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Tech Transfer Roundup: Neon Licenses NKI IP To Advance Cell Therapy Into Clinic

Executive Summary

Continuing a partnership between Neon co-founder and two Netherlands Cancer Institute researchers, agreement will help autologous T-cell candidate move into Phase I. Plus Scrip's monthly roundup of tech transfer deals.

Continuing a collaborative research agreement signed in 2016, Neon Therapeutics licensed technology from the [Netherlands Cancer Institute] (NKI) that will enable it to advance its personalized neoantigen T-cell therapy candidate NEO-PTC-01 into clinical development.

The agreement announced Oct. 4 grants the Cambridge, Mass.-based biotech exclusive intellectual property rights for an ex vivo induction platform developed in collaboration by NKI researchers John Haanen and Joost van den Berg and Neon’s co-founder Ton Schumacher. Richard Gaynor, Neon’s president of R&D, told Scrip the IP builds on the NKI researchers’ work of several years on manufacturing adoptive cell therapies.

The partners have been working together for about a year on the technology, which produces autologous T-cells targeting a patient’s own neoantigens. [See Deal] The licensed IP will support Neon’s continued work on NEO-PTC-01, a personalized neoantigen autologous T-cell therapy in preclinical cancer studies.

Neon and the NKI will work together towards a Phase I trial, which is expected to begin by the end of 2018. Gaynor said GMP up-scaling toward that goal is underway.

“Our collaboration with the NKI is about complementary capabilities – bringing together our expertise in neoantigen biology with the NKI’s experience in developing T-cell therapies,” he added. “We have co-developed these technologies to enable us to generate multiple neoantigen responses in an ex vivo induction protocol. Our focus is now on completing up-scaling so it can be used to generate patient-specific T-cell therapy products.”

Neon’s goal is to develop cell therapies that can yield tumor regression in multiple types of cancer, Gaynor said. The biotech raised a $55m Series A financing in 2015, with investment by Third Rock Ventures, Clal Biotechnology Industries and Access Industries [See Deal]. It then raised $70m in a Series B round this past January led by Partner Fund Management and including new investors Fidelity Management & Research, Wellington Management Company, Inbio Ventures and Nextech Invest. [See Deal]

Below is Scrip’s monthly roundup, compiled by Strategic Transactions, of recent technology transfer agreements between companies and academic or other research institutions.

Casebia, Seattle Children's Study Gene-Edited Regulatory T-Cell Therapies

Casebia Therapeutics is joining forces with Seattle Children’s Research Institute to research and develop autoimmune disease treatments using CRISPR/Cas9 gene-edited regulatory T-cells (Tregs). Under terms of the deal announced Sept. 27, Casebia will sponsor research at the institute – it also licensed exclusive global rights develop and will sell the related Treg intellectual property to potentially treat a wide range of autoimmune conditions. Funding contributions from Casebia could reach $12m.

The deal stems from discoveries made by Seattle Children’s researchers David Rawlings and Andrew Scharenberg related to engineering, manufacturing and expanding T-cells. Casebia, formed as a joint venture between Bayer AG and CRISPR Therapeutics AG, already has three gene editing-based autoimmune candidates in its pipeline, including one for severe combined immunodeficiency (SCID). [See Deal] It is also working on treatments for hemophilia and inherited retinal disease.

Glythera Licenses CDK11 Inhibitor Program From Cancer Research UK

Cancer Research UK granted Glythera Ltd. exclusive global rights Sept. 26 to its CDK11 inhibitor program, which Glythera will use in the development, manufacture and sale of new antibody-drug conjugates for cancer. Glythera paid an undisclosed sum up front and is responsible for milestone payments and royalties.

Together, the partners will select toxins from Cancer Research’s CDK11 inhibitor payload series, and Glythera will use its PermaLink conjugation platform to develop multiple ADCs. PermaLink is a stable and controlled conjugation platform comprised of a portfolio of linker designs, and offers benefits including controlled drug-to-antibody ratio and low immunogenicity. The company hopes to have its first project in clinical trials by 2019.

The deal is the second in a week for Glythera. It also announced a partnership with Iontas Ltd. on Sept. 20 in which the latter firm will use its generation platforms to discover fully human monoclonal antibodies, and Glythera will get an option to develop the mAbs into ADCs using PermaLink. [See Deal]

ConverGene Partners With MD Anderson On Ovarian Carcinoma Research

ConverGene LLC announced a collaboration with University of TexasMD Anderson Cancer Center Sept. 18 to characterize CVG-101 – the biotech’s first-in-class dual inhibitor of BET bromodomains and dopamine receptor 2 – in ovarian carcinoma.

Based in Cambridge, Mass., ConverGene will undertake the work in tandem with MD Anderson’s Sherry Wu and Anil Sood. Slated for two distinct phases, the project first will investigate CVG-101’s unique mechanism of action in vitro, followed by determination of clinically relevant activity for the compound utilizing in vivo model systems developed in the Sood lab.

ConverGene leverages rational drug design for small molecule discovery with a mission to develop therapeutics offering novel mechanisms of action for patients with limited or no treatment options. Its lead program, CVG-101, is formulated to be selective against BET proteins. It contains multiple clinical candidates that exhibit dual activity, simultaneously inhibiting epigenetic processes within cancer cells as well as D2R, which is a driver of proliferation and metastasis in cancer.

