June IPOs to Watch in Tech, Biotech, Automotive and Mining (Nasdaq: $LIF) (Nasdaq: $WAY) (Nasdaq: $GAUZ) (Nasdaq: $FLYE) (Nasdaq: $RAPP) (Nasdaq: $TEM) (TSXV: $ONAU.V)

 June IPOs to Watch in Tech, Biotech, Automotive and Mining (Nasdaq: $LIF) (Nasdaq: $WAY) (Nasdaq: $GAUZ) (Nasdaq: $FLYE) (Nasdaq: $RAPP) (Nasdaq: $TEM) (TSXV: $ONAU.V)

 

 

 

 

July 12, 2024 – (Investorideas.com Newswire) Investorideas.com, a global news source and expert investing resource, announces today’s roundup of stocks – all recent IPO’s.

 

This month’s IPOs cover the tech, biotech, transportation and mining industry sectors.

 

Read this in full at https://www.investorideas.com/news/2024/main/07121Stocks.asp

 

Investor Ideas is always researching and searching for new stocks to add to our growing list of free stock directories. The directories are not meant as recommendations but as a research tool to discover opportunities and trading ideas in a particular sector.”

 

New Stocks Added to the Tech Directories:

Life360, Inc. (Nasdaq:LIF) a family connection and safety company, keeps people close to the ones they love. The category-leading mobile app and Tile tracking devices empower members to stay connected to the people, pets, and things they care about most, with a range of services, including location sharing, safe driver reports, and crash detection with emergency dispatch. Life360 delivers peace of mind and enhances everyday family life in all the moments that matter, big and small.

 

Waystar Holding Corp. (Nasdaq:WAY) mission-critical software is purpose-built to simplify healthcare payments so providers can prioritize patient care and optimize their financial performance. Waystar serves approximately 30,000 clients, representing over 1 million distinct providers, including 18 of 22 institutions on the U.S. News Best Hospitals list. Waystar's enterprise-grade platform annually processes over 5 billion healthcare payment transactions, including over $1.2 trillion in annual gross claims and spanning approximately 50% of U.S. patients. Waystar strives to transform healthcare payments so providers can focus on what matters most: their patients and communities.

 

New Stocks Added to the Automotive/Transportation Directories:

Gauzy Ltd. (Nasdaq:GAUZ) is a fully-integrated light and vision control company, focused on the research, development, manufacturing, and marketing of vision and light control technologies that are developed to support safe, sustainable, comfortable, and agile user experiences across various industries. Headquartered in Tel Aviv, Israel, the Company has additional subsidiaries and entities based in Germany, France, the United States, Canada, China, Singapore, and Dubai. Gauzy serves leading brands in over 30 countries through direct fulfillment and a certified and trained distribution channel. Automotive grade LCG® smart glass technologies support glare mitigation, thermal comfort, interior protection, and transparent displays for an array of applications in passenger and commercial vehicles. Fully customized turn-key shading solutions provided as a Tier 1 with LCG® smart glass, or traditional shades supporting on demand cabin shading, ambiance, and cockpit shading for commercial and private jets.

 

Fly-E Group, Inc. (Nasdaq:FLYE) is an electric vehicle company that is principally engaged in designing, installing and selling smart electric motorcycles, electric bikes, electric scooters and related accessories under the brand “Fly E-Bike.” Our commitment is to encourage people to incorporate eco-friendly transportation into their active lifestyles, ultimately contributing towards building a more environmentally friendly future.

 

New Stocks Added to the Biotech Directories:

Rapport Therapeutics, Inc. (Nasdaq:RAPP)         is a clinical-stage biotechnology company dedicated to discovering and developing transformational precision neuromedicines for patients suffering from central nervous system (CNS) disorders. The Company’s founders have made pioneering discoveries related to the function of receptor associated proteins (RAPs) in the brain. Their findings form the basis of Rapport’s RAP technology platform, which enables a differentiated approach to generate precision small molecule product candidates with the potential to overcome many limitations of conventional neurology drug discovery. Rapport’s precision neuroscience pipeline includes the Company’s lead clinical program, RAP-219, designed to achieve neuroanatomical specificity through its selective targeting of a RAP expressed in only discrete regions of the brain. The Company is currently advancing RAP-219 in clinical trials in focal epilepsy, peripheral neuropathic pain, and bipolar disorder. Additional preclinical and late-stage discovery stage programs are also underway, targeting CNS disorders including chronic pain and hearing disorders.

