Cancer Vaccine Breaking News

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

Jun 9, 2021 • 12:44 pm CDT

Idaho-based Therapeutic Solutions International, Inc. reported potent synergy between its tumor blood vessel targeting StemVacs-V iPSC immunotherapy and several classical tumor-specific therapeutic vaccines. 

In a series of experiments, tumor growth administration of dead tumor cells together with StemVacs-V resulted in potent immunological memory to the tumor cells, which could be transferred to immunologically naïve mice. 

Additionally, the experiments demonstrated killing tumor cells using conventional approaches such as chemotherapy, when performed together with StemVacs-V iPSC, led to the development of immunological memory towards specific cancer.

"By targeting the blood vessels that feed the cancer, StemVacs-V iPSC causes enhanced necrotic cell death, which stimulates systemic immunity against cancer throughout the whole body," said Dr. James Veltmeyer, Chief Medical Officer of the Company, in a press release issued on June 7, 2021.

"We are extremely enthusiastic by the current data showing that our blood vessel targeting approach can be used to synergize with both cancer immunotherapies and non-immune therapies of cancer such as chemotherapy and radiation therapy."

The company stated It is believed that by starving the cancer of its blood supply, StemVacs-V iPSC may convert cold tumors to immunotherapy sensitive "hot tumors."

"This new finding is extremely exciting because it opens the door to synergies with other immunotherapies such as CAR-T therapies, which although successful in liquid tumors, to date have not yielded promising results in solid and/or cold tumors," commented Timothy Dixon, President, and CEO of the Company. 

Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases.

Jun 1, 2021 • 7:26 pm CDT

New Jersey-based Janssen Pharmaceutical Companies announced today that the U.S. Food and Drug Administration (FDA) had granted Breakthrough Therapy Designation (BTD) for teclistamab to treat relapsed or refractory multiple myeloma.

This distinction for teclistamab, an off-the-shelf, T-cell redirecting, a bispecific antibody targeting both B-cell maturation antigen (BCMA) and CD3 receptors, follows a PRIME designation from the European Medicines Agency received earlier in 2021.

“We are pleased to have received Breakthrough Therapy and PRIME Designations for our novel bispecific antibody, teclistamab,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC., in a press release.

Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, says Cancer.net. When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2021, it is estimated that nearly 35,000 people will be diagnosed, and more than 12,000 will die from the disease in the USA.

The FDA grants BTD to expedite the development and regulatory review of an investigational medicine intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

The Breakthrough and PRIME designations are supported by data from the Phase 1 MajesTEC-1 study, an open-label, multicenter clinical trial evaluating the safety and efficacy of teclistamab in adults with measurable multiple myeloma that is relapsed or refractory to established therapies or be intolerant of those established multiple myeloma therapies.

Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson. Learn more at www.janssen.com.

Jun 1, 2021 • 1:17 pm CDT

Norway-based Ultimovacs ASA announced that a peer-reviewed article on the ongoing NIPU Phase II trial of the Company’s universal cancer vaccine, UV1, in malignant pleural mesothelioma (MPM) was published. Journal of Translational Medicine.

This article outlines the mechanistic rationale for using the combination of UV1 with two checkpoint inhibitors, ipilimumab and nivolumab.

The dual-use of ipilimumab and nivolumab was recently approved as first-line therapy in MPM, where few therapeutic options are currently available.

However, as Haakensen et al. explains in the article, observed response rates with checkpoint inhibitors have been moderate in MPM compared to documented performance for the combination of checkpoint inhibitors in other cancers, suggesting that checkpoint inhibitors alone may be insufficient to trigger an immune response.

Earlier phase I/II studies have shown that UV1 is safe on its own and when used in combination with checkpoint inhibitors and that it induces vaccine-specific immune response associated with survival.

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. UV1 is being developed as an “off-the-shelf” therapeutic cancer vaccine which may serve as a platform for use in combination with other immunotherapy, which requires an ongoing T cell response for their mode of action.

