Cancer Vaccine Breaking News

Cancer vaccine breaking news brought to you by Vax Before Cancer.

Jun 23, 2022 • 1:13 pm CDT
U.S. FDA

Massachusetts-based VBI Vaccines Inc. announced yesterday that the U.S. FDA granted Orphan Drug Designation for VBI-1901, a bivalent vaccine candidate for the treatment of glioblastoma (GBM).

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens gB and pp65. 

“This orphan drug designation is another significant milestone for our VBI-1901 program, and it underscores the urgency of our effort to develop meaningful new treatment options for patients with this devastating cancer,” said Jeff Baxter, President, and CEO of VBI, in a press release issued on June 22, 2022.

“With this orphan drug status, we look forward to working closely with the FDA and clinical investigators to build on that data, advancing the potential of this program to be a valuable part of the fight against GBM.”

GBM is among the most common and aggressive malignant primary brain tumors in humans.

In the U.S. alone, 14,000 new cases are diagnosed each year.

The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy.

Unfortunately, even with aggressive treatment, GBM progresses rapidly and has a high mortality.

In June 2021, the FDA also granted Fast Track Designation for VBI-1901 to treat recurrent GBM in patients with first tumor recurrence.

Note: This press release was manually curated for mobile readers.

May 17, 2022 • 9:17 am CDT
CDC

The U.S. CDC recently published updated COVID-19 testing suggestions when traveling and confirmed an at-home test solution available for free. As of May 16, 2022, the CDC's website encourages anyone planning to travel to get tested for the SARS-CoV-2 coronavirus within three days of departure.

And to get tested after travel if your trip involved visiting situations with an increased risk of virus exposure, such as a crowded, indoor place.

Furthermore, suppose you are returning to the U.S. from overseas.

In that case, air passengers (2+ yrs.) must show a negative test result taken no more than one day before departure or Documentation of Recovery from COVID-19 in the past 90 days.

To help travelers access these tests, the CDC confirmed every home in the U.S. is now eligible to order the 3rd round of free at-home tests.

These self-tests are rapid antigen at-home tests, not PCR, can be taken anywhere, and most importantly, deliver results within 30 minutes (no lab drop-off required).

Each order now includes eight rapid antigen COVID-19 tests, which arrive in separate packages (4 tests in each box), and are shipped for free!

Fill in this form with your contact and shipping information, or call 1-800-232-0233 to order your tests.

At-home tests are also available for sale. Check with local pharmacies to see where they are available.

Whether you test positive or negative for COVID-19, you should take preventive measures to protect yourself, and others, says the CDC.

As of May 13, 2022, the CDC has confirmed over 847 million COVID-19 test results.

Note: The CDC information was manually curated for mobile readership.

Mar 8, 2022 • 7:15 am CST
Image by Claudio Scott

The Annals of Internal Medicine published Original Research on March 1, 2022, 'Estimation of Breast Cancer Overdiagnosis in a U.S. Breast Screening Cohort,' funded by the National Cancer Institute.

Mammography screening can lead to overdiagnosis—that is, screen-detected breast cancer that would not have caused symptoms or signs in the remaining lifetime. 

Based on an authoritative U.S. population data set, the analysis projected that among biennially screened women aged 50 to 74 years, about 1 in 7 (14%) cases of screen-detected cancer is overdiagnosed.

The study cohort included 35,986 women, 82,677 mammograms, and 718 breast cancer diagnoses. 

'This information clarifies the risk for breast cancer overdiagnosis in contemporary screening practice and should facilitate shared and informed decision making about mammography screening,' concluded these researchers.

Feb 21, 2022 • 4:33 pm CST

Positive topline results from the pivotal DESTINY-Breast04 phase 3 clinical trial announced today showed ENHERTU® (trastuzumab deruxtecan) demonstrated a statistically significant and clinically meaningful improvement in both progression-free survival (PFS) and overall survival (OS) in patients with HER2 low unresectable and/or metastatic breast cancer regardless of hormone receptor (HR) status versus physician's choice of chemotherapy, which is the current standard of care.

