Blood Cancer Symptoms That Shouldn’t Be Ignored: Early Warning Signs and Diagnosis

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Published on October 9, 2023

By Dr. Suraj D Chiraniya, Clinical Hematologist,  Hemato-oncologist and BMT Physician, HCG Cancer Centre, Borivali

A type of cancer that affects the blood cells. Some common types of blood cancer include leukaemia, lymphoma, and myeloma. Though rare, Myelodysplastic Syndromes (MDS) and Myeloproliferative Neoplasms (MPN) are some other types of blood cancer.

Caused by mutations in DNA within the blood cells, blood cancer results in blood cells behaving abnormally. Treatment, symptoms, and prognosis will vary depending on the type of blood cancer. Further, some types of blood cancer affect children, for which treatment can vary.

Understanding The Various Types Of Blood Cancer

One of the most common types of blood cancer affecting most blood cancer patients is leukaemia, defined as cancer of the blood cells. There are three main categories of blood cells: Red Blood Cells (RBC), White Blood Cells (WBC) and platelets. Leukaemia affects the WBC within the bloodstream and causes them to divide rapidly and eventually crowd out the normal cells. As a result of this, the WBC is unable to perform its function of fighting infections within the human body.

Lymphoma is another type of blood cancer that affects the human immune system. It specifically affects white blood cells called lymphocytes, a vital part of the immune system. Lymphoma can also be called a cancer of the lymphatic system or lymphatic cancer.

In general, there are two main types of lymphoma, namely Hodgkin lymphoma and non-Hodgkin lymphoma, which both affect the blood’s lymphocytes.

The third most common type of blood cancer is myeloma, which affects the blood’s plasma cells. Plasma cells help fight infections in the body, and when a person has myeloma, the body cannot make these antibodies properly. Additionally, abnormal cells multiply in this situation, causing various symptoms.

The majority of the people diagnosed with myeloma are men over the age of 70, although cases of it occurring in young men are also possible.

Who is at risk for blood cancer?

Although there are no specific conditions for blood cancer, the risk can sometimes increase with age. Further, a family history of this disease can also increase the risk.

In addition to the above, the risk of leukaemia can also increase due to the following factors:

•            exposure to radiation

•            treatment with certain chemotherapy drugs

•            a past diagnosis of blood cancer

•            Down syndrome and other genetic syndromes

•            Smoking

Risk factors for lymphoma, on the other hand, include the following:

•            exposure to radiation

•            Epstein-Barr or human T-cell lymphotropic virus

•            HIV, organ transplant, or genetic immune disorders

Some risk factors for myeloma include exposure to radiation, obesity, and other plasma cell diseases.

What are some common signs and symptoms of blood cancers?

Since blood cancer is a chronic condition that progresses slowly, symptoms may not appear until the end stage. While coughing, chest pain, frequent infections, fever and unexplained weight loss can occur in all the types of blood cancer, other symptoms may be specific to the condition.

Some symptoms of leukaemia include fever and lethargy, paleness and shortness of breath due to anaemia and increased bruising and bleeding. When it comes to lymphoma, symptoms may include swollen lymph nodes, fever, night sweats and fatigue and unexplained weight loss. Finally, in the case of myeloma, specific signs may include bone pain, particularly in the back and ribs, weakness, fatigue, and paleness due to anaemia and frequent bacterial infections, such as pneumonia.

Can blood cancer be prevented?

While there is no way to prevent blood cancer completely, certain lifestyle factors can reduce the risk of developing cancer. These include maintaining a healthy diet, exercising regularly, and not indulging in habits like smoking and excessive alcohol consumption.

Preventive health check-ups are highly recommended, especially if a person has a personal or family history of blood cancer, previous sessions of radiation therapy/chemotherapy or radiation exposure. If a person has experienced any symptoms related to blood cancer, it is best to consult an expert, as early diagnosis can ensure the best possible outcome.

