On May 11, 2021 NANOBIOTIX (Euronext : NANO – NASDAQ: NBTX), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported a partnership with LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and major Asian markets, to develop and commercialize Nanobiotix lead product candidate NBTXR3, a potential first-in-class radioenhancer, in Greater China (mainland China, Hong Kong, Taiwan, and Macau), South Korea, Singapore and Thailand. (Press release, Nanobiotix, MAY 11, 2021, View Source [SID1234579720])

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Shanghai and Princeton-based LianBio was founded by Perceptive Advisors with an innovative partnership model to develop and commercialize therapeutics in China and other Asian markets. LianBio’s cross-border development and commercialization expertise includes strong capabilities in oncology and NBTXR3 is the third investigational cancer treatment in the company’s portfolio. Given the global high unmet need in locally advanced and metastatic cancers, along with data supporting the potential of NBTXR3 across solid and metastatic tumor types, Nanobiotix continues to leverage strategic relationships with world class collaborators to expand and accelerate its pipeline while prioritizing Company resources to support development in head and neck cancer and immunotherapy.

"Discovery of practice-changing therapeutics for major diseases through a physics-based approach, with the support of people committed to making a difference for humanity, is our mission at Nanobiotix. We have long believed that NBTXR3 will significantly improve treatment outcomes for patients with any solid or metastatic tumor, and we are grateful that a partner with the talent and capability of LianBio has agreed to walk with us in our journey. Cancer is a disease that knows no borders and bringing our innovation to patients in Greater China with speed and determination is an absolute necessity," said Nanobiotix CEO and co-founder Laurent Levy.

"We purpose-built LianBio with a next-generation licensing model that enables us to meaningfully contribute to our partners’ global development initiatives in order to accelerate the availability of transformative therapeutics for patients throughout Asia," said Konstantin Poukalov, Executive Chairman of LianBio and Managing Director of Perceptive Advisors. "We believe NBTXR3 has a highly targeted nature and broadly applicable mechanism of action and thus has the potential to change radiotherapy and immuno-oncology treatment paradigms by addressing key limitations of current standards of care."

"The NBTXR3 therapeutic approach offers a promising avenue to address the significant burden of disease across multiple tumor types," said Debra Yu, M.D., President and Chief Business Officer, LianBio. "Our partnership will provide Nanobiotix with access to LianBio’s regional expertise and is designed to enable Nanobiotix to reach the growing number of cancer patients in China and other Asian markets who are in need of improved treatment options."

Under the terms of the agreement, LianBio will obtain exclusive rights to develop and commercialize NBTXR3 in Greater China, South Korea, Singapore, and Thailand. Nanobiotix will receive a $20 million upfront payment and is entitled to receive up to an aggregate of $220 million in potential contingent, development and commercialization milestone payments. Nanobiotix will also be eligible to receive tiered, low double-digit royalties based on net sales of NBTXR3 in the licensed territories. LianBio will participate in the Nanobiotix global phase III registrational study evaluating NBTXR3 for patients with locally advanced head and neck squamous cell carcinoma (HNSCC; head and neck cancer) by enrolling 100 patients in China in the study. In addition to the phase III head and neck cancer study, LianBio has committed to enroll patients in four additional registrational studies conducted by Nanobiotix across indications and therapeutic combinations potentially including immunotherapy. LianBio will fund all development and commercialization expenses in the collaboration territory, and Nanobiotix will continue to fund all development and commercialization expenses in all other geographies.

About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

NBTXR3 is being evaluated primarily in locally advanced head and neck squamous cell carcinoma (HNSCC). The company-sponsored phase I dose escalation and dose expansion study has produced consistently favorable safety data and early signs of efficacy; and a phase III global registrational study is planned to launch in 2021. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the planned phase III study.

Nanobiotix has also prioritized an Immuno-Oncology development program—beginning with a Company-sponsored phase I clinical study evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/metastatic HNSCC and lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a strategic collaboration strategy with world class partners to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 The University of Texas MD Anderson Cancer Center entered into a broad, comprehensive clinical research collaboration with Nanobiotix to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations.

