Janssen Announces U.S. FDA Breakthrough Therapy Designation Granted for Talquetamab for the Treatment of Relapsed or Refractory Multiple Myeloma

Novel GPRC5DxCD3 Bispecific Antibody Receives Breakthrough Therapy Designation Based Upon Results from the Phase 1/2 MonumenTAL-1 Study

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for talquetamab for the treatment of adult patients with relapsed or refractory multiple myeloma, who have previously received at least 4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Talquetamab is an investigational, off-the-shelf, T-cell redirecting bispecific antibody targeting both GPRC5D, a novel drug target, on multiple myeloma cells and CD3 on T-cells. This distinction for talquetamab follows a PRIME (PRIority MEdicines) designation from the European Medicines Agency (EMA) on January 29, 2021, and an Orphan Drug Designation (ODD) from the FDA on May 3, 2021. Today’s milestone marks the 12th BTD received by Janssen in oncology and the third such designation for the company’s portfolio of bispecific antibodies.

“This Breakthrough Therapy Designation marks an important step in the continued development of talquetamab, a first-in-class bispecific antibody T-cell engager using GPRC5D, a novel target for the treatment of patients with relapsed or refractory multiple myeloma,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. “Despite the therapies available for patients with relapsed or refractory multiple myeloma, new targets and treatments are needed because of the heterogeneity of the disease, which can impact a patient’s response to treatment. We are resolute in our commitment to advance science and develop new therapies and regimens for patients with the goal of delivering the best possible outcomes while driving toward cures.”

The Breakthrough Therapy Designation is supported by data from the Phase 1/2, first-in-human dose-escalation MonumenTAL-1 study of talquetamab (Phase 1: NCT03399799; Phase 2: NCT04634552) for the treatment of heavily pretreated patients with relapsed or refractory multiple myeloma.[1]

Data from the MonumenTAL-1 study were featured during the 2022 European Hematology Association (EHA) Annual Congress as an oral presentation (Abstract #S182)[2] and were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #8015).[3]

The FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement in at least one clinically significant endpoint over available therapy.[4]

About Talquetamab

Talquetamab is a potential first-in-class, investigational T-cell redirecting bispecific antibody targeting both GPRC5D, a novel multiple myeloma target that does not shed over time, and CD3, a component of the T-cell receptor.[1] CD3 is involved in activating T-cells, and GPRC5D is highly expressed on multiple myeloma cells.[5],[6] Results from preclinical studies in mouse models demonstrate that talquetamab induces T-cell-mediated killing of GPRC5D-expressing multiple myeloma cells through the recruitment and activation of CD3-positive T-cells and inhibits tumor formation and growth.[7]

Talquetamab is currently being evaluated in a Phase 1/2 clinical study for the treatment of relapsed or refractory multiple myeloma (NCT03399799) and is also being explored in combination studies (NCT04586426).

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that affects white blood cells called plasma cells, which are found in the bone marrow.[8] When malignant, these plasma cells rapidly spread and replace normal cells in the bone marrow. In 2020, an estimated 176,000 people worldwide were diagnosed with multiple myeloma.[9] In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S.[10] While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.[11]

About the Janssen Pharmaceutical Companies of Johnson & Johnson

At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine in which we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.

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