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NCCN-Directed Guidelines Driving PV Treatment in Clinical Practice

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September 22, 2023

Aaron Gerds, MD, MS: The NCCN [National Comprehensive Cancer Network] guidelines for polycythemia vera and the other MPNs [myeloproliferative neoplasms] are updated regularly. So certainly there’s a large effort to annually update them, where we go over the entire sets of guidelines to update and refine them. But as new developments come along, we update on the fly. Say a new therapy is approved for polycythemia vera. We would quickly add that to the guidelines in an ad hoc update, just simply keeping the link to the NCCN and guidelines readily available, so whatever is on the NCCN guidelines website is the most up-to-date version. Printing it out and having it on your desk might [allow it to] become outdated at some point. So certainly relying heavily on the website. The NCCN also has apps available where the guidelines are automatically updated within the app, so you don’t have to worry about visiting the website or printing out new guidelines. And the NCCN, as well as other entities, support regular education efforts, both virtually and in person. There’s the annual NCCN Hematologic Malignancies conference, where the guidelines are a key piece of that, where updates are given not only within the disease state, but specifically with an eye to the guidelines. Within the MPN world, there are lots of conferences and educational opportunities as well, both virtually and in-person. The big event here in North America is the American Society Hematology annual meeting, where updates are given within the disease field. But, certainly, looking toward the NCCN, the resources available there can keep you up to date, especially a lot of people have found the app to be very, very helpful.

Adherence to the NCCN guidelines for polycythemia vera is actually kind of a chicken and the egg issue. What came first? I think NCCN guidelines reflected everyday practice of taking care of patients with polycythemia vera before they were invented. So the MPN guidelines in general are relatively new compared to other guidelines for breast cancer or colon cancer. At that time, we focused on what the current practice was. Identifying patients [as] high risk, low risk,…reductive therapies in the high-risk cases, and so on and so forth. Now, over time, we’ve had to adapt the guidelines for new therapies, in particular the approval of ropeginterferon, and incorporate that in additional data has been published about ruxolitinib, particularly the MAJIC-PV trial. We’ve incorporated this information into the guidelines, which has altered or strengthened some of our recommendations. So certainly I think the guidelines…will help guide someone learning how to take care of patients with polycythemia vera. But I think it really does reflect the best practice in how practice is sustained throughout North America. So it’s kind of a bidirectional effort, where absorbing what is currently the standard of care practice [and] putting [it] in the guidelines to then help unify practice throughout the country. And while there [is] some data out there that looks at adherence to the guidelines in everyday practice, I would say that polycythemia vera is actually one of the ones that is a little bit more stringent. There are fewer treatment options. Kind of clear, low risk, high risk, “what do you do” process. So I think it’s a lot easier to take those guidelines and adhere to them closely.

Transcript is AI-generated and edited for clarity and readability.

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MWTX-003 Wins FDA Fast Track Designation for Polycythemia Vera

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Kristi Rosa

The FDA has granted fast track designation to the investigational, anti-TMPRSS6 monoclonal antibody, MWTX-003 (DISC-3405), for use in the treatment of patients with polycythemia vera, according to an announcement from Disc Medicine, Inc.1

Patients with hematologic diseases such as polycythemia vera, myelodysplastic syndrome (MDS), and beta-thalassemia are known to develop high levels of iron, which leads to survival and quality-of-life complications.2 MWTX-003 was designed to boost the production of hepcidin, which suppresses serum iron. Preclinical data in animal models of beta-thalassemia and polycythemia vera have confirmed this ability.

“We are delighted to have received fast track designation for MWTX-003, which highlights the unmet need for [patients with] polycythemia vera and the potential of MWTX-003 in a disease where there are few treatment options,” John Quisel, JD, PhD, president and chief executive officer of Disc Medicine, Inc., stated in a press release.1 “We believe MWTX-003 is uniquely positioned to address the needs of [patients with] polycythemia vera and are excited to initiate a phase 1 trial in the coming months.”

