On April 13, 2021 BridgeBio Pharma, Inc. (NASDAQ: BBIO) reported a collaboration with Oregon Health & Science University (OHSU) in Portland, Oregon, to advance research and the development of investigational medicines for patients with genetically driven conditions (Press release, BridgeBio, APR 13, 2021, View Source [SID1234577967]).

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"We feel privileged to have the opportunity to work with OHSU to focus on developing potential therapies for diseases with clear genetic drivers and severe unmet need," said BridgeBio founder and CEO Neil Kumar, Ph.D. "OHSU was one of the first research institutions we connected with in the early days of BridgeBio, and we look forward to deepening our relationship through close collaboration with their scientists as we strive to help patients together."

This arrangement builds on five years of informal collaborations between the two organizations. Under the agreement, the BridgeBio team will work closely with OHSU scientists and investigators to advance potential medicines for patients with genetically driven conditions, including cancers.

"BridgeBio’s approach to identifying and developing novel therapies allows them to interact with multiple investigators at OHSU who are studying a wide variety of diseases," said OHSU Chief Research Officer and Executive Vice President Peter Barr-Gillespie, Ph.D. "We believe their expertise and infrastructure will help lead to the rapid development of new drugs. We see this collaboration as an important part of our efforts to bring OHSU innovations to clinical significance."

BridgeBio collaborates with stand-out academic institutions, including OHSU, to support research around genetically driven conditions and is focused on rapidly translating findings into meaningful treatments for patients. Today BridgeBio also announced formal partnerships with Brown University, GlycoNet, The Lundquist Institute, 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 partners with, please visit Our Partners page.

With a diverse pipeline encompassing investigational therapies for rare diseases and genetically validated cancers, BridgeBio provides the insights and support needed to rapidly progress therapeutic research from labs to clinical development. BridgeBio intends to develop similar long-term partnerships based on trust, engagement, science and respect to support its mission of developing life-changing medicines for patients with genetically driven conditions as quickly and safely as possible.

On April 13, 2021 BridgeBio Pharma, Inc. (NASDAQ: BBIO), and Brown University reported a formal collaboration to advance research in genetically driven neurological disorders into potential life-changing medicines for patients (Press release, BridgeBio, APR 13, 2021, View Source [SID1234577966]).

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"Brown University is a leader in critical neurology research, and we look forward to partnering with their scientists to focus on discovering and advancing new targeted treatment approaches to potentially address challenging and complex diseases of the brain," said BridgeBio founder and CEO Neil Kumar, Ph.D.

Under the partnership, BridgeBio will work with scientists at Brown to evaluate new discoveries in neurology research that have promise to advance toward clinical investigation. Select therapeutic programs may be spun out and advanced in BridgeBio affiliate companies, with an opportunity for Brown University scientists to support as company leaders and guide ongoing development.

"BridgeBio’s unique approach and support for having researchers stay involved in development made it an easy decision to partner with them in translating our discoveries into potential medicines for patients in need," said Neil Veloso, Executive Director of Brown Technology Innovations at Brown University. "We look forward to the collaboration and empowering Brown faculty to progress their research."

BridgeBio partners with stand-out academic institutions, including Brown University, to support research around genetically driven conditions and is focused on rapidly translating findings into meaningful treatments for patients. Today BridgeBio also announced formal partnerships with GlycoNet, 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 part of an ongoing effort to translate research in genetically driven conditions toward therapeutic development, BridgeBio will continue to seek long-term partnerships with similar institutions that are rooted in trust, engagement, science and respect in order to drive forward its mission of developing transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers as quickly and safely as possible.

On April 13, 2021 Repertoire Immune Medicines, a clinical-stage biotech company decoding the immune synapse to create novel immune therapies for cancer, immune disorders, infectious disease, and other serious diseases, reported the completion of a $189 million Series B financing (Press release, Repertoire, APR 13, 2021, View Source [SID1234577963]). Joining Flagship Pioneering, Repertoire’s institutional founder and principal backer, are new and existing investors including Softbank Vision Fund 2i, the Public Sector Pension Investment Board (PSP Investments), the Alaska Permanent Fund, Invus, and others. Proceeds from this financing will support further expansion of the company’s proprietary DECODE discovery platform, accelerate its clinical and preclinical pipeline, expand Repertoire’s team, and enhance its manufacturing capabilities. Founded by Flagship Pioneering, Repertoire has raised more than $350 million in the aggregate to date.

