On September 23, 2021 Immunocore Holdings Plc (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infection and autoimmune disease, reported that data from a phase 3 randomized trial comparing tebentafusp (IMCgp100) with investigator’s choice in first-line metastatic uveal melanoma (mUM) has been published in The New England Journal of Medicine (NEJM) (Press release, Immunocore, SEP 23, 2021, View Source [SID1234590238]).

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The paper concluded that tebentafusp is the first systemic treatment to show a survival benefit in mUM and should become a new treatment option for this poor prognosis disease.

"The publication of these phase 3 data in a leading peer-reviewed scientific publication like NEJM demonstrates the significance of Immunocore’s work in the field of TCR therapy," said Bahija Jallal, Chief Executive Officer of Immunocore. "This further validates the potential of tebentafusp to provide a much needed treatment option for patients with metastatic uveal melanoma, making a meaningful difference to patients’ lives. In addition, we believe these data show the broader potential of Immunocore’s TCR technology for the treatment of other solid tumors."

Results from the randomized, open-label, phase 3 trial of tebentafusp vs. investigator’s choice in previously untreated HLA-A*02:01-positive patients with mUM demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) as a first-line treatment in mUM. The OS Hazard Ratio (HR) in the intent-to-treat population favored tebentafusp, HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, over investigator’s choice (82% pembrolizumab; 12% ipilimumab; 6% dacarbazine). Treatment-related adverse events were manageable and consistent with the proposed mechanism.

Tebentafusp has been granted Breakthrough Therapy Designation, Fast Track designation and orphan drug designation by the U.S. Food and Drug Administration (FDA) and Promising Innovative Medicine (PIM) designation under the UK Early Access to Medicines Scheme for metastatic uveal melanoma. Immunocore’s biologics license application for approval of tebentafusp for the treatment of HLA-A*02:01-positive adult patients with metastatic uveal melanoma was recently accepted by the FDA. In addition, the European Medicine Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) accepted Immunocore’s Marketing Authorisation Application (MAA).

On September 23, 2021 Allarity Therapeutics A/S, a clinical-stage precision medicine company actively advancing a pipeline of in-licensed oncology therapeutics for patients with difficult-to-treat cancers, and Lonza, a CDMO partner to the biopharma industry, reported an agreement to develop and manufacture dovitinib (Press release, Allarity Therapeutics, SEP 23, 2021, View Source [SID1234590237]). The agreement aims to commence manufacturing of dovitinib in 2022 to meet Allarity’s projected needs for bringing dovitinib to market following anticipated regulatory approvals.

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Dovitinib represents Allarity’s most advanced clinical asset, targeting metastatic renal cell carcinoma (RCC) with possible use in other indications, such as liver cancer, breast cancer and various solid tumors. This pan-tyrosine kinase inhibitor targets fibroblast growth factor receptor, vascular endothelial growth factor receptor and other receptor tyrosine kinases.

Steve Carchedi, CEO, Allarity commented: "Entering this agreement with Lonza is an important step in our long-term preparations to take dovitinib towards commercialization. Allarity now has a robust agreement covering the production and ongoing supply of dovitinib that we will need in the years to come."

Under the terms of the agreement, Lonza will leverage its capabilities for commercial manufacturing of small-molecules and oral solid dosage forms to provide Allarity with cGMP compliant drug product supply and regulatory support towards commercialization. Allarity will leverage Lonza’s global network, technical capabilities, and integrated solution covering both drug substance and drug product. The drug substance manufacturing and particle size reduction by micronization will be performed at Lonza’s facility in Visp (CH). The drug product manufacturing will take place at the Tampa, FL (US) facility.

Christian Dowdeswell, VP and Global Head, Commercial Development – Small Molecules, Lonza, added: "Our collaboration with Allarity Therapeutics demonstrates our commitment to supporting companies with their development pipeline. Our unique and comprehensive set of capabilities supporting drug substance through to drug product development and manufacturing enable Allarity to focus on dovitinib commercialization."

On September 23, 2021 KemPharm, Inc. (NASDAQ: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported that Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm, will present at the Cantor Fitzgerald Virtual Global Healthcare Conference taking place September 27-30, 2021 (Press release, KemPharm, SEP 23, 2021, View Source [SID1234590236]). The live presentation will be available via webcast from the conference’s virtual platform.

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Details of the presentation are as follows:

Event: Cantor Fitzgerald Virtual Global Healthcare Conference
Date: September 28, 2021
Time: 1:20 p.m., ET
Webcast: View Source
Dr. Mickle and members of the KemPharm management team will host virtual one-on-one meetings with registered investors and pharmaceutical company executives. Investors may register for the event by using the webcast link provided above, or by contacting their Cantor Fitzgerald sales representative.

Following the conclusion of the event, a recording of Dr. Mickle’s presentation will be available under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source

On September 23, 2021 VaxEquity, developing transformative RNA vaccines and therapeutics based on its next generation self-amplifying RNA (saRNA) platform originating from Imperial College London, reported a collaboration with AstraZeneca to progress the company’s platform technology through proof of concept to enable the development of multiple products (Press release, AstraZeneca, SEP 23, 2021, View Source [SID1234590235]). VaxEquity could receive development, approval and sales-based milestones totalling up to $195 million and royalties in the mid-single digits per drug target. VaxEquity also received an upfront equity investment from AstraZeneca and global life sciences investor Morningside Ventures.

