Arrowhead Pharmaceuticals

Calando Pharmaceuticals is a biopharmaceutical company focused on creating targeted therapeutics using its patented and proprietary cyclodextrin-based polymer technologies for the development of nanoparticle drug formulations.   Calandoâ€™s initial emphasis has been to bring targeted siRNA and small molecule therapeutics to the clinic for oncology applications; however, the technologies are not limited to these classes of molecules and are applicable to a very broad range of therapeutic entities and diseases.

Arrowhead Pharmaceuticals is a biotechnology company that focuses on the development of medicine to treat diseases with a genetic origin, characterized by the overproduction of one or more proteins. Its medical solutions are aimed at the genes that trigger the diseases. Its portfolio includes drugs targeting hepatitis B, Alpha-1 Antitrypsin Deficiency, thrombosis and angioedema, clear-cell and renal cell carcinoma, and cardiovascular disease.

<div>Arrowhead Pharmaceuticals develops medicines for the treatment of intractable diseases. The company's pipeline includes several drug candidates targeting various conditions. Plozasiran, in Phase 2 billion and Phase 3 trials, aims to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome. Zodasiran, in Phase 2 billion, is being developed for dyslipidemia and hypertriglyceridemia. ARO-PNPLA3, in Phase 1, targets non-alcoholic steatohepatitis. ARO-RAGE, in Phase 1/2a, focuses on inflammatory pulmonary conditions, while ARO-MUC5AC, also in Phase 1/2a, is intended for muco-obstructive pulmonary diseases. Arrowhead's technology platform utilizes RNA interference (RNAi) to silence specific genes involved in disease processes. As of May 2024, the company reported negative financial results, with a quarterly loss of USD 1.02 per share, missing analyst estimates. Arrowhead's revenue was down 100% year-over-year for the same quarter. The company had a market capitalization of approximately USD 3.51 billion as of July 2024.</div>

Natixis Makes New Investment in Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR)

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Hennion & Walsh Asset Management Inc. Acquires 4,523 Shares of Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR)

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Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) Receives Consensus Rating of “Moderate Buy” from Analysts

Russell Investments Group Ltd. Has $6.94 Million Holdings in Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR)

Retirement Systems of Alabama Has $9.98 Million Stake in Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR)

Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) Receives $47.89 Average Target Price from Analysts

RNA Therapeutics

Arrowhead Pharmaceuticals Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4) - December 19, 2024

Sarepta Therapeutics

To develop treatments for rare genetic diseases by leveraging Arrowhead's Targeted RNAi Molecule (TRiM) platform and Sarepta's expertise in genetic medicines, with Sarepta conducting clinical development and commercialization activities. The deal is valued at up to USD 10.825 billion, including USD 825 million upfront, USD 250 million over five years, and potential milestone payments.

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Companies that offer support services to gene-editing companies, which include genomic research companies, gene-editing component manufacturing, and designing tools

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Takeda

A collaboration and licensing agreement to develop ARO-AAT, a Phase 2 investigational RNA interference (RNAi) therapy in development to treat alpha-1 antitrypsin-associated liver disease (AATLD).

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 Arrowhead Pharmaceuticals develops medicines for the treatment of intractable diseases. The company's pipeline includes several drug candidates targeting various conditions. Plozasiran, in Phase 2 billion and Phase 3 trials, aims to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome. Zodasiran, in Phase 2 billion, is being developed for dyslipidemia and hypertriglyceridemia. ARO-PNPLA3, in Phase 1, targets non-alcoholic steatohepatitis. ARO-RAGE, in Phase 1/2a, focuses on inflammatory pulmonary conditions, while ARO-MUC5AC, also in Phase 1/2a, is intended for muco-obstructive pulmonary diseases. Arrowhead's technology platform utilizes RNA interference (RNAi) to silence specific genes involved in disease processes. As of May 2024, the company reported negative financial results, with a quarterly loss of USD 1.02 per share, missing analyst estimates. Arrowhead's revenue was down 100% year-over-year for the same quarter. The company had a market capitalization of approximately USD 3.51 billion as of July 2024.
 
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 PYC Therapeutics is a clinical-stage biotechnology company focused on developing RNA therapies for genetic diseases. The company utilizes a proprietary drug delivery platform based on Cell-Penetrating Peptides (CPPs) to enhance the potency of precision medicines within the RNA therapeutic class. PYC's technology aims to overcome the delivery challenges associated with traditional RNA therapeutics, allowing for more effective treatment of genetic ailments.
 
