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Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases

On December 19, 2024 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported they have entered into a license agreement allowing Astellas to leverage Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and neuronal transduction in nonhuman primates (Press release, Astellas, DEC 19, 2024, View Source [SID1234649211]). The agreement grants Astellas a worldwide exclusive license to utilize the STAC-BBB capsid for one target, with the right to add up to four additional targets after paying additional licensed target fees to deliver their intravenously administered genomic medicines to treat certain neurological diseases.

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Sandy Macrae, Chief Executive Officer, Sangamo
"We strongly believe in the potential of STAC-BBB, our industry-leading, intravenously delivered AAV capsid, to overcome the challenges associated with delivering therapies to the central nervous system. This agreement underscores the continued industry interest in our STAC-BBB capsid and reinforces our ongoing commitment to partnering with collaborators who understand its unique potential. We are delighted to license STAC-BBB to Astellas to advance potential treatments for neurological diseases with significant unmet medical needs."

Adam Pearson, Chief Strategy Officer, Astellas
"Delivering treatments to the brain and central nervous system remains a highly complex challenge in the field of gene therapy. We believe that technologies such as Sangamo’s STAC-BBB capsid could prove critical in helping us deliver effective transformational treatments to patients suffering from serious genetic neurological conditions. We continue to build a world-class gene therapy pipeline and end-to-end discovery, development, manufacturing, and commercial capabilities. This agreement is another example of our commitment to delivering meaningful therapies for patients with genetic diseases."

Under the terms of the agreement, Sangamo is responsible for completing a technology transfer related to the STAC-BBB capsid. Astellas is responsible for all research, preclinical and clinical development, regulatory interactions, manufacturing, and global commercialization of the resulting gene therapy products. Sangamo will receive a $20 million upfront license fee from Astellas and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across the five potential neurology disease targets, as well as tiered mid-to-high single digit royalties on potential net sales of such products, subject to certain specified reductions.

Artios Pharma to Present at the 43rd Annual J.P. Morgan Healthcare Conference

On December 19, 2024 Artios Pharma Limited ("Artios"), a clinical-stage biotech company led by pioneers of DNA damage response ("DDR") drug development, reported that it will present at the 43rd Annual J.P. Morgan Healthcare Conference taking place in San Francisco from January 13 to 16, 2025 (Press release, Artios Pharma, DEC 19, 2024, View Source [SID1234649210]).

Artios Pharma to Present at the 43rd Annual J.P. Morgan

Niall Martin, PhD, Artios’ Chief Executive Officer, will present the company’s strategic vision for 2025 and beyond on January 16 at 08:00 am PST (11:00 am EST / 04:00 pm UTC) in the Elizabethan C room at The Westin St. Francis, 335 Powell Street, San Francisco.

AFYX Therapeutics A/S Secures DKK 25 million in Funding

On December 19, 2024 AFYX Therapeutics A/S, a pioneering Danish biopharmaceutical company specializing in reversed innovation and the repurposing of established pharmaceutical substances, reported it has successfully raised DKK 25 million in its latest funding round (Press release, AFYX Therapeutics, DEC 19, 2024, View Source [SID1234649209]). This milestone will support the company’s efforts to advance its product pipeline, scale operations, and expand its footprint across the European Union and other global markets.

The funding round was led by existing investors and employees and highlights the robust internal and external trust in AFYX’s mission and strategic direction. The new funds will accelerate the development of the company’s leading products, including its clobetasol patch for oral lichen planus and esketamine therapy for cluster headaches. Additionally, the capital will fuel AFYX’s commercial expansion into new markets, organizational growth, and broader international partnerships.

"We are thrilled to have secured this significant investment, which will enable us to advance our mission of developing transformative biopharmaceutical solutions," said Dr. Claus Møller San Pedro, CEO and co-founder of AFYX Therapeutics. "The continued trust from our investors reflects their confidence in our team and vision. With their ongoing support, we’re well-positioned to address critical unmet medical needs and deliver groundbreaking therapies that improve patient outcomes."