Mitobridge Licenses EFPL's IP For Mitochondrial, Aging Diseases

Mitobridge Inc. gained exclusive worldwide rights Sept. 18 to intellectual property related to modulating the nicotinamide adenine dinucleotide (NAD+) pathway from the [Ecole Polytechnique Federale de Lausanne] (EPFL).

Mitobridge will apply EPFL's IP to drug compounds to boost NAD+ levels, hoping to generate candidates to treat a variety of mitochondrial dysfunction-associated diseases as well as conditions of aging. The IP complements Mitobridge's existing platforms that target multiple points in the NAD+ biosynthetic and metabolic pathways, including poly ADP ribose polymerase (PARP), aminocarboxymuconate semialdehyde decarboxylase (ACMSD) and N'-nicotinamide methyltransferase (NNMT).

The Cambridge, Mass.-based firm also has programs in gene regulation (including PPAR IP licensed from the Salk Institute) and mitochondrial dynamics. Mitobridge hopes to build its pipeline of compounds for muscular dystrophies – lead MA0211 is in Phase I for Duchenne muscular dystrophy – and other kidney, liver, musculoskeletal, and neurodegenerative diseases that will benefit from improved mitochondrial function.

The research originated from the laboratory of EPFL's Johan Auwerx, who specializes in metabolism and its effects on health, aging and disease. Mitochondria are organelles controlled by a network of proteins, including NAD+, an essential metabolic regulator coenzyme known to play a key role in mitochondrial energy production and cell signaling. Studies have demonstrated reduced NAD+ levels are linked with mitochondrial dysfunction, which can cause multiple medical disorders and age-related diseases, but compounds that protect or elevate NAD+ levels could potentially provide therapeutic benefits.

Bayer, Vanderbilt Will Collaborate On Novel Kidney Disease Therapies

Bayer and Vanderbilt University announced a five-year strategic research alliance on Sept. 11 to evaluate new drug candidates for the treatment of kidney diseases with a goal of accelerating the translation of innovative lab research findings to preclinical development. No financial terms were disclosed.

Under the agreement, Bayer and Vanderbilt’s University Medical Center will jointly undertake target validation, assay development and lead optimization work. Each will contribute personnel and infrastructure to the effort; Bayer receives an option for the exclusive use of the collaboration results.

The partners noted the limited therapeutic options for patients with end-stage renal disease – dialysis, transplantation or palliative care – and that there is no effective treatment available to prevent the cardiovascular consequences of chronic kidney disease and at the same time reduce the progression to ESRD. With that in mind, the collaboration’s goal is to develop two investigational new drugs addressing these medical needs within the five-year timeframe.

Pascal Biosciences Options Leukemia Antibody From U Of New Mexico

STC.UNM, the tech transfer unit of the University of New Mexico, has granted Pascal Biosciences Inc. a six-month option to license exclusive global rights to its technology surrounding a therapeutic monoclonal antibody for B-cell precursor acute lymphoblastic leukemia (BCP-ALL). Announced Sept. 11, the option can be extended for another 12 months.

The technology licensed by Pascal covers antibody-drug conjugates, CAR-T therapy and VpreB1-targeted treatments. The partners will also work together to develop a companion diagnostic for patient selection.

UNM researchers Bridget Wilson and Stuart Winter discovered an antibody specific for VpreB1, a subunit of the pre-B cell receptor expressed in most ALL patients during the early stages of the disease. Targeting the marker helps to kill off leukemia cells but spares healthy “memory B-cells” that are responsible for stimulating the immune system to continue fighting the disease.

PureTech Health Licenses Lymphatic Biology Tech From Monash University

Monash University granted PureTech Health PLC exclusive rights Aug. 29 to a lymphatic targeting program called the “Glyph technology,” for use in the development of therapies that selectively target certain lymph nodes such as the mesenteric lymph nodes.

The technology was designed by Monash’s Christopher Porter and is intended to help immune-modulating drugs navigate the immune-gut-brain axis in a way that bypasses first-pass metabolism, improves oral bioavailability and lowers liver toxicity risk. PureTech’s Glyph Biosciences subsidiary, together with Porter’s laboratory, will continue work on the technology to explore its implications in autoimmune diseases and cancer.

Grid Therapeutics Licenses Antibody Rights From Duke

Duke University granted its spin-out Grid Therapeutics LLC exclusive rights on Aug. 22 to all IP and patents related to complement factor H (CFH) antibodies, which Grid will develop into therapeutics and diagnostics for cancer.

Grid was spun out of Duke by company CEO Edward Patz and his team who discovered a tumor-specific immunoglobulin (IgG) antibody in early-stage cancer patients who do not develop metastases. The antibody against complement factor H helps to stop immune protection that cancer cells have, and makes the cells more vulnerable to drugs, resulting in increased tumor cell death and adaptive immune response modulation to help the body attack cancer cells anywhere else in the body.

Grid’s first compound is expected to enter Phase I trials in early 2019. The biotech announced a Series A financing backed by Duke and Longview International on Sept. 14 – the amount raised was not disclosed. [See Deal]

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