BioMarin to Host Third Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, October 26, at 4:30pm ET

On October 4, 2022 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 Wednesday, October 26th, at 4:30 p.m. ET to discuss third quarter 2022 financial results and provide a general business update (Press release, BioMarin, OCT 4, 2022, View Source,-October-26,-at-4-30pm-ET [SID1234621671]).

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BioMarin to Announce 3Q22 Financial Results October 26th, 2022 After the Market Closes
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.

Abcuro Initiates Phase 1/2 Trial Evaluating ABC008 in Patients with T Cell Large Granular Lymphocytic Leukemia

On October 4, 2022 Abcuro, Inc., a clinical-stage biotechnology company developing therapies for the treatment of autoimmune diseases and cancer through precise modulation of cytotoxic T and NK cells, reported initiation of a Phase 1/2 dose escalation trial to evaluate the safety, tolerability, and proof-of-concept of ABC008 in patients with T cell large granular lymphocytic leukemia (T-LGLL) who suffer from anemia and/or neutropenia (Press release, Abcuro, OCT 4, 2022, View Source [SID1234621668]). ABC008 is a first-in-class anti-KLRG1 antibody capable of selectively depleting highly cytotoxic T cells, while sparing regulatory T cells, central memory T cells, and other immune cells.

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"Initiating the Phase 1/2 clinical trial of ABC008 represents an important step toward a potential breakthrough in the treatment of T-LGLL," said Jeffrey Wilkins, MD, Chief Medical Officer of Abcuro. "This trial builds upon prior published data demonstrating the ability of ABC008 to deplete highly cytotoxic T cells, which attack and destroy muscle tissue in inclusion body myositis, an autoimmune disease."

T-LGLL is an autoimmune disorder characterized by clonally expanded CD3+ CD8+ cytotoxic T lymphocytes which attack neutrophils and red blood cell precursors, leading to neutropenia and anemia. Neutropenia can lead to frequent infections, a major cause of premature death in patients with T-LGLL, while anemia results in transfusion dependence in approximately one third of patients.

"The limited efficacy of current standard of care is reflected in an overall reduced life expectancy for T-LGLL patients," said Paul Shami, MD, Professor of Medicine in the Division of Hematology and Hematologic Malignancies at the University of Utah and Principal Investigator. "Without any approved treatment options for T-LGLL, many patients turn to off-label therapies, such as methotrexate, a non-specific immunosuppressant, which often have limited effectiveness."

About ABC008
ABC008 is a first-in-class anti-KLRG1 antibody capable of selectively depleting highly cytotoxic T cells, while sparing regulatory and central memory T cells. ABC008 has been designed to treat diseases mediated by highly cytotoxic T cells, including the autoimmune muscle disease inclusion body myositis (IBM), T cell large granular lymphocytic leukemia (T-LGLL), and mature T cell malignancies. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABC008 for the treatment of IBM.

AbbVie to Host Third-Quarter 2022 Earnings Conference Call

On October 4, 2022 AbbVie (NYSE: ABBV) reported that it will announce its third-quarter 2022 financial results on Friday, October 28, 2022, before the market opens (Press release, AbbVie, OCT 4, 2022, View Source [SID1234621667]). AbbVie will host a live webcast of the earnings conference call at 8 a.m. CT. It will be accessible through AbbVie’s Investor Relations website investors.abbvie.com. An archived edition of the session will be available later that day.

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SyntheX and Bristol Myers Squibb Enter Into a Research Collaboration to Discover and Develop Targeted Protein Degradation (TPD) Therapeutics

On October 4, 2022 SyntheX, Inc., an emerging growth biotechnology company innovating next-generation functional drug discovery engines, reported a research collaboration and license agreement with Bristol Myers Squibb to discover, develop and commercialize novel small molecules using SyntheX’s ToRNeDO platform for protein degradation (Press release, SyntheX, OCT 4, 2022, View Source [SID1234621666]).

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The collaboration will focus on discovering molecular glue degraders. Under the terms of the agreement, SyntheX will receive a combined upfront of cash and an investment and is eligible for up to $550 million in performance-based milestone payments, as well as royalties on global net sales of products.

