On July 27, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer,reported the acceptance of five oral presentations at the International Society of Pediatric Oncology ("SIOP") Virtual Annual Congress held October 14 through October 17, 2020 in Ottawa, Canada (Press release, Y-mAbs Therapeutics, JUL 27, 2020, View Source [SID1234562454]). The abstracts are publicly available online at View Source

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Naxitamab

The abstracts include the following presentations of naxitamab, one of the Company’s lead product candidates, which is currently being evaluated for the treatment of pediatric patients with relapsed or refractory high-risk neuroblastoma, osteosarcoma and other GD2-positive tumors:

"High-dose naxitamab (humanized-3F8) plus stepped-up dosing of granulocyte-macrophage colony-stimulating factor (GM-CSF) for resistant osteomedullary neuroblastoma: major responses and outpatient treatment in a Phase II trial," submitted by MSK in New York

"Naxitamab-based chemoimmunotherapy for resistant high-risk neuroblastoma: preliminary results of HITS treatment," submitted by SJD Barcelona Children’s Hospital in Barcelona, Spain

"Telemedicine blood pressure monitoring after anti-GD2 monoclonal antibody immunotherapy during the COVID-19 pandemic," submitted by MSK in New York
Naxitamab has been accepted for priority review by the U.S. Food and Drug Administration ("FDA") for the treatment of patients with relapsed/refractory high-risk neuroblastoma. The FDA set an action date of November 30, 2020, under the Prescription Drug User Fee Act ("PDUFA").

Omburtamab

The abstracts also include the following presentation of omburtamab, the Company’s other lead product candidate, which is currently being evaluated for the treatment of patients with CNS/Leptomeningeal metastasis from neuroblastoma, diffuse intrinsic pontine glioma ("DIPG"), and desmoplastic small round cell tumors ("DSRCT"):

"Intracerebroventricular radioimmunotherapy using 131I-omburtamab for neuroblastoma central nervous system/leptomeningeal metastases, interim results from multi-center trial 101," submitted by Memorial Sloan Kettering Cancer Center ("MSK") in New York
During June 2020, we initiated the submission of the Biologics License Application ("BLA") for omburtamab under the FDA’s Rolling Review process and completion of the BLA submission is currently expected to take place over the next few weeks.

GD2-GD3 Vaccine

The abstracts also include the following presentation of the GD2-GD3 Vaccine, the Company’s vaccine candidate, which is currently being evaluated for high-risk neuroblastoma patients in remission:

"Favorable toxicity profile of bivalent GD2/GD3 neuroblastoma vaccine," submitted by MSK in New York

On July 27, 2020 CARISMA Therapeutics Inc., a biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the Company’s lead product candidate, CT-0508, an anti-human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M) (Press release, Carisma Therapeutics, JUL 27, 2020, View Source [SID1234562419]). Under this IND, CARISMA intends to initiate its Phase 1, first-in-human, multi-center study in patients with recurrent or metastatic HER2 overexpressing solid tumors after failure of approved HER2 targeted agents.

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"This will be the first time that an engineered macrophage has progressed successfully to the in-patient study phase and represents a new chapter for CARISMA: advancing from a preclinical discovery-stage company to a clinical development stage company," said Steven Kelly, President and Chief Executive Officer. "The clearance of our IND application for CT-0508 is a ground-breaking milestone in the field of cell-based cancer immunotherapy."

Historically, cell therapies have encountered key challenges treating solid tumors, including limited trafficking to the tumor site, an immunosuppressive tumor microenvironment, and the heterogeneous expression of tumor-associated antigens, but CARISMA’s preclinical findings suggest that CAR-M therapy could overcome these challenges.

"Our preclinical findings indicate that CAR-M have the ability to mount a broad immune response against cancers, and the acceptance of the CT-0508 IND brings this technology to patients with incurable solid tumors," said Michael Klichinsky, PharmD, PhD, co-inventor of the CAR-M technology, Scientific Co-founder, and Vice President of Discovery at CARISMA Therapeutics.

CARISMA Scientific Co-founder and Assistant Professor of Hematology-Oncology in Penn’s Abramson Cancer Center, Saar Gill, MD, PhD, added, "I’m incredibly pleased and excited to see the technology that was originally developed in my lab progress to the clinic. Getting a chance to translate your preclinical research and evaluate its potential impact for patients is the reason why I do this type of work."

The planned clinical trial will be conducted at two trial sites—University of Pennsylvania in Philadelphia, Pennsylvania, and University of North Carolina in Chapel Hill, North Carolina—and will enroll patients with different types of recurrent or metastatic cancers with HER2 overexpressing solid tumors.

"HER2 is overexpressed not only in breast and gastroesophageal cancers, but in a wide variety of epithelial origin solid tumors, such as non-small cell lung, colorectal, bladder, and pancreatic cancers," said Debora Barton, MD, Chief Medical Officer at CARISMA Therapeutics. "There is an important unmet medical need that remains to be addressed and we aim to achieve that during this clinical trial."

