On January 12, 2021 OX2 Therapeutics, Inc., a privately held Minneapolis company, reported that they treated their first patient in a phase one human trial of a new treatment developed to combat recurrent high-grade brain tumors (Press release, OX2 Therapeutics, JAN 12, 2021, View Source [SID1234573915]).

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"This is a first-of-its kind immunotherapy that works to treat one of the most aggressive and deadly cancers today," said Christopher Moertel, MD, OX2 Therapeutics, Inc. "Central nervous system cancers are the number one cause of cancer related mortality in children, and a major cause of morbidity and mortality in adults."

OX2 Therapeutics developed the first immune checkpoint peptide platform targeting the immune system to attack solid tumors. Focusing on high-grade gliomas, OX2 Therapeutics has been treating dogs diagnosed with spontaneous high-grade glioma in a canine clinical trial at the University of Minnesota, Veterinary hospital. "This is the first therapy Dr. Olin and I have used to significantly extend the life of dogs with high-grade gliomas with no adverse events," said G. Elisabeth Pluhar, DVM, PhD, director of canine brain tumor program.

The OX2 peptide, known as CD200AR-L, is a single peptide that has the potential to replace the toxic antibody therapies that are currently used to block immune checkpoints," stated Michael Olin, PhD, OX2 Therapeutics, Inc. "CD200AR-L provides a one-two punch in the fight against cancer through the activation of the immune system while simultaneously protecting it against tumor induced suppression allowing the immune system to both reach the cancer cells and then fight them."

The FDA approved the treatment for an adult phase one human trial in June 2020. OX2 Therapeutics treated their 1st patient January 6th in a phase one single center, open-label, dose-escalation clinical trial for recurrent glioblastoma under the direction of Elizabeth Neil, MD at the University of Minnesota. This will be followed by a pediatric trial for recurrent malignant brain tumors based on its safety and pharmacokinetic profile led by Emily Greengard, MD.

The phase one human trial of this potentially groundbreaking treatment is an important milestone for OX2 Therapeutics and the Brain Tumor Program at the Masonic Cancer Center, University of Minnesota.

On January 12, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development, and commercialization of drugs for the treatment of cancer, reported the initiation of a pivotal trial for CLR 131 in Waldenstrom’s macroglobulinemia (WM) (Press release, Cellectar Biosciences, JAN 12, 2021, View Source [SID1234573911]).

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The pivotal trial is designed as a global, non-comparator, single arm, expansion cohort of the currently ongoing Phase 2 CLOVER-1 study of CLR 131. This design is in alignment with the feedback received from the U.S. Food and Drug Administration (FDA) during the guidance meeting held in September 2020.

The study will enroll 50 WM patients who have failed first-line therapy and have failed to respond to, or have progressed while on treatment with a BTK inhibitor (i.e. ibrutinib). Patients in the trial will receive up to 4 doses of CLR 131 over 2 cycles (cycle one days 1, 15, and cycle two days 57, 71). The primary endpoint of the trial is response rate as defined as a partial response (a minimum of a 50% reduction in the biological marker IgM) or better in patients that receive a minimum total body dose of 60 mCi with secondary endpoints of treatment free survival, duration of response and progression free survival. An independent data monitoring committee (iDMC) will perform an interim safety and futility evaluation on the first 10 patients enrolled. The assessment will occur patient by patient and will conclude after the tenth patient is evaluated; there is no planned study stoppage. The trial has been initiated at select US cancer centers and will roll out to additional US and international sites in early 2021.

"CLR 131 has the potential to be an important therapeutic option for patients with Waldenstrom’s macroglobulinemia, an indication with limited treatment alternatives. The 100% overall response rate achieved to date at comparable doses bodes well for CLR 131 to deliver meaningful outcomes for patients," said Jim Caruso, president and CEO of Cellectar. "The company now possesses an accelerated route to commercialization with Fast Track and Orphan Drug designations further facilitating a clear regulatory pathway and a balance sheet to support development through NDA approval."

