On June 23, 2022 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on research and development of therapies for difficult-to-treat hematological diseases, reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), has unanimously adopted a positive opinion recommending a full marketing authorization approval (MAA) of Pepaxti (melphalan flufenamide, also called melflufen) in EU (Press release, Oncopeptides, JUN 23, 2022, View Source [SID1234616217]). The European Commission (EC) will make a legally binding decision based on the EMA recommendation within 60 days. Once granted by EC, the marketing authorization is valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Lichtenstein, and Norway.

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The positive opinion is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study which was utilized as confirmatory study. No specific post-marketing commitments were issued. Oncopeptides intends to submit a type II variation in Q4 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).

Pepaxti is indicated, in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

"Pepaxti helps patients with multiple myeloma, an incurable hematologic cancer. Today’s positive CHMP opinion confirms that Pepaxti provides benefit to these patients and is foundational for the future of Oncopeptides and our development pipeline," says Jakob Lindberg, CEO of Oncopeptides. "Based on the scientific evaluation by EMA, our dialogue with the US Food and Drug Administration (FDA) has now been intensified to achieve a clear path forward also for US patients."

Efficacy results for triple-class refractory patients who have received at least 3 prior lines of therapies and who had no ASCT or progressed more than 36 months after an ASCT in the HORIZON study

"The recommendation for full approval of Pepaxti by EMA is really good news for patients with triple class refractory disease, where the unmet medical need remains high and treatment options often are exhausted," says Pieter Sonneveld, professor of Hematology at the Erasmus University Medical Center in Rotterdam, the Netherlands and principal investigator of the OCEAN study.

"EMA´s assessment of Pepaxti corroborates our scientific conclusion that the overall survival result in the OCEAN study constitutes a case of true survival heterogeneity which is reflected in the indication statement in accordance with the agency´s guidelines," says Klaas Bakker, MD, PhD, Executive Vice President, and Chief Medical Officer. "In addition, EMA confirms that there are no toxicological safety signals in both studies and there is a positive benefit risk profile in the indicated patient population. The non-transplanted, often older patient population, which represents the largest group of RRMM patients, particularly benefits from treatment with Pepaxti."

As previously disclosed, Oncopeptides has an EIB loan facility. Oncopeptides and EIB are currently in negotiations, to update tranche definitions to reflect the current regulatory situation. In addition, the Company is considering additional financing options to capture the opportunities with the upcoming EU-approval. This may include new share issues and other public or private financing options.

Oncopeptides will advance market access activities after an approval by the European Commission, to pave the way for a successful launch of Pepaxti in Germany in Q4, 2022. The Company is actively considering various options to commercialize the drug, making it available for patients across Europe, and maximizing shareholder value.

Conference call for investors, analysts, and media 

Investors, financial analysts, and media are invited to participate in a webcast with a Q&A session on June 27, 2022, at 11:00 (CET). The event will be hosted by CEO Jakob Lindberg, CMO Klaas Bakker and CFO Annika Muskantor.

Webcast

The webcast will be streamed via View Source
The link can also be found on the website: www.oncopeptides.com.

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person above, on June 23, 2022, at 17:55 (CET).

About Pepaxti

Pepaxti (melphalan flufenamide, also called melflufen) is a lipophilic peptide conjugated alkylating drug that rapidly and selectively is delivering cytotoxic agents into tumor cells. The drug is composed of a di-peptide and an alkylating moiety. The lipophilicity allows a faster cellular uptake whereas the peptide hydrolysis mediated by aminopeptidases, results in accumulation of alkylating moieties in cancer cells. This results in an improved efficacy without an increased toxicity compared to melphalan. Pepaxti inhibits proliferation and induces apoptosis of haematopoietic and solid tumour cells. It shows synergistic cytotoxicity in combination with dexamethasone in melphalan resistant and non-resistant multiple myeloma cell lines.

Pepaxti is indicated in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapies. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

About Multiple Myeloma

Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cells which produce antibodies to help fight infection, and cause cancer cells to accumulate in the bone marrow. Multiple Myeloma is the second most common hematologic malignancy, and accounts for approximately 1-2% of all new cancer cases, with a global incidence rate of 1.7 per 100,000 and an age-standardized incidence rate of 2.1-3.4 per 100,000 in France, Germany, Italy, Spain, and the UK. An estimated 35,842 patients were diagnosed in the EU27 during 2020, with an estimated 23,275 deaths due to the disease (ECIS 2020).

