On July 7, 2021 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted a supplemental Biologics License Application (sBLA) for anti-PD1 antibody tislelizumab for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy (Press release, BeiGene, JUL 7, 2021, View Source [SID1234584649]).

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"We are excited to submit the eighth marketing application for tislelizumab, of which five have been approved in China. As the first global Phase 3 ESCC clinical trial, the differences in clinical practice in various countries and regions have been fully taken into account, which indicates the result is fully globally representative." commented Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. "The results demonstrated improved efficacy of tislelizumab monotherapy over chemotherapy in second-line treatment for patients with locally advanced or metastatic ESCC and the potential to benefit patients with improved overall survival (OS) compared to chemotherapy. We look forward to further communication with CDE and hope this medicine will benefit Chinese patients with locally advanced or metastatic ESCC soon."

The sBLA is supported by clinical results from a randomized, open-label, multi-center, global Phase 3 clinical trial RATIONALE 302 (NCT03430843) to evaluate the efficacy and safety of tislelizumab as a second-line treatment for patients with locally advanced or metastatic ESCC compared to chemotherapy. The primary endpoint of this trial is overall survival (OS) in the intent-to-treat (ITT) population; a key secondary endpoint is OS in patients with high PD-L1 expression (defined as visually-estimated combined positive score [vCPS] ≥10%); and other secondary endpoints include progression-free survival (PFS), objective response rate (ORR), duration of response (DoR), and safety. A total of 512 patients were enrolled in the trial in 11 countries and regions in Asia, Europe, and North America, randomized 1:1 to either tislelizumab arm or chemotherapy arm (investigator’s choice of paclitaxel, docetaxel, or irinotecan). Results of this trial were presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

About Esophageal Squamous Cell Carcinoma (ESCC)

Esophageal cancer is one of the most common malignant tumors in the digestive tract. As the country with a high incidence of esophageal cancer, China accounts for 53.7% of the world’s new cases and 55.7% of the world’s deaths every yeari. Esophageal cancer is mainly divided into squamous cell cancer and adenocarcinoma, among which adenocarcinoma is the dominant cancer in European and American countries, accounting for about 70% of the overall incidence. In China, more than 95% of patients are esophageal squamous cell carcinomaii.

About Tislelizumab

Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

The China National Medical Products Administration (NMPA) has granted tislelizumab approval in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.

In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors and for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.

BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.

In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

Tislelizumab is not approved for use outside of China.

About the Tislelizumab Clinical Program

Clinical trials of tislelizumab include:

Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
BeiGene Oncology

BeiGene is committed to advancing best and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines to patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy volunteers. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries or regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. The Company currently markets three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks. BeiGene has also entered into a collaboration with Novartis granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

On July 7, 2021 Alector, Inc. (Nasdaq: ALEC) and GlaxoSmithKline plc (LSE/NYSE: GSK), reported a strategic global collaboration for the development and commercialization of two clinical-stage, potential first-in-class monoclonal antibodies (AL001 and AL101) designed to elevate progranulin (PGRN) levels (Press release, Alector, JUL 7, 2021, View Source [SID1234584646]). PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments.

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The collaboration brings together Alector’s leading immuno-neurology expertise with GSK’s R&D focus on the science of the immune system and human genetics, proven late-stage drug development capabilities and global footprint. Enrollment is currently underway for a pivotal Phase 3 trial for AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). FTD-GRN is a rapidly progressing and severe form of dementia found most frequently in people less than 65 years old at the time of diagnosis and has no approved treatments. AL001 is also currently in a Phase 2 study in symptomatic FTD patients with a mutation in the C9orf72 gene and is planned to enter Phase 2 development for amyotrophic lateral sclerosis (ALS) in the second half of 2021. AL101 is in a Phase 1a clinical trial and is designed to treat patients suffering from more prevalent neurodegenerative diseases, including Parkinson’s disease and Alzheimer’s disease.

Dr Hal Barron, Chief Scientific Officer and President R&D, GSK, said: "Our focus on human genetics and the science of the immune system gives us unique insights into the potential of targets such as progranulin to help patients with a number of neurodegenerative diseases. Working with Alector’s world class scientists will allow us to investigate the potential of these immuno-neurology therapies to help patients with frontotemporal dementia, a devastating disease without any currently approved treatments, as well as explore the ability to help patients with other neurodegenerative diseases, such as ALS, Parkinson’s and Alzheimer’s."

Arnon Rosenthal, Ph.D., Chief Executive Officer, Alector, said: "This transformative collaboration brings together Alector’s leading immuno-neurology expertise with GSK’s commitment to immunology and human genetics, proven drug development capabilities and global footprint, to help expand and accelerate the development of our progranulin franchise into large indications, while bolstering the build

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out of our own late-stage development and commercial capabilities. Importantly, this collaboration is designed to fully support AL001 and AL101’s development and to enable Alector to continue building a fully integrated company as we strive to address the high unmet medical need in patients suffering from neurodegenerative diseases. We are confident that GSK’s extensive experience launching ground-breaking medicines at the intersection of immunology and human genetics, will ensure that AL001 and AL101 are developed to their full potential."

As part of the recent Investor Update day on 23 June 2021, GSK committed to an R&D approach focused on maximizing opportunities by leveraging an increased understanding of the science of the immune system and human genetics. The collaboration with Alector on AL001 and AL101, two antibodies designed to elevate PGRN levels and potentially slow the progression of FTD and other neurological disorders, provides GSK access to a promising clinical program in immuno-neurology.

