A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered to Patients With Phlebotomy Dependent Polycythemia Vera (PD-PV)

The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.

Start Date30 Dec 2021

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT04059406

/ TerminatedPhase 2

A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients With Non-Transfusion Dependent β-Thalassemia Intermedia

The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.

Start Date24 Sep 2020

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

ACTRN12617000458336

/ CompletedPhase 1

A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-TMPRSS6-Lrx Administered Subcutaneously to Healthy Volunteers

Start Date08 Jun 2017

Sponsor / Collaborator-

100 Clinical Results associated with Sapablursen

100 Translational Medicine associated with Sapablursen

100 Patents (Medical) associated with Sapablursen

News (Medical) associated with Sapablursen

14 Mar 2025

·www.fiercepharma.com

Fierce Pharma Asia—Takeda's oncology focus; Sun's Checkpoint buy; Ono-Ionis antisense deal

Takeda's narrowed focus on oncology, Sun Pharma's proposed acquisition of Checkpoint Therapeutics and Ono's antisense oligonucleotide deal with Ionis made our news this week. Takeda is sharpening its oncology focus amid an R&D revamp. Sun Pharma will acquire Checkpoint Therapeutics and an FDA-approved PD-L1 inhibitor. Japan's Ono Pharmaceutical bought an antisense oligonucleotide from Ionis Pharmaceuticals. Plus more.1. 'I don't feel we lost momentum': Takeda’s oncology execs sharpen focus after restructuringTakeda likely won’t do any cell therapy development deals in the near future, even though it’s still advancing an allogeneic gamma delta T-cell therapy in acute myeloid leukemia. Takeda’s oncology leaders Teresa Bitetti and P.K. Morrow, M.D., explained the company’s cancer drug strategy in an interview. In addition, the Japanese pharma is not counting out China as a source for partnering despite geopolitical risks. 2. On the heels of FDA nod for cancer drug, Sun Pharma buys Checkpoint Therapeutics for up to $416MSun Pharma has agreed to buy Checkpoint Therapeutics for $355 million upfront, plus a contingent value right that could bring the deal value to about $416 million. The acquisition bolsters Sun’s oncology-dermatology franchise with Unloxcyt, a PD-L1 inhibitor recently approved by the FDA for advanced cutaneous squamous cell carcinoma.3. Ono pays $280M upfront for Ionis' antisense oligonucleotide for rare blood cancerOno Pharmaceutical is paying $280 million upfront to acquire Ionis Pharmaceuticals’ antisense oligonucleotide sapablursen. The drug is designed to reduce the production of the TMPRSS6 protein and is currently in a phase 2 trial for the rare blood condition of polycythemia vera. The pact includes up to $660 million in potential milestones.4. J&J, Legend to invest $150M in Belgium cell therapy facility as new Carvykti rival loomsLegend Biotech and its partner Johnson & Johnson will invest $150 million to expand their so-called Tech Lane cell therapy facility in Belgium. The plant is slated to begin clinical production of Carvykti in a few weeks. The partners are further expanding manufacturing capacity even as Gilead Sciences and Arcellx’s rival BCMA CAR-T anito-cel is expected to enter the market in 2026.5. Illumina aims to cut $100M in costs following China import banAs China bans Illumina’s importation of DNA sequencers in retaliation to new U.S. tariffs, the company is looking to cut $100 million in expenses this year. Last year, Illumina counted about 7% of its sales, or about $300 million, from China. The cost reduction “includes optimizing stock-based compensation and non-labor spending and accelerating certain productivity measures,” Illumina said.6. Insilico Medicine raises $110M for new trials and robotic helping handsHong Kong-based Insilico Medicine has raised $110 million to advance its artificial-intelligence-powered drug design platforms. The series E round, led by Value Partners, will help refine Insilico’s AI models as well as support pivotal clinical trials of the company’s lead drug candidate, rentosertib (ISM001-055), in idiopathic pulmonary fibrosis.7. Frazier arms Callio with $187M series A to fly Hummingbird's HER2 dual payload ADC to clinicSingapore’s Hummingbird Bioscience has created a new company together with its investor Frazier Life Sciences and a few other venture capital funds. The new firm, called Callio Therapeutics, has launched with a $187 million series A and a phase 1-ready HER2-targeted dual-payload antibody-drug conjugate licensed from Hummingbird.Other News of Note:8. Daiichi taps Nosis to deliver RNA therapies beyond the liver9. Harbour BioMed launches new obesity biotech on mission to preserve muscle mass10. Kelun's TROP2 ADC picks up another approval in China11. Indian contractor Syngene throws down $37M for Emergent's Baltimore biologics plant12. Nasdaq stock market plans move to 24-hour trading in US13. Samsung backs Alzheimer’s blood test developer C2N Diagnostics with $10M14. Astellas, Yaskawa cement deal for robotic cell therapy production joint venture15. Otsuka debuts new kid-friendly resources about Alzheimer's to ease burden on 'sandwich generation' caregivers

