Clinical Catch-up from Last Week: May 5-10

Biospace | May 13, 2019

Almost every week, biopharma companies release results from ongoing clinical trials. Here’s a roundup of some of the top clinical trial news from the previous week. AveXis, a Novartis company, released interim data from the Phase I STRONG trial, additional data from the STR1VE trial, and the first data from the Phase III SPR1NT trial for Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1. Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMA gene. SMA is a severe neuromuscular disease noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis. Neurocrine Biosciences presented data from two Phase III clinical trials of opicapone, a once-daily, oral catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s disease. They found that patients receiving opicapone 50 mg with levodopa, the standard of care, had a significant and sustained improvement in symptoms. Neurocrine reported on two Phase III studies, BIPARK-1 and BIPARK-2. BIPARK-1 looked at about 600 patients with Parkinson’s and motor fluctuations. BIPARK-2 looked at about 400 patients with Parkinson’s and motor fluctuations. They received once-daily doses of opicapone of either 25 mg or 50 mg or placebo for 14 to 15 weeks. The data from these two Phase III trials showed that adding once-daily opicapone to levodopa significantly increased ON time without troublesome dyskinesia.

Spotlight

Europe has a complex biotech landscape, with hundreds of companies, multiple paths to innovation and financing, and marked differences among countries.

Spotlight

Europe has a complex biotech landscape, with hundreds of companies, multiple paths to innovation and financing, and marked differences among countries.

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MEDTECH

GP-write Partners with DNA Script to Accelerate DNA-Writing Technology and Accessibility

Genome Project-write | October 18, 2021

GP-write’s CAD is a one-stop shop for microbe, plant and animal genome writing and redesign. Its automated workflow allows users to rapidly upload a genome, redesign it and synthesize the new sequence. The tool enables researchers to directly order synthetic DNA or related products and services from GP-write’s affiliated members. DNA Script’s SYNTAX System, a benchtop DNA printer powered by their groundbreaking enzymatic DNA synthesis (EDS) technology, enables users to print sequences designed on GP-write’s CAD tool right in their lab. The first-of-its-kind DNA printer expedites workflows, making DNA writing as simple and efficient as next-generation sequencing. DNA Script will host a roundtable at the GP-write 5.0 conference on October 22 at 12:30 p.m. ET to engage attendees in a discussion centered on biosecurity as it relates to emerging technologies, including GP-write’s new CAD tool and DNA Script’s SYNTAX System. “We’re pleased to join GP-write and their industrial partners to drive innovation on the forefront of DNA printing technologies. Just as NGS, or DNA 'read,' and CRISPR, or DNA 'edit,' have brought significant advances to research and clinical care, we believe the broad accessibility of synthetic DNA printing, or DNA 'write,' offered by our SYNTAX System will be equally transformative and power the next bio-revolution.” Thomas Ybert, co-founder and CEO of DNA Script GP-write President and General Counsel, Amy Cayne Schwartz, notes that the organizations are partnering to work toward realizing “a shared vision of a future where writing genomes is facile, democratized and safely accessible.” Schwartz explains that “this will open up new frontiers for development of novel therapeutics and solutions for environmental health.” About Genome Project-write GP-write, conceived as a sequel to the Human Genome Project, applies lessons learned from HGP to pursue scientific exploration fully integrated with the development of genome engineering technologies. The primary goal of the project is to drive dramatic cost reductions and expedite whole-genome writing and redesign over the next decade, empowering researchers to uncover complex biological behavior and reprogram organisms to address defining global challenges in medicine, biotechnology and environmental health. About DNA Script Founded in 2014, DNA Script is a pioneering life sciences technology company developing a new, faster, more powerful and versatile way to design and manufacture nucleic acids. The company has developed an alternative to traditional DNA synthesis called Enzymatic DNA Synthesis, or EDS, allowing this technology to be accessible to labs with the first benchtop enzymatic synthesis instrument, the SYNTAX System. By putting DNA synthesis back in the lab, DNA Script aims to transform life sciences research through innovative technology that gives researchers unprecedented control and autonomy.

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Unum Therapeutics announced The Transfer of BOXR Cell-Based Therapy Technology to SOTIO

Cision PR Newswire | August 31, 2020

Unum Therapeutics Inc. (Nasdaq: UMRX), a biopharmaceutical company focused on developing novel, best-in-class precision kinase inhibitors for a range of patients living with cancer and other unmet medical needs, announced today the sale of its cell-based BOXR programs to SOTIO, a clinical stage immuno-oncology company owned by PPF Group. Under the terms of the agreement, SOTIO will make an upfront payment of $8.1 million for the BOXR technology and will assume development of Unum's lead candidate, BOXR1030, which is on track for near-term entry into the clinic. In addition, Unum will be eligible to receive downstream milestones of up to $3.4 million. The sale to SOTIO will enable it to further advance its goal to develop the next generation of potent immunotherapies for patients with cancer. Unum will retain its antibody coupled T cell receptor (ACTR) technology and continues to explore strategic opportunities for the technology and assets. The sale is final as of August 28, 2020.