 

Tempus AI, Inc. (Nasdaq:TEM) is a technology company advancing precision medicine through the practical application of artificial intelligence in healthcare. With one of the world’s largest libraries of multimodal data, and an operating system to make that data accessible and useful, Tempus provides AI-enabled precision medicine solutions to physicians to deliver personalized patient care and in parallel facilitates discovery, development and delivery of optimal therapeutics. The goal is for each patient to benefit from the treatment of others who came before by providing physicians with tools that learn as the company gathers more data.

 

New Stocks Added to the Mining Directories:

OnGold Resources Ltd. (TSXV:ONAU) (formerly 1348515 B.C. Ltd.) is a reporting issuer in the provinces of British Columbia and Alberta with no current activities or operations. ONGold owns significant exploration assets in Northern Ontario, highlighted by the district-scale TPK Project and October Gold Project. These projects represent a strategic footprint in one of Canada's most prolific gold-producing regions.

 

About Investorideas.com - Big Investing Ideas

Investorideas.com is the go-to platform for big investing ideas. From breaking stock news to top-rated investing podcasts, we cover it all. Our original branded content includes podcasts such as Exploring Mining, Cleantech, Crypto Corner, Cannabis News, and the AI Eye. We also create free investor stock directories for various sectors, including gaming, biotech, tech and sports. Public companies within the sectors we cover can use our news publishing and content creation services to help tell their story to interested investors. Paid content is always disclosed.

 

Disclaimer/Disclosure: Investorideas.com is a digital publisher of third party sourced news, articles and equity research as well as creates original content, including video, interviews and articles. Original content created by investorideas is protected by copyright laws other than syndication rights. Our site does not make recommendations for purchases or sale of stocks, services or products. Nothing on our sites should be construed as an offer or solicitation to buy or sell products or securities. All investing involves risk and possible losses. This site is currently compensated for news publication and distribution, social media and marketing, content creation and more. Disclosure is posted for each compensated news release, content published /created if required but otherwise the news was not compensated for and was published for the sole interest of our readers and followers. Contact management and IR of each company directly regarding specific questions. More disclaimer info: https://www.investorideas.com/About/Disclaimer.asp Learn more about publishing your news release and our other news services on the Investorideas.com newswire https://www.investorideas.com/News-Upload/

Global investors must adhere to regulations of each country. Please read Investorideas.com privacy policy: https://www.investorideas.com/About/Private_Policy.asp

 

Learn more about our news, PR and social media, podcast and ticker tag services at Investorideas.com

https://www.investorideas.com/Investors/Services.asp

 

Learn more about advertising and guest posts

https://www.investorideas.com/Advertise/

 

Follow us on Twitter https://twitter.com/Investorideas

Follow us on Facebook https://www.facebook.com/Investorideas

Follow us on YouTube https://www.youtube.com/c/Investorideas

 

Contact Investorideas.com to be added to our stock directories or learn more about us

Dawn Van Zant and Cali Van Zant

 

800 665 0411

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Bloodstream Infections – From Threats to Solutions; Stocks to Watch: Citius Pharmaceuticals, Inc. (Nasdaq: $CTXR), BD (Becton, Dickinson and Company) (NYSE: $BDX), CorMedix Inc. (Nasdaq: $CRMD), Vir Biotechnology, Inc. (Nasdaq: $VIR), Abbott Laboratories (NYSE: $ABT)

 

Bloodstream Infections – From Threats to Solutions; Stocks to Watch: Citius Pharmaceuticals, Inc. (Nasdaq: $CTXR), BD (Becton, Dickinson and Company) (NYSE: $BDX), CorMedix Inc. (Nasdaq: $CRMD), Vir Biotechnology, Inc. (Nasdaq: $VIR), Abbott Laboratories (NYSE: $ABT)

 

July 11, 2024 - Investorideas.com, a go-to investing platform releases the second of a two-part series looking at recent news and developments in the blood infections treatment market, featuring Citius Pharmaceuticals, Inc. (Nasdaq:CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products. The Company's diversified pipeline includes two late-stage product candidates.