ENHERTU is a HER2-directed antibody-drug conjugate (ADC) jointly developed by Daiichi Sankyo and New Jersey-based AstraZeneca.

All patients in the trial received a HER2 test, and the results were centrally confirmed.

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors and is one of many biomarkers expressed in breast cancer tumors.

HER2 low status was defined as an immunohistochemistry (IHC) score of 1+ or IHC2+ with a negative in-situ hybridization (ISH) score.

HER2 expression is currently defined as either positive or negative.1 HER2 positive cancers are defined as IHC 3+ or IHC 2+/ISH+, and HER2 negative cancers are defined as IHC 0, IHC 1+, or IHC 2+/ISH-. 

Up to 55% of all patients with breast cancer have tumors with a HER2 IHC score of 1+ or a HER2 IHC score of 2+ in combination with a negative ISH test, an expression level not currently eligible for HER2 targeted therapy. 

Low HER2 expression occurs in both hormone receptor (HR) positive and HR negative disease.

HER2 testing is well-established to determine an appropriate treatment strategy in metastatic breast cancer.

Targeting the lower range of HER2 expression may offer another approach to delay disease progression and extend survival in patients with metastatic breast cancer. 

Currently, chemotherapy remains the only treatment option for patients with HR-positive tumors following progression on endocrine (hormone) therapy and for those who are HR negative.

"Today's historic news from DESTINY-Breast04 could reshape how breast cancer is classified and treated," said Susan Galbraith, MBBChir, Ph.D., Executive Vice President, Oncology R&D, AstraZeneca, in a press release issued on February 21, 2022.

"A HER2 directed therapy has never before shown a benefit in patients with HER2 low metastatic breast cancer."

"These results for ENHERTU are a huge step forward and could potentially expand our ability to target the full spectrum of HER2 expression, validating the need to change the way we categorize and treat breast cancer."

The companies stated the data will be presented at an upcoming medical meeting and shared with global health authorities.

Daiichi Sankyo is responsible for the manufacturing and supplying ENHERTU and datopotamab deruxtecan.

Feb 18, 2022 • 1:47 pm CST

Cambridge-based Moderna, Inc. today announced that it is expanding its mRNA vaccine pipeline. One of the new vaccines is a checkpoint cancer vaccine candidate, mRNA-4359.

Moderna's mRNA-4359 expresses Indoleamine 2,3-dioxygenase and programmed death-ligand 1 antigens.

Moderna designed mRNA-4359 to stimulate effector T-cells that target and kill suppressive immune and tumor cells that express target antigens. Moderna plans to explore initial indications for advanced or metastatic cutaneous melanoma and non-small cell lung carcinoma (NSCLC).

NSCLC frequently goes undetected, remaining asymptomatic until it has progressed to later stages.

Approximately 115,000 people are diagnosed with metastatic NSCLC or progress to the metastatic disease annually, according to the U.S. CDC.

"We are pleased to announce these new development programs, which reflect the continued productivity of our platform and the potential of our mRNA technology to impact the lives of hundreds of millions of people," said Stéphane Bancel, Chief Executive Officer of Moderna, in a media statement issued on Feb. 18, 2022.

In 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA) to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production. 

Feb 6, 2022 • 4:55 pm CST

An innovative clinical research team at The Clatterbridge Cancer Centre in the UK announced on February 4, 2022, they have given a cancer patient an injection of a therapy tailor-made to his personal DNA and designed to help his immune system ward off cancer permanently.

He will have a year-long course of immunotherapy injections in a bid to keep him cancer-free, part of a research project designed to reduce deaths and recurrence in head and neck cancers, including of the throat, neck, mouth, and tongue.

In a news article, the West Kirkby resident said that he was not worried about being the first person in the UK to receive this pioneering treatment and that it "opened new doorways," which gave him hope that cancer would not come back.

