On May 3, 2022 Agendia, Inc. , a commercial-stage company focused on facilitating optimized decision-making by providing clinicians with next-generation information and diagnostic solutions that can be used to help improve outcomes of breast cancer patients worldwide, reported that it will present new data at the European Society for Medical Oncology Breast Cancer Conference 2022 (ESMO Breast 2022) in Berlin, Germany, from 3 to May 5, 2022 (Press release, Agendia, MAY 3, 2022, View Source [SID1234613434]).

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The first of two poster presentations, titled Equivalence of NGS-based MammaPrint 70-gene signature risk of recurrence and BluePrint 80-gene signature of molecular subtyping tests to the centralized microarray tests [234P], concludes that the sequencing version MammaPrint and BluePrint [1] Next-Generation Microarray Testing (NGS) is equivalent to centralized microarray testing, as evidenced by results from several laboratories worldwide. This enables a more accurate decentralized solution for breast cancer care, promoting global accessibility to more reliable genomic tests.

"Accessibility to breast cancer care is crucial and by validating the interchangeability of the NGS version of MammaPrint and BluePrint compared to standardized microarray tests, it opens new doors for clinicians around the world who may need to rely on platforms in the country to secure the MammaPrint and BluePrint results that their patients may need," says Annuska Glas, Senior Vice President of Research and Development and Innovation at Agendia. "MammaPrint is a valuable tool to aid treatment planning for breast cancer patients and with the decentralized NGS platform, it can be offered in even more countries. These results confirm that MammaPrint and BluePrint can provide the same accurate results through of NGS," he adds.

Agendia is creating new ways to ensure accessibility of care around the world, advancing solutions with a decentralized option for test kits to run on an NGS platform, and innovative modalities such as AI digital pathology. This data presented at the ESMO (Free ESMO Whitepaper) Breast 2022 conference follows Agendia’s announcement earlier this year that it was advancing its Digital MammaPrint artificial intelligence (AI) platform in Brazil to potentially radically change the way breast cancer is treated, providing essential information faster and elevating the capabilities of global clinicians treating women with breast cancer in their local communities.

A second poster presentation, entitled Budget impact analysis for the Health Care Package by using MammaPrint in Belgium [238P],builds on earlier research from the landmark MINDACT trial and combines it with interim analysis results from the pilot study conducted in Belgium (unpublished data). The budget impact model shows that the use of MammaPrint results in savings in breast cancer care in Belgium for the Belgian health package compared to no gene expression profiling testing. According to the budget impact model, the use of MammaPrint in patients selected according to the criteria of the pilot study represents a great saving. If gene expression profiling were performed on all clinically high-risk patients, the savings would be even greater.

Combining the cost of genomic testing with the savings from the net reduction in chemotherapy use, the annual savings amount to more than €5.8 million, which is an average saving of €3,900 per patient, compared to standard clinical care of a patient with breast cancer in Belgium. MINDACT has carried out previous analyzes of health savings in several countries, published in the European Journal of Cancer ,showing that genomic testing is beneficial as it provides valuable information that potentially allows patients to avoid the complexity of chemotherapy, resulting in significant financial savings for them, while generating significant cost savings for large healthcare systems around the world.

"A partir de los datos de impacto utilizados en el estudio piloto realizado en Bélgica, podemos determinar nuevas ideas de que el uso de MammaPrint podría conceder un ahorro sustancial de costes a las mujeres que no requieran quimioterapia y, en última instancia, podría aliviar parte de la carga del sistema sanitario belga en su conjunto", señala Pino Cusumano, MD, cirujano de mama del Centre Hospitalier Chrétien de Lieja en Bélgica. "Los datos han demostrado previamente que al desescalar a las mujeres con casos de bajo riesgo del tratamiento de quimioterapia, los resultados siguen siendo igual de buenos y las mujeres pueden seguir un plan de tratamiento más personalizado. Creemos que estos datos demuestran que el análisis anterior es válido para el estado de la atención del cáncer de mama en Bélgica en 2022,and that the overall benefit to the patient extends beyond physical care, to financial well-being as well," he continues.

Taken together, the two posters presented at ESMO (Free ESMO Whitepaper) Breast 2022 suggest that no matter where a patient is located, she can receive the same high-quality results with the added benefit of cost-effective treatment planning.

