On January 6, 2022 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported that Christian S. Schade, Chairman and Chief Executive Officer, will present a corporate update at the 40th Annual J.P. Morgan Healthcare Conference on Thursday, January 13, 2022 at 11:15 a.m. ET (Press release, Aprea, JAN 6, 2022, View Source [SID1234598321]).

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A live webcast of the presentation can be accessed under the "Events Calendar" in the Investors section of the Aprea website at Link.

On January 6, 2022 Eli Lilly and Company (NYSE: LLY) reported that has acquired exclusive rights to Entos Pharmaceuticals’ Fusogenix nucleic acid delivery technology to research, develop and commercialize nucleic acid products targeting the central and peripheral nervous system (Press release, Eli Lilly, JAN 6, 2022, View Source [SID1234598320]). This technology provides an opportunity for Lilly to access a novel delivery platform technology with the potential to solve a key delivery challenge for many nucleic acid therapeutic modalities.

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Under the terms of the research and collaboration agreement, Lilly and Entos have agreed to multiple programs focused on the development of proteo-lipid vehicles (PLV) for delivery of therapeutic cargo supplied by Lilly to targets in the central and peripheral nervous systems. Entos will be responsible for the generation, development and optimization of PLVs using its proprietary Fusogenix platform technology. Lilly will be responsible for selecting PLVs for clinical development and commercialization. Entos will receive an initial payment of $50 million, which includes an equity investment by Lilly in Entos. For each of the programs under the collaboration, Entos is also eligible to receive up to $400million in potential developmental and commercial milestone payments, as well as royalties upon the successful development and commercialization of products.

"Nucleic acid-based therapies hold great promise in addressing the biologic cause of many serious diseases that have significant unmet medical need," said Andrew Adams, vice president, Lilly Genetic Medicine. "Overcoming barriers to the safe and effective delivery of such therapies to specific target cells is essential to realizing their potential. We look forward to working with Entos as part of our efforts to overcome this challenge and develop potential new therapies that may improve patient outcomes."
"For more than 145 years, Lilly has demonstrated its ability to incorporate cutting-edge scientific advances into drug development strategies," said John Lewis, Ph.D., founder and CEO of Entos. "We are excited for the opportunity to play a part in Lilly’s ongoing quest to develop high-quality medicines and believe our collaboration with Lilly is an important validation of the potential value of our expertise and technology platform."
This transaction will be reflected in Lilly’s reported results and financial guidance according to Generally Accepted Accounting Principles (GAAP). There will be no change to Lilly’s 2021 or 2022 non-GAAP earnings per share guidance as a result of this transaction.

On January 6 2022 ONK Therapeutics, an innovative NK cell therapy platform company, reported the closing of its $21.5 million Series A financing, led by current investors Acorn Bioventures and ALSHC, who were joined by Cormorant Asset Management (Press release, ONK Therapeutics, JAN 6, 2022, View Source [SID1234598318]).

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The financing will enable ONK to maintain its strong momentum as it advances pre-clinical programs through comprehensive IND-enabling studies, including in-vivo proof-of-concept models across multiple programs. ONK will also continue progress towards a GMP manufacturing capability, as it optimizes both its proprietary cell engineering platform and process development.

ONK’s pipeline currently has three programs in pre-clinical development across both hematological malignancies and solid tumors. The Company is pioneering optimally engineered natural killer (NK) cell therapies, utilising a suite of engineering strategies to optimize cytotoxic potential, metabolic health and persistence of NK cells, while reducing the potential for their exhaustion in the tumor microenvironment. This is made possible through the Company’s ability to achieve unique proprietary gene edits.

ONK’s comprehensive owned and exclusively licensed patent estate covers CISH knockout (KO) in the field of human NK cells for the treatment of cancer, irrespective of the NK cell source; the expression of high affinity, membrane bound,TNF-related apoptosis-inducing ligand variants (TRAILv); and the deletion of checkpoint receptors in NK cells, including extracellular proteins CD96, TIGIT, Siglec-7 and PD-1.