Since its formation in January 2024, through the merger of three highly experienced pharmaceutical innovators, AFYX Therapeutics has made remarkable progress. Over its first year, the company has accelerated its research and development activities, expanded its commercial reach, and secured new international distribution partnerships, paving the way for sustained growth and innovation.

Vivesto strengthens Cantrixil program with new preclinical results and patent application

On December 19, 2024 Vivesto AB, an oncology-focused development company, reported that positive results were obtained from preclinical studies with combination treatments within the company’s Cantrixil program, supporting continued development in hematological cancer (Press release, Vivesto, DEC 19, 2024, View Source [SID1234649208]). Vivesto also announced that a new patent application covering the treatment of hematological cancer with Cantrixil was filed, with the aim to strengthen the IP position.

The drug candidate Cantrixil has been evaluated in further combination treatments with other anti-cancer drugs generating new in vitro data in hematological cancer cell lines. The results demonstrate clear positive effects of Cantrixil in combination with other anti-cancer drugs. The positive results confirm previous preclinical efficacy results and support continued development in hematological cancer. New results from hematological cancer models are expected to be presented throughout 2025.

"Hematological cancer is one of Vivesto’s priority focus areas, and we are pleased to report successful results from yet another preclinical study supporting the further development of the Cantrixil program. We believe that Cantrixil can play a key role in the treatment of hematological cancer and look forward to reporting new data from additional ongoing studies early next year that will be of great importance as the program is developed towards clinical phase," said Erik Kinnman, CEO of Vivesto. "Vivesto also filed a new patent application for the treatment of hematological cancer with Cantrixil as a robust patent portfolio is fundamental for value creation in clinical development, and eventually, commercialization of the product."

Cantrixil has previously shown strong cytotoxic effects at low doses in cell lines derived from patients with hematological cancer. The recently generated data provides important input to the dosing selection and treatment regime in upcoming preclinical and clinical studies.

Sprint Bioscience adds new cancer drug program to portfolio

On December 18, 2024 Sprint Bioscience AB (publ) reported that the company is broadening its portfolio with a drug development program for the treatment of acute myeloid leukemia (AML). The program targets the decapping scavenger enzyme (DCPS), a target protein that has been validated both in scientific literature and by Sprint Bioscience in collaboration with Associate Professor Julian Walfridsson at Karolinska Institutet.

The initiation of Sprint Bioscience’s DCPS program is part of the company’s strategy to capitalize on its platform for small-molecule drug discovery to broaden the portfolio in the cancer area and to bring more opportunities for future licensing deals.

The DCPS program focuses on a target protein that degrades a metabolite resulting from the processing of mRNA. Both small-molecule inhibition and genetic knock-down of DCPS affects the differentiation and proliferation of several AML cell lines as well as patient-derived samples. Furthermore, healthy tissue seems to be insensitive to DCPS inhibition, indicating that DCPS inhibitors could offer a safe and effective treatment option for AML patients. Sprint Bioscience has also identified biomarkers to predict response to DCPS inhibition, enabling patient selection and clinical success for patients with unmet therapeutic needs.

"We have identified a target protein that constitutes a very attractive approach for the treatment of AML. We aim to advance the project to a stage where it can be licensed to an international pharmaceutical company, enabling further clinical development and progression towards the market to benefit AML patients in need of new treatment options," said, Johan Emilsson, CEO of Sprint Bioscience.

AML is a severe type of blood cancer. The Global Burden of Disease study estimates that about 140,000 people around the world are diagnosed with AML on a yearly basis. There is an urgent medical need to identify safe and effective therapies to improve treatment outcomes.

Sprint Bioscience’s portfolio now consists of six internal drug development programs (five of which are in cancer) and one program that has been licensed to Day One Biopharmaceuticals.

(Press release, Sprint Bioscience, DEC 18, 2024, View Source [SID1234660961])