To date, UV1 has been tested in four phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

“The NIPU trial is important in understanding the potentially synergistic activities of checkpoint inhibitors and our universal cancer vaccine, UV1,” stated Jens Bjørheim, Chief Medical Officer at Ultimovacs, in a press release. 

“Malignant pleural mesothelioma is a challenging disease to treat even with checkpoint inhibitors that have been effective in other types of cancer. The article published today explains how we think UV1 may help in meeting that challenge.”

NIPU is a randomized, multi-center, open-label, proof of concept study comparing the efficacy and safety of nivolumab and ipilimumab with or without UV1 in patients with inoperable malignant pleural mesothelioma after first-line platinum-based chemotherapy.

Article details:  Haakensen, V.D., Nowak, A.K., Ellingsen, E.B. et al. NIPU: a randomized, open-label, phase II study evaluating nivolumab and ipilimumab combined with UV1 vaccination as second-line treatment in patients with malignant mesothelioma. J Transl Med 19, 232 (2021).

About Ultimovacs: Ultimovacs seeks to become a leader in developing immune-stimulatory vaccines to treat a broad range of cancers. For further information, please see www.ultimovacs.com.

May 27, 2021 • 5:17 pm CDT

Sweden-based BioInvent International AB and Transgene announced that their Investigational New Drug application for BT-001 had been granted by the U.S. Food and Drug Administration. BT-001 is being co-developed through a 50/50 collaboration between BioInvent and Transgene.

This news enables patients in the USA to enroll in the ongoing Phase 1/2a clinical trial of this novel oncolytic virus BT-001.

The ongoing Phase 1/2a study is a multicenter, open-label, dose-escalation trial evaluating BT-001 as a single agent and in combination with pembrolizumab (anti-PD-1 treatment). The Phase 1 part of the trial has already been initiated in Europe, where it is enrolling patients in several countries.

BT-001 is expected to elicit a strong and effective antitumoral response by selectively targeting and modulating the tumor microenvironment. In addition, delivering the anti-CTLA4 antibody directly to the tumor aims to induce local Treg depletion and strong therapeutic activity.

“We are pleased to receive IND approval for this Phase 1/2a clinical trial of BT-001, which is BioInvent’s fourth clinical program. This unique oncolytic virus has very exciting potential as it combines multiple mechanisms of action and anti-cancer properties, and we are looking forward to developing it further with our partners at Transgene,” said Martin Welschof, CEO of BioInvent, in a press release.

BT-001 is a novel oncolytic virus developed with Transgene’s Invir.IO™ platform. Invir.IO™’s viruses are based on the patented large capacity Vaccinia virus Copenhagen strain genetically modified with the double deletion TK-RR-. This optimization enhances the safety profile of the virus.

BT-001 is engineered to encode both a highly differentiated Treg depleting anti-CTLA4 antibody and the human GM-CSF cytokine.

The recombinant antibody recognizing human CTLA4 was generated by BioInvent’s proprietary n-CoDeR®/F.I.R.S.T™ platforms. The use of an oncolytic virus to deliver the anti-CTLA4 locally and selectively in the tumor microenvironment allows high intratumoral concentrations of both transgenes, eliciting a stronger and more effective antitumor response by reducing systemic exposure to a very low level.

BioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently three drug candidates in four ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors, respectively. 

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May 27, 2021 • 9:07 am CDT

Prostate-specific antigen (PSA) screening was associated with better oncologic outcomes in African American patients with prostate cancer, according to data presented by Edmund M. Qiao, BS, of the University of California San Diego, reported Audrey Sternberg with The Cancer Network on May 20, 2021.

An increase in the frequency of PSA screening was linked to a nearly 25% reduction in prostate cancer-specific mortality and an approximately 40% lower risk of having the metastatic disease at the time of a prostate cancer diagnosis.

The “high PSA screening” group had received an average of 3 prior screening tests, and the “low PSA screening” group had received an average of 0.5 prior screening tests.