Terms of the Collaboration

Under the terms of the collaboration agreement, Alector will receive $700 million in upfront payments. In addition, Alector will be eligible to receive up to an additional $1.5 billion in clinical development, regulatory and commercial launch-related milestone payments.

Alector will lead the global clinical development of AL001 and AL101 through Phase 2 proof-of-concept. Thereafter, Alector and GSK will share development responsibilities for all late-stage clinical studies for AL001 and AL101 and all costs for global development will be divided between the two companies.

The companies will be jointly responsible for commercialization in the U.S. and will share profits and losses. Alector will lead commercial efforts associated with AL001 in orphan indications and GSK will lead the commercialization of AL101 in Alzheimer’s and Parkinson’s disease. Outside the U.S., GSK will be responsible for commercialization of AL001 and AL101 and Alector will be eligible for tiered royalties.

The collaboration agreement is conditional upon customary conditions including review by the appropriate regulatory agencies under the Hart-Scott-Rodino Act.

About the Progranulin-Elevating Monoclonal Antibodies – AL001 and AL101

Decreased levels of PGRN, a key regulator of immune response, lysosomal function, and neuronal survival in the brain, are genetically linked to many neurodegenerative disorders. AL001 and AL101 are novel human monoclonal antibodies that elevate levels of progranulin by blocking the sortilin receptor responsible for progranulin degradation. AL001 is currently in a pivotal Phase 3 clinical study in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). AL001 is also currently in a Phase 2 study in symptomatic FTD patients with a C9orf72 mutation, with another Phase 2 study in patients with ALS planned to begin in the second half of 2021. AL101, is designed to treat people suffering from more prevalent neurodegenerative diseases and is currently in a Phase 1a study in healthy volunteers. AL101 is intended to be developed for treatment of Parkinson’s disease and Alzheimer’s disease.

About Frontotemporal Dementia (FTD)

Frontotemporal dementia is a rapidly progressing and severe form of dementia. It affects 50,000 to 60,000 people in the United States and roughly 110,000 in the European Union, with potentially higher prevalence in Asia and Latin America. There are currently no FDA-approved treatment options for FTD.

There are multiple heritable forms of FTD. In one form, FTD-GRN, people have a mutation in the progranulin gene. This population represents 5% to 10% of all people with FTD. Mutations in a single copy of a progranulin gene leads to a 50% or greater decrease in the level of progranulin protein and invariably leads to development of FTD. In another form, people with mutations in the chromosome 9 open reading frame 72 (C9orf72) gene can develop FTD. FTD-C9orf72 is associated with abnormal accumulation of the protein TDP-43, which is also a hallmark in FTD-GRN. To date researchers have identified more than 120 inherited loss of function mutations in the progranulin gene that lead to FTD.

Alector Conference Call Information

Alector management will host a conference call to discuss the collaboration today at 8:30 a.m. ET. Analysts and investors are invited to participate in the conference call by dialling (888) 705-0365 from the U.S. and Canada or (415) 817-9241 internationally and using the conference ID 9476664. The live webcast can be accessed on the investor page of Alector’s website at investors.alector.com. A replay of the webcast will be available on Alector’s website approximately two hours after the completion of the event and will be archived for up to 30 days.

On July 7, 2021 Akari Therapeutics, Plc (Nasdaq: AKTX) ("Akari" or the "Company"), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, reported that it has entered into definitive securities purchase agreements with certain accredited and institutional investors, led by existing investors of the Company, including Dr. Ray Prudo, the Company’s Chairman, to receive gross proceeds of approximately $12 million through a private placement of its equity securities (Press release, Akari Therapeutics, JUL 7, 2021, View Source [SID1234584645]).

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In connection with the offering, the Company will issue unregistered American Depository Shares, each representing 100 of the Company’s ordinary shares ("ADSs"), at a purchase price of $1.55 per ADS. The closing of the offering is expected to take place during or before the week of July 12, 2021, subject to the satisfaction of customary closing conditions.

Paulson Investment Company, LLC, is acting as the exclusive placement agent in connection with this offering.

The ADSs described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act") and Regulation D promulgated thereunder, and have not been registered under the Act or state securities laws, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission or an applicable exemption from such registration requirements.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein. There shall not be any offer, solicitation of an offer to buy, or sale of securities in any state or jurisdiction in which such an offering, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On July 7, 2021 Aadi Bioscience, Inc. ("Aadi"), a privately-held clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, reported that Founder, Chief Executive Officer and President, Neil Desai, Ph.D., will present at the Ladenburg Thalmann 2021 Virtual Healthcare Conference on Tuesday, July 13, 2021 (Press release, Aadi Bioscience, JUL 7, 2021, View Source [SID1234584644]).

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Presentation Information:

Date: Tuesday, July 13, 2021

Time: 2:30 – 2:55 pm ET

Location: Track 2

The live webcast can be accessed here: View Source

On July 7, 2021 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that Dmitri Wiederschain, Ph.D., Chief Scientific Officer will participate in a panel discussion at the William Blair’s Biotech Focus Conference taking place virtually on Wednesday, July 14, 2021 at 4:20 PM ET (Press release, Jounce Therapeutics, JUL 7, 2021, View Source [SID1234584643]).

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A webcast of the panel will be available by visiting "Events and Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. A replay of the webcast will be archived for 30 days following the presentation.