On December 30, 2020 Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported that the primary objectives were met in the initial part of its Phase 1 trial, which evaluated a single ascending dose of exoIL-12 in healthy volunteers (Press release, Codiak Biosciences, DEC 30, 2020, View Source [SID1234573329]). In this randomized, placebo controlled, double-blind study, exoIL-12 demonstrated a favorable safety and tolerability profile, with no local or systemic treatment-related adverse events and no detectable systemic exposure of IL-12.

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"This is an important milestone, as these results show that exoIL-12 acts in humans as we had expected, based on our preclinical evaluations. The safety and tolerability profile observed here support the target profile that we are hoping to achieve with this candidate," said Benny Sorensen, M.D., Ph.D., Senior Vice President and Head of Clinical Development, Codiak. "We’re looking forward to advancing exoIL-12 into the multi-dose part of the study in cutaneous T cell lymphoma patients and presenting the detailed results from the healthy volunteer part of this study at an upcoming medical conference."

exoIL-12 is the first engineered exosome therapeutic candidate to be evaluated in humans and one of two Codiak programs currently in clinical development. exoIL-12 was engineered using the company’s proprietary engEx Platform and designed to display functional IL-12 on the exosome surface using the exosomal protein, PTGFRN, as a scaffold, the capability of which was identified by Codiak scientists.

IL-12 is a potent anti-tumor cytokine, but prior clinical development conducted by others1 of recombinant IL-12 (rIL-12)-based therapies has generally been hindered by significant safety and tolerability concerns. To overcome these limitations, exoIL-12 was designed to facilitate dose control of IL-12 and limit systemic exposure and associated toxicity by localizing IL-12 in the tumor microenvironment (TME) in order to potentially expand the therapeutic index.

Initial Data from Healthy Volunteers

A total of five cohorts each with five subjects, randomized 3:2 active drug to placebo, were enrolled and dosed in the first part of the Phase 1 study. Each cohort received a subcutaneously administered single ascending dose of exoIL-12: 0.3 µg, 1.0 µg, 3.0 µg, 6.0 µg or 12.0 µg, respectively.

No treatment-related adverse events were observed throughout 10 days of follow-up. In particular, no chills, fever, fatigue, dizziness, myalgia, headache or back pain were reported. These symptoms have been observed in previous clinical studies of subcutaneously administered rIL-12 at comparable doses (ranging from 2 to 12 µg)1 to those used in Codiak’s study of exoIL-12.

Plasma pharmacokinetic (PK) measurements of subjects that received exoIL-12 showed no systemic exposure with levels of IL-12 below the limit of quantification. In contrast, previous rIL-12 clinical studies showed dose-dependent systemic exposure with dosages of 5 and 12 µg resulting in Cmax plasma levels of approximately 15 to 45 pg/ml within 6 to 12 hours after dosing.1

Codiak’s analyses of pharmacodynamic (PD) data from the healthy volunteer portion of the exoIL-12 trial, including skin IL-12 levels and IL-12 signaling from skin punch biopsies collected before and at 24 hours, Day 8 and Day 15 after subcutaneous administration around the injection site, are ongoing and are expected to be available in early Q1 2021. The company intends to use these results to identify an optimal pharmacological dose to carry forward into the second part of the trial, which is on track to begin in Q1 2021 and will evaluate repeat dosing of exoIL-12 in patients with early-stage cutaneous T cell lymphoma (CTCL).

exoIL-12 Development and Ongoing Phase 1 Clinical Trial

Codiak is initially focusing development of exoIL-12 on tumors that have previously shown clinical responses to IL-12 used as a monotherapy, such as CTCL. While the biological rationale for IL-12 as a cancer treatment has been validated in previous human clinical studies, its utility has been severely limited due to serious adverse events caused by systemic exposure.

Codiak has engineered exoIL-12 to display fully active IL-12 on the surface of the exosome, which is designed to facilitate potent local pharmacology at the tumor injection site with precisely quantified doses. Exosomal delivery has demonstrated limited systemic exposure to IL-12 in preclinical models and resulted in significant and prolonged PD activity and both local and systemic anti-tumor immune responses.