About Waldenstrom’s macroglobulinemia

Waldenstrom’s macroglobulinemia (WM) is a rare and incurable disease defined by specific genotypic subtypes that defines patient responses and long-term outcomes. The annual incidence is 6,500 with prevalence of approximately 60,000 patients globally. WM is a lymphoma, or cancer of the lymphatic system. The disease occurs in a type of white blood cell called a B-lymphocyte or B-cell, which normally matures into a plasma cell whose job is to manufacture immunoglobulins (antibodies) to help the body fight infection. In WM, there is a malignant change to the B-cell in the late stages of maturing, and it continues to proliferate into a clone of identical cells, primarily in the bone marrow but also in the lymph nodes and other tissues and organs of the lymphatic system. These clonal cells over-produce an antibody of a specific class called IgM.

WM cells have characteristics of both cancerous B-lymphocytes (NHL) and plasma cells (multiple myeloma), and they are called lymphoplasmacytic cells. For that reason, WM is classified as a type of non-Hodgkin’s lymphoma called lymphoplasmacytic lymphoma (LPL). About 95% of LPL cases are WM; the remaining 5% do not secrete IgM and consequently are not classified as WM.

There is no standard treatment for WM. Several drugs have demonstrated activity either alone or in combinations but only a single drug has received regulatory approval. Treatment is mainly focused on the control of symptoms and the prevention of organ damage. Front-line treatments for WM include rituximab alone or in combination with other agents. In the salvage therapy (second line or later) setting, ibrutinib, combinations of proteosome inhibitors and immunomodulatory drugs and stem cell transplantation are considered. Ibrutinib is the only drug to receive regulatory approval (2015) as a salvage therapy; in late 2019, it was approved for front-line treatment in combination with rituximab. Factors such as long-term cytopenias, age, hyper viscosity, the need for quick disease control, lymphadenopathy, co-morbidities, and IgM-related end-organ damage are key consideration in the choice of treatment.

About CLR 131

CLR 131 is a small-molecule Phospholipid Drug Conjugate designed to provide targeted delivery of iodine-131 (radioisotope) directly to cancer cells, while limiting exposure to healthy cells unlike many traditional on-market treatment options. The company’s lead PDC therapeutic, CLR 131, is currently in two clinical studies. The CLOVER-1 Phase 2 study in hematologic malignancies and the Phase 1 pediatric safety study. The CLOVER-1 study met the primary efficacy endpoints from the Part A dose-exploration portions conducted in r/r B-cell malignancies and remains under further evaluation in highly refractory multiple myeloma patients. A global, pivotal expansion cohort was launched in December 2020 in BTK inhibitor failed or suboptimal response Waldenstrom’s macroglobulinemia (WM) patients. The WM cohort will enroll up to 50 patients to evaluate the efficacy and safety of CLR 131 for marketing approval.

The U.S. Food and Drug Administration (FDA) granted CLR 131 Fast Track Designation and Orphan Drug Designation (ODD) for relapsed/refractory Waldenstrom’s macroglobulinemia, multiple myeloma and diffuse large B-cell lymphoma. Rare Pediatric Disease Designations and ODDs were granted for the treatment of, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The European Commission granted an ODD for r/r multiple myeloma.

On January 12, 2021 Acorda Therapeutics, Inc. (NASDAQ: ACOR) announced that Ron Cohen, M.D., President and Chief Executive Officer, will present at the 39th Annual J.P. Morgan Health Care Conference on Thursday, January 14, at 5:20 PM ET (Press release, Acorda Therapeutics, JAN 12, 2021, View Source [SID1234573910]). A live audio webcast of the presentation can be accessed under "Investor Events" in the Investor section of the Acorda website at www.acorda.com. An archived version of the webcast will be available following the presentation.

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On January 12, 2021 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) and Incyte (NASDAQ: INCY) reported that Health Canada has accepted the New Drug Submission (NDS) for tafasitamab, an anti-CD19 antibody (Press release, MorphoSys, JAN 12, 2021, View Source [SID1234573909]). The application seeks approval of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from low grade lymphoma, who are not eligible for, or refuse, autologous stem cell transplant (ASCT).

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"With the acceptance of the NDS by Health Canada, review of the data can begin, an important step on the path to making tafasitamab available in Canada for use in combination with lenalidomide in eligible patients with relapsed or refractory DLBCL," said Josée Brisebois, Ph.D., Head of Medical Affairs, Incyte Biosciences Canada. "We intend to work closely with Health Canada as we seek to bring this innovative targeted therapeutic option to the clinical community and to appropriate patients for whom few treatment options exist."

