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Emerging Paradigms in the Sickle Cell Disease Treatment Market

11 Dec Emerging Paradigms in the Sickle Cell Disease Treatment Market

According to an estimate by the Centers for Disease Control and Prevention (CDC), SCD affects over 100,000 Americans and is a major public health concern as it resulted in 75,000 hospitalizations between 1989 through 1993, and cost over $475 million. The Medical expenditure of children with SCD averaged $11,702 for those with medical coverage, and $14,772 for children with employer-sponsored insurance, with at least one hospital stay in 2005.

There is no cure for this disorder and the treatment is aimed at alleviating the symptoms and delaying the progression of the disease. New Born Screening and blood tests are used for initial diagnosis and are followed by medication and monitoring to prevent complications. Stem Cell transplant, bone marrow transplantation and blood transfusions are the more advanced treatment options but are limited by the availability of donors, efficacy of treatment in adults and costs.

The field of Sickle Cell Treatment is witnessing the emergence of groundbreaking research in the form of gene therapy, nitric oxide therapy and inhibition of the formation of sickle cells by boosting hemoglobin production. We take a look at few companies that are at the forefront of innovation in this area.

Syros Pharmaceuticals (NASDAQ: SYRS)

Market Cap: $202M; Current Share Price: 4.77 USDChart
Data by YCharts

The Company’s core area of interest is controlling and regulating gene expression, to develop therapeutics that leverages its proprietary gene control platform. Syros uses its path breaking platform to identify disease-causing genes, and develop therapeutics to control them, thereby addressing the need for a cure for disease, which was hitherto sidelined for drug discovery and development, owing to a poor understanding of their gene expression.

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The Company has identified and validated a novel fetal hemoglobin repressor through the use of its broad broader drug discovery effort in sickle cell disease, which it intends to highlight at the 61st American Society of Hematology (ASH) Annual Meeting taking place December 7-10 in Orlando, Florida, as per a Company statement.

SY-1365, its lead candidate for the treatment of ovarian and breast cancers, is a first-in-class potent and selective inhibitor of the cyclin-dependent kinase 7 (CDK7). It is currently undergoing phase I clinical trials to study its efficacy and safety. In preclinical trials, it has demonstrated an increased biological activity, a strong anti-tumour activity with complete regressions, lowered uncontrolled cell cycle progression and lowered expression of cancer causing genes.

In addition, its pipeline consists of SY-1425, a first-in-class oral selective retinoic acid receptor alpha (RARα) agonist for the treatment of acute myeloid leukemia (AML), which is currently undergoing phase 2 clinical trials. SY-5609, an oral l CDK7 inhibitor is undergoing preclinical studies for treatment of Cancer, while the Company is also focused on discovering candidates Myeloproliferative neoplasms.

Syros has a worldwide strategic collaboration and option agreement with Incyte Corporation (NASDAQ: INCY), for discovering and developing up to seven novel therapeutic targets for myeloproliferative neoplasms (MPNs).


Market Cap: $3.8B; Current Share Price: 67.63 USDChart
Data by YCharts

The Company uses a CRISPR-associated (CAS) endonuclease, or enzyme, that acts as “molecular scissors” to cut DNA at a location specified by a guide RNA to deliver cutting–edge therapies for treatment of numerous genetic diseases. The technology was co-invented by Dr. Emmanuelle Charpentier, one of its scientific founders. It is focused on creating solutions for unmet medical needs by targeting gene editing and cellular engineering using its proprietary methodology.

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Crispr Therapeutics’ Sickle Cell Disease and Beta-Thalassemia treatment approach is centered on editing-genes to increase the fetal hemoglobin count, by substituting HbF for the diseased hemoglobin in β-thalassemia and SCD patients. Its lead product candidate CTX001, uses a patient’s own blood cells and edits them using CRISPR/Cas9 to promote HbF expression. This program is being developed in association with Vertex Pharmaceuticals (NASDAQ: VRTX). It is currently being evaluated in a phase 1 / 2 study, with a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs), in Subjects with Transfusion-Dependent β-Thalassemia. It is expecting topline results from the study in May 2022. In April 2019, CTX-001 received a fast track designation from the FDA.

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In addition its pipeline consists of CTX-110 for Immuno-oncology applications, Regenerative medicine aimed at Diabetes Mellitus Type I and genetic diseases such as Duchenne Muscular Dystrophy (DMD), Cystic Fibrosis and Hurler Syndrome.

Akebia Therapeutics (NASDAQ: AKBA)

Market Cap: $762M; Current Share Price: 6.41 USDChart
Data by YCharts

The Company’s lead product candidate, Vadadustat, is a hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, which can aid in improved red blood cell production and oxygen delivery to tissues. The candidate is based on Nobel Prize-winning science, and utilizes HIF PHIs to replicate the body’s response to lower levels of oxygen, thereby increasing the availability of HIF, a protein essential for erythropoietin synthesis and the regulation of iron metabolism.