The Phase 1 clinical trial is designed in two parts to evaluate safety, tolerability, PK and PD of exoIL-12 after single, ascending, subcutaneous doses in healthy volunteers, followed by repeat dose exoIL-12 into the lesions of stage IA-IIB CTCL patients. Patients with CTCL will be monitored for safety, PK, and PD effects through analysis of blood and tumor biopsies, and for local and systemic anti-tumor efficacy using validated CTCL assessment criteria. Safety, biomarker and preliminary anti-tumor efficacy results from CTCL patients are anticipated in mid-2021.

About exoIL-12

exoIL-12 is Codiak’s exosome therapeutic candidate engineered to display fully active IL-12 on the surface of the exosome, using the exosomal protein, PTGFRN, as a scaffold, and designed to facilitate potent local pharmacology at the injection site with precisely quantified doses. By limiting systemic exposure of IL-12 and associated toxicity, Codiak hopes to enhance the therapeutic index with exoIL-12, delivering a more robust tumor response, dose control and an improved safety profile.

Codiak intends to focus development of exoIL-12 on tumors that have, in previous clinical testing, shown clinical responses to IL-12 used as a monotherapy. This includes cutaneous T cell lymphoma (CTCL), melanoma, Merkel cell carcinoma, Kaposi sarcoma, glioblastoma multiforme and triple negative breast cancer.

About the engEx Platform

Codiak’s proprietary engEx Platform is designed to enable the development of engineered exosome therapeutics for a wide spectrum of diseases and to manufacture them reproducibly and at scale to pharmaceutical standards. By leveraging the inherent biology, function and tolerability profile of exosomes, Codiak is developing engEx exosomes designed to carry and protect potent drug molecules, provide selective delivery and elicit the desired pharmacology at the desired tissue and cellular sites. Through its engEx Platform, Codiak seeks to direct tropism and distribution by engineering exosomes to carry on their surface specific targeting drug moieties, such as proteins, antibodies/fragments, and peptides, individually or in combination. Codiak scientists have identified two exosomal proteins that serve as surface and luminal scaffolds. By engineering the exosome surface or lumen and optimizing the route of administration, Codiak aims to deliver engEx exosomes to the desired cell and tissue to more selectively engage the drug target, potentially enhancing the therapeutic index by improving potency and reducing toxicity.

On December 30, 2020 Incyte (Nasdaq:INCY) and Cellenkos, Inc., a privately held, clinical stage biotech company, reported a development collaboration to investigate the combination of ruxolitinib (Jakafi) and CK0804, Cellenkos’ cryopreserved CXCR4 enriched, allogeneic, umbilical cord blood-derived T-regulatory cells, in patients with myelofibrosis (MF) (Press release, Incyte, DEC 30, 2020, View Source [SID1234573327]). In addition, Incyte has an exclusive option to acquire sole rights to develop and commercialize CK0804, and genetically-modified variants of CK0804, in benign and malignant hematology indications.

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"This collaboration supports our continued commitment to developing new therapeutic options that may improve and expand treatment options for patients with MF, part of a group of rare blood cancers known as myeloproliferative neoplasms (MPNs)," said Steven Stein, M.D., Chief Medical Officer, Incyte. "We are excited to partner with Cellenkos to initiate this study as part of our LIMBER clinical development program, designed to evaluate new monotherapy and combination strategies for patients with MPNs."

"We are delighted to partner with Incyte, a global biopharmaceutical company, to further study and develop CK0804. Incyte’s investment in strong science and R&D excellence makes them an ideal partner to evaluate CK0804 in combination with ruxolitinib as a potential treatment for the many patients living with myelofibrosis, especially those who are transfusion dependent," said Tara Sadeghi, Vice President, Clinical Operations, Cellenkos, Inc. "Our innovative strategy of exploiting the CXCR4/CXCL12 axis to redirect the immune modulatory T-regulatory cells specifically to the diseased bone marrow holds the promise of resolving inflammation to allow for normal hematopoeisis resulting in clinical improvement. This collaboration is in line with our corporate strategy to partner with world-leading major pharma companies in order to maximize access to our innovative cellular medicines."

Per the terms of the agreement, the companies plan to initiate a Phase 1b single arm, open-label study evaluating ruxolitinib in combination with CK0804 in patients with MF. Incyte will fund the study, which will be operationalized by Cellenkos.

In addition, per the agreement, Incyte will have an option to acquire an exclusive global license to develop and commercialize the program. Upon exercising the global licensing option, Incyte would be responsible for all activities and costs associated with research, development and commercialization of the program. Cellenkos would be eligible to receive a $20 million licensing fee and, for each distinct product under the agreement, development, regulatory and sales milestones totaling up to $294.5 million as well as tiered royalties ranging from mid-single digit to low-double digits, if approved.

