Stevanato Group | January 25, 2024
Stevanato Group S.p.A. a leading global provider of drug containment and delivery solutions to the pharmaceutical, biotechnology, and life sciences industries, unveiled today two new offerings for efficient small batch pharmaceutical manufacturing: the EZ-fill® Kit and the non-GMP laboratory fill and finish service at its Technology Excellence Centers (TEC).
In a context where reducing time-to-market is key, Stevanato Group’s new EZ-fill® Kit offers fast access to a customizable and versatile solution to pharma and biotech companies needing to efficiently fill small batches with high-quality injectable formulations during clinical trials or commercial phases.
The surge the industry has seen in biopharmaceuticals treating various diseases in several therapeutical areas may signal a significant growth potential for pharmaceutical companies. Injectables, comprising more than 60% of the over 21,000 drugs currently in development, of which 44% are biopharmaceuticals, require high-performance primary packaging and delivery technologies for proper storage and administration.
Stevanato Group’s EZ-fill® Kit builds on its existing ready-to-use platform of pre-sterilized containment solutions – vials, cartridges, and syringes – allowing customers to effectively screen different primary packaging in combination with drug products. The kit will be available as a modular box made up of glass ready-to-use drug containers and add-on components on request, shipped by courier. As a result, the platform can bring enhanced levels of quality and safety to patients across the entire drug life cycle and improve the rate at which new drugs enter the market. EZ-fill® Kit will be presented at Pharmapack and is expected to be available commercially beginning in the second quarter of 2024.
Additionally, to better support customers, Stevanato Group has introduced a non-GMP laboratory filling and finishing service at its Technology Excellence Centers (TEC) in Boston, USA, and Piombino Dese, Italy. This new service allows customers to assess and identify the possible effects of the fill-and-finish process on their product performance as early as the container selection stage, thus de-risking processes and enhancing development and commercialization strategy.
“In the race to bring new advanced biopharmaceutical products to market and to patients, Stevanato Group is tackling the challenge of supporting customers with flexible drug containment and filling solutions in a timely manner," says Fabio Bertacchini, Senior Director EZ-fill® Vials & Cartridges at Stevanato Group. "Stevanato Group remains focused on developing new offerings that can help raise pharmaceutical manufacturing standards. By leveraging the expertise provided by our global TEC Centers, we aim to support customers' innovation, helping them face tests and de-risking processes and making right-the-first-time choices throughout their drug development journey.”
About Stevanato Group
Founded in 1949, Stevanato Group is a leading global provider of drug containment, drug delivery and diagnostic solutions to the pharmaceutical, biotechnology and life sciences industries. The Group delivers an integrated, end-to-end portfolio of products, processes and services that address customer needs across the entire drug life cycle at each of the development, clinical and commercial stages. Stevanato Group’s core capabilities in scientific research and development, its commitment to technical innovation and its engineering excellence are central to its ability to offer value added solutions to clients.
Jnana Therapeutics, Inc. | February 02, 2024
Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.
“There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.”
JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day screening period focused predominantly on ensuring an average blood Phe level of >600µM, study participants were randomized with no run-in period to 75mg of JNT-517 twice daily (BID) or placebo.
The planned interim analysis was based on 13 participants, eight dosed with JNT-517 and five dosed with placebo over 28 days, and demonstrated the following results
JNT-517 led to a statistically significant (p=0.0019 vs. placebo) mean blood Phe reduction from baseline of 51%, measured per-protocol at day 28.
A high response rate was seen where seven of eight (88%) treated participants achieved >30% reduction in blood Phe from baseline; five of eight (63%) achieved >45% reduction; and two of eight (25%) achieved >65% reduction.
A robust response was seen across participants treated with JNT-517 irrespective of baseline blood Phe levels, which ranged from 593µM to 1,526µM with a mean of 1,124µM.
A rapid onset of effect was observed with significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing.
JNT-517 was safe and well tolerated with no serious adverse events and no clinically significant changes in laboratory parameters, consistent with the safety profile demonstrated in the Phase 1a healthy volunteer study.
"JNT-517 represents a completely new therapeutic approach that could transform the current treatment paradigm in PKU, in particular for individuals with severe, or classical, PKU where there is the highest unmet medical need,” said Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine. “I am encouraged by the clinical results to date and look forward to working with Jnana and the PKU community to continue to advance this program.”
