On February 4, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported the launch of an Expanded Access Program (EAP) in the USA for tafasitamab, an anti-CD19 antibody currently under development (Press release, MorphoSys, FEB 4, 2020, View Source [SID1234553831]). Under certain conditions, patients with relapsed or refractory diffuse large B-cell lymphoma (r / r DLBCL) can have access to tafasitamab in combination with lenalidomide via the EAP. Tafasitamab is an active ingredient in development (investigational product), the safety and efficacy of which have not been established.

According to the FDA, programs for expanded access , sometimes referred to as "compassionate use", offer patients an opportunity to gain access to an investigational medication for the treatment of a serious illness. They are often provided when there are no comparable or satisfactory alternative therapies to treat the disease, when the patient cannot be enrolled in a clinical trial, when the potential benefit to the patient justifies the potential treatment risk, and when it is made available of the investigational medicinal product does not affect studies that could support the marketing authorization of the drug for the corresponding treatment indication.

To be eligible for the Tafasitamab EAP, r / r DLBCL patients must meet the EAP inclusion / exclusion criteria that correspond to those in the MorphoSys L-MIND study. Treatment of DLBCL patients under the EAP is recommended with tamasitamab in combination with lenalidomide according to the L-MIND treatment plan. The EAP will be available for a limited time while the FDA is reviewing MorphoSys’s application for a Biologics License Application (BLA) for Tamasitamab. Applications for inclusion in the Tafasitamab EAP must be made by a medical doctor approved in the United States.

The Tafasitamab EAP is from Clinigen Healthcare Ltd. managed. Questions or inquiries regarding the Tafasitamab EAP should be directed to [email protected] .

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About tafasitamab
Tafasitamab is a humanized Fc-modified monoclonal antibody against CD19. In 2010, MorphoSys licensed Xencor, Inc.’s worldwide development and marketing rights for tafasitamab. Tafasitamab has an Fc part modified with the XmAb (R) technology, which is said to lead to a significant increase in antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), and thus to improve a key mechanism of tumor cell killing. MorphoSys is investigating tafasitamab as a therapeutic option for malignant B-cell disorders in a number of ongoing combination studies. An open phase 2 combination study (L-MIND study) examined the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed / refractory DLBCL who were not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) Question come. Based on preliminary data from L-MIND, the FDA granted therapeutic breakthrough status for tafasitamab plus lenalidomide in this patient population in October 2017.

On February 4, 2020 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage biopharmaceutical company pioneering a novel class of immunotherapies, reported that clinical translational data supporting the mechanism of action of its lead compound, DPX-Survivac, will be presented during the 2020 ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium, being held on February 6 – 8, 2020 in Orlando, FL (Press release, IMV, FEB 4, 2020, View Source [SID1234553830]).

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"These translational data continue to validate the mechanism of action of our lead program in advanced ovarian cancer," said Frederic Ors, President and Chief Executive Officer at IMV. "We continue to believe DPX-Survivac may offer significant clinical utility and a potentially meaningful treatment option for patients in this setting, as well as in other hard-to-treat indications in which survivin is highly expressed. We look forward to reporting topline results from our Phase 1b/2 study evaluating DPX-Survivac in advanced ovarian cancer, in the first quarter of 2020."

As part of this analysis, the Company measured systemic immune responses, tumor immune infiltrates and clinical tumor response from pre- and post-treatment patient samples in connection with three Phase 1 and/or Phase 2 clinical studies, each evaluating DPX-Survivac alone or in a combination regimen in patients with platinum sensitive or resistant, advanced ovarian cancer. Highlights from these translational data include:

DPX-Survivac generated survivin-specific T cells in the blood of 80% of patients sampled

Clinical anti-tumor responses were correlated with increased infiltration of T cells into tumors following treatment with DPX-Survivac

DPX-Survivac induced enrichment in T cell, cytotoxic lymphocytes and B cell-specific signatures which correlate with clinical response

Antigen-specific T cells retained their functionality throughout the duration of treatment

DPX-Survivac is currently being evaluated in three Phase 2 studies in advanced ovarian cancer, relapsed/refractory diffuse large B-cell lymphoma and a basket trial of five solid tumors, all of which are expected to report topline results in the first half of 2020.