Preclinical studies have demonstrated strong pharmacodynamic effects that are reflective of TMPRSS6 inhibition.3 Specifically, a single administration of MWTX-003 led to an approximate 70% suppression of serum iron that lasted for 3 weeks. Moreover, in non-clinical GLP safety studies, the agent showcased a strong toxicity profile.

In a model of beta-thalassemia, treatment with MWTX-003 resulted in significant effects on disease hallmarks such as iron overload, ineffective erythropoiesis, and splenomegaly. The production of hepcidin was boosted up to 4-fold, serum and liver iron was reduced by approximately 60% to 65%, red blood cell production increased, and spleen weight decreased.

MWTX-003 was in-licensed from Mabwell Therapeutics, and in November 2022, the FDA accepted an investigational new drug application for the agent.1 In January 2023, the clinical-stage biopharmaceutical company shared development plans for MWTX-003 which consisted of establishing phase 1 proof-of-mechanism; this was planned for initiation in the second half of 2023, and would examine hepcidin, iron, and other hematologic parameters.3

They also shared plans to advance the agent into point-of-care studies focused on polycythemia vera. In a phase 1b/2a proof-of-concept study, they hope to evaluate the safety and pharmacokinetic profile of MWTX-003 in patients with polycythemia vera. These data could provide clarity on the regulatory development path for the agent, according to Disc Medicine.

There is interest in examining the agent in additional POC studies spanning a range of indications, including hereditary hemochromatosis, beta-thalassemia, and MDS.

References

  1. Disc Medicine receives FDA fast track designation for MWTX-003 for the treatment of polycythemia vera. News release. Disc Medicine, Inc. September 20, 2023. Accessed September 21, 2023. https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-receives-fda-fast-track-designation-mwtx-003
  2. MWTX-003. Disc Medicine, Inc. website. Accessed September 21, 2023. https://www.discmedicine.com/our-pipeline/mat-2-inhibitor/
  3. Novel anti-TMPRSS6 monoclonal antibody portfolio: exclusive in-licensing agreement with Mabwell Therapeutics. Disc Medicine, Inc. January 20, 2023. Accessed September 21, 2023. https://ir.discmedicine.com/static-files/549caf12-e7be-45ff-8667-86908e4e6bdd

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Ruxolitinib Receives Positive NICE Opinion for Polycythemia Vera

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Russ Conroy

The National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending the approval of ruxolitinib (Jakafi) as a treatment for adult patients with polycythemia vera that is intolerant or resistant to hydroxycarbamide or hydroxyurea, according to a press release from Novartis.1 This may help to make the agent available for patients residing in England and Wales.

“We welcome this recommendation from NICE, as polycythemia vera can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms,” Jon Mathias, co-chair of MPN Voice, said in the press release. “Ruxolitinib addresses a significant unmet need in patients who cannot tolerate or no longer respond to [hydroxycarbamide/hydroxyurea].”

According to a previous report, treatment with hydroxycarbamide or hydroxyurea leads to the development of resistance or intolerance in 24% of patients with polycythemia vera, which correlates with a higher risk of disease progression.2

In an international MPN Landmark survey, 72% of patients with polycythemia vera reported that they experienced reduced quality of life (QOL) due to disease symptoms.3 Additionally, 14.0% of surveyed patients with polycythemia vera reported that they were experiencing emotional hardship due to their condition, and 29.0% stated that they felt worried or anxious about their disease. A further 33.0% of the surveyed polycythemia vera population stated that they experienced impairment at work, and 40.3% reported impairment with respect to overall activity.

“There is a significant unmet need for people with polycythemia vera in England and Wales, who live with a large symptom burden as a result of their condition,” Claire Harrison, MD, FRCP, FRCPath, consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, said in the press release.1 “Today’s decision is a step in the right direction for providing additional treatment options that reduce the burden of these symptoms and improve disease progression, in this under-represented patient population.”