"When I had my first cancer treatment in 2011, I was under the impression that cancer would not return."

"My biggest fear was realized in 2016 when it came back and then in 2019 and then two cases in 2021."

Chief Investigator for the UK trial, Professor Christian Ottensmeier, a Consultant Medical Oncologist at The Clatterbridge Cancer Centre and Professor of Immuno-Oncology at the University of Liverpool, commented, "It is a really exciting day in this important and potentially game-changing research."

"To have reached the stage of a patient receiving this treatment that only a few years ago was thought of as science fiction is truly amazing."

Prof Ottensmeier said more patients were joining the trial at Clatterbridge, and they would be offered individualized treatments, which could benefit them greatly, as well as expand the team's knowledge of personalized cancer therapies that could eventually be help people with other cancer types.

The Transgene study, undertaken at Clatterbridge Centre, Liverpool, also involves cancer researchers from Liverpool Head & Neck Centre, Liverpool University Hospitals, and the University of Liverpool.

Feb 5, 2022 • 2:08 pm CST

The peer-reviewed journal Nature Communications published a new article on February 1, 2022, demonstrating that a DNA methylation signature in easy-to-access Müllerian Duct-derived cervical cells from women with and without ovarian cancer could identify women with ovarian cancer in the absence of tumor DNA.

The WID-OC test identifies 71.4% and 54.5% of <50 and ≥ 50-year-old ovarian cancer patients with a specificity of 75%.

This and the observation that the cervical cell WID-OC-index mimics the epigenetic program of those cells at risk of becoming cancerous in BRCA1/2 germline mutation carriers (i.e., mammary epithelium, fallopian tube fimbriae, prostate) further suggest that the epigenetic misprogramming of cervical cells is an indicator for cancer predisposition.

'This concept has the potential to advance the field of risk-stratified cancer screening and prevention,' stated these researchers.

This finding is essential since epithelial ovarian cancer is by far the most common cause of gynecological cancer-associated death, says the U.S. CDC. 

Currently, 75% of ovarian cancers are picked up at an advanced stage where the tumor has spread within the entire abdominal cavity and beyond.

Feb 4, 2022 • 10:45 am CST

People who have been treated with the cancer drug rituximab or similar drugs respond poorly or not at all to subsequent COVID-19 mRNA vaccines, according to a study published by researchers at Stanford Medicine on January 31, 2022.

Rituximab, marketed under the brand name Rituxan, is widely used alone or in combination with other treatments in people with lymphomas, a type of blood cancer.

It targets a molecule called CD-20 found on the surface of immune cells called B cells.

In 2021, around 90,000 people were diagnosed with the disease in the United States.

However, the researchers did not directly assess whether patients treated with rituximab or with other drugs targeting CD-20 before being vaccinated subsequently had higher infection rates with the SARS-CoV-2 coronavirus that causes COVID-19.

But the findings strongly suggest that people who are newly diagnosed with lymphoma should be offered an mRNA vaccine before beginning rituximab or similar drugs.

“This finding is likely to be practice-changing,” said Ronald Levy, MD, professor of oncology, in a press statement issued on February 2, 2022.

“We found that antibody responses to the COVID-19 vaccine were blunted in people who received rituximab up to a year before vaccination. But if they were vaccinated before treatment, most responded and were able to hold on to that response during their rituximab treatment.”

Note: On December 2, 2021, the U.S. FDA Approved rituximab (Rituxan, Genentech, Inc.) in combination with chemotherapy for pediatric patients (≥6 months to <18 years) with previously untreated, advanced stage, CD20-positive diffuse large B-cell lymphoma, Burkitt lymphoma, Burkitt-like lymphoma, or mature B-cell acute leukemia. 

Feb 3, 2022 • 11:11 am CST

Norway-based Ultimovacs ASA today announced it had completed treatment of the second dose cohort in a Phase I clinical study (TENDU) designed to evaluate the Tetanus-Epitope Targeting (TET)-platform in patients with prostate cancer.