 

“Bloodstream infections (BSI) are a growing threat to public health worldwide. The 2 million cases of BSIs that occur annually in North America and Europe are associated with 250,000 deaths, making BSIs the leading cause of mortality from infection”, says a recent report in the Journal of Clinical  Microbiology.

 

Citius Pharmaceuticals, Inc. (Nasdaq:CTXR) just announced expected milestones in the second half of calendar 2024 and recapped key achievements year-to-date.

 

Citius reported it has significantly advanced its two late-stage product candidates in the first six months of 2024, and expects several key milestones in the near term.

 

Paid news dissemination for Citius Pharmaceuticals.

 

Read this article, featuring CTXR in full at: https://www.investorideas.com/news/2024/biotech/07114Bloodstream-Infections-Stocks.asp

 

Citius recently announced positive topline data from the Phase 3 study of Mino-Lok® in catheter-related bloodstream infections (CRBSIs). Primary and secondary endpoints were met with statistical significance.

 

Next steps for the Mino-Lok program are to prepare a submission to the U.S. Food and Drug Administration (FDA) and schedule a Type B meeting.  

 

Mino-Lok (MLT), a novel antibiotic lock solution that combines minocycline, ethanol and edetate disodium, is designed to treat patients with catheter-related blood stream infections. Citius licensed Mino-Lok from an affiliate of The University of Texas MD Anderson Cancer Center. Mino-Lok is designed to offer an alternative to removing and replacing a central venous catheter (CVC), which may lead to a reduction in serious adverse events and cost savings to the healthcare system. If approved, Mino-Lok would be the first and only FDA-approved treatment that salvages central venous catheters that cause central line-related blood stream infections.

 

In late May, Citius announced positive topline results of its pivotal Phase 3 clinical trial of Mino-Lok®, a novel antibiotic lock solution designed to salvage catheters in patients with central line-associated infections (CLABSI) or catheter-related bloodstream infections (CRBSI). The study met its primary endpoint with a statistically significant improvement in the time to failure event in patients receiving Mino-Lok compared to Control arm patients receiving clinician-directed anti-infective lock solution. The data demonstrate that Mino-Lok is well-tolerated.

 

"We are extremely pleased by the strong results of the trial, which demonstrate the safety and efficacy of Mino-Lok in preserving indwelling catheters in patients with bloodstream infections. The data indicate, with a high degree of statistical significance, that Mino-Lok outperforms anti-infective lock solutions utilized in patients with CLABSI or CRBSI in the study's control arm. We believe Mino-Lok could potentially set a new standard of care (SOC) as an adjunct therapy in the treatment of patients with bloodstream infections. This therapy offers a non-invasive treatment option compared to catheter removal and replacement," stated Leonard Mazur, Chairman and Chief Executive Officer of Citius.

 

"We look forward to engaging with the US Food and Drug Administration (FDA) to determine the optimal path forward for Mino-Lok. Our focus remains on improving outcomes for patients and offering a much-needed alternative to the current practice of catheter removal and replacement," added Mazur.

 

Mino-Lok Phase 3 Trial Design

 

MDA 2013-0039 (NCT02901717) is a completed Phase 3 multicenter, randomized, open-label, active-controlled assessor-blinded trial conducted to evaluate the safety and efficacy of Mino-Lok as an adjunctive therapy to systemic anti-infectives for the treatment of catheter-related bloodstream infections (CRBSI) and central line-associated infections (CLABSI).

