July 13, 2023
The Latest in Precision Medicine
Thanks to innovations in drug development, genomics and big data, medicine is moving away from the traditional one-size-fits-all approach and becoming more personalized for patients, especially those with chronic diseases. The National Institutes of Health defines precision medicine as an emerging practice that uses an individual’s genetic profile to guide decisions made in regard to the prevention, diagnosis and treatment of disease. Although many of us don’t experience precision medicine in our day-to-day yet, it’s not too far off in the future: new therapies are being researched and developed, and the field has grown over the last several years. Ten years ago, Bristol Meyers Squibb made a major breakthrough when its immunotherapy, Opdivo, was approved by the FDA for the treatment of multiple types of cancers, and was the first immunotherapy approved for gastric cancer (it’s also on the World Health Organization’s list of essential medicines). In 2017, Merck hit a milestone with Keytruda, the first precision immunotherapy and FDA-approved drug to treat cancer tumors based on its genetic makeup. Last December, Mirati’s accelerated approval of Krazati, a new gene therapy for adults with a specific type of non-small cell lung cancer, was a big advancement in cell and gene therapy.
Targeted drug therapy is a highly sought-after innovation today in the biotech world, as several large companies recently paid billions to acquire such assets and technologies. Read on for some of the latest news and what’s to come in precision medicine, a healthcare sector that Technavio estimates will grow into a market worth over $54 billion by 2027.
First Gene Therapy for Severe Hemophilia
Just a few weeks ago, the FDA approved Roctavian, the first gene therapy to treat severe hemophilia in adults. Developed by Bay Area-based BioMarin Pharmaceuticals, Roctavian is an adeno-associated virus vector-based gene therapy for Hemophilia A, a rare genetic disorder where blood is not able to clot to stop bleeding and can result in blood entering the vital organs. Roctavian is administered in one dose intravenously.
Hot Off the Press
Precision Biomarker Laboratories markets itself as “not your typical CRO.” The facility was founded at Cedars-Sinai Medical Center in Los Angeles and blends the traditional services a CRO offers biotech companies along with the research capabilities of academia. The laboratories leaned into the power of proteomics—studying the makeup/composition of proteins and how they interact with cells—which has been a hot topic in medicine, as the technique can help identify biomarkers and lead to the discovery of new drugs. In a recent study, scientists at Precision Biomarker Laboratories were able to identify subjects’ personalized proteomics biosignatures from a single drop of blood, which it says has the potential to provide precise insights about a person’s health. The study appeared as the cover story in a recent edition of Analytical Chemistry Journal.
Next Steps for Prometheus
San Diego-based Prometheus Biosciences has been making headlines since Merck announced it was acquiring the precision therapy biotech for $10.8 billion, which officially closed last month. Prometheus develops precision drugs for a variety of autoimmune conditions, and says its Prometheus360 discovery engine platform includes the world’s largest biobank of specimens from inflammatory bowel disease patients—around 200,000 samples were collected from more than 20,000 patients over the course of two decades. The company’s lead candidate, an investigational monoclonal antibody to treat ulcerative colitis and Chron’s disease known as PRA023, will enter late-stage studies later this year, and it’s also evaluating a new therapy for Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD).
$3 Billion Dollar Acquisition for Kidney Disease Innovations
Last month, Novartis announced its plan to acquire Chinook Therapeutics, a Seattle-based biopharma for $3.2 billion. Chinook developed two late-stage medicines to treat rare and chronic kidney diseases, including Immunoglobulin A Nephropathy (IgAN), a disease that mostly affects young adults and has a high chance of progressing to kidney failure. Novartis says the acquisition is part of its larger strategy to expand its pipeline of innovative medicines and treatments for renal conditions.
Path to Progress
San Diego-based Kura Oncology reported positive data from its Phase 1/Phase 11 clinical trial of ziftomenib, its genetically targeted cancer drug for acute myeloid leukemia. The study, which the company presented last month at the European Haematology Association (EHA) Annual Congress in Frankfurt, Germany, says that 35 percent of patients who took 600 milligrams of the drug (via a tablet) once a day had their cancer go into remission. The company notes the next phase of the study is expected to enroll 85 patients, and a future series of studies will examine how ziftomenib works with current standards of care for the disease and across multiple patient populations.
One Step Closer to Hope for Gastric Cancer Patients
Earlier this month, Astellas was granted priority review by the FDA for its Biological License Application of zolbetuximab, a targeted monoclonal antibody for patients with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma—a cancer that forms in glands that line the organs. In a press release, the company says 26,000 people in the U.S. will be diagnosed with gastric cancer in 2023 and 11,130 people will die from disease this year. Early-stage gastric cancer symptoms frequently overlap with more common stomach-related conditions, meaning the disease is often diagnosed at an advanced stage. “The FDA’s acceptance of the Biologics License Application filing and Priority Review designation for zolbetuximab confirms the urgent therapeutic need and brings us one step closer to delivering on this commitment to patients, families and caregivers,” Moitreyee Chatterjee-Kishore, senior vice president and head of immuno-oncology development at Astellas, said in the release.