Poster Presentation Details:

Poster Title: DPX-Survivac, a novel T cell immunotherapy, induces robust T cell responses in advanced ovarian cancer with significant anti-tumor efficacy Presenter: Oliver Dorigo, M.D., Ph.D., Associate Professor of Obstetrics and Gynecology (Oncology), Stanford University Medical Center

Abstract Number: 6 – Poster Session A

Date and Time: Poster will be displayed all day on February 6, 2020

ASCO-SITC has published the official abstracts on its meeting website in advance of the Clinical Immuno-Oncology Symposium on February 3rd, 2020 at 5:00PM EST.

The final conference poster presentation will include additional data collected between the abstract submission on October 15, 2019 and the presentation itself. The poster will be available under Events, Webcasts and Presentations in the investors section of IMV’s website on the day of presentation.

About DPX-Survivac

DPX-Survivac is the lead candidate in IMV’s new class of targeted immunotherapies designed to elicit antigen-specific functional, robust and sustained de novo T cell response. IMV believes this mechanism of action (MOA) is key to generating durable solid tumor regressions. DPX-Survivac consists of five unique HLA-restricted survivin peptides formulated in IMV’s proprietary DPX drug delivery platform and known to induce a cytotoxic CD8+ T cell response against survivin expressing cancer cells.

Survivin, recognized by the National Cancer Institute (NCI) as a promising tumor-associated antigen, is broadly over-expressed in most cancer types and plays an essential role in antagonizing cell death, supporting tumor-associated angiogenesis and promoting resistance to chemotherapies. IMV has identified over 20 cancer indications in which survivin can be targeted by DPX-Survivac.

DPX-Survivac has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as maintenance therapy in advanced ovarian cancer, as well as orphan drug designation status from the U.S. FDA and the European Medicines Agency (EMA) in the ovarian cancer indication.

On January 4, 2020 The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) reported that it has helped more than 30,000 patients gain access to vital medications valued at more than $500 million, and a new program expansion will further increase access to vital cancer therapies for patients with the greatest financial need (Press release, The Ohio State University, FEB 4, 2020, View Source [SID1234553829]).

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"Financial toxicity is a very real concern for families facing a cancer diagnosis. As an institution, we want to do all that we can to reduce additional stressors for patients so they can focus on getting well," explains Julie Kennerly-Shah, PharmD, a pharmacist and associate director of pharmacy at the OSUCCC – James. "Our financial counselors work with patients so they don’t have to make decisions about whether they can afford potentially life-saving treatment."

In February 2019 the pharmacy assistance program expanded with the implementation of a drug repository program that allows patients to donate no-longer-needed oral cancer therapy drugs for the benefit of other cancer patients through new state rules spearheaded by the State of Ohio Board of Pharmacy and OSUCCC – James.

"In cancer, it is quite common for patients to switch to a new medication or experience a medication dose reduction. As a result, we end up with a lot of wasted medication that must be disposed of," explains Kennerly-Shah. "This new program allows patients to donate these unneeded medications for re-dispensing to patients in financial need through our existing hospital-based Medical Assistance Program."

New rules adopted in November 2019 by the State of Ohio Board of Pharmacy states donated medications must be within expiration dates, stored as prescribed and otherwise untampered with. Pharmacists will then go through an eight-point inspection of the drug to ensure that it is safe to re-dispense at a future date to patients in need. Previous rules allowed only for the collection of unopened medication that was dispensed for the prescribed patient but never picked up.

The cancer drug repository initiative is a new component of the overall Medical Assistance Program (MAP) at the OSUCCC – James. The MAP consult service was established to help patients who are unable to afford their medications due to financial hardship. The program consists of pharmacists, medical assistance program coordinators, clinical financial case managers and other support personnel who work one-on-one with patients to reduce healthcare costs associated with cancer treatment.

"Ohio State has been a leader in medication-assistance programs and has taught many people across the country how to optimize various manufacturer programs and grant programming. We want to be an asset to other hospitals considering implementation of a cancer drug repository as well," adds Kennerly-Shah. "Our hope is that our repository is a first step toward a much bigger solution long-term that could be modeled beyond our individual hospital and state."

The OSUCCC – James is the first hospital in Ohio, and among the first in the United States, to launch a cancer drug repository program. The new cancer drug repository program is housed at the OSUCCC – James Outpatient Pharmacy. The program will accept donations of unused medications from individual patients, pharmacies, hospitals and non-profit clinics to be re-dispensed to patients in treatment at the OSUCCC – James who cannot afford the cost of the medications. Patients interested in donating non-expired, no-longer-needed capecitabine or temozolomide should contact the OSUCCC – James Outpatient Pharmacy.