Cell TherapyPhase 1AcquisitionPhase 2Oligonucleotide

12 Mar 2025

·pipelinereview.com

Ono Enters into License Agreement with Ionis Pharmaceuticals for Sapablursen for the Treatment of Polycythemia Vera

OSAKA, Japan I March 11, 2025 I Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “ONO”) announced that it has entered into a license agreement with Ionis Pharmaceuticals, Inc. (Carlsbad, CA,U.S.; CEO, Brett P. Monia, Ph.D.; “Ionis”) for sapablursen, an investigational RNA-targeted medicine for the treatment of polycythemia vera (PV). Sapablursen is currently being evaluated in adult patients with PV in the fully enrolled Phase 2 IMPRSSION study. Sapablursen was granted Fast Track designation in January 2024 and orphan drug designation in August 2024 by the U.S. Food and Drug Administration (FDA). Under the agreement, ONO will obtain an exclusive license to develop and commercialize sapablursen worldwide. Ionis will remain responsible for the completion of the ongoing Phase 2 IMPRSSION study, while ONO will be solely responsible for subsequent development, regulatory filings and commercialization. ONO will make an upfront payment of 280 million US dollars, with up to a maximum of 660 million US dollars in additional payments based on the achievement of development, regulatory and sales milestones. ONO will also pay to Ionis royalties in the mid-teens on annual net sales of sapablursen. The transaction is subject to the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act). “We are delighted to enter into this collaboration with Ionis Pharmaceuticals, a pioneer in RNA-targeted medicines, for sapablursen. This partnership aligns with our strategy to strengthen our pipeline in hematology. We expect sapablursen to become a new treatment option for PV patients worldwide,” said Toichi Takino, President and COO of ONO. “Ionis and ONO share a commitment to advancing innovative medicines to help meet the needs of people living with serious diseases. We are pleased to entrust sapablursen to ONO, whose unique capabilities will help maximize its value by ensuring broad access for people living with PV,” said Brett P. Monia, Ph.D., CEO of Ionis. The impact of this matter on the Company’s consolidated financial results for the current fiscal year ending March 31, 2025, is expected to be minor. Should there be any events to be disclosed in the future, we will announce them in a timely manner. About Sapablursen Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, which is the key regulator of iron homeostasis. By increasing production of hepcidin, sapablursen has the potential to positively impact blood diseases such as PV. About Polycythemia Vera Polycythemia Vera (PV) is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, especially in critical organs like the lungs, heart and brain. Patients with PV also experience severe iron deficiency and commonly have symptoms of fatigue, which can lead to reduce quality of life (QOL). About Ionis Pharmaceuticals Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. For further information, visit https://www.ionis.com/ . SOURCE: Ono Pharmaceutical Co

Orphan DrugLicense out/inPhase 2Fast Track

12 Mar 2025

·www.fiercebiotech.com

Ono pays $280M upfront for Ionis\' antisense oligonucleotide for rare blood cancer

