Algorithm Hunts for Anomalies in Gene Expression Data

Computational biologists at Carnegie Mellon University have devised an algorithm to rapidly sort through mountains of gene expression data to find unexpected phenomena that might merit further study. What's more, the algorithm then re-examines its own output, looking for mistakes it has made and then correcting them. This work by Carl Kingsford, a professor in CMU's Computational Biology Department, and Cong Ma, a Ph.D. student in computational biology, is the first attempt at automating the search for these anomalies in gene expression inferred by RNA sequencing, or RNA-seq, the leading method for inferring the activity level of genes. The researchers report that they have already have detected 88 anomalies — unexpectedly high or low levels of expression of regions within genes — in two widely used RNA-seq libraries that are both common and not previously known. "We don't yet know why we're seeing those 88 weird patterns," Kingsford said, noting that they could be a subject of further investigation. Though an organism's genetic makeup is static, the activity level, or expression, of genes varies greatly over time. Gene expression analysis has thus become a major tool for biological research, as well as for diagnosing and monitoring cancers.

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Amicus Therapeutics and Blackstone Enter into $430 Million Strategic Financing Collaboration

GlobeNewswire | October 03, 2023

Amicus Therapeutics a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases, today announced that it has entered into a definitive agreement for a $430 million financing collaboration with funds managed by Blackstone. As part of the collaboration, Blackstone Life Sciences and Blackstone Credit have agreed to provide Amicus with a $400 million senior secured term loan facilitating a refinancing of existing debt and a $30 million strategic investment in Amicus’s common stock. The financing collaboration allows Amicus to grow revenues and move toward profitability while delivering on its mission for patients and its vision of being one of the leading biotechnology companies focused on rare diseases. Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, stated: “This new financing with Blackstone strengthens our balance sheet and financial profile by reducing the interest rate versus our current debt, pushing out the amortization schedule and extending the amortization period. This strategic investment demonstrates Blackstone's commitment to Amicus’ future and belief in the strong growth potential of Galafold and Pombiliti™ + Opfolda™ as we continue on our mission to develop medicines for people living with rare diseases.” Key features of this transaction include $400M senior secured term loan facility; interest rate at adjusted Term SOFR plus 6.25%, subject to a 2.50% floor on Term SOFR $30M investment in Amicus common stock Requires interest-only payments until late 2026 and matures in October 2029 The full amount of the loan and equity purchase will be available and fully drawn at the initial funding The proceeds will be used to refinance Amicus’s existing debt and fund ongoing operations Commenting on the arrangement, Craig Shepherd, Senior Managing Director with Blackstone Life Sciences and Brad Colman, Senior Managing Director with Blackstone Credit said: "Blackstone aims to provide customized financing solutions for the world’s leading biotech and pharma companies across therapeutic areas to support mission critical scientific innovation. We are excited to collaborate with Amicus and provide capital to advance their mission of bringing important new medicines to people living with rare diseases around the world." Simon Harford, Amicus Chief Financial Officer, added: “Securing this financing with Blackstone as we launch Pombiliti™ + Opfolda™ around the world, allows us to better align our borrowing with anticipated cash flows while at the same time enhancing our ability to maximize access to our therapies for people living with rare diseases.” Subject to completion of customary conditions, the loan is expected to be funded and the equity investment is expected to close on October 5, 2023. About Amicus Therapeutics Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. About Blackstone Life Sciences Blackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical technologies that improve patients’ lives and currently has more than $8 billion in assets under management.

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BenevolentAI Signs Strategic Collaboration with Merck

