Sickle Cell Disease - Competitive landscape, 2023

This “Sickle Cell Disease - Competitive landscape, 2023” report provides comprehensive insights about 40+ companies and 50+ drugs in Sickle Cell Disease Competitive landscape. It 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

Sickle Cell Disease: Understanding

Sickle Cell Disease: Overview

Sickle cell disease (SCD) is a group of inherited red blood cell disorders that affect hemoglobin, the protein that carries oxygen through the body. Healthy red blood cells are round, and they move through small blood vessels to carry oxygen to all parts of the body. In someone who has SCD, the hemoglobin is abnormal, which causes the red blood cells to become hard and sticky and look like a C-shaped farm tool called a “sickle.”
A baby born with sickle cell disease inherits a gene for the disorder from both parents. When both parents have the genetic defect, there's a 25 percent chance that each child will be born with sickle cell disease. Moreover, If a child inherits only one copy of the defective gene (from either parent), there is a 50 percent chance that the child will carry the sickle cell trait. People who only carry the sickle cell trait typically don't get the disease, but can pass the defective gene on to their children.

Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia which is why it is commonly known as sickle cell anemia. Sickle cell disease is a serious and lifelong condition, although long-term treatment can help manage many of the problems associated with it. The disease affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. There is an atypical hemoglobin molecule called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. Sickle cell disease is caused by a mutation in the hemoglobin-Beta gene found on chromosome 11.

SCD can be prevented prenatally, diagnosed in utero or in the newborn period by screening, or be detected at any time during life. The type of testing done depends on the age of the patient. DNA testing can be used for prenatal diagnosis or to confirm a diagnosis of the sickle cell genotype. Screening of neonates is available in most US states and involves hemoglobin electrophoresis. Screening and diagnosis in children and adults involve examination of the peripheral smear, hemoglobin solubility testing, and hemoglobin electrophoresis. The gold standard for pain assessment and diagnosis is patient self-report. There are no reliable diagnostic tests to confirm the presence of acute or chronic pain in individuals with SCD except when there are identifiable causes like avascular necrosis on imaging or leg ulcers on exam.

Treatment for sickle cell anemia is usually aimed at avoiding crises, relieving symptoms and preventing complications. There are some medical treatments and home remedies available to make you feel better. Treatments might include medications to reduce pain and prevent complications, and blood transfusions, as well as a bone marrow transplant. Researchers are exploring whether inserting a normal gene into the bone marrow of people with sickle cell anemia will result in normal hemoglobin along with exploring the possibility of turning off the defective gene while reactivating another gene responsible for the production of fetal hemoglobin. Researchers are studying various drugs to devise a way to boost the production of fetal hemoglobin.

Report Highlights

• In May 2023, The US Food and Drug Administration (FDA) granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD).
• In April 2023, bluebird bio, Inc. announced the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs). The BLA includes a request for Priority Review, which, if granted, would shorten the FDA's review of the application to six months from the time of filing, versus a standard review timeline of 10 months. If approved, lovo-cel will be bluebird bio's third ex-vivo gene therapy approved by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder, building on more than a decade of leadership in gene therapy.
• In March 2023, BioLineRx Ltd. announced a collaboration with Washington University School of Medicine in St. Louis to advance a Phase I clinical trial that will evaluate the safety and feasibility of the Company's lead clinical candidate motixafortide to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in SCD.
• In November 2022, C4X Discovery Holdings plc (AIM: C4XD), signed an exclusive worldwide licensing agreement with AstraZeneca worth up to $402 million, for its NRF2 Activator programme.
• In October 2022, Pfizer announced the completion of its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities starting with sickle cell disease (SCD).
• In October 2022, Novo Nordisk completed the acquisition of Forma Therapeutics Holdings, Inc. (Forma Therapeutics). Forma Therapeutics is a clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders.
• In June 2022, Precision BioSciences, Inc., a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG.

Sickle Cell Disease: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, Novartis use innovative science and digital technologies to create transformative treatments in areas of great medical need. In quest to find new medicines, the company consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments.

Product Description: ADAKVEO

ADAKVEO (crizanlizumab) previously known as SEG101 is indicated to reduce the frequency of VOCs, or pain crises, in adults and pediatric patients aged 16 years and older with sickle cell disease. It is the first and only targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion in sickle cell disease. By binding to P-selectin on the surface of the activated endothelium and platelets, ADAKVEO blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes.