Patients with multiple myeloma may have symptom-free periods, but the disease always relapses, and patients may become refractory to all available treatment options due to mutations and/or clonal evolution of the tumor cells. A growing subset of patients are triple-class refractory, and develop disease refractory to immunomodulatory drugs, proteasome inhibitors, and CD38- targeting monoclonal antibodies. These patients have a very short expected overall survival.

On June 23, 2022 Paige, a global leader in clinical AI applications in pathology, reported it received CE-IVD and UKCA marks for HER2Complete, an artificial intelligence (AI) software designed to identify patients with breast cancer whose tumors have expressions of human epidermal growth factor receptor 2 (HER2) protein (Press release, Paige AI, JUN 23, 2022, View Source [SID1234616216]).* This is the first CE-IVD and UKCA designated tool to explore the novel space of HER2-low. In a recent study, HER2Complete was able to detect levels of HER2 expression in HER2-negative (IHC-0) and HER2-low (IHC1+/2+) hematoxylin and eosin (H&E)-stained tissue samples, the first and only AI biomarker assay capable of doing so.

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HER2 is a protein that promotes breast cancer cell growth, and breast cancer cells with excess levels of HER2 are called HER2-positive.1 Targeted therapies for HER2-positive tumors have been a mainstay of cancer treatment over the past two decades. Recent evidence suggests there may be a subgroup of patients who are considered HER2-negative through standard immunohistochemistry (IHC) and fluorescence in situ hybridization (FISH) testing but may actually have low or ultra-low levels of HER2, and therefore may be responsive to particular investigative therapies.2,3 It has been challenging to identify these patients reliably and reproducibly using conventional methods, calling for the development of novel ways to assess a patient’s HER2 status that are more accurate and sensitive than traditional assays, which were designed and optimized to detect high levels of HER2 expression.

In contrast to traditional IHC tests for HER2, Paige deploys HER2Complete to detect HER2 expression based on protein and mRNA levels on digital images of H&E-stained tissue samples, with results generated rapidly at the click of a button. Recent work has shown that HER2Complete can also identify HER2 expression in patients currently classified as IHC negative (or IHC-0), in addition to expression in HER2-low (IHC1+ and 2+/FISH negative) patients. This approach complements existing IHC testing to potentially identify true HER2-expressing breast cancers without the need for special staining approaches and with a very rapid turnaround using only the diagnostic biopsy or resection slides. Further, this tool has been used to detect true HER2-negative disease, whose definitive identification based on a lack of IHC and mRNA HER2 expression may accelerate efforts for developing new therapies for this important group of patients who may not benefit from HER2 targeted therapies. This approach doesn’t require any special labelling of the tissue sample and can be done rapidly and easily on standard tissue preparations using methods that already exist in labs across the globe.

"AI brings a transformational approach to diagnostics and allows us to identify low levels of HER2 in tissue that we would not be able to detect using current assays," said David Klimstra, M.D., Founder and Chief Medical Officer at Paige. "We are working to enable the next generation of HER2 testing to provide the information physicians need to guide the use of next generation HER2 therapy. We believe that this assay will allow us to reliably identify increased likelihood of HER2 expression, even on samples where HER2 expression was misclassified by legacy diagnostics as low or null."

"Our product is potentially a very targeted and cost-effective solution to identify a subset of patients who have previously been unidentified," said Jill Stefanelli, Ph.D., President and Chief Business Officer at Paige. "Paige is developing an end-to-end solution to detect and further characterize breast cancer so that pathologists and oncologists get comprehensive insights into an individual’s cancer. We look forward to assessing the clinical utility with multiple potential partners in order to link this test to treatment outcomes."

*In the United States, the software is available for Research Use Only and not for use in diagnostic procedures.

On June 23, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported findings from a study that evaluated uveal melanoma (UM) patients’ attitudes toward prognostic testing and specifically with respect to DecisionDx-UM (Press release, Castle Biosciences, JUN 23, 2022, View Source [SID1234616215]). Highlights from the study were shared in a poster presentation at the 20th congress of the International Society of Ocular Oncology (ISOO), recently held in Leiden, The Netherlands.

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The poster, titled "Uveal Melanoma Patient Attitudes Towards Prognostic Testing Using Gene Expression Profiling" and presented by Basil K. Williams, Jr., M.D., assistant professor in the Department of Ophthalmology and Mary Knight Asbury Chair of Ocular Oncology at the University of Cincinnati College of Medicine, can be viewed here.

"Most patients impacted by the rare but quite deadly disease, uveal melanoma, want and expect to play an active role in their care to ensure the best possible outcome," said Williams. "We believe the survey data supported this sentiment and found that regardless of their overall prognosis, patients value the information provided by their DecisionDx-UM test results."