A total of six patients have been treated, three in each dose level (40 and 400 μg).

The Drug Safety Monitoring Board (DSMB) found no safety concerns related to the first two dose cohorts.

The conclusion from the DSMB enables the dose-escalation study to proceed with the enrollment of patients in the third and last dose cohort (960 μg).

"The continued progress of the Phase 1 safety evaluation of the TET platform is very encouraging," said Jens Bjørheim, Chief Medical Officer of Ultimovacs, in a press release issued on February 3, 2022.

"Our main focus at this stage in the TENDU study is the safety and tolerability of our new therapeutic vaccine candidate for prostate cancer, a cancer type where the unmet medical need is high."

"This result also feeds into Ultimovacs' plans for the development of the TET platform more broadly as an extension of the company's pipeline."

The TET platform, an innovative adjuvant technology, allows for designing and producing multiple therapeutic cancer vaccines.

It can potentially be used to strengthen and increase T cell responses to cancer cells by targeting antigens specific to one type of cancer or common to many tumor types.

The vaccine used in the TENDU study contains prostate cancer-specific antigens.

By combining cancer antigens and the vaccine adjuvant in the same molecule, the TET platform can generate vaccine candidates with a potential beneficial safety and administration profile, including presenting an opportunity to treat patients at an early stage of their disease.

The TENDU study is a first-in-human, dose-escalation study designed to generate initial safety and immune activation data. In addition, this study investigates a prostate cancer-specific therapeutic TET-based vaccine in patients who have relapsed following radical prostatectomy.

Ultimovacs is developing immune-stimulatory vaccines to treat a broad range of cancers. 

Feb 2, 2022 • 4:02 pm CST

Recent data in the UK has shown COVID-19 vaccine booster take-up among certain British people is almost double that of some ethnic minorities.

An analysis of NHS England data, obtained by Blood Cancer UK after a Freedom of Information request, shows 84% of immunocompromised people from a white British background had three vaccine doses by mid-December 2021.

This finding compared to third doses among other ethnic minorities include:

  • 46% for people from an African background;
  • 47% for people from a Caribbean background; and
  • 49% for people from a Bangladeshi background.

And the 539,000 immunocompromised compromised people in England (230,000 with blood cancer) are at higher risk from severe COVID-19 and are less likely to respond well to the vaccines.

While some of the differences in third dose uptake may be because of varying levels of vaccine hesitancy, this does not explain why the gap for third doses is much larger than the gap for the first two doses.

For example, there was a 14-percentage point difference in take-up of the first two doses between White British immunocompromised people (95%) and those from a Pakistani background (81%).

Still, the gap between the two groups' uptake of the third dose was 41% points.

Gemma Peters, Chief Executive of Blood Cancer UK, commented in a press release issued on January 31, 2022, "We know that people from ethnic minorities have been disproportionately affected by the pandemic, and it is alarming."

"Much of the public debate around lower vaccination rates in some communities has centered around vaccine hesitancy."

Note: The data in this press release are until December 14, 2021, and we expect the racial disparity to have narrowed since then as more people have been given third doses. But this would still mean that people from these groups were disproportionately at risk from Covid between October and December, a period when the infection rate was high.

Feb 2, 2022 • 8:33 am CST

California-based Mosaic ImmunoEngineering, Inc. today announced a new article published in the journal Molecular Pharmaceuticals that details broad immune activation of inactivated CPMV (termed inCPMV) and potent, systemic, and durable antitumor activity with the treatment combination in an aggressive two-tumor model of melanoma.

Co-authors of the new article, "Inactivated Cowpea Mosaic Virus in Combination with OX40 Agonist Primes Potent Antitumor Immunity in a Bilateral Melanoma Mouse Model," include Mosaic co-founders Nicole F. Steinmetz, Ph.D., director of the UC San Diego Center for Nano-ImmunoEngineering, and Steven N. Fiering, Ph.D., professor of microbiology and immunology at the Geisel School of Medicine at Dartmouth University.