The collaboration is effective immediately.

About Myelofibrosis (MF)

MF is a rare, chronic blood cancer that is part of a group of diseases known as MPNs. In MF, scar tissue forms in the bone marrow and impairs its ability to produce normal blood cells. This can result in an enlarged spleen, and symptoms such as fatigue, itching and night sweats, which can impact a patient’s quality of life. About 16,000 to 18,500 people in the United States are living with MF.

About LIMBER

Incyte is a leader in the discovery and development of therapies for patients with myeloproliferative neoplasms (MPNs). The Leadership In MPNs BEyond Ruxolitinib (LIMBER) program is designed to evaluate multiple monotherapy and combination strategies to improve and expand treatments for patients with myeloproliferative neoplasms (MPNs). The program currently has three key areas of focus: development of a new, once-daily formulation of ruxolitinib; ruxolitinib-based combinations with new targets such as PI3Kδ, BET and ALK2; and new therapeutic options.

About Ruxolitinib (Jakafi)

Ruxolitinib (Jakafi) is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for the treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, in adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF and for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

About CK0804

CK0804 is a novel allogenic cell therapy product consisting of T-regulatory cells that exploit the CXCR4/CXCL12 axis and are derived from clinical-grade umbilical cord blood units and manufactured using Cellenkos’ proprietary process. The product is cryopreserved and readily available off-the-shelf, without any requirement for HLA matching, and is infused intravenously. One manufacturing campaign can result in multiple doses of cryopreserved product that can be shipped to the clinical site, where it can be stored for an extended period or made available for immediate treatment, as needed.

Important Safety Information for Jakafi (ruxolitinib)

Jakafi can cause serious side effects, including:

Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.

Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.

Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.

Increases in cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.

The most common side effects of Jakafi include: for certain types of MF and PV – low platelet or low red blood cell counts, bruising, dizziness, headache, and diarrhea; and for acute GVHD – low platelet, red or white blood cell counts, infections, and fluid retention.

These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, have a high level of fat in your blood (high blood cholesterol or triglycerides), had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider.

Women should not take Jakafi while pregnant or planning to become pregnant. Do not breast-feed during treatment with Jakafi and for 2 weeks after the final dose.

On December 30, 2020 Propanc Biopharma, Inc. (OTC: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that the Proenzymes Optimization Project 1 ("POP1") joint research and drug discovery program advanced towards producing commercial scale quantities of the two proenzymes trypsinogen and chymotrypsinogen (Press release, Propanc, DEC 30, 2020, View Source [SID1234573326]). The Company’s product candidate is targeting the global metastatic cancer treatment market, projected to be worth US$111.2 Billion by 2027, according to current analysis by Emergen Research.

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The program’s lead research scientist, Mr. Aitor González, synthesized and purified both proenzymes in the laboratory. Once purified, the proenzymes were lyophilized (freeze dried) and each formed a stable, dry white powder. Mr. González then determined the sequence of proteins of each proenzyme by mass spectrometry. He recently started to produce larger quantities of the proenzymes with the objective of establishing their combined anti-cancer effects against pancreatic and colorectal cancers. In addition, research activities were transferred to the MEDINA Foundation Research Center to scale up production. MEDINA is a Non-Profit Research Organization established in 2008 through a public-private alliance between the Regional Government of Andalusia, Spain, the pharmaceutical company Merck Sharp & Dohme de España S.A. (MSD), and the University of Granada. Medina’s scientific platforms support the development of multidisciplinary research programs in Microbiology, Natural Product Chemistry and Screening & Target Validation. For further information, please click on the following link: View Source

The POP1 program is designed to produce a backup clinical compound to the lead product candidate, PRP. The objective is to produce large quantities of trypsinogen and chymotrypsinogen for commercial use that exhibits minimal variation between lots and without sourcing the proenzymes from animals. Propanc is undertaking the challenging research project in collaboration with the Universities of Jaén and Granada, led by research scientist Mr. Aitor González, supported by Dr. Macarena Perán, Ph.D., representing the Universities and Propanc’s Chief Scientific Officer, Dr. Julian Kenyon, M.D.

