Blueprint Medicines and Zai Lab Announce Strategic Collaboration and License Agreement for BLU-945 and BLU-701 in Greater China

On November 9, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) and Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported an exclusive collaboration and license agreement for the development and commercialization of BLU-945 and BLU-701 for the treatment of patients with epidermal growth factor receptor (EGFR) -driven non-small cell lung cancer (NSCLC) in Greater China, including mainland China, Hong Kong, Macau and Taiwan (Press release, Blueprint Medicines, NOV 9, 2021, View Source [SID1234594834]). Discovered by Blueprint Medicines, BLU-945 and BLU-701 are investigational next-generation EGFR inhibitors with first-in-class potential.

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By combining Blueprint Medicines’ precision therapy expertise with Zai Lab’s development capabilities and established lung cancer franchise in Greater China, the collaboration aims to accelerate global development of BLU-945 and BLU-701 while addressing significant medical needs in China, where 40-50 percent of patients with NSCLC are believed to harbor EGFR mutations.1,2,3 Blueprint Medicines will retain all rights to BLU-945 and BLU-701 in the rest of the world.

"With deep development and commercial expertise in oncology across a broad portfolio including multiple precision therapies for lung cancer, Zai Lab is the ideal partner to help us bring to China our vision for transforming the care of patients with EGFR-driven lung cancer," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Through this collaboration, we will also propel forward our development program for BLU-945 and BLU-701 with a broad clinical trial footprint in Greater China that complements our development efforts."

"We are excited to enter into this collaboration with Blueprint Medicines, a leader in precision medicine, to bring forward two potential first-in-class EGFR inhibitors exquisitely designed to treat or prevent on-target resistance," said Dr. Samantha Du, Founder, Chairwoman and Chief Executive Officer of Zai Lab. "With more than 800,000 newly diagnosed lung cancer patients annually, one of the highest EGFR mutation rates in the world, and with no available therapies to address on-target resistance to early-generation EGFR therapies, we believe we have a tremendous opportunity to improve patient care in China." 1-4

While targeted therapies have improved treatment for patients with EGFR-driven NSCLC, resistance inevitably emerges, with the T790M and C797S mutations being highly common on-target resistance mechanisms. Designed to address these challenges, BLU-945 and BLU-701 have the potential to be used either as a monotherapy or in combination, together or with other agents, to overcome or prevent on-target resistance across multiple lines of treatment. In addition, this collaboration enables opportunities to combine BLU-945 or BLU-701 with other Zai Lab lung cancer drug candidates to address off-target resistance mutations.

BLU-945 is a selective, potent EGFR tyrosine kinase inhibitor with activity against EGFR activating mutations combined with the T790M and C797S resistance mutations. It is highly selective over wild-type EGFR and off-target kinases, highlighting its potential to enable tolerable combinations. BLU-945 is currently being evaluated in the Phase 1/2 SYMPHONY trial in patients with previously treated EGFR-driven NSCLC (NCT04862780). BLU-701 is a selective, potent EGFR tyrosine kinase inhibitor with activity against EGFR activating mutations combined with the C797S resistance mutation. It has shown significant central nervous system (CNS) penetration in preclinical studies, which is meaningful because in EGFR-mutant NSCLC patients with baseline brain metastases, up to 40 percent of disease progressions involve CNS metastases.5

Subject to the terms of the agreement, Blueprint Medicines will receive an upfront cash payment of $25 million and will be eligible to receive up to $590 million in potential development, regulatory and sales-based milestone payments, and tiered royalties on a product-by-product basis ranging from the low-teens to mid-teens on annual net sales of BLU-945 and BLU-701 in Greater China, subject to adjustment in specified circumstances. In addition, Zai Lab will be responsible for all the development costs for BLU-945 and BLU-701 occurring in Greater China and will receive the rights to develop and exclusively commercialize BLU-945 and BLU-701 in the region.

About EGFR-Driven NSCLC in China

Lung cancer is the most commonly diagnosed cancer type and the leading cause of cancer death in China.1 Annually, there are more than 800,000 new cases of lung cancer in China, of which approximately 85 percent are NSCLC.1,6 EGFR mutations are more common in China than in the United States, occurring in 40-50 percent of NSCLC patients.1 Third-generation EGFR-tyrosine kinase inhibitors, including osimertinib, are commonly prescribed in China and have emerged as the standard of care for the first-line setting. However, resistance inevitably emerges, leading to disease progression. There are no approved therapies for patients with disease progression following third-generation EGFR treatment.

About BLU-945 and BLU-701

Derived from Blueprint Medicines’ proprietary research platform, BLU-945 and BLU-701 are investigational next-generation EGFR non-covalent tyrosine kinase inhibitors. Both treatments are specifically designed to provide comprehensive coverage of the most common activating and on-target resistance mutations, spare wild-type EGFR and other kinases to limit off-target toxicities and enable a range of combination strategies, and treat or prevent central nervous system metastases. BLU-945 is currently being evaluated in the Phase 1/2 SYMPHONY trial in patients with previously treated EGFR-driven NSCLC (NCT04862780). In addition, Blueprint Medicines plans to initiate a Phase 1/2 trial of BLU-701 in the fourth quarter of 2021.

