Repare Therapeutics to Participate in Two Upcoming Investor Conferences

On September 7, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported that members of its senior management team will participate at two investor conferences in September (Press release, Repare Therapeutics, SEP 7, 2022, View Source [SID1234619183]). Details for the fireside chats are as follows:

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Morgan Stanley 20th Annual Global Healthcare Conference
Date: Wednesday, September 14, 2022
Time: 2:55 p.m. Eastern Time
Location: New York, NY

Guggenheim Nantucket Therapeutics Conference
Date: Wednesday, September 28, 2022
Time: 8:00 a.m. Eastern Time
Location: Nantucket, MA

A live webcast of the fireside chats can be accessed in the Investor section of the Company’s website at View Source A replay of the webcasts will be archived on the Company’s website for 90 days.

Orbital Therapeutics Launches to Advance New Frontiers of Science with the Next Generation of Innovative RNA Medicines

On September 7, 2022 Orbital Therapeutics reported that launched with a vision of enhancing global health by unleashing the full potential of RNA medicines to treat human disease in ways that were not previously possible (Press release, Beam Therapeutics, SEP 7, 2022, View Source [SID1234619182]).

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"The breakthroughs in RNA therapeutics over the last decade have been remarkable achievements by the biopharmaceutical industry, with several approved products for a range of diseases and many more in development. This frontier of science represents the future of medicine, and we are just beginning to realize the full breadth of its applications in treating a wide range of serious diseases," said John Maraganore, Ph.D., Orbital co-founder and chairman of the board of directors. "Orbital Therapeutics has a unique opportunity to integrate and apply a spectrum of innovative RNA technologies to advance a portfolio that could dramatically expand the potential of today’s RNA therapeutic approaches."

Game-Changing RNA Vision
RNA-based medicines represent a fast-growing and disruptive class of therapeutics for a breadth of disease areas. First-generation RNA treatments successfully overcame multiple hurdles, such as the rapid degradation of exogenous RNA, delivery of RNA and complications associated with immunogenicity.

To further broaden the application of this important class of treatments, Orbital is building a first-in-kind RNA platform that integrates both established and emerging technologies and delivery mechanisms, excluding RNAi. This platform is designed to extend the durability and half-life of Orbital’s novel RNA therapeutics, while also expanding their delivery to a larger number of cell types and tissues. As the company grows, Orbital intends to continue investing in the growth of its platform capabilities and technologies.

With a platform that provides access to state-of-the-art RNA and novel delivery technologies, Orbital plans to build an expansive portfolio across a range of human diseases, including in the areas of vaccines, immunomodulation, protein replacement and regenerative medicine.

Collaboration with Beam Therapeutics
As part of its launch, Orbital and Beam Therapeutics have entered into a license and research collaboration under which Orbital and Beam have each granted the other access to respective RNA technology and non-viral delivery technology. Orbital’s exclusive field of use consists of vaccines and certain therapeutic proteins, while Beam’s exclusive field of use consists of gene editing and conditioning for use in cell transplantation.

"The field of RNA medicines is advancing rapidly, and we are excited to participate in the launch of Orbital. It’s also an exciting opportunity for Beam, as we continue our strategy of pursuing creative partnerships that can generate value from the full breadth of our platform and accelerate the development of novel and diverse medicines for patients," said John Evans, chief executive officer of Beam Therapeutics. "The collaboration with Orbital also enables us to leverage cutting-edge advancements in RNA science for the development of our pipeline of potential transformative medicines focused on gene editing."

World-Class Founders
Orbital is founded by a collective group of scientific pioneers and proven biopharma company builders, including:

Howard Chang, M.D., Ph.D., professor of cancer research and professor of genetics, Stanford University
Ravi Majeti, M.D., Ph.D., professor of medicine, chief of division of hematology, Stanford University
Drew Weissman, M.D., Ph.D., professor in vaccine research, Perelman School of Medicine, University of Pennsylvania
Gene Yeo, Ph.D., MBA, professor of cellular and molecular medicine, UC San Diego
Giuseppe Ciaramella, Ph.D., president and chief scientific officer, Beam Therapeutics
John Maraganore, Ph.D., former founding chief executive officer of Alnylam Pharmaceuticals
Kristina Burow, managing director, ARCH Venture Partners
Carol Suh, partner, ARCH Venture Partners
In addition, Orbital is initially funded by ARCH Venture Partners, a16z Bio + Health and Newpath Partners.