Study highlights:

An online questionnaire was distributed by the Melanoma Research Foundation’s CURE OM (Ocular Melanoma) initiative to capture anonymous information regarding patient-reported experiences following prognostic testing.
Patients were asked questions regarding the decision to undergo prognostic testing and the extent to which they felt decision regret.
Of the 177 survey participants, 90% reported wanting prognostic information at diagnosis.
Of the patients who received prognostic testing with DecisionDx-UM, there was no significant difference in decision regret levels among those receiving a low- (Class 1A), intermediate- (Class 1B) or high-risk (Class 2) test result (Kruskal-Wallis rank sum test, X2=4.1, p=0.13).
Patients tested with DecisionDx-UM reported gaining value from their test result, including:
Increased knowledge and understanding of their disease
More personalized treatment options
Information relevant to life planning
Relief from uncertainty about the future
About DecisionDx-UM

DecisionDx-UM is Castle Biosciences’ 15-gene expression profile (GEP) test that uses an individual patient’s tumor biology to predict individual risk of metastasis in patients with uveal melanoma. DecisionDx-UM is the standard of care in the management of newly diagnosed uveal melanoma in the majority of ocular oncology practices in the United States. Since 2009, the American Joint Committee on Cancer (AJCC; v7 and v8) Staging Manual for UM has specifically identified the GEP test as a prognostic factor that is recommended for collection as a part of clinical care. Further, the National Comprehensive Cancer Network (NCCN) guidelines for uveal melanoma include the DecisionDx-UM test result as a prognostic method for determining risk of metastasis and recommended differential surveillance regimens based on a Class 1A, 1B, and 2 result. DecisionDx-UM is the only prognostic test for uveal melanoma that has been validated in prospective, multi-center studies, and it has been shown to be a superior predictor of metastasis compared to other prognostic factors, such as chromosome 3 status, mutational status, AJCC stage and cell type.

It is estimated that nearly 8 in 10 patients diagnosed with uveal melanoma in the U.S. receive the DecisionDx-UM test as part of their diagnostic workup.

On June 23, 2022 Hummingbird Diagnostics GmbH, a leader in reading blood-based microRNAs for early disease detection and characterization, reported a publication in the Journal of Thoracic Oncology Clinical and Research Reports that demonstrates their blood 5-microRNA signature (miRisk) to predict survival following immunotherapy in advanced non-small cell lung cancer (NSCLC) patients with high PD-L1 expression (Press release, Hummingbird Diagnostics, JUN 23, 2022, View Source [SID1234616214]).

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The study builds on results published in March 2022 in npj Precision Oncology [1] and addresses a current clinical dilemma; which advanced-stage NSCLC patients with high PD-L1 expression (≥ 50%) should be treated with immunotherapy (IO) alone versus immunotherapy in combination with chemotherapy (ICT)? Both are currently licensed therapies in international guidelines, yet no biomarkers exist to guide this decision. Optimizing this choice using principles of precision medicine will have significant impacts on patient survival and quality of life, by including toxic chemotherapy only when deemed necessary. Hummingbird’s results demonstrate a significant association between overall survival (OS) and the miRisk score in IO treated patients and furthermore highlight its value as a predictive biomarker for type of treatment (IO or ICT). The score could identify high-risk patients who might benefit from treatment with ICT, as opposed to IO, and support treatment decisions as a blood-based complementary diagnostics.

"Patients with advanced, non-oncogene-driven, non-small-cell lung cancer (NSCLC) with high PD-L1 expression are eligible for treatment with immunotherapy. There is, however, an urgent medical need for biomarkers identifying cases that require additional combination with chemotherapy," says Timothy Rajakumar, MD PhD, Medical Director of Hummingbird Diagnostics and first author on the study. "The miRisk score represents an immune focused biomarker that is specifically predictive of response to immunotherapy and could serve as the foundation for a complementary diagnostic to guide therapeutic decisions and thereby allow physicians to more accurately choose between treating patients with IO alone vs. ICT."

The study was performed in collaboration with Professor Petros Christopoulos from the Thoraxklinik at Heidelberg University Hospital, as well as with Professor Martin Reck, Grosshansdorf. A valuable study cohort was assembled from a total of 155 whole blood samples, prospectively collected from patients with stage IV NSCLC with PD-L1 TPS ≥50%, before they had been initiated on treatment with IO or ICT. These samples were subject to small RNA sequencing and used to train and validate a 5-microRNA model to predict OS (miRisk). This model was significantly associated with OS and, most importantly, with the type of therapy (IO or ICT) therefore offering the promise to support clinical decisions surrounding choice of therapy.