Overall, the study included 4,726 African American men diagnosed with prostate cancer. The mean patient age was 51.8 years. The mean number of previous PSA screening tests was 1.9.

The US Preventive Services Task Force PSA screening policy is a grade C recommendation for men aged 55 to 69 years, meaning in this population, an individual decision on screening should be made based on a physician-clinician discussion of the potential benefits and risks.

May 25, 2021 • 7:19 am CDT

Zydus Cadila announced on May 24, 2021, it launched Trastuzumab Emtansine, the first Antibody Drug Conjugate (ADC) biosimilar and a highly effective drug for treating both Early and Advanced HER2 positive Breast Cancer, under the brand name ‘Ujvira’.

HER2-positive Breast Cancer is considered an aggressive form and constitutes 20 to 25% of all breast cancer cases.

In a step that can significantly reduce treatment cost by almost 80%, the drug is being offered at Rs. 32495 for a 100 mg vial. The current MRP of the existing Trastuzumab Emtansine drug is Rs. 1,59,225 for a 100 mg vial.

Ujvira will be available in two strengths, 100 mg, and 160 mg.

Speaking on this milestone, Dr. Sharvil Patel, Managing Director, Cadila Healthcare Limited stated in a press release, “The launch of Ujvira reinforces the innovation capabilities that India has to be able to create complex therapies like ADCs and Zydus' ongoing commitment to offer breakthroughs backed by science and innovation."

"This research breakthrough enables access to a critical drug for patients who are undergoing therapy for breast cancer. We hope that with this innovation, patients will be able to adhere to the treatment and stand to benefit from the advanced technology without worrying about the cost of the treatment.”

Trastuzumab Emtansine ADC biosimilar is a developmental breakthrough due to its complexity in manufacturing and similarity assays. This drug is made by combining Trastuzumab and the cytotoxic compound Emtansine with the help of a stable linker by a process called Antibody Drug Conjugation.

Due to this technology, the targeted delivery of the cytotoxic agent is enabled and the other toxicities on the body are reduced.

Patients already treated with Trastuzumab may still have the disease and would require this therapy as the next step. The high cost of therapy is a barrier to availing this therapy and Ujvira bridges this need, said the company.

Based in India, Zydus Cadila is an innovation-driven, global pharmaceutical company that discovers, develops, manufactures, and markets a broad range of healthcare therapies, including small molecule drugs, biologic therapeutics, and vaccines. The company employs nearly 25,000 people worldwide, including 1,400 scientists engaged in R & D.

May 24, 2021 • 5:37 pm CDT

Cancer immunotherapy has shown great potential in cancer therapy as, unlike traditional chemotherapy and radiation therapy, it aims to evoke the immune response instead of killing cells indiscriminately.

Cancer vaccines consisting of tumor-associated antigens (TAAs) can initiate a powerful anti-tumor immune response through antigen-presenting cells, such as dendritic cells (DCs) and macrophages, stated researchers in a new study published in the July 2021 edition of ScienceDirect.

And they have shown great potential in cancer prevention and therapy.

However, poor anticancer efficacy and an uncertain immunization process have previously limited the application of cancer vaccines.

A new multifunctional nano-vaccine comprising ovalbumin (OVA), MnO2, and polydopamine (OMPN) was prepared by a facile one-pot method.

The OMPN displayed excellent anticancer efficacy against an orthotopic melanoma and could also prevent liver metastasis in a tumor re-challenge mice model.

Additionally, the migration behavior of DCs in the inguinal lymph node after vaccination was tracked by MRI contrasted with OMPN, indicating successful DC activation and immune response.

The superior anticancer efficacy, especially the high efficiency against tumor metastasis, and the capability of tracking the immunization process make OMPN a very promising multifunctional nano-vaccine for cancer therapy, concluded these researchers.

