Pomalidomide

Johnson & Johnson’s (J&J) Tecvayli (teclistamab) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat adults in England and Wales with relapsed and refractory multiple myeloma (RRMM). The final draft guidance specifically applies to patients who have received at least three prior treatments, have progressed on their last treatment, and would otherwise be offered pomalidomide plus dexamethasone (pom-dex). Approximately 5,900 people are diagnosed in the UK every year with multiple myeloma, an incurable blood cancer that affects a type of white blood cell called plasma cells. The majority of patients will relapse and require subsequent therapy, with efficacy declining after each line of treatment. Administered as a subcutaneous injection, Tecvayli is a bispecific antibody that works by redirecting T cells to multiple myeloma cells and helping to destroy them. NICE’s decision was supported by clinical trial results showing that RRMM patients who received Tecvayli after three prior therapies had a median overall survival of 22 months and a median progression-free survival of 11 months. Further data comparing Tecvayli to pom-dex demonstrated that J&J’s therapy reduces the risk of disease progression by 44% and the risk of death by 48%. Amanda Cunnington, senior director of patient access, J&J Innovative Medicine UK, said: “Patients with multiple myeloma face several challenges – not least coping with recurrent relapses – and it’s vital that they can access novel treatment options that offer the potential for sustained remission and improved quality of life.” Cunnington added that, while the draft guidance “meets the needs” of the majority of these patients, the company is “very concerned” that the proposed restriction applied by NICE removes this option from those who have been waiting for Tecvayli to become available on the NHS. The recommendation comes just over two weeks after J&J received approvals from both the European Commission (EC) and the US Food and Drug Administration for its tuberculosis treatment, Sirturo (bedaquiline). In addition, the company’s bispecific antibody Rybrevant (amivantamab) was recently approved by the EC for use alongside carboplatin and pemetrexed chemotherapy as a first-line treatment for non-small cell lung cancer in adults with epidermal growth factor receptor exon 20 insertion mutations.

SAN DIEGO, CA, USA I July 17, 2024 I Biotheryx, Inc., a biopharmaceutical company discovering and developing a portfolio of first-in-class protein degraders with a focus on validated targets in cancer and inflammatory diseases, today announced that the first patient has been dosed in its Phase 1 clinical trial evaluating BTX-9341, an investigational oral and bifunctional degrader of cyclin-dependent kinase 4 (CDK4) and cyclin-dependent kinase 6 (CDK6), as a monotherapy and in combination with fulvestrant for patients with advanced and/or metastatic hormone receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) breast cancer who have previously received CDK4/6 inhibitor therapy either in the adjuvant or metastatic setting. “BTX-9341 is a potent and selective CDK4/6 degrader that has shown significant anti-tumor activity in both CDK4/6 inhibitor naïve and resistant preclinical models. We are optimistic that it will meet a critical unmet medical need for patients with HR+/HER2- breast cancer who have received prior CDK4/6 inhibitor therapy,” said Leah Fung, Ph.D., CEO of Biotheryx. “Dosing the first patient in this trial represents a significant milestone for Biotheryx, the patients we aim to serve and the scientists who have made this possible.” The Phase 1 clinical trial will begin with dose escalation of BTX-9341 as a monotherapy, followed by a combination with fulvestrant and will conclude with dose expansion of BTX-9341 in combination with fulvestrant. The trial will assess safety, tolerability, pharmacokinetic and pharmacodynamic activity of BTX-9341 as a monotherapy and in combination with fulvestrant. Once the recommended Phase 2 dose of the combination has been determined, there will be a formal evaluation of efficacy in an expansion cohort. “We are thrilled to have dosed the first patient with BTX-9341 at The START Center for Cancer Research,” stated START Co-Founder and Co-Director of Clinical Research, Dr. Amita Patnaik, MD, FRCPC. “BTX-9341 is a highly novel, first-in-class, potent and selective degrader of CDK4/6, representing an innovative therapeutic approach. It has the potential to transform the care of patients with advanced and/or metastatic HR+/HER2- breast cancer, particularly those who have received prior CDK4/6 inhibitor therapies. This milestone is perfectly aligned with START’s mission to accelerate drug development and provide early access to cutting-edge anticancer therapies, bringing hope to patients and their families.” In preclinical studies, orally administered BTX-9341 demonstrated in vivo CDK4/6 degradation and improved anti-tumor activity as a monotherapy compared to current standard of care treatment regimens. BTX-9341 also demonstrated synergies with selective estrogen receptor degraders including fulvestrant, elacestrant and camizestrant in CDK4/6 naïve and resistant models. About BTX-9341 BTX-9341 is a first-in-class, oral degrader of CDK4/6, important targets for a range of cancers and clinically validated in HR+/HER2- breast cancer. In preclinical breast cancer models, BTX-9341 demonstrated superiority to CDK4/6 inhibitors through potent and highly selective catalytic degradation of CDK4 and CDK6, robust inhibition of Cyclin E and CDK2 transcription, cell cycle arrest and ultimately superior in vivo efficacy in breast cancer xenografts. Beyond this increased efficacy potential, BTX-9341 is differentiated from CDK4/6 inhibitor approaches through the ability to overcome key resistance mechanisms that limit the impact of inhibitors in second line HR+/HER2- breast cancer. About Biotheryx, Inc. Biotheryx is a clinical-stage biopharmaceutical company discovering and developing a portfolio of first-in-class protein degraders, including bifunctional degraders and molecular glues. Our focus is on deploying the differentiated potential of degraders towards validated targets in cancer and inflammatory disease. Members of our founding and scientific teams previously developed the IMiDs, the first U.S. Food and Drug Administration (FDA) approved modulators of Cereblon, the most widely validated E3 ligase involved in protein degradation, and we have applied our expertise in Cereblon binding to build our proprietary PRODEGY platform. Biotheryx’s clinical program, BTX-9341, a bifunctional degrader of CDK4 and CDK6, began a Phase 1 clinical trial in the third quarter of 2024. Biotheryx’s pipeline also includes BTX-10908, a first-in-class degrader of SOS1 for pan-KRAS mutant cancers and PDE4 degraders for inflammatory diseases. For more information, please visit biotheryx.com and engage with us on LinkedIn . SOURCE: Biotheryx