INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 28, 2023
Hansa Biopharma AB, "Hansa" a pioneer in enzyme technology for rare immunological conditions, and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, announced they have entered a research and development collaboration.
The collaboration will, in a clinical study, evaluate the safety and efficacy of Hansa's antibody cleaving enzyme imlifidase as a pre-treatment prior to the administration of Genethon's gene therapy product candidate GNT-0003 in Crigler-Najjar syndrome in patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus serotype 8 (AAV8). The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
Søren Tulstrup, President and CEO, Hansa Biopharma stated: "Genethon is a pioneer at the cutting-edge of research and development of gene therapies for rare diseases and we are thrilled to be collaborating with them. This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
GNT-0003 is currently being evaluated in a pivotal clinical study in France, Italy, and the Netherlands and has received PRIME (PRIority MEdicines) status from the EMA. Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003. The outcome of the ongoing clinical study of GNT-0003 could potentially form the basis for a MAA or BLA application in Europe or the US.
Crigler-Najjar is a genetic disease-causing bilirubin accumulation which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy. This collaboration with Hansa Biopharma is thus an important next step in the development of our gene therapy treatment for Crigler-Najjar syndrome, Hansa Biopharma's proven enzyme technology coupled with its scientific expertise will help us advance the critical research we are conducting in Crigler-Najjar and could enable gene therapy treatment for patients who are today not eligible because of their immunological status".
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA.
Read More
MEDICAL, INDUSTRY OUTLOOK
PRNewswire | May 16, 2023
PhenomeX Inc. the functional cell biology company, announced the launch of Beacon Select™ optofluidic system with Opto® B Discovery workflows for antibody discovery.
The Opto B Discovery workflows, with their cutting-edge technologies and features, enable scientists to find lead candidate antibodies tailored to their specific challenges and changing needs. Combined with the value-matched Beacon Select system, more customers may access these propriety antibody discovery applications, but with the optimum level of features that meet their research and budgetary needs. PhenomeX's optofluidic platform is now a standard to deliver optimal antibodies with speed, as highlighted recently in the high-impact journal Cell, where antibodies against COVID-19 variants were discovered on the Beacon system six months before Omicron emergence.
Beacon Select for Antibody Discovery Workflow Feature
Opto B Discovery workflows on the Beacon Select system enable scientists to accelerate antibody screening and hit generation to find lead candidate antibodies and are ideally suited for small- to mid-sized biopharma and CDMOs/CROs. This contrasts with the flagship four-chip Beacon system enabled for antibody discovery, optimized for the highest screening throughputs demanded by large biopharma and established CDMOs/CROs.
The Beacon Select system enables rapid function-first selection of single B cells with the best characteristics for any antibody application, including antibody therapeutics, diagnostics, and reagents. In addition, the Opto B Discovery workflows offer users highly customizable assays to assess antigen and epitope specificity, species or variant cross-reactivity, and functional properties, all in a single-day workflow. By selecting and recovering sequences of only the most desirable antibodies with favorable attributes, users can reduce overall bioprocessing costs, improve the probability of campaign success, and shorten timelines to lead candidates. When combined with PhenomeX's catalog of best-in-class reagents and consumables for mouse, human, and rabbit B cell repertoires, customers can do all this against even the hardest antigen targets, including G coupled-protein receptors (GPCRs) and ion channels, to accelerate antibody discovery beyond traditional methods available today.
PhenomeX participation at 19th Annual PEGS Boston Conference & Expo
PhenomeX is participating at the 19th annual Protein Engineering & Cell Therapy Summit (PEGS) conference and expo being held from May 15-19 at the Hynes Convention Center in Boston, Mass. At the conference, the Company will feature its optofluidic and proteomic platform technologies, including the new Beacon Select system, in booth #517 and present two posters and podium presentations. Attendees can explore demonstrations of the technology and learn more about how PhenomeX's products can advance and accelerate our understanding of cellular function along the continuum of scientific discovery, bioprocessing, clinical research, and immunotherapy development.
About PhenomeX Inc.
PhenomeX is empowering scientists to leverage the full potential of each cell and drive the next era of functional cell biology that will advance human health. We enable scientists to reveal the most complete insights on cell function and obtain a full view of the behavior of each cell. Our unique suite of proven high-throughput tools and services offer unparalleled resolution and speed, accelerating the insights that are key to advancing discoveries that can profoundly improve the prevention and treatment of disease. Our award-winning platforms are used by researchers across the globe, including those at the top 15 global pharmaceutical companies and approximately 85% of leading U.S. comprehensive cancer centers.
Read More
INDUSTRY OUTLOOK
Puma Biotechnology, Inc. | March 16, 2023
Puma Biotechnology, Inc., a biopharmaceutical company, recently announced the online publication of the Phase II TBCRC041 randomized clinical trial results in JAMA Oncology. The trial investigated the use of alisertib, an adenosine triphosphate–competitive and reversible inhibitor of aurora kinase A, alone or in combination with fulvestrant in postmenopausal women with endocrine-resistant, HER2-negative metastatic breast cancer who were previously treated with fulvestrant.
The Phase II randomized clinical trial was conducted via the Translational Breast Cancer Research Consortium and included patients previously treated with CDK 4/6 inhibitors and everolimus.
The trial enrolled 91 evaluable patients, with baseline characteristics balanced between the two arms of the trial. However, more patients in the alisertib plus fulvestrant arm had previously received chemotherapy for metastatic disease (47.8% in the alisertib alone arm vs. 68.9% in the alisertib plus fulvestrant arm). In each arm of the trial, all patients had earlier been treated with CDK 4/6 inhibitors. Everolimus was previously administered to 37% of patients in the alisertib alone arm and 57.8% in the alisertib plus fulvestrant arm.
The trial's efficacy results indicated that nine partial responses were observed in the 46 evaluable patients in the alisertib alone arm, leading to an overall response rate of 19.6%. The median duration of response was 15.1 months, with a clinical benefit rate of 41.3% at 24 weeks. The projected median progression-free survival (PFS) was 5.6 months. Nine of the 45 evaluable patients in the alisertib plus fulvestrant arm of the study responded, for an overall response rate of 20.0%. There was one patient who had a complete response and eight patients who showed partial responses. The median duration of response was 8.5 months, with a clinical benefit rate of 28.9% at 24 weeks. The median PFS was expected to be 5.4 months.
The most prevalent grade 3 or higher adverse events in the alisertib alone arm of the trial were neutropenia (43.4%), anemia (19.6%) and leukopenia (17.4%). The most prevalent grade 3 or higher adverse events in the alisertib plus fulvestrant arm of the study were neutropenia (42.2%), leukopenia (31.1%), lymphopenia (15.6%), fatigue (11.1%), and anemia (8.9%).
About Puma Biotechnology, Inc.
Puma Biotechnology, Inc. is a leading biopharmaceutical company specializing in the development and commercialization of innovative treatments for cancer. Its flagship product is Nerlynx (neratinib), an oral tyrosine kinase inhibitor approved by the USFDA for the extended adjuvant treatment of HER2-positive early-stage breast cancer. In addition to Nerlynx, it has a robust pipeline of product candidates in various stages of development. The company is firmly committed to research and development and collaborates with leading academic institutions and research organizations to advance the understanding of cancer and develop new therapies.
Read More