The study's authors declared that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

This work was financially supported by the National Natural Science Foundation of China, the Zhejiang Provincial Natural Science Foundation of China, the Zhejiang University Education Foundation Global Partnership Fund. The China Postdoctoral Science Foundation, the National Postdoctoral Program for Innovative Talent, the Zhejiang Provincial Natural Science Foundation of China and the Zhejiang University.

May 24, 2021 • 4:30 pm CDT

A large-scale randomized clinical trial of annual screening for ovarian cancer did not succeed in reducing deaths from the disease, despite one of the screening methods tested detecting cancers earlier.

The UK Collaborative Trial of Ovarian Cancer Screening (UKCTOCS) was published in The Lancet on May 12, 2021.

The UKCTOCS was designed to test the hypothesis that a reliable screening method that picks up ovarian cancer earlier, when treatments are more likely to be effective, could save lives.

The latest analysis looked at data from more than 200,000 women aged 50-74 at recruitment who were followed up for an average of 16 years.

The researchers found that, while the approach using multimodal testing succeeded in picking up cancers at an early stage, neither screening method led to a reduction in deaths.

Earlier detection in UKCTOCS did not translate into saving lives. Researchers said this highlighted the importance of requiring evidence that any potential screening test for ovarian cancer actually reduced deaths and detected cancers earlier.

Professor Usha Menon, the lead investigator of UKTOCS, said in a press statement, “UKCTOCS is the first trial to show that screening can definitely detect ovarian cancer earlier. However, this very large, rigorous trial shows clearly that screening using either of the approaches we tested did not save lives."

"We, therefore, cannot recommend ovarian cancer screening for the general population using these methods."

“We are disappointed as this is not the outcome we and everyone involved in the trial had hoped and worked for over so many years. To save lives, we will require a better screening test that detects ovarian cancer earlier and in more women than the multimodal screening strategy we used.”

May 21, 2021 • 3:04 pm CDT

Basel-based Roche announced on May 20, 2021, interim results from the Phase III IMpower010 study, showing for the first time that treatment with Tecentriq® (atezolizumab) following surgery and chemotherapy reduced the risk of disease recurrence or death (disease-free survival; DFS) by 34% (hazard ratio [HR]=0.66, 95% CI: 0.50–0.88) in people with Stage II-IIIA non-small cell lung cancer (NSCLC), whose tumors express PD-L1 ≥1%, compared with best supportive care (BSC).

In this population, median DFS was not yet reached for Tecentriq compared with 35.3 months for BSC.

In the larger population of all randomized Stage II-IIIA study participants, Tecentriq reduced the risk of disease recurrence or death by 21% (HR=0.79, 95% CI: 0.64–0.96) after a median follow-up of 32.2 months.

Tecentriq increased DFS by a median of seven months (42.3 months versus 35.3 months with BSC).1 Safety data for Tecentriq were consistent with its known safety profile, and no new safety signals were identified.

Tecentriq is a monoclonal antibody designed to bind with a protein called Programmed Death Ligand-1 (PD-L1), which is expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors.

“These landmark Phase III data demonstrate for the first time that cancer immunotherapy can bring a clinically meaningful improvement to certain people with early lung cancer in the adjuvant setting,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “These results lay the groundwork for a new approach to the treatment of early-stage lung cancer and bring us closer to our goal of providing an effective and tailored treatment option for every person diagnosed with this disease.”

The full results of IMpower010 will be presented in the lung cancer oral abstract session (Abstract #8500) on Sunday 6 June (08:00–11:00 EDT) at the 2021 ASCO Annual Meeting.

May 21, 2021 • 2:52 pm CDT

The U.S. Food and Drug Administration (FDA) announced the approval of Rybrevant (amivantamab-vmjw) as the first treatment for adult patients with non-small cell lung cancer whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

The FDA's approval of Rybrevant was granted to Pennsylvania-based Janssen Pharmaceutical Companies of Johnson & Johnson.

“The approval of RYBREVANT, along with the companion diagnostic test, addresses a high unmet need in the treatment of people with genetically defined non-small cell lung cancer,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC.