Mr. James Nathanielsz, Propanc’s Chief Executive Officer said, "By expanding our product pipeline, our vision is to establish a new product class that provides a solution for the treatment and prevention of many recurring and spreading malignant tumors, perceived as untreatable, with less toxicity compared to standard treatments and no immune suppression. This is critical for patients who are at risk of dying from secondary infection, especially in the context of a global pandemic. Through our PRP development and POP1 drug discovery programs we are making positive steps towards achieving our vision."

"Despite a challenging year due to the global pandemic, we made significant advancements in producing synthetic recombinant versions of trypsinogen and chymotrypsinogen," said Dr. Julian Kenyon. "These two active pharmaceutical ingredients combine to form our lead product candidate, PRP, which are currently of animal origin. Our objective is to further stabilize and enhance the combined effects of the two proenzymes when administered to patients."

On December 30, 2020 Hutchison China MediTech Limited ("Chi-Med") (Nasdaq/AIM: HCM) reported that surufatinib has been granted approval for drug registration by the National Medical Products Administration of China ("NMPA") for the treatment of non-pancreatic neuroendocrine tumors ("NETs") (Press release, Hutchison China MediTech, DEC 30, 2020, https://www.chi-med.com/chi-med-announces-the-nmpa-approval-of-surufatinib-sulanda-in-china-for-epnet/ [SID1234573325]). Surufatinib will be marketed in China under the brand name Sulanda. Surufatinib is Chi-Med’s first self-discovered oncology drug to be approved in China without the support of a development partner, and follows the approval of Chi-Med’s first oncology drug, Elunate (fruquintinib capsules), in 2018.

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Christian Hogg, Chief Executive Officer of Chi-Med commented, "We are very pleased to have achieved this major milestone for Chi-Med. The approval of surufatinib, our first un-partnered oncology drug, is a strong testament to our in-house research and development capability."

"Today’s approval also marks an important development for NET patients, for whom there are currently limited treatment options. Compared with other NET therapies available in the market, surufatinib has a unique mode of action by both inhibiting angiogenesis and promoting the body’s immune response against tumor cells. If our second new drug application ("NDA") in pancreatic NET is successful, this therapy would be approved in China to address all NET patients regardless of the tumor origin."

"We look forward to making this unique therapy available to patients as quickly and broadly as possible through our own expanded oncology commercial team."

Chi-Med has established an oncology commercial team that today covers more than 2,000 hospitals across China. The team is led by a leadership team highly experienced in oncology products commercialization in China with deep knowhow in the field of NETs.

The NMPA approval of Sulanda was based on results from the SANET-ep[1] study, a Phase III trial (clinicaltrials.gov identifier: NCT02588170) in patients with advanced non-pancreatic NETs conducted in China. The study met the pre-defined primary endpoint of progression-free survival ("PFS") at a preplanned interim analysis. The positive results of this trial were highlighted in an oral presentation at the 2019 ESMO (Free ESMO Whitepaper) Congress and published in The Lancet Oncology in September 2020.[2] Median PFS was significantly longer for patients treated with surufatinib at 9.2 months, compared to 3.8 months for patients in the placebo group (HR 0.334; 95% CI: 0.223-0.499; p<0.0001). Surufatinib had an acceptable safety profile, with the most common treatment-related adverse events of grade 3 or worse being hypertension (36% of surufatinib patients vs. 13% of placebo patients), proteinuria (19% vs. 0%) and anemia (5% vs. 3%).

In China, there were an estimated 67,600 newly diagnosed NET patients in 2018. Considering the current incidence to prevalence ratio, there may be as many as 300,000 patients living with the disease.[3]

About NETs
NETs form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. NETs are typically classified as pancreatic NET ("pNET") or non-pancreatic NET ("epNET"). Approved targeted therapies include Sutent (for pNET only) and Afinitor for pNET and well-differentiated, non-functional gastrointestinal or lung NET.

According to Frost and Sullivan, there were 19,000 newly diagnosed cases of NETs in the U.S. in 2018. Importantly, NETs are associated with a relatively long duration of survival compared to other tumors. As a result, there were approximately 141,000 estimated patients living with NETs in the U.S. in 2018.

About Surufatinib (Sulanda in China)
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.

Chi-Med currently retains all rights to surufatinib worldwide.