 

More from the news: A total of 241 patients in the U.S. and India, with an indwelling central venous catheter before the onset of a bloodstream infection, were randomized in a 1:1 ratio to receive either Mino-Lok or SOC site-specific anti-infective lock solution plus SOC systemic antibiotics. Enrollment was not limited to patients with specific types of catheters. Patients in the Mino-Lok arm received one Mino-Lok dose daily with a dwell time of two to four hours for a total of seven doses over a period of up to 15 days.

 

The primary endpoint for this study is the time to a catheter failure event between randomization and test of cure (TOC) at six weeks, measured in days following randomization. Additional secondary outcome measures include overall success, microbiological eradication, and clinical cure, among others.

 

For subjects in the Control arm, the investigator determined the antibiotic used in the lock, dose, dwell time, and number of days of administration based on institutional standards or Infectious Diseases Society of America (IDSA) guidelines.

 

TOPLINE RESULTS

 

Efficacy:

The primary endpoint in the study was time to catheter failure between randomization and six-weeks following the first dose of Mino-Lok or SOC lock solution. Catheter failure was defined as the inability to administer study lock solution, catheter removal for any infection-related reason (including worsening clinical signs and symptoms or persistence or recurrence of baseline pathogen, or new infection), and all-cause mortality.

 

Results of blinded-assessor clinical outcome analyses in all randomized patients demonstrated that Mino-Lok achieved its primary endpoint:

 

Time to catheter failure events in patients receiving Mino-Lok therapy occurred substantially later than in patients in the Control arm (p value = 0.0006)

 

Median time-to-failure (MTF) of Control arm: 33 days; 95% confidence interval (CI) (14 days – 44 days; n = 122)

 

Median time-to-failure (MTF) of Mino-Lok arm: MTF exceeded the time the patients were on trial and therefore not estimable (NE); 95% CI (50 days - NE; n = 119)

 

The hazard ratio of this study was 0.53

 

The critical secondary endpoints included the proportion of patients at six weeks with overall treatment success including no catheter failure, clinical cure (absence of baseline clinical signs and symptoms of infection or improvement of clinical signs and symptoms such that no additional therapy was necessary), or microbiological eradication (absence of the baseline pathogen).

 

Results in all randomized patients who received at least one dose of study lock solution demonstrate overall treatment success in 57.1% of patients in the Mino-Lok arm and 37.7% of patients in the Control arm (p=0.0025).

 

Safety:

Mino-Lok is designed for intracatheter instillation and is not for intravenous injection or systemic circulation.

 

No serious adverse events were drug-related

 

Serious adverse events occurred in 45.1% of patients in the Mino-Lok arm and 46.1% of patients in the Control arm

 

BD (Becton, Dickinson and Company) (NYSE:BDX), a leading global medical technology company, today issued the following statement related to a US FDA letter to health care providers regarding the supply of blood culture vials:

 

Nikos Pavlidis, worldwide president of BD Diagnostic Solutions said, "BD is experiencing reduced availability of blood culture vials from our supplier. We understand the critical role that blood culture testing plays in diagnosing and treating infections and are taking all available measures to address this important issue, including providing the supplier our manufacturing expertise, using air shipments, modifying BD manufacturing schedules for rapid production, and collaborating with the U.S. Food and Drug Administration to review all potential options to mitigate delays in supply. As an additional stopgap measure, our former supplier of glass vials will restart production to help fill the intermittent gap in supply.

 

"We take our responsibility to support health care providers and patients very seriously and, while we are able to meet the majority of global demand, we are taking all available measures to address this important issue. BD BACTEC™ blood culture vials are being allocated manually so we can best mitigate any impact to patient care. Customers have been advised to prioritize the use of blood culture vials based on clinical needs and following guidelines, such as the most recent update from the Infectious Disease and Society of America and/or the World Health Organization.

 

"While this supplier issue is not expected to have a material financial impact on BD, we are fully focused on returning the supply of blood culture vials to normal levels."

