Inovio Presents Cancer Killing Data of Its Transformative DNA-Encoded Bi-specific T Cell Engagers (dBiTEs) at AACR

On April 2, 2019 Inovio Pharmaceuticals, Inc. (NASDAQ: INO) reported the company’s novel DNA-Encoded Bi-specific T Cell Engagers (dBiTEs) generated potent anti-tumor activities in a preclinical study (Press release, Inovio, APR 2, 2019, View Source [SID1234534896]). Results were presented as a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta. For this study, Inovio, with its collaborators at The Wistar Institute, developed a novel dBiTE targeting the HER2 molecule which was tested in therapeutic models for the treatment of ovarian and breast cancers. Importantly, just a single dose of Inovio’s HER2 dBiTE resulted in high levels of corresponding BiTE in mice for four months, far exceeding what is typically displayed with conventional BiTE’s short half-life of only a few hours. The HER2 dBiTE effectively generated T cell cytotoxicity against HER2-expressing tumor cells resulting in a near-complete tumor clearance. Also presented was Inovio’s CD19 dBiTE which can kill B cell cancers by targeting B cell specific marker CD19.

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Dr. J. Joseph Kim, Inovio’s President and CEO, said, "In layman’s terms, dBiTEs are like a double-sided tape that binds to a tumor and to a cancer-killing T cell. By allowing the products be expressed directly and efficiently in the patient, our dBiTEs could finally fulfill the therapeutic promise of BiTEs. Based on these promising preclinical results, we are rapidly preparing for the clinical development of our dBiTE candidates, as well as constructing more cancer tumor targeting dBiTE candidates using our transformative dBiTE platform."

Dr. Kim added, "Leveraging Inovio’s in vivo synthetic nucleic expression platform, we have shown that just one dose of Inovio’s dBiTE could generate corresponding BiTEs at high levels in mice for several months, demonstrating a dramatic advantage over conventional BiTEs. Our CD19 dBiTE has the potential to treat multiple B cell cancers and to compete favorably with CD19 CAR-T products with potentially improved tolerability and safety profiles. Similarly, the HER2 dBiTE could be used to treat multiple solid tumors which express HER2 such as breast, ovarian, and gastric cancers."

BiTEs are a class of artificial bi-specific monoclonal antibodies that has the potential to transform the immunotherapy landscape for cancer. They direct a host’s immune system, more specifically the T cells’ cytotoxic activity, against cancer cells. BiTEs have two binding domains. One domain binds to the targeted tumor (like HER2 or CD19 expressing cells) while the other engages the immune system by binding directly to CD3 molecules on T cells. This double-binding activity drives T cell activation directly at the tumor resulting in a killing function and tumor destruction.

The biggest drawback of conventional protein-based BiTEs is the delivery and expression. The BiTEs have a half-life of only about two hours, which requires patients to undergo continuous intravenous infusion for several weeks to maintain therapeutic levels, making treatment adherence more difficult and resulting in high levels of infusion-associated adverse events. In addition, just like other traditional monoclonal antibodies, conventional BiTEs are also manufactured in bioreactors, typically requiring costly large-scale manufacturing facility development and laborious production as well as having to deal with improper product folding and stability. They must also be kept and distributed frozen at all times. These difficulties collectively have limited the development and commercialization of conventional BiTEs as only one licensed product is currently on the market (BLINCYTO (blinatumomab)).

Inovio’s dBiTE is a new transformative application of Inovio’s dMAb platform. The dBiTEs share many advantages of Inovio’s dMAbs as they both are composed of engineered DNA sequences which encode two antibody fragments. When administered by Inovio’s CELLECTRA delivery device, the patient’s own cells become the factory to manufacture functional BiTES encoded by the delivered dBiTE sequences.

Inovio’s dBiTEs provide major potential advantages over a conventional protein-based BiTE therapy because of dBiTEs’ better product expression and availability as well as simplicity in administration and manufacturing. Inovio has demonstrated that a single dose of dBiTE construct delivered with CELLECTRA expressed the product at high levels in mice for four months. Inovio’s dBiTEs are developed with simplified design using novel plasmid vectors and unique formulations allowing for rapidity of development, long-term product stability at refrigeration, ease of validated and scalable manufacturing and deployability.

Earlier this year, Inovio initiated the first clinical trial for a dMAb. Funded fully by the Bill & Melinda Gates Foundation, this trial’s focus is on evaluating the dMAb’s (INO-A002) ability to prevent or treat Zika virus infection. The clinical results will help to broadly advance Inovio’s dMAb and dBiTE programs in infectious diseases and cancer.

About Inovio’s dBiTE program

Inovio’s dBiTEs are able to target the cytotoxic T cells to tumors by engaging proteins expressed in the tumor surface. The current preclinical models have shown proof that DNA technologies are in an advantageous position to launch a more ambitious BiTE program. The tumor-binding domain can be modified to engage multiple targets, of which preclinical data targeting HER2 and CD19 will be presented. Of these, the CD19dBiTE can be used to target B cell cancers and the HER2dBiTE can be used to treat advanced breast, ovarian, gastric, esophageal and endometrioid cancers.

