With the J.P. Morgan Healthcare Conference in full swing, companies did not waste time in announcing deals and providing updates to their pipelines. BioSpace takes a look at some of the announcements made on day one of the annual conference in the Bay Area.
With the J.P. Morgan Healthcare Conference in full swing, companies did not waste time in announcing deals and providing updates to their pipelines. BioSpace takes a look at some of the announcements made on day one of the annual conference in the Bay Area.
Guardant Health – California-based Guardant Health, Inc. kicked off J.P. Morgan with an announcement it forged a strategic collaboration with Amgen to develop a blood-based companion diagnostic test for Amgen’s AMG 510, an investigational oral therapy that inhibits KRAS G12C mutant protein. Under the agreement, Guardant Health plans to pursue pre-market approval for the device dubbed Guardant360 CDx in the United States, Europe and Japan for use with AMG 510 in metastatic non-small cell lung cancer (NSCLC) patients with the KRAS G12C mutation. Amgen is currently enrolling patients in a potentially registrational Phase II study with AMG 510. The FDA granted Orphan Drug Designation to AMG 510 for previously treated metastatic NSCLC and colorectal cancer with KRAS G12C mutation and Fast Track Designation for previously treated metastatic NSCLC with KRAS G12C mutation. AMG 510, a first-in-class KRAS G12C inhibitor, is designed to selectively and irreversibly target a specific mutant form of KRAS called G12C that is present in nearly 13% of all NSCLC patients.
AmirAli Talasaz, president of Guardant Health, said that “development of Guardant360 CDx will lead to consistently delivered guideline-complete genotyping results along with other important genomic information to patients and their providers through blood, which will ultimately increase the number of patients who are identified as eligible for targeted therapies, including AMG 510, and thus improve access to these potentially life-changing treatments.”
BridgeBio Pharma – San Francisco-based BridgeBio added an additional four assets to its pipeline, bringing the company’s total to more than 20 developmental medications. The four new assets are aimed at the areas of cardiovascular and renal disease, gene therapy, and ophthalmology.
Encaleret is a small molecule antagonist of the calcium-sensing receptor targeting conditions related to hypoparathyroidism including Autosomal Dominant Hypocalcemia Type 1 (ADH1). BridgeBio completed the submission of an Investigational New Drug Application to the U.S. Food and Drug Administration and plans a Phase IIb study in ADH1 early 2020. For retinal diseases, BridgeBio is focused on BBP-551, a novel treatment for the genetically determined retinal diseases phenotypically classified as Retinitis Pigmentosa and Leber’s Congenital Amaurosis. BBP-551 has been granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States. BBP-815 is an AAV gene therapy for the treatment for nonsyndromic hearing loss caused by recessive mutations in the TMC1 gene. By replacing the dysfunctional TMC1 protein using AAV gene therapy, BBP-815 is aiming to repair the deficient inner ear sensory cells at the source of this disease. BBP-472 is a novel treatment designed to balance kinase signaling in the brain for the treatment of children with autism-spectrum disorders characterized by loss of the PTEN protein. This program, currently in the lead-finding phase, is focused on advancing a brain-permeable inhibitor of PI3KB, a kinase shown to signal unabatedly in the absence of PTEN.
Oyster Point – Oyster Point Pharma, Inc. announced positive top-line results from its Phase II MYSTIC study in Dry Eye Disease. The study focused on the company’s OC-01 nasal spray, which showed a statistically significant improvement in Schirmer’s score at Day 84 in both doses tested compared to control. OC-01 nasal spray is a preservative-free, aqueous, nicotinic agonist nasal spray designed to activate the trigeminal parasympathetic pathway to stimulate natural tear production, the company announced. The study demonstrated that OC-01 nasal spray was well-tolerated at the two dose levels tested.
Adaptimmune – At the J.P. Morgan Healthcare Conference, Adaptimmune Therapeutics plc announced two confirmed partial responses in a liver cancer patient and melanoma patient who were both treated with the company’s SPEAR-T platform for solid tumors. Additionally, Adaptimmune said it observed two partial responses in a patient with gastroesophageal junction cancer and one in a patient with head and neck cancer. The company said these data further confirm the potential of its SPEAR T-cell platform for patients with multiple solid tumors. Data were previously reported showing compelling efficacy with ADP-A2M4 in synovial sarcoma.
Adrian Rawcliffe, chief executive officer of Adaptimmune, said the responses it has seen in its platform demonstrates that the SPEAR T-cell program can overcome the challenges of treating a range of solid tumors with a T-cell therapy product. He expressed the fact that these results are still early and more data is needed to support the research. However, he noted that the data is a “critical demonstration of the value of our SPEAR T-cell therapies for people with cancer and a validation of the importance of our proprietary affinity engineering.”
Eagle Pharmaceuticals – New Jersey-based Eagle Pharmaceuticals announced it agreed to terms with the University of Pennsylvania for the development of dantrolene sodium for the potential treatment of people living with Alzheimer’s disease. Last year, a proof-of-concept study showed that intranasal administration of dantrolene sodium provided therapeutic effects on memory and cognition in a mouse model of Alzheimer’s. Eagle believes that the homeostatic balance of calcium inside cells is essential for the proper function of the nervous system.