"This important milestone moves tafasitamab in combination with lenalidomide into the regulatory review process in Canada, with the potential to significantly advance patient care in the treatment of relapsed or refractory DLBCL," said Nuwan Kurukulasuriya, Ph.D., Senior Vice President Global Medical Affairs, MorphoSys.

The NDS, submitted by Incyte, is based on data from the L-MIND study evaluating tafasitamab in combination with lenalidomide as a treatment for patients with relapsed or refractory DLBCL not eligible for autologous stem cell transplant, and is supported by the RE-MIND study, an observational retrospective study in relapsed or refractory DLBCL.

Incyte has exclusive commercialization rights for tafasitamab outside of the United States and, if approved, Incyte will hold the marketing authorization for tafasitamab in Canada. This NDS marks the second marketing application that Incyte Biosciences Canada has made to Health Canada since establishing operations in Canada in April 2020.

About Diffuse Large B-cell Lymphoma (DLBCL)
DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide1, characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about 40% of patients not responding to initial therapy or relapsing thereafter2, leading to a high medical need for new, effective therapies3, especially for patients who are not eligible for an autologous stem cell transplant in this setting.

About L-MIND
The L-MIND trial is a single arm, open-label, multicenter Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have had at least one, but no more than three prior lines of therapy, including an anti-CD20 targeting therapy (e.g. rituximab), who are not eligible for high-dose chemotherapy or refuse subsequent autologous stem cell transplant. The study’s primary endpoint is Overall Response Rate (ORR). Secondary outcome measures include Duration of Response (DoR), Progression-Free Survival (PFS) and Overall Survival (OS). In May 2019, the study reached its primary completion.

For more information about L-MIND, visit View Source

About RE-MIND
RE-MIND, an observational retrospective study (NCT04150328), was designed to isolate the contribution of tafasitamab in combination with lenalidomide and to prove the combinatorial effect. The study compares real-world response data of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who received lenalidomide monotherapy with the efficacy outcomes of the tafasitamab-lenalidomide combination, as investigated in MorphoSys’ L-MIND trial. RE-MIND collected the efficacy data from 490 relapsed or refractory DLBCL patients in the U.S. and EU. Qualification criteria for matching patients of both studies were pre-specified. As a result, 76 eligible RE-MIND patients were identified and matched 1:1 to 76 of 80 L-MIND patients based on important baseline characteristics. Objective Response Rates (ORR) were validated based on this subset of 76 patients in RE-MIND and L-MIND, respectively. The primary endpoint of RE-MIND was met and shows a statistically significant superior best ORR of the tafasitamab-lenalidomide combination compared to lenalidomide monotherapy.

For more information about RE-MIND, visit View Source

About Tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP). In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Following approval by the U.S. Food and Drug Administration in July 2020, tafasitamab is being co-commercialized by MorphoSys and Incyte in the United States. Incyte has exclusive commercialization rights outside the United States.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

XmAb(R) is a registered trademark of Xencor, Inc.

The safety and efficacy of tafasitamab is under review and the market authorization in Canada has not yet been obtained.

On January 12, 2021 Primmune Therapeutics reported the close of its Series A financing round with the addition of $4.0 million from Bioqube Ventures, a European life sciences venture capital firm (Press release, Primmune Therapeutics, JAN 12, 2021, View Source [SID1234573908]). This brings the total of the Series A financing raise to $31.4 million. These funds will be used to support the development of PRTX007, a novel orally-administered, small molecule toll-like receptor 7 (TLR7) agonist as a therapeutic-adjuvant for acute viral diseases and cancer.

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Concurrently, Debbie Dumont, Co-founder and Managing Partner at Bioqube Ventures has joined Primmune’s Board of Directors as an observer. Elina Zuniga, Ph.D., Professor of Molecular Biology at the University of California, San Diego has joined the company’s scientific advisory board.

"We are excited to have Bioqube Ventures join our investor syndicate because of their expertise and experience in establishing European operations. Bioqube Ventures will facilitate the establishment of our Belgium subsidiary to complement our Australian presence and enable us to effectively partner and run clinical studies in the European Union as well as in the United States and Australia," said Charlie McDermott, Chairman and Chief Executive Officer of Primmune Therapeutics. "Dr. Elina Zuniga will be important in guiding our TLR7 agonist strategy as part of our scientific advisory board given her deep knowledge regarding the interplay between toll-like receptor signaling, endogenous poly-interferon antiviral responses, and host innate immune modulation."