On April 13, 2021 BridgeBio Pharma, Inc. (NASDAQ: BBIO) and the Canadian Glycomics Network (GlycoNet), a pan-Canadian Network of Centres of Excellence, reported a collaboration to translate scientific research in glycomics into potential treatments for patients with genetic diseases (Press release, BridgeBio, APR 13, 2021, View Source [SID1234578024]).

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"We are thrilled to be partnering with GlycoNet, an institution at the forefront of developing carbohydrate-based drugs to address areas of unmet need," said BridgeBio founder and CEO Neil Kumar, Ph.D. "We understand the clinical benefit research in glycomics could have for certain genetic diseases and by partnering together, we hope to advance potentially life-changing medicines as rapidly as possible."

BridgeBio will work alongside GlycoNet researchers to identify research programs that may have the potential to become treatments for genetic diseases. BridgeBio will potentially sponsor research programs and support clinical investigation through its licensing and affiliate development model. Genetic disease research has benefitted substantially from the study of glycomics, which relates to the functions of glycans, or sugar, in biological systems.

"The opportunity to partner with BridgeBio is invaluable," said GlycoNet CEO Elizabeth Nanak, Ph.D., MBA. "Our joint effort could potentially address areas of unmet needs and get treatments to patients more efficiently. We are hopeful that our network’s expertise in glycomics coupled with BridgeBio’s vigor to advance therapies into the clinic, will improve the quality of life of patients with genetic disorders."

BridgeBio partners with top academic and research institutions throughout the world, including GlycoNet, to support early, promising research. Today BridgeBio also announced formal partnerships with Brown University, The Lundquist Institute, Oregon Health & Science University, Roswell Park Comprehensive Cancer Center, University of California, Davis and University of California, San Diego – for a total of 20 partnerships between BridgeBio and leading academic and research institutions to-date. For a list of some of the institutions BridgeBio is partnered with, please visit Our Partners page.

As one of its guiding principles for drug development, BridgeBio believes in the importance of developing long-term partnerships through trust, respect and science in order to pioneer critical medicines for patients with genetically driven conditions as quickly and safely as possible.

On April 13, 2021 Harbour BioMed ("HBM"; HKEX: 02142.HK), a global clinical-stage biopharmaceutical company, reported its newly discovered fully human anti-B7H7 monoclonal antibody at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Harbour BioMed, APR 13, 2021, View Source [SID1234578000]). This meeting is being held virtually from April 10-15, 2021. The anti-B7H7 antibody is a novel immune-oncology antibody with potent anti-tumor activity generated using HBM’s proprietary H2L2 Harbour Mice platform.

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Details of the presentation:

Harbour BioMed’s presentation
By leveraging its innovative discovery engine based on the Harbour Mice antibody platforms, HBM has built a sustained capability to deliver novel antibody therapeutics in the field of oncology and immunology. Following the previously reported monoclonal antibody against a novel target CCR8, HBM now reports another first-in-class therapeutic antibody targeting a novel immune checkpoint – B7H7. B7H7 is mainly expressed in some PD-L1 negative/low tumors and may serve as an alternative immune escape pathway for these tumors. This antibody generated from H2L2 Harbour Mice demonstrates excellent preclinical activity and anti-tumor efficacy.

About B7H7

B7H7 is a novel immune checkpoint of B7 family, which is highly expressed on a variety of human cancers including colon, pancreatic, kidney, breast, bladder, lung cancers, etc. and is associated with metastatic disease and poorer survival rate of these patients. B7H7 antibody may present a novel anti-tumor therapy complementary to PD-L1/PD1 based therapy. B7H7 antibodies show significant T cell activation effect in vitro and tumor growth inhibition in several mouse tumor models.

On April 13, 2021 Biomica Ltd., an emerging biopharmaceutical company developing innovative microbiome-based therapeutics and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), reported additional positive pre-clinical results in its immuno-oncology program demonstrating efficacy of its live biotherapeutic product (LBP) consortium BMC128, this time in melanoma (Press release, Biomica, APR 13, 2021, View Source [SID1234577998]). In these studies, Biomica tested BMC128, which consists of four live bacterial strains, in a mouse model of melanoma.

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Dr. Elran Haber, CEO of Biomica, stated: "We are very excited with the results of this study demonstrating the effectiveness of BMC128 in treating additional types of solid cancer tumors. These positive pre-clinical results indicate the potential of BMC128 to become best-in-class in the treatment of solid cancer tumors, and help validate Biomica’s computational-based drug design approach. We look forward to providing incremental updates as we work towards a first-in-human, proof of concept clinical trial."

Treatment with BMC128 in combination with Immune Checkpoint Inhibitors (ICI) immunotherapy significantly enhanced anti-tumor activity, resulting in an increased response of melanoma tumors to anti-PD1, as demonstrated in an improved Objective Response Rate (ORR) and Percent Tumor Growth Inhibition (%TGI). The group treated with only anti-PD1 showed no response (ORR = 0%) as measured by the Response Evaluation Criteria in Solid Tumors (RECIST), while the group treated with a combination of BMC128 and anti-PD1 demonstrated a total of 13% response (ORR = 13%). The %TGI was increased by 100% in the BMC128 and anti-PD1 combination treated group compared to the group treated by anti-PD1 alone. Response to BMC128 was correlated with a desired anti-tumor immunological profile. BMC128 changed the course of response to ICI, leading to stimulation of the immune system which shifted cold-tumors into hot-tumors.

These positive results supplement previous pre-clinical data using BMC128 in combination with ICI in a breast cancer mouse model that demonstrated pronounced anti-tumor activity as manifested in an increase of almost 50% in ORR. The current results demonstrate the potential applicability of BMC128 and its relevance to treating multiple types of solid tumors.