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On May 11, 2021 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, reported that Dr. William V. Williams, President & CEO of BriaCell, is scheduled to participate in the following virtual conferences (Press release, BriaCell Therapeutics, MAY 11, 2021, View Source [SID1234579657]):

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• Benzinga Global Small Cap Investor Conference: May 13-14, 2021
o Dr. Williams will deliver his corporate presentation at 12:20pm ET on May 13, 2021.
o Dr. Williams will also participate in a panel discussion titled "Investing Post COVID – Back to the Fundamentals" at 11:50am ET on May 13, 2021. The panel will discuss healthcare investing in the post COVID world.
o Investors may request one-on-one meetings with Dr. Williams.
o Investors may register for the conference here: View Source

• Q2 Investor Summit: May 17-18, 2021
o Dr. Williams will deliver his corporate presentation at 2:00pm ET on May 17, 2021.
o Investors may request one-on-one meetings with Dr. Williams.
o Investors may register for the conference here: View Source

• 2021 LD Micro Invitational Investor Conference: June 8-10, 2021
o Dr. Williams will deliver his corporate presentation at 12:00pm ET on June 9, 2021.
o Investors may request one-on-one meetings with Dr. Williams.
o Investors may register for the conference here: View Source

• MarketsandMarkets 4th Annual Next Gen Immuno-Oncology Virtual Congress-US Edition: June 28-30, 2021
o Dr. Williams will be speaking at the scientific conference at 11:30am ET on June 29, 2021.
o The topic is "Personalized, off-the-shelf cellular immunotherapy for cancer".
o A copy of the presentation will be posted here: View Source
o For additional information on the conference and the speaker, please visit:
View Source

Separately, BriaCell has recently appointed Miguel A. Lopez-Lago, Ph.D. as Senior Director, Research and Development. Since 2000, Dr. Lopez-Lago has been working as a cancer scientist at Memorial Sloan-Kettering Cancer Center, New York (MSKCC). Specifically, he has investigated various aspects of tumor biology, including the development of targeted therapies for Mesothelioma and the characterization of the biological mechanisms underlying cancer metastasis. More recently, Dr. Lopez-Lago has been interested in the study of the tumor immune-microenvironment and in the development of immunotherapies for thoracic cancers using chimeric antigen receptor (CAR) T cell technologies. Since 2013, Dr. Lopez-Lago has been working as Senior Research Scientist at MSKCC. Dr. Lopez-Lago received his Bachelor of Science in Bio-Sciences and his doctorate in Molecular Biology from Santiago of Compostela University, Spain.

On May 11, 2021 Aileron Therapeutics (NASDAQ:ALRN), a chemoprotection oncology company focused on fundamentally transforming the experience of chemotherapy for cancer patients, reported business highlights and financial results for the first quarter ended March 31, 2021 (Press release, Aileron Therapeutics, MAY 11, 2021, View Source [SID1234579672]).

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"We continue to work diligently to initiate our first randomized, double-blind, placebo-controlled clinical trial of ALRN-6924 as a novel therapeutic to protect non-small cell lung cancer (NSCLC) patients receiving first-line chemotherapy against various chemotherapy-induced toxicities. Approximately 50% of NSCLC patients have a p53 mutation, suggesting the broad potential impact of ALRN-6924 in this indication alone," said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer.

Dr. Aivado further commented, "Simultaneously, we are actively planning for the rapid advancement of ALRN-6924 into late-stage development for patients with p53-mutated NSCLC pending the outcome of the Phase 1b trial. Bolstered by our successful capital raises in the first quarter of 2021, we are making near-term strategic investments in CMC, p53 companion diagnostic development, and team scale-up which we expect will not only enable the future advancement of our NSCLC clinical program, but also will provide foundational support for future clinical programs of ALRN-6924 across multiple p53-mutated cancer indications."

Aileron is developing ALRN-6924 to selectively protect healthy cells in patients with cancers that harbor p53 mutations, which are present in over half of all cancer patients, to reduce or eliminate chemotherapy-induced side effects while not interfering with chemotherapy’s attack on cancer cells. This novel concept is known as chemoprotection. By reducing or eliminating multiple chemotherapy-induced side effects, ALRN-6924 may improve patients’ quality of life and help them better tolerate chemotherapy. Enhanced tolerability may result in fewer dose reductions or delays of chemotherapy and the potential for improved efficacy as a result. Given Aileron’s p53 biomarker approach, designed to ensure selective chemoprotection only in healthy cells, coupled with the high prevalence of p53-mutated cancers, the company’s strategy is to ultimately pursue a tumor-agnostic label for ALRN-6924 as a chemoprotective agent in p53-mutated cancers.