"The formation of Orbital by a group of visionary leaders and scientific pioneers establishes a single organization that integrates a wide expanse of RNA technologies with a goal of delivering new medicines with a far-reaching impact on human health worldwide," said Ms. Burow. "The ability to combine the highest caliber science with potentially revolutionary technologies and a dynamic organizational structure led by proven experts in RNA, provides a special opportunity to create a therapeutically transformational company, and we at ARCH are thrilled to be a part of such a bold and impactful mission with Orbital."

Expert Leadership and Board
Orbital will be led by Giuseppe Ciaramella, Ph.D., who will serve as interim chief executive officer and a member of the board of directors, in addition to his ongoing role as president and chief scientific officer at Beam Therapeutics. Dr. Ciaramella has more than 25 years of drug discovery expertise and is a leader in the field of RNA research and drug development. Prior to Beam, Dr. Ciaramella served as chief scientific officer of the infectious diseases division at Moderna Therapeutics, where he led the establishment of its initial mRNA vaccine pipeline and the execution of its first investigational new drug application submission.

"In the history of medicine, certain therapeutic classes have revolutionized the treatment of both prevalent and rare diseases, and RNA-based therapeutics is undoubtedly one of them," said Dr. Ciaramella. "The creation of Orbital brings together a critical mass of the latest innovations in RNA technology under one roof to enable near-term clinical readiness, while advancing the durability, tissue-specific programmability and breadth of clinical applications. We stand at the forefront of science with an opportunity to treat human diseases in ways that have not yet been done, and I am excited to be leading the company in this endeavor."

Orbital has also appointed Gilles Besin, Ph.D., as chief scientific officer, bringing more than 15 years expertise in immunology and vaccines for infectious diseases, oncology and metabolic disorders. Dr. Besin joins Orbital from Affinivax Inc., where he served as vice president, head of discovery, leading all research efforts. Previously, Dr. Besin had increasingly senior roles at Moderna Therapeutics, where he led the platform immunology group and the efforts to modulate T cell responses in cancer and autoimmune diseases using mRNA lipid nanoparticles. Earlier in his career, Dr. Besin led research and discovery groups at In-Cell-Art, a biopharmaceutical company specializing in the development of DNA/RNA based vaccines and therapeutics. Dr. Besin earned an Engineering degree in biotechnology (Master’s-equivalent) from École Supérieure de Biotechnologie de Strasbourg (Strasbourg Graduate School of Biotechnology), as well as a Ph.D. in immunology from the Max Planck Institute of Immunobiology and Epigenetics. Dr. Besin is a member of the Scientific Advisory Board of Ovensa Inc.

In addition to Dr. Ciaramella, Orbital’s highly experienced board of directors includes:

Vineeta Agarwala, M.D., Ph.D., general partner, a16z Bio + Health
Kristina Burow, managing director, ARCH Venture Partners
John Evans, chief executive officer, Beam Therapeutics
John Maraganore, Ph.D., former founding chief executive officer of Alnylam Pharmaceuticals, and chairman of the Orbital board of directors
Carol Suh, partner, ARCH Venture Partners
To support the near-term growth of the company, Orbital will leverage the resources and talent of the Beam team in addition to Dr. Ciaramella, for leadership capabilities, operational support, and research and development.

Positive Readouts for Transgene’s Clinical Stage Candidates Generated by its Two Innovative Platforms, with Further Clinical Data Expected in the Second Half of 2022

On September 7, 2022 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for the six-month period ended June 30, 2022 and provides an update on the progress of its portfolio of clinical-stage drug candidates (Press release, Transgene, SEP 7, 2022, View Source [SID1234619181]).

