CELL AND GENE THERAPY

BeiGene Initiates First-in-Human Phase 1 Clinical Trial of Investigational TYK2 Inhibitor BGB-23339

BeiGene | November 23, 2021

BeiGene a global, science-driven biotechnology company focused on developing innovative and affordable medicines, announced that the first patient has been dosed in a Phase 1 clinical trial of BGB-23339, a potent, allosteric investigational tyrosine kinase 2 (TYK2) inhibitor internally developed by BeiGene scientists.

TYK2 is a member of the JAK family and functions as a critical mediator in cytokine signaling pathways implicated in multiple immune-mediated disorders, such as psoriasis and inflammatory bowel disease. BGB-23339 is a potent, highly selective, investigational TYK2 inhibitor targeting the regulatory pseudokinase (JH2) domain.

Discovered and developed by BeiGene, BGB-23339 is a highly selective, potent, allosteric TYK2 inhibitor that has shown promising activity in preclinical evaluation. Building on our proven track record in oncology, BeiGene is expanding its clinical focus to discover new modalities and platforms in areas of high unmet need, including inflammation and immunology, to bring innovative, impactful medicines to patients.”

Lai Wang, Ph.D., Global Head of R&D at BeiGene

The first-in-human Phase 1 trial (NCT05093270) is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary activity of BGB-23339. The trial is expected to enroll up to 115 healthy volunteers in Australia and/or China.

In addition to its broad portfolio focused on hematological malignancies and solid tumors, BeiGene is applying its research excellence and clinical expertise to address inflammation and immunology, an area of high unmet medical need. BeiGene’s internally developed, highly selective next-generation BTK inhibitor BRUKINSA® (zanubrutinib) is currently being evaluated in a Phase 2 trial in patients with active proliferative lupus nephritis.

About BGB-23339
BGB-23339 is a potent, highly selective, allosteric, investigational tyrosine kinase 2 (TYK2) inhibitor discovered and being developed by BeiGene. TYK2 is a member of the JAK family and functions as a critical mediator in cytokine signaling pathways implicated in multiple immune-mediated disorders. Designed to target the regulatory pseudokinase (JH2) domain on TYK2, BGB-23339 has demonstrated strong selectivity in preclinical studies with potent inhibition of interleukin (IL)-12, IL-23, and Type 1 interferons (IFNs)—pro-inflammatory cytokines that play a determinant role in the induction of inflammation. BGB-23339 is currently being evaluated in a Phase 1 clinical study.

About BeiGene
BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 7,700 colleagues across five continents. 

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding plans for the Phase 1 trial and development of BGB-23339, the potential for BGB-23339 to address unmet medical needs, BeiGene’s plan to expand its clinical focus to discover new modalities and platforms in areas of high unmet need, including inflammation and immunology, and BeiGene’s plans, commitments, aspirations and goals under “About BeiGene”. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates and achieve and maintain profitability; the impact of the COVID-19 pandemic on the BeiGene’s clinical development, regulatory, commercial, and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

Spotlight

International Service for the Acquisition of Agri-biotech Applications (ISAAA) is a not-for-profit international organization that shares the benefits of crop biotechnology to various stakeholders, particularly resource-poor farmers in developing countries, through knowledge sharing initiatives and the transfer and delivery of proprietary biotechnology applications.

Spotlight

International Service for the Acquisition of Agri-biotech Applications (ISAAA) is a not-for-profit international organization that shares the benefits of crop biotechnology to various stakeholders, particularly resource-poor farmers in developing countries, through knowledge sharing initiatives and the transfer and delivery of proprietary biotechnology applications.