 

CorMedix Inc. (Nasdaq:CRMD), a biopharmaceutical company focused on developing therapeutic products for life-threatening diseases and conditions, recently announced that it has commenced commercialization of DefenCath® (taurolidine and heparin) to outpatient dialysis centers and vascular access clinics where patients receive outpatient dialysis and catheter placement services. The outpatient launch coincides with the effective date of July 1st for outpatient reimbursement of DefenCath® as directed by the Center for Medicare & Medicaid Services (CMS).

 

Late last year, CorMedix announced that the US Food and Drug Administration (FDA) had approved DefenCath® (taurolidine and heparin) catheter lock solution (CLS) to reduce the incidence of catheter-related bloodstream infections (CRBSIs) for the limited population of adult patients with kidney failure receiving chronic hemodialysis through a central venous catheter (CVC). DefenCath® is the first and only FDA-approved antimicrobial CLS in the U.S. and was shown to reduce the risk of CRBSIs by up to 71% in a Phase 3 clinical study.

 

Vir Biotechnology, Inc. (Nasdaq:VIR), an immunology company focused on powering the immune system to transform lives by treating and preventing infectious diseases and other serious conditions, including viral-associated diseases, recently announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application and granted Fast Track designation for the combination of tobevibart and elebsiran for the treatment of chronic hepatitis delta infection. Tobevibart, an investigational monoclonal antibody, and elebsiran, an investigational small interfering ribonucleic acid, are currently being evaluated in the Company’s Phase 2 SOLSTICE hepatitis delta clinical trial, with complete 24-week treatment data on track to be reported in the fourth quarter.

 

"The IND clearance and Fast Track designation from the FDA, along with the encouraging preliminary data from our Phase 2 hepatitis delta trial, underscore the potential of tobevibart and elebsiran to transform the treatment landscape for people living with this severe and life-threatening disease," said Marianne De Backer, M.Sc., Ph.D., MBA, Vir’s Chief Executive Officer. "We are committed to working closely with health authorities to bring this potential groundbreaking treatment to patients as quickly as possible, addressing a critical unmet medical need."

 

Some of the biggest pharma companies including Abbott Laboratories (NYSE:ABT) are grabbing market share in the blood infections testing market, estimated at over 875 Million in 2023.

 

From testing to treatments, the life threatening bloodstream infection market is seeing breakthroughs in technology to answer the call.

 

Research and find more biotech stocks at Investorideas.com

https://www.investorideas.com/BIS/Stock_List.asp

About Investorideas.com - Big Investing Ideas

Investorideas.com is the go-to platform for big investing ideas. From breaking stock news to top-rated investing podcasts, we cover it all. Our original branded content includes podcasts such as Exploring Mining, Cleantech, Crypto Corner, Cannabis News, and the AI Eye. We also create free investor stock directories for sectors including mining, crypto, renewable energy, gaming, biotech, tech, sports and more. Public companies within the sectors we cover can use our news publishing and content creation services to help tell their story to interested investors. Paid content is always disclosed.

 

Disclaimer/Disclosure: This news article featuring Citius Pharmaceuticals, Inc. (CTXR) a paid for news release creation and dissemination on Investorideas.com.  Our site does not make recommendations for purchases or sale of stocks, services or products.  This is not investment opinion: Nothing on our sites should be construed as an offer or solicitation to buy or sell products or securities. All investing involves risk and possible losses. More disclaimer info: https://www.investorideas.com/About/Disclaimer.asp Learn more about publishing your news release and our other news services and prices on the Investorideas.com newswire https://www.investorideas.com/News-Upload/ Global investors must adhere to regulations of each country. Please read Investorideas.com privacy policy: https://www.investorideas.com/About/Private_Policy.asp

 

Follow us on X @investorideas

Follow us on Facebook https://www.facebook.com/Investorideas

Follow us on YouTube https://www.youtube.com/c/Investorideas

 

Contact Investorideas.com

800-665-0411

Biotech Industry Stocks- investing ideas in biotechnology stocks, medical technology and life sciences

Get more biotech and medical tech news, articles, podcasts and stock directories

 

 