Bruno Steinkraus, PhD, Chief Scientific Officer of Hummingbird Diagnostics remarked: "We believe our analysis will provide a blueprint for host-based integrative biomarker usage in a field of pressing medical need with the emergence of more complex immunotherapy regimens. Taken together with the relatively simple use of a peripheral whole blood test that does not require pipetting at point of care, we envision applicability of this technology to non-invasive therapy guidance for the IO or ICT decision in PD-L1 high patients."

The open-access article can be found online on the journal’s website: View Source(22)00093-5/fulltext

1Rajakumar T et al. A blood-based miRNA signature with prognostic value for overall survival in advanced stage non-small cell lung cancer treated with immunotherapy. NPJ Precis Oncol 2022; 6:19

On June 23, 2022 McKesson Corporation (NYSE: MCK) and HCA Healthcare, Inc. (NYSE: HCA) reported an agreement to form a joint venture combining McKesson’s US Oncology Research (USOR) and HCA Healthcare’s Sarah Cannon Research Institute (SCRI) (Press release, McKesson, JUN 23, 2022, View Source [SID1234616213]). USOR is the research arm of McKesson’s The US Oncology Network and draws from a network of experienced investigators and dedicated clinical staff who specialize in oncology clinical trials. SCRI, which is the research arm of Sarah Cannon, HCA Healthcare’s Cancer Institute, offers end-to-end clinical trial site support services with a deep expertise in early-phase oncology research and drug development as well as a specialized contract research organization (CRO).

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Together, USOR and SCRI will create a fully integrated oncology research organization aimed at expanding clinical research, accelerating drug development and increasing availability and access to clinical trials for community oncology providers and patients, including those in underserved communities. The joint venture plans to offer an expanded clinical research network, a broader portfolio of clinical trials, and enhanced data and analytics capabilities to better match patients with clinical trials.

"McKesson and HCA Healthcare share a commitment to advancing the next generation of cancer care," said Brian Tyler, chief executive officer, McKesson. "This new joint venture is an important step forward in increasing access to clinical trials, particularly within the community setting, where the majority of all cancer patients are initially treated. Additionally, the joint venture directly aligns with McKesson’s strategic growth priorities by further expanding our differentiated oncology ecosystem and improving the value proposition for provider and biopharma partners."

"Clinical research and partnerships are fundamental to how we address our communities’ most pressing healthcare needs," said Sam Hazen, chief executive officer of HCA Healthcare. "We believe this joint venture with McKesson, which unifies our oncology research experts, will promote the development of individualized therapies and provide more opportunities for cancer patients to receive new treatments."

The transaction is expected to close in 2022. Following the close of the transaction, McKesson will own 51% of the joint venture and have operating control. The joint venture will be governed by a Board of Directors with representation from both McKesson and HCA Healthcare. The new company will be led by SCRI’s Dee Anna Smith, who will serve as chief executive officer, and SCRI’s Howard A. "Skip" Burris, III, MD, who will serve as president. Additionally, USOR’s Robert Coleman, MD will become the new organization’s chief medical officer and SCRI’s David Spigel, MD will be its chief scientific officer.

"When it comes to the fight against cancer, building a network of collaborators is essential. Our impact on patients is evident through our longstanding partnerships with experts from Tennessee Oncology, Florida Cancer Specialists and many other valued oncology programs," said Dee Anna Smith, chief executive officer of Sarah Cannon. "In the last three decades, our collaborations have led to advancing treatment standards for the majority of new cancer therapies that are available to patients today. We believe that coming together with US Oncology Research will complement and strengthen our network so that we can continue to make a greater impact for people living with cancer."

Separately, McKesson will acquire Genospace, SCRI’s personalized medicine platform. Genospace is a leading innovator in precision medicine and clinical trial matching. By becoming a part of McKesson, Genospace will power the oncology data and analytics capabilities for the joint venture as well as enhance the ability of its provider partners to more efficiently identify the most appropriate therapies or clinical trials for their patients.

Research is essential to advancing cancer care from diagnosis through survivorship, and as such, the joint venture will enhance both companies’ cancer care offerings. McKesson’s The US Oncology Network and HCA Healthcare’s cancer network, Sarah Cannon Cancer Institute, are not part of the transaction and will continue to operate independently of the joint venture.

Financial terms of the agreement were not disclosed. The transaction is subject to necessary regulatory approvals and other customary closing conditions.