Related News

MEDICAL

Novalis Biotech Announces the Closing of a €25 Million Biotech Acceleration Fund

Novalis Biotech | July 01, 2021

Novalis Biotech, an early-stage venture capital investor in healthcare-related technology, announced today the completion of its €25 million second fund, the Novalis Biotech Acceleration Fund. The fundraising has included both new and returning investors. The second fund's investor base comprises institutional investors, including Participate Maatschappij Vlaanderen (PMV), family offices, and high-net-worth individuals (HNWIs). The Biotech Acceleration Fund will continue to invest in start-up and scale-up companies that use "enabling technologies" to improve healthcare. Genomic and bioinformatics technologies, drug research and manufacturing tools, diagnostics, and personalized medicine are examples. The funds will be divided between early-stage incubation initiatives and late-stage acceleration programs. Grapheal and RheaVita have already made investments in the enabling technologies they are developing. Novalis will continue to use its strong network in the Benelux area and intends to expand its investments throughout Western Europe and the United States. Novalis invests early and focuses on capital-light ideas where modest investments can generate substantial value using software, intellectual property, tools, and other enabling technologies that can transform healthcare. Novalis has a strong track record of turning ideas into profitable businesses by using extensive networks. Novalis Fund 1 was established in September 2018 with a total capitalization of €4 million, which was invested in eight companies, the majority of which were located in the Benelux region. BioLizard, Cergentis, Enzyre, Epify, Fertiga, myNEO, and OHMX.bio are among them. A first exit occurred when doc.ai, a US portfolio company, was acquired by Sharecare as part of an IPO. About Novalis Biotech Acceleration Novalis Biotech is an early-stage venture capital investor in healthcare-revolutionizing technologies. The company's main competence is digitalization in the life sciences, focusing on capital-light "enabling technologies" such as bioinformatics, artificial intelligence, genomics, personalized medicine, research or manufacturing tools, and diagnostics.

Read More

Novartis Acquires Vedere Bio, a Novel Optogenetics AAV Gene Therapy Company

Vedere Bio | October 29, 2020

Vedere Bio, Inc. (Vedere Bio), a stealth-stage company focused on advancing photoreceptor-protein-based optogenetic therapies that are delivered to the retina intravitreally to restore functional vision, announced today that it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio was formed in the Atlas Venture incubator in June 2019. The company was launched with a $21 million Series A financing and began lab operations at LabCentral in Cambridge, MA where it advanced its lead programs from concept to development candidate within one year. Immediately prior to the acquisition, certain earlier-stage vision restoration and vision preservation assets leveraging the company's ocular gene therapy toolbox were spun out into a newly formed entity – Vedere Bio II, Inc.

Read More

MEDTECH

Patritumab Deruxtecan Granted U.S. FDA Breakthrough Therapy Designation in Patients with Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