The candidate is undergoing clinical evaluation for its safety, efficacy and tolerability in the treatment of anemia due to CKD, in dialysis dependent and non-dialysis dependent adult patients, in two global Phase 3 programs – PRO2TECT (non-dialysis) and INNO2VATE (dialysis). The results from the studies are expected in mid-2020 and Q2, 2020 respectively. In addition the candidate has also undergone two Phase 3, active-controlled, pivotal studies in Japanese subjects with anemia due to CKD, besides two additional Phase 3 single-arm studies in peritoneal and hemodialysis subjects, conducted by Mitsubishi Tanabe Pharma Corporation (OTCPINK: MTZPY), the Company’s partner in Japan and other Asian markets.

Mitsubishi Tanabe Pharma Corporation submitted a Japanese New Drug Application (JNDA) in June 2019, on the basis of positive topline data from the above mentioned studies. In addition the Company has entered into numerous strategic collaborations with Otsuka,Vifor Pharma, Janssen, Panion & BF Biotech and Japan Tobacco Inc.

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The Company’s core focus remains kidney disease and it is focused on adding to its portfolio through internal research and development, in-licenses, strategic collaborations and acquisitions. Akebia markets AURYXIA (ferric citrate) in the U.S, which is indicated for the control of serum phosphorus levels in adult patients with chronic kidney disease on dialysis as well as for the treatment of iron deficiency anemia in adult patients with chronic kidney disease not on dialysis as per the Company.

Pieris Pharmaceuticals (NASDAQ: PIRS)

Market Cap: $207M; Current Share Price: 3.55 USDChart
Data by YCharts

Pieris Pharmaceuticals develops Anticalin-based drugs for treatment of conditions related to immuo-oncology, respiratory diseases such as Asthma and Anemia. Anticalin proteins are engineered versions of lipocalins that are human proteins having the ability to bind, store and transport molecules, which the Company has leveraged for delivering drug candidates binding to a broad spectrum of therapeutic targets, in the clinic and in partnerships with leading pharmaceutical companies as per the Company. The technology was licensed from the Technische Universität München (TUM) and provides it exclusive access to Anticalin-related IP.

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In 2017, the Company signed deals with Servier in immuno-oncology and with AstraZeneca (NYSE: AZN) in respiratory diseases, retaining the co-development and co-commercialization rights for several programs focused on addressing challenging diseases. Pieris has more than fifty patent families as of January 2017, comprising of key patents granted in the United States, Europe and Asia, covering the Anticalin® protein as a drug class, libraries of Anticalin proteins and all derived products.

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Pieris has entered into a Japanese partnership for PRS-80, its lead candidate for Anemia, which is undergoing a Phase IIa clinical study. The Company intends to out-license the candidate in other markets following the completion of its trials. The candidate has demonstrated a favorable safety profile and efficacy, showing a significant decrease in plasma hepcidin and an increase in serum iron concentrations and transferrin saturation. The Company is currently engaged in a clinical trial in anemic patients with chronic kidney disease undergoing hemodialysis.

Fulcrum Therapeutics (NASDAQ: FULC)

Market Cap: $301M; Current Share Price: 12.91 USDChart
Data by YCharts

FTX-6058, an oral therapeutic that regulates expression of fetal hemoglobin (HbF), is the Company’s lead candidate for the treatment of SCD. The main focus of the Company is to treat genetic diseases by targeting their root cause, this is achieved by adopting a biomarker-driven drug discovery and development strategy that uses patient-derived cell models. In addition, the acquired cells undergo a long-term culturing to allow target identification that are then screened using robust assays using relevant models.

Fulcrum uses it proprietary small molecule compound database named FulcrumSeek and a customized CRISPR-Cas9 guide library to test multiple chemogenomic and genomic screening approaches for candidate identification and validation. Furthermore the Company uses a genome-wide profiling to understand drug/target interaction and understand its impact on cell health.

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The Compnay’s pipeline consists of candidates intended for the treatment of  Facioscapulohumeral muscular dystrophy(FSHD), beta-thalassemia DMD, Friedreich Ataxia, Myotonic Dystrophy 1 and α‑Synucleinopathies covering Muscle, CNS and Blood Disorders. Fulcrum plans to introduce 6 additional screens in 2020, besides filing an Investigational New Drug (IND) application for FTX-6058 for the treatment of SCD and β-Thalassemia in mid-2020.

Robert J. Gould, Ph.D., Fulcrum’s president and chief executive officer, commented

“We look forward to advancing these two programs through our ongoing Phase 2 trials with losmapimod and our IND-enabling studies with FTX-6058 and utilizing our product engine to identify and validate drug targets to address diseases caused by the mis-expression of certain genes”

As per its Q3, 2019 financial results, as of September 30, 2019, the Company’s cash and cash equivalents were $101.6 million, as compared to $72.8 million as of December 31, 2018, which are sufficient to fund the operation until 2021.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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