Transformative Biopharma Stocks - Citius Pharmaceuticals (Nasdaq: $CTXR), Vertex Pharmaceuticals (Nasdaq: $VRTX), CRISPR Therapeutics (Nasdaq: $CRSP), Recursion (NASDAQ: $RXRX)

 

Transformative Biopharma Stocks - Citius Pharmaceuticals (Nasdaq: $CTXR), Vertex Pharmaceuticals (Nasdaq: $VRTX), CRISPR Therapeutics (Nasdaq: $CRSP), Recursion (NASDAQ: $RXRX)

 

July 11, 2024 - Investorideas.com, a go-to investing platform releases the first of a two-part series looking at transformative innovation in biopharma, featuring Citius Pharmaceuticals, Inc. (Nasdaq:CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products. The Company's diversified pipeline includes two late-stage product candidates.

 

The global biopharmaceutical market size was USD 411.4 Billion in 2022, 436.7 Billion in 2023 and analysts say it will grow at 15, 4% from 2024 to 2033.

 

Looking at key trends in the sector, including AI and CRISPR, Labiotech reports, “2024 stands as a pivotal year that could potentially mark a turning point in the biotech industry. With the right blend of innovation, collaboration, and regulatory oversight, the industry is well-positioned to harness these emerging trends and transform the landscape of healthcare and therapeutic interventions. The future of biotech is not just about technological advancements, but also about how these technologies are integrated responsibly and ethically into our healthcare systems to create a healthier, more sustainable world.”

 

Citius Pharmaceuticals, Inc. (Nasdaq:CTXR) just announced expected milestones in the second half of calendar 2024 and recapped key achievements year-to-date.

 

Leonard Mazur, Chairman and CEO of Citius stated, "Our solid execution since the beginning of the year sets us up for potentially transformative catalysts in the coming months. Mino-Lok's strong Phase 3 topline results support its potential to become part of the standard of care for treating catheter-related bloodstream infections. Mino-Lok would also have a first and only advantage in a market with no approved or investigational products for salvaging infected central venous catheters. For LYMPHIR™, the FDA is currently reviewing our Biologics License Application, with an expected decision on August 13th. We are preparing for near-term commercialization of LYMPHIR if approved. These near-term catalysts should enable the company to optimize its current cash runway, future cash needs, as well as create potential non-dilutive cash opportunities.”

 

Paid news dissemination for Citius Pharmaceuticals.

 

Read this article, featuring CTXR in full at: https://www.investorideas.com/news/2024/biotech/07113Transformative-Biopharma-Stocks.asp

 

"Our primary purpose and mission for these late-stage assets is to provide best-in-class, highly effective treatment options for patients and caregivers," Mazur added. "Ultimately, we believe our achievements and milestones offer powerful levers for value creation."

 

Late-Stage Assets: Mino-Lok® and LYMPHIR™

 

Citius significantly advanced its two late-stage product candidates in the first six months of 2024, and expects several key milestones in the near term.

 

Mino-Lok: Citius recently announced positive topline data from the Phase 3 study of Mino-Lok® in catheter-related bloodstream infections (CRBSIs). Primary and secondary endpoints were met with statistical significance.

 

Next steps for the Mino-Lok program are to prepare a submission to the U.S. Food and Drug Administration (FDA) and schedule a Type B meeting.

 

LYMPHIR™: In March 2024, the FDA accepted the Company's Biologics License Application (BLA) for LYMPHIR™ (denileukin diftitox), an IL-2-based immunotherapy for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL).

 

A decision on the LYMPHIR BLA is expected on August 13, 2024, the FDA's assigned Prescription Drug User Fee Act (PDUFA) action date. If approved, Citius is preparing for LYMPHIR commercialization in 2024.

 

Considered a new biologic by the FDA, LYMPHIR would potentially be eligible for 12 years of exclusivity, if approved.

 

In addition to the initial indication, two investigator-initiated Phase 1 studies of LYMPHIR are underway to explore how the differentiated mechanism-of-action may offer potential indications beyond CTCL. The studies are in progress at the University of Pittsburgh and the University of Minnesota.

