Investor Ideas, Trading Ideas and Stocks to Watch : 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.

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"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.

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