Sickle Cell Disease: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: Pfizer

Pfizer prioritizes the health and well-being of people and the planet to deliver breakthroughs around the world in a responsible way. Pfizer's commitment to ESG spans product innovation; equitable access and pricing; product quality and safety; diversity, equity, and inclusion; climate change; and business ethics. The company focuses on the science and global resources to bring therapies to people that extend and significantly improve their lives. The company strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of time. Consistent with the responsibility as one of the world's premier innovative biopharmaceutical companies, the company collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, the company has worked to make a difference for all.

Product Description: Inclacumab

Inclacumab, a fully human IgG4 anti-P-selectin monoclonal antibody, is being developed for the reduction of vaso-occlusive crises (VOCs) in patients with SCD. P-selectin is a protein that mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab has the potential to be a best-in-class option for reducing VOCs in people with SCD, with the potential for quarterly, rather than monthly dosing. The drug was being developed by the Global Blood Therapeutics (GBT), which is acquired by the Pfizer. GBT has exclusive worldwide rights to inclacumab as part of the company's licensing agreement with Roche. Inclacumab is in Phase III stage of the development for SCD.

2. Company Overview: Agios Pharmaceuticals

Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company's most advanced drug candidate is a first-in-class pyruvate kinase (PK) activator, mitapivat, which is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development.

Product Description: Mitapivat

Mitapivat is an oral small molecule designed to activate the enzyme pyruvate kinase-R (PKR). This enzyme is important for glycolisis - the process of generating cellular energy by breaking down sugar molecules. When PKR is active, it uses up 2, 3-diphosphoglycerate (2, 3-DPG). In SCD, 2, 3-DPG is thought to accumulate in red blood cells and cause them to become misshapen by prompting hemoglobin molecules to clump together in a process known as polymerization. By activating PKR, Mitapivatis expected to lower the 2, 3-DPG accumulation and thereby lessen hemoglobin polymerization and red blood cell sickling. Agios Pharmaceuticals has initiated a Phase II/III trial called RISE UP to test its investigational therapy Mitapivat (AG-348) in people with sickle cell disease (SCD).

3. Company Overview: Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, Novartis use innovative science and digital technologies to create transformative treatments in areas of great medical need. In quest to find new medicines, the company consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments.

Product Description: Canakinumab

Novartis is developing Canakinumab (ACZ885), a selective, high-affinity, fully human monoclonal antibody that acts as an interleukin-1β (IL-1β) blocker in Phase II stage of development for the treatment of Sickle Cell Anemia. IL-1β is a key cytokine in the inflammatory pathway known to drive the continued progression of inflammatory atherosclerosis. ACZ885 acts by blocking the action of IL-1β for a sustained period of time, therefore inhibiting inflammation that is caused by its over-production. Canakinumab is already approved under the brand name Ilaris for the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) including Familial Cold Auto inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS). Novartis is also developing ACZ885 for preventing cardiovascular risk. Company believe that ACZ885 is the first and only agent which has shown that selectively targeting inflammation significantly reduces cardiovascular risk in patients who have had a prior heart attack and have an increased cardiovascular inflammatory burden. Canakinumab is administered subcutaneously.

Sickle Cell Disease Analytical Perspective

In-depth Commercial Assessment: Sickle Cell Disease Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

Sickle Cell Disease Competitive Landscape

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

Sickle Cell Disease Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

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

Key Players

• CRISPR therapeutics
• Bluebird Bio
• Pfizer
• Novo Nordisk
• Agios Pharmaceuticals
• Alexion Pharmaceuticals
• Takeda
• Prolong Pharmaceuticals
• Roche
• Beam Therapeutics
• Editas Medicine
• Sangamo Therapeutics
• Bellicum Pharmaceuticals
• Invenux
• EpiDestiny
• Hillhurst Biopharmaceuticals
• CSL Behring
• Fulcrum Therapeutics
• Sana Biotechnology

Key Products

• CTX001
• LentiGlobin BB305
• Inclacumab
• Etavopivat
• Mitapivat
• ALXN1820
• TAK-755
• Sanguinate
• Crovalimab
• BEAM101
• EDIT 301
• BIVV003
• BPX-501
• SCD-101
• Nicotinamide
• HBI-002
• CSL889
• FTX-6058
• SG418

This product will be delivered within 3-5 business days.