 

Specialty Pharma Asset: Halo-Lido

 

An end of Phase 2b trial meeting with the FDA was held to discuss the positive data for Halo-Lido, a prescription-strength topical for symptomatic hemorrhoid treatment. The data showed a meaningful reduction in symptom severity when compared to individual components alone.

 

Continuing engagement with the FDA will guide the Company's next phase of development for Halo-Lido.

 

Mino-Lok (MLT), a novel antibiotic lock solution that combines minocycline, ethanol and edetate disodium, is designed to treat patients with catheter-related blood stream infections. Citius licensed Mino-Lok from an affiliate of The University of Texas MD Anderson Cancer Center. Mino-Lok is designed to offer an alternative to removing and replacing a central venous catheter (CVC), which may lead to a reduction in serious adverse events and cost savings to the healthcare system. If approved, Mino-Lok would be the first and only FDA-approved treatment that salvages central venous catheters that cause central line-related blood stream infections.

 

LYMPHIR is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. In 2011 and 2013, the FDA granted orphan drug designation to LYMPHIR for the treatment of PTCL and CTCL, respectively. In 2021, denileukin diftitox received regulatory approval in Japan for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). Subsequently in 2021, Citius acquired an exclusive license with rights to develop and commercialize LYMPHIR in all markets except for Japan and certain parts of Asia.

 

Halo-Lido (CITI-002) is a proprietary topical formulation intended to provide symptomatic relief to individuals suffering from hemorrhoids. Hemorrhoids are a gastrointestinal disorder characterized by pain, swelling, itching, tenderness, and bleeding. Although hemorrhoids are not life-threatening, individual patients often suffer painful symptoms that can limit social activities and have a negative impact on the quality of life. More than half of the U.S. population will experience hemorrhoidal disease at least once in their life. Each year, nearly 10 million patients in the U.S. report symptoms.

 

Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX), a  global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, recently announced that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy (vanza triple) for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to the vanza triple. Vertex used a priority review voucher for this submission reducing the review time from 10 months to 6 months, resulting in a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025.

 

"The FDA acceptance of our vanza triple application and the MAA validation by the EMA represent important milestones in the decades-long development of CFTR modulators and another example of our track record of serial innovation in CF," said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. "Vanzacaftor raises the high bar set by TRIKAFTA® and gives more people with CF the chance to get to levels of sweat chloride below the diagnostic threshold for CF, and even to levels of sweat chloride seen in those without CF."

 

From the news: Vertex also received validation of its Marketing Authorization Application (MAA) submission by the European Medicines Agency (EMA) in the EU for patients ages 6 years and older. The company has also submitted in Canada, Australia, Switzerland and the U.K.

 

CRISPR Therapeutics (Nasdaq:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, previously announced new preclinical data presented at the 27th Annual Meeting of the American Society of Cell and Gene Therapy (ASGCT) highlighting the Company’s approach to developing lipid nanoparticle (LNP) based delivery for in vivo ocular gene editing. In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs. CTX340™ and CTX450™ utilize LNP-based delivery of CRISPR/Cas9 gene editing cargo to the liver, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively.

 

“Over the past two years, we have made significant progress on the development of our lipid nanoparticle platform for the delivery of CRISPR/Cas9 to the liver and are now in clinical trials with CTX310 and CTX320,” said Samarth Kulkarni, Chief Executive and Chairman of the Board of CRISPR Therapeutics. “The expansion of our in vivo pipeline speaks to the scalability of the platform and the exceptional translation capabilities of our team. We continue to add programs to treat both common and rare diseases, as we look to broaden the number of areas where CRISPR could have transformational impact.”

 

From the news: In Vivo Pipeline Expansion

CRISPR Therapeutics has established a proprietary LNP platform for the delivery of CRISPR/Cas9 to the liver. The first two in vivo programs utilizing this proprietary platform, CTX310™ and CTX320™, are directed towards validated therapeutic targets associated with cardiovascular disease, and are in on-going clinical trials. The addition of two more programs, CTX340 and CTX450, utilizing this LNP delivery technology demonstrates the modularity and scalability of the platform.