First Quarter 2021 Highlights and Recent Updates

Aileron to host upcoming KOL Fireside Chat, "Protecting Cancer Patients from Chemotherapy-Induced Toxicities – A New Paradigm". Moderated by Soumit Roy, Ph.D., Vice President, Healthcare Analyst with JonesTrading Institutional Services LLC, the event will include a discussion on the unmet need and potential for chemoprotection with Alan List, M.D. and Lodovico Balducci, M.D. Dr. List, who recently was appointed Chief Medical Officer of Precision Biosciences, is a renowned hematologist with extensive academic and clinical experience in the research and development of hematology and oncology products. Dr. List is also a member of Aileron’s Scientific Advisory Board. Dr. Balducci, a prominent geriatric oncologist, previously served as Senior Member of the Senior Adult Oncology Program and Medical Director of Affiliates and Referring Physician Relations at the Moffitt Cancer Center. Link here to register for the event, which will take place on Wednesday, May 26, 2021.
Abstract co-authored by Aileron and Foundation Medicine, Inc. to be presented at 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. The abstract, titled, "Gene Sequencing of Serial Tumor Biopsies from a Large Cohort of Cancer Patients Shows Longitudinal Changes in TP53 Mutation Status Are Uncommon" (Abstract #3124), was accepted as a poster presentation at the meeting. Additional details will be announced upon ASCO (Free ASCO Whitepaper)’s online publication of this year’s abstracts later this month.
Announced enrollment expansion for upcoming Phase 1b clinical trial of ALRN-6924 in patients with advanced NSCLC. In February 2021, Aileron announced a 50% expansion of its enrollment target for its upcoming Phase 1b clinical trial of ALRN-6924 in patients with NSCLC undergoing chemotherapy. Aileron plans to enroll 60 patients with advanced p53-mutated NSCLC undergoing treatment with first-line carboplatin plus pemetrexed (with or without immune checkpoint inhibitors). Aileron anticipates this enrollment expansion will enable a more robust exploration of ALRN-6924 as a novel chemoprotective agent to prevent chemotherapy-induced toxicities in the NSCLC patient population and better position the company to rapidly advance ALRN-6924 into late-stage clinical development for NSCLC following the Phase 1b trial.

In the Phase 1b NSCLC trial, patients will be randomized 1:1 to receive either carboplatin/pemetrexed plus 0.3 mg/kg ALRN-6924 or carboplatin/pemetrexed plus placebo for at least four 21-day treatment cycles. Evaluations will include the proportion of treatment cycles free of severe hematological and other toxicities, transfusions and the use of growth factors, as well as the impact on quality of life. Aileron plans to initiate the NSCLC trial in the second quarter of 2021 and anticipates reporting early interim data at the end of 2021 and topline results in mid-2022.
During the first quarter, Aileron completed its Phase 1b evaluation of ALRN-6924 at the recommended phase 2 dose of 0.3 mg/kg in patients with SCLC receiving ALRN-6924 24-hours prior to topotecan and conducted preliminary evaluation of data from additional cohorts. These preliminary findings from 11 additional patients (n=7 patients receiving 0.3 mg/kg ALRN-6924 six hours before topotecan and n=4 patients receiving 0.2 mg/kg ALRN-6924 twenty-four hours before topotecan) were in line with data presented last October and with Aileron’s expectation that administering ALRN-6924 at 0.3mg/kg and 24 hours before topotecan remains the optimal dose and schedule in this patient population. Aileron expects to present results of the Phase 1b SCLC trial at a scientific conference in the second half of 2021.

Raised $55.7 million in aggregate proceeds from the sale of common stock during first quarter 2021, which based on Aileron’s current operating plan, is expected to provide funding into the second half of 2023 and approximately 12 months beyond the anticipated topline results from the NSCLC trial. In January 2021, Aileron completed a registered direct offering of common stock, for a purchase price of $1.10 per share, raising $33.1 million in aggregate net proceeds, after deducting placement agent fees and other offering expenses payable by Aileron. New fundamental and institutional investors, including Acorn Bioventures, BVF Partners, L.P., Maven Investment Partners and Grand Oaks Capital, participated in the offering, in addition to several existing Aileron investors, including Satter Medical Technology Partners and Lincoln Park Capital Fund, LLC. In addition to the registered direct offering, since January 1, 2021, Aileron sold an aggregate of 13,775,399 shares of its common stock for an average purchase price of $1.64 per share, for aggregate net proceeds of $22.6 million, after deducting fees and offering expenses, in "at the market" offerings and under its structured equity line with Lincoln Park Capital Fund, LLC.
Continuing healthy volunteer study to support long-term clinical development strategy for ALRN-6924. The ongoing study, initiated in November 2020, is designed to characterize the time to onset, magnitude, and duration of cell cycle arrest in human bone marrow relative to ALRN-6924 administration. Another goal of the study is to develop a universal dosing regimen for ALRN-6924 for use as a chemoprotection agent across a range of chemotherapies and p53-mutated tumor indications. Aileron expects to present results from the healthy volunteer study at a scientific conference in the second half of 2021.
First Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and investments at March 31, 2021 were $63.4 million, compared to $13.8 million at December 31, 2020. The company expects, based on its current operating plan, that its existing cash, cash equivalents and investments will fund operations into the second half of 2023.