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"We are making tremendous progress toward unlocking the full potential of therapeutically modulating the immune system by decoding the interactions between T cells and antigens to create new classes of immune medicines for cancer, immune disorders, infectious diseases, and other serious diseases," said John Cox, CEO of Repertoire. "This financing round will support the broad application of our DECODE platform to define and understand the complete repertoire of T cell receptor-antigen pairs that drive cellular immunity. The insights we glean from this information will enable a new era of novel immune medicines designed to exploit these critical interactions."

This financing also supports the advancement of multiple clinical programs in immuno-oncology using Repertoire’s proprietary antigen-primed multi-clonal cytokine enhanced T cell products in solid tumors. The PRIME IL-15 program in advanced metastatic solid tumors is underway, with a data readout anticipated later this year. The PRIME IL-12 program in HPV-16+ solid tumors will be initiated by mid-year. Both clinical programs represent novel first of their kind immune medicines in oncology.

"All roads to cellular immunity pass through the immune synapse," said Douglas Cole, M.D., co-founder, and Chairman of the Board of Repertoire Immune Medicines and Managing Partner of Flagship Pioneering. "Repertoire’s innovative platform makes it possible to elucidate the features of the immune synapse that drive clonal behavior. Applying this knowledge will revolutionize the discovery and development of immune medicines."

On April 13, 2021 Arcellx, a privately held clinical-stage biopharmaceutical company, reported that it raised $115 million in a Series C financing to advance its pipeline of adaptive and controllable cell therapies (Press release, Arcellx, APR 13, 2021, View Source [SID1234577962]). The proceeds will support the company’s development of CART-ddBCMA, a BCMA-specific CAR-modified T-cell therapy currently in Phase 1 and anticipated to begin a pivotal trial in 2022. In addition, the funding will support initiation of clinical trials evaluating ACLX-001 and ACLX-002, cell therapies derived from Arcellx’s uniquely controllable ARC-SparX platform, in multiple myeloma (MM) and acute myelogenous leukemia (AML), respectively.

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This financing follows FDA clearance of Arcellx’s IND for ACLX-001, the first ARC-SparX program to enter clinical trials, and Arcellx’s initial release of clinical results at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) meeting. In the ASH (Free ASH Whitepaper) release, the CART-ddBCMA data showed all six multiple myeloma patients responded per IMWG criteria, with four of those patients achieving stringent complete response. The therapy was also well-tolerated, and CAR-T related toxicities resolved rapidly.

Participants in the Series C financing include both existing and new investors to Arcellx. The financing was co-led by Samsara BioCapital and CAM Capital, joined by new investors Adage, Asymmetry, CaaS Capital, Cambrian Bio, Sixty Degree, Soleus Capital, Surveyor Capital (a Citadel company), Suvretta, and Terra Magnum Capital Partners, and existing investors NEA, Novo Holdings, SR One, Takeda Ventures, LG Tech, and Clough Capital.

"With support from this high caliber syndicate, Arcellx is poised to elevate the field of cell therapy by advancing our treatments for a range of cancers," said Rami Elghandour, Chairman and Chief Executive Officer of Arcellx. "Our platform of both single infusion and controllable CAR-Ts based on our novel synthetic binding domain is built to address the limitations of cell therapy with the opportunity to improve efficacy, reduce toxicity, and shorten the time to intervention while expanding into new indications. This financing positions us to advance to a registrational study in multiple myeloma and to initiate a Phase 1 study in AML in 2022 as well as progress our solid tumor targets toward the clinic. It’s also a reflection of our incredibly talented and diverse team that is powering Arcellx forward. We appreciate the support of our new and existing investors as we advance our novel therapies to the benefit of cancer patients most in need."

"Based on the early clinical data, we believe that CART-ddBCMA represents a potential best-in class therapy for multiple myeloma and with the support of this financing will be positioned to move into pivotal trials next year. We’re also excited about the opportunity for CART-ddBCMA to move into earlier lines of treatment for multiple myeloma based on the safety profile in this early data set. In addition, the ARC-SparX platform will be the first adaptive and controllable CAR-T system to enter the clinic and provides a unique approach to building next generation cell therapies. We look forward to partnering with the Arcellx team to help bring these important therapies to patients," said Mike Dybbs, Ph.D., Partner, Samsara BioCapital.