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"Our goal is to transform the outlook for cancer patients with solid tumors by developing immunotherapies that can become vital components of the therapeutic arsenal. To achieve this, Transgene has created two highly innovative technology platforms that have delivered multiple therapeutic vaccine and oncolytic drug candidates now being evaluated in clinical trials," commented Hedi Ben Brahim, CEO of Transgene.
"We have made remarkable progress in the first half of 2022, with initial results in patients reinforcing my confidence that we are on track to progress through the clinic successfully. With several further important clinical milestones to be announced before the end of the year, we are approaching a critical moment to become a world leader in innovative cancer therapies."

Highlights and expected news flow

TG4001 – Result of the Phase II interim analysis to be released in Q4 2022

Just over 50 patients have been enrolled in the randomized Phase II trial evaluating the combination of TG4001 and avelumab in HPV-induced anogenital cancers. Transgene expects to report the results of the interim analysis in Q4 2022.

The objective of this trial is to demonstrate the superiority of the combination of TG4001 + avelumab versus avelumab as a single agent to improve progression-free survival (primary endpoint of the trial) and, in the longer term, overall patient survival.

In this Q4 2022 communication, Transgene will specify, based on the results of the interim analysis, the total number of patients that must be included in the trial to confirm the efficacy of the therapy and achieve statistically significant progression-free survival improvement. Indeed, the design of the trial allows for the total number of patients to be adjusted according to the results obtained in the interim analysis and based on the recommendations of the IDMC (independent data monitoring committee).

TG4050 – Patients treated with our neoantigen vaccine show a robust, vaccine specific immune response

Transgene presented new positive preliminary data from TG4050, its proprietary cancer vaccine based on the myvac platform, at the AACR (Free AACR Whitepaper) (April 2022) and ASCO (Free ASCO Whitepaper) (June 2022) meetings. These data show good safety and robust immunogenicity. The induction of immune responses is particularly efficient (100% of patients evaluated at the data cut-off date showed a specific cellular response) and is associated with disease regression.

Transgene has also produced data on circulating tumor DNA (ctDNA); signals of this increasingly validated surrogate marker of efficacy are particularly encouraging. Combined with the first signs of clinical activity, these results suggest that the individualized TG4050 vaccine has the potential to extend the period of remission, potentially offering a new treatment option for cancer patients.

Additional data from the two ongoing Phase I trials (ovarian cancer and head and neck cancers) are expected in the second half of 2022. The information generated will be key to designing the Phase II trial of TG4050, which could start as early as 2023.

In addition, Transgene has completed patient inclusion in the two Phase I trials. These patients are now being monitored until they become eligible for treatment per study protocol.

An article on Transgene’s personalized vaccine technology was published in the Journal for ImmunoTherapy of Canceri. The publication demonstrated that Transgene successfully developed a patient specific vaccine within a few weeks for a study case patient with a low tumor mutational burden (TMB).

TG6002 – Phase I trials completed – Confirms the strong potential of IV administration of the Invir.IO oncolytic viruses

Patient enrollment has been completed in the Phase I trial evaluating the intravenous (IV) route of administration: 51 patients received TG6002 at different doses and with different schedules.

This route of administration has considerable therapeutic and market potential as it allows targeting many types of internal lesions and metastases inaccessible by intratumoral injection (injection of the oncolytic virus directly into the tumor) which is currently the only approved route of administration for an oncolytic virus.

New data from this trial will be presented in a poster at the ESMO (Free ESMO Whitepaper) Congress on September 11, 2022 at 12:00 pm CET. The data confirm the safety and feasibility of the IV route for TG6002 and support the potential of IV administration of Invir.IO-based oncolytic viruses, extending the use of these therapies to a broad range of solid tumors.

In the TG6002 Phase I trial evaluating the intrahepatic artery route of administration (IHA), enrollment has been completed. Data are currently being analyzed and will be presented at a conference in the first half of 2023.