 
 
 
 PYC's lead drug candidate, VP-001, is designed to treat Retinitis Pigmentosa type 11 (RP11), a blinding eye disease that begins in childhood. VP-001 targets the U4/U6 small nuclear ribonucleoprotein (PRPF31) gene and is administered through intravitreal injection. In August 2023, the US Food and Drug Administration (FDA) granted Fast Track designation to VP-001, potentially expediting its development and review process. In May 2024, PYC reported encouraging first clinical data for VP-001 in RP11 patients.
 
 
 
 
 The company's pipeline includes other preclinical programs for Inherited Retinal Diseases and Central Nervous System disorders. In May 2024, PYC received orphan drug designation from the FDA for PYC-001, a drug candidate targeting autosomal dominant optic atrophy (ADOA). PYC is also developing VP-002 and exploring multiple candidates for eye and CNS indications.
 
 
 
 
 In January 2024, PYC announced plans to collaborate with Google Cloud to develop new precision medicines. This project aims to utilize machine learning models, merging AlphaFold and its successors, to predict ideal peptide sequences and structures for delivering precision medicine cargoes to cells in the human body.
 
 
 
 
 For the half-year ended December 31, 2023, PYC reported revenue of AUD 9.12 million, up from AUD 3.49 million in the previous year. The company's net loss for the period was AUD 15.09 million. As of March 31, 2024, PYC had a pro-forma cash balance of approximately AUD 84 million, providing runway into the second half of 2025 for its ongoing clinical trials.
 
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PYC Therapeutics

Flamingo Therapeutics is a biotechnology company specializing in RNA-targeted therapies for oncology. Founded in 2019, the company focuses on developing treatments for difficult-to-treat cancers using state-of-the-art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo's lead program is danvatirsen, an antisense oligonucleotide (ASO) that selectively targets STAT3 and has shown clinical activity in two Phase II studies. Danvatirsen binds to STAT3 mRNA, inhibiting translation of the transcript, resulting in reduced tumor cell growth and activation of anti-tumor immunity. Flamingo initiated the PEMDA-HN Phase II trial evaluating danvatirsen in combination with pembrolizumab for patients with recurrent/metastatic head and neck squamous cell carcinoma (HNSCC). The company is also advancing FTX-001, its most advanced long non-coding RNA (LncRNA) program targeting MALAT-1, through readiness for Phase 1 trials in solid tumors. Flamingo's proprietary discovery engine, FLAME (Flamingo LncRNA Antisense Mining Engine), addresses lncRNAs, a large and untapped class of disease-causing targets within the "dark matter" of the human genome. In March 2023, Flamingo merged with Dynacure to create a leading RNA therapeutics company focused on clinical oncology, combining their pipelines and expertise.
Key customers and partnerships
Flamingo Therapeutics has established strategic alliances and partnerships to advance its RNA-targeted therapies. The company has an expanded alliance with Ionis Pharmaceuticals, a world leader in RNA-targeted antisense oligonucleotide (ASO) therapeutics. This partnership, announced in September 2021, combines three clinical-stage programs from Ionis with Flamingo's novel lncRNA discovery engine and preclinical lncRNA program. The alliance leverages Flamingo's expertise in oncology and long non-coding RNAs with Ionis' proficiency in RNA-targeted drug discovery and development. Additionally, Flamingo collaborates with academic partners at VIB, KU Leuven, Ghent University, and the University of Michigan, whose pioneering work in non-coding RNA genes in cancer contributed to the company's foundation. In June 2023, Flamingo received a EUR 1.7 million research grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance its RNA-targeting oncology portfolio, further validating its approach in creating a pipeline of programs targeting undruggable transcription factors and non-coding RNA.