Ionis Pharmaceuticals previously investigated sapablursen in another blood disorder called beta thalassemia.\n Japan\'s Ono Pharmaceutical is handing over $280 million upfront to acquire Ionis Pharmaceuticals’ phase 2-stage antisense oligonucleotide for a rare type of blood cancer.The RNA-targeted medicine, called sapablursen, is designed to reduce the production of a protein called TMPRSS6. The idea is that this increases the expression of hepcidin, which is the key regulator of iron homeostasis, thereby helping treat various blood diseases.Sapablursen is currently in a phase 2 trial for polycythemia vera (PV), a rare blood condition characterized by the overproduction of red blood cells. As part of the licensing deal with Ono, Ionis will remain responsible for the midstage study.Beyond that, Ono will become solely responsible for sapablursen’s development, regulatory filings and commercialization, according to a post-market release March 11.In return, Ono is handing over $280 million upfront, with potentially up to $660 million to follow in additional development, regulatory and sales milestones, along with mid-teen royalties on annual net sales.Ionis had previously investigated sapablursen in another blood disorder called beta thalassemia before ending those plans in 2023 after looking at midphase efficacy data and instead focusing on PV.At the time, Ionis CEO Brett Monia, Ph.D., said the company had seen “quite remarkable” target engagement in phase 1 plus “very significant elevations in hepcidin levels.” However, “in beta-thalassemia intermedia, we didn\'t see those changes,” the CEO explained back in 2023.In yesterday’s release, Monia said the company was “pleased to entrust sapablursen to Ono, whose unique capabilities will help maximize its value by ensuring broad access for people living with PV.” Ionis recently became a commercial-stage company as a result of the approval of Tryngolza to treat the rare genetic disorder familial chylomicronemia syndrome in December.“Ionis remains committed to advancing the wholly owned medicines we choose to commercialize ourselves, which includes our first independent launch currently underway and three additional anticipated launches in the next three years,” Monia added. “Streamlining our Ionis-owned portfolio provides financial flexibility, supporting our commitment to invest in and focus on our near and mid-term commercial opportunities and generate substantial revenue growth.”Meanwhile, Ono—which codeveloped blockbuster cancer immunotherapy Opdivo with Bristol Myers Squibb—is active in the oncology space, where it is working on a slate of antibodies as well as some multispecific molecules and cell therapies. Last year, the biotech got in on the antibody-drug conjugate action via a $700 million biobucks deal with LigaChem Biosciences.The partnership with Ionis “aligns with our strategy to strengthen our pipeline in hematology,” said Ono President and Chief Operating Officer Toichi Takino, who added that the company “expects sapablursen to become a new treatment option for PV patients worldwide.”The deal comes a week after Takeda and Protagonist Therapeutics posted a phase 3 win for their own PV prospect, an injectable hepcidin mimetic called rusfertide.“While rusfertide is likely to be first to market in the hepcidin-boosting drug class, we see sapablursen as a potential improvement in treatment regimen that will be attractive to patients and healthcare providers,“ William Blair analysts said in a March 12 note, adding that Ono\'s deal with Ionis was “positive for both sides.“

License out/inPhase 2Phase 3OligonucleotideDrug Approval

100 Deals associated with Sapablursen

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 2	United States	Ionis Pharmaceuticals, Inc.	30 Dec 2021
Polycythemia Vera	Phase 2	Australia	Ionis Pharmaceuticals, Inc.	30 Dec 2021
Polycythemia Vera	Phase 2	Canada	Ionis Pharmaceuticals, Inc.	30 Dec 2021
Polycythemia Vera	Phase 2	Poland	Ionis Pharmaceuticals, Inc.	30 Dec 2021
Polycythemia Vera	Phase 2	United Kingdom	Ionis Pharmaceuticals, Inc.	30 Dec 2021
Beta-Thalassemia	Phase 2	Australia	Ionis Pharmaceuticals, Inc.	24 Sep 2020
Beta-Thalassemia	Phase 2	Greece	Ionis Pharmaceuticals, Inc.	24 Sep 2020
Beta-Thalassemia	Phase 2	Lebanon	Ionis Pharmaceuticals, Inc.	24 Sep 2020
Beta-Thalassemia	Phase 2	Thailand	Ionis Pharmaceuticals, Inc.	24 Sep 2020
Beta-Thalassemia	Phase 2	Turkey	Ionis Pharmaceuticals, Inc.	24 Sep 2020

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04059406 (CTgov)	Phase 2	Beta-Thalassemia \| Non-transfusion dependent thalassaemia	29	sapablursen (Cohort A: Sapablursen)	wecjvwssiv = jwaofcyxrs djwtgarmiu (rrkyxbccwa, ptrrmnkltk - vxcjrgcbim) View more	-	18 Feb 2025
				sapablursen (Cohort B: Sapablursen)	wecjvwssiv = aguqfnqmsq djwtgarmiu (rrkyxbccwa, hbfyvmumbc - ewydxfysto) View more

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