Corporate Developments

CARISMA also announced today the expansion of its Board of Directors to include Briggs W. Morrison, MD, as well as additions to the Scientific Advisory Board, Nina Bhardwaj, MD, PhD, and Prasad S. Adusumilli, MD.

"CARISMA is at an exciting juncture," said Mike Heffernan, Chairman of the Board of Directors. "Evolving from a preclinical stage company, having demonstrated reduced tumor burden and significantly improved overall survival with our CAR-M technology in humanized mouse models, to quickly approaching the in-clinic study of this first-of-its-kind therapy. We are eager to have Drs. Morrison, Bhardwaj and Adusumilli’s counsel as CARISMA embarks on this new chapter."

The CARISMA Board of Directors welcomes Dr. Briggs Morrison, who brings with him over 25 years of pharmaceutical, global regulatory and business development leadership experience. Joining the Scientific Advisory Board are Drs. Nina Bhardwaj and Prasad S. Adusumilli. Dr. Bhardwaj brings extensive immunology experience: she is currently the Director of Immunotherapy, Medical Director of the Vaccine and Cell Therapy Laboratory, and Co-Director of the Cancer Immunology Program at The Tisch Cancer Institute and holds the Ward Coleman Chair in Cancer Research. She has made influential contributions to human dendritic cell biology, specifically regarding isolation, biology, antigen presenting function and use as vaccine adjuvants in humans. Dr. Adusumilli, Deputy Chief of Thoracic Service at Memorial Sloan Kettering Cancer Center, brings his extensive experience with the investigation of the tumor immune microenvironment and the development of CAR T-cell-mediated immunotherapies.

On July 27, 2020 Agendia, Inc., a world leader in precision oncology for breast cancer, reported the publication of MINDACT trial data in the July 27 issue of Communications Biology, a Nature Publishers Journal (Press release, Agendia, JUL 27, 2020, View Source [SID1234562418]). In the largest data set seen to date, the study yielded a full transcriptome array for each tumor and demonstrated the successful removal of technical variation in gene expression profiles while retaining expected biological signals. The resulting dataset could serve as an invaluable tool to discover or test gene expression signatures in the Company’s quest to better elucidate the complexities of breast cancer.

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The paper, entitled, "Controlling technical variation amongst 6693 patient microarrays of the randomized MINDACT trial," is available online here.

"As part of our mission to bring patients and their doctors the data they need to make treatment decisions, we continue to conduct a number of studies and push research within our existing trials to ensure that our diagnostic tests, MammaPrint and BluePrint, offer reliable and actionable results," said Annuska Glas, PhD, Vice President of Research and Development at Agendia. "This is a major research and development milestone, achieved over 13 years, that will be used in future studies to help patients and physicians better understand breast cancer."

Gene expression data obtained in large studies are promising but in general are prone to technical variation, and removal of these variations is essential to avoid inaccurate conclusions. The publication provides a robust resource how for ensuring that initial variation affecting the full transcriptome expression data of a large dataset can be adjusted by removing over-time technical variations without losing biological signals for future analysis purposes.

The paper concluded that the analysis pipeline for this unprecedented achievement and sample size is entirely reproducible, and both adjusted and unadjusted breast cancer data sets are available and ready-to-use for translational research.

"We are happy to make this data and our process accessible for the community working to uncover every complexity of breast cancer," said Laura van’t Veer, PhD, Chief Research Officer and Co-Founder of Agendia.

The multi-faceted, real-world data library being built for the MINDACT trial underscores Agendia’s commitment to both long-term and immediately actionable research and dedication to consistently pushing the boundaries of previous findings to improve patient outcomes.

About the MINDACT Trial

MammaPrint, the 70-gene risk of recurrence assay for patients with early-stage breast cancer, is supported by level 1A clinical evidence from MINDACT, a landmark trial sponsored by the EORTC (EORTC-10041/BIG3-04). The study found that clinically high-risk patients with a MammaPrint Low Risk result could safely de-escalate treatment and forgo chemotherapy. Long-term follow-up data from MINDACT, presented at the 2020 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), confirmed and built upon the findings published in the New England Journal of Medicine in 2016.

The MINDACT trial enrolled 6,693 breast cancer patients. At the five-year follow-up mark, the trial showed that tumor analysis and reclassification by the MammaPrint 70-gene expression signature enabled a 46 percent reduction in the use of chemotherapy for clinically high-risk patients that were reclassified by the genetic assay to be genomically Low Risk. Nine-year follow-up data will be published in depth later this year, and confirm MINDACT as a positive de-escalation study for chemotherapy and continues to demonstrate MammaPrint’s clinical utility when determining a breast cancer patient’s need for chemotherapy.