Flamingo Therapeutics

Korro Bio is a biotechnology company focused on developing RNA editing therapeutics for both rare and common diseases. Founded in 2018, the company is headquartered in Cambridge, Massachusetts. Korro's proprietary platform, called OPERA (Oligonucleotide Promoted Editing of RNA), leverages the body's natural ADAR (adenosine deaminase acting on RNA) system to make precise edits to RNA transcripts. This approach allows for the correction of genetic mutations without permanently altering DNA, potentially offering a safer alternative to traditional gene editing techniques.
The company's lead program targets alpha-1 antitrypsin deficiency (AATD), a genetic disorder affecting the lungs and liver. Korro's AATD candidate aims to repair inherited mutations and restore production of normal functional alpha-1 antitrypsin (A1AT) protein in the liver. preclinical studies have demonstrated an increase of normal A1AT protein to 85% of total protein in circulation. Korro plans to submit a regulatory filing to initiate clinical trials for this candidate in the second half of 2024.
Beyond AATD, Korro is developing RNA editing therapies for conditions such as Parkinson's disease, severe alcoholic hepatitis, amyotrophic lateral sclerosis (ALS), subsets of pain, and cardiometabolic diseases. The company's initial focus is on liver-targeted therapies, with plans to expand into central nervous system indications and other tissue types.
In July 2023, Korro Bio announced plans to go public through a reverse merger with Frequency Therapeutics. The combined company will operate under the Korro Bio name and trade on the Nasdaq stock exchange under the ticker symbol KRRO. This transaction, along with a concurrent USD 117 million financing, is expected to provide Korro with approximately USD 170 million in cash upon closing, which is anticipated to fund operations into 2026.

Korro Bio

Halo-Bio is a biopharmaceutical company focused on developing innovative therapies. The company has a licensing agreement with Tekmira Pharmaceuticals related to certain lipid nanoparticle (LNP) technologies.

Halo-Bio RNAi Therapeutics

Oncotelic Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing RNA therapeutics and small molecule drugs for cancer and infectious diseases. The company's lead drug candidate is OT-101, a first-in-class RNA therapeutic designed to target TGF-β2, a protein that suppresses immune cell response and contributes to tumor growth in cancer tissues. OT-101 has demonstrated notable single-agent activity in clinical trials for relapsed/refractory cancer patients, with overall survival increasing in a dose-dependent manner at levels of 140mg/m2 and higher.
Oncotelic is currently conducting a Phase 2 billion/Phase 3 clinical trial called STOP-PC to evaluate OT-101 in combination with mFOLFIRINOX compared to mFOLFIRINOX alone for patients with advanced and unresectable or metastatic pancreatic cancer. The company believes this trial could deliver a decisive win against pancreatic cancer based on previous data showing improved survival in patients with low TGF-β2 expression treated with irinotecan.
In addition to its oncology pipeline, Oncotelic has explored OT-101's potential against SARS-CoV-2, completing a Phase 2 trial for Covid-19 in November 2021 with positive top line data for safety and efficacy. The company has also acquired AL-101 for intranasal delivery of apomorphine, which it intends to develop for conditions including Parkinson's Disease, erectile dysfunction, and female sexual disorders.

Oncotelic

Companies developing treatments using molecules like antisense oligonucleotides (ASOs) and phosphorodiamidate morpholino oligomers (PMOs). These molecules target specific genes to treat genetic diseases by either silencing or modifying gene expression.

Companies using RNA interference (RNAi) to target and silence specific genes involved in diseases for treating various conditions by blocking harmful gene activity.

Companies involved in altering RNA molecules to modify gene expression or correct genetic defects. This can include substitutions, insertions, or deletions of nucleotides, leading to alterations in the RNA sequence and the regulation of RNA molecules to control gene expression or cellular functions.

Companies focused on other RNA-based therapies using approaches such as tRNA, circRNA, lncRNA, and saRNA to develop treatments targeting specific RNA molecules to regulate gene expression.

RNA-based developments form a foundation for precision medicine.

RNA plays essential roles in gene expression, regulation, and protein synthesis, making it a fundamental focus in genomic research and understanding genetic mechanisms.

PROMINENT_CATEGORY

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 Sarepta Therapeutics, a gene therapy company in Massachusetts, and Arrowhead Pharmaceuticals, a biopharmaceutical company in California, have formed a global licensing and partnership deal to develop treatments for rare genetic diseases. Arrowhead will receive $825 million, split between cash and an equity investment, as well as $250 million paid over five years, plus up to $10 billion in milestone payments and royalties on sales.
 This licensing agreement includes four clinical-phase programs, three preclinical candidates, and possibly six discovery programs. Arrowhead will use its Targeted RNAi Molecule (TRiM) platform to carry out discovery and preclinical development tasks while Sarepta will manage clinical development and commercialization of these programs. The agreement is set to be finalized in early 2025.

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Arrowhead Pharmaceuticals

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