Daiichi Sankyo | December 24, 2021

Daiichi Sankyo Company, Limited announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to patritumab deruxtecan, a potential first-in-class HER3 directed antibody drug conjugate, for the treatment of patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer with disease progression on or after treatment with a third-generation tyrosine kinase inhibitor and platinum-based therapies. Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 While the efficacy of targeted therapy with EGFR TKIs is well-established in the treatment of advanced EGFR-mutated NSCLC, which comprises approximately 30% of patients, the development of a broad range of resistance mechanisms commonly leads to disease progression.4,5,6 After failure of an EGFR TKI, platinum-based chemotherapy has limited efficacy with progression-free survival (PFS) of approximately 4.4 to 6.4 months.7 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 The U.S. FDA’s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines. The FDA granted the BTD based on data from the dose escalation portion and two expansion cohorts of a three-cohort phase 1 study of patritumab deruxtecan. Extended follow-up data from the dose escalation portion and dose expansion cohort 1 of the study were recently presented at the 2021 American Society of Clinical Oncology annual meeting and published in Cancer Discovery. This is the first BTD for patritumab deruxtecan and the seventh BTD across Daiichi Sankyo’s oncology portfolio. “The Breakthrough Therapy Designation for patritumab deruxtecan acknowledges the need for new treatment approaches to overcome resistance and improve survival in patients with metastatic TKI-resistant, EGFR-mutated non-small cell lung cancer. We are proud that the FDA has once again recognized our innovative science and technology and we look forward to bringing this potential first-in-class HER3 directed antibody drug conjugate to patients with this specific type of lung cancer as quickly as possible.” Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo About Non-Small Cell Lung Cancer Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 There were an estimated 2.2 million new cases of lung cancer and 1.8 million deaths in 2020.9 NSCLC is diagnosed at an advanced stage in more than 50% of patients and often has a poor prognosis with worsening outcomes after each line of subsequent therapy.10,11,12 The introduction of targeted therapies and checkpoint inhibitors in the past decade has improved the treatment landscape for patients with advanced or metastatic NSCLC. For patients with advanced EGFR-mutated NSCLC, targeted therapy with EGFR TKIs offer higher response rates and PFS compared to chemotherapy.13 However, most patients eventually develop resistance to these therapies and subsequent therapy after EGFR TKI with platinum-based chemotherapy have limited efficacy with PFS of approximately 4.4 to 6.4 months.7,14 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 New treatment approaches are needed to overcome resistance and improve survival in this subtype of NSCLC. About HER3 HER3 is a member of the EGFR family of receptor tyrosine kinases, which are associated with aberrant cell proliferation and survival.15 Approximately 25% to 30% of lung cancers have an EGFR-activating mutation, and it is estimated that about 83% of all NSCLC tumors express the HER3 protein, which can be associated with an increased incidence of metastases, reduced survival and resistance to standard of care treatment.16,17,18 Currently, no HER3 directed medicines are approved for the treatment of cancer. About the Phase 1 Non-Small Cell Lung Cancer Study The global, multicenter, open label, two-part phase 1 study is evaluating patritumab deruxtecan in previously treated patients with metastatic or unresectable NSCLC. The dose escalation part of the study evaluated patients with EGFR-mutated disease either with progression on osimertinib or T790M-negative after progression on erlotinib, gefitinib or afatinib. The primary objective of this part of the study was to assess the safety and tolerability of patritumab deruxtecan and determine the recommended dose for expansion. The dose expansion part of the study is evaluating patritumab deruxtecan at the RD in three cohorts. Cohort 1 includes patients with locally advanced or metastatic EGFR-mutated NSCLC who experienced disease progression after taking one or more EGFR TKIs and one or more platinum-based chemotherapy regimens. Cohort 2 includes patients with squamous or non-squamous NSCLC without EGFR-activating mutations following platinum-based chemotherapy and following an anti-PD-1 or anti-PD-L1 antibody regimen. Cohort 3 includes patients with NSCLC with EGFR-activating mutations including any histology other than combined small cell and non-small cell lung cancer; patients in Cohort 3 are randomized 1:1 to receive the 5.6 mg/kg RDE regimen or an escalating up-titration regimen of patritumab deruxtecan. The primary objective of the dose expansion part of the study is to assess efficacy of patritumab deruxtecan as measured by confirmed objective response rate assessed by blinded independent central review. Secondary study endpoints include investigator-assessed ORR, safety and pharmacokinetics. The study enrolled patients at multiple sites in Asia, Europe and North America. For more information, visit ClinicalTrials.gov. About Patritumab Deruxtecan Patritumab deruxtecan is one of three lead DXd ADCs in the oncology pipeline of Daiichi Sankyo. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, patritumab deruxtecan is comprised of a fully human anti-HER3 IgG1 monoclonal antibody attached to a topoisomerase I inhibitor payload via a stable tetrapeptide-based cleavable linker. Patritumab deruxtecan is currently being evaluated in a comprehensive development program across multiple cancers as both a monotherapy and in combination with other anticancer treatments. The development program includes HERTHENA-Lung01, a pivotal phase 2 study in patients with locally advanced or metastatic EGFR-mutated NSCLC previously treated with a TKI and platinum-based chemotherapy; a phase 1/2 study in HER3 expressing metastatic breast cancer; a phase 1 study in combination with osimertinib in locally advanced/metastatic EGFR-mutated NSCLC; and, a phase 1 study in previously treated patients with metastatic or unresectable NSCLC. Patritumab deruxtecan is an investigational medicine that has not been approved for any indication in any country. Safety and efficacy have not been established. About Daiichi Sankyo in Oncology The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon, our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.”

Read More