The FDA also approved the Guardant360 CDx (Guardant Health Inc.) as a companion diagnostic for Rybrevant.

“Advances in precision oncology continue to facilitate drug development, allowing diseases like lung cancer to be subset into biomarker-defined populations appropriate for targeted therapies,” commented Julia Beaver, M.D., chief of medical oncology in the FDA’s Oncology Center of Excellence and acting deputy director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a press release.

“With today’s approval, for the first time, patients with non-small cell lung cancer with EGFR exon 20 insertion mutations will have a targeted treatment option.”

Researchers evaluated Rybrevant’s efficacy in a study of 81 patients with non-small cell lung cancer and EGFR exon 20 insertion mutations whose disease had progressed on or after platinum-based chemotherapy.

The main outcome measured was the overall response rate (proportion of patients whose tumor is destroyed or reduced by a drug). In the trial population in which all patients received Rybrevant, the overall response rate was 40%. The median duration of response was 11.1 months, with 63% of patients had a duration of 6 months or more.

The most common side effects of Rybrevant include rash, infusion-related reactions, skin infections around the fingernails or toenails, muscle and joint pain, shortness of breath, nausea, fatigue, swelling in the lower legs or hands or face, sores in the mouth, cough, constipation, vomiting, and changes in certain blood tests. Rybrevant should be withheld if patients develop symptoms of interstitial lung disease and permanently discontinued if interstitial lung disease is confirmed.

Patients taking Rybrevant should limit sun exposure during and for two months after treatment. Rybrevant may cause problems with vision. Rybrevant can also cause fetal harm when administered to a pregnant woman; therefore, the pregnancy status of females of reproductive potential should be confirmed before treatment is started.

Rybrevant received Priority Review and Breakthrough Therapy designation for this indication.

According to the American Cancer Society, lung cancer is the most common cancer type and the leading cause of cancer-related deaths worldwide, with non-small cell lung cancer accounting for 80% to 85% of all lung cancers. Approximately 2% to 3% of patients with non-small cell lung cancer will have EGFR exon 20 insertion mutations, a group of mutations on a protein that causes rapid cell growth, and consequently, helps cancer spread. EGFR exon 20 insertion mutations are the third most common type of EGFR mutation.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. 

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May 19, 2021 • 1:28 pm CDT

The US Preventive Services Task Force (USPSTF) announced on May 18, 2021, it has published a final recommendation statement on screening for colorectal cancer. It has concluded with high certainty that screening for colorectal cancer in adults aged 50 to 75 years has a substantial net benefit (B rating).

Overall, people 45 to 75 should be screened to reduce their risk of dying from this disease. For adults 76 to 85, the Task Force recommends that the decision to screen be made individually.

Colorectal cancer is the third leading cause of cancer death for both men and women, with an estimated 52,980 persons in the US projected to die of colorectal cancer in 2021. It is estimated that 10.5% of new colorectal cancer cases occur in persons younger than 50 years.

In 2018, about 31% of eligible people were not up to date with screening.

Recommendations made by the Washington DC-based USPSTF are independent of the U.S. government. They should not be construed as an official position of the Agency for Healthcare Research and Quality or the U.S. Department of Health and Human Services.

May 18, 2021 • 6:51 pm CDT

According to a new study by immunologists from the University of Konstanz, a new cancer vaccine candidate could boost the positive effects of existing immunotherapy drugs, improving the success rate of treatments up to 75% of cases.

The vaccine, which incorporates a new immunostimulant that is safe for use in humans, was shown to eliminate tumors in mice partially.

However, the study further demonstrated that combining the vaccine with an immune checkpoint inhibitor -- an established immunotherapy drug with a 20% success rate overall for patients -- can vastly improve the proportion of individuals who respond to treatments, eliminating tumors in 75% of cases in mice.

The researchers suggest that these promising pre-clinical results should be transitioned into clinical application based on the study's findings. "This might have a very beneficial impact on immunotherapy in certain types of cancer," commented Marcus Groettrup, senior author on the study, in a press statement.