On April 13, 2021 Adagene Inc. ("Adagene") (Nasdaq: ADAG), a platform-driven, clinical-stage biopharmaceutical company committed to transforming the discovery and development of novel antibody-based immunotherapies, reported updated preclinical data from its lead SAFEbody program, ADG126, are being presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, Adagene, APR 13, 2021, View Source [SID1234577961]).

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"There is a critical unmet need for new anti-CTLA-4 therapies, and we are encouraged by the data generated to date, which demonstrate ADG126 has the potential to overcome the severe immune-mediated adverse reactions associated with the class," said Peter Luo, Ph.D., Co-founder, Chief Executive Officer and Chairman of Adagene. "By leveraging our SAFEbody platform technology, designed to precisely and very efficiently mask the antibody binding interface and activate specifically within the tumor microenvironment, our preclinical data continues to highlight the opportunity to effectively deliver treatment; ADG126 has demonstrated superior systemic safety profile at efficacious dose levels with a significantly enlarged therapeutic index (TI). Our ongoing global Phase 1 trial is expected to provide clinical validation for our SAFEbody platform and our SAFEbody product candidate, ADG126. We have successfully finished DLT evaluation of 3 patients at 0.1 mg/kg of ADG126 and remain on track to report topline safety and efficacy data in the second half of 2021."

A copy of the poster presentation, entitled "A Novel Anti-CTLA-4 Checkpoint Inhibitor Prodrug to Address On-target Off-tumor Toxicity for Cancer Immunotherapy," is available on the AACR (Free AACR Whitepaper) website and is also available for download via our website (View Source).

The data presented show:

Notable Findings: ADG126 demonstrated an impressive safety margin while maintaining its potent antitumor activity.
Unique Epitope with Broad Species Cross-Reactivity: ADG126 targets a conserved epitope of CTLA-4 with broad species cross-reactivity and is an activatable prodrug for tumor suppression in multiple syngeneic mouse models in single and combination therapies.
Differentiated Mechanism of Action: Although the activated ADG126 is softer in blocking CTLA-4 binding with its ligands than ipilimumab, the activated ADG126 exhibited more potent antibody dependent cellular cytotoxicity (ADCC) in the tumor microenvironment.
Intra-Tumoral Treg Depletion: Treg cells in tumor tissues exhibited higher CTLA-4 expression than in peripheral tissues and were efficiently depleted upon treatment with ADG126 in the immune-competent mouse syngeneic colon tumor model.
Human T-Cell Activation in Vitro: Activated ADG126 potently enhanced T-cell activation, measured by IL-2 secretion, whereas the masked ADG126 did not.
In Vivo Monotherapy Antitumor Activity: ADG126 exhibited potent antitumor activity as a single agent in different immune-competent syngeneic mouse tumor models.
In Vivo Combination Antitumor Activity: ADG126 combined synergistically with other IO agents, such as anti-PD-1 antibody, to inhibit tumor growth in vivo. Combination therapy significantly slowed tumor growth and caused complete regression in 50% of Lewis lung cancer mouse models.
Safety and Tolerability: ADG126 was well tolerated in animals suggesting the potential for high therapeutic index. The highest non-severely toxic dose (HNSTD) was determined to be 200 mg/kg/dose, which is one of the highest reported HNSTD for anti-CTLA-4 antibodies.
About ADG126
ADG126 is a fully human antagonistic mAb targeting a novel epitope of CTLA-4 and has been shown to specifically deplete regulatory T-cells in tumors. ADG126 is Adagene’s lead SAFEbody product candidate. The SAFEbody technology, developed using Adagene’s AI-powered platform, enables binding of an antibody to a specific target only after conditional activation of the antibody in target tissues.

In preclinical studies, ADG126 was well tolerated in cynomolgus monkeys and demonstrated an encouraging antitumor response in multiple immune-competent mouse tumor models in a dose-dependent manner both as a single agent and in combination with anti-PD-1 and other therapies.

Unlike anti-PD-1/PD-L1 check point inhibitors, anti-CTLA-4 is known for its dose-dependent clinical response in single and combination therapies, which is severely limited by the narrow therapeutic window available to current anti-CTLA-4 therapies. The large safety margin shown by ADG126 GLP toxicology studies of up to 200 mg/kg in targeting CTLA-4 will make it possible to dose patients for their optimal clinical benefits in single and combination therapies.