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The strategic, long-term research collaboration with AstraZeneca aims to optimise and validate VaxEquity’s saRNA platform and apply it to advance novel therapeutic programmes. AstraZeneca will support VaxEquity with research and development funding and has the option to collaborate with VaxEquity on up to 26 drug targets. VaxEquity’s modified saRNA platform uses similar technology to mRNA but with the added ability to self-amplify, thereby expressing proteins for longer, resulting in higher protein levels per dose level.

Michael Watson, Executive Chairman of VaxEquity, said, "We are delighted to collaborate with AstraZeneca given its strong track record in innovation and welcome them as a new investor. We are also grateful for the ongoing support of our existing investor, Morningside Group. With our self-amplifying RNA platform, we aim to underpin the next generation of RNA-delivered medicines enabling not only vaccines but also broad range of therapeutic applications."

Professor Robin Shattock, Head of Immunology of Infection within the Department of Infectious Diseases at Imperial College London, and co-founder of VaxEquity, said, "We have all seen how technologies based around RNA have been fundamental to preventing ongoing severe disease and death in major global pandemics. The prospect of further therapeutic applications adds to this technology’s great potential."

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D at AstraZeneca, said "This collaboration with VaxEquity adds a promising new platform to our drug discovery toolbox. We believe self-amplifying RNA, once optimised, will allow us to target novel pathways not amenable to traditional drug discovery across our therapy areas of interest."

Professor Alice Gast, President of Imperial College London, said: "I am deeply proud of my colleagues’ work in pioneering self-amplifying RNA technology. This collaboration will help realise our ambition of building a lasting legacy from the great scientific advances Imperial made in this pandemic."

The proprietary, flexible platform enables the simultaneous expression of a broad range of targets and immunomodulatory proteins that can be rapidly produced and delivered at scale. Using saRNA, rather than mRNA, means that a lower (1/3 to 1/10th) dose of RNA is required to provide greatly enhanced protein expression as the RNA replicates for longer post-administration. VaxEquity modifies the RNA to include elements (called ‘Innate Inhibitory Proteins’ or IIPs) that finely tune the innate immune response (based on interferons) preventing suppression of RNA replication and thereby maximizing protein expression by saRNA.

As part of this investment and collaboration, Tyrell Rivers and Anders Holmén from AstraZeneca will join VaxEquity’s Board as Investor Directors. Will West and Jason Dinges will represent Morningside. Charles Mallo will represent Imperial. Robin Shattock will remain on the Board, while Michael Watson will assume the role of Executive Chair.

On September 23, 2021 Lion TCR Pte Ltd, a clinical-stage biotech company specialized in T Cell Receptor (TCR) T cell therapy reported that it has received clearance from U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) Application for LioCyx-M004, autologous T-cells transfected with mRNA encoding Hepatitis B surface antigen (HBsAg) specific TCR (Press release, Lion TCR, SEP 23, 2021, View Source [SID1234590234]).

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Lion TCR intends to initiate a Phase 1b/2 multi-center study for its lead investigational product, LioCyx-M004, for patients with advanced Hepatitis B Virus (HBV)-related hepatocellular carcinoma (HCC). This will be the first Phase 1b/2 study that uses specific T cell receptor (TCR)-T cell therapy to target HBV-related HCC.

In 2020, there are more than 900,000 new cases with liver cancer worldwide, of which 90% been hepatocellular carcinoma (HCC). In Asia, HBV is an important inducer of HCC, for example, 80% – 90% of HCC is related to HBV infection in China. The use of LioCyx-M004 has been tested in Phase 1 study in primary HBV-related HCC. LioCyx-M004 infusions were well-tolerated with no occurrence of cytokine release syndrome (CRS) nor neurotoxicity. Disease control rate was 60% and partial response (PR) as per RECIST 1.1 was observed with a duration of response of 27.7 months. Median overall survival (OS) was 33.1 months.

The upcoming study aims to evaluate LioCyx-M004 as a monotherapy treatment and the safety and efficacy of LioCyx-M004 in combination with lenvatinib. Considering lenvatinib as a well-established first-line treatment for advanced HCC and its potential to revert the immunosuppressive tumor microenvironment towards an immune-supportive profile, the combination of lenvatinib and LioCyx-M004 treatment is expected to further improve clinical outcome.

"LioCyx-M004, with its unique mechanism of action and its excellent safety profile and radiological tumor response, has the potential to become a first-in-class TCR T cell therapy for HCC. We also have a strong scientific rationale for the use of our TCR-T therapy in combination with other therapies to extend its effectiveness. We are proud of our significant progress in recent years and look forward to initiating the Phase 1b/2 study in the upcoming weeks for the benefits of HCC patient refractory or relapsed to current systemic treatment. Patient recruitment for this study will begin at City of Hope Comprehensive Cancer Center in United States, a leading research and treatment center for cancer," said Dr Tina Tingting Wang, COO and CMO of Lion TCR.

"This Phase 1b/2 IND clearance is a very important milestone for Lion TCR! We will continue our dedication to be the world leading biotech company using cutting-edge TCR-T immunotherapy to provide innovative therapeutic options for patients in need. Lion TCR has developed various TCR-T technology platform including TCR discovery platform, autologous and allogeneic TCR platform incorporating with gene editing technologies. With the potential of our revolutionary TCR-T cell immunotherapy, we aim to be the pioneer company for benchmarking in the field of liver cancer treatment," said Dr Peng Xiaoming, Managing Director & CEO of Lion TCR.