BT-001 – Good safety and first signs of antitumor activity as a monotherapy

Transgene and BioInvent provided an update on the progress of the clinical trial of BT-001, an oncolytic Vaccinia virus encoding an anti-CTLA-4 antibody and GM-CSF, in June 2022. Initial data from Part A of the Phase I trial demonstrated that BT-001 as a single-agent is well tolerated, with first signs of anti-tumor activity observed in a hard-to-treat patient population. The mechanism of action for BT-001, as a single agent, was also confirmed: the virus replicates in the tumor and the anti-CTLA-4 antibody is expressed in the tumor.

A clinical collaboration and supply agreement for KEYTRUDA (pembrolizumab) was signed with MSD (Merck & Co) at the end of June 2022. The Phase Ib part of the clinical trial (combination with pembrolizumab) is expected to start in the second half of 2022.

Promising preclinical data with BT-001 were presented at AACR (Free AACR Whitepaper) 2022 and published in the Journal for ImmunoTherapy of Cancerii demonstrating the broad and robust antitumor activity of this Invir.IO oncolytic virus.

Change in governance and appointment within the executive committee

Transgene reinforced its corporate governance by separating the roles of Chairman and CEO. Dr. Alessandro Riva, MD, became Non-Executive Chairman of the Company (May 25, 2022). With 30 years’ experience in the Life Sciences industry, Dr. Riva is closely working with Transgene’s CEO Hedi Ben Brahim to realize the potential of the Company’s technology platforms and products to benefit cancer patients.

Prof. Jean‑Yves Blay, MD, PhD, and Laurence Espinasse were appointed as members of the board of directors.

Steven Bloom joined Transgene as Vice President, Chief Business Officer (CBO). In this position, he has become a member of the executive committee, leading global business development strategy, alliance management and program management. In particular, he is focused on building the profile of Transgene in the USA, where he is based, as part of establishing the Company as a world leader in virus-based immunotherapies.

R&D Day

Transgene is organizing an R&D Day intended for the financial community, in particular analysts and institutional investors. It will be accessible to investment professionals in person and, online to everyone on Transgene’s website (via webcast). It will take place on September 27, from 2 p.m. to 6 p.m. CET (8 a.m. EST to 12 p.m. EST).

The event will include presentations by several Key Opinion Leaders, independent and active in Transgene’s clinical trials, as well as a review of Transgene’s immunotherapy portfolio and its positioning in the immuno-oncology landscape. In particular, Transgene will disclose new data on TG4050 and provide insights on the progress of the Invir.IO platform.

Summary of key ongoing clinical trials

TG4001

+ avelumab
Phase II

NCT03260023

Targets: HPV16 E6 and E7 oncoproteins

Recurrent/metastatic anogenital HPV-positive – 1st (patients ineligible for chemotherapy) and 2nd lines

Randomized Phase II trial comparing the combination of TG4001 with avelumab versus avelumab alone
Active patient enrollment in Europe (France and Spain) and in the USA
Results of the interim analysis expected in Q4 2022 (N≈50)

myvac

TG4050

Phase I

NCT03839524

Targets: tumor neoantigens

Codeveloped with NEC
New positive data in first patients demonstrating the immunogenicity of the vaccine as well as first signs of clinical activity presented at AACR (Free AACR Whitepaper) and ASCO (Free ASCO Whitepaper) 2022
Additional data on the 2 trials expected in H2 2022
Ovarian cancer – after surgery and first-line chemotherapy

Trial ongoing in the USA and in France
Patient enrollment completed
TG4050

Phase I

NCT04183166

HPV-negative head and neck cancer – after surgery and adjuvant therapy

Trial ongoing in the UK and in France
Patient enrollment completed

TG6002

Phase I/IIa

NCT03724071

Payload: FCU1 for the local production of a 5-FU chemotherapy

Advanced gastro-intestinal cancer (colorectal cancer for Phase II) – Intravenous (IV) administration