"The studies presented at EMSO 2022 support Agendia’s key initiatives to ensure that our genomic tests are widely accessible and affordable, without compromising scientific integrity or decision-making, to provide the clarity that women with breast cancer seek to guide treatment planning," says Bastiaan van der Baan, Director of Clinical and Commercial Development at Agendia. "With our strong commitment to facilitating the global adoption of MammaPrint and BluePrint, we look forward to continuing studies like these to increase the evidence supporting our goal of caring for women with breast cancer throughout their treatment," he concludes.

1 The MammaPrint and BluePrint next-generation sequencing kits have not yet been cleared by the Food and Drug Administration for sale in the United States; BluePrint has received CE marking for use in Europe. BluePrint is also marketed as a Laboratory Developed Test (LDT) and is CAP/CLIA audited in the United States.

On May 3, 2022 Nordic Nanovector ASA (OSE: NANOV), a clinical-stage biotech company focused on CD37-targeted therapies for haematological cancers and immune diseases, reported the publication of two new research papers highlighting approaches to improve the potential therapeutic effect of its novel CD37-targeting radioimmunoconjugate Humalutin (177Lu-DOTA-NNV003) in B-cell malignancies, such as Non-Hodgkin Lymphoma (NHL) (Press release, Nordic Nanovector, MAY 3, 2022, View Source [SID1234613451]).

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The first publication by the Company’s scientists and its collaborators, published in PLOS One (Ref. 1), reports on the combined effect of Humalutin with olaparib, a member of the class of anticancer therapies known as PARP inhibitors, on NHL cell lines.

In the studies, the combination of Humalutin and olaparib was found to be synergistic or conditionally synergistic leading to cell death in 6 of 7 NHL cell lines (diffuse large B cell lymphoma and mantle cell lymphoma). Where the combination was conditionally synergistic (i.e. both synergistic and antagonistic), the effect was dependent on the concentration of each drug, showing the importance of optimising the parameters for further studies.

Humalutin acts by inducing potentially cytotoxic DNA breaks in the NHL cells, sensitising these cells to olaparib, which prevents the repair of DNA breaks by blocking the activity of DNA repair enzymes poly (ADP ribose) polymerase 1 and 2 (PARP1 and PARP2). Olaparib is approved in the US and most markets globally for BRCA mutated ovarian and breast cancer.

The authors concluded that further in vivo studies evaluating the anti-tumour effect of the combination of radioimmunotherapies, including Humalutin, and PARP inhibition are warranted.

Separately, Nordic Nanovector reports the publication of a paper in the high-impact open access journal Scientific Reports (Ref. 2) on the potential of a non-invasive diagnostic imaging approach to select NHL patients who are more likely to respond to or are at risk for developing CD37-induced haematological toxicities from CD37-targeted radioimmunotherapy.

The imaging approach used a radioimmunoconjugate ([89Zr]Zr-N-sucDf-NNV003) comprising the Company’s proprietary anti-CD37 antibody NNV003 (a component of Humalutin), and zirconium-89, a radioisotope that is well-suited to commonly used positron emission tomography (PET) imaging, to assess CD37-expression, biodistribution and tumour-uptake levels in mice bearing human B cell lymphomas and to predict the possible therapeutic effects of Humalutin in NHL patients.

A good manufacturing practice (GMP)-compliant production process has also been established to enable administration to patients in future studies.

Jostein Dahle, Nordic Nanovector’s Chief Scientific Officer, said: "These two publications add to the growing scientific evidence supporting CD37 as a valuable tumour target both for therapeutic and diagnostic applications in NHL. This evidence provides important validation of our pipeline approach, building on the significant data we have collected from our preclinical and clinical studies with Betalutin and now expanding to our next-generation CD37-targeting radioimmunoconjugate Humalutin. We look forward to continuing to grow our understanding around CD37 and the potential of our emerging pipeline."

References

1. Malenge, M.M. et al. Anti-CD37 radioimmunotherapy with 177Lu-NNV003 synergizes with the PARP inhibitor olaparib in treatment of non-Hodgkin’s lymphoma in vitro. PLOS One (2022): 17(4): e0267543

2. Giesen, D. et al. 89Zr-PET imaging to predict tumor uptake of 177Lu-NNV003 anti-CD37 radioimmunotherapy in mouse models of B cell lymphoma. Sci Rep 12, 6286 (2022). View Source

On May 3, 2022 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported that Dr. William G. Rice, Chairman, President and CEO of Aptose, will participate in the RBC Capital Markets Global Healthcare Conference (Press release, Aptose Biosciences, MAY 3, 2022, View Source [SID1234613360]):

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2022 RBC Capital Markets Global Healthcare Conference

The Aptose management team also will be hosting 1×1 meetings during the events.