Investigators sent the MPN LANDMARK survey to patients with polycythemia vera, myelofibrosis, and essential thrombocythemia, and physicians across the United Kingdom, Australia, Germany, Canada, Japan, and Italy from April 2016 to October 2016. The survey was designed to evaluate how MPNs affected QOL, patients’ ability to work, and implementation of disease-management strategies.

Patients 18 years and older diagnosed with myelofibrosis, polycythemia vera, or essential thrombocytopenia were able to respond to the survey. Those enrolled on clinical trials were not eligible to take the survey.

Overall, 219 physicians and 699 patients—including 174 with myelofibrosis, 223 with polycythemia vera, and 302 with essential thrombocythemia—completed the survey.

The FDA approved ruxolitinib as a treatment for patients with polycythemia vera who are intolerant to hydroxyurea in December 2014.4 Supporting data for the FDA approval came from the phase 3 RESPONSE trial (NCT01243944), in which treatment with ruxolitinib produced improvements in hematocrit control and spleen volume reductions compared with best available therapy. Additionally, a higher number of patients receiving ruxolitinib experienced complete hematologic remission. Investigators reported that the most common hematologic adverse effects included thrombocytopenia and anemia.

References

  1. NICE recommends Novartis Jakavi® (ruxolitinib) for patients living with polycythaemia vera (PV). News release. Novartis. September 14, 2023. Accessed September 15, 2023. https://shorturl.at/hixAD
  2. Alvarez-Larran A, Pereira A, Cervantes F, et al. Assessment and prognostic value of the European LeukemiaNet Criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera. Blood. 2012;119(6):1363-1369. Doi:10.1182/blood-2011-10-387787
  3. Harrison CN, Koschmieder S, Foltz L, et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol. 2017;96(10):1653-1665. doi:10.1007/s00277-017-3082-y
  4. FDA approves Jakafi® (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera. News release. Incyte Corporation. December 4, 2014. Accessed September 15, 2023. https://shorturl.at/aARU3

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Novartis makes sculptures to shape knowledge of cancer symptoms

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By Nick Paul Taylor

September 18, 2023

Novartis has enlisted artists to help raise awareness of rare blood cancers, commissioning sculptures that portray the 10 key symptoms of the conditions to create a temporary exhibition that will tour the U.K.

The art exhibition is focused on symptoms of myeloproliferative neoplasms (MPN), a group of rare blood cancers that affect around 4,100 people in the U.K. each year. Novartis sells Jakavi for the treatment of MPNs in the U.K., recently securing coverage for one of the three main types of the blood cancer, and wants to spread knowledge that enables more cases to be identified early.

MPNs cause symptoms, such as fatigue, loss of concentration and night sweats, that overlap with other conditions, leading to delayed diagnoses. Novartis wants to bump MPNs up the list of potential causes that patients and physicians consider when they encounter the symptoms.

“More needs to be done to recognize and identify these symptoms early so that people living with the condition can seek help from medical professionals to better manage their symptoms, and those who are undiagnosed can seek a potential diagnosis sooner,” Alisia O’Sullivan, a MPN patient and MPN Voice volunteer, said in a statement.

To support that goal, the Swiss drugmaker has identified 10 key symptoms and commissioned sculptures to represent each of them. Each sculpture is in the shape of the number assigned to the symptom and features art that portrays the symptom. For example, symptom two, inactivity, displays a painting of someone sleeping under a blue sky and symptom three, weight loss, features a quote about being thin.

Novartis worked with MPN Voice and five mural artists from around the U.K. to create the sculptures and began showing them at Westfield, a shopping center in London, last week. The art exhibition will run for one week at Westfield and then go on a tour of U.K. cities.

Encouraging people to consider MPNs as an explanation for the 10 symptoms could support growth products for the condition. Novartis’ sales of Jakavi increased by 11%, rising to $435 million, in the second quarter on the back of demand in emerging markets, Japan and Europe.