Based on these interim results, Jnana has adapted the protocol of the ongoing trial to include dose exploration. Jnana expects topline data from the second dose cohort in mid-2024 and plans to submit full data from the two dose cohorts for presentation at a scientific meeting in the second half of 2024. Jnana anticipates the company will engage regulators in the second half of 2024 and seek to advance JNT-517 directly into a pivotal Phase 3 study in the first half of 2025.
JNT-517 Phase 1b Clinical Trial
The ongoing clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399).
JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The U.S. Food and Drug Administration granted JNT-517 Rare Pediatric Disease Designation in late 2022.
PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe.
About Jnana Therapeutics
Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures.
Vernal Biosciences | January 03, 2024
<p> Vernal Biosciences, a leading manufacturing and services company specializing in mRNA and lipid nanoparticle (LNP) formulation, proudly announces the additions of Sven Lee, Chief Commercial Officer, and Richard P. Hamel, Chief Financial Officer to its leadership team. Both distinguished leaders will strengthen Vernal’s position as a premier mRNA and LNP Contract Development and Manufacturing Organization (CDMO) while using their deep experience to deliver Vernal’s integrated mRNA manufacturing solutions to a growing marketplace.</p> <p> Chief Commercial Officer - Sven Lee</p> <p> Sven will lead the Commercial team, including business development, marketing, technical sales, and alliance management. His team will help customers define their manufacturing needs and provide creative solutions that leverage Vernal’s innovative technologies and manufacturing operations. Sven has a 30-year track record achieving remarkable business growth across the biotech and pharma sectors. Prior to joining Vernal, Sven was a Board Member and the Chief Commercial Officer at BioCina, an Australian based CDMO. Prior to BioCina, Sven spent six years as the Chief Business Officer of Abzena, a fully integrated, global biologics and bioconjugate CDMO. Sven has also held prominent roles in sales, marketing, and strategic planning at other CDMOs and life sciences companies including Global VP of Business Development at Catalent Biologics, Sr. Director of Global Business Development & Sales at TerumoBCT, Crucell (now Janssen), and Biogen.</p> <p> As Chief Commercial Officer, Lee says, “I am thrilled to be a part of a dynamic team specializing in high purity mRNA and LNP solutions for client partners in the rapidly advancing field of biotechnology of vaccines and therapeutic development for clinical trials and commercial use.”</p> <p> Chief Financial Officer – Richard P. Hamel</p> <p> Richard will be responsible for Vernal’s finance strategy and will lead the finance organization, including planning, treasury, tax and reporting, Richard is an accomplished financial executive with over 25 years of experience managing finance teams, improving business performance, and developing systems, processes, and procedures to streamline operations for high growth life science companies. Most recently, Richard was CFO at ReciBioPharm where he played a key role in integrating and managing several business units within ReciBioPharm. Prior to joining ReciBioPharm, he was CFO at United BioSource in Blue Bell, PA helping carve out the business from Express Scripts and building a stand-alone finance organization. Prior to joining United BioSource, Richard spent over seven years at inVentiv Health/Syneos in various financial roles of increasing responsibility including finance, shared services and treasury. Earlier in his career, Richard held a range of finance positions at ThermoFisher, AB Volvo, Kodak, Diageo and EY.</p> <p> As CFO, Richard will support Vernal Biosciences in achieving its ambitious goals and driving long-term success. “I am excited to join the Vernal team and apply my experience to further bolster the company’s already strong mRNA and LNP portfolio and further expand its unique capabilities,” said Hamel.</p> <p> CEO and Founder of Vernal Biosciences, Christian Cobaugh says, “On behalf of all Vernal employees and our investors, I am excited to welcome Sven and Rich to Vernal’s talented leadership team. Their collective experience and success that they bring to Vernal and our clients complements our expansion, including the recent addition of our GMP facilities and operations. Sven has spent most of his career helping customers find creative solutions in clinical manufacturing and Rich has successfully built and integrated several businesses in the life sciences sector, from early stage to global scale. Their additions to our team will help Vernal democratize access to high purity mRNA and LNPs for all stages of research and development.”</p> <p> <strong>About Vernal Biosciences</strong></p> <p> Vernal Biosciences is a leading CDMO specializing in mRNA and LNP manufacturing. Vernal provides comprehensive high purity platform manufacturing solutions for mRNA-based medicines and vaccines, from research to clinical development. Our dedicated team of experts, our platform solutions, and GMP facility show our commitment to our partners, enabling them to focus on accelerating their programs to clinical development to improve patient lives.</p>