The article highlights include the following:

  • Broad, durable, and systemic antitumor immunity observed using inCPMV with OX40 agonist antibodies,
  • Intratumoral inCPMV administration in combination with systemic anti-OX40 controlled the progression of the primary as well as untreated secondary tumors, and
  • 70% of animals survived for at least 100 days post tumor challenge without the development of recurrence or metastatic disease.

"Our studies demonstrate that intratumoral administration of inCPMV and systemic administration of an OX40 agonist antibody generates potenantitumorc antitumor immunity," said Dr. Steinmetz in a press release issued on February 2, 2022.

"We observed that synergistic efficacy can be achieved through a combination of inCPMV with an OX40 agonist."

"These data further support the rationale for combining our lead immuno-oncology product candidate, MIE-101, with antibodies targeting OX40, which are currently showing promise in clinical development for the treatment of cancer."

"The potential of MIE-101 to turn immunologically cold tumors hot could allow checkpoint treatment approaches such as OX40 agonists to be effective in more patients who do not currently respond," added Steven King, president, and CEO of Mosaic.

"Checkpoint targeted treatments have shown promising and durable results in melanoma patients, yet there remains a significant unmet medical need because only a minority of patients receive the full potential clinical benefit to currently approved therapies. These results published by Mosaic's co-founders reinforce our goal to continue identifying strategic partners to work with us as we advance MIE-101."

Mosaic ImmunoEngineering, Inc. is a development-stage biotechnology company located in Novato, CA, focused on bridging immunology and engineering to develop novel immunotherapies to treat and prevent cancer and infectious diseases. 

Feb 1, 2022 • 12:20 am CST

The Netherlands-based Public health institute RIVM confirmed on January 31, 2022, children ten years of age and older would be invited to get vaccinated against the Human Papillomavirus (HPV).

"Vaccinating both boys and girls contribute to the creation of herd immunity," said Jeanne-Marie Hament, RIVM program manager of the National Immunization Program, in a news article.

The RIVM previously offered the HPV vaccine to teenage girls and older to protect them against vagina-, labia-, and cervix cancer.

But new research shows that HPV can also cause cancer in boys and men.

"After advice from the Health Council, we are now starting to offer the HPV vaccination to boys. In this way, they are also protected against HPV cancer," added Hament.

Annually, almost 400 men in the Netherlands get cancer from HPV.

However, about 80% of these cancer cases can be prevented with the HPV vaccination, according to the RIVM.

The Netherlands' National Vaccination Immunisation Programme uses a vaccine called Cervarix®.

Jan 30, 2022 • 2:10 pm CST

Denmark-based researchers announced on January 26, 2022, that chemists are copying cancer cell carbohydrates in the laboratory for inclusion in a vaccine that activates the immune system.

The surface of all cells is covered with different carbohydrates.

On cancer cells, however, the carbohydrates often differ from the carbohydrates located on the surface of healthy cells.

In a press release, Professor Mads Hartvig Clausen and Postdoc Cecilia Romanò from Technical University of Denmark (DTU) Chemistry stated they are trying to 'exploit this difference to develop a cancer vaccine.'

"With such a vaccine, it will be possible to teach our immune system to recognize the difference between a healthy cell and a cancer cell so that the cancer cells are attacked and destroyed by the immune cells," says Mads Hartvig Clausen.

The vaccine is based on imitations of the surface carbohydrates of cancer cells—which are called tumor-associated carbohydrate antigens (TACA)—and which DTU chemists can produce artificially in the laboratory using chemical synthesis.

"The carbohydrates are only found in small quantities on the surface of cancer cells, but we can produce large quantities in the laboratory."

"When the vaccine is injected under the skin, the immune system will be presented with our copies of the TACAs."

"They are to act as antigens, which means that they must activate the immune system to produce antibodies."

