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,
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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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