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FDA Grants Approval to GSKs Ojjaara A GameChanger in Myelofibrosis Treatment

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by Yasmim Mendonça
 September 18, 2023

As of September 18, 2023, the United States Food and Drug Administration (FDA) has granted approval to GSK’s groundbreaking medication, Ojjaara (momelotinib), for the treatment of intermediate or high-risk myelofibrosis in adults suffering from anemia. This approval encompasses both primary myelofibrosis and secondary myelofibrosis, including post-polycythemia vera and post-essential thrombocythemia cases. Ojjaara is the sole authorized treatment that effectively addresses the primary symptoms of myelofibrosis, such as anemia, constitutional symptoms, and splenomegaly, making it a game-changer in the field of medicine.

Myelofibrosis, a form of blood cancer, affects a significant number of individuals in the United States, with an estimated 25,000 patients grappling with this condition. The approval of Ojjaara marks a monumental milestone for GSK, as it introduces a formidable rival to the widely-used Incyte drug, Jakafi. With this groundbreaking development, patients diagnosed with myelofibrosis now have an additional treatment option that holds immense promise in effectively managing their condition and improving their quality of life.

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Inflammation and the Development of Leukemia Are Connected

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Penny Min

Published September 15, 2023

In blood stem cells with p53 mutations — the so-called “guardian of the genome” — research has uncovered hitherto unrecognized impacts of persistent inflammation on the emergence of cancer.

The protein p53, generated by the gene TP53, is regarded as “the guardian of the genome,” according to research published in Nature Genetics. Apoptosis, a process by which cells “self-destruct” to stop themselves from procreating additional damaged cells, is triggered when p53 is activated. However, mutations can make p53 ineffective, which allows injured cells to continue dividing unchecked.

As many as 50% to 60% of human malignancies have a TP53 mutation, which can result in cancer development. Hematopoietic stem cells (HSCs) with TP53 mutations have been associated with acute myeloid leukemia (AML), an aggressive kind of blood cancer.

By creating all different types of blood cells, they are in charge of preserving a healthy blood system. The processes behind how these mutant HSCs multiply to produce cancer were largely unknown. In the current study, researchers from the University of Oxford examined how chronic inflammation affects TP53-mutant HSCs in cancer development.

The study team used TARGET-seq, a single-cell method, to examine the impact of the mutation. This enabled them to use cells provided by individuals with myeloproliferative neoplasms, a condition that predisposes them to leukemia, to explore how TP53 mutations in HSCs impact cancer progression.

What did the results entail?

Researchers discovered that cells from individuals with TP53 mutations exhibited higher levels of inflammation-related gene activation. They established, using laboratory mice, that these mutant cells multiplied when the animals were exposed to inflammatory stimuli.

Additionally, compared to healthy HSCs, the mutant HSCs generated fewer white blood cells and were more resistant to cell death, which is normally brought on by inflammation. This indicates that compared to non-mutant HSCs, the mutated HSCs were better able to grow when exposed to inflammation and were more “fit” to survive.

The inability of TP53-mutated cells to effectively repair mistakes in their genetic coding when subjected to inflammation may exacerbate this impact and aid in cancer growth.

“Overall, these findings offer valuable insights into how genetic defects and inflammation interact in the development of blood cancer.”

– Co-first author Alba Rodriguez-Meira

Additionally, she continues that this research may lead to novel approaches for TP53-mutant leukemia therapy and other cancer types, improving results for cancer patients.

The connection between inflammation and genetic evolution in cancer has broad implications, says senior author Adam Mead. The challenge is to figure out how scientists might intervene in this process to treat or prevent the inflammation linked to cancer progression more effectively.

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Momelotinib Receives FDA Approval for Myelofibrosis With Anemia

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Ariana Pelosci

The News

The FDA has approved momelotinib (Ojjaara) for the treatment of patients with intermediate- or high-risk myelofibrosis, including primary and secondary myelofibrosis, who are experiencing anemia, according to a press release from GSK.1

The approval was based on results from the phase 3 MOMENTUM trial (NCT04173494), which was previously presented at the 2022 American Society of Clinical Oncology Annual Meeting, with other supporting data coming from a subpopulation of the phase 3 SIMPLIFY-1 trial (NCT01969838). 2,3

Momelotinib, a once daily oral JAK1/2 inhibitor, is the only approved treatment for this indication.