The therapeutic concept developed in this study is currently being tested in a first small phase 1 clinical trial by project partners in the Netherlands to determine if it is similarly effective in humans.

The microparticle-based cancer vaccine, which uses the immunostimulant Riboxxim that has the approval for application in humans, can generate the body's T-cell response necessary for immune checkpoint blockade drugs to be effective.

The results suggest that this new approach of using a vaccine combined with established drugs may be potent anti-cancer immunotherapy to be tested in future clinical trials.

The findings appeared in Nature Communications on May 18, 2021.

May 13, 2021 • 1:36 pm CDT

New Jersey-based Merck announced positive results from the pivotal neoadjuvant/adjuvant Phase 3 KEYNOTE-522 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, combined with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent (adjuvant) treatment after surgery.

Merck confirmed that on May 13, 2021, KEYNOTE-522 met its dual primary endpoint of event-free survival (EFS) to treat patients with high-risk early-stage triple-negative breast cancer (TNBC).

Based on an interim analysis conducted by the independent Data Monitoring Committee (DMC), neoadjuvant KEYTRUDA plus chemotherapy followed by adjuvant KEYTRUDA as monotherapy showed a statistically significant and clinically meaningful improvement in EFS compared with neoadjuvant chemotherapy alone.

As previously communicated, KEYNOTE-522 met its other dual primary endpoint of pathological complete response (pCR). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified.

“KEYTRUDA is the first immunotherapy to show positive results for event-free survival in patients with high-risk early-stage TNBC, a particularly aggressive form of breast cancer,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, in a press statement. 

“The improvement in pathological complete response rates initially observed following pre-operative treatment was encouraging, and now that we are seeing the data mature after four years to include a statistically significant improvement in event-free survival, we look forward to working with the FDA and other global authorities to bring this new option to patients as quickly as possible."

"We are grateful to the study participants who are critical to our efforts to advance potential treatment options for patients with TNBC.”

An analysis of pCR from KEYNOTE-522 was presented at the European Society for Medical Oncology 2019 Congress and published in the New England Journal of Medicine.

Triple-negative breast cancer is an aggressive type of breast cancer that characteristically has a high recurrence rate within the first five years after diagnosis, says Merck.

Merck, known as MSD outside of the United States and Canada, has been inventing for life, bringing forward medicines and vaccines for many of the world’s most challenging diseases in pursuit of our mission to save and improve lives.

May 11, 2021 • 3:52 pm CDT

Osla-based Ultimovacs ASA announced the publication of its positive long-term Overall Survival (OS) data from the Phase I trial evaluating the Company's universal cancer vaccine, UV1, in combination with checkpoint inhibitor ipilimumab in patients with metastatic malignant melanoma.

As published on May 11, 2021, in addition to the achievement of the primary endpoints of safety and tolerability, 50% of the patients were still alive at the data cut-off, supporting the combination of the Company's proprietary UV1 vaccine candidate with ipilimumab, a CTLA-4 checkpoint inhibitor, and standard-of-care treatment, in this late-stage patient population.

"As we continue to evaluate UV1 in various combinations and indications, it is valuable to gain increased international recognition from the clinical community for this study," stated Jens Bjørheim, Chief Medical Officer at Ultimovacs in a company press release.

"Historical data on the use of ipilimumab as monotherapy in malignant melanoma have shown a 5-year survival rate below 20%; therefore, the results published today reinforce UV1's potential in this indication."

The data published in Frontiers in Immunology covers 4.8 years of follow-up on the total of 12 metastatic malignant melanoma patients that were enrolled in the Phase I trial. As reported in the journal, the OS was 50% at 4.8 years, which was confirmed.

Ultimovacs was established in 2011, and its proprietary technology is based on pre-clinical and clinical research on immunotherapies conducted at the Oslo University Hospital. Ultimovacs is headquartered at the Oslo Cancer Cluster Innovation Park in Oslo, Norway, and has an office in Uppsala, Sweden. The company is a limited public liability company listed on the Oslo Stock Exchange in Norway.