These different strategies are being employed across the ONK pre-clinical product portfolio, including:

ONKT102, the Company’s lead program, an optimized affinity CD38 CAR (chimeric antigen receptor) NK cell therapy being developed for the treatment of patients with relapsed refractory multiple myeloma
ONKT104, a CLL-1 CAR-NK program targeting AML stem cells, which is advancing through later stages of its pre-clinical evaluation
ONKT103, which is being optimized to treat solid tumors such as Ovarian, Breast and Non-Small Cell Lung Cancer (NSCLC) and is based on tumor associated MUC-1 targeting CAR NK cells
In addition, these programs will explore incorporating further novel gene edits, such as CISH KO and TRAIL variants targeting DR5 or DR4.

ONK has also recently started two programs focused on the potential of highly functional CISH KO NK cells, namely:

ONKT105, exploring CISH KO cord blood derived NK cells
ONKT106, exploring CISH KO iPSC derived NK cells
The Company also continues to make significant progress on the manufacturing front, optimizing its process development, gene editing capability, efficient expansion techniques and its cryopreservation capability. In combination, these will support manufacturing as part of ONK’s progress towards GMP manufacturing of large numbers of NK cell therapy batches from a single cord.

Chris Nowers, ONK Therapeutics’ CEO said, "We thank our existing investors for their continued support and are pleased to welcome Cormorant Asset Management as a new investor and Andy Phillips to our Board, as we continue against our goal to optimally engineer, off-the-shelf, NK cell therapies to potentially cure patients with cancer. This financing will allow us to continue to deliver against our focused strategy, funding significant program progression, organizational development, and company growth. Within the next 18 months, we have the potential for multiple IND approvals to enable our evolution into a clinical-stage company."

Commenting on the investment, Isaac Manke, Ph.D., Partner at Acorn Bioventures said, "Over the 12 months of our investment, ONK Therapeutics has made excellent progress in advancing both its innovative next-generation NK cell therapy platform and across its exciting portfolio of pre-clinical assets. We are pleased to continue our support and to have introduced Cormorant Asset Management as a new investor to join with ourselves and founding investor ALSHC."

Andy Phillips, Ph.D., Managing Director at Cormorant Asset Management said, "We are impressed by the Company’s potential to make a highly innovative contribution to the quickly evolving field of NK cell therapies, which we believe have the opportunity to improve the lives of patients in need of new treatments for their disease. We are pleased to join the current investor groups in financing ONK as it looks forward to important proof of principle in vivo data and additional value catalyzing milestones."

On January 6, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, reported its expected milestones and the following strategic priorities for 2022 (Press release, Intellia Therapeutics, JAN 6, 2022, View Source [SID1234598317]):

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Accelerating clinical validation of in vivo pipeline: Further characterize the safety and efficacy of NTLA-2001, including in patients with cardiomyopathy and complete enrollment of the Phase 1 study; establish the initial clinical profile of NTLA-2002 as a single-dose therapy for the treatment of hereditary angioedema (HAE).
Strategic pipeline expansion: Significantly progress in vivo and ex vivo pipeline, including determining the initial safety profile of NTLA-5001 for acute myeloid leukemia (AML), advancing in vivo insertion candidates and nominating multiple new development candidates.
Platform innovation: Broaden the Company’s industry-leading platform through expansion of Intellia’s genome editing, delivery and cell engineering capabilities.
"Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases," said Intellia President and Chief Executive Officer John Leonard, M.D. "As we begin 2022 with great momentum, we are poised to significantly expand our full-spectrum pipeline of potentially curative therapies with the nomination of at least two new in vivo candidates and our first allogeneic development candidate during the year. Importantly, we look forward to sharing additional data from the ongoing study of NTLA-2001 and interim results from the Phase 1/2 study of NTLA-2002, which we expect will further demonstrate the modularity of our genome editing platform."

