On October 16, 2019 Navire Pharma, Inc., a BridgeBio Pharma, Inc. subsidiary developing small molecule inhibitors of the protein tyrosine phosphatase SHP2 (Src homology 2 domain-containing phosphatase), reported preclinical data demonstrating the potential for the Company’s SHP2 inhibitor to lung cancer tumor cells that have acquired resistance to EGFR inhibitors, which are common targeted cancer medicines (Press release, Navire Pharma, OCT 16, 2019, View Source [SID1234542303]). The data will be presented at AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on October 29, 2019.

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"Our work with MD Anderson’s Institute for Applied Cancer Science (IACS) adds to the growing body of evidence that SHP2 is an important node in MAPK signaling and supports combining a SHP2 inhibitor with an RTK inhibitor in RTK-driven cancers," said Shafique Virani, CEO of Navire Pharma. "The aim of BridgeBio’s BBP-398 program is to rapidly translate this exciting science into the clinic to evaluate the program’s potential to manage treatment-resistant cancers. We are preparing the program to be ready for clinical testing in 2020."

In a poster entitled "Discovery of IACS-13909, an allosteric SHP2 inhibitor that overcomes multiple mechanisms underlying osimertinib resistance," Yuting Sun, Ph.D., a member of the Institute for Applied Cancer Science at MD Anderson Cancer Center, from which Navire licensed its SHP2 inhibitors, will present preclinical data demonstrating that the allosteric SHP2 inhibitors were able to reduce growth of EGFR-driven non-small cell lung cancer (NSCLC) in vitro and in vivo. Importantly, IACS-13909 enhanced the anti-tumor activity of osimertinib, a front-line therapy for EGFR mutated NSCLC, when used in combination with osimertinib in preclinical models.

SHP2, a conserved protein tyrosine phosphatase, is a critical node in growth factor, cytokine and integrin signaling, all of which are important in the progression of cancer. SHP2 regulates multiple downstream signaling pathways including RTK/MAPK and the adaptive immune response through checkpoint inhibition. Alterations in RTK/MAPK and checkpoint inhibition pathways are common in cancer. Thus, targeting SHP2 may offer a potential new approach to treat this disease.

On October 16, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that an abstract related to the Company’s Phase 2 clinical trial of tipifarnib in HRAS mutant head and neck squamous cell carcinomas (HNSCC) has been accepted for presentation at the upcoming AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), which will be held from October 26-30, 2019 in Boston (Press release, Kura Oncology, OCT 16, 2019, View Source [SID1234542302]). Details are as follows:

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Title: Preliminary results from a phase 2 trial of tipifarnib in squamous cell carcinomas (SCCs) with HRAS mutations
Session Title: Clinical Trials
Session Date: Sunday, October 27
Session Start Time: 12:30
Session End Time: 16:00
Location: Hall D, Hynes Convention Center
Abstract Number: A087

The abstract has been selected as a newsworthy candidate for the meeting’s official press program and will be embargoed until 11:00 a.m. ET on Friday, October 25, 2019. A copy of the poster will be available at www.kuraoncology.com following presentation at the meeting.

On October 16, 2019 PharmaCyte Biotech (OTCQB: PMCB) reported that it has now moved one step closer to submitting an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) to request a clinical trial in locally advanced, inoperable pancreatic cancer (LAPC) in the United States (Press release, PharmaCyte Biotech, OCT 16, 2019, View Source [SID1234542301]). The company has successfully completed the first of two manufacturing runs to produce the clinical trial product (Cell-in-a-Box capsules) it needs for its upcoming Phase 2b clinical trial in LAPC.

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Kenneth L. Waggoner, PharmaCyte’s Chief Executive Officer, said that the completion of the first manufacturing run "is a major milestone" towards the completion of the IND.

A few other key events will play themselves out in the weeks to come—all leading to the submission of the IND. The first of which has already begun. PharmaCyte stated this week that the second of two staggered and back-to-back manufacturing runs is already underway, and that the cells from the company’s Master Cell Bank (MCB) are growing well in this second run and will be encapsulated within the next week or two.