Research and Development (R&D) Expenses: R&D expenses for the quarter ended March 31, 2021 were $4.3 million, compared to $4.1 million for the corresponding quarter in 2020. The increase of $0.2 million is primarily a result of increased spending on clinical development of ALRN-6924 in connection with the preparation for the upcoming Phase 1b clinical trial in patients with advanced NSCLC patients being treated with first-line chemotherapy of carboplatin and pemetrexed and is partially offset by the effect of cost savings measures implemented in 2020 resulting in decreased spending on employee, facility and other development expenses.

General and Administrative (G&A) Expenses: G&A expenses for the quarter ended March 31, 2021 were $2.7 million, compared to $2.8 million for the corresponding quarter in 2020. The decrease in general and administrative expense is the result of lower headcount and facility costs during the three months ended March 31, 2021 as a result of cost savings measures as compared to the three months ended March 31, 2020 and is partially offset by increased spending on professional services.

Net Loss: Net loss for the quarter ended March 31, 2021 was $7.0 million, compared to $6.7 million for the corresponding quarter in 2020. The basic and diluted net loss per share for the first quarter of 2021 was $0.08 compared to $0.24 for the first quarter of 2020. The change in basic and diluted net loss per share is primarily a result of increased shares outstanding in connection with sales of common stock during the first quarter of 2021.

On May 11, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported preclinical data from its HiT targeting mesothelin, being co-developed with Astellas, during a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) (Free ASGCT Whitepaper) meeting today (Press release, Adaptimmune, MAY 11, 2021, View Source [SID1234579688]).

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"These data are very exciting because our HLA-independent TCR targeting mesothelin induced complete regression in a mouse tumor xenograft model and also outperformed a TRuC construct that we developed in-house," said Karen Miller, SVP, Pipeline Research. "The natural signaling machinery of the TCR, as well as our proprietary TCR engineering and affinity optimization expertise, offer distinct advantages over cell therapies that rely on antibody moieties to engage antigen. We are evaluating further HiT targets, as we deliver against our 2-2-5-2 strategic plan to bring new cell therapy products forward for clinical development and launch."

Adaptimmune has developed a T-cell receptor (TCR) that can directly bind to the cell surface protein mesothelin, independent of HLA, using the Company’s proprietary naïve phage display libraries and affinity optimization techniques. The HLA-independent TCR or HiT platform enables the Company to target extracellular proteins, such as those utilized by CAR or TRuC T-cell therapies. Data from the ASGCT (Free ASGCT Whitepaper) presentation are summarized below.

Adaptimmune’s HiTs are CD8 and HLA-independent and kill mesothelin expressing human tumor cells

Human T-cells expressing the mesothelin-targeted HiT kill human tumor cell lines and primary human lung tumor cells expressing mesothelin
Data demonstrate that the mesothelin HiT is not dependent on CD8 to kill target cells, and CD4 cells expressing the HiT are cytotoxic towards tumor cells
A TRuC construct was developed by Adaptimmune as a comparator for these experiments, which also killed mesothelin target cells independently of CD8
Adaptimmune’s HiTs are not neutralized by free mesothelin, but the TRuC construct is

Data from additional in vitro experiments clearly show that Adaptimmune’s HiT is not neutralized by soluble mesothelin, unlike the TRuC construct
It has been described that CAR-T and similar therapies, which rely on high affinity antibody moieties to engage antigen, can be neutralized by soluble target protein, inhibiting their function1
This is particularly important for many tumor targets that are both cell-surface bound and secreted, as is the case with mesothelin
Adaptimmune’s HiT T-cells targeting mesothelin induce complete tumor regression in a mouse xenograft tumor model and outperform a comparator TRuC construct

Immunodeficient mice were implanted with human pancreatic Capan-2 mesothelin-expressing tumor cells
Mice with pre-established tumors received HiT T-cells or TRuC comparator T-cells by a single IV injection
Data showed a strong, dose-dependent, and persistent tumor regression with HiT T-cells
TRuC T-cells only inhibited tumor growth at comparative doses to those used with the HiT T-cells