Multicenter trial ongoing in France, Belgium and Spain
Proof-of-concept data of the IV administration presented in 2021 (ESMO & AACR (Free AACR Whitepaper))
Patient enrollment completed in Phase I part
Poster to be presented at ESMO (Free ESMO Whitepaper) 2022 (September 11, 2022)

TG6002

Phase I/IIa

NCT04194034

Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration

Multicenter trial ongoing in the UK and in France
Patient enrollment completed in Phase I part
Data to be presented in H1 2023

Invir.IO

BT-001

Phase I/IIa

NCT04725331

Payload: anti-CTLA4 antibody and GM-CSF cytokine

Solid tumors

Co-development with BioInvent
Trial ongoing in France, Belgium and approved in the USA
Initial data showing safety and first signs of clinical activity
Collaboration agreement with MSD, supplying pembrolizumab for the trial
Start of part B of the Phase I trial in H2 2022

Key financials

The Board of Directors of Transgene met on September 7, 2022 and approved the financial statements for the six-month period ended June 30, 2022. The Statutory Auditors have conducted a limited review of the interim consolidated financial statements.

The half-year financial report is available on Transgene’s website, www.transgene.fr.

Key elements of the income statement

Operating income amounted to €6.1 million for the first six months of 2022 compared to €5.0 million for the same period in 2021.

In 2019, the Company entered into a collaboration agreement with AstraZeneca, which included exclusive licensing options to co-develop oncolytic immunotherapies derived from the Invir.IO platform. As a result, in the first half of 2019 Transgene received €8.9 million (US$10 million) in fees for access to its platform. This initial payment is recognized as revenue based on the stage of completion of the related activities. In the first half of 2022, the income recognized under this collaboration agreement was €2.3 million (€1.3 million in the first half of 2021). Of this amount €0.4 million reflects recognition of the initial payment for work done during the period and €1.9 million in respect of the supply of candidates and the performance of R&D services.
The research tax credit amounted to €3.7 million for the first half of 2022, compared to €3.5 million for the first half of 2021.
Research and Development (R&D) expenses amounted to €17.0 million in the first half of 2022 compared to €15.3 million for the same period in 2021, reflecting the acceleration of clinical trials.

General and administrative expenses amounted to €3.9 million for the first half of 2022 compared to €3.1 million for the same period in 2021.

Financial income is a loss of €0.4 million in the first half of 2022 compared to a gain of €1.6 million for the same period in 2021.

As a consequence, the net loss amounted to €15.3 million for the first half of 2022 compared to a loss of €11.8 million for the same period in 2021.

Transgene’s cash burn amounted to €6.8 million in the first half of 2022 compared with €11.9 million for the same period in 2021.

As of June 30, 2022, the Company’s cash, cash equivalents and other financial assets amounted to €42.8 million (€49.6 million as of December 31, 2021).

The Company has financial visibility until the end of 2023.

A conference call in English is scheduled today, on September 7, 2022, at 6 p.m. CET / 12 p.m. EST.

Fusion Pharmaceuticals to Present at the Morgan Stanley 20th Annual Global Healthcare Conference

On September 7, 2022 Fusion Pharmaceuticals Inc. ( Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported that the Company will present in a fireside chat at the Morgan Stanley 20th Annual Global Healthcare Conference on Wednesday, September 14, 2022 at 12:55 p.m. ET (Press release, Fusion Pharmaceuticals, SEP 7, 2022, View Source [SID1234619180]). Presenting on behalf of Fusion will be Chief Financial Officer John Crowley, and Chief Technology Officer Eric Burak, Ph.D.

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors & Media" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 60 days following the presentation.

Cue Biopharma to Present at Two September 2022 Scientific Conferences

On September 7, 2022 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of injectable biologics to selectively engage and modulate tumor-specific T cells directly within the patient’s body, reported members of their management and scientific teams will participate in two scientific conferences this September, The Promise of Interleukin-2 Therapy taking place September 14-17 in Paris, France and the Next Generation Protein Therapeutics Summit, held in Boston, Massachusetts and virtually on September 28-30 (Press release, Cue Biopharma, SEP 7, 2022, View Source [SID1234619179]).