Business Wire | September 26, 2023

BenevolentAI a leader in the development of advanced AI that accelerates biopharma discovery, announces that it has signed a strategic collaboration with Merck, a leading science and technology company headquartered in Darmstadt, Germany. The collaboration will enable Merck to leverage BenevolentAI’s powerful end-to-end AI platform capabilities and gain access to an expert team of interdisciplinary drug discovery scientists with the aim of identifying and developing innovative compounds, through Hit Identification (Hit ID) to pre-clinical stage. BenevolentAI will leverage its suite of AI chemistry design tools, in combination with its fully equipped wet lab facility in Cambridge (UK), to deliver small molecule drug development candidates into the Merck pipeline, ready for onward pre-clinical and clinical development. Under the terms of the agreement, BenevolentAI will be eligible for payments of up to $594 million, consisting of a low double-digit million dollar upfront payment on signing and then potentially discovery, development and commercial milestones. Tiered royalties will also be payable on net sales of any commercialised products. Joanna Shields, Chief Executive Officer of BenevolentAI, said: “Our strategic collaboration with Merck exemplifies BenevolentAI’s unique capability to leverage AI to accelerate drug discovery. Our proprietary technology and extensively validated approach of combining AI, molecular biology, medicinal chemistry, and in vivo pharmacology supports the discovery of innovative drug candidates in fewer cycles. We look forward to working with the Merck team and are excited at the opportunities in our partnership.” Public disclosure of inside information in accordance with article 17(1) of the Regulation (EU) No 596/2014 on market abuse (Market Abuse Regulation) About BenevolentAI BenevolentAI is a leading developer of advanced artificial intelligence technologies that unlock the value of multimodal data, surface novel insights, and accelerate biomedical discovery. Through the combined capabilities of its AI platform, its scientific expertise, and wet-lab facilities, the Company is developing an in-house drug pipeline of high-value assets. The Company is headquartered in London, with a research facility in Cambridge (UK) and a further office in New York.

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Ichnos Sciences Enters Licensing Agreement For Ox40 Antagonist Monoclonal Antibody Portfolio With Astria Therapeutics

PR Newswire | October 12, 2023

Ichnos Sciences, a global clinical-stage biotechnology company developing innovative multispecific antibodies for oncology, announced that the company has entered into an exclusive worldwide licensing agreement for its OX40 antagonist monoclonal antibody portfolio with Astria Therapeutics, a biopharmaceutical company developing therapies for rare allergic and immunological diseases. With the execution of this agreement, Ichnos has successfully licensed its two assets for inflammatory and immunological diseases, a key milestone in the company's prioritization of its pipeline of oncology drug candidates. Within the terms of the agreement, Astria will assume full cost and responsibility for the global development and commercialization of the licensed therapeutic program for all indications. In exchange, Ichnos will receive up to $320 million in upfront, development, regulatory and sales milestone payments in addition to up to low double-digit royalties. Ichnos has also agreed to allow Astria to draw down on its existing investigational drug substance and drug product stocks at normalized costs to facilitate development. Telazorlimab is a novel, humanized IgG1 monoclonal antibody that targets OX40 on T cells responsible for inflammation and immunity diseases. Excessive OX40 signaling, expressed on activated T cells, is the feature of several inflammatory diseases, including atopic dermatitis (AD). Astria plans to develop an affinity-matured version of telazorlimab and apply YTE half-life extension technology to create a product that aims to address the need for a safe, effective, and infrequently administered AD treatment. "As Ichnos continues to grow as a biotechnology company, this agreement enables our team to focus on advancing our robust pipeline of clinical-stage multispecific antibodies in oncology generated by our proprietary BEAT® platform1, as well as continue the discovery and development of our NK-cell engaging programs for solid tumors," said Cyril Konto, M.D., President and CEO of Ichnos. "I am proud of the work achieved by the Ichnos team in successfully completing the Phase 2b with telazorlimab in atopic dermatitis and potentially opening up a new therapeutic class for this disease. Ichnos is confident in Astria Therapeutics' capabilities and vision for pursuing the development of its OX40 program and delivering a potentially life-changing treatment to patients with inflammatory and immune diseases." "We are looking forward to building on the foundational work that Ichnos has done with their OX40 portfolio," said Jill Milne, Ph.D., co-founder and CEO of Astria Therapeutics. "We believe that by using Ichnos' affinity-matured next generation monoclonal antibody OX40 antagonist and applying YTE half-life extension technology, we have the potential to deliver a best-in-class profile for atopic dermatitis patients; one that we think can be safe, effective, and long-acting. In addition to OX40 antagonism already being a clinically validated mechanism in atopic dermatitis, we also are excited about the opportunity for potential expansion into additional indications." About Ichnos Sciences Inc. A fully integrated, global biotech with the spirit of a start-up, Ichnos is shifting the way the world thinks about innovation in medicine through its research and development of transformative, disease-centric treatments in oncology. The company, with headquarters in New York, N.Y., is rapidly advancing a clinical-stage pipeline of novel, first-in-class candidates designed to address complex diseases and to treat patients holistically. With its patented BEAT® technology platform and pioneering teams, Ichnos Sciences has a mission to provide breakthrough, curative therapies that will extend and improve lives, writing a new chapter in healthcare.

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