On June 11, 2020 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for DKN-01 for the treatment of gastric and gastroesophageal junction cancer (Press release, Leap Therapeutics, JUN 11, 2020, View Source [SID1234561000]). DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Orphan Drug Designation for DKN-01 in gastric and gastroesophageal junction cancer is another significant milestone in our DKN-01 development program and underscores the need for new treatment options for these indications," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "We believe DKN-01 has the potential to be an important new therapy for this patient population that remains an area of high unmet medical need."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides to Leap certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

DKN-01 is currently being evaluated in Phase 1/2 and Phase 2 clinical trials for gastroesophageal, gynecologic, hepatobiliary, and prostate cancers. Site initiation activities are now underway for the Company’s combination study of DKN-01 plus tislelizumab, BeiGene, Ltd.’s anti-PD-1 antibody, in patients with gastric or gastroesophageal junction cancer with dosing of the first patients expected in the third quarter of 2020.

About DKN-01

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling, a signaling pathway frequently implicated in tumorigenesis and suppressing the immune system. DKK1 has an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK ligands on tumor cells.

On June 11, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the European Patent Office has announced the intent to grant patent number EP3229810 (B1) titled "Phospholipid Ether Analogs as Cancer-Targeting Drug Vehicles (Press release, Cellectar Biosciences, JUN 11, 2020, View Source [SID1234560999])." The patent provides composition of matter and use protection for the company’s proprietary PLE, targeted delivery vehicle analogs in combination with a broad range of chemotherapeutics such as paclitaxel, gemcitabine, and other classes of small molecule chemotherapeutic agents. The cancer-targeting PLE delivery vehicle serves as the foundation for the company’s lead product candidate CLR 131, which continues to advance through clinical studies in both adult and pediatric cancer indications.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This patent represents a key step towards the expansion of our PDC franchise into the European and the rest of the world markets. It provides intellectual property protection for many of our ongoing preclinical programs, similar to the previously granted U.S. patent," stated James Caruso president and chief executive officer of Cellectar Biosciences. "We plan to explore other therapeutic modalities with the PLE and seek partnerships with pharmaceutical and biotechnology companies interested in utilizing our technology to potentially improve the delivery and the efficacy and tolerability of their drugs."

The combination of the PLE with a small molecule chemotherapeutic is known as a Phospholipid Drug Conjugate or PDC. PDC’s provide targeted delivery and release of therapeutic payloads inside tumor cells including primary and metastatic tumor sites as well as cancer stem cells and has also demonstrated the capacity to cross the blood-brain-barrier and target brain tumors.

About Phospholipid Drug Conjugates

Cellectar’s product candidates are built upon a patented delivery platform that utilizes optimized phospholipid ether-drug conjugates (PDCs) to target cancer cells. The PDC platform selectively delivers diverse oncologic payloads to cancerous cells and cancer stem cells, including hematologic cancers and solid tumors. This selective delivery allows the payloads’ concentration within tumor cells to be increased while reducing the concentration in normal tissue, which may enhance drug potency while reducing adverse events. This platform takes advantage of a metabolic pathway utilized by all tumor cell types. Compared with other targeted delivery platforms, the PDC platform’s mechanism of entry does not rely upon specific cell surface epitopes or antigens which can be modified or removed by tumor cells resulting in resistance to the treatment. In addition, PDCs can be conjugated to molecules in numerous ways, thereby increasing the types or classes of molecules that can be selectively delivered. Cellectar believes the PDC platform holds potential for the discovery and development of the next generation of cancer-targeting agents.

On June 11, 2020 BeyondSpring Inc. (the "Company" or "BeyondSpring") (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative cancer therapies, reported its financial results and provided an operational update for the three months ended March 31, 2020 (Press release, BeyondSpring Pharmaceuticals, JUN 11, 2020, View Source [SID1234560998]).

"During the first quarter, we continued to make progress in the two lead indications for Plinabulin for both the prevention of chemotherapy-induced neutropenia (CIN) and treatment of non-small cell lung cancer (NSCLC)," said Dr. Lan Huang, Co-Founder, Chairman and Chief Executive Officer. "Our recent data from PROTECTIVE-2 Phase 2 showed Plinabulin combined with G-CSF improves chemotherapy compliance compared to G-CSF alone, which potentially leads to better clinical outcomes. The Plinabulin-G-CSF combination’s potential to prevent infection and hospitalization becomes even more important to the physicians, patients and the healthcare system in the COVID-19 pandemic. We expect to reach the pre-specified interim analysis for PROTECTIVE-2 Phase 3 this month to evaluate superiority in CIN, which has the potential to mark the first significant enhancement in preventing neutropenia in 30 years."

