Ewing Sarcoma - Pipeline Insight, 2024

This “Ewing Sarcoma - Pipeline Insight, 2024,” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Ewing Sarcoma 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.

Geography Covered

• Global coverage

Ewing Sarcoma Understanding

Ewing Sarcoma: Overview

Ewing sarcoma is the second most common primary malignant bone tumor, mostly affecting adolescents in the second decade of their life, and it is a highly metastatic class of sarcoma. Despite the use of radiotherapy or surgery, historically, an estimated 85% to 90% of patients died within a few months from a metastasis. Due to the considerable progress in the treatment of disease with both local therapy and multi-agent adjuvant chemotherapy, 5-year survival rate improved from less than 20% to greater than 70%, but the recurrence rate remains high. It must be promptly recognized and treated to avoid high morbidity and mortality. Ewing sarcoma family tumors (ESFT) are characterized by the presence of non-random chromosomal translocations producing fusion genes that encode aberrant transcription factors. The t(11;22)(q24;q12) translocation is associated with 85% of tumors and leads to EWS-FLI-1 formation, whereas t(21;12)(22;12) and other less common translocations induced EWS-ERG fusion comprises the remaining 10% to 15% of cases. Ewing sarcoma is composed of small round cells with an increased nuclear-cytoplasmic ratio that represents a family of small round blue cell tumors of childhood (e.g., retinoblastoma, neuroblastoma, rhabdomyosarcoma, and nephroblastoma). Ewing cells have scant eosinophilic cytoplasm containing abundant glycogen that is often detected by staining with periodic-acid-Schiff. The standard of care for patients with or without metastasis includes interprofessional treatment with chemotherapy and local therapy, including surgery and radiotherapy (RT).

Ewing Sarcoma - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Ewing Sarcoma pipeline landscape is provided which includes the disease overview and Ewing Sarcoma treatment guidelines. The assessment part of the report embraces, in depth Ewing Sarcoma 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, Ewing Sarcoma collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• In April 2022, AI Therapeutics announced the acquisition of Entre Chem’s, investigational drug candidate EC-8042 (redesignated AIT-102), a novel targeted cancer therapy in development for rare pediatric and other cancers. AIT-102 specifically targets the mutations responsible for the formation and progression of two notable pediatric tumors, rhabdoid tumors and Ewing sarcoma, and holds the potential to treat a broader family of tumors that share a common mechanism of mutated or dysregulated SWI/SNFactivity.
• In December 2021, Rakovina Therapeutics announced that it has been selected to join a three year US$975,000 (CDN$1,297,000) research program funded by the St. Baldrick’s Foundation Martha’s BEST Grant for All, which is aimed at developing new treatments for Ewing sarcoma.

Ewing Sarcoma Emerging Drugs Chapters

This segment of the Ewing Sarcoma 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.

Ewing Sarcoma Emerging Drugs

Vigil: Gradalis Gradalis is developing and testing Vigil, a fully personalized, patient-specific cancer immunotherapy that can be applied to virtually any cancer. Vigil utilizes the patient’s own cancer cells to create a fully personalized cancer immunotherapy with the goal to activate the patient’s own T-cells against their cancer cells. When those cells are reintroduced back into the patient, the two modifications are designed to help activate the immune system to detect and kill any cancer cells that may remain locally and in circulation. All together, the goal is to stimulate the existing components of the immune system with the intent to improve their anti-tumor responses. Vigil enhances specific functions that assist in cancer antigen recognition and dampen other functions that cancer cells often employ to evade the immune system. A Phase III clinical trial of Vigil in combination with irinotecan and temozolomide for the treatment of patients with Ewing’s sarcoma (EWS) is currentlyongoing.

ONCT216: Oncternal Therapeutics ONCT-216 (formerly called TK216) is a first-in-class small molecule inhibiting the biological activity of ETS-family (E26 Transformation Specific) transcription factor oncoproteins in a variety of tumor types. It is currently being evaluated in the Phase II portion of a study in patients with relapsed or refractory Ewing sarcoma, a serious pediatric bone cancer. ONCT-216 was developed based on the discoveries of Jeff Toretsky, MD, at Georgetown University, who identified a small molecule that was shown to kill Ewing sarcoma cells and inhibit growth of tumors in preclinical studies. Oncternal scientists developed and tested a large series of derivatives of the research molecule and after extensive evaluation, ONCT-216 was selected as a lead product candidate. ONCT-216 has been extensively evaluated in preclinical studies, where it has been confirmed to kill Ewing sarcoma cells and inhibit Ewing sarcoma tumors in animal models. The company continues to collaborate with Dr. Toretsky and his colleagues in order to advance the research underlying the molecular pathways of ONCT-216 and optimize its development across multiple tumor types.

SP-2577: Salarius Pharmaceuticals SP-2577 is being studied in an ongoing Phase I/II trial treating three different patient groups with sarcomas, including Ewing Sarcoma, Myxoid Liposarcoma and additional FET-rearranged soft tissue sarcomas. These are cancers with high-unmet need and represent Salarius’ “speed-to-market strategy” given the potential for accelerated approval. Salarius’ LSD1 technology was licensed from the University of Utah Huntsman Cancer Institute where it was developed in the laboratory of Dr. Sunil Sharma.

Ewing Sarcoma: Therapeutic Assessment

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

Major Players in Ewing Sarcoma

There are approx. 25+ key companies which are developing the therapies for Ewing Sarcoma. The companies which have their Ewing Sarcoma drug candidates in the most advanced stage, i.e. Phase III include, Gradalis.

Phases

This report covers around 25+ 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

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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

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

Ewing Sarcoma: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Ewing Sarcoma 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 Ewing Sarcoma drugs.

Ewing Sarcoma Report Insights

• Ewing Sarcoma Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Ewing Sarcoma 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 Ewing Sarcoma drugs?
• How many Ewing Sarcoma drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Ewing Sarcoma?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Ewing Sarcoma 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 Ewing Sarcoma and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Gradalis, Inc.
• Shanghai Pharmaceuticals Holding Co., Ltd
• Tyme, Inc
• Pfizer
• Hutchison Medipharma Limited
• Bristol-Myers Squibb
• Salarius Pharmaceuticals
• Bio Atla,Inc.
• Cellectar Biosciences
• Valent Technologies
• Inhibrx, Inc.
• Eisai Co Ltd
• Amgen
• Y-mAbs Therapeutics
• Nano ValentPharmaceuticals.
• Oncternal Therapeutics
• Aptadel Therapeutics
• Gibson Oncology
• Edison Oncology
• Oncoheroes Biosciences
• CotheraBio
• Rakovina Therapeutics

Key Products

• Vigil
• TK216
• SM-88
• Pembrolizumab
• Palbociclib
• Surufatinib
• Nivolumab
• Seclidemstat
• CAB-AXL-ADC
• CLR 131
• VAL-413
• Eribulin mesylate
• INBRX-109
• AMG 479
• Naxitamab
• NV101
• NV103
• ADEL101
• LMP400
• Orotecan
• PC002

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