Expert Perspective

“I think [momelotinib] will make an immediate impact. There clearly are individuals now who are on JAK inhibitors like ruxolitinib [Jakafi] or fedratinib [Inbrec] who have significant anemia who will immediately be potential candidates,” Ruben A. Mesa, MD, said in an interview with CancerNetwork® prior to the approval. “We’ll see how the [National Comprehensive Cancer Network] guidelines form but there’s a case to be made for consideration [for momelotinib] as the initial JAK inhibitor selected for people who have significant anemia.”

Mesa is the president of the Enterprise Cancer Service Line and senior vice president at Atrium Health; executive director of the National Cancer Institute-designated Atrium Health Wake Forest Baptist Comprehensive Cancer Center; and vice dean for Cancer Programs at Wake Forest University School of Medicine

The MOMENTUM Trial

The MOMENTUM trial included 195 patients who were randomly assigned 2:1 to receive either momelotinib (n = 130) at 200 mg per day plus placebo or danazol (n = 65) at 600 mg per day plus placebo. At week 24 patients in the danazol arm were allowed to crossover to the momelotinib arm. The primary end point was total symptom score at week 24, and the secondary end points included transfusion independence and splenic response rate.

In the momelotinib arm, 27.7% of patients discontinued treatment for several reasons including adverse effects (AEs; n = 16), patient decision (n = 6), or death (n = 4). In the danazol arm, 41.5% of patients discontinued treatments because of AEs (n = 11), patient decision (n = 5), or death (n = 3). Additionally, 4 patients crossed over to the momelotinib arm early.

The median age in the momelotinib arm was 71.0 years vs 72.0 years in the danazol arm, 60.8% vs 67.7% were male, and 82.3% vs 76.9% were White, respectively. In terms of myelofibrosis subtype, 60.0% of those in the momelotinib arm had a primary subtype vs 70.8% in the danazol arm, 20.8% vs 16.9% had post-polycythemia vera, and 19.2% vs 12.3% had post-essential thrombocytopenia.

At week 24, the total symptom score response rate was 24.6% (95% CI, 17.49%-32.94%)in the momelotinib arm vs 9.2% (95% CI, 3.46%-19.02%) in the danazol arm (P = .0095). Moreover, 40.0% (95% CI, 31.51%-48.95%) of patients in the momelotinib arm had a 25% reduction in splenic volume vs 6.2% (95% CI, 1.70%-15.01%; P <.0001) in the danazol arm. Additionally, 35% reduction in spleen volume was observed in 23.1% (95% CI, 16.14%-31.28%) in the momelotinib arm and 3.1% (95% CI, 0.37%-10.68%; P = .0006) in the danazol arm.

At baseline, the transfusion independence rate at baseline was 13% in the momelotinib arm vs 15% in the danazol arm. Comparatively, the rate week 24 was 31% in the momelotinib arm vs 20% in the danazol arm (P = .0064).

SIMPLIFY-1 Trial

In this randomized, multicenter study, momelotinib was investigated vs ruxolitinib (Rituxan) in patients who had not received prior treatment with a JAK inhibitor. A total of 432 patients were analyzed with patients received 200 mg orally daily of momelotinib or 20 mg of ruxolitinib once per day.

A 50% of more reduction in the total symptom score was observed in 28.4% of patients receiving momelotinib vs 42.2% receiving ruxolitinib (P = .98). Momelotinib improved the transfusion rate, transfusion independence, and transfusion dependence (P ≤ .19).

Safety

In terms of safety findings from the MOMENTUM trial, the most common grade 3 or higher nonhematologic AEs included acute kidney injury (3.1% vs 9.2%), nausea (2.3% vs 3.1%), and dyspnea (2.3% vs 1.5%) in the momelotinib and danazol arms, respectively. Hematologic AEs of grade 3 or higher included anemia (60.8% vs 75.4%), thrombocytopenia (27.7% vs 26.2%), and neutropenia (12.3% vs 9.2%) in the momelotinib and danazol arms, respectively.