SyntheX has developed proprietary platforms that leverage the power of synthetic biology to build next generation drug discovery engines. Using genetic engineering, SyntheX’s platform technologies rely on functional intracellular drug selection as opposed to in vitro screening, which allows the company to discover functional molecular glues using a pre-specified E3 ligase and a neosubstrate of interest. The platform is versatile and can exploit many human E3 ligases.

"Bristol Myers Squibb is a globally recognized pharmaceutical leader, and we are excited to work with them in targeted protein degradation (TPD), an extremely exciting scientific field with vast drug development opportunities," said Maria Soloveychik, Ph.D., Co-Founder and Chief Executive Officer of SyntheX. "We look forward to combining our ToRNeDO platform with Bristol Myers Squibb’s clinical development expertise as we work to discover and deliver the next-generation of TPD therapies for patients in need."

miRecule Enters into Strategic Collaboration with Sanofi to Accelerate Discovery and Development of a Best-in-Class Antibody-RNA Conjugate to Treat Facioscapulohumeral Muscular Dystrophy (FSHD)

On October 4, 2022 miRecule, Inc., an innovator of next-generation RNA therapeutics, reported a strategic collaboration and exclusive license agreement with Sanofi to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD) (Press release, miRecule, OCT 4, 2022, View Source [SID1234621663]). The collaboration marks miRecule’s first licensing transaction leveraging its proprietary DREAmiR platform.

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FSHD is the second most common type of muscular dystrophy – affecting more than one million individuals worldwide with no approved treatments. Patients with causative genetic mutations experience lifelong deterioration of muscle function and progressive disability. The collaboration will combine miRecule’s anti-DUX4 RNA therapy (discovered through its proprietary DREAmiR platform) with Sanofi’s proprietary muscle-targeted NANOBODY technology to join the two molecules into an ARC utilizing miRecule’s NAVIgGatorTM conjugation and formulation chemistry. The potential resulting best- in-class therapy may become a disease-modifying treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. This groundbreaking approach would enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive disease.

Under the terms of the collaboration agreement, miRecule will grant Sanofi an exclusive worldwide license to intellectual property rights to the FSHD therapy. miRecule and Sanofi will collaborate on research activities through lead candidate selection. Following candidate selection, Sanofi will assume sole responsibility for IND enabling studies and subsequent development and commercialization activities globally. miRecule will receive an upfront payment and is eligible for near-term milestone payments, which combined could exceed $30 million. Additional development, regulatory, and commercial milestone payments could raise the total to nearly $400 million for advancement of the FSHD drug candidate, which will be exclusively developed and commercialized by Sanofi. miRecule is also eligible to receive tiered royalties on global net sales of the approved collaboration product.

Pablo Sardi
Global Head of Rare and Neurologic Diseases Research, Sanofi

"We look forward to working with miRecule to bring together our two groundbreaking technologies synergizing in a best-in-class therapy designed to suppress the underlying cause of FSHD. We hope that this will enable patients to live a life free from the debilitating symptoms of the disorder. We are excited to embark on this collaboration with miRecule as we work together to bring hope to the FSHD community."

Anthony Saleh
Founder and Chief Executive Officer, miRecule

"We are thrilled to enter our first major licensing transaction with a partner of Sanofi’s caliber and capabilities as a global leader in the development and commercialization of rare disease therapies. Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life. The philosophy driving our DREAmiR discovery platform focuses on patient centered drug development and strong scientific decision making. We believe Sanofi shares these core values on creating life changing therapies, making this is an ideal partnership to bring forward our anti-DUX4 RNA therapy."

miRecule’s founder and CEO has a strong commitment to developing a therapy to treat FSHD as the disorder runs in his family. miRecule started its program in 2019 with strong support from the patient and academic community, receiving funding from the FSHD Society, the Friends of FSH Research, and National Institute of Neurological Disorders and Stroke (NINDS, Grant 5R44NS119147-02). Sanofi and miRecule look forward to continuing to work with these organizations to help bring forward a groundbreaking treatment for FSHD.