 

Refractory hypertension is a serious unmet medical need affecting approximately 1.5 million patients in the U.S. alone. CTX340 is designed to inhibit production of hepatic angiotensinogen (AGT), a validated target to modulate the renin-angiotensin-aldosterone system (RAAS) and normalize blood pressure durably with a one-time treatment. In preclinical studies, CTX340 showed ~60% liver editing and ~90% AGT protein reduction, resulting in sustained ~30 mmHg blood pressure (BP) reduction out to 3 months in the spontaneously hypertensive rat (SHR) model.

 

Acute hepatic porphyria (AHP) is a group of rare genetic diseases of heme biosynthesis.

 

More from news; Symptomatic patients have acute attacks, characterized by debilitating neurovascular symptoms, as well as multiple chronic symptoms, such as pain. There are approximately 5,000 patients diagnosed with AHP in the U.S., although the disease remains underdiagnosed. CTX450 is specifically designed to inhibit production of ALAS1 in the liver, preventing accumulation of neurotoxic aminolevulinic acid (ALA) and porphobilinogen (PBG). In preclinical studies, CTX450 showed ~70% liver editing and ~97% ALAS1 protein reduction, resulting in reduction of ALA and PBG disease biomarkers to normal levels in an AHP mouse model.

 

CRISPR Therapeutics has initiated IND/CTA-enabling studies for CTX340 and CTX450 and expects to initiate both clinical trials in the second half of 2025.

 

One of the biggest trends transforming the sector is Artificial Intelligence (AI) and Machine Learning (ML) and Recursion (NASDAQ:RXRX), a leading clinical stage TechBio company, is betting on it.

 

Recursion is decoding biology to industrialize drug discovery. Central to its mission is the Recursion Operation System (OS), a platform built across diverse technologies that continuously expands one of the world’s largest proprietary biological, chemical and patient-centric datasets. Recursion leverages sophisticated machine-learning algorithms to distill from its dataset a collection of trillions of searchable relationships across biology and chemistry unconstrained by human bias. By commanding massive experimental scale — up to millions of wet lab experiments weekly — and massive computational scale — owning and operating what Recursion believes is one of the fastest supercomputers deployed in the sector, Recursion is uniting technology, biology, chemistry and patient-centric data to advance the future of medicine.

 

Transformative is a key word looking at the future of biopharma/biotech stocks as innovation keeps the sector one to watch for investors.

 

Research and find more biotech stocks at Investorideas.com

https://www.investorideas.com/BIS/Stock_List.asp

About Investorideas.com - Big Investing Ideas

Investorideas.com is the go-to platform for big investing ideas. From breaking stock news to top-rated investing podcasts, we cover it all. Our original branded content includes podcasts such as Exploring Mining, Cleantech, Crypto Corner, Cannabis News, and the AI Eye. We also create free investor stock directories for sectors including mining, crypto, renewable energy, gaming, biotech, tech, sports and more.

 

Disclaimer/Disclosure: This news article featuring Citius Pharmaceuticals, Inc. (CTXR) a paid for news release creation and dissemination on Investorideas.com.  Our site does not make recommendations for purchases or sale of stocks, services or products.  This is not investment opinion: Nothing on our sites should be construed as an offer or solicitation to buy or sell products or securities. All investing involves risk and possible losses. More disclaimer info: https://www.investorideas.com/About/Disclaimer.asp Learn more about publishing your news release and our other news services and prices on the Investorideas.com newswire https://www.investorideas.com/News-Upload/ Global investors must adhere to regulations of each country. Please read Investorideas.com privacy policy: https://www.investorideas.com/About/Private_Policy.asp

 

Follow us on X @investorideas

Follow us on Facebook https://www.facebook.com/Investorideas

Follow us on YouTube https://www.youtube.com/c/Investorideas

 

Contact Investorideas.com

800-665-0411

Biotech Industry Stocks- investing ideas in biotechnology stocks, medical technology and life sciences

Get more biotech and medical tech news, articles, podcasts and stock directories