Biomica’s immuno-oncology program is based on the premise that the gut microbiome affects the efficacy of cancer immunotherapy, specifically that of the ICI involving the blockade of PD-1 or PD-L1 and CTLA-4 as suggested in scientific literature.[1],[2] Fecal microbial transplantation has been recently reported to increase response in patients resistant to immune-checkpoint therapy[3],[4], however the specific microbial entities driving this response are currently unknown. BMC128 is a rationally-designed microbial consortium identified and selected through a detailed functional microbiome analysis using PRISM, a proprietary high-resolution microbiome analysis platform powered by Evogene’s MicroBoost AI platform.

As previously reported, Biomica has initiated scale-up processes and Good Manufacturing Practice (GMP) production of its drug candidate in its immuno-oncology program in preparation for the first-in-human proof-of-concept clinical trial expected later this year.

Mr. Ofer Haviv, Chairman of Biomica and Evogene President & CEO, stated: "We are proud of the results that Biomica reported today. These results support the computational biology capabilities developed by Evogene and Biomica which predicted that the microbes that make up BMC128 can be utilized to improve the efficacy of ICI in solid tumors. We look forward to validating the same computational forecasting capabilities in additional successful programs led by Biomica such as IBD and IBS."

[1] Zitvogel et al. 2018, Science 359 (6382)
[2] Thompson J, et al. Microbiome & immunotherapy: Antibiotic use is associated with inferior survival for lung cancer patients receiving PD-1 inhibitors. J Thorac Oncol 12(suppl 2):S1998, 2017
[3] Baruch E, et al. 2021. Fecal microbiota transplant promotes response in immunotherapy-refractory melanoma patients. Science, 371 (6529)
[4] Davar D, et al. 2021. Fecal microbiota transplant overcomes resistance to anti–PD-1 therapy in melanoma patients. Science, 371 (6529)

On April 13, 2021 Iterion Therapeutics, Inc., a venture-backed, clinical stage biotechnology company developing novel cancer therapeutics, reported that it has confirmed the safety of Tegavivint, a novel, potent and selective nuclear beta-catenin inhibitor, after completing enrollment and dosing the final patient in a multicenter Phase 1/2a dose expansion clinical study of Tegavivint in patients with desmoid tumors (Press release, Iterion Therapeutics, APR 13, 2021, View Source [SID1234577997]).

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Nuclear beta-catenin is a highly-studied oncology target associated with numerous cancer types. Tegavivint is unique among nuclear beta-catenin inhibitors in that it binds to TBL1 (Transducin Beta-like Protein One), a novel downstream target in the Wnt-signaling pathway. As such, Tegavivint enables silencing of Wnt-pathway gene expression without affecting other Wnt/beta-catenin functions in the cell membrane, thus avoiding toxicity issues common to other drugs in this pathway.

The Phase 1/2a clinical trial of Tegavivint in patients with progressive desmoid tumors was designed as an open-label, non-randomized dose-finding study. The primary objectives of the study were to evaluate the safety and tolerability of Tegavivint. Secondary objectives were to determine the durability of response (DOR) to Tegavivint after the achievement of best response. The total study enrolled 24 patients. During the dose expansion portion of the trial 16 of these patients were treated with a recommended Phase 2 dose (RP2D) that was established based on pharmacokinetic exposure levels and clinical responses in a recently completed Phase 1 study.

Data from patients treated in the dose expansion portion of the trial reaffirmed Tegavivint’s safety at the RP2D level. No dose-limiting toxicities or significant adverse events were observed. This data will enable Iterion to accelerate clinical activity in additional cancer indications where nuclear beta-catenin signaling has been identified as a potential therapeutic target, including AML, NSCLC, and certain pediatric cancers. Iterion expects to initiate clinical programs investigating Tegavivint for these indications in 2021.

"We have seen very good tolerability with no dose-limiting toxicities and no significant adverse events in escalating clinical doses," said Casey Cunningham, Chief Medical Officer of Iterion. "We are seeing a very strong safety signal in patients who have been on Tegavivint for over a year and are also observing tumor activity in patients. We continue to follow the patients that are still receiving treatment and look forward to sharing efficacy results at an upcoming medical conference."

Rahul Aras, CEO of Iterion, stated: "The completion of enrollment in the dose expansion phase of our desmoid tumor clinical trial and demonstration of safety and clinical activity at the RP2D represent important milestones in our clinical development of Tegavivint. We look forward to advancing the clinical development of Tegavivint in desmoid tumors as this disease target is greatly underserved. The results of this study also provide a ‘green light’ to initiate clinical development of Tegavivint in additional, high-value cancer settings, including AML, NSCLC, and certain pediatric cancers, that are characterized by nuclear beta-catenin overexpression."

About Desmoid Tumors

Desmoid tumors are rare, non- metastasizing sarcomas that overexpress nuclear beta-catenin, a historically "undruggable" oncology target implicated in cell proliferation, differentiation and immune evasion. An estimated 1,500 patients in the US are newly diagnosed with desmoid tumors each year. Desmoids are most commonly diagnosed in young adults between 30-40 years of age and are associated with significant morbidities, including severe pain, disfigurement, internal bleeding and organ damage, range of motion loss and, in rare cases, death. Iterion has received Orphan Drug Designation for Tegavivint to treat desmoid tumors, a disease for which there are no FDA approved therapies.

On April 13, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, April 29, at 4:30 p.m. ET to discuss first quarter 2021 financial results and provide a general business update (Press release, BioMarin, APR 13, 2021, prnewswire.com/news-releases/biomarin-to-host-first-quarter-2021-financial-results-conference-call-and-webcast-on-thursday-april-29-at-430pm-et-301266955.html [SID1234577996]).

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Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.biomarin.com. A replay of the call will be archived on the site for one week following the call.