Myelodysplastic Syndrome - Pipeline Insight, 2025

This “Myelodysplastic Syndrome - Pipeline Insight, 2025” report provides comprehensive insights about 120+ companies and 125+ pipeline drugs in Myelodysplastic Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Myelodysplastic Syndrome: Understanding

Myelodysplastic Syndrome: Overview

Myelodysplastic syndrome (MDS) is a heterogeneous group of hematologic neoplasms classically described as a clonal disorder of hematopoietic stem cells leading to dysplasia and ineffective hematopoiesis in the bone marrow. Some patients with MDS may have a transformation into acute myeloid leukemia (AML). MDS is usually diagnosed in older patients over the age of 65. Clinical manifestations include a decrease in the number of red blood cells (RBC), platelets, and white blood cells (WBC). The disease course is variable.

Development of MDS may occur due through various mechanisms such as environmental exposures to chemicals like benzene, radiation, prior exposure to chemotherapeutic agents, or may be idiopathic, which are typically seen in the elderly population. Bone marrow failure syndromes like acquired aplastic anemia and Fanconi anemia have a risk of developing MDS and sometimes mimic this syndrome.

MDS can be de novo or secondary to other causes, also known as treatment-related MDS. Chemotherapeutic agents such as alkylators or topoisomerase II inhibitors have been implicated as known causes of MDS, usually occurring 2 to 7 years after exposure. The mechanism for the development of MDS has been implicated by various genetic and chromosomal abnormalities, which may occur de novo or secondary to one of the above etiologies. Cytogenetic abnormalities are seen in more than 80% of patients and include translocations or more commonly, aneuploidy (loss or gain of a chromosome). Changes in cytogenetics play a large role in the International prognostic scoring system (IPSS). Deletion of the long arm of chromosome 5 (5q) is the most common abnormal karyotype and may be subdivided into 2 categories: treatment-related MDS with 5q deletion, usually with exposure to alkylating agents, versus de novo isolated 5q deletion. Patients with 5q deletion related to prior chemotherapeutic agents usually also have other cytogenetic abnormalities and/or TP53 mutations and usually portends a poor prognosis. Isolated 5q deletion without other cytogenetic abnormalities has a significantly better prognosis. Other cytogenetic abnormalities commonly studied include normal karyotype, deletion 7q (-7), trisomy 8 and -Y.

The mainstay of treatment for MDS involves assessment of symptoms and potential morbidity attributed to the disease. Patients do not always require treatment as long as they are asymptomatic and most can be treated with supportive measures such as intermittent blood or platelet transfusions. MDS often portends an indolent or gradual course, though some patients have risk factors that put them at risk for transformation into AML. Oncologists will use the IPSS or R-IPSS scoring system to help guide the course of treatment. Treatment options include supportive measures, low intensity treatment with systemic agents, or high intensity treatment such as allogeneic stem cell transplant. The only curative modality remains an allogeneic stem cell transplant, but this is often difficult as MDS occurs more commonly in the elderly population. Candidates for allogeneic stem cell transplant must be carefully selected, as the transplant process itself can be morbid for patients with potentially significant treatment-related mortality, especially in the elderly population. However, high-risk patients who are able to undergo transplant have around 50% survival at 3 years. Treatment decisions are often individualized to each patient and based upon potential morbidity and mortality from treatment. Patients in intermediate or high-risk categories are generally considered for treatment.

'Myelodysplastic Syndrome- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myelodysplastic Syndrome pipeline landscape is provided which includes the disease overview and Myelodysplastic Syndrome treatment guidelines. The assessment part of the report embraces, in depth Myelodysplastic Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Myelodysplastic Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Myelodysplastic Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Myelodysplastic Syndrome.