Grade 3 or higher AEs occurred in 53.8% vs 64.6%, and serious AEs occurred in 34.6% vs 40.0% of patients in the momelotinib and danazol arms, respectively. Investigators reported a hazard ratio (HR) of 0.734 (95% CI, 0.382-1.409; P = .3510) for overall survival overall and 0.506 up to week 24 (95% CI, 0.238-1.076; P = .0719).

Safety data from the SIMLIFY-1 trial indicated that AEs occurred in 7% of patients who received momelotinib vs 3% who received ruxolitinib. In 10% of patients, treatment-related neuropathy occurred with momelotinib treatment vs 5% with ruxolitinib.

References

  1. Ojjaara (momelotinib) approved in the US as the first and only treatment indicated for myelofibrosis patients with anaemia, News release. GSK. September 15, 2023. September 15, 2023.
  2. Mesa RA, Gerds AT, Vannucchi A, et al. MPN-478 MOMENTUM: phase 3 randomized study of momelotinib (MMB) versus danazol (DAN) in symptomatic and anemic myelofibrosis (MF) patients previously treated with a JAK inhibitor. J Clin Oncol. 2022;40(suppl 16):7002. doi:10.1200/JCO.2022.40.16_suppl.7002
  3. Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: A phase III randomized trial of momelotinib versus ruxolitinib in janus kinase inhibitor–naïve patients with myelofibrosis. J Clin Oncol. 2017;34(suppl 34):3844-3850. doi:10.1200/JCO.2017.73.4418

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PharmaEssentia and MPN Advocacy & Education International Launch New Educational Initiative to Empower People Living With Polycythemia Vera (PV)

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Published: Sep 14, 2023

PV&ME™ campaign features personal stories from people living with PV and their journeys navigating the rare blood cancer

BURLINGTON, Mass. & EAST LANSING, Mich.–(BUSINESS WIRE)– PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, and MPN Advocacy & Education International, a leading advocacy group dedicated to providing the knowledge, support and resources patients will need as they adjust to living with a myeloproliferative neoplasm (MPN), today announced a new educational initiative for the MPN community called PV&ME. The goal of the campaign is to bring to light the unique and challenging experiences of living with polycythemia vera (PV) in the hopes of raising awareness, empowering patients to advocate for themselves and ensuring newly diagnosed patients feel supported in their journeys. PV&ME features the stories of four inspiring individuals – Buzz, Deb, Patti and Steven – living with this chronic cancer and their perspectives on navigating diagnosis, addressing burdensome symptoms and seeking comprehensive care.

PV is the most common MPN and a long-term, potentially life-threatening cancer that has had limited treatment options for many years. Patients with PV are at a more significant increased risk of developing thromboembolic events than the general population with cardiovascular disease, due to increased blood cell counts. They also have a long-term risk of progression to myelofibrosis or transformation to acute myeloid leukemia.1-5

“People living with PV often face feelings of isolation as they navigate a long and confusing road to diagnosis and adjust to extreme fatigue or other often debilitating symptoms,” said Ann Brazeau, Chief Executive Officer, MPN Advocacy & Education International. “The stories shared in this new PV&ME campaign show just how important the right support and resources can be for this community. We hope this new initiative will help people with PV feel connected and empowered to advocate for themselves on their PV journeys.”

“At PharmaEssentia, we are committed to being an essential partner for the MPN community and know that a critical component of that is listening to and amplifying stories from individuals living with PV themselves,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at PharmaEssentia. “This MPN Awareness Day, we are proud to partner with MPN Advocacy & Education International to share these inspiring stories with the MPN community and help encourage patients to take a proactive approach in their care.”