A successful second run should be regarded as an even greater milestone and extremely good news for the company and its shareholders as it will represent the conclusion of all necessary manufacturing runs on the way to submitting an IND. cGMP Validation is the company that will take responsibility for PharmaCyte’s clinical trial product coming into the United States and being used in human patients, so two successful manufacturing runs undoubtedly gives cGMP Validation the comfort level it needs to take on this responsibility.

Waggoner said of the second manufacturing run, "Our cGMP expert has recommended that a second manufacturing run be done because, by doing so, we can firmly validate to the FDA that our manufacturing process is both reproducible and robust. Also, additional information on duplicate manufacturing runs may be beneficial to our cGMP expert, who will also serve as our ‘Release Agent’ so that our clinical trial product can be used in human patients in the U.S. in a clinical trial."

Validating that the company’s manufacturing process is both "reproducible and robust" to the FDA is significant today and well into the future for PharmaCyte, according to the company’s CEO.

"Although we have been advised that the IND for a Phase 2 clinical trial doesn’t require information related to successful duplicate manufacturing runs, it’s important for us to take the extra time to complete the second manufacturing run because the manufactured product is not only the ‘centerpiece’ of our planned clinical trial in LAPC, but it will also likely play a similar role in the treatment of other forms of cancer."

The most important event left to complete outside of the final manufacturing run is "release testing" of the clinical trial product. PharmaCyte said that a representative sample of frozen syringes from the first successful manufacturing run, which are filled with 300 Cell-in-a-Box capsules each, are in the process of being shipped to external testing labs for the release testing that is required by the FDA.

The data that those tests will produce are all that remains for the company to complete the IND and then submit it to the FDA.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

On October 16, 2019 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized chimeric antigen receptor T cell ("CAR T") platform technology engineered to target patient-specific tumor neoantigens, reported that Jeffrey Eisenberg, Chief Executive Officer of Xenetic, will present at BIO Investor Forum on Wednesday, October 23, 2019 at 2:45 PM PT in San Francisco, CA (Press release, Xenetic Biosciences, OCT 16, 2019, View Source [SID1234542300]).

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As part of his presentation, Mr. Eisenberg will provide a Company overview and discuss the Company’s novel CAR T platform technology, called "XCART," a proximity-based screening platform capable of identifying CAR constructs that can target patient-specific tumor neoantigens, with a demonstrated proof of mechanism in B-cell Non-Hodgkin lymphomas. Xenetic is currently advancing the development program for XCART to confirm the positive preclinical results shown to date and to demonstrate a more attractive safety profile than existing therapies.

In addition to the presentation, management will also be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference.

A live audio webcast of the presentation will be available on the IR Calendar in the Investors section of Xenetic’s website (www.xeneticbio.com) or by accessing the conference website here. Within three days of the event, a webcast replay will be made available on the Company’s website.

About BIO Investor Forum

Now in its 17th year, the BIO Investor Forum is an international biotech investor conference focused on early and established private companies as well as emerging public companies. The event features plenary sessions, business roundtables, therapeutic workshops, company presentations, and BIO One-on-One Partnering meetings. For more information, please visit the conference website here.

On October 16, 2019 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain (Press release, Intellia Therapeutics, OCT 16, 2019, View Source [SID1234542299]).

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Intellia’s data includes important updates about the company’s programs and platform development activities:

Oral Presentation:

"In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression"

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellia’s gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellia’s similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.
Abstract number: OR48
Session 5b: New delivery systems and technologies
Presentation date/time: Friday, October 25, 2019, 11:30 a.m. – 1:30 p.m. CET
Location: Room 113-115

Poster Presentations:

"In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics"

This poster presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel O’Connell, Ph.D.
Poster ID Number: P655
Date: Wednesday, October 23, 2019

"Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia"

This poster presentation focuses on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San Raffaele
Poster ID Number: P111
Date: Wednesday, October 23, 2019

"Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout"

This poster presentation focuses on the company’s T cell engineering technology, which is being applied in its Wilms’ Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.
Poster ID Number: P162
Date: Thursday, October 24, 2019

"CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria"

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.
Poster ID Number: P552
Date: Thursday, October 24, 2019