Anticipated 2022 Milestones:

In Vivo Programs

NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, and has the potential to be the first single-dose treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Today, Intellia announced that the first patient in the cardiomyopathy arm of the Phase 1 study has been dosed with NTLA-2001. This follows the Company’s recent announcement that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study now includes patients with ATTR-CM enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Intellia expects to complete enrollment of the Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022.
Intellia intends to present additional interim clinical data of NTLA-2001 in ATTRv-PN patients from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four ATTRv-PN dose cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In December 2021, Intellia dosed the first patient in its second clinical study of a CRISPR-based therapeutic candidate evaluating NTLA-2002 for HAE. The first-in-human Phase 1/2 trial is expected to evaluate the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE, and will continue to leverage insights gained from the development of NTLA-2001.
The Company expects to present interim data from the Phase 1/2 study in the second half of 2022. These results are expected to characterize the emerging safety and activity profile of NTLA-2002 and demonstrate preliminary proof-of-concept.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s wholly owned CRISPR-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001 with plans to file an IND or IND-equivalent in 2023. The Company also continues to explore additional editing strategies for AATD.
Ex Vivo Programs

NTLA-5001 for AML: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML).
In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company expects to dose its first patient in the coming weeks and enroll patients throughout the year. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its TCR-based platform.
Modular Platform and Pipeline Expansion

Platform Innovation: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that will enable broader in vivo and ex vivo applications.
Intellia plans to advance at least two new in vivo development candidates by the end of 2022.
The Company expects to nominate its first allogeneic ex vivo development candidate by the first half of 2022.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Corporate Updates:

In January, Intellia and Kyverna Therapeutics announced a licensing and collaboration agreement for the development of KYV-201, an allogeneic CD19 CART-cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases. Intellia may exercise an option to lead U.S. commercialization for KYV-201 under a co-development and co-commercialization agreement.
In December 2021, Intellia appointed Derek Hicks as Executive Vice President, Chief Business Officer. Mr. Hicks joins Intellia with more than 25 years of combined business, leadership and biotechnology experience, having most recently served as Head of Business Development at Spark Therapeutics.
Cash Position

Intellia ended the fourth quarter of 2021 with approximately $1.1 billion in cash, cash equivalents and marketable securities.
Intellia’s Presentation at the 40th Annual J.P. Morgan Healthcare Conference

Intellia is scheduled to present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, at 2:15 p.m. ET. A live audio webcast of Intellia’s presentation can be accessed under the Events and Presentations page of the Investors & Media section on the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least two weeks following the presentation.

On January 6, 2022 F-star Therapeutics, Inc. (NASDAQ: FSTX), a clinical-stage biopharmaceutical company dedicated to developing next generation bispecific immunotherapies to transform the lives of patients with cancer, reported that Ares Trading S.A., an affiliate of Merck KGaA, Darmstadt, Germany has exercised a fourth licensing option to develop another bispecific program under the ongoing immuno-oncology collaboration (Press release, F-star, JAN 6, 2022, View Source [SID1234598316]). Under the terms of the agreement, Merck KGaA, Darmstadt, Germany will be responsible for all future development and commercialization costs and will pay future success-based milestones and royalties on any net sales, resulting from programs covered by the agreement.

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The two companies have had a long standing and successful partnership and in May of 2019 Merck KGaA, Darmstadt, Germany, exercised its option to bring a first program from the collaboration into its pipeline. In July 2020, Merck KGaA, Darmstadt, Germany took an early option to a second program and in March 2021, a third option was exercised. With the fourth option now exercised, the total potential remaining milestone-based revenue due to F-star from this immuno-oncology collaboration with Merck KGaA, Darmstadt, Germany is approximately $765 million.

Eliot Forster, CEO of F-star Therapeutics, Inc, said: "We were delighted to see the recent data presented by Merck KGaA, Darmstadt, Germany, from our long-standing collaboration at the recent SITC (Free SITC Whitepaper) conference, demonstrating the unique potential of the F-star bispecific platform. This latest licensing option marks another positive milestone in our collaboration and it is exciting to see our partner successfully progress another mAb2 based program. Along with our internal pipeline, we believe all of these next generation immuno-oncology programs will make a real difference to the lives of patients with cancer."