"The idea is that if a person's immune system subsequently encounters these carbohydrates because he or she has developed cancer, it will be able to recognize them and consequently combat the cancer cells," says Mads Hartvig Clausen.

This concept sounds surprisingly simple.

Yet the chemists had already faced several challenges that needed to be addressed before they were ready with an initial cancer vaccine candidate. And the chemists were aware that they required close collaboration with other disciplines at DTU if they were to succeed with their ambition.

"When we started, we knew the strong competencies that exist at DTU in immunology and drug delivery."

"We're experts in chemical synthesis, but we need to collaborate on the other aspects if we're to succeed in developing the vaccine, and we, therefore, got together with colleagues from DTU Health Tech and DTU Bioengineering," explains Mads Hartvig Clausen.

The first challenge for Mads Hartvig Clausen and Cecilia Romanò was to select the types of TACAs to be produced in the laboratory.

Because—with cancer—there is not just one kind of carbohydrate on the surface of all cancer cells. The TACAs vary from cancer type to cancer type, just as each cancer type has several kinds of TACAs on the surface of the cells.

At DTU, the choice fell on a group of carbohydrates associated with malignant melanoma cancer, neuroblastoma (a type of tumor in the body of children), and small cell lung cancer.

"With such a vaccine, it will be possible to teach our immune system to recognize the difference between a healthy cell and a cancer cell, so that the cancer cells are attacked and destroyed by the immune cells."

"These are primarily three cancers that have the same type of carbohydrate on their cell surface, namely the carbohydrates known as gangliosides. Therefore, they can be produced in the laboratory from simple sugars that we can buy for this purpose," says Cecilia Romanò.

The next challenge was to ensure that the carbohydrate activates an immune response in the body when injected.

According to the two chemists, there is an obstruction, as the body is 'a friend of carbohydrates'—meaning that carbohydrates are generally not very strong antigens.

"Carbohydrates generally don't activate a robust response from our immune system."

"Therefore, we had to find a way to help our artificial carbohydrates elicit the desired immune response," says Cecilia Romanò.

Amplifying the effect of a pharmaceutical is a well-known challenge, and the solution may be to use an excipient—an adjuvant.

Mads Hartvig Clausen and Cecilia Romanò chose to use the excipient α-galactosylceramide as an adjuvant.

This substance consists of carbohydrates and fatty acids—a so-called glycolipid—which stimulates the immune system. To boost the immune response further, the chemists compounded the antigen and the adjuvant in the laboratory.

This means that they 'coupled' their selected TACA with α-galactosylceramide, so they became one compound instead of two' loose substances.'

"We've subsequently demonstrated that it gives a stronger immune response when the two substances are joined than if they are given individually," explains Mads Hartvig Clausen.

Assimilation in the body is ensured.

Before the chemists could inject their antigens, they were aware that something was needed to help the substances be assimilated into the immune system.

"We needed a kind of carrier to ensure that the immune system can detect and respond to our antigens. Otherwise, we will not be able to start the cascade of events which makes up an immune response and which results in the actual production of antibodies," explains Mads Hartvig Clausen, who approached his colleagues at DTU Health Tech, Associate Professor Jonas Henriksen and Professor Thomas Andresen.

They have been collaborating for many years. As a result, the chemists were familiar with their colleagues' work with liposomes (a fat globule the size of a nanoparticle) used for drug delivery and vaccine formulation.

Mads Hartvig Clausen and Cecilia Romanò were thus able to have their cancer antigens 'mounted' on a liposome, which was well described and documented assimilation by the immune system.

The chemists could now start testing their vaccine. So far, they have performed one trial with mice.

They were all cancer-free because the researchers were initially interested in finding out whether their vaccine would create the desired immune response at all.

The results were really good, say Mads Hartvig Clausen and Cecilia Romanò.

"We've shown that our vaccine triggered the immune system of the mice into producing antibodies that can recognize cancer cells with our selected carbohydrate on the surface. The antibodies can also communicate that components from the immune system—the so-called complement system—can kill cancer cells in the laboratory," said the two chemists.