"With over 1,200 patients enrolled to date for Plinabulin clinical programs, we believe we are well-positioned to capitalize on our upcoming regulatory milestones with multiple New Drug Application (NDA) filings followed by near term commercial opportunities. Looking ahead, we continue to advance our clinical studies to support our view of Plinabulin as a ‘pipeline in a drug’ and believe its potential in improving standard of care in CIN prevention and cancer treatments will help many patients in need globally."

Select First-Quarter 2020 and Recent Operational Highlights

Chemotherapy-Induced Neutropenia (CIN)

PROTECTIVE-2 Phase 2 for Chemotherapy-Induced Neutropenia Shows Positive Results in Chemotherapy Optimization with Potentially Better Clinical Outcomes

In June 2020, BeyondSpring announced that PROTECTIVE-2 Phase 2 superiority trial for CIN shows that Plinabulin in combination with Neulasta (pegfilgrastim), a long-lasting G-CSF, which is a predominant therapy to treat CIN, enables more cancer patients to receive the optimal chemotherapy dose and regimen, which potentially leads to better clinical outcomes.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In breast cancer patients treated with docetaxel, doxorubicin and cyclophosphamide (TAC, a high-risk chemotherapy) with 20mg/m2 of Plinabulin combined with 6mg of Neulasta (n=16) compared with 6mg of Neulasta alone (n=22), Plinabulin + G-CSF improved compliance with targeted chemotherapy

Dose reduction (over 15 percent): only 6.3 percent of patients in the Plinabulin-Neulasta combination arm versus 22.7 percent in Neulasta arm – a 72 percent improvement

Downgraded regimen (from TAC, to docetaxel and cyclophosphamide, or TC): No (0 percent) patients in the Plinabulin + G-CSF arm downgraded chemotherapy from the TAC regimen to the TC regimen versus 18.2 percent in the Neulasta arm – p < 0.05

Plinabulin’s Mechanism of Action Complements Neulasta in Cancer Treatment

In May 2020, two Company abstracts were presented at this year’s American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program, evaluating Plinabulin alongside Neulasta.

BeyondSpring’s e-publication, titled, "Comparison of CD34+ mobilization effects of standard dose pegfilgrastim (Peg) versus low-dose peg combined with plinabulin (Plin)," demonstrates the efficacy of Plinabulin-Neulasta combination in increasing CD34+ counts for patients, with fewer adverse events

Additionally, BeyondSpring’s poster presentation, titled, "Head-to-head comparison of the non-G-CSF small molecule single agent (SA) plinabulin with SA pegfilgrastim for the prevention of docetaxel chemotherapy (chemo)-induced neutropenia (CIN) in the protective-1 trial," compares Plinabulin versus Neulasta as an effective monotherapy for CIN prevention

Non-Small Cell Lung Cancer (NSCLC)

DSMB Recommends DUBLIN-3 Phase 3 NSCLC to Continue Without Modification

In June 2020, BeyondSpring reported it had reached the pre-specified second interim analysis for DUBLIN-3 for NSCLC treatment with Plinabulin.

Upon reviewing the efficacy and safety data of over 500 patients at an approximately 300-patient death event, DSMB advised BeyondSpring to continue the study without any modifications

DUBLIN-3 is a global Phase 3 trial for Plinabulin, in combination with docetaxel versus docetaxel alone, for the treatment of second- / third-line EGFR wild-type NSCLC

Thus far, over 600 cancer patients have been dosed with Plinabulin, which has demonstrated good tolerability and satisfies the safety database standard of both the U.S. Food and Drug Administration (FDA) and China’s National Medical Products Administration (NMPA)

Intellectual Properties

BeyondSpring Granted U.S. Patent for Plinabulin to Treat Severe CIN from Taxane in Cancer Patients

In May 2020, the U.S. Patent and Trademark Office (USPTO) granted BeyondSpring a new patent for methods of treating severe CIN in cancer patients treated with taxane with protection through 2033. This patent establishes Plinabulin’s beneficial effects in reducing CIN associated with taxane, one of the most commonly used chemotherapies

The Company currently owns 76 patents, including 17 issued U.S. patents, for Plinabulin and its analogs with protection through 2036

Financial Results for the Three Months Ended March 31, 2020

Research and development ("R&D") expenses were $13.7 million for the quarter ended March 31, 2020, compared to $6.3 million for the quarter ended March 31, 2019. The $7.4 million increase was largely attributable to an increase of $4.4 million in clinical trial expenses and an increase of $3.0 million in non-cash share-based compensation.