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Presentation Details

The Promise of IL-2 Therapy
Location: Centre international de Conférence Sorbonne Université, Paris, France
Presentation title: Leveraging the Immuno-STAT platform to enhance anti-tumor immunity by selectively delivering IL-2 to tumor-specific T cells
Presenter: Matteo Levisetti, M.D., senior vice president, Clinical Development, Cue Biopharma
Session: Session IX – IL-2 in Cancer Therapy
Date and time: September 17, 2022, 9:00 am CET

Dr. Levisetti will present an overview of Cue Biopharma’s Immuno-STAT (Selective Targeting and Alteration of T cells) platform including the interleukin 2 (IL-2)-based CUE-100 series of biologics designed to enable selective targeting of IL-2 to tumor-specific T cells. The presentation will highlight data from clinical trials evaluating CUE-101, the company’s lead clinical candidate from the CUE-100 series, in patients with human papilloma virus positive recurrent/metastatic head and neck squamous cell carcinoma (HPV+ R/M HNSCC) as a monotherapy and in combination with pembrolizumab. Dr. Levisetti will also discuss the company’s second clinical candidate, CUE-102, currently in evaluation in a Phase 1 trial for patients with Wilms’ Tumor 1 (WT1)-positive cancers as well as other platform developments that support versatility for the potential treatment of several diverse cancers.

Presentation title: CUE-401: a novel IL-2/TGF-beta fusion protein for induction and expansion of regulatory T cells
Presenter: Steven Quayle, Ph.D., vice president Translational Pharmacology, Translational Medicine, Cue Biopharma
Session: Session X – Novel IL-2s and Combination Therapies
Date and time: September 17, 2022, 11:30 am CET

Dr. Quayle will provide an overview on the Company’s lead CUE-400 series drug product candidate CUE-401, a novel bispecific molecule designed for differentiation and expansion of induced regulatory T cells (iTregs). Dr. Quayle will discuss preclinical data supporting CUE-401’s mechanism of action and potential opportunity for the treatment of patients with autoimmune and graft-versus-host disease. Cue Biopharma plans to advance the CUE-400 series programs through strategic partnerships.

Presentation Details

Next Generation Protein Therapeutics Summit
Location: Boston Convention and Exhibition Center & virtual. The event will be simultaneously live streamed: https://informaconnect.com/next-gen-protein/
Presentation title: Immuno-STATs: TCR-selective engagers and activators of anti-tumor T cells
Presenter: Anish Suri, President and chief scientific officer, Cue Biopharma
Session: T Cell Engager Design and Challenges
Date and time: September 28, 2022, 4:30 pm ET

Dr. Suri will present an overview of Cue Biopharma’s Immuno-STAT platform and biologics designed to present tumor-specific proteins to T cell receptors (TCR) of the target tumor-specific T cells, to modulate that T cell repertoire for the anti-cancer response, as well as clinical data demonstrating the single-agent anti-tumor activity of CUE-101, the Company’s first IL-2 based clinical candidate, in late-stage head and neck cancer patients.

About Immuno-STAT
The company’s Immuno-STAT (Selective Targeting and Alteration of T cells) biologics are designed for targeted modulation of disease-associated T cells in the areas of immuno-oncology and autoimmune disease. Each of our biologic drugs is designed using our proprietary scaffold comprising: 1) a pMHC to provide selectivity through interaction with the T cell receptor (TCR), and 2) a unique co-stimulatory signaling molecule to modulate the activity of the target T cells. The simultaneous engagement of co-regulatory molecules and pMHC binding mimics the signals delivered by antigen presenting cells (APCs) to T cells during a natural immune response. This design enables Immuno-STAT biologics to engage with the T cell population of interest, resulting in selective T cell modulation. Because our drug candidates are delivered directly in the patient’s body (in vivo), they are fundamentally different from other T cell therapeutic approaches that require the patients’ T cells to be extracted, modified outside the body (ex vivo), and reinfused.