The researchers will optimize the vaccine in the next two to three years and further understand the immune response.

The next step will be testing on mice with cancer to see if the antibodies produced by the vaccine will fight the disease.

In parallel, the researchers applied and received funding for starting processes with a consultant that will pave the way for getting the vaccine out of the laboratory and onto a commercial market.

"If society is to benefit from our vaccine one day, we will need a company to take over our invention so that it can be tested in humans and then—hopefully—be put into production," says Mads Hartvig Clausen.

The researchers hope to hand over their cancer vaccine candidate to a pharmaceutical company in about three years.

Note: DTU is an independent and self-governing university with a Board of Governors. Hans Christian Ørsted founded DTU in 1829 with a clear vision to develop and create value using science and engineering to benefit society.

Jan 29, 2022 • 10:22 am CST

In a media release issued on January 18, 2022, Epic Research confirmed that as the COVID-19 pandemic continues, clinicians are concerned cancer screening rates have not returned to pre-pandemic baselines.

'We found that rates of breast and colon cancer screenings remain slightly below historical baselines, down 2.7% and 3.4% respectively.'

And 'cervical cancer screening rates are still 10% below historical baselines.'

'These rates equate to an estimated 68,000 missed breast cancer screenings, 27,000 missed colon cancer screenings, and 9,000 missed cervical cancer screenings from January 2021 through October 2021.'

'Further delays in cancer screening could lead to delayed cancer diagnoses, which could increase morbidity and mortality and exacerbate existing health care disparities, as well as increase health care costs.'

'Ongoing efforts to increase patient access to affordable cancer screenings are important to our nation's COVID recovery.'

These data come from Cosmos, a HIPAA-defined Limited Data Set of more than 126 million patients from 156 Epic organizations, including 889 hospitals and 19,420 clinics, serving patients in all 50 states.

Epic Research is a public benefit corporation owned by Epic Systems Corporation.

Jan 28, 2022 • 1:40 pm CST

California-based Sierra Oncology, Inc. today announced positive topline data from the pivotal Phase 3 MOMENTUM study evaluating momelotinib (MMB) in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor.

The trial met all of its primary and key secondary endpoints. The Topline data announced based on 195 patients (MMB n = 130; DAN n = 65) include:

  • Primary Endpoint of Total Symptom Score (TSS) of >50%: 25% in the MMB arm vs. 9% in the control arm (p=0.0095)
  • Secondary Endpoint of Transfusion Independence (TI): 31% in the MMB arm vs. 20% in the control arm (one-sided p=0.0064; non-inferiority)
  • Secondary Endpoint of Splenic Response Rate (SRR) >35%: 23% in the MMB arm vs. 3% in the control arm (p=0.0006)
  • The rate of Grade 3 or worse adverse events in the randomized treatment period was 54% in the MMB arm and 65% in the control arm. Serious treatment-emergent adverse events were 35% in the MMB arm and 40% in the control arm.
  • Mean baseline characteristics for all patients were TSS of 27, Hemoglobin (Hgb) of 8 g/dL, and platelet count of 145 x 10 9/L

"As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anemic or at risk of becoming anemic," said Ruben Mesa, MD, FACP, Executive Director of the Mays Cancer Center, home to UT Health San Antonio, MD Anderson Cancer Center, and co-Principal Investigator of the study, in a press statement. 

"Anemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival."

"Half of all myelofibrosis patients present with anemia at diagnosis, and virtually all become anemic over time."

"With currently approved therapies being myelosuppressive, it's wonderful to know that we may soon have such an effective treatment option for these patients."

Myelofibrosis is a rare blood cancer that results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen), and progressive anemia.

The full data set will be presented at an upcoming medical meeting, says the Company.

Sierra Oncology is a late-stage biopharmaceutical company located in San Mateo, CA, on a mission to deliver targeted therapies that treat rare forms of cancer.