Myelodysplastic Syndrome Emerging Drugs Chapters

This segment of the Myelodysplastic Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Myelodysplastic Syndrome Emerging Drugs

• Tamibarotene: Syros Pharmaceuticals

Tamibarotene (formerly SY-1425) is an oral selective retinoic acid receptor alpha (RARα) agonist. The company is developing treatments for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene. Approximately 50% of MDS patients and 30% of AML patients have RARA overexpression. When RARα is expressed in excess of its tightly controlled natural ligand, cells in the bone marrow may not differentiate into healthy myeloid cells, which can lead to hematological malignancies. However, when oral tamibarotene is administered, tamibarotene binds to RARα, allowing for the restoration of gene expression and myeloid differentiation. The company is currently investigating tamibarotene in the Phase III SELECT-MDS-1 trial in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA overexpression.

CA-4948: Curis

CA-4948, which is being developed by Curis, is a small-molecule IRAK4 kinase inhibitor. Inhibition of IRAK4-L activity with emavusertib (CA-4948) blocks leukemic growth in non-clinical experiments. Because IRAK4 plays a central role in this pathway, it is considered an attractive target for the generation of therapeutics to treat these B-cell malignancies as well as certain inflammatory diseases. As part of the collaboration with Aurigene, in October 2015, Curis exclusively licensed a program of orally available, small molecule inhibitors of IRAK4 kinase, including emavusertib (CA-4948). Currently, the drug is in the Phase II stage of its development for the treatment of Myelodysplastic syndromes.

RVU120: Ryvu Therapeutics

RVU120 (SEL120) is a specific, selective inhibitor of CDK8 and its paralog, CDK19. Preclinical studies indicated the strong antileukemic potential of RVU120, which was often associated with the multilineage commitment of CD34+ AML cells. Moreover, RVU120 could improve proliferation and induce erythroid differentiation of CD34+ cells derived from Diamond-Blackfan anemia (DBA) patients. Currently, the drug is in the Phase II stage of its development for the treatment of Myelodysplastic syndromes.

MNV-201: Minovia Therapeutics

MNV-201 is an investigational cell therapy developed by Minovia Therapeutics, targeting low-risk Myelodysplastic Syndromes (MDS). It utilizes a novel approach known as Mitochondrial Augmentation Technology (MAT), which involves enriching a patient's own hematopoietic stem and progenitor cells (HSPCs) with mitochondria derived from allogeneic placental sources. This therapy aims to address mitochondrial dysfunction, which is implicated in the pathophysiology of MDS. Currently, MNV-201 is undergoing a Phase I clinical trial (NCT06465160) that is actively recruiting participants. The study's primary objective is to evaluate the safety and therapeutic effects of MNV-201 in patients diagnosed with low-risk MDS.

Myelodysplastic Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Myelodysplastic Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Myelodysplastic Syndrome

• There are approx. 120+ key companies which are developing the therapies for Myelodysplastic Syndrome. The companies which have their Myelodysplastic Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Syros Pharmaceuticals.

Phases

The report covers around 125+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Myelodysplastic Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Myelodysplastic Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myelodysplastic Syndrome therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myelodysplastic Syndrome drugs.

Myelodysplastic Syndrome Report Insights

• Myelodysplastic Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Myelodysplastic Syndrome Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Myelodysplastic Syndrome drugs?
• How many Myelodysplastic Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Myelodysplastic Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Myelodysplastic Syndrome therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Myelodysplastic Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Novartis
• Syros Pharmaceuticals
• Curis
• Ryvu Therapeutics
• Chia Tai Tianqing Pharmaceutical Group
• Amgen
• Sanofi
• Forma Therapeutics
• Agios Pharmaceuticals
• AbbVie
• Daiichi Sankyo Company
• Geron Corporation
• Astex Pharmaceuticals
• Jazz Pharmaceuticals
• Maxinovel Pty., Ltd.

Mabwell (Shanghai) Bioscience Co., Ltd.

BeiGene

• Ellipses Pharma
• Treadwell Therapeutics
• Bellicum Pharmaceuticals

Key Products

• MBG453
• Tamibarotene
• CA-4948
• RVU120
• TQB2618
• AMG 176
• SAR443579
• Etavopivat
• AG-946
• Venetoclax
• Quizartinib
• Imetelstat
• Azacitidine
• Vyxeos
• MAX-40279-01
• 6MW3211
• BGB-11417
• EP0042
• CFI-400945
• BPX-501

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