On May 3, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the first quarter ended March 31, 2022 (Press release, Blueprint Medicines, MAY 3, 2022, View Source [SID1234613385]).

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"The first quarter was marked by a number of important milestones across our business. We have strong momentum in our commercial execution, our pipeline is advancing rapidly, and we continue to build on our scientific leadership," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our ongoing global launch of AYVAKIT/AYVAKYT (avapritinib) in the U.S. and now also in Europe is establishing a new standard of care for the treatment of advanced SM, targeting the underlying cause of the disease. We are on track to have topline data from our registration-enabling PIONEER trial in late summer, further expanding our leadership in SM as we potentially bring the first and only medicine to patients with the non-advanced form of the disease. In addition, we continue to progress our pipeline of innovative investigational medicines in difficult-to-treat and prevalent cancers such as non-small cell lung cancer and breast cancer. At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, we presented five abstracts highlighting data across four programs within our EGFR and CDK2 franchises, while earlier in the quarter we announced a targeted protein degradation discovery collaboration with Proteovant Therapeutics. The strength of this past quarter demonstrates that we are well on our way of delivering on our 2022 goals while also building the foundation to drive further value for the years ahead."

First Quarter 2022 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): advanced systemic mastocytosis (SM) and PDGFRA gastrointestinal stromal tumor (GIST)

Recorded global net product revenues of $23.8 million for the first quarter of 2022.
Received European Commission approval for AYVAKYT for the treatment of adult patients with advanced SM, including aggressive SM, SM with an associated hematological neoplasm, or mast cell leukemia, after at least one systemic therapy, and treated the first commercial patients in Germany. Read the press release here.
GAVRETO (pralsetinib): RET-altered cancers

As previously recorded and reported by Roche, GAVRETO product sales for their region were 5 million CHF, which excludes sales in the Greater China territory driven by CStone Pharmaceuticals.
BLU-945, BLU-701 and BLU-451 (formerly LNG-451): EGFR-driven NSCLC

Reported proof-of-concept data at AACR (Free AACR Whitepaper) from the Phase 1/2 SYMPHONY clinical trial of BLU-945, showing early evidence of safety and clinical activity, with dose-dependent decreases in circulating tumor DNA (EGFR variant allele fractions) and radiographic tumor reductions, including an unconfirmed partial response (PR) in a patient treated with 400 mg once daily (QD). BLU-945 was generally well-tolerated, with no significant adverse events (AEs) associated with wild-type EGFR inhibition. The maximum tolerated dose and recommended Phase 2 dose have not yet been identified, and dose escalation is continuing. These results support plans to expand the development of BLU-945 in combination with multiple agents, including osimertinib, with the goal of preventing or treating tumor resistance to prolong patient benefit. Read the press release here.
Entered into a clinical trial supply agreement with AstraZeneca (LSE/STO/Nasdaq:AZN), under which Blueprint Medicines will evaluate BLU-945 and BLU-701 in combination with osimertinib in the ongoing SYMPHONY and HARMONY trials, respectively.
Also at AACR (Free AACR Whitepaper), reported preclinical data supporting the development of BLU-451 in EGFR exon 20 insertion-positive NSCLC.
Initiated patient dosing in the CONCERTO trial, a Phase 1/2 trial of BLU-451 in patients with EGFR-driven NSCLC harboring exon 20 insertion mutations.
BLU-222: breast, ovarian, and other CDK2-vulnerable cancers, including CCNE1-amplified tumors

Reported preclinical data in a CCNE1-amplified ovarian tumor model at AACR (Free AACR Whitepaper) supporting the development of BLU-222 in CDK2-vulnerable cancers.
Initiated the VELA trial, a Phase 1/2 trial of BLU-222 in CDK2-vulnerable cancers, including estrogen-receptor-positive breast cancer and a range of other CCNE1-amplified tumors, and dosed the first patient in Part 1 dose escalation.
Corporate

Announced strategic collaboration with Proteovant Therapeutics to advance novel targeted protein degrader therapies to address important areas of medical need. Under the terms of the collaboration, the companies will jointly research important targets and advance up to four novel protein degrader therapies into development candidates. Read the press release here.
Recognized a $30 million milestone payment from Clementia related to the initiation of a Phase 2 trial of BLU-782, which is now called IPN60130, our out-licensed ALK2 inhibitor in development for the rare bone disease fibrodysplasia ossificans progressiva.
Key Upcoming Milestones

