Celltrans, Inc.

PolTREG plans to start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. Image Credit: Anatoliy Cherkas / Shutterstock. PolTREG’s has presented positive long-term data for its cell therapy, PTG-007, showing that the therapy induced clinical remission for type 1 diabetes for up to 12 years. The follow-up data from 54 patients with early-onset type 1 diabetes who took part in Phase I and II trials was presented at the European Association for the Study of Diabetes (EASD) taking place from 9-13 September in Spain. In light of the positive data, the Polish company will start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. “Today’s results are extremely encouraging and show that some patients remain in clinical remission for up to 12 years after initial treatment,” said Professor Piotr Trzonkowski, PolTREG’s CEO. “This makes us very eager to launch a pivotal study of PTG-007 as soon as we find a suitable partner.” The 54 patients with early-onset type 1 diabetes were followed for 7-12 years after receiving initial treatment as part of Phase I and Phase II trials. The study showed that a “proportion of patients” remained insulin-independent up to 18 to 24 months after treatment. See Also: LACTIN-V by Osel for Human Papillomavirus Infections: Likelihood of Approval Cadisegliatin by VTv Therapeutics for Type 1 Diabetes (Juvenile Diabetes): Likelihood of Approval Additionally, some of the patients remained in clinical remission for 7-12 years after being treated with PTG-007. The trial defined clinical remission as having a low need for external insulin, while retaining proper metabolic/glycaemia control. The study also found that the “best results” were seen in patients who received the combination therapy of Treg therapy and Roche and Biogen ’s anti-CD20 treatment Rituxan (rituximab). The company plans to release detailed data and publish the results in a peer-reviewed scientific publication. PolTREG plans to initiate a Phase II trial evaluating PTG-007 in patients with presymptomatic type 1 diabetes. Cell therapies as a treatment for type 1 diabetes are gaining popularity. Last year saw the US Food and Drug Administration (FDA) approving the first cell therapy, CellTrans’s Lantidra (donislecel) for the treatment of type 1 diabetes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

PolTREG plans to start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. Image Credit: Anatoliy Cherkas / Shutterstock. PolTREG’s has presented positive long-term data for its cell therapy, PTG-007, showing that the therapy induced clinical remission for type 1 diabetes for up to 12 years. The follow-up data from 54 patients with early-onset type 1 diabetes who took part in Phase I and II trials was presented at the European Association for the Study of Diabetes (EASD) taking place from 9-13 September in Spain. In light of the positive data, the Polish company will start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. “Today’s results are extremely encouraging and show that some patients remain in clinical remission for up to 12 years after initial treatment,” said Professor Piotr Trzonkowski, PolTREG’s CEO. “This makes us very eager to launch a pivotal study of PTG-007 as soon as we find a suitable partner.” The 54 patients with early-onset type 1 diabetes were followed for 7-12 years after receiving initial treatment as part of Phase I and Phase II trials. The study showed that a “proportion of patients” remained insulin-independent up to 18 to 24 months after treatment. See Also: Bavarian Nordic promises two million mpox vaccines for Africa by end of 2024 IBA venture PanTera gains €134m in funding to scale up actinium-225 production Additionally, some of the patients remained in clinical remission for 7-12 years after being treated with PTG-007. The trial defined clinical remission as having a low need for external insulin, while retaining proper metabolic/glycaemia control. The study also found that the “best results” were seen in patients who received the combination therapy of Treg therapy and Roche and Biogen ’s anti-CD20 treatment Rituxan (rituximab). The company plans to release detailed data and publish the results in a peer-reviewed scientific publication. PolTREG plans to initiate a Phase II trial evaluating PTG-007 in patients with presymptomatic type 1 diabetes. Cell therapies as a treatment for type 1 diabetes are gaining popularity. Last year saw the US Food and Drug Administration (FDA) approving the first cell therapy, CellTrans’s Lantidra (donislecel) for the treatment of type 1 diabetes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Vertex has acquired access to an advanced cell therapy manufacturing platform developed by TreeFrog which will allow the Boston company to boost production of its three pipeline candidates to treat Type 1 diabetes. Following success in cystic fibrosis and sickle cell disease, Vertex’s next big leap is Type 1 diabetes (T1D). Helping the Boston company develop three pipeline candidates in the indication will be French cell therapy developer and manufacturer TreeFrog Therapeutics. The companies announced their partnership on Tuesday, with Vertex licensing exclusive use of TreeFrog’s cell manufacturing platform, C-Stem, which will optimize production by allowing cells to grow “exponentially in 3D,” Vertex said. “The technology will enhance Vertex’s ability to generate large amounts of fully differentiated cells for its portfolio of cell therapies,” the companies wrote. Terms of the milestone-heavy partnership include a $25 million payment upfront, with $215 million available related to the development of a process to produce differentiated islet cells. TreeFrog can also gain sales royalties and another $540 million when clinical, regulatory and commercial milestones are met on up to two future products. Vertex will apply TreeFrog’s technology to its primary T1D candidate VX-880, which successfully completed a phase 1/2 trial last year. Both VX-880 and VX-264 are stem-cell-derived, fully differentiated insulin-producing islet cell therapies. “Our expectation is that C-Stem will enhance our ability to generate large amounts of fully differentiated cells for our portfolio of T1D cell therapies,” a Vertex spokesperson wrote in an email. “We will be implementing TreeFrog’s technology into our current processes to potentially enhance stem cell production and scale-up.” In addition to developing a pipeline of cell therapy products—including a lead candidate for Parkinson’s disease—six-year-old TreeFrog licenses out its C-Stem technology.  Three years ago, the company touted C-Stem as a “leap forward in cell manufacturing,” allowing companies to mass-produce off-the-shelf cell therapies with standardized quality. “This is a fantastic opportunity to apply and further develop our C-Stem technology,” Frédéric Desdouits, Ph.D., CEO of TreeFrog, said in a release. “This collaboration with Vertex advances our business strategy of helping bring best-in-class cell therapies to millions of people—both through our own therapeutic programs and with best-in-class partners like Vertex.” Last year, Vertex struck a manufacturing deal with Lonza in which the companies are co-investing in a new facility in Portsmouth, New Hampshire, which will employ up to 300 people. The plant will support the scale-up and manufacture of Vertex’s two clinical T1D programs. “Our scale up at Lonza is ongoing and we will determine the right time to incorporate TreeFrog’s technology into that process as the collaboration progresses,” the Vertex spokesperson said. Last year, Chicago startup CellTrans scored the first approval for a T1D cell therapy when the FDA signed off on Lantidra (donislecel). Stem cell treatments—like those under development by Vertex—are believed to be more reliable and healthier than those from deceased donors, as is the case with Lantidra.