About Surufatinib Development
NETs in the U.S. and Europe: In the U.S., surufatinib was granted Fast Track Designations for development in pNET and epNET in April 2020, and Orphan Drug Designation for pNET in November 2019. A U.S. FDA NDA rolling submission was initiated in December 2020, to be followed by a marketing authorization application (MAA) submission to the European Medicines Agency (EMA) in Europe. The basis to support these filings includes the completed SANET-ep and SANET-p[4] studies, along with existing data from surufatinib in U.S. non-pancreatic and pancreatic NET patients (clinicaltrials.gov identifier: NCT02549937).

Pancreatic NETs in China: The SANET-p study is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pNET in China. It was terminated early as the pre-defined primary endpoint of PFS was met (clinicaltrials.gov identifier: NCT02589821) at an preplanned interim analysis, leading to a second NDA accepted by the China NMPA in September 2020. The results of this study were presented at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020 and published simultaneously in The Lancet Oncology. [5]

Biliary tract cancer in China: A Phase IIb/III study was initiated in March 2019, comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532).

Immunotherapy combinations: Chi-Med has entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317, developed by BeiGene, Ltd.), Tuoyi (toripalimab, developed by Shanghai Junshi Biosciences Co. Ltd.) and Tyvyt (sintilimab, developed by Innovent Biologics, Inc.), which are approved as monotherapies in China.

On December 29, 2020 Xspray Pharma AB (publ) (Nasdaq Stockholm: XSPRAY) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its product candidate, HyNap-Nilo, for the treatment of chronic myeloid leukemia (CML) (Press release, Xspray, DEC 29, 2020, View Source [SID1234649572]). HyNap-Nilo is under development as an improved version of TasignaTM (nilotinib), a leading drug for CML.

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The FDA’s Orphan Drug Designation (ODD) has been granted to Xspray Pharma for nilotinib oral formulation intended for the treatment of chronic myeloid leukemia. The ODD program provides orphan status to drugs intended for the treatment of rare disorders that affect fewer than 200,000 people in the United States. This designation allows developers benefits such as the waiver of filling fees under Prescription Drug User Fee Act (PDUFA), as well as potential marketing exclusivity in the U.S for a period of 7 years, should clinical superiority over the originator drug be demonstrated upon approval of the product.

"We are pleased to have received the orphan designation from the FDA based on the potential clinical benefit of HyNap-Nilo. In this case, the reference product has a food effect on drug exposure that is clearly described in a black box safety warning," says Per Anderson, CEO of Xspray Pharma.

A black box warning appears on a prescription drug’s label and is designed to call attention to serious or life-threatening risks associated with the treatment. In an earlier reported study in healthy volunteers, the results showed that HyNap-Nilo can eliminate the clinically relevant food effect.

"This is also a strong confirmation of our strategy, showing that products developed based on our unique technology platform can play a key role in improving established cancer drugs for the clinical benefit of the patients," continues Per Andersson.

About HyNap-Nilo
HyNap-Nilo is being developed as an improved version of the originator drug TasignaTM (nilotinib) with potential to significantly reduce food effect on the plasma concentration and absorption, increase bioavailability as demonstrated in a previous pilot clinical trial, and decrease the drug-drug interactions with gastric pH modulating drugs. According to the drug label for the marketed PKI nilotinib, patients need to refrain from food intake for two hours before and one hour after administration of the drug which is given twice daily. In a completed cross-over Phase I clinical trial, Xspray Pharma measured the exposure of its proprietary HyNap-Nilo formulation in healthy individuals. When administered in the fasted state, a HyNap-Nilo dose of 150 mg produced the same area under the curve values as those reported for a dose of 400 mg of the marketed product. After a high-fat meal the study showed an increase in drug exposure of 25% for HyNap-Nilo, measured both as peak concentration (Cmax) and AUC. For the marketed product, the corresponding increases after a high-fat meal are reported to be 112% and 82%, respectively. In 2019 sales of TasignaTM amounted to USD 804 million in the U.S.

Chronic myeloid leukemia – CML
CML is a type of blood cancer where the body produces malignant white blood cells. Almost all patients with CML have an abnormality known as the "Philadelphia chromosome," which produces a protein called BCR-ABL. This protein aids the proliferation of malignant white blood cells in affected patients. About 15% of all leukemia is CML. In 2020 it is projected that 8,450 people in the US will be diagnosed with CML and in 2017, there were an estimated 58,000 people living with the disease in the US.