On February 4, 2020 Navrogen, Inc., a biopharmaceutical company specialized in developing therapies for cancer and immune-related disorders, and Levena Biopharma, a company specialized in developing antibody drug conjugates (ADCs), reported the expanded collaboration to develop ADCs targeting humoral immuno-suppressed cancers (Press release, Navrogen, FEB 4, 2020, View Source [SID1234553828]). ADCs developed under this collaboration combine Levena’s linker and cytotoxic payload chemistry expertise along with Navrogen’s cancer-targeting antibodies discovered using its proprietary Humoral Immuno Oncology (HIO) platform technologies.

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Navrogen’s antibodies are able to specifically target and combat HIO-suppressed cancers that produce factors that dampen antibody-mediated immune-effector activity as well as decrease ADC internalization and subsequent release of their cytotoxic payloads which is required for killing. This collaboration will also include the use of Levena’s process development and GMP capabilities.

"Our collaboration with the exceptional scientists at Levena has enabled us to create a number of ADCs to specifically treat HIO-suppressed cancers," stated Luigi Grasso, Ph.D., Chief Scientific Officer at Navrogen. "The expansion of this collaboration will enable us to proceed with their advancement towards clinical trials."

Tong Zhu, Ph.D., Executive Director, Chemistry at Levena commented, "we have employed several of our technologies with the Navrogen team over the past year to identify HIO-refractory ADC formats. We look forward to continue supporting their development plans that can benefit from our expertise and technologies."

On February 4, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported several achievements for its GMI-1359 development program, including the issuance of a new patent and key designations granted by the U.S. Food and Drug Administration (FDA) that may provide future development support and marketing protections (Press release, GlycoMimetics, FEB 4, 2020, View Source [SID1234553827]). GMI-1359 is the Company’s novel drug candidate designed to simultaneously inhibit both E-selectin and CXCR4, two adhesion molecules involved in tumor trafficking and metastatic spread. Duke University investigators recently dosed the first patient in a proof-of-concept Phase 1b study to evaluate GMI-1359 drug candidate in patients with advanced breast cancer with bone metastases.

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New Patent

GlycoMimetics today said that the United States Patent and Trademark Office has issued a patent for GMI-1359, covering composition of matter as well as pharmaceutical formulations, and will provide protection through 2035, excluding any patent term adjustments or extensions.

"The new patent for GMI-1359 will help protect the composition of matter of this innovative approach in oncology. GMI-1359 may have a role in treating rare pediatric cancers, such as osteosarcoma, as well as breast cancer and other solid tumors that metastasize to bone," stated Rachel King, Chief Executive Officer of GlycoMimetics. "This intellectual property, as well as that previously granted in Europe, will play a key role as the company advances the drug candidate, especially with the new orphan and rare pediatric disease designations granted by the FDA."

New FDA Designations

In parallel, GlycoMimetics reported that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GMI-1359 for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States. These designations will aid in the development of this drug candidate, including making it eligible for the FDA’s Pediatric Priority Review Voucher.

"It’s encouraging for us as well as for patients and providers that the FDA recognizes the urgent need for new, more effective treatments for this devastating pediatric disease," stated Ms. King.

In addition to its clinical work in breast cancer, GlycoMimetics has conducted preclinical studies that have demonstrated strong support for the potential use of GMI-1359 in osteosarcoma. At the 2018 American Association of Cancer Research Annual Meeting, GlycoMimetics presented data establishing the biologic rationale for the use of a dual e-selectin/CXCR-4 inhibitor in pediatric and young adult patients with osteosarcoma. In that study, GMI-1359 was shown to inhibit tumor progression in an orthopedic model of osteosarcoma as well as inhibit the development of pulmonary metastases from primary osteosarcoma lesions. (View Source)

About Orphan Drug Designation

The FDA Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.

About Rare Pediatric Disease Designation

The FDA defines a "rare pediatric disease" as a serious or life-threatening rare disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the FDA’sRare Pediatric Disease Priority Review Voucher program, a sponsor who receives an initial approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

About Osteosarcoma

Osteosarcoma is a rare cancer of the bone that usually affects the large bones of the arm or leg, often growing quickly and spreading to other parts of the body. It occurs most often in children and young adults between the ages of 10 and 30. Each year, about 800 to 900 new cases of osteosarcoma are diagnosed in the United States. For more information, please see the osteosarcoma fact sheets at the National Cancer Institute and the American Cancer Society.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as acute myeloid leukemia and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. The newly initiated Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective dose of the drug candidate and to generate initial biomarker data around the drug’s activity.