The PV&ME educational video series launched on MPN Awareness Day (September 14) and can be found by visiting us.pharmaessentia.com/patients/patient-stories/. Throughout Blood Cancer Awareness Month, PharmaEssentia and MPN Advocacy & Education International will continue to share important educational content for the MPN community.

Follow PharmaEssentia USA on Twitter and LinkedIn for news and updates.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.6

About PharmaEssentia

PharmaEssentia (TPEx: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan.

For more information about PharmaEssentia USA, visit the website, LinkedIn or Twitter.

About MPN Advocacy & Education International

MPN Advocacy and Education International provides educational programs, materials, and resources for patients, caregivers, physicians, and entire healthcare teams to improve their understanding of myelofibrosis, polycythemia vera, and essential thrombocythemia. They are dedicated to making a difference in the lives of those affected by MPNs and strive to grow awareness and advocate on behalf of the MPN community.

For more information about MPN Advocacy and Education International, visit the website, Facebook or Twitter.

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1 Griesshammer M, Kiladjian J-J, Besses C. Thromboembolic events in polycythemia vera. Ann Hematol. 2019;98:1071–82. DOI: 10.1007/s00277-019-03625-x
2 Antithrombotic Trialists (ATT) Collaboration. Aspirin in the primary and secondary prevention of vascular disease: collaborative meta-analysis of individual participant data from randomised trials. Lancet. 2009;373:1849-1860. https://doi.org/10.1016/S0140-6736(09)60503-1
3 Yusef S, et al. Cholesterol lowering in intermediate-risk persons without cardiovascular disease. NEJM. 2016;374(21):2021-2031. DOI: 10.1056/NEJMoa1600176
4 Risk and Prevention Study Collaborative Group;​ Roncaglioni M, et al. N-3 fatty acids in patients with multiple cardiovascular risk factors. NEJM. 2013;368:1800-1808. DOI: 10.1056/NEJMoa1205409
5 Barbui T, et al. In contemporary patients with polycythemia vera, rates of thrombosis and risk factors delineate a new clinical epidemiology. Blood. 2014;124:3021-3023. https://doi.org/10.1182/blood-2014-07-591610
6 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J. 2015;5, e366; DOI:10.1038/bcj.2015.95

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Ezobresib by Bristol-Myers Squibb for Myelofibrosis: Likelihood of Approval

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September 14, 2023

Ezobresib is under clinical development by Bristol-Myers Squibb and currently in Phase II for Myelofibrosis. According to GlobalData, Phase II drugs for Myelofibrosis have a 40% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Ezobresib’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. 

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Ezobresib overview

Ezobresib (BMS-986158) is under development for the treatment of solid tumors including triple-negative breast cancer, small-cell lung cancer, epithelial ovarian cancer, peritoneal cancer, renal cell carcinoma, fallopian tube cancer, Burkitt’s lymphoma/leukemia, Uveal melanoma, Uterine carcinosarcoma, NUT-midline carcinoma, Non-small cell lung cancer, metastatic hormone refractory (castration resistant, androgen-Independent) prostate cancer, blood cancer (hematologic malignancies), primary myelofibrosis (PMF), post-essential thrombocythemia (ET) or post-polycythemia vera (PV) myelofibrosis. It is administered orally as a capsule. The drug candidate acts by targeting bromodomain and extra-terminal (BET) proteins. It was under development for Ewing sarcoma.

Bristol-Myers Squibb overview

Bristol-Myers Squibb (BMS) is a specialty biopharmaceutical company that is engaged in discovery, development, licensing and manufacturing, marketing, distribution and sale of medicines and related medical products to patients with serious diseases. Its primary focus is on cancer, cardiovascular, immunology and fibrotic therapeutic projects. The company offers its products across the world to wholesalers, retail pharmacies, medical professionals, hospitals and government entities. BMS provides its products in the US, Europe, and Japan. The company conducts research to focus on the discovery and development of novel medicines that address serious diseases in areas of significant unmet medical need. BMS is headquartered in New York City, New York, the US.

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