On July 27, 2020 Foresee Pharmaceuticals Co., Ltd. (6576.TWO) ("Foresee"), reported that it has submitted to the U.S. Food and Drug Administration a 505(b)(2) New Drug Application for Camcevi 42mg (FP-001 LMIS 50mg), a ready-to-use 6-month depot formulation of leuprolide mesylate (Press release, Foresee Pharmaceuticals, JUL 27, 2020, View Source [SID1234562417]). The application seeks approval for the use of this product for the palliative treatment of advanced prostate cancer.

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This NDA submission is supported by a previously communicated successful Phase 3 study in 137 Advanced Prostate Carcinoma patients, where treatment with LMIS 50 mg injection every 6 months was demonstrated to be effective, safe and well tolerated.

"In the NDA submission in 2019, FDA suggested that additional device design verification data on the combination product be provided. Since then, with tireless efforts and commitment from the Foresee team and collaborators, as well as continued support of our investors, we have undertaken diligent preparation for the requested information," said Dr. Ben Chien, Founder and Chairman of Foresee. "We are confident in the resubmission of the NDA."

"Next step for the FP-001 franchise will be to establish a strong commercial partnership in the US and to prepare for the NDA submission of the 3-month depot of Camcevi. We look forward to the successful launch of the FP-001 franchise, providing patients with its differentiated ready-to-use profile." said Dr. Ben Chien.

On July 27, 2020 Medpace Holdings, Inc. (Nasdaq: MEDP) ("Medpace") reported financial results for the second quarter ended June 30, 2020 (Press release, Medpace, JUL 27, 2020, View Source [SID1234562416]).

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Second Quarter 2020 Financial Results

Revenue for the three months ended June 30, 2020 decreased 4.3% to $205.0 million, compared to $214.1 million for the comparable prior-year period. On a constant currency organic basis, revenue for the second quarter of 2020 decreased 4.1% compared to the second quarter of 2019.

Backlog as of June 30, 2020 grew 14.6% to $1.3 billion from $1.2 billion as of June 30, 2019. Net new business awards were $254.1 million, representing a net book-to-bill ratio of 1.24x for the second quarter of 2020, as compared to $279.2 million for the comparable prior-year period. The Company calculates the net book-to-bill ratio by dividing net new business awards by revenue.

For the second quarter of 2020, total direct costs were $148.4 million, compared to total direct costs of $150.3 million in the second quarter of 2019. Selling, general and administrative (SG&A) expenses were $21.9 million in the second quarter of 2020, compared to SG&A expenses of $23.6 million in the second quarter of 2019.

GAAP net income for the second quarter of 2020 was $24.1 million, or $0.64 per diluted share, versus GAAP net income of $27.5 million, or $0.73 per diluted share, for the second quarter of 2019. This resulted in a net income margin of 11.8% and 12.8% for the second quarter of 2020 and 2019, respectively.

EBITDA for the second quarter of 2020 decreased 12.9% to $35.0 million, or 17.1% of revenue, compared to $40.2 million, or 18.8% of revenue, for the comparable prior-year period. On a constant currency basis, EBITDA for the second quarter of 2020 decreased 15.0% from the second quarter of 2019.

A reconciliation of the Company’s non-GAAP financial measures, including EBITDA and EBITDA margin to the corresponding GAAP measures is provided below.

Balance Sheet and Liquidity

The Company’s Cash and cash equivalents were $160.9 million at June 30, 2020, and the Company generated $44.3 million in cash flow from operating activities during the second quarter of 2020. During the second quarter of 2020, the Company repurchased approximately 0.11 million shares at an average price of $68.65 per share for a total of $7.6 million. The Company had $49.2 million remaining under its authorized share repurchase program at the end of the quarter.

Financial Guidance

The Company forecasts 2020 revenue in the range of $880.0 million to $920.0 million, representing growth of 2.2% to 6.9% over 2019 revenue of $861.0 million. GAAP net income for full year 2020 is forecasted in the range of $136.0 million to $144.0 million. Additionally, full year 2020 EBITDA is expected in the range of $180.0 million to $190.0 million. Based on forecasted 2020 revenue of $880.0 million to $920.0 million and GAAP net income of $136.0 million to $144.0 million, diluted earnings per share (GAAP) is forecasted in the range of $3.62 to $3.83. This guidance assumes a full year 2020 tax rate of 15.0% to 16.0% and does not reflect the potential impact of any share repurchases the Company may make pursuant to the share repurchase program.

Conference Call Details

Medpace will host a conference call at 9:00 a.m. ET, Tuesday, July 28, 2020, to discuss its second quarter 2020 results.

To participate in the conference call, dial 800-219-7113 (domestic) or 574-990-1030 (international) using the passcode 7182886.

To access the conference call via webcast, visit the "Investors" section of Medpace’s website at medpace.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A supplemental slide presentation will also be available at the "Investors" section of Medpace’s website prior to the start of the call.

A recording of the call will be available at 12:00 p.m. ET on Tuesday, July 28, 2020 until 12:00 p.m. ET on Tuesday, August 11, 2020. To hear this recording, dial 855-859-2056 (domestic) or 404-537-3406 (international) using the passcode 7182886.