The company plans to achieve the following near-term milestones:

Report topline data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in late summer 2022 and submit a supplemental new drug application to the U.S. Food and Drug Administration for AYVAKIT in non-advanced SM in the second half of 2022.
Present initial data from the dose escalation cohort of the Phase 1/2 SYMPHONY trial evaluating BLU-945 in combination with osimertinib in the second half of 2022.
Present initial clinical data from the Phase 1/2 HARMONY trial of BLU-701 in the second half of 2022.
Present initial data from Part 1 of the HARBOR trial of BLU-263 in non-advanced SM in the second half of 2022.
Share the company’s research and portfolio vision, including scientific platform expansion plans, at an R&D Day in the second half of 2022.
First Quarter 2022 Results

Revenues: Revenues were $62.7 million for the first quarter of 2022, including $23.8 million of net product revenues from sales of AYVAKIT/AYVAKYT and $38.9 million in collaboration revenues. Blueprint Medicines recorded revenues of $21.6 million in the first quarter of 2021, including $7.1 million of net product revenues from sales of AYVAKIT/AYVAKIT, $1.8 million of net product revenues from sales of GAVRETO and $12.6 million in collaboration revenues.
Cost of Sales: Cost of sales was $5.1 million for the first quarter of 2022, as compared to $0.1 million for the first quarter of 2021.
R&D Expenses: Research and development expenses were $103.1 million for the first quarter of 2022, as compared to $79.7 million for the first quarter of 2021. This increase was primarily due to increased costs associated with the progression of our clinical trials, increased costs related to early discovery efforts, and a decrease in reimbursement from the global development cost sharing arrangement under our collaboration with Roche for GAVRETO. Research and development expenses included $10.0 million in stock-based compensation expenses for the first quarter of 2022.
SG&A Expenses: Selling, general and administrative expenses were $57.1 million for the first quarter of 2022, as compared to $42.0 million for the first quarter of 2021. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. General and administrative expenses included $13.4 million in stock-based compensation expenses for the first quarter of 2022.
Net Loss: Net loss was $106.0 million for the first quarter of 2022, or a net loss per share of $1.79, as compared to a net loss of $99.7 million for the first quarter of 2021, or a net loss per share of $1.72.
Cash Position: As of March 31, 2022, cash, cash equivalents and investments were $893.4 million, as compared to $1,034.6 million as of December 31, 2021.
Financial Guidance

Blueprint Medicines continues to anticipate approximately $180 to $200 million in total net revenues in 2022, including approximately $115 to $130 million in AYVAKIT net product revenues. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss first quarter 2022 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 694684. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in three upcoming investor conferences:

Jefferies Healthcare Conference on Tuesday, June 9, 2022 in New York, NY.
JMP Securities 2022 Life Sciences Conference on Thursday, June 16, 2022 in New York, NY.
Goldman Sachs 43rd Annual Global Healthcare Conference on Thursday, June 16, 2022 in Rancho Palos Verdes, CA.
A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.

On May 3, 2022 Heat Biologics, Inc. / NightHawk Biosciences (NYSE American: HTBX; NHWK), a fully integrated biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, reported that it has completed its name change from Heat Biologics, Inc. to NightHawk Biosciences, Inc. to better reflect the Company’s evolution, including expansion of the therapeutic pipeline, vertical integration of capabilities from drug discovery to manufacturing and commercialization, as well as the Company’s new biodefense capabilities (Press release, NightHawk Biosciences, MAY 3, 2022, View Source [SID1234613401]). In connection with the name change, the Company’s ticker will change to "NHWK," effective today, May 3, 2022. The name and symbol changes do not affect the Company’s share structure or the rights of the Company’s shareholders, and no further action is required by existing shareholders.

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Jeff Wolf, CEO of Nighthawk, commented, "The NightHawk brand better reflects our evolution towards a fully integrated ecosystem, designed to efficiently move drugs from discovery through commercialization. The NightHawk ecosystem includes an expanded development pipeline, new biodefense capabilities and enhanced manufacturing capabilities. As a result, we have organized the Company around five key subsidiaries: Skunkworx Bio, Heat Biologics, Pelican Therapeutics, Elusys Therapeutics and Scorpion Biological Services. NightHawk is laser focused on addressing key industry challenges, including the slow pace and high cost of new drug development through a fully integrated ecosystem of drug discovery, preclinical testing and manufacturing. We look forward to progressing these efforts under the NightHawk banner."