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CELL AND GENE THERAPY

X-Therma Closes Oversubscribed $13M Series A Financing Led by LOREA AG

X-Therma Inc. | December 23, 2021

X-Therma Inc., a biotechnology company developing breakthrough technology for regenerative medicine and organ preservation - has closed an oversubscribed $13M Series A funding round. The financing is led by LOREA AG, an entrepreneur-operated investment firm, with participation from Zen11 Holdings, Graphene Ventures, VU Venture Partners, 2b AHEAD Ventures, Methuselah Foundation, Catalytic Impact Foundation, along with notable return angel investors. X-Therma’s technology, a nontoxic biopreservation platform enabled by biomimetic peptoid, is transforming global accessibility to organ transplantation, as well as enabling “off-the-shelf” cell and gene therapy products and engineered tissues by providing safe & effective chemically defined cryopreservation that is DMSO-, serum-, and protein-free. More and Better Organ Transplants “We’re excited about the transformative impact of X-Therma’s technology and potential to better meet society’s demands of safely and successfully transplanting organs across the globe,” says Dr. Christoph Swarovski, owner and founder of LOREA AG. “Our conviction in this technology was inspired by the world’s dire need of a better solution for organ transport and we are fortunate to help X-Therma bring its vision to fruition.” X-Therma has made a quantum leap in demonstrating a significant extension of organ shelf-life from a few hours to a few days in collaboration with world-leading surgeon, Dr. Gerald Brandacher at Johns Hopkins University. Less than 10% of the worldwide demand for transplantable organs is being met. Despite many improvements in surgical techniques and immunosuppression, innovations to extend the time window for safe and reliable organ preservation have, until now, fallen short. X-Therma’s turn-key solution XT-ViVo® and TimeSeal® in organ preservation removes this time constraint bottleneck without changing the peri-transplant workflow. “Most organs are transported in a cooler filled with ice at 4°C. This provides very limited time to deliver organs to patients before they perish or to provide accurate matching. Our chemistry encodes specific protein-like functions into a synthetic molecule to shape ice crystals and prevent further growth into a detrimental size. This fundamentally enables us to safely store organs at sub-zero temperature, which improves transplantation from both organ accessibility and quality perspectives with extended time.” Dr. Mark Kline, CTO & Co-founder of X-Therma “Off-the-Shelf” Regenerative Medicine Currently, many regenerative medicine companies suffer significant logistical barriers to scale up and from the proteomic and epigenetic changes induced by DMSO, decreasing therapy efficacy. Cryopreservatives used today are toxic to cells which can lead to cell damage, reduced product yields, and inefficiencies in production. X-Therma’s flagship product XT-Thrive®, a DMSO-, Serum-, Protein-free and chemically defined cryopreservation solution directly plugs into the current CMC workflow, resolves current bottlenecks in the cell manufacturing process, and enables “off-the-shelf" living medicines, and now is receiving hyper-growth customer demand. “Founded with the mission to enable global organ banking, our platform technology has expanded to all living medicines. Biopreservation is crucial in the evolving regenerative medicine market. It underlies the sourcing of cellular material, manufacture, storage, and delivery of final products,” says Dr. Xiaoxi Wei, CEO & Co-Founder of X-Therma. “Since the onset of the pandemic, the demand for advanced biopharma cold chain solutions has become extremely hot. COVID vaccines have accelerated this growth and exposed cold-chain weaknesses. From day one, our vision has been focused on the heavy lifting of fundamental scientific breakthroughs to solve the soon-to-be bottlenecks still in existence due to a lack of innovation. Our technology supports cell and gene therapy CMC by greatly improving the critical cell preservation process that can make-or-break the delivery of a successful medicine.” With this new round of funding, the company plans to scale commercialization of XT-Thrive® to meet customer demand for cGMP-grade cell preservation products aimed at Cell and Gene Therapies and tissue engineering users in chemistry, manufacturing, and control processes. Moreover, through its continued partnership with Johns Hopkins University, X-Therma will expand collaboration efforts worldwide and accelerate the regulatory process for organ preservation. X-Therma will also continue to expand its multi-disciplinary team, filling positions remotely in its San Francisco Bay Area headquarters and Austria subsidiary. About X-Therma Inc. X-Therma is headquartered in Richmond, part of the San Francisco Bay Area, with research centers in Richmond and Berkeley, and now has expanded to Europe in Austria. X-Therma pioneers ground-breaking cold chain technology via a convergent biopreservation platform, to advance Regenerative Medicine and make available safe and on-demand organs, engineered tissues, cell & gene therapies, vaccines, and beyond to patients in need. X-Therma’s technology could be a paradigm shift that makes worldwide organ sharing possible and eliminates organ waiting lists across the globe. X-Therma has been a selected Industrial User at the Lawrence Berkeley National Laboratory and received over $10M contracts and grant support from the U.S. Department of Defense, the National Science Foundation, and the California Institute for Regenerative Medicine.

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