Rubius Therapeutics to Participate in Jefferies London Healthcare Conference

On November 9, 2021 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune diseases, reported that Pablo J. Cagnoni, M.D., president and chief executive officer, will provide a corporate update at the Jefferies London Healthcare Conference via a virtual presentation, which will be available to view on the conference website on November 18-19, 2021 (Press release, Rubius Therapeutics, NOV 9, 2021, View Source [SID1234594833]).

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The recorded audio presentation will be available within the Investors & Media section of the Rubius Therapeutics website on November 18th beginning at 8:00 a.m. GMT/3:00 a.m. ET. An archived replay will be accessible for 90 days following the event.

Preclinical Data Supporting Therapeutic Potential of Surface Oncology’s Lead Clinical Programs, SRF617 and SRF388, Presented at the Society for Immunotherapy of Cancer 2021 Annual Meeting

On November 9, 2021 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that two scientific posters sharing updated preclinical data from Surface Oncology’s two lead clinical-stage antibody therapies, SRF617 (targeting CD39) and SRF388 (targeting IL-27), will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2021 Annual Meeting, to be held virtually and at the Walter E. Washington Convention Center in Washington, D.C. November 10-14, 2021 (Press release, Surface Oncology, NOV 9, 2021, View Source [SID1234594832]).

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"The data to be presented highlight SRF617’s potent CD39 inhibition in preclinical models, and its effect on promoting proinflammatory therapeutic activity," said Vito Palombella, Ph.D., chief scientific officer at Surface Oncology. "Additionally, we will provide evidence that the IL-27 gene signature is highly expressed in patients with lung cancer that have treatment-resistant disease, supporting our continued development of SRF388, our first-in-class IL-27 antibody, across numerous tumor types."

Highlights of the posters are provided below. Full posters will be placed on Surface Oncology’s website following the presentations.

SRF617 Highlights
In a poster entitled "The fully human antibody SRF617 is a potent inhibitor of ecto-enzyme CD39 in vivo," Surface researchers describe preclinical studies demonstrating the therapeutic potential of targeting CD39 for cancer treatment:

SRF617 is an inhibitor of CD39 enzymatic activity in vivo.
Anti-CD39 therapy promotes a pro-inflammatory tumor microenvironment by increasing CD8+ T cell accumulation and downregulating CD39 protein on immune cells.
In combination with gemcitabine, anti-CD39 therapy promotes an increase in tumor macrophages while reducing CD39 expression and activity.
SRF388 Highlights
In a poster entitled "IL-27 signaling drives a type 1 interferon-like gene expression program of immunoregulatory pathways associated with cancer progression," Surface researchers demonstrate that blockade of IL-27 alleviates an immunosuppressive gene transcriptional program implicated in treatment resistance in cancer:

IL-27 induces robust gene expression in human immune cells and cancer cell lines that include several inhibitory receptors and canonical interferon-regulated genes.
Both IL-27 and IFNβ can counteract some of the immune stimulatory properties of PD-1 blockade.
IL-27 is expressed by a macrophage population associated with progressive disease in patients with non-small cell lung cancer (NSCLC).
These studies elucidate the transcriptional networks engaged after IL-27 signaling in immune and cancer cells and highlight the parallels with interferon-associated immune regulation.

Ziopharm Oncology Presents Preclinical Data Supporting TCR-T Library Approach at the Society for Immunotherapy of Cancer 2021 Annual Meeting

On November 9, 2021 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported the presentation of preclinical data highlighting the potential of neoantigen-specific TCR-T cells for the treatment of solid tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2021 Annual Meeting (Press release, Ziopharm, NOV 9, 2021, View Source [SID1234594831]).

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"We are pleased to share preclinical data demonstrating the versatility of our Sleeping Beauty technology to develop neoantigen-specific TCR-T cells with the potential to address a wide range of solid tumor indications," commented Raffaele Baffa, M.D., Ph.D., Chief Medical Officer & EVP, Research & Development. "Our TCR-T library approach allows us to develop safe, effective and durable therapies for any patient with a matching neoantigen/HLA combination within our library. We are working diligently to initiate our Phase 1/2 TCR-T Library trial in the first half of 2022 as well as continue to expand our library of TCRs in order to increase the pool of eligible patients who could benefit from these therapies."

Details of Ziopharm’s SITC (Free SITC Whitepaper) 2021 presentation are as follows:

Title: Neoantigen-specific TCR-T cells targeting shared hotspot mutations for adoptive cell therapy in common epithelial cancers
Presenter: Drew Deniger, Ph.D., Ziopharm Oncology
Date/Time: Saturday, Nov. 13, 2021 from 7:00 a.m. – 8:30 p.m. ET
Location: Walter E. Washington Convention Center, Hall E
Abstract Number: 226

In the study presented at SITC (Free SITC Whitepaper), Ziopharm’s non-viral gene transfer technology, Sleeping Beauty, was used to develop TCR-T cells targeting EGFR, KRAS and p53 neoantigens, which are present in cancer cells but not in normal tissue, and are presented by a variety of human leukocyte antigen (HLA) molecules on the cancer cell surface. Ziopharm’s Sleeping Beauty technology exhibited greater than 60% expression of the introduced neoantigen-specific TCRs in the generated TCR-T cells. The study demonstrated that the neoantigen-specific TCR-T cells successfully targeted tumor cells expressing EGFR, KRAS and p53 neoantigens in conjunction with the matching HLA restriction. Importantly, these TCR’s did not recognize cells lacking the matching mutations and HLA restrictions.