Conatus Pharmaceuticals to Participate in H.C. Wainwright Global Life Sciences Conference

On April 2, 2019 Conatus Pharmaceuticals Inc. (NASDAQ:CNAT) reported has accepted an invitation to participate in the H.C. Wainwright Global Life Sciences Conference (April 7-9 in London) (Press release, Conatus Pharmaceuticals, APR 2, 2019, View Source [SID1234534895]). Conatus senior management will conduct a series of scheduled meetings with investment professionals and present a company overview on Tuesday, April 9, 2019, from 10:30 a.m. to 10:50 a.m. BST (5:30 a.m. to 5:50 a.m. EDT). A webcast of the presentation will be available live and archived in the Investors section of the Conatus website at www.conatuspharma.com.

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Pacira Pharmaceuticals to Present at the 18th Annual Needham Healthcare Conference

On April 2, 2019 Pacira Pharmaceuticals, Inc. (NASDAQ: PCRX) reported that it will present at the 18th Annual Needham Healthcare Conference at 3:30 PM ET on Tuesday, April 9, 2019 (Press release, Pacira Pharmaceuticals, APR 2, 2019, View Source;p=irol-newsArticle&ID=2393147 [SID1234534894]). Live audio of the presentation can be accessed by visiting the "Events" page of the company’s website at investor.pacira.com. A replay of the webcast will also be available for two weeks following the event.

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Onconova Therapeutics To Present Update At The 2019 HCW Global Life Sciences Conference In London, April 7-9

On April 2, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes (MDS), reported that the Company will be presenting at the 2019 HCW Global Life Sciences Conference at the Grosvenor House/JW Marriott Hotel in Mayfair, London (Press release, Onconova, APR 2, 2019, View Source [SID1234534893]). Dr. Steven Fruchtman, President & CEO, will present at the conference and meet with investors.

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Presentation details
Date/Time:
Venue:
Presenter:
Webcast link: Monday, April 8th at 10:10 a.m. BST
Grosvenor House/JW Marriott Hotel – Spencer Suite
Steven M. Fruchtman, President & CEO
View Source
About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

Kura Oncology Identifies Farnesylated Proteins Associated with CXCL12 Expression, Potential Biomarker of Clinical Benefit from Tipifarnib in Lymphoma Indications

On April 2, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported new findings regarding the mechanism of action of the Company’s lead drug candidate tipifarnib and its potential clinical applications (Press release, Kura Oncology, APR 2, 2019, View Source [SID1234534892]). These findings are being presented today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta. A copy of the poster is available on Kura’s website at www.kuraoncology.com.

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Tipifarnib is a potent and selective farnesyl transferase inhibitor currently in a registration-directed clinical trial in patients with head and neck squamous cell carcinoma (HNSCC) that carry mutations in HRAS, an exclusively farnesylated oncogene. Tipifarnib has also been shown to downregulate production of the chemokine CXCL12 in tumor models and cancer patients. New findings, presented today, suggest that gene expression of the exclusively farnesylated proteins RHOE (RND3) and PRICKLE2 is strongly associated with CXCL12 expression in bone marrow stroma, which may provide a mechanistic rationale for why the CXCL12 pathway is a therapeutic target of tipifarnib and other farnesyl transferase inhibitors.

In addition, an analysis of a subset of patients from a previously conducted Phase 2 trial of tipifarnib in patients with relapsed and refractory lymphomas identified pre-treatment tumor CXCL12 expression as a potential biomarker of clinical benefit in patients with diffuse large B-cell lymphoma (DLBCL) and mycosis fungoides, the most common form of cutaneous T-cell lymphoma (CTCL). This observation is consistent with similar findings from Kura in other indications such as peripheral T-cell lymphoma (PTCL), acute myeloid leukemia (AML) and pancreatic cancer. CXCL12 and its receptors, CXCR4 and CXCR7, have been implicated in cancer progression and poor prognosis across a large spectrum of solid tumor and hematologic indications.

"The target of farnesyl transferase inhibitors has been elusive for several decades. These findings provide key evidence supporting the inhibition of the CXCL12 pathway as a mechanism of action mediating the activity of tipifarnib in the clinic," said Antonio Gualberto, M.D., Ph.D., Head of Development and Chief Medical Officer of Kura Oncology. "CXCL12-expressing cancers represent a major unmet medical need, and we believe that CXCL12 pathway biomarkers could enable registrational strategies for tipifarnib in multiple hematologic and solid tumor indications."

In December 2018, Kura reported activity from an ongoing Phase 2 trial of tipifarnib in patients with relapsed or refractory PTCL, including a significant association between CXCL12 expression and clinical benefit, as well as proof-of-concept in angioimmunoblastic T-cell lymphoma (AITL), an aggressive form of PTCL often characterized by high levels of CXCL12 expression. The Company anticipates providing an update on this trial, including duration of response data from the AITL cohort and additional data from the CXCL12-high PTCL cohort, in mid-2019.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent and highly selective inhibitor of farnesylation, a key cell signaling process implicated in cancer initiation and development. Tipifarnib was previously studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets, however no molecular mechanism of action had previously been determined that could explain its activity across a range of diverse clinical indications, including squamous tumors that carry mutant HRAS, as well as in lymphoid, myeloid and solid tumors that do not carry HRAS mutations. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib