP1244, is a novel DNA binding agent that has shown in preclinical studies that it is 500 times more potent than the chemotherapeutic agent daunorubicin in inhibiting tumor cell proliferation. 

CNS Pharmaceuticals (NASDAQ: CNSP) (“CNS” or the “Company”), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, today announced that it has submitted an Investigational New Drug (“IND”) application, which has been accepted for review, to the U.S. Food and Drug Administration (“FDA”) for Berubicin in the treatment of Glioblastoma Multiforme (“GBM”). The Company plans to evaluate the efficacy of Berubicin in a Phase 2 Trial for adults with GBM who have failed first-line therapy and commence the trial within the first quarter of 2021, pending the FDA's acceptance of the Company's filing. To view the original press release, visit https://nnw.fm/1Qib1

CNS Pharmaceuticals (NASDAQ: CNSP) (“CNS” or the “Company”), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, today announced that it has submitted an Investigational New Drug (“IND”) application, which has been accepted for review, to the U.S. Food and Drug Administration (“FDA”) for Berubicin in the treatment of Glioblastoma Multiforme (“GBM”). The Company plans to evaluate the efficacy of Berubicin in a Phase 2 Trial for adults with GBM who have failed first-line therapy and commence the trial within the first quarter of 2021, pending the FDA's acceptance of the Company's filing. To view the original press release, visit https://nnw.fm/1Qib1

What We Covered00:44 – Ed, Brian and Meranda join the show to share their experiences in all phases of the pharmaceutical development industry5:12 – The process of filing an Investigational New Drug (IND) and a New Drug Application (NDA) 18:54 – What it means to have ‘rest-of-world awareness' as you author an NDA 22:37 – Ed, Brian and Meranda speak to the importance of storyboards and telling a story from the right perspective 28:55 – Ed, Brian and Meranda break down the detailed development process, including the work that Design Space InPharmatics does 39:28 – The document review process, timelines for submissions and tracking projects 49:00 – Ed, Brian and Meranda conclude the podcast and thank the audience for listening Tweetable Quotes“Filing an IND is important to get a trial started. And folks start clinical trials for a variety of reasons.” (05:43) (Ed)“Out of academia we see a lot of this very little information filed into an IND, and then very little information generated up to the point where you get to a Phase Two or Three where you see efficacy data and then suddenly you have to file an NDA.” “It's hard to become an expert. There's no real training. I think a lot of it is just experience. It's going into meetings with FDA when you don't have all the data.”“Make sure it looks good, because it does reflect on the actual data and content in there, just like anything in life.” “As the author goes through the process with the client, there's still another piece. And that's the publishing group.” “One way to alleviate that [stress] is to be able to give an update on any given subject or any given section at any given time.” Relevant LinksDesign Space InPharmatics - LinkedInDesign Space InPharmatics - TwitterEdward Narke on LinkedInBrian Lihou on LinkedInMeranda Parascandola on LinkedIn

By Michael Tetreault and Dr. Joshua Hare Today on the DocPreneur Leadership Podcast at Concierge Medicine Today we sit down with Dr. Joshua Hare Chief Science Officer at Longeveron, a life sciences company developing biological solutions for aging and aging-associated diseases through the testing of allogeneic human Mesenchymal Stem Cells (MSCs) that are derived from the bone marrow of young, healthy donors. Dr. Hare is also the Founding Director of the University of Miami Interdisciplinary Stem Cell Institute (ISCI). Through his work with ISCI, Hare has made significant advances in the field of stem cell regenerative medicine, including the creation of relevant intellectual property. Dr. Joshua Hare co-founded Longeveron in 2014 utilizing intellectual property and technology exclusively licensed from the University of Miami, where he is also the Founding Director of the university's Interdisciplinary Stem Cell Institute (ISCI). Through his work with ISCI, Hare has made significant advances in the field of stem cell regenerative medicine, including the creation of relevant intellectual property. A graduate of the University of Pennsylvania (1984) and the Johns Hopkins University School of Medicine (1988), Hare completed fellowships at Johns Hopkins and Brigham and Women's Hospital in Boston, Ma. He served as a research fellow at Harvard University and is Board Certified in cardiovascular medicine. During his career, Hare has championed the field of allogeneic cell therapy, and he has conducted five clinical trials using allogenic cell therapy. Hare's publications in the area of cell-based therapy are widely cited, and he holds six Investigational New Drug (IND) authorizations from the FDA for cell therapy drugs and treatments in patients with heart disease. Hare co-founded Longeveron as a means to bring his academic findings to patients with unmet medical needs. Hare's extensive list of accolades and professional experience includes: Director, Interdisciplinary Stem Cell Institute (ISCI) Louis Lemberg Professor of Medicine (cardiology), Leonard M. Miller School of Medicine, University of Miami Professor of Biomedical Engineering Professor of Molecular and Cellular Pharmacology Professor of Cell Biology and Anatomy Nominated in 2011 to the Association of American Physicians Stem cell therapy is emerging as a promising new treatment option for chronic diseases and injuries affecting various organ systems. One of the most exciting ideas emerging from the field of regenerative medicine is the theory that stem cells can treat aging-related disability and frailty, reducing inflammation and improving functional capacity and quality of life for human beings. Stem cells have the potential to increase longevity and could ameliorate diseases and disorders associated with aging. Currently there are no FDA-approved allogeneic mesenchymal stem cell treatments in the United States. Longeveron's LMSCs are currently undergoing rigorous testing for safety and efficacy in Phase I and II clinical trials. They hope to be the first to offer stem cells as a safe, effective, FDA-approved treatment for some of the world's most difficult chronic and life-threatening conditions such as Aging Frailty, Alzheimer's disease and the Metabolic Syndrome.