Istari Oncology Presents Data on Its PVSRIPO Immunotherapy at the Annual November Meetings for the Society for Immunology of Cancer (SITC) and the Society for Neuro-Oncology (SNO)

On November 9, 2021 Istari Oncology, Inc., a clinical-stage biotechnology company developing novel immunotherapies for the treatment of solid tumors, reported it will be presenting data on its investigative immunotherapy PVSRIPO for the potential treatment of solid tumors at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC; Washington D.C.; November 10-14) and the 26th Annual Meeting of the Society for Neuro-Oncology (SNO; Boston; November 18 – 21) (Press release, Istari Oncology, NOV 9, 2021, View Source [SID1234594830]).

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PVSRIPO is an investigational immunotherapy based on the live–attenuated Sabin type 1 poliovirus vaccine that has been genetically modified for safety. PVSRIPO has been shown to activate a patient’s innate and adaptive immune system to facilitate a systemic anti-tumor immune response. Because PVSRIPO utilizes CD155 (the poliovirus receptor) to enter both solid tumor cells and antigen–presenting cells (APCs) in the tumor microenvironment, PVSRIPO has the potential to treat a variety of cancers.

Details of Istari Oncology’s SITC (Free SITC Whitepaper) Presentations – Focusing on PVSRIPO’s recall MOA and recent expansion into head and neck cancer
Title: Poster Presentation: (517) LUMINOS-103: A basket trial evaluating the safety and efficacy of PVSRIPO and PVSRIPO in combination with anti-PD-1/L1 checkpoint inhibitors in patients with advanced solid tumors
Presenter: Brant A. Inman MD, MS
Presentation time: 11/12/2021 from 7:00 – 8:30 p.m. EST
Location: Poster Hall, Walter E. Washington Convention Center, Washington D.C.

Title: Poster Presentation: (739) Intratumor childhood vaccine-specific CD4+ T cell recall helps antitumor CD8 T cells
Presenter: Michael Brown, PhD
Presentation time: 11/12/2021 from 7:00 a.m. – 8:30 p.m. EST
Location: Poster Hall, Walter E. Washington Convention Center, Washington D.C.

Details of Istari Oncology’s SNO Presentations – Focusing on preclinical and clinical safety data supporting the company’s work in glioblastoma
Title: Poster Presentation: (CTIM-18) LUMINOS-101: Initial safety and tolerability of PVSRIPO and pembrolizumab combination therapy in recurrent glioblastoma
Presenter: Andrew Sloan, MD
Presentation time: 11/18 – 11/21 from 7:30 – 9:30 p.m. EST
Location: Exhibit Hall D, Hynes Convention Center, Boston, MA

Title: Poster Presentation: (BIOM-20) Tumor-intrinsic and peripheral features associate with survival after polio virotherapy in recurrent GBM
Presenter: Michael Brown, PhD
Presentation time: 11/18 – 11/21 from 7:30 – 9:30 p.m. EST
Location: Exhibit Hall D, Hynes Convention Center, Boston, MA

Title: Abstract Presentation: (IMMU-26) Safety and efficacy of PVSRIPO in recurrent glioblastoma: long-term follow-up and initial multicenter results
Presenter: Annick Desjardins, MD, FRCPC
Presentation time: 11/19 from 4:50 – 4:55 p.m. EST
Location: Room 207, Hynes Convention Center, Boston, MA

Title: Abstract Presentation: (EXTH-77) Polio virotherapy of murine brain tumors includes microglia proliferation and inflammation that is potentiated by immune checkpoint blockade
Presenter: Yuanfan Yang, MD
Presentation time: 11/21 from 11:50 – 11:55 a.m. EST
Location: Room 208, Hynes Convention Center, Boston, MA

For more information about Istari Oncology and its ongoing clinical trials, visit www.istarioncology.com.

About PVSRIPO
PVSRIPO is an investigational immunotherapy based on the live-attenuated Sabin type 1 poliovirus vaccine that has been genetically modified for safety. PVSRIPO targets cells using the poliovirus receptor CD155, which is widely expressed on both the malignant cells of most solid tumors and key antigen-presenting cells (APCs) within the tumor microenvironment. PVSRIPO targets tumors using three key mechanisms: 1) engagement and activation of APCs, leading to T cell priming and sustained, systemic anticancer immunity; 2) direct tumor cell killing and antigen release; and 3) amplification of the immune response via recall of poliovirus vaccine-specific T cells. PVSRIPO has been granted Breakthrough Therapy and Orphan Drug Designation status by the U.S. Food and Drug Administration in recurrent glioblastoma, and Fast Track and Orphan Drug Designation status in refractory melanoma.