Selling, general and administrative ("SG&A") expenses were $2.9 million for the quarter ended March 31, 2020, compared to $1.6 million for the quarter ended March 31, 2019. The $1.3 million increase was mainly due to a $0.6 million increase in commercial and marketing expense, a $0.3 million increase in salary, wages and benefits expense, and a $0.4 million increase in other expenses.

Net loss attributable to the Company was $16.1 million for the quarter ended March 31, 2020, compared to $7.3 million for the quarter ended March 31, 2019.

As of March 31, 2020, the Company had a cash balance of $24.9 million. The Company believes currently available financial resources will be sufficient to support its clinical trials and submit NDAs in the U.S. and China for Plinabulin for the CIN and NSCLC indications, as well as to advance its immuno-oncology pipeline and ubiquitination protein degradation research platform.

Anticipated Milestones

The following outlines the Company’s anticipated upcoming milestones and projected timelines:

Interim topline data readout for PROTECTIVE-2 Phase 3 for CIN – June 2020

Final data readout for PROTECTIVE-2 Phase 3 for CIN – H2 2020

Final data readout for PROTECTIVE-1 Phase 3 for CIN – H2 2020

NDA submission for Plinabulin for CIN in the U.S. – H2 2020

Final data readout for DUBLIN-3 for NSCLC – H2 2020

NDA submission for Plinabulin for NSCLC in China – H2 2020

NDA submission for Plinabulin for NSCLC in the U.S. – H1 2021

On June 11, 2020 The Board of Directors of Bristol Myers Squibb (NYSE:BMY) reported a quarterly dividend of forty-five cents ($0.45) per share on the $.10 par value Common Stock of the corporation (Press release, Bristol-Myers Squibb, JUN 11, 2020, View Source [SID1234560997]). The next quarterly dividend will be payable on August 3, 2020, to stockholders of record at the close of business on July 6, 2020.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The directors also declared a quarterly dividend of fifty cents ($0.50) per share on the $2.00 Convertible Preferred Stock of the corporation, payable September 1, 2020 to stockholders of record at the close of business on August 4, 2020.

On June 11, 2020 Immunomic Therapeutics, Inc. reported that it will present preclinical data on its investigational nucleic acid platform, UNITE (UNiversal Intracellular Targeted Expression), that elicits potent immune responses when used with its investigational UNITE vaccine, ITI-3000, in mice (Press release, Immunomic Therapeutics, JUN 11, 2020, View Source [SID1234560996]). UNITE fuses a tumor-associated antigen, here a mutated form of the large T antigen (LT) of Merkel cell polyomavirus (MCPγV), with lysosomal-associated membrane protein 1 (LAMP1). This lysosomal targeting technology results in enhanced antigen presentation and a balanced T cell response, as ITI-3000 activated antigen-specific CD4+ T cells in vivo. The data will be presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting II, June 22-24, 2020.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"ITI-3000 is an innovative and novel targeted approach to a MCPγV vaccine," says Teri Heiland, Ph.D., Immunomic’s Chief Scientific Officer. "We are highly encouraged by this data and look forward to continuing to advance ITI-3000 toward the clinic."

The majority of Merkel cell carcinomas (MCC) are associated with Merkel cell polyomavirus (MCPyV) infection, making LT an attractive target for therapeutic cancer vaccines. MCPγV integrates into the host genome, resulting in expression of a truncated form of the viral LT in infected cells. While induction of tumor-reactive CD8+ T cells is a major goal of cancer therapy, CD4+ T cells provide essential support to CD8+ T cells by promoting their expression of cytotoxic effector molecules and increasing their migratory capacity. Cytokines secreted by CD4+ T cells, such as IFNγ, can also exert desirable effects on the tumor microenvironment. Hence, a cancer vaccine that promotes potent, antigen-specific CD4+ T cell responses to MCPγV-LT may drive robust anti-tumor immune responses.

Poster Title: LAMP1 targeting of the large T antigen of Merkel cell polyomavirus elicits potent CD4+ T cell responses and prevents tumor growth

Session Category: Immunology

Session Type: Poster Session

Session Title: Vaccines

Poster Number: 4585

Poster Session Date and Time: June 22, 2020 9AM-6PM

Location: Virtual Poster Presentation

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.