May 8, 2021 • 12:59 pm CDT

A global biotechnology company announced that its PARP inhibitor pamiparib had received conditional approval from the China National Medical Products Administration (NMPA) for the treatment of patients with germline BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy.

BeiGene, Ltd.'s new drug application was previously granted priority review by the Center for Drug Evaluation (CDE) in July 2020. 

Beijing and Cambridge, MA-based BeiGene stated in its press release issued on May 7, 2021, it is preparing to launch pamiparib this month (May'21).

“Today’s NMPA approval makes pamiparib the third BeiGene internally discovered and developed medicine to receive marketing authorization, an incredible company milestone validating our scientific innovations,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene, in a press statement.

The NMPA conditional approval of pamiparib for treating patients with advanced ovarian, fallopian tube, or primary peritoneal cancer is based on clinical results from a pivotal Phase 2 portion of the Phase 1/2 trial (NCT03333915). A total of 113 patients in China with high-grade, non-mucinous, epithelial ovarian cancer (including fallopian or primary peritoneal cancer), harboring gBRCA mutations, following at least two prior lines of standard chemotherapy, were enrolled in the pivotal Phase 2 portion of the trial, including 90 patients with advanced platinum-sensitive ovarian cancer (PSOC), and 23 patients with advanced platinum-resistant ovarian cancer (PROC).

Clinical efficacy data in the pamiparib label in China, as assessed by the independent review committee (IRC) per RECIST v1.1, were based on 101 patients evaluable for efficacy analysis, including 82 patients with PSOC and 19 patients with PROC.

For patients with PSOC, with a median follow-up time of 17.0 months, the objective response rate (ORR) was 68.3% (95% CI: 57.1, 78.1), and the median duration of response (DoR) was 13.8 months (95% CI: 10.97, 20.73); for patients with PROC, the median follow-up time was 11.6 months, the ORR was 31.6% (95% CI: 12.6, 56.6) and the median DoR was 11.1 months (95% CI: 4.21, 16.59).

The safety profile of pamiparib in the label in China was based on 317 patients who received pamiparib as monotherapy in three clinical trials. The most common adverse reactions (≥10%) were anemia, nausea, leukopenia, neutropenia, vomiting, fatigue, thrombocytopenia, decreased appetite, diarrhea, abdominal pain, aspartate aminotransferase (AST) increased, alanine aminotransferase (ALT) increased, blood bilirubin increased, and lymphopenia.

Grade ≥3 adverse reactions occurred in 55.8% of patients, with the most common (≥1%) being anemia, neutropenia, leukopenia, thrombocytopenia, lymphopenia, vomiting, fatigue, diarrhea, nausea, and AST increased. Serious adverse reactions occurred in 21.5% of patients, with the most common (≥1%) anemia and leukopenia.

The recommended dose of pamiparib is 60 mg twice daily (BID) taken orally.

In China, ovarian cancer is the deadliest gynecologic cancer, responsible for approximately 22,500 deaths every year, and the five-year survival rate among Chinese patients is about 40%.

Pamiparib is an inhibitor of PARP1 and PARP2, which has demonstrated pharmacological properties such as brain penetration and PARP-DNA complex trapping in preclinical models. Discovered by BeiGene scientists, pamiparib is currently in global clinical development as a monotherapy or in combination with other agents for a variety of solid tumor malignancies.

To date, more than 1,200 patients have been enrolled in clinical trials of pamiparib.

In China, pamiparib received conditional approval to treat patients with germline BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy in May 2021. Full approval for this indication is contingent upon results from ongoing corroborative trials confirming the clinical benefit of pamiparib in this population.

BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. BeiGene is a headquarter-less company by design, with a growing global team of approximately 6,000 colleagues across five continents. To learn more about BeiGene, please visit www.beigene.com.

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